Research Overview
Molecular therapy targeting cancer and cancer stem cells, via novel nanovectors and rational drug design: 1) Novel nanovector delivery systems targeting cancer and cancer stem cells - Drug/gene delivery. - Funded by multiple NIH and DOD grants, my lab works on the tumor-targeted delivery of molecular cancer therapeutics using novel nanotechnology. A self-assembled, virus-mimicking, non-viral nanovector for systemic p53 gene therapy has shown promise in chemo- and radiosensitization of breast, prostate, head and neck cancer, is now in a first-in human Phase I clinical trial of non-viral tumor-targeted p53 gene therapy. Multiple US and International Patents are issued or pending. Currently, we are using this patented nanovector for tumor-targeted delivery of siRNA/miRNA-based therapeutics and novel small-molecule therapeutics. Currently, we are working on the next generation nanovectors for targeting cancer stem cells. 2) Novel small-molecule cancer therapeutics - Drug discovery and molecular cancer therapy - My lab has been working on drug discovery for novel small molecule cancer therapeutics targeting cell death pathways, especially for cancer cell radiosensitization and chemosensitization by molecular modulation of apoptosis and autophagy. One of the first lead compounds tested in vitro and in vivo in my lab has entered Phase I-IIb clinical trials as the World's first small-molecule inhibitor of Bcl-2/Bcl-xL/Mcl-1 entered clinic trials. Currently, we are working on molecular cancer radio-sensitization with novel Mcl-1 inhibitors and with Beclin-mimetics inducing autophagy, as well as novel small molecule inhibitors of RNA-binding proteins Musashi and HuR.