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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

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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
    Show allShow less

    Date
    2021-06-08
    Journal
    Frontiers in Cell and Developmental Biology
    Publisher
    Frontiers Media S.A.
    Type
    Article
    
    Metadata
    Show full item record
    See at
    https://doi.org/10.3389/fcell.2021.675738
    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. © 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.
    Rights/Terms
    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.
    Keyword
    clinical investigation
    expanded access IND
    mesenchymal stem cells
    schwann cell
    single patient IND
    Identifier to cite or link to this item
    http://hdl.handle.net/10713/16108
    ae974a485f413a2113503eed53cd6c53
    10.3389/fcell.2021.675738
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