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HK1223547A1 - Methods for the treatment of cancer - Google Patents

Methods for the treatment of cancer
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HK1223547A1
HK1223547A1HK16111783.0AHK16111783AHK1223547A1HK 1223547 A1HK1223547 A1HK 1223547A1HK 16111783 AHK16111783 AHK 16111783AHK 1223547 A1HK1223547 A1HK 1223547A1
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Hong Kong
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entinostat
administration
administered
patient
fed
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HK16111783.0A
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Chinese (zh)
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.古德諾
R.古德诺
.奧登特里科
P.奥登特里科
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欣达克斯制药公司
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Abstract

Described herein are methods for the treatment of cancer in a subject. In particular, methods are provided for the treatment of lung cancer with a combination of entinostat and an EGFR inhibitor, or the treatment of breast cancer with a combination of entinostat and an aromatase inhibitor. Furthermore, a food effect was evident for the oral administration of entinostat.

Description

Method of cancer treatment
Cross-referencing
This application claims the benefit of U.S. provisional application No. 61/819,505 filed on 3/5/2013, which is incorporated herein by reference in its entirety.
Technical Field
The present invention relates to methods of treating cancer based on co-administration of HDAC inhibitors.
Background
Cancer, tumors, tumor-related disorders, and neoplastic disease states are serious and often life-threatening disorders. These diseases and conditions, characterized by rapidly proliferating cell growth, remain the subject of research efforts aimed at identifying therapeutic agents that are effective in their treatment. Such agents can prolong the life of a patient, inhibit the growth of rapidly proliferating cells associated with a neoplasm, or effect regression of a neoplasm.
In general, surgery and radiation therapy are the first modes of consideration in the treatment of locally restricted cancers and provide the best prognosis. Chemotherapy of some cancers often results in disappointing survival rates but still provides a survival benefit. For example, in lung cancer patients, chemotherapy regimens with Epidermal Growth Factor Receptor (EGFR) inhibitors, such as erlotinib and gefitinib, are used. Additional conventional treatments currently employed provide limited benefit if the patient is unresponsive to treatment with EGFR inhibitors. In breast cancer patients, aromatase inhibitor chemotherapy regimens are used, such as the use of letrozole, anastrozole or exemestane. Other conventional treatments provide limited benefit if the patient does not respond to aromatase inhibitor therapy.
Although several EGFR inhibitors have been approved for the treatment of lung cancer, EGFR inhibitor treatment is limited, such as by side effects resulting from their use. There is a growing concern that although treatment of tumors with EGFR inhibitors may initially reduce the size of the tumor, the size of the tumor may eventually become larger, which especially implies the development of resistance. Erlotinib, a widely used EGFR inhibitor, may represent the type of therapeutic agent used in cancer therapy due to its effect on cancer, but due to other factors that are not completely understood, tumors develop resistance and progress.
Although several aromatase inhibitors have been approved for the treatment of early and late breast cancer, their use produces side effects as with most therapeutics. For example, common side effects include hot flashes, vasodilation, and nausea. There is a growing concern that although treatment of tumors with aromatase inhibitors may initially reduce the size of the tumor, the size of the tumor may eventually become larger, which in particular implies the development of resistance. Letrozole, a widely used aromatase inhibitor, may represent a type of therapeutic agent for cancer treatment; its use has an impact on cancer, but due to other factors that are not completely understood, tumors develop resistance and progress.
Histone Deacetylase (HDAC) inhibitors are an emerging class of therapeutic agents that promote differentiation and apoptosis in blood and solid malignancies through chromatin remodeling and gene expression regulation. Several HDAC inhibitors have been identified, including benzamides (entinostat), short chain fatty acids (i.e., sodium phenylbutyrate); hydroxamic acids (i.e., suberoylanilide hydroxamic acid (suberoylanilide hydroxamic acid) and trichostatin a (trichinostatin a)); cyclic peptides containing a 2-amino-8-oxo-9, 10-epoxy-decanoyl moiety (i.e., trapoxin a) and cyclic tetrapeptides without a 2-amino-8-oxo-9, 10-epoxy-decanoyl moiety (i.e., FK 228). Entinostat is a benzamide HDAC inhibitor that is being clinically studied for various types of solid tumors and hematologic cancers. Entinostat is rapidly absorbed and has a half-life of about 100 hours, and, importantly, the change in histone acetylation persists for weeks after administration of entinostat.
Accordingly, there is a need for compositions and/or methods for treating cancer that utilize the synergistic effects found in therapeutic combinations that can increase the effectiveness of the agents and reduce and/or eliminate the side effects typically associated with conventional therapies.
Disclosure of Invention
One embodiment provides a method of treating cancer in a patient in need thereof, the method comprising orally administering entinostat, wherein Cmax of entinostat is increased when the entinostat is administered in a fasted condition as compared to when the entinostat is administered in a fed condition.
One embodiment provides a method of treating cancer in a patient in need thereof, the method comprising orally administering entinostat, wherein the Tmax of entinostat is increased when the entinostat is administered at a fed condition compared to when the entinostat is administered at a fasted condition.
One embodiment provides a method of treating breast cancer in a patient in need thereof, the method comprising orally administering exemestane and entinostat, wherein the entinostat is administered to a fasting patient.
One embodiment provides a method of treating non-small cell lung cancer in a patient in need thereof, the method comprising orally administering erlotinib and entinostat, wherein the entinostat is administered to a fasting patient.
One embodiment provides a method of treating cancer in a patient in need thereof, the method comprising orally administering entinostat, wherein administration of entinostat at fasting results in an increase in Cmax as compared to administration of entinostat at fed, and wherein the ratio of Cmax after administration at fasting to Cmax after administration at fed is at least about 2: 1.
one embodiment provides a method of treating cancer in a patient in need thereof, the method comprising orally administering entinostat, wherein administration of entinostat at fed results in an increase in Tmax compared to administration of entinostat fasted, and wherein the ratio of Tmax after administration at fed to Tmax after administration at fasted is at least about 2: 1.
Is incorporated by reference
All publications, patents, and patent applications mentioned in this specification are herein incorporated by reference to the same extent as if each individual publication, patent, or patent application was specifically and individually indicated to be incorporated by reference. Patent application PCT international patent application No. PCT/US2012/053551, U.S. patent application No. 14/342,354, and U.S. patent application publication No. 2013/0150386 are incorporated herein by reference.
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The novel features of the invention are set forth with particularity in the appended claims. A better understanding of the features and advantages of the present invention will be obtained by reference to the following detailed description that sets forth illustrative embodiments, in which the principles of the invention are utilized, and the accompanying drawings of which:
figure 1 provides a pharmacokinetic analysis of the food effect study described in example 1.
Detailed Description
Provided herein are methods of treating breast cancer based on the administration of a Histone Deacetylase (HDAC) inhibitor and an aromatase inhibitor. The method comprises administering the HDAC inhibitor without food. The treatment method may incorporate patient selection based on the level of protein lysine acetylation observed during treatment. The method may further comprise a treatment wherein the administration of the HDAC inhibitor and aromatase inhibitor is supplemented with one or more therapeutic agents or therapies.
Provided herein are methods of treating lung cancer based on the administration of an HDAC inhibitor and an Epidermal Growth Factor Receptor (EGFR) inhibitor. The method comprises administering the HDAC inhibitor without food. The method may further comprise a treatment wherein the administration of the HDAC inhibitor and EGFR inhibitor is supplemented with one or more therapeutic agents or therapies.
One embodiment provides a method of treating cancer in a patient in need thereof, the method comprising orally administering entinostat, wherein administration of entinostat at fasting results in an increase in Cmax as compared to administration of entinostat at fed, and wherein the ratio of Cmax after administration at fasting to Cmax after administration at fed is at least about 2: 1. another embodiment provides a method of treating cancer, wherein the ratio of Cmax after administration under fasting conditions to Cmax after administration under fed conditions is at least about 3: 1. another embodiment provides a method of treating cancer, wherein the ratio of Cmax after administration under fasting conditions to Cmax after administration under fed conditions is at least about 4: 1. another embodiment provides a method of treating cancer, wherein the ratio of Cmax after administration under fasting conditions to Cmax after administration under fed conditions is at least about 5: 1. another embodiment provides a method of treating cancer, wherein the ratio of Cmax after administration under fasting conditions to Cmax after administration under fed conditions is at least about 6: 1. another embodiment provides a method of treating cancer, wherein the ratio of Cmax after administration under fasting conditions to Cmax after administration under fed conditions is at least about 7: 1. another embodiment provides a method of treating cancer, wherein the cancer is lung cancer. Another embodiment provides a method of treating cancer, wherein the lung cancer is non-small cell lung cancer. Another embodiment provides a method of treating cancer, wherein the cancer is breast cancer. Another embodiment provides a method of treating cancer, the method further comprising orally administering an EGFR inhibitor. Another embodiment provides a method of treating cancer, wherein the EGFR inhibitor is erlotinib. Another embodiment provides a method of treating cancer wherein erlotinib is administered at a different time than the day that entinostat is administered. Another embodiment provides a method of treating cancer wherein the patient does not eat within 2 hours prior to administration of erlotinib. Another embodiment provides a method of treating cancer, wherein the patient does not eat within 1 hour after administration of erlotinib. Another embodiment provides a method of treating cancer wherein about 150mg of erlotinib is administered. Another embodiment provides a method of treating cancer wherein erlotinib is administered once daily. Another embodiment provides a method of treating cancer, the method further comprising orally administering an aromatase inhibitor. Another embodiment provides a method of treating cancer, wherein the aromatase inhibitor is exemestane. Another embodiment provides a method of treating cancer wherein exemestane is administered at a time different from the day of entinostat administration. Another embodiment provides a method of treating cancer wherein exemestane is administered after a meal. Another embodiment provides a method of treating cancer wherein exemestane is administered with a meal. Another embodiment provides a method of treating cancer wherein about 25mg of exemestane is administered. Another embodiment provides a method of treating cancer wherein exemestane is administered once daily. Another embodiment provides a method of treating cancer, wherein the patient is administered about 10mg of entinostat. Another embodiment provides a method of treating cancer, wherein the patient is administered about 5mg of entinostat. Another embodiment provides a method of treating cancer, wherein the patient is administered about 1mg to about 20mg of entinostat. Another embodiment provides a method of treating cancer, wherein the patient is not fed within 2 hours prior to administration of entinostat in fasted conditions.
Another embodiment provides a method of treating cancer, wherein the patient is not fed within 1 hour prior to administration of entinostat in fasted conditions. Another embodiment provides a method of treating cancer, wherein the patient is not fed within 2 hours after administration of entinostat under fasting conditions. Another embodiment provides a method of treating cancer, wherein the patient is not fed within 30 minutes after administration of entinostat in a fasted condition. Another embodiment provides a method of treating cancer, wherein the patient consumes a high fat meal in a fed condition.
One embodiment provides a method of treating cancer in a patient in need thereof, the method comprising orally administering entinostat, wherein administration of entinostat at fed results in an increase in Tmax compared to administration of entinostat fasted, and wherein the ratio of Tmax after administration at fed to Tmax after administration at fasted is at least about 2: 1. another embodiment provides a method of treating cancer, wherein the ratio of Tmax after administration at fed to Tmax after administration at fasted is about 2:1 to about 5: 1. another embodiment provides a method of treating cancer, wherein the ratio of Tmax after administration at fed to Tmax after administration at fasted is about 5:1 to about 8: 1. another embodiment provides a method of treating cancer, wherein the ratio of Tmax after administration at fed to Tmax after administration at fasted is about 8:1 to about 12: 1. another embodiment provides a method of treating cancer, wherein the ratio of Tmax after administration at fed to Tmax after administration at fasted is about 12:1 to about 15: 1. another embodiment provides a method of treating cancer, wherein the cancer is lung cancer. Another embodiment provides a method of treating cancer, wherein the lung cancer is non-small cell lung cancer. Another embodiment provides a method of treating cancer, wherein the cancer is breast cancer. Another embodiment provides a method of treating cancer, the method further comprising orally administering an EGFR inhibitor. Another embodiment provides a method of treating cancer, wherein the EGFR inhibitor is erlotinib. Another embodiment provides a method of treating cancer wherein erlotinib is administered at a different time than the day that entinostat is administered. Another embodiment provides a method of treating cancer wherein the patient does not eat within 2 hours prior to administration of erlotinib. Another embodiment provides a method of treating cancer, wherein the patient does not eat within 1 hour after administration of erlotinib. Another embodiment provides a method of treating cancer wherein about 150mg of erlotinib is administered. Another embodiment provides a method of treating cancer wherein erlotinib is administered once daily. Another embodiment provides a method of treating cancer, the method further comprising orally administering an aromatase inhibitor. Another embodiment provides a method of treating cancer, wherein the aromatase inhibitor is exemestane. Another embodiment provides a method of treating cancer wherein exemestane is administered at a time different from the day of entinostat administration. Another embodiment provides a method of treating cancer wherein exemestane is administered after a meal. Another embodiment provides a method of treating cancer wherein exemestane is administered with a meal. Another embodiment provides a method of treating cancer wherein about 25mg of exemestane is administered. Another embodiment provides a method of treating cancer wherein exemestane is administered once daily. Another embodiment provides a method of treating cancer, wherein the patient is administered about 10mg of entinostat. Another embodiment provides a method of treating cancer, wherein the patient is administered about 5mg of entinostat. Another embodiment provides a method of treating cancer, wherein the patient is administered about 1mg to about 20mg of entinostat. Another embodiment provides a method of treating cancer, wherein the patient is not fed within 2 hours prior to administration of entinostat in fasted conditions. Another embodiment provides a method of treating cancer, wherein the patient is not fed within 1 hour prior to administration of entinostat in fasted conditions. Another embodiment provides a method of treating cancer, wherein the patient is not fed within 2 hours after administration of entinostat under fasting conditions. Another embodiment provides a method of treating cancer, wherein the patient is not fed within 30 minutes after administration of entinostat in a fasted condition. Another embodiment provides a method of treating cancer, wherein the patient consumes a high fat meal in a fed condition.
To facilitate understanding of the disclosure described herein, a number of terms are defined below.
As used herein, "abnormal cell growth" refers to cell growth that is not dependent on normal regulatory mechanisms (e.g., loss of contact inhibition), including abnormal growth of normal cells and growth of abnormal cells.
