Childhood Cancer Research Results and Study Updates
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For Some Kids with Brain Cancer, Targeted Therapy Is Better than ChemoPosted:
The combination of dabrafenib (Tafinlar) and trametinib (Mekinist) shrank more brain tumors, kept the tumors at bay for longer, and caused fewer side effects than chemotherapy, trial results showed. The children all had glioma with a BRAF V600 mutation that could not be surgically removed or came back after surgery.
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NCI launches program to offer molecular characterization of childhood cancersPosted:
The Molecular Characterization Initiative will offer state-of-the-art diagnoses to children, adolescents, and young adults with central nervous system tumors.
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Good News and Cautions about Pregnancy for Childhood Cancer SurvivorsPosted:
As adults, survivors of childhood cancer aren’t any less likely to have healthy babies than those without a history of cancer, a new study shows. However, they may have more health risks during pregnancy, warranting more intensive obstetric care.
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Test Detects Early Signs of Remaining Cancer in Kids Treated for MedulloblastomaPosted:
A new test could potentially be used to identify children treated for medulloblastoma who are at high risk of their cancer returning. The test detects evidence of remaining cancer in DNA shed from medulloblastoma tumor cells into cerebrospinal fluid.
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Drug combination helps children with acute promyelocytic leukemia avoid conventional chemotherapyPosted:
A Children’s Oncology Group trial shows that the combination of all-trans retinoic acid and arsenic trioxide is highly effective in children with acute promyelocytic leukemia. The therapy avoids or minimizes the use of conventional chemotherapy.
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Seeing a Promising Future for Progress against Childhood CancerPosted:
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.
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FDA Approval of Rylaze Will Address Drug Shortage for Childhood ALLPosted:
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.
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For Kids with Medulloblastoma, Trial Suggests Radiation Can Be TailoredPosted:
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.
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International study of rare childhood cancer finds genetic clues, potential for tailored therapyPosted:
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.
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Brentuximab May Mean Less Radiation for Children, Teens with Hodgkin LymphomaPosted:
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.
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Blinatumomab Improves Survival in Children with Relapsed LeukemiaPosted:
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.
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For Childhood Eye Cancer, Researchers Investigating “Packaged” CAR T CellsPosted:
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.
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Pazopanib Shows Promise for Children, Adults with Soft Tissue SarcomasPosted:
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.
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Scientists Focus on Fusion Proteins in Childhood CancersPosted:
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.
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CCDI Activities Enhance NCI’s Childhood and AYA Cancer ResearchPosted:
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.
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New Drug Regimen Cures More Children with Aggressive B-Cell LymphomaPosted:
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.
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Selumetinib Approved by FDA to Treat Children with NF1Posted:
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.
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NCI study provides genetic insights into osteosarcoma in childrenPosted:
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.
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In NIH trial, selumetinib shrinks tumors, provides clinical benefit for children with NF1Posted:
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.
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For Children with Retinoblastoma, Disparities Seen across the GlobePosted:
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.
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Immunotherapy drug improves outcomes for some children with relapsed leukemiaPosted:
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.
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Children with Acute Lymphoblastic Leukemia Can Skip Radiation to the BrainPosted:
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.
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For Children with Neuroblastoma, Trial Results Highlight Continued Evolution of TreatmentPosted:
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.
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FDA Approves Entrectinib Based on Tumor Genetics Rather Than Cancer TypePosted:
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.
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The Childhood Cancer Data Initiative: Transforming the Pediatric Cancer Landscape through Sharing DataPosted:
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.
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Pediatric MATCH Study Finds More Targetable Genetic Changes than ExpectedPosted:
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.
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Some Children with Liver Cancer May Need Less Chemotherapy, Study SuggestsPosted:
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.
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NCI’s Rare Cancer Clinics: Engaging Patients and Fostering CollaborationPosted:
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.
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A CAR T-Cell Therapy for Multiple Childhood Cancers?Posted:
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.
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Blood Test Shows Promise for Detecting Genetic Changes in Brain TumorsPosted:
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.
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Childhood Cancer Survivor Study: An OverviewUpdated:
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.
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Sodium Thiosulfate Prevents Cisplatin-Induced Hearing Loss in Some ChildrenPosted:
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.
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Trial Produces Practice-Changing Findings for Some Children, Young Adults with LeukemiaPosted:
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).
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Selumetinib Continues to Show Promise in Children with NF1Posted:
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.
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Some Children with Wilms Tumor Can Receive Less Therapy, Study SuggestsPosted:
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.
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Testing an Interactive Approach to Promote Exercise in Young Cancer SurvivorsPosted:
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.
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TARGET Study Finds Major Differences between Childhood and Adult AMLPosted:
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.
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Dasatinib Approved by FDA for Some Children with Chronic Myelogenous LeukemiaPosted:
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.
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Kids First Pediatric Research Program Moves ForwardPosted:
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.
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CAR T-Cell Therapy Approved for Some Children and Young Adults with LeukemiaPosted:
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).
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Crizotinib Shows Promise for Childhood CancersPosted:
In a small clinical trial, the drug crizotinib shrank tumors in children with cancers that have alterations in the ALK gene.
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Studies Identify Potential Treatment Strategies for Pediatric DIPG Brain TumorsPosted:
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.
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New Mouse Model Closely Mimics Most Common Leukemia in InfantsPosted:
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.
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Early-phase trial demonstrates shrinkage in pediatric neural tumorsPosted:
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.
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TARGET Initiative Fueling Progress against Childhood LeukemiaPosted:
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.
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The Impact and Future of the Childhood Cancer Survivor Study: An Interview with Greg Armstrong, M.D.Posted:
Dr. Greg Armstrong of the Childhood Cancer Survivor Study discusses this long-running study and its future.
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Toward Precision Therapy for Children with Cancer: An Interview with Dr. Javed KhanPosted:
NCI’s Dr. Javed Khan discusses a new NCI clinical program and the promise and challenges of genome-guided therapy for children with cancer.
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Gene Fusion May Drive Rare Childhood Brain TumorPosted:
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.