Initial tests indicate that a CRISPR-based treatment for sickle cell disease may be working, NPR reports.
In August, Vertex Pharmaceuticals and CRISPR Therapeutics began a trial in which doctors are removing cells from patients' bone marrow, editing them to produce fetal hemoglobin, and re-introducing the now-altered cells back into the patients. The trial, which is to have 45 patients, started with one, Victoria Gray, as NPR reported at the time.
It now reports that Gray's cells are producing fetal hemoglobin, and more than the researchers estimated would be needed to treat her sickle cell disease. Additionally, Gray has not had any painful attacks associated with the condition, has not had to receive any blood transfusions, and has been able to go watch her son play sports, it adds. This, NPR's Rob Stein says, is "a first hint that this [treatment] might be working" and safe.
"It's amazing, you know, to have a chance at a different type of life," Gray tells NPR.
Gray's doctor, TriStar Centennial Medical Center's Haydar Frangoul, notes that she will continue to be monitored, but that this is a "huge achievement."