(ORLANDO) – In what researchers are calling a breakthrough, patients with thyroid cancer that is resistant to radioactive iodine therapy were found to respond well to sorafenib, a University of Pennsylvania School of Medicine researcher reported today at the annual meeting of the American Society of Clinical Oncology (ASCO). The phase II clinical trial data highlight an intensive effort at the Abramson Cancer Center to develop effective, personalized therapies for these patients, who have previously had few options for treatment.

“This is not a transition, this is a breakthrough for our patients,” says Marcia S. Brose, MD, PhD, assistant professor of hematology/oncology, who led the trial. “This is the first significant progress in 30 years, since doxorubicin was approved in 1974, which is toxic and produces responses in only five percent or patients.”

What is also unique to the program led by Brose is the intensive lab-based efforts underway to understand the molecular basis of the responses, so that future therapies can be tailored to each patient. “We have a full set of biospecimens on this group of patients, which together with the clinical data will allow us to answer many questions related to target therapy for these patients,” Brose says.

An interim analysis published recently in the Journal of Clinical Oncology reported on the first 30 patients enrolled in the trial and first showed that sorafenib is an effective agent at treating advanced disease. Twenty-three percent of those patients achieved an objective partial response to therapy, with another 54 percent achieving durable stabilization of their disease. Updated results presented at ASCO reveal that the responses translate into a median overall survival of more than 140 weeks -- a three-fold improvement over doxorubicin. (Progression-free survival data for the entire study cohort and the differentiated thyroid cancer patients which make up a majority of the patients studied will be presented at the meeting). Brose will present compelling data showing that a major component of the sorafenib response is anti-angiogenic – inhibiting the growth of blood vessels needed to support tumor growth – consistent with the mechanism seen in other tumors. A total of 55 patients have enrolled in the trial so far, and Brose and her team continue to follow their progress and characterize their samples.

Based on these findings and data from Dr. Brose’s and other phase II trials, the National Comprehensive Cancer Network clinical guidelines now recommend use of sorafenib for patients with radioactive iodine-refractory thyroid cancer who are not able to enter clinical trials. About 10 percent of the 30,000 patients diagnosed with thyroid cancer each year ultimately progress to this advanced disease, with tumors appearing in the bones, lungs and other sites.

“What became clear is that there was an enormous unmet need out there,” Brose says. “I have had patients come from as far away as Hawaii, Uruguay and northern Ontario.”

Brose became interested in thyroid cancer when she realized that it shared some genetic changes with renal cancer and melanoma, malignancies in other Penn clinical trials she was involved in. “Once I started studying it, I realized that very few oncologists treated or understood the disease due to the lack of successful treatments. So once these patients failed radioactive-iodine, they really had no options,” she says. “No one wants to be sent home and told there is no treatment available.”

Brose hopes to launch additional clinical trials this year as part of her effort to have a trial for patients with every type and stage of thyroid cancer, including a phase II trial for patients whose cancers have progressed despite sorafenib treatment.

Genetic mutations and immunohistochemistry data uncovered in tumor tissue from each of the patients enrolled is helping guide new treatment approaches, allowing each patient to receive the most effective therapy for their cancer.

“If you have tissue from patients and know how they respond to treatment, you have the chance to predict where you should go next,” Brose says. “We are really setting the foundation for a new treatment paradigm for this disease –– a paradigm which didn’t exist three years ago.”

Editor’s note: Bayer/Onyx provided the study drug for the trial, and Dr. Brose has received consulting fees from Bayer/Onyx.


PENN Medicine is a $3.6 billion enterprise dedicated to the related missions of medical education, biomedical research, and excellence in patient care. PENN Medicine consists of the University of Pennsylvania School of Medicine (founded in 1765 as the nation's first medical school) and the University of Pennsylvania Health System.

Penn's School of Medicine is currently ranked #3 in the nation in U.S.News & World Report's survey of top research-oriented medical schools; and, according to the National Institutes of Health, received over $366 million in NIH grants (excluding contracts) in the 2008 fiscal year. Supporting 1,700 fulltime faculty and 700 students, the School of Medicine is recognized worldwide for its superior education and training of the next generation of physician-scientists and leaders of academic medicine.

The University of Pennsylvania Health System (UPHS) includes its flagship hospital, the Hospital of the University of Pennsylvania, rated one of the nation’s top ten “Honor Roll” hospitals by U.S.News & World Report; Pennsylvania Hospital, the nation's first hospital; and Penn Presbyterian Medical Center, named one of the nation’s “100 Top Hospitals” for cardiovascular care by Thomson Reuters. In addition UPHS includes a primary-care provider network; a faculty practice plan; home care, hospice, and nursing home; three multispecialty satellite facilities; as well as the Penn Medicine Rittenhouse campus, which offers comprehensive inpatient rehabilitation facilities and outpatient services in multiple specialties.

The Abramson Cancer Center (ACC) of the University of Pennsylvania is a national leader in cancer research, patient care, and education. The pre-eminent position of the Cancer Center is reflected in its continuous designation as a Comprehensive Cancer Center by the National Cancer Institute for 30 years, one of 39 such Centers in the United States. The ACC is dedicated to innovative and compassionate cancer care. The clinical program, composed of a dedicated staff of physicians, nurse practitioners, nurses, social workers, physical therapists, nutritionists and patient support specialists, currently sees over 50,000 outpatient visits, 3400 inpatient admissions, and provides over 25,000 chemotherapy treatments, and more than 65,000 radiation treatments annually. Not only is the ACC dedicated to providing state-of-the-art cancer care, the latest forms of cancer prevention, diagnosis, and treatment are available to our patients through clinical themes that developed in the relentless pursuit to eliminate the pain and suffering from cancer. In addition, the ACC is home to the 400 research scientists who work relentlessly to determine the pathogenesis of cancer. Together, the faculty is committed to improving the prevention, diagnosis and treatment of cancer.

Penn Medicine is one of the world’s leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of the Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania (founded in 1765 as the nation's first medical school) and the University of Pennsylvania Health System, which together form a $7.8 billion enterprise.

The Perelman School of Medicine has been ranked among the top five medical schools in the United States for the past 20 years, according to U.S. News & World Report’s survey of research-oriented medical schools. The School is consistently among the nation’s top recipients of funding from the National Institutes of Health, with $405 million awarded in the 2017 fiscal year.

The University of Pennsylvania Health System’s patient care facilities include: The Hospital of the University of Pennsylvania and Penn Presbyterian Medical Center — which are recognized as one of the nation’s top “Honor Roll” hospitals by U.S. News & World Report — Chester County Hospital; Lancaster General Health; Penn Medicine Princeton Health; Penn Wissahickon Hospice; and Pennsylvania Hospital – the nation’s first hospital, founded in 1751. Additional affiliated inpatient care facilities and services throughout the Philadelphia region include Good Shepherd Penn Partners, a partnership between Good Shepherd Rehabilitation Network and Penn Medicine, and Princeton House Behavioral Health, a leading provider of highly skilled and compassionate behavioral healthcare.

Penn Medicine is committed to improving lives and health through a variety of community-based programs and activities. In fiscal year 2017, Penn Medicine provided $500 million to benefit our community.

Share This Page: