The intended product is an orally available novel small molecule CXCR4 inhibitor for the safe and effective treatment of recurrent or metastatic rhadbomysarcoma in children, with the goal of improving survival rates and long term outcomes. Rhabdomyosarcoma (RMS) is a rare pediatric cancer, with an incidence in the US of 300 to 400 new cases per year, more than half of which are in children less than 10 years old. RMS treatment usually involves a combination of treatments, including surgery, chemotherapy, and RT. Almost 20% of children with RMS have metastatic disease at the time of diagnosis. Metastasis is a major cause of treatment failure and death. Despite heavy multi-modal therapeutic regimens, metastatic RMS has a dismal prognosis, with 5-year survival rates of less than 30% and those with bone or bone marrow metastases or metastases in more than 3 sites have survival rates of lt10%. Their prognosis has not improved in decades and novel therapies are desperately needed to halt tumor progression and reduce long term side effects in these young patients. CXCR4 is highly expressed in RMS and correlates with metastatic potential and poor prognosis. NovoMedix has developed proprietary libraries of novel, orally available, small molecules with issued composition of matter patents which reduce CXCR4 in cancer cells. These compounds have a unique combination of properties that target the tumor as well as the tumor microenvironment while protecting the heart. The goal of this project is to develop a novel, safer, more effective treatment for metastatic RMS to extend life and improve long term outcomes.
