With sponsorship from CARRA and the Arthritis Foundation, Maya Breitman and Theresa Wampler-Muskardin attended the Utrecht University Summer School course “Why Translational Medicine Fails-And What to do About it” this past July in the Netherlands. This course was created and is directed by the EUREKA Institute of Translational Medicine (http://eurekainstitute.org). EUREKA is a not-for-profit organization established in 2008 by leaders in the field of translational medicine (including pediatric rheumatologist Berent Prakken).
“The birth of Eureka arises from the realization that the itinerary from molecular to clinical medicine requires a seamless trajectory to insure that talent, ideas, and potential cures are captured. An important resource and one that is a fundamental unmet need is human capital: a person who is a builder of bridges, who has the knowledge, the skill and the desire to navigate the challenges and components of this complex field. System-wide networks which can inspire, catalyze and sustain Translational Medicine on an international scale simply do not exist. These unmet needs are perceived as the main hurdle to the integration between Molecular and Clinical Medicine into a seamless gradient leading to a patient-centered approach to healing (Precision Medicine).” (http://eurekainstitute.org/about-us/)
As part of their education and community building effort, EUREKA (jointly with the Science in Transition team from UMC Utrecht) offers the summer school course, which focuses on translational medicine issues in rare and orphan diseases. Designed with insights gleaned through EUREKA’s experience, the summer school course masterfully tackles the intrinsic difficulties of effective translational medicine in rare and orphan diseases. Whereas EUREKA’s Certificate course is intended for more established investigators, the Utrecht Summer School course is meant for early stage researchers. The days are full but high-yield, interactive, and truly engaging.
During the course they heard from Dr. Frank Miedema, UMC Utrecht dean and initiator of Science in Transition (http://scienceintransition.nl/english). Science in Transition is a Dutch initiative in which four distinguished scientists gave a piercing analysis of the current state of Dutch universities. The “current state” is really the same in the U.S.: “Current incentives combined with hyper competition for limited funds have severe negative effects: many publications of poor quality and limited societal impact; risk aversion and avoiding complex, multidisciplinary problems; systematic under-appreciation of education and other academic duties; very poor career perspectives for young scientists.” Science in Transition received a lot of attention from public, policy makers and scientists, and started a tide of change in the Netherlands. As early stage clinician-scientists in the U.S., learning about Science in Transition was inspiring and empowering. Through the remainder of the course we were able to gain insight into how modern science works and were able to reflect upon their own role, expectations and potential actions we may want to make to achieve our goals as scientists.
Working with their classmates and experienced translational researchers in rare disease, we got to know the road from bench to bedside guided by a real-life case. We learned about their personal communication styles and had practice collaborating across disciplines and cultures. They learned to appreciate and align different interests of different stakeholders. The course emphasized the importance of collaboration across disciplines and institutes to benefit patients and the importance of patient/parent involvement in all stages of research. We learned why protecting intellectual property is crucial to successful innovation and translational medicine, and gained insights from others in navigating innovation between academia and industry. The fact that science is team work was reinforced and we learned about leadership development in translational medicine.
During the course, we had time directed toward personal reflection. This allowed us to create our own “personal manifesto” and start our own leadership and innovation toolkit within the context of translational medicine. We had personal mentoring by experienced senior mentors, and were able to have inspirational speed counseling with several established and successful junior clinical scientists.
The focus on rare diseases was perfect for translational researchers who focus on pediatric inflammatory and rheumatic diseases. It was an outstanding opportunity to learn from leaders in translational medicine in rare diseases and to connect with others who desire to do the same. The course exceeded their expectations. Not only were we able to learn about the latest developments in the field of translational medicine but we were also given excellent opportunity to better understand the interconnection between the basic science to the clinical aspects of translational medicine. For translational scientists involved mostly in basic research, the course also delivered new insights, tools and competence to navigate the difficult track from the bench to the bedside. This course is a wonderful opportunity to make new connections with other translational medicine researchers from around the globe and actually build a foundation for future collaboration. We both highly recommend this course to anyone who has a desire to pursue a career in translational medicine, and are tremendously grateful to CARRA and the Arthritis Foundation for the support to be able to attend!
Maya Breitman, Ph.D., and Theresa Wampler-Muskardin, M.D.
Drs. Hanna Kim, Rebecca Sadun, and Grant Schulert were each selected for the CARRA-PReS Travel Award, which covers up to $2500 in travel expenses related to attending the Paediatric Rheumatology European Association (PReS) Annual Scientific Congress (September 14- 17, 2017) and the preceding Young Investigators Meeting (YIM) (September 13-14, 2017), both in Athens, Greece. At the YIM, all young investigators had the opportunity to present their research. All three travel award winners also presented their research at the main PReS Scientific Congress. In addition, on behalf of the CARRA Early Investigator (EI) group, Hanna Kim presented an overview of both CARRA and the CARRA EI group, including discussing opportunities for international member involvement in CARRA.
Drs. Kim and Sadun also represented the United States at the PReS Trainee Working Group, where they learned more about the newly formed group for PReS Trainee members called EMERGE (EMErging RheumatoloGists and rEsearchers). One key new initiative they have developed is the PRES EMERGE fellowship, which provides financial support for a fellow from any country to visit an institution in Europe to gain additional training in a clinical or research domain; more information on this opportunity can be found at http://www.pres.eu/activities/young-investigators/fellowship-programs.html. EMERGE is also developing a mentorship program similar to CARRA’s AMIGO program.
In addition, EMERGE is interested in developing several other initiatives, including an immunology “boot camp,” an interactive way to provide research/grant development support for young investigators, and a reciprocal travel scholarship to permit PReS early investigators to attend CARRA. Attendees were intrigued to hear about CARRA’s experiences with the AMIGO program and the Aims page from grant proposal review available through the CARRA EI group. The international audience of primarily European trainees and early investigators expressed significant interest in becoming CARRA members and opportunities to attend the next CARRA Annual Meeting. Dr. Sadun noted, “The young investigators present were grateful for our participation and collaboration as they work to form support structures for pediatric rheumatology trainees world-wide. We were glad to help CARRA be part of that conversation.”
Dr. Sadun presented her work on “Teaching adult rheumatology fellows to help young adult patients ‘stick the landing’ when transferring from pediatric to adult rheumatology care.” Dr. Kim presented on “Novel serum broad-based proteomic discovery analysis identifies proteins and pathways dysregulated in juvenile dermatomyositis (JDM).” Dr. Schulert presented on “Single cell RNA-sequencing of bone marrow macrophages identifies a distinct subpopulation in systemic JIA with features of interferon response, endocytic vesicles and phagocytosis.” Of note, Dr. Schulert was awarded the gold medal for best abstract by an early investigator.
All three early investigators reported appreciating the opportunity through the CARRA-PReS Travel Award to discuss their research with an international audience of peers and established researches, including sharing new ideas to improve the research and promote collaboration. Dr. Kim stated, “It was a great opportunity to present my research to distinguished physician scientists from around the world, making connections with them face to face.” According to Dr. Schulert, “This was an invaluable experience both to increase the visibility of my research, and to nurture collaborations with international partners.” Dr. Sadun added, “The PReS conference itself was wonderful, as was the opportunity to engage with other young investigators, from across the globe, who are dedicated to advancing the science that guides the field of pediatric rheumatology.