A groundbreaking advancement in pediatric oncology is on the horizon as the NHS in England prepares to introduce the first-ever targeted treatment for brain tumors in children and young adults. This innovative treatment, designed to be administered at home, has shown remarkable effectiveness in slowing down the progression of the disease, offering a brighter outlook and improved quality of life for young patients over an extended period.
The pressing need for better treatment options for pediatric brain tumors is underscored by the challenges posed by gliomas, which are abnormal growths in the brain or spinal cord. These tumors vary in severity, with low-grade gliomas progressing slowly and high-grade variants presenting a more aggressive and often fatal threat.
Each year in the UK, approximately 150 children are diagnosed with low-grade gliomas and 30 with high-grade gliomas, highlighting the urgent demand for improved care options.
The groundbreaking treatment involves a combination of dabrafenib and trametinib, which has demonstrated the ability to halt disease progression for significantly longer than standard chemotherapy. Specifically tailored for children diagnosed with low-grade gliomas carrying the BRAF V600E mutation, this treatment not only extends progression-free survival but also mitigates the harsh side effects commonly associated with traditional chemotherapy.
This targeted therapy, endorsed by the National Institute for Health and Care Excellence (NICE), is poised to transform the management of gliomas in children and young adults aged one to 17. By targeting the proteins produced by the altered BRAF gene, which fuels tumor growth, the treatment aims to disrupt the disease’s progression at a molecular level.
Helen Knight, director of medicines evaluation at NICE, emphasized the devastating impact of glioma brain tumors on children and their families and highlighted the limited and often brutal treatment options available. She expressed satisfaction in recommending this new combination therapy, which not only extends the time without tumor growth but also enhances the quality of life for affected children and their families.
Clinical trials have demonstrated the superiority of dabrafenib with trametinib over conventional chemotherapy in terms of both effectiveness and tolerability. The treatment has shown a median progression-free survival of 24.9 months for low-grade gliomas, compared to just 7.2 months with standard chemotherapy.
Furthermore, the oral administration of the treatment, with dabrafenib in dissolvable tablets taken twice daily and trametinib in an oral solution taken once daily, offers added convenience for young patients and their families.
The NHS is gearing up to roll out this groundbreaking brain tumor treatment in the coming months, providing renewed hope to children and young people facing this challenging diagnosis.
- : public
