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<strong>Translational</strong><br />

<strong>Medicine</strong><br />

at the University<br />

Medical Center<br />


<strong>Translational</strong> <strong>Medicine</strong>…<br />

… is the process of translating research outcomes to tangible products that<br />

can be used in clinical practice;<br />

… also works the other way around: research questions should arise from<br />

clinical care and unmet medical needs;<br />

… is teamwork, and requires collaboration between researchers, clinicians,<br />

patient organizations, and industry;<br />

… unites the knowledge of these different fields and experts to increase the<br />

societal relevance of research and ultimately improve patient care.<br />

UMC Utrecht 1

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2 <strong>Translational</strong> <strong>Medicine</strong>

<strong>Translational</strong> <strong>Medicine</strong> at the University Medical Center Utrecht<br />

Berent Prakken<br />

Vice-dean for Education, UMC Utrecht, and Co-founder of the Eureka Institute<br />

One day, my friend and colleague Salvo Albani and<br />

I realized how many mistakes we had made while<br />

trying to develop immunotherapy. We committed<br />

ourselves to prevent young scientists from making the<br />

same mistakes. This started a long and unforeseen<br />

journey. We met like-minded people along this<br />

journey, and together we started the Eureka Institute.<br />

The aim of Eureka is to create a community of<br />

<strong>Translational</strong> <strong>Medicine</strong> professionals to increase the<br />

impact of science on patients. The initial<br />

accomplishment of Eureka was our certificate course,<br />

an international course in worldwide <strong>Translational</strong><br />

<strong>Medicine</strong> that was first offered in 2009.<br />

The mission of the University Medical Center Utrecht<br />

(UMC Utrecht) is to generate, evaluate, share and apply<br />

knowledge about health, illness, and care for the<br />

benefit of patients and society. This is in line with the<br />

vision and ambitions of Eureka. From that perspective,<br />

it was no surprise that UMC Utrecht joined<br />

Duke-National University of Singapore Medical School<br />

in becoming the first university partners of Eureka.<br />

Today, <strong>Translational</strong> <strong>Medicine</strong> is blooming at<br />

UMC Utrecht, in alignment with the philosophy of<br />

Science in Transition. In this book, you will find<br />

examples of that. But above all, it gives voice to<br />

scientific role models, most of whom are Eureka<br />

alumni. They explain what <strong>Translational</strong> <strong>Medicine</strong><br />

means to them, and how it is incorporated in their daily<br />

work. They share their expectations and hopes for the<br />

future. May their words convince you that we can<br />

increase the impact of biomedical research, and that<br />

we indeed can, and will, make good on our promise of<br />

improving patient’s lives!<br />

www.umcutrecht.nl/translationalmedicine<br />

www.eurekainstitute.org<br />

UMC Utrecht 3

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4 <strong>Translational</strong> <strong>Medicine</strong>

Science in Transition<br />

Frank Miedema<br />

Dean and Vice-Chairman of the Board, UMC Utrecht, and Co-founder of Science in Transition<br />

With the promise of new treatments and therapies,<br />

<strong>Translational</strong> <strong>Medicine</strong> is at the heart of biomedical<br />

research. But for a host of reasons, it may not always<br />

get the appreciation it deserves. With the Science in<br />

Transition initiative, which is rooted in UMC Utrecht<br />

and started in 2013, we aim to shed light on the<br />

incentive and reward system in biomedical science.<br />

We hope to push researchers to value efforts that bring<br />

results from the laboratory into clinical practice over<br />

publications in peer-reviewed journals.<br />

Science in Transition is one voice in a choir. Many<br />

analyses strongly suggest that current incentives,<br />

combined with hyper-competition for limited funds,<br />

contribute to the reproducibility crisis; to waste in<br />

research; and to poor translation of promising findings<br />

to clinical application. The inescapable conclusion is<br />

that we need new rewards and incentives for scientists,<br />

and that we should involve societal stakeholders in<br />

setting the research agenda. This conclusion and<br />

possible solution is echoed in the European Union’s<br />

‘Open Science’ agenda for science policy.<br />

UMC Utrecht takes its responsibility and intends to<br />

cultivate first-rate biomedical research and innovation.<br />

With Science in Transition, we make a plea for science<br />

that produces socially robust knowledge. We want to<br />

produce knowledge that answers relevant questions<br />

and benefits society. In the field of medicine, this<br />

means we have to make research ‘translational’.<br />

That is why I fully support Eureka and the efforts at<br />

UMC Utrecht to improve and expand <strong>Translational</strong><br />

<strong>Medicine</strong>.<br />

www.scienceintransition.nl<br />

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“In my research, I collaborate with fundamental researchers, clinical researchers,<br />

and with other disciplines – for example with people who build bridges between<br />

research, clinical practice and society.”<br />

Sanne Nijhof<br />

Pediatrician and Postdoctoral Researcher<br />

UMC Utrecht 7

“Wietse Kuis is my role model, because he has a brilliant mind, is honest, is engaged<br />

with what he does, feels for his patients, and wants to dive into details in the lab to<br />

understand everything he sees in his patients.”<br />

Joost Swart<br />

Pediatric Rheumatologist and Immunologist<br />

8 <strong>Translational</strong> <strong>Medicine</strong>

UMC Utrecht 9

10 <strong>Translational</strong> <strong>Medicine</strong>

“The main problem is an increased gap between the amount<br />

of data that we generate in research and the ability to apply<br />

it into clinical practice, in a meaningful and impactful way.”<br />

Salvatore Albani<br />

Director of <strong>Translational</strong> Immunology Institute, Professor, Duke-NUS Graduate Medical School<br />

Singapore, Co-founder of the Eureka Institute for <strong>Translational</strong> <strong>Medicine</strong><br />

