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Neuro-oncology: The emergence of a promising targeted therapy

A ground-breaking study to investigate novel treatment avenues for young brain cancer patients, initiated by Dr. Sébastien Perrault in 2018, continued this year. The aim is to test the efficacy and neuropsychological impact of trametinib, a drug that has the potential to limit the sequelae of benign brain tumours.  Close to 70 patients were recruited, and the preliminary statistics compiled are very encouraging. 

  • 24% of patients saw their lesion shrink or, in one case, disappear altogether; 
  • 74% of tumours remained stable; 
  • Only one patient’s tumour grew; 
  • Improvements were also observed in neurological symptoms and, in some cases, pain reduction.  

The non-malignant brain tumours targeted by this research — namely low-grade gliomas and plexiform neurofibromas — are the most frequent types of pediatric tumours. Neurofibromas occur in association with a disorder known as neurofibromatosis type 1, one of the most frequently encountered genetic conditions, affecting one child out of every 3,000.  

These noncancerous lesions can nevertheless have serious consequences, including significant comorbidities, partial or total loss of vision, motor impairments, coordination problems, bone deformities, and more.  

Thank you to the Fondation des Gouverneurs de l’espoir for their support of this vital and pioneering research. 

We are constantly thinking about finding more effective treatments with as few side effects as possible; it’s what guides us through more difficult times, such as therapeutic impasse for our patients. Donor funding, especially for clinical trials, allows us to transform our dreams into reality.

Dr Sébastien Perreault