HOPE For People With AS, SS Genotype As Doctors Successfully Treat Sickle Cell Patient With Novel Gene Therapy
A groundbreaking development in gene therapy has brought new hope to SCD patients. Scientists recently treated Branden Baptiste, a sickle cell patient, using a novel technique called base editing. This method, tested in the BEACON trial, involved collecting Branden’ s blood stem cells and exposing them to a virus engineered to deliver genetic instructions for producing normal hemoglobin. Afterward, the edited cells were reinfused into his body.
Unlike traditional gene therapy, which may involve cutting the genome and risking unintended effects, base editing precisely swaps one DNA base for another, reducing the potential for harm. Branden is the first person in the world to undergo this procedure for SCD and one of the earliest to receive base editing for any condition.
Branden’ s journey with SCD began at age two, and his symptoms worsened as he aged. Before the treatment, he experienced significant pain and was unable to perform everyday activities, including exercise.
The treatment required a multi- step process. First, Branden’ s blood stem cells were harvested and edited outside his body. To make room for the modified cells, his existing blood stem cells were eliminated. Branden was closely monitored in the hospital for several weeks after the procedure.
Remarkably, he made such a significant recovery that he was able to leave the hospital earlier than anyone had expected. Now, more than a year after completing his treatment, Branden has completely weaned off SCD medication and has successfully regained the ability to engage in physical activities. He is now enjoying a life free from pain, allowing him to fully embrace the activities he loves and pursue a healthier lifestyle.
Dr. Matthew Heeney, Branden’ s hematologist at Boston Children’ s Hospital, highlighted the transformative effects of the therapy on Branden’ s life. He stated, ” Not only have we seen his blood counts and disease markers return to normal levels, but he is also able to participate in activities that were once out of his reach. It’ s truly a joy to witness him setting and pursuing goals that felt impossible just a year ago.
