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New breakthroughs drive research into Amyotrophic Lateral Sclerosis (ALS) treatments
Research into regenerative medicine may have profound implications in the fight against ALS

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.

Chief Scientist Prof. Michel Revel (center, with beard), CEO Yossi Ben-Yossef (in blue glasses) and the Kadimastem team
Dr. Angela Genge
Dr. Eran Hornstein

MNI – McGill University and the Weizmann Institute of Science

Another potential therapy for ALS developed from the work of Weizmann scientists is set to move to the clinical stage right here in Canada through a collaboration between the Montreal Neurological Institute (MNI) of McGill University and the Weizmann Institute of Science.

Prof. Eran Hornstein of the Weizmann Institute and his team discovered that the activity of the endoribonuclease DICER, which is essential for microRNA biogenesis, is inhibited in ALS. They further demonstrated that enhancing DICER activity with a molecule called Enoxacin was beneficial for neuromuscular function in ALS in animal models, making Enoxacin a potential new therapy for patients suffering from ALS.

What makes this discovery particularly exciting is that Enoxacin is an antibiotic which is already approved for use in the treatment of other diseases. This decreases the risk of unforeseen health issues occurring during clinical trials which may in turn result in a shortened timeline to approval for this new potential ALS therapy.

Prof. Hornstein and his team are partnering with Dr. Angela Genge, Director of the Clinical Research Unit and of the ALS Clinic at the Montreal Neurological Institute and Hospital at McGill University to drive the research into Enoxacin forward into the clinical trial phase.

It is thanks to Apotex that these clinical trials are moving forward! We are so very grateful.

“This trial is a direct result of exceptional research in the lab of Dr. Hornstein. We hope it will be a gamechanger for our ALS patients.”
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