8-year perspective on Gleevec -- the “selling” of a drug innovation

Since its approval in 2001, Gleevec has turned a rare form of leukemia from a death sentence into a manageable disease. But with only an estimated 10,000 patients worldwide afflicted with chronic myelogenous leukemia, Novartis initially had little interest in the drug.

Dr. Brian J. Druker at Oregon Health & Science University's Knight Cancer Institute, whose lab did the basic work on Gleevec, persuaded Novartis to take a chance on it.

In a 2003 Science Watch interview, Druker explains how he made the case.
Science Watch correspondent Gary Taubes asked Dr. Druker, “How did you convince a major pharmaceutical company to develop a drug for a disease that strikes a comparatively small group of roughly 5,000 Americans a year?”

Here is his reply:

“That was certainly the hard part, and a lot of things had to fall into place. We faced three major hurdles. One was that it was a drug for a small market. Second, there was some liver toxicity in early animal tests. For a variety of reasons, Ciba-Geigy was adverse to taking a risk with a potentially toxic drug. But for me, I was dealing with cancer patients who were dying, and I was accustomed to giving drugs with significant toxicity. Thus, I was much less concerned about potential risks. Here was a compound that might help patients, and I absolutely wanted it to be given a fair trial in people. If it proved to be toxic in people, then of course it wouldn’t move forward. But if it worked with a therapeutic window, that would be worth developing.

“The last hurdle was that Ciba Geigy had just merged with Sandoz to create Novartis. That led to down-sizing and re-evaluations of all the programs. STI571 wasn’t even on anybody’s radar screen. In addition, Nick Lydon, who was the lead investigator of the tyrosine kinase inhibitor program at Ciba Geigy and the main internal proponent for the development of STI571, had left the company.

“Ultimately, I was able to convince a few key people at Novartis to move this compound forward to clinical trials. One of the factors that helped was that this compound also inhibited the platelet-derived growth factor receptor (PDGF-R) tyrosine kinase. The PDGF-R is expressed in most cancers, including most of the common ones, such as breast and prostate cancers. Whether its activity is required for the growth and survival of these cancers is unknown, but if STI571 worked in these diseases, this would be the market size that would interest a large pharmeuctical company. So the view was that if it worked in CML, Novartis had an easy way to the market. It could then be tested broadly and might have a very large market potential.”

You can read the full interview on the journal’s website archive.