First Child Cured of Brain Stem Glioma

Medical progress in recent decades has boosted the survival rate of children with cancer to 85%, but brain stem glioma remains a severe outlier. Dr. Jacques Grill, a French doctor, initially predicted a grim outcome for 6-year-old Lucas diagnosed with this rare and deadly tumor. However, an experimental treatment, randomly assigned to Lucas, led to the complete disappearance of the tumor, a unique case globally.

Officially known as diffuse intrinsic pontine glioma (DIPG), this rare cancer affects around 300 children annually in the United States and 100 in France. The standard two-year survival rate is only 10%, with radiotherapy as the primary treatment option.

Lucas participated in the Biomede trial in France, where he was randomly assigned the drug everolimus. Remarkably, he took the medication for over 5 years, resulting in the complete disappearance of his tumor. While seven other children from the trial survived, none experienced a complete tumor disappearance like Lucas. His case is now being considered a potential breakthrough for improving long-term outcomes in DIPG cases.

Lucas’s tumors had a rare genetic mutation, making them exceptionally responsive to everolimus. Biomedical researchers are now aiming to replicate this mutation in vitro, potentially paving the way for further trials to confirm its effectiveness. Although the journey toward an approved medication may take 10 to 15 years, scientists are optimistic about the accelerated pace of technological advancements.

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