Genomic medicine for pediatric and adolescent and young adult (AYA) cancers

Abstract

Although the number of pediatric and adolescent and young adult (AYA) patients with cancer is much smaller than that of adult cancer patients, malignant disease is one of the significant causes of death among pediatric and AYA patients. In particular, patients with relapse or metastatic disease still have a dismal prognosis, and effective standard therapies have not been established for these patients with intractable cancers. On the other hand, recent advances in genome-wide approaches provide specific gene-mutation-based therapies for patients with intractable pediatric and AYA cancers. However, gene targets, novel molecular targeted drugs, and clinical trials for patients with pediatric and AYA cancers are currently limited in Japan. In addition, since the gene mutation spectrum in pediatric and AYA patients differs from that of adult patients, development of specific cancer multigene panels in pediatric and AYA cancers is necessary. Thus, further improvement of genomic medicine for pediatric and AYA cancers is necessary.