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Translational medicine

Translational medicine, also called translational medical

science,preclinical research, evidence-based
Research, or disease-targeted research, area of research that
aims to improve human health and longevity by determining the
relevance to human disease of novel discoveries in the biological
sciences. Translational medicine seeks to coordinate the use of new
knowledge in clinical practice and to incorporate clinical observations
and questions into scientific hypotheses in the laboratory. Thus, it is a
bidirectional concept, encompassing so-called bench-to-bedside
factors, which aim to increase the efficiency by which new therapeutic
strategies developed through basic research are tested clinically, and
bedside-to-bench factors, which provide feedback about the
applications of new treatments and how they can be improved.
Translational medicine facilitates the characterization of disease
processes and the generation of novel hypotheses based on direct
human observation.

The term translational medicine was introduced in the 1990s but only
gained wide usage in the early 2000s. Its definition varies according to
the stakeholder. Patients, physicians, and other practitioners tend to
use the term to refer to the need to accelerate the incorporation of
benefits of research into clinical medicine and to close the gap
between what we know and what we practice. Academics tend to
interpret translational medicine as the testing of novel concepts from
basic research in clinical situations, which in turn provide opportunity
for the identification of new concepts. In industry it is used in
reference to a process that is aimed at expediting the development
and commercialization of known therapies. Although different, these
interpretations are not mutually exclusive. Rather, they reflect
different priorities for achieving a common goal.

The clinical benefits of translational medicine are realized on a

timeline measured in decades, whereas applied research aspires to
shorter-term results without pretense of generating radical
breakthroughs. None of the goals encompassed by translational
medicine, however, are unique to the discipline, since most
biomedical scientists and practitioners firmly believe that their work is
to some extent relevant to the cure of disease. As a result,
translational medicine, in enhancing the efficiency of biomedical
discovery and application, rather than attempting to modify existing
processes within disciplines, has come to serve as a unifying concept
in the increasingly complex, specialized, and fragmented field of
biomedical research.

There are many compelling reasons to find cost-effective solutions to

health care delivery. For example, the rapidly growing life
expectancy in most world populations has resulted in an increased
prevalence of chronic disease, for which treatments are costly,
prolonged, and, in many cases, largely ineffective. Such conditions
represent more than 70 percent of health care spending in most
developed countries. Their continued rise in prevalence, however, has
resulted in a projected growth of health care spending to unrealistic
proportions of gross national product in most countries. The problem
is compounded by the lack of useful surrogate endpoints for clinical
testing, particularly in the case of new treatments for chronic disease.
Surrogate endpoints are biological markers that can be measured to
assess the benefits of a given treatment in the early stages of clinical
testing. Without them, however, the duration of trials that seek to
advance the treatment of chronic conditions can be prolonged by
decades. Translational medicine could help relieve this situation by
expediting the incorporation of novel endpoints into clinical testing,
thereby shortening the duration of clinical trials.

Translational medicine is also needed to deal with the numerous new

diagnostic and therapeutic tools that are supplied by modern
technology and that must be tested in human subjects before they
can become incorporated into medicine. The number of testable
agents is significantly larger than the number of patients available,
and the cost of clinical testing is astronomical (see below). These
problems are aggravated by the limited predictive accuracy of models
that do not allow reliable preclinical screening of candidate products.
Overcoming these issues may be possible with translational medicine,
which can facilitate the transfer of testable agents into the clinic,
thereby leading to more rapid validation of new products and reducing
costs associated with preclinical testing.

Challenges In Translational Medicine

There are several obstacles to the effective translation of biomedical
discovery into clinical benefit. One of the most significant of these is
cost. Carrying a product through production, laboratory testing, and
clinical trials to gain approval by regulatory agencies costs tens of
millions of dollars. Moreover, products can fail to generate projected
revenue once on the market. To overcome these issues, more accurate
preclinical testing and creative cost-effective solutions to clinical
testing must be identified. At the same time, insufficient funding for
both basic science and large-scale clinical investigation, as well as
unintended financial competition between basic and clinical research,
must be addressed.

Regulatory burdens aimed at protecting the privacy of individuals and

public safety are an important component of the biomedical
enterprise. But as the threshold for privacy and safety is raised, the
cost, complexity, and length of testing increase. The increased
sophistication of modern biotechnology has enabled scientists to
refine therapeutic and clinical strategies to improve their safety and
effectiveness. However, this has led to increased complexity among
therapeutic agents, with many newer agents based on the use of
cellular substances, the genetic modification of cells and tissues, and
the administration of substances that act indirectly on target tissues
by altering specific physiological functions in patients. Such products
complicate safety and efficacy evaluations, however, since most act
through distinct mechanisms, which may not be fully understood,
making it difficult to standardize validation strategies.

Obstacles to translational medicine are not limited to the interface

between basic and clinical research. They are also endemic to the way
clinical research has been performed. An administrative structure that
segregates clinical scientists according to discipline
(e.g., surgery, pathology, radiology, and nursing) functions according
to fixed rules and does not optimize translational science. A goal-
oriented, adaptive adhocracy model, on the other hand, with
departments built around a goal rather than a discipline, better suits
interactions among experts and fosters communication on a daily
basis. Thematic areas, built on pathogenetic mechanisms, such
as cancer and inflammation, are much better suited to assist in
fulfilling the missions of health care, teaching, and research. This
approach, in the context of translational medicine, could be more
effective in the clinic than the existing administrative model of
fragmentation into different disciplines.

Increased complexity in biomedical research has distanced the

laboratory from clinical scientists, and thus there is a need for clinical
scientists who can serve as facilitators of the translational process.
However, the training of such individuals is lengthy and expensive,
and incentives are needed. Moreover, the complexity of translational
studies that require the participation of several experts and often
require interinstitutional collaboration does not suit existing models in
which biomedical scientists are rewarded according to individual

Opportunities In Translational Medicine

The goals of translational medicine in academia and industry are
complementary. While most spending by the commercial sector funds
clinical investigations that aim to validate the effectiveness of known
entities, researchers in academia tend to focus on the identification of
novel and creative solutions. Thus, a balanced approach that
encourages partnership between these entities, with small
biotechnology enterprises bridging the gap, could establish a positive
feedback loop in which benefits in the clinic fuel advances in
academia, which in turn lead to the development of new products in
The completion of the Human Genome Project in 2003, which marked
the advent of rapid, automated (high-throughput) biotechnology and
expedited the testing of concepts encompassing thousands of
variables at once, represented one of the major scientific revolutions
of the modern era. It made possible the investigation of humans
individually across the heterogeneity of their genetic backgrounds and
their diseases. Accompanying advances in bioinformatics led to
unprecedented computational power, resulting in large databases
and comprehensive data analyses. Led by these technological
advances, which expedited the efficiency of the learning process,
several other disciplines emerged that helped increase scientists
understanding of human physiopathology in the context of
the environment. Examples include nutrigenomics, which studies the
effects of foods on gene expression, and the study of the human
microbiome, which involves the analysis of interactions between
commensal flora and the human organism. The latter revealed that, in
certain genetic backgrounds, alteration of the intestinal flora can
affect the responsiveness of cancer to standard treatment through
modulation of the immune response. Such studies demonstrate how
the natural history of complex human diseases and their
responsiveness to treatment are multifactorial phenomena, affected
by interactions between genetically determined characteristics of
each person, which in turn affect the persons interaction with
environmental factors. Achievements in linking the genetics of
individuals to their predisposition to disease and responsiveness to
treatment are expected to lead to cost-efficient approaches to
therapeutic intervention. Following such a patient-specific road map is
referred to as personalized medicine.