The cancer gene MYC has been called the "Mount Everest" of cancer research because of the difficulty of designing medications that can disable it, and the expectation that an effective MYC drug could help so many cancer patients. A collaboration among RNA scientists, chemists and cancer biologists in Florida and Germany has climbed that peak, while opening new routes to summit other similarly hard-to-treat diseases.
The researchers describe their strategy today in the journal Nature.
The scientists' approach directs cells' recycling enzymes to cancer genes' RNA and cuts up key segments to prevent them from doing harm. The tactic worked against the MYC cancer gene, and also two other challenging cancer genes, JUN and MIR155. All three of the cancer genes regulate the transcription of other genes, igniting rapid tumor growth.
"For cancer patients whose disease is driven by these common but challenging oncogenes, the RNA degrader approach may offer new hope," said Herbert Waldmann, Ph.D., director of the Max Planck Institute of Molecular Physiology in Dortmund, Germany.
Their study also opens new possibilities for targeting RNA with medicines, opening potentially many other genetic diseases to this treatment approach, said chemist and institute professor Matthew D. Disney, Ph.D., of The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology and the UF Health Cancer Center.
"We discovered around 2,000 new RNA structures that are able to bind drug-like small molecules, and identified six new chemotypes able to bind RNA," Disney said. "We basically have created an encyclopedia of druggable RNA folds."
