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In a phase 1/2a study, 5 patients with amyotrophic lateral scleroris (ALS) have had healthy astrocytes derived from stem cells (AstroRx; Kadimastem, Ness Ziona, Israel) transplanted intrathecally. The Phase 1/2a trial is expected to include a total of 21 patients, with results expected in 6 months. The therapy contains functional, healthy astrocytes derived from human embryonic stem cells. Healthy astrocytes are expected to support and protect motor neurons from damage that results in ALS.
For the therapy, the astrocytes are injected into the patient’s spinal fluid, with the goal of supporting the malfunctioning cells in the brain and spinal cord, thereby slowing the progression of the disease and improving quality of life and life expectancy.
“The continued collection of neurologic data in ALS patients before and after treatment will allow us in the near future to evaluate the results of the first group of patients,” said Michel Revel, chief scientist at Kadimastem.
Gordon H. Baltuch, MD, PhD
Rashmi B. Halker Singh, MD; Amaal J. Starling, MD; and Juliana VanderPluym, MD
Brian E. McGeeney, MD, MPH, MBA