BrainStorm Cell Therapeutics have received approval from the United States Food and Drug Administration to proceed with Phase 2 trials for their stem cell treatment for progressive multiple sclerosis (1). The treatment utilizes a proprietary autologous stem cell treatment, injecting patient-derived mesenchymal stem cells that secrete neurotrophic factors to stem the advance of disease (2).
Mesenchymal stem cells are commonly found in bone marrow, placenta, and adipocytes, and have been the subject of clinical trials in many conditions. BrainStorm alters the stem cells by growing them in proprietary conditions, giving the cells the ability to secrete neurotrophic factors (2). These cells, termed MSC-NTF cells, are able to treat neurodegenerative disease by delivering neurotrophic factors, such as BDNF and GDNF, to the site of degeneration.
Multiple sclerosis (MS) is characterized by the loss of myelin in the central nervous system due to an immune response. Myelin is responsible for insulating the axons of neurons, allowing for efficient signaling in the brain and spinal cord. When myelin is lost, neurons lose the ability to communicate with each other, resulting in myriad symptoms, from vision problems to sensory issues to muscle weakness. MS is thought to be caused by environmental cues in genetically predisposed people. MS can further be described as relapsing-remitting or progressive. Relapse-remitting MS is characterized by distinct attacks on the disease, followed by periods of time without additional symptoms of the disease. Progressive MS does not have the quiet periods, instead showing steady worsening of the disease. Additionally, many patients with relapsing-remitting MS eventually advance to progressive MS. Treatment options are limited for progressive MS, providing an impetus for finding new therapeutic options.
MSC-NTF cells have been tested in animal models of many neurodegenerative diseases and physical injuries, including ALS, Parkinson’s disease, MS, and peripheral nerve injury. These studies have suggested that MSC-NTF treatment may be a viable option for slowing the progression of neurodegenerative disease and being neuroprotective after injury compared to injecting untreated mesenchymal stem cells. These studies from the Offen and Melamed labs form the foundation for clinical trials to treat neurodegenerative diseases with the cells.
Phase 2 clinical trials for MSC-NTF cell treatment in progressive MS are expected to begin in early 2019. The MSC-NTF cells from the NurOwn treatment have already undergone Phase 1 clinical trials for treating amyotrophic lateral sclerosis (ALS), which is currently in Phase 3 clinical trials. Based on their other pre-clinical studies, it is possible that BrainStorm will also pursue clinical trials for other neurodegenerative diseases and potentially Autism Spectrum Disorder. The potential of NurOwn cells to protect against neuronal death and dysfunction could lead to relief for millions of people worldwide suffering from incurable diseases and is a promising advance in the field of regenerative medicine.