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What do we mean by experimental medicine?

by Guest Author on 15 Apr 2015

Earlier this month we launched the call for our third round of Experimental Medicine Challenge Grants. But what exactly do we mean by experimental medicine, and why is now a good time to be doing such research? Professor Stephen Holgate, Chair of our Translational Research Group, explains.  

A researcher analysing fMRI data

(Image: NIMH on Wikimedia Commons in the public domain)


Medical research would be very different without models of health and disease. We use cells, tissues and animals to determine what healthy biological processes look like, how they change with disease, and to test new interventions.

Traditionally, we made discoveries in models and then, once it was appropriate, tested potential interventions in people. All kinds of models are used, from cells in dishes to macaque monkeys.

Cell and animal models will continue to be a cornerstone of medical research, but it’s time to start experimenting in another important model organism: humans. What could teach us more about human health than the human body itself?

We now have the tools we need to allow us to ask questions about the human body that we are used to asking of cells, flies and mice. Questions such as which brain pathways and neurotransmitters may be involved in the onset of schizophrenia, or what is the role of placental infection in adverse pregnancy outcome?

For example, the technologies ushered in by the human genome project, used in the ‘omics’ fields of genomics, proteomics and metabolomics, mean that we can track the intricacies of disease mechanisms from blood or urine samples. And new imaging technologies such as hyperpolarised magnetic resonance imaging give us non-invasive access to the human body at detailed resolutions, allowing not only visualisation of structure, but also function.

Here at the MRC, we call doing this kind of basic or discovery science in humans ‘experimental medicine’, and in late March we launched a call for proposals to the third round of Experimental Medicine Challenge Grants.

We’ve funded some really strong projects through the scheme in its first two rounds, but we’re confident that there are more researchers out there with ambitious ideas for experiments in people which will allow fascinating new insights into disease mechanisms.

It sounds obvious, but experimental medicine is all about the experiment, perturbing a system and recording the effect, not simply characterising a group of patients using these powerful new technologies. Instead we want ideas that address specific gaps in our knowledge of disease.

For example, in a project funded under the first round of grants, Prof Vincenzo Cerundolo, Director of the MRC Human Immunology Unit, is infecting volunteers with the S. Typhi or S. Paratyphi variants of Salmonella to fill in the gaps in our knowledge of how the immune system responds to the bacteria and how the infection spreads throughout the body.

And at Imperial College London Prof Steve Bloom is answering the question of whether the effects of gastric bypass surgery are largely down to the boost in gut hormones that they cause. He is comparing a group of patients receiving a bypass with a similar group of patients who will receive gut hormones via a pump. By comparing the metabolism, body weight and appetite of the two groups, he will begin to see whether hormone therapy could be a new treatment for obesity and diabetes.

So, what do we want to see from applications?

We want to see projects that make use of the clinical research resources established in the UK, from technologies recently funded under our Clinical Research Infrastructure Initiative, to Cancer Research UK’s Experimental Cancer Medicine Centres and NIHR’s Biomedical Research Centres and Units. Projects can use patient cohorts or healthy volunteers.

We want to see collaborations with industry partners, whether it’s under structured arrangements such as the MRC-Industry Asset Sharing Initiative or using existing relationships.

We also want to make it clear that experimental medicine isn’t solely a march to the clinic. It’s easy to imagine that because research involves people, it must be about testing a new drug or other intervention. While we’re happy to see proof-of-principle experiments that lay the groundwork for further clinical trials, we want to see findings from experimental medicine returned to the laboratory for further study.

But most of all, we want to see ambitious proposals which set out to address significant and specific gaps in our understanding of human disease, paving the way for a deeper understanding of disease and for effective new therapies.

Stephen Holgate

As well as being Chair of the MRC Translational Research Group, Stephen Holgate will chair the panel for this round of the Experimental Medicine Challenge Grants.


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