New hope for sufferers of thyroid cancer

Patients with advanced thyroid cancer have for many years faced bleak prospects and no viable treatment options.

But now, building on recent discoveries about the genetics and cell signaling pathways of thyroid tumors, researchers are developing exciting new weapons against the disease, using kinase inhibitors that target tumor cell division and blood vessels.

Two recent clinical trials led by a researcher from the Perelman School of Medicine at the University of Pennsylvania showcase the great promise of these new approaches.

The first study provides additional data from the phase III DECISION trial of the drug sorafenib, a kinase inhibitor already approved for treatment of kidney and liver cancer, which was presented as a plenary during the 2013 annual American Society of Clinical Oncology meeting.

In the newly released findings, lead author Marcia Brose, MD, PhD, an assistant professor in the department of Otorhinolarlyngology: Head and Neck Surgery and the division of Hematology/Oncology in the Abramson Cancer Center, and her colleagues examined the effectiveness of sorafenib on thyroid cancers that harbor BRAF and RAS mutations.

Of the 417 patients enrolled in the trial, 256 had tumors collected for genetic analysis. As they expected, the most common mutations were found in the BRAF and RAS genes.

However, the analyses show that all groups, regardless of the presence of a BRAF and RAS mutation benefited from treatment with sorafenib.

The use of sorafenib for the first line treatment for advanced differentiated thyroid cancer is now being evaluated for approval by the FDA, which would represent the first effective drug for advanced thyroid patients in more than 40 years.

The second study Brose will present during the European Cancer Congress focused on the subgroup of patients with papillary thyroid cancer (PTC), which is the most prevalent form of advanced thyroid cancer.

About half of PTC patients harbor the BRAFV600E mutation, which is also present in melanomas that can be successfully treated with BRAF inhibitor drugs.

Taken together, the two trials offer substantial new hope for patients with progressive thyroid cancer.