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Guest Blog–Conversations on Cancer – Pediatric Cancers: Navigating the Challenges Together

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 on November 19, 2024, 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). Because of the rarity of cancers arising in childhood and adolescence and high unmet medical need, pediatric oncology drug development is inherently complex. As a result, pediatric oncology clinician investigators are faced with challenges related to [...] Read more

CAC2 Childhood Cancer Community News Digest (November 11-17)

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

CAC2 Childhood Cancer Community News Digest (November 4-10)

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

CAC2 Childhood Cancer Community News Digest (October 28-November 3)

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

CAC2 Childhood Cancer Community News Digest (October 21-27)

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

Readout from October 6 Federal Advocacy Drop-In Session

The October 10 Federal Advocacy drop-in session covered three pieces of authorizing legislation and provided an update on possible appropriations for FY2025.  Each of the legislative initiatives have a strong possibility for passage in this Congress, and advocates may be called on to make final pushes between now and the end of the year. Legislation: Give Kids a Chance Act has passed the US House and is awaiting approval in the Senate. This bill would help ensure kids with cancer have access to the most modern clinical trials by authorizing the FDA to direct companies to perform a pediatric cancer study for […]

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CAC2 Childhood Cancer Community News Digest (October 7-20)

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

CAC2 Childhood Cancer Community News Digest (September 30-October 6)

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

CAC2 Childhood Cancer Community News Digest (September 23-29)

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

CAC2 Childhood Cancer Community News Digest (September 16-22)

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

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