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Ebola Drug Could Be Ready For Human Testing Next Year

In this colored transmission electron micrograph, an infected cell (reddish brown) releases a a single Ebola virus (the blue hook). As it exits, the virus takes along part of the host cell's membrane (pink, center), too. That deters the host's immune defenses from recognizing the virus as foreign.

The Ebola outbreak in West Africa is terrifying because there's no drug to treat this often fatal disease. But the disease is so rare, there's no incentive for big pharmaceutical companies to develop a treatment.

Even so, some small companies, given government incentives, are stepping into that breach. The result: More than half a dozen ideas are being pursued actively.

And these are boon days for drugs that can treat viruses. Think of treatments for AIDS and hepatitis C.

Potential treatments for Ebola pursue many strategies. These include conventional drugs, custom-built antibodies, and vaccines that are designed not simply to prevent the spread of a disease, but to treat it in people who are in the early stages of infection.

Each idea has shown some promise in animals, but nothing has yet passed critical human testing as yet. So there's nothing ready to be tried during the current outbreak.

And Dr. Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases, is hopeful about this multi-pronged approach, but says, "I think it's really too early to make a prediction about what is the more or less promising one among them."

One challenge is there are several different species and strains of Ebola-like viruses so there may not be a one-size-fits-all solution.

But one experimental drug could conceivably fit that bill. It's called BCX4430. Travis Warren at the US Army Medical Research Institute for Infectious Diseases lab in Frederick, Md., has been working on this anti-viral drug.

"It worked great against both Ebola virus and (the closely related) Marburg virus" when tested in mice, he says. It also protected guinea pigs from these viruses and yellow fever, as well.

So the next set of tests was in a small number of monkeys who had been infected with Marburg virus.

"When we started the drug ether 24 hours or 48 hours after the infection, 100 percent of those animals survived," Warren says.

It's notable that this drug is being developed by a small company, called BioCryst Pharmaceuticals.

"It just wouldn't make the cut at a major company," says Dr. William Sheridan, BioCryst medical director, who once worked at the drug giant, Amgen.

Ebola, scary as it is, has only made about 2,500 people sick since it was discovered in 1976, killing about 1,700 of them. So the market for a drug like this is tiny.

Still, Sheridan says his company has good reason to pursue it, beyond his desire to address an important public health issue.

"There's a market, and the market is the U.S. government," he says.

The government has promised to buy a stockpile of drugs that are effective against Ebola, in case someone should try to use it as a biological agent on the battlefield, or in an act of terrorism. And federal agencies also are helping to pay for the research. So the company is pushing forward as quickly as it can.

"We're currently [manufacturing the] drug and will be conducting typical animal safety experiments that you typically do before you put drugs in humans," Sheridan says. "And once that's successfully completed, I anticipate by the middle of next year that we should have completed phase one studies in people."

Phase one tests will tell them whether the drug is relatively safe, but it won't tell them whether it would actually be an effective treatment. Assuming the drug seems safe, it would be ready for testing in a future outbreak of Ebola.

Of course, most potential drugs don't live up to their early promise. And there's no assurance this one will, either.

But Travis Warren at the Army lab says, given all the advances in developing drugs to treat viruses, something will emerge out of this broad quest for an Ebola drug.

"I'm absolutely certain it will happen," Warren says. "It's just a matter of time."

Copyright 2014 NPR. To see more, visit http://www.npr.org/

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