View
215
Download
1
Category
Tags:
Preview:
Citation preview
KATRINA L. KELNER, PHDEDITOR, SCIENCE TRANSLATIONAL MEDICINESCIENCE FAMILY OF JOURNALS
KIKI FORSYTHE, MLSSENIOR PUBLISHER RELATIONS SPECIALISTSCIENCE FAMILY OF JOURNALS
Defining and Publishing Translational
Medicine
Translational and Precision Medicine
The expanding Science family of journals (Kiki Forsythe)
Publishing in Science journals
Defining Translational Research: Standard Definition
BASIC
BIOMEDICAL
RESEARCH
PRECLINICAL RESEARCH
CLINICALTRIALS
IMPLEMENTATION
BENCH BUSINESS BEDSIDE
Defining Translational Research:More Complicated Definition
Research that moves basic and preclinical biomedical or engineering science discoveries toward improved patient care
Clinical observations that are moved back into the research lab for mechanistic studies
The use of research findings to inform policy changes that affect community health and heath care
Translation: The Road is Long
SLOW:
EXPENSIVE:
HIGH FAILURE RATE:
NUMBER OF NEW DRUGS:
PROBLEM:
Average time to approval, 10 y
Cost per successful drug: $4B
1 in 40 (not 1 in 10) drug candidates reach clinic
27 FDA drug approvals in 2013(only 9 first in class)
Limited access to patients except during clinical trials
KNOWLEDGE
INNOVATION
REGULATORY
POLICY
S
Let’s give it a try. If all else fails, Sara has a corkscrew.
KNOWLEDGE GAP:
We don’t know as much about human biology as we thought we did
We must decipher the natural histories of human diseases and use this knowledge to select therapeutic targets and approaches
Progress requires investigations at the interfaces of established and emerging disciplines
BEDSID
INNOVATION GAP:
• Fear of risk
• Lack of funding; rewards for safe science, not new ideas
• Pharmaceutical and Biotech companies must satisfy shareholders and make profits
• “Ultimately, translational research requires a leap of faith because … there is always the risk of unanticipated adverse events.”
--Christopher Breuer, MD, Deputy Vice Chair of Research and Director, Tissue Engineering,
The Ohio State University
REGULATORY GAP:
Regulation is a bottleneck VS Regulation protects the public
Do we need to approve drugs and devices faster or be more vigilant about keeping potentially dangerous drugs off the market?
Challenge: We could do both at the same time if we could learn how to accurately predict, from preclinical data, the human health risks associated with a new product
REGULATORY GAP:
What Is the Greatest Regulatory Challenge in the Translation of Biomaterials to the Clinic?
G. D. Prestwich et al., What Is the Greatest Regulatory Challenge in the Translation of Biomaterials to the Clinic?. Sci. Transl. Med. 4, 160cm14 (2012).
POLICY GAP:
stm.sciencemag.org/site/misc/current_policy_collection.xhtml
• FUNDING (e.g., ideas for early-venture funding)
• EDUCATION (e.g., how to train the next generation of translational scientists)
• REWARD SYSTEMS for team science (industry and academics)
• GLOBAL HEALTH ISSUES (large philanthropic entities, the Gates Foundation)
• REGULATORY OVERHAUL (requires government intervention)
• Make available to the world's scientists screening facilities, validated animal disease models, and chemical compound libraries
• Earlier access to patients • Facilitate drug repurposing
B. Onaral, Emerging Economies, Enduring Partnerships. Sci. Transl. Med. 4, 158ed8 (2012). Podcast: stm.sciencemag.org/content/4/158/158pc7.abstract
Solutions
• Build worldwide innovation networks with diverse expertise
• Make available to the world's scientists screening facilities, validated animal disease models, and compound libraries
• Build worldwide innovation networks with diverse expertise
B. Onaral, Emerging Economies, Enduring Partnerships. Sci. Transl. Med. 4, 158ed8 (2012). Podcast: stm.sciencemag.org/content/4/158/158pc7.abstract
USAEurope
China
Collaboration
PUBLIC AND PRIVATE PARTNERSHIPS“Share everything … Play fair.”
--Robert Fulghum
DATA SHARING
Sharing of knowledge drives science and innovation. It fosters cross-pollination, an essential driver of creativity
A proprietary culture threatens to stall the engine that has given us so many valuable treatments
Industry, academia, foundations, and government need to engage in collaborations early in the research process
Expand the precompetitive space!
