Researchers in his laboratory made this key discovery through genetic tests that demonstrate that healthy genes in charge of protein production can become cancerous when mTOR is hyperactivated. To combat this, the scientists employed a new drug called PP242. This drug was discovered at UCSF in the lab of Kevan Shokat, PhD, Howard Hughes Medical Investigator and professor of cellular and molecular pharmacology at UCSF.

"This drug has shown promising results by bringing protein synthesis and cell proliferation levels back down to normal rates," Ruggero said. "In addition, PP242 helps fight the process of immortalization that cancer cells go through."

In their findings, PP242 proved to be more effective than similar drugs in its ability to jumpstart translational control in both live mice and human cells tested in the lab. PP242 is currently in Phase 1 clinical trials.

"We demonstrated that the drug kills the cancer cells more effectively because it blocks the abnormal production of proteins," said Ruggero. "The other drugs we tested did not show clinical effectiveness in blocking cancer development in this manner.''

The authors say that PP242 could become a potent cancer treatment. The findings are a positive step, Ruggero said, because what have previously been considered unresponsive tumors can now be treated with the second generation of inhibitors that halt mTOR's action on protein production.

"We are extremely excited about our findings and the potential of targeting aberrant protein synthesis and mTOR in cancer as we should be able to block cancer's main source of growth," said Ruggero. "We are working with clinicians to test our hypothesis in a variety of human tumors."

Source: University of California - San Francisco