Experimental Medicine is the study of the pathogenesis and treatment of disease. Modern experimental medicine represents a rapidly growing body of knowledge involving the determination of diseases processes and the development of appropriate therapies. The Experimental Medicine Program is intended for individuals seeking a career in research.

Experimental Medicine is the study of the pathogenesis and treatment of disease. Modern experimental medicine represents a rapidly growing body of knowledge involving the determination of diseases processes and the development of appropriate therapies.

experimental medicine study of the science of healing diseases based on experimentation in animals.

Investigation undertaken in humans, relating where appropriate to model systems, to identify mechanisms of pathophysiology or disease, or to demonstrate proof-of-concept evidence of the validity and importance of new discoveries or treatments.

Experimental medicine is “the use of innovative measurements, models and designs in studying human subjects for establishing proof of mechanism and concept of new drugs, for exploring the potential for market differentiation for successful drug candidates, and for efficiently terminating the development of unsuccessful ones”.67 These paradigms are being developed to allow early reads on efficacy with using patients, or more commonly, studies of mechanism in volunteers. For example, a number of models of pain and sensitization have been established that can be used on either patients or volunteers to allow an early read on potential for clinical efficacy.68 These include electrical hyperalgesia and UV burn to the skin. These can be used in addition to the routine measurement of normal pain threshold to mechanical or thermal sensation after chronic or acute drug treatment. Currently, these models mostly rely on psychophysical markers; further progress in this area will involve refinement of pain markers and end-points, including fMRI and EEG/evoked potentials.69 There remains much validation to be done with these pain models with standard agents before they can become decision-making in drug discovery and development. A longer term objective would be to correlate efficacy in the disease with efficacy in volunteer models and in preclinical pain models although sufficient data with a range of compounds for this comprehensive analysis does not yet exist.

Cognitive enhancement is also amenable to experimental medicine studies. Particularly powerful is a combination of imaging with tests of memory and attention. These studies can be done in young or old volunteers under normal conditions or where performance is impaired either by the use of a pharmaceutical agent such as the muscarinic antagonist scopolamine or a stressor such as sleep deprivation. Under these conditions cholinesterase inhibitors such as donepezil (Aricept®) have been shown to reverse deficits caused by scopolamine or sleep deprivation. Recently Gregory et al.70 demonstrated that a combination of fMRI and cognitive assessment could detect changes after the administration of an H3 antagonist compared to placebo. A single dose of GSK-189254 could produce discreet changes in brain activity during associative learning and paired attention tasks probably via effects on histaminergic neurons in the posterior hypothalamus.