It was a moment Anthony Carbajal will never forget: standing on the stage of one of TV’s most popular talk shows, fighting for a cause that touches him and his family profoundly. All while his mom looked proudly on.
The breakthrough discovery deals with a a protein called TDP-43, which can be found clumped outside the brain cells nuclei of ALS patients. When researchers introduced a protein designed to mimic TDP-43 into the neurons of mice, cells came back to life and were fully restored.
This was an incredible breakthrough for the field, as treatment could have the potential to slow down or even stop effects of the disease, which is currently incurable.
The next step is to create a therapeutic model to treat mice as whole organisms rather than just treating their cells. After that, researchers hope to be ready for human clinical trials.
On July 25, the ALS Association announced that researchers have identified a new gene, NEK1, which is one of the most common genes that contribute to the neurodegenerative disease ALS. A press release issued by the ALS Association notes that funding for this research came largely from donations raised by the 2014 viral ice bucket challenge, in which people posted videos of themselves dumping buckets of ice water on their heads as a way to raise awareness for the disease.
According to the ALS Association the challenge raised $220 million for research over the last two years—money that has been the catalyst behind two groundbreaking discoveries. Here’s how they could help end ALS.