‘Will it really work?’: Young sickle cell patient among the first to start new gene therapy

By Jason Kane, Yamiche Alcindor and Fiona Glisson

Since two groundbreaking treatments were approved in late 2023, only a handful of patients have been able to access the multimillion dollar, medically-intensive therapies.

WASHINGTON — For Wedam Minyila, hospital rooms have always meant blinding pain. “Like someone is jamming a knife in me,” he said. 

But for a brief moment on a recent December morning, Wedam, 19, who has sickle cell disease, allowed himself to believe what his doctors had been telling him for months: This visit could be the first step to a cure. As one of the first patients in the world to undergo commercial treatment for the genetic condition, he could start imagining a future free of excruciating pain. 

“I choose to partially believe it,” he said the day he was admitted to Children’s National Hospital in Washington, D.C., for his stem cell collection — the first major step of the revolutionary sickle cell gene therapy process. “But that also comes with the skepticism of: Will it really work?”

It’s the same question sickle cell patients nationwide have been asking since the Food and Drug Administration approved two gene therapies that could cure the symptoms of the disease just over a year ago.

Since then, only a few dozen patients nationwide have been able to access it. Paperwork snarls, a multimillion-dollar price tag and patient concerns over serious side effects mean only a limited number of hospitals have treated patients so far. 

Keep reading about the state of this illness in the US.

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