FDA To Review New Sickle Cell Treatment

Sunday, October 29th, 2023 -- 9:45 AM

(AP) -The only cure for painful sickle cell disease today is a bone marrow transplant. But soon there may be a new cure that attacks the disorder at its genetic source.

On Tuesday, advisers to the Food and Drug Administration will review a gene therapy for the inherited blood disorder, which in the U.S. mostly affects Black people. Issues they will consider include whether more research is needed into possible unintended consequences of the treatment. If approved by the FDA, it would be the first gene therapy on the U.S. market based on CRISPR, the gene editing tool that won its inventors the Nobel Prize in 2020. The agency is expected to decide on the treatment in early December, before taking up a different sickle cell gene therapy later that month. The disorder affects hemoglobin, the protein in red blood cells that carries oxygen. A genetic mutation causes the cells to become crescent-shaped, which can block blood flow and cause excruciating pain, organ damage, stroke and other problems.

Millions of people around the world, including about 100,000 in the U.S., have the disease. It occurs more often among people from places where malaria is or was common, like Africa and India, and is also more common in certain ethnic groups, such as people of African, Middle Eastern and Indian descent. Scientists believe being a carrier of the sickle cell trait helps protect against severe malaria. Current treatments include medications and blood transfusions. The only permanent solution is a bone marrow transplant, which must come from a closely matched donor without the disease and brings a risk of rejection.

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