$1B Effort Yields No Bioterror Defenses

Mass. labs in line to join scaled-back Pentagon program

By Bryan Bender
Globe Staff

WASHINGTON — The Pentagon is scaling back one of its largest efforts to develop treatments for troops and civilians infected in a germ warfare attack after a $1 billion, five-year program fell short of its primary goal.

Even the heavy infusion of research cash and a unified effort by university labs and biotech companies from Boston to California were insufficient to break through limitations of genetic science, according to government officials and specialists in biological terrorism.

Instead, the Pentagon’s next $1 billion for the Transformational Medical Technologies program will focus on better ways to identify mutant versions of Ebola, Marburg, and other deadly viruses. Those are among the genetically modified agents that officials fear could be used by terrorists or rogue states against urban or military targets.

The continued flow of money, even with the shift in strategy, should help Massachusetts and other states retain jobs and research labs focused on this arena.

“There is tremendous potential for further development of a biodefense subcluster in the state,’’ said James D. Rooney, vice president of the Massachusetts High Technology Council.

Among Bay State firms that have received contracts under the germ warfare effort is Worcester-based Microbiotix. Representatives from Microbiotix did not respond to requests for comment.

The new strategy represents a return to the drawing board for an ambitious program conceived after the Sept. 11 terrorist strikes and subsequent mailing of anthrax to members of Congress and media organizations — events that helped US military planners realize that the nation lacked adequate defenses against bioterrorism.

Scientists initially set out to develop new medicines capable of attacking viruses that might be altered by terrorists to make them more deadly. But after more than 50 research projects by more than 100 contractors — including biotech firms, pharmaceutical companies, and universities, including several in the Boston area — only two experimental medicines have shown promise. And even those are far from being ready for limited clinical tests, according to project officials.

“They are trying to come up with new medical technologies that are more difficult to develop,’’ said Crystal Franco, a specialist at the Center for Biosecurity at the University of Pittsburgh Medical Center who specializes in biological defense policy. “They are really trying to push the envelope.

Another hurdle in the government’s effort: such treatments cannot be tested in human clinical trials, which are typically required for Food and Drug Administration approval, because it is unethical to expose people to deadly virus in such a study, requiring animals with similar traits as humans to serve as surrogates.

Alan S. Rudolph, director of science and technology at the Defense Threat Reduction Agency, said in an interview that the agency will now focus more attention on ways of identifying new pathogens. That research could lay the groundwork for further advances in the development of antidotes that could eventually win FDA approval.

The new focus of the program will be making a “cadre of investments that are able to take an unknown sample that may contain different agents, and be able to determine very quickly what is in there,’’ Rudolph said. “It is our intent to continue to grow this capability.’’

He added the ultimate goal will still be to someday develop therapeutic remedies that could treat someone infected with any number of deadly viruses — what the Pentagon called “one size fits all’’ or “one drug, many bugs.’’

In addition to Ebola and Marburg, some of the potential biological threats on the Pentagon’s target list are Lassa, Sabia, Machupo, and Junin, especially modified versions designed to cause more severe symptoms of hemorrhagic fever that are more resistant to traditional drugs.

The difficulty in developing medicines so far, however, demonstrates how much more research is needed, say biological warfare specialists.

It turns out it is easier to modify a germ or virus for an offensive threat than it is to develop an effective defense, they said.

“The offensive capabilities outrun the defensive capabilities as the march of biology continues,’’ said Richard J. Danzig, a former Navy secretary and noted expert on bioterrorism who sits on the Pentagon’s high-level Defense Policy Board.

“The theory behind [the program] was these same advances should empower the defenses,’’ he said. “I think that intuition is worth exploring and investing in, but it is easier to conceive than to execute.’’

Margaret Kosal, an assistant professor at Georgia Tech who worked on the program between 2006 and 2007, said “there is a fundamental need for basic science. The low-hanging fruit has all been picked.’’

One Pentagon contractor involved in the program who was not authorized to speak publicly put it more bluntly: “We’re years away from any reasonable FDA certification, let alone production.’’

Franco said the project’s hurdles also highlight the need for ongoing taxpayer-investment commitments from government, to encourage private-sector focus on such technologies that will generate little in sales, compared to, say, cholesterol and diabetes treatments.

“These are not going to be blockbuster drugs,’’ said Franco. “It is different when the government is your only market. There needs to be incentives for companies to participate, to take it on for the public good.’’

Bryan Bender can be reached at bender@globe.com.