Type 1 Diabetes — A Novel Drug Development Opportunity

The Wisconsin Alumni Research Foundation (WARF) is seeking dedicated partners focused on translational research into therapeutics for treatment of type 1 diabetes using novel vitamin D analogs.


According to the Juvenile Diabetes Research Foundation, more than three million people in the United States have type 1 diabetes. Type 1 diabetes is an autoimmune disease that results when the immune system destroys insulin-producing beta cells in the pancreas. It can lead to serious complications, including blindness, cardiovascular disease, kidney damage and lower-limb amputations.

Treatment consists of daily insulin injections. There is no cure, and no clinically useful means of preventing this disease currently exists.

While the cause of type 1 diabetes is still not fully understood, available evidence suggests analogs of the hormonally active form of vitamin D, known as calcitriol or 1,25 dihydroxyvitamin D3, could play an important role in understanding the disease and possible therapeutic solutions. Vitamin D is essential for normal insulin secretion, and vitamin D receptors have been described in pancreatic beta cells.

In addition, current research into therapeutic solutions focuses on immune modulation and protection of beta cells, two characteristics of calcitriol. Therefore, it is believed that these compounds may play an important role as therapeutics that can be used to preserve islet cell function and/or prevent diabetes. In non-obese diabetic (NOD) mouse models, calcitriol has been shown to prevent the development of diabetes.

To modulate the immune system and protect insulin-producing beta islet cells, vitamin D analogs can target antigen presenting dendritic cells (DCs) and inhibit their differentiation and maturation. These analogs cause DCs to become tolerogenic by selectively decreasing the T effector "attack" cells (T helper 1 cells) that can destroy islet cells and/or by stimulating T-regulatory cells (T helper 2 cells), which suppress the generation and function of T-effector cells.

While promising, the concentrations of calcitriol required for effective diabetes prevention can be toxic. The hormone mobilizes calcium from bones and increases intestinal absorption of dietary calcium. Effective therapeutic concentrations can lead to hypercalcemia; a condition characterized by elevated blood calcium levels, alterations in mental status, muscle weakness and calcification of soft tissues and organs such as the heart and kidneys.

A Novel Approach to Prevention and Treatment: Non-calcemic analogs of vitamin D

UW–Madison researchers are designing non-calcemic analogs of calcitriol. These analogs may provide the solution to the dose limitations of calcitriol by offering a wider dose range and lower potential for causing hypercalcemia and its complications. They show significant ability to preserve islet cell function and prevent diabetes at concentrations that do not cause bone calcium mobilization, intestinal calcium transport or increase blood calcium to dangerous levels.

WARF maintains a robust and growing portfolio of low and non-calcemic analogs of calcitriol. Intellectual property rights and special development incentives for commercialization in the type 1 diabetes space are currently available.

Business Opportunity

  • Diabetes affects 246 million people worldwide and nearly 8% of the U.S. population. By 2025 experts predict 380 million people will be afflicted with diabetes.
  • In 2007, the five countries with the largest numbers of people with diabetes were India (40.9 million), China (39.8 million), United States (19.2 million), Russia (9.6 million) and Germany (7.4 million).
  • Global diabetes treatment market was valued at over $21 billion in 2006.
  • The Juvenile Diabetes Research Foundation estimates there are over 3 million people in the U.S. with T1D and nearly 15,000 new cases of T1D diagnosed yearly.
  • Multiple source funding is available for diabetes translational research.


  • Prevention of type 1 diabetes in humans and other animals.

Key Benefits

  • A proven biologically active compound with beneficial effects on beta cell and immune function.
  • Provides a safer, less calcemic compound than the natural hormone calcitriol.
  • Offers a fresh therapeutic approach for gaining access to the diabetes market.
  • Provides a drug development opportunity in a growing market space.
  • Innovative licensing and/or development terms available.

Stage of Development

The analogs offered in this portfolio have been subjected to in vitro/in vivo models for early-stage preclinical evaluation.

Please contact our office for updates as study data sets may be evolving with compounds under development.

Additional Information

For more information about the inventor, see Hector DeLuca.

Contact Information

Please contact our licensing team at licensing@warf.org or 608.960.9850 to explore and discuss innovative development pathways that are available to qualified development interests.