Development of stem cell therapy to treat amyotrophic lateral sclerosis (ALS) continued with two studies in mice, one by an Israeli biotech company and one by an Italian research team.
BrainStorm Cell Therapeutics has released data from a recently completed toxicity study showing that repeat doses of the company's experimental NurOwn stem cell therapy for ALS were safe and well-tolerated in mice.
To create NurOwn cells, scientists harvest stem cells from a person's bone marrow, then reprogram them to become specialized neuron support cells. The NurOwn cells are then transplanted back into the person from whom they originated.
"We believe repeat dosing is the key to the long-term clinical efficacy of NurOwn, and we are anxious to begin testing in ALS patients," BrainStorm CEO Adrian Harel said in a company press release.
Mice in the study received as many as three intramuscular injections of NurOwn at concentrations 50 to 100 times the current dose set for clinical trial participants. The treatment was well-tolerated and investigators noted no adverse clinical effects.
Brainstorm currently is launching a phase 2a combined treatment, dose-escalating trial of NurOwn in people with ALS at the Hadassah Medical Center in Jerusalem. According to a BrainStorm press release issued Jan. 7, 2013, approval for the acceleration was based on the promising early safety results of the treatment in the first 12 (of an expected total of 24) study participants in the company’s phase 1-2 trial at Hadassah.
Pending FDA approval, the company plans to add U.S. trial sites to the ongoing NurOwn clinical trial in 2013. Planned trial site locations include the University of Massachusetts Medical School in Worcester and Massachusetts General Hospital in Boston.
Mice with an ALS-like disease that were treated with injections of human neural stem cells lived an average of 20 days longer and had improved neuromuscular function. Investigators confirmed that after injection the stem cells migrated to the spinal cords of the mice, where they matured and multiplied.
The cells used in the study were derived from human induced pluripotent stem cells (iPSCs) — adult cells that have been genetically reprogrammed back to a stem-cell-like state, where they can then be coaxed along a desired path of development to become a specific cell type.
"Stem cell transplants may represent a promising avenue for effective cell-based treatment for ALS and other neurodegenerative diseases," study author Stefania Corti, at the University of Milan in Italy, said in a press release.
Complete study results will be presented at the American Academy of Neurology’s 65th Annual Meeting in San Diego, March 16-23, 2013.