Genetic scientists, physicians, and other researchers have for several years been probing the unruly frontier of precision medicine. That’s the promising new approach to studying and cataloguing genetic variations linked to a particular disease, then tailoring treatments — a custom-formulated anticancer drug, for example — to attack them. It’s the medical equivalent of using a sniper to kill a single lethal enemy, as opposed to the carpet-bombing strategy of standard chemo- and radiation therapies.
Now the research appears headed for the mainstream as President Obama presses Congress for a $215 million Precision Medicine Initiative. Obama's proposal should attract bipartisan support when Congress votes on the initiative later this year. Congress should approve funding in the spirit that neither dreaded disease nor triumphant cures have a political hue.
A centerpiece of the initiative would be creation of a biobank containing comprehensive medical records and detailed genetic information from 1 million Americans in various stages of sickness and health. The goal is to give clinicians highly specific information about each individual's genome — the total genetic makeup, flaws and all — and use that to devise personalized treatment for, say, a distinctive lung tumor instead of scattershot treatment for generic lung cancer. By close tracking of the tumor, or mutation, clinicians might decide to use different drugs in different combinations at different times — all the while analyzing genomic side-effects to avoid causing damage to one part of the body while curing another.
"Precision medicine is really what medicine is going to become,'' says Kimberly Stegmaier, pediatric oncologist at the Dana-Farber/Boston Children's Cancer and Blood Disorders Center.
The envisioned revolution is a sign of the rapid innovation in biomedicine that has been funded, in large part, by much-needed government grants. Customized treatments are possible because of genomic analysis and sharp reductions in sequencing costs.
NIH director Francis Collins notes that 15 years ago it cost $400 million to sequence an entire human genome; today the cost is about $1,000. Research centers in the US have mapped the genomes of roughly 20,000 people. Sequencing a million individuals and making the information available to clinicians, laboratory analysts, drug designers, and medical scientists across the country "would be an enormous advance,'' says Collins.
While cancer looms largest in the crosshairs, precision medicine could help doctors grapple with an array of diseases, including certain forms of diabetes, Alzheimer's, Parkinson's disease, and even severe mental illness.
Under the president's proposal, the National Institutes of Health would receive some $130 million for the genetic mapping project; $70 million would go to the National Cancer Institute for deeper study into the genetic roots of cancer; the FDA would get $10 million to evaluate new drugs and diagnostic tests; and $5 million would go toward building the computer and data-sharing networks that would make research rapidly available to clinicians and scientists.
Research points toward precision diagnosis and treatment as one of the most viable and promising new approaches for 21st century medicine. The initiative is a smart use of government funding and deserves support.