Dec 09 2012
For many years, the development of pharmaceutical drugs has been kind of hit or miss. New and interesting bioactive compounds are discovered and tested in different laboratory animals until someone figures out what a particular compound might be good for. It isn't terribly common that a pharmaceutical company comes up with an idea for an effect and then works backward until they find a compound that will do what they hope to accomplish.
That shows signs of changing.
A company called Parabon Nanolabs in Reston, Virginia has announced the development of a new drug which is effective in treating one of the most common and yet deadly forms of brain cancer called glioblastoma multiforme. However, rather than using the traditional ways of finding and developing drugs, they used a highly interesting approach: After identifying the metabolic processes in the cancer cells they wanted to attack and figuring out which chemical receptors they could access, they used a CAD application they'd developed to piece together a molecule with specific, desired properties that they hoped would be effective. CAD has been used since the 1980's in the design of buildings, machinery, and other products but Parabon Essemblix and inSçquio is the first CAD package for molecular compounds.
In a process very much like assembling Tinkertoys, chemical engineers figure out which molecules are suited for certain tasks, such as binding to a particular set of receptor sites on a cell membrane common to cancel cells, protecting other functional groups from some metabolic pathways within those cells, or interfering with the replication of the cells directly and assemble a larger molecule out of those functional groups. The design is then fed into a massively parallel virtual computer called the Parabon Computation Grid which optimizes and tests the design by spreading the analytic tasks across hundreds of CPUs on a network. It's not explicitly stated in any articles I've seen, but I wouldn't be surprised if genetic algorithms that assist in the optimization and simulation processes were part of their software, but I might also be completely off base here. That pretty firmly falls into the "secret sauce" part of the spectrum. Then, the compounds that the grid computer outputs are synthesized using the same molecular self assembly techniques used by DNA. Total time to synthesize a medically usable quantity of the new drug: Days to weeks, instead of months to years.
This represents a significant step forward in the field of pharmaceutical engineering because it's a much faster and more accurate process. Rather than hoping to stumble across a compound that can be used to treat a particular disease, custom-designed compounds are now becoming a more feasible means of treatment. In fact, this technology may very well become crucial in the battle against antibiotic resistant bacteria, which medical science is not winning. To give you an idea of what the stakes are, drug-resistent tuberculosis is becoming more common throughout the world, and in fact at least one strain was discovered that every drug tried so far on it has proven ineffective. It's bad enough that outbreaks are being covered up because they can't be contained. Sometimes the only drugs we have that do work are arguably just as dangerous as the disease because the most common side effects observed include kidney or nerve damage. However, WYSIWYG chemical engineering coupled with fast and cheap genetic analysis might tip the scales back in the favor of medical science.
Keep an eye on this, folks. The life this technology saves might one day be your own.