Sunday, October 21, 2012

The Role of Translational Research in Medicine





What is the point of research if it is reserved to only laboratories?  It is when research is practically applied to treat illnesses and increase society's knowledge of human health that research has achieved one of its ultimate goals.  Translational research aims to do just this, to create meaningful health outcomes from bench to bedside.  Scientific discoveries begin at the "bench" where scientists study diseases at the molecular level.  This research then progresses to clinical applications at the patient's "bedside" to improve their well-being.  Dr. Robert Lipsky, Director of Translational Research in the Department of Neurosciences at Inova Fairfax Hospital, founded the Inova-GMU Neuroscience Translational Research (INTR) Laboratory.  Dr. Lipsky's research revolves around functional genomics and its role in translational medical science. 




Genetics makes each and every one of us unique, whether it is in our eye color or our susceptibility to illnesses. Medication too, should be tailored to each individual's needs.  Pharmacogenetics is the study of how genetic variations affect patients' individual responses to drugs, both therapeutic and adverse effects.  This study allows for the design of drug therapy based on patients' individual genetic profiles, as well as the analysis of the safety and efficacy of drugs.  

Dr. Lipsky regarded to Warfarin as an example of the role of pharmacogenetics in translational research.  Warfarin is a drug used to inhibit the formation of blood clots, however it has a complex dose-response relationship.  Research has found that variations in the vitamin K epoxide reductase complex 1 (VKORC1) and Cytochrome P450 CYP2C9 genes both affect patients' responses to Warfarin.  Knowledge of the role of genotype variation in response to Warfarin was a biomarker for determining the ideal dosage for individual patients. In another study for the development of biomarkers in Alzheimer's disease, GRIN2B SNP along with neuroimaging were established as biomarkers for mild cognitive impairment and neurodegeneration.  

To better treat Major Depressive Disorder (MDD) patients, the Sequenced Treatment Alternatives to Relieve Depression (STAR*D) Project was developed to find more effective treatments for patients who did not improve after an initial treatment with Citalopram, an antidepressant of the selective serotonin reuptake inhibitor (SSRI) class.  In the STAR*D Project, patients' DNA were screened for long and short forms of HTTLPR, a serotonin transporter. It was found that S and Long G (LG) alleles express low levels of serotonin transporters.  Patients with S and/or LG alleles had earlier and greater side effects with the SSRIs and were considered "poor-responders" to the treatments due to the adverse side effects.  

Spinal cord injuries occur more than some of us may think.  Here are some surprising statistics about spinal cord injuries:  about 250,000 Americans have spinal cord injuries (SCI) and about 52% of spinal cord injured individuals are considered paraplegic and 47% quadriplegic.  Although this is a growing problem, there has been little progress made to help patients with SCI.  Treatments that include steroids or surgery failed to improve SCI symptoms.  Fortunately a device called the oscillating field stimulator (OFS) was developed to promote the regeneration of the spinal cord and neurological recovery.  Along with stimulating axonal growth, the OFS device improves sensation in patients with complete SCI, the worst-case scenario for the injury, and overlays the injury by creating an "electric feel."  15 weeks after the implant of the device, patients experienced fewer urinary tract infections, fewer pressure sores, improved bladder and bowel control, and had better physical therapy and rehabilitation outcomes. 



 Understanding the relationship between genes and symptoms is the core of pharmacogenetics, helping enhance the field of translational research in developing drug therapy. Translational research has benefited the medical field greatly.  Scientists provide clinicians with effective methods to treat patients.  The overall effect of the treatment on the illness is observed, and may spark leads for new studies.  It is an ongoing cycle of discoveries and application, science being used for the greater good of humanity.  In order to enhance translational research, scientists must take initiative if they find that a treatment or device shows medical potential by developing clinical trials to test the efficacy of the treatment or device.  Translational science is the bridge connecting scientific research to clinical practice.  The road to achieving this goal is not easy however.  How can translational research be improved to strengthen and accelerate the process from bench to bedside?  Solutions that should be utilized include:  finding a way to improve and advance multidisciplinary research teams and creating new research tools to encourage the development of new ideas.  As translational research continues to advance, so will the health of humanity.