A new drug, known as OTS964, can eradicate aggressive human lung cancers transplanted into mice, according to a new report. The drug, given as a pill or by injection, inhibits the action of a protein that is overproduced by several tumor types, including lung and breast, but is rarely expressed in healthy adult tissues. Without this protein, cancer cells begin the cell-division process but fail to complete it and die.
When taken by mouth, the drug was well tolerated with limited toxicity. An intravenous form, delivered within a liposome, was just as effective with fewer side effects. Both approaches—described in the Oct. 22, issue of Science Translational Medicine—led to complete regression of transplanted tumors.
“We identified the molecular target for this drug 10 years ago, but it took us nearly a decade to find an effective way to inhibit it,” said study author Yusuke Nakamura, professor of medicine at the University of Chicago and deputy director of the University’s Center for Personalized Therapeutics. “We initially screened 300,000 compounds, then synthesized more than 1,000 of them and found a few that were likely to work in humans. We focused on the most effective, and now we think we have something very promising.”