SHARING OUR STORIES
This quarter, St. Jude continued to lead transformative advancements in pediatric medicine, public health, and scientific innovation. From groundbreaking research and precision therapies to global health collaborations, our efforts have shaped conversations around disease prevention, cancer survivorship, and emerging technologies.
Highlights include new genetic markers that refine brain tumor diagnoses, successful use of targeted therapies for high-risk neuroblastoma, and expanded access to cutting-edge research tools. Our teams also shared long-term success in treating hemophilia B, reinforcing the power of sustained, focused research.
Finally, faculty earned recognition for outstanding contributions in cancer genomics, immunology, and neurotherapeutics—further affirming St. Jude’s leadership in pediatric research and care.
ST. JUDE AND NATURE PARTNER TO PROFILE PROGRESS
St. Jude’s pioneering expertise is being showcased through featured content on Nature platforms—sharing stories of research, discovery, and impact with a global audience. We work directly with Nature Research Custom Media to develop high-quality content that adheres to the Nature editorial standards. These articles create visibility and awareness around the research enterprise, initiatives, and those representing the organization.
In Q4FY25, we highlighted publications that reveal genomic insights into pediatric cancer and innovations that could advance clinical trials and transform treatment development. Read more in the Genomic insights in cancer section of our collections page on nature.com.
Learn more: Genomic insights in cancer
SELECT SCIENTIFIC DISCOVERIES
Chromatin remodeling captured in comprehensive structural study
Mario Halic, PhD
Cell Research
• Chromatin remodeling, including “nucleosome sliding,” is a crucial process that regulates DNA access for gene expression and replication. Disruptions in this process are linked to cancer and other diseases.
• Scientists used advanced cryo-EM technology to capture the chromatin remodeler SNF2H at multiple stages of its activity. They revealed 13 distinct structures that provide a complete picture of how nucleosome sliding works.
• This foundational knowledge could lead to better understanding disease pathology and developing targeted therapies.
Study provides scaffold to selectively target drug breakdown process
Taosheng Chen, PhD, PMP
Nature Communications
• Cytochrome P450 (CYP) proteins help break down most approved drugs, but existing CYP inhibitors (coadministered during cancer treatments) often block multiple CYPs, causing dangerous drug interactions.
• Scientists developed new inhibitors that selectively target CYP34A, a key protein involved in breaking down drugs like paclitaxel (a cancer treatment) and Paxlovid (a COVID-19 therapeutic).
• Structural studies revealed how these inhibitors achieve their selectivity, paving the way for future drug development to create safer and more targeted therapies.
Learn more: Visit the St. Jude Newsroom to read the full press release.
Learn more: Visit the St. Jude Newsroom to read the full press release.
Discovery of FOXR2 activation in various brain tumors refines diagnosis to improve care
Jason Cheng-Hsuan Chiang, MD, PhD
Society for Neuro-Oncology
• The specific genes expressed by brain tumors play an instrumental role in a tumor’s classification, treatment strategy, and prognosis.
• Activation of the oncogene, FOXR2, has long been used as a diagnostic marker exclusive to central nervous system (CNS) neuroblastoma.
• St. Jude scientists found activated FOXR2 in multiple types of CNS tumors, such as high-grade gliomas and embryonal tumors, and noted that these tumors had inferior prognoses compared to CNS neuroblastoma.
• These findings suggest that FOXR2 expression should not be used as an exclusive clinical marker for CNS neuroblastoma and should be considered during therapeutic decision-making for many CNS tumors.
Researchers crack the code of cell movement
M. Madan Babu, PhD, FRS
Cell
• Understanding cell movement is crucial for advancing disease treatments and improving knowledge of healthy development. Scientists learned how chemokines and GPCRs work together to guide cellular movement through the body.
• Researchers developed a breakthrough framework to decode how these proteins interact. They discovered that specific regions in chemokines and GPCRs act like unique “encryption keys,” enabling precise chemical interactions that guide cell migration.
• By designing changes to these encryption keys, scientists demonstrated the ability to alter immune cell movement, paving the way for improved treatments like cancer immunotherapy and stem cell-based therapies.
Learn more: Visit the St. Jude Newsroom to read the full press release.
Learn more: Visit the St. Jude Newsroom to read the full press release.
Unique model of rare epileptic disease helps pinpoint potential treatment route
Heather Mefford, MD, PhD
Science Translational Medicine
• UBA5-associated developmental and epileptic encephalopathy is a rare disease causing severe developmental delays and early-onset seizures.
