Early study results hold promise for people with severe sickle cell disease

A rectangular image with a dark crimson background. The image shows a cartoon depiction of the globe ringed by sickled blood cells.  A dark red ribbon is shown in the middle of the globe.
Credit: Jill George, NIH

An NHLBI researcher shared encouraging results from a human clinical trial testing a novel gene replacement therapy in people with severe sickle cell disease. The preliminary findings were presented at the 60th Annual Meeting of the American Society of Hematology (ASH), Dec. 1-4, in San Diego.

According to the scientist, the results suggest that the approach has an acceptable level of safety and might help patients consistently produce normal red blood cells instead of the sickle-shaped ones that mark this painful, life-threatening disease.

The experimental treatment involves removing hematopoietic stem cells from the patients’ bone marrow or blood and adding a therapeutic beta globin gene, which is defective in people with sickle cell disease. The cells are then returned to the patients, leading to the production of anti-sickling hemoglobin (T87Q).

STUDY: Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 Study