ROUNDTABLE Regulating MSC Clinics: Are we Doing Enough to Protect Patients?
Friday, June 2, 2023 08:00 AM - 09:00 AM
Room 251
Regulation & Reimbursement
Roundtable
Moderator
Sowmya Viswanathan, PhD, Scientist at the Osteoarthritis Program/Associate Professor at the Institute of Biomedical Engineering and Department of Medicine, University Health Network, Canada
Christian Jorgensen, MD, PhD, University Hospitale Lapeyronie, France
Panelists
Bambi Grilley, RPh, RAC, CIP, cCRC, CCRP, Director, Clinical Research and Early Product Development, Baylor College of Medicine, USA
Laertis Ikonomou, PhD, Associate Professor, The State University of New York at Buffalo, USA
Miguel Forte, MD, PhD, President-Elect ISCT; CEO Kiji Tx; EiR AdBio - Kiji Tx/AdBio Partners
How do we balance patient access to novel MSC therapies while ensuring they meet sufficient levels of efficacy?
Background Materials
Session Description
This roundtable will discuss how minimally manipulated cellular therapies, and/or more than minimally manipulated, culture-expanded mesenchymal stromal cells (MSCs) are used/marketed in musculoskeletal (MSK) indications in different jurisdictions. Often these are provided to patients for a fee, which is typically paid out-of-pocket. The level of evidence for the minimally manipulated therapies is mixed, and the level of evidence for culture-expanded MSCs remains investigational. Despite this, many jurisdictions can offer these to patients, reflective of jurisdictional variation in regulatory requirements. At this roundtable, panelists will discuss this jurisdictional variation in legislative regulations across regions, for use of i) autologous, minimally manipulated homologous cells in MSK indications, and ii) for more than minimally manipulated and/or non-homologous use of cells for MSK indications.
Learning Objectives
1. Understand research, industry, clinical and ethical consequences of recent US federal court ruling in favor of “stem cell” clinics to use culture-expanded MSCs for musculoskeletal indications
2. Understand differences in jurisdictional regulatory approaches to enabling patient access to culture-expanded MSCs (EAP, HE)
Sowmya Viswanathan, PhD, Scientist at the Osteoarthritis Program/Associate Professor at the Institute of Biomedical Engineering and Department of Medicine, University Health Network, Canada
Christian Jorgensen, MD, PhD, University Hospitale Lapeyronie, France
Panelists
Bambi Grilley, RPh, RAC, CIP, cCRC, CCRP, Director, Clinical Research and Early Product Development, Baylor College of Medicine, USA
Laertis Ikonomou, PhD, Associate Professor, The State University of New York at Buffalo, USA
Miguel Forte, MD, PhD, President-Elect ISCT; CEO Kiji Tx; EiR AdBio - Kiji Tx/AdBio Partners
How do we balance patient access to novel MSC therapies while ensuring they meet sufficient levels of efficacy?
Background Materials
- FDA page on regenerative medicine therapies: https://www.fda.gov/vaccines-blood-biologics/consumers-biologics/important-patient-and-consumer-information-about-regenerative-medicine-therapies
- ISCT press release on federal judge ruling in U.S. v. California Stem Cell Treatment Center case: https://www.isctglobal.org/telegrafthub/blogs/lauren-reville/2022/09/12/isct-issues-response-to-us-federal-judge-ruling-on
- Medicine Maker interview of Dr. Laertis Ikonomou, Chair of ISCT Committee on the Ethics of Cell & Gene Therapy: https://themedicinemaker.com/business-regulation/stem-cell-therapys-unwelcome-surprise
Session Description
This roundtable will discuss how minimally manipulated cellular therapies, and/or more than minimally manipulated, culture-expanded mesenchymal stromal cells (MSCs) are used/marketed in musculoskeletal (MSK) indications in different jurisdictions. Often these are provided to patients for a fee, which is typically paid out-of-pocket. The level of evidence for the minimally manipulated therapies is mixed, and the level of evidence for culture-expanded MSCs remains investigational. Despite this, many jurisdictions can offer these to patients, reflective of jurisdictional variation in regulatory requirements. At this roundtable, panelists will discuss this jurisdictional variation in legislative regulations across regions, for use of i) autologous, minimally manipulated homologous cells in MSK indications, and ii) for more than minimally manipulated and/or non-homologous use of cells for MSK indications.
Learning Objectives
1. Understand research, industry, clinical and ethical consequences of recent US federal court ruling in favor of “stem cell” clinics to use culture-expanded MSCs for musculoskeletal indications
2. Understand differences in jurisdictional regulatory approaches to enabling patient access to culture-expanded MSCs (EAP, HE)
Sowmya Viswanathan PhD
Scientist at the Osteoarthritis Program/Associate Professor at the Institute of Biomedical Engineering and Department of Medicine
University Health Network
Moderator
Scientist at the Osteoarthritis Program/Associate Professor at the Institute of Biomedical Engineering and Department of Medicine
University Health Network
Moderator
Bambi Grilley RPh, RAC, CIP, CCRC, CCRP
Director, Clinical Research and Early Product Development
Baylor College of Medicine
Panelist
Director, Clinical Research and Early Product Development
Baylor College of Medicine
Panelist