If the US FDA (Food and Drug Administration) approves liver and kidney cancer drug Nexavar for use in radioactive iodine-refractory differentiated thyroid cancer, it would be the first effective agent for this disease. Nexavar is a kinase inhibitor that mediates tumor cell division and growth of tumor blood vessels.
Thyroid cancer is the fastest-increasing cancer worldwide. It is the sixth most common cancer in females. Over 213,000 new thyroid cancer diagnoses are made annually. 30,000 people die from the disease each year globally.
In 90% of cases thyroid cancer can be effectively treated and cured with surgery and radioactive iodine treatment. However, for the remaining 10% who do not respond to standard therapies, the tumors spread and appear in the lungs, bones, lymph nodes and other parts of the body.
Doxorubicin is the only other drug for thyroid cancer that has metastasized. It was approved in 1974. However, doctors do not prescribe it because it is highly toxic and ineffective.
Study leader, Marcia S. Brose, MD, PhD, said:
"Until we began using sorafenib, we had no medical options for these patients who suffered due to progression of their disease. 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 multicenter, international trial, known as DECISION, involved 417 metastatic thyroid cancer patients. 207 of them were randomly selected to receive oral drug sorafenib (400 mg twice daily), while 210 took a matching placebo. The DECISION trial started in October 2009.
The researchers reported that:
- 12% of the sorafenib patients experienced tumor shrinkage
- 0.5% of those on placebo experienced tumor shrinkage
- 42% of patients in the sorafenib group had stable disease after 6 months
- 33% of patients in the placebo group had stable disease after 6 months
- PFS (progression-free survival) in the sorafenib arm was 10.8 months
- PFS among the placebo patients was 5.8 months
- In this cross-over trial, 70% of the placebo patients switched over to sorafenib
Side effects reported among the sorafenib patients included rash, fatigue, weight loss, elevated blood pressure, diarrhea, and skin reaction. All adverse events are similar to those reported in patients receiving sorafenib for liver and kidney cancers.
Nexavar is approved by the FDA for the treatment of unresectable hepatocellular carcinoma (approved for liver cancer in November 2007) and advanced renal cell carcinoma. Nexavar is approved in more than 100 countries for these indications and is currently being evaluated for a range of cancers.
Dr. Brose presented the team's findings on Sunday, June 2nd in Room E353a, McCormick Place in Chicago. The findings of the trial were also presented in a plenary session in N Hall B1 of McCormick Place.
Bayer HealthCare Pharmaceutical and Onyx Pharmaceuticals funded the trial. (Editor's note: Dr. Brose has received consulting fees and honoraria from these companies)
Kemal Malik, MD, Member of the Bayer HealthCare Executive Committee and Head of Global Development, said "This trial is representative of our continued commitment to fully understanding sorafenib's potential applicability, especially in hard-to-treat cancers where there are limited treatment options."
Pablo J. Cagnoni, M.D., Executive Vice President, Global Research & Development and Technical Operations, Onyx Pharmaceuticals, said "In the past 30 years, there have been no new treatment options approved for patients with radioactive iodine-refractory differentiated thyroid cancer. We are pleased with the results of the DECISION study, which demonstrate sorafenib's activity in patients who have no other treatment options."
According to Bayer HealthCare Pharmaceutical, the Phase 3 DECISION data will form the basis for sorafenib's regulatory submissions for the treatment of RAI-refractory differentiated thyroid cancer. An sNDA (supplemental New Drug Application) in the USA is planned for the middle of this year, with further submissions to follow worldwide.
DECISION stands for "stuDy of sorafEnib in loCally advanced or metastatIc patientS with radioactive Iodine refractory thyrOid caNcer".
Written by Christian Nordqvist