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[IP] Research aim: Make Insulin-producing cells in lab

Research aim: Make insulin-producing cells in lab
Knight Ridder Newspapers

MIAMI -- Doctors expect the next decade to bring a revolution in treatment
options for the 30,000 people who develop new cases of diabetes every year in
the United States.

They foresee these patients moving beyond the rigors of insulin therapy,
pancreas transplants, and even the current focus of research --
transplantation of insulin-producing islet cells.

If researchers, including a new initiative at the University of Miami,
succeed, cells that can make insulin, which converts food into energy, will be
produced in the lab from readily available stem cells, ending diabetics' wait
for rare donor organs.

Parallel research seeks the secrets to preventing the body's rejection of
transplanted organs and cells, freeing recipients from the dangers of
anti-rejection drugs.

"If we can advance this, it will be the treatment of choice," said Dr. Juan
Dominguez-Bendala, recently recruited by the university's Diabetes Research
Institute from the Roslin Institute in Scotland, famous for cloning Dolly the
sheep. "Hopefully it will get us closer to curing diabetes within 10 to 15

The potential for stem cells, the early cells that can turn into any type of
tissue, to cure disease and restore the function of damaged organs was hailed
by university researchers and biomedical industry representatives at a recent
conference of the University of Miami's new Cellular Therapies and Tissue
Engineering Initiative.

Cancer physicians, transplant surgeons, spinal cord injury researchers and
others are joining the diabetes team to discover how replacement tissues might
be manufactured, in many cases using adult stem cells from muscles, skin, even

"This is not an ivory tower kind of initiative," said Dr. Camillo Ricordi,
scientific director of the Diabetes Research Institute and director of the new
initiative. "It has potential application in a lot of degenerative diseases.

"We want to speed up the path of university discoveries from the bench to the

In the case of Type I diabetes, the most serious form, pancreas transplant is
an extremely limited option: Only 1,300 are performed each year, reaching a
tiny percentage of the patients who might benefit.

The transplantation of islet cells, pioneered years ago at the DRI, has
received lots of attention recently because of the success of what's known as
the Edmonton Protocol in keeping diabetics off insulin. The DRI is
participating in trials of the protocol, which will be even more limited
because it takes cells from two donor pancreases for each patient.

Hence the enthusiasm for finding new sources of insulin-producing cells.
Scientists are first trying to figure out, using mice, how the pancreas
develops and what mechanisms cause some cells to become islet cells.

"The hope is that once we can understand that, we can basically recapitulate
those very events starting with other cells and teach them to become
pancreatic islets," said Dr. Luca Inverardi, University of Miami professor of
medicine, microbiology and immunology.

Several sources for those other cells are being studied. Researchers are
working with embryonic stem cells from mice and adult stem cells from various
human tissues -- including fat.

"The idea of having such easy access to these precious cells is quite
remarkable," Inverardi said. "It would make this access a clinical reality
without the need of any type of invasive procedure."

Scientists would prefer to build replacement tissue using the recipient's own
stem cells. "This is the ideal scenario, the perfect goal that we have with
this research," Inverardi said.

Central to the DRI's research on islet cell development is a new lab under the
direction of Swedish investigator Helena Edlund, a world leader in the field.

Re-educating the immune system to accept transplanted cells or organs is also
a major goal. "Even if we were able to obtain enough islets today, they would
not be a perfect cure because the body would attack them," Inverardi said.

Now recipients are given lifelong anti-rejection drugs, which can cause other
complications including cancer and infection. Researchers are pursuing several
approaches to inducing what they call tolerance of the donated organ or cells.

The tissue engineering initiative, which pulls together the work of more than
60 researchers, was designed in large part to help the University of Miami
compete for more of the growing pool of research funding from the federal
government and industry. The co-director is Dr. Herman Cheung, professor in
the schools of medicine and engineering.

"This will capitalize on the unique position we have in some fields and create
synergies between existing fields," Ricordi said. "This is the way to be
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