ByKatie Camero
Published June 21, 2023
• 11 min read
In the summer of 2014, the ALS Ice Bucket Challenge quite literally drenched the internet, quickly becoming what is now considered the largest social media movement in medical history.
Seventeen million people uploaded videos of themselves to Facebook dumping buckets of ice water over their heads and challenging friends, family, and even celebrities to do the same. The viral sensation raised $115 million in just six weeks, and boosted critical awareness for a fatal disease that has few treatments and no cure—not yet at least. As the effects of the Ice Bucket Challenge continue to ripple, hope is on the horizon for ALS patients and their families.
“The Ice Bucket Challenge was incredible and unprecedented for any disease charity,” says Neil Thakur, chief mission officer of the ALS Association (ALSA). “When you have more money to invest, you have more chances for things to work—and that’s what we’re seeing now.”
ALS, or amyotrophic lateral sclerosis, is a disease that degrades nerve cells in the brain and spinal cord over time, leading to loss of muscle control and and, ultimately, inability to talk, move, eat, and breathe. Also known as Lou Gehrig’s disease, ALS affects more than 31,000 people in the United States, estimates show; most live only two to five years after symptoms develop.
(“Zombie cells” could hold the secret to Alzheimer’s cure.)
The Ice Bucket Challenge “dramatically accelerated the fight” against the disease, according to ALSA. It funded the work that led to the discovery of five new genes associated with ALS and novel drug therapies.
In the last year alone, the U.S. Food and Drug Administration approved two new treatments for ALS, both developed with funds from the challenge. Today, researchers are considered closer than ever to making ALS a livable, and eventually, curable disease.
Perhaps no one understands how impressive this progress has been, and the challenges that still lie ahead, better than Patrick Quinn—father of Pat Quinn, who was one of the Challenge’s three co-founders and who died of ALS in 2020.
“This was one of my son’s biggest feats,” Quinn says. “It’s what powered him through his seven-and-a-half year battle with ALS.”
An influx of funding changes the pace of research
Despite being described for the first time more than 150 years ago, ALS continues to puzzle scientists.
Researchers are still trying to understand how the disease works, what biomarkers can help diagnose and treat it, and the role genetics plays—both in the 10 percent of cases where people have a family history of the disease and the 90 percent of those without that family history who suffer from sporadic ALS. A better understanding of what genes contribute to the disease could ultimately lead to the development of effective treatments.
It took 126 years to find the first therapy for the disease: Riluzole, which can extend patients’ lives by about three months. That was in 1995—and it took 16 years to approve another treatment, Nuedexta in 2011.
Then the Ice Bucket Challenge happened in 2014.
It was both an exhilarating and exhausting several months for the ALSA. Brian Frederick, a spokesperson for the organization, says rooms were overflowing with checks, while organizations raising money to fight other diseases were calling desperate for advice to start their own campaigns. Accusations of slacktivism poured in as the organization struggled to quickly decide where and when to invest all the money. Despite the chaos, funds began to make their way to the ALS community a month after the challenge went viral.
As a result, ALS treatment clinics in the U.S. nearly doubled, and funding from the U.S. National Institutes of Health rose from $49 million a year in 2015 to a projected $220 million in 2024. Funds from the challenge also helped scientists at Dartmouth launch a research program to study environmental causes of ALS; in 2018, they found the disease is associated with increased mercury levels in toenails.
(CDC, FDA, NIH—what’s the difference?)
Most profoundly, the Ice Bucket Challenge led to an influx of new treatments by accelerating research already in progress before that summer. In 2017, the FDA approved Radicava, a drug that prevents nerve damage and slows ALS progression. Five years later, Relyvrio, which prevents nerve cell death by blocking stress signals in cells, gained approval. And in April 2023, the agency gave conditional approval to Qalsody, which shuts down the production of a toxic protein in the SOD1 gene—the first-ever treatment for a rare form of the disease.
