Novel Cancer Drug Targets RNA Weak Points in Tumor Cells

Researchers from the Hebrew University of Jerusalem have developed a novel class of drug molecule that specifically targets and destroys TERRA, an RNA molecule crucial for the survival of certain cancer cells.

This innovative approach employs a technique known as RIBOTAC, short for Ribonuclease-Targeting Chimera, allowing the compound to locate TERRA within cancer cells and dismantle it while sparing healthy RNA.

The findings, published in Advanced Sciences, are led by Dr. Raphael I. Benhamou, Elias Khaskia, and Dipak Dahatonde, who emphasize the significance of TERRA in maintaining chromosome stability. When TERRA functions abnormally, it can lead to unchecked cell growth and division, contributing to various cancers, including some brain and bone tumors.

Dr. Benhamou described their creation as a tool that acts like a guided missile targeting harmful RNA. The RIBOTAC molecule identifies a specific structural feature of TERRA called a G-quadruplex, and it recruits the natural enzyme RNase L to facilitate the breakdown of the RNA.

This marks the first successful demonstration of a targeted approach that can accurately eliminate TERRA without affecting other RNA molecules. In laboratory experiments with cancer cell lines such as HeLa and U2OS, which represent challenging cancer types, the treatment effectively reduced TERRA levels and inhibited cancer cell proliferation.

This discovery points to a paradigm shift in cancer treatment strategies, suggesting a focus on RNA as a therapeutic target rather than solely on proteins, which are the primary focus of current medications.

The researchers assert that this new perspective could pave the way for innovative medicines that tackle diseases previously deemed untreatable, highlighting the potential for RNA-targeting therapies to reshape future cancer treatments.