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From bench to bedside

Researcher transforms lab discoveries into hope for patients

Dr. Elliott Richelson

In more than two decades of research to develop new drugs, Dr. Elliott Richelson, a psychiatrist and scientist at Jacksonville's Mayo Clinic, has compiled a list of achievements that would be the envy of most researchers. It includes everything from large federal research grants to noteworthy awards and a number of patents. But despite all these accomplishments, he has yet to achieve his ultimate ambition: creating a safe, effective drug that is used in people.

"I've lost count of the number of patents I have," he says with a laugh. "To be honest, they don't matter. After 20 years, it would just be nice to get something into the clinic to help patients."

He may soon reach that goal. Recently Mayo Clinic licensed one of Richelson's discoveries to a start-up company. The company will perform all of the remaining testing, production and government filings necessary to launch the first clinical trial of the drug, which will be tested first for its potential as an anti-pain drug.

"We would like to test it first in the postoperative setting, for patients coming out of surgery," says Richelson. "Our studies show it has a potent pain-relieving effect — more so than morphine — and it will likely have a much more favorable side effect profile."

If all goes well, the first clinical study could begin in about a year. And that may just be the beginning of what is possible for the drug, which is itself an interesting story and compelling example of Mayo's strengths in medical research.

The drug is based on a naturally occurring substance in the brain called neurotensin, which has a powerful role in certain brain functions. For example, animal studies have shown that, in addition to its anti-pain properties, neurotensin has potential as a possible treatment for schizophrenia and Parkinson's disease and as a tool to help wean people from addictions. But these possibilities have been difficult to pursue because scientists had been unable to develop a practical way to deliver neurotensin treatments. Mayo's tradition of collaboration, which is prominent not just in patient care but also in research, helped Richelson solve this problem. Wo neurotensin with a synthetic molecule. The resulting compound can be administered like other treatments and still pass through the brain's security system — the blood-brain barrier — to reach its therapeutic targets.

The collaborative environment at Mayo also led Richelson and his staff to pursue studies of the drug as a possible tool to help people quit smoking. Dr. Paul Fredrickson, a psychiatrist at the Jacksonville campus and director of the Nicotine Dependence Center helped launch this research. Dr. Mona Boules, a member of Richelson's lab, has since received a large grant from the state of Florida to study the idea further, and so far, her results have been exciting. In laboratory rats that were conditioned to self-infuse nicotine through a tube inserted into a vein, the neurotensin compound completely removed their urge for nicotine.

If the drug produces a similar result with equal safety in smokers, it will help address a major public health concern, says Fredrickson, who believes the research also serves as an important example of another Mayo specialty: translational research.

Highly dependent on the ability of scientists and physicians to work together, translational research focuses on transforming scientific discoveries into practical applications that directly benefit patients.

Even if all goes well with the testing, it will still be years before the neurotensin compound is evaluated in people as a therapy for schizophrenia and Parkinson's disease, two of its most exciting possibilities. The drug has the potential for offering more favorable side effects compared to existing treatments for these disorders.

In the meantime, Richelson says, he won't spend a lot of time worrying about a negative laboratory test result stopping the drug from going forward. One of the benefits of studying something for 20 years? Time to develop a back-up plan. "You do worry a little bit about a test result killing the whole thing, but if that happens, we have other compounds we can test," Richelson says.

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