St. Jude Children's Research Hospital scientists have developed an integrated system to better understand and possibly manipulate gene expression for treatment of disorders like sickle cell disease and beta thalassemia. The new method identified dozens of DNA regulatory elements that orchestrate fetal-to-adult hemoglobin switch, offeri...
A phase 2 clinical trial found that brentuximab vedotin achieved excellent outcomes in high-risk pediatric Hodgkin lymphoma patients, with a three-year survival rate of 99% and reduced radiation exposure. The targeted therapy also minimized side effects, particularly neuropathy.
Researchers studied stress signals in skeletal muscle and found they prevent misfolded protein aggregates in the brain and retina. Tailoring this signaling may help combat neurodegenerative conditions like age-related dementia and Alzheimer's disease.
Researchers at St. Jude Children's Research Hospital discovered three molecular groups of ATRT with varying clinical outcomes, highlighting the importance of age and metastasis in prognosis and treatment planning. The study sheds light on the biology of this disease and provides valuable insights for developing targeted therapies.
The Crazy 8 Initiative aims to tackle major obstacles in pediatric oncology through collaborative research and innovative approaches. Grants will fund studies using cutting-edge technologies, such as targeted degradation and molecular glues, to develop new treatments for aggressive cancers.
A study at St. Jude Children's Research Hospital found that socioeconomic status is significantly linked to IQ, academics, and self-care skills in children with brain tumors treated with radiation. The gap between these groups widens over time.
Immunologists at St. Jude Children's Research Hospital have mapped the biological machinery that generates T cells to kill bacteria, viruses, and tumor cells. The study reveals mechanisms that inhibit development of long-lived memory T cells, which can be blocked with pharmacological or genetic approaches to boost protective immunity.
Researchers at St. Jude Children's Research Hospital have discovered a mechanism that tumors use to switch on protective regulatory T cells, raising the potential for drug treatments that render tumors more vulnerable to cancer immunotherapy. The researchers showed that blocking tumor-associated regulatory T cell activity eliminated tu...
Researchers at St. Jude Children's Research Hospital characterized genomic abnormalities of 84 therapy-related myeloid neoplasms in children, identifying notable mutations and increased expression of a transcription factor called MECOM. Early detection of these mutations could lead to early interventions and potentially benefit patients.
Researchers at St. Jude Children's Research Hospital published a clinical trial report providing molecular profiling insights into pediatric medulloblastoma. The study confirmed the need for integrated molecular assessment of these tumors, highlighting differences in prognosis based on molecular groups.
African American childhood cancer survivors are at a higher risk of developing cardiomyopathy, with genetic variants on chromosomes 15q25.3 and 1p13.2 identified as key factors. The study has implications for long-term follow-up and surveillance of these patients.
Research reveals Aspergillus fumigatus activates inflammasome through galactosaminogalactan, increasing inflammation. The fungal pathogen's GAG is essential for NLRP3 inflammasome activation.
Researchers discovered a potential strategy to prevent life-threatening inflammation, lung damage, and organ failure in patients with COVID-19. They identified specific cytokines that trigger inflammatory cell death pathways, leading to tissue damage and multi-organ failure. Treatment using existing drugs targeting these cytokines show...
Scientists at St. Jude Children's Research Hospital have identified how metabolic signaling pathways influence key immune cells, including eTreg cells. Understanding this regulation may aid in developing more specific drugs to target these pathways and treat diseases such as lupus, rheumatoid arthritis, and cancer.
A study by St. Jude Children's Research Hospital and Washington University found that most adults with moderate-to-severe COVID-19 have a suppressed viral immune response, unlike flu patients. Steroids like dexamethasone are only effective in a small subset of patients with life-threatening hyperinflammation.
A comprehensive genomic analysis of neuroblastoma has revealed possible approaches for developing precision medicines to improve patient outcomes. The study found associations among common mutational traits that could be exploited therapeutically, including an association between a chromosomal mutation and oxidative stress.
