Treatment of refractory epilepsy patients with autologous mesenchymal stem cells reduces seizure frequency: An open label study

Purpose: Existing anti-epileptic drugs (AED) have limited efficiency in many patients, necessitating the search for alternative approaches such as stem cell therapy. We report the use of autologous patient-derived mesenchymal stem cells (MSC) as a therapeutic agent in symptomatic drug-resistant epilepsy in a Phase I open label clinical trial (registered as NCT02497443). Patients and methods: The patients received either standard treatment with AED (control group), or AED supplemented with single intravenous administration of undifferentiated autologous MSC (target dose of 1 x 10(6) cells/kg), followed by a single intrathecal injection of neurally induced autologous MSC (target dose of 0.1 x 10(6) cells/kg). Results: MSC injections were well tolerated and did not cause any severe adverse effects. Seizure frequency was designated as the main outcome and evaluated at 1 year time point. 3 out of 10 patients in MSC therapy group achieved remission (no seizures for one year and more), and 5 additional patients became responders to AEDs, while only 2 out of 12 patients became responders in control group (difference significant, P = 0.0135). Conclusions: MSC possess unique immunomodulatory properties and are a safe and promising candidate for cell therapy in AED resistant epilepsy patients. (C) 2017 Medical University of Bialystok. Published by Elsevier B.V. All rights reserved.