Panelists: Dana Ball, Program Director, The Leona M. & Harry B. Helmsley Charitable Trust Alan Lewis, President and Chief Executive Officer, Juvenile Diabetes Research Foundation Douglas A. Melton, Co-Director, Harvard Stem Cell Institute and Investigator, Howard Hughes Medical Institute Marie Schiller, Partner, Health Advances

This panel featured a unique collaboration that was a case in point for the premise of the Partnering for Cures meeting. For many years, The Leona M. & Harry B. Helmsley Charitable Trust (the Trust) has been giving philanthropically, supporting a diverse range of organizations with a major focus on health, medical research, and human services. In April 2009, the Trust began a new chapter in its giving with an emerging focus on Type 1 Diabetes (T1D) as part of its program portfolio.

(L-R): Dana Ball, Program Director, The Leona M. & Harry B. Helmsley Charitable Trust and David Panzirer, Trustee, The Leona M. & Harry B. Helmsley Charitable Trust

David Panzirer, one of five Helmsley trustees, opened the discussion by noting his personal goal to pave the path to a cure for Type 1 Diabetes, as soon as possible. The Trust's program director, Dana Ball shared the lessons learned from a comprehensive due diligence process aimed at identifying the current gaps in diabetes research and finding ways to fill these gaps. The Trust found that the current state of diabetes research had very little applied research, limited human proof-of-concept testing, non-integrated approaches, and insufficient information sharing. A new model was needed - one that featured cross-discipline programs, operational and support systems, and extensive patient outreach that will drive substantial change in a fragmented community.

With this as backdrop, the Trust developed a four-pronged T1D program that includes a 1) research consortium, 2) biorepository and patient registry, 3) technology focus including work on the artificial pancreas, and 4) outreach and support to current patients.

The first pillar of the T1D program is the Helmsley Type 1 Diabetes Research Consortium, an interconnected and interdisciplinary 3 year, 28 grant program engaging 11 institutions and organizations, totaling $21.8 million. The focus of the program is to advance the understanding of T1D with the goal of developing therapeutic solutions until a cure can be found. Doug Melton of Harvard University leads one of the Consortium's projects and said that it was critical to align the project's goals with those of the Trust. For example, Melton noted that they selected researchers who want cures, not publications. When asked later in the panel why this cure-focus was a better approach, Melton responded that "collaboration means early sharing of knowledge, real-time reporting," which would be a difficult thing to do if the goal is to get into a peer-reviewed publication. He added that "some things you have to do are not publishable. Half the results are unreplicable - and the Consortium helps sort this out."

Marie Schiller of Health Advances had assisted the Trust in identifying the need for a biorepository and defining the solution for that need – T1D Exchange – which is the second pillar of the T1D program. She said that there needs to be a comprehensive systems approach that banks patient samples and coordinates clinical trials. She added that "the status quo is no longer an option." There remains major gaps in the fundamental knowledge of T1D. Closed registries are not getting what we need fast enough: more resources for driving better outcomes, increased patient involvement involved, and emerging technologies that allow new approaches. T1D Exchange combines a biorepository and clinical registry that provides samples to researchers and clinical data to researchers but also matches patients with clinical trials.

Alan Lewis of the Juvenile Diabetes Research Foundation (JDRF) spoke of the third pillar of the T1D program – a focus on technology, including work on the artificial pancreas and next generation technologies. JDRF and the Trust have established a partnership that focuses in the research and development of T1D related technologies including Artificial Pancreas Project Acceleration Program and the immunomodulation/immunoisolation project at the Diabetes Research Institute. Lewis stated that a major driver for this is developing strategic collaborations that create value for all involved is key. Hallmarks of the partnership include sharing of ideas and resources including technical and grantmaking expertise.

The panel also noted the role of philanthropic investments in medical research. Ball noted that philanthropic capital allows organizations to show other funders how new models of research can make a difference.

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