leading the world to a cure - the Shell Key West Challenge

LEADING THE
WORLD TO
A CURE
Mission
To provide the Diabetes Research Institute with the funding
necessary to cure diabetes now.
>
Type 1 diabetes is a devastating disease that inflicts
immeasurable pain and suffering on people of all
ages. It robs children of the freedom and innocence
of childhood. It takes lives prematurely. Patients worry
about the future as they try to manage this relentless
disease every minute of every day. Families face many
ongoing challenges in their attempts to cope with
the consequences of diabetes. Their dream: a cure!
Thankfully, there are many organizations devoted
to the care of diabetes patients and the education
of the public about diabetes. However, there is only
one national organization solely devoted to finding
a cure for diabetes: the Diabetes Research Institute
(DRI). Already, through the tireless commitment
and philanthropic support of the Diabetes Research
Institute Foundation (DRIF), the DRI has made
significant contributions to the field of diabetes
research. It has pioneered new therapies to
restore insulin production and reversed diabetes
in patients involved in ongoing clinical trials.
The DRI’s progress has truly changed the lives of
those affected by diabetes.
CASE
STATEMENT:
LEADING
THE WORLD
TO A CURE
As a result of its numerous achievements, the Diabetes
Research Institute is regarded as the global leader in
cure-focused diabetes research. Much has been
accomplished and the cure is closer than ever before.
But even tomorrow isn’t soon enough for the millions
of children and adults now living with this disease.
To ensure that no time or resources are wasted, the
Foundation has adopted a bold new mission - to cure
diabetes now - by providing the Diabetes Research
Institute with the funding necessary to fulfill this
life-saving goal.
The DRI is Leading the World to a Cure,
and this campaign will provide the
financial resources required to capitalize
on the unprecedented opportunity to
eradicate diabetes.
“We cannot work efficiently if we don’t break
the barriers of traditional academic research
and science…if you don’t make a difference in the
way you perform research, and how you link basic
science to translational, pre-clinical and eventually
clinical trials, making scientists from different
levels communicate with each other, don’t let
anyone sit and study just a mouse model or a
basic mechanistic concept for years without
verifying, as soon as possible, the potential
relevance for translation to the clinical setting.”
Camillo Ricordi, M.D.
Stacy Joy Goodman Professor of Surgery and Medicine;
Distinguished Professor of Medicine; Chief, Division
of Cellular Transplantation, Scientific Director and
Chief Academic Officer, Diabetes Research Institute,
University of Miami Miller School of Medicine
>
LEADING
WITH EXCELLENCE
The DRI is credited with changing the international research paradigm, ensuring
that promising findings in the lab can be translated to patients in the fastest,
safest and most efficient way possible. The most comprehensive diabetes research
center of its kind in the world, the DRI is uniquely structured to house state-of-theart laboratories for the three fundamental phases of research – basic, pre-clinical
and clinical. The Institute’s multidisciplinary teams of scientists working within
these areas share knowledge and build upon each other’s results to quickly move
closer to a cure.
The DRI’s groundbreaking findings are routinely published in leading, peerreviewed medical journals and widely reported in the national and international
media. An innovator in several research areas, its principle focus is the biological
replacement of insulin function to restore blood sugar control. It was one of
the first centers to pursue transplantation of the insulin-producing cells of
the pancreas and its scientific director, Dr. Camillo Ricordi, invented the
system that makes this procedure possible. The DRI’s pioneering work in islet
transplantation has resulted in normalized blood sugar levels in study patients,
with nearly 90 percent of trial participants able to discontinue insulin
injections during the first year.
Despite needing to resume small doses of insulin over time, these patients
maintain some insulin production, allowing them to more easily manage their
blood sugar levels, halt the progression of crippling diabetes-related complications
and experience an improved quality of life. Most importantly, their risk of severe
hypoglycemia is greatly minimized. Though progress has been substantial, islet
transplantation remains an experimental procedure and continues to play a
central and important role as a platform technology.
Today, the Institute is taking science to a higher level by bridging cellular
therapies with emerging technologies. In fact, scientists are developing a new
roadmap in cure-focused diabetes research to overcome the few research
challenges that remain.
The DRI’s commitment to a cure also extends beyond its walls through its long
history of scientific collaboration with investigators worldwide. The Institute is
now the hub of the most focused and far-reaching global research alliance ever
assembled, the Diabetes Research Institute Federation. Comprised of leading
researchers from around the world, the DRI Federation expands the Institute’s
expertise to ensure that the finest minds are working together to cure this
disease as quickly as humanly possible.
