Pediatric high-grade gliomas (pHGG), including H3K27M-DMGs, remain a major cause of cancer mortality in children. ~15% harbor PDGFRA alterations - making this receptor an attractive therapeutic target 🧵 1/n 👇👇 www.cell.com/cancer-cell/...

Key findings from UKALL2011: 1. Shortening dexamethasone from 28 to 14 days during induction didn’t reduce toxicity or osteonecrosis in children and young adults with ALL👇👇👇 ascopubs.org/doi/full/10....

Adding ifosfamide to MAP chemotherapy didn’t improve outcomes for osteosarcoma patients with poor response to initial treatment, and caused more side effects, says JCOG0905 trial. ascopubs.org/doi/full/10....

Survivors of childhood cancer face aging-related diseases nearly 18 years earlier than the general population, with a 2.7-fold higher risk by age 65. Early cancer and heart disease prevention should start much sooner in this group 👇👇👇 jamanetwork.com/journals/jam...

Point-of-care CAR T-cell therapy using automated production showed strong results in 10 patients, with lower costs, good safety, and high remission rates—promising for wider access, also in low-resource settings 👇👇 www.cell.com/molecular-th...

Targeted therapies like ALK inhibitors and brentuximab vedotin show strong responses in young patients with relapsed ALK+ ALCL, but questions remain about treatment duration and the need for stem cell transplant. ashpublications.org/bloodadvance...

Check out this new study led by Profs Duque-Afonso and Heinaniemi utilizing a mouse model of TCF3-PBX1+ leukemia that mimics human relapse after chemotherapy. About 40% of treated mice survived, with CNS infiltration, similar to clinical setting 👇👇 🧵 1/n onlinelibrary.wiley.com/doi/10.1002/...

Multiomics profiling of resistant Ph like ALL cells revealed MYC dependency and a dormant leukemia subpopulation. Dual inhibition of BCL-2 and JAK/STAT or SRC/ABL pathways effectively eradicated these cells, offering a potential therapeutic strategy 👇 ashpublications.org/blood/articl...

Interesting study using 27 genetically engineered mouse models that closely mimic human hepatocellular carcinoma. 4 subtypes were identified and cladribine, combined with standard therapy, showed promising efficacy for a specific subtype 👇👇 www.nature.com/articles/s41...

By age 60, about 60% of the normal oesophageal epithelium is occupied by cell clones with driver mutations. In contrast, this proportion is much lower in the intestines (~1%) and *stomach* (~5%), see new study 👇👇👇 www.nature.com/articles/s41...

This article challenges the traditional view that cancer is primarily a "genetic disease", proposing instead that tumorigenesis arises from disruptions in cellular and tissue organization 👇👇👇 journals.plos.org/plosbiology/...

Glioblastoma has a dismal prognosis and resists also immunotherapy. Here, a case report with a neoadjuvant dose of triple checkpoint inhibitors before surgery led to strong immune activation and no recurrence after 17 months 👇👇👇 www.nature.com/articles/s41...

This study reveals a surprising and tumor-specific mechanism by which GABAergic neurons promote the growth of diffuse midline gliomas (DMGs), particularly those with H3K27M mutations 👇👇 🧵🧵 1/n www.nature.com/articles/s41...

In high-risk neuroblastoma, catecholamine positivity at complete remission (CR) predicted worse event-free (38% vs. 80%) and overall survival (52% vs. 86%). 3-methoxytyramine at CR was a strong prognostic marker for poor OS (HR 7.5, 95% CI 2.0–28.6) 👇 ascopubs.org/doi/10.1200/...

Thirty percent of children with Wilms cancer have a predisposition gene mutation. In a cohort of 137 cases, Wilms tumour development was observed to vary by gene and timing of mutation, influencing driver events and clonal architecture 👇👇👇 pmc.ncbi.nlm.nih.gov/articles/PMC...

Osteosarcoma's evolution driven by chromothripsis, found in 74% of cases, causing genomic instability and tumor heterogeneity. Novel mechanism, LTA chromothripsis, triggers TP53 inactivation and oncogene amplification. Genome-wide LOH predicts prognosis 👇👇👇 www.cell.com/cell/fulltex...

VenEx trial showed that ex vivo drug sensitivity is beneficial for selecting patients with R/R or sAML for venetoclax-azacitidine therapy 👇👇👇 ashpublications.org/blood/articl...

ALLO_GD2-CART01, an allogeneic CAR T-cell therapy targeting GD2, showed promise in 5 children with relapsed/refractory high-risk neuroblastoma. Despite cytokine release syndrome and GVHD, 2 complete and 1 partial response were achieved 👇👇👇 www.nature.com/articles/s41...

