Kadimastem (TASE: KDST) has reported positive interim results from a trial of its treatment for Amyotrophic Lateral Sclerosis (ALS) (Lou Gehrig's disease) using fully differentiated cells that are derived from embryonic stem cells. These are the first human trial results reported by the company, which was founded in 2009.
According to the report, development of the disease was significantly slower than the projected rate of deterioration for five patients in the trial, according to the historic figures. As is usually the case in diseases like ALS, there was no control group in the trial. The trial is an initial one that is not yet supervised by the US Food and Drug Administration (FDA). The company also monitored the patients during the three months preceding the treatment, during which their deterioration was the same speed as in the accepted historic measurement, so the good results were not caused by deliberate or random selection of patients whose deterioration rate was in any case slow. The difference between the periods before and after treatments was also statistically significant.
ALS is a degenerative muscular disease that usually results in the patient's death in 2-5 years, during which the patient's condition deteriorates rapidly, with disability culminating in complete paralysis. The genetic basis of the disease is unknown, and its mechanism is also not completely understood. Most patients are over 60, but 10% of them are younger, and there are even babies who suddenly contract the disease. The Israls and Prize4life non-profit organizations consider ALS a rare disease, but not an ultra-rare one. A number of Israeli companies are developing a drug for the disease, including BrainStorm Cell Therapeutics, Kadismastem, NeuroSense Therapeutics, and Immunity Pharma. Kadimastem is the only one other than BrainStorm that has reached a human trial.
Kadimastem's product is based on the use of human brain cells called astrocytes that are injected into the spinal fluid. These astrocyte cells are manufactured by Kadimastem by a differentiation process from human embryonic stem cells. According to the company, the cells discharge materials that protect motor nerve cells, the degeneration of which is related to the development of ALS. In contrast to BrainStorm, which also injects cells into the vicinity of the spinal column, Kadimastem's product is a shelf product. BrainStorm's product is also based on stem cells, but is produced from the cells of each adult patient.
Kadimastem was founded by Yossi Ben Yosef, and Prof. Michel Revel. Ben Yosef resigned from the company last May without seeing the company he founded, and for which he abandoned his venture capital activity, reach its first milestone in treating patients. Several months after his resignation, Ben Yosef was investigated for insider trading in an affair in the which the main suspect was Menachem Weinberg from the Summit company.
Ben Yosef was replaced as CEO by Rami Epstein, a cofounder of IDgene Pharmaceuticals, which searched for genes that increase the risk of diseases. He later became one of the founders of BiondVax Pharmaceuticals, a company that became listed on Nasdaq after many years of being listed on the Tel Aviv Stock Exchange. Before being appointed CEO of Kadimastem, Epstein was a director in the company for six years. He became involved in the company through Revel, who was a member of IDG's advisory committee. The two men became acquainted on the committee, and Revel later recruited Epstein from Kadimastem. Revel is known as the inventor of Rebif, a drug for treatment of multiple sclerosis sold by German pharma company Merck.
In addition to its ALS product, Kadimastem is also developing a drug for diabetes, also based on embryonic stem cells. In this product, however, the function of the stem cells is to turn into beta cells that discharge insulin. The aim is to implant the cells, so that they will become a functioning pancreas to replace the patient's damaged pancreas.
Published by Globes, Israel business news - en.globes.co.il - on September 25, 2019
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