“It has completely changed my life.
What a wonderful feeling going to
bed, not worrying if I was not going
to wake up the next day because I
was going to be in a coma, because
my blood sugars went too low. I don’t
have those fears anymore…It was the
best thing that’s ever happened to
me, ever.”
Judy Barker, islet transplant recipient
With its singular focus on curing diabetes, multidisciplinary teams
of scientists, unequalled research expertise, unmatched record of
accomplishment and global outreach, the Diabetes Research
Institute is Leading the World to a Cure.
“I basically looked far and wide at every
organization that was doing work in
type 1 diabetes. The answers kept coming
back that some of the best work was being
done at the DRI…I felt like this is someplace
that will make a difference and where
I can make a difference.”
Thomas D. Stern
Chairman of the Board, DRIF
>
LEADING
WITH PASSIONATE
COMMITMENT
Throughout its 35-year history, the support of the Diabetes Research Institute Foundation
has been the driving force behind the Institute’s progress and continues to serve as its
main funding source. Founded in 1971 by a group of parents of children with diabetes who
were committed to finding a cure for this crippling disease, the Foundation has evolved
into an international coalition of families, patients, business leaders, celebrities, scientists,
clinicians and other concerned individuals who have helped change both the scope and
direction of diabetes research.
Over the last three decades there has certainly been a proliferation of worthwhile diabetes
causes. With almost 24 million children and adults affected by this devastating disease in
the United States alone, these myriad of organizations provide a great service to help lift
the burdens of diabetes.
But the DRIF is unique among diabetes causes by choosing to concentrate solely on the
cure and by dedicating its resources – both financial and human – to this very goal. This
narrow focus has attracted the attention of those who seek a clear mission, accountability
and transparency when supporting a diabetes organization. Many of them have canvassed
the world in search of the most worthy place to devote their commitment and resources.
They’ve come to the DRI to make a difference. And they’ve come to the Foundation because
its funding provides scientists with the unparalleled opportunity to pursue new ideas,
capitalize upon promising research avenues and maintain a strict focus on a cure.
The Foundation’s donors know that their contributions are being stewarded in the most
responsible and efficient manner possible. The DRI Foundation is among the top 15 percent
of all charities, as recognized by Charity Navigator for its sound fiscal management. Having
earned back-to-back, four-star ratings, the highest possible, the Foundation outperforms
the vast number of charities in America.
It is for all of these reasons and more that the Diabetes Research Institute Foundation has
been and continues to be the organization of choice for those who are serious, passionate
and committed to finding a cure for their loved ones and millions of others with diabetes.
Join the Diabetes Research Institute Foundation and join with others
whose personal stake motivates them to work harder, whose passion
drives them to delve deeper and whose financial investment is Leading
the World to a Cure.
“The DRI embraced us
and they gave us hope.
There is no other place in
the world like it. They have
so many advances and
they’re so close…My hope
is that one day there will
be a cure for Matthew
and so many kids like
him, and the DRI gives
me hope for that.”
Crystal Blaylock Sanchez,
mother of a child with
type 1 diabetes
“I think that all the people associated with
the DRI are very open-minded, and one of
the most open-minded persons I’ve ever
met is Dr. Camillo Ricordi. He is so open to
new ideas and analyzing every emerging
technology that could promote diabetes
research and the cure.”
Sarah Ferber, Ph.D.
Director, Molecular Endocrinology, Chaim
Sheba Medical Center, Tel-Hashomer, Israel
“I don’t think any other diabetes research
facilities have embraced technologies quite
as much as at the DRI. That, I think, is
relatively unique.”
Jeffrey Hubbell, Ph.D.
Director, Integrative Biosciences Institute;
Professor, Institute for Chemical Sciences
and Engineering, Ecole Polytechnique
Fédérale de Lausanne, Switzerland
>
LEADING
WITH VISION
DEFINING RESEARCH FOR A CURE
IN THE 21ST CENTURY
The DRI has established a successful transformational
research model, one that puts the patient front and center
of the research strategy and process, and translates new
findings from the lab to clinical application in the fastest,
safest and most efficient way possible.
The model not only shifts the traditional paradigm from
the lab to the patient and thus speeds overall research, but
it also unifies the research process in what can best be called
a “sequential, integrated” approach to biomedical research.
