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Ulcer Pill That Fights Cancer? Scientists Find New Uses for Old Drugs

Thursday, August 18, 2011 2:13 PM

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A computer program that pairs gene information with drug data may help resurrect old medicines for new uses, including an over-the-counter ulcer pill that may be used to treat lung cancer, Stanford University scientists said.

By searching through a National Institutes of Health database that catalogs the results of genomic studies, researchers matched symptoms of diseases with the effects of drugs, yielding 1,000 potential new treatments, said Atul Butte, the study’s lead researcher. The scientists described their work in two reports published Thursday in the journal Science Translational Medicine.

The system, which correctly matched current drugs with the diseases they treat, also suggested GlaxoSmithKline’s Tagamet, a nonprescription ulcer medication, may have use in treating lung cancer, and Johnson & Johnson’s Topamax, an antiseizure medicine sold as generic topiramate, might treat a type of bowel disorder. Finding new uses for old drugs could become much easier using the approach, Butte said.

“If there are additional uses for drugs coming off patents, one could be thinking creatively, how do we find the value in that,” Butte, the chief of divisions of systems medicine at Stanford’s School of Medicine near Palo Alto, Calif., said in an interview.

It takes an average of 15 years and about $1 billion to bring a single new drug to the market, researchers said in one of the reports. So-called repurposing of old drugs can cut that cost because drugmakers wouldn’t have to do the toxicity tests required in early development.

1,000 Potential Treatments

Butte’s team focused on 100 diseases and 164 generic drugs, which yielded about 1,000 potential treatments, Butte said. The matchmaking method provided possible therapeutic drug-disease relationships for 53 of the 100 diseases, he said. Cancers of the skin and stomach showed the most matches, according to his study.

Topiramate was tested in rats after the program proposed its use as a treatment for inflammatory bowel disease. After administering the drug to the animals with the disease’s symptoms, topiramate was effective, sometimes even better than the standard treatment, prednisolone.

That result was compelling, said Pankaj Agarwal, director of computational biology at London-based Glaxo, in an interview.

“The impact of repurposing drugs would be huge,” said Agarwal. “If you find a new use for a drug that’s already on the market and somebody sponsors it, you wouldn’t have to go through safety and have a new indication within three to five years.”

Tagamet Test

Cimetidine, the generic form of Tagamet, was targeted by the system for its potential for lung cancer, and was shown to slow tumor cell growth in mice, according to one report.

“Unless someone focuses on this, the additional uses are never going to be captured,” Butte said. “Companies have to get creative and remarket and rebrand these to tests in clinical trials.”

Pfizer’s $1.9 billion erectile dysfunction drug, Viagra, was originally designed for chest pain, Butte said. Topiramate has been used off-label as a treatment for obesity, researchers said. The drug is also part of Vivus Inc. (VVUS)’s experimental weight- loss compound Qnexa.

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