Neuralstem, Inc. (CUR-NYSE Amex) announced on Monday of this week that it would begin enrolling the next group of patients into its ongoing Phase I ALS clinical trial. This trial seeks to demonstrate the safety of surgically injecting stem cells into the spinal cords of patients who are diagnosed with amyotrophic lateral sclerosis (ALS) or Lou Gehrig’s disease. To date, six non-ambulatory individuals have received stem cell injections in the lumbar spinal cord, and the trial’s Safety Monitoring Board has unanimously approved enrolling the next group of patients. The trial’s next patients will be ambulatory, which represent earlier stages of the disease than have been tested thus far. Ultimately, 18 ALS patients are expected to be enrolled in the Phase I trial, which is underway at Emory University in Atlanta, Georgia.
This Phase I trial is the first FDA-cleared ALS stem cell trial. CNN Health has recently covered the trial in a video segment posted here. Neuralstem is evaluating the safety, and potentially efficacy related to motor and other functions, of its patented process for growing neural stem cells to treat central nervous system (CNS) diseases. The neurodegenerative conditions that the Company targets are incurable and are usually associated with severe side effects, such as muscle atrophy, partial or complete paralysis, or death. Neuralstem’s proprietary Human Neural Stem Cell technology isolates stem cells from CNS tissue of the developing human brain and spinal cord and expands these cells in the laboratory for up to 60 doublings. By amplifying the natural ability of fetal stem cells to self-renew for long time periods, the Company creates billions of cells from each original stem cell, enabling commercial quantities from a sole donated tissue. Neuralstem has also submitted an application to the FDA to begin a Phase I safety trial in chronic spinal cord injury as well.
Peregrine Pharmaceuticals, Inc. (PPHM-NASDAQ) has also reported favorable clinical data this week. In Peregrine’s ongoing Phase II trial of Cotara®, a unique radioisotope-linked monoclonal antibody, the Company announced that 14 patients afflicted with relapsed glioblastoma multiforme (GBM), a severe brain cancer, were still alive an average of 86 weeks into the trial. In contrast, standard expected survival for relapsed GBM patients is typically only 24 weeks from the time of disease recurrence. Cotara® had been administered to patients as a single infusion, where the treatment travels directly into the tumor to destroy the tumor from the inside out, with minimal exposure to healthy tissue. Peregrine is working to complete enrollment in its Phase II trial by the end of 2010, with data anticipated by mid-2011.
The Company presented these interim data at the 2010 Congress of Neurological Surgeons Annual Meeting. The FDA has granted Cotara® Orphan Drug status and Fast Track designation for the treatment of GBM and anaplastic astrocytoma (AA).