BYU
poli sci professor publishes medical journal article about possible cystic
fibrosis treatment
Her two sons suffer from the disease; next step, clinical trials
PROVO, Utah (June
11, 2001)—This week, Brigham Young University political science professor
Valerie Hudson will publish a scientific article in a medical journal advocating
a new approach to treating cystic fibrosis. Inspired by her research, a
clinical trial testing the approach is underway in Germany, with an American
team ready to begin a similar trial as soon as funding is secured.
The mother of
two boys diagnosed with cystic fibrosis, Hudson grew impatient with the
rate of medical research on her sons' fatal hereditary disease and wanted
more information than health care professionals had to give.
So the professor,
who teaches the graduate students in her research methods class that research
acumen can open doors in any realm of knowledge, taught herself human physiology
and molecular biology from scratch. Although she didn't plan to, along
the way she came up with a new understanding of the way cystic fibrosis
attacks the lungs and digestive tract, which pointed to a novel therapy.
If funding can be acquired and subsequent clinical trials bear out her
hypothesis, it could mean immediate alleviation of many of the disease's
symptoms at a fraction of the cost of current treatments.
"It's unglamorous,
it's old, it's unpatentable; nobody is going to get intellectual property
rights off of it so nobody's going to make a fortune," said Hudson."So
most researchers want nothing to do with the ideas of the person they view
as the wacky political science woman out in Utah."
It is that inexpensiveness
and Hudson's unorthodox credentials that she believes have caused the traditional
cystic fibrosis research community to turn up its collective nose at her
work, published in this week's issue of Free Radical Biology and Medicine,
and, without seeking to understand it, deny funding to the trial she and
some medical professionals advocate.
Cystic fibrosis
is an incurable genetic disorder in which patients suffer from abnormally
thick mucus that clogs their lungs and digestive tract. Their chronically
inflamed lungs are plagued by persistent infections that eventually waste
away their lung capacity and kill them. Life expectancy for the 30,000
Americans with the disease is about 30 years. The condition results from
a mutation of the gene responsible for manufacturing a protein known as
CFTR that normally serves as a channel out of most cells. In cystic fibrosis
patients, the channel is blocked and doesn't allow the passage of certain
biochemicals. One of them is called glutathione, which performs a number
of functions that protect healthy people's lungs.
As teens, cystic
fibrosis patients have between 5 and 20 percent of the levels of glutathione
on the surface of their lungs that healthy people do since the chemical
is trapped inside their lung cells. Hudson's article shows how this deficiency
could cause many of the symptoms of cystic fibrosis. The therapy she advocates
in the journal article involves administering a solution of supplemental
glutathione to patients. That approach is already used to treat some people
with emphysema and adult respiratory distress syndrome. (For a more details
of the science behind Hudson's proposal, visit here).
She cautions
that she is not claiming to have understood or solved the entire mechanism
with which cystic fibrosis attacks patients. Rather, Hudson says, she has
identified a new approach that further research could show to alleviate
many symptoms.
A few scientists
outside the mainstream cystic fibrosis research community were willing
to look at her paper - stacked with a bibliography of 360 studies that
support parts of her hypothesis - and offer her advice and encouragement.
Anonymous reviewers at Free Radical Biology and Medicine accepted
the paper for publication. (For more on Hudson's abrupt introduction to
medical research, visit here).
Henry J. Forman,
chairman of the Department of Environmental Health Sciences and co-director
of the Center for Free Radical Biology at University of Alabama-Birmingham,
has published studies on the role of glutathione in cystic fibrosis and
is the co-editor of the journal that published Hudson's article.
"(The paper)
went through a fairly tough review. It's valid as a hypothesis - it's testable
and the chemistry underlying the potential protective role of glutathione
is there," Forman said.
"In general,
most people would agree it's a good idea to restore glutathione to normal,"
he said of Hudson's assertion. "It's hard to
say if this
therapy will make a major difference - it would certainly seem that increasing
glutathione in the (lung's) airspace would have an advantageous effect."
Forman believes
a properly controlled clinical trial that gives careful attention to the
amount and form of the glutathione administered, "is likely to be, at worst,
innocuous, and at best, helpful."
While her paper
was being reviewed, Hudson sought credentialed medical researchers to conduct
clinical trials to test if giving cystic fibrosis patients supplemental
glutathione improves their condition.
Clark Bishop,
a pulmonologist and regional medical director at 330-bed Utah Valley Regional
Medical Center in Provo, reviewed Hudson's research and agreed to introduce
her to fellow physicians who might conduct a trial. He was so disappointed
by the indifference of the cystic fibrosis research community that he reluctantly
volunteered to conduct a trial himself, provided funding can be secured.
He feels obligated not to turn his back on an approach he believes has
the potential to relieve suffering.
"I'm not looking
for extra things to do," said Bishop, who works with the 600 physicians
on the medical staff of the IHC's Urban South Region. "But this is a hypothesis
that deserves to be tested. The basic science is sound, and the mechanism
is well worked out.
"Right now we
do very little to get at the underlying pathology of the disease," he said,
acknowledging that future gene therapy will be the best avenue for completely
curing cystic fibrosis. "In the meantime, we have the opportunity to make
a leap forward and it is being ignored by the cystic fibrosis community.
This isn't the root, but it's certainly a lot closer to the tree."
Hudson is practically
begging for any organization to fund Bishop's trial, which he said would
cost less than $50,000. After meeting Hudson's husband at the European
research conference on cystic fibrosis, Matthias Griese, a cystic fibrosis
researcher at the children's hospital at the University of Munich, is in
the middle of a clinical trial in which he is administering inhaled glutathione
to 18 patients. But Hudson believes American doctors will only listen to
American doctors, so she holds out hope for funding for an American trial.
"It was never
my intent to carry the baton forever, but to pass it to the professional
community," Hudson said. "In none of my material have I attempted to prove
that glutathione works, but rather have attempted to demonstrate that there
is ample warrant for a serious clinical investigation. I hope the fact
that I'm a political scientist and a 'CF mom' doesn't doom this therapy,
which I believe could help all cystic fibrosis children. It would be simple
to do this trial and find out for sure."
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