During last night's State of the Union Address, President Barack Obama called for a new initiative to fund precision medicine. Here's what the proposed initiative entails and what it could mean to your health.
Here's what the President had to say last night:
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. In some patients with cystic fibrosis, this approach has reversed a disease once thought unstoppable. 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.
Obama was short on details, but his administration will likely commit more funding to the National Institutes of Health and other related institutes and organizations. More details are expected in the coming weeks.
A Targeted Approach
By precision medicine, Obama is referring to a burgeoning approach in healthcare in which biotechnology, science, and medical records are used to improve our understanding of the causes of disease in order to develop individualized (i.e. targeted) therapies. A fundamental goal of precision medicine is to get away from the longstanding "one size fits all" approach. Ultimately, it will result in more effective treatments and quicker drug development turnarounds.
As a multidisciplinary approach to healthcare, it will involve physicians, geneticists, pharmaceutical developers, public policy wonks, venture investors, and regulators like the FDA.
"Precision medicine is about being much more precise about the diagnosis to make better and more effective medicines," says Genetech's CMO Hal Barron. To which Onyx Pharmaceuticals CEO Anthony Coles adds: "The biology of the body and the biology of disease can to an extent be predicted by the human genome."
Indeed, a fundamental assumption of precision medicine is that genetic information and molecular diagnostics about a person's condition can be used to diagnose and treat their disease. Thanks to new sequencing technologies, we can analyze individual genomes, both in part and in whole, which will in turn present an optimal path to treatment.
Both Barron and Coles participated at the two-day OME Precision Medicine Summit in 2013 at UC San Francisco. The event brought together 170 of the world's foremost thinkers, creators, and innovators to "identify new approaches and spur action to make medicine more predictive, preventive and precise."
Here's an approach to precision medicine as conceived by UCSF:
As this chart makes clear, the success of this strategy is quite dependent on genomic data, hence the call for "data donors." To boost the cause, UC San Francisco has launched meforyou.org as a way to promote the concept and educate the public.
Treatments for "Subpopulations"
It's important to note that precision medicine does not involve the development of drugs or treatments for single individuals. That area is typically referred to as personalized medicine. The National Research Council explains that precision medicine refers to:
the tailoring of medical treatment to the individual characteristics of each patient. It does not literally mean the creation of drugs or medical devices that are unique to a patient, but rather the ability to classify individuals into subpopulations that differ in their susceptibility to a particular disease, in the biology and/or prognosis of those diseases they may develop, or in their response to a specific treatment. Preventive or therapeutic interventions can then be concentrated on those who will benefit, sparing expense and side effects for those who will not.
Put another way, it's where pharmacogenetics and personalized medicine meet. And by "precision" the NRC means it in a colloquial sense, to mean both "accurate" and "precise."
Areas of Focus
By pouring through genetic profiles, molecular and cellular analyses, and medical records, scientists will increasingly be able to identify the genetic influencers of a patient's specific illness and use this data to develop treatments. In future, scientists will also have to consider the proteome and various microbiomes.
An added benefit of the precision approach is that it will allow researchers to isolate causes of drug resistance in patients who aren't responding to treatments, while directing them to more promising therapies.
Areas of research include diabetes, cancer, neurodegenerative diseases, cardiovascular disease, epilepsy, and many others. Cancer research in particular looks to benefit tremendously from this approach. As noted by the National Cancer Institute:
Cancer is a disease of the genome and as more is learned about cancer tumors, the more we are finding that each tumor has its own set of genetic changes. Understanding the genetic changes that are in cancer cells is leading to more effective treatment strategies that are tailored to the genetic profile of each patient's cancer.
Cancer genomics aims to advance personalized medicine through the DNA sequencing and analysis of patient tumors to find new genetic alterations associated with specific cancers. Providing researchers with comprehensive catalogs of the key genomic changes in many major types and subtypes of cancer will support advances in developing more effective ways to diagnose, treat and prevent cancer.
And as noted during the State of the Union Address, the strategy can be used to reverse the effects of cystic fibrosis.
Immediate challenges to precision medicine include funding (which the President is clearly trying to resolve), acquiring enough data to make meaningful diagnoses and to create accurate sets of subpopulations, data analysis, and ensuring the confidentiality of sensitive information. Given the tremendous benefits to be had, it's a safe bet that we'll overcome many of these hurdles.
Top image: Lonely/Shutterstock