The real meaning of translational research

Abstract

The buzzword in academic institutions and in the National Institutes of Health for the past several years has been "translational research." This was supposed to be a novel concept: perform research that has application to human disease. For years, we have all been writing grants, abstracts, and manuscripts, making the claim that our reductionist systems, in vitro or animal models, had clear implications for the understanding of or therapy for a variety of disease states. Although great strides have been made during the past 2 decades, we have still not cured cancer (despite having declared war on this illness in the 1980s) or permanently corrected genetic disorders (a few missteps along the way). Maybe the problem comes from the realization that the diseases that we deal with as specialists are not uniform. Unique expression of disease in a specific population of patients stems from the critical role that environmental and genetic influences play. Even in inbred strains of mice, the same genetic defect or in vivo manipulation can yield very disparate patterns of disease. So how can we deal with these problems and perform research that has true implications for disease pathogenesis and therapy? The answer comes from a combinational, interactive approach that uses the expertise of laboratories with strength in genetics, human biology, and animal modeling. The success of the Human Genome Project is evident but still has not realized its full potential. Random mapping strategies have put new genes and loci at our disposal to study in in vitro systems, animal models, and select populations in humans. The results of preliminary studies using such approaches have underscored the complexity of disease. Answers are not readily apparent. None of the genetic defects defined exist in a vacuum. They occur in an intact organism-a human. Generally, we are constructed with sufficient backup systems such that single defects do not always result in overt disease. That makes the researcher's job even more difficult