As for possible efficacy of MSC-NTF cell transplantation, meaningful change in the rate of progression of the ALS-FR-S score and FVC was not seen in patients receiving IM injections but was seen in those receiving IT injections. The mean monthly rate of progression of the ALS-FRS-R score was reduced from −1.56 to 0.28 and the FVC was reduced from −3.5% to −2.3%. Improvement was stronger for patients in the 2a study, with a monthly rate of decline of ALS-FRS-R score from −1.4 to−0.6 and a reduction in FVC from −2.6% to 0.86%.
When results from the IT and IT plus IM groups were combined and analyzed via linear regression analysis, change between baseline and 6-month-follow-up results was significant for VFC (from −5.1% to −1.2%/month; P = .036) and approached significance for ALS-FRS-R score (from −1.2 to 0.6/month; P = .052). Eighty-seven percent of patients in this study cohort were responders, defined as having at least 25% improvement at 6 months after treatment in the slope of progression in either FVC or ALS-FRS-R score.2
The results demonstrate that IT and IM administration of MSC-NTF cells—even administered concomitantly—is safe in patients with ALS and may ultimately provide clinical benefit. A double-blind, placebo-controlled multicenter phase 2 clinical trial is ongoing.
- Petrou P, Gothelf Y, Argov Z, et al. Safety and clinical effects of mesenchymal stem cells secreting neurotrophic factor transplantation in patients with amyotrophic lateral sclerosis Results of phase 1/2 and 2a clinical trials. JAMA Neurol. 2016; doi: 10.1001/jamaneurol.2015.4321.
- Castrillo-Viguera C, Grasso DL, Simpson E, et al. Clinical significance in the change of decline in ALSFRS-R. Amyotroph Lateral Scler. 2010;11(1-2):178-180.