Results from a study led by Professor Eric Bouffet at the University of Toronto have been shared on the Journal of Clinical Oncology. The paper covers the phase I and II paediatric study, exploring how a new drug combination effects particular mutations in childhood cancer.
About the study
The study focused on individuals with the BRAF V600 mutation, which occurs in 20% childhood low-grade gliomas. This mutation potentially increases the growth of a tumour by producing a protein that is involved in sending signals in cells and in cell growth.
During this trial, the team aimed to:
- Establish the correct dosage for the two drugs being tested (trametinib and dabrafenib)
- Understand efficacy and safety
- Establish the pharmacokinetics of the drugs. (Pharmacokinetics is the study of how an organism affects a drug.)
139 patients aged 18 or younger with relapsed malignant tumours were included in the trial. 91 of them received the cancer cell-inhibiting drug trametinib, and the remaining cohort of 48 people received trametinib and the BRAF-specific growth limiting drug, dabrafenib.
Results and next steps
The research concluded that the trial provided a strong evaluation of the aims discussed. Tolerability of the drug combination was positive for those who received both drugs, this means the drug combination can be used to treat those with this mutation in the future.
The paper states that other cohorts will be reported elsewhere, so there are further results to be expected regarding the efficacy and future of this drug combination.
With further insights on the horizon and the success regarding tolerability and safety results, those in the paediatric community with the unfortunate diagnosis of a low-grade BRAF-mutated tumour could see their pathway improved. These developments could provide a greater chance of limiting tumour growth and a better quality of life.
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