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Key issues in translational
medicine: A perspective from
scientific journals
Juan Carlos López
Chief Editor
Nature Medicine
What is translational research?
 NIH definition: “To improve human health, scientific
discoveries must be translated into practical applications.
Such discoveries typically begin at “the bench” with basic
research [...] then progress to the clinical level, or the
patient's “bedside.”
 “Scientists are increasingly aware that this bench-tobedside approach [...] is really a two-way street. Basic
scientists provide clinicians with new tools for use in
patients [...] and clinical researchers make novel
observations about [...] disease that often stimulate basic
investigations.”
 But where are the new medicines?
Limited success in drug development
Munos, B. Nat. Rev. Drug Disc. 8, 959–968 (2009)
Where do we go from here?
 Three projects to foster translational research
Herrenhausen Symposia
Eureka Certificate Program in Translational Medicine
Science Café
Herrenhausen Symposia
 Goal: To identify the most important roadblocks
to translational research in different fields of
biomedicine and to propose potential solutions
 Methodology
 Outcomes
Herrenhausen Symposia
What have we learned?
 The roadblocks are remarkably similar across
disciplines:
We need to learn more about the biology of disease
We need to develop better animal models
We need to find better biomarkers
We need to design better preclinical and clinical trials
We need to invest in infrastructure and human
resources
 Ways to overcome the roadblocks have not
emerged yet
Grand challenge:
Developing human resources (I)
 There is a profound disconnect between the
goals of translational research and what is
expected of scientists to advance in their careers
Scientists are evaluated in a time scale much shorter
than that of any truly translational project
Funders and universities commonly value individual
contributions over teamwork
Journals favor big conceptual advances over the kind
of work that is ultimately translated into a therapeutic
advance
High-profile journals’ criteria
vs. translational research
Dronedarone—Atrial fibrillation
Golimumab—Rheumatoid arthritis
Sapropterin—Phenylketonuria
Prasugrel—Atherothrombosis
Ustekinumab—Psoriasis
Tocilizumab—Rheumatoid arthritis
Febuxostat—Gout
Etravirine—HIV
Lacosamide—Epilepsy
Alvimopan—Postoperative ileus
Icatibant—Angioedema
Romiplostim—Immune
thrombocytopenic purpura
Dabigatran etexilate—Venous
thromboembolism
Plerixafor—Hematopoietic stem cell
transplantation
Tolvaptan—Hyponatremia
Journal of Cardiovascular Pharmacology
Annals of the Rheumatic Diseases
Journal of Pediatrics
British Journal of Pharmacology
Journal of Immunology
Journal of Experimental Medicine
European Journal of Pharmacology
Antimicrobial Agents and Chemotherapy
Journal of Medicinal Chemistry
Journal of Medicinal Chemistry
British Journal of Pharmacology
Cytokine
Journal of Medicinal Chemistry
Blood
Journal of Pharmacology and
Experimental Therapeutics
Two ways to think about
the results
Grand challenge:
Developing human resources (I)
 Every field of biomedicine needs to attract, train
and retain new talent
 Every field equally needs to develop ways to
recognize the contribution of individual scientists
to translational projects, which take place over
very long periods and involve very large teams
Grand challenge:
Developing human resources (II)
 Physician-scientists are often markedly unaware
of all the pieces that must fall into place to
develop a new therapy
Intellectual property protection
Interaction with regulators
Target validation and toxicology
Fundraising
The importance of teamwork, etc.
 Enter the Eureka Certificate Program in
Translational Medicine
The Eureka Program
 Goal: To expose budding translational
researchers to a lot of what they don’t know
about the long road from bench to bedside
Mentoring
Case studies
Lectures
Workshops
 An equally important goal is the formation of
international networks that the trainees can
harness to advance their research
The Eureka Program
http://www.eurekainstitute.org
Grand challenge:
Developing human resources (III)
 Biotech is in dire straits, and the pharmaceutical
industry needs a new source of innovation
 Academia is the obvious place to go to, but the
terms need to be right for the partnership to work
 Scientists (and their employers) often have
unrealistic expectations about the value of their
research
 Industry wants to discuss with academics the
reality of the drug-discovery process
The Science Café
 A Nature Medicine-Nature Biotechnology project
 It puts young scientists with a track record on
translational research in contact with investors
 Goals: to foster both the development of
scientist-investor networks and investment in
science with translational/commercial potential
 Three sites: Boston, San Francisco and London
 Frequency: two or three times a year per city
 Inspiration: SciBX
SciBX
The Science Café—Methodology
 Review 40–50 biomedicine and biotechnology journals
 Identify those articles from each of the three regions that,
in our opinion have the highest translational potential
 Invite the authors of 3–4 articles to give a 10-minute talk
to 20–30 investors/VCs
 Mentor the scientists to make their talk as suitable to this
audience as possible
 We very rarely find more than the 3–4 papers necessary
for each SciCafé, and most scientists we invite need a
lot of mentoring before they can give their presentation
The Science Café—Results
 Top VC fund. 800 requests for money from researchers
trying to commercialize their science. How many were
funded?
 ZERO
 12 scientists spoke at the SciCafé in the same city and
year. How many struck deals with the VCs?
 EIGHT
 The model works, and we are developing it to suit the
needs of other parties (e.g. companies, governments)
Conclusions
 There are scientific and extra-scientific obstacles
to translational research
 Among the extra-scientific obstacles, careerdevelopment priorities and lack of formal training
on translational research are critical, but they
tend to receive limited attention. This is partly
the motivation for some of our projects
 The large investment in translational research
being made globally may not bear fruit without a
similar commitment to advanced training and
career development