“With our broad scope, we try to cover the whole<br />

gradient of <strong>Translational</strong> <strong>Medicine</strong>, ranging from<br />

molecular biology to new products. This is very rare to<br />

find. The course offered by Eureka differs from most<br />

programs because of the international setting, and the<br />

way it is adapted to promote critical thinking and<br />

create awareness.” Most of all, Salvatore Albani wants<br />

to emphasize that Eureka is more than a program.<br />

“It establishes collaborations, it creates an international<br />

network. One could talk about the individual<br />

achievements, but Eureka is all about a group quest.<br />

In my opinion, the main problem in the medical world<br />

is an increased gap between the amount of data that<br />

we generate in research and the ability to apply it into<br />

clinical practice, in a meaningful and impactful way,”<br />

Salvatore explains. That is the reason why Salvatore<br />

and his colleagues founded the Eureka Institute: “to<br />

contribute to the growth of <strong>Translational</strong> <strong>Medicine</strong> in<br />

a way that is directly impactful to patients.”<br />

www.eurekainstitute.org<br />

UMC Utrecht 11

12 <strong>Translational</strong> <strong>Medicine</strong>

“I am very proud to be building the infrastructure, setting up the network, and<br />

obtaining the funding to study several diseases that have frustrated me in the clinic.<br />

I hope to provide better answers and therefore better treatments and guidance in<br />

the near future.”<br />

Michiel Schreuder<br />

Pediatric Nephrologist, Radboud University Medical Center, Amalia Children’s Hospital, Nijmegen<br />

UMC Utrecht 13

“I am most proud of having initiated and continued to build the Scientific Alliance<br />

between the Wilhelmina Children’s Hospital and Nutricia Research, with a focus on<br />

immune development and nutrition.”<br />

Belinda van ‘t Land<br />

Affiliated Senior Scientist<br />

14 <strong>Translational</strong> <strong>Medicine</strong>

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16 <strong>Translational</strong> <strong>Medicine</strong>

“The future of <strong>Translational</strong> <strong>Medicine</strong> will be especially<br />

bright if we can direct our combined efforts towards<br />

diseases with a large burden in difficult-to-reach<br />

populations around the world.”<br />

Joyce Browne, Assistant Professor, Global Health<br />

Lilly Verhagen, Postdoctoral Researcher and Pediatric Resident, Global Health<br />

“<strong>Translational</strong> <strong>Medicine</strong> is a continuous process in which<br />

you try to solve a problem in a lab setting, and then<br />

translate that back to your patients. We see whether it<br />

works, and go through the whole routine again until we<br />

reach clinical impact, rather than using a one-step bench<br />

to bedside translation.” In Lilly’s case, the focus is on<br />

respiratory tract infections and vaccination in global<br />

pediatric risk groups. These patients could be at risk<br />

because they live in a setting with low resources, or it<br />

could be a group of patients in The Netherlands with<br />

immune deficiencies - with an immune system that fails to<br />

properly protect against infections. “For me, one of the<br />

challenges is to tailor our vaccination strategies to the<br />

specific risk groups across the world, rather than the<br />

one-size-fits-all approach that we currently take:<br />

embracing rather than masking differences between<br />

populations. Joyce adds, ”I made the decision to focus on<br />

Global Health education and research. I want to focus on<br />

areas where the burden is high, and where I feel I can add<br />

the most value and impact with what I do. For<br />

<strong>Translational</strong> <strong>Medicine</strong>, this means thinking about how we<br />

can make innovations available at the bedside of patients<br />

all over the world. One of my research projects focuses on<br />

a ‘poly pill’, a pill that combines aspirin and calcium to<br />

prevent hypertensive disorders of pregnancy. This is one<br />

of the major killers of mothers and babies in the world. I<br />

set up the project to develop this pill for Low and Middle<br />

Income Countries. But at the moment, I am actually<br />

preparing a clinical trial in the Netherlands, using the same<br />

pill for Dutch women.” Both Lilly and Joyce agree that<br />

Global Health and <strong>Translational</strong> <strong>Medicine</strong> are related. “I<br />

would say that addressing global health issues requires<br />

the same methods and techniques in all fields and<br />

settings, plus some context-specific logistical challenges”,<br />

says Lilly. “You need to cross political, administrative,<br />

linguistic and cultural boundaries to address the patient’s<br />

most relevant questions. You need to look beyond the<br />

borders of your country,” adds Joyce.<br />

UMC Utrecht 17

18 <strong>Translational</strong> <strong>Medicine</strong>

“<strong>Translational</strong> <strong>Medicine</strong> requires teams consisting of clinicians and fundamental<br />

researchers, and aims to translate findings with the help of all team members to<br />

ensure that patients can benefit.”<br />

Sylvia Brugman<br />

Assistant Professor,<br />

Cell Biology and Immunology Group, Wageningen University<br />

UMC Utrecht 19

“I am most proud of having created an organoid-based screening platform that uses<br />

patient-specific organoids as models of disease.”<br />

Sabine Middendorp<br />

Associate Professor, Pediatric Gastroenterology<br />

20 <strong>Translational</strong> <strong>Medicine</strong>

UMC Utrecht 21

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22 <strong>Translational</strong> <strong>Medicine</strong>

“In my heart I am still a basic scientist, but it is amazing what<br />

kind of impact you can have on peoples’ lives.”<br />

Jeffrey Beekman<br />

Group Leader, <strong>Translational</strong> Research on Cystic Fibrosis, UMC Utrecht<br />

