The possible reversal of disability that was observed in a subset of patients warrants a larger phase II placebo-controlled study to establish efficacy of IT MSC-NP treatment in patients with MS.
Here are exerts from the paper which is open acess
"The clinical feasibility of IT MSC-NP treatment in MS was initially investigated in six patients with advanced MS treated with two to five injections of escalating doses of autologous MSC-NPs. PwMS were followed an average of 7.4 years after initial injection. There were no serious adverse events or safety concerns noted, and the treatments were well-tolerated. Four of the six patients showed a measurable clinical improvement following MSC-NP treatment".
In this current study "eligible patients had clinically definite SPMS or PPMS with significant disability (EDSS ≥ 3.0) that was not acquired within the 12 months prior to enrollment. The inclusion of patients with a relatively stable disease state was designed to allow better discernment between natural disease progression and treatment-related events. To minimize additional variables, patients who were already receiving disease-modifying therapies (DMT) upon entering the study continued as a concomitant treatment through the course of the study".
"There were no serious adverse events or hospitalizations associated with IT MSC-NP treatment"
"The safety data was further supported by a lack of any change in brain MRI scans during the study. Specifically, no new T2 lesions or changes in disease burden were observed".
"The study design was not blinded, and there were no placebo controls".
Therefore, be warned the power of the placebo should not be under-estimated. The placebo effect where you get better after taking nothing can be immense and has sent many good ideas to their graves.
The primary post-treatment clinical assessments were conducted at three and six months following the third treatment and compared to baseline (pre-treatment) in order to determine trends in efficacy. Of the 20 study subjects, 15 (or 75%) demonstrated neurological improvement associated with IT MSC-NP treatment.
Improvements were documented in the following areas: EDSS, MRC muscle strength scale, timed 25-ft walk (T25FW), and/or bladder function.Of the remaining subjects, two showed disease worsening despite the treatment, and three subjects showed no change.
The predefined endpoint was adverse effects but secondary endpoints were evidence of efficacy allowing the centre to pick and choose and so data hack to spin the best story.
So as you can see 4 people showed qute a bit of improvement and there were reports of improvement for other outcomes notably the lower limbs and bladder rather than upper limbs and cognition.