T1D is an autoimmune disease in which the immune system attacks beta cells that produce insulin.  Insulin is a hormone that enables the body to metabolize glucose. Although the immune system targets only beta cells, the production of other significant hormones like glucagon released by neighboring cells, the alpha cells, is also significantly disrupted. A clinically relevant therapy for people with T1D has to ensure functional beta cells that will not be the subject of an autoimmune response. This can be achieved through three approaches: prevention of beta cell loss, renewable beta cell sources and control of autoimmunity. The T1D program is already investing or plans to invest in the near future in all three areas. The strategy of the research portfolio is to identify and fund innovative ideas and develop a network to facilitate their transition to the clinic. The main driver of the research portfolio is to promote a collaborative effort that enables the exchange of scientific ideas and data.

Understanding the Causes of T1D

Despite making great strides in our understanding of T1D, we still do not know how and why the disease develops. The Trust supports scientists across the U.S. and Europe who are using a variety of approaches to better understand the pathogenesis of T1D, including developing animal models that will allow researchers to recapitulate human T1D in an environment amendable to scientific study and exploring the role that beta cells play in the development of autoimmunity. These efforts will provide invaluable insights and research tools for future research in T1D and, potentially, a range of other diseases.

Developing Renewable Beta Cell Sources

For decades, scientists have worked assiduously to develop renewable beta cells that can provide glucose responsive insulin. The Helmsley T1D Program aims to accelerate this work by supporting promising efforts to either develop beta cells from a range of biological sources or increase the number of endogenous beta cells. Collaboration among multidisciplinary teams and data sharing among leading scientists is essential to advancing the T1D field and bringing such novel therapies to people with T1D. The Trust has created a comprehensive network of leading scientists who bring together expertise from stem cells, beta cells, biomaterials and immunology. 

Controlling the T1D Immune System

Mitigating the autoimmunity caused by T1D will significantly improve the lives of people with T1D. Recognizing the need of therapies that will control the T1D active immune system, the research portfolio supports a phase 1 clinical trial that is treating subjects with established T1D with a combination of two FDA-approved drugs.  In parallel, the T1D program recently released launched the Breakthrough Therapeutic Initiative, a request for grant proposals seeking support for novel ideas that will have the potential to become therapies for people with T1D.