A pilot gene therapy treatment for sickle cell disease, restoring patients’ ability to make fetal hemoglobin, has produced good results in the first three patients to receive it. Investigators at Boston Children’s Hospital reported the findings of their ongoing clinical trial this week at the American Society of Hematology (ASH) annual meeting.
The three adult patients, all treated at Boston Children’s, have now been followed for 8, 10, and 18 months, respectively. None have shown adverse events from the treatment. And all indicators suggest that the gene therapy has “taken” and that sickling of red blood cells has been reduced. The trial has now begun to enroll adolescents with sickle cell disease.
