12-22-2025, 03:27 PM
Aligning Development Pathways for Cell-Based Therapies in Small Patient Populations
December 3, 2025
Genetically modified cell-based therapies, such as chimeric antigen receptor T-cell (CAR T) therapies, hold extraordinary promise for patients with rare cancers, but progress can be hampered by regulatory, manufacturing, and financial systems that are not designed to serve very small patient populations. Friends of Cancer Research (Friends) and the Parker Institute for Cancer Immunotherapy (PICI) outlined strategies in a newly published commentary in the Journal for ImmunoTherapy of Cancer (JITC) that address these barriers for therapies under development in small or rare patient populations.
Access the full commentary here
The commentary, “Enabling Access to Genetically Modified Cell Therapies Through Flexible Approaches to Manufacturing and Cost Recovery,” proposes three key strategies to align regulatory and manufacturing expectations with the operational realities of developing therapies for small populations, enabling timely and sustainable patient access.
Tailored manufacturing and quality measures that maintain rigor while enabling safe and reproducible manufacturing for small populations.
Adaptable regulatory frameworks that support diverse manufacturing models, including centralized, decentralized, and point-of-care approaches.
Sustainable financial mechanisms, such as public-private partnerships or protocols supported by the National Institutes of Health, to sustain development and access, and generate evidence needed for approval.
“Genetically modified cell therapies are advancing rapidly, but progress will depend on addressing the regulatory, manufacturing, and financial barriers that limit both their development and ability to reach patients.” – John Connolly, Chief Scientific Officer, Parker Institute for Cancer Immunotherapy
“Sustainable development and access to cell therapies, particularly for rare cancers, will require coordinated approaches across sectors. This commentary reflects a shared effort to ensure that innovation in the lab can be readily translated into new treatments.” – Jeff Allen, President & CEO, Friends of Cancer Research
A special thank you to the working group members who contributed to the proposals in this commentary through their participation in planning discussions and review of the accompanying meeting white paper.
For more information on Friends Cell and Gene Therapies project, please visit: https://friendsofcancerresearch.org/cell...therapies/
Authors
Mark D. Stewart,1 Christopher R. Cabanski,2 Jeff D. Allen,1 John E. Connolly,2 Ben M. Beneski,3 Boro Dropulić,4 Steven A. Feldman,5 Lee A. Fleisher,6 Patrick J. Hanley,8 Kristen Hege,9 Natasha Kekre,10 Holly Fernandez Lynch,7 Crystal L. Mackall,5
Affiliations
1.Friends of Cancer Research 2. Parker Institute for Cancer Immunotherapy 3. Allogene Therapeutics 4. Caring Cross 5. Stanford University School of Medicine 6. Rubrum Advising 7. Perelman School of Medicine at the University of Pennsylvania 8. Children’s National Hospital; The George Washington University 9. Independent Advisor 10. University of Ottawa
https://friendsofcancerresearch.org/news...pulations/
December 3, 2025
Genetically modified cell-based therapies, such as chimeric antigen receptor T-cell (CAR T) therapies, hold extraordinary promise for patients with rare cancers, but progress can be hampered by regulatory, manufacturing, and financial systems that are not designed to serve very small patient populations. Friends of Cancer Research (Friends) and the Parker Institute for Cancer Immunotherapy (PICI) outlined strategies in a newly published commentary in the Journal for ImmunoTherapy of Cancer (JITC) that address these barriers for therapies under development in small or rare patient populations.
Access the full commentary here
The commentary, “Enabling Access to Genetically Modified Cell Therapies Through Flexible Approaches to Manufacturing and Cost Recovery,” proposes three key strategies to align regulatory and manufacturing expectations with the operational realities of developing therapies for small populations, enabling timely and sustainable patient access.
Tailored manufacturing and quality measures that maintain rigor while enabling safe and reproducible manufacturing for small populations.
Adaptable regulatory frameworks that support diverse manufacturing models, including centralized, decentralized, and point-of-care approaches.
Sustainable financial mechanisms, such as public-private partnerships or protocols supported by the National Institutes of Health, to sustain development and access, and generate evidence needed for approval.
“Genetically modified cell therapies are advancing rapidly, but progress will depend on addressing the regulatory, manufacturing, and financial barriers that limit both their development and ability to reach patients.” – John Connolly, Chief Scientific Officer, Parker Institute for Cancer Immunotherapy
“Sustainable development and access to cell therapies, particularly for rare cancers, will require coordinated approaches across sectors. This commentary reflects a shared effort to ensure that innovation in the lab can be readily translated into new treatments.” – Jeff Allen, President & CEO, Friends of Cancer Research
A special thank you to the working group members who contributed to the proposals in this commentary through their participation in planning discussions and review of the accompanying meeting white paper.
For more information on Friends Cell and Gene Therapies project, please visit: https://friendsofcancerresearch.org/cell...therapies/
Authors
Mark D. Stewart,1 Christopher R. Cabanski,2 Jeff D. Allen,1 John E. Connolly,2 Ben M. Beneski,3 Boro Dropulić,4 Steven A. Feldman,5 Lee A. Fleisher,6 Patrick J. Hanley,8 Kristen Hege,9 Natasha Kekre,10 Holly Fernandez Lynch,7 Crystal L. Mackall,5
Affiliations
1.Friends of Cancer Research 2. Parker Institute for Cancer Immunotherapy 3. Allogene Therapeutics 4. Caring Cross 5. Stanford University School of Medicine 6. Rubrum Advising 7. Perelman School of Medicine at the University of Pennsylvania 8. Children’s National Hospital; The George Washington University 9. Independent Advisor 10. University of Ottawa
https://friendsofcancerresearch.org/news...pulations/