In a pivotal Phase III study led by MD Anderson researchers, the oral anti-angiogenic therapy lenvatinib has shown dramatic improvement in progression-free survival in patients with advanced radioiodine-refractory thyroid cancer.
The global study, led by Steven Sherman, M.D., associate vice provost for Clinical Research and chair of Endocrine Neoplasia and Hormonal Disorders, was published in the New England Journal of Medicine. It could offer a new treatment model for a group of patients for whom, until recently, there has been no new effective treatment since the 1940s. Preliminary findings were first reported at last year’s American Society of Clinical Oncology annual meeting. The published study includes updated data.
According to the American Cancer Society, 62,450 people will be diagnosed with thyroid cancer in 2015, and 1,950 will die from the disease. It’s the fastest growing cancer type, says Sherman, with rates of refractory disease also on the rise. Until recent therapeutic advances, historically, radioactive iodine has been the only treatment available to patients with metastatic thyroid disease, he explains. While it does offer a cure to a select group of patients, more than half do not respond to the therapy.
“For decades in this patient population, the treatment was often to repeat ineffective doses of radioactive iodine, and possibly salvage therapy with chemotherapy,” says Sherman, the study’s senior author and the international principal investigator.
The international, randomized, Phase III, double-blind study enrolled 392 patients — all of whom had progressive, refractory disease — from 21 countries. Patients were randomized at a 2-1 ratio to receive either the study drug or placebo, respectively. In total, 261 received lenvatinib and 131 received a placebo. At the time of disease progression, patients in the placebo arm of the study could receive lenvatinib. The primary endpoint was progression-free survival; secondary endpoints tested response rate, overall survival and safety.
For those who received the study drug, the median progression-free survival rate was 18.3 months, compared with 3.6 months in those who received a placebo. The overall response rate in the study arm group was 64.8% (with four complete and 165 partial responses), and 1.5% in the placebo arm. The median overall survival was not reached in either group.
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