Genetic Data Sharing and the Internet May Radically Change Drug Discovery, Scientist Says

Genetic data generated through thousands of experiments and freely available to access may — with the help of the Internet and a little ingenuity — change the future of how new drugs are brought to the market.

According to Professor Atul Butte, a director of the University of California Institute of Computational Health Sciences who spoke at the recent Science on the Swan conference in Perth, Australia (May 3–5), almost anyone could start the process of commercializing a new drug from their garage, using only a computer, the Internet, and freely available data.

Professor Butte and his team suggested that experimental and open-access data from sources such as the National Institute of Health (NIH) and the European Molecular Biology Laboratory (EMBL) can be used to repurpose existing drugs. For example, the researchers looked at genomic data from people with different genetic conditions, such as muscular dystrophy and genetic heart disease, and saw many resemblances. This led them to think that a drug that is already being used for heart disease could also be beneficial for people with muscular dystrophy.

But hypothesizing that a certain drug could work for a disease other than the one it was originally intended is only a first step in repurposing it — the drug still needs to be tested experimentally. According to Professor Butte, this challenge can also be overcome with the help of the Internet. Websites such as Assay Depot offer a research service to anyone. Users can “order” a drug to be tested on the model organism of their choice, and even choose from a range of laboratories worldwide for the experiments to be carried out.

Following animal testing, the next steps would be to test the safety and efficacy of the drug in clinical trials, after receiving approval from regulators such as the U.S. Food and Drug Administration (FDA), and finally commercializing the drug for the selected disease. According to Professor Butte and his team, these steps can be covered with the help of spinoff companies now emerging from universities, and with resources from investors such as venture capitalists.

Bringing a drug to the market now involves the work of thousands of people over many years, and estimates of its cost can run from $2.6 billion to $5 billion, according to a press release. However, the future of drug discovery may be very different.