The Children's Oncology Group Foundation, Inc.

PHOENIX, Oct. 3, 2024 /PRNewswire/ -- Scientists at Phoenix Children's Research Institute at the University of Arizona College of Medicine — Phoenix have achieved remarkable progress in medical research thus far in 2024, advancing new cures, securing significant funding and improving medical care through more than 700 active studies. "Our researchers are stretching the boundaries of science," said Stewart Goldman, MD, senior vice president of research at Phoenix Children's and Sybil B. Harrington endowed chair and professor, Department of Child Health at UArizona College of Medicine — Phoenix. "In recent months, their work has improved understanding and treatments for kids facing some of the most challenging childhood diseases, from neuromuscular disorders and congenital heart disease to fragile lungs, sickle cell disease and cancer." Just this year, the institute has published more than 310 studies and secured more than $5.9 million in funding from sources including the National Cancer Institute through Children's Oncology Group, as well as the Pediatric Orthopaedic Society of North America (POSNA). Highlights from this research include: Preventing the Spread of Lung Cancer A study led by Tanya Kalin, MD, PhD, vice chair of translational research for Phoenix Children's Center for Cancer and Blood Disorders and professor of Child Health at UArizona College of Medicine — Phoenix, revealed a novel method for preventing the spread of lung cancer. The research, featured in EMBO Molecular Medicine in April, demonstrates a new treatment approach that improves the effectiveness of chemotherapy by stabilizing tumor blood vessels. This approach contrasts with traditional methods that aim to starve tumors, which can make cancer cells more aggressive. This work represents a significant discovery that could change cancer treatment in the future. Generating Transplantable Lungs Vlad Kalinichenko, MD, PhD, director of Phoenix Children's Research Institute at UArizona College of Medicine — Phoenix, and Bingqiang Wen, Ph.D., assistant professor of Child Health at UArizona College of Medicine — Phoenix, made history when they successfully generated mouse lungs in a rat using advanced genome editing and blastocyst complementation. This breakthrough, published in March in the American Journal of Respiratory and Critical Care Medicine, is the first of its kind in the world. It provides a path forward in generating transplantable lungs, providing new hope to children in need of transplant. Identifying a New Treatment for Aggressive Brain Tumors Dr. Goldman is now leading the PBTC-048 Trial to evaluate the efficacy of the Optune device in treating recurrent, progressive or refractory supratentorial high-grade glioma or ependymoma as well as newly diagnosed cases of diffuse intrinsic pontine glioma (DIPG) in children. The Pediatric Brain Tumor Consortium represents 15 children's hospitals throughout North America. This study assesses the feasibility and safety of combining Optune, which uses low-intensity electric fields to disrupt cancer cell growth, in conjunction with and following radiation therapy. The research aims to develop a new treatment modality for children with these highly aggressive brain tumors, which currently have very poor prognoses. Treating Lung Disease in Premature Infants Dr. Kalinichenko secured a $3 million NIH grant to develop innovative new treatments for lung diseases in premature infants. Using gene and cell therapies, he is working to improve lung function and blood vessel formation in babies with bronchopulmonary dysplasia, offering a potential breakthrough in treating this life-threatening condition. Improving Outcomes Following Congenital Heart Surgery New research examining the impact of respiratory viral infections on congenital heart surgery outcomes in pediatric patients was published in the Journal of Thoracic and Cardiovascular Surgery in January. The study provides important insights for preoperative assessment and postoperative stay. It was published by a collaborative team at Phoenix Children's including Lucia Mirea, Ph.D., senior biostatistician; Arash Sabati, MD, pediatric cardiologist and medical director of the Exercise Stress program; Mohamad Alaeddine, MD, pediatric cardiothoracic surgeon; and Daniel Velez, MD, co-director, Phoenix Children's Center for Heart Care and division chief of cardiothoracic surgery. Treating Neuromuscular Conditions In March, Matthew Halanski, MD, division chief of orthopedics and sports medicine, and the Herbert J. Louis Endowed Chair in Orthopedics at Phoenix Children's, received the prestigious POSNA research award, which supports his work to enhance treatment outcomes for patients with neuromuscular hip disorders. Among many areas of research, Dr. Halanski is working to develop new surgical techniques and post-operative care protocols to improve mobility and quality of life for children with neuromuscular conditions. Diagnosing Tumors Kristian T. Schafernak, MD, MPH, Phoenix Children's pathologist, contributed to the WHO Classification of Hematolymphoid Tumors, also known as a WHO Blue Book, which was published