Helmsley Charitable Trust Awards $2.1 Million to Penn, Stanford and Seattle Children's Research Institute to Explore Cell-Based Therapies for Type 1 Diabetes

NEW YORK, April 7, 2016 -- The Type 1 Diabetes (T1D) Program of The Leona M. and Harry B. Helmsley Charitable Trust has announced $2.1 million in grants to support research toward the creation of new T1D-specific cell therapies that could help delay or halt the progression of the disease. The new investment will support three complementary projects at the University of Pennsylvania, Stanford University and Seattle Children's Research Institute, which will collaborate with the Benaroya Research Institute.

Approximately two million Americans live with T1D, an autoimmune disease in which the body's insulin-producing beta cells are abnormally attacked by the immune system. Current treatment requires patients to receive insulin through injections or an insulin pump, a process that can be difficult and time-consuming to manage and can lead to acute and chronic complications.

However, an emerging class of therapies that uses immune cells as therapeutic agents has found success in the cancer field, encouraging rapid development of such therapies and suggesting that they may be safe and effective for other diseases as well.

For T1D-specific cellular immunotherapy, researchers hope to engineer cells that suppress T1D autoimmunity by developing a process to modify a patient's own regulatory T cells and re-inject them back into the blood to protect insulin-producing beta cells. These T cells would specifically suppress T1D autoimmunity, potentially making the approach safer and more effective than immunotherapies that broadly suppress the immune system.

"Today, a T1D diagnosis means patients and their families have to monitor insulin levels on a grueling hour-by-hour basis, and there is a significant unmet opportunity to develop therapies that could reduce this burden," said Dr. Gina Agiostratidou, Program Director of the Helmsley Charitable Trust's T1D Program. "We believe that targeted cellular immunotherapies could fill this need and deserve to be developed, de-risked and brought into the T1D field."

The Helmsley grants will support three research projects:

The University of Pennsylvania will engineer and test a panel of protein receptors that could direct immune cells to suppress T1D autoimmunity.

"We have seen from cancer research that engineered T cells can be a powerful and safe approach to provide durable cures of leukemia," said Dr. James Riley, Associate Professor of Microbiology at the Perelman School of Medicine, University of Pennsylvania. "We hope that the lessons learned from re-directing T cells to recognize tumors can be used to reduce or even halt the body's destruction of its own insulin-producing cells, making T cell-engineering a promising therapeutic agent for people with T1D."

Stanford University will test a new approach to target immune cells as well as determine whether they can deliver additional factors that could enhance the survival of beta cells.

"Cellular immunotherapies are an exciting and promising frontier to explore for treating autoimmune diseases," said Dr. Seung Kim, Professor of Developmental Biology at the Stanford University School of Medicine. "We are excited to be part of this effort to leverage the progress in existing research and apply it to type 1 diabetes."

Seattle Children's Research Institute will collaborate with the Benaroya Research Institute to convert immune cells into long-acting cells that specifically suppress T1D autoimmunity.

"Recently we have made major advances in the ability to edit genes in primary human T cells including generation of novel regulatory T cell populations. Use of these cells in immunotherapy applications may provide a way to turn off the destructive T cell responses that lead to pancreatic injury in T1D and, possibly, a way to achieve long-term tolerance" said Dr. David Rawlings, Director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute and Chief of Immunology at Seattle Children's Hospital.

Collectively, the three projects will aim to demonstrate a preclinical proof of principle that engineered immune cells can offer a safe, effective therapy worthy of further preclinical refinement.

ABOUT THE HELMSLEY CHARITABLE TRUST

The Leona M. and Harry B. Helmsley Charitable Trust aspires to improve lives by supporting exceptional nonprofits and other mission-aligned organizations in health, selected place-based initiatives, and education and human services. Since 2008, when the Trust began its active grantmaking, it has committed more than $1.5 billion for a wide range of charitable purposes. The Helmsley Type 1 Diabetes Program is one of the largest private foundation funders of T1D in the United States focused on understanding the disease, developing better treatments and improving care and access.

CONTACT: Laura Fahey, [email protected], 212-953-2814