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GUIDELINES/RECOMMENDATIONS The precision medicine strategy of Mass General Cancer Center led to a remarkable 91 percent shrinkage in tumor size, and every one of the 15 patients who underwent at least one cycle of treatment responded positively.
A groundbreaking study led by Mass General Cancer Center has shed light on a revolutionary approach to treating papillary craniopharyngiomas (PCPs), utilizing precision medicine to considerably reduce these brain tumors.
Key Points:
- Papillary craniopharyngiomas (PCPs) are rare brain tumors that cause significant health challenges for patients.
- Existing cancer treatments can leave patients with long-term health issues, such as neuroendocrine dysfunction or vision or memory loss.
- The Mass General Cancer Center spearheaded the first multicenter treatment protocol for PCPs.
- The study utilized BRAF/MEK inhibitors in a phase II clinical trial with 16 patients, resulting in an average tumor shrinkage of 91 percent.
Additional Points:
- All patients who completed one or more cycles of therapy responded to treatment, indicating the highest response rate to date of any medical therapy for brain tumors.
- The trial was based on the discovery that approximately 95 percent of PCPs have a BRAF V600E mutation, which drives their cancerous activity.
- The BRAF/MEK inhibitors used in the trial have been approved by the FDA for treating other cancers like melanoma.
- Though some patients experienced adverse reactions, many patients tolerated the therapy well and elected to continue therapy beyond the prescribed cycles due to their positive response.
Conclusion:
- The study’s findings indicate a paradigm shift in brain tumor treatment, showing the powerful potential of precision medicine when applied to the right target with the right drugs.
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“These unprecedented results signal a paradigm shift for targeting brain tumors because they show that, with the right target and the right drugs, precision medicine can have a dramatic impact on brain tumors.”
Priscilla Brastianos, MD
Director of the Central Nervous System Metastasis Center
Mass General Cancer Center