Many in the biotech community have held that some of the greatest promise for regenerative medicine therapies may rest in their uses for neurodegenerative disorders, diseases such as Alzheimer's, Huntington's, Parkinson's and Amyotrophic Lateral Sclerosis for which traditional therapies provide no real solutions. That's not to discount the potential benefits of stem cell therapies across a wide array of diseases and conditions, such as stroke and heart disease; only to highlight that neurodegenerative disorders represent a tremendous area of unmet medical need for which regenerative medicine could make a clinical difference. Research on stem cells, which was fought tooth-and-nail by too many for too long, is starting to unwind some of the mysteries and landmarks in cell development, which can be integral to viable therapies in the future. BrainStorm Cell Therapeutics Inc. (OTCQB:BCLI) is hoping to deliver the first stem cell therapeutic for Amyotrophic Lateral Sclerosis (ALS, or "
Lou Gehrig'sDisease" as it's often called), a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord and one of the most common neuromuscular diseases in the world. ALS is a painful disease to watch someone suffer, and eventually die from, as motor neurons rapidly degenerate, halting communication with muscles that eventually atrophy, leaving the afflicted with difficulty talking, swallowing or breathing and sometimes totally paralyzed before death. While physical functions are destroyed, cognitive function and all the senses often remain unaffected. BrainStorm is building its clinical database showing the safety and efficacy of its NurOwn cell therapy in early human trials. In the NurOwn therapy, autologous, bone marrow-derived, mesenchymal stem cells are removed and differentiated into neuron-supporting cells and re-injected into the body. These cells, officially called MSC-NTF (Mesenchymal Stem Cells Secreting Neurotrophic Factors), secrete elevated levels of nerve growth factor for inhibiting damage to motor neurons, while supporting motor neuron growth and nerve-muscle communication. NurOwn as a novel therapy to ALS has been studied in a Phase 1/2 trial and a Phase 2a trial at Hadassah Medical Centerin Jerusalem, Israel. In both trials, no serious adverse events were reported in patients receiving NurOwn therapy. Further, there was a statistically significant improvement in neurological function as measured by the ALS Functional Rating Scale and Forced Vital Capacity, a measurement of respiratory function, in patients treated with the BrainStorm therapy. The data from the trials has been presented at multiple conferences, including most recently at the Joint Congress of European Neurologythis week. Shares of BCLI have more than doubled in 2014 on the data, but investors – and the scientific community – are looking for the treatment population size of the clinical research to increase to lend validity to the data. Ultimately, investors like to see the studies conducted in the States as well, both things are now happening. Only 26 ALS patients were treated in total in the first two trials, but that number is going to be almost doubled in a Phase 2 trial that will be conducted at leading institutions in the U.S. On April 28, the FDAgave BrainStorm the go ahead to commence the trial of NurOwn in AML patients being conducted at Massachusetts General Hospitalin Boston, the University of Massachusetts Memorial Hospitalin Worcesterand Mayo Clinicin Rochester, Minnesota. Dana-Farber Cancer Institute'sConnell O'Reilly Cell Manipulation Core Facility is manufacturing the NurOwn cells for the sites in Massachusetts. On Friday, BrainStorm said that the first patient has been enrolled in the Phase 2 trial at Mass General. From an industry perspective, this enrollment represents the first ever mid-stage trial authorized by the FDAusing stem cells to treat ALS. With that in mind, it's somewhat surprising that the stock is tepidly lower today, but there was somewhat of a buying spree in the six days prior to Wednesday that lifted shares by 29% on increased volume. As the trial progresses, it will be interesting to see how Wall Streetreacts in anticipation of results from this micro-cap for a disease that has been notoriously hard to treat. Proper due diligence is, as always, encouraged.