The company announced that, after reviewing the safety data from the first nine patients, the trial's Safety Monitoring Board has unanimously approved moving to the last group of ALS patients in this part of the safety trial. These next three patients, all of whom are ambulatory, will each receive ten injections, bilaterally, in the lumbar spinal cord. After this cohort, the FDA will review the trial data to date before approving it to move into the final cohort of patients, who will receive injections in the cervical region of the spinal cord.
"We are delighted that we've advanced to this final cohort of patients in the first part of the safety trial," commented Dr. Eva Feldman, PhD, MD, Principal Investigator of the trial and a consultant to Neuralstem. Dr. Feldman is Director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of Michigan Health System. "We have been pleased with the smooth progress of the trial thus far, from non-ambulatory to ambulatory patients who have an earlier stage of the disease."
"We are greatly encouraged by the SMB's approval to advance the trial to the final three patients to receive full lumbar injections," said Karl Johe, PhD, Chief Scientific Officer and Chairman of Neuralstem's Board of Directors. "The trial has progressed as expected, and we are deeply indebted to the patients and families who have taken part."
About The Trial
The Phase I trial to evaluate the safety of Neuralstem's spinal cord stem cells in the treatment of ALS, the first FDA-approved ALS stem cell trial, has been underway since January, 2010. The trial plans to enroll up to 18 ALS patients, who will be examined at regular intervals post-surgery.
All of the first six patients treated in the trial were non-ambulatory. Of these, the first three received five injections each, unilaterally, in the lumbar region of the spinal cord. The next three patients received ten injections each, bilaterally in the lumbar region. All remaining trial patients are ambulatory, and therefore represent earlier stages of disease progression. Of the ambulatory group, the first three patients received five injections each, unilaterally, in the lumbar region. The next three, which have just been approved, will receive ten injections each, bilaterally, in the lumbar region. After the required FDA approval, the final six patients in the trial will receive injections in the cervical region.
While the trial is evaluating only the safety of the cells and procedure, it includes exploratory endpoints including attenuation of motor function loss, maintenance of respiratory capacity, and stabilization of patients along the ALS functional rating scale.
The Emory ALS Center has posted the relevant trial information for patients on its website at www.neurology.emory.edu/ALS/Stem%20Cell.html.
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 is in a FDA-approved Phase I safety clinical trial for amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig's disease, which has been rewarded Orphan Drug Status by the FDA.
In addition to ALS, the company is also targeting major central nervous system diseases, including traumatic spinal cord injury, ischemic spastic paraplegia, and Huntington's disease. The company has also submitted an IND (Investigational New Drug) application to the FDA for a Phase I safety trial in chronic spinal cord injury.
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 has been approved to commence a Phase Ia safety trial evaluating NSI-189, its first small molecule compound, for the treatment of major depression. Additional indications could include schizophrenia, Alzheimer's disease, traumatic brain injury, post-traumatic stress syndrome, and stroke.
For more information, please go to www.neuralstem.com.
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, 2009, and in its quarterly report on Form 10-Q for the period ended September 30, 2010.