Researchers in the UK and the US have developed a method of designing drugs that can act on multiple gene targets, enabling them to treat complex diseases.
The new drug development process could revolutionise the treatment of major diseases including diabetes, cancer and bipolar disorder.
Whereas current genetic therapies target individual genes, leading to a highly specialised or ‘personalised’ drug regime for the patient, the new drugs could be effective across a wide patient spectrum.
Susceptibility to complex diseases such as cancer is known to depend on a considerable number of genetic and non-genetic factors – so the impact of a drug that can treat all known genetic factors could be massive.
Ironically, this research breakthrough could reverse the trend of pharmaceutical innovation towards ‘personalised’ medicine over the last decade, renewing the role of broad-spectrum drugs in treating major diseases.
The research collaboration by scientists at the Universities of Dundee (UK) and North Carolina (US) has developed a method of computerised drug design, based on large databases of drug-target interactions.
The scientists tested 800 predicted drug-target interactions using new drugs designed by this method: 75% were confirmed by in vitro tests.
In addition, the concept of using one new drug to treat a complex disease across a range of genetic factors has been proven in mouse models.
A drug designed to treat ADHD was shown to be effective in preventing typical hyperactive behaviours in two mouse groups with different genetic defects: a missing dopamine receptor and a missing brain neuropeptide.
Study co-leader Brian L. Roth, Professor of Pharmacology at the UNC School of Medicine, commented that for a complex disease, “we know there are likely hundreds of different genes that can influence the risk for disease and, because of that, there’s likely no single gene and no one drug target that will be useful for treating it.
“And so the realisation has been that perhaps one way forward is to make drugs that hit collections of drug targets simultaneously. Here we show how to efficiently and effectively make designer drugs that can do that.”