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Assorted News from the Last Week: The emotional toll of chronic illness in childhood: Doing more to promote mental well-being in pediatric patients. Young children treated for brain tumors are less ready for school than their peers. The FDA has awarded rare pediatric disease designation and orphan drug designation to INV724 for the treatment of patients with neuroblastoma. U.K. charities give $36 million to start new drugs for childhood tumors: New consortium hopes to advance drug candidates in long-neglected area of cancer treatment. (gated) Childhood Cancer Awareness Month is in full swing.  National Cancer Institute highlighter several important streams of [...] Read more

Assorted News from the Last Week: White House issues proclamation on National Childhood Cancer Awareness Month, 2024 CAC2 Member Blog–Wisconsin Pediatric Cancer Action Plan Published! Improving care in pediatric oncology: why patient- and family-centered support is essential throughout the cancer survivorship continuum. FDA grants Orphan Drug designation to Opaganib for neuroblastoma. The Rare Cancer Initiative is enabling the research community to access more clinical data to improve health outcomes for children diagnosed with rare cancers. Dr. Mary Frances Wedekind, a pediatric oncologist and assistant research physician with the Pediatric Oncology Branch at NCI, explains how the Childhood Cancer Data Initiative follows [...] Read more

Assorted News from the Last Week: Early interventions may improve long-term academic achievement in young childhood brain tumor survivors. Early onset psychosis in a pediatric oncology setting: a case report. Approximately 80% of pediatric tumors occur in low- and middle-income countries (LMIC), where diagnostic tools essential for treatment decisions are often unavailable or incomplete. Technology could be part of the solution of addressing the diagnostic limitations. CAC2 Member Pine Tree Apple Classic Fund and Children’s Minnesota announce new $1 Million cancer research endowment fund. Work in childhood cancer informs adult cancer options: Menin inhibitors in pediatric acute leukemia. Lions Club donates [...] Read more

Assorted News from the Last Week: The NCI Budget Fact Book summarizes the distribution of the fiscal year 2023 budget among the various NCI research programs and funding mechanisms, funding policies influencing grant awards, and comparisons with prior year allocations. Additional information on the NCI budget is accessible from the NCI home page. A collaborative study supported by the Gabriella Miller Kids First Pediatric Research Program (Kids First) and National Institutes of Health (NIH) Common Fund, published today in the journal Nature, found that approximately 60% of the genetic changes driving T-ALL cancer cells are non-coding changes. Researchers announced a [...] Read more

Assorted News from the Last Week: Lifestyle habits can alleviate the effects of cancer treatment in children. A study into precision medicine for high-risk Neuroblastoma has revealed encouraging results. The DNA methylome of pediatric brain tumors appears shaped by structural variation and predicts survival. Late-onset tumors in rhabdoid tumor predisposition syndrome type-1 (RTPS1) and implications for surveillance. Pharmacists have a crucial role in the treatment of sarcomas in the pediatric setting. Feel good story of the week: 224th Officer Basic Course (OBC) student at The Judge Advocate General’s Legal Center and School and his 4-year-old daughter Davey. Researchers identified genetic [...] Read more

Assorted News from the Last Week: Congratulations to CAC2 Members Autumn Gentry (MACC Fund, Inc) and Mariah Forster Olson (Neuroblastoma Children's Cancer Society) with inspiration and guidance from CAC2 Board Member Ann Ramer on the publication of the Wisconsin Pediatric Cancer Action Plan. A tale of resilience: Physician fights Blue Cross of Idaho to cover her baby's brain cancer treatment with lorlatanib. The National Institutes of Health’s (NIH) All of Us Research Program has started limited enrollment of children from birth through age 4 at five partner health care organizations across the country. Expanding participation to children offers researchers the [...] Read more

Assorted News from the Last Week: CAC2 Supporting Member Day One Biopharmaceutical and Ipsen unveiled an ex-U.S. licensing agreement for tovorafenib (approved as Ojemda in the U.S.) in pediatric low-grade glioma (pLGG) and "any future indications developed by Day One" outside the U.S. Several CAC2 Members contributed to the recently published manuscript that was produced by the ACCELERATE Paediatric Strategy Forum for Medicinal Product Development of PI3 K-inhibitors. The FDA has accepted the resubmission of a biologics license application (BLA) seeking the approval of remestemcel-L (Ryoncil) for the treatment of pediatric patients with steroid-refractory acute graft-vs-host-disease (SR-aGVHD). It Mattered Later: [...] Read more

Assorted News from the Last Week: Congratulations to CAC2 Supporting Organization Day One Biopharmaceuticals and CAC2 member authors and reviewers who contributed to the white paper “Advancing Pediatric Cancer Research and Drug Development through Multi-Stakeholder Collaboration: A Framework Based on the Tovorafenib Journey.” Read about the collaborative effort that made this paper possible on the CAC2 Collaborative Achievement Blog. More frequent use of genetic testing in pediatric oncology, along with the application of advanced testing methods, holds great promise for identifying patients with CPS as well as detecting somatic variants that inform diagnosis, prognosis, and treatment. The FDA has granted [...] Read more

Assorted News from the Last Week: Psychological and ethical issues raised by genomic testing in paediatric care pathway, a qualitative analysis with parents and childhood cancer patients. Immuno-oncologic profiling of pediatric brain tumors reveals major clinical significance of the tumor immune microenvironment. CAR-T therapy, which harnesses a person’s own immune cells, racks up some astonishing success stories against deadly brain tumours in children. Researchers has identified and located a population of stem-like cells that initiates and maintains Group 3 medulloblastoma (Gr3-MB) in the developing brain. They showed that eliminating the small population of stem-like cells present in Gr3-MB tumors led [...] Read more

Assorted News from the Last Week: Building a case for incorporating patient advocates throughout the research continuum in pediatric oncology. The NCI Council of Research Advocates met virtually on June 26 and heard the latest legislative report, with updates on appropriations, cancer-related legislation, and other ways that NCI is engaging and advocating. An article in support of the Creating Hope Reauthorization Act: "The rare pediatric disease voucher program creates new treatments. I have new data to prove it." Benefits for children with suspected cancer from routine whole-genome sequencing. Upcoming Webinars, Online Opportunities, and Meetings: 56th Congress of the International Society [...] Read more