Emerging Research and Future Treatment Directions

Reviewed by: HU Medical Review Board | Last reviewed: February, 2022.

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 certain forms of blood and marrow cancer. In certain forms of melanoma, it works by blocking a cell receptor called c-KIT. c-KIT mutations are found in approximately 6% of melanoma cases.5 The mutation causes the c-KIT receptor to be constantly “on.”

Studies continue to look at medications such as nilotinib that target the c-KIT gene mutation to determine their safety and effectiveness for this type of 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 Certain treatments are available, and others continue to be studied, that treat melanoma by boosting your own immune system.

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 relatively early stages of study, and research is ongoing.

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. Clinical trial: NCT02557321 is an ongoing study treating metastatic melanoma.

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.

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