SAN DIEGO — The approval one year ago of two genetic medicines for sickle cell disease, including the first therapy powered by CRISPR gene editing, was a milestone for people living with a debilitating blood disease long neglected by the medical establishment. But access to the near-curative treatments has been slowed by their high cost and the complicated procedures required to administer them.
New research presented here Sunday at the annual meeting of the American Society of Hematology highlighted emerging efforts to remove one of the most concerning obstacles for patients: a “conditioning” course of a highly toxic chemotherapy called busulfan required to eliminate diseased cells from the bone marrow and make room for the genetically modified cells.
Gene editing is 21st century technology, but in blood diseases, it remains shackled to a chemotherapy approved in the 1950s.
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