The Strategic Communication, Education and Outreach team curates stories for press releases using scientific impact and institutional strategic alignment as central criteria.
The stories featured represent an esteemed fraction of discovery and accomplishment across the St. Jude research and clinical enterprise. Additional media engagement is pursued for each release, accompanied by promotion across our channels and platforms.
This was a remarkably prolific period for St. Jude investigators. Work published in Q1FY25 was representative of the breadth and depth of the fundamental and translational research enterprise. From unraveling the biophysical properties of molecular condensates to deciphering novel mechanisms regulating tumor growth, revealing epigenetic mechanisms of neurological disease to improving diagnoses and treatment for the most challenging forms of pediatric cancer, St. Jude investigators made significant strides in advancing our mission.
SELECT SCIENTIFIC DISCOVERIES
Nature Physics
Biomolecular condensate ‘molecular putty’ properties found encoded in protein sequence
• Cells organize their components in temporary hubs called biomolecular condensates, akin to oil droplets in water. The elasticity of condensates changes over time, becoming more putty-like with age.
• Scientists at St. Jude discovered that the elasticity of these condensates is regulated by the structure of the proteins they contain.
• This work provides quantitative evidence for the phenomenon of condensate aging, a process associated with neurodegenerative diseases.
Cell Reports
Lab-grown human neuron assembloids effectively model synaptic plasticity
• Scientists have historically relied on preclinical models, or postmortem tissues with neuropathologies, to understand neuronal development and plasticity.
• St. Jude researchers created a model of the human thalamocortical network by reprogramming human cells and engineering organoids: mini models of biological systems.
• When cultured in close proximity, the organoids created physical and functional connections that accurately represent human neural networks.
• This is a significant advancement in leveraging lab-grown models to investigate synaptic plasticity in the developing brain and decipher mechanisms of neurological disease.
Learn more: Biomolecular condensate ‘molecular putty’ properties found encoded in protein sequence
Learn more: Lab-grown human neuron assembloids effectively model synaptic plasticity
Tanja Mittag, PhD
Stanislav Zakharenko, PhD
Clinical Cancer Research
St. Jude gene panel for pediatric cancers increases access to high-quality testing
• The Pediatric Cancer Genome Project (PCGP), co-led by St. Jude, identified many genes involved in pediatric cancer. Sequencing a tumor’s whole genome can reveal driver mutations identified by the PCGP, but few institutions have the physical and digital infrastructure to perform this analysis.
• St. Jude scientists created a gene panel that tests genes known to be involved in pediatric cancer, which is more cost-effective and accessible than whole genome sequencing.
• St. Jude is freely providing the panel’s content, which could lead to better, earlier diagnoses of pediatric cancers and, thus, better outcomes.
• Ependymomas are pediatric brain tumors with no available targeted therapies, largely due to a lack of molecular understanding of the tumor microenvironment.
• St. Jude scientists used a novel murine model to experimentally confirm that serotonin is taken up by tumor cells and deposited on chromatin, facilitating tumor growth.
• Work also showed that tumor cells also secreted factors that modulate normal activity, thus promoting hyperactivity and creating a feedback loop that drives tumorigenesis.
• Targeting tumor neuronal interaction pathways offers an untested but hugely promising route for future therapeutic discovery for ependymoma and many other types of cancer.
Learn more: St. Jude gene panel for pediatric cancers increases access to high-quality testing
Improved chemokine homing enhances CAR T-cell therapy for osteosarcoma
• Chimeric antigen receptor (CAR) T cells are patients’ own immune cells reengineered to destroy cancer cells. They have been successfully used in leukemia but not yet in osteosarcoma due to their limited homing to osteosarcoma sites.
• St. Jude investigators found that osteosarcomas secrete chemokines, chemicals that drive CAR T–cell homing, for which CAR T cells don’t express receptors. Researchers modified CAR T cells, targeting the osteosarcoma antigen B7H3 to express CXCR2 or CXCR6 receptors, which recognize chemokines secreted by osteosarcomas.
• CXCR2 or CXCR6 expression enhanced CAR T cells’ ability to home to metastatic disease sites and improved their antitumor activity compared to unmodified CAR T cells in preclinical models.
• Optimizing CAR T–cell homing to osteosarcomas presents a promising strategy to improve antitumor activity and warrants early-phase clinical testing.
Learn more: Improved chemokine homing enhances CAR T-cell therapy for osteosarcoma
Journal of Clinical Oncology
Age impacts pharmacogenomics and treatment outcomes for most common form of leukemia
• Adults with B-cell acute lymphoblastic leukemia (B-ALL) have worse responses to treatment compared to children with the disease, but no biological drivers have been identified that could cause this difference.