“The Ice Bucket Challenge offered rapid resources and flexible funding: two things not usually found in the research environment,” says Don Cleveland, chair of the department of cellular and molecular medicine at University of California San Diego, whose research team helped develop Qalsody.
And there are clinical trials underway for more treatments. Cleveland credits the Ice Bucket Challenge money with helping his team in 2019 identify what they believe is one of, if not the most promising gene to target to ease symptoms in those with sporadic ALS. (An independent team led by Kevin Eggan at Harvard University discovered this gene at the same time.) Called STMN2, a clinical trial is testing how existing therapies affect it—just four years after the gene’s discovery, which is “very, very, fast in the pharma world,” Cleveland says.
Finding genes or mutations associated with a disease is time-consuming. “It’s like giving you 5,000 copies of War and Peace and asking you to find one typo of one word,” says John Landers, a neurology professor at UMass Chan Medical School who runs a lab focusing on developing ALS treatments.
Since the Ice Bucket Challenge, stellar progress has been made in improving ALS patients’ quality of life too. The influx of cash helped the startup Pison develop technologies that allow ALS patients who have lost the ability to speak to communicate and that can help doctors detect changes in patients’ motor neurons to monitor disease progression.
Greater collaboration changed the game
Since the Challenge’s inception, ALSA has committed more than $118 million to global scientific collaborations that have changed the course of ALS research.
“Early in my career someone said to me that ALS genetics is a full-contact sport. It can be very competitive, but one tremendous thing the Challenge did is bring more and more groups together,” Landers says. “And in genetics, more is always better.”
(Should you get tested for the gene mutation related to breast cancer?)
Ice Bucket funds made it possible to add an American arm to Project MinE, a global consortium for ALS genetics research that is the largest whole-genome sequencing effort of its kind. With the help of hundreds of researchers from dozens of institutions, the group, led by Landers, was able to identify a mutation related to ALS in the NEK1 gene, which plays a role in DNA damage repair and other cell functions. The group also discovered a mutation in the gene KIF5A that causes ALS.
Meanwhile, the ALS Research Collaborative (ARC), run by the nonprofit ALS Therapy Development Institute, also hit the ground running in 2014 after receiving funds from the challenge. It began collecting clinical and longitudinal data from more than 800 ALS patients and is now the longest-running natural history study of the disease, according to Carol Hamilton, vice president of development at ALS TDI.
Just last month, the group launched its ARC Data Commons program, which gives anyone in the world access to that data and will help scientists develop treatments to help slow, stop, or reverse ALS at a much faster pace.
“We would not have had the money to advance those programs into what they are today, and do it without sacrificing the rest of our work, had those ice buckets not fallen,” Hamilton says. “Sharing data with researchers around the world can raise the tide for every ship.”
There’s still more work to be done
Despite the Ice Bucket Challenge’s tsunami-like impact on ALS research, scientists agree much more work is needed to cure the disease. The first step: make ALS livable by 2030.
“We have to shift our science to keep up with the pace of change,” Thakur says. “Now that there are new therapies, we have to figure out how these therapies can work together, as well as how people can afford them and get treated in a timely manner. Both of those things are really hard.”
Even before ALS took his son’s life, Quinn understood that it takes a village to help ALS patients navigate the complexities of the medical system. For years he’s been working closely with Quinn for the Win to raise money for ALS research and ALS members of his Yonkers community, ensuring their journey with the disease isn’t any harder than it needs to be.
“Pat would say, ‘Every day, no matter how much adversity you think you may have, find a reason to find your smile,’” Quinn says. “We don’t want you to have a death sentence. We want to be able to say, ‘Yes, you have ALS, but there’s treatment and you can live a normal life.”
“This awareness we created back in 2014 is one of the major reasons ALS is still in the public eye,” he adds. “I promised my son I wouldn’t stop fighting, either until I’m gone or ALS is, whichever comes first.”
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