Transcript Precision Medicine A New Initiative The Concept of Precision Medicine (PM) The prevention and treatment strategies that take individual variability into account.

Precision Medicine
A New Initiative
The Concept of
Precision Medicine (PM)
The prevention and treatment strategies that
take individual variability into account.
The prospect of applying PM broadly
has been dramatically improved by:
1. The development of large-scale biologic databases
(such as the human genome sequence)
2. Patient characterization such as proteomics,
metabiolomics, genomics, cellular assays, and
mobile health technology
3. Computational tools for the analysis of large data
sets
Oncology is well suited for studying the
near-term impact of PM
•
Research has revealed many of the molecular lesions that drive cancers.
Each cancer has its own genomic signature, with tumor-specific features
and features common to multiple tumor types.
•
Cancers are a consequence of accumulating genomic damage.
•
Inherited genetic variations contribute to cancer risk.
•
This understanding has influenced risk assessment, diagnostic categories,
and therapeutic strategies.
•
Drugs and antibodies have been developed designed to counter specific
molecular drivers.
•
Molecular signatures may be strong predictors of benefit.
Cancer-focus is designed to address obstacles
encountered in “Precision Oncology”:
1. Unexplained drug resistance
2. Genomic heterogeneity of tumors
3. Insufficient means for monitoring responses and
tumor recurrence
4. Limited knowledge about the use of drug
combinations
PM’s more individualized, molecular
approach may:
1. Modify, but not replace, prevention, diagnostics,
screening, and effective treatments
2. May provide a framework for accelerating the
adoption of precision medicine in inherited genetic
disorders and infectious diseases
The Precision Medicine Initiative will
generate the scientific evidence needed to
move the concept of precision
medicine into clinical practice.
NEAR TERM GOALS
Innovative clinical trials of targeted drugs for
adult, pediatric cancers
Use of combination therapies
Knowledge to overcome drug resistance
LONGER TERM GOALS
Create a research cohort of > 1 million
American volunteers who will share genetic
data, biological samples, and diet/lifestyle
information, all linked to their electronic health
records if they choose.
LONGER TERM GOALS
Pioneer a new model for doing science that emphasizes engaged
participants, responsible data sharing, and privacy protection.
Research based upon the cohort data will:
1.
Advance pharmacogenomics, the right drug for the right patient at
the right dose
2.
Identify new targets for treatment and prevention
3.
Test whether mobile devices can encourage healthy behaviors
4.
Lay scientific foundation for precision medicine for many diseases
www.nih.gov/precisionmedicine
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