Childhood Cancer Research Results and Study Updates
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Helping Kids with Cancer Speak for the Supportive Care They NeedPosted:
Younger cancer patients who filled out surveys about their symptoms experienced fewer cancer-related symptoms than those who did not complete the surveys, results from two clinical trials have shown. The findings show patient-reported outcome (PRO) surveys for children can give an accurate picture of how a child is feeling during the stress of treatment.
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Tovorafenib Approved for Some Children with Low-Grade GliomaPosted:
FDA has granted an accelerated approval to tovorafenib (Ojemda) for kids and teens who have low-grade glioma with changes in the BRAF gene. In a small clinical trial, the drug shrank or completely eliminated tumors in nearly 70% of patients.
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For childhood cancer survivors, inherited genetic factors influence risk of cancers later in lifePosted:
Common inherited genetic factors that predict cancer risk in the general population may also predict elevated risk of new cancers among childhood cancer survivors. Findings could potentially inform screening and long-term follow-up of those at greatest risk.
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How a Simple Tool Is Saving Lives of Children with Cancer in Latin AmericaPosted:
A system for catching treatment-related complications in children with cancer has proven to be highly effective in many Latin American hospitals. An NCI-funded study aims to help make these early warning systems sustainable.
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Blinatumomab Increases Survival for Infants with an Aggressive Type of ALLPosted:
Giving the drug blinatumomab (Blincyto) after standard chemotherapy substantially increased survival for infants with an aggressive form of acute lymphoblastic leukemia (ALL), a recent study showed. If confirmed in larger studies, the treatment may become standard therapy for infants with ALL caused by KMT2A rearrangements.
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Liquid Biopsies on the Horizon for Children with Solid CancersPosted:
Results from a new study highlight the progress being made toward developing liquid biopsies specifically for use in children with solid cancers like Ewing sarcoma and Wilms tumor. The tests can help detect and diagnose cancer and monitor for response to treatment and recurrence.
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Dexrazoxane Protects the Heart Long Term for Kids Being Treated for CancerPosted:
Doxorubicin is used to treat many types of childhood cancer, but it can damage the heart. Giving dexrazoxane (Zinecard) before each dose substantially decreases a child’s risk of treatment-related heart problems in adulthood, new study results show.
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ETV6 Protein Could Be an Important Target for Ewing Sarcoma TreatmentPosted:
The protein ETV6 appears to promote tumor growth by affecting the behavior of the EWS-FLI1 fusion protein that drives most Ewing sarcomas. The research groups that made the discovery hope it leads to a targeted therapy for the aggressive childhood cancer.
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Brentuximab Approved for High-Risk Hodgkin Lymphoma in Children and AdolescentsPosted:
Based on an NCI-sponsored clinical trial conducted by the Children’s Oncology Group, FDA has approved the drug brentuximab vedotin (Adcetris) in combination with chemotherapy for some children and adolescents with Hodgkin lymphoma.
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Drug Combination Shows Promise for Rhabdomyosarcoma, but Can It Get to Clinical Trials?Posted:
An NCI study in mice has identified a drug combination that may help treat children with rhabdomyosarcoma. But one of the drugs, ganitumab, is no longer being made. Based on the study's promising results, the NCI researchers who led the study want to test the combination in humans.
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Sodium Thiosulfate Approved to Reduce Chemo-Related Hearing Loss in Children with CancerPosted:
The chemotherapy cisplatin often causes permanent hearing loss. Sodium thiosulfate (Pedmark) is the first treatment approved by FDA that can reduce the risk of hearing loss and the severity of damage to the inner ear in children treated with cisplatin.
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Making Transformative Advances against Childhood Cancer: A Conversation with Dr. Doug HawkinsPosted:
Dr. Doug Hawkins, chair of the NCI-funded Children’s Oncology Group, discusses advances in treating children with cancer, COG’s role in conducting clinical trials, and efforts like the Childhood Cancer Data Initiative and Molecular Characterization Initiative.
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Study Confirms Dinutuximab Extends Life for Children with High-Risk NeuroblastomaPosted:
Researchers have confirmed that the immunotherapy drug dinutuximab (Unituxin) can help children with high-risk neuroblastoma live longer. The finding is based on a trial of nearly 1,200 children with the disease.
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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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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.