A recent issue of the New England Journal of Medicine features the results of a multi-site clinical trial in which Dr. Mo Milhem and the University of Iowa’s Holden Comprehensive Cancer Center participated. Sorafenib, a kinase inhibitor primarily used to treat renal, hepatic, and thyroid carcinomas, was used against a placebo in a double-blind, phase 3 trial to treat desmoid tumors. Also known as aggressive fibromatosis, these debilitating and symptomatic tumors can attack internal organs and connective tissue throughout the body. There is no known accepted treatment for this difficult and rare disease.
Nearly 20 medical centers throughout the country enrolled 87 participants, with 6 people treated at Iowa. The trial results, detailed here, show dramatic promise. Against this poorly understood disease with no previous standard of care, the sorafenib group had a two-year, progression-free survival rate of 81%, compared to the placebo group rate of 36%. “These results,” Dr. Milhem said, “are very encouraging. It is very valuable for Iowa to participate in the study of such a rare disorder, truly showing our impact to all kinds of diseases.” The US Food and Drug Administration will now review the study and consider the drug for widespread use.
For more information, the National Cancer Institute press release is here: https://bit.ly/2SZD97B