Scientists are crediting the ALS Ice Bucket Challenge — which helped raise awareness about amyotrophic lateral sclerosis, or Lou Gehrig's disease — with supporting a major breakthrough in the search for a treatment, the Washington Post reports.
Researchers at the Johns Hopkins University School of Medicine told the Post and Reddit that the campaign, which went viral in the summer of 2014 and has raised more than $220 million globally over the past year, has provided the public support and financial stability needed to facilitate "high risk, high reward" experiments. In a study published last week in the journal Science, Johns Hopkins researchers detailed how in mice cells, TDP-43, which is supposed to decode DNA, can break down and become unable to do so, but when a special protein designed to mimic TDP-43 is inserted into the neurons, the cells return to normal. The findings have sparked hopes that the treatment could be used to slow or even halt the progress of the disease.
"Without [the Ice Bucket Challenge], we wouldn't have been able to come out with the studies as quickly as we did," said Philip Wong, who led the team and has been studying ALS for about a decade. "The money came at a critical time when we needed it."
The next step would be to conduct more experiments to see if the TDP-43-mimicking protein could be used as a therapeutic model to treat mice as a whole organism and not just at the cell level — and, if that goes well, to move it into clinical trials involving people. With hundreds of millions of dollars now available to the ALS research community, those trials are already funded. The $115 million the ALS Association collected last year amounts to a 4,100 percent increase in fundraising from the $2.8 million the organization raised in 2013.
About fifteen thousand Americans currently live with ALS, which usually results in death two to five years after someone has been diagnosed. Critics have argued that such concentrated attention on a single disease leads to "funding cannibalism" — that money given to support ALS research is diverting resources from other research areas, including diseases that affect more people.
However, Wong argues that the money isn't coming from a fixed pie. "[Y]ou can take similar strategies to bring about awareness and have people come in to support [research into other diseases]," he said. Researchers also told the Post that understanding the proteins implicated in ALS could have impact beyond the ALS community and lead to treatments for Alzheimer's disease, muscular dystrophy, and inclusion body myositis. "The lesson that we learned can be applied to other human diseases," said Wong. "There's a much greater number of people affected by this."