Accelerating the development of genetically engineered cellular therapies: a framework for extrapolating data across related products

Authors

Mark D. Stewart, Friends of Cancer Research, Washington, DC, USA. Electronic address: mstewart@focr.org.
Michael Kalos, Next Pillar Consulting, Philadelphia, PA, USA.
Vicki Coutinho, Geni Consulting Ltd., London, UK.
Marc Better, Pharmefex, Bellevue, WA, USA.
Jonathan Jazayeri, Kite, A Gilead Company, Collegeville, PA, USA.
Jennifer Yohrling, Johnson & Johnson Innovative Medicine, Raritan, NJ, USA.
Julie Jadlowsky, Center for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Miriam Fuchs, Novartis, Basel, CH.
Shalini Gidwani, Allogene Therapeutics, San Francisco, CA, USA.
Carsten Goessl, GSK, Waltham, MA.
Patrick J. Hanley, Center for Cancer and Immunology Research, Children's National Hospital, The George Washington University, Washington, DC, USA.
Jane Healy, Merck & Co., Inc., Rahway, NJ, USA.
Wen Liu, Lyell Immunopharma, South San Francisco, CA, USA.
Brittany A. McKelvey, Friends of Cancer Research, Washington, DC, USA.
Laura Pearce, Canadian Cancer Trials Group, Queens University, Kingston, Ontario, Canada.
Shari Pilon-Thomas, Department of Immunology, Moffitt Cancer Center, Tampa, Florida, USA.
Hillary S. Andrews, Friends of Cancer Research, Washington, DC, USA.
Monica Veldman, Genentech Inc., Washington, DC, USA.
Judy Vong, A2Bio, Los Angeles, CA, USA.
Susan P. Weinbach, Bristol Myers Squibb, San Diego, CA, USA.
Jeff D. Allen, Friends of Cancer Research, Washington, DC, USA.

Document Type

Journal Article

Publication Date

7-1-2024

Journal

Cytotherapy

Volume

26

Issue

7

DOI

10.1016/j.jcyt.2024.03.009

Keywords

CAR-T cell therapy; drug development; regulatory policy

Abstract

BACKGROUND: Significant advancements have been made in the field of cellular therapy as anti-cancer treatments, with the approval of chimeric antigen receptor (CAR)-T cell therapies and the development of other genetically engineered cellular therapies. CAR-T cell therapies have demonstrated remarkable clinical outcomes in various hematological malignancies, establishing their potential to change the current cancer treatment paradigm. Due to the increasing importance of genetically engineered cellular therapies in the oncology treatment landscape, implementing strategies to expedite development and evidence generation for the next generation of cellular therapy products can have a positive impact on patients. METHODS: We outline a risk-based methodology and assessment aid for the data extrapolation approach across related genetically engineered cellular therapy products. This systematic data extrapolation approach has applicability beyond CAR-T cells and can influence clinical development strategies for a variety of immune therapies such as T cell receptor (TCR) or genetically engineered and other cell-based therapies (e.g., tumor infiltrating lymphocytes, natural killer cells and macrophages). RESULTS: By analyzing commonalities in manufacturing processes, clinical trial designs, and regulatory considerations, key learnings were identified. These insights support optimization of the development and regulatory approval of novel cellular therapies. CONCLUSIONS: The field of cellular therapy holds immense promise in safely and effectively treating cancer. The ability to extrapolate data across related products presents opportunities to streamline the development process and accelerate the delivery of novel therapies to patients.

APA Citation

Stewart, Mark D.; Kalos, Michael; Coutinho, Vicki; Better, Marc; Jazayeri, Jonathan; Yohrling, Jennifer; Jadlowsky, Julie; Fuchs, Miriam; Gidwani, Shalini; Goessl, Carsten; Hanley, Patrick J.; Healy, Jane; Liu, Wen; McKelvey, Brittany A.; Pearce, Laura; Pilon-Thomas, Shari; Andrews, Hillary S.; Veldman, Monica; Vong, Judy; Weinbach, Susan P.; and Allen, Jeff D., "Accelerating the development of genetically engineered cellular therapies: a framework for extrapolating data across related products" (2024). GW Authored Works. Paper 5394.
https://hsrc.himmelfarb.gwu.edu/gwhpubs/5394

Department

Pediatrics