Enhancing pediatric access to cell and gene therapies

Authors

Crystal L. Mackall, Center for Cancer Cell Therapy, Stanford Cancer Institute, Stanford University School of Medicine, Stanford, CA, USA. cmackall@stanford.edu.
Catherine M. Bollard, Center for Cancer and Immunology Research and Department of Pediatrics, Children's National Hospital and The George Washington University, Washington, DC, USA.
Nancy Goodman, KidsVCancer, Washington, DC, USA.
Casey Carr, Center for Cancer Cell Therapy, Stanford Cancer Institute, Stanford University School of Medicine, Stanford, CA, USA.
Rebecca Gardner, St. Jude Children's Research Hospital, Memphis, TN, USA.
Rayne Rouce, Center for Cell and Gene Therapy, Baylor College of Medicine, Houston Methodist Hospital and Texas Children's Hospital, Houston, TX, USA.
Elena Sotillo, Center for Cancer Cell Therapy, Stanford Cancer Institute, Stanford University School of Medicine, Stanford, CA, USA.
Rich Stoner, Exthymic, San Diego, CA, USA.
Fyodor D. Urnov, Innovative Genomics Institute, University of California at Berkeley, Berkeley, CA, USA.
Alan S. Wayne, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
Julie Park, St. Jude Children's Research Hospital, Memphis, TN, USA.
Donald B. Kohn, Departments of Microbiology, Immunology & Molecular Genetics; Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA.

Document Type

Journal Article

Publication Date

6-17-2024

Journal

Nature medicine

DOI

10.1038/s41591-024-03035-1

Abstract

Increasing numbers of cell and gene therapies (CGTs) are emerging to treat and cure pediatric diseases. However, small market sizes limit the potential return on investment within the traditional biopharmaceutical drug development model, leading to a market failure. In this Perspective, we discuss major factors contributing to this failure, including high manufacturing costs, regulatory challenges, and licensing practices that do not incorporate pediatric development milestones, as well as potential solutions. We propose the creation of a new entity, the Pediatric Advanced Medicines Biotech, to lead late-stage development and commercialize pediatric CGTs outside the traditional biopharmaceutical model in the United States-where organized efforts to solve this problem have been lacking. The Pediatric Advanced Medicines Biotech would partner with the academic ecosystem, manufacture products in academic good manufacturing practice facilities and work closely with regulatory bodies, to ferry CGTs across the drug development 'valley of death' and, ultimately, increase access to lifesaving treatments for children in need.

APA Citation

Mackall, Crystal L.; Bollard, Catherine M.; Goodman, Nancy; Carr, Casey; Gardner, Rebecca; Rouce, Rayne; Sotillo, Elena; Stoner, Rich; Urnov, Fyodor D.; Wayne, Alan S.; Park, Julie; and Kohn, Donald B., "Enhancing pediatric access to cell and gene therapies" (2024). GW Authored Works. Paper 5090.
https://hsrc.himmelfarb.gwu.edu/gwhpubs/5090

Department

Pediatrics