An Israeli company reports the first patient group in an early stage clinical trial indicates a new drug manages to significantly slow down disease progression of amyotrophic lateral sclerosis (ALS).
ALS Has No Cure & Lots of Costs
ALS, which leads to paralysis and eventual death, has an average life expectancy of 2 to 5 years. The Times of Israel’s Shoshanna Solomon reported that The ALS Therapy Development Institute notes that there about 450,000 ALS patients worldwide with 30,000 of them in America. The average healthcare costs of a ALS patient in the U.S. is $200,000—totaling $6 billion in America annually.
Sponsored by Kadimastem, they are conducting a study investigating the transplantation of Astrocytes derived from human embryonic stem cells in ALS patients. They are not changing the routine ALS treatment in participating patients. Rather, they are administering the treatment along with the standard of care treatment.
The study hypothesis: that transplantation of Astrocyte (AstroRx) cells can compensate for the malfunctioning of patients’ own astrocytes by restoring physiological capabilities like the reuptake of excessive glutamate, reducing oxidative stress, reducing other toxic compounds, as well as by secreting different neuroprotective factors.
The study commenced April 2018 and will complete August 2020.
The Therapy: AstroRx
AstroRx is a stem cell therapy developed by Kadimastem for the treatment of ALS. Comprised of full matured human astrocytes derived from human embryonic stem cells that are injected into the fluid surrounding the spinal cord of the patient to support damaged motor neurons reports ALS News Today. AstroRx was granted orphan drug status by the U.S. Food and Drug Administration for the treatment of ALS in November 2018.
The Clinical Investigative Site
The study is being conducted by Hadassah Medical Center in Jerusalem. The Principal Investigator is Dr. Marc Gotkine with the Neurology Department. Established in 1934, Hadassah operates two university hospitals at Ein Kerem and Mount Scopus in Jerusalem in addition to schools of medicine, dentistry, nursing and pharmacology affiliated with the Hebrew University Jerusalem. It was founded by Hadassah, the Women’s Zionist Organization of America, which continues to offer funding. It ranks as the sixth largest hospital complex in Israel. The two campuses the complex includes 1,000 beds, 31 operating theatres and nine special intensive care units. The founding mission is to extend a hand to all without regard of race, religion or ethnic origin.
Publicly traded Kadimastem Ltd develops cell therapies targeting ALS, known as Lou Gehrig’s disease, a neurodegenerative condition causing loss of muscle control. It is fatal. The news of the ALS clinical trial progress was reported in a filing with the Tel Aviv Stock Exchange. The company was founded by Professor Michel Revel, an emeritus faculty member of the Weizmann Institute of Science and winner of the Israel Prize. He developed Rebif, a multiple sclerosis drug. He founded Kadimastem in 2009 with Yossi Ben-Yossef, based on cell therapy research from Weizmann Institute. This research focuses on live cells transplanted into the body to replace malfunctioning, disease-causing cells.
The company is pre-revenue like many small biotech ventures and its future rides with this latest clinical trial. By the end of 2018, the company lost about $3.2 million.
The Times of Israel notes the firm runs two operations, including neurological diseases with the goal of replacing nervous system cells that don’t function and type 1 diabetes, where they develop cells that actually secrete insulin.
Call to Action: Do you or a loved one have ALS? This study in Israel should be monitored. It is still early, but if the results continue the sponsor, Kadimastem Ltd, may truly be on to something special. The investigator, Dr. Marc Gotkine, undoubtedly is an important person when it comes to this cell therapy study. TrialSite News tracks this effort. But there is a ways to go. The current Phase 1/2a study will conclude toward the end of 2020 and then the sponsor plans to commence an additional study with that they hope is a larger group of patients in more medical centers (sites).