Nanotech is promising, but faces hurdles

Nanotech is promising, but faces hurdles

Dwight Seferos, PhD, a researcher at the International Institute for Nanotechnology at Northwestern University, held a sizable audience in thrall for nearly an hour at the 13th European Federation of Biotechnology meeting in Barcelona.

He had a good topic. Researchers at the institute have used oligonucleotides--a short piece of DNA known for its ability to bind to complementary DNA--to effectively disperse nanomaterials inside a variety of cell lines. There's good potential to use this technology to dispatch nanoparticles inside a patient's body to deliver therapeutic agents, Seferos told the group. The same approach, for example, could be effective in targeting cancer cells, killing them or leaving them vulnerable to chemotherapy.

Chad Mirkin, the director of the institute and a founder of Nanosphere and NanoInk, has been a leader in using atomic force microscope tips to make nanoscale materials, putting on one atomic layer at a time as he delves into a world that is one to 100 nanometers in size.

Translating that kind of technology to the bedside won't be easy, though--particularly in Europe, where government regulators have turned a cold shoulder to expensive new therapies. That message was sent loud and clear just a few hours after Seferos wrapped his presentation.

In front of a smaller but no less avid audience, Mike Eaton, PhD, the head of biological chemistry at the giant UCB, outlined the barriers that are standing in the way of nanotechnology.

As promising as many of these research projects are, he said, developers have to keep in mind that financial obstacles can easily thwart new therapeutic approaches. To illustrate that, he noted the fate of abatacept, a nanotherapeutic known commercially in the U.S. as Orencia, which had achieved a 50 percent reduction in symptoms among 40 percent of patients taking the therapy--solid proof of efficacy. Abatecept works at the T cell level, preventing a chain cellular reaction that causes rheumatoid arthritis. But abatacept costs about €9,300 a year, and the National Institute for Health and Clinical Excellence, the U.K.'s drug watchdog, turned it down flat.

Anyone looking to advance a nanotech approach to medicine, he says, needs to factor in that European regulators, at least, are unlikely to give a green light to any therapy that costs more than €5,000 a year. Said Eaton: "Significant benefits will be needed to counteract complexity."

But there are also some important clinical considerations to consider, as well, added Eaton. For example, if gold nanoparticles are used in the way Seferos described in his presentation, regulators will want to know precisely how the metals will be disposed in the body and exactly how it will be entirely flushed out of the system. In vivo analysis will be essential.

The ability to efficiently manufacture nanotherapeutics will also be tough, he says. Small companies have a key role to play here, said Eaton, because they have the kind of flexibility that can advance innovative approaches to new therapies. But the demands on nanotherapeutics will be considerable, demanding the involvement of big companies with deep pockets. A company like UCB, he noted, has 14,000 employees, and it would take a company at least half that size to advance a nanotherapeutic into clinical practice.

The bottom line here: The science of small will need Big Pharma to make it in the commercial world of approved therapeutics. - John Carroll