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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

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 treatmen...

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Autores principales: Khan, Aisha, Bellio, Michael A., Schulman, Ivonne H., Levi, Allan D., Longsomboon, Bangon, Brooks, Adriana, Valasaki, Krystalenia, DiFede, Darcy L., Pujol, Marietsy V., Yavagal, Dileep R., Bates, Karen E., Si, Ming-Sing, Kaushal, Sunjay, Green, Barth A., Anderson, Kimberly D., Guest, James D., Burks, Stephen Shelby, Silvera, Risset, Santamaria, Andrea J., Lalwani, Anil, Dietrich, W. Dalton, Hare, Joshua M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8217825/
https://www.ncbi.nlm.nih.gov/pubmed/34169074
http://dx.doi.org/10.3389/fcell.2021.675738
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author Khan, Aisha
Bellio, Michael A.
Schulman, Ivonne H.
Levi, Allan D.
Longsomboon, Bangon
Brooks, Adriana
Valasaki, Krystalenia
DiFede, Darcy L.
Pujol, Marietsy V.
Yavagal, Dileep R.
Bates, Karen E.
Si, Ming-Sing
Kaushal, Sunjay
Green, Barth A.
Anderson, Kimberly D.
Guest, James D.
Burks, Stephen Shelby
Silvera, Risset
Santamaria, Andrea J.
Lalwani, Anil
Dietrich, W. Dalton
Hare, Joshua M.
author_facet Khan, Aisha
Bellio, Michael A.
Schulman, Ivonne H.
Levi, Allan D.
Longsomboon, Bangon
Brooks, Adriana
Valasaki, Krystalenia
DiFede, Darcy L.
Pujol, Marietsy V.
Yavagal, Dileep R.
Bates, Karen E.
Si, Ming-Sing
Kaushal, Sunjay
Green, Barth A.
Anderson, Kimberly D.
Guest, James D.
Burks, Stephen Shelby
Silvera, Risset
Santamaria, Andrea J.
Lalwani, Anil
Dietrich, W. Dalton
Hare, Joshua M.
author_sort Khan, Aisha
collection PubMed
description 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.
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spelling pubmed-82178252021-06-23 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 Khan, Aisha Bellio, Michael A. Schulman, Ivonne H. Levi, Allan D. Longsomboon, Bangon Brooks, Adriana Valasaki, Krystalenia DiFede, Darcy L. Pujol, Marietsy V. Yavagal, Dileep R. Bates, Karen E. Si, Ming-Sing Kaushal, Sunjay Green, Barth A. Anderson, Kimberly D. Guest, James D. Burks, Stephen Shelby Silvera, Risset Santamaria, Andrea J. Lalwani, Anil Dietrich, W. Dalton Hare, Joshua M. Front Cell Dev Biol Cell and Developmental Biology 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. Frontiers Media S.A. 2021-06-08 /pmc/articles/PMC8217825/ /pubmed/34169074 http://dx.doi.org/10.3389/fcell.2021.675738 Text en Copyright © 2021 Khan, Bellio, Schulman, Levi, Longsomboon, Brooks, Valasaki, DiFede, Pujol, Yavagal, Bates, Si, Kaushal, Green, Anderson, Guest, Burks, Silvera, Santamaria, Lalwani, Dietrich and Hare. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Cell and Developmental Biology
Khan, Aisha
Bellio, Michael A.
Schulman, Ivonne H.
Levi, Allan D.
Longsomboon, Bangon
Brooks, Adriana
Valasaki, Krystalenia
DiFede, Darcy L.
Pujol, Marietsy V.
Yavagal, Dileep R.
Bates, Karen E.
Si, Ming-Sing
Kaushal, Sunjay
Green, Barth A.
Anderson, Kimberly D.
Guest, James D.
Burks, Stephen Shelby
Silvera, Risset
Santamaria, Andrea J.
Lalwani, Anil
Dietrich, W. Dalton
Hare, Joshua M.
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
title 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
title_full 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
title_fullStr 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
title_full_unstemmed 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
title_short 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
title_sort 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
topic Cell and Developmental Biology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8217825/
https://www.ncbi.nlm.nih.gov/pubmed/34169074
http://dx.doi.org/10.3389/fcell.2021.675738
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