• Work at St. Jude showed that children responded better to seven chemotherapy drugs compared to adults. This is due to distinct molecular subtypes of B-ALL that occur in different age groups.
• Children whose cancer has a gene expression pattern similar to that of adults also experience drug resistance and poorer outcomes despite their chronological age.
• These findings suggest that treatments should be tailored based on underlying molecular groups beyond age.
Learn more: Age impacts pharmacogenomics and treatment outcomes for most common form of leukemia
Lindsay Talbot, MD
Jun J. Yang, PhD
Nature Communications
Genetic ‘episignatures’ guide researchers in identifying causes of unsolved epileptic neurological disorders
• Current testing methods can identify the root cause of developmental and epileptic encephalopathies (DEEs), a group of complex and severe epilepsies, in approximately 50% of cases, which can be attributed to changes in any one of more than 825 genes. The remaining cases are rarer occurrences, but treatment and support cannot be given without an accurate diagnosis.
• St. Jude investigators demonstrated the benefit of DNA methylation “episignatures” to track the cause of unsolved rare DEE cases to their relevant gene.
• In a sample of unsolved DEE cases, the method successfully identified 2% of cases, representing a significant addition to the diagnostic landscape of rare DEE events.
Nature
St. Jude identifies SARS-CoV-2 “mimic” contributing to pediatric inflammatory syndrome
• Multisystem inflammatory syndrome in children (MIS-C) is a severe condition that typically occurs after SARS-CoV-2 infection, but whether the onset is connected to the viral infection was unknown.
• Work at St. Jude identified that a SARS-CoV-2 nucleocapsid protein has a region that is molecularly similar to the human protein SNX8. Because of the similarity, the immune system mistakes SNX8 for a viral antigen, triggering an autoimmune response.
• Understanding the link between SARS-CoV-2 and MIS-C can lead to better diagnosis and the development of targeted treatments, potentially improving outcomes for affected children.
Learn more: Genetic ‘episignatures’ guide researchers in identifying causes of unsolved epileptic neurological disorders
Learn more: St. Jude identifies SARS-CoV-2 “mimic” contributing to pediatric inflammatory syndrome
Heather Mefford, MD, PhD
Paul Thomas, PhD
Journal of Clinical Oncology
Scientists identify genes linked to relapse in the most common form of childhood leukemia
• About 15% of children with standard risk B-cell acute lymphoblastic leukemia (SR B-ALL) experience relapse, but the genetic factors driving this phenomenon are not well understood.
• St. Jude researchers used molecular profiling to pinpoint genetic alterations linked to relapse risk and treatment response, aiding in predicting patient outcomes.
• They found that specific genetic alterations and aneuploidy patterns were associated with varying degrees of relapse risk and time to relapse in childhood ALL.
• The findings provide a molecular foundation for tailored treatments based on individual relapse risk, potentially enhancing the efficacy of therapies for patients in the future.
Cell
Study reveals oleoyl-ACP-hydrolase underpins lethal respiratory viral disease
• Respiratory infections can be severe or even deadly, but it is not fully understood why some cases are more severe than others. Researchers studied the molecular drivers that provoke fatal disease.
• Work from St. Jude found that high oleoylACP-hydrolase (OLAH) gene expression, a gene encoding an enzyme involved in fatty acid metabolism, modulates severe disease outcomes. High OLAH expression can be found early and stays high as severe disease progresses lethally, but high OLAH expression was not detected in those with normal disease.
• Researchers can use this information to inform treatment strategies as patients with high OLAH gene expression early in the course of disease may benefit from more intense initial treatment.
Learn more: Scientists identify genes linked to relapse in the most common form of childhood leukemia
Learn more: Study reveals oleoyl-ACPhydrolase underpins lethal respiratory viral disease
Charles Mullighan, MBBS (Hons), MSc, MD | Ti-Cheng Chang, PhD
Jeremy Chase Crawford, PhD
Nature
New research from Children’s Hospital of Philadelphia and St. Jude poised to transform approach to diagnosing and treating acute leukemia in children
• There is a limited understanding of the genetics underlying T-lineage acute lymphoblastic leukemia (T-ALL), an aggressive, high-risk form of cancer.
• Work at St. Jude showed that genetic alterations in noncoding regions of DNA drive this cancer. Whole genome analysis of T-ALL patients revealed that about 60% of the genetic changes promoting oncogenesis are in the noncoding DNA.
• Scientists also identified 15 distinct molecular subtypes of T-ALL.
