First wave of sickle cell gene therapy patients begin treatment despite cost, complexity barriers

Wedam Minyila knows hospital rooms the way other nineteen-year-olds know their college dorms. For him, they have always meant a particular kind of agony—a sensation he describes as a knife being driven through his body. But on a December morning at Children's National Hospital in Washington, D.C., he was there for something different. He was there to begin a process that his doctors had been telling him for months might actually cure him.

Wedam has sickle cell disease, an inherited condition that warps red blood cells from their normal round shape into crescents that jam up in blood vessels, triggering waves of excruciating pain, strokes, organ damage, and shortened lives. More than 100,000 Americans live with it—nine out of ten of them Black. Until very recently, the only cure was a stem cell transplant from a matched donor, a procedure that worked for Wedam's younger brother Wekem but was impossible for Wedam himself because antibodies in his blood rejected the match. Then, in late 2023, the FDA approved two new gene therapies that could offer what had seemed impossible: a genuine cure, tailored to his own cells.

On the morning of his stem cell collection, Wedam allowed himself a careful kind of hope. "I choose to partially believe it," he said. "But that also comes with the skepticism of: Will it really work?" His mother Sylvia watched from the corner of the room, holding her breath. She had already experienced a miracle with Wekem's successful transplant. Now, months after that procedure, doctors were calling to say Wedam might be eligible for something new. "It felt unreal," she said.

The process began with medication to draw stem cells from Wedam's bone marrow into his bloodstream. Hours later, he was connected to an apheresis machine—a device that pulls blood from the body, spins it at high speed to separate out millions of stem cells, and returns the rest back through his veins. As the first light orange liquid containing his cells began collecting in an IV bag, Dr. Andrew Campbell, director of the hospital's Comprehensive Sickle Cell Disease Program, stepped to his bedside. "You are one of the few in the country and even in the world taking this big step," Campbell told him. Wedam, unfazed, simply said: "I had to. There's not something I could really pass up."

What happens next will take months. Wedam's cells will be shipped to a laboratory in Tennessee, where technicians will use CRISPR—a gene-editing tool—to modify them so they produce red blood cells less likely to sickle and block blood flow. Then he will undergo intensive chemotherapy to clear out his existing stem cells and make room in his bone marrow for the edited ones to take hold. The chemotherapy alone carries serious risks: four days of treatment that makes patients severely ill and vulnerable to infection, often causing ulcerating sores in the mouth and throat that make eating nearly impossible. Longer term, it raises the risk of cancer and infertility.

Yet despite the promise of cure, uptake has been slow. Since the FDA approvals, only a few dozen patients nationwide have started treatment. At Children's National—which was ahead of the curve after participating in clinical trials—only ten patients, including Wedam, have begun or completed the process, out of roughly 1,500 sickle cell patients the hospital treats in the Washington area. The barriers are substantial. Casgevy, made by Vertex Pharmaceuticals, carries a list price of $2.3 million. Bluebird Bio's Lyfgenia is priced at $3.1 million, neither including hospital stays or chemotherapy costs. Insurance companies have implemented extensive pre-authorization procedures. The procedure itself is resource-intensive, requiring multiple days for stem cell collection and, months later, multiweek hospital stays for chemotherapy and cell reintroduction. Dr. David Jacobsohn, chief of blood and marrow transplantation at Children's National, acknowledged the reality: "It's been a learning curve for the hospitals, and it's been a learning curve for the insurance companies." His team is ramping up to start one or two patients per month—a pace he called "exciting" but also cautious, given the high-risk nature of the treatment.

For Wedam, the calculus is personal. The disease has stolen most of his life. He attends college classes online because he cannot reliably leave his bed. His mother describes how he was once a happy boy who loved school and dancing, but pain crises in middle school changed him. He became withdrawn, moaning in pain throughout the house. "It's taken almost everything," Sylvia said. "It's taken our joy." Wedam's ambitions now are modest: to attend classes in person, to make new friends, to be what he calls "a normal person, doing what a normal person would." He dreams of becoming a filmmaker. His younger brother, transformed by his own successful transplant and now riding a bike for the first time, is rooting for him. "I feel like it changed the life that he is going to live," Wekem said.

Wedam remains cautious about his own odds. "Maybe 60 percent," he said when asked if he believed the cure would work. But he has decided that even with the life-threatening complications that may come, any chance at a less painful future is worth the risk. His willingness to be an early adopter matters. At City of Hope Children's Cancer Center in Los Angeles, only one patient has completed the full process so far, but Dr. Leo Wang, a pediatric hematologist-oncologist there, expects rapid increases as early successes accumulate. "We're very hopeful that uptake will increase," Wang said, "and become more acceptable to people who are maybe a little bit more risk-averse." Wedam knows this. "If all of those people could see me and see that it works, they'll have some hope," he said. "Even if it's a tiny bit."