In all the publicity surrounding the "moonshot" for cancer announced recently by President Obama, there was little attention given to another huge cancer research project, which is also shooting for the moon.
The other shot at the moon was announced, albeit with a little less fanfare, by Patrick Soon-Shiong, MD, founder and CEO of the biotech firm NantWorks. At a press event in San Francisco held on January 11, he laid out provocative plans for the launch of the National Immunotherapy Coalition (NIC), which will form the basis of Cancer MoonShot 2020.
"Sometimes people do things and it creates exponential change in mankind," Dr Soon-Shiong said during the press event. "I truly believe with the advent of whole-genomic sequencing proteomics, that we can truly move cancer and lead ourselves to a cure."
In a statement, he pointed out that the "era of immunotherapy has taken the oncology world by storm. For the first time in 40 years, there is a glimmer that we may be able to win this war against cancer."
Large pharmaceutical and biotech companies are developing dozens of agents to activate the immune system, he explained. "The problem is that while these drugs are being developed individually in silos by each entity, they need to act together when it comes to activating the immune system. If we follow the current path of drug development, it may take 40 or 50 years before we have worked out the right cocktail combination and countless lives will be lost as a result of this inefficiency."
He has collected many of these efforts into a coalition that has a single focus: to accelerate the potential of combination immunotherapies as the next-generation standard of care for cancer patients.
The hope is to get competing drug and biotech companies to work together on combination therapies and share data, rather than developing and testing their own compounds in isolation.
To that end, the NIC plans to design, initiate, and complete randomized clinical trials in cancer patients, at all stages of the disease, in up to 20 tumor types, and to have as many as 20,000 participants in randomized phase 2 trials by 2020.
These findings will inform phase 3 trials, with the goal of developing an effective vaccine-based immunotherapy to treat cancer.
Thus far, the NIC network includes large pharmaceutical companies, such as Celgene and Amgen, and biotech companies, such as NantWorks, NantKwest, Etubics, Altor Bioscience, and Precision Biologics. Participating academic cancer centers include the Cancer Center at the Beth Israel Deaconess Medical Center in Boston, Columbia University in New York City, the University of Utah in Salt Lake City, Tufts Cancer Center at Tufts Medical Center in Boston, and the University of Arizona in Tucson.
In addition, this alliance includes Independence Blue Cross, one of the nation's largest payers and the first major insurer to offer reimbursement for next-generation whole-genome sequencing, which is an integral part of the envisioned clinical trial program.
As for the cost of bringing 2020 to fruition, no estimates or hard numbers have been released. But a full-genome sequencing costs around $30,000 and a panel of about 300 genes costs around $9000, which can get very pricey for 20,000 patients. Independence Blue Cross has said it will cover genomic testing for its members and Dr Soon-Shiong has reportedly promised to help cover funding from his own foundation, the Chan Soon-Shiong Family Foundation. In addition, Bank of America, one of the largest self-insured companies in the United States, has partnered with the coalition.
In his speech about the cancer moonshot, President Obama highlighted the fact that Congress had agreed to a $264 million funding boost for the National Cancer Institute.
Shooting the Moon in Unison?
The launch of two giant cancer initiatives aimed at shooting for the moon undoubtedly has invoked confusion among the general public and healthcare professionals alike. Are these separate launches, or are they part of the same medical space race?
The answer to that question is yes and no. The vice president is not directly involved in Cancer Moonshot 2020, and Dr Soon-Shiong is not seeking federal funding, but there is definitely an intermingling of minds and ideas.
This past December, a meeting of the NIC was held at Vice President Biden's Naval Observatory in Washington, DC. "Mr Biden had asked Patrick Soon-Shiong — who he had previously been in contact with regarding his son's health — to convene a meeting of experts to discuss his own moonshot, and also as a way for Mr Biden to learn about all the different aspects of advancing cancer therapy as part of his own initiative," said Mark C. Poznansky, MD, PhD, director of the Vaccine and Immunotherapy Center at Massachusetts General Hospital in Boston.
"The vice president said that he had met with about 200 experts, but only about 15 to 20 were convened by Patrick," said Dr Poznansky, who is involved with the NIC coalition. "I would say that we were part of a bigger national mission, and Patrick's mission is concordant with that. It has components that are part of it and has components that are separate from it."
The fact that it is a great mission means that other factions must be involved, Dr Poznansky told Medscape Medical News. "It has government involvement, but it has to have industry involvement and academic involvement as well."
Dr Poznansky said that what he finds most interesting about Cancer Moonshot 2020 is that it had all of those components from the get-go. It has "government, industry, payers, insurance, the FDA — which I believe are all stakeholders in the ultimate quest to find better therapies for cancer."
As for potential cross pollination, Dr Poznansky believes that is a distinct possibility. "This is like anything when you have a complex problem to solve," he said. "There may not be one single approach, but instead many approaches."
A Man on a Mission
Dr Soon-Shiong, who was born and raised in South Africa during the apartheid era, is a surgeon, medical researcher, businessperson, philanthropist, and professor at the University of California, Los Angeles. He is currently chair of the Chan Soon-Shiong Family Foundation and chair and CEO of the Chan Soon-Shiong Institute for Advanced Health, National LambdaRail, the Healthcare Transformation Institute and NantWorks, LLC.
