- Posted Tuesday June 12, 2012
NEJM hails new skin cancer drug as 'greatest advance yet'
World's first patient treated at Virginia G. Piper Cancer Center
by Scottsdale Healthcare, TGen physicians; Follow-up study led by
Mayo Clinic-TGen physician proved key to early FDA approval of
June 12, 2012
Vismodegib, a new skin cancer drug for patients with advanced
basal cell carcinoma tested by TGen, Virginia G. Piper Cancer
Center at Scottsdale Healthcare and Mayo Clinic, is hailed as "the
greatest advance in therapy yet seen" for advanced basal cell
carcinoma in an editorial in the New England Journal of
Vismodegib (marketed under the name Erivedge) was administered for the first time in the world on Jan. 23, 2007 in a Phase I clinical trial at Virginia G. Piper Cancer Center Clinical Trials at Scottsdale Healthcare, a partnership with the Translational Genomics Research Institute (TGen).
This is the first drug tested under the Scottsdale Healthcare-TGen partnership to receive FDA approval, and is the first to receive FDA approval to treat inoperable basal cell carcinoma. Successful early trial results led to additional study sponsored by Genentech.
The successful Phase I study at Virginia G. Piper Cancer Center Clinical Trials at Scottsdale Healthcare, headed by Scottsdale Healthcare Chief Scientific Officer and TGen Physician-In-Chief Daniel Von Hoff, M.D, led to a broader study, published June 7 by the New England Journal of Medicine.
"The first patient put on this drug had advanced basal cell cancer, so we suspected that the tumor had the mutation this drug is targeted against," said Ramesh K. Ramanathan, M.D., Medical Director at Virginia G. Piper Cancer Center Clinical Trials and Clinical Professor and Deputy Director of the Clinical Translational Research Division at TGen. "And our partnership with Dr. Ronald Korn and his team who did advanced PET imaging helped to really demonstrate the drug's efficacy for Genentech to continue pursuing the additional study."
The follow-up study, a Phase 2 clinical trial of basal cell carcinoma (BCC), was headed by Dr. Aleksandar Sekulic, Assistant Professor of Dermatology for the Mayo Clinic in Arizona, and an Assistant Professor in TGen's Integrated Cancer Genomics Division.
This Phase 2 study provided the efficacy data for vismodegib that led to its approval by the U.S. Food and Drug Administration (FDA) on Jan. 30, 2012.
"As a result of a strong collaboration between the Valley institutions, clinical development of this new medication, from the first clinical trial to the FDA approval, was led by the Valley researchers," said Dr. Sekulic, M.D and Ph.D., whose study found that vismodegib shrank advanced basal cell carcinoma tumors in 43 percent of patients with locally advanced disease and in 30 percent of patients whose disease spread to other organs.
The drug blocks the Hedgehog signaling pathway and was approved for treatment of locally advanced and metastatic basal cell carcinomas. Additional research noted encouraging results for patients with inherited genetic susceptibility for development of large numbers of BCC tumors, known as basal cell nevus syndrome.
"The availability of vismodegib and similar medications will really change the way we treat the patients with advanced forms of BCC, who had very limited options to date. In addition, this drug will likely offer a life-changing treatment for patients with basal cell nevus syndrome. It is possible that the drug may also be of benefit in treatment of earlier stages of BCC, and this is currently being evaluated in several clinical trials," Dr. Sekulic said.
"It is a landmark day for patients with basal cell carcinoma and all those involved in their care," said the NEJM editorial. However the editors cautioned that additional study of Hedgehog pathway inhibitors like vismodegib warrant further study so a larger number of patients may benefit.
FDA approval in five years is a remarkable achievement because clinical trials typically progress through three phases and can take up to 15 years to successfully complete, according to Mark Slater, Ph.D., Vice President of Research at Scottsdale Healthcare.
Most instances of basal cell cancer can be effectively treated, but in some cases, the cancer cells spread and develop an aggressive form of the cancer that does not respond to standard surgical treatment.
"For a small percentage of patients, basal cell carcinoma can progress to inoperable life-threatening, locally advanced or metastatic tumors. Vismodegib, taken as a once a day pill, represents an opportunity to improve quality of life for these patients," said Dr. Glen Weiss, Director of Thoracic Oncology at Virginia G. Piper Cancer Center Clinical Trials and Clinical Associate Professor of TGen's Cancer and Cell Biology Division.
San Francisco-based Genentech developed vismodegib. Successful results of early clinical trials at the Virginia G. Piper Cancer Center at Scottsdale Healthcare, Johns Hopkins University and Karmanos Cancer Institute were published in the Sept. 17, 2009, New England Journal of Medicine and led to interest in increased access to the drug.
Individuals seeking information about eligibility to participate in clinical trials at the Virginia G. Piper Cancer Center at Scottsdale Healthcare may contact the cancer care coordinator at 480-323-1339; toll free at 1-877-273-3713 or via email at [email protected]
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About the Virginia G. Piper Cancer Center at Scottsdale Healthcare
The Virginia G. Piper Cancer Center at Scottsdale Healthcare in Scottsdale, Ariz. offers comprehensive cancer treatment and research through Phase I clinical trials, diagnosis, prevention and support services in collaboration with leading scientific researchers and community oncologists. Scottsdale Healthcare is the nonprofit parent organization of the Virginia G. Piper Cancer Center at Scottsdale Healthcare, Scottsdale Healthcare Research Institute, Scottsdale Healthcare Osborn Medical Center, Scottsdale Healthcare Shea Medical Center and Scottsdale Healthcare Thompson Peak Hospital. For more information, visit www.shc.org.
Public Relations Director, Virginia G. Piper Cancer Center
About Mayo Clinic
Mayo Clinic is a nonprofit worldwide leader in medical care, research and education for people from all walks of life. For more information, visit MayoClinic.com or MayoClinic.org/news.
The Translational Genomics Research Institute (TGen) is a Phoenix, Arizona-based non-profit 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. For more information, visit: www.tgen.org.
TGen Senior Science Writer