Researchers have announced that an experimental drug showed promising results for a rare and inherited form of amyotrophic lateral sclerosis (ALS). The drug, referred to as ‘tofersen’, was evaluated based on safety and efficacy in both phase 1 and phase 2 clinical trials carried at Washington University School of Medicine in St. Louis, Massachusetts General Hospital in Boston.
Tofersen showed evidence in lowering the culprit protein concentration that leads to the symptoms of ALS. Such promising results, published July 9 in the New England Journal of Medicine, have launched the phase 3 clinical trial with hopes to continue evaluation.
"ALS is a devastating, incurable illness," said principal investigator Timothy M. Miller, MD, PhD, the David Clayson Professor of Neurology at Washington University and director of the ALS Center at the School of Medicine. "While this investigational drug is aimed at only a small percentage of people with ALS, the same approach -- blocking the production of specific proteins at the root of the illness -- may help people with other forms of the illness.
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"This trial indicated that tofersen shows evidence of safety that warrants further investigation and that the dose we used lowers clinical markers of disease. There are even some signs that it slowed clinical progression of ALS, although the study was not designed to evaluate effectiveness at treating the disease, so we can't say anything definitive. Overall, the results are just what we hoped for, and a phase 3 trial is currently underway."
Source: Science Daily