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.
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.
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.