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Clinical Trial of ALS Stem Cell Therapy Begins Treatments

Eva Feldman

Eva Feldman (University of Michigan)

The first two of up to 15 patients received their first injections of stem cells to treat amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, in an intermediate-stage clinical trial at University of Michigan in Ann Arbor and Emory University in Atlanta. The study is testing stem cell treatments made by Neuralstem, a biotechnology company in Rockville, Maryland that is also funding the trial.

ALS 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 five dosing regimens of Neuralstem’s NSI-566 spinal cord-derived stem cell therapy. 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.

The therapy received orphan drug status from the U.S. Food and Drug Administration, which also approved the new clinical trial. An early-stage trial confirmed the safety of NSI-566 injections of doses with 100,000 stem cells. The researchers in that trial also reported that for some ALS patients, the progression of the disease did not accelerate.

Michigan neurology professor Eva Feldman is leading the new trial where four groups of three ALS patients each receive between 10 and 20 injections in the cervical region of the spinal cord, of 200,000 or 400,000 stem cells. A fifth group of three patients receives 20 injections of 400,000 stem cells in the lumbar region of the spinal cord, as well as 20 injections in the cervical region.

One group a month will receive the NSI-566 injections, followed by a month of observation before the next group’s treatments. Treatments are expected to be completed by the second quarter of 2014.

The study focuses particularly on finding the maximum safe dosage of NSI-566, as well as determining the safety and feasibility of treatments. The trial also gauges any clinical benefit from the treatments, including reduction in the loss of motor functions, maintenance of respiratory capacity, stabilization of scores on a standard ALS functional rating scale.

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