Kanneganti's research focuses on the innate immune system, inflammation, and cell death in health and disease, particularly in colorectal cancer. She aims to understand how innate immune pathways contribute to tumor development and improve cancer treatment strategies.
Researchers have identified the structure of double-strand DNA break repair by PARP enzymes, which can bridge broken DNA ends together. The study provides insight into the mechanisms underlying PARP activation and catalytic cycle, potentially aiding in understanding resistance to cancer drugs that inhibit PARP.
A study found that an inherited GATA3 gene variant influences how children respond to leukemia treatment and is linked to relapse. The variant also predicts minimal residual disease levels.
Researchers identified the LANDO pathway as a key factor in neuroinflammation, which is a major hallmark of Alzheimer's disease. By disrupting this pathway, scientists found that they could reduce neuroinflammation and even improve cognition and memory in a mouse model.
Researchers at St. Jude Children's Research Hospital found that severe hearing loss in childhood cancer survivors is associated with neurocognitive deficits independent of therapy type. The study evaluated 1,520 survivors and discovered that more than one-third had severe hearing loss, leading to increased risk of neurocognitive problems.
St. Jude Children's Research Hospital scientists have developed a computational tool called cis-X to identify alterations that drive tumor formation in the human genome. The method analyzes abnormal expression of tumor RNA and identifies novel pathogenic variants and oncogenes activated by such variants in regulatory noncoding DNA.
The Childhood Solid Tumor Network data portal on St. Jude Cloud provides comprehensive data for studying pediatric solid tumors, accelerating discoveries and novel therapies. The portal offers interactive tools for analyzing genomic sequences, epigenetic data, and preclinical pharmacokinetic reports.
Scientists have discovered a common TP53-R337H variant among people of Brazilian descent that increases cancer risk when combined with an inherited XAF1 mutation. The study found individuals with both mutations are at a greater risk of cancer, highlighting the importance of genetic screening and public health approaches.
Scientists at St. Jude Children's Research Hospital have created orthotopic patient-derived xenograft models representing a variety of pediatric brain tumor types. These models are molecularly characterized and available through a cloud-based data portal, enabling researchers worldwide to access them.
Researchers at St. Jude Children's Research Hospital have developed a software system called CICERO that enables better detection of gene fusions in cancers. The system distinguishes fusion events by comparing the cells' RNA sequence with the human genome, identifying potential cancer-causing fusions.
Researchers found that interleukin-2 is critically important to driving resistance to dexamethasone in HLH. Blocking interleukin-2's effect can re-sensitize T cells to dexamethasone, making the combination of dexamethasone and ruxolitinib a promising treatment for HLH.
A study published in Journal of Clinical Oncology finds that combined effects of therapy and inherited mutations in DNA-repair genes increase the risk of developing subsequent cancers. The research uses whole genome sequencing to analyze DNA from blood samples of 4,402 pediatric cancer survivors.
St. Jude awarded a National Science Foundation grant to develop new tools for studying pediatric diseases by integrating biology and engineering at the molecular level. The collaboration will provide St. Jude faculty with a new chemical and engineering toolbox to address gene regulation disorders, including childhood cancer.
Caspase-6 plays a critical role in regulating the ZBP1-NLRP3 inflammasome, facilitating PANoptosis during viral infection. Modulation of caspase-6 could be beneficial for treating viral diseases like influenza and other inflammatory diseases including cancer.
A new anti-malarial compound called SJ733 has shown promising results in its first clinical trial, demonstrating a rapid and effective treatment against malaria parasites. The study involved 38 healthy volunteers who received the drug after being infected with malaria, with no significant side effects identified.
A significant, sustained global investment in treating children with cancer could save 11 million lives. The potential return on investment is a triple gain of almost $2 trillion to the global economy. The Lancet Oncology Commission report analyzed the benefits of investing $594 billion in interventions such as primary care and treatment.
Researchers at St. Jude Children's Research Hospital discovered a novel predisposition gene, ELP1, in the SHH subgroup of pediatric medulloblastoma, affecting 40% of cases. The study found that patients with this mutation tend to do well on therapy and may benefit from tailored treatment in the future.
