The director of the Mellen Center for MS Treatment and Research at Cleveland Clinic provided context on whether efficacy outcomes should be weighed more than mechanistic action when evaluating MSC-NTF cell therapies. [WATCH TIME: 3 minutes]
WATCH TIME: 3 minutes
"Both are important. We knew that, from the outset, this was going to be a relatively small study. Our primary goal was to confirm the feasibility of the procedures and safety, and then to get some preliminary indications that the cells were biologically active.”
Autologous cellular therapy has not only recently emerged as a credible and practical option for cancer and other highly debilitating diseases, but a potential option for patients with progressive multiple sclerosis (PMS). A phase 2 study that evaluated the use of autologous mesenchymal stromal cells secreting neurotrophic factors (MSC-NTF) cells in this patient population found it to be a safe approach to treatment, with preliminary evidence of efficacy and relevant cerebrospinal fluid biomarker outcomes.
The results, presented at the 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), October 13-15, by Jeffrey Cohen, MD, showed that 14% and 13% of patients had 25% improvement on Timed 25-Foot Walk and 9-Hole Peg Test, respectively. There are currently no FDA-approved autologous cell therapy options for MS, and the therapeutic landscape of options for those with PMS remains limited.
The primary end point of the study was safety, with secondary efficacy end points that included T25W, 9HPT, Low Contrast Letter Acuity, Symbol Digit Modalities Test, 12 item MS Walking Scale, as well as cerebrospinal fluid, and blood biomarkers. Cohen, director, Mellen Center for MS Treatment and Research, Cleveland Clinic, sat down to discuss whether observing a mechanistic effect is more valuable than achieving efficacy end points at this stage of the treatment’s pipeline.
For more coverage of ECTRIMS 2021, click here.