B. Onaral, Emerging Economies, Enduring Partnerships. Sci. Transl. Med. 4, 158ed8 (2012). Podcast: stm.sciencemag.org/content/4/158/158pc7.abstract
Successes
Cell-based Therapeutics
TumorImmunotherapy
Engineered microbes
iPSCs
Engineered microbes
NEU
RO
PR
OS
TH
ETIC
S
IMAGING THE LIVING HEART
Nanoparticle imaging and corresponding diagnostic modalities
GENOMIC MEDICINE
PRENATALDIAGNOSIS
GENETIC DISEASES
INCOMPLETE PENETRANCE
DISEASE SUSCEPTIBILITY
PREDICTIVE ANIMAL MODELS
CANCER
Precision Medicine
Emerging in the last 5 years
……an innovative approach to disease prevention and treatment that takes into account individual differences in people’s genes, environments, and lifestyles. Precision medicine gives clinicians tools to better understand the complex mechanisms underlying a patient’s health, disease, or condition, and to better predict which treatments will be most effective.
United States of America President Obama announces
the Precision Medicine Initiative Jan 30 2015
Epilepsy patients who carry an HLA-B*1502 gene should not receive the antiepileptic drug carbamazepine. This genetic marker predicts with high certainty that they will react to the drug with the deadly skin-blistering side effect “Stevens–Johnson syndrome” Another drug should be chosen.
The Genetics of the Individual Patient Guides Drug Selection
Carbamazepine, HLA-B*1502 and risk of Stevens–Johnson syndrome and toxic epidermal necrolysis: US FDA recommendations. PB Ferrell and HL McLeod, Pharmacogenetics 9, 1543 (2008)
Precision Medicine
Cancer leads the way for Precision Medicine
Lung cancer Some patients recover, some do not
Gefitinib
Gefitinib
Two types of lung cancer Two precision treatments, all recover
Bisphosphonate plus rapamycin
EGFR +
K-RAS+
Alexis Borisy (left) and Michael Pellini founded Foundation Medicine to determine the genetics of cancers for individual patients
A patient with salivary gland cancer who had failedconventional treatment sent cells from his tumor toFoundation Medicine for testing. They were found toshow overactivity in HER2, a gene often defective inbreast cancer. This result suggested to the patient’sdoctors that his cancer would respond to Herceptin,a drug often prescribed for breast cancer patients
Remote continuous vital sign monitoring via iPhone. Delivers real-time data about the vital signs of any patient who is in a hospital intensive care unit to a doctor’s or nurse’s smart phone.
Published by AAAS
Digital Medical Devices
Another example ofpersonalized medicine for cancer:
Inject many drugs into aTumor to find the most effective
Other Precision Medicine Approaches
Personalized pharmacokinetics
Companion diagnostics for precision therapies
Nanotechnology for targeted therapies
Submitting to the Science Family of Journals
Physics
Ecology: Cave fish without eyes
Materials Science
Biomedicine
Magazine News
Research Articles
Print and online
Shorter format
Interdisciplinary Science
Open AccessOnline onlyResearch ArticlesFull papersLong format
Comparing the four Science journals
Staff editors and Board of Reviewing Editors
Peer review for top qualityReject, revise, accept
SUBSCRIPTION via INSTITUTIONPAPERS FREE AFTER 12 MONTHS
Academic editorsPeer review for top qualityReject, revise, accept
OPEN ACCESS via AUTHOR FEEPAPERS FREE IMMEDIATELY
Submissions and Acceptance Rate
Submissions/ >11500 >700 >2600 3000++ year
Acceptance 6% 19% 10% 16%
Papers/year ~800 ~150 ~250 ~250 to 500++
34
How to Publish in the Science Journals is NOT a
secret Read the Science Journals
Become a trusted, critical reviewer for the Science Journals
Work on important problems
Know when you have a reasonable result worthy of publication in a high-profile journal
Present the results of your work in an unbiased, dispassionate manner
Get some honest advice from impartial critics prior to submission
WHICH JOURNAL IS RIGHT FOR MY RESEARCH?
READ THE JOURNALS
BE FAMILIAR WITH WHICH JOURNALS PUBLISH PAPERS IN YOUR FIELD
LEARN WHAT MAKES A GOOD PAPER
ASK YOURSELF THESE QUESTIONS:
Is your finding a big step forward with broad implications?
Will many scientists find your results exciting?
Did you apply a new technique to investigate difficult scientific questions?
Is your research in the biological, physical, or social sciences?
Is your study self-contained and suitable for the Science format?
IF YOU ANSWERED YES TO THESE QUESTIONS, YOUR PAPER MAY BE SUITABLE FOR SCIENCE.
Should I submit to Science?
ASK YOURSELF THESE QUESTIONS:
Is your finding a step forward with a broad implication?
Will many scientists find your results exciting?
Is your research in the biological, physical, or social sciences?
Does your paper require a longer format?
Does your funding agency require publication in an open access journal?
Was your paper recommended for transfer from a Science journal?
