Columbia University Irvine Medical Center recently issued a press release published in EurekAlert! that a in a phase 3 clinical trial, sorafenib stopped the progression of desmoid tumors for two years in 80 percent of the patients who completed the treatment. Sorafenib (co-developed by Bayer and Onyx Pharmaceuticals as Nexayar) is a kinase inhibitor drug approved for the treatment of primary kidney cancer (advanced renal cell carcinoma), advanced primary liver cancer (hepatocellular carcinoma), and radioactive iodine resistant advanced thyroid carcinoma.
Desmoid tumors (also called aggressive fibromatosis) are abnormal growths that arise in connective tissue and can occur anywhere in the body. Desmoid tumors are not considered cancerous, because they do not spread to other parts of the body. They do not often cause death, but these tumors can cause significant health problems by invading surrounding tissues, causing pain, limiting mobility, and interfering with organ function. About 1,000 new cases, mostly in young adults, are diagnosed in the U.S. each year. There is no standard of care for patients with desmoid tumors. “In general, desmoids are locally aggressive and often painful tumors for which there are no effective therapies,” says Gary K. Schwartz, MD, chief of hematology/oncology at NewYork-Presbyterian/Columbia University Irving Medical Center and a senior author of the paper. “Sorafenib is an oral agent that provides a new means to directly target the ability of desmoid tumors to grow.”
In a phase three clinical trial, a drug called sorafenib stopped progression of desmoid tumors for two years in 80 percent of patients who completed treatment, a significant increase in progression-free survival compared with placebo. (Progression-free survival is the length of time a patient lives without worsening of the disease).
Gary K. Schwartz, MD