Recently, Jeffrey Beekman was one of the speakers at<br />

the largest conference on Cystic Fibrosis (CF) in the<br />

United States. Many parents of children with CF were<br />

present. “The vibe at this conference gave me goose<br />

bumps. I could feel the response to our research when<br />

everyone saw our experiments for the first time. When<br />

I talked to the parents afterwards, I learned that our<br />

research really had a large impact. It still gives me<br />

goose bumps.”<br />

Jeffrey has not always worked in the field of<br />

<strong>Translational</strong> <strong>Medicine</strong>. “As a basic scientist, you focus<br />

on scientific concepts and questions. Basic research is<br />

obviously needed, but I wanted to have more impact<br />

on patients’ lives. That is why I switched to the field of<br />

CF.” Jeffrey now collaborates with scientists from<br />

different disciplinary backgrounds. His team designed<br />

a test focussing on stem cells that could potentially be<br />

used for CF treatment. ”Together with the CF patient<br />

organisation we co-fund a program to enable<br />

individualized therapy for CF. What I like about having<br />

direct patient involvement: they keep you oriented on<br />

the impact of research for the patient. In the end, with<br />

our research we provide a service for the community.<br />

In my heart I am still a basic scientist, but it is amazing<br />

what kind of impact you can have on peoples’ lives.<br />

You hope and dream about what you may achieve, but<br />

this time it turned out to be even better in real life.”<br />

UMC Utrecht 23

24 <strong>Translational</strong> <strong>Medicine</strong>

“I hope the future of <strong>Translational</strong> <strong>Medicine</strong> will be bright and based on teamwork.<br />

In my view, the only way to succeed is to bring together people who have the<br />

creativity and freedom to think outside the box.”<br />

Hester den Ruijter<br />

Associate Professor, Experimental Cardiology<br />

UMC Utrecht 25

“The solution to major medical problems can often only be solved by a <strong>Translational</strong><br />

<strong>Medicine</strong> approach.”<br />

Louis Bont<br />

Pediatrician and Researcher<br />

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28 <strong>Translational</strong> <strong>Medicine</strong>

Education and Research on <strong>Translational</strong> <strong>Medicine</strong><br />

at UMC Utrecht<br />

<strong>Translational</strong> <strong>Medicine</strong> is a complex and<br />

interdisciplinary field. Clinicians, researchers, patients,<br />

and industry all need to be involved to ask research<br />

questions that are clinically relevant and translate<br />

research outcomes to tangible products at the bedside.<br />

<strong>Translational</strong> scientists, who are either PhDs with an<br />

interest in clinical research or clinician-scientists, need<br />

to obtain a broad range of skills to navigate the<br />

translational pathway. They need to learn how to work<br />

effectively in interdisciplinary teams and communicate<br />

with a broad range of stakeholders. Clinician-scientists<br />

have a dual role, as they are trained in both research<br />

and clinical practice, and can therefore play an<br />

important role in further closing the gap between<br />

research and clinical practice.<br />

UMC Utrecht invests in educating the next generation<br />

of translational scientists and in research to further<br />

improve training and career pathways for translational<br />

scientists. This is necessary, as becoming a successful<br />

translational scientist or clinician-scientist is<br />

challenging. The competitive funding system, the<br />

pressure to publish, and the lack of training and career<br />

pathways make this a challenge .<br />

The educational strategy of UMC Utrecht shows that<br />

educating translational professionals is of high<br />

importance for UMC Utrecht. This is illustrated by its<br />

focus on inter-professional education, strengthening<br />

the connection with patients, improving patient<br />

participation in education, and creating awareness of<br />

the societal impact of research.<br />

The Selective Utrecht Medical Master (SUMMA) is a<br />

concrete example of this, as it focuses on the training<br />

of clinician-scientists. The four-year SUMMA program is<br />

for students with a Bachelor’s degree in biomedical or<br />

life sciences, and combines a medical degree with a<br />

degree in clinical science. After four years, SUMMA<br />

graduates are prepared for a career that combines<br />

UMC Utrecht 29

clinical practice with research activities. These<br />

graduates can play an important role in the translation<br />

of research outcomes to patient care.<br />

UMC Utrecht also participates in further improving and<br />

developing the education of translational scientists<br />

and clinician-scientists in an international context.<br />

Crucial for this development is the partnership with<br />

the Eureka Institute. Together with Eureka, the<br />

UMC Utrecht embarked on several initiatives.<br />

In collaboration with University College London,<br />

University of Granada, Ghent University and Nutricia<br />

Research, UMC Utrecht successfully applied for a<br />

European Erasmus+ grant that resulted in the<br />

‘PATHWAY project’. The objectives of this project are to<br />

create efficient and sustainable career pathways for<br />

clinician-scientists, to integrate research and clinical<br />

components in the training of clinician-scientists, and<br />

to generate impact by raising awareness amongst<br />

stakeholders throughout Europe. Altogether, this<br />

contributes to an overall aim to bridge the gap<br />

between bench and bedside by training clinicianscientists<br />

for the academic and industrial workforce.<br />

In 2015, UMC Utrecht and the University of Toronto<br />

started an international research collaboration to<br />

support the clinician-scientist workforce. Research on<br />

the professional identity development, training, and<br />

career pathways of clinician-scientists is needed, as this<br />

workforce is both ageing and declining. The aim is to<br />

investigate how clinician-scientists develop their<br />

professional identity around their dual roles in both<br />

clinical practice and research, and how training and<br />

career pathways should improve to support<br />

early-career clinician-scientists. These studies are<br />

performed within the Eureka Network. The Julius<br />

Center for Health Sciences and Primary Care also<br />

performs research on general practitioners who<br />

become clinician-scientists, because a need for an<br />

increasing number of professionals with a research<br />

degree has also been identified within primary care.<br />

Together, the educational and research activities that<br />

take place within UMC Utrecht aim to improve the<br />

education and career support for these professionals,<br />

who are vital players in the field of <strong>Translational</strong><br />

<strong>Medicine</strong>.<br />

30 <strong>Translational</strong> <strong>Medicine</strong>

UMC Utrecht 31

32 <strong>Translational</strong> <strong>Medicine</strong>

“I am most proud of having contributed to the training and mentoring of young<br />

