Sorafenib is an oral multiple kinase inhibitor that targets both tumor-cell proliferation and angiogenesis(1). Approved in December 2005 for advanced renal cell carcinoma, it is being further developed by the manufacturer for metastatic melanoma, advanced primary liver cancer, and NSCLC. Several small studies have been presented or published(2,3). In one recent Phase II clinical trial, 30 patients with advanced iodine-refractory thyroid cancer were treated with Nexavar for a minimum of 16 weeks. The partial response rate was 23%, and the disease stabilization rate was 53%; this made for a disease control rate of 76%. The median progression-free survival was 79 weeks. Seventeen of 19 evaluable patients showed a “marked and rapid response in thyroglobulin levels.” One patient had liver failure, possibly due to Nexavar toxicity. These authors concluded that these results were better than would have been expected from chemotherapy treatment.
On Jan 13, 2013, Bayer HealthCare Pharmaceuticals and Onyx Pharmaceuticals, Inc announced that a Phase 3 trial of Nexavar (sorafenib) tablets in patients with locally advanced or metastatic radioactive iodine-refractory (RAI) differentiated thyroid cancer has met its primary endpoint of a statistically significant improvement of progression-free surviva(4)l. The study, called DECISION, evaluated the efficacy and safety of Nexavar compared to placebo. It has not been reflected in guidelines as of yet.
1.Priya Kundra, MD Kenneth D. Burman, MD Thyroid Cancer Molecular Signaling Pathways and Use of Targeted Therapy Endocrinology and Metabolism Clinics – Volume 36, Issue 3, 2007
2.Mazzaferri EL, Kloos RT. Clinical review 128: current approaches to primary therapy for papillary and follicular thyroid cancer. J Clin Endocrinol Metab 2001; 86: 1447-63.
3.Gupta-Abramson V, Troxel AB, Nellore A, et al. Phase II trial of sorafenib in advanced thyroid cancer. Journal of Clinical Oncology [early online publication] 2008; on June 9.
4.Marcia S Brose et al,Rationale and design of DECISION: a double-blind, randomized, placebo-controlled phase III trial evaluating the efficacy and safety of sorafenib in patients with locally advanced or metastatic radioactive iodine (RAI)-refractory, differentiated thyroid cancer BMC Cancer 2011, 11:349