European Patients’ Academy on Therapeutic Innovation Translational Medicine: An introduction

European Patients’ Academy on Therapeutic Innovation  There is no clear definition of translational medicine because translational medicine means different things to different people.  Here we define Translational medicine (also referred to as translational science) as a rapidly growing discipline in biomedical research which aims to expedite the discovery of new diagnostic tools and treatments by using a multi-disciplinary, highly collaborative approach.  Often described as the practice of transferring scientific knowledge "from bench to bedside“ (B2B). 2 Introduction

European Patients’ Academy on Therapeutic Innovation  Translational medicine – a bidirectional concept  Translational medicine will encourage the flow of information from the laboratory to the clinic, and in the same way, it should be encouraged from the clinic back to the laboratory. This means that translational medicine, as a concept, is a bi- directional concept, encompassing: 3 Translational medicine: A bidirectional process

European Patients’ Academy on Therapeutic Innovation  Bench-to-bedside factors, which aim to increase the efficiency by which new therapeutic strategies, developed through basic research, are tested clinically.  Bedside-to-bench factors, which provide feedback about the applications of new treatments and how they can be improved. 4 Translational medicine: A bidirectional process

European Patients’ Academy on Therapeutic Innovation 5 Translational Medicine: The patient perspective In order to ensure continuous feedback and communication among the diverse stakeholders in this field which are essential for success, patients are playing a very important role. The bi-directional feed back loop: bench to bedside to bench

European Patients’ Academy on Therapeutic Innovation Professor Asbjörn Fölling Reference: Centerwall, S., & Centerwall, W. (2000). The Discovery of Phenylketonuria: The Story of a Young Couple, Two Retarded Children, and a Scientist. Pediatrics, Phenylketonuria (PKU) is cited as the prototype of the first treatable genetic condition (or rare disorder). Prof Fölling’s research in partnership with Egeland family began the long journey to discover what we know about PKU today. This journey led doctors, biochemists and geneticists to discover more congenital metabolic diseases, which can also cause neurological damage. 6 Translational Medicine: Actual examples

European Patients’ Academy on Therapeutic Innovation  Children with Marfan syndrome were treated by Harry Dietz and his colleagues at Johns Hopkins University. This resulted in findings that a medicine already approved in the United States as a treatment for high blood pressure could also prevent the aortic aneurisms found in mice with Marfan syndrome.  This syndrome affects the body's connective tissue and the complications are normally defects of the heart valves and aorta, which often lead to early death.  The medicine has now been tested as a therapy in a group of children with this syndrome and was found to inhibit the development of the potentially deadly abnormalities in the aorta. 7 Translational Medicine: Actual examples

European Patients’ Academy on Therapeutic Innovation  In patients with early stage prostate tumours, Anant Madabhushi and colleagues at Rutgers University use sophisticated image processing algorithms and analyse the texture in medical images made with high-resolution magnetic resonance imaging (MRI), to detect and locate the tumours in an early stage.  This led to a more sensitive and reliable technique for clinical application than other existing approaches.  The clinical implication is that tumours can be detected and treated at an earlier stage, thereby significantly improving the clinical outcome to the benefit of patients. 8 Translational Medicine: Actual examples

European Patients’ Academy on Therapeutic Innovation As illustrated in the given examples, 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. It also facilitates the characterisation of disease processes and the generation of novel hypotheses based on direct human observation. 9 Final remarks