Drug Blocks Gene That Drives Cancer

Researchers have discovered a gene that's key to cancer growth. By blocking it, they were able to reverse the health issue. 

The protein known as Ral, can drive tumor growth and metastasis in several human cancers when it is active state, including bladder, colon, lung, pancreatic and prostate cancer. However, drugs that block activity of the protein are not yet available.

Recent findings published in the journal Nature provide a novel approach that targets activation of Ral proteins.

"When you want to keep an alligator from biting you, you can tie its mouth shut. We took another approach - we put a stick in its mouth to hold it open," said lead study author Dan Theodorescu, MD, PhD, professor of Urology and Pharmacology and director of the University of Colorado Cancer Center who led a multidisciplinary team of investigators from the University of Colorado, Indiana University, the University of Virginia and Yale University, in a news release. 

The study found that inactive ‘Ral' cavity disappears when the protein becomes active. Furthermore, researchers took the findings to human cells and treated these with compounds to see which resulted in the greatest reduction of Ral activitation.

Findings revealed that a derivative of the RBC8 dubbed BQU57 had entered the tumor tissue and also slowed the growth of the tumors.

"We still need to optimize these compounds and then characterize these agents for toxicity in several animal species and determine their optimal route of delivery, such as oral or intravenous before moving to the clinic," Theodorescu concluded. "But we see this work as a valuable first step in the development of a novel class of therapeutic agents directed at Ral. The concept of targeting sites on proteins that collapse upon activation, and whose collapse is required for activation, could in principle be used to discover drugs aimed at other proteins driving human disease as well."