Breakthroughs in Treating Advanced Melanoma

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Melanoma treatment is very successful when doctors find the cancer before it starts growing in other areas of the body. Advanced melanoma that has spread is harder to treat. For many years, surgery, X-ray treatment, and drugs that kill cancer cells were the only treatments available for advanced melanoma. None of them worked very well.

Immunotherapy (using injectable drugs like pembrolizumab, nivolumab, and ipilimumab) is a newer treatment. It stimulates the immune system to fight cancer cells. This treatment proved to be more effective. Although immunotherapy was more effective that mainstay treatments, people with advanced melanoma needed a breakthrough.

The good news is that could change with the newest treatment option: targeted therapy.

What Is Targeted Therapy?

Researchers found that some people with melanoma have abnormal genes that trigger the growth of melanoma cancer cells. In fact, about half of all melanomas show a gene mutation that causes the tumor to grow. These genes include BRAF, MEK and C-KIT. Doctors can now do a special type of biopsy to tell whether a gene is causing a melanoma tumor to grow.

If you have these genes, there are new drugs that target them. The U.S. Food and Drug Administration (FDA) approved these drugs as treatment for advanced melanoma. The drugs attack the genes and stop the tumor from growing. It's important to note, they are used only for melanomas that cannot be removed by surgery.

Targeted Therapy Drugs


These drugs have a big advantage over chemotherapy and radiation therapy because they target only cancer cells. Chemo and radiation kill normal cells along with cancer cells. Targeted therapy may also have fewer side effects because it spares normal cells. 

Current targeted therapy drug options include:

  • Vemurafenib. The FDA approved this drug for targeted therapy in 2011. It is approved for people who are positive for the BRAF gene. Several studies show that it helps extend survival. Vemurafenib is a pill you take twice a day. Possible side effects include fatigue, rash, sun sensitivity, and an increased risk of other types of skin cancers.
  • Dabrafenib. The FDA approved this drug in 2013. It also targets BRAF. You take this drug as a capsule twice a day. It appears to be as effective as vemurafenib. Side effects can include flu-like symptoms and thickening of the skin. It is less likely than vemurafenib to cause other skin cancers.
  • Trametinib. The FDA approved this drug in 2013. It blocks the MEK gene. You take this pill once a day. It may work best in combination with a BRAF inhibitor. Side effects include rash and diarrhea. Heart, lung and vision problems are rare but serious possible complications.
  • Cobimetinib.The FDA approved this drug in 2015. It also blocks the MEK gene. You take this pill once a day in combination with Vemurafenib. Side effects include skin photosensitivity, decreased heart function, metabolic imbalances, and vision problems.

What the Future Holds

There's still a lot to learn about the best ways to use targeted therapies for advanced melanoma. For instance, doctors and researchers are now doing clinical trials on drugs that target the C-KIT gene. It's also likely that researchers will discover more genes to target and develop new drugs to use in different combinations.

Talk with your doctor about the best options for you. If you have advanced melanoma, a clinical trial might be the best place to get access to a new treatment. Medications are free, and you will get to work with doctors on the cutting edge of melanoma research. You may not only benefit from a breakthrough treatment, but you could also be a part of developing new treatments for others who follow.

Key Takeaways

  • New genes and medicines for advanced melanoma are being actively investigated.

  • Researchers discovered abnormal genes that encourage melanoma growth.

  • Medications that attack these specific genes and slow down growth are now available. 

  • A clinical trial can be a good option for getting the latest advanced melanoma treatment. 

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Medical Reviewer: William C. Lloyd III, MD, FACS
Last Review Date: 2018 Oct 5

  1. New therapies giving hope for patients with advanced melanoma. American Academy of Dermatology.

  2. Treatment of melanoma skin cancer, by stage. American Cancer Society.

  3. Targeted therapy for melanoma skin cancer. American Cancer Society.

  4. Targeted Therapies. Melanoma Institute Australia.

  5. Treating Melanoma Through Clinical Trials. Melanoma Research Foundation.

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