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Medical Space Race: Inside The Two Moonshots To Cure Cancer

Key players agree that cancer cures are within reach. Two major, sometimes overlapping, efforts are underway—one public and one private.

In his final State of the Union address, President Obama announced a "moonshot" program to cure cancer. Obama named Vice President Joe Biden, whose son Beau died from brain cancer last year, to lead the government effort, which could even have bipartisan appeal. In 2015, House Republicans and Democrats overwhelmingly passed the 21st Century Cures Act, which raises funding and lowers barriers for audacious medical research. (The Senate is considering its own version.) The bill's sponsor (and Chairman of the House Energy and Commerce Committee), Fred Upton, tweeted his support for the Obama-Biden effort.

Theirs was the second cancer moonshot announcement of the week. On Monday (January 11), Patrick Soon-Shiong, M.D., founder and CEO of biotech firm NantWorks, announced at a JP Morgan Health conference the launch of Cancer MoonShot 2020, an ambitious program to get new anticancer immunotherapies approved by the end of the decade. And the afternoon before Obama's address, Soon-Shiong convened a press conference of pharma and biotech CEOs, physicians, and others to announce their participation in the program.

Obama's Moonshot and Soon-Shiong's MoonShot share supporters, and Soon-Shiong has advised Biden on cancer treatments, beginning with a discussion of Beau Biden's own case in the winter of 2015. The two programs also share the same philosophy: Personal genetic sequencing will enable more effective, customized cancer treatments.

This breaks from the typical one-size-fits-all approach: high doses of general chemotherapy and radiation. "You'd find something from the Amazon rainforest [for example], you purify it, and it kills cancer cells in a dish," says William Nelson, M.D., director of the Johns Hopkins Sidney Kimmel Comprehensive Cancer Center. "And then the game in clinical development was, how much could you give a person…that you could kill the cancer cells without hurting them too badly." One side effect is weakening the patient's immune system.

Soon-Shiong wants to focus instead on boosting the immune system to fight cancer, in combination with lower doses of chemotherapy and/or radiation. "Is immunotherapy-based treatment going to give you much better quality of life and better outcomes than high-dose chemotherapy?" he asks. He's not alone. "The immune system might be better unleashed by cancer cells," says Nelson, noting successful efforts to goose the body's own defenses against such nasty cancers as melanoma.

While the two men agree on much of the science, there is tension between the research community, the government, and private-sector efforts like Soon-Shiong's. "He's gotten rich, and I'm glad he did, but that's not our goal," says Nelson. And using essentially the same name for Soon-Shiong's program has caused confusion. "Maybe the term moonshot has been utilized by a lot of people over time," says National Institutes of Health director Francis S. Collins. "Make no mistake, what the Vice President announced…is not the same thing." Yet Soon-Shiong has become inextricably linked with the Obama-Biden initiative because he has created such media buzz, pulled in support from hefty players including corporate CEOs and leaders of cancer centers, and claimed a significant role influencing the Vice President's education on the issues.

Shooting for the Same Moon

Fundamental to immunotherapy and other new treatments is custom genetic sequencing. "All cancers are disorders of acquired defects in genes," says Nelson. Genetically, no two cancers are exactly alike, he says. Knowing a patient's specific mutations may allow doctors to target the malformed proteins that cause the cancer. With this knowledge, doctors can also develop custom vaccines to teach the immune system to attack a patient's unique cancer the same way it would attack a virus like influenza, says Soon-Shiong. His MoonShot aims to enroll 20,000 patients, covering 20 types of cancer, in clinical trials of immunotherapy treatments and get as far as phase III—broad-based testing—within three years, with the goal of getting approved treatments by 2020.

Vice President Biden hit the same themes in a posting on Medium published just before the State of the Union address. "Several cutting-edge areas of research and care — including cancer immunotherapy, genomics, and combination therapies — could be revolutionary," he writes. Soon-Shiong sees the White House program jibing with his own. "The fact that President Obama has adopted Vice President Biden's recommendations to go down this path of immunotherapy and lower-dose chemotherapy and genetic testing is absolutely the right one," he says.

Biden, Collins, Nelson, Soon-Shiong and many others are working from the same scientific consensus and talk with many of the same people, sometimes with each other. "I had the opportunity on October the 6th [2015] to visit with Vice President Biden in the White House and shared with him a white paper of exactly this plan, called the MoonShot," says Soon-Shiong. (The plan was leaked, says Soon-Shiong, leading to a New York Times article about Soon-Shiong's influence on the VP.) It was neither their first nor their last meeting. However, the Vice President has met with about 200 experts and philanthropists in dozens of meetings, his office points out, including top people from cancer centers at Memorial Sloan Kettering, The University of Texas and Massachusetts General Hospital.

A few weeks after that meeting with Soon-Shiong, in an October 21 White House Rose Garden address, Biden announced that he would not run for President but would focus other priorities. "And I believe we need a moonshot in this country to cure cancer. It’s personal. But I know we can do this," said Biden. "The President and I have already been working hard on increasing funding for research and development — because there are so many breakthroughs just on the horizon in science and medicine. The things that are just about to happen, we can make them real with an absolute national commitment to end cancer as we know it today."

The National Institutes of Health gave a rough outline of the Administration's plan two days after the President's address in a phone conference with reporters. It focuses on genetically tailored treatments, with a big role for immunotherapy.

More whole-genome sequencing will be key, and it's looking more likely to happen. Independence Blue Cross, with about 10 million subscribers, will be the first health care provider to cover the tests, said its president and CEO Daniel Hilferty at the MoonShot press conference. At the same time, Bank of America, which provides its own health insurance for employees, announced that it would also cover personal sequencing. The CEOs of both corporations are members of MoonShot 2020.

The next step is to unify research as well as treatment programs. "Innovations in data and technology offer the promise to speed research advances and improve care delivery," wrote Biden on Medium. "But the science, data, and research results are trapped in silos, preventing faster progress and greater reach to patients. The NIH, which includes the National Cancer Institute (NCI) got a funding boost in its 2016 budget (the biggest in more than 10 years), which it will use to support more research. It can also require organizations it funds, such as universities, to share their research, said NIH director Collins. And NCI is building a cloud database to hold up to 50,000 anonymized patient records, including their full genetic workups.

"Not just doctors, but patients, doctors, academia, industry, institutions, government, philanthropy—we all have to work together," said Azra Raza, M.D., Director of MDS Center at Columbia University (covering rare blood cancers), during the MoonShot 2020 conference. She and other members of MoonShot 2020 met with Vice President Biden on December 1, along with senior government officials such as Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research. "For the first time, we got the right people at the table in front of the Vice President—the FDA, NCI, academics, oncologists and pharma to all say one word: we will work together," says Soon-Shiong. The FDA does already have some tools to fast-track treatments: A 2012 law allows it to use Breakthrough Therapy Designation to accelerate the evaluation of drugs for serious or life threatening diseases.

Soon-Shiong says he will be submitting clinical trial designs of immunotherapies for several types of cancers to the FDA within 6 weeks, based on a MoonShot 2020 scientific symposium held January 15-17. The meeting included representatives of several major biotech companies as well as scientific investigators from the NCI. The Institute, provides a caveat, however, saying in an email to Fast Company, "The participation by NCI investigators should not be taken in any way as endorsement of the coalition that Dr. Soon-Shiong has recently announced."

I ask Soon-Shiong if his MoonShot could be a duplication of the government's Moonshot, running a parallel path. "It's not a competition," he says. "Whatever they [the government] are doing will be fantastic."

All the players agree that treatments have to get to patients sooner, and that could be an important measure of success from any efforts. Biden, Soon-Shiong and others cite the statistic that only around 5% of cancer patients make it into clinical trials. "Having more people on clinical trails with cancer is a really key issue," says the NCI's acting director, Douglas R. Lowy, M.D. "It's also important for people all over the country to have access to the latest, first-quality treatment available." Soon-Shiong constantly hammers that point, as well, as did Biden in his post on Medium: "It’s not just about developing game-changing treatments — it’s about delivering them to those who need them."

This article has been updated to reflect the role of NCI employees in the recent MoonShot 2020 meeting.

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