What is it about?
NP001, a regulator of inflammation, failed to delay disease in a placebo controlled clinical trial in ALS patients. However, a large subset of patients didn't progress over 6 months while taking NP001. These patients were between the ages of 40-65 , had a mildly elevated blood marker of inflammation, and represented the majority (74%) of patients in the trials. When this subset restriction was applied, to patients from 2 NP001 six month trials, 35% of NP001 recipients had stopped progressing and all showed a 50% improvement in breathing function. No significant side effects were noted.
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Why is it important?
ALS is a heterogeneous disease and it's important to identify subsets that respond to specific therapies. For example, no one drug works on all cancer patients, why should one work in all ALS patients? In order to best serve patients with ALS it's important not to discount drugs that "fail" in clinical studies unless a subset analysis proves no beneficial activity. In this paper, a large subset (74%) of ALS patients identified retrospectively benefited from receiving NP001, an immune regulator. Most importantly is the finding that NP001 treatment is the only drug ever shown in any type of analysis to improve breathing function as compared to controls.
Perspectives
ALS is such a devastating disease that it's important not to quit pursuit of an effective therapy, even if it is effective in only a subset of patients.
Michael McGrath
University of California San Francisco
Read the Original
This page is a summary of: Phase
2B
randomized controlled trial of
NP001
in amyotrophic lateral sclerosis: pre‐specified and post‐hoc analyses, Muscle & Nerve, January 2022, Wiley,
DOI: 10.1002/mus.27511.
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