BrainStorm Cell Therapeutics Inc., in New York and Israel, reports a patient with the neurodegenerative diseases amyotrophic lateral sclerosis (ALS) and myasthenia gravis responded with improved motor and cognitive functions after stem cell treatments developed by the company. Researchers from Israeli medical centers and research institutes, and the Mayo Clinic in the U.S. report the case in an upcoming issue of the journal Muscle and Nerve (paid subscription required).
The case involves a patient with a rare condition, having both ALS and myasthenia gravis. ALS, also known as Lou Gehrig’s disease, is a progressive neurological disease that is invariably fatal. The disease attacks nerve cells that control voluntary muscles, such as those used in the limbs and face, causing them to stop sending signals to the muscles, leading to the muscles weakening and wasting away. Eventual failure of muscles in the diaphragm and chest lead to the inability to breathe without a ventilator.
Myasthenia gravis is an autoimmune disorder, also involving the voluntary muscles. The disease is a result of a defect in the transmission of nerve signals to muscles, where antibodies are tricked into blocking the passage of a neurotransmitter substance that activates muscle movement. The condition leads to frequent periods of muscle weakness, and when that weakness affects breathing muscles, can require the use of a ventilator. Treatments are available to control the disease.
The patient in this case is a 75 year-old man diagnosed with both disorders, who received two treatments of BrainStorm’s stem cell therapy. The technology, which the company calls NurOwn, takes a patient’s own mesenchymal or adult stem cells from bone marrow and cultures the stem cells into cells that secrete growth factors that promote nerve growth and restore neurological functions. The new cells are then transplanted with injections into the patient’s spinal cord and muscles. The company licenses the technology from Tel Aviv University.
BrainStorm says one month after the initial stem-cell transplants, the patient experienced significant improvement in speech, muscle activity, and cognition, based on measurements from a standard scale of muscular functions related to ALS, and respiratory and cognitive capacity. The patient’s speech improved, the company reports, to the point of delivering a speech to an audience.
Over the next six months, however, the patient experienced progressive weakness and cognitive impairment, requiring a second set of stem cell transplants. After the second transplants, the patient had improvements in muscle activity, speech, and cognition experienced similar to the first transplants, but the authors note (quoted in a company statement), “the outcome of the second transplantation was even more remarkable.” The company says the patient had no adverse effects from the treatments.
BrainStorm and Haddash Medical Center in Jerusalem are conducting an intermediate-stage clinical trial with 12 ALS patients in Israel. In December, the company reported in a statement only one adverse reaction to the treatments among the patients with “some initial indications of clinical improvement.” The company is planning another clinical trial in the U.S., with Massachusetts General Hospital, University of Massachusetts Medical School, and Mayo Clinic. That trial is awaiting FDA approval.
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