Follow the Money: The Ice Bucket Challenge raised big bucks on social media. Now, the ALS Association tells us where that cold, hard cash is going.

Shaw, Gina

doi: 10.1097/01.NNN.0000461242.12944.cb
Features: The Ice Bucket Challenge

The ALS Association tells us where the cold, hard cash from the Ice Bucket Challenge is going.

As snowstorms and icy winds batter much of the country, it's hard to imagine willingly pouring a bucket of ice water over your head for any reason. But last August—when temperatures were much more forgiving—that's exactly what about 3 million people did. In an astonishing example of the power of social media, a July 31 dare from former Boston College baseball player Pete Frates, who was diagnosed with amyotrophic lateral sclerosis (ALS) in 2012, quickly went viral.

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The dare? Dump ice water on your head within 24 hours of being challenged or donate $100 (or whatever amount you could afford) to the ALS Association or another organization supporting ALS research.

Pretty soon, the Ice Bucket Challenge meant dumping ice on your head and donating money, and everyone from your neighbor's preschooler to Bill Gates (with a custom-built ice douser), Oprah Winfrey, Jon Bon Jovi, and entire casts of Broadway shows were doing it—and posting their videos online. Even 86-year-old Ethel Kennedy took the challenge. (President Obama donated but declined the ice; actor Patrick Stewart posted a video of himself writing a check.)

In La Jolla, CA, prominent ALS researcher Don Cleveland, PhD, who pioneered a novel therapy that turns off the defective genes that contribute to neurodegenerative diseases, watched the online commotion with skepticism. But as the buzz grew into a cacophony of donations and awareness, he, too, saw the value of the challenge. By the end of August, when Dr. Cleveland and his lab taped their own Ice Bucket Challenge video, Frates's dare had raised more than $100 million. As of last fall, donations totaled approximately $115 million.

“Every time I think about it, I am overwhelmed,” says Nicole Ferrari, a mother of two grade-school children who was diagnosed with ALS three years ago at age 39. “I would not have been able to tell you what ALS was before I was diagnosed. But something as simple as dumping ice over your head has meant that now millions of people across the world are aware of this disease and the impact it has on people like me.”

For scientists like Dr. Cleveland, who have been cobbling together research grants from the National Institutes of Health (NIH) and much smaller donations to fund their ALS research, the challenge means their work can move forward faster.

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AND THE MONEY GOES TO…

In early October, the ALS Association announced an initial commitment of $21.7 million to four major programs; with matching gifts, the funds actually total $34.2 million. The programs were chosen because they “had synergies that strengthened each other,” says Lucie Bruijn, PhD, the ALS Association's chief scientist.

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NEURO COLLABORATIVE: $5 MILLION

The Neuro Collaborative will harness the efforts of four major ALS research groups around the country, including Dr. Cleveland's lab, that are working on several promising avenues of research. These include studies focusing on novel gene therapies to fix or replace defective genes associated with ALS, the therapeutic potential of human stem cells, and new targets for drug development.

Dr. Cleveland says that by the fourth quarter of 2015, the group hopes to begin human trials of an “antisense” drug that will attempt to block mutated genes from producing proteins that contribute to ALS in some patients. Research on gene therapy, which involves delivering an “improved” version of a defective gene directly to a targeted cell, where it will be expressed as a “good” gene, is further back in the pipeline. Still, Dr. Cleveland is optimistic that within three years a gene therapy will be nearly ready to go to trial. “Without the ice bucket funding, this would be going at a much slower pace,” he says.

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ALS ACT: $10 MILLION

ALS ACT (Accelerated Therapeutics), an academic-industry partnership that aims to accelerate testing treatments in clinical trials, has received a matching gift of $10 million from the ALS Finding a Cure Foundation, in addition to the $10 million they received from the ALS Association. The organization plans to use that money to develop brain imaging tools to look for biological signs, or biomarkers, of ALS progression. “Good, objective biomarkers not only help us see how the disease is progressing, but also help us determine whether new treatments may be slowing progression,” says Dr. Bruijn.

The biomarker initiative will help reveal the underlying biology of ALS, says Merit E. Cudkowicz, MD, MSc, co-chair of the Northeast ALS Consortium, chief of neurology and director of the ALS Program at Massachusetts General Hospital, and a member of the American Academy of Neurology (AAN). “Is this one disease or 100? I hope this helps us target patient populations for treatment trials—maybe some of the past trials have failed because we're lumping together people with different disease biologies.”

That's very important to Madeline Kennedy, a 61-year-old nurse from Troy, NY, who was diagnosed with ALS in 2012. She now uses a power wheelchair most of the time and has recently noticed that the strength and dexterity in her hands is deteriorating. “It's still hard to believe that this is consuming me, relentlessly,” she says. “When I talk to researchers, I ask, ‘What if some of these rejected therapies might work in some of us, just not all of us?’ People with ALS are a diverse group. It's more of a syndrome than a single disease. We can't let therapies that might work for some people sit on a shelf because we don't have a way to understand the differences among us.”

Funding for ALS ACT will also support the development of therapies targeting two biological processes that contribute to ALS: the production of misfolded proteins—proteins that have developed structural “errors”—within motor neurons, and inflammation in brain cells. And it will buttress big data-sharing projects, including NeuroBANK, which gathers data from many clinical studies, and the Northeast ALS Consortium Biorepository, which is building a large bank of biological samples for ALS investigators. The samples are matched with extensive clinical, environmental, and demographic information.

ALS ACT has also requested proposals for larger clinical trials to test the efficacy of different therapies, which it is hoping to fund, with $1.5 million from the Ice Bucket Challenge, by March 2015.

