Dr. David Fajgenbaum
๐ค SpeakerAppearances Over Time
Podcast Appearances
And I recommend, you know, it's early days, but like you could try a JAK inhibitor in Kyla.
You know, we've shown the stuff in the lab.
Maybe it'll work.
And so Kyla became the first patient ever with Castleman's to be treated with a JAK inhibitor.
And she responded so well, Sean.
And she is doing so well today.
I mentioned she's...
training to become a nurse now.
And like, it's just incredible that like this drug was always there.
We actually have just opened up a clinical trial of that drug.
We're going to study it in more Castleman's patients because when you do these, they're called like N of ones where you try a drug off label in one patient and it works, that's great.
But you really want to get more data to prove that it works.
That way you can get more patients on and you can really figure out who's it going to work for.
So, so we have a trial that we just opened up for that.
How do you, I mean, how many test subjects do you need for a successful trial?
It's a great question.
So it really depends on a couple things.
The more effective the drug is, the fewer test subjects you need in the trial.
Because if it's clearly effective, like you could have a trial,
of just 20 people but if like the 10 people that got the drug all do better and survive and the 10 people don't get the drug none of them survive that's sort of you know night and day it so it's one part is how how effective is it like what's the effect size and the other one is how common is the disease because really rare diseases sometimes you're forced to like have very small numbers but if it's a common disease you know the bigger the better in terms of how confident you are in the result