Neural Stem cells to the Rescue?. Phase I trial results

VK Harris et al. Phase I Trial of Intrathecal Mesenchymal Stem Cell-derived Neural Progenitors in Progressive Multiple Sclerosis
EBiomedicine DOI:


Multiple sclerosis is one of the leading causes of disability in young adults. Our study is the first evidence suggesting that a cell-based therapeutic approach is capable of reversing disability in multiple sclerosis. Results of our study add to existing evidence demonstrating the safety and tolerability of intrathecal administration of autologous mesenchymal stem cell-derived neural progenitors. The study was associated with repeated administrations of cells freshly harvested from culture, as opposed to cryopreserved cells thawed at the bedside, which may have contributed to the observed efficacy of the treatment. The continued development of this therapeutic approach will likely have implications for treating other neurological diseases.

Multiple sclerosis (MS) is an immune-mediated demyelinating disease of the central nervous system and is one of the leading causes of disability in young adults. Cell therapy is emerging as a therapeutic strategy to promote repair and regeneration in patients with disability associated with progressive MS.
We conducted a phase I open-label clinical trial investigating the safety and tolerability of autologous bone marrow mesenchymal stem cell-derived neural progenitor (MSC-NP) treatment in 20 patients with progressive MS. MSC-NPs were administered intrathecally (IT) in three separate doses of up to 1 × 107 cells per dose, spaced three months apart. The primary endpoint was to assess safety and tolerability of the treatment. Expanded disability status scale (EDSS), timed 25-foot walk (T25FW), muscle strength, and urodynamic testing were used to evaluate treatment response. This trial is registered with, number NCT01933802.
IT MSC-NP treatment was safe and well tolerated. The 20 enrolled subjects completed all 60 planned treatments without serious adverse effects. Minor adverse events included transient fever and mild headaches usually resolving in <24 h. Post-treatment disability score analysis demonstrated improved median EDSS suggesting possible efficacy. Positive trends were more frequently observed in the subset of SPMS patients and in ambulatory subjects (EDSS ≤ 6.5). In addition, 70% and 50% of the subjects demonstrated improved muscle strength and bladder function, respectively, following IT MSC-NP treatment.
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.

So first things first you can see it is not a miracle cure and therefore we need to view these results positively, but also objectively. 

The myth being spun is that stem cells will turn back the clock and for most people here, this is not the case, so we need to understand the reality of these studies. It is a start

This study injected live growing cells and so they are producing a group of "potential goodies" that may be growth factors.