Scientists at St. Jude Children's Research Hospital have identified 156 potential targets for CAR T-cell immunotherapy, which could lead to curative approaches for various cancers. The study validated at least one target, COL11A1, and built a data resource for the community.
Researchers identified over 500 functional non-coding DNA variants associated with chemotherapy resistance in acute lymphoblastic leukemia, a common childhood cancer. These variants were linked to a specific gene and mechanism of resistance, offering new insights into the underlying causes of treatment failure.
Scientists at St. Jude Children's Research Hospital discovered a new compound that selectively targets parts of the cancer-related protein EP300/CBP in Group 3 medulloblastoma, significantly reducing cancer cell growth. The targeted approach using region-specific inhibitors resulted in profound effects on Group 3 medulloblastoma cells.
Researchers identified four genetic variants associated with increased type 2 diabetes risk in childhood cancer survivors. The findings also showed that polygenic risk scores derived from diverse ancestry datasets were more informative in assessing diabetes risk in survivors of both European and African ancestries, highlighting the imp...
Scientists at St. Jude Children's Research Hospital found that improving metabolic health in obese mice before flu vaccination but not after, protects against influenza virus. A healthy diet improved T-cell function and a robust anti-flu response during later exposure.
Scientists have developed a new preventative treatment that can prevent lethal lung injury from the flu by targeting inflammation protein. The drug UH15-38 achieves a balance between shutting down runaway inflammation and allowing the immune system to stop the virus, resulting in increased survival rates.
The St. Jude survivorship portal provides a rich dataset for researchers to study childhood cancer survivors, including genomic and clinical information. The portal facilitates breakthroughs in pediatric cancer survivorship research through dynamic visualization and customizable charts.
Researchers at St. Jude Children's Research Hospital designed a combination therapy to effectively treat relapsed or resistant B-cell leukemia by targeting the developmental stages of cancer cells. The findings revealed that a protein called BCL-2 is a hidden vulnerability in asparaginase-resistant tumor cells.
Dr. Ching-Hon Pui will be honored for his contributions to pediatric cancer research, revolutionizing the treatment of childhood acute lymphoblastic leukemia and improving survival rates above 90%. St. Jude Children's Research Hospital is recognized as a leading center in understanding, treating, and curing childhood cancer.
Researchers at St. Jude Children's Research Hospital discovered that removing the quality control protein DCAF5 can reverse cancer cell identity in rhabdoid tumors. The study suggests a new treatment paradigm where cancer cells are returned to an earlier, more normal state rather than killed with toxic therapies.
Researchers identified a small set of proteins governing anaplastic large cell lymphoma (ALCL) identity and found that drugs targeting the STAT family of proteins already exist for treatment. The study provides insight into potential vulnerabilities in ALCL cells.
Researchers at St. Jude Children's Research Hospital discovered a potential therapeutic target for high-risk neuroblastoma by studying the cellular personality switch driven by the MYCN protein. Inhibiting the protein KDM4 showed promising anticancer activity, blocking MYCN expression and repressing key oncogenes in disease models.
Scientists discovered an immune cell protein that can target and destroy fibrolamellar carcinoma, a rare liver cancer with a poor prognosis. T-cell therapy may effectively treat the disease by recognizing the fusion protein, providing a novel approach for FLC treatment.
Researchers at St. Jude Children's Research Hospital have discovered that myeloid cell leukemia-1 (MCL-1) plays a critical role in regulating long-chain fatty acid oxidation in mitochondria, revealing new insights into its anti-apoptotic functions and potential therapeutic applications.
Scientists at St. Jude Children's Research Hospital found that ruxolitinib, a drug inhibiting both Janus Kinase 1 (JAK1) and JAK2, is a safe and potentially more effective therapy for Hemophagocytic lymphohistiocytosis (HLH), a rare and aggressive syndrome of hyperactive inflammation. The study showed that ruxolitinib increased surviva...
Researchers created an additional means for therapy to find and eliminate cancer cells using a small peptide, demonstrating better efficacy in lab tests and in vitro experiments. The study used computational analysis and predicted protein models to understand how structure impacts antigen recognition and therapy efficacy.
