[IPu] Fw: [IP] Re: Human clinical trials this spring
This one looks promising ...
From: "Julie Errichetti" <email @ redacted>
To: <email @ redacted>
Sent: Thursday, March 30, 2006 12:40 AM
Subject: [IP] Re: Human clinical trials this spring
Just thought you might be interested in human clinical trials currently
way which focuses on the destruction of the Tcells, not just the blocking of
By SHARON BEGLEY
DOW JONES REPRINTS
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After Initial Rejection,
Scientists Back Work
On Cure for Diabetes
March 24, 2006
When Denise Faustman announced that she had cured mice of diabetes, funders
didn't exactly beat a path to her door, and colleagues didn't shower her
To the contrary. After her 2001 breakthrough, Dr. Faustman, an associate
professor of medicine at Harvard Medical School, couldn't interest drug
companies or the Juvenile Diabetes Research Foundation in supporting the
next step she proposed: testing in people a version of what cured the mice.
When she published a similar study two years later, reaction from colleagues
wasn't much better. Two fellow Harvard diabetes experts sent a letter to the
New York Times, which had run an article describing Dr. Faustman's work,
calling the claim that she was the first scientist to cure diabetes in mice
"patently false." They also apologized to people with diabetes "on behalf of
Dr. Faustman" for "having their expectations cruelly raised." JDRF, getting
flak for not funding her, circulated the (unpublished) letter to show that
scientific verdict on her results was far from unanimous, explains spokesman
But JDRF did approve grants to three competing teams, including one led by
an author of the critical letter, to attempt to replicate Dr. Faustman's
Now all three are announcing they have confirmed the aspect of her study
is the basis for a clinical trial planned at Harvard. By keeping the mice's
immune system from destroying their insulin-making beta cells, the three
report in today's issue of the journal Science, they got beta cells in some
(but not all) of the animals essentially to come back from the dead, curing
In the three studies -- from the University of Chicago, Harvard and
Washington University -- about one-third of diabetic mice were cured. They
normal blood-sugar levels even though they had only a few beta cells.
"Autoimmune diabetes can be reversed," the Chicago team says. It isn't clear
why only some mice were cured, but scientists are working on getting higher
response rates, says Chicago's Anita Chong, who speculates that tweaking the
treatment might help.
Biomedical science has a long history of mouse cures that never become
cures, but this one may be different. The mice had long-established
due to the same mechanism that causes Type-1 diabetes in people: the immune
system's destruction of beta cells. Without enough functioning beta cells,
there is too little insulin to keep blood sugar in check. That can lead to
blindness, kidney failure and amputations. Although a number of treatments
keep mice from developing diabetes, "few can induce its reversal," wrote Dr.
In the 2003 study that the three labs tried to confirm, Dr. Faustman and
colleagues gave diabetic mice a compound that destroys killer T-cells. They
also transplanted cells from the spleens of healthy mice into diabetic mice.
The transplants bloomed into beta cells, they reported.
That suggested the spleen contains adult stem cells that can morph into
specialized cells. Dr. Faustman attributed the cure largely to this, landing
her smack in the middle of the stem-cell debate. The juvenile diabetes
foundation and a number of scientists argue passionately for research on
stem cells obtained from embryos. Some who oppose that research for ethical
reasons talk up the potential of adult stem cells.
None of the three teams found that transplanted spleen cells differentiated
into beta cells. "Denise Faustman was extremely helpful to us in duplicating
her protocol, but it's possible we did something wrong, and so can't
absolutely rule out the possibility that the spleen contains stem cells that
can become beta cells," says Chicago's Louis Philipson.
For patients, it may not matter. Harvard's David Nathan will soon launch a
clinical trial, funded with some of the $11.5 million grant the Iacocca
Foundation gave Dr. Faustman when others turned her down. It will not use
spleen cells. It will inject diabetic volunteers only with a compound called
BCG; like the one given to mice, it stimulates the immune system in a way
eliminates T-cells that attack beta cells. With the T-cells gone, they hope,
surviving or regenerated beta cells will yield enough insulin to reverse
That a diabetes-ravaged pancreas contains enough beta cells to support a
cure is arguably better news than finding that spleen-cell transplants are
key. Harvard's Diane Mathis and her colleagues discovered that even in mice
with long-established diabetes, there is "substantial beta-cell mass, which
can be rejuvenated/regenerated to reverse disease." If so, then cell
transplants, from cadavers or embryonic stem cells, wouldn't be necessary.
she cautions that earlier trials of BCG have failed.
"The good news is that all three groups cured mice as we did," says Dr.
Faustman. "They showed that it was due to regeneration in the pancreas, and
that's the beauty of it: The animals' own pancreas did this."
She still thinks transplanted cells from the spleen might produce beta
cells. "The pancreas is too smart to cure itself in only one way," she says.
"I think there will be many sources of regeneration, and we're only at the
beginning of understanding what they are."
See these links for just published replication efforts.
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