In the viral video craze of the summer, millions of people dumping freezing water on their heads generated an unexpected deluge of more than $100 million to support amyotrophic lateral sclerosis charities. The ALS Association took in $112 million between July 29 and September 11 alone. Where should all that money go? 

“These funds will be used to fund cutting-edge research as well as care and support for people living with the disease,” promised ALSA chief executive officer Barbara Newhouse in a press release. Experts interviewed by Alzforum had no shortage of ideas for spending the research slice of the pie. Their priorities coalesced around tissue banking, biomarker discovery, and therapy development.

In the ice bucket challenge, social media users “challenge” others with a choice: Undergo a frigid dunk and post the video online, or donate money to the ALS charity of your choice—ideally, of course, both. Challenge recipients have 24 hours to respond, and once they do they can nominate others. The wave engulfed public figures from former president George W. Bush to the family of physicist Stephen Hawking, who has ALS. Others, such as President Obama, preferred to write a check. ALS clinics have challenged each other, and research groups have gotten soaked en masse. (Timothy Miller led Washington University St. Louis’s neuromuscular team in this video.) “It is fantastic,” said Jeffrey Rothstein of the Johns Hopkins University School of Medicine in Baltimore. “It is raising awareness and money for a disease that badly needs it.”

Neurologist Terry Heiman-Patterson of Drexel University College of Medicine in Philadelphia, Pennsylvania, takes the Ice Bucket Challenge.

As with many fads, the precise origin of the ice bucket challenge is uncertain, but it was not originally about ALS. Earlier this year, golfers and other sporting enthusiasts were challenging each other to either douse themselves or donate to a charity of choice. For ALS, the challenge took off with the involvement of an intrepid trio: pro golfer Chris Kennedy of Sarasota Florida, who has a relative with ALS, and Pat Quinn of Yonkers, New York, and former Boston College baseball player Pete Frates, both of whom have ALS. From there, the challenge spread to Frates’ network of sporting buddies and the ensuing bucket brigade made ALS fundraising history. (See story). 

Breaking the Bank
As a result, ALSA has been taking in record donations since the end of July. Last year, by comparison, it received $2.8 million between July 29 and August 29. This year it accepted $100.9 million over the same time period. For a while this August, about $9 million a day flowed into ALSA coffers. “Every day we saw the opportunities get bigger and better and more exciting,” ALSA communications officer Carrie Munk said in an interview with Alzforum. The outpouring of support has ALS nonprofits “euphoric … thankful … exhausted,” said Steven Perrin, president of the ALS Therapy Development Institute in Cambridge, Massachusetts. Staffers have been working 12- to 18-hour days just to keep up contact with new supporters, he told Alzforum. ALS-TDI has collected about $3.5 million so far.

The $100 million-plus windfall is more than a drop in the bucket. In a typical year, the National Institute of Neurological Disorders and Stroke (NINDS) slates approximately $40 million for ALS research, said neurodegeneration program officer Amelie Gubitz, and she estimates that ALSA and other nonprofits together contribute a similar amount. This compares with about $500 million annually the National Institutes of Health spends on Alzheimer's disease research, and approximately $1.3 billion per year on HIV/AIDs research. As for AD, there is no known prevention for ALS, and only one treatment that extends life by a few months. At the moment the NINDS supports a single ALS clinical trial, a Phase 2 stem cell transplant study

The difference between government and ice bucket money, of course, is that the government ponies up every year, noted George Vradenburg, chairman of USAgainstAlzheimer’s in Washington, D.C. “As good as this is, can you make it sustainable?” he asked. Even if this summer’s fad is unlikely to repeat, Brian Kraemer of the University of Washington, Seattle, said that the challenge has raised public awareness of this orphan disease, and that bodes well for future fundraising efforts.

ALSA has not yet decided how to spend the sudden wealth, Munk said. The association's national office normally operates on an annual budget of about $25 million, with 25 percent going to research. The decision on the new funds rests with ALSA’s Board of Trustees, who will meet in mid-October to develop a plan. Grant applications will undergo the same peer-review process as always, though the new funds may allow ALSA to support projects that had merit but went unfunded in the past, Munk said. “I suspect [ALSA] will do the right thing,” commented Rothstein. “They have a long history of funding really good research.” According to its website, ALSA currently funds nearly 100 studies and has spent more than $99 million on research since 1985.

