This week’s cool medicine is the drug Fomivirsen. Fomivirsen is an antiviral drug of a rather new sort. It’s the first antisense drug approved by the FDA. For those who aren’t sure what “antisense” means, a quick description follows. The link goes to a much lengthier discussion on Wikipedia.
Nucleic Acids are the “letters” in the genetics instructions every living thing contains about how to build itself. They can be either DNA or RNA. In this case, we’ll be talking about DNA. DNA molecules have a number of interesting properties, one of which is called complementary base-pairing. There are 4 bases that can be used to make a molecule of DNA. Each molecule uses only one of these bases. Each base is capable of binding (think of it like holding hands, of you don’t know what binding is) with one other base. The bases are represented by letters: C, G, A and T. C binds with G, and A with T. DNA is also read in a specific direction. For simplicity’s sake, we’ll just say it’s read from Start (S) to Finish (F). When two pieces of DNA bind to each other, it happens that the Start end of one piece binds to the Finish end of the other piece, so if you have a piece of DNA that reads “S-CATTCGTCACT-F”, the piece that would bind to it would read “S-AGTGACGAATG-F”. These pieces are called “complementary”. Lined up like they would be when paired with each other, they’ll look like this:
S-CATTCGTCACT-F
F-AGTGACGAATG-S
Now, when a piece of DNA is being copied, either strand of the pair can be copied to make the new piece, but when a piece of DNA is being used as instructions for making something else, only one strand is used. Let’s say the top one in the example above is the one that’s used as instructions, it’s called the “sense” strand. The “antisense” strand is its complementary strand, the bottom strand in our example.
Now to get back to Fomivirsen. Fomivirsen is a drug that is actually a piece of DNA. This piece of DNA is antisense to an important part of the DNA for the virus Fomivirsen is used to treat. What happens is that Fomivirsen pairs with that piece of DNA when it isn’t supposed to be paired with anything. This keeps it from being used as instructions for making part of the virus, and keeps the virus from growing within the person who is taking the drug.
Antisense molecules have been used for years in research labs to study what different genes do, but Fomivirsen is the first drug made of an antisense molecule approved by the FDA for use in people. It’s a new approach that could lead to the creation of drugs that are very, very specific for the bug they’re trying to kill, which will help reduce side-effects and increase potency.
Very cool. The only down-side of this drug is the way it’s given. The drug has to be injected directly into the eye of the patient, incurring an “ick-factor” almost large enough for me to have not written about it.