Astrocyte Cell Therapy for ALS Has Interim Positive Results in Phase 1/2a Trial
In interim analysis of phase 1/2a clinical trial (NCT03482050) data, a cohort of 5 participants with amyotrophic lateral sclerosis (ALS) had improved muscle function over time after treatment with an astrocyte cell product (AstroRX; Kadimastem, Nes-Ziona, Israel). At 3 months after treatment, participants mean change in ALS Functional Rating Scale-Revised (ALS-FRS-R) was +0.26 per month compared with -0.87 per month over the 3 months before treatment.
In this open-label dose-escalating study, functional healthy astrocytes derived from human embryonic stem cells (hESC) are delivered with an intrathecal injection. The aim is to protect diseased motor neurons in the spinal cord and brain through several mechanisms of action and thereby slow disease progression.
In the first cohort of 5 participants, treated with the lowest planned dose of 100 x 106 cells, no serious adverse events or dose-limiting toxicities have occurred. Expected enrollment in the trial is 21 participants.
"These preliminary results on a small subset of patients are encouraging, as the treatment seems to be safe, at this lower dose,” said Marc Gotkine, MD, department of neurology, Hadassah Medical Center. “Initial data analysis presented to us appears to demonstrate a slowing down of the reported ALS-FRS-R deterioration rate over the 3 months following treatment. The trial is now continuing with higher doses and repeated injections.”