Spending Priorities
What tops the wish list for ALS researchers? At ALS-TDI, Perrin already has a plan for $2.5 million. “What do you do when you have a windfall of cash and a sense of urgency?” he asked in a Web post. “Spend it.” One million dollars will go to support a new program in which the institute has invited people with ALS to come in for a multipronged evaluation that includes whole-genome sequencing, creation of personal induced pluripotent stem cell (iPSC) lines, and continuous movement evaluation via wrist and ankle accelerometers. ALS-TDI will use those iPSC lines to screen drugs, and Perrin hopes that once therapeutics become available, those cells will help researchers select the treatment that works best for each ALS patient. Initially, he had hoped to enroll 20 people in the program by the end of 2014; now, he has upped his goal to 100 participants by year’s end. And while the program was intended to be available only to people who could visit the Cambridge office, he now expects to roll it out at four other centers across the country.

Furthermore, Perrin has designated $1.5 million to speed up a clinical trial of a potential new ALS treatment. Negotiations with the company that owns the drug are underway, and he hopes to announce a collaboration soon. As for the remaining $1 million ALS-TDI has received so far, Perrin is still pondering where it would be most useful.

Experts who spoke with Alzforum offered plenty of suggestions for how to invest the ice bucket money. Charities both large and small need to think about how they can “leverage” donations for research benefits in the long run, said Terry Heiman-Patterson of Drexel University College of Medicine in Philadelphia. She would like to see ALS tissue banks for biomarker research expanded. The Northeast ALS Consortium, which Heiman-Patterson co-chairs, runs such a repository (see Oct 2011 news story), but more samples could help researchers hunt for potential biomarkers, she suggested. She said that local ALS clinics need funds to hire personnel, including specialists who can run clinical trials, so they can spring into action as soon as new drugs are ready for testing.

James Heywood, founder of ALS-TDI and chairman of PatientsLikeMe, a networking site for people with ALS and other conditions, suggested that the primary task would be to do large-scale natural history studies of patients. These kinds of studies are transforming Alzheimer’s and Parkinson’s research. A natural history study of ALS patients would require multiple time points and have a price tag of more than $5,000 per patient, and so would have been nearly unthinkable until this summer, Heywood said.

One-Time Funds Make Major Advances
While $100 million could fund big projects like Heywood’s proposal, even small investments sometimes yield big benefits, noted Heiman-Patterson. She is president of the ALS Hope Foundation, a small Philadelphia nonprofit that has netted $60,000 so far from the ice bucket trend. For example, in the past the Foundation funded a $25,000 study to change the splice forms of glial and neuronal AMPA receptors, which had been implicated in ALS pathology. That project ended up receiving large federal grants and growing into LifeSplice Pharma, LLC, of Malvern, Pennsylvania, which is developing two splice-modifying treatments that extend life in ALS model mice.

“Bumps in funding can make a huge difference,” agreed Rothstein. He and Gubitz likened the ice bucket cash infusion to the additional research funds from the 2009 American Recovery and Reinvestment Act. The extra money doled out to the NIH allowed NINDS to invest in large-scale ALS projects that were considered “bold” at the time, Gubitz said. It was the first time NINDS had funded whole-genome sequencing for ALS, and that and other projects aimed at iPSC banks and biomarker studies were considered risky, with no guarantee of success. Thanks to those extra funds, Rothstein today runs a program with more than 50 iPSC lines for ALS research (see Sep 2010 news story). 

The ice bucket phenomenon may not be over yet. It was featured on “The Ellen Degeneres Show” season premiere on September 8. The unprecedented donations illustrate how social media and a simple challenge can make a difference for one disease. Other fundraisers are taking note with new challenges (see NBC News story). In one version, participants douse themselves in soapy water and give away hand sanitizer to fight the spread of the Ebola virus. In an Indian variant, people post pictures of themselves holding buckets of rice and then donate that rice to a food bank. In another spinoff, Vradenburg and USAgainstAlzheimer’s initiated a tongue-twister challenge to raise money to fight Alzheimer’s.—Amber Dance


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News Citations

  1. NEALS: Sharing Among ALS Researchers and Participants
  2. Hereditary Diseases: A Natural Fit For iPSC Modeling

External Citations

  1. ALS Association
  2. press release
  3. George W. Bush
  4. Stephen Hawking
  5. video
  6. story
  7. Phase 2 stem cell transplant study
  8. USAgainstAlzheimer’s
  9. website
  10. Web post
  11. repository
  12. PatientsLikeMe
  13. LifeSplice Pharma, LLC
  14. “The Ellen Degeneres Show”
  15. NBC News
  16. tongue-twister challenge

Further Reading