Type 1 diabetes, Type 2 diabetes -- signif-
icant step forward in regenerative medicine:
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August 28, 2008
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ScienceDaily (Aug. 28, 2008) - In a feat of
biological prestidigitation likely to turn the
field of regenerative medicine on its head ...
- directly turning one type of fully formed
adult cell into another type of adult cell.
... using a technique it is calling "direct repro-
gramming," the team is able to turn mouse
exocrine cells, which make up about 95 per-
cent of the pancreas, into precious and rare
insulin-producing beta cells. These beta cells,
which comrpise about one percent of the pan-
creas, are the cells that die off in Type 1 dia-
In addition to its value for the field of regen-
erative medicine, the work also is a major
step forward toward eventually developing a
treatment for Type 2 - and eventually Type 1 -
"We're intrigued by the possibility that this
approach, which has worked for pancreatic
insulin-producing cells, could be more widely
applied to many kind of cells, especially those
that are lost in disease or following injury,"
Melton said. "And at the same time, we are
exploring the possibility of using this general
approach in a clinical context to make new
beta cells for patients."
The work was principally supported by the
Harvard Stem Cell Institute and Howard
Hughes Medical Institute.
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In a discovery that's being hailed as a leap
forward in regenerative medicine, researchers
have found a way to transform common pan-
creatic cells in an adult mouse into the rare,
insulin-producing beta cells that are destroyed
in type 1 diabetes. Previously, researchers
believed that the only way to transmute an
adult cell was to first coax it back into stem
cell form and then to reprogram it; this new
research removes the first step entirely.
The accomplishment raises the tantalizing
prospect that patients suffering from not only
diabetes but also heart disease, strokes and
many other ailments could eventually have
some of their cells reprogrammed to cure
their afflictions without the need for drugs,
transplants or other therapies.
"It's kind of an extreme makeover of a cell,"
said [lead researcher] Douglas A. Melton..
The goal is to create cells that are missing or
defective in people. It's very exciting" [Wash-
Excerpts (from Washington Post article):
The experiments, detailed online yesterday in
the journal Nature, raise the prospect that
patients suffering from not only diabetes but
also heart disease, strokes and many other ail-
ments could eventually have some of their cells
reprogrammed to cure their afflictions without
the need for drugs, transplants or other therapies.
"It's kind of an extreme makeover of a cell," said
Douglas A. Melton, co-director of the Harvard
Stem Cell Institute, who led the research. "The
goal is to create cells that are missing or defec-
tive in people. It's very exciting."
"I'm stunned," said Robert Lanza, chief scientific
officer of Advanced Cell Technology in Worcester,
Mass., a developer of stem cell therapies. "It intro-
duces a whole new paradigm for treating disease."
Although the experiment involved mice, Melton
and other researchers were optimistic that the
approach would work in people.
Melton has already started experimenting with
human cells in the laboratory and hopes that
within a year he can start planning the first stu-
dies involving people with diabetes. "I would
say within five years, we could be ready to start
human trials," Melton said.
Other scientists have begun trying the approach
on other cells, including those that could be
used to treat spinal cord injuries and neurode-
generative disorders such as Lou Gehrig's
"Embryonic stem cells offer a unique window
in human disease and remain a key to the long-
term progress of regenerative medicine," Melton
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"And now that it's shown that you can turn one
of your cells into another, it makes you think of
what other cells you'd like to convert" [Wired
Excerpts from Wired News article:
In an unprecedented flourish of genetic alchemy,
scientists used a virus to coax one type of cell to
become another, without the intermediate stem
The research, conducted with cells from the pan-
creas, could soon be used to treat people with
diabetes -- but its long-term impacts could be
"This represents a parallel approach for how to
make cells in regenerative medicine," said Doug-
las Melton, co-director of the Harvard Stem Cell
Institute. "And now that it's shown that you can
turn one of your cells into another, it makes you
think of what other cells you'd like to convert."
Melton's team avoided stem cells, and their bag-
gage, altogether by using a virus to tweak three
developmental genes in pancreatic tissue cells
in mice. Three days later, these became insulin-
producing beta cells, and appear free from the
complications that have frustrated stem cell
If the technique, described today in Nature, is
replicated in humans, it could be used to treat
insulin deficiencies in people with diabetes
-- and that's just the start.
"Neurodegenerative diseases come to mind, as
does cardiovascular disease," said Melton.
Arthur Caplan, a University of Pennsylvania bio-
ethicist who wasn't involved in the study, called
the findings a "breakthrough" for both diabetes
and the field of regenerative medicine.
"It's a system that's easier to manipulate than get-
ting a new stem cell to turn into something you
want," he said. "The kind of work done here has
the promise to go into clinical practice in a rela-
tively short time."
Caveats remain, the foremost being the replica-
tion of the work in human tissue.
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