Drug ‘repurposing’ comes to CFS, Wall Street Journal health blog, 23 February 2012

February 23, 2012

From the Wall Street Journal health blog, 23 February 2012 (story by Amy Dockser Marcus)

The CFIDS Association of America says that $2 million in funding — backed by individual gifts including a $250,000 donation from best-selling writer Laura Hillenbrand, who has chronic fatigue syndrome– 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 — including muscle and joint pain, cognitive dysfunction, headaches, and unrefreshing sleep — 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’s scientific director. “CFS is in many ways in the norm when it comes to chronic diseases,” she says.

Hillenbrand, who says the success of her recent book “Unbroken” enabled her to make the $250,000 gift, tells the Health Blog she supports an integrated approach to CFS research. “Things are finally happening,” 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’s repurposing project will be run by the biotech Biovista. The company created a technology that is able to analyze huge quantities of information — drugs in the public domain, adverse event databases, gene targets, patents, medical literature — and look for novel connections between drugs’ mechanisms of action and key symptoms of CFS, says Aris Persidis, president of Charlottesville, Va.-based Biovista.

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

Click on the following link to read the following PRNewsire story: The CFIDS Association of America Breaks Ground on Its ‘Institute Without Walls' to Transform Chronic Fatigue Syndrome Research

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