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Emerging Research and Future Treatment Directions

Survival rates for advanced skin cancer are low. Chemotherapy used to be the main treatment for widespread melanoma, but it only worked for 10% to 15% of patients.1

Innovative treatments are needed. Cancer develops when cell growth becomes uncontrolled. Promising medications block the signals for cell growth and survival. Other medications help your immune system to destroy cancer cells.

Clinical trials

The US Food and Drug Administration (FDA) is a government agency. The FDA makes sure medications are effective and safe. A drug is tested in clinical trials before the FDA approves it. Clinical trials take place in steps, called “phases.”2

  • Phase I: These trials include a small group of people. One goal is to find the safe dose range. Another goal is to figure out what side effects the medication causes.
  • Phase II: These trials are done in larger groups of people. The goals are to see if the medication works and if it is safe.
  • Phase III: These trials are done with large groups of people. One goal is to confirm that the drug works. Another goal is to see how frequently side effects occur. These trials are usually the last step before the FDA decides whether to approve the medication.

Targeted therapy

Cancer cells often have mutations that make them different from normal cells. Targeted therapies interfere with these mutated cell features. Usually, the medications block processes that help a cancer cell grow and survive.3,4 Some targeted therapies are already approved. More are being studied.

Tasigna® (nilotinib)

Tasigna is approved to treat blood and marrow cancer. It works by blocking a cell receptor called c-KIT. c-KIT mutations are found in 20% of mucosal melanoma and 15% of acral lentiginous melanoma.5 About 2% of melanomas on sun damaged skin have c-KIT mutations.5 The mutation causes the c-KIT receptor to be constantly “on.”

Tasigna was studied for melanoma treatment in a Phase II clinical trial.6 The 19 study participants had stage III or IV melanoma. Eight participants had brain metastases, which is melanoma that has spread to the brain. Nearly all had used other medications before the trial. While taking Tasigna, the cancer did not progress for about 3 months. The median overall survival was 14 months for patients without brain metastases. Median overall survival was 4 months in patients with brain metastases (Median overall survival is the length of time from treatment that half the study population is still alive). The most common side effects were fatigue, muscle and joint pain, and gastrointestinal discomfort. There will be more studies of Tasigna in people with melanoma.


There is a well-known link between the immune system and melanoma. For example, people who take medications that weaken their immune system are at high risk of melanoma. Melanoma cells are able to hide from the body’s immune defenses.7 It may be possible to treat melanoma by boosting your own immune system.

Keytruda® (pembrolizumab) and epacadostat

Keytruda is a drug approved to treat melanoma. Keytruda is an immune checkpoint inhibitor. This medication prevents melanoma cells from hiding from the immune system’s T-cells. Keytruda is being studied together with epacadostat, a new medication. Epacadostat seems to work by activating the immune system. A phase I study showed promising results with this combination. The study included 19 patients with advanced or metastatic melanoma.8 Four patients had a complete response. A complete response means that all signs of cancer disappeared. Another 7 patients had a partial response. A partial response is a decrease in the size of the tumor or the extent of the cancer. A phase III trial is currently recruiting participants (Clinical Trial NCT02752074).

Adoptive T-cell transfer therapy

T-cells are immune cells that recognize and attack invaders. Patients live longer if T-cells naturally infiltrate (sneak into) melanoma tumors.7 Adoptive T-cell therapy is based on this idea. T-cells that have entered a melanoma tumor are collected from a patient. More T-cells are grown in a laboratory. The T-cells are given back to the patient. The idea is that the T-cells will fight the cancer cells. This treatment is still in early stages of study. Several Phase I and II studies are recruiting patients.

Intralesional therapy: PV-10

PV-10 (Rose Bengal). For this treatment, medication is injected into the tumor. The term for this is intralesional therapy. Rose Bengal is a dye used for medical purposes. Researchers noticed that the dye builds up in the tumor lysosomes. (Lysosomes are the cell’s “garbage disposal.” It is the part of the cell that digests cellular waste.) As the dye builds up, the tumor cells quickly start to break down.9 PV-10 is a formulation of Rose Bengal made for people with melanoma. In a phase II study, 26% of patients with advanced or metastatic melanoma had a complete response. The side effects were mild to moderate.9 Most related to the injection site. Examples include pain, swelling, or appearance of fluid-filled blisters at the injection site. A phase III trial of PV-10 for recurrent melanoma is recruiting (Clinical Trial NCT02288897).

Participating in a clinical trial

Many researchers are working on finding new and better treatments for skin cancer. Clinical trials provide important information about these treatments. By participating in a clinical trial, you may get access to new treatments. You also help future patients, who will benefit from better treatments. However, clinical trials are not right for everyone. You must weigh the potential benefits with the potential risks.

The National Cancer Institute has a list of questions for patients to ask before participating in a study.10 These questions include:

  • What is the purpose of this trial?
  • Why do the researchers think that this treatment is better than the current treatments?
  • What kinds of tests and treatments are involved?
  • How will the doctor know if the treatment is working?
  • What are the possible risks and side effects?
  • What are the possible benefits?
  • Will I have to pay for any of the treatments or tests?

If you are considering a clinical trial, talk with your doctor.

Written by: Sarah O'Brien | Last reviewed: December 2018.
  1. Johnson DB, Sosman JA. Therapeutic advances and treatment options in metastatic melanoma. JAMA Oncol. 2015;1:380-386.
  2. National Library of Medicine. FAQs. Clinical trial phases. Accessed April 15, 2017 at:
  3. National Cancer Institute. Targeted cancer therapies. Accessed January 18, 2017 at:
  4. American Cancer Society. What is Targeted cancer therapy? Accessed January 18, 2017 at:
  5. Lovly C, Pao W, Sosman J. 2016. Molecular profiling of melanoma. My Cancer Genome (Updated January 26).
  6. Carvajal RD, Lawrence DP, Weber JS, et al. Phase II study of nilotinib in melanoma harboring KIT alterations following progression to prior KIT inhibition. Clin Cancer Res. 2015;21:2289-2296.
  7. Tang T, Eldabaje R, Yang L. Current status of biological therapies for the treatment of metastatic melanoma. Anticancer Res. 2016;36:3229-3241.
  8. Gangadhar TC, Hamid O, Smith DC, et al. Epacadostat plus pembrolizumab in patients with advanced melanoma and select solid tumors: Updated phase 1 results from ECHO-202/KEYNOTE-037. Abstract 1110PD. Presented at ESMO 2016 Congress. Available at:
  9. Thompson JF, Agarwala SS, Smithers BM, et al. Phase 2 study of intralesional PV-10 in refractory metastatic melanoma. Ann Surg Oncol. 2015;22:2135-2142.
  10. National Cancer Institute. Questions to ask your doctor about treatment clinical trials. Accessed February 26, 2017 at: