STAT+: Historic approval of sickle cell gene therapies met with cautious optimism by experts at ASH
News of the FDA's historic approval of the first gene therapies for sickle cell disease sparked discussion, debate, and, above all, measured optimism at this year’s ASH meeting.
SAN DIEGO — News of the Food and Drug Administration’s historic approval of the first gene therapies for sickle cell disease sparked discussion, debate, and, above all, measured optimism at this year’s meeting of the American Society of Hematology.
Doctors and researchers noted that the approval of both therapies, one made by Vertex Pharmaceuticals and CRISPR Therapeutics and the other by Bluebird Bio, marked a landmark shift in the treatment of sickle cell, a disease researchers have understood for decades but one that has long been ignored and underfunded. But they also questioned whether these cutting-edge treatments will reach the patients who need them most, citing issues ranging from their cost to the limited number of treatment centers, as well as the challenge of educating both patients and health care providers about the medicines.
“From a clinician, from a scientist’s perspective, I think that it’s just groundbreaking. Medicine has changed forever,” said Akshay Sharma, a physician who treats children with sickle cell at St. Jude Children’s Research Hospital. “Is this really going to transform the care for patients with sickle cell disease in the next five years? I remain skeptical.”
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