BrainStorm Cell Therapeutics Inc., a leading developer of adult stem cell therapeutics for neurodegenerative diseases, announced that it has validated its cryopreservation process for NurOwn in preparation for the upcoming phase 3 clinical study in amyotrophic lateral sclerosis (ALS).  

The validation involved a comparison of NurOwn (MSC-NTF cells) derived from fresh mesenchymal stem cells (MSC) to those derived from cryopreserved MSC.  Company scientists were successful in showing that the MSC can be stored in the vapor phase of liquid nitrogen for prolonged periods of time while maintaining their characteristics.  The cryopreserved MSC are capable of differentiating into NurOwn, similar to the NurOwn derived from fresh MSC of the same patient/donor, prior to cryopreservation. This will allow the Company to provide repeated doses of autologous NurOwn from a single bone marrow aspirate in its upcoming multidose clinical trial. Cryopreservation will avoid the need for patients to undergo repeated bone marrow aspirations.

"Developing a robust and reproducible cryopreservation process has been an important goal for our R&D team as we prepare for the upcoming multi-dose phase 3 clinical study in ALS," commented BrainStorm's CEO Chaim Lebovits. "One of the challenges with autologous stem cell transplantations such as NurOwn has been that treating patients with multiple doses would have required them to undergo a number of aspiration procedures. Cryopreservation will streamline the process and help reduce the burden both on patients and on the treatment centers. Importantly, it will also result in lower cost of goods for NurOwn.  This development, along with the other improvements we have made to the NurOwn manufacturing process, is part of our strategy to continuously advance our stem cell platform technologies."  

BrainStorm Cell Therapeutics Inc. is a biotechnology company engaged in the development of first-of-its-kind adult stem cell therapies derived from autologous bone marrow cells for the treatment of neurodegenerative diseases.