University of Miami Archives - Hyundai Hope On Wheels

2019

Scholar Grant

Matteo Trucco

Coral Gables, FL

Title: High-throughput drug screen for pediatric sarcomas

Executive Summary:
Sarcomas are cancers of bones and soft tissues. We have been successful in curing many cancers in children, but the cure rate for children with sarcomas lags behind and has plateaued for decades. The current treatments for pediatric sarcomas have many side effects and are often ineffective.
We recently developed a platform for testing 215 FDA-approved drugs against a patient’s cancer telling us specifically to what drugs it is and is not sensitive. This “drug sensitivity testing” (DST) allows for the generation of personalized treatments within 10-14 days based on directly testing drugs against that patient’s cancer cells. The DST incorporates comparing the sensitivity of the cancer cells to the sensitivity of normal cells, highlighting drugs that are exquisitely toxic to the cancer but not normal tissue.
Developed originally for adult acute myeloid leukemia (AML), our aim is to do the necessary testing to fine-tune this powerful tool to work for pediatric sarcomas. To accomplish this, the process for purifying the cancer cells from sarcomas needs optimization and we need to establish a library of sensitivity profiles for normal bone, muscle and other soft tissue cells for comparison. We will use samples of sarcomas grown in mice to perfect the cell collection technique and performing the DST. The platform’s success in AML has led to a clinical trial using it for this cancer. Our goal is to adapt this platform to pediatric sarcomas so that a corresponding trial can one day lead to developing and testing personalized treatments for children with sarcomas.

Goals:
– Establish drug sensitivity profile for normal bone and soft tissue cells.
– Adapt drug sensitivity testing platform for sarcomas.
– Produce preclinical data to support the opening of a clinical trial in pediatric sarcomas incorporating drug sensitivity testing to develop and test personalized treatment regimens.