Scientists at St. Jude Children's Research Hospital have identified the ABCC4 transporter as critical to the SHH signaling pathway in medulloblastoma. Targeting this transporter shows promise for treating this disease, with increased expression correlating to poor overall survival.
Researchers at St. Jude Children's Research Hospital created an 'atlas' of gene-regulatory mechanisms in immune cells that facilitate Type 1 diabetes. The study revealed a dual biological personality of T cells, enabling them to retain the ability to attack insulin-producing cells across successive generations.
Researchers identified a gene, CELSR2, associated with glucocorticoid resistance in leukemia cells. A new drug, venetoclax, may reverse resistance by inhibiting a cell death pathway. The findings suggest combining venetoclax with current therapy could improve ALL chemotherapy effectiveness.
Researchers identified subpopulations of leukemia cells present at diagnosis that have distinct characteristics, leading to relapse in children and adults. These findings provide new avenues for overcoming resistance and improving treatment approaches.
Scientists at St. Jude Children's Research Hospital have identified two critical microRNAs driving ventricular enlargement in models of schizophrenia. Reduced expression of these microRNAs leads to increased motile cilia movement and brain ventricle enlargement, highlighting a potential target for future treatments.
A collaborative clinical trial has led to changes in the treatment of leukemia driven by the Philadelphia chromosome, a high-risk subtype associated with poor outcomes. Dasatinib was found to provide greater benefit than the standard of care, resulting in improved event-free survival rates.
Researchers discovered an alternate mechanism by which the molecule RIPK1 triggers cell death in infected or damaged cells. The finding breaks existing dogma that RIPK1 kinase activity is required for cell death and holds promise for treating inflammatory diseases and cancer.
Researchers discovered that myokine Dpp, produced by skeletal muscle, regulates feeding behavior by modulating dopamine levels in the brain. This finding sheds new light on the mechanisms governing feeding behavior and has implications for understanding metabolic diseases.
Researchers integrated genomic and transcriptomic sequencing to classify AML and MDS into distinct biologic subgroups. This new understanding may lead to personalized treatment approaches, improving patient outcomes.
Clinical fellow Harry Lesmana at St. Jude Children's Research Hospital has been selected for the 2019 ASH Research Training Award to advance his research training in hematology genetics. He will have protected time for hands-on research and gain expertise in the field.
Researchers used smFRET to study the MhsT transporter and discovered that different cargo have distinct rate-limiting steps. This finding reveals the presence of secondary binding sites on transporter proteins, crucial for regulating activity.
Researchers identified therapy-induced mutations that cause drug resistance in children with acute lymphoblastic leukemia who relapse. Thiopurine treatment may prime patients for relapse, and precision medicine approaches, such as immunotherapies, are being explored.
A clinical trial at St. Jude Children's Research Hospital found that adding chemotherapy doses in the cerebrospinal fluid improved CNS control without increasing toxicity for high-risk patients. The study reduced CNS relapse rates from 5.7% to 1.8%, making it the lowest among reported studies.
St. Jude researchers have identified key biological switches controlling regulatory T cells, which can be boosted or suppressed for cancer and autoimmune disorder treatment. The study reveals the critical role of enzymes Rag and Rheb in activating these cells, offering new avenues for immunotherapy against cancers.
Lindsay Schwarz, a St. Jude Children's Research Hospital neuroscientist, has won the prestigious NIH Director's New Innovator Award for her groundbreaking research on norepinephrine neurons and neural circuits. The award supports her innovative approaches to developing new molecular tools that target specific cell populations.
Scientists at St. Jude Children's Research Hospital have identified DDX3X as a crucial molecule in determining the fate of stressed cells. The molecule plays a pivotal role in regulating the innate immune response and helps cells interpret and respond to various stressors with measures meant to ensure cell survival or death. This findi...
Scientists created a 3D map of a mouse genome using ultra-deep chromosome conformation capture technology, revealing surprising changes in genomic organization during development. The study used machine learning to gauge how easily accessible genes are for transcription, shedding light on gene regulation and its role in disease.