MDA SUPPORTS FIRST U.S. TRIAL OF LITHIUM IN ALS RELEASE HIGHLIGHTS

MDA to support study to treat ALS with lithium.
Ten sites throughout U.S. to conduct clinical trials.
A previous Italian trial showed that lithium dramatically delayed the progression of ALS.
30,000 Americans are estimated to have ALS.


TUCSON, Ariz., May 7, 2008 - An MDA-supported clinical trial of lithium carbonate, a medication commonly used to treat bipolar disorder, is set to begin in people with ALS following reports that the drug may dramatically slow the progress of this devastating, paralyzing disease.

The study will involve 10 sites throughout the United States. Principal investigator is neurologist Robert Miller, director of the Forbes Norris MDA/ALS Research Center at California Pacific Medical Center in San Francisco.

The study is an effort to confirm or refute the findings of a recent Italian trial of lithium carbonate in ALS conducted by Francesco Fornai at the University of Pisa (Italy) and colleagues at other Italian institutions. The results of that group's study, published online Feb. 4 in Proceedings of the National Academy of Sciences, showed that lithium dramatically delayed progression of the disease compared to standard treatment alone.

"As soon as we saw the intriguing results from Italy, we started working with ALS researchers to launch a U.S.-based trial," said Valerie Cwik, MDA's medical director and vice president of research. "If the drug is helpful, we want all ALS patients to have access to it as soon as possible; and if not, we want to be able to advise them not to take it."

The Italian study involved 44 people with ALS, all of whom were taking riluzole, the only drug approved specifically for ALS. Sixteen trial participants were randomly selected to receive two daily doses of lithium carbonate, and 28 to receive standard care only. All continued taking riluzole.

After 15 months, about 30 percent of the patients in the standard-care group had died, while all those receiving lithium had survived. The disease had progressed markedly in the standard-care group, but very slowly in the lithium group.

The MDA-supported trial will enroll 100 participants who will take twice-daily doses of lithium carbonate to reach and maintain the same blood level used in the Italian study. The trial will run for 12 months, during and after which investigators will compare changes in the ALS Functional Rating Scale (Revised), or ALSFRS-R, to those in people who participated in a recent ALS study but received only a placebo (inactive substance). This "historical control" design allows all participants in the trial to receive the study drug.

"The results from the Italian study were the most important and positive ever reported for ALS," Miller said. "We really need to know that it's real, and this study should give us that answer."

Miller said specific contact information for the trial sites is not yet available.

About ALS

ALS is a progressive, degenerative disease of the motor neurons (nerve cells that control muscles) that usually begins in middle or late adulthood. ALS gradually causes total paralysis of all voluntary muscles, and often leads to death within five years of diagnosis. It's called Lou Gehrig's disease after the famed New York Yankees baseball player who died of the disease in 1941.

About MDA

MDA is the world leader in providing health care services for people with ALS, and in funding research that seeks treatments and a cure for the disease and other related diseases through programs of worldwide research, comprehensive services and far-reaching professional and public health education. Since 1950 MDA has allocated more than $230 million for ALS research and services, and the Association is currently funding ALS research projects totaling nearly $30 million.