Leveraging Ex Vivo Gene Therapy Advancements in Hemoglobinopathies and Metabolic Diseases

In a special co-branded episode between Oncology On the Go hosted by CancerNetwork^® and the American Society for Transplantation and Cellular Therapy (ASTCT)’s program ASTCT Talks, Alexis K. Kuhn, PharmD, BCOP, spoke with Katie Bruce, PharmD, BCPPS, and Susie Long, PharmD, about the use of approved cell-based gene therapies for patients with sickle cell disease, beta thalassemia, adrenoleukodystrophy (ALD), and metachromatic leukodystrophy (MLD). These panelists shared the pharmacist’s perspective on ensuring quality care with these ex vivo gene therapies across all treatment phases, including mobilization, conditioning, and infection prophylaxis.

Kuhn is an ambulatory Pediatric Hematology/Oncology/BMT Pharmacist at the Mayo Clinic in Rochester, Minnesota, and an assistant professor of Pharmacy at the Mayo Clinic College of Medicine. Bruce is a pediatric clinical pharmacy specialist at the Sarah Cannon Pediatric Hematology/Oncology & Cellular Therapy program of Tristar Centennial Medical Center in Nashville, Tennessee. Long is a pediatric clinical pharmacist in the Blood and Marrow Team at the University of Minnesota Masonic Children's Hospital.

Specifically, the panelists spoke about the use of agents like elivaldogene autotemcel (Skysona) and atidarsagene autotemcel (Lenmeldy), which are FDA-approved for ALD and MLD, respectively.^1,2 They also discussed the use of exagamglogene autotemcel (Casgevy) and lovotibeglogene autotemcel (Lyfgenia), which the FDA approved for treating patients 12 years and older with sickle cell disease in December 2023.³ The conversation broke down each stage of treatment, detailing optimal strategies for the cell manufacturing and storing processes as well as the management of toxicities like cytopenias. They also reviewed key considerations during the post-infusion period that may help maximize the quality of life for patients after they complete their therapy.

“It has been so amazing to be able to be a part of gene therapy and gene editing,” Bruce stated regarding the potential long-term impacts of these treatments. “We have patients who are able to hold full-time jobs they never were able to have before. We have patients who are climbing mountains and backpacking through Europe, which would have never been an option before because their sickle cell disease would have prevented them from [doing] that…. It’s not an easy process, and it has a lot of steps for the patient to go through, but the reward at the end of it all is worth it.”

References

1. bluebird bio receives FDA accelerated approval for SKYSONA® gene therapy for early, active cerebral adrenoleukodystrophy (CALD). News release. bluebird bio, Inc. September 16, 2022. Accessed October 7, 2024.https://tinyurl.com/mp8crxes
2. FDA approves first gene therapy for children with metachomatic leukodystrophy. New release. FDA. March 18, 2024. Accessed October 7, 2024. https://tinyurl.com/mrh659yk
3. FDA approves first gene therapies to treat patients with sickle cell disease. News release. FDA. December 8, 2023. Accessed October 7, 2024. https://tinyurl.com/3zbdnf4c