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Health Information Under the Microscope: Testing a Targeted Therapy for Melanoma

Rush participates in an international clinical trial for a promising new drug More than a century ago, chemotherapy pioneer Paul Ehrlich, MD, theorized about creating "magic bullets" — drugs that went directly to specific cellular targets — to fight disease. Ehrlich's vision is now being realized in cancer treatment with the development of targeted therapies, including a promising new drug for melanoma.

In November, Rush University Medical Center began enrolling patients in an international clinical trial of nilotnib, a novel drug to treat advanced melanomas arising from the mucosa (the moist tissue that lines some organs and body cavities throughout the body, including the nose, mouth, lungs and digestive tract) and foot.

A Stealthy Saboteur

Historically, treatments for metastatic melanoma haven't been shown to improve survival rates. And those therapies that have had modest success are highly toxic because they attack both malignant and healthy cells, so not all patients can tolerate them.

Rather than blocking or killing all rapidly dividing cells, whether malignant or not, nilotnib is one of a new class of targeted agents designed to zero in on abnormal molecules characteristic of individual cancers — in this case, the c-kit protein.

Howard Kaufman, MD, director of the Rush University Cancer Center and principal investigator of the study, says that this kind of targeted therapy holds out hope of transforming cancer from a lethal disease into a chronic, but manageable disease.

As a small molecule, nilotnib is able to slip across the cell's membrane and into the machinery inside. There it targets, and turns off, the abnormal c-kit protein, created by a mutated c-kit gene, shutting it down. Once this happens, the protein is unable to send the signals that spur cell growth and cause melanoma lesions to proliferate. Because the drug spares healthy cells, nilotnib should have less toxicity and fewer side effects than traditional therapies.

"For advanced melanoma, there are currently few satisfactory treatments," Kaufman says. "But new targeted therapies, including vaccines, antibodies and small molecules like nilotnib are in clinical trials now, adding to an arsenal of treatments that appear to be promising. This trial is especially significant since the c-kit mutation is found more commonly in melanomas arising from the mucosa and foot, which are historically very difficult types of melanoma to treat."

Already approved by the Food and Drug Administration for the treatment of chronic myelogenous leukemia, nilotnib is now being studied for the first time for the treatment of melanomas that express the c-kit gene.

More Information at Your Fingertips ...

  • If you are interested in exploring your eligibility for the nilotnib trial at Rush, call Darilyn Greenhow at (312) 563-2330. Or click here to view open cancer clinical trials on the Rush Web site.
  • The new Rush University Cancer Center provides leading-edge, comprehensive, compassionate care for the treatment of a wide array of cancers, including melanomas. To schedule an appointment, call (888) 352-RUSH (7874).
  • Rush is committed to helping patients and their families cope with the psychological, emotional and spiritual effects of cancer. Through the Cancer Integrative Medicine Program, patients have access to therapies — such as acupuncture, biofeedback, nutritional and herbal counseling, massage and yoga — that complement their medical treatments.
  • Looking for a doctor? Call toll free: 888 352-RUSH (888 352-7874)
  • Stay in touch with Rush with Rush Blogs, Facebook, Twitter, YouTube and more.

Please note: All physicians featured in Discover Rush Online are on the medical faculty of Rush University Medical Center. Some of the physicians featured are in private practice and, as independent practitioners, are not agents or employees of Rush University Medical Center.

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Under the Microscope: Testing a Targeted Therapy for Melanoma

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