- Post-hoc comparison of combined cohorts from Phase 1 and 2 trials vs. historical controls revealed statistically significant difference in scores in functional status and in a composite statistic combining survival and functional status in subjects receiving human spinal cord-derived neural stem cells
- Study provides validation for additional trials to evaluate the benefit of neural stem cells in ALS patients
GERMANTOWN, Md., May 03, 2018 (GLOBE NEWSWIRE) -- Neuralstem, Inc. (Nasdaq:CUR), a biopharmaceutical company developing novel treatments for nervous system diseases, today announced the results from a study published in the Annals of Clinical and Translational Neurology that support the potential of transplanted human spinal cord-derived neural stem cells (HSSC) to stabilize functioning of amyotrophic lateral sclerosis (ALS) patients. The publication can be found here.
The manuscript entitled “Long-term Phase 1/2 Intraspinal Stem Cell Transplantation Outcomes in Amyotrophic Lateral Sclerosis” had as senior author Study Principal Investigator Eva Feldman, MD, PhD, Russell N. DeJong Professor of Neurology at the University of Michigan. It evaluated the impact of HSSC transplantation on functional outcomes, as measured using the ALSFRS-R scale, and on a composite statistic that combined functional and survival outcomes. Results were evaluated against matched controls derived from two historical datasets and showed significantly better ALSFRS-R scores at 24 months, as well as the composite functional/survival score in subjects receiving HSSC. The ALS Functional Rating Scale-Revised (ALSFRS-R) is a validated questionnaire that measures physical function in performing activities of daily living (ADLs).
“We are looking forward to completing a larger Phase 2b/3 multicenter study,” said Dr. Feldman.
“These data are encouraging and indicate that human spinal cord-derived neural stem cells may provide functional benefit to ALS patients,” said Rich Daly, Neuralstem’s Chief Executive Officer. “We are grateful to the subjects who have participated in these trials and to the clinicians and caregivers involved in performing them. These long-term follow-up data are extremely valuable to us in designing the Company’s Phase 2b/3 clinical study to further evaluate NSI-566, Neuralstem’s cell therapy candidate, in this patient population.”
The Phase 1 and 2 open label dose escalation studies were carried out at the University of Michigan in Ann Arbor, Emory University in Atlanta, and Harvard Medical School in Boston. They enrolled a total of 30 subjects and had as their primary objective to evaluate the safety of intraspinal transplantation of HSSC, while also measuring survival and functional endpoints. Previous publications from the investigators have concluded that the treatment is safe.
About Amyotrophic Lateral Sclerosis (ALS)
ALS is a progressive disease that affects nerve cells, or neurons, in the brain and the spinal cord, leading to degeneration and eventual atrophy of the surrounding muscles. As the condition worsens, motor neurons die, and the brain can no longer control the affected muscles. In time, this causes the loss of patients' ability to speak, eat, move and breathe, eventually resulting in death. According to the ALS Association, it is estimated that more than 6,000 people in the U.S. are diagnosed with ALS each year, amounting to 15 new cases per day, totaling approximately 20,000 Americans living with the disease at any given time. There is currently no cure or treatment that that halts or reverses the progression of the disease.
Neuralstem is a clinical-stage biopharmaceutical company developing novel treatments for nervous system diseases of high unmet medical need. The Company has two lead development candidates:
- NSI-189, is a small molecule in clinical development for major depressive disorder and in preclinical development for Angelman syndrome, irradiation-induced cognitive impairment, Type 1 and Type 2 diabetes, and stroke.
- NSI-566 is a stem cell therapy being tested for treatment of paralysis in stroke, Amyotrophic Lateral Sclerosis (ALS) and chronic spinal cord injury (cSCI).
Neuralstem’s diversified portfolio of product candidates is based on its proprietary neural stem cell technology.
Cautionary Statement Regarding Forward Looking Information
This news release contains “forward-looking statements” made pursuant to the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements relate to future, not past, events and may often be identified by words such as “expect,” “anticipate,” “intend,” “plan,” “believe,” “seek” or “will.” Forward-looking statements by their nature address matters that are, to different degrees, uncertain. Specific risks and uncertainties that could cause our actual results to differ materially from those expressed in our forward-looking statements include 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, 2017 filed with the Securities and Exchange Commission (SEC), and in other reports filed with the SEC. We do not assume any obligation to update any forward-looking statements.
Argot Partners (Investor Relations)