Possible Lou Gehrig’s Treatment Fails To Outperform Placebo

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A potential treatment for Lou Gehrig’s disease has dissipated as researchers have found the potential new medication for the neurological condition is no more effective than a placebo.

The study, published in Lancet Neurology, focused on the safety and effectiveness of a drug called dexpramipexole in patients with Lou Gehrig’s disease, but the researchers noted those receiving the medication fared no better than those not receiving the medication.

Amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease, is a neurological disease which rapidly attacks nerve cells, or neurons, necessary for the voluntary control of muscles all across the body, including in the arms, legs, and face.

Treatments for the condition have been hard to come by, though researchers have recently focused in on the idea that a focus on mitochondria may unlock a new treatment. Mitochondria are important energy producers for neurons.

DexpramipexoleDexpramipexole was believed to improve the function of the mitochondria, thereby helping to treat Lou Gehrig’s. But the findings of the study from the Neurological Clinical Research Institute at Massachusetts General Hospital have dealt a heavy blow to the hypothesis. The groups administered the drug showed no significant difference in patients.

Even worse, DailyRx reports 8 percent of patients from the group given dexpramipexole developed neutropenia, a condition characterized by low levels of a specific type of white blood cell called neutrophils. Only 2 percent of the control group developed the condition.

“Dexpramipexole was generally well tolerated but did not differ from placebo on any prespecified efficacy endpoint measurement,” said the study. “Our trial can inform the design of future clinical research strategies in amyotrophic lateral sclerosis.”

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