Scientists at the Hebrew University of Jerusalem have developed a new drug molecule that specifically destroys TERRA, an RNA essential for some cancer cells' survival. Using a technique called RIBOTAC, the drug identifies and breaks down this RNA without harming healthy cells. Tests in cancer cell lines showed reduced growth, suggesting potential for novel therapies.
A team from the Hebrew University of Jerusalem's Faculty of Medicine has created a drug-like molecule to target TERRA, an RNA molecule that maintains chromosome ends and supports cell stability. When TERRA malfunctions, it can enable cancer cells in tumors like those of the brain and bone to grow uncontrollably.
The researchers employed RIBOTAC, or Ribonuclease-Targeting Chimera, to build a small molecule that recognizes a unique G-quadruplex fold in TERRA. This structure allows the molecule to recruit the cell's RNase L enzyme, which degrades the RNA. As Dr. Raphael I. Benhamou explained, "We've created a tool that acts like a guided missile for bad RNA. It can find TERRA inside cancer cells and make it disappear—without harming healthy parts of the cell."
In experiments with HeLa and U2OS cancer cell lines—representing difficult-to-treat cancers—the treatment lowered TERRA levels and slowed cell growth. This is the first demonstration of selectively destroying TERRA without affecting similar RNAs.
The study, conducted by Dr. Benhamou, Elias Khaskia, and Dipak Dahatonde, was published in Advanced Science in 2025 (DOI: 10.1002/advs.202512715). It highlights a shift toward RNA-targeting therapies, beyond traditional protein-focused drugs. Benhamou noted, "This is a new way of thinking about medicine. Instead of focusing only on proteins, we're now learning how to target the RNA that controls them. That could open the door to treating diseases we once thought were impossible to reach."
This approach could reshape cancer treatments by addressing genetic drivers at their core.