The ALS Association Works with Investigators on
Follow Up Study of Lithium for Treating ALS

[Quick Summary: An Italian study of 44 people reports that a 15-month trial found that daily doses of the drug lithium, used to treat bipolar disorder, significantly slowed the progression of ALS in patients also taking riluzole, the only FDA-approved for the treatment of Lou Gehrig’s disease.]

The ALS Association is working with investigators and funding organizations to plan a follow up clinical study of lithium as a treatment for ALS.

The 15-month trial conducted on 44 people with amyotrophic lateral sclerosis, commonly known as Lou Gehrig’s disease, reported that the progressive neurodegenerative disease was slowed significantly in patients receiving daily doses of lithium along with riluzole, the only drug approved by the U.S. Food & Drug Administration for the treatment of ALS.

Francesco Fornai at the University of Pisa (Italy) with colleagues at the University of Novara (Italy) and the Santa Lucia Foundation in Rome, demonstrated that lithium was neuroprotective and increased survival in the mouse model of ALS. These promising results led to the clinical study.

“The results of the study are very encouraging, however the number of patients in this trial is small, and a larger trial would be needed to confirm that lithium is beneficial in ALS,” said Lucie Bruijn, Ph.D., science director and vice president of The ALS Association. Lithium has known benefit in bipolar disease and is currently being explored in other neurological disorders. The increasing numbers of studies aimed at understanding the biological mechanisms that may be involved in the neuroprotective effect of this agent and the pharmacological data that is available make this an attractive compound to pursue in further studies.

“It will be important to determine the appropriate dose and whether the beneficial effects are due to lithium or the combination of lithium and riluzole as all participants were also taking riluzole,” Bruijn said.

“The safety of administering lithium to people with ALS has not been carefully explored, and if lithium is helpful for ALS, the optimum dose would need to be determined,” Bruijn said. Lithium is toxic at higher doses and careful monitoring of blood levels of lithium as well as laboratory blood tests of kidney and liver function need to be performed. Clinicians caution that lithium may interact with other medications.

The ALS Association urges people with ALS to consult their primary care physician and discourages the use of lithium before appropriate trials have been coordinated. The Association is working to expedite these studies and will keep the community informed.