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.
“Today there are only two FDA-approved drugs used to treat ALS – riluzole, which extends life about 10 percent, and Nuedexta, which treats the emotional instability that characterizes ALS and other neurological disorders,” said Merit Cudkowicz, MD, director of the Massachusetts General Hospital (MGH) Neurology Clinical Trials Unit and ALS Center, lead author of the study.
“We need more therapies to slow, halt and ultimately reverse the course of disease and also therapies to treat the symptoms,” he noted.
Also known as Lou Gehrig’s disease, ALS is a progressive neurodegenerative disease affecting motor neurons in the brain and spinal cord.
Death of these nerve cells stops the transmission of neural impulses to muscle fibers, leading to weakness, paralysis and usually death from respiratory failure.
Mitochondrial dysfunction is among several factors believed to underlie nerve cell death.
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.
“Confirmation of these findings in the phase 3 clinical trial, which is currently ongoing, would give us even more confidence in this study design for phase 2 testing in ALS,” Cudkowicz said.
“We hope that adding this drug to the approved treatments for ALS would give patients a chance to stay healthier longer, with slower decline of function and increased survival,” he added.
Results of the study will be published online in Nature Medicine.