Mayo Clinic Taking Part in ALS Stem Cell Clinical Trial

Cortical neuron in a culture dish (Don Arnold, University of Southern California, via NIH)

29 July 2014. BrainStorm Cell Therapeutics, a biotechnology company in New York and Israel says the Mayo Clinic in Rochester, Minnesota is now taking part in a clinical trial of the company’s treatments for amyotrophic lateral sclerosis (ALS) that harness the patient’s own bone marrow stem cells. Financial aspects of the agreement were not disclosed.

ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease where neurons or nerve cells controlling muscles in the body begin to waste away, and can no longer send or receive signals from the brain or spinal cord. As the nerve cells stop functioning, the muscles in the limbs, and later speech and breathing muscles, begin weakening and eventually stop functioning. Most people with the disease die of respiratory failure.

BrainStorm’s technology, called NurOwn, extracts stem cells from the patient’s bone marrow that are transformed into cells supporting development of nerve cells. These transformed stem cells, says the company, secrete proteins that protect nerve cells, as well as encourage their growth and interactions with muscles. Because the original cells come from the patient, they have little risk of rejection by the immune system.

The clinical study of BrainStorm’s therapy is an intermediate-stage test of NurOwn against a placebo with 48 patients, expected to begin in April 2014. The trial is already planned to take place at Massachusetts General Hospital in Boston and University of Massachusetts Medical School in Worcester. Patient recruitment has not yet begun at these sites.

The new agreement adds Mayo Clinic in Rochester as a trial site, under the direction of Anthony Windebank, director of the clinic’s regenerative neurobiology lab. In addition, Mayo Clinic will be able to to generate NurOwn cells for the trial in its Human Cellular Therapy Laboratory, which has facilities to harvest and transform a patient’s own stem cells.

Earlier this month, BrainStorm reported results of a patient with both ALS and myasthenia gravis, an autoimmune disorder also involving the voluntary muscles, who responded with improved motor and cognitive functions after receiving the company’s stem cell treatments. That patient was treated at the Mayo Clinic.

Read more:

• Stem Cell Therapy Aids Patient with ALS, Myasthenia Gravis
• Myelin Foundation to Assess Multiple Sclerosis Treatment
• Clinical Trial of ALS Stem Cell Therapy Begins Treatments
• NIH Funds Three New Neurologic Drug Discovery Projects
• Trial Fails to Show Biogen Idec ALS Drug Efficacy

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