In 2014, millions of people uploaded videos of themselves pouring ice water over their heads to raise awareness of amyotrophic lateral sclerosis (ALS). ALS is a disease where motor neurons, the cells that control muscles, die. As those cells die, people lose their ability to walk, speak, move, and breathe. There is no cure; most people die within five years of diagnosis.
The Ice Bucket Challenge was a playful way to fight a horrific disease orchestrated by charismatic leaders who themselves have ALS — Pat Quinn and Pete Frates. And it worked. More than 17 million people posted videos to Facebook, raising over $200 million around the world. The ALS Association received $115 million, which was many times more than our annual operating budget.
The Ice Bucket Challenge was a "lightning strikes" moment with a lasting legacy. And as we've learned over the past five years, the challenge — and the funds it raised — serve as an important case study for research funding in general. With help from RTI International, an independent nonprofit research institute, we evaluated the impact of Ice Bucket Challenge spending on our own research program and have identified research funding practices that have proven effective.
First, the funds raised by the challenge helped support the ALS research infrastructure, enabling us to better coordinate clinical trials across the nation, share data and specimens with scientists at no charge, and identify new genes (five to date) that cause ALS. In 2014, we were able to support 42 principal investigators, and by the end of 2018 that support had grown five-fold, to 237 principal investigators. In addition, the number of researchers collaborating on ALS-related scientific papers has nearly doubled.
Supporting collaboration in all its forms — whether on studies, helping each other stay focused and productive, and unfettered sharing of resources and ideas — is imperative. The ALS research community is getting too big to rely on informal information exchange as the sole means of preventing wasteful duplication of effort. As our space grows in complexity, we need to ensure that we do not create silos or bottlenecks in the flow of resources and ideas. That means more collaboration, of the kind we have supported with Ice Bucket Challenge funds, is needed if we want to see more researchers working urgently and efficiently toward a cure for ALS.
Collaboration in the ALS space has already borne fruit. Some of the recent discoveries in ALS research simply would not have been possible without the infrastructure and collaboration we've supported. We were able, for example, to fund multiple gene studies and databanks that pooled information and talent. More than 250 scientists around the world analyzed over 100,000 samples to identify a new genetic link to ALS, the KIF5A gene. That work is critical, as each new gene discovery is a new opportunity to develop ALS treatments.
Collaboration also creates an imperative for transparency and accountability. When ALS charities are transparent about what they fund, scientists and other funders can better plan ahead. We post our funded awards here, and we are working on approaches to make it easier to track our funding and impact. We've also adopted open science practices that encourage aggressive sharing of work; things like sharing scientific papers with colleagues at no cost, publishing preprints (complete public drafts of scientific papers), and publicly pre-registering scientific protocols before research commences are key steps to moving us all forward.
Finally, RTI found our Ice Bucket Challenge spending served as an important "multiplier" of funding from other sources: as a direct result of the $40 million in Ice Bucket research funding we awarded, ALS scientists reported receiving an additional $122 million in follow-on grants from other funders, including the federal government.
That multiplier, both in terms of dollars spent on ALS and collaboration by scientists working on ALS, leads to faster research and additional discovery, which in turn creates greater impact. Looking ahead, we expect to see an increase in the number of drugs tested in clinic, an acceleration in the speed of those trials, and for these trials to result in new treatments. We will accept nothing less, as people who are living with ALS have no time to spare.
The Ice Bucket Challenge was a transformative gift from the world to the ALS community. It's a phenomenon with staying power that has created an important blueprint for research funding, one in which sharing and collaboration increase the pace of discovery and make all the difference in the world. It's up to us, the scientists and funders who fight ALS, to embrace that legacy and move even faster.
Neil Thakur, PhD, is executive vice president, mission strategy, the ALS Association.