Immunotherapy involves taking medications that stimulate your immune system to destroy cancer cells. It can sometimes treat oral cancer that can’t be removed surgically or has returned.

The American Cancer Society estimates that 54,540 people will receive an oral cancer diagnosis in 2023. Oral cancers start inside your mouth or throat.

The main treatments for oral cancer are:

Immunotherapy is a rapidly developing type of cancer treatment. The Food and Drug Administration (FDA) has approved two immunotherapy drugs to treat oral cancer — pembrolizumab and nivolumab. Researchers continue to examine how best to use these drugs to treat oral cancer and whether new immunotherapy drugs might also be effective.

Read on to learn more about how immunotherapy can help treat oral cancer.

Doctors can usually treat oral cancer contained in your mouth or surrounding area with a combination of surgery and radiation therapy.

Immunotherapy, with or without chemotherapy, is a potential treatment for cancers that have spread too far for surgery and radiation therapy to be effective or cancers that have returned after treatment. Your doctor may also recommend immunotherapy if chemotherapy drugs stop working.

Pembrolizumab and nivolumab

Pembrolizumab (Keytruda) and nivolumab (Opdivo) are the most studied immunotherapy drugs for oral cancer. They fall into a class of immunotherapy drugs called immune checkpoint inhibitors.

Your T cells are white blood cells that attack cancer cells and foreign cells. They contain “checkpoint” proteins on their surface that keep them from being too aggressive. Some cancer cells produce proteins that bind to these checkpoint proteins to turn them off, allowing the cancer cells to avoid detection.

Immune checkpoint inhibitors block this binding process so that your T cells stay activated.

More specifically, pembrolizumab and nivolumab are programmed cell death protein 1 (PD-1) inhibitors. PD-1 is a checkpoint protein on the surface of T-cells. Some cancer cells produce a protein called PD-L1, which can bind to PD-1. Pembrolizumab and nivolumab are able to prevent this binding.

Other immunotherapy drugs

Many other immunotherapy drugs are under investigation for treating oral cancer. These drugs include:

  • cemiplimab
  • bevacizumab
  • anti-EGFR monoclonal antibody
  • cetuximab
  • sintilimab
  • toripalimab
  • anti-OX40 antibody

How effective is immunotherapy at treating oral cancer?

In a phase 3 clinical trial of 882 people with incurable, recurrent, or metastatic head and neck cancer, researchers gave participants one of:

  • pembrolizumab alone
  • pembrolizumab with chemotherapy
  • cetuximab with chemotherapy

The researchers concluded that pembrolizumab plus chemotherapy is an appropriate first-line treatment for recurrent or metastatic head and neck cancer. Pembrolizumab by itself was effective for treating PD-L1-positive recurrent or metastatic head and neck cancer.

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Side effects of PD1 inhibitors include:

Rare but serious side effects include:

  • infusion reactions, which can cause:
    • trouble breathing
    • facial flushing
    • chills
    • fever
    • itchy skin
    • dizziness
    • wheezing
  • autoimmune reactions, which can cause life threatening problems with your liver, lungs, or other organs

You’ll receive immunotherapy at a doctor’s office, clinic, or hospital.

Healthcare professionals administer nivolumab through an IV (intravenous) infusion every 2–4 weeks.

They administer pembrolizumab with or without chemotherapy as an IV infusion every 3–6 weeks.

You’ll likely be able to go home shortly after your appointment unless you develop a side effect that requires further treatment.

Other treatment options for oral cancer include:

  • Surgery: Surgery involves removing the tumor and tissue around it. Your doctor may combine this with radiation therapy.
  • Radiation therapy: Radiation therapy uses high-energy waves to destroy cancer cells.
  • Chemotherapy: These medications destroy cancer cells and other cells in your body that replicate quickly.
  • Targeted therapy: Targeted medications specifically attack cancer cells. They tend to cause fewer side effects than chemotherapy.

Here are some frequently asked questions people have about immunotherapy.

What is the cure rate for most mouth cancers?

Your outlook with oral cancer depends on where the cancer develops. The 5-year relative survival for lip cancer is 91%, but only 52% if the cancer starts in your throat.

The 5-year relative survival rate measures how many people with the cancer are alive 5 years later compared to people without the cancer.

Which cancers are most successfully treated with immunotherapy?

Immunotherapy is a relatively new treatment option still under investigation for treating many different types of cancer. Some of the most studied cancers include:

Is pembrolizumab (Keytruda) FDA-approved to treat oral cancer?

Pembrolizumab gained FDA approval in June 2019 as a first-line treatment for people with head and neck squamous cell carcinoma that can’t be removed with surgery or has spread to distant tissues.

Doctors use immunotherapy to treat oral cancers that they can’t surgically remove or that have spread to other locations or returned after treatment. Researchers are still investigating how best to use immunotherapy to treat oral cancer.

Your healthcare team can help you decide if you may benefit from immunotherapy. They can also help you understand the potential benefits and risks of undergoing immunotherapy.