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University Spin-Off Begins Trial of Stem Cell ALS Treatment

Neuron illustration (NIH)

(National Institute on Aging, NIH)

A technology developed at Tel Aviv University in Israel and licensed to a spin-off company invokes the potential of bone-marrow stem cells as treatment for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. A clinical trial, now in Israel and later in the U.S., to test the discovery is recruiting participants.

The technology involves taking stem cells from a patient’s own bone marrow and causes them to differentiate into astrocyte-like cells, which are responsible for the well-being of the brain’s neurons. Astrocytes are star-shaped cells — thus their name — that interact with non-nervous tissues and neurons, and monitor the integrity of nervous tissue by a complex set of receptors and channels.

Tel Aviv medical researchers Daniel Offen and Eldad Melamed focus on astrocytes because the cells release neuroprotectants, which have been shown to play a key role in reducing the progress of ALS, a debilitating disease characterized by the progressive degeneration of motor neurons, resulting in paralysis of a patient’s limbs and organs.

In preclinical research with mice, Offen and Melamed found the stem cells from bone marrow prevented degeneration in the brain following injection of some neurotoxins. Transplanting the stem cells also increased the survival rate and delayed the degeneration of motor functions of mice with ALS.

The university licensed the technology to a spin-off company, BrainStorm Cell Therapeutics, that has developed it into a product called NurOwn. The company is recruiting participants for a clinical trial to test the safety and efficacy of NurOwn at Jerusalem’s Hadassah Medical Center, and has recently agreed to expand the trials to Massachusetts General Hospital in Boston.

Daniel Offen and Eldad Melamed serve as chief scientist and chief medical adviser respectively of BrainStorm Cell Therapeutics that has offices in Israel and the U.S.

Read more: Mayo Clinic, Korean Biotech to Collaborate on ALS Research

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