Eric Topol
๐ค SpeakerAppearances Over Time
Podcast Appearances
phenomenal stories of your work as a molecular sleuth.
So maybe if you could give us a synopsis, and of course, we'll link to that so people could watch the whole talk.
But I think that Mucin 1 or MUC 1, as you call it, discovery is really important to kind of ground our discussion.
Yeah, and it's pretty extraordinary.
We'll put the figure from your classic cell paper, 2019, where you have a small molecule that targets the cargo receptor called Tmed9, I guess.
And what's amazing about this, of course, is the potential to reverse this toxic protein disease.
And as you say, it may have applicability well beyond
of this MUC1 kidney story, but rather eye disease with retinosis, retinosis pigmentosa and the familial Alzheimer's and who knows what else.
And what's also fascinating about this is how, as you said, you know, there were these limited number of families with the kidney disease.
And then you found another one, uromedulin, I guess.
So there's now
you know, as you say, thousands of families.
That gets me to part of your sleuth work is not just, you know, hardcore science.
You started an entity called the Ladders to Cure Accelerate.
Tell us about that, because this is really pulling together all the forces, which includes the patient advocacy groups and how we're going to move forward like this.
Yeah, well, there's many things to get to to unpack what you've just been reviewing.
So one thing for sure is that of these 8,000 monogenic diseases, they have relevance to the polygenic common diseases, of course.
Of course.
And then also the fact that the people that the patient family advocates, they are great at scurrying the world internet, finding more people, bringing together communities with each of these, as you point out aptly,