Opinion: After living with sickle cell disease for 39 years, I’m both excited and skeptical about the newly approved gene therapies
What a sickle cell disease patient thinks is the biggest challenge facing a newly approved gene therapy.
For 39 years, I have lived with sickle cell disease. As of late, the sickle cell community has heard a lot of excitement about gene therapy’s potential to be the cure we’ve all been waiting for. That potential took a step toward fulfillment Friday when the Food and Drug Administration approved Casgevy from Vertex Pharmaceuticals’ and Lyfgenia from Bluebird Bio, both gene therapy treatments for people with sickle cell.
Yet I find myself teetering between excitement and skepticism. I am thrilled that research for sickle cell disease treatments and possible cures has blossomed. However, I worry that the celebration around this approval is ignoring a significant barrier: a historic legacy of medical mistrust.
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