The Food and Drug Administration has approved the first new drug to treat advanced thyroid cancer in 40 years. Now patients have new hope for more time — and more treatment possibilities down the road.
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In its priority review program, which fast-tracks review of drugs for serious conditions, the FDA approved use of the cancer drug sorafenib for patients with a late-stage (metastatic) differentiated thyroid cancer that no longer responds to radioactive iodine treatment.
The development opens up, too, the possibilities of new treatments of this type of advanced thyroid cancer with cancer drugs. Thyroid cancer is usually treated by endocrinologists because medical oncology had no effective treatment for those patients who became resistant to radioactive iodine.
Sorafenib (Nexavar®) is also used to treat liver and kidney cancer.
Drug shown to improve progression-free survival
Oncologist Tobenna Nwizu, MD, says this is an exciting development for these cancer patients. “Progressive metastatic radioactive resistant thyroid cancer is incurable, but sorafenib can help control and prevent progression of the disease,” he says.
Though not a cure, sorafenib was shown in a phase 3 clinical trials to safely and effectively improve cancer progression-free survival by five months. In the study patients treated with sorafenib had a progression-free survival of median 10.8 months as compared to 5.8 months in patients treated with placebo.
This was a long-awaited approval, says Christian Nasr, MD, Medical Director of Cleveland Clinic’s Thyroid Center.
“Because of the need for effective therapy, the FDA streamlined the approval process to give an option for patients with this type of malignancy,” says Dr. Nasr. “Otherwise these patients would have no good therapeutic options.”
Along with the encouraging development, Dr. Nwizu and Dr. Nasr raise a few cautions about:
How long the drug will be effective. “With time patients will develop resistance and will require another treatment,” says Dr. Nwizu. As differentiated thyroid cancer can also follow an indolent course, it’s important that the cancer is progressing or getting worse before initiating treatment. And Dr. Nasr notes that it “remains to be seen whether life without cancer will be prolonged beyond the duration of the phase 3 study that led to the approval of the drug.”
The high cost of treatment. “Cost might be a big hurdle because thyroid cancer is only second to lung cancer as a cause of medical bankruptcy,” says Dr. Nasr.
The drug’s side effects. They can be difficult, ranging from diarrhea, nausea and rashes to skin reactions on the hands and feet and hypertension.
Despite these factors, use of the drug may give precious extra time to some thyroid cancer patients. And it may be a sign of a new, oncology-based approach to treatment that expands the possibilities of success in the battle against this dangerous cancer.