Story 3 begins — ALS: what’s failing and why models didn’t translate

1:37:42it's been in the news recently um because of the uh recent death of Eric Dayne. Um, many of you have maybe saw the uh Netflix documentary that came out. Um, for those who may not know, uh, Eric Dayne uh is quite a famous and popular uh actor and father and husband. Uh, he was Dr. McTeami in Grey's Anatomy. uh and he uh while was while having been diagnosed with l uh I think it was just over 10 months ago uh excuse me um when he had found out about his diagnosis he was just wrapping up the shooting of season 3 of Euphoria um uh unfortunately

1:38:26accelerated quite quickly and so we just wanted to um you know take this opportunity to I think cover you know what is the latest in the the fight against ALS um understanding it a little bit and then we'll end with uh a funding piece that's related to this research area. Um, so you know, we know it's something that a lot of folks have been not only talking about, but have been personally impacted by because of the way in which um, Eric communicated uh, just his, you know, worldview, his what it's like to be a father, his message to his kids, the stories that he had to tell about

1:39:07life, I think really resonated with a lot of people. Um, and so with that, uh, we're going to kind of sort of look at the current landscape. >> Yes. I wanted to take this opportunity to talk about ALS, the disease, and one particular scientific paper that came out last year that is just, you know, one of the latest in a series of breakthroughs. Obviously, it's still very much a crisis in medicine. amotrophic lateral sclerosis. Okay, it's a catastrophic motor neuron degradation that happens with motor neurons specifically. These are the neurons that

1:39:50relay signals to our muscles in order to have them contract. ALS in its most advanced phase gets so bad that even the contraction of the muscles like the diaphragm >> that allow you to breathe because everything every every bit of movement in our body is from muscular contraction, right? And like even the the the act of breathing becomes something that the brain cannot sustain because these motor neurons have degraded so much. The survival is really quite bad. 2.3 to five years post diagnosis is on average. One of the most famous cases is Stephen Hawking. He

1:40:30survived for like 50 years, but it's extremely rare to do that. Um, you know, now he's no longer the scientific hero that we that he once was, but he's he's one of these like rare rare cases. >> Um, annual patient care is about 250,000 plus per patient, right? and the current incidents it's about one to two per 100 thousand. Um by 2040 there's approximately going to be a 25% increase in the global prevalence. >> Mhm. >> And if we talk about like there's there's really two types of ALS. There's familial ALS which comes from genetic

1:41:11mutations that you inherit from your family. That's only about 10% of cases. 90% of cases are sporadic ALS. There's all these polygenetic landscapes in our genome that causes sporadic ALS and that happens just there's no there's no familial history of it. >> You know, you didn't carry a mutation from your parents. It just happened. >> So, [clears throat] let's get into like what exactly is going on, okay, in our nervous system when ALS happens. So, ALS as I as I described, it's motor neurons, right? >> Yes. Motor neurons are extreme in scale. >> Okay?