• Scientists developed a groundbreaking organoid model using patient-derived cells to uncover the disease’s root causes.
• Researchers also found that increasing expression of the partially functioning UBA5 gene reversed the effects of the mutation, pointing to a promising potential treatment for further study.
Study reveals targetable mechanism behind high-risk predisposition gene in pediatric medulloblastoma
Paul Northcott, PhD
Cancer Cell
• Children with a nonfunctional ELP1 gene are predisposed to a specific type of medulloblastoma, a malignant pediatric brain tumor.
• Researchers discovered that ELP1 deficiency reduces the activity of the tumor suppressor p53, increasing the risk of SHH-medulloblastoma.
• The study revealed a promising targeted therapy for SHHdriven medulloblastoma associated with ELP1 germline deficiency — inhibiting MDM2 — which restored p53 activity, extending survival in model systems.
Learn more: Visit the St. Jude Newsroom to read the full press release.
Learn more: Visit the St. Jude Newsroom to read the full press release.
Infant immune systems respond differently to severe COVID-19
Octavio Ramilo, MD and M. Asuncion Mejas, MD, PhD
Nature Communications
• Researchers studied how hospitalized infants respond to SARS-CoV-2, revealing unique immune features compared to other age groups.
• Infants showed increased antiviral interferon activity and inflammatory cytokines, along with a robust new antibody response, as maternal antibodies did not offer protection.
• These findings highlight the distinct nature of infant immune responses to severe COVID-19, underscoring the importance of studying this age group to develop effective treatments for early-life infections.
Research reveals unexpected roles of TEAD proteins in neurodevelopment
Xinwei Cao, PhD
Genes and Development
• TEAD proteins play a key role in the Hippo signaling pathway, which is essential for organ development, regeneration and tumor formation.
• Scientists discovered that TEAD proteins function differently depending on their interacting partners in specific cell types, particularly in neurodevelopment.
• The findings reveal challenges for developing drugs targeting TEAD proteins. Such treatments could lead to unintended effects due to the proteins’ contextdependent roles in different cells.
Learn more: Visit the St.
read the full press release.
Learn more: Visit the St. Jude Newsroom to read the full press release.
Jude Newsroom to
Research untangles role of stress granules in neurodegenerative disease
Tanja Mittag, PhD
Molecular Cell
• Mutations in stress granule-forming proteins are linked to neurodegenerative diseases like ALS, as they lead to harmful amyloid fibril formation. Stress granules are biomolecular condensates, but their role in fibril formation has been unclear.
• Researchers found that disease-causing fibrils can form with or without condensates, and mutations weaken condensates while accelerating fibril formation.
• Stabilizing stress granules suppressed fibril formation, showing their protective role and offering insights for development of therapies for neurodegenerative diseases.
Genetic and therapy type determine second cancer risk after childhood treatment
Yadav Sapkota, PhD
The Lancet Oncology
• Research from the St. Jude Lifetime Cohort Study (St. Jude LIFE) and the Childhood Cancer Survivor Survey (CCSS) found that pediatric cancer treatment type and genetics are the biggest contributors to the risk of developing a second cancer.
• Chemotherapy and genetics can contribute up to 35% and 37% of second cancer risk, respectively, depending on cancer type. This suggests that, in some cases, inherited genetic risk can be more impactful than chemotherapy.
• These findings emphasize the need for clinicians to consider both genetic factors and treatment history when caring for childhood cancer survivors to manage long-term risks better.
Learn more: Visit the St. Jude Newsroom to read the full press release.
Learn more: Visit the St. Jude Newsroom to read the full press release.
Hemophilia B gene therapy demonstrates long-term success
Andrew Davidoff, MD and Ulrike Reiss, MD
The New England Journal of Medicine
• Hemophilia B is a rare genetic disorder caused by low levels of factor IX, a crucial protein for blood clotting.
• A 13-year follow-up of a one-time gene therapy trial showed sustained factor IX levels, a nearly tenfold reduction in bleeding episodes, and minimal side effects in participants.
• This study provides the longest evidence of gene therapy’s effectiveness for hemophilia B, supporting its potential as a long-term treatment option.
Imaging-based STAMP technique democratizes single-cell RNAresearch
Jasmine Plummer, PhD
Cell
• Single-cell RNA sequencing is vital for understanding health and disease, but high costs and limited cell throughput have restricted its use.
• Researchers developed STAMP (Single-cell Transcriptomics Analysis and Multimodal Profiling through Imaging), a new technique that combines microscopy with RNA detection, enabling analysis of over a million cells per slide at a fraction of the cost of current methods.
