Research in thyroid cancer

We are always learning more about cancer. Researchers and healthcare professionals use what they learn from research studies to develop better ways to treat thyroid cancer. The following is a selection of research showing promise for treating thyroid cancer.

We’ve included information from the following sources. Each item has an identity number that links to a brief overview of the research.

  • PubMed, US National Library of Medicine (PMID)
  • Canadian Cancer Trials and (NCT)
  • American Society of Clinical Oncology (ASCO)


Researchers are looking for the best ways to treat thyroid cancer using surgery and improve how surgery is done.

Transaxillary robotic thyroidectomy surgery (TARS) allows the surgeon to remove the thyroid using a cut (incision) in the armpit rather than the neck. The surgeon uses a computer to move surgical instruments that are connected to robotic arms. Studies have confirmed that TARS is safe and can successfully remove the thyroid. It has a similar risk for complications and leads to similar treatment outcomes as the traditional surgery that uses a cut in the front of the neck. This surgical technique can also be used to remove lymph nodes from the neck (Journal of Laparoendoscopic & Advanced Surgical Techniques Part A, PMID 30835159; Surgical Endoscopy, PMID 27572060; The Journal of Surgical Research, PMID 29907208).

TARS followed by radioactive iodine (RAI) therapy seems to be an effective treatment for differentiated thyroid cancer, even for people with lymph node metastases (European Thyroid Journal, PMID 30023348).

Chemotherapy and other drugs

The following is noteworthy research in chemotherapy for thyroid cancer.

Zoledronic acid is a bisphosphonate drug used to treat osteoporosis (loss of bone density). It is also sometimes used to treat bone metastases in people with cancer. Bone metastases in people with differentiated thyroid cancer cause skeletal-related events (SREs), which are complications that affect the bone, including fractures, spinal cord compression, bone pain and hypercalcemia (high calcium level). Researchers evaluated the treatment of thyroid cancer bone metastases with zoledronic acid and RAI therapy. The group that received the drug had better overall survival and fewer SREs. There was significant improvement in overall survival when the bone metastases were treated with zoledronic acid and responded to the RAI (European Thyroid Journal, PMID 30800641).

Radiation therapy

Researchers are looking for the best ways to treat thyroid cancer using radiation therapy.

Radioactive iodine (RAI) therapy is used to treat differentiated thyroid cancers that take up iodine. The thyroid gland absorbs almost all the iodine in the body, so radioactive iodine can be used to kill thyroid cells. This treatment is used to destroy thyroid cells left behind after surgery or to treat some types of thyroid cancer that have spread to lymph nodes or other areas of the body. It is not known if this therapy is needed for thyroid cancers that are considered low risk. Researchers are trying to determine if radioactive iodine therapy is helpful in treating well-differentiated thyroid cancer following surgery to remove the whole thyroid. Participants in the study will be treated with either RAI or no iodine. The researchers will analyze the results to see if there is a difference in disease-free survival between the 2 groups (, NCT01398085).

Targeted therapy

The following is noteworthy research in targeted therapy for thyroid cancer.

Larotrectinib (Vitrakvi) is tropomyosin receptor kinase (TRK) inhibitor. Rearrangements of the genes that code for the TRK proteins happen in several types of cancer. This can lead to “TRK fusion proteins” that are always active and can promote cancer. Researchers are studying larotrectinib to see how well it can treat cancers with TRK fusion proteins. The drug was effective in stopping cancer progression in up to 80% of study participants (New England Journal of Medicine, PMID 29466156). This is important for thyroid cancer since genetic studies have shown that TRK mutations occur in papillary thyroid cancer, which is the most common type of thyroid cancer. Although only a small number of people with thyroid cancer have been treated with larotrectinib so far, all of them had their tumour shrink in size.

Targeted therapy for RAI-refractory thyroid cancer

Researchers are looking at how targeted therapy drugs can help treat thyroid cancer that does not respond to RAI therapy (called RAI-refractory thyroid cancer). RAI is used to treat differentiated thyroid cancers. It cannot be used to treat undifferentiated thyroid cancers because these cancers do not take up iodine.

