Last week I spoke on a couple of panels at the BIO Global Convention in San Diego. At one point I found myself seated next to Joshua Boger, the CEO of Vertex Pharmaceuticals, which recently announced the development of a new drug for cystic fibrosis. He told the audience, in as many words, that without the Cystic Fibrosis Foundation (CFF) as a 10-year funding partner, this $70 million therapy would not exist. That's a head-turning statement to say the least, both for people with CF and for Vertex's shareholders.
Yet The Michael J. Fox Foundation and CFF were among only a few medical research philanthropies who chose to come and be counted among BIO's 20,000-plus attendees. I fielded huge curiosity about how industry could best obtain foundation funding to pursue "high risk/high reward" work. But I heard from countless industry reps that MJFF and CFF are the exception, not the rule, in partnering with for-profits.
Why the low penetration of this approach in spite of the obvious and tangible benefits and the efforts of groups like Michael Milken's FasterCures to build momentum? Maybe it's got something to do with the idea, eloquently refuted in a recent Boston Globe Op-Ed by David A. Shaywitz and Dennis A. Ausiello, that academic researchers who partner with industry are "intrinsically tainted." Maybe other factors are at work. But in disease research funding circles, there is endless talk about the broken medical research enterprise and the pressing need for new funding paradigms. That's why over the past 10 years (starting with Bob Beall's efforts at CFF), a handful of foundations have explored different ways of driving toward cures -- and evidence continues to accumulate that funding companies could be a very big wave to ride.
I can speak only for MJFF's experience, but I think it's pretty compelling. With 21 active industry partnerships at the time of this writing, we're funding the pursuit of multiple disease-modifying PD treatments that likely otherwise would have been shelved for lack of capital. Our investments are frequently leveraged, with companies attracting both external and internal follow-on funding once we've signed on. We routinely seek out the opinions of industry decision-makers -- both on the science and business development sides -- to inform our opinion of the field. Their expertise on drug development is a force multiplier for interpretable outcomes from our academic and industry investments alike.
No one advocates a complete shift away from funding academic research. But medical research philanthropy, funded largely by patients themselves, is uniquely motivated to bridge the gap between discoveries in university labs, drug development efforts within companies, and therapies in the clinic. And to do that effectively, we must work as intellectual and financial partners with industry, from tiny biotechs to Big Pharma.
So what will it take to make this happen on a larger scale? I'd argue the field needs two things in greater measure: cross-talk among leaders and boards of different foundations, and honesty with donors about what their contributions are achieving.
Private philanthropists need to break out of the collective comfort zone of a traditional, "academics only" approach. Identifying a prize-winning researcher or a celebrated institution to support is simply not an effective way to get to the life-transforming therapies that are so desperately needed. But that wouldn't be obvious to an individual donor. It doesn't become clear until you get a comprehensive view of the field -- the bird's-eye-view of an MJFF or a CFF, reviewing hundreds of grant proposals in our respective indications each year, working day in and day out with a who's-who of academic and industry scientists toiling in the field.
So MJFF forges ahead with the experiment. It's early days yet, and we're still part of a lonely minority. But we believe the payoff in terms of field-advancing results is likely to convince far more organizations -- and their donors -- that this is an approach foundations can't afford not to take.