Gene therapy is about ready to be the next big thing. The prospect of treating diseases by tinkering with DNA has a long history of both promise and frustration. Steady progress means 2017 should be the year the technology finally hits the US market. The problem may be figuring out how to pay for cures.
In the 1990s, researchers used viruses to replace defective genetic code, essentially healing an immune-system disease. The resulting boom was short-lived, however. A death and several cases of cancer in subsequent trials ended most of the related research.
Scientists who stuck around in the field have made advances. Infective agents used today are less likely to insert their payload into a location that causes cancer, and are more likely to successfully modify targeted cells.
Two or more gene therapies soon could be approved in the United States. GlaxoSmithKline's treatment for a rare and deadly immune disease probably will get the green light from regulators on the strength of the data. Smaller biotech Spark Therapeutics also will seek signoff for a therapy to restore vision in an inherited form of progressive blindness.
Neither may generate bountiful cash. GSK estimates there are perhaps 12 new American patients for its drug each year. It charges about 600,000 euros per person in Europe, where it already has been approved. The $90 billion pharmaceutical company hopes, however, to use its viruses, factories and know-how to roll out a different new treatment annually.
Spark's bigger promise is in hemophilia. Subsequent potential cures could be blockbusters. Sickle-cell disease, for example, can cut decades off a patient's life, but may be amenable to a cure. It afflicts 100,000 Americans.
These genetic developments are also having an effect in bigger diseases. One of the hottest areas in oncology is modifying immune cells to recognise cancerous ones. Regenerating damaged tissue offers even bigger, more distant prizes.
They won't come cheaply. The industry initially thought most one-shot cures would be priced at $1 million apiece. Given that hundreds could be developed, the resulting sums would be prohibitive for many societies. Payment for therapies based on efficacy and evidence of savings elsewhere, such as reduced hospitalisation, may offer a way to solve the conundrum. These genetic problems could be passed down starting next year.
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