Phase 2 DFMO preventative trials


(Kyle Matthews) #41

The paper has been submitted, and is awaiting publication…


(Riza) #42

Do you know estimated publication time? And is the paper about preventive or maintenance treatment?


(Mark Liu) #43

Any update for DFMO?


(Sam Saffron) #44

Not sure, perhaps @KyleMatthews or @Patrick_Lacey would have an update? I have not come across anything new published in the last 12 months or so.


(Kyle Matthews) #45

The most recent numbers I’ve heard are 97% OS and 86% EFS.

Even more encouraging is the durability of these numbers (they are showing to stay true at 4 years, 5 years).

This is huge compared to the results the ch14.18 antibody (UNITUXIN/dinutuximab) has shown. They showed a big jump at 2 years in OS/EFS. Sadly, at 5 years there is no statistical difference in EFS or OS for kids that did - or did not - get antibody.


(Nick) #46

p-value was 0.03 at 5-years
74.2% vs 57.0%
for OS on ANBL0032 (antibody vs. isotretinoin)


(Nick) #47

@KyleMatthews when will the maintenance trial complete and manuscript published and peer-reviewed? If those numbers persist then people will have to take a serious look - and possibly think about bringing it in earlier somehow? If DFMO is going to start being given with ch14.18 + GM-CSF + Temo-Irino for R/R then theoretically starting it concurrently with, say, the last round of antibody (or sooner) shouldn’t prove an issue? Unless it interacts with 13-cisRA somehow? I do wonder about the argument that pre-clinical evidence is it doesn’t have efficacy at the dose used in the preventative trial. I’m no a scientist but clearly there are literally dozens of examples of things that do work in vitro and in vivo and then, because cell lines and mice are not human beings, have no effect in children. Is it completely beyond the realms of all possibility for the opposite to also be true? I don’t know.


(Mark Liu) #48

is there any official release of this statistics?


(Kyle Matthews) #49

No published paper yet, no.