translational research

Translational research is a catchphrase in biomedical circles these days. But if you’re confused about what the term really means, you’re not alone. 

It was 35 years ago when I first heard the term “translation” in a scientific context. It was about enabling basic research undertaken by the National Institutes of Health (NIH) to find its way into treatments for patients. Though that sounds pretty straightforward, I still have to explain it not only to the lay public but even to my colleagues. Why? One reason may be that translational research no longer just describes a process, but characterizes an entirely new discipline. 

A lot has happened since the 1980s, including the creation in 2012 of a new NIH institute—the National Center for Advancing Translational Sciences. This is one of the only NIH centers that is not disease-focused (unlike, for example, the National Cancer Institute or the National Institute of Mental Health). Its director, Dr. Christopher Austin, spoke at Einstein last fall and described NCATS’s mission: to “catalyze the generation of innovative methods and technologies that will enhance the development, testing and implementation of interventions that tangibly improve human health across a wide range of human diseases and conditions”

What Have You Done for Me Lately?
You might say, “Okay, that’s all well and good, but why should I care about translational research?” One answer is that industry, scientists and research agencies have all noted the alarming lack of innovative treatments being approved over the past several years despite strong overall research funding. Data suggest that the explosion in translational research in the past decade has caused it to outstrip its NIH funding. (See a recent paper by Dr. Lloyd Fricker.) This implies that the real problem may be a breakdown in the long road from the science lab to the prescription pad. 

“Translational research” can most simply be defined as investigation that is informed and framed by a robust understanding of human biology and disease. This all-encompassing approach thus bridges the spectrum from basic research to health outcomes. However, the terminology can be somewhat confusing because there are many types of translational science disciplines. One way to say it is that multiple translational sciences link together to make translational research happen. 

Science Silos
Translational research involves teams of investigators who have different areas of expertise. Our biomedical disciplines, such as “pharmacology,” “cardiology,” “cell biology,” “epidemiology” and “genetics,” can create artificial boundaries that are difficult to overcome. (See A. Casadevall and F. C. Fang, “Specialized Science,” Infection and Immunity, 2014.) These days, every scientist has become a superspecialist and may have trouble communicating, even with those in adjacent disciplines.

Translational research requires a special breed who are eager to work with and integrate disparate fields. In translational research, genetics may be as important as informatics for an epidemiologist, and “big data” are as vital for understanding a biological pathway as computer models are for health outcomes.

Valleys of Death
Translational research also brings us face to face with “valleys of death” that can hinder research as it advances through phases labeled T0T1T2T3T4. In this shorthand, T0 is basic scientific discovery; T1 tests discoveries in model animal systems; T2 spans the translation of knowledge from the lab into treatments for people; T3, or efficacy research, applies new interventions to larger patient populations; and T4, or effectiveness research, evaluates the methods used to affect healthcare for patients in real-life, community settings.

At the end of the day, what links all translational researchers regardless of their specific disciplines is their ability to foresee these gaps, and to think creatively about how to bridge them.

Engineering Solutions
Translational research is at a crossroads. While research funding is threatened by many of today’s societal and economic challenges, the research community must also take responsibility for its own shortcomings. The best way to advance translational research would be to better understand and address the gaps among disciplines, since that’s where teams of scientists will—as Tom Friedman would say—invent the future.

1 comment

Student gets study help from a professorEditors’ Note: Before physicians with M.D. degrees can practice medicine in the U.S., they must pass a multipart professional exam called the United States Medical Licensing Examination (USMLE). Preparation for the challenging three-part exam can provoke anxiety. In this blog post, we look at best practices for preparing for Step 1—the eight-hour, 300-plus-question first portion of the exam that assesses whether a student has a proper command of the basic science concepts needed to practice medicine. Passing Step 1 is a requirement at nearly all U.S medical schools in order to continue into third year medical school curriculum.

by Kristina Petersen, Ph.D.
Assistant Professor of microbiology & immunology
Co-Director of the Office of Academic Support & Counseling

Not looking forward to locking yourself away in a room to study for Step 1? Almost every med student has to undergo the grueling experience of studying for and passing Step 1. Is it simply a rite of passage? An unpleasant experience everyone must survive? Not necessarily. There are a few things you can do to help yourself thrive on Step 1 from the time you arrive at medical school…. Interested? Read on! [click to continue…]

1 comment

PET (positron emission tomography) scan of the brain

One typically thinks of advances in medical science and technology as having unalloyed benefits. The ability to cure illness, the mitigation of pain and the possibility of making diagnoses that are more accurate are some of the uncontroversial results of medical progress. Yet as a new study of vegetative states demonstrates, such advances can raise ethical quandaries for physicians and the families of patients diagnosed as vegetative.

