by Researchers have made significant progress in understanding and potentially expanding treatment options for medulloblastoma, an aggressive childhood cancer, according to a study published in Molecular Cell. Medulloblastoma affects both young and school-aged children and involves the development of a brain tumor that obstructs the flow of fluid around the brain, resulting in symptoms such as severe headaches, vomiting, sleepiness, fussiness, and difficulty walking and coordinating movements.
The current treatment for medulloblastoma is intensive and typically includes high-dose chemotherapy, surgery, and sometimes radiation or stem cell transplants. However, the one-size-fits-all approach to treatment proves ineffective for many patients, particularly those with certain genetic variations of the disease.
Dr. John Prensner, one of the senior aauthors of the study and an Assistant Professor of Pediatrics and Biological Chemistry, states that gaining a deeper understanding of medulloblastoma at the genetic and cellular level is crucial for tailoring treatment options and developing new therapies.
To achieve this, Prensner’s team collaborated with Dr. Sebastiaan van Heesch and his research group at the Princess Máxima Center for pediatric oncology in the Netherlands. Together, they examined multiple groups of patients, focusing on those who had the most aggressive form of medulloblastoma and the worst outcomes.
Several of these patients exhibited genetic and biological factors that worsened their cancers, including the presence of the cancer gene MYC. The researchers explored how MYC functioned and its potential implications in cancer development.
“What if this MYC is doing important functions outside of what we know to be genes in these cancer cells?” Prensner questioned. This new perspective led his team to investigate the role of essential microproteins in medulloblastoma, specifically the micro-protein ASNSD1-uORF, which appeared to be vital for the survival of MYC cancer cells.
The discovery that these overlooked tiny proteins play a significant role in the growth of cancer cells is groundbreaking and emphasizes the need for expanded research into childhood cancers.
By further understanding the role of MYC and other genetic, biological, and cellular factors, healthcare providers can identify interventions and develop therapeutics that target the molecular processes enabling cancer cell growth.
Dr. Prensner’s interest in medulloblastoma began early in his career when he encountered patients living with the tumor as a medical student. He was deeply moved by their experiences, driving him to utilize his scientific training and expertise to advance the understanding and treatment of the disease.
“I’ve never forgotten, not just the patients, but the families. That’s why it’s been so important to me to try to use the interests and training that I have in the scientific fields to think about how we can really unlock new areas of understanding about this disease to get these patients better choices,” Prensner explained.
Overall, this research is a significant step towards improving treatment options and outcomes for children battling medulloblastoma. The findings provide valuable insights into the genetic and cellular workings of the disease, ultimately leading to personalized therapies and better quality of life for patients and their families.
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1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it
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