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Neuralstem Cell Therapy for ALS

  • Product status: Phase I Safety Trial in final phase: cervical transplantations
  • Mechanism of Action: Rebuilding neural circuitry
  • Route of Administration: Direct injections into the spinal cord

Neuralstem is seeking to treat the symptoms of ALS via transplantation of its human spinal cord stem cells (HSSCs) directly into the gray matter of the patient’s spinal cord. In ALS, motor neurons die, leading to paralysis. In preclinical animal work, Neuralstem cells both made synaptic contact with the host motor neurons and expressed neurotrophic growth factors, which are protective of cells.  View published papers here: 1, 2, 3.

Neuralstem initiated the first FDA-approved stem cell trial for ALS in January 2010, at Emory University. This Phase I safety trial, to evaluate the safety of the cells and surgical technique, is designed to enroll up to 18 patients. The Principal Investigator is Eva Feldman, MD, PhD, Director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of Michigan Health System. The Site Investigator is Jonathan Glass, MD, Professor of Neurology, Emory School of Medicine and Director of the Emory ALS Center. The trial was awarded an Orphan Drug Designation by the FDA in February 2011.

In humans, Neuralstem expects that the transplanted cells will:

  • GRAFT permanently into the region where they were transplanted
  • REBUILD circuitry with the patient motor neurons
  • PROTECT patient neurons from further ravages of the disease

In a review of the safety data from the initial nine patients, Neuralstem cells were deemed to be safe, with no adverse reactions reported believed to be related to cells or surgical technique.

Neuralstem ALS Trial

This Phase I safety trial is designed to enroll up to 18 patients. The trial began with 12 late- to mid-stage patients who received a series of injections in the L2-L4 lumbar region. The first six patients were all non-ambulatory with permanent paralysis. Of these, the first three patients (Cohort A1) were treated with five unilateral cell injections, while the next three patients (Cohort A2) received ten bilateral injections in the same region. The trial then progressed to patients who were ambulatory. The first three of these (Cohort B) received five unilateral injections. The next three patients (Cohort C) received ten bilateral injections in the same lumbar region.

The trial was designed to halt for a comprehensive safety data review by the FDA and the trial’s safety monitoring board (SMB) before beginning transplantation to the cervical spinal cord of ambulatory patients. Principal Investigator Eva Feldman, MD, PhD presented primary and secondary endpoint data on the first 12 patients at the American Neurological Association’s annual meeting in late September 2011. Neuralstem expects to move into this phase of the trial in the fall of 2011.

Neuralstem received approval from the FDA to move into the cervical (upper back) stage of the trial in the fall of 2011. The first of six patients in the cervical cohorts to receive stem cells was treated on November 18, 2011, which marked the first FDA-approved intraspinal surgical transplantation of stem cells into the cervical region. The entire 18-patient trial concludes six months after the final surgery.

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Amyotrophic Lateral Sclerosis, also known as ALS or Lou Gehrig’s disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, leading to complete paralysis, and eventually, death. According to the ALS Association, as many as 30,000 Americans have the disease, and about 5,600 people in the U.S. are diagnosed with ALS each year.

There is no cure.

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