• STAMP uses standard lab equipment, avoids biases in traditional sequencing, and makes advanced singlecell analysis more accessible for research and clinical applications worldwide.
Learn more: Visit the St. Jude Newsroom to read the full press release.
Learn more: Visit the St. Jude Newsroom to read the full press release.
FACULTY HONORS AND AWARDS
St. Jude Children’s Research Hospital leukemia researcher Charles G. Mullighan, MBBS (Hons), MSc, MD, senior deputy director of the St. Jude Comprehensive Cancer Center, has been elected as a Fellow of the Royal Society of London, the oldest scientific academy in continuous existence. Mullighan was selected to join the Royal Society for his trailblazing contributions to genomic research, which have advanced the understanding, diagnosis and treatment of acute leukemia, notably childhood acute lymphoblastic leukemia (ALL). His studies have redefined the disease by identifying novel subtypes, uncovering critical genomic drivers and advancing precision medicine approaches.
TIME has named Richard S. Finkel, MD, Center for Experimental Neurotherapeutics (CENT) director at St. Jude Children’s Research Hospital, to TIME100 Health, the news magazine’s list of 100 individuals who are positively influencing global health in 2025. Finkel, a leading pediatric neurologist, joined St. Jude in 2020 to lead the creation of CENT, the clinical arm of the Pediatric Translational Neuroscience Initiative (PTNI). His arrival signified an expansion of the institution’s focus on pediatric catastrophic diseases to include neurologic disorders.
Hongbo Chi, PhD, a faculty member and the Robert G. Webster Endowed Chair in Immunology, was named chair of the Department of Immunology. In his new role, Chi will recruit world-class scientists to broaden the scope of immunology research at St. Jude. Future expansion areas include investigations of cell–cell interaction networks that regulate immunity in tissue microenvironments and the intersection of the immune system with other organ systems. Chi will also foster the applications of cuttingedge systems biology and immuno-engineering approaches to advance immunological science.
St. Jude Investigators Recognized by the American Association of Cancer Research
This year, AACR has elected Charles W.M. Roberts, MD, PhD, executive vice president and director of the St. Jude Comprehensive Cancer Center, to serve on the organization’s Board of Directors. Roberts leads research in the field of cancer epigenetics, and his work has led to new investigational cancer therapies for both children and adults. He studies how chromatin remodeling complexes, which normally allow access to DNA so genes can be transcribed, contribute to cancer.
Adam Durbin, MD, PhD, has been named one of AACR’s NextGen Stars, which recognizes up-and-coming researchers in the field of pediatric cancer. Durbin is an assistant member in the division of Molecular Oncology, where his research focuses on translating laboratory discoveries into clinical breakthroughs for patients with difficult-to-treat malignancies.
Jinghui Zhang, PhD, has been named a member of the 2025 AACR Class of Fellows. Each year, clinicians and researchers are selected as fellows to highlight their groundbreaking contributions that have driven significant innovation and progress in the field of cancer research. Zhang is the former chair of the St. Jude Department of Computational Biology and is known for her computational expertise in pediatric cancer genomics. Zhang is the mind behind St. Jude Cloud, conceptualizing the resource and overseeing its development and launch.
AACR honored Charles Mullighan, MBBS (Hons), MSc, MD, for his contributions to pediatric cancer research at this year’s annual meeting. Mullighan received the St. Baldrick’s Foundation Award for Outstanding Achievement in Pediatric Cancer Research. This award is given to those who have made significant discoveries in the pediatric cancer research community and to honor their improvement in the understanding and treatment of pediatric cancer.
MEDIA SPOTLIGHTS
St. Jude in the Headlines: Protecting
Children in a Shifting Infectious Disease Landscape
Recent national media coverage has spotlighted a central truth: Vaccines remain our strongest line of defense in protecting children. This quarter’s headlines — from measles surges to new RSV approvals and avian flu concerns — highlight the urgency of advancing vaccine access and education. These stories also underscore how St. Jude continues to shape the conversation through research, preparedness and public engagement.
Vaccines in the Spotlight: A Public Health Imperative
The resurgence of measles has driven media focus on the critical role of childhood vaccines. In NBC New York’s report, Diego Hijano, MD, a St. Jude investigator specializing in pediatric infectious diseases, was interviewed to provide expert insight on the safety and efficacy of the MMR vaccine. Hijano emphasized the importance of vaccination in preventing outbreaks, particularly among vulnerable children, highlighting St. Jude’s frontline role in research and advocacy for immunization.
