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Stem-Cell Therapy Shows Promise in ALS Patients

Mid-stage trial meets primary safety endpoints

Positive results have been reported from a phase II trial of NSI-566 (Neuralstem, Inc.), a spinal cord-derived neural stem-cell line, in patients with amyotrophic lateral sclerosis (ALS). The study met its primary safety endpoints. The maximum tolerated dose of 16 million transplanted cells and the surgery were well tolerated.

The open-label, dose-escalating trial included 15 ambulatory patients, who were divided into five dosing cohorts. The dosing increased from 1 million to 8 million cells in the cervical region of the spinal cord. The final trial cohort also received an additional 8 million cells in the lumbar region of the spinal cord.

Secondary efficacy endpoints at 9 months after surgery indicated a 47% response rate to the stem-cell treatment, as measured by either a near-zero slope of decline or a positive slope in the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) score in seven of the 15 patients, and by either a near-zero decline or an increase in grip strength in seven of the 15 patients.

Grip strength is an indicator of direct muscle strength of the lower arm. ALSFRS is a standard clinical test used to evaluate the functional status of ALS patients.

The average ALSFRS score for responders at 9 months after treatment was 37. Nonresponders scored an average of 14. These scores represented 93% and 35% of the baseline score retained by the responders compared with the nonresponders, respectively, at 9 months, which represented a statistically significant difference. As measured by an average slope of decline in the ALSFRS, the responders’ disease progression was –0.007 point per day, whereas the nonresponders’ disease progression was –0.1point per day, which was again statistically significant.

Lung function, as measured by seated vital capacity, showed that responders remained within 94% of their starting scores compared with 71% for nonresponders.

A later-stage, multicenter study of NSI-566 for the treatment of ALS is expected to begin in 2015. The treatment received an “orphan product” designation from the FDA.

In addition to ALS, NSI-566 is being evaluated for the treatment of chronic spinal cord injury in a phase I trial. NSI-566 is also in clinical development to treat neurological diseases, such as ischemic stroke and acute spinal cord injury.

NSI-532.IGF, another stem-cell product from Neuralstem, Inc., consists of human cortex-derived neural stem cells that have been engineered to secrete human insulin-like growth factor-1 (IGF-1). In preclinical data presented at the Congress of Neurological Surgeons’ 2014 annual meeting, the cells rescued spatial learning and memory deficits in an animal model of Alzheimer’s disease.

Source: Neuralstem, Inc.; March 12, 2015.

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