IN THE PIPELINE: Progress Seen In Battle Against Skin Cancer

16th Jun 2010 12:35

(This article was originally published Tuesday.) By Peter Loftus Of DOW JONES NEWSWIRES Recent research is raising hope that new drugs are on the horizon that could prolong the lives of people with the most serious form of skin cancer. The experimental drugs aren't cures, and the long-term prognosis for people with advanced melanoma may remain grim. In addition, some drugs may not make it to market if problems arise in further testing or regulatory reviews. But patients and doctors are nonetheless hopeful the recent advances set the stage for more significant gains down the road. "For the first time we have a bunch of drugs that are working," said Patrick Hwu, chairman of the melanoma medical oncology department at the University of Texas M.D. Anderson Cancer Center in Houston, which has participated in some of the clinical trials for melanoma drugs. The need for better treatments is great because current options are limited and the incidence of melanoma is on the rise. According to the American Cancer Society, about 68,130 new cases of melanoma are diagnosed annually in the U.S.; about 8,700 people die from the disease each year. Over-exposure to sunlight is one of the risk factors for melanoma. If the cancer is caught early, it can be removed surgically and survival rates are relatively high. But if it's diagnosed at a stage when the cancer has already begun to spread, or metastasize, the prognosis is poorer--average survival is less than a year, doctors say. Now, however, doctors, patients and drug companies are finally seeing signs of hope. In a study whose results were presented at a cancer meeting earlier this month, Bristol-Myers Squibb Co.'s (BMY) ipilimumab extended median overall survival to about 10 months in people with metastatic melanoma who had gotten previous treatments, versus nearly 6.5 months in people taking an experimental vaccine believed to have limited effectiveness. Experts say it was the first time any compound prolonged overall survival in metastatic melanoma in a late-stage, randomized clinical trial. Even current treatments haven't been definitively proven to significantly extend survival in clinical studies. Bristol-Myers plans to file for regulatory approval of ipilimumab this year. The company hasn't discussed pricing, but as a biologic drug, ipilimumab is likely to command a high price. Wall Street analysts have predicted its peak annual sales to be in the vicinity of $800 million. Ipilimumab is an immunotherapy, designed to stimulate the body's immune system to fight off cancer. The downside is that it makes the body vulnerable to immune-related side effects, like severe diarrhea and inflammatory conditions. The side effects can be managed by medicine, but patients need to be closely monitored, doctors say. Another limitation of ipilimumab is that a minority of patients in the study experienced significant tumor shrinkage, while the disease progressed in a majority of patients. Researchers want to figure out if there's a way to identify ahead of time which patients will respond best. Targeted therapies, or those that home in on a specific gene or molecule, represent another tack to fighting melanoma. About half of patients with metastatic melanoma have a mutation in a gene known as BRAF, which is believed to fuel tumor growth. Researchers think blocking the BRAF mutation could help treat the disease. Roche Holding AG (RHHBY), in partnership with privately held Plexxikon, has taken this approach with PLX4032, now in late-stage testing. An earlier stage study suggested the drug helped shrink tumors. Lynn Wilkinson, a 47-year-old corporate event planner in northern Virginia, is taking PLX4032 in a clinical trial. She had a recurrence of melanoma in 2006 and the cancer spread to her lymph nodes. She enrolled in a PLX4032 trial last fall after testing positive for the BRAF mutation. Successive scans since then have shown tumor shrinkage, she said; full results of the trial reflecting all patients aren't yet known. GlaxoSmithKline PLC (GSK) also is studying a BRAF inhibitor and another targeted therapy known as a MEK inhibitor. Eli Lilly & Co. (LLY), Delcath Systems Inc. (DCTH), Synta Pharmaceuticals Corp. (SNTA) and Celldex Therapeutics Inc. (CLDX) are among the companies studying various approaches to battling melanoma. (Peter Loftus covers the pharmaceutical industry for Dow Jones Newswires. He can be reached at 215-656-8289 or by email at [email protected].) (TALK BACK: We invite readers to send us comments on this or other financial news topics. Please email us at [email protected]. Readers should include their full names, work or home addresses and telephone numbers for verification purposes. We reserve the right to edit and publish your comments along with your name; we reserve the right not to publish reader comments.) (END) Dow Jones Newswires June 16, 2010 07:35 ET (11:35 GMT)