Researchers at St. Jude Children's Research Hospital have developed a molecular glue that sticks to the cancer-related protein casein kinase 1 alpha (CK1α), leading to its destruction. The compound, SJ3149, displays broad anti-cancer activity and may have clinical utility as an alternative to conventional small molecule inhibitors.
Researchers identified RBM5 as a key regulator of HOXA9 expression in leukemia cells, revealing its dual function in DNA and RNA handling. Removing RBM5 from cells significantly reduced HOXA9 mRNA levels, suggesting its potential as a therapeutic target for acute myeloid leukemia treatment.
Researchers found that two common biomarkers, global longitudinal strain and N-terminal-pro-B-type natriuretic peptide, can identify asymptomatic childhood cancer survivors at elevated risk of declining heart muscle function. These biomarkers may lead to earlier treatment to prevent further heart damage.
The study classifies pediatric acute myeloid leukemia (pAML) into 23 distinct molecular categories, revealing unique biological characteristics and drivers of the disease. This classification provides a path forward for clinicians to identify distinct pAML sub-types and guide treatment decisions.
A new, universal definition of acute pediatric critical illness has been reached by consensus among researchers and clinicians from 40 countries. The definition focuses on the patient's status rather than resource availability, enabling global research and potentially leading to improvements in patient outcomes.
Researchers at St. Jude Children's Research Hospital have designed a new version of the drug spectinomycin that overcomes efflux, a key mechanism driving antibiotic resistance in Mab infections. The modified compound, eAmSPCs, shows enhanced antimicrobial efficacy and works well with various classes of antibiotics.
Researchers at St. Jude Children's Research Hospital have visualized how cytonemes transfer signals across vast distances during neural development, establishing an 'express route' for communication between cells. This discovery provides insights into the complex process of mammalian nervous system development and highlights the crucia...
Scientists at St. Jude Children's Research Hospital have determined the complex structure of Parkinson’s disease-related proteins LRRK2 and Rab29, revealing how they work synergistically to cause the disease. The structures provide an atomic-scale map to trace how different mutations affect function within this complex, with implicatio...
Scientists at St. Jude Children's Research Hospital have discovered that only about 80 amino acids in the β2-adrenergic receptor contribute to its pharmacological properties, with specific residues controlling efficacy and potency. Understanding these molecular origins can help design more potent and efficacious drugs.
Researchers found that pre-zygotic germline variants and post-zygotic hypermethylation at 11p15.5 predispose patients to bilateral Wilms tumor. This study provides new insights into the genetic and epigenetic mechanisms underlying the disease, with potential implications for treatment decisions and clinical trials.
A study found childhood cancer survivors have twice the risk of developing prediabetes as the general public, increasing their risk of life-threatening diseases. Lifestyle interventions may help reduce this risk and improve their life expectancy.
Scientists at St. Jude Children's Research Hospital created a highly adaptable system to improve the safety and efficacy of immunotherapy for solid tumors. By adding modular chimeric cytokine receptors to CAR T cells, the therapy can target multiple types of cancer without generating significant toxicity.
Scientists at St. Jude Children's Research Hospital identified genes directly regulated by the oncogenic HOXA9 protein in high-risk pediatric leukemias. The study found two major targets, FLT3 and CDK6, which can be therapeutically targeted with drugs, showing promising outcomes in preclinical models. Additionally, researchers discover...
Scientists at St. Jude Children's Research Hospital validated GRP78 as a promising but complex target for CAR T-cell immunotherapy. However, they discovered that some tumors trick the immune cells into expressing GRP78, turning off their own cancer-killing ability.
Researchers created a comprehensive chromatin map to understand gene regulatory networks contributing to differences between pediatric acute lymphoblastic leukemia subtypes. The study identified key transcription factor footprints and chromatin accessibility patterns, which can predict leukemia subtype with 89% accuracy.
Researchers mapped transcription factors in T cells infiltrating tumors, finding ways to increase anticancer efficacy by promoting or blocking T-cell differentiation. The findings have implications for cancer immunotherapy and provide new potential strategies to enhance immunotherapy.
Scientists at St. Jude Children's Research Hospital discovered that a fusion oncoprotein forces cells to become tumor-like, regardless of their original muscle or endothelial cell origin. The study suggests that focusing on the effects of this protein may be a better approach for identifying potential therapeutic interventions.