This new approach ensures that each and every improvement
made in a basic lab experiment, for example, impacts the next
level of research as the study moves forward into pre-clinical
studies, and finally to clinical trials. This approach has been
optimized at the DRI, where unlike other diabetes research
centers, all levels of translational research are found under
one roof so as to maximize collaborations and crossfertilization of ideas – from basic to clinical as well as
across disciplines.
Since its inception, the Diabetes Research Institute has been
at the forefront of cure-focused research, pioneering many of
the approaches to restore natural insulin production in those
living with diabetes. Its outstanding contributions to the field
have been recognized throughout the international scientific
community, adopted in research centers worldwide and
embraced as gold-standard approaches to resolve the
challenges of diabetes.
Building upon more than three decades of expertise,
dedication and progress in cure-focused research, the DRI
has laid out a scientific roadmap to cure type 1 diabetes
that is at the forefront of medicine today.
With its visionary research strategy, diverse
medical and technical expertise and unique
spirit of collaboration, the Diabetes Research
Institute is Leading the World to a Cure.
> UNDERSTANDING CELLS:
OUR BODY’S BUILDING BLOCKS
Understanding cellular function, and dysfunction, is key to finding a cure for
diabetes. Therefore, the focus of the diabetes research community must be on
multiple cell types, not just those that produce insulin. For that reason, the DRI’s
cellular and molecular biology efforts go well beyond islets and include many
other cell types that impact metabolism, transplantation and regeneration. These
include dendritic cells, mesenchymal stem cells, endothelial cells, bone marrow cells,
embryonic and adult stem cells, amniotic and cord blood progenitor cells, to name
a few. The study of their similarities and differences, cellular architecture, response
to cellular signals, and a host of other molecular characteristics all play a role in
furthering the understanding of diabetes and the development of new therapies
to restore normal metabolic function.
LEADING WITH VISION:
The DRI has developed the
first ever “living window”
for the study of transplanted
insulin-producing cells.
This groundbreaking DRI
accomplishment enables
Armed with powerful scientific tools, researchers are now able to look deep within
cells at the very molecules that trigger biological and metabolic responses. Cellular
response to inflammation and other known stressors in the cells’ environment help
put the disease process in context, and DRI scientists are focusing on them at the
molecular level. Understanding the building blocks of our body, the living cell, is a
vital area of research. DRI scientists have developed novel ways of viewing complex
cellular interactions, observing distinct cellular signals and tracking dangerous
warning signs that indicate cell destruction and that can serve as predictive markers
for tissue rejection.
Scientists have also developed methods to assess the health and quality of cells
prior to transplantation into patients, an important advance that will help predict
their long-term survival and improve outcomes for patients with diabetes.
scientists to view critical cell
functions in a living organism
in real time. Scientists can now
observe how insulin-producing
cells sense glucose and
secrete insulin, as well as
monitor and assess the
effects of new treatments
for type 1 diabetes.
LEADING WITH VISION:
DRI scientists have patented a new technology to turn pancreatic stem cells into insulin-producing
cells. The revolutionary device, called the “Oxygen Sandwich,” closely mimics the cells’ oxygen-rich
environment, a critical element to successful cell survival and growth that was previously overlooked
by researchers. The DRI found that simply saturating the cells in oxygen resulted in dramatic growth
compared to conventional cell culture methods in use in laboratories around the world.
> HARNESSING AND DEVELOPING UNLIMITED SOURCES OF
INSULIN-PRODUCING CELLS FOR CELLULAR THERAPIES
Scientists are focusing on a number of strategies to optimize cellular therapies for the treatment of
diabetes, including finding alternative cell sources for use in transplantation to bridge the supply gap;
transdifferentiating cells (reprogramming one cell type to grow into another); and even working in
the area of cell regeneration, which may ultimately replace transplantation altogether.
At the DRI, hepatic (liver) cells are already being reprogrammed and driven towards becoming
insulin-producing cells. The use of hyperbaric oxygen is being studied for the role it may play
in enhancing engraftment and perhaps assisting in regeneration. Generating new insulin-producing
cells from one’s own tissues is no longer as remote a possibility as it once was. The DRI will continue
to be at the forefront of that type of groundbreaking research.
