International Society for Cell & Gene Therapy Position Paper: Key considerations to support evidence-based cell and gene therapies and oppose marketing of unproven products

Laertis Ikonomou; Natividad Cuende; Miguel Forte; Bambi J Grilley; Aaron D Levine; Megan Munsie; John E J Rasko; Leigh Turner; Hamid R Bidkhori; Rachele Ciccocioppo; Felix Grignon; Alok Srivastava; Daniel J Weiss; Patricia Zettler; Bruce L Levine; ISCT Committee on the Ethics of Cell and Gene Therapy (ECGT)

doi:10.1016/j.jcyt.2023.03.002

International Society for Cell & Gene Therapy Position Paper: Key considerations to support evidence-based cell and gene therapies and oppose marketing of unproven products

Cytotherapy. 2023 Sep;25(9):920-929. doi: 10.1016/j.jcyt.2023.03.002.

Authors

Laertis Ikonomou¹, Natividad Cuende², Miguel Forte³, Bambi J Grilley⁴, Aaron D Levine⁵, Megan Munsie⁶, John E J Rasko⁷, Leigh Turner⁸, Hamid R Bidkhori⁹, Rachele Ciccocioppo¹⁰, Felix Grignon¹¹, Alok Srivastava¹², Daniel J Weiss¹³, Patricia Zettler¹⁴, Bruce L Levine¹⁵; ISCT Committee on the Ethics of Cell and Gene Therapy (ECGT)

Affiliations

¹ Department of Oral Biology, University at Buffalo, The State University of New York, Buffalo, New York, USA; Cell, Gene and Tissue Engineering Center, University at Buffalo, The State University of New York, Buffalo, New York, USA. Electronic address: laertisi@buffalo.edu.
² Andalusian Transplant Coordination, Servicio Andaluz de Salud; Sevilla, Spain.
³ AdBio Partners, Paris, France.
⁴ Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston Methodist Hospital, Houston, Texas, USA; Department of Pediatrics, Baylor College of Medicine, Houston, Texas, USA.
⁵ School of Public Policy, Georgia Institute of Technology, Atlanta, Georgia, USA.
⁶ Melbourne Medical School, University of Melbourne, Parkville, Victoria, Australia; Murdoch Children's Research Institute, Parkville, Victoria, Australia.
⁷ Gene and Stem Cell Therapy Program Centenary Institute, University of Sydney, Sydney, NSW, Australia; Faculty of Medicine and Health, University of Sydney, Sydney, NSW, Australia; Cell & Molecular Therapies, Royal Prince Alfred Hospital, Camperdown, NSW, Australia.
⁸ Department of Health, Society, and Behavior, Program in Public Health; Stem Cell Research Center; Institute for Clinical and Translational Science; Department of Family Medicine; University of California, Irvine; Irvine, California, USA.
⁹ Stem Cell and Regenerative Medicine Research Department, Academic Center for Education, Culture and Research (ACECR)-Khorasan Razavi, Mashhad, Iran.
¹⁰ Gastroenterology Unit, Department of Medicine, A.O.U.I. Policlinico G.B. Rossi & University of Verona, Verona, Italy.
¹¹ International Society for Cell & Gene Therapy, Vancouver, Canada.
¹² Department of Haematology, Christian Medical College, Ranipet Campus, Ranipet & Centre for Stem Cell Research (a unit of inStem Bengaluru) CMC Campus, Vellore, India.
¹³ University of Vermont College of Medicine, Burlington, Vermont, USA.
¹⁴ Moritz College of Law, Drug Enforcement and Policy Center, Comprehensive Cancer Center, The Ohio State University, Columbus, Ohio, USA.
¹⁵ Center for Cellular Immunotherapies, Department of Pathology and Laboratory Medicine, Perelman School of Medicine, The University of Pennsylvania, Philadelphia, Pennsylvania, USA.

PMID: 37517865
DOI: 10.1016/j.jcyt.2023.03.002

Abstract

The field of regenerative medicine, including cellular immunotherapies, is on a remarkable growth trajectory. Dozens of cell-, tissue- and gene-based products have received marketing authorization worldwide while hundreds-to-thousands are either in preclinical development or under clinical investigation in phased clinical trials. However, the promise of regenerative therapies has also given rise to a global industry of direct-to-consumer offerings of prematurely commercialized cell and cell-based products with unknown safety and efficacy profiles. Since its inception, the International Society for Cell & Gene Therapy Committee on the Ethics of Cell and Gene Therapy has opposed the premature commercialization of unproven cell- and gene-based interventions and supported the development of evidence-based advanced therapy products. In the present Guide, targeted at International Society for Cell & Gene Therapy members, we analyze this industry, focusing in particular on distinctive features of unproven cell and cell-based products and the use of tokens of scientific legitimacy as persuasive marketing devices. We also provide an overview of reporting mechanisms for patients who believe they have been harmed by administration of unapproved and unproven products and suggest practical strategies to address the direct-to-consumer marketing of such products. Development of this Guide epitomizes our continued support for the ethical and rigorous development of cell and cell-based products with patient safety and therapeutic benefit as guiding principles.

Keywords: cell and gene therapies (CGTs); regulatory agencies; reporting mechanisms; tokens of scientific legitimacy; unproven cell and gene interventions.

MeSH terms

Cell- and Tissue-Based Therapy*
Genetic Therapy
Humans
Marketing*
Regenerative Medicine