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.
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.
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.
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.
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).
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.
When Quincy was diagnosed with a rare type of leukemia called JMML, he was just a baby. Standard treatment didn’t help, but research funded by Alex's Lemonade Stand Foundation brought him hope. This lead to a therapy that worked – and gave him his cure!
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