Show simple item record

dc.contributor.authorKhan, Aisha
dc.contributor.authorBellio, Michael A
dc.contributor.authorSchulman, Ivonne H
dc.contributor.authorLevi, Allan D
dc.contributor.authorLongsomboon, Bangon
dc.contributor.authorBrooks, Adriana
dc.contributor.authorValasaki, Krystalenia
dc.contributor.authorDiFede, Darcy L
dc.contributor.authorPujol, Marietsy V
dc.contributor.authorYavagal, Dileep R
dc.contributor.authorBates, Karen E
dc.contributor.authorSi, Ming-Sing
dc.contributor.authorKaushal, Sunjay
dc.contributor.authorGreen, Barth A
dc.contributor.authorAnderson, Kimberly D
dc.contributor.authorGuest, James D
dc.contributor.authorBurks, Stephen Shelby
dc.contributor.authorSilvera, Risset
dc.contributor.authorSantamaria, Andrea J
dc.contributor.authorLalwani, Anil
dc.contributor.authorDietrich, W Dalton
dc.contributor.authorHare, Joshua M
dc.date.accessioned2021-06-29T12:43:40Z
dc.date.available2021-06-29T12:43:40Z
dc.date.issued2021-06-08
dc.identifier.urihttp://hdl.handle.net/10713/16108
dc.description.abstractThe 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.en_US
dc.description.urihttps://doi.org/10.3389/fcell.2021.675738en_US
dc.language.isoenen_US
dc.publisherFrontiers Media S.A.en_US
dc.relation.ispartofFrontiers in Cell and Developmental Biologyen_US
dc.rightsCopyright © 2021 Khan, Bellio, Schulman, Levi, Longsomboon, Brooks, Valasaki, DiFede, Pujol, Yavagal, Bates, Si, Kaushal, Green, Anderson, Guest, Burks, Silvera, Santamaria, Lalwani, Dietrich and Hare.en_US
dc.subjectclinical investigationen_US
dc.subjectexpanded access INDen_US
dc.subjectmesenchymal stem cellsen_US
dc.subjectschwann cellen_US
dc.subjectsingle patient INDen_US
dc.titleThe Interdisciplinary Stem Cell Institute's Use of Food and Drug Administration-Expanded Access Guidelines to Provide Experimental Cell Therapy to Patients With Rare Serious Diseasesen_US
dc.typeArticleen_US
dc.identifier.doi10.3389/fcell.2021.675738
dc.identifier.pmid34169074
dc.source.volume9
dc.source.beginpage675738
dc.source.endpage
dc.source.countrySwitzerland


This item appears in the following Collection(s)

Show simple item record