A budding U.S. biodefense industry — working largely without
the major pharmaceutical companies — is working on medical
countermeasures intended to treat deadly agents. But many of the
companies seeking government funds for biodefense work are small
firms with no experience in bringing a drug to market. They face
a significant challenge.
see an emerging new industry, new capabilities” in the medical
biodefense field, says Vidadi M. Yusibov, executive director of
the Fraunhofer USA Center for Molecular Biotechnology in Newark,
Del., who oversees work on vaccines and therapeutics administered
in edible plants.
Ninety-five U.S. companies are working on vaccines or therapeutics
to combat bioweapons, according to a 2005 report by BioAbility,
a provider of biotechnology- related information services located
in Research Triangle Park, N.C. However, a large portion of those
biodefense products are still just in the pre-clinical stage of
Since the 9/11 attacks, the federal government has dramatically
increased funding to help create such products. For instance, funding
for biodefense by the National Institute of Allergy and Infectious
Diseases (NIAID) increased from $3.2 million in fiscal year 2001
to an estimated $561.51 million in fiscal year 2005. Project Bioshield
legislation enacted last year provided $5.6 billion over the next
10 years to help generate medical countermeasures.
The president’s requested fiscal year 2006 budget calls for
$4.77 billion for civilian biodefense for the Departments of Homeland
Security and Health and Human Services, in addition to funds for
biodefense provided under Project Bioshield.
Experts say at least 50 biological agents are potential bioweapons.
The development of a medical response to protect against them is
an expensive and slow process. The estimated cost of creating a
new vaccine is about $800 million, and takes typically about 10
years or more.
Government officials have created priority lists of pathogens of
greatest concern, and further assessment of threats is under way.
The only licensed biodefense vaccines being manufactured in the
United States are against anthrax and smallpox, and each of those
has shortcomings that companies are working to overcome in new vaccines.
Only a few companies are working on biodefense countermeasures
as their only product. Instead, they frequently are drawing upon
work on their other products. For some, government funds available
for biodefense have helped ensure their survival after recent years
of bad economic times.
“I’m not yet convinced there is a sustainable industry
only in biodefense,” says Una S. Ryan, president and chief
executive officer of Avant Immunotherapeutics, based in Needham,
Mass., which is working on vaccine protection against anthrax and
plague. “I don’t think I would have set up a company
just for anthrax and plague.”
Her company has received commitments of about $10 million from
the Defense Department for its work on a next-generation injectable
anthrax vaccine and an oral vaccine that would protect against both
anthrax and plague.
Avant is drawing upon technology the company uses for other products,
including its protection against cholera. It employs genetically
modified bacteria as “buses” to deliver plague and anthrax
antigens to the immune system and stimulate it. Ryan says of its
work on biodefense vaccines, “We’re really manufacturing
a cholera bus or a salmonella bus with different soldiers.”
In Brisbane, Calif., VaxGen also is receiving federal funds. The
Department of Health and Human Services awarded the company an $877.5
million contract to produce 75 million doses of its next-generation
anthrax vaccine. Because it is being produced using modern manufacturing
techniques, expectations are that it will be purer than the existing
licensed vaccine. And it will take effect with fewer doses than
the six injections over an 18-month period called for in the current
VaxGen’s contract provides BioShield funds for delivering
doses of the vaccine starting early next year, even though the Food
and Drug Administration will not have approved it by then. This
and other unapproved products are being added to the Strategic National
Stockpile for use in the event of an emergency. The contract requires
that VaxGen continue developing the vaccine until its licensing.
In the meantime, the producer of the approved vaccine, BioPort
Corp. of Lansing, Mich., has a $122.7 million BioShield contract
to provide 5 million doses of its licensed anthrax vaccine.
Enough smallpox vaccine is now available for everyone in the United
States, and new smallpox vaccines are being created. Stewart Simonson,
assistant HHS secretary for public health emergency preparedness,
informed a congressional subcommittee that efforts are under way
for a next-generation smallpox vaccine.
Scientists are also working on countermeasures against other pathogens
considered to be among the most likely bioweapons including botulism,
tularemia, Venezuelan equine encephalitis and the Ebola virus as
well as genetically engineered threats.
Many of the involved researchers are in the forefront of medical
“Vaccines really are at a crossroads,” Ryan says, noting
improvements under way that could make them less expensive and faster
to produce as well as more stable in varying temperatures.
Companies are also working on different ways to administer vaccines
including using inhaled drugs, patches and multi-agent vaccines.
Ryan believes Avant might eventually be able to combine protection
against three pathogens in one dose.
“It’s very exciting and very interesting research,”
says Michael G. Kurilla, director of the Office of Biodefense Research
Affairs and associate director for Biodefense Product Development
for NIAID. “Small companies have gotten very innovative.”
Some experts are raising concerns over a “one-bug, one-drug”
approach because of the expense of developing countermeasures for
each pathogen, the logistics of stockpiling and administering them
and the potential adverse health effects from receiving so many
vaccines. Researchers are exploring alternatives, including non-specific
boosters of the immune system.
