By Meg Tirrell
Last summer, neurology professor John Landers was stuck. His grant proposal for funding to sequence the genomes of patients with amyotrophic lateral sclerosis, or Lou Gehrig's disease, as part of a worldwide project to better understand the illness, had been rejected. The ALS Association, a nonprofit that supports research, didn't have the funds. Then about 3 million people dumped buckets of ice water over their heads-and everything changed.
"We had a grant in with them asking for about $1 million, and although they were very excited about the project, it was beyond what they were able to do," Landers said in an interview from his lab at University of Massachusetts Medical School. "However, luckily the Ice Bucket Challenge kicked in and as a result of that, they were able to fund this."
The Ice Bucket Challenge -- a viral sensation that involved daring friends to dump ice on themselves or donate money to ALS research -- raised $115 million for the ALS Association last summer (many people both dumped and donated). That compares with its total annual budget, including regional chapters, of $60 million. It's also spurred international awareness about a crippling neurodegenerative disease with no good treatments, and which is often fatal within two to five years of diagnosis.
Understanding the Cause
ALS afflicts about 30,000 people at any given time in the U.S., and about 5,600 people are diagnosed with it each year. But its causes aren't well understood, making research like Landers' -- called Project MinE -- crucial.
"To the extent that we can understand what's going on on the genetic side, those are things we can target through drug development," Landers said. "The best way to think about this is: You bring your car into the garage and it's just not working right; until you figure out what's going wrong with the car, you can't fix it."
The grant to Project MinE was one of four the ALS Association announced in October as part of the initial $22 million it allocated. The others are going to a partnership between academia and industry, called ALS Accelerated Therapeutics, to speed drug development; the New York Genome Center, to further explore the genetic basis of the disease; and three medical labs in California in a project called Neuro Collaborative, which also works on drug development. The association aims to invest $21 million to $25 million a year in research projects, spokeswoman Carrie Munk said.
The funding has also enabled more support for patients and their caregivers. The Greater Philadelphia chapter, for example, used Ice Bucket Challenge funding to buy assistive technology devices to loan to people with ALS who can't afford them: power wheelchairs, or eye-gaze technology for communication, she said.
"Another chapter was able to reinstate a respite care program, giving caregivers of people with ALS a break," Munk said by telephone. "Previous to the Ice Bucket Challenge, they had to cut the program; now they can provide it to people who need it."
The funds, Munk said, have energized the research community. The ALS Association has received triple the number of applications for grants for young scientists than in previous years. "A lot of times that funding enables them to continue a lifetime career in the area of ALS," she said.
Other nonprofits saw more funding as well, though the ALS Association took in the bulk of it. Project ALS, a group that aims to fund collaborative research on the disease, took in $750,000 from the Ice Bucket Challenge, almost 10 times more than in the same period the year before.
The organization has already allocated all of the funds to research, including projects focused on testing existing drugs for use in ALS. Others supported research aimed at better understanding whether lipid metabolism, central to rare maladies like Gaucher's disease and Fabry's disease, plays a role in ALS. "We wouldn't have been able to do it without that money," Valerie Estess, director of research at Project ALS, said in a telephone interview.
There's a concern, though, that the challenge will have provided just a one-time boost in funding, and then could peter out. "Of course you'd want it to continue, but what you're hoping with the initial burst in support is that you're seeding pilot studies that are going to bear fruit," said Estess, whose sister was diagnosed with ALS at age 35. "To me, nothing speaks louder than actual data."
Promising projects may then be able to draw funding from the National Institutes of Health, researchers said. And while it may be unlikely to see a repeat of the viral videos this summer, the ALS community is hopeful the effects of a challenge that encompassed so many people will continue to pay off.
"Even if people don't dump ice over their heads in August, hopefully every year, people will remember the Ice Bucket Challenge," Munk said. "We asked people, 'Why did you participate in the Ice Bucket Challenge?' The No. 1 answer was: 'Because I was asked to.'"
By Meg Tirrell