Precision vision

Details about the precision medicine initiative presented at the White House on Friday are relatively sparse but presumably will keep trickling out over the next weeks and months, especially as the National Institutes of Health (NIH) defends its budget proposal to Congress.

NIH will ask that this initiative be a supplement to regular funding, and will probably get the money since advanced biomedical research is just about the last refuge of bipartisan support.

It also helps that the initiative is not asking for  big bucks by federal standards—the pricetag for the million-patient-powered research cohort is pegged at about a third of the Broad Institute’s annual budget, for example.

Cancer, unsurprisingly, is a poster child for precision medicine, aka personalized medicine, in which therapies are tailored to individual patients on the basis of genetic mutations or other individual factors. initiative. And the new effort would give the National Cancer Institute (NCI) an extra $70 million in the fiscal year 2016 budget, to accelerate its current lines of precision medicine research and (with some unspecified sum) start building a cancer knowledge network.

As it happens, NCI is far from the first with plans for such a knowledge network. It joins some of its leading cancer centers such as Memorial Sloan Kettering and MD Anderson (both working with IBM’s Watson supercognitive crunching system), companies such as Foundation Medicine and Flatiron Health, the American Society of Clinical Oncology’s CancerLinQ, the OncoArray Consortium and dozens of U.S. and international research and clinical care projects that target cancer or include cancer as one target.

But the feds can provide leadership that no other group can provide—not just in funding, although that will be needed to fill in gaps particularly in the early days before these knowledge networks offer major payoffs our healthcare system recognizes.

More importantly, NCI and NIH overall can promote the vision, help to set goals, act as a convener and fair broker, and aid in setting standards (regulatory and technical) for all the clinicians and researchers and their tools to talk with each other.

NIH also can take on the critical challenges of exploration and education about patient rights and roles as partners in research, which if handled carefully seem quite solvable.

Its knowledge network vision is based on Towards Precision Medicine, a 2011 report from the Institute of Medicine that still reads well. Two of the report’s main points:

Precision medicine “is not achievable without a massive reorientation of the information systems on which researchers and health-care providers depend: these systems, like the medicine they aspire to support, must be individualized.”

And we can’t envision where this revolution in medicine will take us in the long run, just as the early architects of the Web couldn’t guess what the Web would become. Mistakes will be made, big mistakes. “In a sense, this challenge has parallels with the building of Europe’s great cathedrals–studies started by one generation will be completed by another, and plans will change over time as new techniques are developed and knowledge evolves.”

Measuring M-PACT

This month the National Cancer Institute began gathering patients for the Molecular Profiling-based Assignment of Cancer Therapeutics (M-PACT) trial among patients with metastatic solid tumors not responding to standard treatments. The study will begin by screening hundred of patients for 391 mutations in 20 genes that may respond to specific targeted therapies. Next, 180 patients will be randomly split into arms that either receive or don’t receive drugs that thought to target a specific mutation or pathway in their tumor. The primary outcomes will be response rates and/or 4-month progression-free survival, with that data collected by March 2017. Among its results, NCI says, M-PACT should give a good snapshot of the state of the art in improving outcomes via targeted therapies.