Researchers at St. Jude Children's Research Hospital have determined the structure of vesicular monoamine transporter 2 (VMAT2), a protein crucial for packaging and releasing neurotransmitters in neurons. The study provides critical information for drug development to treat hyperkinetic disorders like Tourette syndrome.
Researchers identified the integration site pattern of lentiviral gene therapies in patient cells, shedding light on treatment safety and efficacy. The study found that integration near nuclear pores is associated with improved safety and effectiveness, avoiding oncogene regions where earlier technologies failed.
A study published in The Lancet Oncology reveals a genetic explanation for increased second-cancer risk and mortality among childhood cancer survivors. The research identified specific genes associated with this increased risk, enabling personalized cancer screening and prevention strategies.
A new study by St. Jude Children's Research Hospital predicts the formation of condensates in cancer-associated fusion oncoproteins, which promotes cancer development. The researchers used machine learning to analyze biomolecular features and established a framework for understanding the mechanisms underlying oncogenic properties.
Researchers at St. Jude Children's Research Hospital refined the definition of hyperdiploidy in childhood B-cell acute lymphoblastic leukemia (B-ALL), using DNA index to predict patient outcomes. The study found that a simpler system captures a significant proportion of patients with excellent prognoses, and that individual chromosome ...
Researchers at St. Jude Children's Research Hospital discovered a subset of immune cells that slows Alzheimer's disease progression by interacting with microglia. The cells, called CD8+ T cells, use a molecular handshake to signal to the microglia to stop causing uncontrolled inflammation, which in turn slows plaque growth and symptoms.
Researchers at St. Jude uncover how ABCG2 protein removes chemotherapies from cells, highlighting a potential path to combat drug resistance. The study identifies key amino acids responsible for this promiscuity and suggests designing inhibitors targeting these sites.
Researchers found that certain immune cells were associated with increased protection from flu symptoms, while others were linked to susceptibility. The study's findings have implications for developing tailored vaccine platforms and improving public health strategies.
The 'survival of the accessible' model provides an alternative explanation for evolutionary changes in flu viruses, highlighting the importance of mutational bias and variant accessibility. The research reveals how specific mutations can gain or lose function, influencing protein activity and potentially driving pandemics.
Researchers at St. Jude Children's Research Hospital created a more accurate hepatoblastoma model to improve therapies, focusing on DNA damage repair pathways. The model identified potential targets and validated the effectiveness of PRKDC inhibition when combined with doxorubicin, enhancing treatment efficacy.
St. Jude researchers found that supplying glutamine to tumors enhances the immune system's cancer-killing activity, while a molecular pathway identified as a potential drug target could improve anti-cancer therapies. Glutamine helps activate dendritic cells, which then activate T cells that kill cancer cells.
Researchers use base editing technology to restart fetal hemoglobin expression in SCD patient cells, achieving higher and more stable levels than other genome editing technologies. The approach has potential as a 'one-size-fits-all' treatment for all mutations that cause SCD and beta-thalassemia.
Researchers from St. Jude Children's Research Hospital discovered NLRP12 to be the key molecule responsible for inducing inflammatory cell death and pathology in response to heme combined with other cellular damage or infection. This finding provides a new potential drug target to prevent morbidity in certain illnesses.
Scientists at St. Jude Children's Research Hospital discovered how the current H5N1 avian influenza virus gained new genes and greater virulence as it spread across North America. The researchers found that the virus could severely infect the brains of mammalian research models, a notable departure from previous related strains.
Researchers at St. Jude Children's Research Hospital discovered that the epigenetic landscape plays a crucial role in regulating pioneer transcription factor binding. By understanding this process, scientists can develop new therapeutics to combat cancer and other diseases. The study reveals how epigenetic modifications affect transcri...
Scientists at St. Jude Children's Research Hospital discovered that subunits of the SWI/SNF chromatin remodeling complex act as bookmarks to safeguard cell identity during mitosis. This finding provides new insights into how cancers develop and how they can be treated.
Scientists at St Jude Children's Research Hospital have captured six cryo-EM structures of a key transporter involved in cancer and immunity, providing unprecedented insight into its function. The findings have significant implications for developing targeted therapies against cancer and autoimmune diseases.