AdaptiveFlow, an AI-informed platform, can virtually screen billions of drug-like molecules with a 1,000-fold reduction in computational costs. The platform identified potent inhibitors for existing and emerging cancer targets, showcasing its potential as a powerful tool for modern drug discovery.
Researchers at St. Jude Children's Research Hospital found that a brain mutation causes cells to remain immature, leading to tumor formation in specific brain regions. This discovery may lead to therapies tailored to childhood brain cancers.
Scientists at St. Jude Children's Research Hospital discovered removing Regnase-1 from engineered immune cells enhances CAR T-cell therapy's ability to control osteosarcoma growth and prevent metastasis. The modified cells also alter their tumor microenvironment, activating immune cells and reducing immunosuppressive signals.
Charles W.M. Roberts, a renowned physician-scientist, will lead St. Jude Children's Research Hospital in accelerating progress against childhood cancer and catastrophic diseases. With his expertise in cancer epigenetics and clinical research, Roberts aims to transform outcomes for children worldwide.
Scientists created a single-cell atlas of spinal neurons to understand motor coordination and recovery from injury. The study identified a small subgroup of interneurons associated with speed, clarifying their role in controlling movement.
Researchers at St. Jude Children's Research Hospital have found a way to reactivate the frataxin gene in Friedreich's ataxia, a neurodegenerative disease, by using a specially designed chemical adaptor. The study challenges a longstanding assumption about histone modifications and offers new insights into gene regulation.
Researchers discovered a lipid regulator, arachidonic acid, boosts Smoothened activity through a newly found binding site, enhancing SHH signaling in tissues like the heart and lungs. Disrupting this interaction impaired cardiopulmonary development, providing insight into how the SHH pathway may be modulated in diseases.
Researchers at St. Jude Children's Research Hospital discovered an RNA-based survival strategy that enables bacteria to tolerate antibiotics. The findings provide new insights into how persistent infections develop and could inform strategies to improve antibiotic effectiveness. Antibiotic tolerance allows bacteria to survive treatment...
Researchers identified RBM5 as a potential vulnerability in childhood leukemias, particularly in cancer stem cells. Removing RBM5 from these cells doubled mouse model survival rates and showed promise for developing more precise therapies. Targeting RBM5 may disrupt the pro-growth feedback loop of MYC, a key cancer-driving protein.
Researchers found that blood stem cells from young patients with sickle cell disease have features of aging, which can increase the risk for other complications. Using senolytics improved disease symptoms in model systems, highlighting a needed improvement to gene therapies.
Researchers at St. Jude Children's Research Hospital discovered that smaller sub-compartments within the nucleolus form to finish the final steps of ribosome assembly. These sub-compartments lock major ribosome building blocks together until they are assembled, preventing a key protein from associating with them prematurely.
Researchers at St. Jude Children's Research Hospital have discovered the 'double-donut' structure of SPOP, a protein critical to regulating gene expression in cells. The study reveals how certain cancer mutations disrupt this balance, leading to disease progression.
Researchers at St. Jude Children's Research Hospital discovered that C1 neurons in mice modulate fear and anxiety, with prolonged activation leading to heightened anxiety. Inhibition of these neurons reduced anxiety-like behaviors, suggesting they may be a valuable therapeutic target for anxiety disorders.
Heather Currier Hunt will lead the evolution of St. Jude's Leadership Academy to develop leaders for its growing workforce and global impact. The program has already trained over 1,600 staff members and focuses on creating leaders who are curious, collaborative, and driven.
St. Jude Children's Research Hospital has been recognized by the World Health Organization (WHO) as a Collaborating Centre for Childhood Cancer. The redesignation aims to improve global pediatric cancer outcomes and expand access to quality care for children worldwide.
Researchers at St. Jude Children's Research Hospital discovered that cells use two pathways to silence invading transposons: RNA interference and heterochromatin. These pathways are triggered by abnormal RNA patterns produced by the transposon, allowing cells to detect and defend against invasive DNA sequences.
