In a significant stride towards advancing paediatric oncology, the University College London (UCL) and Great Ormond Street Hospital (GOSH) have recently published groundbreaking results from a Phase II clinical trial. The study focused on treating BRAF-mutated low-grade gliomas (BM-LGG), a form of brain tumour prevalent in children. The promising findings were shared in prestigious medical journals, the New England Journal of Medicine, and the Journal of Clinical Oncology.

The Trial:

The Phase II clinical trial targeted 73 children diagnosed with BRAF-mutated low-grade gliomas. This specific type of brain tumour is characterised by alterations in the BRAF gene, making it a prime target for novel therapeutic approaches. The treatment regimen employed a combination of two drugs developed by Novartis: Tafinlar (dabrafenib) and Mekinist (trametinib). Both drugs are designed to interfere with the cancer cells’ signalling pathways, ultimately inhibiting their growth.


The trial reported encouraging results, showcasing the effectiveness of the Tafinlar and Mekinist combination in managing BRAF-mutated low-grade gliomas in paediatric patients. Not only did the treatment demonstrate a significant reduction in tumour size, but it also exhibited a favourable safety profile, a crucial aspect when dealing with young patients.

The publication of these results in the New England Journal of Medicine and the Journal of Clinical Oncology underscores the significance of this breakthrough. It not only adds valuable insights to the field of paediatric oncology but also serves as an optimistic step forward for families facing the challenges of BRAF-mutated low-grade gliomas.

Implications for Paediatric Oncology:

The success of this clinical trial holds profound implications for the field of paediatric oncology. BRAF-mutated low-grade gliomas, though considered less aggressive than other brain tumours, pose unique challenges in terms of treatment. The use of Tafinlar and Mekinist presents a targeted and effective strategy that can potentially revolutionise the standard of care for these cases.

Also, the positive safety profile observed in the trial is particularly significant for paediatric patients. The reduced toxicity of the treatment is crucial in minimising side effects and ensuring a better quality of life for the young participants. This aspect can significantly impact the long-term wellbeing of children undergoing treatment for BRAF-mutated low-grade gliomas.

Future Directions and Challenges:

While the results are undoubtedly promising, further research and clinical trials will be necessary to solidify the long-term efficacy and safety of the Tafinlar and Mekinist combination. Additionally, the identification of potential resistance mechanisms and the development of strategies to overcome them will be vital in ensuring sustained success in treating BRAF-mutated low-grade gliomas.


The collaboration between UCL and GOSH, coupled with the innovative approach of utilising Tafinlar and Mekinist, marks a significant milestone in paediatric oncology. The successful results from the Phase II clinical trial offer hope to families and healthcare professionals dealing with BRAF-mutated low-grade gliomas, bringing us one step closer to more effective and targeted treatments for childhood brain tumours. As the research progresses, these findings pave the way for a brighter and healthier future for children facing the challenges of paediatric brain cancer.

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