"neoplasia" as described herein is abnormal, unregulated and disturbed cell proliferation that is distinguished from normal cells by spontaneous growth and somatic mutation. As neoplastic cells grow and divide, they transmit their genetic mutations and proliferative characteristics to progeny cells. A neoplasm or tumor is an accumulation of neoplastic cells. In some embodiments, the neoplasm can be benign or malignant.
As used herein, "metastasis" refers to the spread of tumor cells through lymphatic or blood vessels. Metastasis also refers to the migration of tumor cells through the serosal cavity or subarachnoid space or other spaces by direct spread. Through the process of metastasis, migration of tumor cells to other areas of the body forms a tumor in areas distant from the site of initial appearance.
As discussed herein, "angiogenesis" is significant in tumor formation and metastasis. Angiogenic factors have been found to be associated with several solid tumors such as rhabdomyosarcoma, retinoblastoma, ewing's sarcoma, neuroblastoma and osteosarcoma. In the absence of a blood supply to provide nutrients and remove cellular waste, the tumor cannot expand. Tumors in which angiogenesis is critical include solid tumors such as renal cell carcinoma, hepatocellular carcinoma, and benign tumors such as acoustic neuroma and neurofibroma. Angiogenesis is associated with hematological tumors such as leukemia. Angiogenesis is believed to play a role in the development of myeloid abnormalities that cause leukemia. Preventing angiogenesis can arrest the growth of cancerous tumors and the damage to the subject due to the presence of the tumor.
The term "subject" refers to an animal, including but not limited to a primate (e.g., human), cow, sheep, goat, horse, dog, cat, rabbit, rat, or mouse. The terms "subject" and "patient" are used interchangeably herein with reference to, for example, a mammalian subject, such as a human subject.
The term "treating" is intended to include alleviating or eliminating a disorder, disease, or condition; or one or more symptoms associated with the disorder, disease, or condition; or to reduce or eradicate the cause of the disorder, disease, or condition itself.
The term "therapeutically effective amount" refers to an amount of a compound that, when administered, is sufficient to prevent the development of, or alleviate to some extent, one or more symptoms of the disorder, disease, or condition being treated. The term "therapeutically effective amount" also refers to an amount of a compound that is sufficient to elicit the biological or medical response of a cell, tissue, system, animal or human that is being sought by the researcher, veterinarian, medical doctor or clinician.
The term "pharmaceutically acceptable carrier", "pharmaceutically acceptable excipient", "physiologically acceptable carrier" or "physiologically acceptable excipient" refers to a pharmaceutically acceptable material, composition or vehicle, such as a liquid or solid filler, diluent, excipient, solvent or encapsulating material. Each component must be "pharmaceutically acceptable" in the sense of being compatible with the other ingredients of the pharmaceutical formulation. It must also be suitable for use in contact with the tissues or organs of humans and animals without excessive toxicity, irritation, allergic response, immunogenicity, or other problems or complications, commensurate with a reasonable benefit/risk ratio. See Remington, the science and practice of pharmacy, 21 st edition; philadelphia, PA,2005, Lippincott Williams & Wilkins; handbook of pharmaceutical excipients, 5 th edition; rowe et al, eds., 2005, the pharmaceutical pressure and American pharmaceutical Association; and handbook of pharmaceutical additives,3 rdEdition; ashandsheds, gowerpublishing company, 2007; pharmaceutical preformulation and formulation, Gibsoned, CRCPress LLC: BocaRaton, FL, 2004).
The term "pharmaceutical composition" refers to a mixture of a compound disclosed herein with other chemical components such as diluents or carriers. The pharmaceutical composition facilitates administration of the compound to an organism. A variety of techniques exist in the art for administering compounds including, but not limited to, oral, injection, aerosol, parenteral, and topical administration. Pharmaceutical compositions can also be obtained by reacting the compounds with inorganic or organic acids such as hydrochloric acid, hydrobromic acid, sulfuric acid, nitric acid, phosphoric acid, methanesulfonic acid, ethanesulfonic acid, p-toluenesulfonic acid, salicylic acid and the like.
The terms "fasted," "fasted," or "food-free" are defined to generally mean the absence of food during a time period from at least about 30 minutes prior to administration of a therapeutic agent as described herein to at least about 30 minutes after administration of a therapeutic agent as described herein. In some cases, there is no food intake from at least about 2 hours prior to administration of a therapeutic agent described herein to at least about 1 hour after administration of a therapeutic agent described herein. In some cases, there is no food intake from at least about 1 hour prior to administration of a therapeutic agent described herein to at least about 1 hour after administration of a therapeutic agent described herein. In some cases, there is no food intake from at least about 1 hour prior to administration of a therapeutic agent described herein to at least about 2 hours after administration of a therapeutic agent described herein.
The term "eating condition" refers to a condition in which a meal has been consumed. In some cases, the food is a high fat or high calorie meal. High calorie meals may include, but are not limited to, meals containing 500 calories or more, about 300 to about 800 calories, about 500 calories to about 1,000 calories, and about 800 calories to about 1,500 calories. In some cases, high fat meals include, but are not limited to, meals in which the percentage of calories from fat to daily caloric intake is from about 20% to about 50%, from about 30% to about 60%, and from about 40% to about 70%. In some embodiments, the diet is not high-fat. In some embodiments, the diet is not high caloric.
The term "bioavailability" generally refers to the rate and extent of active ingredient absorption from a therapeutic agent and becoming available at the site of action. For oral dosage forms, bioavailability is associated with the process by which the active ingredient is released from the oral dosage form and migrates to the site of action. In terms of amount, the term "oral bioavailability" or "% F" is defined as AUCoral/AUCivWherein AUCoralIs AUC measured after oral administration, and AUCivIs the AUC measured after intravenous administration.
"AUC" refers to the area under the drug-concentration curve. "AUC0-t"refers to the area under the drug-concentration curve from zero to time t. "AUClast" refers to the area under the drug-concentration curve from zero to the last data point of the drug-concentration curve. "AUC0-∞"or" AUCinf "refers to the area under the drug-concentration curve from zero to infinite time.
“t1/2"refers to the elimination half-life of the species in question. "t" smax"refers to the time of maximum concentration of the species in question. "Cmax"refers to the maximum concentration of the species referred to.
Treatment of breast cancer
Histone deacetylase
HDACs are a family comprising at least 18 enzymes, which are classified into classes 3 (I, II and III). Class I HDACs include, but are not limited to, HDACs 1,2, 3, and 8. Class I HDACs are found in the nucleus and are thought to be associated with transcriptional control repressors. Class II HDACs include, but are not limited to, HDACs 4, 5, 6, 7, and 9, and can be found in the cytoplasm and nucleus. Class III HDACs are believed to be NAD-dependent proteins and include, but are not limited to, members of the Sirtuin family of proteins. Non-limiting examples of Sirtuin proteins include SIRT 1-7. As used herein, the term "selective HDAC inhibitor" refers to an HDAC inhibitor that does not interact with all class 3 HDACs.
HDAC inhibitors
HDAC inhibitors can be broadly divided into pan HDAC inhibitors and selective HDAC inhibitors. Despite the great structural diversity of known HDAC inhibitors, they share common features: a moiety that interacts with the active site of the enzyme and a side chain that is located inside the channel leading to the active site. This is seen in hydroxamic acids such as SAHA, where the hydroxamic acid group is believed to interact with the active site. In the case of depsipeptides, it is believed that the reduction of the intracellular disulfide bond produces a free sulfhydryl group attached to a 4-carbon alkenyl chain (which interacts with the active site). The difference between HDAC inhibitors is the way they interact with the edge of the HDAC channel (which is at the opposite end of the channel leading to the active site). It is believed that this interaction between HDAC inhibitors and the channel borders accounts, at least in part, for some of the differences in HDAC selectivity observed between pan HDAC inhibitors, such as SAHA, and selective HDAC inhibitors, such as depsipeptides. A particularly preferred HDAC inhibitor is entinostat. Entinostat has the chemical name N- (2-aminophenyl) -4- [ N- (pyridin-3-yl) methoxycarbonylamino-methyl ] -benzamide and the chemical structure shown below.
Chemical structure of entinostat
Aromatase enzymes
Estrogen is a female sex hormone and has many functions in the body. It has been found that about 80% of breast cancer tumors overexpress estrogen receptors and respond positively to the presence of estrogen. In postmenopausal women, ovarian estrogen production is reduced and plasma estrogen levels are generally lower than in premenopausal women.
The residual source of estrogen in postmenopausal women is the synthesis of estrogen from androgens, catalyzed by aromatase. Inhibition of aromatase activity will result in a reduction in estrogen levels, thereby reducing the growth of breast cancer tumors that respond positively to the presence of estrogen.
Aromatase is an enzyme of cytochrome P450 family and is a product of the CYP19 gene. The chemical functions of aromatase are to convert testosterone into estradiol and androstenedione into estrone.
Aromatase inhibitors
Aromatase inhibitors lower the body's estrogen by blocking the aromatase enzyme from converting androgens to estrogens. For the treatment of early breast cancer, some aromatase inhibitors can be used as adjuvant therapy instead of tamoxifen or after 2 or more years of tamoxifen use. For the treatment of metastatic breast cancer, aromatase inhibitors are being tested in clinical trials to compare them with hormone therapy of tamoxifen.
As used herein, an "aromatase inhibitor" is a molecule that inhibits the activity of aromatase. Those skilled in the art use procedures such as standard pharmacological testing procedures (which measure 1,2-3Inhibition of conversion of H-androstenedione to estrone) and the like can be readily identified as aromatase inhibitors.
Briefly, microsomal fractions were prepared from human placenta by the method described by Thompson and Siiteri (j.biol.chem., vol.249, p.5364 (1974)). The microsomal preparation thus obtained was lyophilized and stored at-40 ℃. Adding said human placenta microsomes to 1,2-3H-androstenedione and incubation at 37 ℃ for 20 min. By passing3H2Loss of O into the incubation medium measures the amount of aromatisation of the labelled substrate. The substrate was removed by chloroform extraction followed by adsorption to charcoal in suspension. The char was removed by centrifugation and the non-steroidal medium was counted in a liquid scintillation counter. The composition is tested for aromatase inhibitory activity by adding the composition to the incubation medium prior to addition of the microsomes. The relative cpm obtained with or without the composition was used to calculate the percent inhibition of androstenedione aromatization to estrone. IC can be determined graphically from the concentration of test compound at which the aromatisation of androstenedione to estrone decreases to 50% of the control value50The value is obtained.
Subcutaneous fat is the major aromatase active site and plasma estrogen levels have been shown to correlate with body mass index (Longcope et al, Metabolism1986,35,235-7). It has been shown that during menopause, plasma estrogen levels drop from about 110pg/mL to much lower levels, about 7 pg/mL. However, it has been found that in postmenopausal women, intratumoral estradiol concentrations are about 10 times higher than in plasma, probably due to intratumoral aromatase activity.
Aromatase inhibition has been studied and has met with some success as a treatment option for breast cancer. Three classes of aromatase inhibitors are currently approved for marketing in the united states for the treatment of various stages of breast cancer in postmenopausal women. LetrozoleIs applicable to several treatment options, including extended adjuvant treatment of early breast cancer in postmenopausal women who were previously treated with tamoxifen for 5 years, treatment of locally advanced or metastatic breast cancer in postmenopausal women who are hormone receptor positive (or unknown), and treatment of advanced breast cancer in postmenopausal women with disease progression after antiestrogen therapy.
AnastrozoleIs applicable to several treatment options including adjuvant treatment of early breast cancer in postmenopausal women at hormone receptor (+), first-line treatment of locally advanced or metastatic breast cancer in postmenopausal women at hormone receptor (+) (or unknown), and advanced breast cancer in postmenopausal women after disease progression after tamoxifen therapy.
Exemestane (A), (B), (C) Is applicable to several treatment options, including the adjuvant treatment of early breast cancer in postmenopausal women who have received tamoxifen for 2-3 years for estrogen receptor (+) and advanced breast cancer in postmenopausal women who have progressed on tamoxifen therapy.
These drugs can be divided into two categories: (class 1) exemestane is based on a steroidal chemical structure, (class 2) letrozole and anastrozole are based on a non-steroidal chemical structure. Clinical trials have shown that letrozole is superior to tamoxifen in the treatment of advanced ER (+) disease. In early disease, adjuvant therapy with anastrozole appears to be superior to tamoxifen therapy in reducing the risk of relapse. Recent clinical trial results have made aromatase inhibitors a standard of care for breast cancer therapy instead of tamoxifen.
Breast cancer
Today, breast cancer remains the most common diagnosed cancer among women in the united states. 1/8 is at risk of developing breast cancer. Age, family history, diet and genetic factors are all identified as risk factors for breast cancer. Breast cancer is the second leading cause of death in women.
HER2/neu positive breast cancer
Cancers associated with overexpression of HER2/neu include breast, ovarian, endometrial, prostate, gastric, salivary gland, pancreatic, colorectal, oral, and non-small cell lung cancers. Breast cancer has been the focus of anti-HER 2/neu therapy.
Approximately 25-30% of breast cancers have HER2/neu gene amplification or overexpression of its protein product. Overexpression of this receptor in breast cancer is associated with increased disease recurrence and poorer prognosis.
Hormone positive cancer
Many breast cancers require estrogen to grow. In menopausal women, the main source of estrogen is the conversion to estrogen by androgens. This process is carried out by aromatase, as described above.
Triple negative breast cancer
In the treatment of triple negative breast cancer (where the cancer is estrogen receptor negative, progesterone receptor negative, and HER2 negative), the compositions and therapies described herein may be combined with other therapeutic agents. Such agents include, by way of example only, cetuximab, paclitaxel, docetaxel, taxane agents, e.g.(ABI-007), paclitaxel-polyoxyethylated castor oil, polyglutame paclitaxel, and Paclitaxel Injectable Emulsion (PIE). These combinations may be advantageous when a cancer associated with overexpression of HER2 is present but not detected due to technical limitations in the assays employed in quantifying HER2 expression.
