As with many other cancers, acute lymphoblastic leukemia (ALL) cases are often driven by over-active proteins, called kinases. Although for some patients targeted therapy is available, many do not have appropriate agents available and many will not survive their disease. We are looking for common targetable pathways which are turned on by these kinases.
Principal Investigator Name:
Joya Chandra, PhD
Project Title:
Targeting PLK1 as a Common Mechanism in Ph-like ALL
Diffuse intrinsic pontine gliomas (DIPG) are aggressive, inoperable brain tumors that affect children. Life expectancy from a DIPG diagnosis is less than 1 year, even with intervention. There are no known survivors of DIPG. DIPG differ from other pediatric cancers because DIPG do not respond to chemotherapy. The blood brain barrier (BBB) insulates DIPG from chemotherapeutic treatment.
Project Goal
Principal Investigator Name:
Mark Souweidane, MD & Richard Ting, PhD
Project Title:
Image Guided Diffuse Intrinsic Pontine Glioma Drug Delivery and Design
Medulloblastoma is the most common malignant pediatric brain tumor and, unfortunately, nearly one-third of affected children ultimately die of the disease despite aggressive therapy. High-risk medulloblastoma and recurrent tumors portend an even poorer prognosis. To improve patient outcomes, our primary goal is to improve methods to noninvasively detect tumor progression or relapse at early stages that will likely offer therapeutic opportunities or alter treatment strategies.
Project Goals
Principal Investigator Name:
David Lyden, MD/PhD & Parveen Raju, MD/PhD
Project Title:
Circulating Exosomes as Biomarkers of Medulloblastoma Progression, Metastasis, and Recurrence
Child's Exposure to Radiation from Medical Imaging and the Risk of Childhood Leukemia: Pooled Analyses from the Childhood Leukemia International Consortium (CLIC)
Children who continue to have small amounts of leukemia after chemotherapy, known as minimal residual disease (MRD), have a high risk of relapse. Since these MRD cells survived through chemotherapy, giving additional standard chemotherapy is not effective and new treatments are urgently needed.
Principal Investigator Name:
Alix Seif, MD
Project Title:
Sepsis as innate immune stimulus: severe sepsis exposures and protection against relapse in children with acute leukemias
The LIN28 family of RNA binding proteins are known drivers of many pediatric cancers. These proteins function by blocking the function of the let-7 family of microRNAs, which exert tumor suppressive effects. To date, despite the panoply of childhood tumors driven by LIN28, efforts to target this protein in pediatric cancer are lacking.
Project Goal
Principal Investigator Name:
George Q. Daley, MD/PhD
Project Title:
Targeted Inhibition of Lin28/let-7 Binding to Treat Pediatric Malignancy
Hux is 7 years old and a party animal who loves dancing. Since he was diagnosed with Phelan-MCDermid syndrome at birth, he gets yearly brain MRIs - and one revealed a tumor when Hux turned 3. After a year of treatment, Hux has remained cancer-free!