Scientists at St. Jude Children's Research Hospital have identified a small RNA embedded within the V. cholerae gene as the key factor controlling its ability to infect humans. Variations in this RNA controlled about 85% of genes related to human infection, enabling the bacteria to evade immune barriers and colonize the gut.
Hongbo Chi's election recognizes his pioneering work in immunometabolism and its impact on next-generation cancer immunotherapies. He continues to contribute to St. Jude's mission-driven institution, advancing fundamental science and meaningful impact.
Researchers at St. Jude Children's Research Hospital discovered that blood stem cells with aplastic anemia escape the immune system by acquiring different gene mutations independently. These mutations, such as HLA loss and CHIP gene changes, allow cells to hide from the immune system and provide long-term remission.
A new study published in Science Advances identifies IRS4 as a promising drug target for multiple solid tumors, offering hope for safer cancer treatments. By using AI and natural mutations, researchers prioritized targets with high therapeutic indexes to minimize toxicity.
Scientists at St. Jude Children's Research Hospital identified a key regulatory enhancer, ecMYC E1, that drives MYC expression in pediatric medulloblastoma, a type of brain cancer with poor outcomes. Silencing this enhancer reduces MYC transcription and can be targeted to treat the disease.
Researchers found that an ASO strategy boosts expression of the closely related HNRNPH1 protein, reducing symptoms of HNRNPH2-related neurodevelopmental disorder. The study provides vital mechanistic data to support advancement of this promising therapy.
Researchers at St. Jude Children's Research Hospital identified therapy-specific mutations that could guide safer treatments among childhood cancer survivors. The study analyzed genetic mutations in subsequent neoplasms of 160 survivors who developed breast, thyroid malignancies and meningiomas after treatment.
Researchers at St. Jude Children's Research Hospital developed patient-derived tumor organoids that accurately reflect the biologic underpinnings of embryonal brain tumors. The models enable faster functional assays and preclinical drug testing without relying on newly obtained tumor samples, advancing the study of pediatric brain tumors.
Scientists mapped the spread of a novel flu strain, D1.1 H5N1, through wild bird populations across North America, revealing its emergence and geographic penetration. The study shows that current human vaccine stockpiles will likely work well against both avian and human infections.
Researchers from St. Jude Children's Research Hospital discovered the gene-regulatory protein PHIP as a critical vulnerability in cancers driven by broad SWI/SNF inactivation. PHIP's loss disrupts cancer growth and cooperates with SWI/SNF to activate transcription, providing evidence for therapeutic development of PHIP-inhibiting drugs.
The ASH HematOmics Program platform combines genomics, gene expression, and clinical information from nearly 6,000 patients with blood cancers. Researchers can explore these datasets together in a single open resource, accelerating discovery in hematological malignancies.
According to a new study published in The Lancet, childhood cancer is the eighth-leading cause of death among children worldwide. Researchers found that over 377,000 new cases and 144,000 deaths occurred in 2023, with most cases in low- and middle-income countries.
Scientists discovered how tumors disable dendritic cell function by decreasing their mitochondrial fitness, leading to weakened immune defenses against cancer. Restoring mitochondrial function in dendritic cells improves antitumor immune responses and enhances immunotherapeutic efficacy.
Researchers developed AI models to analyze conversations between children with cancer and their caregivers, detecting severe symptoms that require extra support. More complex prompting strategies outperformed simpler ones in accurately identifying symptom severity and its impact.
Robert Webster, Ph.D., a leading expert in influenza research, has been elected as an AAAS Fellow for his contributions to understanding the reservoir and spread of avian influenza. His work has had a direct impact in protecting immunocompromised children at St. Jude and safeguarding children worldwide from pandemic threats.
The study reveals that ZFTA-RELA exploits open regions of the genome to trap immature brain cells in developmental limbo. This finding may lead to novel therapeutic strategies to test, such as forcing cells past immature developmental states.
A comprehensive protein atlas has been developed to understand the molecular underpinnings of neurodegenerative diseases. By analyzing protein levels, modifications, and interactions across six major diseases, researchers identified alterations unique to and shared between diseases, as well as distinct subtypes within individual diseases.
