President Barack Obama committed his administration to launching a patient-specific “precision medicine initiative” to deliver new and more effective treatments for diseases last night during his “State of the Union” address, leaving most of the details to emerge in coming weeks and months.
“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,” Obama declared, drawing applause from members of his own party. He offered scant details on the initiative, and didn't say how much he will ask Congress to pay for such an effort in the budget he is expected to submit next month.
The president did say that the precision medicine effort was intended to not only deliver on cures, but “give all of us access to the personalized information we need to keep ourselves and our families healthier.”
“We can do this,” Obama interjected, briefly veering from his prepared text.
Obama sought to put a human face on his precision-medicine message by highlighting the story of Bill Elder, who attended the address seated next to First Lady Michelle Obama as her guest. Elder was diagnosed with cystic fibrosis (CF) at age 8, at a time when people with the disease commonly lived to early adulthood.
Instead Elder is now 27, an alumnus of Stanford University and now a third-year medical student at Boonshoft School of Medicine at Wright State University, “studying to become a family practitioner with a focus on preventive care,” Obama said.
Elder is among patients who have been successfully treated with Kalydeco (ivacaftor)®, commercialized by Vertex Pharmaceuticals through an R&D collaboration with researchers sponsored by the Cystic Fibrosis Foundation.
“His story is a testament to the promise of precision medicine,” the White House declared on its website.
Obama's initiative won kudos from Research!America, an advocacy group for greater NIH funding. Research!America President and CEO Mary Woolley said in a statement last night that Elder's story “shows that science can deliver breakthroughs for patients with cystic fibrosis, cancer, Alzheimer's and other diseases.”
“The remarkable ability of our nation’s researchers to advance precision medicine to hone targeted treatments to improve individual patient outcomes is another compelling example of what can be achieved through public-private partnerships,” Woolley stated.
In a GEN article last year, Harry Glorikian, senior executive, board director, and consultant in the life sciences/healthcare industry, observed that emerging technologies, big data, and increased pressure to contain health costs are creating a major opportunity advancing precision medicine.
“To succeed in this new rapidly changing environment, however, precision medicine test makers will need to optimize their strategy,” Glorikian wrote. “That means picking the right targets, developing them along the appropriate path, providing substantive evidence of their value and cost-effectiveness, and determining exactly when, where, and how the tests should be implemented in the healthcare system workflow.”
Among companies hoping to benefit from federal investment in precision medicine is Edico Genome, a San Diego startup that won $10 million in financing last July from investors that included Greg Lucier. Edico’s technology promises to enable the clinical use of genomics by radically reducing the cost and time of analyzing next-generation sequencing (NGS) data.
“Our technology is particularly suited for analysis of the huge amounts of data and whole genome sequences that are being generated,” Edico’s CEO, Pieter van Rooyen, Ph.D., told GEN. “It’s one of the big challenges that is out there today in genomics, to address this bottleneck of all the data that is being generated. The whole point of all of these projects and studies is to get to the meaning of all these genomic markers and genes and how that relate to medicines and illnesses. The issue is to get to those results as quickly as possible, and to do things efficiently.”
Large-scale efforts such as the initiative should also agree on common standards for genomic analysis, Dr. van Rooyen added.
Joseph Panetta, president and CEO of BIOCOM, the industry group for San Diego and southern California, said Edico is among the region’s numerous companies focused on genomic tools and tech that stand to benefit from a precision medicine initiative. Others include Thermo Fisher Scientific and Illumina—whose share price zoomed 7% yesterday to $196.73, near its record high of $199.47 on January 8, fueled by speculation that the precision medicine effort will boost sales of the company’s sequencers.
“There are the companies that will benefit in terms of the ability to do more targeted drug development based on the information that comes out of a project like this, and there are companies that are going benefit in terms of the ability to do more targeted clinical trials as well,” Panetta said. “We’ve got about 600 companies here, and I would say that in one way or another, it’s going to benefit 90% of those companies.”
Such businesses, he said, should play more of a partnering role with government in advancing genomic efforts.
In 2011 Eric Lander, Ph.D., envisioned the need for a “one million genomes” large-scale sequencing project to fulfill the promise of genomics for medicine. Last year, the keynote speaker at the 3rd Annual Scientific Symposium of The Cancer Genome Atlas (TCGA) discussed the data storage and cost needed to go beyond the 10,000 cancer genomes sequenced, to one million. TCGA is a joint effort of two NIH institutes, the National Cancer Institute (NCI) and the National Human Genome Research Institute (NHGRI).
“Sequencing of one million genomes would likely change the understanding of genetic variation,” Robert Grossman, Ph.D., of The University of Chicago told attendees of TCGA’s 3rd Annual Scientific Symposium on May 12, 2014, according to the meeting summary. “One million genomes would produce approximately 1,000 petabtyes or one Exabyte of data. With compression, these data could perhaps be reduced to approximately 100 petabytes. At a cost of $1,000 per genome, the sequencing would cost approximately $1 billion dollars.”
While acknowledging the great advancement that much data would make possible in developing new drugs, Panetta questioned whether large-scale genomic efforts shouldn’t instead be addressed through the private sector rather than government. He noted the progress and greater efficiency introduced by J. Craig Venter, Ph.D.'s Celera nearly two decades ago, during the scramble to map the human genome begun by a federal effort headed by NIH's current Director Francis S. Collins, M.D., Ph.D.
The precision medicine proposal was not addressed during the Republican rebuttal delivered by Sen. Joni Ernst (R-IA), who was elected last November to succeed Tom Harkin (D), who chose not to seek re-election. Harkin was a longtime advocate of increased NIH spending for basic research.