professionals and scientists, each with their own career paths. They are going to<br />

make the difference.”<br />

Femke van Wijk<br />

Associate Professor<br />

UMC Utrecht 33

“At the Neonatal Intensive Care Unit, continuous improvement in care for these<br />

small and very vulnerable human beings is mandatory to improve long-term<br />

outcome and quality of life. Historically, this population has been underrepresented<br />

in basic science and therapeutic research. I feel a responsibility to make research on<br />

neonates possible in the best clinical way.”<br />

Sanne Hoeks<br />

Pediatrician and Neonatologist<br />

34 <strong>Translational</strong> <strong>Medicine</strong>

UMC Utrecht 35

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36 <strong>Translational</strong> <strong>Medicine</strong>

“There is a bright idea or healthcare innovation and a firstin-man<br />

study has been done, so it is beautifully translated<br />

from the lab to human. But then, boom, still nobody knows<br />

whether it works or has impact in daily medical practice.”<br />

Carl Moons<br />

Professor of Clinical Epidemiology, Julius Center for Health Sciences and Primary Care<br />

“From my perspective, we can only speak of<br />

<strong>Translational</strong> <strong>Medicine</strong> when research results are<br />

applicable to the end-users. These end-users would<br />

include patients or care providers, and not only<br />

research going from the bench to the first-in-man<br />

studies.” For Carl it is obvious, “Medical science is often<br />

too discovery-driven. It is sexy to discover things or<br />

develop innovations, but not to validate or test these<br />

discoveries in the context for which the innovation is<br />

eventually to be applied. And that is what is needed<br />

before taking wide scale implementation of any<br />

innovation into practice. I used to be the same, in a<br />

way. We finished a phase 3 trial, described it in a<br />

beautiful paper, published it in a high impact journal<br />

and went on with the next trial. What I should have<br />

done is pick up the result, further explain it where<br />

needed, and bring it to the relevant stakeholders to<br />

make sure it entered the clinical guidelines.”<br />

Carl continues, “To me, <strong>Translational</strong> <strong>Medicine</strong> often<br />

seems less translational than it should be. Researchers<br />

and research teams often stop too early. From the start<br />

of the idea or conception of the innovation, we should<br />

work much more in multidisciplinary teams. This is<br />

needed in the entire chain to bring the idea into daily<br />

practice. Then <strong>Translational</strong> <strong>Medicine</strong> will be fully<br />

translational, and much more effective and impactful.<br />

It moves from discovery- and technology-driven to<br />

impact- and fit-for-purpose-driven. If you talk about<br />

decision support systems, for example, the end-users<br />

are you and I, and actually the whole population.<br />

Developing a decision support system or some<br />

e-health app is one thing, but evaluating whether it<br />

indeed has true impact on the health of the actual<br />

end-users in the intended context of the app is<br />

something else.”<br />

UMC Utrecht 37

38 <strong>Translational</strong> <strong>Medicine</strong>

“In my research, I try to keep in mind how patients can benefit from the results.<br />

I find this hard to accomplish, due to the fact that most research findings represent<br />

small steps ahead and need further research and optimizations before they can be<br />

really applied in daily clinical practice.”<br />

Pieter van Dijkhuizen<br />

PhD student, Pediatric Rheumatology<br />

UMC Utrecht 39

“I would call someone a translational scientist when the main objective of their<br />

research is to cross the ‘Valley of Death’ between promising pre-clinical results and<br />

making the drug or device part of daily clinical practice.”<br />

Roel Deckers<br />

Assistant professor, Imaging Division<br />

40 <strong>Translational</strong> <strong>Medicine</strong>

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42 <strong>Translational</strong> <strong>Medicine</strong>

“At the UMC Utrecht, people talk about how the translational<br />

pathway works and if we need to challenge that paradigm.<br />

That is part of the uniqueness of the UMC Utrecht.”<br />

Norman Rosenblum<br />

Staff Nephrologist at The Hospital for Sick Children, and Senior Scientist Developmental<br />

& Stem Cell Biology, University of Toronto<br />

Asked about the relevance of <strong>Translational</strong> <strong>Medicine</strong>,<br />

Norman Rosenblum answers, ”<strong>Medicine</strong> exists today at<br />

a time where there is potential to bring a variety of<br />

discoveries to the clinical context and to wider<br />

populations. Populations of not necessarily sick people,<br />

but even healthy people for whom we seek to optimize<br />

health. There is tremendous potential to do this<br />

because of real revolutions in the biological sciences<br />

over the last 30 years, as well as in information<br />

technology, population health sciences, and artificial<br />

intelligence. The old way of thinking was about<br />

bringing discoveries to the market, but we now focus<br />

on technology and patient interfaces, information, and<br />

healthcare models. The patient has become a real<br />

partner, whereas before the patient was a target.”<br />

“The University of Toronto plays a big role in the field<br />

of <strong>Translational</strong> <strong>Medicine</strong> as a top-20 university in Life<br />

