Honest Opinions on DFMO

Hi, I’m Lindsey, mom to Maci, who was diagnosed with stage 4 HR NB MYCN amp on 8/10/19 at 21 months of age. As I write this I look at my daughter napping while getting her unituxin infusion of her last inpatient cycle. My husband and I are currently debating about DFMO. I should say upfront that we are strongly leaning towards NOT doing it, but i feel we have to make sure we cover all our bases, consult with our doctors (which we have done) and do as much research as possible before we make a final decision.

I am currently searching through old threads about dfmo and looking for any information I may have missed. Any input on your thoughts of dfmo are welcome.

I have the frustration that no study for dfmo has used a constant factor to get true results. I wish that since it started being trialed back in 2012 that there would be a reputable study done by now, 2020, and there’s not.


Hi Lindsey,

Unfortunately all of us are on the same boat here, and of course none can guarantee you that a specific treatment is going to be efficient or enough for this disease. If this can be of any help to you, I can list our own rationale on why we are leaning towards travelling to the US in order to enroll to the maintenance trial:

  • Our son (almost 3 yo at diagnosis, HR Stage 4, primary tumor adrenal gland/abdomen and metastasic disease almost everywhere literally - eye/orbits, bone marrow, skull, bones), had the worst prognosis, and still is considered a very difficult case to treat. So everything that could assist in keeping him clean is welcome.
  • DFMO doesn’t seem to have any major long-term side effects as opposed to most of the therapies he has received so far
  • He is nmyc amplified. That’s a huge factor in my opinion in favour of DFMO, as the drug itself is designed to indirectly target this gene, even though studies are showing that non-nmyc amp kids may benefit from it.
  • Most importantly, the recently published paper (May 2020) shows an obvious survival advantage for kids that received DFMO after completing the frontline therapy NED, compared to the ones who didn’t do DFMO at all. This retrospective study used patients from the same clinics; you can find it below:

As with almost every medical study of course, it has its vague points, for example there seems to be a minor disadvantage for the patients selected as a control group (“no DFMO”) in terms of response to induction chemo, plus after further breaking down the results to nmyc amp/non amp groups the population is reduced in size, so statistically significant results cannot be obtained.

However the DFMO outcomes by themselves are really imperssive in this study: for MYCN amplified population the 5 year EFS is almost 92%, which is very far from anything I have read or been told by our oncologists here.

Anyway, that’s my train of throught. Since we are in Europe, the covid situation adds an extra consideration on whether to pursuit it; if we were in the US, that would be a no-brainer for me, but I totally understand a parent who would choose not to extend their child’s therapy for 2 years. Good luck with your little girl, whatever you decide.