Treating Head and Neck Cancer: The Medications That Are Improving Outcomes

Knowing all your options will help you find the best treatment.

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Head and neck cancer is a collective term for several types of cancers that may start in the moist lining of the mouth, nose, and throat. Usually, though not always, the cancer affects the flat squamous cells that line these mucosal surfaces (this is called squamous cell carcinoma of the head and neck). Learn more about the different types of head and neck cancer here.

Because of this, treatment for head and neck cancer can vary as well. “There's no one way to treat these types of cancers,” says Mark Persky, MD, an otolaryngologist and head and neck surgeon at NYU Langone Health in New York City.

Cancer treatment is split into two categories: local and systemic.

  • Local treatments treat only the cancer (surgery + radiation) and often do not affect the rest of the body.

  • Systemic treatments are medications that go through whole body (chemo + targeted therapy) and can reach all cells in the body.

Treatment for head and neck cancer may include surgery, radiation, chemotherapy, targeted therapy, or immunotherapy. “Treatment of head and neck cancer can vary depending upon the site, the type of tumor, whether it's HPV-positive or not, and the staging of the tumor,” says Dr. Persky. Learn more about how head and neck cancers are diagnosed.

Some of the newer medications available are targeted therapies and immunotherapies.

Targeted therapy

Targeted therapy “targets” and kills the mutations in cancer cells that help them grow. Unlike chemotherapy, which aims to kill any rapidly dividing cell, target therapy affects mainly cancer cells and preserves normal cells.

Epidermal growth factor receptor (EGFR)  inhibitors are so far the only targeted agents that have been approved for head and neck cancer. Anti-EGFR drugs are a man-made version of an immune system protein, called a monoclonal antibody. These drugs target a protein on the surface of EGFR that helps cells grow and divide. By blocking EGFR, it helps slow or stop cell growth.


Immunotherapy is a type of therapy that stimulates the immune system to combat cancer cells “in a very effective way,” says Dr. Persky. Immunotherapies that treat head and neck cancer are called checkpoint inhibitors, more specifically, PD-1 inhibitors.

Here’s how checkpoint inhibitors work: The immune system uses a “checkpoint” system, in which proteins on an immune cell can be switched on or off to trigger—or inhibit—the immune response. Cancer cells can manipulate this checkpoint system to turn off the immune response, allowing rapid and uninhibited cancer growth.

That’s where checkpoint inhibitors come in. They block those proteins on the immune cell, signaling to the immune system to recognize and attack the cancer cells. PD-1 inhibitors target a protein on the immune cells called PD-1. Learn more about how immunotherapy treats cancer.

Clinical trials

Another option patients have is to participate in a clinical trial. Clinical trials are research studies that test new drugs or other treatments in people. Clinical trials are one way to get state-of-the art cancer treatment.

“There are many, many institutional studies, multi-institutional studies, that are now focusing on improving the cure for head and neck cancer,” says Dr. Persky.

Ask your doctor if there are any clinical trials that may be of benefit to you.

“Every patient should be aware of the options of treatment,” says Dr. Persky. “The doctor hopefully will present ... the best option of treatment, but the patient has the final decision.”

Remember: You and your doctor are a team. It’s important to be open with your doctor and bring up any questions or concerns you may have when considering different treatment options.

“A good doctor will always be there for the patient throughout the evaluation process, the treatment process, and the follow-up process,” says Dr. Persky. “That's important for the patient to realize and to expect from any physician treating him, no matter whether it's head and neck or any other type of tumor.”