CD5-targeting CAR-T therapy achieved 100% remission by day 30 in a study of 19 r/r T-ALL patients, including those who failed CD7 CAR-T. Late infections and relapse remain challenges. Transplantation may improve long-term outcomes 👇👇👇 www.nature.com/articles/s41...

I'm delighted to announce that the Pharma Proteomics Project will commence full-scale proteomic profiling of the UK Biobank in 2025. We have selected the Olink Proteomics Explore HT platform and Ultima Genomics UG 100 sequencers for this unprecedented study. www.ukbiobank.ac.uk/learn-more-a...

Children with ETV6::RUNX1 or high-hyperdiploid B-ALL have excellent 5-year EFS (97.7% and 94.7%) with low-intensity therapy. However, high-hyperdiploid B-ALL with slow MRD response needs improved treatments 👇👇👇 ashpublications.org/blood/articl...

I was walking along a trail yesterday and noticed this Madagascar periwinkle (Catharanthus roseus) - the plant that “vinca alkaloid” anti-cancer drugs vincristine and vinblastine were isolated from back in 1961. They’re still used today, especially vincristine for acute lymphoid leukemia and NHL.

A phase 1 trial of B7-H3 CAR T cells for #DIPG showed tolerability with repetitive intracerebroventricular dosing and a median survival of 10.7 months post-infusion. Three patients survived over 3 years, supporting further phase 2 investigation 👇👇👇 www.nature.com/articles/s41...

Blood review: "How I treat infant acute lymphoblastic leukemia"👇👇👇 ow.ly/suhm50UyVKJ

Novel GET model accurately predicts gene #expression across 213 cell types using chromatin accessibility data as input, uncovering regulatory networks and interactions. It identifies novel regulatory regions and disease-relevant mutations 👇👇👇 www.nature.com/articles/s41...

A vision- language foundation model, MUSK, integrates pathology images and text for clinical decision-making. Pretrained on large datasets, it excels in tasks like cancer prognosis, biomarker prediction, and therapy response 👇👇 www.nature.com/articles/s41...

From #ASH24 to inpatient leukemia service! Instead of posting articles I will be posting an abstract a day Day 1 - our IIT looking at InO plus TKI in newly diagnosed Ph+ ALL ashpublications.org/blood/articl...

Day 2 - TP53 mutations drive cd19- relapse in blina-treated ALL ashpublications.org/blood/articl...

Another great day ahead! Thesis defence by MSc Mari Lahnalampi @UniEastFinland, with Prof Hanna Mikkola serving as opponent #leukemia

Gene therapy for hemophilia A with the use of lentiviral vector–transduced autologous HSCs resulted in stable factor VIII expression, with factor VIII activity correlating to vector copy number in the peripheral blood 👇👇👇 www.nejm.org/doi/full/10....

Fascinating findings from the @cmullighan.bsky.social team: ctDNA was identified in all cases of hematological malignancies, in 19 out of 38 patients with solid tumors, and in just 1 out of 18 brain tumor patients 👇👇👇 www.nature.com/articles/s41...

#ASH24 #leusm Baruchel: Intro to Plen1. In spite of great strides in mgmt of peds B-ALL 15% still relapse and toxicity remains high. There is still room for improvement. Blin in 1L has been explored in smaller studies in peds, and now Ph3 RCT study AALL1731 adding Blin to 1L therapy being presented.

Symposia: Late-Breaking Abstracts Program: Marquee Sessions 7:30 am - 9 am PST Meeting Hall B #ASH24 #BloodJournals https://buff.ly/3BeZb26

#ASH2024 Reducing daunorubicin during induction therapy for standard-risk acute lymphoblastic #leukemia is safe and results in a 4-fold decrease in invasive fungal infections 👇👇👇 ashpublications.org/blood/articl...

#ASH2024 This is a huge leap forward! 3-y disease-free survival (DFS) with blinatumomab was 97.5% vs. 90.2% without blina in SR-Average patients, and 94.1% vs. 84.8%, respectively, in SR-High patients. Obs! 10/19 relapses in the blina arm involved CNS 👇👇 ashpublications.org/blood/articl...

#ASH2024 In PIVOT trial, hydroxyurea treatment at 20 mg/kg/day was well-tolerated in both children and adults with HbSC, and was associated with significantly fewer painful vaso-occlusive events and fewer hospitalizations 👇👇👇 ashpublications.org/blood/articl...

Presented at #ASH24: The addition of blinatumomab after induction chemotherapy in children with standard-risk disease of average or high risk of relapse significantly improved disease-free survival. Full AALL1731 trial results: nej.md/49CVkc1 #medsky

Leukaemia cell intrinsic and extrinsic factors cooperate to facilitate the survival and proliferation of KMT2A-rearranged B-ALL in the CNS niche. https://www.biorxiv.org/content/10.1101/2024.12.03.626602v1