Finally, a Nuclear Apocalypse Drug Gets Approved by the FDA

The Radiation and Nuclear Countermeasures Program is preparing for the worst.

Apr 23 2015, 12:00pm

Photo: Thomas Williams/Flickr

The US government is developing and stockpiling drugs to treat radiation poisoning in the event of a nuclear attack on American soil, and the first of them just got approved by the Food and Drug Administration.

Last month, the Radiation and Nuclear Countermeasures Program (RNCP) succeeded in getting filgrastim, a chemotherapy drug, approved for use in a nuclear emergency. Filgrastim, which was approved under the name "Neupogen," resulted in a 38 percent increase in survival in animals irradiated to the point of internal organ injury. It's also the first-ever drug approved for use in the event of a nuclear catastrophe.

The RNCP was formed in 2004 to fund basic research into drugs and treatments for severe radiation dosage, which can result in extreme illness and death. The explicit goal was to fill the Strategic National Stockpile—the US government's vault of emergency medication for outbreaks like Ebola—with medication that could be used in the event of a nuclear catastrophe, where a large portion of the population is left irradiated and at risk of dying.

"They would be used in a public health emergency," said Bert Maidment, director of the RNCP. "A terrorist event, a nuclear accident—something like that—or a nuclear detonation, God forbid."

The program is a product of the post-9/11 era, which stoked fear of international terrorism in the minds of policy makers, and a joint initiative between the White House Office of Science and Technology and the Homeland Security Council, which directed the National Institute of Allergy and Infectious Diseases (NIAID) to form the program under its auspices.

The program works by acting as a "virtual pharmaceutical firm," in that it doles out grants and contracts to universities and hospitals to undertake key research. The reason for this is that commercial pharma companies are unlikely to put money into developing a drug that will only be purchased by the government, and then given away for free only in the event of a far-flung emergency. It's not the most profitable endeavour.

"If your board of directors said you're going to spend all this money to develop a drug that's going to sit on a shelf and hopefully never be used, that's not a very good business model," said Maidment. "This program is to bridge that gap, or reduce that risk such that we set up our program so we can develop a drug from discovery from licensure."

"There are certain cases where a drug will have no use other than for a specific radionuclide, and there's no other clinical or medical need for it," Maidment added. "In that particular case, the government would need to fund everything from start to finish."

In this respect, the RNCP mirrors other government programs under the NIAID. When the ongoing Ebola outbreak began concerning Americans, for example, drugs were already under development and money was ready and waiting to be given to researchers who could help the cause, thanks to research programs that had already been looking into the deadly disease for decades. Private companies weren't even slow on the take up; they just weren't interested.

But research is only one part of what the RNCP does—as previously mentioned, they also have to get these drugs approved.

Since you need to bring someone close to the point of death with heavy doses of radiation in order to prove they work, the FDA had to make an exception for the program. The drugs it develops only need to be tested on animals, not humans. This presents yet another huge research hurdle: how do you make sure that a drug that works on a monkey will work on a person?

"So, the animal disease represents what could happen in humans," said Maidment. "What about the mechanism of action in the animal? Is the mechanism of that drug the same as what would happen in a human? We won't know that until something actually happens, God forbid."

After phase one safety studies in humans and extensive testing on non-human animals, Maidment said, "One can sit back and do an equation with irradiated monkeys, non-irradiated monkeys, and non-irradiated humans, and then solve for x, where x is irradiated humans."

Another drug the program is looking into—Neulasta, an analogue of Neupogen—resulted in a 46 percent increase in survival, Maidment said. If America ever goes nuclear, the Radiation and Nuclear Countermeasures Program will be ready.