PITA Fiscal Year 2007 Projects

Biomedical and Health Engineering

Developing Artificial Cell-Inspired Systems for Therapeutic Applications
Medical treatment has focused on molecular therapies to address many diseases, including cancer and heart disease. Current therapies usually entail manufacturing products in a large-scale factory and ultimately delivering them into the body. Although systemic delivery is useful in many cases, targeted drug delivery to specific locations with controlled release schemes can be much more desirable, increasing local concentrations and reducing unwanted side effects. While this therapy approach provides a valuable first line solution, developing a methodology that utilizes or mimics characteristics of biological systems inherent in the body could potentially open a diversity of avenues for novel solutions. One of the grand challenges of medicine is to treat disease within the body. The human body is inherently capable of self-medication, actively adapting molecular production in response to health threats signaled and sensed by the intrinsic biochemistry of the body. Our proposed solution to this grand challenge is to utilize and convert naturally available molecules in vivo into active therapeutics by designing “artificial cell” factories. These small-scale factories would have the ability to respond to a localized health threat by targeting the appropriate region of the body and then manufacturing and delivering a biological product to treat the medical condition over an extended period. While there appear to be at least six essential components for realizing an artificial cell factory, we will initially focus on the first three in the framework of this proposal. These six components are: (i) a structural shell or scaffold, (ii) encapsulation of biochemical machinery, (iii) transport to convey biomolecules to and from the environment, (iv) sensing functionality, (v) targeting of the factory within the body, and (vi) externally triggered degradation to terminate a treatment in a controlled fashion. This approach has the potential to provide a new paradigm for therapeutics.

The primary goal of this project is to construct artificial cells that will act as a nanofactory for producing biologically-relevant molecules from a source of raw materials. The ultimate goal is to develop the artificial cells as a commercial product either by forming a spin-off company based in Pennsylvania or through our continued collaboration with a Pennsylvania corporation such as NanoDynamics Life Sciences, Inc., our industrial participant. This effort will involve also the training of talented graduate and undergraduate students in this venture as well with one of the graduate student being a minority (Native American).