Sciences and top-3 university in pediatrics,” Norman<br />

explains. “But the UMC Utrecht does not just talk about<br />

<strong>Translational</strong> <strong>Medicine</strong>; it is trying to promote it as a<br />

way for people to engage in their science and in the<br />

application of their science. They are asking about<br />

patient’s priorities, asking about waste in science.<br />

From the Dean down, they are willing to do it.”<br />

UMC Utrecht 43

44 <strong>Translational</strong> <strong>Medicine</strong>

“I am most proud of having brought together different people into a<br />

multidisciplinary group. All had something to bring to the table, and the<br />

outcome will be bigger than all the individual input.”<br />

Joris van Montfrans<br />

Pediatrician<br />

UMC Utrecht 45

“I would call myself a clinician-scientist because I am translating clinical findings<br />

into new research projects, and translating lab findings into potential clinical<br />

applications. I do this within my own field of interest as well as in the projects of my<br />

colleagues or researchers that I meet.”<br />

Marije Bartels<br />

Pediatric Hematologist<br />

46 <strong>Translational</strong> <strong>Medicine</strong>

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UMC Utrecht Eureka Summer Course<br />

on <strong>Translational</strong> <strong>Medicine</strong><br />

Participating in the Eureka/UMC Utrecht Summer<br />

Course on <strong>Translational</strong> <strong>Medicine</strong> is like entering a<br />

bubble. With a group of about 30 students and almost<br />

as many faculty members, you go through an intense<br />

week with a surprising mix of presentation formats and<br />

content, of social and scientific activities, and of regular<br />

and unorthodox teaching methods.<br />

And you are all in it together.<br />

The first thing you will notice is that the many<br />

inconvenient truths of the scientific enterprise are<br />

openly discussed. This reflects the fact that the<br />

UMC Utrecht is home to Science in Transition, an<br />

initiative that criticizes how incentives and rewards<br />

shape the quality and relevance of science.<br />

At the same time, inside the bubble you will find all the<br />

tools and knowledge to make a positive difference as a<br />

translational researcher. The dedicated faculty has an<br />

outstanding reputation as both translational scientists<br />

and as teachers. The curriculum is developed with the<br />

Eureka Institute, which has a decade of experience in<br />

<strong>Translational</strong> <strong>Medicine</strong>.<br />

But the most important part, of course, is your fellow<br />

travelers inside the bubble. They are young and<br />

motivated researchers from around the globe that<br />

meet each other in Utrecht with a common belief:<br />

that biomedical research can improve our lives, and<br />

that they can add to that.<br />

The inspiration and energy are contagious, and long<br />

after leaving the bubble you will feel the effects of the<br />

one-week course. You will no longer take ‘how science<br />

works’ for granted. You will feel empowered to make a<br />

change by reaching out to colleagues, peers and<br />

stakeholders, and together you will give biomedical<br />

research the impact that it should have.<br />

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UMC Utrecht Eureka Summer Course<br />

on <strong>Translational</strong> <strong>Medicine</strong><br />

Edition 2016<br />

“I am particularly impressed by the diversity of the groups and the fact that we will face the same<br />

situation when we would practice <strong>Translational</strong> <strong>Medicine</strong>. Also, I learned a lot about how to cope with<br />

challenges and hurdles. I will spread the concept of <strong>Translational</strong> <strong>Medicine</strong> in my home institution by<br />

writing an article in local journals.”<br />

Nikmah S Idris, Pulmonary Hypertension Clinical Fellow, Great Ormond Street Hospital and Paediatric<br />

Cardiology, Staff University of Indonesia.<br />

Alumna of the Summer Course, 2016 edition<br />

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UMC Utrecht Eureka Summer Course<br />

on <strong>Translational</strong> <strong>Medicine</strong><br />

Edition 2017<br />

“There are many examples of individuals and institutions who take the initiative to close the gap between research<br />

and clinical practice. However, it is impossible for individual institutions to change the system as a whole. During the<br />

summer school on <strong>Translational</strong> <strong>Medicine</strong> we came up with the idea of an ‘Impact Index’: a label that indicates what<br />

institutions already do to close the research-practice gap.<br />

This index would have two goals: to create transparency on ‘best-practices’ of institutions that already invest in<br />

improving the quality of research and translating research outcomes to the patient, and to encourage institutions<br />

that have fallen behind to invest in improving the quality of research, using the<br />

best-practices available.”<br />

Anneke Monninkhof, Nienke de Graeff, and<br />

Margot Weggemans<br />

Alumni of the Summer Course, 2017 edition<br />

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54 <strong>Translational</strong> <strong>Medicine</strong>

“I call myself a translational scientist because I directly implement the findings from<br />

our nephrogenetics research line to diagnostics, thereby translating research results<br />

to medical care.”<br />

Kirsten Renkema<br />

Assistant Professor, Genetics<br />

UMC Utrecht 55

“I am proud of the improved health care in the UMC Utrecht due to better<br />

collaboration in professional care teams and more focus on the patient perspective.”<br />

Petra Baarendse<br />

Senior Expert / Architect<br />

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“From the beginning to the end, it should be about patients.<br />

So they need to be involved in promising research as often<br />

and as soon as possible.”<br />

Ingrid Lether<br />

Manager of Research and Innovation at Reumafonds (arthritis patient organization and research funder)<br />

“Our organization tries to involve patients in the<br />

different phases of research. Patients are part of our<br />

scientific advisory board. They look at the feasibility<br />

and relevance of the proposals. If the result of a<br />

proposal will not change clinical practice, it will not<br />

benefit them. A proposal like that may be scientifically<br />

interesting, but the patients tell us what is really<br />

important. We also encourage researchers to set up<br />

their own groups of patients to help them draft<br />

relevant research questions. A patient can tell the<br />

researcher what is bothering him or her most, for<br />

example. This is of course the preferred option:<br />

involvement of patients before a proposal is written.<br />

We can also ask patients to bring in their perspectives<br />

at meetings with the government and health insurance<br />

companies, to inform our research. From the beginning<br />

to the end, it should be about the patients. So they<br />

need to be involved in promising research as often and<br />

as soon as possible,” Ingrid explains.<br />

In the Netherlands, arthritis researchers from Utrecht<br />

enjoy a good reputation, Ingrid thinks. “There is a<br />

concentration of knowledge about arthritis at Utrecht<br />

University and the University Medical Center. Utrecht<br />

could also be a leader when it comes to spinning off<br />

small companies in this field.” Ingrid says<br />

enthusiastically, “In my opinion, one of the main<br />

achievements of our charity in relation to <strong>Translational</strong><br />

<strong>Medicine</strong> is that we realized that we need these small<br />

companies to bring new treatments to the market.”<br />

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60 <strong>Translational</strong> <strong>Medicine</strong>