Hormone therapy is the primary treatment for estrogen receptor positive (ER +) Breast Cancer (BC). Due to the clinical activity and the overall beneficial side effect profile and tolerability of hormonal agents, standard of care typically includes the sequential use of hormonal agents until resistance develops and/or visceral crisis requires a shift to chemotherapy. In postmenopausal women, Aromatase Inhibitors (AI) are a preferred class of antiestrogen therapy which act by blocking the synthesis of endogenous estrogen. Exemestane is a steroidal aromatase inhibitor that reversibly binds to and inactivates aromatase, which has been shown to be potent in the non-steroidal aromatase inhibitor, NSAI; i.e.the metastatic profile of progression after letrozole or anastrozole treatment (ChiaS, GradisharW, MauricL et al: Double-blind, randomizedplacebostrontrollerdtrials of fulvestrant and multiparticulate and phenastanene after priorrononotranslationsiala palladatomasein hibitanephyropastor-positive, advantderastcandor: resultsfromore. JClinoncolol 26: 1664-.
The development of resistance to hormone therapy in advanced breast cancer is a major challenge, putative resistance mechanisms include estrogen-independent growth, hypersensitivity to low estrogen concentrations, cyclin D1 overexpression, constitutive nuclear factor kappa B (NF kappa B) activation, upregulation of growth factor signaling pathways, and downregulation of estrogen receptor α (ER α) expressionAnd romidepsin (romidepsin)It has been demonstrated, pre-clinically, that entinostat inhibits the growth of ER α positive tumors and restores hormone sensitivity due to down-regulation of the estrogen independent growth factor signaling pathway, normalizing ER α levels and increasing aromatase levels (SabnisGJ, golubeybeo, chumrsi et al: functional activity inhibition chemochronotherapy- α and rostatic inhibition chemotherapeutics, cancer response 71:1893-903,2011, SabnisGJ, KaziA, golubeyo, brodifiamh.
Other therapies
Other breast cancer treatments that may be advantageously employed in combination with the therapies disclosed herein include, but are not limited to, radiation therapy, chemotherapy, antibody therapy, and tyrosine kinase inhibitors as adjunctive therapies.
Radiation therapy is a method of cancer treatment that uses high-energy X-rays or other types of radiation to kill or prevent the growth of cancer cells. Chemotherapy is a method of cancer treatment that uses drugs to stop the growth of cancer cells by killing them or stopping their division. When a chemotherapeutic agent is administered orally or injected intravenously or intramuscularly, the drug enters the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapeutic agents are placed directly in the spine, organs or body cavities, such as the abdomen, the drugs primarily affect the cancer cells in these areas (local chemotherapy). The method of administering chemotherapy depends on the type and stage of cancer being treated.
Different chemotherapeutic agents for treating breast cancer are known in the art. Cytotoxic agents for the treatment of breast cancer include doxorubicin, cyclophosphamide, methotrexate, 5-fluorouracil, mitomycin C, mitoxantrone, paclitaxel, taxane agents, by way of example only, such as(ABI-007), paclitaxel-polyoxyethylated castor oil, polyglutame paclitaxel and Paclitaxel Injectable Emulsion (PIE), gemcitabine, docetaxel, capecitabine and epirubicin.
Other chemotherapy for breast cancer includes treatment with one or more of the following agents: bendamustine, carboplatin (e.g.,) Carmustine (e.g.,) The compound (a) is chlorambucil (e.g.,) The amount of cisplatin (e.g.,) Cyclophosphamide injection (e.g.,) Oral administration of cyclophosphamide (e.g.,) The compound(s) of dacarbazine (e.g.,) An acid addition salt of ifosfamide (e.g.,) The amount of lomustine (e.g.,) Mechloroethylamine (mechlorethamine) (e.g., mechlorethamine, and mixtures thereof,) The amount of melphalan (e.g.,) The amount of procarbazine (e.g.,) The amount of bleomycin (for example,) The amount of doxorubicin (e.g.,) Epirubicin, idarubicin (e.g.,) The amount of mitoxantrone (e.g.,) The location of the gemcitabine (e.g.,) Oral administration of mercaptopurine (e.g.,) Methotrexate, pentostatin IV (e.g.,) Oral administration of thioguanine (e.g.,) Oral etoposide (e.g., VP-16,Etopophos), etoposide IV (e.g., VP-16,etopophos), vinblastine (e.g.,) A vincristine (e.g.,) The concentration of vinorelbine (for example,) Dexamethasone (e.g.,) The amount of methylprednisolone (for example,) And prednisone (e.g.,)。
monoclonal antibody therapy is a cancer treatment using antibodies made in the laboratory from a single type of immune system cell. These antibodies can recognize substances on cancer cells or normal substances that can help cancer cells grow. The antibodies attach to these substances and kill cancer cells, block their growth or block their spread. Monoclonal antibodies were administered by infusion. They can be used alone or to carry drugs, toxins or radioactive substances directly to cancer cells. Monoclonal antibodies may also be used in combination with chemotherapy as an adjunct therapy.
TrastuzumabIs a monoclonal antibody that blocks the action of the growth factor protein HER2, which transmits growth signals to breast cancer cells.
Trastuzumab can elicit a clinical response as a single agent and can improve survival when added to chemotherapy for advanced HER2 positive breast cancer. However, some patients do not respond to trastuzumab, and most eventually develop clinical resistance. Little is known about the mechanisms of resistance to natural and acquired trastuzumab. A study has been reported to describe genetic and protein changes associated with resistance using a cell line-based approach (d. tripathiy et al journal of clinical oncology,2005Vol23, No16S, 3121). These investigators studied two HER2 positive breast cancer cell lines (BT474 and SKBR3) that were serially passaged in the presence of trastuzumab until in vitro resistance was recorded. Resistant cell lines appeared after 12 months and exhibited a 3-fold faster growth rate in the absence of trastuzumab. After trastuzumab exposure, G0/G1 arrest was observed in sensitive cells (84% versus 68%) versus resistant cells, with fewer cells in S phase (3% versus 14%). Resistant cell lines show less change in gene expression using trastuzumab, upregulation of chemokine receptor CXCR4 and mitotic checkpoint modulators, and downregulation of PTEN compared to sensitive cells.
Other illustrative treatments that may be advantageously combined with the compositions and therapies disclosed herein may include, but are not limited to, administration of an agent including, but not limited to, lapatinib, alone or in combination with capecitabine, docetaxel, epirubicin, epothilone A, B or D, goserelin acetate, paclitaxel, pamidronate, bevacizumab, or trastuzumab.
In some embodiments, the other treatment comprises chemotherapy comprising administering to the subject one or more of doxorubicin, cyclophosphamide, paclitaxel, lapatinib, capecitabine, trastuzumab, bevacizumab, gemcitabine, eribulin, or albumin-bound paclitaxel (nab-paclitaxel).
Methods for treating breast cancer
One embodiment provides a method of treating breast cancer in a patient comprising: (i) determining the level of protein lysine acetylation prior to administration of the entinostat-aromatase inhibitor combination therapy, (ii) administering the entinostat-aromatase inhibitor combination therapy, (iii) determining the level of protein lysine acetylation after administration of the entinostat-aromatase inhibitor combination therapy, (iv) comparing the level of protein lysine acetylation after administration of the entinostat-aromatase inhibitor combination therapy with the level of protein lysine acetylation before administration of the entinostat-aromatase inhibitor combination therapy, and (v) continuing treatment with the entinostat-aromatase inhibitor combination treatment if the level of acetylation of protein lysine after administration of the entinostat-aromatase inhibitor combination treatment is higher than the level of acetylation of protein lysine before administration of the entinostat-aromatase inhibitor combination treatment. In some cases, entinostat is administered to fasting patients.
One embodiment provides a method of treating breast cancer in a patient comprising: (i) administering an entinostat-aromatase inhibitor combination therapy, and (ii) determining the change in the level of protein lysine acetylation during said treatment as compared to the level of protein lysine acetylation prior to treatment. In some cases, entinostat is administered to fasting patients.
One embodiment provides a method of treating breast cancer in a patient comprising: (i) determining the level of protein lysine acetylation prior to administration, (ii) administering an entinostat-aromatase inhibitor combination therapy, and (iii) determining the level of protein lysine acetylation during the course of treatment. In some cases, entinostat is administered to fasting patients.
It is desirable to improve the oral bioavailability of therapeutic agents such as entinostat, thereby increasing the degree of therapeutic effect on the patient. Generally, food has a variable effect on the bioavailability of a therapeutic agent. Interactions between therapeutic agents and food can result in reduced, delayed, or increased systemic drug availability. The food may interact with the therapeutic agent at the following stages: (i) before and during gastrointestinal absorption; (ii) during the distribution period; (iii) during metabolism; and (iv) during excretion. In one embodiment, the bioavailability of entinostat decreases when administered with food.
Food can affect the peak exposure (Cmax) and the time to reach the peak exposure (Tmax) by delaying gastric emptying and prolonging intestinal transit time. In some cases, food affects total exposure, or area under the concentration-time curve (AUC). In some embodiments, the Cmax is higher when entinostat is administered without food than when entinostat is administered with food. In some embodiments, the ratio of Cmax after entinostat administration in fasted to Cmax after entinostat administration in fed conditions is at least about 2: 1. In one embodiment, the ratio of Cmax after entinostat administration in fasted to Cmax after entinostat administration in fed conditions is at least about 3: 1. In one embodiment, the ratio of Cmax after entinostat administration in fasted to Cmax after entinostat administration in fed conditions is at least about 4: 1. In one embodiment, the ratio of Cmax after entinostat administration in fasted to Cmax after entinostat administration in fed conditions is at least about 5: 1. In one embodiment, the ratio of Cmax after entinostat administration in fasted to Cmax after entinostat administration in fed conditions is at least about 6: 1. In one embodiment, the ratio of Cmax after entinostat administration in fasted to Cmax after entinostat administration in fed conditions is at least about 7: 1.
In some embodiments, the Tmax for entinostat administered without food is lower compared to the Tmax for entinostat administered with food. In some embodiments, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 2: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 3: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 4: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 5: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 6: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 7: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 8: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 9: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 10: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 11: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 12: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 13: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 14: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 15: 1.
In some embodiments, exemestane is administered at a time different from the day of entinostat administration. In one embodiment, exemestane is administered after a meal. In one embodiment, exemestane is administered with a meal.
Another embodiment provides a method wherein the change in protein lysine acetylation level during the course of treatment is determined after about 2 days of treatment, about 5 days of treatment, about 7 days of treatment, about 15 days of treatment, or about 21 days of treatment.
Another embodiment provides such a method, wherein said protein lysine acetylation level is obtained from a tissue sample selected from B cells, T cells, or monocytes.
Another embodiment provides such a method, wherein the aromatase inhibitor is exemestane. Another embodiment provides such a method, wherein the aromatase inhibitor is anastrozole. Another embodiment provides such a method, wherein the aromatase inhibitor is letrozole. Another embodiment provides such a method wherein the aromatase inhibitor is administered daily. Another embodiment provides such a method, wherein the aromatase inhibitor is exemestane and is administered daily. Another embodiment provides a method wherein the entinostat is administered every 7 days in a 28-day cycle. Another embodiment provides a method wherein the entinostat is administered every 14 days in a 28-day cycle. Another embodiment provides the method wherein the entinostat-aromatase inhibitor combination therapy comprises oral administration of entinostat every 7 days and daily oral administration of exemestane in a 28-day cycle. Another embodiment provides the method wherein the entinostat-aromatase inhibitor combination therapy comprises oral administration of entinostat every 14 days and daily oral administration of exemestane in a 28-day cycle. Another embodiment provides a method wherein entinostat is administered to a fasted patient every 7 days in a 28-day cycle. Another embodiment provides a method wherein entinostat is administered to a fasted patient every 14 days in a 28-day cycle. Another embodiment provides the method wherein the entinostat-aromatase inhibitor combination therapy comprises oral administration of entinostat to fasted patients every 7 days in a 28-day cycle and daily oral administration of exemestane. Another embodiment provides the method wherein the entinostat-aromatase inhibitor combination therapy comprises oral administration of entinostat to fasted patients every 14 days in a 28-day cycle and daily oral administration of exemestane.
Another embodiment provides a method wherein the step of determining the level of protein lysine acetylation is performed more than once during the course of treatment. Another embodiment provides a method wherein the step of determining the level of protein lysine acetylation is performed once during the course of treatment.
Another embodiment provides a method further comprising selecting the patient for further treatment if the level of protein lysine acetylation is increased during the course of treatment.
Another embodiment provides a method further comprising selecting the patient for further treatment if the level of protein lysine acetylation increases in the first week of the course of treatment. Another embodiment provides a method further comprising selecting the patient for further treatment if the level of protein lysine acetylation increases in the first week and second week course of treatment.
One embodiment provides a method of selecting a patient for further entinostat-aromatase inhibitor combination therapy comprising comparing the level of protein lysine acetylation in a tissue sample obtained after initiation of treatment with the level of protein lysine acetylation determined prior to initiation of treatment.
One embodiment provides a method of selecting a patient for further entinostat-aromatase inhibitor combination therapy comprising comparing the level of protein lysine acetylation in a tissue sample obtained after initiation of treatment with the level of protein lysine acetylation determined before initiation of treatment, wherein an increase in the level of protein lysine acetylation after initiation of treatment indicates that the patient will benefit from further treatment.
Another embodiment provides a method wherein the level of protein lysine acetylation in a tissue sample obtained after initiation of treatment is determined more than once. Another embodiment provides a method wherein the level of protein lysine acetylation is increased for one week after initiation of treatment. Another embodiment provides methods wherein the protein lysine acetylation level after initiation of treatment is determined on days 2, 8, and 15.
Another embodiment provides a method wherein the increase is from about 10% to about 500%. Another embodiment provides a method wherein the increase is from about 10% to about 400%. Another embodiment provides a method wherein the increase is from about 10% to about 300%. Another embodiment provides a method wherein the increase is from about 10% to about 200%. Another embodiment provides a method wherein the increase is from about 10% to about 100%. Another embodiment provides a method wherein the increase is about 10%, about 20%, about 30%, about 40%, about 50%, or about 60%. Another embodiment provides a method wherein the increase is about 25%, about 50%, about 75%, about 100%, about 125%, or about 150%.
Another embodiment provides such a method, wherein the tissue sample is selected from B cells, T cells or monocytes.
Another embodiment provides a method wherein the tissue sample obtained after initiation of treatment is obtained at least 2 days after initiation of treatment. Another embodiment provides the method wherein the tissue sample obtained after initiation of treatment is obtained between day 2 and day 28 after initiation of treatment. Another embodiment provides the method wherein the sample obtained after initiation of treatment is obtained at day 2,3, 4, 5, 6, 7, 8, 9,10, 11, 12, 13, 14 or 15 after initiation of treatment.
