Schizophrenia drug shows promise in clinical trial

In a clinical trial of about 200 patients, an experimental drug from Eli Lilly reduced schizophrenia symptoms without the serious side effects of current treatments, according to a paper published Sunday in the journal Nature.

The drug must still be evaluated on many more patients to test for the possibility of side effects that have not yet emerged, and it is at least three to four years from completing regulatory review.

But schizophrenia researchers said the trial's results were surprising and impressive, especially since the drug works in a different way from existing anti-psychotic medicines, all of which have serious side effects, including substantial weight gain and tremors.

Lilly will begin a larger clinical trial for the drug this month. If that trial confirms the results seen so far, the new drug could mark a breakthrough in the treatment of schizophrenia — and open the way to a broad new class of treatments for the disease. Schizophrenia, a devastating mental illness that affects 1 percent of adults, or about 2.5 million in the United States, usually begins in the late teens or 20s and is marked by psychotic delusions as well as social withdrawal and cognitive impairment.

"This is potentially one giant step forward for patients," said Dr. Jeffrey Lieberman, chairman of the psychiatry department at Columbia and the lead investigator on a federally sponsored clinical trial of schizophrenia medicines. "This drug may turn out to be not just a comparably good anti-psychotic agent, but a better anti-psychotic agent."

Lieberman has not been involved with the development of the medicine and does not receive any payments or consulting fees from Lilly.

Existing schizophrenia medicines, whether older drugs such as Thorazine or newer medicines like Zyprexa, all work by blocking the brain's dopamine receptors.

But the new Lilly drug does not directly affect dopamine. Instead, it modulates brain activity through a different set of receptors. As a result, it has the potential to be the first truly novel treatment for schizophrenia since Thorazine was introduced 1954, Lieberman and other researchers said.

Lilly's new drug — which does not have a name yet and is referred to as LY2140023 — emerged from almost two decades of research by Dr. Darryle D. Schoepp, a toxicologist and pharmacologist who joined Lilly in 1988.