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


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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.

References

  1. 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.
  2. 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.