Global Impact of Monoclonal Antibodies (mAbs) in Children: A Focus on Anti-GD2

SIMPLE SUMMARY: Despite being in use for almost 50 years, monoclonal antibodies face limitations in their implementation in clinical practice, particularly in pediatrics and pediatric cancer. Although technological advancements and research into new therapeutic targets have led to the development of sophisticated and effective molecules, translational barriers still exist. Integrating monoclonal antibodies (mAbs) into current treatment protocols and ensuring accessibility for all children with cancer globally remains a challenge. This review examines the biological, clinical, economic, and social limitations hindering the global implementation of mAbs in pediatric cancer, with a particular focus on anti-GD2 mAbs. ABSTRACT: Monoclonal antibodies (mAbs), as the name implies, are clonal antibodies that bind to the same antigen. mAbs are broadly used as diagnostic or therapeutic tools for neoplasms, autoimmune diseases, allergic conditions, and infections. Although most mAbs are approved for treating adult cancers, few are applicable to childhood malignancies, limited mostly to hematological cancers. As for solid tumors, only anti-disialoganglioside (GD2) mAbs are approved specifically for neuroblastoma. Inequities of drug access have continued, affecting most therapeutic mAbs globally. To understand these challenges, a deeper dive into the complex transition from basic research to the clinic, or between marketing and regulatory agencies, is timely. This review focuses on current mAbs approved or under investigation in pediatric cancer, with special attention on solid tumors and anti-GD2 mAbs, and the hurdles that limit their broad global access. Beyond understanding the mechanisms of drug resistance, the continual discovery of next generation drugs safer for children and easier to administer, the discovery of predictive biomarkers to avoid futility should ease the acceptance by patient, health care professionals and regulatory agencies, in order to expand clinical utility. With a better integration into the multimodal treatment for each disease, protocols that align with the regional clinical practice should also improve acceptance and cost-effectiveness. Communication and collaboration between academic institutions, pharmaceutical companies, and regulatory agencies should help to ensure accessible, affordable, and sustainable health care for all.