St. Jude Children's Research Hospital

Don’t miss the opportunity to gain insights into the mechanisms of cancer and emerging therapies for cancer treatment in our next The Science of Childhood Cancer virtual lecture on September 5th at 12PM CT/1PM ET. Join us to hear Charles Roberts, St. Jude Executive Vice President, and Director of the Comprehensive Cancer Center, share groundbreaking insights into how mutations fuel cancer and discuss the latest advancements in targeted therapies that are paving the way for more effective treatments. Register here: https://ow.ly/sREg50TaYTx

Biostatisticians at St. Jude are utilizing progressive tools and methodologies to push the boundaries of clinical care advancements. By incorporating adaptive designs and novel statistical tools, trials are more agile and precise, allowing researchers to bring new treatments to children faster and more effectively. Led by experts like Arzu Onar-Thomas, PhD, the biostatistics team is at the forefront of innovative clinical trial designs, particularly in pediatric oncology. These designs are not just about analyzing data—they start from the very beginning and are involved in every step of the process to maximize patient outcomes by delivering reliable, ethical and impactful results while prioritizing patient care. "Adaptations have been commonly used since the early days of clinical trials. What's different now is that we can incorporate more extensive adaptations thanks to the computational power we have access to," said Onar-Thomas, a leading voice in the Children’s Oncology Group (COG). This is possible in part due to cutting edge resources at St. Jude. In addition, recent advances by Haitao Pan, PhD, have further strengthened the approach to dose-finding and immunotherapy in later-phase clinical trial design. Pan developed user-friendly software tools and packages to design efficient dose-finding phase I and phase II clinical trials. "We must intelligently utilize data to better assign incoming patients the appropriate dosages. This is especially important in pediatric oncology where even phase II studies can take up to 10 years because there are very few patients," Pan said. "We streamlined the process by integrating phase I data to inform phase II to enhance our understanding of the toxicity and efficacy of the treatment, ensuring we don't overlook valuable insights as we progress to later phases." With the development of novel clinical trial design strategies, future biostatistics innovation will likely streamline drug discovery and clinical treatments. Learn more on St. Jude Progress: https://ow.ly/5Tkk50Ta76P

Don’t miss the annual Pediatric Palliative Oncology Symposium (PPOS) on Thursday, September 26.     The virtual seminar will feature a panel of healthcare professionals working in institutions across the world including St. Jude, Courageous Parents Network, International Children’s Palliative Care Network and many others to discuss "High Quality Pediatric Palliative Care Delivery for Every Child, Everywhere". The symposium will be broadcast in six languages: English, Spanish, French, Portuguese, Russian, and Ukrainian. Register here: https://ow.ly/uXMb50T9KbI

Congratulations to Nidhi Bhatt, MD, Assistant Member in Hematology and Associate Director of Pediatric Hematology/Oncology Fellowship, for being named the 2024 St. Jude Outstanding Physician. Nominated by several peers, Dr. Bhatt received recognition for exemplifying the award’s criteria, including compassion, excellent patient care, educational leadership and innovative program development. Along with her work with the Pediatric Hem/Onc Fellowship, she works with the local and international hemophilia communities to address health literacy through audiovisual educational materials. In a collaborative effort with the Department of Psychology at St. Jude, Bhatt’s research line involves assessing a patient’s health literacy and neurocognition to create patient education materials that are understandable, accurate and accessible. Additionally, her work with the St. Jude Global initiative aims to remedy any disconnections in health care communications so patients and health care providers can make shared decisions on appropriate treatment and care. Thank you, Dr. Bhatt, for all you do for patients and colleagues. #StJude #Research #Physician #Hematology #Oncology #ChildrensHospital

Lozano Guillermina, PhD at MD Anderson Cancer Center conducts groundbreaking research on the p53 tumor suppressor, a crucial transcription factor that activates hundreds of genes. Her team has identified a pan-tissue p53 signature and developed innovative conditional mutant p53 alleles to study their role in tumor progression. Join us Thursday, August 29, at 12 pm CT/1 pm ET to hear about these invaluable models for testing novel therapeutic combinations. Registration is free: https://ow.ly/V2Gi50T4uFG

We were saddened to learn that Phil Donahue, longtime television show host, St. Jude champion, and husband to our National Outreach Director Marlo Thomas died Sunday. Even as we grieve his loss, we celebrate Phil’s tireless work on behalf of the patients and mission of St. Jude.   Phil became a pioneer of the issues-based daytime talk show when he launched his first TV show in 1967. “The Phil Donahue Show” rocketed in popularity, making “Donahue” a household name across America. In 1977, he met Marlo while she was a guest on his show, and the couple later said it was “love at first sight.” They were married for 44 years. His example of selfless service, unending generosity, and constant love have inspired millions of people throughout his life. Whether it was visiting patients, raising funds for St. Jude, or supporting the FedEx St. Jude Championship, he was always ready to do what he could to help advance cures and treatment of childhood illnesses.   I hope you’ll join me in remembering Phil and offering our heartfelt condolences to Marlo and his family during this difficult time. 

