ROCKVILLE, Md., April 29, 2013 /PRNewswire/ -- Neuralstem, Inc. (NYSE MKT: CUR) announced that final data on the intraspinal delivery method employed in its NSI-566 Phase I trial to treat amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) was presented today at the American Association of Neurological Surgeons Annual Meeting. In a presentation called "Intraspinal Stem Cell Transplantation in ALS, A Phase I Trial: Cervical Microinjection Safety Outcomes," Jonathan Patrick Riley, MD, of the Department of Neurological Surgery at Emory University, in Atlanta, GA, presented data from all 18 procedures, in 15 patients, treated in Phase I. (Three of the patients returned to the trial for subsequent treatments in a different region of the spinal cord.) The study found that none of the patients experienced neurological worsening from injections into either the upper (cervical) or lower (lumbar) region of the back, nor was there any evidence of spinal cord injury. The cells also appeared to be safe with no evidence of toxicity. Researchers further noted that, even in the vulnerable ALS spinal cord, serial injections were well-tolerated. They concluded that the approach of starting in the lower region of the spine, where the potential risk to the patient was less, and then progressing upwards, might not be required in future trials, and that this intraspinal delivery technique is an option for treating other neurodegenerative and traumatic spinal cord disorders.
"This is an extremely important confirmation of the safety and tolerability of both the route of administration and our cells," said Karl Johe, PhD, Neuralstem Chairman and Chief Scientific Officer and a study author. "These patients are in many ways more fragile, and at greater risk from spinal surgery, than patients with other types of diseases or injuries where our cell therapy may be applicable. That our cells and method of delivery are safe in ALS patients bodes very well for expanding to other indications. We expect to commence our FDA-approved Phase I trial in chronic spinal cord injury later this year using the same methodology. We want to thank the surgeons at Emory, who developed these techniques, as well as the patients and their families who have taken part in the trial."
Neuralstem's patented technology enables the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells constitutively into mature, physiologically relevant human neurons and glia. Neuralstem completed an FDA-approved Phase I safety clinical trial for amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig's disease, in February 2013, and has received FDA approval to begin Phase II. Neuralstem has been awarded orphan status designation by the FDA for its ALS cell therapy.
In addition to ALS, the company is also targeting major central nervous system conditions with its NSI-566 cell therapy platform, including spinal cord injury, ischemic stroke and glioblastoma (brain cancer). The company received approval to commence a Phase I safety trial in chronic spinal cord injury in January 2013.
Neuralstem also has the ability to generate stable human neural stem cell lines suitable for the systematic screening of large chemical libraries. Through this proprietary screening technology, Neuralstem has discovered and patented compounds that may stimulate the brain's capacity to generate new neurons, possibly reversing the pathologies of some central nervous system conditions. The company is in the last cohort of a Phase Ib safety trial evaluating NSI-189, its first neurogenic small molecule compound, for the treatment of major depressive disorder (MDD). Additional indications could include traumatic brain injury (TBI), Alzheimer's disease, and post-traumatic stress disorder (PTSD).
Cautionary Statement Regarding Forward Looking Information
This news release may contain forward-looking statements made pursuant to the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that such forward-looking statements in this press release regarding potential applications of Neuralstem's technologies constitute forward-looking statements that involve risks and uncertainties, including, without limitation, risks inherent in the development and commercialization of potential products, uncertainty of clinical trial results or regulatory approvals or clearances, need for future capital, dependence upon collaborators and maintenance of our intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in Neuralstem's periodic reports, including the annual report on Form 10-K for the year ended December 31, 2012.