PHOENIX [October 3, 2024] – 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.