Translational medicine involves the application of pharmacology tools, in vivo assays, biomarkers, pre-clinical and clinical methods and studies integrated to improve understanding of various disease states and advancement and approval of therapy against diseases. The development of a new drug is a cumbersome and expensive process. It involves a series of steps. The traditional method of drug development involves understanding the pathophysiology of the disease, followed by identification and validation of a treatable drug target. The next step involves identification of lead molecule or compound or an antibody specific for the target protein. The preclinical toxicology involves in vitro, in vivo assessment and initial regulatory interactions of the lead compound. The next step involves the progression of the lead compound through the phase I, II and III of the clinical trial. The phase I involves pharmacokinetic characterization of the ‘drug’ followed by toxicity study and validation of biological effect. Phase II involves evaluation of drug efficacy in patients’, dose selection, safety profile initiation, and alignment with regulatory agencies. The III phase of a clinical trial involves confirmation of the drug activity on patients’, confirmation of safety and benefit/risk profile and approval of the drug for the intended use (Butler, 2008).
The focus of translational research is to apply the various observations generated in the lab and preclinical phase to the development of efficient and effective clinical trials that can lead to approval of a drug in humans. However, following approval of a drug, the translational work continues. The focus of translational research is to develop practices focused at enhancing the cost effective use of prevention and treatment in the community (Zahrouni, 2005).
Development of a drug is a highly expensive process and even after billion of dollars spent on research; very few molecules and discoveries made in the laboratory are approved for clinically relevant use in patients. Translational medicine involves both the areas of translational research. The first area of translational research focuses on the development and approval of a drug. It often involves the progression of bench side research to its use in the clinic in patients and redirection of data and observations in the patients’ back to basic research to develop better treatment strategies, use of biomarkers and development of resistance to drugs to be utilized for effective development of new treatment strategies. The second area of translational medicine is focused towards ensuring that the treatment and prevention strategies that are approved are actually available to the community since it has been demonstrated that patients in the US only receive half of the recommended services and treatment (Drolet & Lorenzi, 2011).
The goal of translational medicine research is to overcome the gaps that often observed between the basic and clinical research and availability of this research for the use of community. The first translational block occurs when a discovery made in basic research is not adequately evaluated for clinical use, and the second translational block occurs when proven treatment or preventive strategies are not used as standard treatment practice for the general population. The overall goal of translational medicine is to ensure fast and adequate delivery of approved treatment and cures for diseases. This can happen when efficient and cross functional collaborations are established between scientists, clinicians, and pharmacy professionals by making use of technology and data analysis tools available (Woolf, 2008).
References
Butler, D. (2008) Translational research: crossing the valley of death. Nature. 12;453(7197):840-2.
Drolet, B. C., & Lorenzi, N. M. (2011). Translational research: understanding the continuum from bench to bedside. Translational Research, 157(1), 1-5.
Woolf, S. H. (2008). The meaning of translational research and why it matters. JAMA 299(2), 211-213.
Zerhouni, E.A. (2005).Translational and clinical science--time for a new vision. N Engl J Med. 13; 353(15):1621-3.
