Diabetes Research Institute Foundation Annual Report 2014

annual report 2014 >

MAKING PROGRESS POSSIBLE Thank you to every individual, family, foundation, and business that has given generously over the last year and through the years.

Highlights of the Year

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National Board of Directors

11

Executive Officers' Message

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Regional Boards of Directors

12

Financial Summary

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DRI Foundation Staff

13

To our Donors

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Heritage Society

10

Highlights of the Year

Throughout the year, Diabetes Research Institute scientists continued to build upon critical research programs while launching new scientific initiatives necessary for developing the DRI BioHub, a bioengineered mini organ that mimics the native pancreas to restore natural insulin production. It was an exciting year in which we witnessed progress across the three main research challenges of the Site, Sustainability and Supply, with some projects advancing to or nearing the clinical trial phase of testing. Below is a summary of the year’s research highlights that were made possible by your generous support.

THE SITE The Food and Drug Administration (FDA) approved the DRI’s submission to initiate a Phase I/II clinical trial that will test islets transplanted in a new site in the body called the omentum, an apron-like lining inside the abdomen. The omentum closely replicates the physiological drainage of insulin from the pancreas and has many other beneficial properties.

In this trial, human donor islet cells will be transplanted within a “biodegradable scaffold,” a DRI BioHub platform. The biodegradable scaffold uses a patient’s own plasma, the liquid part of the blood, together with thrombin, a commonlyused, clinical-grade enzyme. Several patients have completed the islet transplantation screening process and have been selected as candidates for the transplant.

Human islet cells embedded in the biodegradable scaffold. The magnification of the mesh shows the fibrin fibers that hold the islets in place.

In its Phase I/II clinical trial, the DRI is testing the omentum as a new transplant site. The omentum is rich with blood vessels, is easily accessed surgically, and has the same insulin drainage characteristics of the pancreas, among other benefits.

SUSTAINABILITY Drs. Alberto Pugliese and Thomas Malek have been collaborating with Paris-based Dr. David Klatzmann, who recently conducted clinical trials using low-dose IL-2 (Interleukin-2) in patients with type 1 diabetes (T1D). The study compared three low doses of IL-2 to determine whether these would be safe and result in increased Regulatory T cells (Tregs), which regulate the immune system and suppress autoimmunity. Results of these collaborative studies were published this year in several peer-reviewed journals, including Diabetes and the Journal of Autoimmunity. New trials are now enrolling patients with recent onset type 1 diabetes (within 3 months from diagnosis) to determine whether low-dose IL-2 can preserve or improve the ability of the pancreas to produce insulin.

Dr. David Klatzmann from the Université Pierre et Marie Curie in Paris, France, (second from left) with the DRI’s Drs. Alberto Pugliese, Jay Skyler, and Thomas Malek. The researchers are collaborating to conduct clinical studies using lowdose IL-2 to boost Treg function, reverse autoimmunity, and restore insulin production.

The DRI team is very encouraged by these results and is now planning to conduct a subsequent clinical trial in Miami that will begin to expand the window to longer time frames post-diagnosis. They believe that patients may benefit from this type of therapy as long as they maintain a certain level of stimulated insulin production. Data has shown that such levels may be present in patients for years after type 1 diabetes develops. The trial being planned will involve patients with residual insulin secretion after one year. They will also be studying whether this therapy can be applicable to patients who receive a pancreas or islet cell transplant as a means of halting the autoimmune attack that caused the onset of T1D. Recent findings in mouse models also suggest that IL-2 may promote some level of beta cell regeneration, in addition to improving immune regulation.

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Dr. Alice Tomei, assistant professor of surgery and cell transplantation, together with collaborators at the École Polytechnique Fédérale de Lausanne (EPFL) in Switzerland, demonstrated that their unique conformal coating process allows efficient encapsulation of islets without compromising viability and function of the cells. The team's novel method encases the islets within complete, uniform, and thin capsules of similar density, and has been designed to specifically address what are considered to be the limitations of traditional cell encapsulation strategies. The results of their study earned the cover position in the prestigious journal Proceedings of the National Academy of Sciences.

