Turning loss into hope, family offers inspiration -- and a few lessons about drug discovery

Cystic Fibrosis Foundation

Article Highlights

  • Raised an estimated $100M for the CF foundation & its support of new drug development and patient care

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  • True advancement & real novelty, if it is to occur at all, is more likely to result from passion than strategy

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  • Collaboration is a necessity, not a choice

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A poignant story in Thursday’s Boston Globe describes how the O’Donnell family of Boston channeled their love of a son, Joey, who died tragically at the age of 12 from cystic fibrosis, into a successful mission to develop impactful new treatments for this terrible affliction.

The narrative – expertly told by Brian McGrory (see here for my favorite example of his wonderful writing) – is a testament to devotion, persistence, the power of parental love, and the resiliency of the human spirit.  It is essential reading for these qualities alone.

There also may be several important lessons for medical discovery and drug development.

"Industry is absolutely essential for delivering new products (so please stop crapping on us)." -- David Shaywitz

According to the Globe piece, the basic biopharma story is this: the CF Foundation, eager to catalyze the sort of risky early-stage research that has become increasingly imperiled, invested $40M in a small San Diego-based company, Aurora Biosciences, focused on CF-related R&D. 

In 2001, Aurora – basically a platform company, specializing in assay development and high-throughput screening —  was purchased by Cambridge-based Vertex Pharmaceuticals (see here for Barry Werth’s classic book recounting the early days of this company), which agreed to continue the CF research Aurora had started – a project it could very reasonably have shuttered.  The CF Foundation, for its part, agreed to continue to fund the work, to the tune of an additional $80M.  Much of this money was raised through the tireless efforts of Joey’s father, Joe, who beat the bushes day and night to keep the funds rolling in.  Over the last 30 years, through mechanisms such as the Joey Fund, he has raised an estimated $100M for the CF foundation and its support of new drug development and patient care.

Read McGrory’s story for the fascinating details, but the upshot is that the research program led to the development and, last week, FDA approval, of a new medication, Kalydeco, for the treatment of CF; while this drug only treats a rare subtype of the disease, McGrory notes that “it is widely believed to be a precursor to drugs that will treat a vast majority of the 30,000 children and young adults in the United States with cystic fibrosis.”

Four lessons here:

Lesson 1: increasingly, impactful translational research is driven by progressive, collaborative disease-focused organizations, such as the CF Foundation – a point Tom Stossel and I also emphasized in this WSJ op-ed a few years back.  We were especially struck by the determination of these foundations to seek out the best talent – whether in the private or public sector – in order to solve difficult problems of great interest.  While self-righteous academics, supposedly devoted to medical progress, would endlessly fret about protecting themselves from the “taint” (or asterisk) of industry association, disease foundations leaders kept their members best interests at heart, and aggressively looked for ways to leverage industry’s capabilities and perspective. 

Lesson 2: vision and passion matter.  As big pharma becomes increasingly process-oriented (see this unusually blunt recent assessment by the always-insightful Bernard Munos), there may be a gain in consistency but a loss in exceptionalism, energy, and delight.  I’ve heard directly from a well-regarded big pharma R&D leader that passion is essentially overrated, and it’s really strategy that is most important.  It’s possible that in a very large organization such as the one this individual runs, that’s effectively true.  But I still believe that precisely because science and progress are just so damn difficult, true advancement, and real novelty, if it is to occur at all, is much more likely to result from passion, appropriately enabled, than strategy, meticulously applied.  Most biophama projects are selected by some version of a balanced scorecard, generally a falsely precise weighting exercise.  I continue to believe we undervalue projects conceived out of the sort of passion that drove much of the CF drug development McGrory describes.  I’ve seen how jazzed a whole team can be working on a highly impactful endeavor – an energy that is typically replaced by a sort of sober professionalism on more typical projects.

Lesson 3: Industry is absolutely essential for delivering new products (so please stop crapping on us).  In many circles, pharma is a dirty word these days; stories like the one above explain why it shouldn’t be.  Fundamentally, industry is about solving problems and delivering solutions.  Making a new medical product is incredible difficult, and while smart academic research is often a vital first step, this fundamental science usually goes only a fraction of the way towards the goal of creating a viable new medicine; the folks in companies have to figure out how to turn a potentially exciting but usually quite preliminary germ of a concept into a fully realized, carefully tested product.  This is difficult, vital work which deserved to be celebrated and valued, its goals lauded, its practitioners recognized, its mission supported. 

I’m especially disappointed by what I’d term the almost casual anti-pharma sentiment that seems to pervade academia, where disparaging asides about industry appear to be the rule rather than the exception.  In many cases, this perception seems to be informed by a world view that is seriously outdated.  For example, in Eric Topol’s generally fascinating, must-read new book, “The Creative Destruction of Medicine,” the usually forward-thinking author (and Scripps Clinic cardiologist) writes, “The life science industry has no motivation to design drugs and devices that are only effective, however striking, for a small, well-defined segment of the population.”  This reflect an outdated view of industry, and is absolutely not the case today, when we see a range of companies – including most big pharmas, as well as a number of start-ups — aggressively pursue orphan disease strategies, and increasingly seek out the use of companion diagnostics to define responder populations.  To his credit, Topol does point to the Kalydeco example as evidence that the industry may be “rebooting” itself — although I suspect this sort of tailored approach to drug discovery has been going on for longer than he may appreciate, and is less unusual than he seems to imagine.

(Special reader challenge: if you have a great idea for a strikingly effective new drug that you feel industry isn’t pursuing because of the small market size, please share your suggestions in the comments section below….)

Lesson 4: Collaboration is a necessity, not a choice.  Making an impact on disease is tough stuff.  At one point, perhaps during the heady, early days of molecular medicine, the question seemed to be, “who is going to get there first?”  Now, as the biopharmaceutical industry has struggled to make a dent in some of the most pernicious diseases, and has seen itself contract in the process, the more relevant question seems to be, “is anyone going to get there at all?”  (Perhaps this is a question the currently cocky but fundamentally unproven digital health industry might contemplate as well.)

For the sake of patients and their families, and to meaningfully honor the lives of courageous souls like Joey O’Donnell who we were not capable enough to help, we must fight our hardest — and collaborate our best – to ensure that the answer to this question is “yes – absolutely.”

Dr. Shaywitz is an adjunct scholar at AEI.

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