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The threat of a nuclear terrorist attack on U.S. soil and abroad looms large at a tiny Eden Prairie pharmaceutical company working on an experimental drug that would treat exposure from radioactive fallout.

Suitcase bombs. Dirty bombs. Accidents or attacks at nuclear power plants. Mass panic. Gridlock. Chaos. The dire scenarios deeply trouble Humanetics Corp. CEO Ronald Zenk.

"Right now, civilians are defenseless in the wake of a nuclear event," he says.

Bolstered by $3 million in Defense Department funding, the privately held company has begun clinical trials in animals and a small number of people testing a drug called BIO 300, which aims to combat acute radiation syndrome (ARS), or radiation sickness.

ARS is a potentially fatal disease caused by a high dose of radiation over a short time, according to the Centers for Disease Control and Prevention. In most fatal cases, the cause of death is the destruction of bone marrow, resulting in infection and internal bleeding.

Humanetics isn't the first company with a potential anti-radiation drug to be courted by the Bush administration, which says it is intent on developing drugs to help the nation defend itself in case of a bioterror attack.

A small San Diego biotechnology firm, Hollis-Eden Pharmaceuticals Inc., spent $85 million over the past decade to develop a similar drug -- only to have the contract canceled by the Department of Health and Human Services. Today, the company's stock price is badly battered and its employee ranks depleted in a recent cutback.

Despite this cautionary tale, Humanetics executives believe BIO 300 has the potential to be approved for treatment of radiation sickness -- which includes damage to the forbears of infection-fighting white blood cells and platelets in bone marrow.

Zenk says that his drug works differently and that the company's broader focus on protecting the civilian population is different, as well. He foresees large companies stockpiling BIO 300, as well as pharmacies and grocery stores; protecting yourself in the wake of a nuclear attack could be as simple as getting a flu shot.

Orphan status

Humanetics cleared an important milestone last month when it was granted an orphan-drug designation from the Food and Drug Administration (FDA). The designation promotes the development of drugs treating rare diseases and conditions -- those typically passed over by large pharmaceutical companies -- that affect fewer than 200,000 people in the United States.

The FDA classification gives companies such as Humanetics seven years of market exclusivity, preventing rivals from developing similar products that siphon away royalties; a 50 percent tax credit for conducting often-expensive clinical trials; and reduced fees for FDA applications.

Although the FDA says the program is successful, the designation doesn't guarantee approval of a drug.

More than 200 drugs and biological products for rare diseases have been approved since the orphan designation was introduced in 1983. Among them: Antizol, used to treat those who have ingested antifreeze, and Acetadote, which mitigates an overdose of Tylenol, which can cause liver damage. (Antizol was marketed by Minnetonka-based Orphan Medical Inc., which was bought by a California company in 2005.)

While Zenk believes the potential market for BIO 300 is "quite large," radiation sickness now is quite rare -- hence its orphan status. The biggest releases of radiation involved the victims of atomic bombs dropped on Hiroshima and Nagasaki during World War II, as well as radiation-related deaths and illnesses associated with the Chernobyl nuclear power plant accident in 1986.

There are drugs now available that treat people exposed to radiation, such as potassium iodide to ward off thyroid cancer. And Prussian Blue, a drug made from blue dye, can remove some radioactive materials from the body, but it must be administered by a doctor.

None of the current remedies protect the body from the damaging effects radiation has on bone marrow, said Dr. John Zenk, Humanetics' chief medical officer and the CEO's first cousin. Ideally, he said, a BIO 300 pill or injection could be taken preventively by the military, first responders and civilians if a radioactive leak has been detected -- especially since people might not know whether they've been contaminated.

Humanetics said it obtained the license for BIO 300 from the Henry M. Jackson Foundation for the Advancement of Military Medicine, a private, not-for-profit organization that supports military research. Terms of the license agreement were not disclosed.