Paediatric brain tumours and solid tumours remain a significant challenge in oncology, with conventional treatment modalities such as chemotherapy often limited by toxicity, resistance, and a lack of specificity. These limitations have led to suboptimal survival outcomes and significant long-term side effects in children. The bottleneck of chemotherapy underscores the urgent need for innovative therapeutic approaches that can improve efficacy while reducing collateral harm.
Advances in diagnostic techniques and molecular classification have revolutionized our understanding of paediatric cancers. High-throughput sequencing and molecular profiling have enabled the identification of distinct genetic and epigenetic alterations driving individual tumours. These discoveries have paved the way for the stratification of childhood cancers into biologically defined subgroups, allowing for more accurate prognostication and tailored therapeutic strategies.
In parallel, the field of precision medicine has seen the rapid development of novel agents, including targeted therapies, immunotherapies, and small-molecule inhibitors, that address the unique molecular vulnerabilities of paediatric tumours. Agents such as tyrosine kinase inhibitors, immune checkpoint inhibitors, and cellular therapies are transforming the therapeutic landscape, offering hope for improved outcomes in diseases previously deemed refractory. However, the translation of these breakthroughs into clinical practice for children remains challenging, with hurdles including limited clinical trial data, regulatory complexities, and the unique biology of paediatric cancers compared to their adult counterparts.
There is no free lunch in precision medicine; it relies heavily on a strong multidisciplinary team to seek and deliver novel agents effectively. Hopefully, after this sharing, you will be convinced that precision medicine is not optional but mandatory for every child suffering from cancer.
