Game-changing clinical trial results offer new hope for children with relapsed paediatric high-grade glioma.
Paediatric high-grade gliomas (pHGG) are the leading cause of childhood cancer-related deaths. Currently, treatment for children with pHGGs is surgery followed by radiotherapy and chemotherapy, but despite this, two-year survival rate is less than 35%, and most patients develop recurrent disease for which there is no accepted standard of care.
Promising results from a phase 2 clinical trial, led by Dr Darren Hargraves from Great Ormond Street Institute of Child Health, London, have shown a dramatic improvement in overall survival for children with relapsed or recurrent pHGG.
The results, published in the Journal of Clinical Oncology, show that patients treated with a new combination of drugs called Dabrafenib and Trametinib, had a median overall survival of 32.8 months, compared to the median overall survival of just 5.6 months for historical groups who received standard treatment. What’s more, patients treated with the new combination experienced fewer adverse side effects than those treated with standard chemotherapy, indicating the treatment may not only improve survival, but could also have a less harmful impact on quality of life.
The trial focused on a subgroup of pHGGs patients who had a BRAF V600E mutation driving their cancer, which accounts for approximately 5-10% of all pHGGs. Dabrafenib and Trametinib are drugs that act on this BRAF gene and have already been approved as treatments for a variety of cancers as monotherapies (on their own), as well as a combination therapy (together).
Dr Karen Noble, our Director of Research, Policy and Innovation, said: “The results from this phase 2 trial are very promising, offering much-needed hope of personalised treatments for high-grade gliomas based on the specific genetic driver of the cancer. Studies like this demonstrate how crucial large-scale genomic research can be.
“Phase 2 clinical trials are designed to establish the efficacy and safety of novel treatment, so more work is needed before this treatment will be available to patients, but the results from this trial and the fact the treatment is FDA approved for the same mutation in other cancers are certainly encouraging. We support any efforts to boost drug repurposing.”
Published in the Journal of Clinical Oncology, 20th November 2023.
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