The perfect drugs for you

Why do some drugs work for so few people - and what can we do about it?

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The appalling outcome of the drug trial in Rennes would be the talking point of any medical conference the week after, but it was likely to come under even more intense scrutiny at the Festival of Genomics in London (19-21 January).

Genomics uses information gained by studying a human genome – the unique biological recipe for constructing a person – to create individualised medical treatments. In this way, drugs can be tailored to fit with someone’s particular biochemical make-up so as to maximise their efficacy.

We have only to look at what happened in Paris to see why this kind of tailoring is necessary. The drug on trial was created as a route to blocking pain signals. It was a phase one study, designed to check the drug’s safety for human consumption. Unless the six participants who were negatively affected were accidentally given the wrong dose, it seems clear that the drug fell at this hurdle. One patient died on Sunday. Five have varying degrees of cerebral damage, with what one of the medical team called “deep, necrotic and haemorrhagic lesions in the brain”.

These were healthy people, aged between 28 and 49. All six would have received the same dose, adjusted for body weight, which points to a remarkable difference in outcomes. The same dose of the same drug has destroyed one person’s brain yet left another apparently unaffected. Hence the need for genomics. The value of individualising drug trials becomes clear when you see what we have discovered by accident. Take the drug Glivec (imatinib). If you have a particular kind of genetic abnormality, it will be twice as effective at helping you survive leukaemia, measured against people who don’t have this “Philadelphia translocation”. If you have colorectal cancer with tumours that have a particular mutated gene, the drug Erbitux (cetuximab) will improve your chances of survival.

This is almost certainly the tip of the iceberg. From asthma to depression to high cholesterol and schizophrenia, we have a host of licensed treatments that help somewhere between a quarter and one in 25 only of the patients taking them. It’s likely that genetic variations are to blame for much of this.

At the moment, the valuable information in these statistics is routinely ignored or discarded. That is because there is no easy way for doctors to share their data on what works for whom, and what individual traits and characteristics might be the root cause of the success or failure. This is what genomics aims to change.

An example presented at the Festival of Genomics is the Department of Health’s 100,000 Genomes Project. Clare Turnbull, its clinical leader, explained how the project’s aim of collecting vast sets of high-quality genome samples, coupled with data about each individual’s health, will lead to breakthroughs in understanding and exposing the subtle, hidden causes behind a range of cancers.

The project is the largest of its kind in the world. This is because nowhere else has the combination of nationalised scientific and health infrastructure – with the resources, will and permission to share data – that makes it possible to link health and genomic information on a grand scale. At a bleak time for the NHS, it is something to celebrate. Breaking down the divide between care provision and medical research will be an essential part of making sure that the Paris drug trial tragedy and budget-wasting medicines become a thing of the past.

Michael Brooks holds a PhD in quantum physics. His most recent book is At the Edge of Uncertainty: 11 Discoveries Taking Science by Surprise.

This article appears in the 21 January 2016 issue of the New Statesman, The Middle East's 30 years war