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Assorted News from the Last Week: CAC2 Individual Member Jennifer Flowers writes compellingly about "When the National Drug Shortage Becomes Personal" for her. Published in JAMA: "Stakeholder Perspectives on Randomized Clinical Trials for Children With Poor-Prognosis Cancers." Many CAC2 Members contributed to this of how and when randomized clinical trials (RCTs) should be deployed to best meet the needs of all involved. Reported at the ASH meeting this week: An investigational CAR T-cell therapy induced responses in nearly every single patient with childhood B-cell acute lymphoblastic leukemia (B-ALL). Children with newly diagnosed B-cell acute lymphoblastic leukemia (B-ALL) lived significantly longer [...] Read more

Assorted News from the Last Week: Last week the FDA reported that it is investigating reports of blood cancers -- including life-threatening cases of myelodysplastic syndrome and acute myeloid leukemia -- in patients with early, active cerebral adrenoleukodystrophy (CALD) who were treated with elivaldogene autotemcel (eli-cel; Skysona). Cervical cancer deaths have plunged dramatically among women under age 25, and researchers at MUSC Hollings Cancer Center believe this is likely due to HPV vaccination. An international research team including researchers from Augsburg University Medicine has made significant progress in the risk assessment and treatment of extracranial malign rhabdoid tumors. Giselle Saulnier [...] Read more

Assorted News from the Last Week: In conjunction with the European Medicines Agency (EMA), the FDA Oncology Center of Excellence (OCE) held a one-hour Conversations on Cancer public panel discussion to examine an array of challenging decisions faced by members of the pediatric oncology community. The panel discussion featured current CAC2 Board Member Nicole Scobie (Zoe4Life) and past CAC2 Board Member Donna Ludwinski (Solving Kids’ Cancer). Regular symptom screening in patients with pediatric cancer can lead to a reduced symptom burden, according to a pair of studies published in JAMA Pediatrics and JAMA Suicide rates for young male cancer survivors [...] Read more

Assorted News from the Last Week: Electronic symptom screening in kids with cancer boosts quality of care. DIPG Research in Nature: brain tumor yields, in some young people, to CAR-T therapy. Plus a profile on Dr. Michelle Manje's approach to DIPG research (gated) Member blog post by Student Member Joshua Omale: "Reflections from International Cancer Week 2024" The NCCR Data Platform is the nation’s first data-sharing resource to link childhood, adolescent, and young adult records across population-based cancer registries and real-world data partners. The Data Platform links social drivers of health, pharmacy claims, and medical claims data, as well as [...] Read more

Assorted News from the Last Week: Navigating gatekeeping challenges in pediatric and young adult palliative oncology and end-of-life research. Active participation by patients and families in studies helps identify more effective therapies that improve quality of life and survival. My daughter's diagnosis of a rare and aggressive brain tumor at 3 years old made me strongly consider whether or not to have her treated with radiation. Genetic and epigenetic bases of long-term adverse effects of childhood cancer therapy. Korean patients still waiting for access and insurance approval for Qarziba (dinutuximab beta), which was approved as therapy for treating high-risk and [...] Read more

Assorted News from the Last Week: The NCI Fiscal Year 2026 Professional Judgment Budget Proposal highlights promising research areas and the optimal funding needed to achieve the most rapid progress against cancer, based on the professional judgment of NCI’s leadership. NCI’s Division of Cancer Epidemiology and Genetics has created inventories of the research conducted by its investigators on pediatric malignancies and its childhood leukemia research studies. FDA expands approval of methotrexate to include pediatric acute lymphoblastic leukemia. Rapamycin and dasatinib combined with IT (RIST) is a novel metronomic combination that can be administered in the outpatient setting and shows promise for [...] Read more

Assorted News from the Last Week: Today, the Biden Cancer Moonshot announced a new private sector pilot program to maintain uninterrupted access to seven pre-selected critical pediatric cancer medications. Year-end Advocacy readout: Three legislative initiatives for kids with cancer that have a strong possibility for passage in this Congress. Alarmingly few childhood cancer survivors undergo recommended surveillance for late effects. A Chineses randomized trial showed the a shorter regimen of three cycles of adjuvant chemotherapy was noninferior to a standard six-cycle regimen among patients with pathologically high-risk retinoblastoma. The needs of families of pediatric cancer survivors: challenges and developments in [...] Read more

Assorted News from the Last Week: Pediatric cancer medications are 90 percent more likely to go into shortage than other medications, reports say, and stay in shortage 30 percent longer on average. The FDA has granted rare pediatric disease designation to galinpepimut-S (GPS) for the treatment of pediatric patients with acute myeloid leukemia (AML). Survivors of childhood brain cancer are more likely to be held back in school. A new cell therapy, targeting CD7 on leukaemia cells, gives a potentially effective treatment for patients with T-cell acute lymphoblastic leukaemia (T-ALL) who have exhausted all standard treatment options. Published in the [...] Read more

Assorted News from the Last Week: In a clinical practice guideline issued by the American Academy of Pediatrics and published online Sept. 30 in Pediatrics, recommendations are presented for opioid prescribing for acute pain management in children and adolescents in outpatient settings. Fear of cancer recurrence common decades after surviving childhood cancer. Bereaved parents’ perceptions of their cancer-ill child's last month with or without palliative care. Children with acute lymphoblastic leukemia (ALL) who are measurable residual disease (MRD) negative at the end of first consolidation do not require further MRD assessments. Curbing blood cancers by teaching immune cells to kill [...] Read more

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