But one year and more than $220 million in donations later, scientists at Johns Hopkins are claiming a major breakthrough in ALS research and are partly crediting the success to the massive influx of public interest.
"Without it, we wouldn't have been able to come out with the studies as quickly as we did," said Philip Wong, a professor at Johns Hopkins who led the research team. "The funding from the ice bucket is just a component of the whole — in part, it facilitated our effort."
Wong and his team have been studying ALS for about a decade, but as Jonathan Ling, another researcher at Johns Hopkins, said in an "Ask Me Anything" thread on Reddit, the millions of dollars brought into the field has given researchers the financial stability to pursue "high risk, high reward" experiments.
"The money came at a critical time when we needed it," Wong said.
The breakthrough research unravels the mystery about a protein called TDP-43. About a decade ago, researchers discovered that ALS patients often had clumps of the protein outside the nuclei of their brain cells, but it was unknown whether that was a cause or an effect of the disease.
In a study of the protein in mice cells, published last week in the journal Science, the Johns Hopkins scientists detail how TDP-43 — which is supposed to decode DNA — break down and become "sticky." They are unable to properly read the DNA and the cell dies within a few days.
When the researchers inserted a special protein designed to mimic TDP-43 into the neurons, the cells came back to life and returned to normal. That's sparked fervent interest that the treatment could possibly be used to slow down or even halt the disease.
It's a big step for the 15,000 Americans living with ALS, which currently has no cure, usually ends up killing people two to five years after they are diagnosed.
Wong said the next step would be to conduct more experiments to see if a the mimicking protein could be used as a therapeutic model to treat mice as an whole organism, rather than just cells. If that goes well, it would move into the clinical trials on people.
And with such a large amount of money just now being allocated to the research community, those trials are already funded.
Despite it's enormous attention on social media, ALS is a relatively rare disease. Somewhere around 7,000 Americans are killed by the disease year, according to the Centers for Disease Control — about two in every 100,000 deaths.
Critics have argued that such large attention to the single cause of ALS leads to "funding cannibalism" — that money given to the ALS cause is diverting resources to other research areas, perhaps for a disease that impacts more people.
Wong argued, though, that the money isn't coming from a fixed pie.
"In other diseases, you can take similar strategies to bring about awareness and have people come in to support it," he said. "This helps to drive other foundations to promote their cause. It's doing more good than bad."
The researchers also believe that understanding these proteins could have much broader impact beyond the ALS community. Wong said other diseases — such as Alzheimer's disease, muscular dystrophy, and inclusion body myositis — may also benefit from the treatment.
"The lesson that we learned can be applied to other human diseases," Wong said. "There's a much greater number of people affected by this."