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| Abbreviation | ALS TDI |
|---|---|
| Founded | 1999 |
| Founder |
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| Type | non-profitbiotech |
| 04-3462719 | |
| Location |
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Key people |
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| Affiliations | International Alliance of ALS/MND Associations |
| Website | als.net |
TheALS Therapy Development Institute (ALS TDI) is anon-profitbiotechnology research organization focused on finding treatments foramyotrophic lateral sclerosis (ALS). With a staff including more than 30 scientists, it operates a research and development program centered on ALS.[1][2]
ALS TDI was founded as the ALS Therapy Development Foundation (ALS TDF) in 1999 byJames Heywood, Robert Bonazoli, and Melinda Marsh Heywood after James' brother,Stephen Heywood, was diagnosed with the disease.[3] Dr. Tennore Ramesh joined ALS-TDF when his sister in law was diagnosed with ALS and setup the research facility and served as Chief Scientific Officer from inception until 2003. The organization was initially funded through a donation from Stephen, as well as one fromAlex and Brit d'Arbeloff. The Foundation's first therapy concept was to replaceEAAT2 protein usinggene therapy.[2][4]
In 2004, the Foundation moved to a 16,000-square-foot (1,500 m2) location inCambridge, Massachusetts with an in-house lab. ALS TDF constructed abiosafety level 2 lab in 2005, allowing for the expansion of "gene therapy and cell-based treatment pipelines."[2]
In 2005, the Foundation started the Tri-State Trek, an annual 270-mile bike ride fromBoston, Massachusetts toGreenwich, Connecticut.[2] The Trek has since grown to include over 400 participants and has raised more than $7 million for research.[5][6]
In 2006 the ALS patientAugie Nieto became chairman of the board.[2] The next year, James Heywood resigned as president but joined the board andSean F. Scott, who ALS runs in his family, replaced him.[7] Scott worked withAugie Nieto as well as with theMuscular Dystrophy Association to bring together the two organizations in 2007. The collaboration allowed for MDA to match ALS TDI's annual budget for three years through Nieto's initiative,Augie's Quest, though the partnership continued after that, with MDA cumulatively providing over $36 million.[8][9] The same year, the organization replaced the "Foundation" part of its name with "Institute".[2]
The Institute received a $1.1 million grant from theU.S. Department of Defense in 2008 and an additional $1.6 million grant in 2010.[2][10]
Steven Perrin, previously onlychief scientific officer, was appointed CEO in 2009 following the death of Sean Scott.[11] In 2011, the institute moved to a new 26,000-square-foot (2,400 m2) facility, also in Cambridge, allowing for the hiring of more scientists and a bigger lab.[2]
Two years later, in 2014, Augie's Quest officially transitioned fromMDA to ALS TDI.[12] The same year, ALS TDI received over $3 million through theIce Bucket Challenge.[1] In 2016, the Institute announced the ALS ONE partnership withMassachusetts General Hospital,Harvard Medical School,University of Massachusetts Medical School, and Compassionate Care ALS to find a treatment for ALS within four years.[13]
In 2018, ALS TDI was the original beneficiary of the ALS Pepper Challenge, where participants eat achili pepper.[14] Various public figures, includingKelly Clarkson,Jimmy Kimmel,Shaquille O'Neal,Nancy O'Dell,Wolf Blitzer, theMiami Heat andAndy Cohen have participated in the challenge.[15][16]
In February 2021, the ALS Therapy Development Institute moved to Watertown, MA.[17] The new location included a lab facility allowing preclinical, clinical and transitional research to happen in one place.[17]
In April 2021, Fernando Vieira, M.D., was appointed the chief executive officer of the ALS Therapy Development Institute by the board of directors.[18]
On February 22, 2023, the chairman of the board, Augie Nieto, passed from ALS. In May, 2023, Augie's wife Lynne Nieto was elected to succeed him as board chair.
In February 2024, ALS TDI launched theALS Trial Navigator, in order to help people with ALS find clinical trials they qualify for.
The institute has raised and spent more than $100 million on research into effective treatments for ALS and practicesopen-source science.[19] After the discovery that themultiple sclerosis drugGilenya might also be a treatment for ALS, the Institute enrolled 30 people in a Phase 2A clinical trial the drug in 2013, though it did not progress further.[20][21][22]
ALS TDI launched the Precision Medicine Program in partnership with Denali Therapeutics in 2013 "to identify subgroups of ALS, potential treatments for them using patient data,genomics andiPS cell technology".[23] By 2015, over 300 people had been registered and pre-screened in the program, significantly funded by money raised in theIce Bucket Challenge.[24][25][26]
In 2018, the Institute entered Phase I clinical trials for AT-1501, a potential treatment for ALS andAlzheimer's[27] that blocks the activation of certain immune cells in order to protect nerves from ALS.[28] The development of the drug allows ALS TDI to be viewed as a successfuldrug development organization.[29] ALS TDI received funding from the ALS ONE partnership to develop the drug.[30]
In 2020 the Institute Published "Type I PRMT Inhibition Protects Against C9ORF72 Arginine-Rich Dipeptide Repeat Toxicity" inFrontiers in Pharmacology with a follow-up paper in 2021 called "Hypothesis and Theory: Roles of Arginine Methylation in C9orf72-Mediated ALS and FTD" inFrontiers in Cellular Neuroscience.[31]
In 2023, the Precision Medicine Programevolved into what is now known as the ALS Research Collaborative (ARC). TheARC Study, is an ongoing observational study that invites people with ALS and asymptomatic gene carriers to share their stories by contributing data about their family, work, medical history, and more. TheARC Data Commons, grants researchers access to over eight years of de-identified natural history data contributed by people with ALS – affording an unprecedented level of insight into this complex disease. This database will continue to grow as people with ALS contribute more data to the ongoing ALS Research Collaborative (ARC) natural history study. ALS TDI has collaborated with Google to develop two new digital biomarkers of ALS using voice recordings and accelerometer data from the ARC Study.[32]
