First Patient Treated in Phase 1/2a Trial of Cell Therapy for Amyotrophic Lateral Sclerosis
The first-ever transplantation of astrocytes derived from stem cells (AstroRx; Kadimastem, Nes Ziona, Israel) into a patient with amyotrophic lateral sclerosis (ALS) has been announced. Beginning in April 2018, 5 patients have now been enrolled as cohort A in this phase 1/2a clinical trial (NCT03482050). An additional 16 patients in 3 more cohorts are being enrolled. The aim of this trial is to examine safety and efficacy of transplanting astrocytes as adjunctive therapy for people with ALS.
The transplanted astrocytes are derived from human embryonic stem cells developed in a technique that includes growing cells on an industrial scale and then causing them to differentiate into astrocytes in clean rooms. This represents a unique approach of producing healthy functioning cells in the laboratory for transplantation with the goal of replacing dysfunctional cells.
Professor Tamir Ben Hur, Director of the Department of Neurology and Head of the Department of Neurosurgery at the Hadassah Medical Center in Jerusalem, where the trial is being conducted said: "The translation of the years of investment and development into the patient's treatment is very exciting. We have an optimal combination of academia, industry and medicine."
Prof. Michel Revel, Kadimastem's Chief Scientist, commented: "Kadimastem has now completed years of research and development for its cell-therapy product designed to treat ALS. This, following years of basic research that succeeded in using human embryonic stem cells to produce astrocytes, the specialized cells that support survival of neurons in the human brain, and following proof of efficacy in ALS disease models in animals. At the end, an industrial pharmaceutical production process has been developed at Kadimastem and has received all the regulatory approvals for use in a clinical trial. The injection of the AstroRx cells to the first patient of the clinical trial is an important milestone in the clinical development of this cell therapy product for the treatment of the ALS disease."