Study data appears in New England Journal of Medicine

PHOENIX, Ariz. - Sept. 2, 2009 - Analyses of clinical trial results published today in the New England Journal of Medicine (NEJM) shows a potential new investigational therapy for advanced and metastatic basal cell skin cancer.

The study, conducted at TGen Clinical Research Service (TCRS) at Scottsdale Healthcare and two other sites appears to demonstrate tumor shrinkage and limited side effects. TCRS is a strategic alliance between the Translational Genomics Research Institute (TGen) and Scottsdale Healthcare.

These findings are significant because no proven therapy exists 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.

The article appears on-line today and in the Sept. 17 print issue of NEJM.

"Until now, no treatment existed that effectively slowed tumor growth in those patients with advanced skin cancer," said lead investigator Daniel D. Von Hoff, MD, Physician in Chief at TGen, Chief Medical Officer for the Scottsdale Clinical Research Institute at Scottsdale Healthcare and chief scientific officer at US Oncology. "By strategically initiating what we call "precision oncology", or using the right drug for each cancer, this study offers great potential against basal cell carcinoma and other cancers."

The trial, sponsored by Genentech, included clinicians at TCRS, the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University in Baltimore, and Karmanos Cancer Institute in Detroit. The results demonstrated that GDC-0449, a Hedgehog Pathway Inhibitor, appears to shrink tumors in locally advanced and metastatic BCC. Known as the "Hedgehog" trial, the clinicians observed a durable clinical benefit -defined as tumor shrinkage visible on X-ray or other physical exam or improvement in symptoms without tumor growth- in 18 of 33 patients evaluated. Others had stable disease for prolonged periods of time. Only 4 patients had progression of disease.

Abnormal activation of the Hedgehog signaling pathway appears to be an important mechanism for numerous types of cancer to develop, survive, or grow. A chemical called cyclopamine found in the California corn lily can inactivate this Hedgehog pathway.

Study investigators selected BCC as the first cancer to study in that 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 TCRS physician Glen J. Weiss, M.D., a contributing author on the 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.

Initial observation and isolation of cyclopamine from the corn lily occurred in 1957. Subsequently, scientists at Genentech developed GDC-0449 (an oral drug), which was jointly validated through a series of preclinical studies performed under a collaboration agreement between Genentech and Curis, Inc. (Cambridge, MA). Genentech is a wholly owned subsidiary of the Roche Group.

Patients seeking more information on new therapies available through either TCRS or Scottsdale Healthcare can e-mail [email protected] or call 480-323-1339 (toll free 1-877-273-3713).

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About the Virginia G. Piper Cancer Center at Scottsdale Healthcare
The Virginia G. Piper Cancer Center at Scottsdale Healthcare offers cancer prevention, diagnosis, treatment, research and support services in its facilities at the Scottsdale Healthcare Shea Medical Center, attracting patients from across Arizona and the U.S. Groundbreaking cancer research is conducted through its Scottsdale Clinical Research Institute and TGen Clinical Research Service. It is a primary clinical research site for the Translational Genomics Research Institute and the Stand Up To Cancer Pancreatic Cancer Research Dream Team. Scottsdale Healthcare is the not-for-profit parent organization of the Scottsdale Healthcare Shea Medical Center, Scottsdale Healthcare Osborn Medical Center and Scottsdale Healthcare Thompson Peak Hospital, Virginia G. Piper Cancer Center, Scottsdale Clinical Research Institute and Scottsdale Healthcare Foundation. For additional information, please visit

About TCRS
TGen's Clinical Research Service (TCRS), a strategic alliance with Scottsdale Healthcare (SHC), provides a direct clinical research site for TGen. Led by Dr. Daniel Von Hoff, TGen's Physician-in-Chief and TCRS Chief Scientific Officer, TCRS clinicians focus on clinical trials with targeted agents and genomics-based individualized therapy. The TCRS, with an initial focus on cancer, allows the unique opportunity for TGen to transition its laboratory-based research to patient care centered on individualized therapy. TCRS will bring new clinical research into the community, to those patients who would otherwise have to travel someplace else for access to new therapies or prevention agents.

About TGen
The Translational Genomics Research Institute (TGen) is a non-profit 501(c)(3) organization dedicated to conducting groundbreaking research with life changing results. Research at TGen is focused on helping patients with diseases such as cancer, neurological disorders and diabetes. TGen is on the cutting edge of translational research where investigators are able to unravel the genetic components of common and complex diseases. Working with collaborators in the scientific and medical communities, TGen believes it can make a substantial contribution to the efficiency and effectiveness of the translational process. TGen's vision is of a world where an understanding of genomic variation can be rapidly translated in a manner tailored to individual patients. For more information about TGen, please visit

Keith Jones, Director of Public Relations
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Steve Yozwiak
TGen Senior Science Writer
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