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Assorted News from the Last Week: The rare pediatric disease PRV program to incentivize drug development for rare pediatric diseases (originally the Creating Hope Act) was extended past its sunset date (September 30) to December 20, 2024.  While the US House has reauthorized it through 2029, the Senate must do so before the end of the Congress for it to remain in effect after Decembermber. In a bold step toward addressing the stark disparities in pediatric cancer care, Dana-Farber Cancer Institute is taking the lead in launching IGNITE—the first national, pediatric hematology-oncology health equity research consortium. Experts have developed a breakthrough [...] Read more

Assorted News from the Last Week: Implementation of the RACE for Children Act has substantially lowered the number of adult cancer drugs exempted from pediatric testing. A systematic review and meta-analysis of 23 randomized clinical trials showed a cannabinoids used for medical purposes in children and adolescents come with a risk of adverse events. Novel treatment regimen yields promising response in advanced-phase chronic myeloid leukemia. Vaccine experts report positive results on Phase 1 trial of personalized vaccine for lymphoplasmacytic lymphoma. Non-Hispanic black children experience poorer post-relapse survival outcomes than white children with nonmetastatic osteosarcoma. AACR report highlights improvements in outcomes [...] Read more

Assorted News from the Last Week: Bullying significant issue for young people in aftermath of eye cancer. Personalized vaccine trial for diffuse hemispheric glioma will expand to include children as young as five. Effectiveness of structured, multidisciplinary long-term care for pediatric cancer survivors. Live birth and maternity outcome in childhood and adolescent cancer survivors under 18 years at diagnosis: a 40-year population-based cohort study RNA-sequencing study provides novel insights into chronic lymphocytic leukemia. How technology is changing pediatric cancer treatment in India. Proteomic profiling of cerebrospinal fluid reveals TKT as a potential biomarker for medulloblastoma. Upcoming Webinars, Online Opportunities, and [...] Read more

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