Novel gene therapy ‘switch’ for sickle cell disease shows encouraging results in pilot trial

Manny Johnson has spent 18 years of his life receiving monthly blood transfusions to keep his sickle cell disease at bay. But for the past six months, Johnson, now 21, has been transfusion-free following treatment with a novel gene therapy that induces his body to produce a form of healthy, oxygen-carrying hemoglobin usually made by fetuses and newborns. 

“You can’t tell that he [Johnson] has sickle cell disease anymore,” said Dr. David Williams, president of the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and one of the scientists involved in the development of this new gene therapy.

I spoke with Johnson and his doctors about this novel gene therapy and the early, encouraging clinical trial results that are being reported for the first time at this meeting. You can read the full story here.