One embodiment provides a method of selecting a patient for further entinostat-aromatase inhibitor combination therapy comprising comparing the percent change in protein lysine acetylation level in a tissue sample obtained after initiation of treatment to the protein lysine acetylation level determined before initiation of treatment, wherein a percent decrease in protein lysine acetylation level after initiation of treatment of between about 5% and about 50% indicates that the patient will not benefit from further treatment.
One embodiment provides a method of treating breast cancer that exhibits resistance to a previous aromatase inhibitor therapy, the method comprising administering to a patient a combination comprising entinostat and an aromatase inhibitor, wherein the patient does not exhibit a complete response, a partial response, or stable disease for greater than 6 months during the previous aromatase inhibitor therapy. In some cases, entinostat is administered to fasting patients.
Another embodiment provides a method wherein said patient relapses within 6 months of the end of or during treatment with a non-steroidal aromatase inhibitor previously administered as adjuvant therapy.
Another embodiment provides such a method wherein said patient exhibits disease progression after at least 3 months of prior non-steroidal aromatase inhibitor treatment.
Another embodiment provides such a method, wherein said breast cancer is ER positive.
Another embodiment provides such a method wherein the aromatase inhibitor administered in combination with entinostat is letrozole. Another embodiment provides such a method wherein the aromatase inhibitor administered in combination with entinostat is anastrozole. Another embodiment provides such a method wherein the aromatase inhibitor administered in combination with entinostat is exemestane.
Another embodiment provides a method wherein entinostat and the aromatase inhibitor are administered sequentially or simultaneously. Another embodiment provides a method wherein entinostat and the aromatase inhibitor are administered simultaneously. Another embodiment provides a method wherein the aromatase inhibitor is administered first. Another embodiment provides a method wherein the aromatase inhibitor is administered daily and the entinostat is administered periodically. Another embodiment provides a method wherein entinostat is administered weekly and the aromatase inhibitor is administered daily. Another embodiment provides a method wherein entinostat is introduced into an ongoing aromatase inhibitor course of therapy.
Another embodiment provides methods further comprising administering to the subject one or more therapies in addition to the combination of entinostat and an aromatase inhibitor selected from letrozole, anastrozole, or exemestane, or a pharmaceutically acceptable salt, solvate, or prodrug thereof.
Another embodiment provides such a method, wherein the one or more therapies comprise one or more of radiation therapy, chemotherapy, high dose chemotherapy with stem cell transplantation, and monoclonal antibody therapy. Another embodiment provides such a method, wherein said radiotherapy comprises in vivo and/or in vitro radiotherapy. Another embodiment provides such a method, wherein the chemotherapy comprises administering to the subject one or more of doxorubicin, cyclophosphamide, paclitaxel, lapatinib, capecitabine, trastuzumab, bevacizumab, gemcitabine, eribulin, or albumin-bound paclitaxel (nab-paclitaxel).
One embodiment provides a method of treating breast cancer in a patient in need thereof, the method comprising orally administering exemestane and entinostat, wherein the entinostat is administered to a fasting patient. Another embodiment provides a method of treating breast cancer, wherein the entinostat Tmax is less than 1 hour after administration. Another embodiment provides a method of treating breast cancer, wherein the entinostat Tmax is less than 90 minutes after administration.
Another embodiment provides a method of treating breast cancer, wherein the entinostat Tmax is less than 2 hours after administration. Another embodiment provides a method of treating breast cancer, wherein the entinostat Tmax is 30 minutes to 2 hours after administration. Another embodiment provides a method of treating breast cancer, wherein the Cmax of entinostat is at least 150ng/mL after oral administration of entinostat. Another embodiment provides a method of treating breast cancer, wherein the Cmax of entinostat is at least 125ng/mL after oral administration of entinostat. Another embodiment provides a method of treating breast cancer, wherein the Cmax of entinostat is at least 100ng/mL after oral administration of entinostat.
Another embodiment provides a method of treating breast cancer, wherein the Cmax of entinostat is at least 80ng/mL after oral administration of entinostat. Another embodiment provides a method of treating breast cancer, wherein the Cmax of entinostat is at least 50ng/mL after oral administration of entinostat. Another embodiment provides a method of treating breast cancer, wherein about 5mg of entinostat is administered. Another embodiment provides a method of treating breast cancer, wherein about 10mg of entinostat is administered. Another embodiment provides a method of treating breast cancer, wherein about 1mg to about 20mg of entinostat is administered. Another embodiment provides a method of treating breast cancer, wherein entinostat is administered once weekly. Another embodiment provides a method of treating breast cancer, wherein the entinostat is administered within a 28-day cycle. Another embodiment provides a method of treating breast cancer, wherein the patient does not eat within 2 hours prior to administration of entinostat. Another embodiment provides a method of treating breast cancer, wherein the patient does not eat within 1 hour prior to administration of entinostat.
Another embodiment provides a method of treating breast cancer, wherein the patient does not eat within 2 hours after administration of entinostat. Another embodiment provides a method of treating breast cancer, wherein the patient does not eat within 30 minutes after administration of entinostat. Another embodiment provides a method of treating breast cancer wherein exemestane is administered at a time different from the day of entinostat administration. Another embodiment provides a method of treating breast cancer wherein exemestane is administered after a meal. Another embodiment provides a method of treating breast cancer, wherein exemestane is administered with a meal. Another embodiment provides a method of treating breast cancer, wherein about 25mg of exemestane is administered once daily.
Treatment of lung cancer
Epidermal growth factor receptor
Over the past few years, knowledge about the molecular mechanisms and cellular transformations associated with cancer behavior has increased. Since the development of specific targeted therapies directed at processes involving carcinogenesis of many types of cancer, much interest has arisen. In the 90 s, Epidermal Growth Factor Receptor (EGFR) was found to play an important role in tumor-like biology and behavior. EGFR stimulation activates intracellular signaling and cascades that affect cell proliferation and activity, angiogenesis, and other mechanisms. Normal cells are affected by external factors and it has been found in tumor cells that activation of cell proliferation mediated by this receptor will no longer require an external stimulus but rather act independently and autonomously. In the case of NSCLC, overexpression of this receptor and specific somatic mutations have been shown to occur in their intracellular domain with tyrosine kinase activity (between exons 18 and 21), which can affect prognosis, with significant correlation to stage, survival and chemotherapy response. These data have led to the development and study of a variety of agents, including monoclonal antibodies against the extracellular domain of EGFR (e.g., cetuximab, c,) And tyrosine kinase intracellular domain inhibiting EGFR(tyrosine kinase inhibitors, TKIs) (e.g., gefitinib and erlotinib). Preliminary results of randomized clinical trials with these TKIs have demonstrated their use in patients with advanced disease to be effective, significantly improving survival of these patients, especially when they have mutations in EGFR in a non-smoking, female subset of patients that are more commonly found in the asian population with adenocarcinoma histological subtypes (especially in the presence of bronchioloalveolar carcinoma). Some of these results are so impressive that this phenomenon is known as the Lazarus effect and results in erlotinib being approved in the us and europe for second and third line therapy in NSCLC patients; and gefitinib was approved in europe for patients with EGFR mutations (delMello et al, worldjclinnconocol, vol.2, p.367 (2011)).
EGFR, also known as ErbB1 or Her1, is a transmembrane glycoprotein encoded by a gene located on chromosome 7 (7p 12.1-12.3). EGFR contains 1186 amino acids (a.a.) and 26 exons. Exons 1-14 encode the extracellular domain, exon 15 encodes the transmembrane region, and exons 16-26 encode the intracellular domain. The glycoprotein belongs to the ErbB receptor family, which also includes: ErbB2(HER2/neu), ErbB3(HER3) and ErbB4(HER 4). Each of these proteins consists structurally of an extracellular domain, a hydrophobic transmembrane domain, and an intracellular domain with intrinsic Tyrosine Kinase (TK) activity (except ErbB 3). These receptors exist as inactive monomers, activated by their interaction with the EGF family of growth factors via the extracellular domain. Binding of an ErbB receptor molecule to one of these ligands results in its interaction with other monomers of the same family (receptor dimerization). This dimerization can occur between two identical receptors (homodimerization, e.g., ErbB1-ErbB1) or between two different receptors (heterodimerization, e.g., ErbB1-ErbB 3). The stimulation by specific ligands triggers a unique pattern of dimerization, which is also specific for the tissue/tumor where this phenomenon occurs. Dimerization of these receptors results in their autophosphorylation, as well as activation of the TK and activation of cascades of intracellular biochemical processes that regulate such diverse activities as proliferation, differentiation, apoptosis, and cell migration.
E cadherin
Epithelial cadherins (E-cadherins), also known as cadherin-1, CAM120/80 or morula adhesion protein, are proteins in humans encoded by the CDH1 gene. E cadherins are typical members of the cadherin superfamily. E cadherins are calcium-dependent cell-cell adhesion glycoproteins consisting of five extracellular cadherin repeats in the extracellular domain (EC1-EC5), a transmembrane domain, an intracellular domain that binds to p 120-catenin and β -catenin, and a highly conserved cytoplasmic tail. The intracellular domain contains a hyperphosphorylated region that is critical for β -catenin binding and thus for E-cadherin function. Beta-catenin may also bind to alpha-catenin. Alpha-catenin is involved in the regulation of actin-containing cytoskeletal filaments. In epithelial cells, cell-to-cell junctions containing E-cadherin are typically adjacent to actin-containing cytoskeletal filaments.
Mutations in this gene are associated with gastric, breast, colorectal, thyroid and ovarian cancer. Loss of function or expression is thought to contribute to the progression of cancer and metastasis. Down-regulation of E-cadherin decreases the strength of cell adhesion within tissues, resulting in increased cell motility. This in turn may allow cancer cells to cross the basement membrane and invade surrounding tissues.
Method for determining E cadherin levels
E-cadherin levels can be measured quantitatively by ELISA. Some E-cadherin ELISA kits, such as the E-cadherin EIA kit provided by TaKaRA, are solid-phase sandwich EIAs that use two mouse monoclonal E-cadherin antibodies (one of which is coated on a plate and the other is labeled with POD) to detect human E-cadherin using a two-step incubation method. In a first step, samples are incubated in antibody-coated microtiter plates. During the second step, the plate was washed and incubated with POD-labeled E-cadherin antibody. Substrate was added and the reaction between POD and substrate (H2O2, TMBZ) resulted in color formation. The amount of soluble E-cadherin in a sample is determined by measuring absorbance using an EIA plate reader. The exact soluble E cadherin sample concentrations can be determined by comparing their specific absorbance to the standard absorbance obtained plotted on a standard curve. In some embodiments, the level of E-cadherin is measured quantitatively by ELISA.
E-cadherin levels can be detected by immunohistochemistry. To detect E-cadherin levels in immersion-immobilized cells, cells were affinity-purified polyclonal antibodies (R) to human E-cadherin antigen at 10. mu.g/mL&DCat No. AF648) were incubated together at room temperature for 3 hours. Northern lights were then usedTM557 conjugated anti-goat IgG Secondary antibody (R)&DCatalog NL001) cells were stained and counterstained with DAPI. The E-cadherin and DAPI can be visualized using a fluorescence microscope, and the filters set to suit the markers used. In some embodiments, E cadherin levels are detected by immunohistochemistry.
E-cadherin levels can be detected by immunocytochemistry. Coverslips for Immunocytochemistry (ICC) can be prepared using gelatin. In some embodiments, the method for preparing a coverslip of ICC comprises a) placing a sterilized coverslip in a well of a 24-well plate, b) adding 400 μ L of gelatin coating solution, and c) incubating the coverslip for 10 minutes at room temperature. The gelatin coating solution was then removed and the coverslips air dried for 15 minutes. The dried coverslip can be stored at room temperature until use. Once the coverslip is prepared, cells can be prepared and fixed as follows. Cells were cultured by adding 500 μ L of medium containing about 5000 cells to the wells of a cell culture plate containing gelatin-coated coverslips. When the cells reached the desired density/phase, the medium was removed from each well and washed twice with PBS. 300-400. mu.L of a 2-4% formaldehyde fixation solution was added to each well and incubated at room temperature for 20 minutes. The wells were washed twice with PBS and covered with 400 μ Ι _ of wash buffer. The coverslip can be stored at 2-8 ℃ for up to 3 months or it can be stained immediately. Once the cells are prepared, the cells can be stained for ICC as follows. The coverslip containing the immobilized cells was washed twice in 400. mu.L of wash buffer. Nonspecific staining was blocked by addition of 400 μ L blocking buffer and incubated for 45 min at room temperature. The blocking buffer was removed. No flushing is necessary. Unconjugated primary antibody (or fluorescently conjugated primary antibody) is diluted in dilution buffer according to the manufacturer's instructions. For fluorescent ICC staining of cells on coverslips with R & DSystems antibodies, incubation for 1 hour at room temperature is recommended. Alternatively, incubation was carried out overnight at 2-8 ℃. Wash twice in 400. mu.L of wash buffer. If a primary antibody with a direct fluorescent conjugate is used, go to step 8. The secondary antibody was diluted in dilution buffer according to the manufacturer's instructions. To the hole add 400 u L, and in the dark at room temperature 1h incubation. From this step, the sample should be protected from light. Wash twice in 400. mu.L of wash buffer. To each well 300. mu.L of diluted DAPI solution was added and incubated at room temperature for 2-5 minutes. DAPI binds to DNA and is a convenient nuclear counterstain. It has an absorption maximum at 358nm and fluorescent blue has an emission maximum at 461 nm. Washed once with PBS and once with water. The coverslip was carefully removed from the well and blotted dry to remove any excess water. 1 drop of anti-fluorescence quenching mounting medium (anti-famotindmedium) was dispensed onto each coverslip microscope slide. The coverslip was covered with cells facing the microscope slide. Visualization was performed using a fluorescence microscope and a set of filters appropriate for the marker used. Slides can also be stored in slide cassettes at < -20 ℃ for subsequent examination. In some embodiments, the level of E-cadherin is detected by immunocytochemistry.
E cadherin gene expression can be determined by measuring E cadherin methylation. E cadherin methylation kits such as the one prepared fromCpG providedE-cadherin amplification kit the methylation status of the E-cadherin promoter is determined by Methylation Specific PCR (MSP). The kit contains primers that target the promoter region where the sequences show the greatest difference after bisulfite treatment. The PCR parameters were determined so that all primer sets in the kit were amplified under the same conditions. Control genomic DNA samples (methylated and unmethylated) against E cadherin were also included. In some embodiments, E-cadherin gene expression is determined by measuring E-cadherin methylation.
