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)

Synopsis

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 & Advantages

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

About the Technology

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

References & Resources

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.