Why developing a drug is not easy and collaboration matters

As a reader of books I am slow. I am also picky, focussed on science, art and jazz topics with the occasional rare novel thrown in at random. Back in January I attended the Science Online 2013 meeting. What I did not expect was some great books as freebies. I picked up 3 which I am making my way through. One of which sparked this blog. OK, its not quite a book review for “the Philadelphia Chromosome” by Jessica Wapner it is more of how history repeats, or at least some patterns repeat.

In 2002 (after several years working in big pharmas) I was working at a very young start-up biotech focussed on computational drug design then based in Boston and it was recommended by my boss that I read a book called “the billion dollar molecule” by Barry Werth. This was originally published in 1994 and told the story of a then pretty new drug company focussed on rational drug design, also in Boston called Vertex. The book was a roller coaster ride representing the scientists on the inside and also the business happening on the outside of the company. Ultimately it focussed on their attempts at finding a drug for HIV. This book for me set the standard for a long time for what was in store for anyone thinking they could find a cure for disease X. I was energized by the writing and optimistic at that time that we could do the same and make a difference.  I do not know if my then boss had seen the writing on the wall or the book was some sort of cautionary tale, but within a few years we would have both decamped to do other things after our then small biotech had moved to Philadelphia and became yet another big pharma wannabee (which was far from the initial lofty goals of the company).

The memories of this time were rekindled when I read “the Philadelphia Chromosome” which tells the long story behind the rare disease chronic myeloid leukemia and a search for a treatment which spans over 40 years. What captured my interest was the story as it merged academic and industrial collaboration which appeared very tense, as the clinicians pushed the drug company (Novartis*) along to develop what became Gleevec. I really do not want to give too much away because I think the book is a must read by anyone; A) interested in rare diseases, B) interested in science and the long and winding road from discovery to the clinic, C) interested in a compelling page turner with a feel good stripe running through it and D) interested in what the future for healthcare may look like.

We are literally just at the begining of how drugs like Gleevec can revolutionize a rare disease and at the same time pump new life into the lumbering giant of a pharmaceutical company, that previously was dismissive of such diseases with little perceived commercial appeal. Now of course we are seeing many companies actively courting rare disease patients to try to follow up on the success of Novartis based on their efforts documented in this book. So what really is just hitting me as I write this is that my career has shifted to work increasingly with very small foundations and patient advocate led groups that are looking to find treatments for ultra-rare and rare diseases. They are leveraging crowdfunding to raise money for the research they need doing in academia through collaborations and also reaching out to other companies to get them to either supply compounds for testing or support for potentially bringing their treatments to market. This is a very different approach to that presented in the “the Philadelphia Chromosome” but again it is useful to think about the challenges the academics had to overcome when working with the merging big pharma’s and how the path is never straightforward. I would stick my neck out and offer that “the Philadelphia Chromosome” is for me the next “the billion dollar molecule”, and documenting how the pharmaceutical industry can perhaps save itself by literally going after small populations of patients and growing  that market. Ironically, Vertex may have ultimately also done the same by developing the drug for a small number (~4%) of cystic fibrosis patients, called Kalydeco, in conjunction with the Cystic Fibrosis Foundation. Could this too become their “billion dollar molecule”, time will tell. Perhaps we should be focussing less on whether a drug becomes a billion dollar molecule but instead collaborating to treat devastating diseases regardless of incidence, and that for me I think is the take home message. These books have both appeared at appropriate and important times for me and also represent valuable historical documents. I hope anyone trying to develop treatments learns from them and they inturn become the starting point for a next generation of drug discovery that will likely be documented in a future book.

*In the interests of full disclosure I should say my wife currently works for Novartis Vaccines. I have no financial or consulting links with the company.

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