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New Jersey man pain-free thanks to revolutionary sickle cell treatment at Children's Hospital of Philadelphia

ByStephanie Stahl

/ CBS Philadelphia

A better treatment for sickle cell disease, which strikes mainly people of color, is transforming medicine in Philadelphia, making this Black History Month especially meaningful for one local family.

This is a new gene therapy that works with the patient's own blood, which gets reprogrammed. It's already changing one young man's life. 

Austin Louis, 20, stays busy almost a year after getting a revolutionary treatment forsickle cell disease, a genetic blood disorder that causes excruciating pain.

"It totally worked, I feel amazing," he said.

For the first time in his life, Louis can enjoy being outside in the cold, which triggered pain before the treatment.

"I feel totally different," he said. "It's a brand new body. I feel like a new human."

Louis was the first patient at Children's Hospital of Philadelphia — outside of a clinical trial — to get a new FDA-approved treatment.

It involves collecting his stem cells, which are then manipulated with a gene editing technology calledCRISPR to normalize the blood.

Last March, Louis was back at CHOP for the infusion of his reconstituted blood.

"I totally feel relieved," Louis said. 

His parents, who moved the family from New York to South Jersey to be closer to CHOP, are relieved, too.

Because sickle cell disease affects mainly people of color, this Black History Month is especially poignant for the family. 

"This is history in the making," Jennifer Louis, Austin Louis' mother, said. "I think it's pretty amazing."

It's also a big accomplishment for the team at CHOP, offering a novel new way of treating — and maybe even curing — sickle cell disease.

"You don't get these very often in your career. So for me, wow, this is a big one," said Dr. Alexis Thompson, attending hematologist at CHOP. "To be able to imagine something that is as extraordinary as gene therapy affecting a population that so often feels overlooked and is underserved by our health care system, it's just so rewarding."

A growing number of patients have received the gene therapy, putting a future within reach where sickle cell disease is not such a devastating diagnosis.

Austin Louis is glad to be living without pain for the first time in his life – and helping others along the way. 

"I'm paving the way for others," he said. "So I feel great about that."

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