How The Ice Bucket Challenge Has Changed ALS Research

One year and $100 million later...
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Last summer was full of people dumping buckets of ice on their heads in the name of funding ALS research. A year later, it looks like it was totally worth it.

The Ice Bucket Challenge raised over $100 million, according to the ALS Association, which is huge when you compare it to the $2.8 million it raised during the same period of the previous year.

And it turns out that this funding has already led to some exciting advances and discoveries in just a year -- a very short period of time in the field of medical research. During a Reddit AMA, researcher Jonathan Ling opened up about how helpful the Ice Bucket Challenge has been.

"I remember reading a lot of stories about people complaining that the ice bucket challenge was a waste and that scientists weren’t using the money to do research, etc. I assure you that this is absolutely false," he wrote. "All of your donations have been amazingly helpful and we have been working tirelessly to find a cure."

In case you need a refresher, ALS (amyotrophic lateral sclerosis), or Lou Gehrig's disease, is a fatal disease that attacks nerve cells responsible for controlling muscles and progresses quickly -- most people with ALS die within 3 to 5 years after being diagnosed, according to the National Institute of Neurological Disorders and Stroke.

Since the massive fundraiser, researchers have been able to further investigate TDP-43, a protein in cells that has a link to ALS. Thanks to all the donations, Ling and researchers were able to confirm that TDP-43 isn't doing its job in 97% of all ALS cases.

Here's how Ling explains TDP-43 in his AMA:

DNA is located in the nucleus of a cell. You can think of a nucleus as a library except that instead of having books neatly lined up on shelves, the books in a nucleus have all of their pages ripped out and thrown around randomly.

To sort through this mess, the cell has great librarians that go around collecting all these pages, collating them and neatly binding them together as books. These librarians then ship these “books” out of the nucleus so that other workers in the cell can do their jobs. Think of these books as instruction manuals.

TDP-43 is a very special type of librarian. TDP-43’s job is to ensure that nucleus librarians don’t accidentally make a mistake and put a random nonsense page (usually filled with gibberish) into the books that they ship out. If one of these nonsense pages makes it into an “instruction manual”, the workers in the cell get really confused and mess things up. For terminology, we call these nonsense pages "cryptic exons."

In the brains of ALS patients, some cells begin to get sick because TDP-43 becomes really sticky and clumps together outside the nucleus, where it can’t do its job.

So, what's next? Ling hopes to have therapies based on his research within the next two to three years. In the meantime, the ALS foundation has plans to bring the challenge back "every August until a cure," Julie Frates, the wife of Pete Frates (the co-founder of the challenge) told CBS Boston's WBZ in July.

In case you haven't noticed, it's August. Let the ice-dumping begin.

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