Donafenib is a tyrosine kinase inhibitor that may be an effective treatment for RAI-refractory thyroid cancer. A phase 3 study is comparing progression-free survival in people who are treated with donafenib and people who are treated with a placebo (, NCT03602495).

Apatinib is a vascular endothelial growth factor receptor (VEGFR) inhibitor, which is a type of drug that inhibits the growth of new blood vessels (angiogenesis). Researchers are studying apatinib to treat RAI-refractory thyroid cancer. A phase 3 study is comparing progression-free survival in people who are treated with apatinib and people who are treated with a placebo (, NCT03048877).

Cabozantinib (Cabometyx) is a tyrosine kinase inhibitor that may be used to treat RAI-refractory thyroid cancer that has progressed after treatment with a VEGFR inhibitor, such as lenvatinib (Lenvima) or sorafenib (Nexavar). In this phase 3 study, participants are treated with either cabozantinib or a placebo. The researchers will analyze the results to see if there is a difference in progression-free survival or the number of participants with a complete response or a partial response to treatment (, NCT03690388).

Targeted therapy for rare thyroid cancers

For rare cancers, it’s not possible to recruit enough participants to do a large randomized phase 3 trial. A new type of research study, called a basket trial, recruits a greater number of participants by using the molecular makeup of their tumours instead of what type of cancer they have. Since the cancers have the same genetic change, they are all treated with the same therapy. Researchers are using basket trials to study targeted therapy drugs for rare thyroid cancers.

Dabrafenib (Tafinlar) and trametinib (Mekinist) are effective in treating anaplastic thyroid cancer (ATC). Dabrafenib is a serine/threonine kinase inhibitor. It inhibits the protein made from the BRAF gene. Trametinib is a mitogen-activated protein kinase kinase (MAP2K) inhibitor. ATC is a rare and aggressive type of cancer with no effective treatment options and a poor prognosis. Up to half of ATC tumours have a specific mutation (change) in the BRAF gene called the BRAF V600 mutation. A phase 2 basket trial was done to study 9 different types of rare cancer, including ATC, that were known to have BRAF V600 mutations based on molecular profiling. Researchers found that 69% of participants with locally advanced or metastatic ATC responded to the dabrafenib plus trametinib combination treatment. Although the study included a small number of people, the results are noteworthy because there are no effective treatments for this rare cancer (Journal of Clinical Oncology, PMID 29072975).

LOXO-292 is an inhibitor of the RET receptor tyrosine kinase. Rearrangements of the gene that codes for the RET protein happen in several types of cancer. This can lead to RET fusion proteins that are always active and can promote cancer. Other mutations can also affect RET. A global phase 1 basket trial was done to study advanced papillary thyroid cancers with RET fusion proteins, advanced medullary thyroid cancers with other RET mutations and other types of cancer with these same mutations (ASCO, Abstract 102). Based on the positive results of this study, the US Food and Drug Administration (FDA) granted a faster type of drug development process (called Breakthrough Therapy designation) to LOXO-292 for the treatment of people with advanced RET fusion thyroid cancer.

Active surveillance

Active surveillance may be an alternative to thyroid surgery for people who are newly diagnosed with papillary thyroid cancer (PTC). A study is looking at using active surveillance in people with low-risk PTC, a small tumour and no lymph node or distant metastases. Participants will be given information about the disease prognosis, surgical treatment and active surveillance. They can then choose if they want active surveillance or thyroid surgery. Researchers will follow the study participants to track disease progression, treatment outcomes, psychosocial outcomes and quality of life (, NCT03271892).

Learn more about cancer research

Researchers continue to try to find out more about cancer. Clinical trials are research studies that test new ways to treat cancer. They also look at ways to prevent, find and manage cancer.

Clinical trials provide information about the safety and effectiveness of new approaches to see if they should become widely available. Most of the standard treatments for cancer were first shown to be effective through clinical trials.

Find out more about clinical trials.