Dr. Luca Inverardi and his team have continued their work with Myeloid-Derived Suppressor Cells (MDSCs), a special population of immunomodulatory cells that help tumors escape destruction by the immune system. MDSCs prevent tumor rejection by recruiting Regulatory T cells to surround the cancer cells. The researchers are investigating the potential of MDSCs to protect insulin-producing cells from autoimmune destruction using a similar mechanism.

MDSCs are usually harvested from the bone marrow, but this past year the team was the first to discover and characterize a novel subset of MDSCs that they have named fibrocyteMyeloid-Derived Suppressor Cells (f-MDSC). The cells were isolated from the umbilical cord blood of healthy newborn babies and have a powerful immunosuppressive effect. These particular cells are also easy to grow, expand, and bank. The team’s discovery and results of their studies were published in the European Journal of Immunology and Genomic Data.

Their efforts are now focused on developing ideal conditions for expanding and preserving f-MDSC for their possible clinical use in achieving tolerance to transplanted insulinproducing cells in those with T1D. Dr. Peter Buchwald, director of drug discovery, and his team are targeting a recently identified signaling pathway that leads to autoimmune destruction of insulin-producing cells. They have had promising results in experimental models demonstrating that new-onset diabetes can be reversed by blocking this pathway with a protein known as Smad 7. There is also scientific evidence supporting his theory that the use of Smad 7 not only controls the autoimmune destruction of the islet cells, but can also lead to islet regeneration. Dr. Buchwald and his team are investigating the possible beta cell-enhancing effects of this treatment with the goal of quickly translating this research to clinical therapies.

In this three-dimensional model of human Smad 7, each colored region is believed to interact with a critical receptor (called TGF-ß) in this important pathway under study at the DRI.

SUPPLY The exocrine, or non-insulin-producing, cells of the pancreas have been shown to give rise to insulin-producing endocrine cells. However, previous attempts to achieve this have thus far relied on the use of genetic manipulation, which remains a translational hurdle for diabetes therapies. Drs. Juan Dominguez-Bendala and Ricardo Pastori and their teams have been able to convert adult human exocrine tissue into insulin-producing cells – and have done so using a single molecule that is already in clinical use for other conditions. The DRI team is the very first group in the world to achieve this result using human cells with a compound that is already FDA-approved. The non-genetic conversion of human pancreatic exocrine to endocrine cells is a novel strategy and represents a safer and simpler alternative to genetic reprogramming, while opening the door to the design of new therapies.

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Executive Officers’ Message

By the very nature of our work at the Diabetes Research Institute Foundation, we meet countless individuals and families who are personally affected by diabetes. Some have been living with this disease for decades, while others are relatively new to it. Throughout the course of their involvement with us, they all come to realize the very same thing: the DRI and Foundation are, together, quite a special place.

We are special for many reasons, chief among them being our unparalleled commitment to see this job to the end by discovering a biological cure for diabetes. The drive to fulfill this mission is palpable throughout our entire organization. It underscores everything that we do.

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"There is no better clinical translational group working on type 1 diabetes in the world."

At the DRI Foundation, where the majority of volunteers and professionals have a loved one with diabetes, our resolve could not be stronger. We want a cure, period.

At the DRI, our team of researchers – many of whom are also touched by this disease – are in lockstep with us.

They passionately share the same mission.

Our passion and commitment drive us, but alone they will not defeat such a complex disease. It takes expertise, experience, and vision to achieve something so challenging.

Thankfully, we have these, too, a fact that is evident to our supporters, as well as to the DRI’s esteemed colleagues throughout the scientific community.

On that point, several months ago we attended the two-day meeting of the DRI’s Scientific Advisory Board (SAB), an external council of distinguished investigators who meet every three years to review, and make recommendations on, the Institute’s research program. Their report is then presented to the Dean of the University of Miami Miller School of Medicine, of which the DRI is a part. The SAB’s members offered a glowing review of the DRI’s work, reporting that, “The committee was unanimous in its feeling that major strides have been made in both basic and translational research programs at the DRI…There is no better clinical translational group working on type 1 diabetes in the world.”

