Researchers Encouraged by Results from First American Clinical Trial for Stem Cell-Based Treatment of ALS

Newswise NEW ORLEANS (April 29, 2013) Researchers who completed the first American clinical trial involving stem cell-based treatment of Amyotrophic Lateral Sclerosis (ALS) were encouraged by the trial studys results, noting that this delivery approach could be a helpful therapeutic approach for other traumatic spine-related problems.

The group focused on the safety of a direct microinjection-based technique and neural stem cell transplantation to the cervical and thoracolumbar spinal cord. Eighteen microinjection procedures delivered NSI-566RSC, a human neural stem cell, to a total of 15 patients in five cohorts. Each of the injection procedures consisted of five injections of 10l at 4mm intervals. Group A (n=6) was non-ambulatory and received unilateral (n=3) or bilateral (n=3) thoracolumbar microinjection. Groups B through E were ambulatory and received unilateral (group B, n=3) or bilateral (group C,n=3) bilateral thoracolumbar microinjection. Groups D and E received unilateral cervical (group D,n=3) or cervical plus bilateral thoracolumbar microinjection (group E,n=3). Detailed pre- and post-operative neurological outcomes were recorded, such as post-operative pain, as well as urologic, sensory and motor functions. The results of this study, Intraspinal Stem Cell Transplantation in ALS, A Phase I Trial: Cervical Microinjection Safety Outcomes, will be presented by Jonathan Patrick Riley, MD, from 10:24-10:35 a.m. on Monday, April 29. Co-authors are Jonathan Glass, MD, PhD; Karl Johe, PhD; Meraida Polak, RN; Thais Federici, PhD; Eva Feldman, MD, PhD; and Nicholas Boulis, MD, FAANS.

In the results, researchers noted that the unilateral cervical (group D,n=3) and cervical plus thoracolumbar microinjections (group E,n=3) have been completed in ambulatory patients, and that no neurological worsening was witnessed to follow either cervical or thoracolumbar microinjection. The researchers did note that one cervical microinjection patient developed a post-operative kyphotic deformity, which prompted the addition of a laminoplasty in subsequent patients. Neurologic morbidity was not observed with the delivery of a cellular payload to the cervical or thoracolumbar spine spinal cord within the test groups of this at-risk patient group. This led the researchers to opinion that more consideration should be given to this delivery approach as a possible option for neurodegenerative, oncologic and traumatic spinal cord disorders.

We are excited that the safety results of this trial have borne out what has been shown by our preclinical studies that both the cervical and thoracolumbar spinal cord are able to safely tolerate multiple targeted injections of a cellular graft, said Jonathan Patrick Riley, MD. These results support the exploration of the cellular graft dose range that may be delivered and safely tolerated. Identification of a safe dose range that the spinal cord tolerates is an important first step prior to completion of Phase II efficacy studies.

Disclosure: Jonathan Patrick Riley, MD, disclosed that Neuralstem, Inc., provided other financial or material support.

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About the 2013 AANS Annual Scientific Meeting: Attended by neurosurgeons, neurosurgical residents, medical students, neuroscience nurses, clinical specialists, physician assistants, allied health professionals and other medical professionals, the AANS Annual Scientific Meeting is the largest gathering of neurosurgeons in the nation, with an emphasis on the fields latest research and technological advances. A record-breaking 1,003 scientific abstracts were presented for review at the 2013 AANS Annual Scientific Meeting, and the scientific presentations given at this years event represent cutting-edge examples of the incredible developments taking place within the field of neurosurgery. Additional information about the AANS Annual Scientific Meeting and the Meeting Program can be found at Meeting/2013/Main/Home.aspx.

Founded in 1931 as the Harvey Cushing Society, the American Association of Neurological Surgeons (AANS) is a scientific and educational association with nearly 8,300 members worldwide. The AANS is dedicated to advancing the specialty of neurological surgery in order to provide the highest quality of neurosurgical care to the public. All active members of the AANS are certified by the American Board of Neurological Surgery, the Royal College of Physicians and Surgeons (Neurosurgery) of Canada or the Mexican Council of Neurological Surgery, AC. Neurological surgery is the medical specialty concerned with the prevention, diagnosis, treatment and rehabilitation of disorders that affect the entire nervous system including the spinal column, spinal cord, brain and peripheral nerves. For more information, visit

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Researchers Encouraged by Results from First American Clinical Trial for Stem Cell-Based Treatment of ALS

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