Dr. Argon investigates the unfolded protein response (UPR) , an essential signaling network that determines life or death of stressed cells and tissues. The IRE1 sensor of UPR responds to metabolic stress through four distinct activities and he focuses on determining which stress condition induces each activity and how they are integrated to enable the cells to cope with stress.
Dr. Ahrens-Nicklas works to understand why patients with inherited biochemical disorders often suffer severe, untreatable neurologic and cardiac symptoms. She strives to elucidate the link between biochemistry and network excitability, in order to drive new approaches to therapy.
Dr. Felix uses molecular, biochemical, cellular and in vivo methods to investigate the pathobiology of leukemias with KMT2A (MLL) translocations. Leukemias with these translocations affect infants and young children or occur as a complication of type II topoisomerase (TOP2) poison chemotherapies used for anti-cancer treatment. She aims to develop better treatment and prevention approaches for these leukemias, which have a poor prognosis.
Physician-scientists in the Cancer Center at Children's Hospital of Philadelphia (CHOP) have developed a preclinical, potent therapy attached to an antibody that targets a surface protein expressed in most childhood neuroblastomas, effectively killing cancer cells.
New preclinical findings from extensive cell and animal studies suggest that a drug already used for a rare kidney disease could benefit patients with some mitochondrial disorders—complex conditions with severe energy deficiency for which no proven effective treatments exist. Future clinical research is needed to explore whether the drug, cysteamine bitartrate, will meaningfully benefit patients.