required to screen for an array of complex diseases touching many molecular pathways in the body is enormous. Complementary genomic, biomolecular, and IT tools are required to translate the promise of molecular screening into a robust gatekeeper for all of molecular medicine.
Can molecular imaging provide a cost-effective tool to confirm disease onset, characterize the disease, and determine its location?
In the last decade, molecular nanosystems have been developed primarily for nuclear medicine (e.g., positron emission tomography scanning). More recently, optical, magnetic resonance imaging, and ultrasound probes have been synthesized, but they generally lack either the sensitivity or targeting specificity required for molecular diagnostics. Also, the cost of current molecular imaging procedures is prohibitive for routine clinical use. A cost-effective molecular imaging procedure with both high sensitivity and high targeting specificity must be developed in the next decade to complement molecular screening tools.
Can robust molecular delivery systems be developed to enable targeted drug and biologic therapies at the individual cell level?
At the start of this century, there was great hope that targeting systems could be developed to deliver drugs and modern biologic therapies (e.g., siRNA) with high specificity, sparing the side effects commonly associated with chemotherapies. Although great progress has been made during the last decade, there still is not a robust family of vehicles for targeted delivery, especially at the intracellular level. One of the primary goals of the next decade is to enable intracellular therapeutics (i.e., therapy directed to intracellular targets within a single diseased cell) through engineered delivery systems of great specificity.
Can molecular therapies be monitored non-invasively and cost-effectively to provide a tight feedback loop guiding personalized therapies?
Molecular therapies must be monitored effectively to maximize therapeutic efficiency while simultaneously minimizing side effects. A key goal for the next decade is to develop multi-functional molecular nanosystems that can simultaneously provide the delivery mechanism for intracellular therapies while at the same time serve as an imaging probe able to monitor the effectiveness of these therapies.
Can molecular nanosystems be developed for targeted diagnostics and therapeutics with no long-term toxicity?
Many molecular nanosystems have been developed in the last decade with little or no short-term toxicity. However, no molecular nanosystem for targeted diagnostics and therapeutics has yet been developed that eliminates all concerns about potential long-term toxicity. Eliminating long-term toxicity must become as much as of a design goal for molecular nanosystems in the next decade as sensitivity and specificity were in the last decade.
