and W.W. Smith Trust's $15.5 Million Gift for Type 1 Diabetes Cure
Peter Van Etten,
President and CEO,
Juvenile Diabetes Research Foundation
Mary L. Smith, Trustee,
W.W. Smith Charitable Trust
Ali Naji, Vice Chairman,
Surgical Research and Director,
Center for Islet Transplantation
James Wilson, Professor,
Medicine and Director,
JDRF Center for Gene Therapy
Diabetes Research Foundation International (JDRF) announced a major
step in its continuing efforts to find a cure for Type 1 diabetes
and its complications. It is awarding over $15.5 million to fund
research at the JDRF-W.W. Smith Charitable Trust Center for Islet
Transplantation and the JDRF Center for Gene Therapy at Penn's School
a five-year period, the JDRF-W.W. Smith Charitable Trust Center
for Islet Transplantation is receiving $8.1 million to meet the
challenges of restoring normal insulin production in patients with
insulin-dependent diabetes mellitus (Type 1 diabetes). The JDRF
Center for Gene Therapy is receiving $7.57 million to conduct five
projects focusing on gene transfer technology and gene therapy for
complications of diabetes.
our major research goals remain the same, the use of cutting-edge
technology will speed up our progress toward finding a cure,"
said Charles J. Queenan III, JDRF's chair of research. "We
rely on the generosity of funders such as the W.W. Smith Charitable
Trust and thank them for their help in stamping out this insidious
JDRF-W.W. Smith Charitable Trust Center for Islet Transplantation
is developing an integrated approach--three projects and two supporting
core units--to deliver a successful clinical islet transplantation
program. One project is using a non-invasive technique, positron
emission topography (PET), to better understand the fate of islets
after transplantation. A radioactive version of a drug has been
developed that would bind to molecules found specifically on islets.
Currently, studies are examining whether the link between the islets
and the radioactive drug is strong enough to produce optimal visual
are working on two important strategies. First, we want to make
islets less vulnerable to attacks from the body's immune system
and secondly, we are investigating new methods of observing those
transplanted cells in action," commented Dr. Ali Naji, director
of the JDRF-W.W. Smith Charitable Trust Center for Islet Transplantation.
another project, Center scientists have already determined that
transplanted islets have a greater chance of surviving in the thymus
of rodents with diabetes. Their next step is to conduct similar
studies in pre-clinical animal models and use a novel protocol of
drug treatment to prevent the immune system from attacking transplanted
Human Islet Transplantation Clinical Program brings the research
from the lab to the patient with Type 1 diabetes. This program will
identify and monitor patients with Type 1 diabetes who are suitable
candidates for islet transplantation. The program will test the
"Edmonton Protocol" for islet transplantation, using a
combination of three steroid-free drugs to prevent rejection of
the donor islets.
two core units were established to focus on procuring human pancreata
and developing a standardized system to isolate high quality islets
for distribution. The islet core facility will generate large quantities
of highly purified and quality controlled human islets which will
be used for transplantation.
Center, now also funded by the NIH as an Islet Cell Resource Center,
is a comprehensive islet transplantation program, focusing on the
isolation, evaluation, transplantation and imaging of high-quality
human islets for treatment of Type 1 diabetes mellitus. As principal
investigator, Dr. Naji is responsible for assessing the feasibility
of islet transplantation in humans and evaluating new immunosupression
protocols associated with the procedure.
JDRF Center for Gene Therapy will use gene transfer technology and
gene therapy to develop new and more effective treatments for Type
1 diabetes and its complications. At the JDRF Center for Gene Therapy
scientists are making progress using gene therapy for wound healing,
foot ulcers, and diabetic retinopathy--the alterations in the blood
vessels of the retina caused by high levels of glucose. Growth factor
genes are delivered directly to the wounded tissue, to promote healing
where it is needed. Researchers are assessing whether transferring
vascular endothelial growth factor (VEGF) genes to veins and arteries
could promote blood vessel growth. They are also using a new technology--magnetic
resonance angiography--to observe how blood vessels are formed.
They are developing new vectors to deliver genes to the eye that
will cause cells to produce proteins in the vitreous fluid. Abnormal
blood vessel growth would be slowed down or stopped.
with Dr. James M. Wilson, director of the JDRF Center for Gene Therapy,
are exploring an important challenge in gene therapy: the ability
to control gene expression. When researchers transfer genes responsible
for insulin production, there needs to be control because unchecked
insulin leads to hypoglycemia which can be life threatening. Dr.
Wilson is developing a way to turn the action of a particular gene
on and off--a "biological insulin pump". Scientists are
using a virus vector to deliver the insulin gene into the body.
The patient would activate this transferred gene and switch on the
pump' by taking an oral drug. When the patient stops the drug,
the insulin would switch off. The goal would be the control of blood
sugar levels in a simple, patient-directed manner.
have gathered a committed group of Penn experts with a clear objective:
to develop viral vectors and use gene transfer to pursue many approaches
to treatment of complications," said Dr. Wilson. "This
collaboration, I believe, will produce some promising research for
Diabetes Research Foundation, JDRF, the world's leading non-profit,
non-governmental funder of diabetes research, was founded
in 1970 by the parents of children in Philadelphia and New
York with juvenile diabetes--a disease which predominantly
strikes children suddenly, makes them insulin dependent for
life, and carries the constant threat of devastating complications.
Since inception, JDRF has provided more than $500 million
to diabetes research worldwide. Eighty-seven cents of every
dollar goes directly to research and education about research.
JDRF's mission is constant: to find a cure for diabetes and
its complications through the support of research.
more information, visit their web site at www.jdrf.org,
or call 1-800-223-1138.
Almanac, Vol. 48, No. 13, November 20, 2001
November 20, 2001
Volume 48 Number 13
receives over $15.5
million from JDRF and W.W. Smith Trust to establish two
centers to find a cure for Type 1 diabetes and its complications
2001, the Associated Investments Fund, Penn's main investment
vehicle, out-performed its benchmark, returning a positive performance.
have been redesigned to be more informative and easier to
Way weekly raffles begin; envelopes must be submitted to
Payroll by this Wednesday for the first drawing.
fall semester classes ending December 10, the Provost reissues
Governing Final Examinations.
types of grants
are available to faculty to conduct cancer-related research
projects; the deadline is January 15 for both.
the holidays approach, there are special events
and performances to attend and there are the annual appeals
to donate and contribute to those less fortunate.