Although Aeolus is currently testing the drug for its role in biodefense, the company says it could also help improve some cancer treatments. As in the radiation therapy example mentioned by McManus, the drug could potentially help put a chemical "fence" around a tumor, ensuring destructive radiation damage didn't travel beyond the targeted therapy site.

Even more intriguing, the drug could possibly help with the condition it was first investigated for, amyotrophic lateral sclerosis (ALS), better known as Lou Gehrig's disease.


In this disease, famously suffered by New York Yankee Lou Gehrig as well as the British scientist Stephen Hawking, motor neurons decay and the patient's muscles atrophy. Patients can become paralyzed and many eventually die from respiratory failure, as their lung muscles are no longer strong enough for breathing.

Around five years ago, Aeolus licensed the 10150 drug technology from Duke University and the hospital National Jewish Health, where it was originally developed by Irwin Fridovich, a biochemist and researcher at the university, in order to see if it could help with ALS. Although some initial testing was done with around 50 patients, the work is now on hold because of the difficulty in getting funding for rare diseases, McManus said. "A million people get cancer radiation therapy; there are 30,000 people with ALS," he observed.

NEXT STEPS FOR APPROVAL

Unlike most drugs, Aeolus 10150 has a slightly streamlined approval process. Because the researchers can't irradiate human volunteers and then give them the drug to see if it will save them, the U.S. Food and Drug Administration allows what's known as the two-animal rule. In this, the researchers have to prove the drug's effectiveness in two animal species, but in humans they only have to show it's safe to take, and doesn't cause any dangerous reactions.

In addition to the work on mice, in January, Aeolus began lung studies with a second animal species, McManus said.

From the safety studies that have so far been done with humans, the side effects have mostly been headaches, nausea and dizziness. Most significantly, there was some burning at the site of injection, which diluting the solution was able to bring down, McManus said. Also, because it is metal-based, the drug can cause discoloration, known as tattooing, at the injection site. "It goes away in about a week or so," said McManus.

Critically, one of the current unknowns is how wide they can make the treatment window: ensuring that the drug can be useful if administered some time after radiation exposure. In a likely biodefense scenario, rescuers might not be able to reach survivors trapped in a contaminated area for hours, if not days, following an attack.

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