Researchers at the Greehey Children’s Cancer Research Institute (Greehey CCRI) at UT Health San Antonio continue to push the boundaries of pediatric cancer research with the support of a new grant awarded by the Pediatric Cancer Foundation.
Luiz O. Penalva, PhD, has received funding for his project, “RNA Therapeutics to Target Aggressive Medulloblastoma,” which aims to develop a novel treatment approach for one of the deadliest forms of childhood brain cancer.
Medulloblastoma is the most common malignant brain tumor diagnosed in children. Among its various subtypes, molecular group 3 (G3) medulloblastoma is particularly aggressive, accounting for approximately one-third of all cases and the majority of medulloblastoma-related deaths. Despite decades of research, survival outcomes for children diagnosed with G3 medulloblastoma have seen only limited improvement, highlighting the need for innovative therapeutic strategies.
Dr. Penalva’s research focuses on a class of molecules known as small nucleolar RNAs (snoRNAs). These molecules belong to a larger family of non-coding RNAs that help regulate gene expression. One of their primary functions is supporting ribosome biogenesis—the complex process by which cells produce ribosomes, the molecular machinery that makes proteins.
Cancer cells rely heavily on ribosome production to sustain their rapid growth and survival. Dr. Penalva’s team has discovered that the cancer-driving gene MYC, a key driver of G3 medulloblastoma, alters the ribosome production process by controlling specific snoRNAs and other factors involved in ribosome biogenesis. This remodeling enables tumor cells to manufacture proteins that promote growth, metastasis, and resistance to treatment.
The project will investigate the use of antisense oligonucleotides (ASOs), short synthetic molecules designed to selectively target and destroy disease-causing RNAs. ASOs have emerged as a promising therapeutic platform because they can precisely target molecular abnormalities while minimizing effects on healthy cells.
Through this grant, Dr. Penalva and his team will conduct preclinical studies to evaluate ASOs that target aberrantly expressed snoRNAs in G3 medulloblastoma. Their goal is to lay the foundation for a new RNA-based therapeutic strategy that can slow tumor growth, overcome treatment resistance, and ultimately improve outcomes for children facing this devastating disease.
“This project represents an exciting opportunity to translate our understanding of RNA biology into a potential new treatment approach for children with high-risk medulloblastoma,” said Dr. Penalva. “By targeting the molecular mechanisms that cancer cells depend upon, we hope to identify vulnerabilities that can be exploited therapeutically.”
The Pediatric Cancer Foundation’s investment in this research underscores the critical importance of supporting innovative science to improve the lives of children with cancer. Discoveries made through projects like this bring researchers one step closer to developing safer, more effective therapies for pediatric patients and their families.
At Greehey CCRI, scientists remain committed to translating today’s discoveries into tomorrow’s cures, advancing research that offers new hope for children battling cancer.
More about the Pediatric Cancer Foundation: