Meeting Coverage
ALS Stem Cell Therapy: Too Soon to Tell
http://www.medpagetoday.com/MeetingCoverage/ANA/42288
Published: Oct 15, 2013
By John Gever, Deputy Managing Editor, MedPage Today
http://www.medpagetoday.com/MeetingCoverage/ANA/42288
NEW ORLEANS — Phase I results of a neural stem cell treatment for amyotrophic lateral sclerosis showed hints of promise but solid evidence of efficacy will have to wait for the next round of tests, a researcher said here.
Among 15 patients receiving stem cell injections into their spinal cords in the dose escalation study, nine have survived and the three receiving the most extensive injections showed stabilization of functional scores after almost 2 years of follow-up, said Eva Feldman, MD, PhD, of the University of Michigan.
The treatment also appeared to be safe, with no evidence of spinal cord damage resulting from the procedures or other major complications, she told attendees at the annual meeting of the American Neurological Association, of which she is president.
But “whether there is a benefit, we simply don’t know” from this small trial, said Feldman.
Phase II studies testing larger stem cell doses have recently begun, with the first three patients having undergone procedures at Feldman’s clinic and at Emory University in Atlanta. Her report here was the first to provide full phase I results, which have also been submitted for journal publication, she said.
There is currently no disease-modifying treatment available for the neurodegenerative disease that affects motor neurons in the spinal cord. A few symptomatic therapies are used but they are only modestly effective.
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These cells are capable of self-renewal and also differentiating into both glial and neuronal progenitor cells. The former differentiate further into astrocytes and oligodendrocytes, while the latter may develop into functional neurons.
Extensive preclinical work in rodents and, later, pigs established that preservation of spinal cord function after mechanical and/or chemical injury could be achieved with the stem cells, and that the spinal cord injections could be done safely, Feldman said.
She said the exact mechanism of benefit remains unclear, but the animal studies suggested that the stem cells act to protect and preserve endangered nerve synapses in the spinal cord. “It’s definitely not replacement” of destroyed synapses, she said.
The human tests began in 2010 with three patients with advanced nonambulatory ALS, who received injections on one side of the lumbar spine. A second nonambulatory cohort received bilateral lumbar injections.
Feldman and colleagues then advanced the trial into ambulatory patients, with three-patient cohorts receiving unilateral lumbar and cervical injections.
The final cohort of three patients received bilateral lumbar injections followed about 18 months later by unilateral cervical injections.
Except in that last cohort, efficacy outcomes were mixed. Six of the 12 patients in the earlier cohorts showed relatively steep declines in revised ALS Functional Rating Scale (ALSFRS-R) scores, while declines were slower in the others. But scores remained essentially the same as at baseline — in the range of 32 to 45 points — in the three patients receiving both lumbar and cervical injections.
One theme emerging from the earlier cohorts was that patients with bulbar signs “did not do well,” Feldman said.
Autopsy findings in phase I participants who died indicated that the stem cells had survived in these patients and that there was no suggestion that the therapy had played a role in their deaths.
In the phase II study, the first groups of patients received 200,000 cells per injection, with five injections per patient along the spinal cord on each side — a total of 2 million cells. Subsequent cohorts will receive 10 injections per side and with more cells per injection, reaching an eventual total dose of 8 million cells.