in March and is considered the gold standard for tumor diagnosis from pathological and genetic perspectives. This work follows his previous contributions to the WHO Classification of Soft Tissue and Bone Tumors. Contributing to WHO Blue Books is a true signifier of Dr. Schafernak's expertise and underscores the global impact of his research. Advancing Sickle Cell Care As a result of Phoenix Children's partnership with Arizona State University's Center for Health Information and Research (CHiR), Arizona is now integrated into the Centers for Disease Control and Prevention's national Sickle Cell Data Collection Program. This initiative aims to quantify the size of Arizona's sickle cell population and enhance understanding and management of the disease through comprehensive data collection and analysis. "There's no time to waste when it comes to discovering new treatments for children facing catastrophic diagnoses," said Dr. Goldman. "Our goal is to advance new hope and healing across every area of child health." Research conducted at Phoenix Children's, as well as at the Phoenix Children's Research Institute at the University of Arizona College of Medicine — Phoenix, comprises more than 700 active studies, 640 research investigators and 100 research staff members including research scientists, associates, biostatisticians, pharmacists, nurses, coordinators and assistants. Scientists engage in research across multiple clinical disciplines including cancer, neurology, cardiology, pulmonology and more. About Phoenix Children's Phoenix Children's is one of the nation's largest pediatric health systems. It comprises Phoenix Children's Hospital – Thomas Campus, Phoenix Children's Hospital – Arrowhead Campus, Phoenix Children's Hospital – East Valley, Phoenix Children's – Avondale Campus, four pediatric specialty and urgent care centers, 11 community pediatric practices, 20 outpatient clinics, two ambulatory surgery centers and seven community-service outpatient clinics throughout the state of Arizona. The system provides world-class inpatient, outpatient, trauma, emergency and urgent care and has been serving children and families for 40 years. Phoenix Children's Care Network includes more than 1,405 pediatric primary care providers and specialists who deliver care across more than 75 subspecialties. Alongside our colleagues, collaborators and communities, we're elevating pediatric care, education and innovation, so we can all grow healthier together. For more information, visit phoenixchildrens.org. SOURCE Phoenix Children's WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Genetic changes in non-coding DNA impacts outcomes PHILADELPHIA and MEMPHIS, Tenn., Aug. 14, 2024 /PRNewswire/ -- Researchers at Children's Hospital of Philadelphia (CHOP), St. Jude Children's Research Hospital (St. Jude) and the Children's Oncology Group (COG) today announced a significant paradigm shift in the understanding of T-lineage acute lymphoblastic leukemia (T-ALL), an aggressive and high-risk form of cancer, to one frequently driven by genetic changes in non-coding portions of our DNA. The collaborative study, supported by the Gabriella Miller Kids First Pediatric Research Program (Kids First) and National Institutes of Health (NIH) Common Fund, was published today in the journal Nature. Continue Reading St. Jude Logo David T. Teachey, MD, is an attending physician, Director of Clinical Research at the Center for Childhood Cancer Research at CHOP and Chair of the Acute Lymphoblastic Leukemia disease committee in the Children's Oncology Group. Charles Mullighan, MBBS, MD, St. Jude Children's Research Hospital, Comprehensive Cancer Center deputy director and Department of Pathology member Many children, adolescents, and young adult patients with T-ALL traditionally respond well to initial treatment. However, patients who relapse or have treatment-resistant disease often face a dire prognosis. Given the aggressive nature and rapid progression of the disease, and limited understanding of the genetic basis of T-ALL, researchers saw an urgent need for new and effective approaches to diagnosis and treatment. "This paper is the first to transcend previous barriers and comprehensively profile the whole genome, uncovering critical insights in more than 1,300 children, adolescents and young adults with T-ALL," said David T. Teachey, M.D., an attending physician, Director of Clinical Research at the Center for Childhood Cancer Research at CHOP and Chair of the Acute Lymphoblastic Leukemia disease committee in the COG. "These findings are a significant clinical advancement, as the goal in treating T-ALL is to prevent relapse, which requires identifying the patients most at risk. This data now makes it possible to risk stratify patients with T-cell leukemia, identifying those with a high-risk of relapsing so we can treat them with newer or alternative medicines." Prior studies were unable to identify important genetic changes in T-ALL, as they focused on the coding genome, the part of DNA that encodes proteins, the building blocks of cells. However, only 1% of DNA is coding, while the other 99% is termed non-coding. Once considered useless, scientists