JDRF Center of Excellence
in New England

To cure type 1 diabetes (T1D), we know that we must restore or replace the insulin-producing beta cells and protect them from autoimmune attack. There are hundreds of people who have been living for years free from needing insulin injections after receiving transplanted donor islets as the source of insulin-producing cells. While these results have restored glycemic control and significantly improved people’s quality of life, the current approach is available to a limited number of people and these individuals need to take immunosuppression drugs for the rest of their lives. Our ambition at JDRF is to build on these advances so that the broader T1D community can benefit and remove the need for chronic immune suppression drugs.

Having developed a way to make an unlimited number of beta cells using stem cells, and establishing the most advanced genome engineering tools and immunology expertise, Douglas A. Melton, Ph.D., at the Harvard Stem Cell Institute, and colleagues will focus on the challenge of protecting these cells from the immune attacks that occur following transplantation. They will undertake four projects the results of which will benefit scientists worldwide aiming to treat and find cures for diabetes.

These world-class researchers will:

  • Develop reliable systems to examine what happens in the autoimmune attack at the cellular and molecular levels, so that we can work to reduce or eliminate these reactions
  • Engineer a cell system  that reproduces and amplifies the beta cell-to-immune cell interactions so that we can better assess the effectiveness of therapies
  • Identify the different subsets of immune cells that target beta cells so that we may apply this knowledge in the development of therapies
  • Explore new technologies, such as gene editing, to create beta cells that can evade immune attacks so we can use them in the development of cell therapies

Research Team Leaders

Aaron J. Kowalski, Ph.D., JDRF

Bio

Douglas A. Melton, Ph.D., Harvard Stem Cell Institute

Sanjoy Dutta, Ph.D., Vice President of Research, JDRF

Esther Latres, Ph.D., Assistant Vice President of Research, JDRF

Project Teams

Project 1: Creating immunologically inert islets from human stem cells

This group is creating and genetically engineering cells to not be recognized as immune targets by the body. These researchers are modifying the stem-cell derived beta cells using CRISPR and other gene editing methods to identify mutations and targets that will protect people against autoreactivity. They’ll then test these engineered stem cell-derived islets and beta cells in humanized models of T1D described in project two, to ensure they produce insulin and function successfully in vivo.

Project 2: Testing the ability of stem cell-derived islets to induce an immune response in humanized models of T1D

This group is testing the cell populations created in project one, in novel humanized models of T1D, to determine if they evade the immune system as intended. By engrafting cells and tissues from consenting human donors with T1D as part of a clinical study, this group will observe the relationships between the modified stem cell-derived islets and peripheral blood cells obtained from people living with T1D, to study how these people’s own blood, cells, and tissues interact in various situations within a human immune setting.

Project 3: Examining immune cell responses in genetically modified human stem cell islets

This group is analyzing the impact of the modified stem cell derived islets on the immune system. They’re investigating the human cells before and after their experience in the humanized models. Immune cells recovered from engrafted islets will be studied to determine how their function and characteristics have changed. New assays are being utilized to examine the cells on both a functional level, but also looking at RNA transcripts and protein markers following interaction within the humanized models. It must be measured and proven to be safe before proceeding towards clinical trials.

News & Blogs

  • JDRF Announces New Center of Excellence in New England to Advance Type 1 Diabetes Research

    February 23, 2021

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Contact us to help us advance this critical work in curing T1D and improving lives.


Betty Gaston
National Director, Principal Gifts
JDRF
bgaston@jdrf.org