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Jeff Bluestone in 2012.

Jeff Bluestone in 2012.

Cindy Chew/CTSI at UCSF (CC BY-NC-ND 2.0)

Q&A: What can $250 million achieve? Chief of new cancer immunotherapy institute explains

Can a tech entrepreneur come up with a better way to do cancer research? That’s a gamble that Napster co-founder and billionaire philanthropist Sean Parker made this week when he put $250 million into a new institute for research on cancer immunotherapy. By comparison, the proposed 2017 price tag for Vice President Biden’s cancer moonshot effort is $680 million for the National Institutes of Health’s (NIH’s) National Cancer Institute. (That amount, however, would likely be an annual funding level continued in subsequent years.)

Parker wants to build on stunning successes with treatments that harness the immune system to fight cancer. Engineered immune cells called T cells and drugs called “checkpoint inhibitors” that make tumors visible to the immune system have beaten back tumors for years—but only in some patients with certain types of cancer. Parker says that expanding the reach of immunotherapy will take “a more entrepreneurial approach.”

His Parker Institute for Cancer Immunotherapy will create a network of centers at six cancer research centers: the University of California, San Francisco (UCSF); Memorial Sloan Kettering Cancer Center in New York City; the University of Pennsylvania; Stanford University in Palo Alto, California; and MD Anderson Cancer Center in Houston, Texas. (Another big player, Johns Hopkins University in Baltimore, Maryland, is not on the list, but received $125 million in gifts last month for its own center for cancer immunotherapy.)

Each Parker center will receive $10 million to $15 million this year, with more to come. The money will go for basic research, training programs, recruitment, and clinical trials, says Parker Institute CEO Jeffrey Bluestone, an immunologist at UCSF. In an unusual arrangement, the centers have agreed to share results from all of their cancer immunotherapy work, even projects funded by other sources, with the institute’s headquarters in San Francisco. In turn, researchers will have access to cutting edge technologies and money to explore new ideas without having to submit grant proposals and wait for months for funding.

The institute has chosen three initial focus areas; finding ways to make engineered T cells more effective; exploring why only some patients respond to checkpoint inhibitors; and searching for tumor antigens—proteins made by tumors that trigger an immune response—that could be used to make anticancer vaccines and better cell therapies.

In an interview, Bluestone brushed off skepticism from some scientists about the institute’s approach. His comments have been condensed and edited.

Q: The focus areas sound like things scientists are working on already. What is new here?

A: Within those areas are high-risk things that wouldn't necessarily get funded by the NIH. Things that might require novel technologies and tools. As one example, we could build a clinical grade cell sorter so that researchers can do cell therapy using a cell sorter that can get FDA [U.S. Food and Drug Administration] approval rather than the makeshift way we do it now.

Trying to knock down in a cell a couple of genes with CRISPR [gene editing tools] and express the receptor, these are not things that you can get funded by the NIH or industry because it’s very expensive and high risk.

A second aspect is being extremely collaborative. Everybody who’s part of the network shares everything. All data gets sent to a central repository so that we can compare experiments from site to site and clinical trial data from trial to trial. We have a model where the intellectual property is managed centrally so we can redeploy money.

The third element is to try to dramatically reduce the bureaucracy. We're not asking people to write a 25-page NIH grant proposal. We're trying to break down the barriers to collaborative clinical trials and aggregate the data and make it rapidly available.

Q: Didn't some of these groups collaborate already?

A: Of course there are situations where people have collaborated. We’re trying to bring it all together in one effort. Everybody here is collaborating. Everyone is deciding on the best possible high risk science together.

All the work that is done by the center in cancer immunotherapy becomes a part of the institute, whether that work is funded independently, by the NIH, or whether that work is outside the initial core, all of those get rolled up into this.

Q: Is there a timeframe for the institute?

A: The centers have committed to working with the institute for 7 years. But through a combination of fundraising and eventually evergreen funds that will come in through the intellectual property, we hope to create an even larger bolus of funding that we can distribute to these sites and potentially go to additional sites.

Q: Sean Parker said cancer is “hackable.” But the biology of cancer is complicated. Is it an engineering problem that can be solved with enough people and money?

A: There are some things in the field that could be more rapidly and effectively done as an engineering effort if we had the resources to do it, whether it’s finding ways to manipulate cells or more rapidly grow them up, or finding new ways to understand the pathway that you modify.

I think that the most important thing to remember is that we’re trying an experiment here. We're not trying to say we're the best, we're special, we're doing things that no one else has ever done or will do. We're just trying something with a bunch of really committed, passionate people who want to work together and see if we can make a difference. And hopefully we will.