[Current status and future of cell-based therapy for patients with cardiogenic cerebral embolism].

BACKGROUND AND AIM Cerebral infarction causes permanent neurological damage. Recently, the intravenous administration of bone marrow-derived mononuclear cells has been shown to improve functional recovery through enhanced angiogenesis in an experimental stroke model. Based on these observations, we have started a phase 1/2a clinical trial of cell-based therapy for patients with cardiogenic cerebral embolism. METHODS The major inclusion criterion was patients in whom severe cerebral embolism was diagnosed (more than 9 in the NIHSS score on day 7 after the onset of stroke) at age 75 or younger. The patients received 25 ml (low-dose group, n=6) or 50 ml (high-dose group, n=6) of bone marrow aspirate on days 7-10 after the onset of stroke. Autologous bone marrow-derived mononuclear cells were purified by the density gradient method and administered intravenously on the day of cell aspiration. The primary endpoint was safety and improvement of the NIHSS score compared with our historical control. RESULTS We treated 6 patients in the low-dose group and 4 patients in the high-dose group. No adverse effects were observed and most of the patients showed a significant improvement in neurological function at 6 months after cell transplantation. No enrolled patients showed worsening of the NIHSS score at 30 days after the treatment compared with the NIHSS score before the treatment. CONCLUSION Autologous bone marrow mononuclear cell transplantation is apparently safe and feasible, and it improves functional recovery.