Research on Childhood Cancers

NCI Director Dr. Ned Sharpless discusses progress against childhood cancers, like CAR T-cell therapy and collecting more comprehensive data on children and adolescents with cancer, and the need to better address disparities in childhood cancer.

FDA has approved a new form of asparaginase called Rylaze. The drug was developed to help alleviate shortages of Erwinia asparaginase, a key part of treatment for children and adults with acute lymphoblastic leukemia.

Standard radiation for medulloblastoma can cause long-term damage to a child’s developing brain. A new clinical trial suggests that the volume and dose of radiation could be safely tailored based on genetic features in the patient’s tumor.

A team of international researchers has identified mutations in several genes, including TP53, MYOD1, and CDKN2A, that appear to be associated with an aggressive form of rhabdomyosarcoma in children. The findings could lead to more targeted treatments for the disease.

In a recent study, a treatment regimen using brentuximab vedotin (Adcetris) instead of the chemotherapy drug vincristine allowed some children and teens with advanced Hodgkin lymphoma to avoid radiation therapy—and the long-term health problems that can come with it.

The results of two trials establish blinatumomab (Blincyto) as a new standard treatment for children and young adults with high-risk relapsed B-cell acute lymphoblastic leukemia after remission has been achieved and before a stem cell transplant.

For children with the eye cancer retinoblastoma, researchers are studying a CAR T-cell therapy in which the engineered immune cells are packaged in a biodegradable material called a hydrogel and then injected directly into tumors.

For children and adults with advanced soft tissue sarcoma, adding pazopanib (Votrient) to chemotherapy and radiation before surgery may be a promising treatment option, early results from a clinical trial suggest.

Fusion proteins drive the development of many cancers in children, yet little is known about their biology. NCI’s Fusion Oncoproteins in Childhood Cancers Consortium brings together experts from varied disciplines to investigate these cancers.

Since launching the Childhood Cancer Data Initiative, NCI has undertaken a range of research activities to support this important effort. In this Cancer Currents post, NCI Director Dr. Norman Sharpless provides an update on these efforts.

For children with aggressive Burkitt lymphoma and other B-cell non-Hodgkin lymphomas, adding rituximab (Rituxan, Truxima) to chemotherapy substantially increases the likelihood of the child being cured, results from a large clinical trial show.

The Food and Drug Administration (FDA) has approved selumetinib (Koselugo) to treat children with neurofibromatosis 1 (NF1), a genetic disorder that causes tumors, called plexiform neurofibromas, to form throughout the nervous system.

A new study offers insight into genetic alterations associated with osteosarcoma, the most common bone tumor of children and adolescents, and the findings have implications for genetic testing of children with osteosarcoma and their families.

An NCI clinical trial finds the drug selumetinib improves outcomes for children with neurofibromatosis type 1 (NF1), shrinking inoperable tumors called plexiform neurofibromas, reducing pain, and improving function and overall quality of life.

Children with retinoblastoma in low- and middle-income countries were, on average, diagnosed at an older age and with more advanced disease than those in high-income countries, an analysis shows. The data provide clues about global disparities in outcomes.

For children and young adults with certain relapsed B-cell acute lymphoblastic leukemia (B-ALL), the immunotherapy drug blinatumomab is superior to standard chemotherapy, an NCI-sponsored Children’s Oncology Group trial shows.

Only 1.5% of children with acute lymphoblastic leukemia who skipped radiation had a recurrence in the central nervous system, according to a recent trial. The therapy, which is intended to prevent such a recurrence, can have devastating side effects.

For many children with high-risk neuroblastoma, receiving two separate stem cell transplants is more beneficial than receiving one, according to the results of an NCI-supported clinical trial conducted by the Children’s Oncology Group.

FDA has approved entrectinib (Rozlytrek) for the treatment of children and adults with tumors bearing an NTRK gene fusion. The approval also covers adults with non-small cell lung cancer harboring a ROS1 gene fusion.

To prepare for the proposed Childhood Cancer Data Initiative, NCI sponsored a 3-day symposium that brought together pediatric cancer researchers, advocates, and other stakeholders.

An early report from the NCI–COG Pediatric MATCH trial shows that 24% of young patients with advanced cancer who had their tumors tested for genetic changes were eligible to receive one of the targeted therapies being tested.

Some children with liver cancer may need less chemotherapy than is typically used, new research suggests. The study included children and infants with hepatoblastoma whose tumors had been surgically removed when the disease was diagnosed.

NCI has created special clinics that bring together clinicians, patients, and advocates to promote more rapid progress against rare cancers. The effort includes both rare pediatric cancers and central nervous system tumors in adults.

An experimental CAR T-cell therapy may have potential as a treatment for several types of childhood cancer, results from a new study in mice suggest. The CAR T cells eradicated tumors in mouse models of several different childhood cancers, including two forms of sarcoma and medulloblastoma.

A liquid biopsy blood test can detect DNA from brain tumors called diffuse midline gliomas, researchers have found. This minimally invasive test could be used to identify and follow molecular changes in children with these highly lethal brain tumors.

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.

The drug sodium thiosulfate can protect the hearing of children with cancer undergoing treatment with the chemotherapy drug cisplatin, results from a new clinical trial show. The trial involved children with a form of liver cancer called hepatoblastoma.

This NCI-funded Children’s Oncology Group trial tested the addition of nelarabine (Arranon) to standard treatment for children and young adults with T-cell acute lymphoblastic leukemia (T-ALL).

In a phase 2 clinical trial, the investigational drug selumetinib shrank tumors in some children with the genetic syndrome neurofibromatosis type 1 (NF1). The tumors, called neurofibromas, can cause pain, difficulty breathing or walking, and disfigurement.

Results from an NCI-sponsored clinical trial may point to an important change in how some children with advanced Wilms tumor, a form of kidney cancer, are treated.

An interactive website designed to promote physical activity among children and adolescents who have completed treatment for cancer may indeed help encourage them to get regular exercise, according to preliminary results from a pilot study.

An NCI-funded study has found significant differences in the genetics of acute myeloid leukemia in younger and older patients. The findings could help guide the development of treatments tailored specifically for childhood AML.

On November 9, the FDA approved dasatinib (Sprycel®) for the treatment of children with chronic myelogenous leukemia (CML) whose cancer cells express the Philadelphia chromosome and whose disease is in a relatively early stage, known as the chronic phase.

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.

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.