Companies are also working on a variety of other biodefense products
and services. For example, Logistics Health, with headquarters in
La Crosse, Wis., has been selling logistical support, including
overseeing anthrax vaccinations for military personnel and vaccine
and antibiotics for civilians exposed to anthrax in the 2001 mail
While many companies are struggling with navigating the bureaucracy
and finding the best way to seek support from federal agencies,
Logistics Health can draw upon the experience of former secretary
of Health and Human Services Tommy Thompson, whom it hired as its
president in February.
Detectors are also a growing market. For example, U.S. Genomics
of Woburn, Mass., has a $7.5 million contract from DHS to develop
a sensor that could detect and identify any airborne pathogen. DHS
also is seeking technology to detect infection through breath checks,
and a preservative that would neutralize anthrax spores in food
and treated clothing.
But despite innovative research and billions of federal dollars,
few new medical countermeasures have yet to be produced. “It’s
too early to see massive industry change,” says Mark D. Dibner,
president of BioAbility. “Although there are a few things
on the market, most of these things are in the early stage of development.”
Critics are calling for more results. They question the speed at
which the government is signing up contractors.
Michael Greenberger, director of the University of Maryland Center
for Health and Homeland Security, criticized the time it is taking
for DHS to determine the agents that are a threat to the United
States, a step required before other agencies can spend BioShield
“DHS has employed an opaque, highly bureaucratized, relatively
lengthy process for determining material threats,” he charged
in a written statement.
Experts disagree on which agency should have the lead in providing
medical countermeasures to protect civilians. Some maintain it should
be HHS while others say Homeland Security should have the major
procurement role because they see HHS as having a contentious relationship
with the industry.
Critics have raised concerns about the progress in the development
of medical countermeasures to protect military personnel. The Defense
Department is continuing work on medical countermeasures with its
own researchers and its Joint Vaccine Acquisition Program, with
DVC, of Frederick, Md., as its prime contractor. But with the growing
concern over protection of civilians from potential bioterrorism,
other departments, especially HHS and Homeland Security, are also
now playing significant roles.
Last year the Institute of Medicine issued a report calling for
the creation of a new Medical Biodefense Agency within the Defense
Department to handle military activities and help avoid fragmentation
of responsibilities. It urged that the Medical Biodefense Agency
focus on unique needs for military protection and coordinate its
efforts with the National Institutes of Health to minimize redundancies.
Other concerns are being raised by doubters who wonder whether
the small biotech companies have the funds, experience and infrastructure
to get the job done.
Eventually many of the smaller companies may partner with large
ones. Authorities are exploring options to try to lure large pharmaceutical
companies to participate and help speed up the process.
The problem is not new. The Defense Department faced similar resistance
during the first Persian Gulf War when it tried to get companies
to produce vaccines to protect against biological weapons. Efforts
extended even to those making animal drug products, but BioPort
emerged as the sole producer of the anthrax vaccine.
Thus far, large companies have shown reluctance to produce biodefense
products for civilians. Vaccines don’t offer the same promise
of profits as lifestyle drugs or medicines for chronic diseases,
which are taken repeatedly throughout long periods of time.
“There’s a real lack of development of drugs and vaccines
for infectious diseases of all types,” says Brad T. Smith,
a University of Pittsburgh medical professor and an associate at
the school’s Center for Biosecurity.
In addition, companies worry about having the government as the
only customer. They point to the government’s treatment of
Bayer AG. The company was pressured to sell its Cipro antibiotic
at a discount after the 2001 anthrax incidents.
Alan Goldhammer, associate vice president of the Pharmaceutical
Research and Manufacturers of America, sees “a lot of technical
and regulatory hurdles.” Among his main concerns is the liability
the companies might incur in producing biodefense products. Though
the Safety Act would provide liability protection during a bioterrorism
emergency, it does not cover products administered before an attack.
Goldhammer views biodefense vaccines as especially risky. Because
it is considered unethical to expose trial participants to a disease
that generally does not occur naturally in the United States, researchers
may now use animals to test the drugs. As a result, Goldhammer says,
people might be vaccinated during an emergency with a drug that
has not been tested for effectiveness on humans.
“The challenge of liability protection is potentially very
expensive,” Smith says. Private insurers are reluctant to
provide coverage. It is among the issues Congress is struggling
with as it considers proposed BioShield legislation that supporters
hope will encourage large companies to participate in medical biodefense.
Goldhammer says his other main concern is that antitrust regulations
prevent companies from working together in responding to the challenge.
Many of the industry, academic and government leaders who were interviewed
during a 2004 study conducted by the Center for Biosecurity and
the Sarnoff Corp. of Princeton, N.J., said they would like to see
a single organization created to facilitate collaboration.
According to the Biosecurity and Bioterrorism journal, “the
measures the U.S. government has taken to date, including the passage
of the BioShield legislation, will not be enough to entice pharmaceutical
industry leaders into that field and will not produce the countermeasures
the nation needs for a truly effective biodefense.”
Margaret Davidson is a medical journalist and a professor at the
University of Wisconsin-Oshkosh.