ALS Research Briefs

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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