BrainStorm Cell Therapeutics announced that it has successfully completed a 12-week repeat dose toxicity study with its NurOwn cells in mice. The company believes that the positive data from the study will support its efforts to obtain approval for a future repeat dose clinical study in ALS patients. The study was conducted at Harlan Israel’s laboratories, according to GLP (Good Laboratory Practice) standards of the FDA. The study protocol was approved by the Israeli Ministry of Health.
“We believe that repeat dosing is the key to the long-term clinical efficacy of NurOwn and we are anxious to begin testing in ALS patients. Our proprietary cryopreservation protocol will enable us to do so without having to perform repeat bone marrow aspirations for each dose,” commented Dr. Adrian Harel, BrainStorm’s CEO.
In the study, the animals received up to three intramuscular injections of NurOwn, produced from cryopreserved, or frozen, cells at concentrations 50–100 times the currently administered clinical trial dose. The treatment was well tolerated, and no adverse clinical effects were observed. Analyses included cytokine profile assays, clinical pathology, necropsy and macroscopic examination, as well as histopathological examinations.
BrainStorm is currently launching a Phase IIa combined treatment, dose-escalating trial of its NurOwn cell therapy candidate in ALS patients at the Hadassah Medical Center in Jerusalem. The company was recently fast-tracked by the Israeli Ministry of Health after reporting positive safety data for 12 patients in a Phase I/II trial.
BrainStorm is planning to expand its ALS clinical development to the United States in 2013, pending FDA approval. Towards that goal, the Company has entered into a Memorandum of Understanding with the University of Massachusetts Medical School and Massachusetts General Hospital to begin ALS clinical trials at these institutions.