The FDA has granted an expanded accelerated approval for entrectinib (Rozlytrek®, Genentech Inc.) for pediatric patients, one month or older, with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion without a known acquired resistance mutation, that are metastatic or where surgical resection is likely to result in severe morbidity, and that have progressed following treatment or have no satisfactory standard therapy. Previously, entrectinib was granted accelerated a...

The FDA has approved ibrutinib (Imbruvica®, Pharmacyclics LLC) for treatment of pediatric patients with chronic graft-versus-host disease (cGVHD) following failure of one or more lines of systemic therapy. "Pediatric cGVHD, a potentially debilitating, life-threatening complication of allogeneic hematopoietic stem cell transplantation, has limited treatment options," wrote Marco Zecca, MD, Director of the Pediatric Oncology/Hematology Unit at the Fondazione IRCCS Policlinico San Matteo in Italy, ...

At the recent American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, Illinois, Dr. Melissa Hudson of St. Jude Research Hospital, spoke with Oncology Data Advisor about the phase II trial of dabrafenib/trametinib for BRAF V600–mutant pediatric low-grade glioma, which was presented at the meeting by Dr. Eric Bouffet of the University of Toronto's Hospital for Sick Children. Dr. Hudson provides commentary on the study's results and shares additional insights into her research regar...

The FDA has approved azacitidine (Vidaza®, Celgene) for patients with newly diagnosed juvenile myelomonocytic leukemia (JMML), prior to allogeneic hematopoietic stem cell transplantation (HSCT). "JMML is a rare, unique myeloproliferative/myelodysplastic neoplasia of early childhood driven by canonical Ras-pathway mutations in PTPN11, NRAS, KRAS, NF1, or CBL," wrote Charlotte M. Niemeyer, MD, Professor and Medical Director in the Department of Pediatric Hematology and Oncology at Medical Center F...

Scientists recently discovered that improved cancer treatment approaches over time correlate with higher life expectancy in adult survivors of childhood cancer. As cancer treatment evolves to be more effective, more patients are surviving, with some even living long lives disease free. In order to determine if life expectancy also rises with evolving treatment, the investigators examined data derived from the Childhood Cancer Survivor Study, which included data on 5-year survivors of childhood c...

Chemotherapy is an effective treatment for many cancers; however, the side effects can have huge consequences, especially on pediatric patients, who are still growing. In particular, children treated for medulloblastoma—the most common malignant pediatric brain tumor—have devastating lifelong side effects from chemotherapy. Less harmful treatments are needed. In a new study published in Nature Communications, scientists have discovered that inhibiting an epigenetic modifier protein called lysine...

In a new study conducted by researchers at Princeton University in collaboration with Michigan State University and the University of Oslo, a unified computational framework called Unveiling RNA Sample Annotation for Human Diseases (URSAHD) analyzed hundreds of molecular patterns of various diseases instantaneously, detecting genes behind a wide variety of diseases. Four of the genes that the researchers identified were linked to neuroblastoma, a rare form of cancer that predominantly affects in...

In vitro fertilization (IVF) is a popular method used to conceive children. However, it can increase chances of birth defects and imprinting disorders, both of which are associated with a higher risk of childhood cancer. Researchers at the University of Minnesota conducted a cohort study in order to investigate whether there is an increased occurrence of cancer among children conceived via IVF compared with children conceived naturally. For the study, published in JAMA Pediatrics, the researcher...

In vitro fertilization (IVF) has been thought to potentially cause an increased risk of cancer in children, especially because IVF is linked to childhood cancer risk factors such as imprinting disorders and birth defects. Logan Spector, PhD, Professor at University of Minnesota, and colleagues found a small correlation between IVF and childhood cancer; however, the increased rate of embryonal cancers, specifically hepatic tumors, could not be attributed to IVF per se rather than to underlying in...

Both chromosomal anomalies and nonchromosomal birth defects substantially increase the risk of childhood cancer, a new study reports. Around one in 33 children has a birth defect. Some birth defects have already been linked to childhood cancer. For example, trisomy 21—the presence of an extra #21 chromosome, which causes Down syndrome—is associated with acute leukemia. However, an overall determination of increase in childhood cancer risk in those with birth defects has been limited by small sam...

Children with central nervous system (CNS) tumors have good prognoses; 75% will live at least five years after diagnosis. Unfortunately, a recent study has shown that these survivors experience neuropsychological problems and lower socioeconomic status later in life compared with their healthy peers. For this study, published in Cancer, the researchers analyzed data on 181 adult survivors of pediatric low-grade gliomas from the Childhood Cancer Survivor Study and compared them with a group of 10...

As technology and health care progress, the rate of children who survive five years after being diagnosed with cancer is 80%; however, this rate only applies to children who live in high-income countries (HICs). Those children that live in low-income and middle-income countries (LMICs) make up more than 90% of children who are at risk of developing cancer, and, unfortunately, children who live in LMICs lack the resources necessary to combat childhood cancer compared with children who live in HIC...

​In their recently published study, Philip J. Lupo, PhD, MPH, and colleagues found that both chromosomal anomalies and nonchromosomal birth defects significantly increase a child's risk of receiving a cancer diagnosis before his or her 18th birthday. In this interview with i3 Health, Dr. Lupo discusses his findings and the future directions of this research. Can you comment on the significance of your results concerning the link between birth defects and pediatric cancer risk? Philip J. Lupo, Ph...