A Small Step, Not a Giant Leap, in Stem Cell Therapy for ALS

Advances in stem cell biology have fueled a growing interest in the use of stem cells as possible therapeutics for amyotrophic lateral sclerosis (ALS). Now, researchers describe the results of early-phase clinical trials of mesenchymal stem cells (MSCs) that were induced in vitro to secrete neurotrophic factors prior to transplantation (MSC-NTF cells).

In the first of these two manufacturer-funded, open-label studies, MSC-NTF cells were administered intramuscularly (IM) to 6 patients with relatively early disease and intrathecally (IT) to 6 patients with more-advanced disease. Following an interim safety analysis, the second study included an additional 14 patients with relatively early disease randomized to receive varying dosages of MSC-NT…