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
vismodegib
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
Medicine.
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].
# # #
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.
Press Contact:
Keith Jones
Public Relations Director, Virginia G. Piper Cancer Center
480-323-1383
[email protected]
*
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.
Media Contact
Julie Janovsky-Mason
Public Affairs
480-301-4222
*
About TGen
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.
Press Contact:
Steve Yozwiak
TGen Senior Science Writer
602-343-8704
[email protected]
