Enabling precision medicine and targeted therapeutics in low- and middle-income country.
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Childhood brain tumors are the leading cause of cancer-related morbidity and mortality in children worldwide. Delayed diagnosis and inadequate diagnostic and treatment infrastructure are significant barriers to an optimal outcome in low- and middle-income countries (LMICs), where 80% of children with cancer live. Pediatric neuro-oncology has undergone a transformation with recent developments in molecular diagnostics and the emergence of targeted therapies. However, the implementation of precision medicine in LMICs is challenging in view of high costs, lack of trained personnel, and restricted access to essential medications. Targeted therapies such as MEK, BRAF & NTRK inhibitors have demonstrated promising efficacy with improved toxicity profiles in pediatric brain tumors with actionable mutations. Despite their potential, availability in LMICs is largely restricted to compassionate use programs and select clinical trials. Encouraging results, albeit from few institutions in LMICs, have demonstrated that targeted therapies have a significant impact on quality of life (QOL), in addition to comparable efficacy. Sustainable integration of precision medicine in LMICs requires improved access to affordable molecular diagnostics, inclusion of more targeted drugs in the WHO Essential Medicines List, and participation in international clinical trials. Collaborative models-such as twinning programs, telemedicine, regional reference laboratories, and capacity-building initiatives-can support implementation. Global programs like the WHO Global Initiative for Childhood Cancer and the ATOM Coalition play a vital role in improving drug access and policy advocacy. Enabling precision medicine in LMICs calls for a coordinated multi-stakeholder approach, including integration into the national cancer care policy, and is critical to bridging the survival gap and ensuring equitable outcomes for children with brain tumors globally.