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Educating the next generation of scientists in translational medicine: Focus on eye diseases

Periodic Reporting for period 1 - transMed (Educating the next generation of scientists in translational medicine: Focus on eye diseases)

Reporting period: 2017-12-01 to 2019-11-30

Medicine today experiences a gap between promising results obtained in basic research and a successful translation into clinical practice. The transMed ETN programme is addressing this discrepancy by educating a new generation of “translational researchers” in a variety of areas relevant for clinical translation. The aim is to give the young researchers the broad overview that they will need to in the future move forward bench-to-bedside development of new treatments.
The medical context on which transMed focusses are neurodegenerative diseases of the retina, an area of research where on the one hand a large medical need exists and where on the other hand the intrinsic properties of the eye allow for relatively easy experimental interventions and direct visualization of outcomes. Moreover, the methods and techniques used in basic retinal research are very similar to what is used in clinical practice, facilitating translational work. Several of the transMed projects build on the predecessor EU-funded DRUGSFORD project in which novel compounds targeting the cGMP-signalling system were produced and tested.
In the 1st reporting period the four academic and three non-academic and industrial groups that make up the transMed consortium have recruited thirteen early stage researchers (ESRs) who work in four different areas of translational research, namely: 1) the design and application of new drugs to be used both for the treatment of retinal diseases and to establish disease mechanisms and targets, 2) the efficient formulation and delivery of such drugs, 3) their validation in various test systems, and 4) the pharmaceutical development of the active substance and formulation that will allow for manufacturing according to the standards of good manufacturing practice (GMP), at appropriate quantities needed for clinical development.
To date, the transMed ESRs have successfully identified new compounds with therapeutic potential and molecular probes that could be used for biomarker purposes. The ESRs also have worked on easier to use cell culture models and have produced and evaluated new nanoscale formulations designed to facilitate entry of therapeutic compounds or molecular biomarker probes into the neuroretina. transMed ESRs have worked on the ocular pharmacokinetics of such compounds and formulations, and developed corresponding new manufacturing and purification processes for their large-scale production.
In parallel to their research work, the transMed ESRs participated in theoretical and practical hands-on training courses held across Europe, at the laboratories of the various academic and industrial partners. They also attended the Vision Camp conference dedicated to give young researchers an international venue for their presentations and networking, and performed a number of inter-sectoral secondments from industry to academia and vice versa, permitting further insight and networking in the European biotech industry.
The transMed consortium has already in the 1st reporting period produced research results that go beyond the current state-of-the art. For instance, new formulations for neuroretinal compound delivery after intraocular application were obtained, new production and purification techniques were developed with higher material yield at lower costs. Moreover, a new approach for the non-invasive in vivo detection of retinal neurodegeneration was conceived, which could potentially be turned into a new kind of biomarker that may strongly facilitate clinical diagnostics and drug testing in the future. In the 2nd part of the transMed project the inventive combination of these results, e.g. new compounds with new formulations and production methods, may result in significant further advances, and possibly the generation of corresponding patent applications.
Eventually this has the outcome of giving back to essential academic and non-academic stakeholders in the society: By improving transitions from bench-to-bedside, by increasing market share and innovative capacity of the biotech/pharma industry, by improving and broadening academic research, and, most importantly, by providing new treatments and diagnostic tools for patients with retinal diseases. On a more political level, transMed is helping to strengthen the industrial and academic development in the EU13 member state Czech Republic by giving the SME INCU and the Czech nanotechnology sector a bridge to universities and companies from traditionally high-performing member states. Together, a stimulation of company growth and creation of new workplaces for highly skilled biomedical graduates can be foreseen. The successful realization of transMed also increases awareness of industrial-academic co-education in EU13 member states.
Finally, transMed enables PhD students to learn about the diversity of Europe by experiencing the culture and national heritage of the participating countries, and realizing the benefits – for science in particular and the society in general – of common values and free movement within the EU. Altogether, transMed is offering its students the opportunity to obtain a competitive PhD degree in several critical areas of biomedical research, providing for a strong employability in both the private and public sector of European research.