Research Roundup uodates as of September 2004:
Xaliproden fails in ALS, but hope remains
The experimental drug xaliproden has failed to fulfill the promise that earlier trials had hinted at, according to a report in the June issue of Amyotrophic Lateral Sclerosis and Other Motor Neuron Disorders.
The drug was developed by the multinational pharmaceutical company Sanofi-Synthelabo and tested in two large-scale multicenter trials in North America and Europe. It’s thought to increase the body’s production of its own neurotrophic (nerve-nourishing) substances.
At this time, Sanofi-Synthelabo is unlikely to pursue approval for the drug for ALS from regulatory agencies in the United States, according to Benjamin Brooks, director of the MDA/ALS Center at the University of Wisconsin-Madison and an investigator on this study.
In one study, in which participants took either a placebo (inert substance) or xaliproden, the group that got the higher dose of xaliproden lost respiratory function significantly more slowly.
In the other study, participants took riluzole with xaliproden. The group that took the lower dose of xaliproden with riluzole also lost respiratory capacity significantly more slowly.
However, trial participants who took xaliproden didn’t survive any longer than those given a placebo.
In a recent interview, Brooks said he and other investigators noted that xaliproden seemed to have an “alerting” effect that was detected in some of the psychological tests conducted during the study. It may also have had a positive effect on strength in the biceps, he said.
Presumably because of the alerting effect, Sanofi-Synthelabo has decided to test the drug in Alzheimer’s disease, in which patients lose cognitive abilities. Brooks speculates that if the drug shows benefit in that condition, the company may take a second look at it for ALS.
Some ALS patients may have a specific, as-yet-undefined genetic makeup that allows them to respond to xaliproden, he said.
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CoQ10 still on trial
According to the summer issue of a newsletter published by the Eleanor and Lou Gehrig MDA/ALS Center at Columbia University in New York, coenzyme Q10 has shown itself to be safe for ALS patients in a small, pilot trial.
The trial, in which 20 people with ALS received either coQ10 or a placebo, showed that the medication was well tolerated in general, although some participants experienced stomach upset or other gastrointestinal symptoms. The pilot trial wasn’t designed to show effectiveness.
CoQ10 is thought to improve the activity of the mitochondria, the energy “factories” inside cells, and also to combat damage from free radicals, toxic compounds produced by cellular metabolism. It’s been shown to be beneficial in mice with genetic ALS and in people with Parkinson’s disease.
The newsletter notes that Petra Kaufmann, assistant professor of neurology at Columbia, has received a grant from the National Institutes of Health to conduct a multicenter trial of coQ10 in ALS.
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Partners cope when speech is lost
Loss of the usual methods of communication is among the most devastating effects of ALS. But a study conducted over three years by Joan Murphy in the Department of Psychology at the University of Stirling in Scotland has shown that, at least between the person with ALS and a spouse, close friend or close relative (“communication partner”), communication is fairly well preserved despite the disease.
Fifteen people with ALS were involved in the study, for which results are reported in the June issue of ALS and Other Motor Neuron Disorders.
The study showed that the most important purpose of communication between the participant and his or her partner wasn’t to indicate needs or wants or to transfer information, but rather to develop and maintain social closeness. Their strategies for doing so varied among individuals and according to the stage of the disease.
For people in the early stages of ALS, simply repeating what had been said was usually enough to improve intelligibility. Some people also spelled or stressed key words as a help to the partner. Two people said breathing more deeply helped their speech, and some noted that they had to pace themselves and take rests before a period of talking.
Several partners used “topic cues” and context to understand their loved ones’ speech. Gestures, facial expressions and eye contact helped considerably.
Murphy concluded that communication “is a collaborative effort, and therapy needs to be provided with that in mind, not concentrating solely on the speech of the person with ALS.” She found “very few real communication breakdowns, as partners worked together to resolve any misunderstandings.”
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Harvard doctors study noninvasive EMG
The EMG, or electromyogram, remains a mainstay of diagnosis in ALS and other neuromuscular disorders. But the technique, which uses needle electrodes to measure electricity-like signals coming from inside muscles, is painful and distressing.
Now, doctors at Beth Israel Deaconess Medical Center in Boston (affiliated with Harvard University) are developing a new technique called electrical impedance myography, or EIM, which they say provides information similar to that gleaned from an EMG, without being as painful or invasive. The study is supported by the National Institutes of Health and the Harvard Center for Neurodegeneration and Repair.
The researchers are seeking adults who have ALS or any nerve or muscle disorder that causes weakness and who meet other study criteria. Participants must make periodic visits to Boston, and some financial compensation is offered.
For more information, contact physicians Seward Rutkove or Gregory Esper at (617) 667-3083 or firstname.lastname@example.org.
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