• The work will allow researchers to better identify high-risk patients who are more likely to relapse from T-ALL.
The New England Journal of Medicine
First successful treatment of pediatric highrisk refractory neuroblastoma with PARP inhibition
• Refractory high-risk neuroblastoma has a poor prognosis, and effective therapies are needed to increase survival rates among these children.
• St. Jude scientists identified a germline pathogenic mutation in the BARD1 gene, causing a deficiency in DNA damage repair in the tumor. They adapted chemotherapy regimens to include a poly ADP ribose polymerase (PARP) inhibitor, which targets the DNA damage repair deficiency.
• The treatment resulted in the eradication of the cancer, and the patient has been off treatment for over two years, making this the first successful treatment of this cancer with PARP inhibition.
• The case study has implications for integrating clinical genomics into pediatric cancer care to identify cancer vulnerabilities for targeted therapies.
Learn more: New research from Children’s Hospital of Philadelphia and St. Jude poised to transform approach to diagnosing and treating acute leukemia in children
Learn more: First successful treatment of pediatric high-risk refractory neuroblastoma with PARP inhibition
Charles Mullighan, MBBS (Hons), MSc, MD
Jinghui Zhang, PhD | Sara Federico, MD
Nature Immunology
T helper cells may be the key to improving annual influenza vaccines
• The annual influenza vaccination provides updated protection against the virus but could be improved as there is considerable variability in its efficacy.
• Work at St. Jude showed that the human immune system can fail to build immunity against the desired viral antigens, hemagglutinin and neuraminidase, because of the other components of the vaccine, which remain unchanged from year to year.
• St. Jude scientists discovered that T-follicular helper cells in lymph nodes guide which proteins are targeted by the rest of the immune system and last for long periods, effectively “locking in” the immune response, which also can block desired responses to new viral antigens.
• The research suggests creating influenza vaccines that only include the viral surface proteins would increase vaccine efficacy by preventing long-lived helper cells from generating and maintaining a suboptimal response.
Learn more: T helper cells may be the key to improving annual influenza vaccines
Journal of the National Cancer Institute
Early interventions may improve long-term academic achievement in young childhood brain tumor survivors
• St. Jude investigators found young children treated for brain tumors show early signs of falling behind in academic readiness, which predicts later academic difficulty. The gap in academic readiness between these children and their peers widens over time and affects long-term reading and math outcomes.
• The findings suggest that early interventions could help, rather than waiting until problems arise in elementary school.
• Work from St. Jude also showed that higher socioeconomic status provides some protection against the academic readiness gap, highlighting the importance of access to early developmental resources.
• Families and medical teams can make a difference by gaining and providing access to enriching experiences and support, which may mitigate the long-term academic impact of brain tumor treatments on young children.
Learn more: Early interventions may improve long-term academic achievement in young childhood brain tumor survivors
Paul Thomas, PhD
Heather Conklin, PhD
Lee, PhD
Cell Research
Urate transporter structures reveal the mechanism behind important drug target for gout
• URAT1, a transporter that regulates urate levels, is a target for drugs used to treat gout, but little is known about how URAT1 works or how mutations or drugs affect its activity.
• St. Jude scientists used cryo-electron microscopy to generate 10 unique structures of URAT1 in combination with substrates and drugs used to treat gout. They identified key amino acids, visualized the protein through its entire transport cycle and discovered that drugs that treat gout inhibit URAT1 by locking it in an inward-facing position.
• The researchers also studied mutations to URAT1 linked to kidney disease, instructing future drug discovery efforts.
Learn more: Urate transporter structures reveal the mechanism behind important drug target for gout
Chia-Hsueh
SOCIAL MEDIA
SMA Awareness Month
Over the past four years, St. Jude has been bringing neurological disease research into focus, and the Pediatric Translational Neuroscience Initiative (PTNI) is accelerating progress towards promising outcomes for patients like Camille who has type 2 spinal muscular atrophy (SMA).
St. Jude Global Transversal Program
While pediatric cancer survival rates in the U.S. now exceed 85%, the global five-year survival rate is less than 20%. Complications from infectious diseases cause more than half of all pediatric cancer deaths worldwide.
How St. Jude is staying one step ahead of pathogens
By investing in understanding the viruses, bacteria, and fungi that pose a risk to patients, St. Jude is leading an entirely new generation of researchers to stay one step ahead of pathogens.
