First-in-human pilot trial improves stem cell collection and delivery for sickle cell gene therapy
Patients with sickle cell disease are often hesitant to undergo transformative gene therapy—the current lengthy process requires multiple hospital visits to collect enough stem cells. Researchers from Boston Children's Hospital have helped smooth the road to gene therapy with a new approach that collects enough of the patient's stem cells in a single hospital admission for most patients,…
Boston Children's Hospital researchers have streamlined the process of collecting stem cells for sickle cell gene therapy, enabling a single hospital visit for most patients. The new method collects enough stem cells in one admission, significantly reducing turnaround time compared to industry standards. The single-session collection improved upon previous methods that required up to five sessions.
The collected stem cells were genetically modified and cryopreserved, then safely infused back into patients, with an average turnaround time of seven weeks. Follow-up results from the first-in-human phase I pilot trial, published in Blood, showed no unexpected safety events or adverse effects related to the modification of stem cells.
The therapy is now approved through the FDA's Expanded Access Program, allowing more patients to be treated before a formal Biologics License Application (BLA) for drug approval. The FDA-approved technology has been licensed to Caring Cross, a nonprofit organization expanding access to cell and gene therapies globally, with trials now opening in Brazil and India.
Written by urgent.news from Medical Xpress's reporting — not their text. Machine-written — may contain errors; check the original before relying on it.
