Fetal olfactory ensheathing cells transplantation in amyotrophic lateral sclerosis patients: a controlled pilot study.

This study was designed to clarify whether transplantation of fetal olfactory ensheathing cells (OECs) would affect the clinical course of patients with amyotrophic lateral sclerosis (ALS). Thirty-five patients with probable or definite ALS were enrolled from December 2004 to September 2006; 15 patients received OECs transplantation and 20 patients did not receive OECs transplantation. OECs were cultured and injected into the bilateral corona radiata involving the pyramidal tracts of the frontal lobes. The primary end point used to indicate effectiveness was the rate of change according to the ALS Functional Rating Scale (ALS-FRS) total score. All patients were tested five times at baseline and monthly intervals during a four-month follow-up period using assessment of ALS-FRS. Thirty-one patients (14 in the OECs treated group and 17 in the controls) completed the four-month study; the remaining four patients were lost to follow-up. Patients' data were analyzed four months after OECs transplantation and at the end of the controlled period. There was no significant difference in the rate of progression as measured by the ALS-FRS total score during the first two months (p > 0.05). The functional deterioration, however, was significantly slower in the treated group than in the control group during the last two months (p < 0.05). The mean (+/-SD) change for the ALS-FRS total score was 0.07 +/- 4.18 for the treated group and 6.12 +/- 5.49 for the control group (p = 0.002) during the four months. Of the 14 patients in the treatment group, seven experienced neurological functional improvements, two were stable compared with their clinical status at entry, and the ALS-FRS scores in the other five decreased by a mean of 4.4. Of the 17 patients in the control group, only one patient's condition remained stable while the ALS-FRS scores in the other 16 decreased by a mean of 6.5. The result indicates OECs transplantation appears to be able to slow the rate of clinical progression of ALS in the first four months posttransplantation.