Doomsday drug: Need for radiation sickness treatment a sign of scary times

Robert Mulroy.
Robert Mulroy.Jim Davis/Globe Staff

Maybe it should be called the Doomsday Drug.

As President Trump and North Korean leader Kim Jong Un trade insults — fueling fears of nuclear war — a local biotechnology startup is preparing to roll out a treatment for a public health threat that until recently seemed remote: radiation sickness.

Partner Therapeutics Inc., which was officially launched last week to find untapped uses for existing drugs, plans to announce Thursday that it has bought the global rights to Leukine, a decades-old cancer medicine. Executives at the Lexington company expect Leukine will soon win another approval, this one to treat radiation poisoning, and are betting that demand will soar globally because of escalating fears of a nuclear attack.


“You would hope you’d never need a drug like this for this kind of incident,” said Robert Mulroy, chief executive of Partner Therapeutics and former head of Cambridge-based Merrimack Pharmaceuticals. “But I think that, should an incident like that occur, being prepared for it is a very important thing.”

He sees a growing market for the drug both in the United States and overseas, including in the European Union, Japan, and South Korea.

Leukine has been approved since 1991 in the United States to bolster immune systems weakened by chemotherapy in patients with acute myeloid leukemia. But Partner Therapeutics, which has $60 million in venture capital to get it up and running, says recent laboratory tests found that rhesus monkeys were far more likely to survive high doses of radiation if injected with Leukine 48 hours after exposure.

Based on those results, the company, also known as PTx, expects the Food and Drug Administration to approve the drug by March 29 to treat acute radiation syndrome. Radiation sickness, experts say, could kill hundreds of thousands — if not millions — of people in a nuclear war.


With the Bulletin of the Atomic Scientists recently moving the minute hand forward on the symbolic Doomsday Clock to two minutes to midnight — closer to the apocalypse than it has been since World War II — scientists and scholars say it’s smart to prepare for the possibility of widespread radiation poisoning from a nuclear bomb.

“There’s no question that things have gotten tenser throughout the world — everyone senses that,” said Robert Rosner, a University of Chicago professor of physics and astronomy who chaired the Bulletin panel that determined it is two minutes to midnight. “Having stockpiles of things that are readily available is a sensible thing to do.”

PTx acquired Leukine for an undisclosed sum from Cambridge-based Sanofi Genzyme, which tested it on the monkeys and sought FDA approval for its use for radiation sickness. PTx has also bought a manufacturing plant in Lynnwood, Wash., just outside of Seattle, where Sanofi Genzyme makes the drug.

Sanofi Genzyme’s parent company, the giant French drugmaker Sanofi, said it sold Leukine to focus on developing other products. The list price of the medicine is $1,237 for a package of five vials.

Sanofi Genzyme has received more than $74 million since 2013 from the federal Biomedical Advanced Research and Development Authority to run the laboratory tests on the drug and to provide doses that the federal government is already stockpiling, according to the Department of Health and Human Services, which oversees the authority.

James Greenwood, who heads the Washington, D.C.-based Biotechnology Innovation Organization trade group and sits on a panel of experts that analyzes US defense capabilities against biological threats, welcomes PTx’s interest in Leukine.


“BARDA is quite aware, and so is the industry, that repurposing existing drugs is often a very good way to innovate a medical countermeasure,” Greenwood said, using the term for products that can be distributed in a public health emergency stemming from a nuclear, biological, or chemical attack.

Greenwood, a former Republican congressman from Pennsylvania, said he’s not especially worried that a drug to treat radiation sickness might be necessary.

“I’m 66 years old,” he said. “I ducked and covered under my desk when I was 7 years old. I’m long over being scared about it.”

In interviews with the Globe, PTx leaders stressed that they also acquired Leukine to treat other conditions besides radiation sickness. The drug has shown promise in patients being treated for melanoma with cutting-edge therapies to bolster their immune systems. And company officials say Leukine may have potential as a medicine for common neurological conditions, including Alzheimer’s disease.

But first they hope the FDA approves Leukine to treat acute radiation syndrome resulting from a nuclear disaster. That could be an atomic bomb detonated by a military power or terrorists, or an accident, like the 1986 Chernobyl nuclear plant disaster in Ukraine.

“If, God forbid, there was an incident, we’d make our entire operation available” to produce Leukine, Mulroy said.

A treatment would probably entail injections once a day for about a week, he said. Common side effects include bone pain, low-grade fever, flu-like symptoms, and loss of appetite.


The FDA in 2015 approved two other drugs to treat radiation sickness, Neupogen and Neulasta, made by Amgen Inc. The federal government paid Amgen $37.7 million to stockpile Neulasta, according to the Health and Human Services agency.

But Dr. Debasish Roychowdhury, a medical oncologist and former global head of oncology for Sanofi who serves as chief medical officer of PTx, said those drugs must be injected almost immediately after exposure to radiation to work.

In contrast, Leukine improved the chance of survival even when it was injected in monkeys 48 hours after exposure in laboratory tests in 2015 and 2016. That’s important, Roychowdhury said, because the widespread devastation wrought by a nuclear blast would make it difficult to quickly get the medicine to patients.

In addition, Leukine stimulated the production of multiple types of infection-fighting white blood cells in the bone marrow, he said, while Amgen’s drugs only stimulated one.

A spokeswoman for Amgen confirmed that its medicines stimulate one important type of white blood cell. She said the first dose should be given as soon as possible after exposure and the second one a week later.

PTx leaders said Leukine has already proved effective outside the lab. It was used to help save the lives of four people in the Brazilian city of Goiania after radioactive contamination in 1987.

They were among nearly 250 people found to have significant levels of radioactive materials in their bodies after being exposed to radioactive salt stolen from an abandoned hospital site.


Jonathan Saltzman can be reached at jsaltzman@globe.com