Stem-Cell Derived Astrocytes for Amyotrophic Lateral Sclerosis: Positive Interim Results May Lead to Amending Phase 1/2a Protocol

  • Amyotrophic Lateral Sclerosis
  • Stem cell treatment

An amendment will be submitted for an ongoing phase 1/2a clinical trial protocol of an “off-the-shelf" clinical-grade cell product (AstroRX; Kadimastem, Ness Ziona, Israel) being investigated for treatment of amyotrophic lateral sclerosis (ALS). 

The cell product, administered with intrathecal injection, contains functional healthy astrocytes derived from human embryonic stem cells. The aim of the therapeutic astrocyte delivery is to protect diseased motor neurons and slow progression of ALS.   

If the amendment is accepted by the Food and Drug Administration (FDA), the trial will assess the safety and efficacy of a repeated low dose (100x106) in the cohort that was to be treated with low then medium dose (250x106).

This protocol change is supported by the positive interim results seen in cohort A, reported in September 2019, for whom statistically significant efficacy (P=.0023) and safety of the single low-dose occurred. Progression of ALS was reduced in the first 3 months posttreatment, compared with participants’ pretreatment baseline. 

Results of cohorts A are expected by the end of 2019; results of cohort C are expected in the first half of 2021. 

"We are the first to treat ALS patients with astrocyte cells,” said Rami Epstein, CEO, Kadimastem. “Following our positive interim results, we look forward to achieving a prolonged therapeutic effect in the repeated low dose administration, bringing new hope for patients with this incurable disease."

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