The 5th keynote interview of today’s morning session at WSCRM conference saw the “father of hMSCs” on its stage. Dr Arnold Caplan, from Case Western Reserve University and OrthoCyte Corp. (US), gave its audience an interesting update on how the field of human mesenchymal stem cells (hMSCs) has progressed since the days of their initial definition back in the ‘80s, when Prof Caplan was conducting studies on embryonic chick limb mesenchymal cells.
The hMSC-based technology discovered by Dr Caplan lab led to the foundation of Osyris in 1992. The tremendous potential of these cell preparations to heal a number of different diseases was then clear but the mechanism of action was not yet completely understood. It later became evident that hMSCs are pericytes derivatives and that they can work as “drugstores” for neighbouring cells by secreting therapeutic molecules when needed and that in an injury setting they can set up immunomodulative responses together with a regenerative microenvironment.
Dr Caplan interestingly pointed out that despite different preparations are being licensed by different companies, using different protocols, the cells’ mechanisms of action are likely to be shared and overlapping. Nonetheless it’s in the understanding of what the actual in vivo indirect therapeutic responses are once these cells are implanted in a patient, that liesthe real challenge. How can hMSCs then be specifically primed for specifically stimulating a host injured site? Indeed, this remains a challenge for hMSC products and not surprisingly the only answer -as Arnold stressed out- is by conducting rigorous evidence-based clinical trials.
When asked by Chair Prof Chris Mason how he projects hMSC-based therapies in a 5-year time, he replied that actually already 2014 could be decisive for hMSC therapy approval, mentioning as an example Mesoblast Ltd promising data for vertebral disc regeneration.
I had the chance to chat with Dr Caplan over a coffee break and he commented again on the crucial importance of performing rigorous clinical trials before using a cell therapy product. He condemned unregulated approaches such as the recently emerged Stamina case as these often do not comply with cGMP regulations and are of not use to industry growth.
hMSCs are often indicated as the ideal candidate for the treatment of a wide variety of life-threatening diseases in virtue of their demonstrated safety in 30-50K patients (100% of the patiens who received treatment). But what if we treated million people with hMSC products? In the era of personalized medicine, will we still be able to claim that the probability to find non-responsive outliers (or even worst, individuals in which these products are non-safe) still be irrelevant? Maybe the market need and economic logic wouldn’t allow space for these considerations at the moment, but once more it’s up to regulatory bodies, as they should, yes, protect from scientifically detrimental cases like Stamina’s, but also consider that market logics can’t be the only driving forces.
Giulia Detela for Total BioPharma at WSCRM Congress, London
Department of Biochemical Engineering
University College London