BrainStorm, a developer of adult stem cell technologies for neurodegenerative diseases, has announced that it has a development for commercialisation of its stem cell therapy candidate, NurOwn. The company has developed a proprietary method for cryopreservation, or freezing, of cells, which will enable long-term storage, and production of repeat patient doses of NurOwn without the need for additional bone marrow aspirations.
“Cryopreservation will enable us to create a personalised NurOwn stem cell bank for each patient, for ongoing, repeat treatments,” said Adrian Harel, BrainStorm’s CEO. “We will be in a position to produce repeat doses of NurOwn by thawing and processing the patients’ cryopreserved cells, and transporting these doses to medical centers for immediate transplantation.”
Cryopreservation of hematopoeitic stem cells for clinical use has been routinely performed for fifteen years. Cryopreservation of mesenchymal stem cells for clinical use is a more recent phenomenon, and is typically achieved using vapor phase nitrogen. At extreme, sub-zero temperatures, any biological activity, including the biochemical reactions that would cause cell death, is effectively stopped.
According to Harel: “Our unique method of expansion and differentiation of mesenchymal stem cells requires its own cryopreservation methodology. The company has been focused on this aspect of product development in recent months in order to be fully prepared for repeat dosing of amyotrophic lateral sclerosis patients.”
BrainStorm is currently launching a Phase IIa combined treatment, dose-escalating dose trial of its NurOwn cell therapy candidate in amyotrophic lateral sclerosis 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 amyotrophic lateral sclerosis clinical development to the USA, pending Food and Drug Administration approval. The company has entered into a Memorandum of Understanding with the University of Massachusetts Medical School and Massachusetts General Hospital to begin amyotrophic lateral sclerosishuman clinical trials at these institutions.