Amyotrophic Lateral Sclerosis (also known as ALS or Lou Gehrig’s disease) affects two out of every 100,000 people each year in North America. It drives a devastating trajectory that begins with the death of motor neurons in the brain and spinal cord, and often ends with losing the ability to walk, talk, swallow, and breathe. With only one FDA-approved drug in existence, a compound that extends ALS patients’ lifespan by no more than three months, there is a critical need for new medical strategies.

This past spring, Kadimastem, a biotech company that licensed the discoveries of emeritus Weizmann Institute faculty member Prof. Michel Revel and his team, began enrolling patients for clinical trials into a new treatment approach for ALS using a cell therapy product developed by the company.

This phase I/IIa trial is taking place at Hadassah Medical Center ALS in Israel and will include 21 patients. It aims to test the efficacy and safety of AsrtoRx®, a stem-cell based therapy developed by Kadimastem that enables the repair and replacement of brain-supporting cells which have been shown to malfunction in ALS patients.

Prof. Revel, formerly of the Department of Molecular Genetics, is Chief Scientist of Kadimastem. The company’s focus is regenerative medicine, a clinical approach that seeks to harness the potential of stem cells to repair or replace damaged tissues in order to combat disease. The treatment involves the use of human embryonic stem cells to produce precursor cells that when transplanted into the spinal cord fluid have been shown to slow the progress of ALS in animal models. The cells injected during the AsrtoRx® treatment are precursors of astrocytes, a type of brain cell that supports nerve cells and keeps them alive.

The use of this stem-cell therapy is expected to significantly slow down the progression of ALS and improve the patient’s life expectancy.