AMXT 1501 plus DFMO

[davidfinn]

https://www.ccia.org.au/blog/researc...ildhood-cancer

I am new to watching this kind of announcements, is this something to be excited about, or just hopeful until further testings is accomplished.

[musella]

I helped fund that study. It is exciting but way too early and I am not happy how they announce findings like this.. it builds up hopes. Many times in the past similiar things happen - something works great in the lab and doesn't work when tried in humans.. so it is something to watch, and we need to try to speed up the pace of the research

[davidfinn]

Glad to hear you helped in the research. Great job.

For someone just getting up to speed watching research, what is the next step after a finding like this?

[musella]

The next step is human trials. Ideally a phase 0 trial where they give the drugs before surgery then they take a sample of the tumor and see how much drug got in.. this lets you know if it can get to the tumor.. then once they know that, a phase 1 trial to figure out the best dosages and make sure it is safe, then a phase 2 trial to show how good it works. If it is really good they may be able to get fda approval. if good but not good enough then goes on to phase 3 trial then approval. (Or failure)

[davidfinn]

How hard is it to get Stage 0 approval? Does the FDA need to approve that or some other organization?

Sorry for the basic questions, I am new to all of this.

[musella]

The FDA has to approve it and the hospitals' Internet Review Board has to approve it. It is pretty hard to get approved. You have to prove to the FDA and the IRB that the drug is safe, and show the preclinical work, which could by itself coust a few million dollars and take a year or so.