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Assorted News from the Last Week: The US National Childhood Cancer Registry (NCCR) is combining data from myriad sources to hasten and improve research into pediatric and young adult cancers. The registry will also draw information from birth defects registries and state departments of health.   CAC2 Supporting Member Day One Biopharmaceuticals today announced that the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) for tovorafenib as a monotherapy in relapsed or progressive pediatric low-grade glioma (pLGG). The frequency of cellular therapy trial activations enrolling child/AYA patients with cancer in the U.S. has increased over time. [...] Read more

Assorted News from the Last Week: Trial results confirm effectiveness of Atezolizumab against a rare sarcoma. By a 14-6 vote, the Oncologic Drugs Advisory Committee (ODAC) decided that the totality of evidence presented was sufficient to show that DFMO maintenance improved 2-year event-free survival (EFS). Charles River Laboratories International, Inc. reported that it has the ability to conduct preclinical cancer research using ITCC-P4’s well-characterized collection of 400 annotated pediatric cancer models, and is offering clients access to this collection. The combination of apatinib, irinotecan, and temozolomide has shown activity in a phase 2 trial of pediatric and adult patients with [...] Read more

Assorted News from the Last Week: Video distraction minimizes anesthesia for children undergoing radiotherapy. Supporting the family affected by a pediatric cancer diagnosis: siblings matter, too. From the Journal of Clinical Oncology: Chromosomal gains as a favorable prognostic factor in pediatric ALL. A teen's guide to everything cancer. Cancer in kids is different from cancer in grown-ups – figuring out how could lead to better pediatric treatment. Perception of infertility risk mostly inaccurate among young female cancer survivors. Increased surgical duration was found to be an independent risk factor for surgical site infection in patients with lower extremity bone tumors [...] Read more

Assorted News from the Last Week: Opinion piece from the New York Times:  It Takes a Lifetime to Survive Childhood Cancer. (with a bonus that it references a paper from Pediatric Blood Cancer co-authored by several CAC2 Member patient advocates).   Precedent Setting: In a 14 to 6 vote, the FDA’s Oncologic Drug Advisory Committee (ODAC) agrees that eflornithine (DFMO) shows sufficient evidence to reduce the risk of relapse in pediatric patients with high-risk neuroblastoma (HRBN) who are in remission and have completed multi-agent, multi-modality therapy. The Food and Drug Administration (FDA) has approved Bosulif (bosutinib) for the treatment of [...] Read more

Assorted News from the Last Week: On September 26, 2023, the FDA approved bosutinib (Bosulif, Pfizer) for pediatric patients 1 year of age and older with chronic phase (CP) Ph+ chronic myelogenous leukemia (CML) that is newly diagnosed (ND) or resistant or intolerant (R/I) to prior therapy. Comedian Rob Delaney found out his youngest son Henry had brain cancer. This is a story about the saddest of places life can take you, but it's also about the biggest of loves and how to scrape up bits of joy. "How caring for my child with cancer changed my approach to clinical [...] Read more

Assorted News from the Last Week: U.S. Congressman Michael McCaul (R-Texas) and Childhood Cancer Caucus Co-Chairs Ami Bera (D-Calif.) and Mike Kelly (R-Pa.) introduced the Childhood Cancer Clinical Trials Act, legislation to ensure pediatric cancer patients can access and afford clinical trials offering cutting-edge treatment. Among pediatric patients with low-grade glioma with BRAF V600 mutations, dabrafenib plus trametinib resulted in significantly more responses, longer progression-free survival, and a better safety profile than standard chemotherapy as first-line therapy. Acute and long-term psychosocial consequences in grandparents when a grandchild is diagnosed with cancer – the GROKids Project. NCI released its Fiscal Year [...] Read more

Assorted News from the Last Week: White House announces new Cancer Moonshot Initiatives, including several initiatives involving CAC2 Members: xCures, a health-data technology platform, is launching a study to enhance the collection and organization of comprehensive data for Diffuse Midline Glioma (DMG) and Diffuse Intrinsic Pontine Glioma (DIPG). The study will streamline and standardize data gathering and identify patterns that can predict disease progression and treatment response, leading to more personalized treatment plans and targeted therapies. xCures pledges to maintain the data infrastructure for this study, and to make the data freely available to academic and government researchers. This will [...] Read more

Assorted News from the Last Week: A retrospective study shows that more intense ALL therapy improves outcomes in adolescents and young adults.  This treatment option was developed by "pediatric-inspired protocols" and showed improved event-free, relapse-free survival for Philadelphia chromosome-negative acute lymphocytic leukemia. Autolus Therapeutics announced the publication of data from the AUTO1/22 Phase 1 study (CARPALL) in Pediatric B-cell Acute Lymphoblastic Leukemia where 83% of patients achieved MRD negative, indicating a significant reduction in disease burden. The Seattle-based drugmaker Seagen will team up with CAC2 Supporting Organization Nurix to develop degrader-antibody conjugates (DAC), in a bid to combine their expertise [...] Read more

Assorted News from the Last Week: Major League Soccer, MLS WORKS – the League’s social responsibility platform – and Continental Tire are teaming up during Childhood Cancer Awareness Month with the 10th annual “Kick Childhood Cancer” (KCC) campaign to raise awareness and funds for pediatric cancer research and treatment. Are cannabis products safe and effective for reducing symptoms in children with cancer? Analysis finds insufficient data from published studies. From Cold Spring Harbor Laboratory: Scientists have successfully transformed rhabdomyosarcoma cells into healthy functioning muscle cells. Could this lab breakthrough help bring differentiation therapy to the clinic? In relapsed/refractory BRAF V600–mutant pHGG, [...] Read more

Assorted News from the Last Week: Despite major advances in cancer research, we collectively fall short in bringing innovative medicines to children with cancer.  A new proposal defines a business model to incentivize investors, private companies and public organizations to invest in drug development for paediatric cancers.   Use of homoharringtonine (HHT) may result in improved responses among patients with pediatric acute myeloid leukemia (AML) compared with etoposide-based induction therapy, according to findings from a study published in the Journal of Clinical Oncology. Researchers at Texas A&M University announced a project to explore the use of 3D printing technology to [...] Read more