Animal drug studies are often just a shot in the dark. And sometimes, investigators get hit with some completely unexpected ricochets.
Take the case of cyclodextrin. The Wall Street Journal notes that researchers were using the old compound as a placebo--it was considered inert and is often used as an additive in treatments--in a mouse study of a separate, intriguing therapy for the lethal Niemann-Pick Type C disease, or NPC.
It turns out that cyclodextrin worked as well or better than their experimental treatment, so they switched focus. Now the NIH is rounding up cash to back a clinical trial of the treatment and other investigators are seeing if they can strike a similar form of preclinical paydirt as they explore the compound's potential for Alzheimer's, HIV and even the Ebola virus.
In the case of HIV, investigators used cyclodextrin to partially disarm HIV by using it to remove cholesterol in the membrane of the virus, preventing it from doing damage to the immune system. A new treatment that protects the immune system of HIV patients would be a valued addition to the cocktail therapies now in use.
The unexpected potential that cyclodextrin has is being used to highlight the NIH's new program to encourage scientists to find new uses for old drugs. It's also a prime example of how open academic drug programs can get investigators to think in new ways about the diseases that they are fighting.
"We need to do this more systematically now instead of hoping for serendipity to strike," says NIH chief Francis Collins.
- here's the story from The Wall Street Journal