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

Assorted News from the Last Two Weeks: Latino children are at greater risk of developing certain cancers, and their short and long-term outcomes are often worse than other children with cancer. Approximately 1 in 15 children receiving cancer treatment in low- and middle-income countries die from treatment-related complications. Although treatment-related mortality has decreased in upper–middle-income countries over time, it remains unchanged in low- and middle-income countries. There is an urgent need for targeted supportive care interventions to reduce global disparities in childhood cancer survival. The FDA has issued a complete response letter to a resubmitted biologics license application (BLA) for remestemcel-L [...] Read more

Assorted News from the Last Week: High-Throughput drug screening of primary tumor cells identifies therapeutic strategies for treating children with high-risk cancer. Tribute to CAC2 Member Donna “Nana” Marie Carmical’s (Journey4ACure) life was a testament to the power of love, determination, and selflessness. Her journey from a grieving grandmother to a fierce advocate for childhood cancer showcased her unyielding commitment to making the world a better place for young patients. Epigenetically guided cancer therapy: Targeting H3K27me3 loss in pediatric brain tumors The addition of blinatumomab (Blincyto) to chemotherapy did not improve overall survival (OS) and disease-free survival (DFS) over chemotherapy [...] Read more