University of Texas Southwestern Medical Center

University of Texas Southwestern Medical Center

5323 Harry Hines Boulevard

Dallas, TX 75235

United States

Boosting antitumor immunity to treat pediatric choroid plexus carcinoma

Choroid plexus carcinoma (CPC) is an aggressive and rare malignant disease mainly observed in young children with a median progression-free survival of ~13 months. Chemotherapy, radiotherapy, and surgery are the only currently available therapeutic options in children with CPC. A prospective clinical trial CPT-SIOP-2000 showed the clinical benefit of carboplatin/etoposide/vincristine combo therapy in CPC patients, with 62% and 47% progression-free survival rates at 5 and 10 years, respectively.

Identifying Therapeutic Vulnerabilities in Pediatric AML through Investigation of the MECOM Transcriptional Network

Leukemia, Acute Myeloid Leukemia (AML)

Pediatric acute myeloid leukemia (AML) is an aggressive cancer of the blood that despite very intense treatment can be very difficult to cure. In particular, a subset of AML characterized by genetic changes affecting a protein called MECOM have very poor outcomes. MECOM is also a critical regulator of normal blood stem cells that helps maintain stem cells throughout an individual?s life. High MECOM levels in AML confer stem-cell like properties to AML cells and make them more resistant to standard treatment and more likely to relapse.

Role of Primary Cilium Signaling and Dynamics in Medulloblastoma Initiation and Progression

Medulloblastoma

Zebrafish Modeling of PAX3-FOXO1 Driven Rhabdomyosarcoma

Rhabdomyosarcoma

Background

Sarcomas are cancers that derive from soft tissue including skeletal muscle, fat, and connective tissue. The Amatruda lab uses zebrafish that express human cancer genes as a model to study sarcoma origins and their underlying biology. Zebrafish share over 70% of their genes with humans, and approximately 84% of known human disease genes have a zebrafish counterpart.

B-lapachone as a Novel Targeted Therapy for ATRTs and other Pediatric Cancers

Atypical Teratoid/Rhabdoid Tumor (AT/RT)

Background

Atypical teratoid rhabdoid tumors (ATRTs) are highly lethal cancers that most commonly occur in the brain of young children. Despite maximally intense therapy, most patients with ATRTs die of their disease and the few children who survive are often significantly disabled due to the effects of radiation on their developing brains. Historically, using targeted therapies for ATRTs has been very difficult as the most common gene mutation in these cancers is not currently druggable.

Screening for New Rhabdomyosaroma Pathogenesis Genes in a Drosophila Model

Rhabdomyosarcoma

The Galindo lab's research is built upon a fundamental interest in the mechanisms responsible for childhood cancer. The lab focuses on tumors that grow from the soft tissues (e.g., muscle, fat), called sarcomas, which are notoriously aggressive, and prefer childhood tissues. Little is understood about sarcoma biology, explaining why they have been difficult to study, and cure. We investigate the most common soft tissue sarcoma, the muscle-type tumor rhabdomyosarcoma (RMS).

Validation of microRNAs as valid targets in a pediatric sarcoma

Rhabdomyosarcoma

Rhabdomyosarcoma is the most common soft tissue cancer in childhood. Despite rigorous clinical trials, the survival for children with high-risk rhabdomyosarcoma has not changed for three decades. The children that do survive often suffer from life-long disfigurements as a result of the aggressive treatment. Thus, there is a tremendous need for the development of novel treatments of this disease. Rhabdomyosarcoma resembles developing skeletal muscle and has been speculated to originate from genetically compromised skeletal muscle progenitors.