ISSN 1470-3947 (print) | ISSN 1479-6848 (online)

Endocrine Abstracts (2019) 65 IN3.1 | DOI: 10.1530/endoabs.65.IN3.1

From basic science to life changing therapies

Waljit Dhillo

Imperial College London, London, UK

Endocrinology is fascinating as new hormones are being discovered every year with the power of genomics and scientific advances. The physiology of these novel hormones needs to be investigated in animal studies to determine their mechanism of action and efficacy. These essential animal studies can then highlight potential translational potential of novel hormones. The next step in this translational pathway is first into human studies followed by studies in patient cohorts to demonstrate the efficacy of novel hormones as potential new therapeutics. This translational pathway has been limited due to funding for early phase human studies of novel hormones. However since the formation of the National Institute for Health Research (NIHR) working closely with the Medical Research Council (MRC) since 2006 UK researchers have benefitted from the potential funding available to be able to carry out studies in animals of a novel hormone and then investigate the effects of these hormones in first into human studies. Importantly, funding is now also available to take these novel hormones and test them in patient cohorts for efficacy and then work with pharma to make new drugs to benefit our patients with endocrine pathologies. Over the last decade basic science discovery led to the discovery of the kisspeptin/neurokinin/dynorphin expressing neurones (KnDy neurones) in the brain as major regulators of the reproductive axis. Funded by the MRC and NIHR for the last 15 years I will describe the route from basic science discovery, first into human studies and then onto studies in patient groups to which have led to kisspeptin based therapes for the potential treatment of infertility as well as neurokinin antagonists as potential new treatments for menopausal flushing.

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