Thursday, February 23, 2012

NOT: With Help from Author Laura Hillenbrand, Drug Repurposing Comes to Chronic Fatigue Syndrome

http://blogs.wsj.com/health/2012/02/23/with-help-from-author-laura-hillenbr=
and-drug-repurposing-comes-to-chronic-fatigue-syndrome/

With Help from Author Laura Hillenbrand, Drug Repurposing Comes to
Chronic Fatigue Syndrome.
By Amy Dockser Marcus
February 23, 2012, 8:40 AM.


The CFIDS Association of America says that $2 million in funding =97
backed by individual gifts including a $250,000 donation from
best-selling writer Laura Hillenbrand, who has chronic fatigue
syndrome=96 is going to grants and projects designed to accelerate the
development of treatments for CFS.

One of the new projects: an attempt at so-called drug repurposing,
when already-approved drugs for one indication are tried in a
different disease.

As the WSJ has reported, drug repurposing is getting a big push from a
number of quarters, including the NIH, which is targeting repurposing
efforts in rare diseases, and organizations such as the ALS Therapy
Development Institute, which recently reported that an approved
multiple sclerosis drug appeared to be effective in mice with ALS.

The high cost of developing new therapies has helped drive recent
interest in drug repurposing. The concept is that a drug already
approved for one condition will cost less and move faster into the
clinic if it turns out to also work in a different disease.
Nonetheless, at first glance, CFS might seem an unusual condition in
which to try drug repurposing.

CFS has no known cause. Doctors usually make the diagnosis by ruling
out other problems. Many of the symptoms =97 including muscle and joint
pain, cognitive dysfunction, headaches, and unrefreshing sleep =97 are
very difficult to treat. There is currently no FDA-approved drug for
CFS.

If repurposing succeeds in coming up with potential new therapies to
test in CFS, it could serve as a model for other complex conditions.
Many chronic diseases are heterogeneous, lack approved biomarkers, and
are not well-defined, says Suzanne Vernon, the CFIDS Association=92s
scientific director. =93CFS is in many ways in the norm when it comes to
chronic diseases,=94 she says.

Hillenbrand, who says the success of her recent book =93Unbroken=94
enabled her to make the $250,000 gift, tells the Health Blog she
supports an integrated approach to CFS research. =93Things are finally
happening,=94 Hillenbrand says.

She points out that one of the most promising recent developments in
CFS was actually a repurposing project. Researchers in Norway
published research last year suggesting that the cancer drug Rituxan
relieved symptoms in some CFS patients.

The CFIDS Association=92s repurposing project will be run by the biotech
Biovista. The company created a technology that is able to analyze
huge quantities of information =97 drugs in the public domain, adverse
event databases, gene targets, patents, medical literature =97 and look
for novel connections between drugs=92 mechanisms of action and key
symptoms of CFS, says Aris Persidis, president of Charlottesville,
Va.-based Biovista.

In any disease where there is =93tremendous data and history=94 and the
available facts are =93interpreted differently by different groups of
experts,=94 it can seem difficult to find new drug candidates, says
Persidis. A computational approach allows novel analysis =93without any
bias, in an agnostic manner,=94 he adds.

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