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Assorted News from the Last Two Weeks: National Cancer Institute’s CCDI Molecular Characterization Initiative adds rare tumors. Study provides first in-depth look at major mix-ups in the genomic terrain of pediatric high-grade gliomas. LLS is working to change the pediatric clinical trial model. The aftermath of childhood cancer:  2000 days without Ana Biden appoints former government scientist as first leader of ARPA-H. The use of tisagenlecleucel (Kymriah) in children younger than 3 years of age with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL) was as effective, and safe, in a retrospective cohort study as it was previously seen to […]

Assorted News from the Last Two Weeks: The Food and Drug Administration (FDA) approved Imbruvica (ibrutinib) for the treatment of children aged 1 year or older with chronic graft-versus-host disease (cGVHD) after one or more lines of systemic therapy, according to a news release from the agency. FDA drug approvals, pediatrics — 2022 midyear review Guidebook for caregivers of children with rare and/or serious illnesses 10 gifts to give a friend whose child has cancer or another serious illness Researchers identified a metabolic vulnerability in neuroblastoma that could be exploited to overturn these tumors’ resistance to therapy. Millions of Americans, […]

Assorted News from the Last Week: Leukemia and Lymphoma Society’s (LLS) has launched its Dare to Dream Project as an extension of itself Children’s Initiative.  It will expand education and support services for patients and families, drives advocacy and policy efforts, and increases investments in childhood blood cancer research. Neuroblastoma cells have an extreme need for amino acids. Scientists at the Hopp Children’s Cancer Center Heidelberg (KiTZ), the German Cancer Research Center (DKFZ), the University of Heidelberg, and HI-STEM gGmbH have now discovered the molecular mechanisms underlying this and how the cancer cells could be turned off. From the Washington […]

Assorted News from the Last Two Weeks: The CCDI Molecular Targets Platform is the National Cancer Institute’s instance of the Open Targets Platform, specific to childhood cancers which will allow researchers to find data from multiple sources on molecular targets—molecules involved in the growth and spread of cancer cells. Identifying these targets, how they affect cancers, and how they interact with drugs can help improve how childhood cancers are treated. To explore the Molecular Targets Platform, visit: moleculartargets.ccdi.cancer.gov. From the Washington Post:  Pushed by parent activists and buoyed by new funding, scientists explore novel strategies to treat deadly pediatric cancers. […]

Assorted News from Last Week: The Biden administration has selected Monica Bertagnolli, a renowned surgical oncologist, as the next director of the National Cancer Institute. (gated) The Pediatric Oncology Group of Ontario issued its latest report on childhood cancer efforts in Ontario. Bringing next-generation CAR T-cell therapies to children with solid cancers The Food and Drug Administration (FDA) recently approved the combination of the targeted drugs dabrafenib (Tafinlar)and trametinib (Mekinist) for the treatment of people with nearly any type of advanced solid tumor that has a specific mutation in a gene called BRAF. Results were reported from a phase I safety […]

Assorted News from Last Week: Researchers have identified the child cancer patients at greatest risk of developing life-threatening infections, in a crucial step towards the development of an early diagnostic test. A report out this month (highlights) highlights some of the data capacity needs to handle patient-centered outcomes  and also describes how U.S. Department of Health and Human Services (HHS)’s Office of the Assistant Secretary for Planning and Evaluation (ASPE) could help to address these data capacity needs. Intratumoral infusion of an oncolytic virus offered safe and feasible results, along with encouraging efficacy, in pediatric patients with diffuse intrinsic pontine […]

Assorted News from Last Week: Three things to know if your child is diagnosed with cancer. CNN Correspondent Rene Marsh tells the story of her pediatric cancer journey with her 9-month old son:  “It was worse than a nightmare because I couldn’t wake up from this.” Gilead Sciences has unveiled a new global pediatric drug development center in Ireland that will have 33 job openings by the end of 2022.   Upcoming Webinars, Online Opportunities, and Meetings: Join advocates from around the country for the 2022 Rally for Medical Research! The Hill Day will take place on Wednesday, September 14, with […]

Assorted News from Last Week: Childhood cancer survivors of certain racial and ethnic groups were prone to more hospitalizations for various conditions years down the road. Survivorship Matters Blog:  Recapturing the Dream NCI’s new Molecular Characterization Initiative (MCI) is expanding comprehensive molecular characterization of tumors to children, adolescents, and young adults (AYAs) with newly diagnosed soft tissue sarcomas receiving care at hospitals affiliated with the Children’s Oncology Group. Intratumoral infusion of an oncolytic virus offered safe and feasible results, along with encouraging efficacy, in pediatric patients with diffuse intrinsic pontine glioma (DIPG). Some children with a type of brain cancer called […]

Assorted News from Last Week: Kazia Therapeutics Limited, an oncology-focused drug development company, is pleased to announce that the United States Food and Drug Administration (FDA) has awarded Orphan Drug Designation (ODD) to Kazia’s paxalisib for the treatment of atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer. Bridge therapy ahead of autologous stem cell transplantation (ASCT) consolidation may benefit certain high-risk neuroblastoma patients with residual disease following induction regimens, according to a multicenter retrospective study. Novartis announced the FDA has granted accelerated approval to the BRAF inhibitor dabrafenib (Tafinlar) combined with the MEK inhibitor trametinib […]

Assorted News from Last Week: The Coalition Against Childhood Cancer is pleased to announce the 2022 Annual Summit hosted by Children’s Hospital of Philadelphia and presented by Day One Biopharmaceuticals.  This annual event reflects the CAC2 values and mission by providing top-notch educational programming, designing multiple opportunities for members to connect and engage, and showcasing the ongoing work of our members who are working for a future without childhood cancers. On the basis of extensive experience in recent years, most notably with mAbs, persisting with administering myeloablative chemotherapy as the standard to treat children with high risk neuroblastoma is not […]