Early research has shown that a derivative of the HIV anti-viral drug cidofovir is effective in combating smallpox, U.S. scientists said yesterday at a medical conference in Prague (see GSN, March 19).

“Although it’s too early to say that this is, quote, a breakthrough, there are some very encouraging components about this,” said Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases.  “We are pushing very vigorously to get this into a trial in humans, to see if it would work.”

The U.S. Food and Drug Administration has already approved cidofovir as an experimental treatment in the event of a smallpox outbreak.  The derivative, made by adding a molecule called a lipid to cidofovir, is 100 times more powerful and can be taken orally instead of through intravenous injections as cidofovir requires, said Karl Hostetler, a professor of medicine at the University of California at San Diego who helped develop the derivative.

“If it tracks all the way through, it could be a very useful addition” to treatment strategies, Hostetler said.  “People could self-administer it instead of having to go to places and stand in line” (Sheryl Gay Stolberg, New York Times, March 20).

Research indicated that five oral doses of the derivative given to mice infected with cowpox, a cousin of smallpox, prevented death.  Scientists also learned that, unlike cidofovir, the derivative reduced the amount of smallpox virus in the lungs of infected animals to undetectable levels, according to the Chicago Tribune.

“Until now, the eradication and control of smallpox relied upon vaccination,” Hostetler said.  “These results suggest that anti-viral drugs given orally in a regimen consisting of as few as five doses over five to 14 days might be used as an alternative to people exposed to smallpox, especially those individuals who cannot safely be vaccinated” (Peter Gorner, Chicago Tribune, March 20).

U.S. Office of Public Health Preparedness Director D.A. Henderson, however, said he was unconvinced that the government should stockpile cidofovir.  Animal studies have shown that the drug is only effective immediately after smallpox exposure, and not when symptoms set in.  He said if the derivative works in the same way, then there is little advantage to it over vaccination.

“I know I look like some kind of troglodyte, as though I am not interested in anything novel or new,” said Henderson, who had a key role in the World Health Organization’s efforts to eradicate smallpox in the 1970s and 1980s.  “But you can see why I am a little lukewarm about this” (Stolberg, New York Times).

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