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Targets Versus Drugs

Feb. 06, 2019 4:52 AM ETPFE, MRK1 Comment
Derek Lowe profile picture
Derek Lowe


  • The '850 patent filed by the University of Rochester had several claims, with several key ones coming down to a method of treating human patients with a compound that was a selective inhibitor of COX-2.
  • After being granted that patent, the University of Rochester filed suit against Pfizer for marketing two COX-2 inhibitors, which Rochester claimed violated their patent.
  • In order to be useful, a drug has to be able to be dosed in a human patient, last long enough to have a beneficial effect, and not by itself (or through its breakdown products) cause so many harmful side effects that it's not worth taking.
  • It also has to be produced on large scale, under very tight tolerances, and be stable enough so that it can be stored and shipped without alteration.
  • But the main point is that a target, a mechanism is most certainly not a drug.

There was a comment on the blog the other day about how there are people in academia who feel that the discovery of a new target or pathway is basically finding a new drug, and that the rest is "technicalities". I've encountered that view of the world before (Donald Light/Rebecca Warburton, Marcia Angell, and similarly, Arnold Relman), so it's not just some aberration, but it's just amazingly wrong. People keep pointing this out, including people who've worked in the industry and people who haven't, but to no avail.

So, here's an example that illustrates the difference. If you pick one of the most famous drugs in the world (aspirin, first reported in 1899), it can come as a surprise to people outside the field how long it took to find out how it worked. It wasn't until 1971 that John Vane at the University of London figured out that it (and the other non-steroidal anti-inflammatory drugs) worked by inhibiting the cyclooxygenase enzyme (COX), which is responsible for producing the signaling molecules (prostaglandins) that produce the downstream effects. Aspirin does other things as well, but that's the big mechanism that everyone had been searching for. During the 1980s, extensive work on this enzyme and its genetic background led to the discovery in 1991 (at Brigham Young, by Dan Simmons and his group) of a second subtype of the enzyme (COX-2).

And that set off quite a chase. Because it looked like if you could inhibit COX-2 and not COX-1, you might be able to get the pain-relieving antiinflammatory effects of aspirin without the gastrointestinal side effects of bleeding, irritation, etc. The idea of an "aspirin 2.0" was immensely appealing, and a great deal of work went into finding such compounds. In 1992, a team from the University of Rochester filed a patent application for an assay to distinguish whether new

This article was written by

Derek Lowe profile picture
Derek Lowe, an Arkansan by birth, got his BA from Hendrix College and his PhD in organic chemistry from Duke before spending time in Germany on a Humboldt Fellowship on his post-doc. He's worked for several major pharmaceutical companies since 1989 on drug discovery projects against schizophrenia, Alzheimer's, diabetes, osteoporosis and other diseases. To contact Derek, email him directly: derekb.lowe@gmail.com (mailto:derekb.lowe@gmail.com)

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Comments (1)

Winston Van profile picture
For sure
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