If approved, new sickle cell disease treatments could offer a step forward for medicine, in both technology and equity.
 | By Alexandra Sifferlin Senior Staff Editor, Opinion |
Before the end of the year, the Food and Drug Administration is expected to decide whether it will approve two gene therapies for sickle cell disease. This could be historic, as the treatments offer a new potential cure for the incredibly painful illness, and one of them, called exa-cel, would be the first CRISPR-based treatment to hit the market. |
It also represents an opportunity for medicine to finally do right by a community of patients. As our contributing writer Daniela Lamas writes in a guest essay today, there are about 100,000 people with sickle cell disease in the United States, the vast majority of whom are Black. There has been "relatively little funding or scientific advances for this medically and socially complex illness," she argues. Many patients have felt neglected by the health system. |
Perhaps that could change. Gene therapy wouldn't be the first functional cure for the disease; there is the option of getting a bone-marrow transplant. But gene therapy uses a patient's own stem cells, alleviating the need to hunt for a donor. There's hope that these new treatments could "transform what it means to have this diagnosis," Lamas writes. |
Yet while the expected arrival of these treatments is exciting, there remains concern about how much the therapy will cost and who will get access to it. "The expected approval of this treatment itself will not erase structural racism or undo decades of medical neglect, of course," writes Lamas. "But it is a start." |
Ultimately, should the therapy make it to market and work as well as it appears to, the medical field will have sickle cell disease patients to thank — specifically, the men and women who agreed to partake in clinical trials to help push the science forward. "There was no going back for me. I had a renewed vigor," one clinical trial participant told Lamas. "If sickle cell was going to take my life, it was going to take it inch by inch. I wasn't going to give it willingly." |
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