Novel Therapeutic Approaches

Abstract

During the latter part of the twentieth century, substantial improvements were made in the treatment of childhood cancers. Fifty years ago, most pediatric cancers were fatal. Today, however, more than 75% of pediatric patients with cancer survive the disease. Most advances have been achieved through extensive research and well-designed clinical trials. Investigators continue to explore novel therapies with the goals of providing salvage treatment to patients whose disease does not respond to current therapies and of improving the efficacy and safety of frontline regimens. Recent advances in the fields of genetics, immunology, and molecular biology have led to a better understanding of childhood cancers, which in turn has led to the rational design of drugs based on a thorough understanding of targets on or inside malignant cells. Such an approach aims to further improve cure rates and reduce toxicity. This chapter describes the novel therapies available to pediatric patients at M. D. Anderson Cancer Center. We describe the difficulties of conducting innovative research in children as well as how multidisciplinary interactions at M. D. Anderson help children quickly and safely benefit from novel therapies, including stem cell transplantation for leukemia, lymphoma, and other solid tumors.