Medical Professionals Online

Stem Cell Therapy In Multiple Sclerosis - Now It Is Time To Really Start

March 24, 2017

Clinical research with human stem cells to repair brain damage in MS patients should be intensified. Although not unanimously, this was the main conclusion of an international medical scientific conference on MS and stem cell therapy in Italy today, organised by the independent European Charcot Foundation.

The promising results of the vast number of experiments in animal models, may not always be predictive for results in man. Hence, to learn if stem cell therapy in MS is as effective and safe as suggested, the focus in R&D must shift from the lab to the clinic.

According to the 300 scientists gathered today, preconditions to this challenging research are:

-- autologous stem cells should be used (derived from patients' own bone marrow, skin, blood). Pre-clinical research shows that effectivenes and safety profile of treatment with autologous bone marrow cells are relatively positive, virtually no rejection occurs and ethical considerations are limited;

-- harvesting, culturing, purification and storage of the human stem cells should be performed in specialised labs under specific GMP-regulations;

-- procedures of clinical stem cell trials should be standardised and centrally coordinated, as well as reporting and central database management of all stem cell transplanted patients;

-- ethical issues should be addressed properly, e.g. how to trade-off the cost/benefit ratio, when the benefit is yet unclear at the given scientific status quo and certain risks cannot be ruled out, while the irreversibly ill patient has no other perspectives anymore.

Some scientists at the conference, pledged for more fundamental research before stem cell treatment in man should start at all. "It is beyond doubt that there is lots of research work to be done on the nature of brain damage and natural repair mechanisms of nerve tissue, on the interaction between immune system and stem cells and on various other aspects in this complex neurobiological arena.

However, there is growing, although sometimes inconclusive or casuistic evidence of clinical relevant brain-repair and protective properties of transplanted stem cells. Given the urgency of finding a cure for this widespread, disabling disease, most scientists argued that it is justifiable to arrange a rapid onset of well managed trials", concluded professor O.R. Hommes, chairman of the European Charcot Foundation.

"The proof of concept is available. Now it is time to proceed to the clinic. Therefore, we presented today a concept of a groundbreaking phaseI/II clinical trial, to be executed with some 60 severe MS patients in 5 or 6 centers across Europe. The goal is primarily to evaluate safety and feasibility of the stem cell treatment, and secondly to investigate the repair and protective effect in the brain, as can be monitored with MRI on a cellular level, and in reporting of development of the functional abilities of the patient - as the ultimate parameter," stated Hommes.

The outline of this decisive trial was thoroughly discussed during this scientific meeting.

European Charcot Foundation
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6584 GG Molenhoek (Nijmegen area)
The Netherlands