His resume is impressive. He has pioneered novel therapies for both diabetes and cancer, published more than 100 scientific papers, and has more than 95 issued patents. Dr Soon-Shiong performed the world's first encapsulated human islet transplant, the first engineered islet cell transplant, and the first pig-to-man islet cell transplant in diabetic patients. In cancer circles, he is best known for inventing and developing nab-paclitaxel (Abraxane, Abraxis Pharmaceuticals), the first protein nanoparticle albumin-bound delivery technology for the treatment of cancer approved by the US Food and Drug Administration. He founded two pharmaceutical companies in the United States, Abraxis and American Pharmaceutical Partners, which he subsequently sold for a combined $9.1 billion. According to a report published in Forbes, Dr Soon-Shiong has an estimated net worth of $12.2 billion.
He is also a member of the Buffett–Gates Giving Pledge, and plans to give away at least half of his fortune to charities. Dr Soon-Shiong has already donated $136 million to St. John's Health Center in Santa Monica, California.
However, in addition to his fame for medical accomplishments, he has also generated a degree of controversy. Some have questioned his business practices, and his research has occasionally clashed with scientific caution. Two years ago, for example, Dr Soon-Shiong was featured in a segment of the popular news show 60 Minutes as the doctor who "wants to disrupt the conventional way we treat cancer" and as a researcher who is "overflowing with ideas on how to do it."
The news show reported that Dr Soon-Shiong had created an infrastructure to enable his researchers to map the entire genome of individual tumors, a process that once could have taken months but with new supercomputers can be done in a day. The idea is to classify cancer by its mutations, and then to treat each one with a specifically targeted therapy.
Targeted therapy, although not a new concept, is one of the up-and-coming goals of cancer therapy, and Dr Soon-Shiong believes that his method is ready for prime time and can be used in mainstream medicine now. However, others do not think it's entirely proven. "There's just too much hard, complex science that has to be done before this is state of the art," Derek Raghavan, MD, president of the Levine Cancer Institute in Charlotte, North Carolina, said on the 60 Minutes segment.
Cancer Moonshot 2020 is a grand extension of this. Its aims are to use genomic sequencing to match specific drugs with specific patients and to continue to sequence DNA during treatment to gain a better understanding of how cancer cells mutate.
Inspiration for the Moonshot
Although the two moonshots may not be working together, at least for now, there were no real surprises in either of the announcements. The relationship between Joe Biden and Patrick Soon-Shiong began way before the State of the Union address, at a time of deep despair for the Biden family. While his oldest son Beau was battling a recurrence of brain cancer, the vice president reached out to Dr Soon-Shiong, who subsequently flew to Washington to meet with the Bidens.
This would turn out to be the first of a series of meetings between the two men. Unfortunately, Dr Soon-Shiong could do nothing for Beau Biden, who died last year from brain cancer at the age of 46, but the meetings appear to have inspired the vice president to take on cancer as a cause celebre. According to a report published in the New York Times, it was Dr Soon-Shiong who coined the phrase "moonshot" in describing the next offensive against this insidious disease.
Vice President Biden used the term when he announced that he would not be seeking the presidential nomination, which was 3 months before the State of the Union address. He noted at that time that he believed "that we need a moonshot in this country to cure cancer." Just a few weeks before his speech, the two men met in the White House, where Dr Soon-Shiong gave the vice president a two-page outline of what he had in mind as far as his cancer initiative.
As a foundation for Cancer MoonShot 2020, a master clinical trial protocol, known as QUILT (Quantitative Integrative Lifelong Trial), is being designed to incorporate a broad range of immune system components (including dendritic-cell, T-cell and NK-cell therapies). These components will be synergistically integrated through the evaluation of novel combinations of cancer vaccines, cell-based immunotherapies, low-dose metronomic chemotherapies, low-dose radiotherapies, and synergistic immunomodulating agents in patients who have undergone next-generation panomic molecular fingerprinting (whole-genome, transcriptome, and quantitative proteomic analysis).
The goals are to help these patients achieve durable and long-lasting remissions, and to provide the highest efficacy with the lowest toxicity and the best quality of life. With support from participating members, patients participating in the QUILT trial will have access to more than 60 therapeutic agents.
Blueprint vs Launchpad
Although it does seem that the two moonshots are on the same basic path, with similar goals, they are at dramatically different stages of development. To use a space-race analogy, it would seem that Vice President Biden's spaceship is only at the blueprint stage, waiting for construction to begin, whereas the NIC's is already sitting on the launchpad waiting for the countdown.
Vice President Biden is at the beginning of the process; he is meeting with experts, examining the logistics of collaborations, and plotting the direction of a new cancer plan. The NIC is there already; Dr Soon-Shiong has already put together an extensive collaboration of industry, academics, community oncologists, and a medical insurance company.
"I think the two [moonshots] will ultimately feed into each other," said Dr Poznansky. "It's almost inevitable, quite frankly."
It's all about everybody working together to achieve this goal, and this is where the moonshot comes in, Dr Poznansky continued. "Going to the moon is not just about the astronauts; it's about the physicists, the astrophysicists, the engineers, the accountants, the workers building the modules. There was an enormous amount of expertise that came together to do that," he said. "I think that gets neglected. When we apply that to cancer, we think of the oncologists and researchers in academia making breakthroughs, but it is really much more than that."
"A comprehensive approach and vision are needed. I think that Patrick Soon-Shiong's work, and that of Vice President Biden, requires a complicated coordination among all of these stakeholders," Dr Poznansky explained. "A single goal and multiple stakeholders. Everyone has a different motivation for wanting to be part of it, but in the end, everyone has a part in it."
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Cite this: Shooting for the Moon: The New Cancer Space Race - Medscape - Jan 28, 2016.