Research Priority Areas

Table of Contents

JDRF’s Research Strategy

Overview

Type 1 diabetes (T1D) is an autoimmune disease that ultimately results in the destruction of insulin-producing beta cells and a life-long dependence on carefully titrated exogenous insulin.  T1D progresses through a protracted, silent and asymptomatic phase until diagnosed with frank diabetes requiring insulin therapy.  While the jury is still out regarding the causal genetic predisposition or delayed trigger factors, or whether the stressed and sick beta cells necessitate an immune reactive pathway, the clinical manifestations and evolving epidemiology of T1D globally remain daunting and unresolved.  The challenge of T1D is further compounded by its heterogeneity with multiple causes, various environmental trigger factors, global prevalence in diverse ethnicities, potential for onset at any age, different clinical presentations with mild to moderate to severe dysglycemia independent of disease duration, and unpredictable rates of progression toward complications.  JDRF and others have lead the charge in defining stages of T1D, its heterogeneity at all stages and ages, and its near- and long-term debilitating consequences both to the individual and the ecosystem around, as well as its socio-economic impact.  All of the above are addressed in our Research program.

The JDRF research goals are to find a Cure for T1D and Treat all T1D to live healthy, happy and long lives (to avail a cure).  The main tenets of our Research programs rest on two pillars: Curing Lives and Improving Lives.

Curing Lives will comprise of addressing both underlying T1D pathologies: restoring and protecting the lost beta cells and controlling the immune system.  Beta cell therapies primarily focus on (i) exogenous replacement using human stem cells or non-human islet cells, and (ii) regeneration of endogenous beta cells, while providing means for survival of remaining cells.  Immune therapies primarily focus on (i) targeted antigen specific and general maintenance immune drugs, as standalone or in combination, and (ii) viral, microbial or nutritional approaches to maintain or reset the immune tone.  This encompasses efforts towards the prevention of T1D, potentially through both immune and beta cell approaches.

Improving Lives will comprise of addressing both underlying T1D consequences: reinstating the lost glycemic control and preventing diabetic complications, while reducing the daily burden of disease management.  Glucose control therapies primarily focus on (i) device based automated insulin delivery systems and their components, also known as artificial pancreas systems, and (ii) non-device (primarily drug) based approaches to achieve metabolic homeostasis.  Complications therapies primarily focus on (i) early prognosis and treatments to prevent (or reverse) progression toward end organ failure of the eye and kidney, and (ii) reduce the psycho-social stress of living with T1D by providing the tools necessary for its management.

JDRF is focused on delivering better outcomes for all individuals with or at risk of developing T1D or its complications, at any age or disease stage (Diabetes Care. 2015;38(10):1964–1974. doi:10.2337/dc15-1419; Diabetes Care 2017;40: 1611–1613; 1622–1630; 1631–1640). JDRF aims to support research and catalyze therapeutic developments that will cure, prevent and better treat T1D. Our focus and funding span the research continuum, i.e., discovery, development and delivery of drugs and devices to people with T1D.

JDRF is committed to creating a diversified pipeline of therapeutic candidates to maximize opportunities for success and account for attrition in the R&D process. The earliest stages of the pipeline for several T1D therapies will need to be catalyzed for the foreseeable future supporting the identification and validation of new disease relevant targets and pathways, and increasing understanding of human T1D disease mechanisms, pathophysiology, and heterogeneity.

At the same time, JDRF is focused on progressing more mature opportunities through the pipeline as quickly as possible to deliver both clinically meaningful benefits and progressive advances on the path to a cure for T1D.

JDRF partners with the academic sector, other funders and foundations, industry, regulatory agencies and payers on a worldwide basis and is currently funding research in over 20 different countries and has partnered with over 45 different companies in the last decade.

Success for JDRF is defined ultimately by the availability of new therapies and devices that we help bring to individuals with T1D or those at risk of developing the disease. Thus, JDRF allocates resources to ensure continuous clinical impact and therapeutic advancements for the largest number of individuals with T1D in the shortest period of time by funding programs ranging from those that are relatively close to the clinic to those that are early in discovery phases and represent potential for significant advancement and impact in longer timeframes.

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Beta Cell Replacement

Vision
Deliver life-changing therapies consisting of a safe and renewable beta cell source capable of restoring glucose control and achieving long-term insulin independence without the need for chronic broad immunosuppression – a functional cure.

Full Program Strategy

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Beta Cell Regeneration

Vision
The JDRF Beta Cell Regeneration Program aims to halt the progression of type 1 diabetes of and ultimately achieve a cure for type 1 diabetes (T1D) through the development of disease-modifying therapies (small molecules and/or biologics) that promote the survival, function, and regeneration of endogenous insulin-producing beta cells.

Full Program Strategy

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Immunotherapies

Vision
The JDRF Immunotherapies Program aims to halt the progression of and ultimately achieve a cure for type 1 diabetes (T1D) through the development of disease-modifying therapies that induce desirable and lasting changes to the immune system that result in the protection of pancreatic beta cells from the immune onslaught of T1D.

Full Program Strategy

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Prevention

Vision
The JDRF Prevention Program aims to prevent or delay the onset or progression to insulin-dependent (stage 3) type 1 diabetes (T1D) through the development of therapies that target the triggers, drivers and underlying pathophysiology of T1D. In addition to preventing T1D onset, the Prevention program also aims to prevent the presentation of acute complications at T1D diagnosis, including diabetic ketoacidosis (DKA).

Full Program Strategy

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Artificial Pancreas

Vision
The Artificial Pancreas (AP) program’s vision is a world in which AP devices/systems are commercially available to effectively and conveniently enable optimal glucose control, improve other metabolic outcomes, and enhance quality of life for people with type 1 diabetes (T1D).

Full Program Strategy

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Metabolic Control

Vision
The Metabolic Control (MC) program’s vision is a world in which drugs and drug combinations are commercially available to effectively and conveniently restore overall metabolic homeostasis in people with type 1 diabetes (T1D).

Full Program Strategy

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Complications

Vision
Prevent or delay progressive kidney decline and vision loss until we have a cure for type 1 diabetes (T1D).

Full Program Strategy

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Behavioral Health and Psychology

Vision
Build resilience and foster strengths, reduce the psychosocial challenges, and engender optimism to manage the chronic condition throughout the lifespan with type 1 diabetes (T1D), and ultimately improve overall health outcomes.

Full Program Strategy

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