Armed with a new grant of nearly $9 million, researchers at Philadelphia's Wistar Institute are trying to create a preventive therapy against Zika that, in theory, has the potential to dramatically expand treatment for a range of diseases around the world.
"Our idea was maybe do something to have the body produce its own drugs," said David B. Weiner, executive vice president of the independent research institute, who is leading the investigation.
It is a radical approach that Weiner has pursued for years, and has been shown to work in animal studies involving dengue and chikungunya, both mosquito-borne viruses. The emergence of Zika, which carries the terrifying threat of birth defects when contracted during pregnancy, led the Bill & Melinda Gates Foundation this week to award $8.8 million to Wistar and its collaborators to try the technique on another virus.
The grant gives them two years to move through animal studies on Zika and, if those are successful, develop a product for clinical trials in humans.
The technology uses synthetic DNA monoclonal antibodies that have been encoded to instruct the body to produce the exact antibody needed to kill the virus.
It "has major public health potential because of the speed and precision at which this technology can be developed," Joseph Kim, president and CEO of Inovio Pharmaceuticals Inc., which is collaborating with Wistar, said in a statement released by the institute. It "could help 'jumpstart' work on tackling the next epidemic, giving us hope for a future where countermeasures for emerging pandemics could be more rapidly deployed," he added.
To understand why, think of it this way:
Preventing widespread outbreaks of infectious diseases currently relies on technology that dates back decades. Basically, pieces of killed or inactivated viruses that have been grown in eggs are injected into the body as vaccines. The body's immune system spots the invaders, figures out what is needed to try to neutralize them, and deploys antibodies to do the job.
Another approach is to design and produce antibodies in factories and inject the custom-made material, known as biologics, into the body. But this is extremely expensive, and not feasible on a wide scale for infectious diseases.
Wistar's innovation combines parts of both processes into a sort of direct-to-deployment version. Rather than relying on the body to identify an invader and decide what kind of antibody it needs to produce, the technology injects synthetic DNA that is encoded with the exact information to create the specific antibody needed to fight the disease. And rather than producing the antibodies in pharmaceutical factories, the body makes them. It is not considered a vaccine.
"We are delivering a set of instructions," Weiner said in an interview Friday.
And since the instructions are delivered as tiny bits of synthetic DNA, the body does not attack them in the way that it does other delivery vehicles; that has been a hurdle for the related field of gene therapy.
Although Wistar has been developing the monoclonal antibody technology for decades, it is still highly experimental. But Zika has now been confirmed in 37,700 people in the United States and its territories, the Department of Health and Human Services said Friday, and has affected 1.3 billion people worldwide. Slowing this threat, along with possibly developing an entirely new technology, led the Gates foundation to award the grant, Weiner said.
Wistar is doing the research and leading the project. Inovio would produce the product. Other collaborators are Humab BioMed SA and the University of Laval in Quebec.
If the technology ultimately proves successful, it could "dramatically change the things we treat, the number of people, and who could benefit from this treatment throughout the world," Weiner said.
There are many steps before that point, however, which is why Weiner referred to the grant as "high-risk, high-reward."