New developments in gene therapy offer a promising perspective of a future where curing sickle cell disease (SCD) is possible.
New developments in gene therapy have offered a promising perspective into a future where curing sickle cell disease (SCD) is possible. Each patient who participated in the study had severe sickle cell disease and had experienced at least 4 vaso-occlusive crises (VOCs) in the 2 years prior to enrollment.
Not only has every patient been free of VOCs, the increases in HbF and total Hb was sustained after the exagamglogene autotemcel (exa-cel) infusion intervention. Following a presentation at the American Society of Hematology (ASH) Annual Meeting and Exposition, lead investigator Haydar Frangoul, MD, Medical Director, Sarah Cannon Pediatric Hematology/Oncology & Cellular Therapy at TriStar Centennial, shared how impactful these data are for treating the rare condition.
"As patients become adults with sickle cell disease, they seem to have a lot of complications related to the disease. Allogeneic transplant from a sibling, which is fully matched, is a great option that can cure more than 95% of the patients, but unfortunately, only around 15 to 20% of patients with sickle cell disease have that opportunity to have it fully unaffected," he explained.
Even if a patient has a sibling that's a suitable match, there must be no evidence of the genetic condition in that individual to pursue a transplant.
"That leaves 85% of the patients with no options," Frangoul continued. "We were lucky enough to participate in this clinical trial about three years in 2019. We're using something called CRISPR technology, where we can edit the stem cells of the patients after taking them out and we can edit them in a way to make the stem cells produce high levels of fetal hemoglobin and put them back into the patient."
The results featured at the ASH Annual Meeting are updated data from the 31 patients that Frangoul and his team have been evaluating for up 3 years, and according to him, it's extremely encouraging that the patients are doing amazing.
"Think about it, those 85% of the patients that had no option moving forward, now potentially can have a treatment that will ameliorate their disease or even totally resolve the complications of the disease with their own cells," he said. "So, everybody carries the cure with them."