November 20, 2003

MILAN MEETING HIGHLIGHTS

Progress in ALS research was the topic at the 11th annual meeting of the International Alliance of ALS/MND (Motor Neurone Disease) Associations and the 14th International Symposium on ALS/MND, held in Milan, Italy, Nov. 13 through 19.

Among the highlights presented at the meeting of internationally recognized ALS experts were reports indicating that the dietary supplement creatine, when taken at 5 grams a day for six months, shows no benefit in ALS.

It was also announced that a set of protein abnormalities found in the fluid surrounding the brain and spinal cord is specific to ALS and probably can be used for diagnosis and tracking of disease progression.

The first finding came from a trial funded by MDA, Avicena (manufacturer of the NEOtine brand of creatine) and other organizations. In the trial 104 participants with ALS were assigned to take either creatine or a placebo.

Creatine was found to be safe in ALS, but the investigators found no significant differences between participants on creatine and those on the placebo in arm strength (maximum voluntary contraction), function (using the ALS Functional Rating Scale) or numbers of preserved motor units. (Each nerve cell and the muscle fibers to which it sends signals is a motor unit.)

There were 48 serious adverse events during the trial; none were thought to be due to creatine. Eight deaths occurred, six in the placebo group and two in the creatine group, a difference that wasn’t statistically significant.

Neurologist and principal investigator Jeremy Shefner, who directs the MDA/ALS Center at SUNY Upstate Medical Center in Syracuse, N.Y., said he was disappointed in the results of this study. He added that it remains possible that creatine at higher doses or for a longer period or in combination with other agents might yet have a role in ALS treatment.

Robert Bowser of the University of Pittsburgh School of Medicine presented the finding of abnormalities in the fluid surrounding the brain and spinal cord (cerebrospinal fluid, or CSF) in ALS patients. Bowser worked with a team from Massachusetts General Hospital in Boston that included neurologist Merit Cudkowicz, a longtime MDA-associated physician and research grantee.

The identification of a “panel” of abnormalities in the CSF that appears to be specific to ALS bodes well for both early diagnosis of the disease and better evaluation of experimental treatments in clinical trials.

Trial results are now evaluated on the basis of how long patients survive and how rapidly they lose strength. These types of measurements require months to years and large numbers of patients to show significant differences between the treated and placebo (untreated) groups.

The current findings are based on the differences in the CSF between 25 people with ALS and 35 people without ALS. Bowser said more people would have to be tested to see how the findings hold up. The panel of protein abnormalities would be studied to see how it changes during disease progression, he added.