A Breakthrough Season for RSV Protection
This quarter marked a significant turning point in the prevention of respiratory syncytial virus (RSV), a leading cause of severe respiratory illness in infants. Media coverage in WebMD, Pharmacy Times and Scripps News highlighted new FDA approvals for RSV preventive therapies, including monoclonal antibodies and a maternal vaccine. Octavio Ramilo, MD, was an investigator in pivotal clinical trials contributing to these approvals, and his expert commentary framed the clinical data and real-world benefits.
“We must listen to families’ concerns and help them understand that vaccinating our children prevents measles, polio, and a variety of other infectious agents and their potentially devastating consequences. I do it to protect my children; will you do it to protect yours?”
- Diego Hijano, MD
“Enflonsia combines dosing convenience with strong clinical data showing significant reductions in RSV disease incidence and hospitalizations, making it a promising new intervention to help protect infants from RSV.”
- Octavio Ramilo, MD
WEBBY WEIGHS IN
In a recent feature by Newswise, Richard Webby, PhD, shared insights on the growing threat of H5N1 bird flu, now spreading among U.S. dairy cattle. Drawing on decades of influenza research at St. Jude, Webby explained how the virus is evolving in new ways — crossing species barriers and posing serious risks to public health. His message was clear: Understanding how these viruses adapt is key to staying ahead of the next potential pandemic.
Listen to the podcast
The recent media attention on infectious disease reflects increasing public engagement with vaccine development and outbreak preparedness. St. Jude’s contributions — through basic and translational research, global health collaborations and evidence-based education initiatives — position the institution as a credible source of expertise. This coverage underscores St. Jude’s role in supporting prevention-focused solutions that are scientifically grounded and globally relevant.
Washington Post Highlights the Leadership of St. Jude in Survivorship
In a May 2025 feature, The Washington Post spotlighted the growing population of childhood cancer survivors—and the leadership of St. Jude in ensuring they thrive. The story prominently features St. Jude experts Greg Armstrong, MD, MSCE, and Melissa Hudson, MD, and highlights the institution’s groundbreaking survivorship research, including the Childhood Cancer Survivor Study and St. Jude LIFE.
St. Jude is recognized as the global thought leader in this space—developing care guidelines, launching tools and shaping how survivorship care is delivered worldwide.
Protein-Slayer Drugs Offer Hope for Treating Childhood Cancers
In a May news feature, Nature spotlighted the promise of PROTACs — novel drugs that destroy cancer-driving proteins — in tackling hard-to-treat pediatric tumors. St. Jude was featured as a key player, with Charles Mullighan, MBBS (Hons), MSc, MD, leading efforts to target fusion proteins that drive childhood blood and brain cancers. St. Jude is advancing this emerging science from lab to clinic, reinforcing its role as a global thought leader in pediatric cancer research.
Targeted Triumph: St. Jude Leads PARP Breakthrough in Neuroblastoma
St. Jude Children’s Research Hospital garnered media attention for its groundbreaking precision-medicine success: using genomic analysis to identify a BARD1 gene mutation in a child with high-risk, refractory neuroblastoma and administering a PARP inhibitor regimen that has resulted in over 2.5 years of disease-free remission. Coverage in Medscape highlighted not only the clinical breakthrough but also the human-interest story of 3-year- old Maria (Aspen)Tangonan, who came from the Philippines to receive no-cost treatment. Leading this pioneering work, Sara M. Federico, MD, St. Jude Solid Tumor Division director and an internationally recognized thought leader in pediatric oncology, served as co-corresponding author and emphasized the power of genomics-guided therapy; she is also spearheading multiple clinical trials aiming to extend such targeted approaches to more children with high-risk solid tumors.
Stamping Cells to Revolutionize Research
National Geographic Espana highlighted the transformative potential of a new technology developed through an international collaborative effort that includes Jasmine Plummer, PhD, St. Jude Center for Spatial OMICs director. The article showcased the potential for this single-cell analysis approach to help scientists around the world study diseases more accurately, develop personalized treatments, and contribute to a fundamental understanding of cell biology. The coverage underscores the pivotal role St. Jude plays in accelerating discovery and sharing innovation globally.
Sharing stories of impact and discovery across the St. Jude research enterprise.
Scientific advances make the mission of St. Jude Children’s Research Hospital possible every day. St. Jude Progress tells the stories of those advances from the investigators, laboratory researchers and clinical scientists who are making them.
Scan the QR code to learn more about St. Jude Progress
