In the Loop

News and views from across Mayo Clinic

November 4, 2014

Finding New Purpose for Discarded Drug

By In the Loop

drug caplets outside of medication bottleOnce upon a time there was a once-promising drug that everyone had given up on. Everyone, at least, inside the pharmaceutical company that developed it to treat chest pain and peripheral artery disease. After clinical trials failed to produce the desired results, it was sent off to the Island of Misfit Medications. Mayo's Rakesh Suri, M.D., D.Phil., and Jordan Miller, Ph.D., however, had a different idea for it.

According to a story in the Minneapolis Star Tribune, Dr. Suri, a heart surgeon, and Dr. Miller, a researcher with a focus on cardiac valve disease," thought perhaps the drug Ataciguat could be "retargeted" to create a less expensive and more effective medication for other diseases. Dr. Miller tells the newspaper that he and Dr. Suri found that the drug manufacturer may not have "fully realized what it had in the drug." While it may not have worked for chest pain and peripheral artery disease, Dr. Miller and Dr. Suri realized that Ataciguat can be effective in treating aortic valve stenosis, a narrowing and calcification of one's aortic heart valve.

The next step was a grant application with the National Institutes of Health to study the drug’s potential in treating that condition. The Strib reports that the grant currently funding their work allows for a clinical trial of 100 patients. After that, though, the money runs out -- regardless of whether the data and results look promising enough for the drug to continue its march toward FDA approval. So, Drs. Miller and Suri have begun laying the groundwork for creating their own company, with the hope of finding the right partners to help usher their baby into the light of day.

It won't be easy. The paper reports that "from their first discussions with venture capitalists," the duo is finding out that it’s a challenge to "design a trial that gets to 'endpoints' that will indicate the drug’s effectiveness," and "that will make sense to both the FDA and the entrepreneurs." But that's a challenge that Dr. Suri tells the paper he and Dr. Miller feel good about meeting if they can get others to buy into the drug's promise. “Just because you are a gem of the field doesn’t mean you’ll get found and picked up,” Dr. Suri tells the paper. “We understand that now, that you have to market yourself appropriately, and have the appropriate partners to get there.”

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Tags: aortic valve stenosis, Dr. Jordan Miller, Dr. Rakesh Suri, Practice story

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