St. Jude has been a driving force behind increasing the survival rate of patients with acute lymphoblastic leukemia (ALL) to 94%. One of the remaining challenges in ALL research is how to effectively translate laboratory findings into meaningful clinical advances. St. Jude is addressing this critical issue through collaboration that brings together the laboratory findings of Jun Yang, PhD, with clinical trials led by Seth Karol, MD, MSCI. Their work is applying pharmacogenomics to clinical care, moving children with ALL closer to a world of personalized medicine. This approach to utilizing genomic data to inform therapeutic strategies puts clinicians in the driver’s seat while researchers function as the navigators. Yang's research in pharmacogenomics focuses on understanding how genetic differences influence how a patient responds to treatment. Yang specializes in pharmacotyping, seeing how a patient’s leukemia cells respond to different therapies and correlating that response with the patient’s genetics to identify the genomic basis of that specific drug response. This insight has enabled the identification of new drug targets and therapeutic strategies, particularly for challenging cases of T-cell ALL (T-ALL). “Using this approach, a whole set of potential new drugs are being explored right now, in trials,” Yang said. “We hope that with our pharmacotyping platform, new targets can be found, and we can then develop a whole new generation of treatments for ALL.” The scale at which pharmacotyping can be performed has allowed clinicians including Karol to approach clinical trial design from a new perspective. Clinical trials, such as SJALL23T and RAVEN, are pioneering personalized treatment and are designed to optimize therapy for individual patients to provide even the most difficult-to-treat cases with effective care. “We have proof-of-principle in relapsed patients that this therapy might be helpful,” Karol said. “We looked at those drugs and tried to identify which ones we might combine with our upfront treatment for newly diagnosed patients so that we can treat them early and hopefully keep them from relapsing at all.” Learn more about how the St. Jude approach to translational medicine is advancing personalized care for children with ALL. https://ow.ly/PvHm50SZMmc #Pharmacogenomics #ALL #PersonalizedMedicine

Join us Thursday, August 22 at 12pm CT/1pm ET to hear Stephen Gottschalk, Chair of the Department of Bone Marrow Transplantation and Co-Director of Cellular Therapy at St. Jude, talk about how St. Jude is developing cell therapies for pediatric solid tumors in “Engineered Cell Therapy for Pediatric Cancer”. The talk will focus on CAR T cell therapy, identification of tumor associated antigens and genetic engineering approaches to improve CAR T cell effector function. Registration is free: https://ow.ly/ZgxH50SYMiC

Research from Stu McAfee, PhD, and the St. Jude Department of Diagnostic Imaging is giving a voice to patients with posterior Fossa syndrome (PFS). PFS can be a devastating outcome following brain surgery involving the cerebellum – a structure vital for movement and balance. Recent studies suggest PFS is a more comprehensive syndrome than initially assumed, affecting emotional regulation and motor planning. This work reveals crucial connections between brain regions, challenging previous assumptions and offering neurosurgeons valuable guidance. Compounding the relative scarcity of PFS cases is the delayed onset of symptoms, which include emotional changes and loss of motor skills and cognition. Patients may wake up with some mental fogginess but with the ability to speak and move, only to transition into a “locked-in” state over the following days. Researchers at St. Jude, including McAfee, are well-positioned to address the gaps in knowledge around PFS. “At St. Jude, we see enough of these patients that it is possible to gather data to arrive at some reasonable conclusions,” McAfee said. Recently, St. Jude hosted the second annual Posterior Fossa Society meeting in collaboration with the Pediatric Biomedical Imaging Initiative. As part of this gathering, neuroradiologists, imaging scientists, neurosurgeons, neuro-oncologists, psychologists, speech therapists, rehabilitation specialists, and families affected by PFS all met to discuss the advancements made and hurdles related to the syndrome. As more is uncovered about the origins of the syndrome, McAfee and the Posterior Fossa Society continue to gain ground toward the prevention and treatment of PFS. “There's an opportunity to start using models to study specific features of the disease and test interventions,” McAfee said. “This was the first meeting where somebody proposed an animal model and showed some evidence that it replicated certain features of the disorder.” Learn more on Progress. https://ow.ly/NTuC50SX6Ta

The Crawford Laboratory in the St. Jude Department of Host-Microbe Interactions brings a data analysis perspective to infectious diseases research. Led by principal investigator Jeremy Chase Crawford, PhD, the lab fosters global collaborations to explore how the body responds to infectious disease. Their work enhances the understanding of immunology and disease, shaping approaches to human healthcare. In a recent study published this week in Cell, Crawford and colleagues from the Peter Doherty Institute for Infection and Immunity, Boston Children’s Hospital, and others found that high expression of OLAH drives life-threatening immune responses. Findings reveal that high OLAH gene expression can be detected early in lethal respiratory viral disease’s progression and remains elevated as severe disease progresses. Conversely, patients with milder disease do not exhibit high OLAH levels. In addition to defining the role of OLAH in severe respiratory viral disease, knowing OLAH is expressed early in severe disease means it may be used to determine if patients need more intense initial treatment. “OLAH directly impacts disease severity in multiple globally relevant, but distinct viral infections,” said Crawford. “We started with the very specific disease context of avian influenza, but by forming these collaborations, we were able to interrogate substantially broader disease contexts for this biological mechanism.” The discovery is a result of several years of collaborative studies by scientists and clinicians around the world. Learn more: https://ow.ly/nZuS50SX58t