Honolulu: So Far, So Good in Stem Cell Safety Study for ALS

Series - American Academy of Neurology: 2011 Annual Meeting:

Injecting neural stem cells into the lumbar spinal cord is a safe procedure for people with amyotrophic lateral sclerosis, according to interim data presented at the American Academy of Neurology 63rd Annual Meeting this week in Honolulu, Hawaii. Researchers working with Neuralstem, Inc., Rockville, Maryland, presented data on the first nine people to receive the treatment, all of whom are still alive four to 15 months after the surgery. The scientists, who have performed three more surgeries since assembling the interim report, plan to continue the Phase 1 study with 18 total participants.

Neuralstem has developed a stable line of neural stem cells from donated fetal tissue. The ALS trial is the company’s first human study; they are also planning to try the cells for spinal cord injury and stroke. The hope, said Neuralstem chief operating officer Karl Johe, is that the stem cells will prolong survival of motor neurons. “The graft will reach out, form synapses with the remaining host motor neurons, and protect them from the further ravages of the disease,” he suggested. That remains to be determined.

The trial, led by Eva Feldman of the University of Michigan combines the neural stem cell line with new surgical technology and a new method to measure ALS progression.

The researchers wanted to deliver the cells directly into the lumbar spinal cord. To prevent movement during the two minutes it takes to complete the injection, Nicholas Boulis, a neurosurgeon at Emory University, developed a device to mount the needle directly on the person’s body. “When the body moves, the whole platform moves with the body,” Johe said.

Although it is too early in this safety study of people with advanced disease to look for benefits from the treatment, the researchers are also using a new method, electrical impedance myography (see ARF related news story), to follow disease progression.

The results so far indicate that the cells cause no harm. The only adverse events were related to the surgery itself, or the immunosuppressive drugs the doctors prescribed to prevent rejection of the cells. Next, the scientists plan to pursue cervical spinal cord injections, which are riskier because any abnormal stem cell growth could push up against the spinal cord and interfere with nerve signals needed for breathing.

The trial is an important one, and the stem cells might indeed have some effect on the neurons around them, said Lucie Bruijn of the ALS Association. “With all of the stem cell studies that are being offered worldwide with so little rigor, it is exciting to see this is so carefully undertaken,” she told ARF.—Amber Dance.

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  1. ARF related news story

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Papers

  1. Lunn JS, Sakowski SA, Federici T, Glass JD, Boulis NM, Feldman EL. Stem cell technology for the study and treatment of motor neuron diseases. Regen Med. 2011 Mar;6(2):201-13. PubMed.
  2. Joyce N, Annett G, Wirthlin L, Olson S, Bauer G, Nolta JA. Mesenchymal stem cells for the treatment of neurodegenerative disease. Regen Med. 2010 Nov;5(6):933-46. PubMed.
  3. Sanberg PR, Eve DJ, Willing AE, Garbuzova-Davis S, Tan J, Sanberg CD, Allickson JG, Cruz LE, Borlongan CV. The treatment of neurodegenerative disorders using umbilical cord blood and menstrual blood-derived stem cells. Cell Transplant. 2011;20(1):85-94. PubMed.
  4. Silani V, Calzarossa C, Cova L, Ticozzi N. Stem cells in amyotrophic lateral sclerosis: motor neuron protection or replacement?. CNS Neurol Disord Drug Targets. 2010 Jul;9(3):314-24. PubMed.
  5. Karussis D, Karageorgiou C, Vaknin-Dembinsky A, Gowda-Kurkalli B, Gomori JM, Kassis I, Bulte JW, Petrou P, Ben-Hur T, Abramsky O, Slavin S. Safety and immunological effects of mesenchymal stem cell transplantation in patients with multiple sclerosis and amyotrophic lateral sclerosis. Arch Neurol. 2010 Oct;67(10):1187-94. PubMed.
  6. Mitrecić D, Nicaise C, Gajović S, Pochet R. Distribution, differentiation, and survival of intravenously administered neural stem cells in a rat model of amyotrophic lateral sclerosis. Cell Transplant. 2010;19(5):537-48. PubMed.

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