Introduction: Despite the significant survival improvement in childhood acutelymphoblastic leukemia (ALL), 15-20% of patients continue to relapse; outcomes following relapse remain suboptimal and have room for further improvement. Advances in genomics have shed new insights on the biology of ALL, led to the discovery of novel genomically defined ALL subtypes, refined prognostic significance and revealed new therapeutic vulnerabilities.Areas covered: In this review, the authors provide an overview of the genomic landscape of childhood ALL and highlight recent advances in molecular-based and antibody-based pharmacological approaches in the treatment of childhood ALL, from emerging preclinical evidence to published results of completed clinical trials.Expert opinion: Molecularly targeted therapies and immunotherapies have expanded the horizons of ALL therapy and represent promising therapeutic avenues for high-risk and relapsed/refractory ALL. These novel therapies are now moving into frontline ALL therapy and may define new treatment paradigms that aim to further improve survival and reduce chemotherapy-related toxicities in the management of pediatric ALL.
Keywords: Acute lymphoblastic leukemia; children; genomics; immunotherapy; precision medicine; targeted therapy.