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The Interdisciplinary Stem Cell Institute’s Use of Food and Drug Administration-Expanded Access Guidelines to Provide Experimental Cell Therapy to Patients With Rare Serious Diseases

Authors :
Aisha Khan
Michael A. Bellio
Ivonne H. Schulman
Allan D. Levi
Bangon Longsomboon
Adriana Brooks
Krystalenia Valasaki
Darcy L. DiFede
Marietsy V. Pujol
Dileep R. Yavagal
Karen E. Bates
Ming-Sing Si
Sunjay Kaushal
Barth A. Green
Kimberly D. Anderson
James D. Guest
Stephen Shelby Burks
Risset Silvera
Andrea J. Santamaria
Anil Lalwani
W. Dalton Dietrich
Joshua M. Hare
Source :
Frontiers in Cell and Developmental Biology, Frontiers in Cell and Developmental Biology, Vol 9 (2021)
Publication Year :
2021
Publisher :
Frontiers Media SA, 2021.

Abstract

The U.S. Food and Drug Administration (FDA) provides guidance for expanded access to experimental therapies, which in turn plays an important role in the Twenty-first Century Cures Act mandate to advance cell-based therapy. In cases of incurable diseases where there is a lack of alternative treatment options, many patients seek access to cell-based therapies for the possibility of treatment responses demonstrated in clinical trials. Here, we describe the use of the FDA’s expanded access to investigational new drug (IND) to address rare and emergency conditions that include stiff-person syndrome, spinal cord injury, traumatic brain stem injury, complex congenital heart disease, ischemic stroke, and peripheral nerve injury. We have administered both allogeneic bone marrow-derived mesenchymal stem cell (MSC) and autologous Schwann cell (SC) therapy to patients upon emergency request using Single Patient Expanded Access (SPEA) INDs approved by the FDA. In this report, we present our experience with 10 completed SPEA protocols.

Details

Language :
English
ISSN :
2296634X
Volume :
9
Database :
OpenAIRE
Journal :
Frontiers in Cell and Developmental Biology
Accession number :
edsair.doi.dedup.....8817f245f69a161154f61596683c1ccd
Full Text :
https://doi.org/10.3389/fcell.2021.675738