New sickle cell treatment cures disease at lower cost than gene therapies
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A new type of bone marrow transplant can cure sickle cell disease with only half of the donor's cell proteins matching, according to new clinical trial results published in the New England Journal of Medicine.
Why it matters: The procedure would greatly expand the pool of potential donors, in addition to costing less than one-quarter of the price of innovative gene therapies for the condition that have earned Food and Drug Administration approval in recent years.
Context: Sickle cell anemia is considered a rare disease, but it's the most common inherited blood disorder. It affects about 100,000 people in the U.S. and 8 million people across the world.
- The disease changes the shape of red blood cells in a way that can block blood flow to the rest of the body. Sickle cell anemia is associated with extreme pain and other health complications including strokes and organ damage.
What they did: The new transplant method uses bone marrow from "half-matched donors," who have some but not all of the same cell proteins as the patient. Patients received low doses of chemotherapy and total body irradiation before the transplant, followed by other drugs for up to a year to prevent adverse reactions.
- Stem cell bone marrow transplants have been used to effectively treat sickle cell since the 1980s, but eliminating the need for a full-match donor opens up the curative procedure to many more patients.
- Nearly every person on the planet has a half match, said Robert Brodsky, a professor at Johns Hopkins University School of Medicine and a co-author of the study.
- The clinical trial was supported by funding from the National Institutes of Health.
What they found: The trial results show half-match donor transplants are just as effective for sickle cell treatment as full-match transplants.
- 95% of the 42 people with severe sickle cell disease in the trial were alive two years post-transplant. 88% of those participants were considered cured of the disease.
- "This is a huge, huge step forward," Brodsky said.
Zoom out: Researchers calculated the median cost of the transplant to be $467,747 per patient.
- Meanwhile, sickle cell gene therapies Casgevy and Lyfgenia cost $2.2 million and $3.1 million, respectively.
- More than half of people in the U.S. with sickle cell disease are enrolled in Medicaid or the Children's Health Insurance Program, and policymakers have been grappling with how states and the federal government can cover gene therapies without breaking the bank.
- Gene therapies also aren't appropriate for many adults with sickle cell disease who've already sustained organ damage as a result of the condition, as they can't tolerate the high dose of chemotherapy necessary for the treatment, the researchers said.
The bottom line: "[T]he fact that we can now discuss multiple curative therapy options with patients facing the challenges of sickle cell disease marks a major advance in their care," the study says.
