.png)
2025-2026 Research Initatives
For 2025–26, research is expanding into hard-to-treat sarcomas like osteosarcoma, Ewing sarcoma, and rhabdomyosarcoma, which have cure rates under 30% when metastatic. Drs. Ferguson and Cai are developing a mouse model of osteosarcoma and testing a modified CAR-T cell designed to resist the effects of TGF-β—a molecule that promotes tumor growth, spread, and blocks immune responses. Their hypothesis is that making CAR-T cells insensitive to TGF-β will improve their effectiveness in this tumor type. Additionally, Dr. Ferguson is working with Oxford Biodynamics to study a blood-based biomarker (EpiSwitch®) that may detect osteosarcoma recurrence earlier than imaging. The hypothesis is that this test can identify relapse before clinical or radiologic signs appear, allowing for earlier and more effective intervention.
2022-2024 Report on Research Fund Utilization
Dr. Tse/Ferguson/Cai – CAR-T
Dr. Tse's team developed a new CAR-T cell therapy for DIPG, a deadly childhood brain tumor with a median survival of 11 months. Using CRISPR gene editing and enhanced signaling pathways, they created stronger, longer-lasting CAR-T cells. In mouse models with human DIPG tumors, a single treatment eliminated tumors in over 75% of cases, with no recurrence after a year. The work, done with Dr. Cai’s team, led to a patent and was featured at the 2025 American Society of Pediatric Hematology Oncology.
Dr. Tse/Ferguson/Cai – CAR-T
Dr. Huang’s research focused on understanding how immune cells behave in high- and low-grade brain tumors, aiming to improve treatment effectiveness by targeting the tumor’s immune environment. Dr. Ferguson has continued this work, using genomics to link immune cell activity with tumor genetics, and is preparing a manuscript with Dr. Muge Sak on key immune differences between tumor types. Dr. Huang also led a major clinical trial studying DFMO, a repurposed anti-parasitic drug, for high-risk medulloblastoma. Following his departure, Dr. Barbour is now helping lead the trial through the Beat Childhood Cancer consortium.
​Dr. Scruggs – Bone Health in Pediatric Cancer Survivors
Children with solid tumors often receive intense treatments that can harm bone health, putting them at risk for low bone density, fractures, and poor growth. While long-term effects are known, no studies have tracked bone health during or shortly after treatment. Dr. Scruggs has proposed a multi-institutional study at Norton Children’s and UK Children’s Hospital to monitor bone health in these patients using DEXA scans, lab tests, and physical therapy assessments. The goal is to explore links between bone density, activity levels, and key lab markers. The study is currently in the approval process.
Dr. LaRosa – Circadian Rhythm Disruption in ALL
Cancer and its treatments can disrupt the body’s circadian rhythm, leading to symptoms like fatigue, sleep issues, depression, and cognitive problems in youth with leukemia. These symptoms may be linked to inflammation caused by treatments like chemo and radiation. Studies show children with ALL have weaker circadian rhythms and are less active than their healthy peers. To explore this, Dr. LaRosa has begun a study using wearable devices to track sleep, activity, and light exposure, along with biomarker collection. Data collection is now underway with a small group of patients.
