Brainstorm cell therapeutics announces publication of nurown ® als phase 2 randomized clinical trial data in neurology

Brainstorm cell therapeutics inc. on november 20, 2019, announced publication of “nurown phase 2 randomized clinical trial in als: safety, clinical and biomarker results,” in the international, peer-reviewed journal neurology:volume 93, number 24. the objective of the randomized placebo-controlled phase 2 clinical trial was to determine the safety and efficacy of a single transplantation of autologous bone-marrow derived msc-ntf cells (nurown) in participants with als. the clinical trial enrolled 48 participants randomized 3:1 (treatment: placebo) at three leading u.s. investigative sites: massachusetts general hospital, mayo clinic and university of massachusetts medical school. after a three-month pre-transplant run-in period, participants received one dose of msc-ntf cells (n=36) or placebo (n=12) and were followed for six-months. csf was collected prior to and two-weeks post-transplantation. the clinical trial confirmed that a single transplantation of msc-ntf (nurown) cells was safe and well-tolerated. brainstorm’s nurown cell therapy is the furthest advanced autologous stem cell treatment in development for als. on october 11, the company announced that the nurown, phase 3 clinical trial for als was fully enrolled. key efficacy findings: the rate of als disease progression (alsfrs-r slope) was stabilized for up to 12-16 weeks in a pre-specified group of participants with rapid progression (p<0.05) following a single transplantation; a higher proportion of msc-ntf treated study participants experienced =1.5 point/month improvement in alsfrs-r slope at all post-treatment time points, and this was statistically significant at 4 and 12 weeks post-transplantation (p=0.004 and 0.046, respectively); csf neurotrophic factors increased and inflammatory biomarkers decreased 2 weeks post-transplantation (p<0.05); csf mcp-1 levels (a marker of microglial activation and neuroinflammation) significantly decreased post-transplantation and correlated with alsfrs-r slope improvement at all time points (p<0.05).
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