BMY Bristol Myers Squibb Co

By Ron Winslow 

The strategy of enlisting the power of the immune system to attack cancer is showing early promise against another tough-to-treat malignancy: the brain cancer called glioblastoma.

In the latest development, researchers reported on Friday that a cancer vaccine being developed by Celldex Therapeutics Inc. reduced the risk of death from the disease by 47% in a 73-patient randomized study. In addition, the survival rate at two years was 25% for patients given the vaccine versus none in the control group.

It marks the first time an immunotherapy treatment has improved survival in a randomized trial of glioblastoma patients, researchers said. It is also among the first cancer vaccines to show a survival benefit.

While the study was small and overall results were modest, said David Reardon, clinical director of neuro-oncology at Boston's Dana-Farber Cancer Institute, the findings indicate "we may have a strategy that will move us forward for the first time in a long time in this very challenging brain cancer."

Dr. Reardon presented the data at a meeting of the Society for Neuro-Oncology in San Antonio. The results generally confirmed an earlier report on the study, called React, presented in May.

The price of Celldex shares rose about 12% in Nasdaq trading on Friday.

Pharmaceutical companies are rushing to develop cancer-immunotherapy medicines against a wide variety of tumors, driven by successes from Bristol-Myers Squibb Co.'s Yervoy against melanoma and its Opdivo and Merck & Co.'s Keytruda, which treat both melanoma and lung cancer.

About 12,000 U.S. patients are diagnosed with glioblastoma each year, according to the American Brain Tumor Association. Until recently, oncologists were skeptical that marshaling the immune system against brain cancer would have much effect.

But amid widening enthusiasm for cancer immunotherapy, researchers tried it anyway. Now the New York-based Cancer Research Institute lists more than 30 clinical trials of various cancer-immunotherapy approaches against glioblastoma and related brain tumors.

The Celldex trial involves its cancer vaccine rindopepimut, which targets a mutant protein called EGFRvIII that is present in about 25% to 30% of glioblastoma cases. The patients in the study carried that mutation.

The mutation is associated with poor outcomes, but it differentiates the glioblastoma cells from normal cells. That makes it an attractive immune-system target because it could minimize the potential for the immune response to cause serious side effects by hitting normal cells as well. Researchers said adverse events associated with the vaccine were minimal.

Patients in the study had previously relapsed after treatments other than Celldex's new vaccine. The study reported on Friday showed tumors in about 30% of patients responded to rindopepimut, compared with 18% in the control arm. Median survival with the vaccine was 11.3 months compared with 9.3 months for those in the control arm.

"They took a group of patients that are probably the toughest to treat," Michael Lim, director of brain tumor immunotherapy at Johns Hopkins Medicine, Baltimore, who wasn't involved in the study. "The fact that they showed any sort of signal is very intriguing."

Despite the modest median survival gains, researchers were struck by the durable survival in 25% of the vaccine patients. The result is similar to the surprising long-term survival seen in trials of Bristol-Myers Squibb's Yervoy, where about 21% of melanoma patients survived at least three years, despite many initially not appearing to respond to the treatment.

"Once patients are surviving long term, they don't die" of the disease, said John Sampson, chief of neurosurgery at Duke University Medical Center and co-principal investigator on the study. "This is the kind of thing we look for with immunotherapy." It will take larger studies and longer follow up to determine the significance of that result. Dr. Sampson is an inventor of the vaccine with a financial stake in its success.

Based in part on earlier data from the study, the U.S. Food and Drug Administration granted the vaccine breakthrough therapy designation last February, a status that could hasten regulatory review. In addition, a late stage, or phase 3, study involving 745 newly diagnosed glioblastoma patients is fully enrolled, with an interim look at the findings expected early in 2016.

Write to Ron Winslow at ron.winslow@wsj.com

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(END) Dow Jones Newswires

November 20, 2015 17:40 ET (22:40 GMT)

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