Clinical trials for ALS launched

Kadimastem, a biotech company that licensed the discoveries of emeritus Weizmann Institute faculty member Prof. Michel Revel and his team, has received approval from the Ministry of Health for clinical trials on a new treatment approach for amyotrophic lateral sclerosis (ALS). The phase I/IIa trial will take place at Hadassah Medical Center ALS, and will include 21 patients.

The trial will test the efficacy and safety of AsrtoRx®, a stem-cell based therapy that enables the repair and replacement of cells and tissues lost in the course of the disease.

ALS, also known as Lou Gehrig’s disease, affects two out of every 100,000 people each year, and 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.

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 shown to slow the progress of ALS in animal models. The cells injected are precursors of astrocytes, a type of brain cells that keep nerve cells alive.

Prof. Revel is also the creator of the blockbuster drug Rebif®, a multiple sclerosis drug now marketed by the German pharmaceutical company Merck. Prof. Revel received the Israel Prize in Medicine in 1999.

The vision for the company, which was established in 2009, “was to develop innovative, groundbreaking treatments to help millions of patients suffering from various diseases worldwide,” says Kadimastem CEO Yossi Ben-Yossef. “The commencement of the clinical trial… is a great achievement for us, both in reaching the clinical stage and in the successful development of a revolutionary, groundbreaking treatment based on a unique approach in the cell-based medicine field.”