“Identifying novel drug therapies for desmoid tumors.” Year 1 of 3

One way to identify new drug therapies is to screen known drugs on cell cultures from tumors to see which drugs will kill the tumor cells. In the past Alman et al had tested 1,000 drugs on desmoid tumor cells and identified one such agent. Unfortunately, this agent is not readily available for use in North America. In this 2010 DTRF-funded study Dr. Alman plans to undertake a comprehensive screen of agents to identify drugs that specifically target cell viability in DTs. Building on previous work, Dr. Alman will focus on drugs that inhibit DT cell growth presumably by blocking Beta catenin signaling in mesenchymal cells. The agents identified will be tested for their ability to alter DT cell proliferation, Beta catenin protein level and differential effects on stem cells. Such drugs will be tested using DT cell cultures from patients and also transgenic mice that develop DTs. Identified agents can then be investigated in clinical trials. The exciting part is that the agents found to have clinical benefit will already have been FDA-approved for use in patients and can therefore be rapidly brought to patient care.