amyotrophic lateral sclerosis

I am an oncology nurse.

I've also just recently had two friends diagnosed with cancer, and this week I had a friend who passed away from ALS that she lived with for 14 years.

So I deal a lot with disease and end of life, but I've never had understood people who use their faith.

You know, they feel that in order to be cured, they must have a strong faith.

And the other exciting thing is there are many new technologies and treatments that I have available today that were not available when I got started eight or 10 years ago in this field.

And then I think the key is to layer different approaches together, new technologies, because both Alzheimer's and Parkinson's and many of these other brain disorders like ALS and MS, there's more than one cause.

So thinking that you're going to find one thing that's going to fix everything, generally, that's not how it works.

I have a bunch of disclaimers.

Yeah, different one.

But yeah, she had a match with Persephone and Rebel, who's ALS has really taken her out.

She's on a motorized wheelchair.

God,

Role model.

Yeah, no, it was really good.

They did a lot of, they had a good informative video about what ALS is and how to seek treatment.

And I think that's something I have to commend Tony Khan and AEW for.

But with that knowledge, we use it in humans.

And his lab is working with people with spinal cord injuries, amputees, and even ALS patients who have lost the ability to move their hands.

So they could actually It could help them with daily living.

It could help them dress themselves or feed themselves or whatever they're doing.

I'm curious, you know, you see these examples where somebody will for the first time be able to hear or to see.

What is the reaction early on when people are having this experience?

The one subject that really impressed me the most was actually an ALS patient.

We did implant an ALS patient.

Now, I have shared Evie McDonald's story before on this podcast, but today I want to come back to her.

In 1980, Evie was 29 years old and working in healthcare when she was diagnosed with ALS and told she had less than a year to live.

Within a few months, she had lost so much strength that she was using a wheelchair, needed help with basic activities, and had begun planning the music for her funeral.

At first, Evie became consumed by what she was losing.

Evie radically changed as a person, even though at first her physical capabilities remained the same.

But over time, something remarkable happened.

Evie's ALS symptoms slowly began to reverse.

Over a period of months, her body gradually regained strength.

The AEW did the tribute for Rebel.

And my goodness, very emotional as well, especially at the end with her and Dr. Britt Baker and you know, her being involved at the beginning of the show and how happy that she was and loved it and everything going on to support ALS and the work that's being done.

And please do everything that you can to help support the, that organization.

Um, it's just, it's, it's just so tragic to see.

Genetic medicine has already begun to transform the treatment of some inherited diseases, but the brain remains one of its most formidable frontiers.

For disorders such as Huntington's disease and ALS, scientists may understand key genetic contributors and have increasingly powerful editing tools.

Yet, getting those tools safely, efficiently, and selectively into vulnerable neurons is another matter entirely.

Evox Therapeutics is developing an approach built around naturally occurring nanoparticles that cells use to communicate, known as exosomes, as delivery vehicles for CRISPR-based medicines.

We're going to talk about neurodegenerative disease, EVOX, and its efforts to develop therapies that use exosomes to deliver genetic medicines to the brain to treat

these conditions.

When you look across neurodegeneration, like Huntington disease, ALS, and other CNS conditions.

Where's the bottleneck from a technical point of view? Is it biology, trial design, or commercial factors that are the biggest constraints?

Well,

I mean, you know, the thing about it is I kind of love the fact that this doesn't start out with like, let's get the ALS characters in a big line.

That it's like, okay, let's get Firebrand.

Like, and I remember when I bought this book, I was like, I don't know who this is, but I mean, the thing is, is like those characters that aren't as familiar, Marv spends more time telling you who they are.

And of course, you know, recently we had the Rebel Heart episode, which obviously much bigger than just another dynamite.

You had Rebels fight with ALS.

Also, the emotions surrounding Andy Williams that week as well.

Right.