Novel drug slows progression of Lou Gehrig’s disease


London: A novel drug dexpramipexole may help slow symptom progression in the neurodegenerative disease amyotrophic lateral sclerosis (ALS), a new study has suggested.

It is believed that the drug, dexpramipexole, work by preventing dysfunction of mitochondria – the subcellular structures that provide most of a cell’s energy.


Initially developed by Knopp Biosciences of Pittsburgh, dexpramipexole appears to protect neurons from mitochondrial dysfunction.

Results of the first stage showed that receiving dexpramipexole appeared to slow the progression of symptoms measured both by the ALS Functional Rating Scale and by pulmonary capacity.

The protective effect was greatest in the 300 mg group – in whom symptom progression was approximately 30 percent slower than in the placebo group – and little effect was seen in those receiving 50 mg.

The second stage had similar results, with slower disease progression and a reduced risk of death in participants receiving the higher dosage.

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Results of the study will be published online in Nature Medicine.