Drug treatment of ameloblastoma associated with BRAF V600E mutation - datamining/contacts help requested

[WM314]

Good luck, and hopefully this is all moot.

There's a balance between what the doctors and their staff can do, and what you can do. They have expertise and experience. You have skin in the game and potentially time to spend. Hopefully you and they can find synergy in the combined efforts.

The above is particularly true, so I wanted to highlight it.

I think unfortunately a factor may be how good your insurance is -- getting buy-in from a clinician for an unusual treatment plan depends at least in part on personality, training background, and other individual factors, so having a big payor network will help (especially if academic centers like MD Anderson are available). The other bit is in getting approval from insurance for the expensive biologic infusions and the (slightly less) expensive molecular genotyping of the tumor.

I am slightly doubtful the Alabama trial will take you, as they seem focused on craniopharyngiomas and I don't think ameloblastomas are a subset of the former? Worth a shot though.

This case report seems also of use, and the latter author superficially remains at UCSF (the former is now at BMC): https://pubmed.ncbi.nlm.nih.gov/27671684/

Lastly, if you run into any research paywalls, please feel free to PM me -- I have access to quite a lot of journal resources and would be more than happy to send anything along.

 

[WM314]

So, would a mandibular ameloblastoma be considered an "advanced solid tumor"?

https://www.cancer.gov/news-events/...2/fda-dabrafenib-trametinib-braf-solid-tumors

The relevant language is, eg, in the prescribing label for darafenib (also see the FDA press release). The full language is really more along the lines of:

unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment and have no satisfactory alternative treatment options

So to stay within the bounds of FDA-approved indications, you'd need a physician to conclude that surgery is impossible or unsatisfactory (and also demonstrate the specific BRAF V600E mutation, which IIRC you're suspecting but won't have definitive genetic evidence until the biopsy?).

"Off-label" use is always possible and at the discretion of the physician, but probably runs a higher risk of insurance fights.

 

[WM314]

Previously someone had offered to get me full research papers, but unfortunately I cannot find the discussion. Admittedly, I'm pretty damn scattered right now.

This was me. Shoot me a PM with any specific papers (direct links or Pubmed is easiest, but I can track down a citation if you're having trouble.)

 

[WM314]

@mooner — U Washington oncology (if they are part of Fred Hutch, which it looks like they are) may also be able to do the same sort of stuff as MD Anderson if it turns out you are unable to get to the latter. The core thing is that you probably need to be working with an academic oncologist who is comfortable with this sort of applied biochemistry.

As with Tom, shoot me a DM if you run into a research paywall — I often can get access one way or another. I am rather busy these days so apologies if it takes a few days to get you a response.