Targeting of EWS-FLI1 and RHA Interaction as Anti-Tumor Therapy

Section: For Industry

Category(ies): Cancer Therapeutics

Reference #: TOJE439502

OTC Contact: David Humphrey (Directory Information | Send a Message)

Description

EWS-FLI1 has been identified as a critical target in Ewing’s Sarcoma Family of Tumors (ESFT) over 15 years ago, yet no therapies have been developed that impact the outcome of the disease. ESFT contains a characteristic translocation t(11:22), which leads to the oncogenic transcription factor EWS-FLI1. EWS-FLI1 is a critical tumor-specific oncogene in patients with ESFT because it is derived from a chromosomal translocation necessary to maintain tumor growth. RNA helicase A (RHA), a member of the DEXH box helicase family of proteins, is an integral component of protein complexes that regulate transcription, splicing and mRNA translation in a distinct class of proteins. EWS-FLI1 and RHA interact to promote and maintain the oncogenic phenotype of ESFT. Novel small molecule compounds and peptides have been developed that disrupt the EWS-FLI1 and RHA interaction, demonstrating a potential therapy for treating Ewing’s sarcoma as well as pancreatic cancer, acute myeloid leukemia, prostate cancer, and congenital fibrosarcoma.

Applications

The novel small molecule compounds and peptides are potential therapies for Ewing’s sarcoma, pancreatic cancer, and prostate cancer.

Advantages

Stage of Development

Stage of Development:  Novel small molecules synthesized by Georgetown are being characterized for efficacy and disruption of the RHA and EWS-FLI1 interaction.

Inventors: Dr. Jeff Toretsky, Dr. Aykut Uren, and Dr. Milton Brown

Relevant Publications

Relevant Publications:  Toretsky et al. Oncoprotein EWS-FLI1 activity is enhanced by RNA helicase A. Cancer Research 66(11): 5574-5581, 2006.

Patent Status

US and Foreign rights are protected.