Targeted Therapy Shrinks Melanoma Tumors in Patients with Mutated BRAF Gene


August 30, 2010

Penn Medicine News Brief

Targeted Therapy Shrinks Melanoma Tumors in Patients with Mutated BRAF Gene

Use of an experimental targeted drug to treat metastatic melanoma tumors with a specific genetic signature was successful in more than 80 percent of patients in a phase 1 clinical trial. Results of the trial of PLX4032, an inhibitor of a protein called BRAF that is overactive in more than half of all melanomas, appear in the August 26 New England Journal of Medicine. The role in melanoma of the BRAF mutation, which keeps the protein constantly activated and driving cell growth, was discovered in 2002 by researchers at the Sanger Institute in Britain... Read More

The Philadelphia Inquirer Penn study makes breakthrough in shrinking tumors of terminal melanoma patients

In a major advance against the deadliest type of skin cancer, a genetically targeted drug shrank tumors throughout the bodies of 80 percent of patients with terminal melanoma.

The study, led by the University of Pennsylvania and conducted at seven medical centers, is the first to successfully exploit a molecular abnormality found in about half of melanoma tumors, but not in healthy cells... Read More

The New York Times
A Drug Trial Cycle: Recovery, Relapse, Reinvention
In the final installment of a three part, front-page series, the New York Times explores the experiences of physicians and patients involved in the Phase 1 trial of the drug PLX4032, a targeted therapy for advanced melanoma patients whose cancers exhibit B-RAF mutations. The article follows Keith Flaherty, MD, a former faculty member in the division of Hematology-Oncology, Lynn Schuchter, MD, chief of the division of Hematology-Oncology, and an Abramson Cancer Center patient, Christopher Nelson, a 42 year old New Jersey father of two. Nelson, who had once been told by other doctors that there were no more treatment options for him and that he should consider hospice care, enjoyed another full year of life from trials for PLX4032 and a second targeted melanoma therapy. At his wake last month, Nelson's wife told relatives she felt blessed that he had lived longer than expected. They had celebrated their 21st wedding anniversary. With the children, he had ridden every water ride at Six Flags Great Adventure. "It's a year I would never trade in," she said. Nelson and Flaherty are also featured in the video accompanying the story. Read More
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After Long Fight, Drug Gives Sudden Reprieve
In the second part of a three-part, front-page series, the New York Times explores the experiences of physicians and patients involved in the trial of the drug PLX4032, a targeted therapy for advanced melanoma patients whose cancer exhibit B-RAF mutations. The article follows Keith Flaherty, MD, a former faculty member in the division of Hematology-Oncology, and an Abramson Cancer Center patient, Christopher Nelson, a 42 year old father of two who enrolled in the Phase 1 trial after another hospital told him they could offer him no more hope and that he should consider seeking hospice care. In just a few weeks he improved dramatically, more than any melanoma patient Flaherty had ever seen -- gaining17 pounds, heading to Atlantic City for a day of gambling, and dining out with family and friends. Maryann Redlinger, RN, a clinical research nurse in the Abramson Cancer Center, is also featured in the story of her role monitoring Nelson's progress before and after he began the trial. Flaherty, Redlinger, and Nelson are also featured in the video accompanying the story. Read More
New York Times View Video

A Roller Coaster Chase for a Cure
The first in a three-part series of articles in the New York Times appeared on the front page profiling Keith Flaherty, MD, a former faculty member in the division of Hematology-Oncology, and the Abramson Cancer Center's work on a trial of the drug PLX4032, a targeted therapy for advanced melanoma patients whose cancers exhibit B-RAF mutations. Lynn Schuchter, MD, chief of the division of Hematology-Oncology, and Maryann Redlinger, RN, a clinical research nurse in the Abramson Cancer Center, are also mentioned in the story, which takes readers inside the world of early-stage clinical trials and the patients who seek experimental drugs for their deadly cancer. The series, "A Doctor's Trial," will continue tomorrow and Wednesday. Read Article

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