Grant ID RP200103
Awarded On February 19, 2020
Title Targeting Twist2 Pathway as a Potential Therapy of Rhabdomvosarcoma
Program Academic Research
Award Mechanism Individual Investigator Research Awards for Cancer in Children and Adolescents
Institution/Organization The University of Texas Southwestern Medical Center
Principal Investigator/Program Director Eric N Olson
Cancer Sites Sarcoma
Contracted Amount $1,137,104
Lay Summary

Rhabdomyosarcoma is the most common malignant soft tissue tumor in children and adolescence, which is further is divided into two major molecular categories: fusion-positive (FP-RMS) and fusion-negative (FN-RMS). FP-RMS is commonly driven by a balanced chromosomal translocation generating an oncogenic Pax3-FOXO1 or Pax7-FOXO1 fusion protein. FN-RMS encompasses tumors that do not contain PAX3/7-FOXO1 fusion-proteins, but exhibits significantly greater genomic instability such as copy number variations and gene amplifications. Despite decades of clinical and basic research, the outcome for children with metastatic or recurrent disease is still very poor, with overall survival at 3 years only o...

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