August 26th, 2010
04:55 PM ET

New therapy promising against metastatic melanoma

Approximately 60,000  new cases of melanoma, will be diagnosed in the U.S. in 2010. The disease, which is the most serious type of skin cancer, accounts for about 10,000  deaths each year. That's according to the American Cancer Society. And although melanoma is treatable when caught early, after it spreads to other parts of the body, a patient is usually given a grim diagnosis of less than a year to live.

Now a  study in the New England Journal of Medicine reports a new drug treatment may help some of these patients live longer.

While looking at genes in cancer patients back in 2002 , scientists found that in 40 to 60 percent of melanoma cases and 7 to 8 percent of all cancers, a protein mutation known as BRAF was present. With this mutation,  the protein becomes  overreactive, causing cancer cells to grow. When researchers targeted this BRAF mutation with a drug known as PLX4032, it inhibited the mutation, shrinking tumors and slowing the progression of the disease in 81 percent of those treated.

According to study investigators, the two FDA-approved drugs – interleukin-2 and dacarbazine – produce a response in only 10 to 20 percent of patients. The goal with this PLX4032 was to find a more effective treatment for many patients who do not benefit from these already approved treatments.

"Metastatic melanoma has a devastating prognosis and is one of the top causes of cancer death in young patients," says Keith Flaherty, M.D., director of Developmental Therapeutics at the Massachusetts General Hospital Cancer Center, lead author of the NEJM article. "Until now, available therapies were few and unreliable, so these findings can really change the outlook for patients whose tumors are fueled by this mutation."

The phase 1 clinical trial,  which had 87 patients, was designed to test effective doses.

The drug, which was taken orally, seemed to carry no serious side effects in lower doses. Some patients developed more serious side effects, such a rashes, as the dose became higher. But for many, according to study authors, the side effects did not outweigh the benefits.

"One of the things that make these results truly remarkable is that this drug works so reliably," Flaherty explains. "And patients who have been experiencing symptoms like pain and fatigue begin to feel better within a week of starting treatment, giving them a much better quality of life."

Two additional Mass General-based clinical trials are now under way – a phase 2 study in patients unsuccessfully treated with the FDA-approved drugs and a larger phase 3 study that compares PLX4032 with dacarbazine in patients with  newly diagnosed disease.

soundoff (9 Responses)
  1. Peter Conway

    Thank you so much for this up lifting story of the new study on melanoma and the drug PLX4032.It gives me hope ,having already survived a bout with a rare form of testicular cancer called Germ cell in which I received chemo 24/7 for 6-8 months in '05.It was in my upper chest ,wrapped around and attaching to my heart,lung and tractia.I was cancer free till I recently got a call from my Derm Doctor saying I might have basile cell on my arms from years of welding.Well ,thanks again for giving me and others info and hope for defeating this disease and God bless Mass.Gen.Hospital for their breakthru.

    August 27, 2010 at 08:41 | Report abuse | Reply
    • Jean

      Peter, I have had melamona surgery three times on my face with the last time being a more serious form than the previous two times. The last surgery was Feb 2004 and I have had none since, thank goodness. I refused chemo because after research I found that the chemo available would only prolong the time to a relapse but would not extend my life if there was a relapse. There are not a lot of options for treating melamona. I was told that for what I had if there was one recurrence that 7 out of 10 would be dead in 10 years. It has been 6 and 1/2 years now and I plan to see that doctor at year 10 and say ha ha told you so that I would still be here. And I do not leave that in his hands to keep me here. For any cancer, I recommend reading a lot on alternative medicine and particularly on ph balance and vitamin D3. I could go on and on because I really believe in thinking of it as saving myself and not looking to doctors to do that, but enough said. Please if you don't do anything else take the D3 and vitamin C and B complex. I wish you well and hope you stay healthy for a very long time. I believe it is possible.

      August 27, 2010 at 10:18 | Report abuse |
  2. Chris F

    It's nice to finally see an article on melanoma that is actually informative. I have been getting extremely tired of every melanoma article basically yelling at me to wear chemical sunscreens. Thank you for this informative and very uplifting article. I hope the clinical trials go through without a hitch and this drug gets approved. From what I read it sounds like a great improvement on its predecesors.

    August 27, 2010 at 09:43 | Report abuse | Reply
  3. Denise M

    What an uplifting article. It is good to know that there are treatments out there that don't consist of just a knife, Efudex orTazarac and staying out of the sun. I had a mole taken off in 2004. They took a 1 and 1/2" circle about 1/2" thick from the back of my right arm. They didn't think that it was anything so they weren't real concerned about clean margins. It ended up being Melanoma and without clean margins....forcing a second surgery in the same area within 7 days. Since then I can't tell you how many times I have been cut into to remove basel and squamous cell carcinoma spots, not to mention all of the topical treatments that make me look like raw meat. I wear sunblock even in the house because I nevr know when I will be going outside. If this could be related in any way to the protein mutation, it would be nice to be able to get some help outside of cutting out sections of my skin every 90-180 days.

    August 27, 2010 at 15:01 | Report abuse | Reply
  4. Debbie

    My husband was never given any time line, when I ask was made to feel horrible for questioning. He was diagnosed and was gone six (6) WEEKS later. Do NOT ignore your body.

    August 27, 2010 at 15:21 | Report abuse | Reply
    • Margaret Earnest

      Debbie, you don't say whether your husband had had previous bouts of Melanoma, I am curious if the final diag was the first or if he had been treated previously? My husband's family has a tendency toward moles and he always had had one in particular on his back just abit different looking than the rest. This mole, in just a very short while had changed shape and color and he went to have it ckd. Dr assured him it was benign, I wasn't satisfied and asked if he'd told dr his family had alot of cancer, a biopsy was done and he was in surgery within 24 hrs! This was a huge surgery, went right to the bone! Had another bout 2 yrs later, another surgery not quite so extensive. Everything was fine for 13 yrs, had blood tests, chest x-rays etc until 2005 and was gone 9 wks after diag of stage 4! I can only pray researchers have found something to give patients hope for, if not a cure at least a control of this cancer that spreads like wildfire!

      August 28, 2010 at 21:35 | Report abuse |
  5. Mr. Davidson

    Problem is you can't talk to people its like talking to a brick wall.They want to burn or tan and thats it,they ignore moles like they're beauty marks and burn all summer long .In winter they hit the taning beds and look at you like you're some kind of an idiot when you say something ,and the women are far worse than the men ,the guys ,they're scared , hats all the time now,hahaha.

    November 20, 2010 at 11:24 | Report abuse | Reply
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