Autologous Mesenchymal Stem Cells for ALS: A Phase 2 Trial

A single administration of intramuscular and intrathecal mesenchymal stem cells induced to secrete neurotrophic factors is relatively safe, reduces levels of cerebrospinal fluid neuroinflammatory biomarkers, and increases neurotrophic factors.

To determine the safety and efficacy of autologous mesenchymal stem cells induced to secrete neurotrophic cells (NurOwn®), investigators randomized 36 patients with amyotrophic lateral sclerosis (ALS) to treatment and 12 to placebo in a phase 2 double-blind trial funded by Brainstorm Cell Therapeutics. Patients had a vital capacity ≥65% and symptom duration 1 to 2 years. After a 3-month lead-in period, participants' mesenchymal stem cells were isolated from bone marrow aspirate and differentiated to secrete neurotrophic factors (MSC-NTF). Patients then underwent MSF-NTF administration intrathecally and intramuscularly at 24 sites in the right arm and were followed for 6 months.

No deaths or serious adverse events were attributed to treatment…

Reviewing Author

Leana Doherty, MD

Disclosures

Editorial BoardsNeurohospitalist Society; InterACTN (Annals of Clinical and Translational Neurology)

Leana Doherty, MD

Disclosures

Editorial BoardsNeurohospitalist Society; InterACTN (Annals of Clinical and Translational Neurology)

Autologous Mesenchymal Stem Cells for ALS: A Phase 2 Trial

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Reviewing Author