Translational Neuroscience - Scientific Goals

Translational Neuroscience - Scientific Goals


The Wellcome Trust 4 Year PhD in Translational Neuroscience: Lifecourse influences on human brain health is a training programme focused on common human brain diseases across the life course, the goal of which is to advance knowledge, expertise and skills in clinical translation.

The problem - lost in translation

The long-term burden of neurological disease and mental illness is well appreciated. Developmental disorders have lifelong effects on independence; mental illnesses are a major healthcare burden; stroke is the commonest cause of dependency in adults and 35 million people have dementia worldwide. The quest for novel therapies for these common brain diseases requires studies of causal mechanism, generation of appropriate models, definition of the pathophysiology and the validation of biomarkers so as to enable development of potentially therapeutic strategies to bring to clinical trials.

To date a considerable amount of this translationally-motivated research has led to relatively few effective treatments, with expensive drug trial failures reducing the enthusiasm of Pharma for the field. Stroke provides an excellent example; despite thousands of animal studies there are still no effective therapies in humans. These failures result from deficiencies in the relevance of current strategies to human disease at all steps of the research process. Thus, i) we still have a poor understanding of the causes of many diseases, ii) preclinical models often have poor construct and predictive validity for the clinical phenotype, iii) experiments to correct pathophysiology are often limited by their statistical and translational design and iv) early clinical trials remain challenged by limited early efficacy markers and may be inappropriately designed for the disorder being examined. The consequence is a predictable failure of these critical clinical trials.

The solution - clinical research training for basic neuroscientists

Our vision for this training programme is predicated on the view that a key reason for these failures in translation is the poor understanding non-clinical neuroscientists have of common brain disorders and clinical research. As a result, and as also highlighted by Jensen and Amara (Neuron 2014; Found in Translation – training the next generation of translational neuroscientists), experimental strategies are often inappropriate for transferring the results to the clinical arena as they fail to take into account key confounding issues such as complex causality, the variability of human disease, the effects of longevity, compliance and the practicalities of outcome assessment in clinical trials. The solution is to train a new generation of basic neuroscientists with a deep understanding of the clinical disorders being examined, the possibilities and limitations of clinical research environments and the utility of patient resources – traditionally the focus of clinician scientists.

Our PhD training programme in Translational Neuroscience draws on unique Edinburgh research strengths in diseases across the life-course to achieve this goal. By training non-clinical students to marry cutting-edge experimental animal modelling, cellular, regenerative, computational, genetic technologies and analytical tools with comprehensive knowledge of the clinical brain research environment, we will equip them with the distinct skills required to bridge the knowledge gap between the design, execution and interpretation of cellular/animal experiments and the challenges of experimental medicine.