President Barack Obama will urge Congress to spend U.S. taxpayers’ money for research in “precision medicine,” a burgeoning field of care in which treatments are tailored to an individual patient.
“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 said in his State of the Union address. “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.”
Obama did not provide details on what the initiative would entail and how much it would cost. He’s expected to detail the program in his fiscal 2016 budget, to be released Feb. 2.
The Obama administration views medical research as an area of health-care policy the Republican-led Congress may be willing to help advance, even as it fights the president over the continued implementation of Obamacare, his signature legislative accomplishment. Obama’s health secretary, Sylvia Mathews Burwell, foreshadowed the precision medicine proposal in a speech last week that was conciliatory toward Republicans.
“The promise precision medicine has is that it gives us the ability to develop medical treatments that are highly tailored to the individual characteristics of patients,” she said in the Jan. 15 speech at the New America Foundation, an advocacy group in Washington. “I hope we can work with the Congress to scale up the initial successes we have seen in this promising avenue of scientific endeavor.”
Precision medicine relies on tests such as genetic sequencing to identify patients who will respond to drugs such as Vertex Pharmaceuticals Inc.’s Kalydeco for cystic fibrosis. Advances in such therapies have been helped by the dramatic drop off in costs for sequencing.
Since the whole human genome was first deciphered in 2003 by the National Institutes of Health and J. Craig Venter’s Celera Genomics, the cost of sequencing an entire genome has fallen rapidly to a few thousand dollars per patient, making it a viable tool for drugmakers to employ in research.
The White House hinted at Obama’s proposal in announcing yesterday that Michelle Obama’s guests at the State of the Union address will include a cystic fibrosis patient, Bill Elder, who has been successfully treated with Kalydeco, which targets mutations in a gene underlying the disease.
“His story is a testament to the promise of precision medicine, an emerging approach to treatment that takes into account patients’ individual characteristics, and a kind of research the president hopes to expand,” the White House said in a blog post.
More than 1,800 genetic links to disease have been identified since the Human Genome Project, according to the NIH, and there are more than 2,000 genetic tests for disease conditions.
The gene that Kalydeco targets was discovered in 1989 by Francis Collins and a team of researchers. Collins led the NIH’s Human Genome Project and now leads the entire agency.
Genes -- pieces of chemical code that contain instructions for building cells and tissues -- are mostly the same in all people. Small variations can lead to differences in hair or eye color. Some may cause disease, so understanding how one individual’s genetic makeup differs from another could help scientists develop treatments.
“Precision medicine means identifying the subset of patients who will be most likely to respond to a specific treatment,” Raju Kucherlapati, a Harvard Medical School genetics professor, said in a telephone interview. “One of the best ways to do that is through genetics,” he said, while other methods include blood tests and imaging.
Researchers have been creating and delving into large databases of detailed patient records to understand the links between individual characteristics, such as genetic variation, and disease. The ability to collect data from widespread sources has bolstered studies of relatively rare conditions, including cystic fibrosis, Huntington’s disease and muscular dystrophy, said Kucherlapati, who has held an annual conference at Harvard on the topic for the last decade.
Knowledge of the genetic drivers of a disease “allows you to put patients into smaller buckets,” Cary Pfeffer, a partner at venture capital firm Third Rock Ventures, which helps start biotechnology companies, said in a Jan. 13 interview. This could lead to highly customized therapies that are tailored to individual patients, or that allow doctors to better select the right treatment from available off-the-shelf medicines, he said.
The potential of genetics as a tool in drug development spurred Roche Holding AG’s Genentech unit to commit as much as $60 million this year to 23andMe Inc. in a deal to study the DNA of customers with Parkinson’s disease.
23andMe will help Genentech gather patients with Parkinson’s in the hope of sequencing the genome of about 3,000 individuals. 23andMe will also gather clinical information on the patients, such as their symptoms and activity level, which can help Genentech draw connections between someone’s genomic profile and the way that person experiences Parkinson’s.
“With sequencing technology, we’re now at the point where speed and costs have reached a point where it’s feasible to sequence 3,000 people,” Alex Schuth, head of technology innovation and diagnostics in business development at Roche’s Genentech, said by telephone. “We also have computation power - - you get an enormous amount of information, which needs to be stored and communicated.”
A national precision medicine effort might help coordinate the work of researchers at large centers across the country in finding potential treatments for rare disorders, Harvard’s Kucherlapati said. People suffering from more common ailments, like Alzheimer’s disease, might be tested and separated into groups that will respond to specific treatments, he said.