Updated

Researchers at New York University’s Clinical Cancer Center and several other U.S. medical colleges believe they may have found a breakthrough vaccine to treat the most deadly form of brain cancer.

The vaccine actually uses proteins found in brain tumors of patients with glioblastoma multiforme, the most common and fatal form of brain cancer, to destroy the tumors themselves.

Several universities including NYU, Duke University and the University of California, Los Angeles, as well as Henry Ford Hospital in Detroit are testing it.

The five-year study will track the effects of the vaccine on patients, who receive radiation and chemotherapy treatments over a three-year period. The participants will receive the vaccines every couple of weeks at first, then once every few months and several times per year for three years and will be screened for two additional years following the treatment.

Northwest Biotherapeutics Inc. developed the vaccine, known as DCVax-Brain.

The vaccine treatment alone was successful in four patients tested at UCLA, but researchers believe that the vaccine coupled with radiation and chemotherapy can be more effective.

"We’re trying to see if this is a much better approach," said Dr. Michael Gruber, clinical professor of neurology and neurosurgery at NYU’s School of Surgery and principal investigator of the study.

"We feel that combining the standard care treatment followed by chemotherapy for six months will be more effective than using the vaccine alone. The hypothesis is that it’s better with chemotherapy and standard treatments."

The vaccine will only be used on newly diagnosed patients. To make the vaccine, a patient’s tumor is removed and shipped to a laboratory where the cells will be broken up to prepare the first component of the treatment.

Separately, patients’ dendritic cells, a powerful type of immune cell made up of white blood cells, also are extracted and sent to a laboratory for purification. Dendritic cells may be able to teach the immune system to recognize and destroy cancer cells, according to the initial report.

"At surgery, we take out the tumor, and this is a large part of process," Gruber said. "We take the tumor and freeze it. The patient goes on to receive radiation and chemotherapy, and at the end of the treatment they have an MRI. White cells are also treated with medicine to encourage growth."

The study underway at NYU Medical Center is an expansion of a phase I trial of the vaccine, which took place at UCLA in 2000. Researchers are hoping to test 225 patients over a five-year period.

NYU will start with 15 to 18 patients per year. Combined with a number of volunteers tested at other universities, researchers should have clearer statistics on the effectiveness of the vaccine on patients once the trial is completed.

The American Cancer Society estimates that 20,500 malignant tumors of the brain or spinal cord will be diagnosed in adults this year in the U.S., and approximately 12,740 people will die from these malignant tumors.

Some of the most common symptoms of brain cancer include: headaches, nausea and vomiting; changes in the ability to talk, hear or see; problems with balance or walking; problems with thinking or memory; muscle jerking or twitching and numbness or tingling in arms or legs.

Patients with brain cancer usually can survive 15 months with surgery and chemotherapy. Just a small number of tumor cells left in the brain can be enough to make the cancer return.

The hope is that the vaccine will work as a kind of immunotherapy that makes the patient’s immune system kill the proteins in the cancer cells.

"We are really excited about the promise of this vaccine," said Dr. Patrick J. Kelly, the chairman of the department of neurosurgery at NYU School of Medicine.

"Everything now depends on something in addition to surgery so that these tumors do not recur. A cancer vaccine like this may make a difference in extending life and maintaining a good quality of life. It is so frustrating because brain tumors don’t metastasize like other tumors. They recur locally but we just can’t cure it."

The study will involve patients aged 18 to 65. They will receive standard treatment followed by radiation and concurrent chemotherapy. Some will receive the standard care alone, and others will receive the standard of care and the vaccine.

Gruber said it is most important to remember when testing this vaccine that the brain is delicate, so human function must be preserved as much as possible throughout the study. The placement of the tumor also must be in the right place to be a candidate for removal. A partial piece of the tumor cannot be used alone with the vaccine.

"This is the most advanced cancer there is," Gruber said. "The tumor must be in area where it can be removed. The brain is very eloquent, so if it’s in a part of the brain that’s delicate it can be too delicate a procedure. For the vaccine to work, we need to remove the entire tumor. When you treat people with a horrible disease like this it’s already hard for them to move and talk, so you also want to preserve as much function as possible."

NYU opened the trial in July and Gruber hopes to have some initial findings by next fall but says there will not be a precise analysis until more people are studied.

"It’s sheer luck," Gruber said. "But the problem with studies is that you can’t go by early numbers unless everyone does horribly or it works perfectly for everyone from a small group. I think in order to get accurate data you have to have the whole number."

There are no known risks or side effects related to the vaccine; however, Gruber said the usual drowsiness from radiation treatment and swelling or redness where vaccinated may occur. There is no known effect on the heart, and allergic reactions will be monitored.

"We’re hoping to cure people with this horrible disease," Gruber said. "It kills most people within a year and is the second most common tumor growth in kids. This could be a ray of sunshine. I’m hoping it makes another dent into disease and the overall outcome."