NCI SBIR Innovative Concept Award to study novel molecules for the treatment of rhabdomyosarcoma (Kurmasheva)

Principal Investigator: Raushan Kurmasheva


Rhabdomyosarcoma (RMS) is a rare pediatric cancer. 15% of children with RMS have metastatic disease and have a 5-year survival rate of < 30%. Their prognosis has not improved in decades.  Metastasis is a major cause of treatment failure and death. CXCR4 is highly expressed in RMS and correlates with metastatic potential and poor prognosis. NovoMedix has developed proprietary libraries of novel small molecules which reduced CXCR4 in cancer cells. The goal of this project is to develop a novel more effective treatment for RMS to extend the life and improve long-term outcomes.

The specific aims of this project are 1) identify and characterize drug candidates for RMS and select 3-5 leads, 2) screen 3 lead candidates for anti-tumor activity in 10 RMS xenograft models by a single-mouse testing approach and confirm activity using conventional testing of selected xenografts (10 mice/group), and 3) test the lead compound in an RMS metastasis model +/- vincristine and evaluate metastases.  Expectations: A novel, safe, small molecule, drug candidate will be identified that significantly reduces tumor volume, increases event-free survival, and decreases metastasis compared to vincristine. The next steps are to complete IND enabling studies to support a Phase I clinical trial.