On the heels of the American Society of Clinical Oncology annual meeting in early June, ACGT Scientific Advisory Council member and Research Fellow Crystal Mackall, MD (Stanford University), and Don Kohn, MD (University of California, Los Angeles), brought together leaders in cell and gene therapy development for pediatric cancers and rare diseases for a one-day think tank.
The event was held in June in Washington, D.C., and also allowed for virtual participation.
The event, entitled Ensuring Pediatric Access to Emerging Cell and Gene Therapies, was designed to align the clinical and research communities around the urgency surrounding more access to cancer cell and gene therapies for pediatric diseases and identify next steps needed to begin to solve the problem.
As summarized by Dr. Mackall, the cell and gene therapy field is witnessing unparalleled advances that hold great promise for children with genetic diseases and cancer. Biopharma investment in this arena is growing, but the high cost of developing these therapies combined with small markets in pediatrics diminishes the likelihood that the private sector will advance these therapies at the pace needed to ensure access to all children who can benefit.
The one-day think tank addressed the challenges to commercializing life-saving cell and gene therapies for pediatric disease using current models and what first steps are required to launch new models to solve the problem. The event drew approximately 50 in-person participants and 35 virtual participants spanning academics, advocates, regulators, public funders, and press from the U.S. and Europe.
Government leaders, including Peter Marks, MD, PhD, Director of the Center for Biologics Evaluation and Research, FDA, Renee Wegrzyn, PhD, Director of the Advanced Research Projects Agency for Health, and Maria T. Millan, MD, President and CEO of the California Institute for Regenerative Medicine, presented efforts their agencies are making to support the development of pediatric therapies. Top research scientists outlined the incredible potential of cell and gene therapies and posited solutions to meet the challenges of commercial development and the promotion of equity in patient access.
Additional speakers included ACGT Research Fellow Stephen Gottschalk, MD, of St. Jude Children’s Research Hospital, who is currently funded by ACGT to develop a novel approach to attack pediatric sarcomas with genetically engineered immune cells, called EDB-CAR T cells, which relies on attacking not only the cancer cell but also the supporting blood vessels, which are critical for tumor growth.
Standout presentations included:
- Potential U.S. Nonprofit Biotech Model for Pediatric Cell and Gene Therapy by Dr. Mackall, who presented a new approach to funding and developing pediatric therapies that is not driven by the large market commercialization strategies necessary within public biotech and pharma companies.
- Envisioning the Future of Gene Editing in Pediatrics by Fyodor Urnov, PhD, of Innovative Genomics Institute and University of California, Berkeley, who outlined his vision of curing disease through gene editing and the challenges of making it reality as featured in his recent guest essay in the New York Times.
In our own efforts to support the field, Alliance for Cancer Gene Therapy has funded the following pediatric research programs:
- Dr. Gottschalk’s development of EDB-CAR T cells for pediatric sarcomas. A clinical trial will be planned based on the results of the current grant.
- Hideho Okada, MD, PhD, of the University of California, San Francisco, who is testing an engineered T-cell receptor therapy in a first-in-human phase 1 clinical trial to target a specific protein mutation expressed in pediatric cases of diffuse midline glioma (DMG).
- Dr. Mackall’s development of GD2-CAR T cells for patients with osteosarcoma and neuroblastoma, which has since translated into the development of a GD2-CAR T-cell therapy in clinical trials for children with Diffuse Intrinsic Pontine Glioma, a rare brain tumor with no available treatments.
If you would like to donate or partner to help Alliance for Cancer Gene Therapy support groundbreaking research, please contact Keri Eisenberg, CFRE at keisenberg@acgtfoundation.org! 100% of your donation supports the research and development of cell and gene therapies that are already changing patients’ lives.
