Andrew Lee Hong, MD

Wilms tumor is a type of kidney cancer that occurs in children. Survival rates for Wilms tumor have increased dramatically over the past few decades, and now about 90% of children will survive at least five years. There is a subtype of Wilms tumor called 1q, which is responsible for poorer outcomes in children. Researchers are attempting to map the genetic structure of this subtype in order to develop targeted treatments that will improve outcomes for this higher risk group.

Andrew Hong, MD, received a grant from CureSearch to map the “region” of the gene involved in 1q amplification. Genetic codes are like maps for understanding how diseases progress. Dr. Hong hopes to discover the portion of the map that will give insight into the 1q subtype of Wilms tumor, in order to better target it. Since there are currently limited cell lines for Wilms tumor, his team is working with the Broad Institute to map Wilms tumor cell lines in order to better understand how the disease initiates and progresses. The Broad Institute uses special machines that translate genetic code to map these cells. Dr. Hong uses this genetic information to determine potential genetic vulnerabilities. This information allows the team to run informed screens for the identification of drugs that can target Wilms tumor. If this work is successful, it could lead to new therapeutic options for this higher risk Wilms tumor subtype.

As the result of this project, Dr. Hong has made significant contributions to the understanding of 1q Wilms tumor:

• 100 patient RNA-sequencing data analyzed
• 7 in vitro Wilms tumor models created
• 2 in vivo Wilms tumor models developed that recapitulate Wilms biology
• 5 RNA interference screens performed
• 4 CRISPR-Cas9 screens performed
• 1 drug target identified