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Immunotherapy research gets a boost from Facebook

Institutions and health-care companies across the country have been investing in immunological research. Notably, the Parker Institute for Cancer Immunotherapy, founded in 2016 with a $250 million kick-start from former Facebook president Sean Parker, has created opportunity for more trials. The institute's Tumor Neoantigen Selection Alliance, which includes Bhardwaj's team and others from the University of Pennsylvania and to Stanford Medicine, is focusing on discovering "cancer markers" and then making predictions on neoantigens to combat them.

The "personalized" nature of vaccines is what researchers describe as most promising. Other traditional treatments, such as chemotherapy or surgery, act more like sledgehammers. The vaccine could even be described as "holistic," according to Fred Ramsdell, vice president of research at the nonprofit Parker Institute.

"You can imagine a scenario for many cancers … where we the community will be able to make you immune to your own tumor," Ramsdell said. "We think we can do that now, based on some preliminary early data that looks quite promising. We want to make sure we understand the rules."

Immunotherapy is praised by researchers because it is targeted, focusing on cancerous cells rather than healthy tissue, as well as potentially durable. At the Parker Institute, the range of expertise, from pharmaceutical companies' commercialization efforts to academics' cutting-edge research, is what Ramsdell hopes will fuel its success.

Last summer the first-ever drug from Novartis using an immunotherapy treatment called CAR-T cell therapy was approved by the Federal Drug Administration . But while thousands of clinical trials on immunotherapy drugs, of which many are predicted to fail, are springing up, the depth of information on neoantigen vaccines is much shallower: Just two studies on neoantigen vaccines for melanoma, recently profiled in Nature , were completed last year in Boston and Germany.

"The companies who nominally could be competitors are really interested in trying to solve a problem, and I find that incredibly heartening," Ramsdell said of the Parker Institute.

The challenges of a cancer vaccine

Cancer is more complex to treat with a vaccine than viruses, like the measles, mumps or flu, and varies according to patient. Timing is another major challenge for cancer vaccines, because of the speed in which a malignancy advances. It's also difficult to identify a tumor versus a healthy cell, even with advanced technology.

"It's only recently that cancer researchers have regarded immunology and the immune system as just as important in cancer biology as mutations," said Charlotte Kuperwasser, who leads the Raymond & Beverly Sackler Convergence Lab at Tufts University. "It's more about the biology of cancer being much more complex than a simple vaccine against the mumps. They're doing this very strategically by trying to incorporate as much of the variability and mutational changes to make sure the vaccine is effective."

In 2015, researchers at the University of Washington's Cancer Vaccine Institute published a study on designing vaccines to prevent breast cancer. The trial on breast cancer followed decades of work by Mary Desis on the HER2 protein, typically found in breast cancer patients, according to co-researcher Sasha Stanton.

"When you have these low levels of disease, or no disease at all, it's the best time to get your body to destroy the cancer," Stanton told CNBC.

The goal of immunotherapy, researchers like Stanton say, is to target precancerous lesions or, of course, to prevent the disease altogether. Take the HPV vaccine for cervical cancer.

"Getting the sample from the patient is fairly straightforward, but once you get the sample, a protein is like a 200-line cord, and each line can have slight changes," said Manisit Das, a postdoctoral candidate in pharmaceutical sciences at the University of North Carolina. "It's really hard. Not all the predictions you make will be transferred into a real [vaccine] candidate."

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Melanoma has shown the most promising response to immunotherapy vaccines, according to researchers. Vaccines for breast cancer have been trickier to develop, Stanton said. Her team has struggled to understand how immunology really affects breast cancer compared to similar results on melanoma trials.

"I'm a breast immunotherapist. Of course, I've drank the Kool-Aid," Stanton said. "But I'd like to make this a more targeted therapy and take away some of the risk we currently have of wide blanketed approach."

At Mount Sinai the technique is to focus on developing as many neoantigens as possible.

"Not everybody does this exactly the same way," Bhardwaj said. "This alliance is working to determine, amongst many, many different sectors and companies, how do we come up with the best way to select these mutations and immunogens?"

— By Natalie Daher, special to CNBC.com

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