Pharma Strategy Blog

Commentary on Pharma & Biotech Oncology / Hematology New Product Development

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:

Picture 53 

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:

STI571_Cancer Res
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.}

ResearchBlogging.orgBuchdunger 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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2 Responses to “Understatement of the decade?”

  1. Claudia Donnet

    Typical cautious statement of a pure academic scientist, although I see that Ciba Geigy Switzerland was involved in this case.
    Many of these suggestions appear in the conclusions of many academic papers coming from good labs from all around the world. I wonder if there aren’t many diamonds in the rough that have not been picked up by the industry and transformed into a solution for human health. Some academic papers may appear on topics that are not “hot” or “current” at the time and I guess that their findings could pass by, unnoticed. Is industry screening all the literature or does it depend on the academics to have an entreprenurial and/or healing-oriented vision and connect with the industrial world to get their interest?

  2. MaverickNY

    Hi Claudia, great to see you on this blog after corresponding on Facebook!
    In this particular case, I was being tongue in cheek a little because the science and biology of CML has evolved in CML from the discovery of the Philadelphia chromosome by Nowell and Hungerford, through David Baltimore and Janet Rowley’s work so it was pretty much clear that the target was the right one, things were just waiting for a drug to be developed to target the abnormality.
    As a result, the authors knew they had a very good chance of success, hence my teasing of the great understatement :-). Druker, a physician and a researcher, was the one one made the connection between the target and the drug, which we can all be very thankful for.
    Your point is well made though, often it takes a while for the industry to catch up and realise the significance of basic research. The two are not always in perfect synchronicity, as this example went.
    We need more Brian Drukers, John Goldmans and Charles Sawyers, all great science-physicians who do both translational research and treat patients.

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