ISCT MSC committee statement on the US FDA approval of allogenic bone-marrow mesenchymal stromal cells

Katarina Le Blanc; Francesco Dazzi; Karen English; Dominique Farge; Jacques Galipeau; Edwin M Horwitz; Nadir Kadri; Mauro Krampera; Manoj Mathew Lalu; Jan Nolta; Nikita M Patel; Yufang Shi; Daniel J Weiss; Sowmya Viswanathan

doi:10.1016/j.jcyt.2025.01.005

ISCT MSC committee statement on the US FDA approval of allogenic bone-marrow mesenchymal stromal cells

Cytotherapy. 2025 Jan 17:S1465-3249(25)00030-1. doi: 10.1016/j.jcyt.2025.01.005. Online ahead of print.

Authors

Affiliations

¹ Department of Laboratory  Medicine, Karolinska Institutet, Stockholm, Sweden; Department of Cell Therapies and Allogeneic Stem Cell Transplantation, Karolinska University Hospital, Stockholm, Sweden.
² School of Cardiovascular and Metabolic  Medicine and Sciences, King's College London, London, United Kingdom; AstraZeneca Biopharma R&D, Cambridge, United Kingdom.
³ Kathleen Lonsdale Institute for Human  Health Research, Maynooth University, Maynooth, Ireland; Department of Biology, Maynooth University, Maynooth, Ireland.
⁴ Internal Medicine Unit (UF 04) CRMR  MATHEC, Autoimmune Diseases and Cell Therapy, Reference Center for Rare Systemic Autoimmune Diseases of Ile-de-France MATHEC, AP-HP, St-Louis Hospital, Paris, France; Université Paris Cité, IRSL, Clinical Research in Hematology, Immunology and Transplantation, Paris, France; Department of Medicine, McGill University, Montreal, Quebec, Canada.
⁵ Department of  Medicine, University of Wisconsin Carbone Cancer Center, Madison, Wisconsin, USA.
⁶ Marcus  Center for Advanced Cellular Therapy, Children's Healthcare of Atlanta, Atlanta, Georgia, USA; Aflac Cancer & Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, Georgia, USA; Department of  Pediatrics, Emory University School of Medicine, Atlanta, Georgia, USA; Graduate Division of Biology and Biomedical Sciences, Emory University Laney Graduate School, Atlanta, Georgia, USA.
⁷ Department of  Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden.
⁸ Hematology and  Bone Marrow Transplant Unit, Section of Biomedicine of Innovation, Department of Engineering for Innovative Medicine (DIMI), University of Verona, Verona, Italy.
⁹ Acute Care Research  Program, Blueprint Translational Group, Ottawa Hospital Research Institute, Ottawa, Ontario, Canada; Department of Anesthesiology and Pain Medicine, The Ottawa Hospital, Ottawa, Ontario, Canada; Regenerative  Medicine Program, Ottawa Hospital Research Institute, Ottawa, Ontario, Canada.
¹⁰ Department of Internal Medicine, Stem Cell Program and Institute for Regenerative Cures, University of California Davis, Sacramento, California, USA.
¹¹ INmune Bio International, Royal Free Hospital, London, UK.
¹² The Third Affiliated Hospital of Soochow University Institutes for Translational Medicine, and Suzhou Medical College, Soochow University, Suzhou, Jiangsu, China; Shanghai Institute of Nutrition and Health, Chinese Academy of Sciences, Shanghai, China.
¹³ Department of Medicine, Larner College of Medicine, University of Vermont, Burlington, Vermont, USA.
¹⁴ Osteoarthritis Research Program, Division of Orthopedic Surgery, Schroeder Arthritis Institute, University Health Network, Toronto, Ontario, Canada; Krembil Research Institute, University Health Network, Toronto, Ontario, Canada; Institute of Biomedical Engineering, University of Toronto, Toronto, Ontario, Canada; Department of Medicine, Division of Hematology, University of Toronto, Toronto, Ontario, Canada. Electronic address: sowmya.viswanathan@uhn.ca.

PMID: 39864015
DOI: 10.1016/j.jcyt.2025.01.005

Abstract

The December 2024 US Food and Drug Administration (FDA) approval of Mesoblast's Ryoncil (remestemcel-L-rknd)-allogeneic bone marrow mesenchymal stromal cell (MSC(M)) therapy-in pediatric acute steroid-refractory graft-versus-host-disease finally ended a long-lasting drought on approved MSC clinical products in the United States. While other jurisdictions-including Europe, Japan, India, and South Korea-have marketed autologous or allogeneic MSC products, the United States has lagged in its approval. The sponsor's significant efforts and investments, working closely with the FDA addressing concerns regarding clinical efficacy and consistent MSC potency through an iterative process that spanned several years, was rewarded with this landmark approval. This approval will revive investment and enthusiasm in MSC products, further approvals in major markets, and will continue to foreshadow the long-predicted success of MSCs as a pharmaceutical.

Publication types

Editorial