Researchers Develop Targeted RNA Drug to Combat Cancer Growth

A team of researchers at the Hebrew University of Jerusalem has developed a groundbreaking drug molecule capable of precisely targeting and destroying TERRA, an RNA molecule essential for the survival of certain cancer cells. This innovative approach, which utilizes advanced RIBOTAC technology, marks a significant step towards a new generation of RNA-based cancer treatments that aim to address the disease at its genetic foundation rather than merely alleviating symptoms.

The research, led by Dr. Raphael I. Benhamou, Elias Khaskia, and Dipak Dahatonde, was published in the journal Advanced Science. Their findings focus on TERRA, a molecule that plays a vital role in protecting the ends of chromosomes, which are crucial for maintaining cellular health and stability. When TERRA malfunctions, it can contribute to issues related to cell aging and division. In various cancers, particularly certain types of brain and bone cancers, cancer cells exploit TERRA to sustain their growth and proliferation.

“We’ve created a tool that acts like a guided missile for bad RNA,” said Dr. Benhamou. “It can find TERRA inside cancer cells and make it disappear—without harming healthy parts of the cell.” This precise targeting is achieved through a small molecule engineered to recognize a specific shape that TERRA adopts, known as a G-quadruplex. Once the molecule identifies TERRA, it recruits a natural cell enzyme, RNase L, to dismantle the RNA.

This research represents a significant milestone as it is the first instance in which scientists have successfully targeted and destroyed TERRA with such accuracy. The molecule selectively targets TERRA while leaving similar RNA molecules intact. In laboratory tests involving cancer cell lines, including HeLa and U2OS cells—known for their resistance to treatment—the new drug significantly reduced TERRA levels and hindered cancer growth.

The implications of this discovery could be profound. By focusing on RNA molecules rather than just proteins, which are the traditional targets for most drugs, this research opens new avenues for treating cancers that have previously been deemed difficult to target. “This is a new way of thinking about medicine,” Dr. Benhamou explained. “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.”

The work conducted by the team at Hebrew University of Jerusalem highlights the potential of RNA-targeted therapies in oncology, suggesting a shift towards more precise and effective cancer treatments in the future. As research continues, it may pave the way for innovative strategies that could transform the landscape of cancer therapy, providing hope for patients battling various forms of this challenging disease.