Provita Pharmaceuticals isn't your average life sciences research and development company. Based out of New Jersey, it doesn't have its own multimillion-dollar research facilities or even much experience within its staff. Its CEO is a 16-year-old high school student, as are every single one of the company's 15 other staff members. Its closest association isn't with a major pharmaceutical company, but with Bergen County Technical School system.
But a conceptual product they're working on might just be one of the most innovative ideas being worked on today, and could pose enormous difficulties for regulators.
That product isn't so much a thing, but a what: a mosquito-borne delivery system for vaccines.
Fast Coexist reports the company is working to genetically engineer mosquitoes to essentially be flying syringes, carrying vaccines intended to be used against the West Nile virus and other deadly diseases. The genetically modified mosquitoes wouldn't just carry the vaccine-they would actually make it themselves.
A Host of Challenges
If all this sounds a bit futuristic, it's because it probably is. But the regulatory issues associated with such a product are extensive.
A living combination of animal (the mosquito), medical device (the mosquito's mandible), biologic (the vaccine, contained in the mosquito's saliva) and manufacturing facility (the mosquito) would certainly represent an extraordinary challenge for regulators.
Challenges are likely to come in several forms, but the most obvious may simply be the combination of conducting environmental analyses and winning over public support.
The ongoing review of genetically modified salmon by the US Food and Drug Administration (FDA) illustrates some of those issues. Biotechnology company AquaBounty has for years been attempting to obtain approval from FDA for its genetically modified salmon product, which it says grows both faster and larger than naturally-bred salmon. FDA regulators recently released a draft environmental assessment and preliminary finding that the salmon would not have a significant impact on the environment, but that finding has been met with fierce pushback from environmental groups and some legislators.
A similar environmental assessment would likely be necessary if the product were to be introduced into the US, though Provita said it envisions the product being deployed in Africa, where it could have a greater impact.
But animals don't respect trade barriers, and once introduced in one area, the mosquitoes could well spread to other countries as well, even if regulators there have explicitly banned their entry on the market. Unlike fish, which can be relatively easily contained in enclosures, mosquitoes are difficult to contain and are adaptable enough to be found in large numbers around the world.
An environmental assessment, in other words, could be far more rigorous for a genetically modified insect than a fish.
Other questions raise interesting conundrums as well. If a mosquito is its own manufacturing facility, what sorts of controls need to be built into it to ensure that the final product meets uniform standards for quality and sterility? Can an animal actually be expected to meet current good manufacturing practices (CGMPs) or Quality System Regulation (QSR) standards? And how do you control the dose of a vaccine given to people if an animal is the one administering it and not a qualified medical professional?
Challenging questions, all.
What About Other Animal/Device Combination Products?
But if the regulatory challenges that Provita faces are immense, the concept is one that has already captured the imaginations of some regulatory analysts for years. Author Max Sherman, a longtime contributor to Regulatory Focus magazine, has already identified the use of animals as potential medical therapies in several instances.
Psoriasis, he wrote in December 2012, could well be treated using a species of fish, Garra rufa, better known as "nibble fish" or "doctor fish." The species is already widely used in some spa treatments in Asia, and is known to consume dead skin cells when starved.
But, as Sherman notes, when looked at as a living medical device, several challenges emerge. The fish cannot be sufficiently cleaned, sanitized or disinfected between uses, he notes. Further, the species isn't native to many regions, posing an issue for countries that actively protect against the introduction of potentially invasive species.
Other animal species, such as dogs, could leverage their well-known olfactory abilities to detect cancer in humans, leveling similar problems. It is far easier to calibrate a single device than the olfactory abilities of multiple animals, for example.
For now, though, all of these remain concepts-fantastical what-ifs dreamt up by researchers.
Regardless of the challenges associated with animal-based treatments, though, regulators around the world might do well to expect them to one day arrive. Because while a flying syringe might only be a concept for the moment, this is one combination product that could be buzzing around the White Oak campus's review offices within the next two decades if all goes well.
Read the entire Fast Coexist article here.