“In the future, <strong>Translational</strong> <strong>Medicine</strong> will become more and more important – but in<br />

our efforts to connect with the clinic we will have to be careful not to lose touch with<br />

basic scientists.”<br />

Caroline Lindemans<br />

Pediatric Immunologist, and Bone Marrow Transplantation Specialist<br />

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“My research and hypotheses are always driven by patients’ needs, and aim to<br />

increase awareness of problems within certain areas of health care. They don’t<br />

always directly or immediately impact patient care, but in my opinion that is okay if<br />

the research still supports patients’ needs or draws attention to healthcare areas in<br />

need of change and innovation. In the long term, we will see the impact of this<br />

research.”<br />

Aisha Gohar<br />

PhD Student<br />

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“<strong>Translational</strong> <strong>Medicine</strong> is not the Holy Grail,<br />

but it really makes science better.”<br />

Jorg van Loosdregt, <strong>Translational</strong> Immunology Researcher, Group Leader of the van Loosdregt/Vastert group<br />

Bas Vastert, Pediatric Rheumatologist and Researcher, Group Leader of the van Loosdregt/Vastert group<br />

“Working together with Bas, a pediatrician, makes my<br />

work more relevant and more fun”, says Jorg, a basic<br />

researcher. “As a fundamental scientist, I learn a lot from<br />

Bas about disease, disease mechanisms, how patients<br />

feel, and what they find important. This applies to all<br />

members of our research group: we learn a lot from each<br />

other. We perform clinical trials based on fundamental<br />

research we did in the lab, and that is just one example of<br />

many tangible results of our collaboration.”<br />

Bas continues, “I need this environment of smart<br />

scientists, with the focus and time to work on clinically<br />

relevant problems. Science gets better when people are<br />

continually present for brainstorming and problem<br />

solving. We both explicitly opted for <strong>Translational</strong><br />

<strong>Medicine</strong>. To us, it is the missing link to make science<br />

relevant. <strong>Translational</strong> <strong>Medicine</strong> is not the Holy Grail, but<br />

it really makes sciences better.” The van Loosdregt/<br />

Vastert lab is engaged in clinical science, but also in<br />

fundamental science. Since these are the two extremes,<br />

everything in between is also present in the group.<br />

Jorg explains, “We do super-fundamental DNA and RNA<br />

research, and we try to understand the biology of<br />

disease.” Bas adds, “But it all has to be translatable, it has<br />

to have impact on our patients. It is unrealistic to think<br />

that we can develop a new drug in a couple of years, but<br />

we can make smarter use of existing drugs, combine<br />

drugs in different ways, or put the right drugs in the right<br />

order for our patients. Embedding our efforts in a bigger<br />

organization or context is very important. We cannot do<br />

this alone in our group; we need local support. We also<br />

need to work with powerful partners, so that we have<br />

sufficient budget and freedom to do our work.<br />

We definitely see a transition here at the University<br />

Medical Center Utrecht in favor of <strong>Translational</strong> <strong>Medicine</strong>,<br />

but there is a long road ahead of us.” For Bas and Jorg it is<br />

obvious that working as a team has a clear advantage,<br />

and that patients benefit from this collaboration. They<br />

joke, “We can’t turn back anymore.” But more seriously,<br />

they add, “We really believe in <strong>Translational</strong> <strong>Medicine</strong> and<br />

that it will improve patient care.”<br />

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66 <strong>Translational</strong> <strong>Medicine</strong>

“My role model is Hans Clevers. He is a pioneering scientist with a great worldwide<br />

scientific and societal impact due to his ground-breaking discoveries. Organoid<br />

technology will revolutionize personalized and <strong>Translational</strong> <strong>Medicine</strong>.”<br />

Antoni Hendrickx<br />

Senior scientist, Hubrecht Organoid Technology<br />

UMC Utrecht 67

“I foresee that precision medicine will become more important with regards to<br />

diagnosis and treatment of diseases, with <strong>Translational</strong> <strong>Medicine</strong> being at the<br />

forefront of these developments.”<br />

Marne Hagemeijer<br />

Postdoctoral Researcher<br />

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“Academia and industry need to work together to bring<br />

new concepts to the market.”<br />

Johan Garssen<br />

Director Platform Immunology at Nutricia Research, and Professor of Immunopharmacology at<br />

Utrecht University<br />

“At Nutricia, the majority of my work focuses on<br />

translatability, to develop things that can directly help<br />

patients. It is always in the back of my mind: can I<br />

translate this into something that can be used by<br />

patients 10 years from now? I think that both academia<br />

and companies like Nutricia have changed over the last<br />

years. Let’s be honest, ten years ago we would just look<br />

at your CV and ask how many publications you have as<br />

a scientist and what the impact factor is. This is still<br />

important, but now there is another aspect: translating<br />

science into something that can help patients and<br />

prevent diseases.”<br />

“I am in a unique position since 2005, having a parttime<br />

position with both Nutricia and the Faculty of<br />

Science of Utrecht University,“ says Johan when he talks<br />

about his two jobs. “I do fundamental work on immune<br />

regulation at the university which is not directly<br />

translatable, but I combine it with experiments that will<br />

hopefully lead to new drugs, medical food, or food for<br />

pregnant women or babies.” He concludes, “You need<br />

academia for the basic knowledge, and you need<br />

industry to translate the scientific concept into the<br />

actual drug or nutritional product or vaccine. Academia<br />

and industry need to work together to bring new<br />

concepts to the market.”<br />

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72 <strong>Translational</strong> <strong>Medicine</strong>

