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Your $220 million to the ALS ice bucket challenge made a difference, study results show


Stashed in: Science!, Restore your faith in humanity., Philanthropy!, ALS Ice Bucket Challenge

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Most of us long ago passed the point of "charity fatigue" with all those marathons and bike rides we sponsored for research into medical conditions that don't seem all that significantly less deadly and debilitating... so I appreciated this timely update from the "ice bucket challenge" people on where the money went, and how a significant therapy might have been discovered!

Yes, it's good to see they've used the money wisely!

This seems like a promising breakthrough.

Philip Wong, a professor at Johns Hopkins who led the research team, told The Washington Post the donations helped. 

Less than 12 months later, Science released a paper by ALS researchers about TDP-43, a protein that is common in patients diagnosed with the disease. Following studies of the protein in mice cells, scientists better understood the purpose of the protein: to sort a nucleus' DNA in an orderly fashion. 

And on Reddit, Jonathan Ling, a researcher on the team, helped moderate a discussion based on the report and described why the results were so important.

"We’ve known about TDP-43 for nearly a decade but never really understood what it did," Ling wrote. "Today, in our Science paper, we actually show evidence of cryptic exons in the brain autopsies of ALS cases, suggesting that some of our theories were right all along: TDP-43 isn’t doing its job correctly in ALS."

Most exciting however, is that when scientists devised and genetically input a special protein to imitate the managerial role of TDP-43 in mouse stem cells, the frail cells came back to life and worked as usual.

"If we are able to mimic TDP-43’s function in the human neurons of ALS patients, there’s a good chance that we could slow down progression of the disease!" Ling explained. "And that’s what we’re putting all our efforts into right now."

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