Ralph Kern, MD, MHSc: The State of Therapies for ALS

“We’re hoping regenerative medicine becomes part of ALS therapy. That’s our hope.”

At the American Academy of Neurology's (AAN) 70th Annual Meeting in Los Angeles, California, NeurologyLive's sister publication MD Mag sat with Ralph Kern, MD, MHSc, to discuss the current state of therapies for amyotrophic lateral sclerosis (ALS). The chief medical officer and chief operating officer of Brainstorm Cell Therapeutics noted that while there are only 2 currently US Food and Drug Administration (FDA) approved therapies, neither of them achieves the ultimate goal of treating ALS—to modify the disease. He spoke about the excitement surrounding the studies of new biologics in this area and expressed the hope that some might be successful.

Then, Kern discussed Brainstorm’s current work with cell-based therapy, utilizing it as a mechanism of delivery for neurotrophic factors (NTFs) and cytokines that alter the motor-neurons of the patient. He also touched on some of the lack of success the field has had in treating ALS, including the specific genetic therapies designed to address genetic mechanisms in the SOD1 or C9ORF72 genes, and how they have not held up to expectations just yet. He also spoke about the general lack of success with pure anti-inflammatory treatments and the failure of target engagement with single neurotropic therapies.

After, Kern spoke about his hope for the successful development of regenerative therapies, before he went on to explain the mechanism of action for Brainstorm’s cell-based therapies that are in development. He also touched briefly on some aspects of the design ofBrainstorm's ongoing phase III study, which is currently enrolling with a target goal of 200 patients. The trial will evaluate the safety and efficacy of repeated administration of MSC-NTF cells (NurOwn) therapy—based on transplantation of autologous bone marrow-derived mesenchymal stromal cells (MSC), enriched from the patient's own bone marrow, propagated ex vivo, and induced to secrete NTFs. The autologous MSC-NTF cells are back-transplanted into the patient intrathecally via a standard lumbar puncture, in which neurons and glial cells are anticipated to take up the NTFs secreted by the transplanted cells.