Enhancing pediatric access to cell and gene therapies

Crystal L Mackall^{1

2

3}, Catherine M Bollard⁴, Nancy Goodman⁵, Casey Carr⁶, Rebecca Gardner⁷, Rayne Rouce⁸, Elena Sotillo⁶, Rich Stoner⁹, Fyodor D Urnov¹⁰, Alan S Wayne¹¹, Julie Park⁷, Donald B Kohn¹²

Affiliations

¹ Center for Cancer Cell Therapy, Stanford Cancer Institute, Stanford University School of Medicine, Stanford, CA, USA. cmackall@stanford.edu.
² Department of Pediatrics, Division of Pediatric Hematology, Oncology, Stem Cell Transplant and Regenerative Medicine, Stanford University School of Medicine, Stanford, CA, USA. cmackall@stanford.edu.
³ Department of Medicine, Division of Bone Marrow Transplant and Cell Therapy, Stanford University School of Medicine, Stanford, CA, USA. cmackall@stanford.edu.
⁴ Center for Cancer and Immunology Research and Department of Pediatrics, Children's National Hospital and The George Washington University, Washington, DC, USA.
⁵ KidsVCancer, Washington, DC, USA.
⁶ Center for Cancer Cell Therapy, Stanford Cancer Institute, Stanford University School of Medicine, Stanford, CA, USA.
⁷ St. Jude Children's Research Hospital, Memphis, TN, USA.
⁸ Center for Cell and Gene Therapy, Baylor College of Medicine, Houston Methodist Hospital and Texas Children's Hospital, Houston, TX, USA.
⁹ Exthymic, San Diego, CA, USA.
¹⁰ Innovative Genomics Institute, University of California at Berkeley, Berkeley, CA, USA.
¹¹ Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
¹² Departments of Microbiology, Immunology & Molecular Genetics; Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA.

PMID: 38886624
DOI: 10.1038/s41591-024-03035-1

Review

Enhancing pediatric access to cell and gene therapies

Crystal L Mackall et al. Nat Med. 2024 Jul.

. 2024 Jul;30(7):1836-1846.

doi: 10.1038/s41591-024-03035-1. Epub 2024 Jun 17.

Authors

Affiliations

¹ Center for Cancer Cell Therapy, Stanford Cancer Institute, Stanford University School of Medicine, Stanford, CA, USA. cmackall@stanford.edu.
² Department of Pediatrics, Division of Pediatric Hematology, Oncology, Stem Cell Transplant and Regenerative Medicine, Stanford University School of Medicine, Stanford, CA, USA. cmackall@stanford.edu.
³ Department of Medicine, Division of Bone Marrow Transplant and Cell Therapy, Stanford University School of Medicine, Stanford, CA, USA. cmackall@stanford.edu.
⁴ Center for Cancer and Immunology Research and Department of Pediatrics, Children's National Hospital and The George Washington University, Washington, DC, USA.
⁵ KidsVCancer, Washington, DC, USA.
⁶ Center for Cancer Cell Therapy, Stanford Cancer Institute, Stanford University School of Medicine, Stanford, CA, USA.
⁷ St. Jude Children's Research Hospital, Memphis, TN, USA.
⁸ Center for Cell and Gene Therapy, Baylor College of Medicine, Houston Methodist Hospital and Texas Children's Hospital, Houston, TX, USA.
⁹ Exthymic, San Diego, CA, USA.
¹⁰ Innovative Genomics Institute, University of California at Berkeley, Berkeley, CA, USA.
¹¹ Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
¹² Departments of Microbiology, Immunology & Molecular Genetics; Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA.

PMID: 38886624
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.

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References

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Enhancing pediatric access to cell and gene therapies

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Enhancing pediatric access to cell and gene therapies

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References

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Medical