In other words, we rely on you and all of our generous donors to help us meet this ongoing need. Highlights of the progress that you helped our researchers achieve are presented in this report.

This past year, many have made a significant investment in our research program. Thousands have led and/or participated in the various events held in our regions and other communities across the country. Others have generously donated whatever they could to help move the science along. We are grateful for each and every gift, regardless of the amount, because we will not get there any other way.

We are all a part of this special place. Our mission is to find a cure, and we need each and every one of you to join us. On behalf of all of us at the DRI and Foundation and the millions counting on us to cross the finish line, thank you for your continued support, trust, and friendship.

Sincerely,

Receiving this impressive validation from such a distinguished panel of experts reinforces our belief that we have invested our time and resources in the right place. Yet for all of the accolades, we know there is still much work to be done, because tomorrow is not soon enough to cure this disease.

That is why we would be remiss if we did not persistently ask ourselves: how do you take something that is clearly special and make it better? That is precisely what we are charged with doing, in the interest of our loved ones, each of you, and the millions of people living with diabetes.

One answer to that question is to continuously employ the highest standards of financial stewardship and accountability.

Over the years, we have gone to great lengths to maintain expenses at acceptable levels, to meet the rigorous guidelines established by various non-profit oversight groups, and to provide the necessary transparency about our operations.

Another answer is to ensure that research progress continues, allowing us to keep moving toward our ultimate goal. Much of that depends on our ability to fund the DRI’s research initiatives.

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Harold G. Doran, Jr.

Joshua W. Rednik

Chairman

President and CEO

Financial Summary

Research Funding is Critical The Diabetes Research Institute has become the world leader it is today as a result of the substantial funding provided by the Diabetes Research Institute Foundation (DRIF). This funding stream is at the heart of the DRI's ability to make significant strides toward a cure. Supported by your donations, the DRIF ensures that our scientists can jump-start new ideas while continuing innovative, cure-focused research projects. Our mission – to provide the DRI with the funding necessary to cure diabetes now – is testament to the belief that tomorrow is not soon enough to cure this disease.

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Diabetes Research Institute Foundation Statement of Activities for the Year ended June 30, 2014.

Through the support of private philanthropy, the Diabetes Research Institute Foundation has funded six chairs totaling almost $14 million.

Support and Revenue

The J. Enloe and Eugenia J. Dodson Chair in Diabetes Research

Contributions Reimbursement Contracts Special Events, Net of Expenses Investment Income Total Support and Revenue

5,300,455 139,017 3,757,157 2,055,447 $11,252,076

Expenses and Fund Balances Program Services Research (Provided to the Diabetes Research Institute) Community Education

6,792,936 930,994

Total Program Services

$7,723,930

Support Services Administration and General Fundraising Total Support Services

Change in Net Assets Net Assets, Beginning of Year Net Assets, End of Year

955,229 1,409,158 $2,364,387

1,163,759 26,307,331 $27,471,090

Fundraising Percentage Fundraising Expense as a Percentage of Support and Revenue

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12.5%

Stacy Joy Goodman Chair in Diabetes Research Mary Lou Held Chair for Diabetes Research Martin Kleiman Endowed Investigatorship Daniel H. Mintz Visiting Professorship Ricordi Family Chair in Transplant Immunobiology.

To our donors

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To Our Generous Donors with Deepest Gratitude...

The Diabetes Research Institute and Foundation wish to gratefully acknowledge all of our donors and volunteers, who are enabling us to make great strides toward a biological cure for diabetes.

Thank you to every individual, family, foundation, and business, many of whom are pictured within this report, that have given generously over the last year and throughout the years.

We would not have been able to come this far without you.

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“We need to continue to bring this cause and our mission to find a cure to the forefront for the millions who suffer with diabetes, including my son.”

– Doug Donaldson (left)

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HERITAGE SOCIETY

The Heritage Society of the Diabetes Research Institute Foundation recognizes individuals who have generously made provisions in their estate plans, through life insurance, charitable remainder trusts and gift annuities, or other deferred giving vehicles to ensure that critical funding for the Diabetes Research Institute continues into the future.