One embodiment provides a method of treating cancer in a patient who has not been treated with an EGFR inhibitor, who has progressed on prior treatment, wherein the patient exhibits high levels of E-cadherin expression, comprising administering to the patient a combination comprising entinostat and an EGFR inhibitor. Another embodiment provides a method wherein the high level of E-cadherin expression is characterized by ELISA, immunohistochemistry, immunocytochemistry or measuring E-cadherin methylation levels. Another embodiment provides a method wherein high E cadherin expression levels are determined by immunohistochemistry. Another embodiment provides a method wherein the high E cadherin expression level score is +3, as determined by immunohistochemistry.
Lung cancer
Lung cancer is the leading cause of cancer death in both women and men in the united states and worldwide. Lung cancer has become a leading cause of cancer death in women beyond breast cancer. 157,300 people are expected to die from lung cancer in the united states in 2010, which is higher than the number of deaths caused by colon and rectal cancer, breast cancer and prostate cancer in total. Although the survival rate of lung cancer diagnosed at the earliest stage is high, with about 49% surviving for five years or more, only about 2% of these people diagnosed with lung cancer that has spread to other parts of the body survive five years after diagnosis.
Cancer occurs when normal cells undergo a transition that leads to their uncontrolled growth and proliferation. The cell forms a lump or tumor that is distinct from the surrounding tissue in which it appears. Tumors are dangerous because they consume space for oxygen, nutrients, and healthy cells and because they invade and destroy or reduce the ability of normal tissues to function.
Most lung tumors are malignant. This means that they invade and destroy the surrounding healthy tissue and can spread throughout the body. The tumor may spread to nearby lymph nodes or to other organs through the bloodstream. This process is called migration. When lung cancer metastasizes, the tumor of the lung is called the primary tumor, while the tumor in other parts of the body is called the secondary or metastatic tumor.
Some tumors of the lung are metastasized from cancer in other parts of the body. The lung is a common site of metastasis. If this is the case, the cancer is not considered lung cancer. For example, if prostate cancer spreads to the lung through the bloodstream, it is metastatic prostate cancer in the lung (secondary cancer), and is not referred to as lung cancer.
Lung cancer comprises a group of different types of tumors. Lung cancer is generally divided into two major groups, which account for about 95% of all cases. The division of these groups is based on the cell types that make up the cancer. Two major types of lung cancer are characterized by the cell size of the tumor when viewed under a microscope. They are known as Small Cell Lung Cancer (SCLC) and non-small cell lung cancer (NSCLC). NSCLC includes several tumor subtypes. SCLC is less common, but grows faster and is more likely to metastasize than NSCLC. Typically, SCLC has spread to other parts of the body when cancer is diagnosed. About 5% of lung cancers have rare cell types, including carcinoid tumors, lymphomas, and others. The term "lung cancer" as used herein includes, but is not limited to, SCLC, NSCLC, carcinoid tumors, lymphomas and various subtypes thereof.
Non-small cell lung cancer
NSCLC is a lung cancer that is not a small cell carcinoma (oat cell carcinoma) type. The term "non-small cell lung cancer" applies to all types of bronchial cancers (those arising from the lining of the bronchi). Examples of specific types of NSCLC include, but are not limited to, adenocarcinoma, squamous cell carcinoma, and large cell carcinoma (i.e., large cell undifferentiated carcinoma).
Adenocarcinomas are cancers that develop in the lining or inner surface of an organ. Adenocarcinoma is the most common type of lung cancer, accounting for 30% -40% of all lung cancer cases. One subtype of adenocarcinoma is known as bronchoalveolar cell carcinoma, which produces a pneumonia-like appearance under chest X-ray.
Squamous cell carcinoma is a cancer that begins in squamous cells. Squamous cells are thin, flat cells that appear microscopically to be fish scales. Squamous cells are found in the tissues that form the surface of the skin, the lining of hollow organs of the body, and the passages of the respiratory and digestive tracts. Squamous cell carcinoma may occur in any of these tissues. Squamous cell carcinoma is the second most common type of lung cancer, accounting for approximately 30% of all cases.
Large cell carcinoma showed no signs of squamous or glandular maturation. Therefore, these tumors are usually diagnosed by default (when all other possibilities have been excluded). These tumors lacked any diagnostic features that indicated their diagnosis prior to biopsy. They tend to grow rapidly, metastasize early, and are closely associated with smoking. Large cell tumors are usually large, fluffy, well-defined, pink-grey masses with extensive bleeding and necrosis. Although they usually have central necrosis, they are rarely cavitated. They tend to be present in the mid to peripheral lung regions. They may be locally expanded to include segmental or sub-segmental bronchi. A variant of large cell carcinoma is giant cell carcinoma. This subtype is particularly aggressive and carries a very poor prognosis. These tumors usually exist as large peripheral masses with focal necrotic portions. They do not include large airways, except by direct expansion. Large cell carcinoma accounts for 10% -20% of all lung cancer cases.
Small cell lung cancer
SCLC is also known as oat cell lung cancer and is a type of lung cancer in which the cells appear small and round under a microscope. SCLC is considered to be different from other lung cancers due to its clinical and biological characteristics. Small cell lung cancer exhibits aggressive behavior and grows rapidly, spreads early to distant sites, is extremely sensitive to chemotherapy and radiation, and is often associated with different paracancer syndromes. Small cell carcinomas appear in locations around the bronchi and infiltrate the bronchial submucosa. Extensive metastasis occurs early in the disease process and often spreads to the mediastinal lymph nodes, liver, bone, adrenal glands and brain. In addition, the production of various peptide hormones leads to a wide range of paracancer syndromes; the most common of these are the production of antidiuretic hormone inappropriate secretion Syndrome (SIADH) and ectopic corticotropin (ACTH) syndrome. In addition, autoimmune phenomena can lead to various neurological syndromes, such as Lambert-Eaton syndrome. SCLC accounts for 20% of all cases. Carcinoid tumor
Carcinoid tumors are tumors that secrete large amounts of the hormone serotonin. Carcinoid tumors are also known as lymphoblastomas. The tumor usually occurs in the gastrointestinal tract, i.e. anywhere between the stomach and rectum (the most favored place is in the appendix) and can metastasize from there to the liver. In the liver the tumor produces and releases large amounts of serotonin into the systemic blood stream. This result is called carcinoid syndrome. It is directly caused by serotonin and includes flushing and blushing, swelling of the face (particularly around the eyes), flattened hemangiomas on the skin (small aggregates of dilated vessels), diarrhea, bronchospasm, coronary arteries, hypotension and stenosis of the tricuspid and pulmonary arteries (narrowing of the tricuspid and pulmonary valves of the heart), often accompanied by regurgitation. One or more of the following four types of treatment are used for carcinoid tumors: surgery (removal of cancer); radiotherapy (cancer cell killing using high dose X-rays); biotherapy (using the body's natural immune system to fight cancer); and chemotherapy (the use of drugs to kill cancer cells). Carcinoid tumors are considered to be a type of endocrine tumor due to their secretion of hormones (serotonin). Carcinoid tumors can occur as part of certain genetic disorders such as multiple endocrine tumors (MEN) type 1 and neurofibromatosis type 1 (NF1 or von lecklinghausen) disease. Carcinoid tumors account for 1% of all cases.
Lymphoma (lymphoma)
Lymphomas are types of cancer that involve cells of the immune system (called lymphocytes) and represent primarily cells that are involved in the body's lymphatic system. Lymphomas are malignant transformations of B or T cells or subtypes thereof. Lymphomas fall into one of two main types: hodgkin lymphoma (HL, previously known as hodgkin's disease) and all other lymphomas (non-hodgkin's lymphoma or NHL). Both types occur in the same location, may be associated with the same symptoms, and generally have a similar appearance upon physical examination. However, they are readily distinguishable by microscopic examination. Hodgkin's disease is developed by specific aberrant B lymphocyte lineages. NHLs can be derived from abnormal B or T cells and are distinguished by unique genetic markers. There are five subtypes of hodgkin's disease and about 30 subtypes of non-hodgkin's lymphoma. Because there are so many different lymphoma subtypes, classification of lymphoma is complex (it includes microscopic appearance as well as genetic and molecular markers). Many subtypes of NHL look similar, but they differ greatly in function and respond to different treatments with different cure probabilities. The HL subtypes differ microscopically and are classified based on microscopic differences as well as the extent of the disease.
EGFR inhibitors
EGFR inhibitors interrupt signaling through the Epidermal Growth Factor Receptor (EGFR) in target cells. Certain EGFR inhibitors such as erlotinib have been approved for the treatment of metastatic NSCLC. For advanced NSCLC, EGFR inhibitors such as gefitinib have been approved. Several more EGFR inhibitors are tested in clinical trials for the treatment of NSCLC and other lung cancers.
An "EGFR inhibitor" as described herein is a molecule that inhibits EGF receptor activity. Compounds (inhibitors of EGFR) can be readily identified by those skilled in the art using methods such as EGFR kinase assays that measure the ADP formed from the kinase reaction.
Inhibition of EGFR as a treatment of choice for lung cancer has been studied and has met with some success. Three EGFR inhibitors, erlotinib, gefitinib, and cetuximab, are currently approved for sale in the united states for the treatment of lung cancer.
ErlotinibApproved for the treatment of metastatic non-small cell lung cancer and pancreatic cancer that cannot be removed by surgery or that has metastasized. The small molecule drug inhibits the tyrosine kinase activity of EGFR.
GefitinibApproved for the treatment of patients with advanced non-small cell lung cancer. The small molecule drug is limited to use in patients whose treating physician appears to be currently benefiting from or to have previously benefited from gefitinib treatment. Gefitinib inhibits tyrosine kinase activity of Epidermal Growth Factor Receptor (EGFR), which is overproduced by many types of cancer cells.
CetuximabIs a monoclonal antibody approved for use in the treatment of some patients with head and neck cancer or squamous cell carcinoma of colorectal cancer. This treatment binds to the exterior of EGFR, thereby preventing the receptor from being activated by growth signals, which can inhibit signal transduction and lead to an anti-proliferative effect.
Other examples of EGFR inhibitors include, but are not limited to, panitumumab, vandetanib, lapatinib, canertinib, afatinib, netilmizumab (necitumumab), nimotuzumab, PF299804, RO5083945, ABT-806, and AP 26113.
PanitumumabApproved for the treatment of some patients with metastatic colon cancer. The monoclonal antibody binds to EGFR and prevents it from signaling growth.
VandetanibApproved for the treatment of patients with metastatic medullary thyroid carcinoma who are not amenable to surgery. The small molecule drug binds to and blocks the growth promoting activity of several tyrosine kinases including EGFR, several receptors for vascular endothelial growth factor receptor (VEGF) and RET.
LapatinibApproved for the treatment of certain types of advanced or metastatic breast cancer. The small molecule drug inhibits tyrosine kinase activity of several tyrosine kinases, including HER-2. Lapatinib treatment prevented HER-2 signaling from activating cell growth.
Canertinib is an orally bioavailable irreversible pan-ErbB tyrosine kinase inhibitor targeting EGFR, HER-2, ErbB-3, and ErbB-4. It effectively inhibits the growth of esophageal squamous cell carcinoma (which co-expresses EGFR and HER2) and inhibits the phosphorylation of MAPK and AKT. In vitro studies of human cancer cell lines indicate that canertinib results in a rapid, robust and durable inhibition of tyrosine kinase activity.
Afatinib is irreversible EGFR/HER 2. Afatinib showed potent activity on wild-type and mutant EGFR and HER2 in a cell-free in vitro kinase assay, which is similar to the potency of gefitinib on L858REGFR, but the L858R-T790MEGFR double mutant was about 100-fold more active than gefitinib for gefitinib resistance. Afatinib was effective in inhibiting the survival of lung cancer cell lines with wild-type (H1666) or L858R/T790M (NCI-H1975) EGFR. Evaluation was performed in a standard xenograft model of the epidermoid cancer cell line a 431. Daily oral treatment with 20mg/kg afatinib for 25 days resulted in significant tumor regression with a cumulative treated/control tumor volume ratio (T/C ratio) of 2%. Like lapatinib and neratinib, afatinib is the next generation Tyrosine Kinase Inhibitor (TKI) that irreversibly inhibits human epidermal growth factor receptor 2(Her2) and Epidermal Growth Factor Receptor (EGFR) kinases. Afatinib is active not only against EGFR mutations targeted by first generation TKIs such as erlotinib or gefitinib, but also against those mutations that are not sensitive to these standard treatments. Due to its additional activity against Her2, afatinib was investigated for breast cancer as well as other EGFR and Her2 driven cancers.
Nemuximab is a fully human IgGl monoclonal antibody directed against Epidermal Growth Factor Receptor (EGFR) with potential anti-tumor activity. Necoxib monoclonal is resistant to binding and blocking the ligand binding site of EGFR, thereby preventing activation of the receptor and subsequent dimerization. This may lead to inhibition of EGFR-dependent downstream pathways and thus inhibition of EGFR-dependent tumor cell proliferation and metastasis.
Nimotuzumab is a humanized monoclonal antibody directed against Epidermal Growth Factor Receptor (EGFR) with potential anti-tumor activity. Nimotuzumab binds to and inhibits EGFR, resulting in growth inhibition of EGFR-overexpressing tumor cells. The preparation can be used for treating radiotherapy.
PF299804 is a potent irreversible inhibitor of the human epidermal growth factor receptor (HER) -1/EGFR, -2, and-4 Tyrosine Kinases (TK), which is active in preclinical models of E-sensitivity and E-resistance. PF299804 has clinical activity in EGFRK inhibitor (TKI) refractory NSCLC in phase I/II trial.
RO5083945 is a glycoengineered (glycoengineered) anti-egfr igg1mAb that exhibits increased binding affinity for all Fc γ RIIIa variants expressed on immune effector cells. RO5083945 showed significantly improved cell killing in ADCC-based assays and greater activity in vivo models compared to cetuximab and panitumumab. Thus, RO5083945 has the potential to exhibit clinical activity in patients with solid tumors including KRAS mutant CRC.
