Only one class of targeted agents (anti-GD2 antibodies) has been incorporated into front-
line therapy for neuroblastoma since the 1980s. The Neuroblastoma New Drug …

The use of targeted small-molecule therapeutics and immunotherapeutics has been limited
to date in pediatric oncology. Recently, the number of pediatric approvals has risen, and …

Abstract Purpose: The 2-part, phase I/IIa, open-label study (NCT01677741) sought to
determine the safety, tolerability, pharmacokinetics, and preliminary activity of dabrafenib in …

Background Immune therapies have revolutionized cancer treatment over the last few years
by allowing improvements in overall survival. However, the majority of patients is still primary …

D-2-hydroxyglutaric aciduria type II (D2HGA2) is a severe inborn disorder of metabolism
caused by heterozygous R140 mutations in the IDH2 (isocitrate dehydrogenase 2) gene …

Background Although the role of epigenetic abnormalities has been studied for several
years in cancer genesis and development, epigenetic-targeting drugs have historically failed …

The impressive progress recently observed in adult cancers through the introduction of new
drugs has not yet been translated to adolescents 12–17years of age [defined according to …

Background The most appropriate design of Phase-II trials evaluating new therapies in
osteosarcoma remains poorly defined. Objective To study consistency in phase-II clinical …

In the past decade, the landscape of drug development in oncology has evolved
dramatically; however, this paradigm shift remains to be adopted in early phase clinical trial …

Incremental improvements in the treatment of children and adolescents with cancer have led
to 5-year survival rates reaching nearly 85%. In the past decade, impressive progress has …