Studying outliers could lead to new therapy targets for Type 1 diabetes

By Lisa Foster-McNulty, MSN, RN, CDE

The Leona M. and Harry B. Helmsley Charitable Trust has a Type 1 Diabetes Program, and in May they announced $1.4 million in grants to support the study of unusual clinical cases of Type 1 Diabetes (T1D).  The goal is that this research will lead to the identification of new targets for therapy to treat the disease.  The grants come from the Helmsley Breakthrough Initiative’s 2015 funding opportunity for cutting edge pilot projects which seek to clarify unexplained cases of T1D.  The idea is that supporting research on clinical outliers and rare patients might just lead to surprising discoveries having the potential to transform our understanding of T1D.

Some of the clinical outliers include people who display an unexplained delay or acceleration of the disease, people who develop T1D in spite of having a strongly protective genetic background, and families with an extremely high incidence of T1D.

Promising therapies have been built upon studies of these types of cases for other disease processes such as high cholesterol and HIV.  The Helmsley Charitable Trust’s Type 1 Diabetes Program wants to support researchers for T1D who have similar goals.  They have awarded nine grants in amounts from $100,000 to $267,000.

  • University of Massachusetts researchers will examine why rare families have a particularly high incidence of T1D.  They will study two families, one with six of eight children having T1D, and another with five of six children affected.  They are hoping to identify genetic markers that might associate with beta cell dysfunction or immunological effects.
  • Boston Medical Center Corporation will look at why some unique people with T1D seem to have residual beta cell function.
  • Benaroya Research Institute will explore why a few rare individuals develop T1D despite having a strongly protective genetic background.
  • Yale University will investigate why identical twins and triplets who have T1D might have dramatically different onsets of the disease.
  • University of Exeter will examine why unique people develop T1D before age one.
  • University of Colorado, Denver will look at why some individuals remain free of diabetes for more than 12 years despite having autoantibody biomarkers which predict T1D.
  • University of Nebraska will seek to clarify why certain people with T1D display a lack of the common autoantibody biomarkers.
  • Garvan Institute of Medical Research will study whether new genetic mutations might cause or impact the onset of T1D.
  • University of California, San Francisco will investigate why some rare people develop T1D in spite of having a highly protective genetic background, and why certain unique families have such a high incidence of the disease. 

These studies  could potentially have a high impact, leading to new insights about how the human disease works, and resulting in discoveries about the best ways to target new treatments, yet the studies are also high-risk.  The Helmsley Breakthrough Initiative exists to support innovation in the study of T1D through the advancement of early-stage or pilot studies that have little to no preliminary data yet display a strong rationale.  In 2014, the Initiative made its first investment in pilot studies of early-stage drug therapies which seek to enhance beta cell function or prevent the loss of beta cells.  For more information, visit helmsleytrust.org.

It’s very exciting to see that there is support for such out-of-the-box thinking, since this has the potential to lead to huge innovation or result in no real answers.  We are grateful that the Helmsley Charitable Trust has an interest in a cause that is so near and dear to us!