ABT-806 is a humanized monoclonal antibody (MoAb) against human Epidermal Growth Factor Receptor (EGFR) with anti-tumor activity. MoAbABT-806 targets EGFR deletion variants, de2-7EGFR, and wild-type EGFR expressed in cells overexpressing the receptor, thereby preventing activation and subsequent dimerization of the receptor; reduction of receptor activation and dimerization results in inhibition of signal transduction and anti-proliferative effects. This MoAb targets cells expressing aberrant EGFR, making it an ideal candidate for the generation of radioisotope or toxin conjugates.
AP26113 is an orally available inhibitor of the receptor tyrosine kinase Anaplastic Lymphoma Kinase (ALK) and Epidermal Growth Factor Receptor (EGFR) with potential anti-tumor activity. The dual ALK/EGFR inhibitor AP26113 binds to and inhibits ALK kinase and ALK fusion proteins as well as EGFR and mutants. This results in inhibition of ALK kinase and EGFR kinase, disrupting their signaling pathways and ultimately inhibiting tumor cell growth in sensitive tumor cells. In addition, AP26113 appears to overcome mutation-based resistance. ALK belongs to the insulin receptor superfamily and plays an important role in nervous system development; ALK dysregulation and gene rearrangement are associated with a range of tumors. EGFR is overexpressed in a variety of cancer cell types.
Other treatments
Other useful lung cancer treatments that may be advantageously used in combination with the treatments disclosed herein include, but are not limited to, radiation therapy, chemotherapy, antibody therapy, and tyrosine kinase inhibitors as adjunctive therapies.
Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill or prevent the growth of cancer cells. Chemotherapy is a cancer treatment that employs drugs to arrest the growth of cancer cells by killing them or by arresting their division. When chemotherapy is administered orally or injected into veins or muscles, the drug enters the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly in the spine, organs or body cavities, such as the abdominal cavity, the drug primarily affects the cancer cells in those areas (regional chemotherapy). The manner in which chemotherapy is administered depends on the type and stage of cancer being treated.
Different chemotherapeutic agents for treating lung cancer are known in the art. Cytotoxic agents used to treat lung cancer include carboplatin (e.g.,) The amount of cisplatin (e.g.,) The amount of crizotinib (e.g.,) Etoposide (e.g. etoposide)) Etoposide phosphate (e.g.,) Gemcitabine hydrochloride (for example,) Gemcitabine-cisplatin, methotrexate (e.g.,FolexMethotrexate) Paclitaxel (e.g. paclitaxel)) The amount of pemetrexed disodium (e.g.,) And topotecan hydrochloride (e.g.)。
Monoclonal antibody therapy is a cancer treatment that uses antibodies prepared in the laboratory from a single type of immune system cell. These antibodies can recognize substances on cancer cells or normal substances that can help cancer cells grow. The antibody attaches to the substance and kills, blocks or prevents the spread of cancer cells. Monoclonal antibodies were administered by infusion. Monoclonal antibodies can be used alone or to carry drugs, toxins, or radioactive substances directly to cancer cells. Monoclonal antibodies are also used in combination with chemotherapy as an adjuvant therapy.
BevacizumabIs a recombinant humanized monoclonal antibody aiming at Vascular Endothelial Growth Factor (VEGF), a pro-angiogenic cytokine. Bevacizumab binds to VEGF and inhibits VEGF receptor binding, thereby preventing growth and maintenance of tumor blood vessels. Bevacizumab is currently used to treat several types of cancer, including colorectal, lung, breast and kidney cancerCancer and certain types of glioblastoma.
Other exemplary treatments that may be advantageously combined with the compositions and treatments disclosed herein may include, but are not limited to, administration of agents including, but not limited to, lapatinib (alone or in combination with capecitabine), docetaxel, epirubicin, epothilone A, B or D, goserelin acetate, paclitaxel, disodium pamidronate, bevacizumab or trastuzumab.
In some embodiments, the other treatment comprises chemotherapy comprising administering to the subject one or more of: doxorubicin, cyclophosphamide, paclitaxel, lapatinib, capecitabine, trastuzumab, bevacizumab, gemcitabine, eribulin, or albumin-bound paclitaxel.
Methods for treating lung cancer
One embodiment provides a method of treating cancer in an EGFR inhibitor naive patient who progressed following a previous treatment, wherein the method comprises: (1) determining the level of E-cadherin expression in the patient; (2) selecting a patient exhibiting high E cadherin expression levels with a score of + 3; and (3) administering to the patient a combination comprising entinostat and an EGFR inhibitor. In some cases, entinostat is administered to fasting patients.
In some embodiments, the Cmax is higher when entinostat is administered without food than when entinostat is administered with food. In some embodiments, the ratio of Cmax after entinostat administration in fasted to Cmax after entinostat administration in fed conditions is at least about 2: 1. In one embodiment, the ratio of Cmax after entinostat administration in fasted to Cmax after entinostat administration in fed conditions is at least about 3: 1. In one embodiment, the ratio of Cmax after entinostat administration in fasted to Cmax after entinostat administration in fed conditions is at least about 4: 1. In one embodiment, the ratio of Cmax after entinostat administration in fasted to Cmax after entinostat administration in fed conditions is at least about 5: 1. In one embodiment, the ratio of Cmax after entinostat administration in fasted to Cmax after entinostat administration in fed conditions is at least about 6: 1. In one embodiment, the ratio of Cmax after entinostat administration in fasted to Cmax after entinostat administration in fed conditions is at least about 7: 1.
In some embodiments, the Tmax for entinostat administered without food is lower compared to the Tmax for entinostat administered with food. In some embodiments, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 2: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 3: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 4: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 5: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 6: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 7: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 8: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 9: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 10: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 11: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 12: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 13: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 14: 1. In one embodiment, the ratio of Tmax after administration under fed to Tmax after administration under fasted conditions is at least about 15: 1.
In some embodiments, the EGFR inhibitor is administered at a different time than the day that entinostat is administered. In one embodiment, an EGFR inhibitor is administered to a fasting patient.
Another embodiment provides a method wherein the prior treatment is a prior chemotherapy.
Another embodiment provides a method wherein the prior treatment is two or more prior chemotherapies.
Another embodiment provides a method wherein high E cadherin expression levels are determined by ELISA, immunohistochemistry, immunocytochemistry or measuring E cadherin methylation levels. Another embodiment provides a method wherein high E cadherin expression levels are determined by immunohistochemistry. Another embodiment provides a method wherein the high E cadherin expression level score is +3, as determined by immunohistochemistry.
Another embodiment provides a method wherein the cancer is lung cancer.
Another embodiment provides a method wherein the lung cancer is non-small cell lung cancer.
Another embodiment provides a method wherein the EGFR inhibitor administered in combination with entinostat is erlotinib.
Another embodiment provides a method wherein entinostat and the EGFR inhibitor are administered sequentially or simultaneously in any order. Another embodiment provides a method wherein the entinostat and EGFR inhibitor are administered simultaneously. Another embodiment provides a method wherein the EGFR inhibitor is administered first.
Another embodiment provides a method wherein the EGFR inhibitor is administered daily and the entinostat is administered periodically. Another embodiment provides a method wherein the EGFR inhibitor is administered daily and the entinostat is administered weekly.
Another embodiment provides a method of treating cancer in an EGFR inhibitor naive patient who progressed on prior therapy, wherein said patient exhibits high levels of E-cadherin expression, comprising administering to the patient a combination comprising entinostat and an EGFR inhibitor.
Another embodiment provides a method of treating cancer in an EGFR inhibitor naive patient that progressed following a previous treatment, wherein said patient exhibits high levels of E-cadherin expression, wherein the method further comprises administering to the subject one or more additional treatments in addition to the combination of entinostat and the EGFR inhibitor. Another embodiment provides such a method, wherein the one or more treatments comprise one or more of: radiotherapy, chemotherapy, high dose chemotherapy plus stem cell transplantation, and monoclonal antibody therapy. Another embodiment provides the method wherein the radiation therapy comprises internal and/or external radiation therapy. Another embodiment provides the method wherein the chemotherapy comprises administering to the subject one or more of the following: doxorubicin, cyclophosphamide, paclitaxel, lapatinib, capecitabine, trastuzumab, bevacizumab, gemcitabine, eribulin, or albumin-bound paclitaxel. Another embodiment provides such a method, wherein the chemotherapy comprises administering to the subject one or more IGF-1R inhibitors. Another embodiment provides such a method, wherein the IGF-1R inhibitor is AEW 541.
One embodiment provides a method of treating non-small cell lung cancer in a patient in need thereof comprising orally administering erlotinib and entinostat, wherein the entinostat is administered to a fasting patient. Another embodiment provides a method of treating non-small cell lung cancer, wherein the entinostat Tmax is less than 1 hour after administration. Another embodiment provides a method of treating non-small cell lung cancer, wherein the entinostat Tmax is less than 90 minutes after administration. Another embodiment provides a method of treating non-small cell lung cancer, wherein the entinostat Tmax is less than 2 hours after administration. Another embodiment provides a method of treating non-small cell lung cancer, wherein the entinostat Tmax is 30 minutes to 2 hours after administration. Another embodiment provides a method of treating non-small cell lung cancer, wherein the Cmax of entinostat is at least 150ng/mL after oral administration of entinostat. Another embodiment provides a method of treating non-small cell lung cancer, wherein the Cmax of entinostat is at least 125ng/mL after oral administration of entinostat. Another embodiment provides a method of treating non-small cell lung cancer, wherein the Cmax of entinostat is at least 100ng/mL after oral administration of entinostat. Another embodiment provides a method of treating non-small cell lung cancer, wherein the Cmax of entinostat is at least 80ng/mL after oral administration of entinostat. Another embodiment provides a method of treating non-small cell lung cancer, wherein the Cmax of entinostat is at least 50ng/mL after oral administration of entinostat. Another embodiment provides a method of treating non-small cell lung cancer, wherein about 10mg of entinostat is administered. Another embodiment provides a method of treating non-small cell lung cancer, wherein about 1mg to about 20mg of entinostat is administered. Another embodiment provides a method of treating non-small cell lung cancer, wherein the entinostat is administered once every 14 days. Another embodiment provides a method of treating non-small cell lung cancer, wherein entinostat is administered for one month. Another embodiment provides a method of treating non-small cell lung cancer, wherein the patient does not eat within 2 hours prior to administration of entinostat. Another embodiment provides a method of treating non-small cell lung cancer, wherein the patient does not eat within 1 hour prior to administration of entinostat. Another embodiment provides a method of treating non-small cell lung cancer, wherein the patient does not eat within 1 hour after administration of entinostat. Another embodiment provides a method of treating non-small cell lung cancer, wherein the patient does not eat within 30 minutes after administration of entinostat. Another embodiment provides a method of treating non-small cell lung cancer, wherein erlotinib is administered at a time different from the day of entinostat administration. Another embodiment provides a method of treating non-small cell lung cancer wherein erlotinib is administered once daily to said fasted patient. Another embodiment provides a method of treating non-small cell lung cancer, wherein the patient does not eat within 2 hours prior to administration of erlotinib. Another embodiment provides a method of treating non-small cell lung cancer, wherein the patient does not eat within 1 hour after administration of erlotinib. Another embodiment provides a method of treating non-small cell lung cancer wherein about 150mg of erlotinib is administered.
Oral preparation
Oral formulations containing the active pharmaceutical ingredient as described herein may include any conventionally used oral forms including tablets, capsules, pills, troches, lozenges, pastilles, cachets, micropellets, medicated chewing gums, granules, bulk powders, effervescent or non-effervescent powders or granules, solutions, emulsions, suspensions, solutions, wafers, dispersible capsules (sprinkles), elixirs, syrups, buccal forms (buccalforms) and oral liquids. Capsules may contain mixtures of the active compound with inert fillers and/or diluents such as the pharmaceutically acceptable starches (e.g., corn, potato or tapioca starch), sugars, artificial sweeteners, powdered celluloses, such as crystalline and microcrystalline celluloses, flours, gelatins, gums, and the like. Useful tablet formulations may be prepared by conventional tableting, wet or dry granulation methods using pharmaceutically acceptable diluents, binders, lubricants, disintegrants, surface modifying agents (including surfactants), suspending or stabilizing agents (including, but not limited to, magnesium stearate, stearic acid, talc, sodium lauryl sulfate, microcrystalline cellulose, carboxymethylcellulose calcium, polyvinylpyrrolidone, gelatin, alginic acid, acacia gum, xanthan gum, sodium citrate, silicate complexes, calcium carbonate, glycerol, dextrin, sucrose, sorbitol, dicalcium phosphate, calcium sulfate, lactose, kaolin, mannitol, sodium chloride, talc, dry starch and powdered sugar). In some embodiments, the surface modifying agent comprises a nonionic surface modifying agent and an anionic surface modifying agent. For example, surface modifying agents include, but are not limited to, poloxamer 188, benzalkonium chloride, calcium stearate, cetostearyl alcohol, cetomacrogol emulsifying wax, sorbitan esters, colloidal silica, phosphates, sodium lauryl sulfate, magnesium aluminum silicate, and triethanolamine. The oral formulations herein may utilize standard delayed release formulations or extended release formulations to modify the absorption of the active compound. The oral formulations may also consist of water or fruit juices containing the active ingredient, which may contain suitable solubilizers or emulsifiers as required.
Oral administration
As described herein, the combination therapies described herein can be administered simultaneously or can be administered in a staggered regimen, with the entinostat being administered at a different time than the aromatase inhibitor during the course of chemotherapy. The time difference between administering the two compounds may range from several minutes, hours, days, weeks, or longer. Thus, the term combination does not necessarily mean that the components are administered at the same time or in a single dose, but rather that each component is administered over the desired treatment period. These agents may also be administered by different routes. Generally for a chemotherapeutic regimen, the course of chemotherapy may be repeated after several weeks and may follow the same schedule of administration of the two compounds, or may be modified based on patient response.
In other embodiments, the pharmaceutical compositions provided herein can be provided in solid, semi-solid, or liquid dosage forms for oral administration. Oral administration, as used herein, also includes buccal, lingual and sublingual administration. Suitable oral dosage forms include, but are not limited to, tablets, capsules, alkyl, lozenges, troches (losenges), pastilles (pastilles), cachets, pellets, medicated chewing gums, granules, bulk powders, effervescent or non-effervescent powders or granules, solutions, emulsions, suspensions, solutions, wafers (wafers), dispersible capsules (sprinkles), elixirs, and syrups. In addition to the active ingredient, the pharmaceutical composition may contain one or more pharmaceutically acceptable carriers or excipients, which include, but are not limited to, binders, fillers, diluents, disintegrants, wetting agents, lubricants, glidants, colorants, dye migration inhibitors, sweeteners, and flavoring agents.
