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

Author:

Lu Katie12ORCID,Brauns Timothy1,Sluder Ann E.1,Poznansky Mark C.1,Dogan Fatma1ORCID

Affiliation:

1. Vaccine and Immunotherapy Center Massachusetts General Hospital Boston Massachusetts USA

2. Department of Biology Stanford University Stanford California USA

Abstract

AbstractWhile 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.

Publisher

Wiley

Subject

Cancer Research,Biochemistry, Genetics and Molecular Biology (miscellaneous),Molecular Medicine,Physiology

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