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The Missing Drugs

Before the human genome was sequenced, many researchers thought that such an accomplishment would change how diseases were treated, says Dan Hurley in the New York Times Magazine.

"The ability to sequence DNA quickly and cheaply will also provide the technological basis for a new era in drug development," James Watson, then the director of the National Center for Human Genome Research, wrote in a letter to the Times in 1990, which Hurley quotes.

But rather than speeding up drug development, Hurley says the inclusion of genomics into the drug discovery and development process may have slowed it down. There have been a series of disappointments, he adds: Common diseases don't seem to be caused by a handful of common variants and when a variant is linked to disease, trying to target it can still be difficult. "So far it has produced fewer returns on greater investments," he says.

Hurley notes, though, that there have been successes like Gleevec, but also that many of these drugs found through target-based discovery have only worked for single-gene mutations and only for a small group of people.

"The target-based research made possible by genomics is cool and fascinating," David Swinney, the chief executive of the Institute for Rare and Neglected Diseases Drug Discovery, tells Hurley. "[But] you know what? We almost never use this information before we discover a drug. . . . This whole idea is too simplistic for the overall complexity of biology."

The Scan

Genome Sequences Reveal Range Mutations in Induced Pluripotent Stem Cells

Researchers in Nature Genetics detect somatic mutation variation across iPSCs generated from blood or skin fibroblast cell sources, along with selection for BCOR gene mutations.

Researchers Reprogram Plant Roots With Synthetic Genetic Circuit Strategy

Root gene expression was altered with the help of genetic circuits built around a series of synthetic transcriptional regulators in the Nicotiana benthamiana plant in a Science paper.

Infectious Disease Tracking Study Compares Genome Sequencing Approaches

Researchers in BMC Genomics see advantages for capture-based Illumina sequencing and amplicon-based sequencing on the Nanopore instrument, depending on the situation or samples available.

LINE-1 Linked to Premature Aging Conditions

Researchers report in Science Translational Medicine that the accumulation of LINE-1 RNA contributes to premature aging conditions and that symptoms can be improved by targeting them.