Binders or granulators impart cohesiveness to the tablets to ensure that the tablets remain intact after compression. Suitable binders or pelletizers include, but are not limited to, starches, such as corn STARCH, potato STARCH, and pregelatinized STARCH (e.g., STARCH 1500); gelatin; sugars such as sucrose, glucose, dextrose, molasses, and lactose; natural and synthetic gums, such as acacia, alginic acid, alginates, Irish moss extract, Panwar gum, Indian gum, isabgolhusks mucilage, carboxymethylcellulose, methylcellulose, polyvinylpyrrolidone (PVP), magnesium aluminum silicate, larch arabinogalactan, tragacanth powder, and guar gum; celluloses such as ethyl cellulose, cellulose acetate, calcium carboxymethyl cellulose, sodium carboxymethyl cellulose, methyl cellulose, hydroxyethyl cellulose (HEC), hydroxypropyl cellulose (HPC), hydroxypropyl methyl cellulose (HPMC); microcrystalline celluloses, such as AVICEL-PH-101, AVICEL-PH-103, AVICELRC-581, AVICEL-PH-105(FMCCorp., Marcushook, Pa.); and mixtures thereof. Suitable fillers include, but are not limited to, talc, calcium carbonate, microcrystalline cellulose, cellulose powder, dextrates, kaolin, mannitol, silicic acid, sorbitol, starch, pregelatinized starch, and mixtures thereof. In the pharmaceutical compositions provided herein, the binder or filler may be present in an amount of about 50% to about 99% by weight.
Suitable diluents include, but are not limited to, dicalcium phosphate, calcium sulfate, lactose, sorbitol, sucrose, inositol, cellulose, kaolin, mannitol, sodium chloride, dry starch, and powdered sugar. Some diluents, such as mannitol, lactose, sorbitol, sucrose, and inositol, when present in sufficient amounts, can impart some compressed tablet properties to allow it to disintegrate in the mouth by chewing. Such compressed tablets may be used as chewable tablets.
Suitable disintegrants include, but are not limited to, agar; bentonite; cellulose such as methyl cellulose and carboxymethyl cellulose; a wood product; a natural sponge; a cation exchange resin; alginic acid; gums, such as guar gum and veegum HV; citrus pulp; crosslinked celluloses, such as crosslinked carboxymethyl cellulose; crosslinked polymers, such as crospovidone; cross-linked starch; calcium carbonate; microcrystalline cellulose, such as sodium starch glycolate; potassium polacrilin; starches, such as corn starch, potato starch, tapioca starch, and pregelatinized starch; clay; align; and mixtures thereof. The amount of disintegrant in the pharmaceutical compositions provided herein varies with the type of formulation and is readily discernible to one of ordinary skill in the art. The pharmaceutical compositions provided herein can comprise from about 0.5% to about 15% or from about 1% to about 5% by weight of a disintegrant.
Suitable lubricants include, but are not limited to, calcium stearate; magnesium stearate; mineral oil; light mineral oil; glycerol; sorbitol; mannitol; glycols, such as glyceryl behenate (glycerylbehenate) and polyethylene glycol (PEG); stearic acid; sodium lauryl sulfate; talc; hydrogenated vegetable oils including peanut oil, cottonseed oil, sunflower oil, sesame oil, olive oil, corn oil and soybean oil; zinc stearate; ethyl oleate; ethyl laurate; agar; starch; stone pine nuts; silica or silica gels, e.g.200(w.r.graceco., Baltimore, MD) and(cabot co. of boston, MA); and mixtures thereof. The pharmaceutical compositions provided herein can comprise from about 0.1% to about 5% by weight of the lubricant.
Suitable glidants include colloidal silicon dioxide,(cabot co. of boston, MA) and asbestos-free talc. Coloring agents include any approved, certified, water-soluble FD&A C dye, and a water-insoluble FD suspended on hydrated alumina&C dyes, and lakes and mixtures thereof. Lakes are prepared by absorbing water-soluble dyes intoHeavy metal hydrous oxides, which give the dye in insoluble form. Flavoring agents include natural flavors extracted from plants such as fruits, and synthetic compound mixtures that produce a pleasant taste sensation such as peppermint and methyl salicylate. Sweetening agents include sucrose, lactose, mannitol, syrups, glycerol and artificial sweeteners such as saccharin and aspartame. Suitable emulsifying agents include gelatin, acacia, tragacanth, bentonite and surfactants such as polyoxyethylene sorbitan monooleate (R: (R) (R))20) Polyoxyethylene sorbitan monooleate 80 (A)80) And triethanolamine oleate. Suspending and dispersing agents include sodium carboxymethylcellulose, pectin, tragacanth, magnesium aluminium silicate, acacia, sodium carboxymethylcellulose, hydroxypropylmethylcellulose and polyvinylpyrrolidone. The antiseptic comprises glycerol, methyl paraben, propyl paraben, benzoic acid, sodium benzoate and alcohol. Wetting agents include propylene glycol monostearate, sorbitan monooleate, diethylene glycol monolaurate and polyoxyethylene lauryl ether. Solvents include glycerol, sorbitol, ethanol and syrup. Examples of non-aqueous liquids utilized in emulsions include mineral oil and cottonseed oil. Organic acids include citric acid and tartaric acid. Sources of carbon dioxide include sodium bicarbonate and sodium carbonate.
It will be appreciated that many carriers and excipients may serve several functions, even in the same formulation.
In other embodiments, the pharmaceutical compositions provided herein can be provided in compressed tablets, molded tablets, chewable lozenges, fast-dissolving tablets, multiple compressed tablets or enteric coated tablets, sugar-coated tablets or film-coated tablets. Enteric coated tablets are compressed tablets coated with a substance that resists the action of gastric acid but dissolves or disintegrates in the intestine, thereby protecting the active ingredient from the acidic environment of the stomach. Enteric coatings include, but are not limited to, fatty acids, fats, phenyl salicylate, waxes, shellac, ammoniated shellac, and cellulose acetate phthalate. Sugar-coated tablets are compressed tablets coated with a sugar coating, which advantageously masks unpleasant tastes or odors and protects the tablets from oxidation. Film-coated tablets are compressed tablets covered with a thin layer or film of water-soluble material. Film-coated tablets include, but are not limited to, hydroxyethyl cellulose, sodium carboxymethyl cellulose, polyethylene glycol 4000, and cellulose acetate phthalate. Film coatings give the same commonality as sugar coatings. Multiple compressed tablets are compressed tablets made by more than one compression cycle, which include layered tablets and compression-coated or dry-coated tablets.
Tablet dosage forms may be prepared from the active ingredient in powdered, crystalline, or granular form, alone or in combination with one or more carriers or excipients described herein, including binders, disintegrants, controlled release polymers, lubricants, diluents, and/or colorants. Flavoring and sweetening agents are particularly useful in the formation of chewable tablets and lozenges.
The pharmaceutical compositions provided herein can be provided in soft or hard capsules, which can be made from gelatin, methylcellulose, starch, or calcium alginate. Said hard gelatin capsules, also known as Dry Fill Capsules (DFC), are composed of two parts, one inserted into the other; thus completely encapsulating the active ingredient. Soft Elastic Capsules (SEC) are soft, spherical shells, such as gelatin shells, which are plasticized by the addition of glycerol, sorbitol or similar polyols. The soft gelatin shell may contain a preservative to prevent microbial growth. Suitable preservatives are those as described herein, including methyl and propyl parabens, and sorbic acid. The liquid, semi-solid, and solid dosage forms provided herein can be encapsulated in a capsule. Suitable liquid and semi-solid dosage forms include solutions and suspensions of propylene carbonate, vegetable oils or triglycerides. Capsules containing such solutions may be prepared as described in U.S. patents 4,328,245, 4,409,239, and 4,410,545. The capsules may also be coated as known to those skilled in the art to improve or maintain dissolution of the active ingredient.
In other embodiments, the pharmaceutical compositions provided herein can be provided in liquid and semi-solid dosage forms, including emulsions, solutions, suspensions, elixirs, and syrups. Emulsions are two-phase systems in which one liquid is dispersed throughout another in the form of globules, which can be either oil-in-water or water-in-oil. Emulsions may include pharmaceutically acceptable non-aqueous liquids or solvents, emulsifiers, and preservatives. Suspensions may include a pharmaceutically acceptable suspending agent and a preservative. The aqueous alcoholic solution may include pharmaceutically acceptable aldehydes, such as di (lower alkyl) acetals of lower alkyl aldehydes (the term "lower" means that the alkyl group has 1 to 6 carbon atoms), such as acetaldehyde diethyl acetal; and water-miscible solvents having one or more hydroxyl groups, such as propylene glycol and ethanol. Elixirs are clear, sweetened, hydroalcoholic solutions. Syrups are concentrated aqueous solutions of sugars, such as sucrose, and may also contain preservatives. For liquid dosage forms, for example, a solution in polyethylene glycol may be diluted with a sufficient amount of a pharmaceutically acceptable liquid carrier, such as water, to be conveniently measured for administration.
Other useful liquid and semi-solid dosage forms include, but are not limited to, those comprising the active ingredients provided herein and dialkylated mono-or polyalkylene glycols including 1, 2-dimethoxymethane, diethylene glycol dimethyl ether, triethylene glycol dimethyl ether, tetraethylene glycol dimethyl ether, polyethylene glycol-350-dimethyl ether, polyethylene glycol-550-dimethyl ether, polyethylene glycol-750-dimethyl ether, where 350, 550 and 750 refer to the approximate average molecular weight of the polyethylene glycol. These formulations may further comprise one or more antioxidants, such as Butylated Hydroxytoluene (BHT), Butylated Hydroxyanisole (BHA), propyl gallate, vitamin E, hydroquinone, hydroxycoumarins, ethanolamine, lecithin, cephalin, ascorbic acid, malic acid, sorbitol, phosphoric acid, bisulfite, sodium metabisulfite, thiodipropionic acid and its esters, and dithiocarbamates.
The pharmaceutical compositions provided herein for oral administration may also be provided as liposomes, micelles, microspheres or nanosystems. Micellar dosage forms can be prepared as described in U.S. patent 6,350,458.
In other embodiments, the pharmaceutical compositions provided herein can be provided in non-effervescent or effervescent granules and powders for reconstitution into liquid dosage forms. Pharmaceutically acceptable carriers and excipients used in non-effervescent granules or powders may include diluents, sweeteners and wetting agents. Pharmaceutically acceptable carriers and excipients used in effervescent granules or powders may include organic acids and sources of carbon dioxide.
Colorants and flavoring agents may be used in all of the above dosage forms.
The pharmaceutical compositions provided herein can be formulated in immediate release or modified release dosage forms, including delayed release, sustained release, pulsatile release, controlled release, targeted release, and programmed release forms.
In other embodiments, the pharmaceutical compositions provided herein can be co-formulated with other active ingredients that do not impair the desired therapeutic effect, or with substances that supplement the desired effect.
Examples
Example 1Phase 1 randomized open label study to evaluate the effect of food on entinostat pharmacokinetics in postmenopausal women with locally recurrent or metastatic ER + breast cancer and in men and women with progressive non-small cell lung cancer
Scheme(s)
Title: phase 1 study to evaluate the effect of food on entinostat pharmacokinetics in postmenopausal women with locally recurrent or metastatic ER + breast cancer and in men and women with progressive non-small cell lung cancer
And (3) a research stage: stage 1
Indications: breast cancer; non-small cell lung cancer
The main aims are as follows:
to assess the effect of food on the pharmacokinetics of entinostat in women with breast cancer and men and women with non-small cell lung cancer (NSCLC).
Secondary objective:
safety: to assess the safety and tolerability of entinostat in combination with exemestane or erlotinib as measured by adverse events, laboratory parameters and electrocardio-evaluation
And (3) exploring a target:
to determine the effect of food on the extent of changes in protein lysine acetylation induced by entinostat administration
To evaluate the degree of acetylation correlated with the entinostat plasma concentration and duration of treatment in patients receiving entinostat.
To determine whether the addition of exemestane or erlotinib reaches the extent of acetylation change induced by entinostat administration
Research and design:
this is an entinostat phase I, randomized, open label, two-stage, two-panel crossover study. Patients were randomized into 1:1 groups to receive entinostat 10mg with or without food on cycle 1 day 1 (C1D 1). Patients randomized to receive entinostat with food at C1D1 will receive a second dose of entinostat 10mg without food on cycle 1 day 15 (C1D 15). Similarly, patients randomized to receive entinostat without food at C1D1 will receive a second dose of entinostat 10mg with food at C1D 15. The random groupings will be ranked by gender. Each cycle in the study will last 28 days. Blood samples will be obtained prior to dosing and serial blood samples will be taken after each dose to assess pharmacokinetics. In addition, blood samples were drawn for evaluation of entinostat acetylation.
For cycle 2 and all subsequent cycles, all patients will continue to receive 10mg of entinostat on days 1 and 15 of each cycle. Those patients suffering from breast cancer will also receive 25mg of exemestane orally (po) once daily (qd) from cycle 2 day 1. Those patients with NSCLC will also receive 150mg of erlotinib orally once daily starting on cycle 2 day 1.
Patients will be evaluated at screening and at pre-specified times during study enrollment using standard clinical and laboratory evaluations. The tumor response of the patient will also be evaluated after every 2 cycles. Tumor progression will be assessed by CT, MRI or other suitable radiological studies. The patient will continue to receive their appropriate study treatment cycle until tumor development or an adverse event occurs, as determined by the investigator, necessitating treatment cessation.
End point:
major pharmacokinetic endpoint
Cmax, maximum plasma concentration
Tmax, time to maximum plasma concentration
AUClast, area under the plasma concentration-time curve from time zero to last measurable concentration
AUCinf, area under the plasma concentration-time curve extrapolated from time zero to infinity by AUClast + Clast/λ z described below
λ z, endpoint elimination rate constant
Secondary pharmacodynamic endpoint
Changes in protein lysine acetylation relative to baseline as measured by peripheral blood mononuclear cells
Safety endpoint
Treatment of urgent adverse events, severe adverse events, adverse events leading to permanent cessation of study drug, and incidence of death occurring within 30 days of the last dose of study drug
Changes in laboratory, vital signs and electrocardiographic results from baseline
Sample size: up to 28 patients (about 14 patients with breast cancer and 14 patients with NSCLC, with a minimum of 4 male patients) will be enrolled to ensure that 24 patients (about 12 per treatment group) complete cycle 1 of study treatment.
