MINOCYCLINE INEFFECTIVE FOR ALS;
NEWS FROM AAN ANNUAL MEETING

Disappointing results of a nine-month trial of the drug minocycline for amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) were announced today at the American Academy of Neurology Annual Meeting in Boston.

Minocycline demonstrated no beneficial effect, and for some patients, worsened measurable outcomes.

People with ALS who are currently taking the drug, an antibiotic in the tetracycline family, are urged to contact their physician to discuss discontinuing its use, said MDA Medical Director Valerie Cwik, who is at the meeting.

The drug did not affect survival or quality of life measures for people with ALS in the trial.

The trial tested minocycline versus a placebo (inert, look-alike substance) in 412 people at 31 U.S. centers. The drug was thought to reduce inflammation and counteract cell death.

ADDITIONAL HIGHLIGHTS FROM THE AAN ANNUAL MEETING (April 28-May 5, Boston)

  • A 20-week trial of sodium phenylbutyrate in 40 people with amyotrophic lateral sclerosis (ALS) at eight centers showed the drug was safe and tolerable for the majority of participants at dosages between 12 and 21 grams per day. 26 individuals were able to complete the study. There were no significant abnormalities noted in blood tests, weight or vital signs of those in the study.

  • An analysis of muscle tissue in 33 people with amyotrophic lateral sclerosis (ALS), ALS with dementia or dementia alone, seven people with various other neuromuscular disorders, and 12 people without a neuromuscular disorder showed that a protein in muscle called Nogo-A was found in 20 out of 23 (87 percent) of the ALS patients. It was also found in five out of five people with ALS and dementia (100 percent) and three out of five with dementia alone (60 percent). Only one of the other 19 study participants, who had another neuromuscular disease, had this protein in their muscle tissue. Further study is needed to determine the significance of these results.

  • Rats with amyotrophic lateral sclerosis (ALS) caused by mutated SOD1 genes were helped by a compound called antisense that was designed to keep cells from following the erroneous genetic instructions that would produce an abnormal and toxic SOD1 protein. Onset of symptoms wasn’t affected, but early disease progression was slowed, and survival was extended by a month. The study supports the use of this type of therapy for SOD1-related ALS. A human clinical trial using antisense in familial ALS is planned, and toxicity studies are underway in preparation for that trial.

  • Cells called microglia taken from the nervous systems of mice with amyotrophic lateral sclerosis (ALS) caused by mutated SOD1 genes are more toxic when active than are microglia taken from healthy mice. Microglia are the cells that carry out an immune response in the nervous system. The study supports the hypothesis that activated microglia play a role in ALS.