Periodic Reporting for period 2 - transMed (Educating the next generation of scientists in translational medicine: Focus on eye diseases)
Periodo di rendicontazione: 2019-12-01 al 2021-11-30
In medicine today there is a clear gap between a wealth of promising results obtained in basic research and their successful translation into clinical practice. To bridge this gap, the transMed ETN programme educated a new generation of “translational researchers” in a variety of areas key for successful clinical translation. This included training on disease mechanisms, biomarkers, pharmaceutical development, regulatory affairs, clinical trials, and many other topics, along with the respective techniques and technology. In this way the young researchers were provided with a broad overview, which in their future careers will enable them to design and forward the bench-to-bedside development of new treatments.
The medical field on which transMed focussed was neurodegenerative diseases of the retina. This is 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. transMed work extended significantly beyond this state-of the-art and, for instance, further promoted the development of compounds and their formulations towards clinical applications.
The medical field on which transMed focussed was neurodegenerative diseases of the retina. This is 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. transMed work extended significantly beyond this state-of the-art and, for instance, further promoted the development of compounds and their formulations towards clinical applications.
The four academic and three non-academic and industrial groups that made up the transMed consortium had recruited thirteen early stage researchers (ESRs) who worked in four different areas of translational research, namely: 1) design and application of new drugs to be used both for treatment of retinal diseases and identification of disease mechanisms and targets, 2) efficient formulation and delivery of such drugs, 3) their validation in various test systems, and 4) pharmaceutical development of active substance and formulation to allow for manufacturing according to the standards of good manufacturing practice (GMP), at appropriate quantities needed for clinical development. These four research areas were combined with an innovative training programme covering all aspects of translational medicine that were difficult to cover by research projects directly and that also included a variety of different soft skills. This concerned among others clinical trial design, scientific writing, career development, research policy, etc.
In the further progress of the transMed project its ESRs have successfully identified new compounds with therapeutic potential, as well as new molecular probes that can be used for biomarker purposes. The ESRs also worked on new and more accessible cell culture models and produced and evaluated new nanoscale formulations designed to facilitate entry of therapeutic compounds or molecular biomarker probes into the neuroretina. transMed ESRs worked on the ocular pharmacokinetics of such compounds and suitable 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. Unfortunately, the COVID-19 pandemic has restricted travels to consortium meetings, conferences, and secondments. The transMed consortium compensated for this by resorting to virtual meetings and promoting information exchanges through video-conferencing or data sharing platforms. Even so, the transMed ESRs could attend a number of in-person events, including the Vision Camp conference that was dedicated to give young researchers an international venue for their presentations. Moreover, most ESRs still managed to perform two inter-sectoral secondments from industry to academia and vice versa, permitting further insight and networking in the European biotech industry.
The results of the transMed project were published in over 20 peer-reviewed scientific journals, with many further publications still set to come out in 2022/23. Project results were also publicly presented in national and international conferences (in person and virtual) in the form of posters or talks. Furthermore, the ESRs also participated and actively organized themselves numerous public outreach activities such as lectures in high-schools and for the general public, virtual patient information days, etc.
In the further progress of the transMed project its ESRs have successfully identified new compounds with therapeutic potential, as well as new molecular probes that can be used for biomarker purposes. The ESRs also worked on new and more accessible cell culture models and produced and evaluated new nanoscale formulations designed to facilitate entry of therapeutic compounds or molecular biomarker probes into the neuroretina. transMed ESRs worked on the ocular pharmacokinetics of such compounds and suitable 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. Unfortunately, the COVID-19 pandemic has restricted travels to consortium meetings, conferences, and secondments. The transMed consortium compensated for this by resorting to virtual meetings and promoting information exchanges through video-conferencing or data sharing platforms. Even so, the transMed ESRs could attend a number of in-person events, including the Vision Camp conference that was dedicated to give young researchers an international venue for their presentations. Moreover, most ESRs still managed to perform two inter-sectoral secondments from industry to academia and vice versa, permitting further insight and networking in the European biotech industry.
The results of the transMed project were published in over 20 peer-reviewed scientific journals, with many further publications still set to come out in 2022/23. Project results were also publicly presented in national and international conferences (in person and virtual) in the form of posters or talks. Furthermore, the ESRs also participated and actively organized themselves numerous public outreach activities such as lectures in high-schools and for the general public, virtual patient information days, etc.
The transMed consortium produced numerous research results that went beyond the previous 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. The identification of at least one new compound with retinoprotective properties has led to interest from a pharmaceutical company outside the consortium and negotiations are currently under way that aim at progressing this new compound to the clinical trial stage. Moreover, a new approach based on the detection of calpain-activity was conceived for the non-invasive in vivo detection of retinal neurodegeneration. Potentially this new approach could be used as new kind of biomarker, to facilitate clinical diagnostics and drug testing in the future. The inventive combination of these results, e.g. new compounds or biomarkers with new formulations and production methods, gave rise to further advances, several of which are still being explored even beyond the duration of the transMed project.
Ultimately all these results and efforts will give back to essential academic and non-academic stakeholders in society: Accelerating bench-to-bedside transitions will benefit patients and industry alike, and will increase market share and innovative capacity of the biotech/pharma industry. At the same time academic research will benefit from inter-sectoral connections and networks, and gain a better understanding for patient and industry needs and requirements. Apart from this, transMed also fostered 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, this has stimulated company growth and the creation of new workplaces for highly skilled biomedical graduates, and has increased the awareness of industrial-academic co-education in EU13 member states.
Finally, transMed provided its PhD students with a unique opportunity 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. Through all this, transMed offered its students the opportunity to obtain a competitive PhD degree in critical areas of biomedical research, providing for a strong employability in both the private and public sector. Accordingly, and as of to date (February 2022), all transMed ESRs have successfully transitioned to new positions in either academia or industry, an outcome that may be highlighted as the ultimate testament to transMed´s success.
Ultimately all these results and efforts will give back to essential academic and non-academic stakeholders in society: Accelerating bench-to-bedside transitions will benefit patients and industry alike, and will increase market share and innovative capacity of the biotech/pharma industry. At the same time academic research will benefit from inter-sectoral connections and networks, and gain a better understanding for patient and industry needs and requirements. Apart from this, transMed also fostered 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, this has stimulated company growth and the creation of new workplaces for highly skilled biomedical graduates, and has increased the awareness of industrial-academic co-education in EU13 member states.
Finally, transMed provided its PhD students with a unique opportunity 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. Through all this, transMed offered its students the opportunity to obtain a competitive PhD degree in critical areas of biomedical research, providing for a strong employability in both the private and public sector. Accordingly, and as of to date (February 2022), all transMed ESRs have successfully transitioned to new positions in either academia or industry, an outcome that may be highlighted as the ultimate testament to transMed´s success.