The study, conducted in Belgium and published in the British medical journal Lancet, showed that using the brain-imaging technique known as positive emission tomography (PET) provided a more accurate neurological assessment than other techniques, such as magnetic resonance imaging. The assessment provides more information about “minimally conscious” states, originally thought not to exist in such patients. Evidence from recent research had demonstrated the existence of minimal consciousness in vegetative patients, but without the details that emerged in the Belgian study. The latter revealed that not only do patients who are minimally conscious have some level of awareness or responsiveness, but they may also have some chance of improving and regaining higher levels of consciousness.

More Information, More Quandaries
Why does this new research pose ethical quandaries? Aside from the acknowledgment by a researcher in the Belgian study that the diagnostic technique is not ready for routine use, a bigger problem is the uncertainty in its ability to predict significant improvement or recovery. This can lead to “false positives”—diagnoses that show minimal consciousness and a prospect of improvement in brain function when that will not occur. This situation produces uncertainty among medical experts and families who are hopeful that their loved ones will recover cognitive function. As the Belgian researcher stated, “We shouldn’t give these families false hope.”

In such circumstances, physicians and families may be unlikely to remove life supports even after significant time has elapsed, creating anguish about whether and when to “pull the plug.” Until recently, the problem of uncertainty was that of “false negatives”: diagnoses that patients in a vegetative state had no consciousness at all when, in fact, they may have been minimally conscious. With the new study, uncertainty about eventual improvement looms as a barrier to timely decision making. And lingering uncertainty often is worse for people who have to make decisions than receiving a bad but definitive prognosis.

Some controversy still exists regarding the ethics of removing ventilators or artificial feeding from patients diagnosed as having no chance of recovery. Although families’ willingness to have life supports removed from their relatives is now more common than refusal, opposition remains strong among members of some religious groups. The new evidence may lead to changes in families’ agreement to terminate life-sustaining treatment. And while the use of PET scans for this purpose is not ready for prime time, as further studies confirm the results people may begin to demand this diagnostic technique on a routine basis.

Should Advance Directives Be Updated?
Advance directives (formerly called “living wills”)—instructions people make while fully competent about what they would want to happen if they were to lose decisional capacity—have been made by only a minority of people in the U.S. Yet among those of us who have executed such documents, the question arises: Should we consider revising them in light of this new research? The standard wording in some advance directives says: “I direct that my healthcare providers and others involved in my care provide, withhold, or withdraw treatment . . . if I become unconscious and, to a reasonable degree of medical certainty, I will not regain consciousness.” (This choice not to prolong life appears in the New York State template for the advance directive.) Should such standard wording in forms provided by states now be amended to include mention of “minimally conscious states”?  

The other option on the form—the choice to prolong life—says: “I want my life to be prolonged as long as possible within the limits of generally accepted healthcare standards.” If and when the use of PET diagnoses of vegetative states becomes an accepted healthcare standard, will people who make advance directives change their decisions?

These questions lie in the future. But as many in the field of bioethics urge, it is best to begin thinking about and discussing future medical scenarios before they are upon us.

Be the first to comment

Hepatitis C - orange and yellow text

Hepatitis C virus (HCV) continues to be the number one cause of cirrhosis and liver cancer and the primary indicator for liver transplantation in the United States, yet it remains vastly underdiagnosed.

More than three million people in the U.S. are infected. Guidelines support widespread screening for HCV, and excellent treatments are now available.

So the question remains: why is HCV underreported? [click to continue…]

1 comment

Nutritional label with focus on fats.

With the Mediterranean diet all the rage, not only because consumers like its tasty composition but because of research touting its health benefits, experts have had to become more specific about recommendations for fats.

There’s a steady drumbeat these days—from everyone from chefs to food writers to health gurus—criticizing nutritionists and the “diet police,” who, they claim, told consumers to avoid fats and keep everything fat-free.

This makes for good hype, but I know of not one registered dietitian/nutritionist or any respectable and knowledgeable physician—not a single one—who ever said that. Ever. [click to continue…]

Be the first to comment

E-cig on top of broken regular cigarette

The debate over e-cigarettes has been heating up. Are the smokeless, battery-powered, nicotine-dispensing devices a gateway to smoking for young people or a helpful way for smokers to quit? Public health experts can be found on both sides of the debate. [click to continue…]


Autism therapy featuring shapes

The recent report from the Centers for Disease Control (CDC) on increasing autism prevalence has created concern among parents, headlines in the media and enough questions to warrant additional exploration.

The findings from 2010 data show that autism now affects 1 in 68 children, up 30 percent from the last analysis, released two years ago, which reported that 1 in 88 children was affected. Should we panic? No. [click to continue…]

Be the first to comment