Madeleine Powys, MBBS, on Lessons Learned With Libmeldy
The locum consultant of pediatric BMT and leukemia at Royal Manchester Children’s Hospital discussed the center’s experience in delivering the approved gene therapy to children with MLD.
“The way to improve access [to Libmeldy] is to diagnose children early enough. There have been clinical trials showing that newborn screening is feasible in the MLD population with good sensitivity and good specificity. But, clearly there needs to be a discussion with regulators and with the Ministry of Health about how best to include that in routine newborn screening for all children.”
Orchard Therapeutics’ atidarsagene autotemcel (arsa-cel; Libmeldy; OTL-200) was approved in Europe in 2020, making it uniquely positioned to garner real-world experience data on gene therapies, as 1 of the first approved gene therapies for rare diseases. Arsa-cel is delivered to children with metachromatic leukodystrophy (MLD) under a limited distribution model that includes 5 centers of excellence trained as qualified treatment centers (QTC) across the continent. The model was presented in a poster at the 2023 Tandem Meetings |Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, held in Orlando, Florida, February 15-19, 2023.
CGTLive spoke with Madeleine Powys, MBBS, a locum consultant of pediatric BMT and leukemia at Royal Manchester Children’s Hospital, to learn more about unmet needs that remain with the QTC model. She discussed the importance of including MLD in newborn screening, as well as other diseases in which disease-modifying treatments are available or becoming available. She also noted lessons learned with the adminstration of arsa-cel, including the feasibility of stem cell collection in young infants and making provisions for impacts on fertility in treated children. Royal Manchester Children’s is the only QTC for arsa-cel in the United Kingdom.
REFERENCE
Wynn RF, Jones S, Calbi V, et al. Atidarsageneautotemcel, a European post-regulatory approval model for delivery of autologous hematopoietic stem cell gene therapy products via a network of qualified treatment centers (QTCs). Presented at: 2023 Tandem Meetings, February 15-18, Orlando, Florida. Poster #296
Sickle Cell Disease Gene Therapy Exa-Cel's Ability to Prevent VOCs
December 12th 2024Haydar Frangoul, MD, the medical director of pediatric hematology/oncology at Sarah Cannon Research Institute and Pediatric Transplant and Cellular Therapy Program at TriStar Centennial, discussed the latest data update from the CLIMB SCD-121 trial evaluating exa-cel.
