ALS Research Briefs

by Margaret Wahl on Fri, 2010-12-10 15:47

Updates on ALS clinical trials of drugs that regulate the immune system and stimulate VEGF

Immune system regulator shows safety in ALS

Neuraltus Pharmaceuticals announced Nov. 30, 2010, that its experimental drug NP001 was safe and well-tolerated at four different dose levels when given as a single intravenous treatment to people with ALS. There was also a statistically significant improvement in blood levels of a biological marker (biomarker) thought to be involved in ALS disease progression.

NP001 is a small molecule designed to regulate the activation of immune system cells called macrophages.

See Neuraltus Pharmaceuticals Reports Clinical Results from Phase 1 NP001 Study in the Treatment of Amyotrophic Lateral Sclerosis (ALS) and The Flip of a Switch.

VEGF-stimulating drug shows safety, hints of efficacy

Sangamo BioSciences announced Nov. 17, 2010, that its experimental ALS drug SB509, designed to increase production of vascular endothelial growth factor A (VEGF-A), was safe and well-tolerated, and showed hints of efficacy in a phase 2 trial in people with ALS.

See Sangamo BioSciences Announces Presentation of Phase 2 Data in Amyotrophic Lateral Sclerosis (ALS) at Society for Neuroscience Meeting and Two ALS Drugs Show Early Promise.

About clinical trials

A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur. MDA has no ability to influence who is chosen to participate in a clinical trial. To learn more, see Understanding Clinical Trials and Being a Co-Adventurer, which is about neuromuscular disease clinical trials.

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