2023 Department of Pediatrics Academic Annual Report

Dr. Lowell is the pediatric director of the CF Therapeutics Development Network (TDN) and the Airways Clinical Research Center at UAB. The UAB/Children’s of Alabama Therapeutics Development Center conducts numerous clinical trials that are either sponsored by the TDN or different pharmaceutical companies or are investigator-initiated. These trials include a study looking at various nutrition, gastroenterology, endocrine, pulmonary and infectious outcomes in children ages two to five years before and, longitudinally, following initiation of highly effective modulator therapy (HEMT), a study aimed at determining whether a study of adeno-associated virus (AAV) vector-mediated gene therapy in CF, a study of whether treating pseudomonas infections in CF with one or two IV antibiotics in the hospital is better and a study of mRNA therapy for CFTR replacement in CF. Drs. Harris, Guimbellot, Fowler and Poore join Dr. Lowell as principal and sub-investigators in these pivotal trials. Dr. Lowell is also involved in investigator-initiated multicenter trials, including a trial aimed at promoting self-efficacy and resilience in adolescents and young adults with CF, a study assessing knowledge and education concerning male reproductive issues in CF amongst male CF adolescents and adults, and a study determining the relationship between oral disease and lung disease in CF. Gabriela Oates, Ph.D., has expertise in the integration of social determinants of health (SDH) measures and geographic information system (GIS) methods with clinical data from medical records, patient registries and large cohort studies for research on health outcomes, health services and population health with a focus on health equity. She investigates the impact of multilevel demographic, socioeconomic and environmental factors for disparities in chronic health conditions across the life course. Her recent work highlighted the adverse effect of tobacco smoke exposure on the clinical benefit from CFTR modulators. Her project, Clinical Effort Against Smoke Exposure in CF (CEASE-CF), demonstrated the feasibility, acceptability and preliminary efficacy of a smoking cessation intervention for caregivers of children with CF. She also leads efforts to reduce the prevalence of vaping among adolescents through the Alabama Youth Tobacco Prevention Program. Dr. Oates conducts research on screening for and addressing food insecurity and other unmet needs across CF centers in the US. Other CF research is focused on the role of social determinants for the increase of obesity among people with CF in the era of highly effective modulator therapy, the use of race correction in pulmonary function testing and psychosocial interventions using mobile health (mHealth) tools. Dr. Oates is the site principal investigator of the CF Foundation’s Success with Therapies Research Consortium (STRC) and leader of the STRC mHealth Project. Dr. Oates is an author of more than 60 peer-reviewed papers and three book chapters. Dr. Poore is the assistant program director for the UAB Pediatric Pulmonary fellowship with a focus on mentoring fellowship research projects and an associate scientist at the Gregory Fleming James Cystic Fibrosis Research Center. His current work focuses on fungal infection correlations in CF populations, cardiovascular disease in people with CF, Aspergillus and Pseudomonas co-infection within the airway, the role of mucus and inflammation in Aspergillus germination, thoracic imaging quantification of pulmonary injury in animal models, and disease modeling in airway epithelium and animals. Outside of CF, he studies disease modeling of chronic airway aspiration via airway epithelium, focusing on the mucosal effects of this injury and the inflammatory response. His research is supported by the Cystic Fibrosis Foundation, the Aerodigestive Society, the Kaul Pediatric Research Institute and the NIH Loan Repayment Program from the National Heart, Lung and Blood Institute (in renewal). He is the lead PI of the UAB Pediatric Pulmonary Bronchoalveolar Lavage and Sputum Biorepository and co-investigator on CFF TDN-sponsored trials. He has additional funding to investigate inflammatory profiles in the national CFF and TDN-sponsored PROMISE study and collaborates with many investigators in the CF research community. Outside of CF, he is the lead site PI for the National Children Interstitial Lung Disease Registry (chILD), collaborating with rare lung disease experts nationwide. Miles Fowler, M.D., is a principal investigator or co-investigator on several CF clinical trials, including studies of optimal treatment for pseudomonas and the effect of high-efficiency modulators on children with CF. NEUROMUSCULAR DISORDERS Bradley Troxler, M.D., leads the Muscular Dystrophy Care Center and the CureSMA Care Center. These centers provide care to more than 300 patients annually and provide the latest disease-modifying therapies, including gene therapy, risdiplam, nusinersen Ammar Saadoon Alishlash, M.D., is the medical director of the Sickle Cell Disease Pediatric Pulmonary Program, which encompasses both clinical and research components. Dr. Alishlash’s lab investigates the pathogenesis and management of pulmonary complications of sickle cell disease (SCD). He uses humanized SCD mouse model to investigate the mechanisms and therapeutic approaches of acute chest syndrome and SCD-associated pulmonary hypertension. The clinical research focuses on the risk factors of SCD pulmonary complications. His research is funded by an R21 from the NIH. The clinical component of the program involves screening for pulmonary complications, developing protocols to manage these complications and managing these complications in children with SCD in his SCD-designated pulmonary clinic. SLEEP MEDICINE Dr. Troxler is the medical director of the Children’s of Alabama Sleep Disorder Center. This center performs around 2,000 pediatric sleep studies annually and provides clinical care to hundreds of patients annually. The Sleep Center faculty (Krizstina Harsanyi, M.D., Mary Halsey Maddox, M.D., Pedro Anis Nourani, M.D., and Dr. Troxler) are involved in several research endeavors, including NIH funded research to ascertain the impact of sleep-disordered breathing in the development of chronic lung injury in premature infants with Dr. Ambalavanan, Division of Neonatology. and exon-skipping therapy. SICKLE CELL DISEASE

2023 Academic Annual Report

114