High-dose Tamoxifen may prolong survival in amyotrophic lateral sclerosis (ALS)

Neurologist Benjamin Brooks, who directs the MDA ALS Center at the University of Wisconsin-Madison, presented results of a trial of tamoxifen, a drug approved for the treatment of breast cancer, at a meeting of the American Academy of Neurology in April. The trial was supported in part by MDA.

In a two-year study of 60 people with amyotrophic lateral sclerosis (ALS) who took varying dosages of tamoxifen, which changes the way the body responds to the female hormone estrogen, Brooks found that participants who took more than 20 milligrams a day had a better survival rate than those who took fewer than 10 milligrams a day.

After two years, 27 out of 37 (73 percent) of the participants in the high-dose tamoxifen group had survived, while only 11 of 23 (48 percent) of those in the low-dose groups were still living.

There was no difference among the various dosage groups on measures of muscle strength, respiratory capacity, activities of daily living or functional abilities.

Brooks cautioned that a longer trial would be necessary to demonstrate with certainty that there’s a survival benefit to taking tamoxifen at the higher doses.

He’s planning further studies to investigate the effects of tamoxifen and also recommends that the metabolic products of tamoxifen be studied.