now recognize that the non-coding region plays a key role in regulating biological processes. It signals the cell when to produce certain proteins, like a crossing guard aiding people to safely cross the street. In this case, researchers studied more than 1,300 patients treated on the COG AALL0434 clinical trial and sequenced both the tumor and non-tumor genomes of each patient. While the researchers previously suspected that non-coding DNA in T-ALL played an important role, this study's findings are the first ever to establish that at a large scale. The study found that approximately 60% of the genetic changes driving T-ALL cancer cells are non-coding changes. This fundamentally alters the way researchers think about T-ALL, offering a better understanding of disease biology. This leads to innovative treatments, including new immunotherapies developed at CHOP and St. Jude. Traditionally, patients with T-ALL have been categorized by risk based on their therapy response and immunophenotype, which profiles cell surface proteins as part of the diagnostic workup. While cell surface protein expression helps define T-ALL subtypes, it hasn't proven effective in consistently identifying which patients have a good prognosis. The new comprehensive data revealed why, strongly suggesting that a genomic approach replace the current immunophenotypic classification. As a result, the researchers developed models that incorporate genetics and response to treatment to risk stratify patients with T-ALL accurately and are currently in the process of validating results using patient samples from the next COG trial of T-ALL. "It was striking how abundant these non-coding changes were and how many of them were enhancer perturbation events, whether it was hijacking or co-option of an existing enhancer, or changes that generated a new enhancer," said Charles Mullighan, MBBS, MD, St. Jude Children's Research Hospital, Comprehensive Cancer Center deputy director and Department of Pathology member. "We now have a much stronger framework to take these alterations back to the lab and say now we've got better information to build the right models to understand the biology, and then to test therapy. We have very clear information that these are the sorts of alterations that people need to focus on to build a diagnostic test." Researchers were able to classify T-ALL into 15 subtypes with distinct gene expression and genomic drivers, including previously undefined subtypes. They refined the classification of known subtypes and showed that driver lesions, other genetic changes, and the original cell type work together to define the genomic subtype and the clinical and biological characteristics of a condition. They also observed a significant link between the type of gene alterations and outcomes in T-ALL. This new observation shows it is not only which gene is altered in the cancer cells, but also how it is altered, that helps define prognosis and chance of a cure. "Future research must continue to determine broader applications for this approach," said Teachey. "These findings offer a strong a roadmap for improving patient outcomes and curing more children and adults with T-ALL." In addition to the Gabriella Miller Kids First Pediatric Research Program and National Institutes of Health (NIH) Common Fund, the study was supported by the Leukemia and Lymphoma Society, Alex's Lemonade Stand Foundation, Hyundai Hope on Wheels, the COG Foundation, ALSAC, the fundraising and awareness organization of St. Jude, and private donations from patients and families, including the Harrison Willing Memorial Research Fund, the Invisible Prince Foundation, and the Aiden Everett Davies Innovation Fund. Polonen, et al. "The genomic basis of childhood T-lineage acute lymphoblastic leukemia." Nature. Online August 14, 2024. DOI:10.1038/s41586-024-07807-0 . About Children's Hospital of Philadelphia: A non-profit, charitable organization, Children's Hospital of Philadelphia was founded in 1855 as the nation's first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals, and pioneering major research initiatives, the hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country. The institution has a well-established history of providing advanced pediatric care close to home through its CHOP Care Network, which includes more than 50 primary care practices, specialty care and surgical centers, urgent care centers, and community hospital alliances throughout Pennsylvania and New Jersey, as well as the Middleman Family Pavilion and its dedicated pediatric emergency department in King of Prussia. In addition, its unique family-centered care and public service programs have brought Children's Hospital of Philadelphia recognition as a leading advocate for children and adolescents. For more information, visit . About St. Jude Children's Research Hospital St. Jude Children's Research Hospital is leading the way the world understands, treats and cures childhood cancer, sickle cell disease and other life-threatening disorders. It is the only National Cancer Institute-designated Comprehensive Cancer Center devoted solely to children. Treatments developed at St. Jude have helped push the overall childhood cancer survival rate from 20% to 80% since the hospital opened more than 60 years ago. St. Jude shares the breakthroughs it makes to help doctors and researchers at local hospitals and cancer centers around the world improve the quality of treatment and care for even more children. To learn more, visit stjude.org, read St. Jude Progress, a digital magazine, [] and follow St. Jude on social media at @stjuderesearch. Contact: Jennifer Lee Children's Hospital of Philadelphia (267) 426-6084 [email protected] Contact: Gary Bridgman St. Jude Children's Research Hospital Desk: (901) 595-2028 [email protected] [email protected] SOURCE Children's Hospital of Philadelphia; St. Jude Children’s Research Hospital

