Lithium found to be of no benefit to Lou Gehrig’s disease

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The hopes of patients with amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease, were dashed Monday by results from the first clinical trial of lithium, which found that the drug was no better than a placebo. Hopes had been raised by preliminary results from a small Italian trial, reported in 2008, which suggested that the drug could slow progression of the disease. But that study had no control group and patients knew what they were taking, so the results were viewed with skepticism.

Had the results of the new trial proved successful, they would have produced the first new drug for ALS since riluzole was introduced 15 years ago. That drug was shown to extend the lifespan of ALS patients by three months. Victims of the disabling disorder had hoped that lithium, which is already used for treating bipolar disease, might yield even better results.

ALS is a degenerative disease of unknown cause that attacks motor neurons and leads to progressive loss of muscle control, paralysis and, ultimately, death. It strikes about 30,000 Americans each year and most victims live only three to five years after diagnosis.

The new study was organized by Dr. Swati Aggarwal of the Massachusetts General Hospital in Boston and Dr. Lorne Zinman of Sunnybrook Health Sciences Center in Toronto. They enrolled 84 patients; 40 of them were given riluzole plus lithium in the doses used in the Italian study and 44 were given a placebo plus riluzole. Their endpoint was a six-point decline in the so-called ALSFRS-R scale, a 48-point scale that measures the patient’s ability to carry out the tasks of everyday life. Past history suggests that the patient treated only with riluzole would show a decline of about one point on the scale every month.

The team reported Monday in the journal Nature Neurology that they observed no difference between the two groups. Twenty-two of the 40 patients in the lithium group had a six-point decline in ALSFRS-R within six months of starting the drug, compared with 20 of 44 patients receiving placebo. The group had hoped to increase the enrollment to 250 patients if the initial results proved promising, but their findings led them to halt the study.

[Updated April 9. The correct name of the journal is Lancet Neurology.]

‘At this time, there remains no convincing evidence for the use of lithium as a treatment for patients with ALS,’ they wrote.

-- Thomas H. Maugh II