IF YOU ANSWERED YES TO THESE QUESTIONS, YOUR PAPER MAY BE SUITABLE FOR SCIENCE ADVANCES.
Should I Submit to Science Advances?
ASK YOURSELF THESE QUESTIONS:
Does your research use basic science knowledge to make real progress toward improved patient care?
Does your research apply engineering discoveries toward improvements in clinical care?
Do you have strong evidence that your findings apply to human beings?
Does your research present novel ideas or approaches?
Was your paper recommended for transfer by Science or Science Signaling?
IF YOU ANSWERED YES TO THESE QUESTIONS, YOUR PAPER MAY BE SUITABLE FOR SCIENCE TRANSLATIONAL MEDICINE.
Should I Submit to Science Translational Medicine?
ASK YOURSELF THESE QUESTIONS:
Are your results reporting a cellular or organismal signaling pathway:
that helps understanding of physiology and disease?
that helps understanding or treating disease?
that is a novel type of signaling?
with computational or modeling analysis leading to a tested prediction?
Was your paper recommended for transfer rom Science or Science Translational Medicine?
IF YOU ANSWERED YES TO THESE QUESTIONS, YOUR PAPER MAY BE SUITABLE FOR SCIENCE SIGNALING
Should I Submit to Science Signaling?
YOU CANT GO WRONG
IF YOUR PAPER IS NOT SUITABLE FOR ONE SCIENCE FAMILY JOURNAL, WE WILL HELP YOU TRANSFER TO A MORE SUITABLE SCIENCE JOURNAL
This is your chance to speak directly to the editor.
Explain the overall context of your results. What problem do they solve?
Explain why you are excited about your work.
Keep it short, preferably 1 page.
Have someone else proofread and edit it, especially if English is not your first language.
If you are re-using your cover letter from submission of the paper to another journal, don’t forget to change the name of the journal!!!
Write a good cover letter
Run your own review process
A scientist in your own specialty
A scientist in an unrelated specialty
A good editor for the English language
Think like a reviewer
43
How to Publish in the Science Journals is NOT a
secret Read the Science Journals
Become a trusted, critical reviewer for the Science Journals
Work on important problems
Know when you have a reasonable result worthy of publication in a high-profile journal
Present the results of your work in an unbiased, dispassionate manner
Get some honest advice from impartial critics prior to submission
Science Immunology
Science Robotics
Announcing!Two, new high-impact
research journals from the publisher of Science Publishing in 2016
A visible high-quality platform devoted exclusively to robotics research across the basic sciences, computer science, engineering, and medicine.
The exciting crossroads of cellular and clinical immunology where human research meets a full host of organisms.
Year Submissions
Reviewed
Published
2010 735 50 14
2011 821 59 10
2012 859 77 11
2013 1106 91 23
2014 1103 98 12
Percentage going to review is increasing
Percentage publish up to 2014 still lags global average of 6%
But 2015 is excellent year: by early September have already published more papers from China than in all of 2014.
China Publications in Science
Year Published
2010 10
2011 11
2012 13
2013 18
2014 24
Relative to 130 total papers published per year
China Publications in Science Signaling
China Publications in Science Advances
26 published (out of 176, or about 15%)
16 additional in press
10 under review
222 rejected
A vanished history of skeletonization in Cambrian comb jellies, by Ou et al.,China University of Geosciences, Beijing.
49
Standing out from the crowd The past: Authors compete to get their
papers published in high-impact journals; IF of journal considered a proxy for quality of paper.
The present: Increased use of citations, altmetrics; importance on raising visibility through conventional and social media
The future: Menu of options for demonstrating impact across the basic to applied spectrum, including predictive factors based on altmetrics; more emphasis on authors getting the word out.
50
51
Standards of conduct
The past: Variations in interpretation of what constitutes COI, contributions worthy of authorship, etc. vary internationally.
The present: Journals increasingly stipulating expectations for conduct and asking authors and reviewers to sign declarations.
The future: The scientific enterprise will truly become one international community with a common set of standards practiced globally.
52
Addressing the
overburdened reviewer
system
The problem: Manuscripts undergo serial rejection from journals (down the “IF ladder”), leading to multiple rounds of review.
Solutions: Increasing use of decisions by editors
Broaden reviewer pool (please register!)
Cascading of manuscripts/reviews within journal families
Growth of 3rd party review services that provide peer review and suggest target journals appropriate for the topic/quality of the manuscript
53
Trusting science
The past: Science assumed to be self-correcting. Irreproducible research would be weeded out. Fraudulent research assumed to be rare.
The present: Increasing attention on raising the standards for transparency and improving practices for open science.
The future: The scientific enterprise will truly become one international community with a common set of standards practiced globally.
Recommended