Basic Research as the Foundation for Translational Medicine. (January 18, 2013)

The Australian National University (ANU) defines translational medicine as: "Translational Medicine is an emerging discipline that targets the pathway from basic science to clinical medicine, and from clinical trials into standard clinical practice and into health policy. This new discipline integrates and articulates separate but related fields, with the ultimate goal of improving medical therapies and health outcomes." And it offers the degree  Master of Translational Medicine.

This week's editorial in Science is writen by Huda Y. Zoghbi an investigator with the Howard Hughes Medical Institute; professor of Pediatrics, Molecular and Human Genetics, and Neuroscience at Baylor College of Medicine; and director of the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital in Houston, TX. She obtained her B.S. at the American University of Beirut, Lebanon in 1976 and her M.D. from the Meharry Medical School, Nashville in TN, 1979. So it is as a trained and practicing clinician as well as a researcher that her plea for basic research as the foundation.

Some excerpts:

Although the dream of personalized treatments has been realized for a few disorders, particularly in the field of cancer, the translation of scientific discoveries into effective treatments for other diseases has been much slower than expected. There are two main reasons for this fact: the complexity of human physiology, and our limited understanding of how the vast majority of genes, proteins, and RNAs work, irrespective of whether they are disease-associated or not.

Traditionally, such fundamental knowledge has come from untargeted, discovery-driven basic research, [and while]targeted and in-depth disease-oriented research is sorely needed... it is at least as important to support investigators dedicated to discovery-driven basic research. Specific outcomes from discovery-driven research are hard to predict, but they often surprise and delight us with their applicability in unexpected contexts.

And click here to view the 2012 Chemistry Nobel Laureate, Robert Lefkowitz, on just this point.

Who, for example, would have predicted that an apparently trivial notched-wing phenotype in the fruit fly would yield a gene that is important for so many developmental processes in humans and so many ills, ranging from cancer to stroke?

When basic research is made to seem silly in public discourse, and when its usefulness is questioned during key points in federal budget cycles, scientists should not yield to the bullying.... Not everything worthwhile can be justified by its market value; what is most meaningful may have no apparent practical impact. Yet we can be sure that human imagination will find applications for knowledge, if we are allowed to develop that knowledge in the first place.

The challenge in translational medicine is that scientists are trying to translate a text with the sophistication and depth of Shakespeare using a first-grader's vocabulary and experience, because our knowledge about the functions of most pathways in various cell types, during different developmental stages, and under normal physiological conditions, is still rudimentary and piecemeal... we must admit the limitations of our knowledge of this language and invest in learning it in full.

The best way to promote discovery is to invest in talented researchers driven by curiosity and passion, whether for disease-oriented questions or the more obscure mysteries of nature.