Preliminary Stem Cell Clinical Trials in ALS Patients Show Promise; Further Research Warranted

May 3, 2018

ANN ARBOR — Transplanted human spinal cord-derived neural stem cells (HSSC) showed a potential to stabilize the functioning of amyotrophic lateral sclerosis (ALS) patients, according to a study that was published today in the Annals of Clinical and Translational Neurology. Stephen A. Goutman, MD, Clinical Director of the ALS Center of Excellence at Michigan Medicine, is the first author of the manuscript entitled “Long-term Phase 1/2 Intraspinal Stem Cell Transplantation Outcomes in Amyotrophic Lateral Sclerosis.” Program for Neurology Research & Discovery (PNR&D) Director Eva L. Feldman, MD, PhD, is the senior author and study principal investigator. Read the complete article here.

Dr. Eva Feldman with Dr. Stephen Goutman.

Stem cell transplantation is a promising therapeutic strategy for the treatment of ALS. The authors evaluated a subset of limb-onset and ambulatory participants who enrolled in a Phase 1 study at Emory University and a Phase 2 study at Emory, University of Michigan, and Massachusetts General Hospital. The overall goal of this analysis was to assess the long-term outcomes of participants in these studies and help determine if subjects may have received any benefit from the stem cells to warrant further trials. The investigators found no new serious adverse events using the long-term dataset.

“We are encouraged to see patients with limb onset ALS who enrolled in the Phase 1 and 2 studies had no additional adverse events during long-term follow up and seemed to fare better than matched subjects from a historical database,” said Dr. Goutman. “Importantly though, we only tested 24 patients and this therapy requires a much larger trial before we know its effectiveness.”

The authors also found when using a measure that combined survival and function, as defined by the revised ALS functional rating scale (ALSFRS-R), that this subgroup of patients had better outcomes when compared to matched historical controls in the PRO-ACT dataset, but had similar outcomes when compared to matched controls from the ceftriaxone study. The Phase 1 and 2 studies of HSSC transplantation for the treatment of ALS were designed to address safety and not prove that this therapy is effective, and therefore the fact that any signal was seen is promising.

“We are looking forward to completing a larger FDA-approved multicenter study,” said Dr. Feldman. “Through further research we hope to show that stem cells can offer ALS patients a functional benefit.”

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 the ALS Center of Excellence at Michigan Medicine (ACEMM): The ALS Center of Excellence at Michigan Medicine is comprised of an active basic science, translational, and clinical research program and the multidisciplinary ALS clinic. This structure engages collaboration between physicians, basic scientists, nurses, ancillary providers, and research coordinators all working towards better treatments, an understanding of why a person develops ALS, and ultimately a cure for ALS. To learn more about the program, visit http://www.umich-als.org/.

About ALS: Amyotrophic lateral sclerosis (ALS), which is commonly known as Lou Gehrig’s disease, is a progressive fatal neurodegenerative disease that leads to the death of nerve cells in the brain and spinal cord. This in turns causes progressive weakness of voluntary skeletal muscle often leading to death in 2 to 4 years. Current therapies only minimally slow disease and new therapeutic options are critically needed.

About Neuralstem: Neuralstem is a clinical-stage biopharmaceutical company developing novel treatments for nervous system diseases of high-unmet medical need. The company supplied the stem cells for the ALS clinical trials.