Friend and fam: Most of you know I’ve been associated with ALS research for the past decade. The Ice Bucket Challenge is spectacularly heartening, but raises in my mind raises a cold truth: We need a new paradigm in ALS research itself. We all know people who have survived cancer, yet no one survives ALS. There are no breakthrough medicines for ALS, and none are even close. You probably know I recently had a front-row seat to one of the greatest disappointments in the history of ALS research—the failure of a promising drug in the largest Phase 3 trial ever conducted in ALS. We do have a path forward for this drug, but in fact the entire field of ALS research needs a new path forward. The problem begins with the nature of ALS itself. This is a disease of multiple subtypes, perhaps even a collection of diseases. Yet we conduct one-size-fits all clinical trials, in which even the most promising treatments fail. ALS is spectacularly complex, involving many intersecting molecular pathways, yet any individual scientist typically works in only one narrow aspect of the disease. This reductionism is reinforced by government and charitable funding policy, because a discovery in one narrow area tends to drive more funding to the same area. A “lock-in” effect takes hold, leaving many fresh questions unfunded, unanswered, and “locked-out,” especially those questions at odds with the prevailing research paradigm, which, let’s face it, has not yet yielded a cause or a cure, or even a meaningful treatment. These problems aren’t unique to ALS research, but they may be uniquely vexing in a disease as complex as ALS. The current paradigm leaves too little funding for integrating specialized discoveries—for systemic, boundary-spanning approaches that cut across funding silos. Attacking a disease that has resisted all attacks demands overcoming research boundaries, not reinforcing them. Most of you know how curious my daughter Janis is. Not long ago she joined me for the final morning of an ALS research forum in Chicago before we spent a weekend touring colleges there. The hall was filled with brilliant, dedicated scientists from around the world reviewing the latest work in the field. “All of these people do ALS research?” she asked. “And no one knows the cause?” A giant THANK YOU to my Facebook family and friends who have participated in or written about the Ice Bucket Challenge, including among others Kevin Salwen, Neil Alexander, Amanda Edwards Petzinger, Amanda Bennett, Steve Frazier, Walt Mossberg, Jerry Gindele, Carolyn E Green, Kerri Walker Edwards, Sandra Block, Jim Mather, Lee Carren Ward Mather, Lorretta Gasper, George Anders, John Harwood, Jenni Franz, John Yang, Rachael Markijohn, Tom Reinsel, Cathy Cawood Bubash, and Liz Holter. We really needed this shot in the arm, and please keep it up. But as the awareness and donations increase, let’s hope we’re providing all of those dedicated researchers with more than just another year or two of funding. Let’s hope they’re now able to ask bold, new questions; that they’ll cross research boundaries instead of remaining stuck in them; and that in discovering new medicines with novel mechanisms, they’ll also employ rapid and innovative ways of testing them in clinical trials. Because wrestling this terrible beast to the ground requires more than just more money. It needs new thinking. Thanks a million.
Posted on: Sun, 24 Aug 2014 01:20:20 +0000
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