According to a story from EurekAlert!, the results of a recent Phase 3 clinical trial suggest that the targeted therapy sorafenib could be an effective treatment option for desmoid tumors. There is currently no established standard of care for these tumors, but these results suggest that sorafenib could play a significant role.
About Desmoid Tumors
Desmoid tumors, also known as aggressive fibromatosis, is a rare condition in which tumors originate from fibroblast cells. Fibroblasts are a type of connective tissue cell that supports internal organs and promotes wound healing. Women in their thirties are most commonly affected. Desmoid tumors are locally aggressive, but they can also grow more slowly in some cases. Their behavior is generally not easy to predict. Risk factors include family history, the genetic disorder familial adenomatous polyposis, history of abdominal surgery, and being female. Symptoms include pain, organ dysfunction, and problems with mobility. They can compress nearby organs if they reach sufficient size, and they can occasionally be life-threatening. Treatment may include chemotherapy, microwave ablation, antiestrogens, radiation, and surgery. They can occasionally reappear even after surgical removal. There is no cure for desmoid tumors. Around 1,000 cases are diagnosed in the US per year. To learn more about desmoid tumors, click here.
In the clinical trial, sorafenib was capable of halting the growth of desmoid tumors in 80 percent of patients who completed the trial. This effect was achieved for as long as two years. Of the 87 patients that participated in the trial, all of them had tumors that were causing symptoms, were progressive, or had begun to recur. These results represent a significant increase in progression free survival for patients with desmoid tumors.
Sorafenib is an inhibitor of tyrosine kinase. This interferes with the growth of cancer tumors and the development of their blood vessels. The drug has already gained approval as a treatment for several different types of cancer, including thyroid carcinoma and renal cell carcinoma. Side effects of the drug in the trial were fairly mild and included high blood pressure, rash, and diarrhea; only 22 percent of patients chose to discontinue treatment because of adverse reactions.
Check out the original study here.