Exciting New Human ALS Trial: Lithium and Riluzole

FROM THE DIRECTOR

Exciting New Human ALS Trial: Lithium and Riluzole
Recently, a study was published in the Proceedings of the National Academy of Science, by an Italian group of scientists and clinicians examining the ability of lithium to increase the neuroprotective actions of riluzole in ALS mice and patients.
This study was highly significant in that it showed, for the first time, a marked potentiation of the actions of riluzole in ALS patients. It showed that riluzole and lithium - combined - were far more effective than riluzole alone at increasing survival and slowing disease in ALS patients.
Although riluzole has been shown in multiple, large, independent human trials to be effective in ALS, its actions to slow disease are quite small. Lithium, a salt, has many, many actions in the nervous system but is best known for its ability to treat a very different brain disease: manic depression.
The combination of both lithium and riluzole in ALS patients appeared to keep patient alive longer and slow loss of breathing capacity and other clinical measures of disease progression.
To those of us who work very hard to translate science into effective therapeutics and to those who diagnose and manage ALS patients, this new study is extremely exciting.
It shows that drug combinations, when studied carefully, might actually very significantly slow down ALS and extend the lives of patients.
The actual study by the Italians was quite small. And the history of most modern medical research has taught us that small studies with patients need to be replicated with larger studies. All too often results of small studies are not reproduced when better and larger studies are carried out.
For this reason Packard Center clinicians and scientists are in the process of working with major ALS non-profits, the government, and European colleagues to move as quickly as possible to replicate this very small study with a larger one.
Details of the study will be reported as soon as we know more.
Does that mean patients should wait until a larger study of lithium and riluzole is complete before they consider taking the drug? Lithium is a drug very widely used for psychiatric disorders like manic depression. Its use must be monitored by physicians with appropriate physical exams and blood tests.
All patients interested in taking lithium plus riluzole should discuss this with their local physicians and/or neurologists.
The community of scientists and patients have all too often been disappointed with outcomes of ALS clinical trials, especially after we all get excited by experimental mouse data. But human trials provide the only data that really count.
We deeply hope that this novel combination of riluzole and lithium will turn out to be the “real thing” and we are collectively working hard to make that happen.

Jeffrey D. Rothstein MD, PhD

Professor of Neurology and Neuroscience Director,

Robert Packard Center for ALS Research