A new drug (sorafenib) showed promising results among patients with desmoid tumors, which are a type of tumor for which patients with Familial Adenomatous Polyposis (FAP) due to APC gene mutations are at risk. These tumors frequently grow and encompass internal organs and can be hard to remove surgically. The newly published research showed that treatment with sorafenib doubled the rate of progression-free survival at 2 years among patients with advanced or refractory desmoid tumors. The drug also reduced the risk of death by 87% compared to the placebo, with no major safety concerns. These advances serve to highlight some of the recent breakthroughs in the treatment of tumors among those with inherited cancer predisposition.
Gounder MM, et al. N Engl J Med. 2018 Dec 20. doi: 10.1056/NEJMoa1805052.