Working Group 4 - Diagnostic & therapeutic potential of ME

WG leader: Bernd Giebel

Co-WG leader: Stefano Fais

Aims:

Translation of preclinical findings into medical applications is of upmost socio-economical impact but generally suffers from poor communication and exchanges between researchers, clinicians and industries. This Action will provide the necessary platform to facilitate the transfer of knowledge between these groups, advancing on successes from appropriate research performed by individual groups scattered throughout Europe.

Working closely with WGs1-3 and, again, bringing together “like-minded” researchers with a particular interest in ME as either biomarkers for a given disease and/or as therapeutic delivery vectors, will help ensure that future studies, facilitated by ME-HAD, are not duplications of disparate and small under-powered studies, but collaborative efforts that can lead to meaningful outcomes. Furthermore, it is realistic to envisage that when the therapeutic potential of ME has been further explored by ME-HAD researchers, this Action will act as the foundation, even beyond its 4 years, for designing inhibitors of ME release as potential therapies for conditions where such an approach would be relevant.

Tasks:

Advancing our collaborative research on ME as minimally-invasive biomarkers

Results from a number of studies by Action Participant and involving a range of diseases including cancer and Alzheimers indicate that both ME quantities and contents (proteins, nucleic acids) have relevance as minimally-invasive biomarkers.
As for all other WGs, former suitable data will be collated, evaluated and used as a foundation for more extensive collaborative studies on relevantly-sized cohorts of cases for more extensive characterising and quantification of ME isolated from human body fluids, in order to identify/validate disease-specific biomarkers (and to set standards for patientspecific biomarkers required for personalized therapies).

Progressing research on ME as therapeutic delivery vectors

The demonstration in preclinical studies, performed by Action participants, that ME can deliver molecules into secondary/recipient cells and that it is possible to pay-load ME or synthetic ME-like vesicles with desired functional siRNA or other therapeutic molecules, supports these vesicles being considered as “biomimetic” vectors for therapeutic delivery in, theoretically, any diseases.
Advancing on previous successes, ME-HAD researchers will accelerate scientific progress in this space though fostering cooperation across WGs and disciplines.
Of course the chances that, in the future, these findings will be translated to the clinic highly depend on the level of communication between basic, pre-clinical and clinical scientists and industry.
This Action will substantially facilitate progress here by enabling collaborative progress, synergism and valorisation of output, through the extensive interaction with the European research groups active in this and relate fields.

Designing inhibitors of ME release as therapy

COST Action researchers have established that in neurodegenerative diseases, inhibition of exosomes could ameliorate amyloid pathology.
A similar approach may be very relevant in prevention of cancer spread.
Through the Actions academic/industry collaborations, inhibitor of ME release will be designed and screened via miniaturised exosomes-based assays, starting during the 4 years of this Action and progressing beyond this to larger scale application.