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NEW YORK GENOME CENTER: $2.5 MILLION

Thanks to Ice Bucket Challenge donations and a matching gift from the Tow Foundation, the recently established New York Genome Center, under the leadership of Robert Darnell, MD, PhD, and Hemali Phatnani, PhD, has carved out a niche just for ALS, says Dr. Bruijn. “The center hopes to identify the unique targets and pathways linked to the diseases,” she says.

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PROJECT MinE: $1 MILLION

Efforts at the New York Genome Center will be complemented by Project MinE, an international program started by Bernard Müller, a Dutch ALS patient. The program aims to identify mutations in genes linked to inherited or familial forms of the disease, as well as to sporadic ALS, which is not linked to a specific cause, in at least 15,000 people.

“A global effort is essential in identifying new genes, as various populations have more prominent representation of one or another gene or may have gene mutations unique to a particular geographic population,” says Dr. Bruijn. The project will be led by John Landers, PhD, at the University of Massachusetts in Worcester, MA, and AAN member Jonathan Glass, MD, at Emory University in Atlanta.

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CERTIFIED TREATMENT CENTERS OF EXCELLENCE: $3 MILLION

The ALS Association awards grants annually to its 43 Certified Treatment Centers of Excellence, which are centers established around the country to define and support a national standard of care, and the Ice Bucket Challenge money allows them to increase these grant amounts from $12,500 to $25,000 per center for the next three years. Some of the funding will also be used to develop regulatory guidance for ALS drug development.

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AND THE REST GOES TO…

The ALS Association plans to work with a wide range of partners and will continue to solicit ideas from the broader ALS community on how to distribute the remaining $90 million in Ice Bucket Challenge funds, says Dr. Brujin. “We're talking with other ALS organizations about partnerships, including more recently Target ALS, the group at Columbia University led by Chris Henderson, PhD.” Dr. Henderson is director of Columbia's Project ALS/Jenifer Estess Laboratory for Stem Cell Research, as well as the ALS foundations Prize4Life and Team Gleason.

Other initiatives in the pipeline include a program to build mouse models of ALS more quickly and a grant program designed to move promising drugs through the preclinical phase of testing so they can get into clinical trials faster.

“There's great work being done in academic medical centers, but you need an industry partner to develop treatments,” says Dr. Bruijn. “We will cast our net wide initially with smaller funds, and then focus on the most promising initiatives.” The call for these proposals will likely be issued this spring, she says.

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Missed the Ice Bucket Challenge?

You don't need to dump ice on your head to donate to ALS research, as Sir Patrick Stewart so eminently proved in his own brilliant video (which featured the actor sitting at a desk and writing a check; see above).

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Leading ALS organizations to which you can donate include:

* The ALS Association: http://www.alsa.org

* Project ALS: http://www.projectals.org

* The ALS Therapy Development Institute: http://www.als.net

* The Muscular Dystrophy Association's ALS Division: http://www.als-mda.org

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Patients Ponder the Challenge

People with ALS say they have been overwhelmed by and thrilled with the outpouring of support from the Ice Bucket Challenge, but some wish there had been more collaboration between the ALS Association and other ALS-focused groups like the ALS Therapy Development Institute (ALS TDI), a patient-driven organization founded in 1999 with the singular focus of developing ALS therapies.

“It seems hard to get the premier ALS organizations to work together,” says Tom Murphy, a 56-year-old retired defense contractor in Virginia who was diagnosed with ALS in 2010. “We need a patient-driven, mutual objective that reduces duplication in a limited funding environment.”

* More Transparency. Murphy also thinks decisions about how to use the funds should have been more transparent. He would have liked to have seen a public summit with patients, caregivers, and the premier ALS research organizations, or at least a global survey asking patients and caregivers how to spend the windfall.Dr. Bruijn says patients were involved in the decision-making process and that the ALS Association held strategic meetings with investigators over the past year. “In addition, the ALS Association has partnered with several ALS organizations in the past, including the Muscular Dystrophy Association (MDA), the ALS Therapy Alliance, the Robert Packard Center for ALS Research, Project ALS, and the Motor Neuron Disease (MND) Association UK, and will continue to do so,” she says. “ALS TDI, unlike the organizations mentioned as partners, is not a funding organization. It has been encouraged to submit proposals to the ALS Association for funding, but to date has not done so.”

* A Bigger Pool of Players. While praising the work of the ALS Association, Catherine Wolf, a psychologist and human-computer interaction expert, as well as a frequent contributor to Neurology Now, who has been living with ALS since 1996, says she wishes the funds had gone to a larger pool of organizations—“specifically the ALS division of MDA, which in previous years was the biggest fundraiser for ALS, and ALS TDI,” she says.

* Emphasis on Quality of Life. Wolf also urged the ALS Association to focus not only on long-term goals of finding cures, but also on improving the quality of life for people living with ALS right now, through both expanded patient services and research. It would be helpful to know more about the role of diet and nutrition in disease progression, for example. And the funds could help patients pay for assistive technology and other products or services that improve their quality of life, Wolf adds.

* Keep Hope Alive. For Denis Rizzuto, whose wife, Christie, was diagnosed with ALS five years ago, the Ice Bucket Challenge represents hope—“hope that there are answers out there,” he says. “But it can't stop. A hundred million dollars is a lot of money, but it's not enough to bring a single drug to market. It helps us understand, but it's not going to answer all the questions. We have to keep raising awareness.”Nicole Ferrari is optimistic about the future. “The ALS Association is so collaborative and well connected, there's real hope that we can work together and move the research forward—if not to find a cure, then at least to delay or prevent the progression of this disease,” she says.

© 2015 American Academy of Neurology