St. Jude scientists setting the standard for RSV research
St. Jude scientists setting the standard in RSV research. Combating infectious diseases begins by understanding immune system development in the early stages of life. This is a critical step in developing novel interventions against these infections. Octavio Ramilo, MD, St. Jude Children’s Research Hospital Department of Infectious Diseases chair, and Asuncion Mejias, MD, PhD, a member in the Department, share a vision to map the formative years of the immune system at a holistic level.
St. Jude Graduate School Convocation
A new class of scholars took a giant step forward on the path to becoming scientific leaders and creative thinkers who can advance cures.
The St. Jude Children’s Research Hospital Graduate School of Biomedical Sciences recently welcomed its latest class of students at the Convocation Ceremony. The new class includes 37 students from 14 different countries, including 10 students pursing a MS in Clinical Investigations, 11 pursuing a MS in Global Child Health, and 16 pursing a PhD in Biomedical Sciences.
Forbes’ Top 10 Best Employers for Women
Inclusion and excellence have always been part of our DNA. Forbes recognized St. Jude in the Top Ten on their 2024 list of America’s Best Employers for Women.
Nurse Residency Match Day
We recently celebrated another successful Nurse Residency Match Day, marking the start of an incredible journey for our newest cohort of nurses. In June, our new graduate nurses arrived on campus to begin their 12-month clinical skills-based curriculum, designed to help them transition from student to professional with a focus on becoming Pediatric Oncology Nurses.
Match Day is the culmination of the weeks the recent nursing school graduates spent rotating through various patient care areas—ranging from inpatient and outpatient settings to surgical services and the ICU. These rotations expose residents to the full spectrum of care and foster connections throughout the organization. At the end of this process, each nurse resident will begin their personalized clinical orientation on their assigned unit, working closely with a preceptor to master the skills needed to care for our patients.
EARNED MEDIA
Halting the Bird Flu Outbreak
in Cows May Require Thinking Beyond Milk
A new study paints a complex picture of the outbreak, suggesting that the virus could be spreading in multiple ways and that it is not always mild in cows.
As the bird flu outbreak in dairy cows has ballooned, officials have provided repeated reassurances: The virus typically causes mild illness in cows, they have said, and because it spreads primarily through milk, it can be curbed by taking extra precautions when moving cows and equipment.
“I think the potential for this virus to cause very serious disease has been downplayed a bit,” said Richard Webby, an influenza expert at St. Jude Children’s Research Hospital, who was not involved in the new study. “That has probably hurt the response.”
Childhood Brain Tumor Survivors Can Lag in School --Interventions Can Help
Brain tumors in young children are rare, but those who survive them can lag in school for years afterwards, new research shows.
For those families that can afford it, intervening when kids are still in the preschool years might help them perform better academically later on, researchers said. “We now know that we don’t need to wait until patients are struggling with math and reading; we can intervene earlier,” said study senior author Heather Conklin, Chief, Division of Neuropsychology at St. Jude Children’s Research Hospital.
Sickle cell disease in India: The quest
for a cure
Currently approved gene therapies are out of reach, but research efforts in the country offer a glimmer of hope.
India has one of the highest prevalences of sickle cell disease in the world, and many of those affected are people from tribal communities and other historically disadvantaged groups. Recently approved gene therapies for sickle cell disease are an exciting new treatment option, but their price tags mean they are out of reach for health-care systems and individuals in lower- and middle-income countries. Researchers at St. Jude and elsewhere are now trying to develop similar gene therapies locally to serve people in India with severe sickle cell disease.
Childhood Acute Lymphoblastic Leukemia Subtypes, Prognostic Features Uncovered in Genomic Study
An international team led by investigators at St. Jude Children’s Research Hospital, Children’s Hospital of Philadelphia, and Children’s Oncology Group has spelled out the genomic alterations involved in a high-risk form of leukemia in children.
St. Jude is forging a new frontier in gene editing for cystic fibrosis
After establishing her lab at Cornell in 2019 and achieving notable success with publications in top journals, Liz Kellogg, Ph.D., associate member in the department of Structural Biology at St. Jude Children’s Research Hospital received a unique opportunity.
St. Jude Children’s Research Hospital had embarked on a major expansion of its structural biology and genome editing initiatives.
St. Jude using a cryo-EM system that resolves down to 1.2 Å
For decades, structural biologists have sought to map the structures of proteins and other macromolecules within cells.
Cryo-electron microscopy (cryo-EM), whose early development traces back to the 1970s, emerged as a promising technique for visualizing cells in their natural, hydrated state. But the technology’s initial inability to produce sharp images was a stumbling block in the early days. Early cryo-electron microscopes, while still a breakthrough, produced images so blurry that the field earned the selfdeprecating nickname “blob-ology.”