“My role model is Leonard Cohen: There is a crack in everything,<br />

that is where the light gets in.”<br />

Gijs van Haaften<br />

Associate Professor, Group Leader<br />

UMC Utrecht 73

“Translating the needs and languages of one discipline to another is truly<br />

challenging, but it is pivotal in order to share knowledge and to come to<br />

novel solutions.”<br />

Christiaan Vinkers<br />

Psychiatrist<br />

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76 <strong>Translational</strong> <strong>Medicine</strong>

“My involvement with Apollo Society makes me think critically<br />

about the topics of my projects and what the contribution is to<br />

the health of patients”<br />

Gautam Kok and Remi Stevelink, Utrecht University students, and Board members of Apollo Society Utrecht<br />

Apollo Society is an active international network of<br />

likeminded students with an interest in <strong>Translational</strong><br />

<strong>Medicine</strong>. It was founded in 2014 by four medical students<br />

from Utrecht. Gautam explains, “There was a lack of<br />

courses on <strong>Translational</strong> <strong>Medicine</strong> in our curriculum so we<br />

decided to teach ourselves. We gather once a month to<br />

discuss topics related to <strong>Translational</strong> <strong>Medicine</strong>. These<br />

discussions tend to be very lively. Everyone chooses a<br />

topic, and we look at these topics from multiple<br />

perspectives, including medical, ethical and political<br />

points of view.” Following the example of Utrecht, the<br />

University of Toronto started an Apollo group as well.<br />

“We are currently writing an article in collaboration with<br />

the Apollo group in Toronto on <strong>Translational</strong> <strong>Medicine</strong><br />

about the role of students in promoting <strong>Translational</strong><br />

<strong>Medicine</strong> globally. In the future, we want to expand Apollo<br />

to different hubs: the universities of Miami and Arizona,<br />

and University College London.” Why is <strong>Translational</strong><br />

<strong>Medicine</strong> important to them? Gautam reflects, “I was<br />

doing lab research, trying different kinds of drugs on cell<br />

lines. I was tucked away somewhere in a lab and did not<br />

know what I was doing exactly and what I was doing it for.<br />

That was the moment when I decided to become a doctor<br />

as well, and combine research with patient care.” Remi<br />

says, “Actually, a similar story applies to me. I found out<br />

that many researchers, including myself, had never met a<br />

patient. I did research on autism, but I had never met<br />

someone with autism. And I think my professor had never<br />

met any patient with autism either. I realised that this was<br />

not the way to go forward and I decided to study<br />

medicine as well as doing research to actually make a<br />

difference on the health of patients.” Remi continues,<br />

“My involvement in Apollo makes me think critically about<br />

the reasons for choosing a particular project and the<br />

contribution to health in general. Currently, I am doing<br />

research on epilepsy. I use genetics to predict treatment<br />

outcomes, eventually leading to personalised medicine.<br />

The parents of my epilepsy patients fund my research.<br />

Since the parents are involved in the questions I try to<br />

answer, I actually produce something that is relevant to<br />

them. That keeps me motivated.”<br />

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78 <strong>Translational</strong> <strong>Medicine</strong>

“My role model is embodied in several different people, both clinician-scientists and<br />

clinicians, whom I encountered during my career so far. They are my role models<br />

because of their integrity, perseverance, passion for their work, belief in what they<br />

do, and belief in the people they do their work with.”<br />

Maja Bulatović Ćalasan<br />

Internal <strong>Medicine</strong> Resident<br />

UMC Utrecht 79

“<strong>Translational</strong> <strong>Medicine</strong> is part of my every day work. I work together with clinicians<br />

and basic scientists, and have numerous collaborations with private partners,<br />

all aiming to improve diagnosis and care by making use of translational research.”<br />

Imo Hoefer<br />

Associate Professor, Head of Central Biobank<br />

80 <strong>Translational</strong> <strong>Medicine</strong>

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82 <strong>Translational</strong> <strong>Medicine</strong>

“The field of medicine is changing very fast. It is difficult<br />

to prepare our current students for future challenges, and<br />

we need students who are able to collaborate with people<br />

from different backgrounds to kickstart innovation.”<br />

Annet van Royen-Kerkhof<br />

Pediatrician, Associate Professor, Program Director of Selective Utrecht Medical Master (SUMMA)<br />

Annet van Royen-Kerkhof speaks enthusiastically about<br />

SUMMA, the four-year Medical Master Programme at<br />

the University Medical Center Utrecht. “The small scale<br />

of the program guarantees close interaction with the<br />

students. Our students already have a Bachelor degree<br />

in a field related to Life Sciences, so they are able to<br />

challenge you as a teacher and add different<br />

perspectives to the discussion.<br />

In my opinion, the field of medicine is changing very<br />

fast. It is difficult to prepare our current students for<br />

future challenges, but what we do know is that we<br />

need students who are able to interact and collaborate<br />

with people from different backgrounds. For example<br />

with technicians, since many innovations start at the<br />

interface of medicine and technique.<br />

In an ideal world, our SUMMA students would<br />

collaborate with students from other master programs<br />

on real life problems that cannot be solved by a single<br />

research group. This would contribute to my main aim:<br />

to teach students to think outside the box, to be<br />

creative, and not to be hampered by existing<br />

structures.”<br />

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84 <strong>Translational</strong> <strong>Medicine</strong>

