Myotrophin Trials Disappointing for ALS Treatment

Kathleen Blanchard's picture

Researchers presented their findings this month on the effect of Myotrophin, a drug under study for ALS treatment. The results have proved disappointing, following impetus to resolve conflicting study results.

The drug, showed promise in North American trials, using the Appel ALS rating scale when studied ten years ago. A large European trial showed no benefit for patients suffering from ALS. The FDA felt that a third study was needed, and they provided recommendations to pursue the ALS treatment further.

There is no cure for ALS (Lou Gehrig 's disease), leading the National Institutes of Health to fund studies that would clarify whether Myotrophin, the brand name of IGF 1 (insulin-like growth factor 1) could halt rapid paralysis and death associated with ALS. Myotrophin is manufactured by the pharmaceutical company Cephalon (Frazer, Pennsylvania) and its partner Chiron (Emeryville, California).


Researchers studied 330 people with ALS. They injected either 0.05 mg/kg body weight of Myotrophin or a placebo in randomly selected patients who did not know which treatment they would receive, nor did the researchers. Muscle testing was scored, and survival rates were measured. ALS Functional Rating Scale-Revised (ALSFRS-R) revealed no differences between the two groups of patients.

ALS survival is typically two to five year as muscle wasting and paralysis results from the death of neurons that allow us to remain active. ALS does not affect mental capacity, making it a tragic disease as patients deteriorate. As neurons die, the brain can no longer initiate movement, and many ALS patients become completely paralyzed.

If you wish to learn more about ALS, visit their website. You can read stories of courage from people of all lifestyles who are afflicted with ALS. Lou Gehrig, who was diagnosed in 1939 brought much attention to ALS when he suddenly retired from baseball. Thus far, the only known cause of ALS is a mutation of the SOD1 gene that is toxic to neurons. SOD1 gene mutation only accounts for 20% of familial cases of ALS.

Treatment options are limited, and include primarily supportive treatment. Proper nutrition, and early intervention of breathing difficulty with assistive devices used at night are shown to enhance survival of ALS patients. The only drug currently approved for treatment is riluzole, a drug that only slightly improves ALS survival time.
Remaining hope for those who suffer from ALS lies in gene therapy and stem cell research. Lucie Bruijn, Ph.D., senior vice president of research and development, for the ALS Association, says, "These results are deeply disappointing to all of us in the ALS community."

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