The collaboration for a cure project screened tens of thousands of compounds that might inhibit cell growth in desmoid tumors. These compounds included drugs that are currently in use for clinical care, but not for treating desmoids; agents that could be used as drugs; or agents that might suggest a possible drug therapy. We identified over 50 compounds that inhibited cell growth in desmoid tumors. We then tested the compounds that could be used as drugs in genetically modified mice that develop desmoid tumors. The mouse work in ongoing, but has already identified six possible new drugs that could be developed into potential therapies. One therapy that can be rapidly applied to patient acre is the use of glucocorticoids. This treatment significantly decreased tumor burden in treated mice. Glucocorticoid drugs are currently being used along with traditional chemotherapy in leukemia, and in that disease adding the glucocorticoids substantially improved survival. We plan to present more on our preliminary work on this at the DTRF annual patient meeting in the fall. Our ongoing work is testing the drugs in combination, towards the goal of developing an effective multi-drug regimen. Our finding that glucocorticoids inhibit tumor burden, raises promise that these drugs can be added to other therapies, much in the same vein as this drug family’s use in leukemia, substantially improving outcome.

Marlene Portnoy | 914.262.6595
Co-Founder/ Executive Director
The Desmoid Tumor Research Foundation