Over the years, planned giving programs have enabled many donors to make substantial gifts to the DRI in ways that have complemented their individual financial objectives. Heritage Society members have chosen to create their own personal legacies and perpetuate their philanthropic goals for all those affected by diabetes.

We are exceptionally grateful to all of our Heritage Society donors, who demonstrate the passion and vision to advance a cure beyond their lifetime.

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NATIONAL BOARD OF DIRECTORS

Chairman Harold G. Doran, Jr.

President and CEO Joshua W. Rednik

Immediate Past Chairman Thomas D. Stern

Directors Diane Beber Marlene Berg Ronald Maurice Darling, Jr. John C. Doscas Piero Gandini Marc S. Goldfarb Esther E. Goodman Marc S. Goodman Arthur Hertz Glenn Kleiman Eleanor Kosow Sandra Levy

Vice Chairmen William J. Rand, M.D. Charles Rizzo Treasurer William J. Fishlinger Secretary Bonnie Inserra

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Sean McGarvey Shelia F. Natbony, D.O. Allan L. Pashcow Ramon Poo Ricardo Salmon David Sherr Bruce A. Siegel Kathy Simkins Jill Viner Bruce Waller

REGIONAL BOARDS OF DIRECTORS Florida Region

Northeast Region

Chairman William J. Rand, M.D.*

Co-chairs Marc S. Goldfarb* Bruce A. Siegel*

Directors Sari Addicott Bernard Beber, M.D. Diane Beber* Crystal Blaylock Sanchez Sabrina R. Ferris Bruce Fishbein Joel S. Friedman Rene W. Guim Javier Holtz Norman Kenyon, M.D.

*Also member of National Board of Directors

Vito La Forgia Sandra Levy* Ramon Poo* Cristina Poo Deborah Rand Michelle Robinson Rosa Schechter James Sensale Jacci Seskin Don Strock Richard P. Tonkinson Stephen Wagman Rita Weinstein

Executive Committee William J. Fishlinger* Marc S. Goodman* Barbara Hatz Bonnie Inserra* Directors Greg Besner John Carrion Diane Cohen Delia DeRiggi-Whitton Peter L. DiCapua Kim Dickstein Douglas R. Donaldson Iris Feldman Joan Fishlinger Lindsey Inserra-Hughes John Luebs Louise Pashcow Hon. C. Raymond Radigan Marie Rizzo Ricardo Salmon* Samantha Shanken Baker

Meryl Lieberman Allan L. Pashcow* Charles Rizzo*

Thomas P. Silver Bruce Waller* Roberta Waller Wendy Waller

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DRI FOUNDATION STAFF Joshua W. Rednik

Mary Revie

President and Chief Executive Officer

Executive Assistant

Deborah L. Chodrow

Laurie Cummings

Northeast Region Anthony E. Childs

Chief Operating Officer

Jeffrey Young Chief Financial Officer

Barbara Tavrow Senior Vice President

Tom Karlya Vice President

Jill Shapiro Miller Vice President of Gift Planning

Lori Weintraub, APR Vice President of Marketing and Communications

Lauren Schreier Director of Marketing and Communications

Communications Assistant

Aurora Nunez Administrative Assistant

Oneida Osuna Accounting Assistant

Mylinda Auguste Data Entry Clerk

Marisol McKay Date Entry Clerk

Eddy Garcia Courier

Florida Region

Regional Director

Amy Epstein Director of Special Events, Manhattan Office

Lily Scarlett Director of Special Events, Jericho Office

Jill Salter Development Manager

Melinda Megale Special Events Coordinator

Tricia Pellizzi Special Events Coordinator

Gloria Keyloun Administrative Assistant

Sheryl Sulkin Director of Special Events

Barbara Singer Director of Special Projects

Nicole Otto Associate Director of Special Events

Karen Paraboo Administration and Database Coordinator

Dena Kawecki

Joelle Parra

Sarah Mehan

Communications and Social Media Coordinator

Special Events Coordinator

Melissa Pe単a Development Coordinator

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Special Events Manager

The organization of choice for those who are serious, passionate, and committed to curing diabetes.

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