CTI to conduct phase II study of pixantrone in patients with HER2-negative breast cancer

February 02, 2016

Specific proteins are present on the surface of tumor cells, such as PSMA on prostate cancer cells. Using nucleic acid technology, the researchers developed a small interfering RNA (siRNA) which inhibits the nonsense mediated mRNA decay process. The siRNA was then linked to a targeting ligand, also made of nucleic acid called "aptamer" which binds to selected proteins present only on the surface of tumor cells. In this research, it bound to PSMA expressed on prostate tumors. Acting as a missile, the aptamer targets the siRNA to tumor cells and spares normal cells. In mice, it eliminated the tumor.

Joseph Rosenblatt, M.D., professor of medicine and interim director of Sylvester, says Dr. Gilboa has developed a "very clever way of embracing the body's immune response" and his research "represents a completely new approach to immunotherapy of cancer."

Pascal J. Goldschmidt, M.D., senior vice president for medical affairs and dean of the UM Miller School of Medicine, describes the work as "groundbreaking and possibly a brand new opportunity for the millions of patients who are victims of cancers."

Gilboa is the Dodson Professor of Microbiology and Immunology thanks to an extraordinary gift from Eugenia Dodson following her death in 2006. Because of her battle with lung cancer, she dedicated one-third of her $35.6 million estate to be used for cure-focused cancer research at Sylvester.

Gilboa, who worked in vitro and with mice, describes his findings as "a potentially significant discovery toward a new therapy." The next step, he believes, is clinical trials at the Sylvester Comprehensive Cancer Center. Gilboa says they would likely start with prostate cancer since the reagents are available, but adds that breast cancer expressing HER2 would be another candidate.

SOURCE University of Miami Miller School of Medicine