Future triumphs over serious diseases will come from further studies of the human genome, despite the disappointing experiences of some drug firms so far, a pioneer in the field said Tuesday at the biotechnology industry's annual conference in San Francisco.
Research on variations in DNA sequences between sick and healthy people has identified tens of thousands of genes that play a role in causing illness, said Dr. Francis Collins, director of the National Institutes of Health's National Human Genome Research Institute.
Drug developers joining Collins in a panel discussion at the BIO 2004 convention at the Moscone Center, however, said the abundance of information produced by the Human Genome Project and follow-up research has overwhelmed companies with choices of genes to go after, without yet boosting the rate of new drug approvals.
"We are now drowning in targets,'' said Toni Schuh, chief executive of Sequenom Inc. of San Diego, which uses rapid gene-sequencing technology to aid its own drug discovery and help other firms set research priorities. "There must be a desperate need for a navigation tool among all these targets.''
Companies that try to develop drugs based on intriguing new leads from genomic research have often faced drawn-out studies and high failure rates, said Nicholas Dracopoli of Bristol-Myers Squibb.
"I think our optimism has led us ahead of what the technology could deliver,'' Dracopoli said. Firms are now focusing greater resources on a more limited number of gene targets, he said. They're also hedging their bets by balancing their efforts between newly discovered genes and better-known disease mechanisms.
It's been only four years, Collins noted, since a draft of the human genome sequence was released, and a year since the final sequence was completed. Even with the genome, developing a drug that's effective and safe can take a decade.
"I think there was a bit of over-optimism and, I think, naive belief that the genome would result in cures for disease in a week,'' Collins said.
But he said the tools of genetic analysis have become faster, cheaper and more accurate. They can be used to sort out the crucial disease genes to block and can help researchers evaluate the genetic make-up of individuals to predict which patients will do best on which medicines, he said. "I would be amazed if the genome hasn't made a major contribution to medicine by 2015,'' he said
