Stem cell biotechnology company Brainstorm Cell Therapeutics Inc. announced positive results for its randomized, double-blind, placebo-controlled Phase II clinical trial of NurOwn in ALS, which enrolled 48 patients at 3 sites in the U.S. (see Oct 2015 news). The NurOwn technology is a candidate stem cell therapy that entails transplantation of autologous mesenchymal stem cells (MSCs) designed to secrete neurotrophic factors known to confer neuroprotection. The company reported that the primary objectives of safety and tolerability were met. Exploratory analysis of secondary outcomes measures of efficacy revealed improvements in the ALSFRS-R slope following treatment in a greater percentage of treated patients than controls, and a CSF biomarker analysis revealed an effect on neurotrophic factors and inflammatory markers. The company next plans to conduct a larger, pivotal study with repeat dosing to assess treatment efficacy.
Click here to read the company press release.