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Combinatorial islet protective therapeutic approaches in β‐cell transplantation: Rationally designed solutions using a target product profile

While progress has been made in the development of islet cell transplantation (ICT) as a viable alternative to the use of exogenous insulin therapy in the treatment of type 1 diabetes, it has not yet achieved its full potential in clinical studies. Ideally, ICT would enable lifelong maintenance of e...

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Autores principales: Lu, Katie, Brauns, Timothy, Sluder, Ann E., Poznansky, Mark C., Dogan, Fatma
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10320848/
https://www.ncbi.nlm.nih.gov/pubmed/37415930
http://dx.doi.org/10.1096/fba.2023-00029
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author Lu, Katie
Brauns, Timothy
Sluder, Ann E.
Poznansky, Mark C.
Dogan, Fatma
author_facet Lu, Katie
Brauns, Timothy
Sluder, Ann E.
Poznansky, Mark C.
Dogan, Fatma
author_sort Lu, Katie
collection PubMed
description While progress has been made in the development of islet cell transplantation (ICT) as a viable alternative to the use of exogenous insulin therapy in the treatment of type 1 diabetes, it has not yet achieved its full potential in clinical studies. Ideally, ICT would enable lifelong maintenance of euglycemia without the need for exogenous insulin, blood glucose monitoring or systemic immune suppression. To achieve such an optimal result, therapeutic approaches should simultaneously promote long‐term islet viability, functionality, and localized immune protection. In practice, however, these factors are typically tackled individually. Furthermore, while the requirements of optimal ICT are implicitly acknowledged across numerous publications, the literature contains few comprehensive articulations of the target product profile (TPP) for an optimal ICT product, including key characteristics of safety and efficacy. This review aims to provide a novel TPP for ICT and presents promising tried and untried combinatorial approaches that could be used to achieve the target product profile. We also highlight regulatory barriers to the development and adoption of ICT, particularly in the United States, where ICT is only approved for use in academic clinical trials and is not reimbursed by insurance carriers. Overall, this review argues that the clear definition of a TPP in addition to the use of combinatorial approaches could help to overcome the clinical barriers to the widespread adoption of ICT for the treatment of type 1 diabetes.
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spelling pubmed-103208482023-07-06 Combinatorial islet protective therapeutic approaches in β‐cell transplantation: Rationally designed solutions using a target product profile Lu, Katie Brauns, Timothy Sluder, Ann E. Poznansky, Mark C. Dogan, Fatma FASEB Bioadv Review Article While progress has been made in the development of islet cell transplantation (ICT) as a viable alternative to the use of exogenous insulin therapy in the treatment of type 1 diabetes, it has not yet achieved its full potential in clinical studies. Ideally, ICT would enable lifelong maintenance of euglycemia without the need for exogenous insulin, blood glucose monitoring or systemic immune suppression. To achieve such an optimal result, therapeutic approaches should simultaneously promote long‐term islet viability, functionality, and localized immune protection. In practice, however, these factors are typically tackled individually. Furthermore, while the requirements of optimal ICT are implicitly acknowledged across numerous publications, the literature contains few comprehensive articulations of the target product profile (TPP) for an optimal ICT product, including key characteristics of safety and efficacy. This review aims to provide a novel TPP for ICT and presents promising tried and untried combinatorial approaches that could be used to achieve the target product profile. We also highlight regulatory barriers to the development and adoption of ICT, particularly in the United States, where ICT is only approved for use in academic clinical trials and is not reimbursed by insurance carriers. Overall, this review argues that the clear definition of a TPP in addition to the use of combinatorial approaches could help to overcome the clinical barriers to the widespread adoption of ICT for the treatment of type 1 diabetes. John Wiley and Sons Inc. 2023-06-02 /pmc/articles/PMC10320848/ /pubmed/37415930 http://dx.doi.org/10.1096/fba.2023-00029 Text en ©2023 The Authors FASEB BioAdvances published by The Federation of American Societies for Experimental Biology. https://creativecommons.org/licenses/by-nc/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc/4.0/ (https://creativecommons.org/licenses/by-nc/4.0/) License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.
spellingShingle Review Article
Lu, Katie
Brauns, Timothy
Sluder, Ann E.
Poznansky, Mark C.
Dogan, Fatma
Combinatorial islet protective therapeutic approaches in β‐cell transplantation: Rationally designed solutions using a target product profile
title Combinatorial islet protective therapeutic approaches in β‐cell transplantation: Rationally designed solutions using a target product profile
title_full Combinatorial islet protective therapeutic approaches in β‐cell transplantation: Rationally designed solutions using a target product profile
title_fullStr Combinatorial islet protective therapeutic approaches in β‐cell transplantation: Rationally designed solutions using a target product profile
title_full_unstemmed Combinatorial islet protective therapeutic approaches in β‐cell transplantation: Rationally designed solutions using a target product profile
title_short Combinatorial islet protective therapeutic approaches in β‐cell transplantation: Rationally designed solutions using a target product profile
title_sort combinatorial islet protective therapeutic approaches in β‐cell transplantation: rationally designed solutions using a target product profile
topic Review Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10320848/
https://www.ncbi.nlm.nih.gov/pubmed/37415930
http://dx.doi.org/10.1096/fba.2023-00029
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