I pulled the following article from the Children with Diabetes
website. I never heard of this study before and was wondering if
anyone knew what the drug is that they are going to test. It sounds
a lot like Dr. Faustman's work in that big pharma is not involved. I
thought it was interesting. Any thoughts or information would be
appreciated.
Ellen Crain

Brookline diabetes researcher makes FDA history with clinical trial
By Jessica Scarpati/Staff writer
Wed Feb 13, 2008, 05:01 PM EST
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Brookline - When that 2-foot-thick stack of paperwork from the FDA
arrived on Dr. Tihamer Orban's desk, he took one look at it and made
his decision about developing a drug on his own and leading the
clinical trial.

"I said, `There's no way I can do this,'" sighed Orban, an Aspinwall
Avenue resident and Hungary native.

Luckily for Type 1 diabetes patients around the world, Orban is not a
man of his word — at least in this respect.

The 15-year researcher at Boston's Joslin Diabetes Center has become
the second person in the history of the federal Food and Drug
Administration to get approval to test a drug he developed without
the help, politics or funding of a big pharmaceutical company.

And if the early successes of his maverick vaccine continue, Orban
said it could pave the way for finding a cure.

"If this drug will do what I hope it would, then we can make a major
impact in Type 1 diabetes," said Orban, a pediatrician by training
who also is an instructor at Harvard Medical School.

Want to get in on it?
Take learn more about the B-chain insulin study, go to the Joslin Web
site or e-mail Dr. Tihamer Orban.

Representatives from the FDA and the American Diabetes Association
could not be reached for comment.

Type 1 diabetes is an autoimmune disease — meaning the body
mistakenly attacks its own mechanisms — that gradually kills off
insulin-producing beta cells in the pancreas. The body needs insulin
to regulate blood sugar to survive.

The disease is also known for having strong genetic ties, though no
genetic test has been found yet. It is often found in children, but
can present itself later in life as well.

"You have a large prediabetic time," Orban said, explaining that
patients can still have 30 to 40 percent of their insulin-producing
cells intact at this time. "You have the disease process, but you
don't have the clinical disease."

As a result, Orban's work focuses as much on prevention as it does
intervention.

"The only thing is to do is to stop the auto-aggressive process," he
said. "This sounds very simple, but is very difficult to do."

Often putting in 10- to 12-hour days, Orban said he still finds the
work rewarding, even though a cure has eluded him and thousands of
others before him.

"I'd rather fail 10 times than not try," he said. "If you think it's
meaningful, then the time passes by, and you go home and
think, `Bloody hell, I haven't done half.'"

Trial of few errors, so far
In his "investigator initiated" drug trial, Orban said he bucked the
usual trends of focusing on immunosuppressive drugs that try to stop
Type 1 patients' bodies from destroying insulin.

"This is actually conceptually different," Orban said. "This is
actually a stimulatory agent."

Here's how it works.
"I came up with the concept that autoimmune Type 1 diabetes is the
loss of self-tolerance," Orban said. "So, what I thought would be
most adequate was to remind the immune system of the antigen — of
what it forgot about."

Orban said the immune system learns to tolerate "self material" early
in life, through T cells in the thymus, a lymphatic organ in the
chest. He called it "thymic education."

"You go to school, you have to learn, `This is mine — don't bother,
don't kill,'" he said. "In autoimmunity, this memory is not lost
completely, but it is mostly forgotten."

How could he remind the immune system to recognize
insulin? "Vigorous" doses of insulin might work, except that a
patient would become hypoglycemic and die.

That is, until Orban started to break up insulin's structure — which
appears as a ladder, an "A chain" and a "B chain," glued together by
chemical bonds.

Orban said he isolated the B chain, which is not metabolized the same
way as the whole insulin structure.

"I can give an unlimited amount," he said, which for his trial,
translated into a one-time shot of 2 milligrams of B-insulin — an
amount the average adult produces over two years.

Orban said he alone did most of the toxicology studies for his
nameless injection before presenting it to the FDA. He hired an
independent toxicology lab to verify the results.

The FDA signed on, and Orban got funding for the human trial from the
National Institutes of Health, the federal government's research
agency.

Having just completed the first phase of a two-year clinical trial
for the new drug on 12 patients — half of whom received a placebo —
Orban is beginning to gather 100 to 120 people for the second phase
of trials to support his results.

"We looked for … whether we can wake up the immune system," he
said. "I can tell you we did."

That first group showed no side effects, which was another big win —
especially for a drug that could be used among infants and children.

Though in addition to securing patients, Orban must also find public
and private funding. A trial such as this, he said, costs "several
millions" of dollars.

"We got a clear immune response that I don't think anybody has really
achieved," Orban said. "But these are very early, so I wouldn't
overstate that. We need to study this more."

What do you think? Add your comments at www.wickedlocal.com/brookline.

Jessica Scarpati can be reached at jscarpat@cnc.com.

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