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Stem Cells Shown to Stop Decline for Some ALS Patients

Neuron in tissue culture

Neuron in tissue culture (EnCor Biotechnology Inc./Wikimedia Commons)

13 March 2015. An intermediate-stage clinical trial of a stem cell therapy to treat amyotrophic lateral sclerosis, or ALS, shows some patients receiving the treatments nearly stopped the decline or improved their muscle functions. Neuralstem Inc., a biotechnology company in Germantown, Maryland sponsoring the study, reported summary results yesterday.

ALS, also known as Lou Gehrig’s disease, is a progressive neurological disease that attacks the nerve cells controlling voluntary muscles, such as those in the arms, legs, and face. In ALS, muscles gradually weaken and waste away, leading to individuals losing their strength and their ability to move their arms, legs, and body. When diaphragm and chest wall muscles fail, people lose the ability to breathe without ventilatory support, often leading to death from respiratory failure.

The trial is testing 5 dosing regimens of Neuralstem’s NSI-566 spinal cord-derived stem cell therapy with 15 patients 3 medical centers in the U.S. The company’s technology produces neural stem cells of the human brain and spinal cord, with the ability to control their differentiation into mature human nerve cells and supporting glia cells. NSI-566, says the company, is designed to graft onto the regions where the cells are transplanted, rebuild circuitry in the patient’s neurons, and protect those nerve cells from further damage.

While the clinical trial’s main objective was to assess the safety of NSI-566, the researchers measured clinical response to treatments, as well as muscle functions according to standard rating scales for ALS patients. The company says 7 of the 15 patients responded to NSI-566 treatments. The 7 patients who responded to treatments showed near-zero decline on the ALS Functional Rating Scale. Among responders, overall functions declined -0.007 per day on the scale, compared to -0.1 for non-responding patients. After 9 months, the average score on the scale for responding patients was 37 of a maximum of 48, compared to 14 for non-responding patients.

The trial also tested lung function in a seated vital capacity test of lung muscles. The results show the 7 patients responding to NSI-566 treatments remained within 94 percent of their starting scores, compared to 71 percent for non-responding patients. A test of grip strength, an indicator of lower arm function, shows patients responding to treatments improved their scores on this measure.

On the main objective of the trial, safety of the stem cell treatments, Neuralstem says the treatments were well tolerated by all 15 patients, while 1 patient undergoing surgery to implant the stem cells experienced an adverse event during the procedure. The maximum tolerated dose was 16 million transplanted cells. The company plans to compile the trial’s full results into a manuscript for publication.

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