A Rockville biotechnology company plans to announce today that it has
received government permission to launch human tests of a drug designed to
combat anthrax, the biowarfare agent that killed five people and terrified
Congress in 2001.
Human Genome Sciences Inc. appears to be ahead of several competitors to
develop such a compound. Its drug, Abthrax, could be submitted to the Food and
Drug Administration for approval in months, presenting the Bush administration
with a test of its stated commitment to buy anti-terrorism drugs if industry
takes the trouble to develop them.
The drug is a synthetic antibody, a type of protein similar to antibodies
produced by the immune system, that neutralizes the main toxin produced by the
anthrax germ. Anthrax can already be treated with antibiotics, but drugs like
the one Human Genome Sciences is developing might be useful as an adjunct to or
replacement for antibiotics in several situations.
The drug is on an unusually fast path.
Anthrax is rare as a naturally occurring disease, and, because it's
potentially fatal, humans can't ethically be exposed to it in tests. That means
the only test available to show the new drug works is in animals. Human Genome
Sciences said the drug has already passed that test, showing marked improvement
in survival for animals that received the new drug and were exposed to anthrax
spores.
The company has now received FDA permission to test the drug in people, but
only to see if it can be administered safely. The test subjects won't be exposed
to anthrax spores. The initial safety tests, in scores of healthy volunteers,
are likely to go rapidly.
But William A. Haseltine, chairman and chief executive of Human Genome
Sciences, said the company won't proceed to the more expensive final phases of
safety testing unless it's clear the government is willing to buy the drug. "It
has to make economic sense, or we will not proceed with it," he said.
Under new legislation, drugs like this may win FDA approval based only on
animal studies of their effectiveness, coupled with human safety tests. That
means the drugs may well enter national anti-terror stockpiles without doctors
having a clear fix on how to use them.
Theory suggests that an anthrax antibody, like Abthrax, may be useful in
several situations. If terrorists create an anthrax strain resistant to
antibiotics, an antibody might be the only treatment for exposed people.
An anthrax vaccine is available, but it requires multiple shots over months.
By contrast, an antibody injection would in theory confer instant immunity, so
it might save the lives of soldiers or workers at risk of exposure to the germ
who haven't already been immunized.
Finally, a high proportion of people who develop anthrax symptoms die in
spite of receiving antibiotics. The antibiotics attack the germ itself, but not
a toxin that it releases into the blood. Drugs like Abthrax are specifically
designed to attack the toxin, so they might, in theory, save the lives of people
who are not responding well to standard anthrax treatment.
Exactly how well the drug would work may be answered only if there's another
terrorist attack with anthrax, like the ones in 2001 that emptied congressional
and media offices, killed several postal workers and others, and forced some
10,000 people, including members of Congress and their employees, onto
antibiotic therapy as a preventive measure.
Because a drug like Abthrax would generally be useful only in an attack,
there's likely to be no routine commercial market for it. Instead, the
government has pledged to create a market for such drugs by stockpiling them if
they meet certain criteria.
The nation already has large stockpiles of anti-terrorism drugs and supplies,
but administrators at the Department of Health and Human Services have
complained that they don't have the authority to make long-term purchase
commitments that would create a stronger incentive for firms to undertake
expensive research and development.
President Bush has asked Congress to fix that by passing Project BioShield,
which would commit some $6 billion to anti-terror defenses over 10 years. The
National Institutes of Health has said the Human Genome Sciences drug may serve
as a test for whether Project BioShield can work.
The legislation has bogged down in Congress. Bush, speaking Monday to
biotechnology executives in Washington, demanded again that Congress resolve the
issues. "For the sake of our national security, . . . Congress must pass the
BioShield legislation as soon as possible," Bush said.