A new cure for sickle cell disease may be coming. Health advisers will review it next week
There soon may be a new cure for sickle cell disease that attacks the disorder at its genetic source
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.
Dr. Allison King, who cares for children and young adults with sickle cell disease, said she's enthusiastic about the possibility of new treatments.
“Anything that can help relieve somebody with this condition of the pain and the multiple health complications is amazing," said King, a professor at Washington University School of Medicine in St. Louis. “It’s horribly painful. Some people will say it’s like being stabbed all over.”
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
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