June 18, 2024- Cleveland Clinic researchers have presented the latest findings from a clinical trial aimed at discovering a cure for sickle cell disease, a painful genetic blood disorder with limited treatment options. The innovative treatment modifies a patient’s own blood-forming stem cells to correct the mutation responsible for sickle cell disease. The 18 patients – two of whom were treated at Cleveland Clinic Children’s – underwent a procedure where their stem cells were first collected for gene editing. They then received chemotherapy to clear remaining bone marrow, making room for the repaired cells which were later infused back into their body.
The treatment was well-tolerated with no serious side effects reported. Following treatment, all patients successfully regained their white blood cells and platelets. Importantly, all patients have remained free of painful events since treatment, and those followed for five months or greater have seen their anemia resolve.