President Obama Heralds New Precision Medicine Initiative in State of the Union

January 21, 2015

By Aaron Krol 

January 21, 2015 | In his last State of the Union address on January 27, 2000, President Clinton told Congress that “later this year, researchers will complete the first draft of the entire human genome, the very blueprint of life… [R]esearchers have identified genes that cause Parkinson’s, diabetes, and certain kinds of cancer. They are designing precision therapies that will block the harmful effect of these genes for good.”

This was the first, brief mention in a State of the Union address of a new kind of medicine: often called precision medicine, and sometimes personalized or individualized medicine. Precision medicine spans a wide range of therapies, diagnostic techniques, and strategies for research and drug development. At heart, though, its premise is that the old methods of diagnosing chronic diseases, based almost entirely on a patient’s symptoms, are due for an upgrade. By understanding the unique genetic mutations and molecular pathways that are contributing to a specific patient’s disease, it should be possible to choose more effective therapies that get at the root cause.

In the fifteen years since President Clinton’s address, the record on precision medicine has been mixed. Scientists around the world have sequenced not one human genome, but hundreds of thousands — but the discovery of new mutations that can be pinned down as “the” cause of a particular disease has stalled, and the drug industry is coming to terms with the fact that most chronic diseases have huge biological complexity, making it trickier to find the right pathways to target. As Clinton suggested, patients with “certain kinds of cancer” have benefitted enormously from precision medicine, with a growing arsenal of genetic tests that pin down key cancer mutations and therapies to attack them. Parkinson’s and diabetes research have not yet yielded similar benefits, and not for lack of trying.

Last night, in his sixth State of the Union address, President Obama proposed to direct more federal research funding to building on these early advances.

“I want the country that eliminated polio and mapped the human genome to lead a new era of medicine — one that delivers the right treatment at the right time,” the President told Congress. “Tonight, I’m launching a new Precision Medicine Initiative to bring us closer to curing diseases like cancer and diabetes — and to give all of us access to the personalized information we need to keep ourselves and our families healthier.”

There are no details yet on what the Precision Medicine Initiative will entail — whether it will have a defined mission like the Human Genome Project, or whether, like the Obama administration’s BRAIN Initiative, it will be more a statement of funding priorities for the National Institutes of Health (NIH), open to the diverse approaches of individual research groups. Nor do we know how much financial backing will be given to the project, although we should see more details by February 2, when the President is due to submit his budget proposal to Congress.

Still, basic researchers, care providers, patient groups and the drug industry alike have been enthusiastic about the promise of precision medicine, and early reaction to the idea of an NIH-supported Precision Medicine Initiative has been positive. Notably, the NIH has been instrumental to the biggest advances in molecular medicine in the genomic age, both through the well-known Human Genome Project, and the quieter but arguably even more consequential Advanced DNA Sequencing Technology grants, which supported the growth of the next-generation sequencers that have made massive human sequencing projects possible.

Here’s a selection of the best first-pass commentary on the President’s Precision Medicine Initiative.

At Bloomberg, Alex Wayne, Caroline Chen and John Lauerman give a useful overview of some of the advances a precision medicine approach has already enabled, including marvelously effective “orphan” drugs for small populations of patients. These include Kalydeco, approved in 2012 to treat a single genetic subtype of cystic fibrosis. The Bloomberg article also details the kinds of projects industry is pursuing under the heading of precision medicine, often through large sequencing studies with the help of organized patient groups.

At National Journal, Sam Baker notes NIH Director Francis Collins’ personal interest in precision medicine, and adds that there may be synergy between the Precision Medicine Initiative and a Congressional effort to reform regulatory rules to respond to 21st-century models of drug development.

Xconomy’s Alex Lash offers a dive into what types of research can likely expect support under the Precision Medicine Initiative. “Liquid biopsies” that diagnose molecular subtypes of cancer through simple blood draws, and “tissue-on-a-chip” technologies that use miniaturized human tissue as a platform for testing drugs on samples with specific genetic profiles, both make the cut. Lash also mentions, in passing, “smarter interpretation of genetic variants,” a crucial but often overlooked aspect of precision medicine. Projects like ClinGen are working hard to collect, curate and share the best information on how individual variants can impact the risks of various diseases; more federal support for these programs could be a great value in enabling precision medicine.

David Kroll at Forbes brings up the issue of drug pricing, pointing out that precision medicine, with its high research costs and small patient populations, has been a big factor in the escalation of drug prices. He advocates for a renewed interest in the value of precision therapies, which takes into account how much they can save the overall healthcare system by heading off drastic interventions and long courses of treatment.

At BioPharma Dive, Sy Mukherjee puts the Precision Medicine Initiative in the context of the NIH budget, which has grown at a historically slow rate in recent years.

Finally, David Malakoff at Science Insider, the news arm of the American Association for the Advancement of Science, takes a broader look at how the nation’s scientific mission fared in this year’s State of the Union address.

Bio-IT World will continue to cover the Precision Medicine Initiative as new details are made available.