Where Are All the Miracle Drugs From Sequencing the Human Genome?

What Have We Learned?
Sept. 30 2013 11:45 PM

Where Are All the Miracle Drugs?

The human genome was sequenced about 13 years ago. We were supposed to have major medical advances in a decade.

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Photo by Wanja Jacob/iStock/Thinkstock

Sequencing the human genome seemed like a discovery so important that it couldn’t be overhyped—we had, after all, transcribed the blueprint for human life—but biotech executives somehow managed the trick. William Haseltine, the founder of Human Genome Sciences, predicted in 2000 that he would halve the time and money required to bring a drug to market. Randy Scott of Incyte Genomics claimed that, “In 10 years, we will understand the molecular basis for most human diseases.”

Not quite. The cost of bringing a drug to market has increased dramatically, quibbles about accounting methods notwithstanding. The process still takes more than a decade. We already had a thorough understanding of diseases linked to single genetic sequences, such as Huntington’s and cystic fibrosis, but if anything, exploring the genome has taught us how complicated the relationship between genes and diseases really is. Last year, for example, researchers in Canada linked 71 genetic regions to inflammatory bowel disease, bringing the total to 163 and counting.

Almost 13 years after the map of the human genome was published to great fanfare in Science and Nature, it’s fair to ask where all the miracle drugs are. The 10 leading causes of death have changed very little since 2000. Life expectancy has risen by 1.9 years, but much of the change is a result of improved health among minorities rather than pharmaceutical breakthroughs.

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What happened? Researchers started with an oversimplified view of the genome’s role in human health.

“The genome provided a full parts list for the first time in biology, which was a huge contribution to biology and also drug discovery, but it didn't describe how things fit together or worked together, and that was a big problem,” says University of California–San Francisco pharmacologist Brian Shoichet.

Another excuse is time. Since developing a drug takes at least 10 years, it’s arguably unfair to expect many breakthroughs already. That line of reasoning, however, forecasts an imminent pharmaceutical revolution. I’d suggest that you not hold your breath, in part because researchers spent the first few years after 2000 chasing shadows.

Before the genome was sequenced, researchers estimated that humans had about 100,000 genes, based on the variety of proteins in the body and the now-laughable assumption that complex organisms must have more genes than simpler forms of life. (Grapes have more genes than humans do.) Many pharmacologists believed that 100,000 genes meant 100,000 potential drug targets. Even after we learned that there are only about 21,000 genes, drug companies salivated over the possibilities.

“We saw a brand-new landscape and tried to explore everything at once,” says Andrew Hopkins, who studies computational pharmacology at the University of Dundee, Scotland. “Most of what we saw turned out to be boggy fens and perilous cliffs. We didn’t know where the good real estate would be, and the price of innovation was failure.”

Hopkins is now famous among pharmacologists for developing a concept called the “druggable genome.” In 2002 he argued that only about 10 percent of human genes produce proteins that could bind to a small molecule, which is the form most drugs come in. Of that number, only 20 percent of genes are likely to be associated with disease. In the end, there are probably only a few hundred “druggable” targets, and we’ve already found drugs that work on about half of them. The field narrowed considerably.

Hopkins’ sober assessment came too late for many drug companies, which were investing heavily while attempting to beat each other to new drug discoveries. As the folly became clear, drug executives at Pfizer used to joke that the value of a new genome-derived target was negative $30 million. Drug companies drowned in opportunities. The industry laid off more than 230,000 workers between 2005 and 2010, due in large part to those failures.

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