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Development of a Pharmacodynamic Marker of EWS-FLI1 Activity to Aid in the Clinical Translation of Targeted Therapies for Ewing sarcoma

Van Andel Research Institute
Patrick Grohar, MD, PhD
Grant Type: 
Reach Grants
Year Awarded: 
Type of Childhood Cancer: 
Ewing Sarcoma
Project Description: 

Ewing sarcoma is the second most common childhood bone tumor with a poor prognosis particularly for patients who have relapsed or metastatic disease. The goal of our lab is to develop and clinically translate novel therapies for Ewing sarcoma.

Project Goal
We are working to develop new approaches to target the gene that causes Ewing sarcoma tumors to grow and spread throughout the body, a gene called EWS-FLI1. As we move these therapies in the clinic, there needs to be a way to determine if our new therapies are blocking the EWS-FLI1 drug target.

In this project, we will develop these methods and apply them to a series of clinical trials in various stages of development.  The resulting assays will allow the proper interpretation of the results of these trials. In addition, one of the assays will utilize PET imaging techniques to follow the inhibition of EWS-FLI1 in real time in patients on these trials. Therefore, we will be able to utilize these scans to monitor the blockade of the EWS-FLI1 drug target in patients to optimize the dosing of our new drugs to maximize the efficacy of the therapy while minimizing the associated side effects.

September 2017 Update

“We have now finished writing the clinical trial. We are working on obtaining the tracer and the funding for the clinical study. In addition, the last experiment we performed with this tracer and the funds from our reach award is providing data for another paper that we are writing. It further characterizes the mechanism of trabectedin in Ewing’s sarcoma patients and provides a rationale for an alternate schedule than what has previously been used in the clinic. Thank you ALSF for the support!” - Dr. Patrick Grohar


Co-funded by: 
I Care I Cure