Pursuing Precision Medicine

The great variation among patients requires treatments that are tailored to the individual.

For most of human history, medicine has taken a ‘one-size-fits-all’ approach to the treatment and prevention of disease. Recently, however, advances in genetic sequencing technology and data science, as well as the proliferation of electronic health records and mobile health technologies, have enabled researchers to design customized medical interventions based on a person’s particular genes, environment, lifestyle, and many other factors. This approach, known as ‘precision medicine,’ has the potential to dramatically alter how clinicians diagnose and treat their patients. Already, IRP researchers have examined how certain genes and other variables influence the risk, progression, and treatment of illnesses from Crohn’s disease to hepatitis C to cancer.

In his 2015 State of the Union address, former president Barack Obama announced the Precision Medicine Initiative (PMI), a research program aimed at greatly expanding the capabilities of individualized treatment. The funding provided by this endeavor, combined with the IRP’s unrivaled access to genetic, molecular, and other data from a large and diverse patient population, have resulted in an unprecedented surge of precision medicine research at the NIH.

In particular, a new PMI-funded NIH program is now poised to hasten our ability to use individuals’ unique attributes to develop more effective, targeted treatments. The All of Us Research Program aims to build a massive, diverse precision medicine dataset by gathering health, lifestyle, and other information from more than one million Americans. Using this rich collection of data, IRP and other researchers will be able to investigate how individual differences in a variety of areas influence patients’ risk for specific conditions as well as their response to treatment.

With the advent of the PMI and a deep database of patient data available at NIH, IRP investigators are leading the way on a wide array of precision medicine projects to:

• Create methods of assessing risk for a variety of diseases based on a person’s individual biology and environment
• Explore the reasons behind individual differences in responses to commonly used drugs
• Isolate biological signals (‘biomarkers’) that indicate the presence of a particular condition or an increased or decreased risk of developing a disease
• Utilize mobile health technologies to gauge the links between health outcomes and lifestyle, physiological, and environmental factors
• Classify diseases into subtypes based on their unique traits, assess the relationships between them, and develop targeted treatment approaches
• Design methods for storing and protecting the private health information on which precision medicine depends

Explore these pages for more information about the past, present, and future of IRP precision medicine research:

• Genetics and Genomics Scientific Focus Area
• All of Us Research Program

Check out all 12 of the domains in which we are Accelerating Science to learn about how IRP scientists are tackling important biomedical challenges.