Understatement of the decade?

At the American Society of Hematology meeting last weekend, I had the pleasure of listening to an enlightening education session on CML from Drs Brian Druker, John Goldman, Moshe Talpaz and Tim Hughes, all leading lights in the field.

Prof Goldman's talk was particularly riveting because he always uses examples from history to illustrate the challenges of today, then offers a fair and thoughtful strategic road map for the future.  One such example was to remind us that way back in 1996 before the clinical trials with tyrosine kinases even began, a paper appeared in Cancer Research describing the synthesis of a new compound:

 

These sort of articles appear in science and cancer journals every day as a new compound is born and the big question is whether is will end up a drug star or wither and be terminated in the Pharma pipeline scrapheap.  However, what was touching about the article was the rather European statement in the abstract noting the following:

May have? 

Wow, I love a droll understatement... CGP 57148 went on to become renamed imatinib (Gleevec) and became a cancer blockbuster, changing the lives of many patients with CML, GIST and some rare myeloproliferative diseases driven by PDGF or KIT.

Later today I will post an update on what's hot in chronic myeloid leukemia from ASH and how the new second generation TKI's nilotinib and dasatinib may offer yet more hope for patients with CML in the near future, as well as other agents in development for the rare T315i mutation that none of the approved TKI's currrently inhibit. 

For now, it is sometimes good to reflect on history and feel a sense of awe and wonder that a small molecule chemical compound could eventually lead to 60-70% of CML patients attaining a complete cytogenetic response.  I'll guarantee those results turned out to be far more stunning than the authors of the paper ever imagined.

{Disclosure: I'm a former Novartis marketing director who was involved in the new product development and subsequent launch of imatinib in CML and GIST, so my bias and enthusiasm for both the drug and the many wonderful and brave CML and GIST patients who participated in the clinical trials is apparent.}

Buchdunger E, Zimmermann J, Mett H, Meyer T, Müller M, Druker BJ, & Lydon NB (1996). Inhibition of the Abl protein-tyrosine kinase in vitro and in vivo by a 2-phenylaminopyrimidine derivative. Cancer research, 56 (1), 100-4 PMID: 8548747

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