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Eligible patients with newly diagnosed glioblastoma multiforme (GBM), the most common and aggressive form of brain cancer, are enrolling in the ACT III clinical trial of a vaccine called CDX-110, which may prevent recurrence and extend survival. Physician-scientists at NewYork-Presbyterian Hospital, together with Weill Cornell Medical College and Columbia University Medical Center, are helping to lead the Phase II multicenter trial of the vaccine, which is thought to work by "training" the immune system to target and kill cancer cells.

"Even after surgery, radiation and chemotherapy, this deadly brain cancer has a high likelihood of recurrence. This experimental vaccine is designed to harness the body's immune system to keep the cancer at bay," says Dr. Theodore Schwartz, site principal investigator and neurosurgeon at NewYork-Presbyterian Hospital/Weill Cornell Medical Center, and associate professor of neurological surgery at Weill Cornell Medical College.

"Results from earlier trials are promising, and suggest that the vaccine may be able to improve both time to disease progression and length of survival," says Dr. Rose Lai, site principal investigator and neuro-oncologist at NewYork-Presbyterian Hospital/Columbia University Medical Center, and assistant professor of neurology in the Division of Neuro-Oncology at Columbia University College of Physicians and Surgeons.

Glioblastoma multiforme commonly causes memory, personality and neurological deficits, but may also produce seizures, nausea and vomiting, headache, and weakness on one side of the body (hemiparesis). The location of the tumor plays a role in what types of symptoms a patient may suffer. Until it is large enough, the tumor may not cause any symptoms. About 10,000 people are diagnosed with the disease each year.

An initial single-arm Phase II trial (ACT II) has reported promising preliminary data in 23 patients who received CDX-110. Median time to disease progression was 16.6 months and estimated median overall survival was 33.1 months. This compared favorably with data for a historical control group in which median time to progression was 6.4 months and median overall survival was 15.2 months.

The ACT III study will look at the effectiveness of the vaccine plus temozolomide in patients who are newly diagnosed with glioblastoma multiforme. Patients will receive injections into the skin every two weeks for three doses shortly after the completion of cranial radiation, followed by monthly injections for as long as the tumor has not returned. Temozolomide chemotherapy will be given one week after each monthly injection of the vaccine and continued for up to 12 cycles.

Qualifying patients must be 18 or older, have newly diagnosed and surgically resected glioblastoma multiforme. Interested patients should be screened for the study immediately following surgery. Qualifying patients must have cancer that expresses the protein epidermal growth factor receptor variant III (EGFRvIII). About 20 percent to 25 percent of patients with GBM express the EGFRvIII mutation. The vaccine is expected to work by activating the immune system against the EGFRvIII protein, shutting down the engine room of cancer growth in these patients. Patients must also have had a near total resection of their tumors and must be able to undergo combined radiation with temozolomide in order to qualify.

"If this approach is validated, vaccine therapy could be added on to the existing regimen of combined chemo-radiation," says Dr. Schwartz.

The study is sponsored by Celldex Therapeutics Inc. of Phillipsburg, NJ.

For more information, patients may call 866-NYP-NEWS.

Cancer Vaccines

Cancer vaccines are intended either to treat existing cancers (therapeutic vaccines) or to prevent the development of cancer (prophylactic vaccines). Therapeutic vaccines, which are administered to cancer patients, are designed to treat cancer by stimulating the immune system to recognize and attack human cancer cells without harming normal cells. Prophylactic vaccines are given to healthy individuals to stimulate the immune system to attack cancer-causing viruses and prevent viral infection. Currently, two vaccines have been licensed by the U.S. Food and Drug Administration to prevent virus infections that can lead to cancer: the hepatitis B vaccine, which prevents infection with the hepatitis B virus, an infectious agent associated with liver cancer; and Gardasil®, which prevents infection with the two types of human papillomavirus that together cause 70 percent of cervical cancer cases worldwide.

NewYork-Presbyterian Hospital

NewYork-Presbyterian Hospital, based in New York City, is the nation's largest not-for-profit, non-sectarian hospital, with 2,242 beds. The Hospital has nearly 2 million inpatient and outpatient visits in a year, including more than 230,000 visits to its emergency departments — more than any other area hospital. NewYork-Presbyterian provides state-of-the-art inpatient, ambulatory and preventive care in all areas of medicine at five major centers: NewYork-Presbyterian Hospital/Weill Cornell Medical Center, NewYork-Presbyterian Hospital/Columbia University Medical Center, NewYork-Presbyterian Morgan Stanley Children's Hospital, NewYork-Presbyterian Hospital/The Allen Pavilion and NewYork-Presbyterian Hospital/Westchester Division. One of the largest and most comprehensive health care institutions in the world, the Hospital is committed to excellence in patient care, research, education and community service. NewYork-Presbyterian is the #1 hospital in the New York metropolitan area and is consistently ranked among the best academic medical institutions in the nation, according to U.S.News & World Report. The Hospital has academic affiliations with two of the nation's leading medical colleges: Weill Cornell Medical College and Columbia University College of Physicians and Surgeons. For more information, visit www.nyp.org.

Weill Cornell Medical College

Weill Cornell Medical College, Cornell University's medical school located in New York City, is committed to excellence in research, teaching, patient care and the advancement of the art and science of medicine, locally, nationally and globally. Weill Cornell, which is a principal academic affiliate of NewYork-Presbyterian Hospital, offers an innovative curriculum that integrates the teaching of basic and clinical sciences, problem-based learning, office-based preceptorships, and primary care and doctoring courses. Physicians and scientists of Weill Cornell Medical College are engaged in cutting-edge research in areas such as stem cells, genetics and gene therapy, geriatrics, neuroscience, structural biology, cardiovascular medicine, transplantation medicine, infectious disease, obesity, cancer, psychiatry and public health — and continue to delve ever deeper into the molecular basis of disease in an effort to unlock the mysteries of the human body in health and sickness. In its commitment to global health and education, the Medical College has a strong presence in places such as Qatar, Tanzania, Haiti, Brazil, Austria and Turkey. Through the historic Weill Cornell Medical College in Qatar, the Medical College is the first in the U.S. to offer its M.D. degree overseas. Weill Cornell is the birthplace of many medical advances — including the development of the Pap test for cervical cancer, the synthesis of penicillin, the first successful embryo-biopsy pregnancy and birth in the U.S., the first clinical trial of gene therapy for Parkinson's disease, the first indication of bone marrow's critical role in tumor growth, and most recently, the world's first successful use of deep brain stimulation to treat a minimally conscious brain-injured patient. For more information, visit www.med.cornell.edu.

Columbia University Medical Center

Columbia University Medical Center provides international leadership in basic, pre-clinical and clinical research, in medical and health sciences education, and in patient care. The Medical Center trains future leaders and includes the dedicated work of many physicians, scientists, public health professionals, dentists, and nurses at the College of Physicians & Surgeons, the Mailman School of Public Health, the College of Dental Medicine, the School of Nursing, the biomedical departments of the Graduate School of Arts and Sciences, and allied research centers and institutions. Established in 1767, Columbia's College of Physicians and Surgeons was the first institution in the country to grant the M.D. degree and is now among the most selective medical schools in the country. Columbia University Medical Center is home to the largest medical research enterprise in New York City and state and one of the largest in the United States. For more information, please visit www.cumc.columbia.edu.