Comment by HWR_14
3 years ago
I would hate to ask the author this, as it seems cruel, but is the FDA denying him access? As he points out, Moderna has two drug trials going. The FDA "right to try" page[0] clearly spells out that Moderna could give him unproven medicine currently in a clinical trial under the right to try laws. So Moderna could let him into one of the two studies, or give him the medicine under the right to try laws if it rejects him.
Likely, Moderna doesn't want him to take the drugs because they will probably be ineffective this late in the cancer stage. It doesn't want its treatment to be associated with his death.
[0]https://www.fda.gov/patients/learn-about-expanded-access-and...
Actually the Moderna trials are recruiting metastatic and treatment resistant disease. From his description he has not met inclusion/exclusion criteria for the studies due to treatments received and not received rather than being too advanced in stage.
It's highly highly highly unusual to be offering an experimental therapy on compassionate grounds when conventional options that have potential to actually work well haven't been tried yet. In this post he only describes locoregional therapy and it appears he has not tried anything systemic yet.
The closest I've seen is we at least try a very short attempt of something in clinical use and then say patient refused/intolerable side-effects/treatment failure and move on. I can't imagine a circumstance where medical ethics would allow to skip a trial of validated therapy for something without even phase I data. It's not like this mRNA by Moderna is known to actually work.
Are there conventional therapies that he has not tried that could be effective? It seems he has never had chemotherapy, but he implies at this point that's a long, almost impossible, shot.
It depends what you consider the outcome to be, by virtue of being a 39 y/o with early recurrent and possibly oligometastatic disease he is in the definitively palliative-intent treatment category so everything is a long shot.
Really the best and only chance for curative intent with most cancers is radical resection with negative margins which unfortunately did not work here (most likely due to micrometastatic disease and perineurial invasion). All of these new and fancy systemic options are not believed or intended to be curative-intent.
If we're talking about meaningful prolongation of life the best treatment would be a PD-L1 inhibitor like pembrolizumab if he has expression. He doesn't mention systemic/immunotherapy in his post but on the donation page his brother does so I'm unsure if he's in the early resistance/treatment failure arm for pembro, if that's the case it's very devastating.
He's correct that gem/plat chemo isn't great but it's something. Patients in his position are best served in clinical trials (also in the treatment guidelines).
With that said it also seems he had an aggressive bone cancer in his childhood, this suggests something unique to this person (e.g. some genetic driver mutation or hereditary cancer syndrome) that may complicate any of this prognostication. Unfortunately something very atypical and aggressive is happening in this person.
H&N SCC in younger patients or HPV+ usually has good outcomes.
Given his history it's probably not relevant to this case but best advice for any HN reader is to go get your HPV vaccine and reduce the risk of SCC.
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Do our “right to try” laws mean: only after you’ve gone through our $5m treatment plans?
“The Right to Try Act is one way for patients who have been diagnosed with life-threatening diseases or conditions who have tried all approved treatment options and who are unable to participate in a clinical trial to access certain investigational treatment options.“
As an aside Keytruda is closer to 150k/year.
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I believe the oncologist has to present the case to Moderna for use. If insurance is involved, insurance will deny it.
Fairly certain that someone who has exhausted all supported treatment options doesn't care about insurance anymore.
I think the issue is that Moderna just doesn’t have much incentive to do it and it’s more a distraction than anything. But yeah it could also just be too late. My dad was recently in a similar position, but by the time he theoretically could get his hands on the drug, he was too weak too handle them.