Development of the First Targeted Therapy for Translocation RCC
Abstract
The FY21 KCRP Focus Areas addressed by this proposal are: Conduct basic biology research to better understand etiology and cancer progression, metastatic disease, refractory disease and therapeutic resistance, genetic and environmental risk factors, and the prevention of kidney cancer. Define the biology of rare kidney cancers and develop treatments to improve outcomes and reduce death. Develop novel therapeutic strategies for the treatment of kidney cancer, such as novel drug targets, therapeutic modalities and agents, treatment combinations and drug delivery systems. Translocation RCC (tRCC) is a rare form of renal cell carcinoma (RCC) that can affect children and young adults. Translocation RCC is caused by the fusion of two chromosomes, one of which contains the TFE3 gene. This causes the TFE3 fusion protein to travel to the nucleus of the cell, where it drives cellular growth. Translocation RCC is very different from other subtypes of RCC, and the treatment of tRCC is a major unmet clinical need. Many advances have been made in the treatment of metastatic clear cell RCC (ccRCC), but there have been few therapeutic advances for tRCC. The proposal is highly innovative because it aims to define the first targeted therapy for tRCC. The fundamental hypothesis of this proposal is that targeting TFE3 will have clinical benefit for individuals with advanced translocation RCC. The project includes a high throughput screen of small molecules to identify drugs that are effective in tRCC. This screen will focus on drugs that are already FDA-approved, thereby enhancing the opportunities for rapid clinical translation. Ultimately, our goal is to develop highly effective therapies for translocation RCC, which disproportionally affects children and young adults. These therapies are urgently needed.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Dec 28, 2022
- Source ID
- W81XWH2210901
Entities
People
- Elizabeth P Henske
Organizations
- United States Army