Leaf Huang, PhD, a Fred Eshelman Distinguished Professor at the UNC School of Pharmacy, has received two grants from the National Institutes of Health, each worth more than $1.5 million over five years, to support cancer-therapy research.
Huang, who chairs the School’s Division of Molecular Pharmaceutics, is an expert in designing new methods of drug delivery that could make gene therapy an effective weapon in the fight against cancer. One of his new grants will support the development of cancer therapy using LPD nanoparticles, a novel vector that Huang’s lab created.
LPD nanoparticles are consisted of lipid, polycations (molecules with multiple positive charges), and DNA. They have been used in a clinical trial to treat children with Canavan disease, a genetic neurological disorder. When mixed in the right amounts and the right order, the ingredients in LPD automatically form nanoparticles with DNA encapsulated inside. The DNA in turn acts as a carrier for siRNA, a much smaller molecule that does the actual work in silencing genes.
“siRNA uses a natural mechanism in eukaryotic cells that the cells use to silence a gene,” Huang says. “What we have done is to design the siRNA and then deliver it into the cell. The siRNA joins the cellular silencing function and silences the target gene.
“We try to silence those genes that are important to the growth of cancer cells. If we succeed in silencing those genes, the cancer can’t grow any more.”
Huang’s other new NIH grant will support a study of the interaction of cationic lipids with dendritic cells. Huang’s lab has discovered that some cationic, or positively charged, lipids can deliver an antigen into dendritic cells and activate those cells at the same time, showing a potent adjuvant activity for a vaccine formulation.
“We have demonstrated that vaccine formulated with cationic lipid can stimulate a strong immune response against cervical cancer cells,” Huang says. “It is a potent therapeutic vaccine for cancer therapy.”