Melanoma Skin Cancer Overview

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Treating Skin Cancer - Melanoma TOPICS

Targeted therapy for melanoma skin cancer

As doctors have found some of the gene changes that make melanoma cells different from normal cells, they have begun to develop drugs that attack these changes. These targeted drugs work in a different way from standard chemo drugs. Sometimes, they work when chemo doesn’t. They can also have less severe side effects. Doctors are still learning the best way to use these drugs to treat melanoma.

Drugs that target cells with changes in the BRAF gene

About half of all melanomas have changes (mutations) in the BRAF gene. These changes cause the gene to make more of the BRAF protein, which tells the melanoma cells to grow and divide quickly. Drugs that target this and related proteins can be used in patients with advanced melanoma who have a BRAF mutation. These drugs are not likely to work in patients whose melanomas have a normal BRAF gene.

For instance, the drugs vemurafenib (Zelboraf) and dabrafenib (Tafinlar) attack the BRAF protein directly. They are taken as pills. The most common side effects are joint pain, fatigue, hair loss, and rash. Less common but more serious side effects can also occur. Some people treated with these drugs get new skin cancers called squamous cell carcinomas, which can be treated by removing them.

The drug trametinib (Mekinist) attacks the MEK protein, which is related to the BRAF protein. It is also taken as a pill. Common side effects include rash, diarrhea, and swelling. Rare but serious side effects can also occur. This drug does not seem to shrink as many melanomas as the BRAF drugs, but it may work better when combined with dabrafenib. In fact, some side effects (such as getting other skin cancers) are actually less common with the combination.

Drugs that target cells with changes in the C-KIT gene

A small portion of melanomas have changes in a gene called C-KIT that help them grow. These gene changes are more common in melanomas that start in certain parts of the body, such as:

  • On the palms of the hands, soles of the feet, or under fingernails (known as acral melanomas)
  • Inside the mouth or other mucosal (wet) areas
  • In parts of the body that get a lot of sun

Some targeted drugs, such as imatinib (Gleevec) and nilotinib (Tasigna), can affect cells with changes in C-KIT. If you have a melanoma that started in one of these places, your doctor may test your melanoma cells for changes in the C-KIT gene, which might mean that one of these drugs could be helpful. Clinical trials are now studying this.

Drugs that target other changes are now being studied as well (see “What’s new in melanoma skin cancer research?”)


Last Medical Review: 02/19/2014
Last Revised: 02/19/2014