Kalydeco (ivacaftor) Credit: MIT Technology Review, Rebecca Schroeder |
Wednesday, November 6, 2013
A reminder of one of the reasons that people want to work in pharma
In the middle of a fascinating article about the concerns about high drug pricing by Barry Werth* in MIT Technology Review, a stark reminder of the good that effective pharmaceuticals (such as Vertex's Kalydeco (a cystic fibrosis drug) can do: (The caption from the article reads: "After her son Brady’s health improved,
Rebecca Schroeder had Kalydeco’s molecular structure tattooed on her foot.")
Not to get all mushy on you guys, but if my kid's life was improved significantly from a drug, I'd think about having its structure tattooed on me. (I'm not really a body art guy, fyi.)
I think that a lot of people get involved in chemistry to do good and to have a positive impact on someone else's life while doing the science that we love. It's one of the weird tragedies of our time that pharmaceuticals (and medicinal chemistry in particular) can do so much direct good, and yet, there are many more people who want to work in the field than can be hired.
*He's coming out to a sequel to "The Billion Dollar Molecule" in February! I can't wait.
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" pharmaceuticals can do so much direct good, and yet, there are many more people who want to work in the field than can be hired. "
ReplyDeleteThats really good, but I prefer:
" pharmaceuticals can do so much direct good, and yet, there are so many people who want to work in the field, they can easily be exploited and made bitter. "
Q: Why would you willingly put two t-butyl groups and a phenol on a drug molecule?
ReplyDeleteA: Why would you think drug discovery is rational?
P.S. I heard the story of Kalydeco at a conference recently and it's clear that the people who deserve the most credit are the biologists. Getting the right assay that would mimic the biology of a CF lung was the real breakthrough.
It IS a very interesting structure, isn't it? Hey, Ash, do you think this molecule could have been synthesized using the knowledge from the 1940s?
DeleteProbably - check out PCT/US2006/049421(p46-47) - none of the chemistry is terribly exotic. Whilst I don't know off-hand when some of these reagents would have become widely commercially available, it's pretty standard stuff. Several of these reactions were things I had my undergrad helpers do back in grad school.
DeleteExotic chemistry is way over-rated in terms of finding a drug. Complexity for complexity's sake is necessary, and sometimes straight-forward is all you need. The trick would be IP space on simple molecules like this one. Doesn't seem to have been an issue, however.
DeleteAs Wavefunction said, the real heavy lifting was done by the biologists to find a system to mimic CF. That's truly the hard part.
Thanks for your thoughts, guys -- much appreciated. #KnuckleDraggingProcessChemist
DeleteIndeed, the chemistry was relatively straightforward. And drug approval might have been even faster in the 40s..
DeleteRan it through our in-house property calculators. Chris Lipinski would not approve...
DeleteStewie Griffin:
ReplyDeleteWhen I started grad school I honestly thought that if I ever completed my total synthesis I was going to get a tattoo of my molecule. By the time I finished the damn thing, I wanted nothing more to do with that molecule.
I don't understand chemistry tattoos for the same reason - I had much more passion for the subject before grad school than afterward!
DeleteAt least they got the structure right. Unlike the one in the recent CEN article http://bit.ly/17cu9D1.
ReplyDeleteI for one am v hopeful the follow-up to Kalydeco is also a big success. Good to see some home grown San Diego drugs out there.