Jose G. Merino, MD, MPhil


May 20, 2016


Stem cell transplants, brain-machine interface technology, stimulation of brain circuits to cure dementia, the neurologic consequence of emerging infectious diseases. These seemingly fantastic topics were the focus of the Hot Topics Plenary Session[1] at this year's American Academy of Neurology annual meeting.

Spinal Stem Cell Transplants for ALS

Amyotrophic lateral sclerosis (ALS) is characterized by degeneration of motor neurons leading to progressive weakness, disability, and death within 3-5 years after diagnosis. Therapeutic options for this condition are limited: Riluzole, the only agent for this condition, prolongs life by only a few months. But recent discoveries using stem cells to replace or promote survival of motor neurons raise new hopes for patients with ALS. Eva Feldman, the Russell N. DeJong professor of neurology at the University of Michigan, spoke about her group's experience with spinal cord stem cell transplants for ALS.

A phase 1 study[2] designed to assess the feasibility and safety of injecting spinal cord neural progenitor cells directly into the anterior horn of patients with ALS enrolled 15 patients. Most patients had unilateral injections, but in three they were bilateral, and in another three both the lumbar and cervical spine were injected. MRI and a frame and cannula designed for this trial were used to ensure that the stem cells were accurately injected, even as the cord moved with each respiration and heartbeat. The study showed that injections were safe and feasible and that they did not accelerate disease progression.

A second phase 2[3] trial was designed to assess the maximum safe dose (from 2 to 16 million cells.) Fifteen patients were enrolled, and follow-up is ongoing. One patient had worsening weakness for 3 months, but other adverse events were less serious. Among the 30 patients enrolled in these two trials, cervical and lumbar intraspinal transplants were safe. Patients tolerated up to 10 million cells. More than 70% of enrolled patients had better outcomes than similar historical controls (particularly preservation of functional vital capacity), and several participants with milder disease have had little or no significant progression for more than 700 days after surgery. A phase 3 study is being planned, but several questions remain unanswered: who should be treated (inclusion/exclusion criteria), the role of experimental disease biomarkers for selection or outcome assessment, the need for immunosuppression, appropriate primary outcome measures, and the feasibility and ethics of including a placebo control group.


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