Gene therapy, against the flu?
Published 5:00 am Tuesday, June 4, 2013
In 2009, a global collaboration of scientists, public health agencies and companies raced to make a vaccine against a pandemic influenza virus, but most of it wasn’t ready until the pandemic had peaked.
Now, researchers have come up with an alternative, faster strategy for when a pandemic influenza virus surfaces: Just squirt genes for the protective antibodies into people’s noses. The method — which borrows ideas from both gene therapy and vaccination, but is neither — protects mice against a wide range of flu viruses in a new study.
James Wilson, a leading gene therapy researcher at the University of Pennsylvania, credits the idea to a meeting with Bill Gates in April 2010. Wilson had studied whether a harmless gene therapy tool called adeno-associated virus (AAV) can serve as a gene delivery vehicle to treat inherited diseases like cystic fibrosis and hemophilia.
Building off of animal studies done by AIDS researchers, he wondered whether a specially engineered AAV could deliver the genes encoding influenza antibodies to the cells that line people’s airways. If it worked, these so-called epithelial cells would produce influenza antibodies right at the site where the virus attempts to establish an infection.
Unfortunately, AAV had petered out in about three months in a monkey experiment in Wilson’s study. Wilson says that three months of protection is “not optimal” during an influenza pandemic.
Wilson sees his work as a stopgap measure until researchers figure out how to make a vaccine that triggers broadly neutralizing antibodies. But as of now, despite intense efforts, researchers have been unable to design a vaccine that triggers the production of these powerful molecules. “We still have a ways to go,” he says, “and until that happens, we’ll just keep plugging away at this.”