05/26/2015 03:36 pm ET Updated May 26, 2016

Promising New Treatments for Melanoma

Shutterstock / Rido

Desiree Ratner, MD
Director, Comprehensive Skin Cancer Program
Mount Sinai Beth Israel
Director, Dermatologic Surgery
Mount Sinai Beth Israel, Mount Sinai St. Luke's, and Mount Sinai Roosevelt

Promising New Treatments for Melanoma

It can be frightening to learn you have melanoma, the least common but most dangerous form of skin cancer. When caught in its early stages, melanoma is almost always curable with surgery, but when it metastasizes -- that is, spreads to other areas of your body -- it is notoriously difficult to treat.

In the past, patients diagnosed with the most advanced stages of melanoma usually had only a few months to live. However, metastatic melanoma is no longer the automatic death sentence that it once was. Chemotherapy, once the standard (but largely ineffective) treatment for advanced melanoma, is giving way to new, more successful therapies that are prolonging survival and improving patients' quality of life.

The promising new drugs approved by the U.S. Food and Drug Administration (FDA) in just the last few years fall into two classes: targeted molecular therapy and immunotherapy. The side effects of both tend to be much more tolerable than those of chemotherapy.

Targeted Molecular Therapy

About half of patients with metastatic melanoma have tumors whose cells contain a genetic mutation, most commonly in a gene called BRAF. As its name suggests, targeted therapy attacks and kills those particular mutated cells.

A recent study of one of the newest targeted drugs, nivolumab, found that 32 percent of patients responded to it and, in a third of those patients, the effects lasted six months or longer. This is good news, considering chemotherapy's traditional response rate of only 5 to 20 percent. In fact, the results represented such a significant improvement over previously available drugs that the FDA approved nivolumab three months ahead of schedule, in December 2014.

When targeted drugs work, the tumors shrink for a time, allowing patients to live normally for a while, rather than feeling continuously ill. Unfortunately, this type of drug eventually stops working for many patients as their genes undergo further mutations. To address this issue, researchers are investigating combinations of targeted therapies.

The New England Journal of Medicine, for example, recently published promising results of a study in which patients were treated with a combination of two targeted drugs. Those who received the double therapy survived for a median of 11.4 months without progression of the disease, versus 7.3 months for patients taking one drug alone.

The bottom line is that even though patients who respond to targeted drugs may still die of melanoma, they are surviving longer and with a better quality of life than ever before.


Immunotherapy strengthens the immune system by supporting activation of the body's cancer-fighting T-cells. For example, the FDA-approved drug Yervoy (ipilimumab) blocks a molecule called CTLA-4, which normally keeps immune cells in check. Blocking CTLA-4 "takes the brakes off" of your immune system, allowing T-cells to proliferate and launch a stronger attack on the harmful melanoma cells.

A 2013 analysis of data from 1,861 patients treated with Yervoy revealed exciting news: the patients' median overall survival was 11.4 months; 22 percent of them survived at least three years; and 17 percent were still alive after seven years, with no deaths due to melanoma after that. Such long-lasting responses to anti-cancer drugs in people with metastatic melanoma were previously unheard of.

Individual Factors

Despite these recent breakthroughs, there is no "one size fits all" treatment for patients with metastatic melanoma. Your medical oncologist (a doctor who specializes in treating cancer with medications) should consider the individual factors of your case to determine which drugs make the most sense for you. And that's the exciting part: these new therapies are giving real options to a set of patients who previously had none.

Even so, nobody wants to get metastatic melanoma. Checking your skin regularly for new or changing growths is crucial because, when caught early enough, melanoma can be stopped in its tracks. To learn what to look for, see the American Academy of Dermatology's online reference for identifying the "ABCDE" warning signs of melanoma, and this guide to melanoma from the American Cancer Society.