LOS ANGELES, July 23, 2024 /PRNewswire/ -- The St. Baldrick's Foundation, the largest charity funder of childhood cancer research grants, is proud to announce the newest round of research grants totaling $9.4 million. The grants will support 23 projects with a goal to find better treatments for childhood cancers. The grants were made possible by the generous support and donations from dedicated donors and volunteers who work hard to Conquer Kids' Cancer. St. Baldrick's is dedicated to advancing the fight against childhood cancers by supporting the most promising research initiatives. These new grants emphasize the commitment St. Baldrick's has to discover cures by providing essential funding for critical research at every stage of the journey, from early discovery to advanced clinical trials. Grants were awarded to:  Alabama University of Alabama at Birmingham, Birmingham Research grant: Emily Johnston, M.D. California Beckman Research Institute of City of Hope, Duarte St. Baldrick's Scholar: Rui Su, Ph.D Children's Hospital Los Angeles, Los Angeles Extended Scholar: Babak Moghimi, M.D. St. Baldrick's Scholar: Wendy Hsiao, M.D. University of California San Diego, La Jolla Research grant: Anusha Preethi Ganesan, M.D., Ph.D Colorado University of Colorado Denver, AMC and DC, Aurora Extended Scholar: Kelly Faulk, M.D. Georgia Emory University, Atlanta Continuing Consortia (additional year): Christopher Porter, M.D. St. Baldrick's Scholar: Hunter Jonus, Ph.D Massachusetts Brigham and Women's Hospital, Boston Research Grant: Benjamin Kann, M.D. Dana-Farber Cancer Institute, Boston Research grant: Jun Qi, Ph.D Minnesota University of Minnesota School of Public Health, Minneapolis Extended Scholar: Tianzhong Yang, Ph.D. Missouri Washington University School of Medicine in St. Louis, St. Louis St. Baldrick's Scholar: Yang Li, Ph.D Research grant: Mark Rutherford, Ph.D New York Cornell University College of Agriculture and Life Sciences, Ithaca St. Baldrick's Scholar: Meng Wang, M.D., Ph.D Pennsylvania Children's Hospital of Philadelphia, Philadelphia St. Baldrick's Scholar: Daniel Zheng, M.D. The Children's Oncology Group Foundation, Philadelphia Douglas Hawkins, M.D. Perelman School of Medicine, University of Pennsylvania, Philadelphia Research grant: Fange Liu, Ph.D Tennessee St. Jude Children's Research Hospital, Memphis Extended Scholar: Kelsey Bertrand, M.B.B.S. Texas Baylor College of Medicine, Houston Research Grant: Carl Allen, M.D., Ph.D University of Texas MD Anderson Cancer Center, Houston St. Baldrick's Scholar: Palaniraja Thandapani, Ph.D Virginia Virginia Tech, Fralin Biomedical Research Institute at VTC, Roanoke, Virginia, and Washington, D.C. Extended Scholar: Cheng-Chia Wu, M.D., Ph.D Washington University of Washington, Seattle St. Baldrick's Scholar: Lisa Force, M.D. Washington D.C. Georgetown University's Lombardi Comprehensive Cancer Center, Washington D.C Research Grant: Gary Kupfer, M.D. Since 2005, St. Baldrick's has granted over $352 million to fund the best childhood cancer research, wherever it takes place. The next set of grants will be announced in November, supported by donations made between now and October. To learn more about the research these specific grants support, check out the St. Baldrick's blog. For more information about St. Baldrick's Foundation grants or ways to get involved, please visit StBaldricks.org. About St. Baldrick's Foundation Every 2 minutes, a child somewhere in the world is diagnosed with cancer. In the U.S., 1 in 5 will not survive. The St. Baldrick's Foundation, the largest charity funder of childhood cancer research grants, has awarded more than $352 million to researchers to Conquer Kids' Cancer. When you give to St. Baldrick's, you don't just give to one hospital – you support every institution with the expertise to treat kids with cancer across the U.S. St. Baldrick's ensures that children fighting cancer now — and those diagnosed in the future — will have access to the most cutting-edge treatment, by supporting every stage of research, from new ideas in the lab to the training of the next generation of researchers, to lifesaving clinical trials. St. Baldrick's has played a role in virtually every advancement in the field over the past 25 years and remains essential in advancing progress and fostering innovation in childhood cancer research. Visit StBaldricks.org and help #ConquerKidsCancer. SOURCE St. Baldrick's Foundation