Journal of Clinical Medicine (Jun 2021)
Arguments against the Requirement of a Biological License Application for Human Pancreatic Islets: The Position Statement of the Islets for US Collaborative Presented during the FDA Advisory Committee Meeting
- Piotr Witkowski,
- Jon Odorico,
- Jordan Pyda,
- Roi Anteby,
- Robert J. Stratta,
- Beth A. Schrope,
- Mark A. Hardy,
- John Buse,
- Joseph R. Leventhal,
- Wanxing Cui,
- Shakir Hussein,
- Silke Niederhaus,
- Jason Gaglia,
- Chirag S. Desai,
- Martin Wijkstrom,
- Fouad Kandeel,
- Piotr J. Bachul,
- Yolanda Tai Becker,
- Ling-Jia Wang,
- R. Paul Robertson,
- Oyedolamu K. Olaitan,
- Tomasz Kozlowski,
- Peter L. Abrams,
- Michelle A. Josephson,
- Kenneth A. Andreoni,
- Robert C. Harland,
- Raja Kandaswamy,
- Andrew M. Posselt,
- Gregory L. Szot,
- Camillo Ricordi,
- on behalf of the Islets for US Collaborative
Affiliations
- Piotr Witkowski
- Transplantation Institute, Department of Surgery, University of Chicago, Chicago, IL 60637, USA
- Jon Odorico
- Division of Transplantation, Department of Surgery, University of Wisconsin, School of Medicine and Public Health, Madison, WI 53792, USA
- Jordan Pyda
- Beth Israel Deaconess Medical Center, Department of Surgery, Harvard Medical School, Boston, MA 02115, USA
- Roi Anteby
- Harvard T.H. Chan School of Public Health, Boston, MA 02115, USA
- Robert J. Stratta
- Section of Transplantation, Department of Surgery, Wake Forest School of Medicine, Winston-Salem, NC 27101, USA
- Beth A. Schrope
- Department of Surgery, Columbia University College of Physicians and Surgeons, New York, NY 10032, USA
- Mark A. Hardy
- Department of Surgery, Columbia University College of Physicians and Surgeons, New York, NY 10032, USA
- John Buse
- Division of Endocrinology, Department of Medicine, University of NC, Chapel Hill, NC 27516, USA
- Joseph R. Leventhal
- Department of Surgery, Northwestern University School of Medicine, Chicago, IL 60611, USA
- Wanxing Cui
- Cell Therapy Manufacturing Facility, Georgetown University Hospital, Washington, DC 20007, USA
- Shakir Hussein
- Detroit Medical Center, Department of Surgery, Wayne State School of Medicine, Detroit, MI 48201, USA
- Silke Niederhaus
- Department of Surgery, University of Maryland School of Medicine, Baltimore, MD 21201, USA
- Jason Gaglia
- Joslin Diabetes Center, Harvard Medical School, Boston, MA 02215, USA
- Chirag S. Desai
- Department of Surgery, Section of Transplantation, University of NC, Chapel Hill, NC 27516, USA
- Martin Wijkstrom
- Department of Surgery, University of Pittsburgh, Pittsburgh, PA 15260, USA
- Fouad Kandeel
- Department of Translational Research and Cellular Therapeutics, Diabetes and Metabolism Research Institute, Beckman Research Institute of City of Hope, Duarte, CA 91010, USA
- Piotr J. Bachul
- Transplantation Institute, Department of Surgery, University of Chicago, Chicago, IL 60637, USA
- Yolanda Tai Becker
- Transplantation Institute, Department of Surgery, University of Chicago, Chicago, IL 60637, USA
- Ling-Jia Wang
- Transplantation Institute, Department of Surgery, University of Chicago, Chicago, IL 60637, USA
- R. Paul Robertson
- Division of Endocrinology and Metabolism, Department of Internal Medicine, University of Washington, Seattle, WA 98133, USA
- Oyedolamu K. Olaitan
- Department of Surgery, Rush University Medical Center, Chicago, IL 60612, USA
- Tomasz Kozlowski
- Division of Transplantation, Department of Surgery, The University of Oklahoma College of Medicine, Oklahoma City, OK 73104, USA
- Peter L. Abrams
- MedStar Georgetown Transplant Institute, Washington, DC 20007, USA
- Michelle A. Josephson
- Department of Medicine, University of Chicago, Chicago, IL 60637, USA
- Kenneth A. Andreoni
- Department of Surgery, University of Florida, College of Medicine, Gainesville, FL 32610-0118, USA
- Robert C. Harland
- Department of Surgery, University of Arizona, Tucson, AZ 85711, USA
- Raja Kandaswamy
- Department of Surgery, University of Minnesota, Minneapolis, MN 55455, USA
- Andrew M. Posselt
- Division of Transplantation, Department of Surgery, University of California San Francisco, San Francisco, CA 94143, USA
- Gregory L. Szot
- Division of Transplantation, Department of Surgery, University of California San Francisco, San Francisco, CA 94143, USA
- Camillo Ricordi
- Diabetes Research Institute and Cell Transplant Center, University of Miami, Miami, FL 33136, USA
- on behalf of the Islets for US Collaborative
- DOI
- https://doi.org/10.3390/jcm10132878
- Journal volume & issue
-
Vol. 10,
no. 13
p. 2878
Abstract
The Food and Drug Administration (FDA) has been regulating human islets for allotransplantation as a biologic drug in the US. Consequently, the requirement of a biological license application (BLA) approval before clinical use of islet transplantation as a standard of care procedure has stalled the development of the field for the last 20 years. Herein, we provide our commentary to the multiple FDA’s position papers and guidance for industry arguing that BLA requirement has been inappropriately applied to allogeneic islets, which was delivered to the FDA Cellular, Tissue and Gene Therapies Advisory Committee on 15 April 2021. We provided evidence that BLA requirement and drug related regulations are inadequate in reassuring islet product quality and potency as well as patient safety and clinical outcomes. As leaders in the field of transplantation and endocrinology under the “Islets for US Collaborative” designation, we examined the current regulatory status of islet transplantation in the US and identified several anticipated negative consequences of the BLA approval. In our commentary we also offer an alternative pathway for islet transplantation under the regulatory framework for organ transplantation, which would address deficiencies of in current system.
Keywords