“I feel that frequent inspiration by clinical problems or patients is the most<br />

important aspect of <strong>Translational</strong> <strong>Medicine</strong>. After all, both fundamental and clinical<br />

scientists can perform <strong>Translational</strong> <strong>Medicine</strong>, but their orientation matters:<br />

are they inspired by patients and their problems, or do they merely wish to solve<br />

their own experimental problems?”<br />

Henk Schipper<br />

Pediatric Cardiology Fellow<br />

UMC Utrecht 85

“My drive in doing translational science is to understand the molecular mechanisms<br />

of disease pathogenesis in the liver and gastrointestinal tract. Together with<br />

partners, such as clinicians, pharmaceutical companies, and patient representatives,<br />

I aim to translate this knowledge into therapy or diagnostic tools for the benefit of<br />

human health.”<br />

Saskia van Mil<br />

Associate Professor, and Group Leader at Center for Molecular <strong>Medicine</strong><br />

86 <strong>Translational</strong> <strong>Medicine</strong>

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88 <strong>Translational</strong> <strong>Medicine</strong>

“We have one research line from the bed of the patient<br />

to the molecules in the lab. It is not a one-way street:<br />

it is dynamic and it goes back and forth.”<br />

Jeroen Pasterkamp<br />

Professor of <strong>Translational</strong> Neuroscience, Director of MIND Facility, and Chair of Strategic Research<br />

Program Brain<br />

The lab of Jeroen Pasterkamp is translational in several<br />

different ways. He explains, “First of all, we try to<br />

understand how the brain normally develops and<br />

works, from a basic research perspective. We use the<br />

normal situation as a standard, and then compare it to<br />

the injured or diseased situation. Another strategy is to<br />

use genetic information, brain resection material, or<br />

cultured human neurons from patients as a starting<br />

point. We can then use this to identify the cellular and<br />

molecular mechanisms that are affected in different<br />

diseases.”<br />

“<strong>Translational</strong> <strong>Medicine</strong> in the field of neurosciences is<br />

different from other fields, because the nervous system<br />

is a complex organ and we do not know much about it.<br />

We are trying to fix the patient’s problem, but at the<br />

same time we are trying to understand how the brain<br />

works,” Jeroen explains.<br />

The enthusiastic professor is proud of having achieved<br />

an efficient method to work with the clinic for studying<br />

both motor neuron disease and epilepsy. “For example,<br />

we have one research line in studying ALS, or<br />

amyotrophic lateral sclerosis, that extends from the bed<br />

of the patient to the molecules we study in the lab. It is<br />

not a one-way street: it is dynamic, it goes back and<br />

forth. And, we collaborate with the entire world.”<br />

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90 <strong>Translational</strong> <strong>Medicine</strong>

“What I am proud of is that, together with my research group, we came up with the<br />

“idea” of radioactive holmium microspheres as a possible treatment of liver tumors<br />

into a real product for treatment of liver tumors, available for all patients in<br />

Europe.”<br />

Frank Nijsen<br />

Associate Professor<br />

UMC Utrecht 91

“We live in a very exciting time with spectacular technological possibilities,<br />

including next generation sequencing, gene editing, stem cell (organoid) in vitro<br />

modelling, transcriptomics, metabolomics, large compound screens... Information<br />

technology enables world wide access to international expertise and patient<br />

sharing. If embedded in a collaborative, patient-oriented, creative, inspiring and<br />

critical environment, we should be able to raise translational medicine to<br />

great heights!<br />

Sabine Fuchs<br />

Pediatrician in Metabolic Diseases with dedicated research time<br />

92 <strong>Translational</strong> <strong>Medicine</strong>

UMC Utrecht 93

Colophon<br />

Final Editing<br />

Maggy Ovaa<br />

Texts<br />

Rinze Benedictus, Sandra Genet, Margot Weggemans<br />

Printed by<br />

De Bondt grafimedia<br />

Sponsored by<br />

K.F. Hein Fonds<br />

With the support of / thanks to:<br />

Salvatore Albani, Petra Baarendse, Marije Bartels, Jeffrey Beekman,<br />

Louis Bont, Joyce Browne, Sylvia Brugman, Maja Bulatovic-Calasan,<br />

Roel Deckers, Pieter van Dijkhuizen, Sabine Fuchs, Johan Garssen,<br />

Aisha Gohar, Gijs van Haaften, Marne Hagemeijer, Antoni Hendrickx,<br />

Sanne Hoeks, Imo Hofer, Gautam Kok, Belinda van ‘t Land,<br />

Caroline Lindemans, Ingrid Lether, Jorg van Loosdregt,<br />

Sabine Middendorp, Frank Miedema, Saskia van Mil,<br />

Joris van Montfrans, Carl Moons, Sanne Nijhof, Frank Nijsen,<br />

Jeroen Pasterkamp, Berent Prakken, Kirsten Renkema, Erica Roks,<br />

Norman Rosenblum, Annet Van Royen-Kerkhof , Hester den Ruijter,<br />

Henk Schipper, Michiel Schreuder, Remy Stevelink, Joost Swart,<br />

Bas Vastert, Lilly Verhagen, Christiaan Vinkers, Femke van Wijk<br />

Translations<br />

Alexandra Cloherty<br />

Photography<br />

Thirza Luijten<br />

Lay-out<br />

Barbara Hagoort<br />

Order management<br />

UMC Utrecht, Design & Producties,<br />

Jelle Westerhoff<br />

Tel +31 (0)88 75 662 00<br />

94 <strong>Translational</strong> <strong>Medicine</strong>

UMC Utrecht 95

96 <strong>Translational</strong> <strong>Medicine</strong>

January 2018


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