Research led by Dr. Jezabel Rodriguez Blanco has shown that a drug called Minnelide, initially developed for pancreatic cancer, can increase symptom-free survival in preclinical medulloblastoma models without any signs of toxicity. Medulloblastoma is the most common malignant brain tumor in children, with Group 3 historically having the worst survival rates. This group was the focus of Blanco’s research due to high levels of the MYC oncogene, which is believed to cause cancer.
Triptolide, the active ingredient in Minnelide, has previously shown the ability to target MYC in other cancers. Blanco initiated research on this property in Group 3 medulloblastoma based on experiences during her postdoctoral fellowship. Despite the oncogene’s poor druggability, her initial results were promising. Mechanism of action remains a challenge due to multiple effects of the drug, but Blanco is confident in its efficacy. Minnelide’s effectiveness in tumors with extra MYC copies was significantly higher than in tumors with normal MYC levels.
While Blanco works on understanding the drug’s mechanisms, she also emphasizes the urgent need to provide potential benefits to patients. The potential impact on children diagnosed with Group 3 medulloblastoma is substantial, with current survival rates being very low. Blanco’s decision to prioritize publication over further research reflects the need to make progress quickly. Minnelide’s success in preclinical models displaying efficacy in reducing tumor growth and enhancing the effects of chemotherapy is a key factor pushing for rapid dissemination of results.
Minnelide has seen success in phase I and phase II clinical trials for pancreatic cancer in adults, and Blanco believes that this research on Group 3 medulloblastoma could lead to a clinical trial for children with the disease. She emphasizes the importance of moving quickly to provide potential benefits to these patients. Further research is needed to fully understand the drug’s mechanisms of action specific to cancer cells, but the initial results are highly encouraging.
The drug’s ability to affect MYC gene expression through RNA pol II activity and protein stability showcases the complexity of its mechanism. Despite these challenges, Blanco remains confident in Minnelide’s effectiveness. The drug has shown remarkable efficacy in reducing tumor growth and preventing the spread of cancer cells, showing promise in improving treatment outcomes for Group 3 medulloblastoma patients. Blanco’s research represents a significant step forward in the search for effective treatments for this aggressive form of brain cancer.
Blanco’s dedication to pushing this research forward, despite challenges, highlights the urgency in addressing the needs of children with Group 3 medulloblastoma. The research provides hope for patients with this aggressive form of brain cancer, and the potential for a clinical trial using Minnelide offers a ray of hope for improved treatment outcomes. This work represents a significant advancement in the fight against medulloblastoma and underscores the importance of continued research and development in finding effective treatments for pediatric brain tumors.