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BrainStorm Partners with ALS Groups to Provide Biospecimens from Phase 3 ALS Study

January 10, 2023

BrainStorm Cell Therapeutics said that it will donate biospecimens from its placebo-controlled phase 3 ALS trial to the Northeast Amyotrophic Lateral Sclerosis Consortium biorepository for use by the research community.

The specimens are being submitted to the biorepository in connection with a $500,000 grant previously awarded to BrainStorm by The ALS Association and I AM ALS, to support biomarker research.

The specimens submitted to the NEALS biorepository include serum and cerebrospinal fluid samples collected from trial participants treated with placebo. Samples of both biofluids were collected in a matched fashion and longitudinally at multiple time points over a 20-week period, allowing for the evaluation of intra-patient biomarker changes over time as well as comparisons of biomarker levels between serum and cerebrospinal fluid.

The samples are gathered from a phase 3 study of BrainStorm’s NurOwn, an experimental therapeutic approach to targeting disease pathways important in neurodegenerative disorders. NurOwn uses autologous MSC-NTF cells that are differentiated bone-marrow derived mesenchymal stem cells that secrete neurotrophic factors. The cells are designed to effectively deliver multiple neurotrophic factors and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.

“Our phase 3 trial included one of the largest longitudinal specimen collection protocols in trial participants with ALS, which has allowed us to perform extensive and unique biomarker analyses,” said Stacy Lindborg, co-CEO of BrainStorm. “These analyses have greatly increased our understanding of both the mechanisms driving ALS progression, as well as the biological changes in participants treated with NurOwn.”  

Author: Rare Daily Staff

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