Recent advances in molecular biology, culminating in the completion of the human genome project, have created unprecedented opportunities for the rational design of new therapies for human diseases. Understanding the structure and function of the genes and their protein products that are involved in the pathogenesis of disease will permit the design of highly selective therapeutic agents. The mission of the Program in Macromolecular Therapeutics (PMT) at UNC-CH is to catalyze the process of translating basic research activities on our campus into novel approaches to therapy. While cancer therapeutics is an important theme, members of the Program also have interests in the therapy of cardiovascular, inflammatory and central nervous system disorders.
A key focus of the Program will be the use of macromolecules, such as peptides, proteins, nucleic acids, and other polymers, as therapeutic agents. Macromolecules are inherently more information-rich than small molecules and therefore potentially have greater specificity as drugs. However, there are important problems to address in the therapeutic use of macromolecules including stability in the biological milieu, entry into cells, and biodistribution in the body. A key theme of the PMT is to fully exploit current advances in nanofabrication and nanotechnology in the design and development of delivery systems for macromolecules. Thus the PMT interacts closely with the Carolina Center for Cancer Nanotechnology Excellence and with a cohort of materials scientists across campus.
There are several elements implicit in the process of translation of basic research into therapeutic strategies. These include: (1) understanding molecular mechanisms of action at the level of the drug target; (2) understanding and regulating the transport and delivery of the drug candidate so as to attain a desirable biodistribution; (3) being able to quantitatively analyze the behavior of the drug in the body. The Program will foster the further development of intellectual and physical infrastructure at UNC-CH that contributes to these elements of therapeutic research.