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Mediators of Metastasis in Alveolar Rhabdomyosarcoma.

University of Texas Health Science Center at San Antonio
Koichi Nishijo, MD, PhD
Grant Type: 
Young Investigator Grants
Year Awarded: 
Type of Childhood Cancer: 
Project Description: 

My research interest is a cancer called rhabdomyosarcoma, and specifically the alveolar variant.  Alveolar rhabdomyosarcoma is an aggressive childhood tumor that is difficult to treat when the disease goes beyond the primary site and is virtually incurable when widely metastatic.

Project Goal

Our laboratory has established a unique genetically-engineered (GEM) mouse tumor model that authentically recapitulates the progression of human alveolar rhabdomyosarcoma by re-creating the mutations known to occur in human tumors. Thanks to the funding from ALSF, we have demonstrated that this mouse tumor has the same molecular and biological characteristics as the human disease and thus this model can be applicable to preclinical drug testing. We have also uncovered the genetic pathways conserved between mouse and human diseases and identified the potential therapeutic targets in this disease.

In addition, this mouse line will be the first GEM model ever to be incorporated into the NCI-supported Pediatric Preclinical Testing Program (PPTP), of which our laboratory is soon to be a member. In addition to the NCI activities in the near future, we are already testing a number of promising drugs using this mouse model and we have shared our results with the Children’s Oncology Group to help design better clinical trials.

I am grateful for this training opportunity in pediatric cancer research as a postdoctoral fellow in the laboratory of Dr. Charles Keller. ALSF’s support afforded me the early career development that has allowed me both training and the opportunity to advance my research interests in rhabdomyosarcoma with two recently published first author papers, including one highlighted in April 2009 issue of Cancer Research (Nishijo et al, Credentialing a preclinical mouse model of alveolar rhabdomyosarcoma). I remain fully committed to a career in pediatric cancer research and, thanks to ALSF, I am now a potential candidate for a NIH K99 grant, which I will submit in June. To read more about my progress and other laboratory activities, please visit our lab’s blog.