A handful of pharmaceutical companies, including local biotech giant Amgen, are joining together to test cocktails of their latest cancer drugs in an ambitious initiative aimed at rapidly developing new treatments — but one that's gotten off to a shaky start.
Cancer MoonShot 2020 was announced Monday by Dr. Patrick Soon-Shiong, a billionaire Los Angeles oncologist who is spearheading the program, at a healthcare investor conference in San Francisco.
It calls for conducting dozens of small-scale clinical trials with as many as 20,000 patients over the next three years, followed by larger-scale trials. Its focus is the emerging field of immunotherapy, which seeks to use the body's own immune system to fight cancer.
Aside from Amgen, confirmed participants include Celgene in New Jersey and several smaller firms, which will make their cancer therapies available for the trials. Soon-Shiong hopes up to 10 drug companies ultimately will participate.
Although firms often cooperate to test drug combinations, W. Martin Kast, a professor of microbiology and immunology at USC's Norris Comprehensive Cancer Center, said the scope of the program, if realized, could help speed the development of treatments by bringing together an array of experimental therapies.
"The surprising thing to me is the buy-in of so many partners," said Kast, who is not involved in the effort. "There will be 60 drugs available that can now be tested. That has never happened."
But the alliance of drug companies was uncertain ahead of Monday's announcement. Three global pharmaceutical companies that Soon-Shiong's camp initially said would participate — Pfizer, Merck and GlaxoSmithKline — said they had not signed on.
There is also confusion over the role of the federal government's National Cancer Institute in the program.
Representatives for Merck and GlaxoSmithKline said the companies support the project's goals but are still considering whether they will join.
Pfizer spokesman Dean Mastrojohn said the company supports efforts that "bring together the health innovation ecosystem" but is not prepared to participate until it has negotiated its own research agreement with the National Cancer Institute. It also wants to learn what role the agency will play in the program, including determining which drugs will be tested for specific cancers.
Institute spokeswoman Cynthia Meals Vitelli said the agency has no formal involvement at the moment.
Soon-Shiong insisted the project has the "enthusiastic" support of researchers at the institute with whom he has spoken to and met. He said the confusion and hesitation by Pfizer, Merck and GlaxoSmithKline are the product of the bureaucratic nature of drug research.
"What you may call a misunderstanding is the administrative level of paperwork that needs to happen," he said.
Dr. Edward Garon, an oncologist at UCLA's David Geffen School of Medicine, said the confusion isn't surprising given the project's scope.
"These are exciting approaches, but there are logistical difficulties," he said. "The complexity is quite significant."
Soon-Shiong's involvement also could be controversial. He's an astute businessman who Forbes estimated has a net worth of $12.4 billion, but he's been criticized for making inflated claims about his work. A recent disputed boast is that one of his firms, NantWorks, can analyze a human genome in 47 seconds.
Soon-Shiong said he's accustomed to such criticism, which he sees as a result of his ambition.
"The fact that there are doubters means it's big and important," he said.
Cancer immunotherapy has become a hot field in recent years, with pharmaceutical companies pouring billions of dollars into the development of treatments that boost or reprogram the immune system to fight off tumors.
USC's Kast said he and other researchers believe such treatments probably would be more effective in combination with one another, boosting the immune system in several ways at once. But there has been limited research on such combinations.
The MoonShot program calls for experimenting with mixtures of immunotherapies as well as low-dose chemotherapy and radiation, targeting as many as 20 types of cancer, including breast, lung, prostate and pancreatic.
"Immunotherapies have demonstrated remarkable benefit, but combining them with chemotherapy is likely to produce even more dramatic benefit," said Mark Alles, president of Celgene, which acquired Soon-Shiong's Abraxis drug development company in 2010 for $2.9 billion.
Soon-Shiong estimated the effort could cost billions of dollars, with much of that covered by insurers and drug companies whose products will be studied.
Kast said that even if the project is fully funded, its goal of creating personalized cancer vaccines by 2020 sounds optimistic.
"The vision is great, but it's hard to imagine you would be successful within three or four years," he said.
The program could benefit two of Soon-Shiong's Southland companies. It could validate drug candidates being developed by NantKwest, a company he acquired in 2014, and show the large-scale efficacy of NantWorks, which combines rapid genetic testing with medical records and other information to help doctors make treatment decisions.
Insurers will pay NantWorks for the genetic testing in the clinical trials and the information will be used to select treatments.
Soon-Shiong said the MoonShot program came together over the last year after meetings with other healthcare executives and Vice President Joe Biden.
The Biden family consulted with Soon-Shiong before Biden's son, Beau, died of brain cancer in May. Soon-Shiong said he met with Biden in October and presented the vice president with a report outlining the MoonShot initiative. Biden aides said the vice president is not connected to MoonShot.