Potential skin cancer breakthrough tested at Scottsdale Healthcare
October 7, 2009
SCOTTSDALE – A study published recently in the New England Journal of Medicine reports a potential new investigational therapy for advanced and metastatic basal cell skin cancer tested at the Virginia G. Piper Cancer Center at Scottsdale Healthcare and other sites appears to demonstrate tumor shrinkage and limited side effects in patients.
“Inhibition of the Hedgehog Pathway in Advanced Basal-Cell Carcinoma” is authored by lead investigator Daniel D. Von Hoff, MD, a world-renowned expert in developing new drugs for patients with cancer. Dr. Von Hoff is an oncologist and chief scientific officer at the Virginia G. Piper Cancer Center at Scottsdale Healthcare, physician-in-chief at Translational Genomics Research Institute (TGen) and chief scientific officer at US Oncology.
The article appears online at NEJM.org and is included in the Sept. 17 issue of New England Journal of Medicine.
These findings are significant because there is no proven therapy for advanced basal cell carcinoma (BCC). BCC is the most common cancer in the United States with about one million new cases diagnosed each year. Arizona has one of the highest incidences of skin cancer in the world.
Typically diagnosed with a simple biopsy, the risk of BCC increases for individuals with a family history or prolonged exposure to ultraviolet (UV) rays. Most patients are cured by surgery, but if left untreated or if spread to other organs, then scarring and disfigurement, and even death may result.
In a Phase I clinical trial conducted at the Virginia G. Piper Cancer Center at Scottsdale Healthcare, Dr. Von Hoff and colleagues at Johns Hopkins University and Karmanos Cancer Institute demonstrated that GDC-0449, a Hedgehog Pathway Inhibitor, appears to shrink tumors in locally-advanced and metastatic basal cell carcinoma (BCC) while having limited side effects including a loss of sense of taste, and a small amount of hair loss and weight loss.
Known as the “Hedgehog” trial, results suggest a durable clinical benefit, defined as tumor shrinkage visible on X-ray or improvement in symptoms without tumor growth.
“Until now, we did not have any treatments that can effectively slow the tumor growth in these patients with advanced skin cancer. Using the right drug for each cancer, or precision oncology as we call it, has great potential against other cancers as well,” said Dr. Von Hoff.
Basal cell cancer was chosen as the first cancer to be studied, as most BCCs have abnormalities or mutations of Hedgehog pathway genes named PATCHED and SMOOTHENED.
“Success of this new therapy is another example of applying genetic information to medicine. We are constantly working to improve treatment options for patients with common and rare cancers,” said Dr. Glen J. Weiss, part of Dr. Von Hoff’s team and contributing author for this study.
Patient response to the therapy was assessed through physical examination and imaging. “Integrating genomic data with state-of-the-art clinical and imaging information to develop and apply targeted therapies has certainly taken a major step forward with the encouraging results from the Hedgehog trial," added Dr. Ron Korn, a Scottsdale Healthcare radiologist and director of Scottsdale Medical Imaging Ltd.
Scottsdale oncologist Dr. Maqbool Halepota agrees that advances in precision medicine are good news for patients with cancer. “More and more targets are being identified that could lead to better options for treating cancer. This looks like a major breakthrough for those with advanced basal cell carcinoma.”
Patients seeking more information on new therapies available through the Virginia G. Piper Cancer Center at Scottsdale Healthcare can e-mail firstname.lastname@example.org or call 480-323-1339 (toll free 1-877-273-3713).