Sickle cell gene therapy rolls out slowly : Shots

As his stem cells were harvested, DeShawn “DJ” Chou spoke with pediatric hematologist-oncologist Leo Wang at City of Hope Children's Cancer Center on May 22 about the sickle cell gene therapy he will receive.

City of Hope

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City of Hope

Olayide Adekanbi has battled sickle cell disease his whole life.

“It feels like I'm at war with myself,” said Adekanbi, 29, who lives in Boston. “I don't know if I'd call it an evil deep inside, but it feels dark. [but] sometimes, [it].”

This rare, inherited blood disorder is caused by a gene mutation that causes red blood cells to become deformed into a sickle shape. These deformed cells can clog blood vessels, damage vital organs, and cause bouts of unpredictable, debilitating pain.

“Sometimes it gets to a point where I think, 'I can't survive like this,'” she says. “It drives me crazy, because sometimes I can't move. I just lie in one place and try to distract myself from the pain.”

Adekanbi said the Food and Drug Administration will First gene therapy approved for sickle cell disease, disease The disease disproportionately affects Black people like her and has long been ignored by medicine.

“I'm really excited about the possibilities,” she says. “This is probably the best time in history to be a patient with sickle cell disease.”

Risks and unknowns complicate decisions

But Adekanbi is unsure which of two approved gene therapies to take.

One of the main reasons she is hesitant is the chemotherapy that would introduce genetically modified cells into her bone marrow to alleviate the symptoms of the disease, but it would also jeopardize her ability to have children.

“I want to have children in the future,” she said, “so I'm very nervous about what my body will go through to undergo gene therapy and how it will affect my fertility.”

And Adekanbi is not the only one wondering what to do. People with sickle cell disease and a related condition known as Beta-thalassemiaBut of the thousands of patients eligible for treatment, only around 60,000 have started it.

Adekanbi says she would like to freeze some of her eggs if she decides to undergo the procedure, but fertility isn't the only thing she and other potential patients worry about: the treatment is demanding and complicated in other ways too.

“I could end up in hospital for months.” Melissa Cleary“Even if you're not hospitalized, you have to be near a hospital, which may or may not be in the state you live in. And once treatment is over, there's a very complicated follow-up process that can take months, which may also take place in a state you don't live in,” said Dr.

Olaide Adekanbi hasn't yet decided whether to undergo gene therapy for sickle cell disease, but she's excited about the option. “This is probably the best time in history to be a sickle cell patient,” she says.

Olide Adekanbi

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Olide Adekanbi

The treatment is extremely expensive, costing between $2.2 million and $3.1 million per patient.

“Cost remains a significant barrier to reaching those who need it most,” Cleary said.

And some patients worry about the long-term risks. Dr. Louis SuChief Medical Officer American Sickle Cell Association.

“What's going to happen to me in 10 to 20 years? Will the gene therapy product stop working? Will it not stay in my body that long? Or will I get secondary leukemia?” Su says. “Right now, no one has lived that long, so we don't have any definite information.”

on the other hand, Vertex Pharmaceuticals Boston and Bluebird Bio The Somerville, Massachusetts, pharmaceutical companies that make the treatments say so far both treatments are safe.

Given the complexity and expense of the treatment, it will understandably take time for it to be widely accepted, but both companies say interest is growing rapidly.

The companies say dozens of hospitals have signed up to provide the treatment, with more coming online every day.

Additionally, companies are working to help patients afford treatment and related care, with more government and private insurers covering the costs of treatment.

“The level of interest has been overwhelming, almost exactly what we expected, and we're very encouraged by what's happening,” said Andrew Obenshain, CEO of Bluebird Bio. “Hospitals are ready to provide it. Insurance companies are paying for it. And patients are interested.”

The Bluebirds' rivals agree.

“It's certainly a huge decision for patients to embark on this journey, but it's going extremely well,” said Stuart Arbuckle, executive vice president and chief operating officer at Vertex. “The response from payers, physicians and patients has been incredibly encouraging.”

Economic disparities may limit access

But getting people to pay for all of it can be difficult, and it's still not entirely clear how they'll access treatment, as most sufferers of these genetic blood disorders live in economically disadvantaged countries in Africa and Asia where new treatments are not yet available.

“There's an unspoken sentiment in the industry that, 'We made a Cadillac, so whoever wants it will get it.' Compassionate Crossa nonprofit that advocates for greater access to new medical technologies, said, “We've made something that's largely irrelevant for the vast majority of people who suffer from sickle cell disease.”

That's a problem Victoria Grey Gray, from Forest, Mississippi, is the first woman with sickle cell disease to receive gene editing treatment, as reported by NPR when she underwent the procedure in 2019.

“Every day so many people are suffering. Not only are they suffering, but they're dying,” said Gray, who is now 39 and works full time at Walmart. “And now we have something that can stop it. I want people to be free from this kind of fear, this anxiety and this unspeakable pain.”

One patient now on that path is Deshawn Chou, 19, of Irvine, California, who began treatment earlier this year at City of Hope Children's Cancer Center in Los Angeles. His insurance is paying for the treatment, and he's not worried it will affect his ability to have children.

“When I first heard about it, I was a little apprehensive,” Chou said, “but I'm hoping this will change my life.”