Summary of subject eligibility criteria: the study will recruit postmenopausal women with histologically or cytologically confirmed estrogen receptor positive (ER +) breast cancer at the time of diagnosis that the disease has progressed to the point where the investigator determined that the patient is a candidate to receive exemestane. Furthermore, adults with cytologically or histologically confirmed stage IIIb or IV NSCLC will be enrolled as qualified candidates for erlotinib treatment. All patients must be at least 18 years old and the Eastern Cooperative Oncology Group (ECOG) status is 0 or 1.
The product studied was:
entinostat is a synthetic small molecule with molecular weights of the formulae C21H20N4O3 and 376.41. Entinostat is classified as an antineoplastic agent, and functions as a histone deacetylase inhibitor, particularly by promoting the hyperacetylation of nucleosome histones. Entinostat is orally bioavailable and will be provided as a yellow coated tablet containing 5.0mg of active ingredient.
Study treatment:
one cycle will be identified as a 28 day study treatment.
All patients were: period 1 only
Group A: oral entinostat 10mg on day 1 in fasted and day 15 in fed conditions
Group B: oral entinostat 10mg on day 1 in the case of test food and on day 15 in the case of test fasting
Breast cancer patients only: cycle 2 and all subsequent cycles:
will administer exemestane 25mg once daily starting on day 1 of cycle 2
Oral administration of a 10mg dose of entinostat at least 2 hours after breakfast on days 1 and 15, followed by a fasting of at least 1 hour
NSCLC patients only: cycle 2 and all subsequent cycles
Will be administered erlotinib 150mg once daily starting on cycle 2 day 1
Oral administration of a 10mg dose of entinostat at least 2 hours after breakfast on days 1 and 15, followed by a fasting of at least 1 hour
Pharmacokinetic evaluation, all patients (study cycle 1, day 1 and 15 only)
Patients were administered one of two treatments, according to their randomized cohort: 10mg entinostat in the fed case of the test or 10mg entinostat in the fasted case of the test. All treatments will be given in a single dose with 240mL of water. Water will be allowed in all treatment groups as needed for up to 2 hours before dosing and then restricted up to 2 hours after dosing (except for liquid consumed during breakfast in the fed treatment group). Blood will be obtained for determining entinostat concentrations at the following times: pre-dose (within 60 minutes of administration), and subsequently 0.25, 0.5, 1, 1.5, 2,3, 4, 6, 8, 10, 12, 24, 72, 120, 168, 240 and 336 hours post-dose. ECGs will be acquired in triplicate at-60 and-45 minutes prior to dosing and at the same time points as PK blood samples. A Holter monitor will be used on days 1 and 15 of cycle 1 from pre-dose to 12 hours post-dose.
Pharmacodynamic evaluation, all patients
Study cycle 1 days 1 and 15
Blood will be obtained for determining entinostat acetylation at the following times: pre-dose (within 60 minutes of administration), and subsequently 12, 24, 168 and 336 hours post-dose.
Study day 1 of cycle 3
Blood will be taken for determination of protein acetylation prior to dosing on day 1 of cycle 3.
End of study evaluation
Blood will be obtained for determining protein acetylation at the final study visit.
Duration of study
The patient will continue to receive regimen therapy until suffering from a progressive disease or unacceptable or intolerable toxicity.
And (4) counting attention items:
the difference in mean bioavailability for fed and fasted states will be compared using the Schuirmann two-way one-sided test procedure for the interval hypothesis. Will pass based on AUClastAnd CmaxThe degree of entinostat exposure to determine the difference in mean bioavailability.
The following set of assumptions will be tested for comparing fed versus fasted states based on logarithmically transformed data:
H0FedFastedless than or equal to 0.80 or muFedFasted≥1.25
H1:0.80<μFedFasted<1.25
Wherein muFedAnd muFastedMeans the overall mean AUC in the fed and fasted conditions, respectivelylast、AUCinfOr Cmax. A total of 24 evaluable patients (12 per treatment group) will be required to test the above mentioned differences in mean bioavailability between fed and fasted conditions with a probability of 90% and a type I error rate of 5%. Inter-patient Coefficient of Variation (CV) is assumedThe value was found to be 22%. The true mean ratio between fed and fasted conditions was assumed to be 1.0.
Patients will be considered evaluable by the PK analysis if they receive entinostat during each treatment period according to the conditions determined by random grouping (i.e., fed or fasted). The patient must also have sufficient plasma concentration-time data from each treatment session to provide a meaningful assessment of PK parameters (e.g., Cmax, AUClast).
Individual patient concentration-time data will be listed and presented graphically in linear and logarithmic scales. Concentration-time data will be summarized descriptively in tabular and graphical form (linear and logarithmic scales). PK parameters estimated using a non-compartmental approach will be calculated using winnonlin version 5.1 or higher. Such estimates will be listed and summarized illustratively in tabular and graphical form.
For selected PK parameters (e.g., Cmax, AUClast), a linear mixed effect ANOVA model will be used for comparison between fed and fasted conditions. The model will include treatment status (fed, fasted), stage and group as fixed influencing factors, and patients within the group as random influencing factors. The evaluation of bioequivalence will be based on the classical (shortest) confidence interval method, which operationally corresponds to the Schuirmann two-way one-sided test procedure of interval hypothesis. Estimates from the ANOVA model will be used to calculate a 90% confidence limit for the ratio of the true mean AUC for fed and fasted conditions. If the 90% confidence interval of the ratio (back-transformed) is fully contained within the 80% to 125% equivalence limit, bioequivalence of mean bioavailability will be inferred.
Descriptive statistics and graphical displays will be used to summarize the time to reach the maximum observed plasma concentration (Tmax) of entinostat in fed and fasted conditions. Single patient differences in Tmax will be calculated between fed and fasted cases; a symmetric nonparametric confidence interval for the median difference will be provided. The Wilcoxon signed rank test (Wilcoxon's ssignandracktest) will be used to make a rational comparison of Tmax between fed and fasted cases.
Changes in protein lysine acetylation, the primary pharmacodynamic parameter of this study, will be analyzed in the same or similar manner as described above for the PK analysis. PK combinatorial analysis via acetylation may also be performed.
Safety data analysis was performed on all entinostat patients receiving at least one dose. The analysis results will consist of a summary of data for clinical and laboratory parameters as well as adverse events. Unless otherwise stated, the safety analysis will be performed by a preliminary diagnosis. The number and percentage of patients with one or more adverse events will be summarized by relationship to single study treatment and by severity grade. NCI-CTCAE (version 4.0) will be used to determine severity level. The adverse events will be encoded using a medical regulatory activity terminology dictionary (MedDRA). Descriptive statistics will be employed to summarize laboratory parameters by change from baseline and a data list of clinically significant abnormalities. Descriptive statistics will be employed to summarize vital signs and ECG data by changes from baseline values.
Results
The results of the food effect study are shown herein. Fig. 1 shows the time plot of the mean concentration after administration of 10mg entinostat in fasted or fed conditions. A summary of pharmacokinetic parameters is shown in table 1.
Co-administration of 10mg entinostat with food resulted in a lag in drug absorption and a delay in Tmax (median Tmax 0.76 h in the fasting case and 11 h in the fed case). A significant decrease in the maximum drug concentration was observed (a 71% decrease in Cmax). The overall exposure, estimated by AUClast and AUCinf, was reduced by about 15-17% when entinostat was administered with a high fat diet. The mean elimination half-life of entinostat was estimated as 140 hours in the fasted case and 178 hours in the fed case. There was a high degree of variation in the half-life estimates for the fed groups (% CV-70%), which may be attributed to the small sample size. A few individuals in the fed group did have significantly prolonged t1/2 values. the median value of t1/2 indicates that the two groups are comparable.
And (4) conclusion: the food effect was evident for entinostat when it was co-administered with a high fat diet, resulting in delayed tmax and decreased Cmax and AUC.
Example 2
A method of treating postmenopausal women with locally recurrent or metastatic estrogen receptor positive breast cancer by administering entinostat and a non-steroidal aromatase inhibitor, exemestane.
The aim of this study was to evaluate the safety and efficacy of entinostat in combination with exemestane in the treatment of advanced breast cancer.
The primary outcome measure was to compare the effectiveness of exemestane alone and exemestane plus entinostat, as determined by the duration of Progression Free Survival (PFS) measured from randomized date.
A secondary outcome measure is the comparison of Objective Response Rate (ORR) and Clinical Benefit Rate (CBR),
and evaluating the safety and tolerability of the combination of entinostat and exemestane as measured by adverse events and laboratory safety parameters.
Design of research
And (4) qualified standard:
age in line with study: not less than 18 years old
Study-compliant gender: female with a view to preventing the formation of wrinkles
Healthy volunteers were received: whether or not
Inclusion criteria were:
postmenopausal female patients
Histologically or cytologically confirmed ER + Breast cancer
Recurrent or progressive after previous AI treatment
Metastatic disease must be measurable
Patients receiving palliative radiation at non-target lesions must have a two-week washout period after treatment is complete, prior to enrollment.
The patient may previously receive a chemotherapy as part of the first line therapy, as long as it was received before the start of the previous AI treatment.
ECOG behaviour: 0 to 1
Laboratory parameters: a) the hemoglobin is more than or equal to 9.0 g/dL; the platelet is more than or equal to 100.0x10<9 >/L; ANC is more than or equal to 1.5x10<9>/L, and hematopoietic growth factors are not used; b) creatinine was below 2.5 times the upper limit of normal for this mechanism; c) for this mechanism, AST and ALT are below 2.5 times the upper limit of normal
Ability to understand and make written informed consent and compliance with research protocols
Exclusion criteria:
relapse less than 12 months after non-steroidal AI treatment of patients in an adjuvant therapy setting
Disease progression in patients with metastatic disease after less than 3 months of recent AI treatment
Rapid progression, life threatening diversion
Any palliative radiotherapy to measurable lesions
Premedication with SNDX-275 or any other HDAC inhibitor (including valproic acid)
Allergy to benzamide or inactive component of research drug
Has a history of allergy to any active or inactive component of exemestane
Any concurrent medical condition that prevents adequate study compliance
Patient is currently enrolled (or ends within 30 days prior to study drug administration) in another trial drug study
Patients are currently receiving treatment with valproic acid, Zolinza (vorinostat) or any other HDAC inhibitor or DNA methyltransferase inhibitor or any systemic anti-cancer therapy (other than leuprolide acetate)
Example 3
A method of treating a patient with non-small cell lung cancer who progressed on erlotinib by administering a combination of erlotinib and entinostat.
And (3) determining main results:
disease control Rate (complete response, partial response or stable disease for at least 3 months)
Secondary outcome determination:
progression free survival at month 2
Progression free survival at month 4
Design of research
Qualification standard
Age in line with study: 18 years old and older
Study-compliant gender: both of them
Healthy volunteers were received: whether or not
And (3) inclusion standard:
● has cytologically or histologically confirmed stage IIIb (pleural effusion) or stage IV NSCLC
● the disease progressed on erlotinib treatment (failed to respond to treatment or subsequently relapsed after objective response) based on at least 2 scans (the last was within 4 weeks of study enrollment and could serve as a baseline scan for patient screening into the study)
● recover from any toxicity associated with most recent cancer treatments (no greater than CTCAE scale grade 1 toxicity or previous baseline condition reached)
● at least 1 measurable lesion ≥ 20mm by conventional CT scan, or at least 1 measurable lesion ≥ 10mm by helical CT scan
● ECOG can score 0, 1 or 2 and have an expected life of at least 3 months
● Paraffin-embedded tumor specimens can be used for related research
● Male or female over 18 years old
● hemoglobin is greater than or equal to 9.0 g/dL; platelet is greater than or equal to 75x109/L;ANC≥1.0x109/L, hematopoietic growth factor not used
● coagulation test is in the normal range
● for this mechanism bilirubin and creatinine were below 2 times the upper limit of normal
● for this mechanism, AST and ALT are below the upper limit of normal by a factor of 3
● for this mechanism, potassium, magnesium and phosphorus are within the normal ranges (supplementation is allowed)
● willing to adopt a recognized and effective contraceptive method (for both men and women) during the study and within 3 months after the last administration of entinostat
● Prior to any study-specific procedures (including special screening tests), patients or legally acceptable representatives had given written informed consent
Exclusion criteria:
● Stem cell transplantation previously performed
● symptomatic CNS involvement
● previous treatment with HDAC inhibitors
● concurrent anticancer therapy, except for radiotherapy for non-targeted study of lesions
● medicine on the list of prohibited medicines is currently being taken
● systemic chemotherapy or treatment with test drugs within 28 days before enrollment
● Valproic acid is currently used
● untreated or unstable brain metastases, or steroids administered to the condition within 4 weeks of study drug administration
● Secondary malignancy currently active, or any malignancy within the last 5 years, with the exception of a cured basal or squamous cell skin carcinoma, carcinoma in situ of the cervix, or superficial bladder carcinoma
● inability to swallow oral drugs or gastrointestinal malabsorption
● uncontrolled infection requiring IV antibiotics, antivirals or antifungals, known HIV infection, or active hepatitis B or C infection
● is defined as abnormal cardiac function (multifocal PVC, ST-T wave changes consistent with myocardial infarction or acute ischemia, QTc greater than 500 milliseconds) found clinically significant on ECG, tachycardia or left ventricular ejection fraction less than 40% on MUGA scan
● Another serious or uncontrolled medical condition such as hypertension, diabetes or suppressed immune system within 3 months of enrollment
● known to be allergic to benzamide
● morbid obesity
● female pregnant or lactating at present
● patients currently participating in (or completing within 28 days of) another study with a test drug
● could not be used for patients in the study (on-study) or on follow-up evaluation
● patients have any kind of medical, mental, or behavioral disorder that increases the risk of, or impairs, patients' ability to make written informed consent and/or impairs their ability to comply with research procedures and requirements when participating in a study
Table 1: summary of pharmacokinetic parameters after administration of 10mg entinostat in fasted and fed conditions
Cmax ratio-Cmax fed/Cmax fasted
AUClast ratio fed AUClast/fasted AUClast
AUCinf ratio fed AUCinf/fasted AUCinf

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