ASCO 2013 Report – Sorafenib shows efficacy in advanced differentiated thyroid cancer

by Bruce Sylvester – over 30,000 clinicians and researchers attended the American Society of Clinical Oncology 2013 annual meeting in Chicago on May 31-June 4. Among the most exciting and groundbreaking studies presented at the meeting are those featured here. They include news about targeted therapy and immunotherapy, as well as a new potential use for sorafenib (Nexavar) to treat advanced thyroid cancer.

The kidney and liver cancer treatment sorafenib prevents disease progression in metastatic thyroid cancer for almost twice as long as placebo, researchers from a randomized phase III trial reported on June 2, 2013  at ASCO.

“Until we began using sorafenib, we had no medical options for these patients who suffered due to progression of their disease,” said Marcia S. Brose, MD, PhD, an assistant professor of Otolaryngology and Head and Neck Surgery and Hematology/Oncology, the Perelman School of Medicine at the University of Pennsylvania in Philadelphia, who led the study, which is known as DECISION. “Now, we can give patients hope – a breakthrough medication that can stop the progression of the disease for 5 months. This trial is the first step in a promising series of clinical trials to identify new drugs that are shifting the horizon for patients with advanced thyroid cancer.”

The authors noted that, if it is approved for use in thyroid cancer by the Food and Drug Administration (FDA), sorafenib (Nexavar), would be the first effective agent for this patient group. The only FDA-approved drug for advanced thyroid cancer, doxorubicin, approved in 1974, is not used because it is highly toxic and is ineffective.

The researchers enrolled 417 metastatic thyroid cancer patients in the multicenter trial; 207 were randomized to oral sorafenib and 210 to placebo.

Twelve percent of sorafenib subjects achieved tumor shrinkage, compared to 0.5 percent of placebo subjects. Notably, sorafenib appeared to halt disease progression even among many subjects whose tumors did not regress.

For sorafenib subjects, median progression-free survival was 10.8 months, compared to 5.8 months for  placebo subjects.

Placebo subjects were permitted to cross over into the sorafenib arm when their disease progressed, and 70 percent did.

Overall survival data is not yet available.

Adverse events among sorafenib subjects — hand-foot skin reaction, diarrhea, alopecia, rash, fatigue, weight loss and hypertension–  were consistent with previous trials of the drug for other indications.

Bayer HealthCare Pharmaceuticals and Onyx Pharmaceuticals provided funding for the trial.