In addition, much of the DRI’s research involves inducing tolerance – donor-specific non-responsiveness
of the patient’s immune system to a new transplant. Transplantation tolerance research involves
extensive work, not only with small molecules and novel drugs under development, but in
pre-clinical and clinical work with new antibodies, new bone marrow protocols, and clinical trials
that cross geographical boundaries. Success in the field of transplantation tolerance would represent
a major achievement that would revolutionize the treatment of many diseases in addition to diabetes.
LEADING WITH VISION:
DRI researchers are designing
implantable devices that can
house and protect transplanted
insulin-producing cells. These
bioengineered “containers” hold
many cells and can be refilled
with more cells if necessary.
DRI research teams are taking
this concept even further to make
transplantation safer for patients.
By equipping the device with an
internal “sprinkler” system, the
DRI is working to administer
anti-rejection drugs only at the
site of the transplant instead of
throughout the patient’s entire
body, thereby eliminating
the side effects of generalized
anti-rejection therapies.
> EMERGING TECHNOLOGIES IMPACT
CELLULAR THERAPIES
Investigators are using many newly available technologies to enhance traditional
cell-based science and, as a result, are creating unparalleled opportunities to treat
and cure diseases.
At the DRI, researchers from such diverse disciplines as engineering, immunology,
biomaterials and computer technology are working together as never before. By
contributing their respective expertise, they are generating cutting-edge ideas to
solve the problems of diabetes.
The design and construction of bio-hybrid devices, which are bioengineered structures
that can house transplanted cells, and nanoparticles, extremely tiny materials that go
unnoticed by the body yet protect cells from an immune attack, are two of the newest
end-products of this quickly evolving field of high-end bioengineered medicine.
Developing scaffolds that hold and protect implanted cells from harsh environmental
conditions and reduce immune system attack, encapsulating biomaterials to protect
cells while ensuring adequate nutrition, and the testing of new vessel-promoting growth
factors all constitute new lines of research currently being explored at the DRI. The areas
of research involving tissue engineering, nanotechnology and new biomaterials hold
tremendous promise for scientists in their race find a cure for this disease.
> GROWTH AND DEVELOPMENT OF ADVANCED
TECHNOLOGY PLATFORMS
Over the last several years, the DRI’s research program has grown exponentially.
Once comprised of only a handful of research teams, the DRI faculty and staff now
numbers more than 200, many of whom are investigators from new and exciting
scientific disciplines that didn’t exist yesterday.
Along the roadmap to the cure, talent will need to come from institutes that share
in the DRI’s open door philosophy to research so that progress can continue into the
21st Century in the fastest, most efficient way possible. New laboratories will be
needed to recruit the best minds to the task and ensure that research can continue
without delay. These new facilities will need to be equipped with the latest
technologies to ensure that they are linked with the DRI’s existing
telemedicine/telescience initiatives.
It is only with the ongoing support of private philanthropy that
the DRI will continue on its path of discovery, to recruit brilliant
investigators, provide the infrastructure needed for a world-class
enterprise and Lead the World to a Cure.
LEADING WITH VISION:
To speed the process of developing and testing promising immune system therapies, the
DRI has assembled a specialized research team to focus on designing next-generation drugs.
Using highly-efficient computer modeling and advanced drug design methods, researchers
are working to develop small molecules that can be specifically used to overcome the unique
immune system challenges posed in type 1 diabetes.
LEADING WITH VISION:
The completion of the Diabetes Research Institute’s facility in 1994 ushered in a new era in diabetes
research. The most comprehensive research center of its kind in the world, the DRI enabled the
recruitment of the finest minds, the creation of many scientific programs, and the housing of all
three research levels under one roof. Today, new opportunities in science have made it necessary
to expand the research infrastructure, to acquire new technologies, and to bring in experts from
other disciplines to work synergistically toward a cure for this disease.
National Office
200 South Park Road
Long Island
Suite 100
410 Jericho Turnpike
Hollywood, FL 33021
Suite 201
Telephone 954.964.4040
Jericho, NY 11753
Toll-free 1.800.321.3437
Telephone 516.822.1700
Fax 954.964.7036
Fax 516.822.3570
Regional Offices
California
Northeast
6624 Melrose Avenue
381 Park Avenue South
Los Angeles, CA 90038
Suite 1118
Telephone 323.857.0080
New York, NY 10016
Fax 323.857.1856
Telephone 212.888.2217
Fax 212.888.2219
www.diabetesresearch.org