Researchers identified structural variants as a key driver of childhood cancer, with RAG-mediated recombination found in nearly all subtypes of acute lymphoblastic leukemias. The analysis highlights the importance of structural variants in pediatric blood cancers and provides new insights into the mechanisms driving these diseases.
St. Jude Children's Research Hospital appoints Carsten Bönnemann, MD, to lead the newly created Department of Genomic and Translational Neuroscience, focusing on catastrophic neurological disorders. The department aims to develop genetic precision therapies and advance research into complex pediatric neurological conditions.
Scientists at St. Jude Children's Research Hospital developed BOUQUET to analyze 3D-enhancer architecture in machine learning-based graph theory framework, identifying protein condensates and predicting gene expression. The findings provide new insight into how cells regulate genes controlling specialized identities.
Researchers found a set of light-sensing genes essential to pineoblastoma formation in the developing pineal gland. They extended the finding to medulloblastoma and retinoblastoma, indicating a common developmental state and potential shared therapeutic dependency.
A new test detects infection-causing pathogens days before standard blood cultures, offering a potential approach to protecting vulnerable patients. The test, named plasma microbial cell-free DNA sequencing (mcfDNA-Seq), reliably identifies the most common bacteria and fungi that cause bloodstream infections in children with cancer.
A recent study published in Science Translational Medicine found that infant immune systems respond very differently to RSV and SARS-CoV-2, with important treatment implications. Infants infected with RSV often develop severe symptoms due to low levels of systemic inflammation and a poorly coordinated early immune response.
A study by St. Jude Children's Research Hospital found that radiation therapy contributes to hearing loss in children treated for brain tumors, leading to greater cognitive decline. Children with severe hearing loss experienced a more significant decline in cognitive measures compared to those without.
Dr. James R. Downing will transition to a faculty role at St. Jude in 2026, leading the institution's global mission after an unprecedented growth period. He played a pivotal role in advancing childhood cancer research and treatment worldwide.
A new AI-powered algorithm, M-PACT, uses liquid biopsy to classify pediatric brain tumors based on DNA methylation patterns. The technology has been shown to achieve a high sensitivity rate of 92% in identifying brain tumors.
A multinational clinical trial found that the long-term HIV treatment regimen is safe and favored by adolescents who received injectable intramuscular antiviral medications cabotegravir and rilpivirine. The study showed continued viral suppression after 48 weeks with no major safety concerns.
Researchers discovered a new class of highly interactive LINE-1 loci that modulate cancer gene expression by altering three-dimensional genome architecture. These structural changes allow for high-level expression of genes driving cancer cell proliferation.
Researchers at St. Jude Children's Research Hospital created CHANGE-seq-BE to evaluate the activity and specificity of base editors, a genome editing technology, ensuring safety and accuracy. The technique outperformed conventional approaches and has already been used in clinical work.
Researchers at St. Jude Children's Research Hospital found that distinct GPCR ligands create different levels of activation by pushing the receptor through its activation steps at varying speeds. This affects the efficacy of agonists, with partial agonists getting stuck in kinetic traps and releasing G-proteins more slowly.
Researchers characterized a hidden intermediate state in Src kinase function, which enables rapid phosphorylation and is essential for T-cell activation and cell migration. This study expands our understanding of kinase function and sets the stage for new therapeutic approaches that selectively target these conformational states.
Researchers found that reactivating and maintaining p53 using idasantulin and selinexor significantly extended survival in ATRT and MRT mouse models, reducing tumor burden and increasing survival rates. The study's findings provide a strong rationale for investigating this treatment approach for rhabdoid tumors.
Combocat integrates miniaturized drug dispensing with machine learning to efficiently uncover promising drug combinations. The platform's capabilities were published in Nature Communications, demonstrating its potential for scaling up drug combination screening.
Researchers at St. Jude Children's Research Hospital identified a protein network that stabilizes ABCC4 transporters in place to maintain cyclic AMP signaling levels. The discovery reveals a previously unappreciated protein neighborhood and opens new therapeutic opportunities for regulating ABC transporters.