Which cancers can be treated with immune checkpoint inhibitors?

Updated June 28, 2024

Immunotherapy is a type of cancer treatment that does not target the cancer itself, unlike traditional treatments. Instead, immunotherapy trains the immune system to fight cancer. Several types of immunotherapy are now approved to treat a wide variety of cancer types.

“At times, our immune system can become tolerant to cancer for various reasons,” says Aung Naing, M.D., who leads an immunotherapy working group in MD Anderson’s Phase I clinical trials program. “With immunotherapy, we are trying to harness or reinvigorate the immune system so that it recognizes and attacks the cancer instead of being tolerant. These agents have transformed cancer care since the approval of the first immune checkpoint inhibitor for metastatic melanoma in 2011.”

What are immune checkpoint inhibitors?

Immune checkpoint inhibitors work by blocking checkpoint proteins – including CTLA-4, PD-L1 and PD-1 – on the surface of T cells. These proteins normally prevent T cells from attacking healthy cells, but treatment with checkpoint inhibitors unleashes the T cells to eliminate cancer cells more effectively. Approved checkpoint inhibitors include:

• anti-CTLA-4 therapies ipilimumab and tremelimumab
• anti-PD-1 therapies cemiplimab, dostarlimab, nivolumab, pembrolizumab, retifanlimab-dlwr and tislelizumab
• anti-PD-L1 therapies atezolizumab, avelumab and durvalumab
• anti-LAG-3 therapy relatlimab

Immune checkpoint inhibitors are one of several types of immunotherapy. Other forms include:

• Adoptive cellular therapies such as chimeric antigen receptor (CAR) T cell therapies, are immune cells taken from a patient or healthy donor and modified to boost their ability to eliminate cancer.
• Targeted antibodies are treatments that mimic naturally occurring antibodies from our immune system.
• Cancer vaccines train the body to recognize specific features of cancer and stimulate an immune response.
• Oncolytic viruses use engineered viruses to infect and destroy cancer cells.
• Immune system modulators, or cytokine therapies, use immune signaling compounds to stimulate immune activity.

Immune checkpoint inhibitors treat many types of cancer

Currently, the Food and Drug Administration (FDA) has approved immunotherapies to treat certain patients with the following cancer types. FDA approval is a carefully regulated process to ensure that medicines are safe and effective for patients.

• Alveolar soft part sarcoma
• Basal cell skin cancer
• B-cell lymphoma
• Bile duct cancer
  
• Bladder cancer
• Cervical cancer 
• Classical Hodgkin lymphoma
• Colorectal cancer 
  
• Cutaneous squamous cell cancer
• Endometrial cancer
• Esophageal cancer
• Head and neck squamous cell cancers
• Kidney cancer
• Liver cancer 
• Lung cancer
• Melanoma
• Merkel cell cancer
• Mesothelioma
• Stomach cancer
• Triple-negative breast cancer 
  

Additionally, some patients with advanced solid tumors and specific tumor characteristics, such as high microsatellite instability (MSI-H), mismatch repair deficiencies (dMMR), or high tumor mutational burden (TMB), may be eligible for treatment with certain immune checkpoint inhibitors, regardless of cancer type.

Immune checkpoint inhibitors may be used alone or in combination with other treatments, such as chemotherapy or targeted therapies. In many cases, patients must meet certain criteria to be eligible for these therapies. Keep in mind that the information shared here should not be considered medical advice.

"Immunotherapies are now not only being used to treat a large number of different cancers, but importantly, in some cancers like melanoma, they are now being used in earlier stages, either with a neoadjuvant approach (before surgery) or in the adjuvant setting. In both cases, the goal is to decrease the risk of cancer recurring at a later point. Other indications now include brain metastases," says melanoma medical oncologist Isabella Glitza Oliva, M.D., Ph.D. "Our research is continuing to push our understanding of why these successful therapies do not work in some patients and to enable us to find ways to help more patients."

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