A new gene-editing treatment is showing remarkable success in patients with severe sickle cell disease, offering what doctors are calling a potential “functional cure.”
Researchers, including a team at the Cleveland Clinic, say nearly all patients in a recent clinical trial saw dramatic improvements after receiving the one-time therapy.
Out of 28 patients treated, 27 experienced no painful sickle cell crises following the procedure.
The treatment works by editing a patient’s own stem cells using advanced gene-editing technology. Instead of replacing cells through a donor transplant, doctors modify the patient’s cells to increase fetal hemoglobin, a natural form of hemoglobin that prevents red blood cells from becoming misshapen.
That’s key in sickle cell disease, where abnormal, crescent-shaped cells can block blood flow, causing severe pain and long-term organ damage.
Dr. Rabi Hanna of Cleveland Clinic Children’s says one major advantage of this approach is that it avoids rejection, which can be a risk with traditional bone marrow transplants.
Patients in the study also saw significant improvements in their overall hemoglobin levels, reaching near-normal ranges within months of treatment.
The findings come from the RUBY clinical trial and were published in the New England Journal of Medicine.
While more research is still needed, doctors say the results are a major step forward in treating a disease that has long had limited curative options.
