A six-year-old boy named Leo from Peterborough has become the first patient in the UK to participate in a global clinical trial evaluating a novel drug for the treatment of low-grade gliomas. Diagnosed in the summer of 2023 after experiencing headaches and sickness, Leo's tumor is located centrally in the brain, making surgical removal unsafe. The trial, conducted at Cambridge University Hospitals NHS Foundation Trust (CUH), aims to determine if the new drug is more effective than standard chemotherapy for this specific group of brain tumor patients.
Trial Design and Objectives
The study focuses on newly diagnosed pediatric low-grade gliomas that cannot be completely removed surgically and contain an activating RAF alteration. This alteration acts as an 'on' switch, driving tumor growth. The investigational drug, administered weekly as a liquid or capsule, is being compared to standard chemotherapy delivered intravenously. The primary objective is to assess the drug's ability to stabilize tumors and induce shrinkage, while also reducing the long-term side effects associated with multiple treatments like surgeries, chemotherapy, and radiotherapy.
Professor Matthew Murray, Honorary Consultant Paediatric Oncologist at CUH and Principal Investigator for the trial in Cambridge, highlighted the drug's potential based on previous results: "This trial is a really exciting development as the investigational drug showed promising results for patients who had relapsed or refractory low-grade gliomas. Now, the trial will test whether it shows benefit for newly diagnosed patients."
Patient Perspective and Clinical Insights
Leo's mother, Corey, expressed hope that the trial would not only benefit her son but also future patients and their families. "Being on this trial isn’t just going to help Leo, it’s going to help the next kid and their family, because they’ll be better prepared, and they’ll know a bit more about treatments," she said.
Dr. Charlie Burns, Leo’s Consultant Paediatric Oncologist at CUH, emphasized a key advantage of the trial design: "The advantage for patients of being on this trial is that if they are assigned to the standard chemotherapy and it is shown not to work, then they can automatically ‘cross over’ to receive the investigational drug."
The Broader Impact
The drug was developed by scientists at a US biotechnology company. The trial is supported by the National Institute for Health and Care Research (NIHR), which facilitates research delivery in the UK. The NIHR's Network Director for the East of England, Dr. Helen Macdonald, expressed gratitude to Leo and his family, as well as others participating in research, for enabling life-changing discoveries through clinical trials.
