Research on Childhood Cancers

Progress continues to be made with the Gabriella Miller Kids First Research Program, which is creating opportunities for investigators from different research communities to share resources and collaborate on research into childhood cancers and certain birth defects.

FDA has approved tisagenlecleucel (Kymriah™), a type of immunotherapy called CAR T-cell therapy, for some children and young adults with advanced acute lymphoblastic leukemia (ALL).

In a small clinical trial, the drug crizotinib shrank tumors in children with cancers that have alterations in the ALK gene.

Health problems that develop years later as a result of a cancer treatment are known as late effects. The Childhood Cancer Survivor Study (CCSS) was started in 1994 to better understand and address these late effects.

Two studies have identified proteins that drive growth of diffuse intrinsic pontine glioma (DIPG) tumor cells. Blocking these targets with investigational drugs slowed tumor growth in animal models.

Researchers have created a long-sought-after mouse model for an aggressive form of acute lymphoblastic leukemia that occurs commonly in infants and that the researchers believe can accelerate the development of new therapies for the disease.

In an early-phase clinical trial of a new oral drug, selumetinib, children with the common genetic disorder neurofibromatosis type 1 (NF1) and plexiform neurofibromas, tumors of the peripheral nerves, tolerated selumetinib and, in most cases, responded to it with tumor shrinkage.

Research from TARGET, an NCI-funded program supporting genomic analyses of childhood cancers, has led to the launch of several clinical trials of targeted therapies to treat children with acute lymphoblastic leukemia.

Dr. Greg Armstrong of the Childhood Cancer Survivor Study discusses this long-running study and its future.

NCI’s Dr. Javed Khan discusses a new NCI clinical program and the promise and challenges of genome-guided therapy for children with cancer.

Researchers have identified a genetic rearrangement that may drive the development of a rare benign brain tumor in children through three distinct biological mechanisms simultaneously.

An international research team has identified a possible new treatment target for pediatric neuroblastoma and is planning an early-stage clinical trial of a drug that inhibits this target.

In the most comprehensive study of its kind conducted to date, more than 8 percent of children with cancer were found to have inherited genetic mutations associated with a predisposition to the disease.

Genomic methods, such as DNA sequencing, may help doctors manage the treatment of children and young adults with cancer.

NCI has awarded grants to five research teams to participate in its Pediatric Preclinical Testing Consortium, which is intended to help to prioritize which agents to pursue in pediatric clinical trials.

Survivors of childhood cancer have an elevated risk of developing second, distinct cancers into their forties and beyond, according to a new study.

NCI convened a workshop of representative research teams that have been leaders in defining the genomic landscape of childhood cancers to discuss the influence of genomic discoveries on the future of childhood cancer research.

New data from the Childhood Cancer Survivor Study suggest that refinements in pediatric cancer treatment over the last few decades have helped to extend the lifespans of many survivors of childhood cancer.

Final results from a phase III randomized clinical trial show that addition of aprepitant to the anti-nausea drug ondansetron, with or without dexamethasone, effectively prevents chemotherapy-induced nausea and vomiting in pediatric patients.

NCI’s Dr. Malcolm Smith discusses future directions of childhood cancer research, including treatment directions and clinical trials.

Researchers have identified genetic variations in children with brain cancer that increased their risk of rapid hearing loss after treatment.

A study published in the June 6, 2012, issue of The Lancet shows that radiation exposure from computed tomography (CT) scans in childhood results in very small but increased risks of leukemia and brain tumors in the first decade after exposure.

A study suggests that at least some children diagnosed with acute lymphoblastic leukemia who respond poorly to initial chemotherapy may do better if they receive additional chemotherapy rather than a stem cell transplant.

In this trial, patients up to age 18 who are newly diagnosed with diffuse intrinsic pontine gliomas (DIPG) or who have other high-grade gliomas that could not be completely resected will undergo radiation therapy and receive oral lenalidomide at increasing doses daily for 6 weeks, followed by lenalidomide alone.

An alliance of several large-scale prospective cohort studies of children to pool data and biospecimens and study various modifiable and genetic factors in relation to cancer risk.