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Gene data slows drug discovery

FAR from speeding up the discovery of drugs, the sequencing of the human genome has actually slowed it down, claims a report unveiled last week.

No one doubts that information revealed by the genome will eventually help researchers develop a vast range of novel drugs, but in the short term the reverse seems to be true. “We are drowning in data,” says Martin Wales of UBS Warburg, the Swiss-based financial services company that produced the report.

“In the long run, perhaps a decade from now, we will see benefits of the genome, but at present the industry is still wrestling with how best to use the information,” he said at a conference in London organised by the BioIndustry Association.

The genome project has revealed thousands of hitherto unknown genes, many of which have been linked to specific diseases. The idea is that if you can work out what protein a gene codes for and what role it plays in a disease, you can understand how to intervene to treat the disease. In other words, you can identify a specific target – a a protein, say – and then develop drugs that “hit” that target.

Up to 70 per cent of the targets identified through genome research may be completely novel, according to an earlier study by the Boston Consulting Group, an international company. The trouble is that it takes expensive and lengthy studies to prove that hitting them will have the desired effect.

A drug called sildenafil, for example, was originally designed to act on a target linked to cardiovascular disease. Only when researchers at Pfizer began giving the drug to people did another effect emerge – and the bestseller Viagra was born.

Most pharmaceuticals companies aren’t so lucky. To avoid costly failures, they will only invest in developing drugs when the target has been “validated” already. So although the genome is throwing up plenty of potential targets, the bottleneck of validation is deterring companies from making use of the information. Instead, they’re trying to develop better drugs that hit known targets.

The UBS report cites estimates from the Boston Consulting Group suggesting that making sense of genome data will add as much as two years to the development of each drug and cost an extra $290 million. “This greater understanding will come at a cost, certainly for the first three to five years from initial discovery,” it says.

The estimates help to explain the woes of genomics companies attempting to sell information derived from the genome. Wales says that their best bet for survival is to try to develop drugs themselves. Celera Genomics, the company that raced to complete the human genome before the publicly funded project, did just that this year.

“People who say the genome is holding things up are talking nonsense because it’s providing new opportunities,” says John Sulston, former head of the Sanger Centre in Cambridge, a key partner in the public project. “If big pharma can’t see that then they should get out of the way and let universities get on with it.” If the industry focuses on tiny improvements on existing “safe” targets, we could miss out on massive, lifesaving breakthroughs, he adds.