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Immunotherapy and Targeted Drugs for Eye Cancer

Most eye cancers are melanomas (also known as ocular melanomas or intraocular melanomas).

While standard chemotherapy is often not very helpful for eye melanoma, newer types of cancer drugs can be more effective. These newer drugs generally fall into 2 groups:

  • Immunotherapy drugs work by helping the body’s own immune system recognize and attack cancer cells.
  • Targeted drugs target parts of melanoma cells that make them different from normal cells.

T cell engager (TCE) for eye cancer

Tebentafusp (Kimmtrak) is a type of immunotherapy known as a T cell engager (TCE). Once it’s injected into the body, one part of the drug attaches to immune cells called T cells, while another part attaches to the gp100 protein on melanoma cells. This brings the two together, which helps the immune system attack the cancer cells.

Tebentafusp can be used to treat uveal melanoma that has spread or that can’t be treated with surgery, in people whose cells have a certain HLA tissue type (known as HLA-A*02:01). Before you get this treatment, your doctor will test you to see if you have this tissue type.

This drug is given into a vein (IV), typically once a week.

Common side effects of tebentafusp include:

  • Itching, rash, redness, and swelling of the skin
  • Fever
  • Feeling very tired
  • Headache
  • Nausea and vomiting
  • Belly pain
  • Swelling
  • Low blood pressure
  • Abnormal liver blood tests

Cytokine release syndrome (CRS) is a serious side effect that can occur when T cells in the body release chemicals (cytokines) that ramp up the immune system. This happens most often within the first day after the infusion, and it can sometimes even be life-threatening. Symptoms can include:

  • High fever and chills
  • Severe nausea and vomiting
  • Trouble breathing
  • Feeling very tired
  • Feeling dizzy or lightheaded
  • Headache

Your cancer care team will watch you closely for possible signs of CRS, especially during and after the first few infusions. Contact them right away if you have any symptoms that might be from CRS.

Immune checkpoint inhibitors for eye cancer

Some immune cells have “checkpoint” proteins on them, which act like switches that need to be turned on (or off) to start an immune response. Melanoma cells sometimes use these checkpoints to avoid being attacked by the immune system.

Immunotherapy drugs called checkpoint inhibitors target these checkpoint proteins to help restore the immune response against the cancer cells. Examples of immune checkpoint inhibitors include:

  • PD-1 inhibitors such as pembrolizumab (Keytruda) and nivolumab (Opdivo)
  • CTLA-4 inhibitors such as ipilimumab (Yervoy)

These drugs aren’t as effective in treating uveal melanoma as they are for melanomas of the skin, but they might be helpful for some people. Combining nivolumab with ipilimumab is more likely to be helpful than nivolumab alone, but it also tends to cause more side effects.

For more on these medicines, see Immunotherapy for Melanoma Skin Cancer.

Targeted drugs for eye cancer

About half of all skin melanomas have a change (mutation) in the BRAF gene. Several drugs that target this gene change are now available. (See Targeted Therapy for Melanoma Skin Cancer.)

This mutation isn't common in uveal melanomas, but when it’s present, these drugs might be helpful.

Drugs targeting other gene changes are also being studied.

For more on some of these newer drugs, see What's New in Eye Cancer Research?

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Developed by the 黑料大湿Posts Cancer Society medical and editorial content team with medical review and contribution by the 黑料大湿Posts Society of Clinical Oncology (ASCO).

Carvajal RD, Harbour JW. Metastatic uveal melanoma. UpToDate. 2025. Accessed at https://www.uptodate.com/contents/metastatic-uveal-melanoma on April 10, 2025.

Houghton O, Gordon K. Chapter 64: Ocular Tumors. In: Niederhuber JE, Armitage JO, Doroshow JH, Kastan MB, Tepper JE, eds. Abeloff’s Clinical Oncology. 6th ed. Philadelphia, Pa. Elsevier: 2020.

National Cancer Institute. Intraocular (Uveal) Melanoma Treatment (PDQ?)–Health Professional Version. 2024. Accessed at https://www.cancer.gov/types/eye/hp/intraocular-melanoma-treatment-pdq on April 10, 2025.

National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology: Melanoma: Uveal. V.1.2025. Accessed at https://www.nccn.org on April 10, 2025.

Last Revised: May 5, 2025

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