Phase 2 DFMO preventative trials

(Alexey) #54

A new recent article:

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(Alexey) #55

One more update regarding DFMO - now its available in Canada as well:

(Neven Dawaf) #56

Any recent update ( numbers ) about the DFMO study on kids after their first remission?

(Sam Saffron) #57

This was just published a few days ago:

It is extremely hard to interpret the results as there are at least 3 protocols being compared here with a reasonably small sample size per protocol. Some had tandem transplants, some had single transplants, some had no transplants. Additionally, not everyone was eligible for DFMO cause frontline was refractory or sadly we lost them prior to DFMO.

For example, if an survival graph usually looks at time since diagnosis

Since this trial starts AFTER treatment, you need to correct the comparison to look at a subset and then extrapolate backwards:

Some people started this say 15 months post diagnosis and others started it 18 months after diagnosis.

In all cases people were in complete remission when they started DFMO. This means that for an accurate comparison we need to compare the subset of patients that completed full treatment by COG/SIOPEN/MSKCC comparing yes DFMO to no DFMO. Extrapolating here is very very very hard.

One graph COG/SIOPEN and MSKCC never publish is “survival rate of NB patients that completed NB treatment and achieved full remission, starting one month after treatment”. Graphs they publish tend to include all the hard cases.

From the paper:

Given that the entire cohort includes subjects who had received various standard-of-care therapies prior to
starting DFMO, it is important to review the outcomes from other groups. While numbers are small, analysis of the subject cohorts that received non-COG frontline therapies also suggests benefit for DFMO maintenance therapy. A European study reports that high risk NB patients who were treated with a similar overall therapy strategy (including anti-GD2 antibody) had 3 year EFS of 46.5% ±4.1% and OS of 86.5% ±3.9%; analysis of their survival curves to correct for a lead-in period suggests that those who were event free at the completion of antibody therapy experienced a subsequent 2-year EFS of < 60%.

This in particular was looking at: which was published in 2004 back before SIOPEN used Bu/Mel as the standard of care transplant (in fact half the people in the paper above had a no transplant). This correction for lead-in is not something that is easy to do especially given you don’t even have a clean cohort to compare to.

Also from the paper:

The median time from start of antibody to start of DFMO was 7.2 months. After statistical correction for this “run in” period, the proportion of ANBL0032 patients remaining event-free for an additional 2 years (the duration of DFMO therapy) is conservatively estimated at 75%, and EFS continues to decrease between 2 and 4 years post therapy. In contrast, the observed two-year EFS for subjects in Stratum 1 who received DFMO after ANBL0032 therapy was 86% ±4%

I am a bit confused about this 75% number cause per: EFS across everyone including the hard cases, if you erase a year or so out of there I would expect it to be much higher.

The basic argument here is that the paper is claiming that without DFMO you would expect 75% survival and with DFMO you would expect 86% ±4%. Everything depends on how good the comparison is and the cohorts are quite small and the data is quite hard to compare.

@ludwinski do you have any contacts at COG or SIOPEN? What are their thoughts on the paper?

(Neven Dawaf) #58

Although Our Oncologist thinks the same way and that these results are likely biased , I would still go for the MSKCC vaccine and then the DFMO , Its so scary to stay off treatment .
We are still midway in the treatment and my son just had his surgery but If he will be eligible at the end of his treatment , I would do everything to prevent this evil from coming back .

(Kyle Matthews) #59

Here’s an article exploring that published paper a bit more.

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(Roland King) #60

Neven, what you have decided in the end? more than a month has passed and i’m really interested in any updates if you have for us. thanks!
Kyle thanks for the article!

(Sam Saffron) split this topic #61

A post was split to a new topic: Photon vs proton radiotherapy

(Alexey) #62

Hi Kyle, thanks for the article!
I think it was asked/discussed for many times, but are there any plans to offer DFMO in Europe?..

(Kyle Matthews) #63

Nothing concrete, no. It’s something we’d like to see happen, but have no official plans for currently.

(jindrich daniel) #64

hi kyle
who is the best to contact in US for dfmo?
I have contacted dr scholler from helen devos children hospital and received already some documents.
we really think about to get there prior or post IT if our daughter stays in remission.
or have you any other usefull informations for us?
br jindrich

(Kyle Matthews) #65

Hey there, Dr Sholler and her team are the correct contact for DFMO discussions. The info at is current.

(Daniel Witt Corell) #66


We are talking about doing the DMFO trial at the moment, when my son is done with frontline in April.

Just got this from Dr. Shollers team:

International patients:

Hello Daniel-
Thank you for your inquiry. Here is some quick

  1. Trial costs- there are 6 required visits during the 2 year trial receiving medications. There are 2 answers for costs…IF Dr. Sholler will accept the scans from your home institution, it will be ~$1000 each visit. If we have to do the scans here, it will be ~$20,000 each visit.
  2. If you scan at home, your visit will be one full day in clinic. If you scan here, it will be 3 days.
  3. You will be outpatient during your visit here.
  4. The DFMO is provided by the study, and there is no cost.
  5. You will have to enroll by 60 days after your last dose of therapy (immunotherapy).

On top of that, you have travel cost etc.

This is recieved yesterday January 2019.

Just wanted to share.


(Kelly) #67

Thank you for your information, Daniel. Its a giant bill for international patient.

(Daniel Witt Corell) #68

Yes it is a massive bill for international patients.

We have just talked to our doctor about it, and he has seen the numbers from the DFMO trial, and are very curious. He would talk to some of his collegues in UK what they thought of it.

Our son will finish his immunotherapy in May, and then we will hopefully join the trial. Our doctor was open to do the scans at home as my son is going to have 8 scans doing a 2 year period anyways and therefore it should be able to align it with the trial.

Then we are “only” looking at a bill of 31.000 dollars over a 2 year period. ( hospital 6000 dollars and travel expenses for the rest )

(Alexey) #69

This is not update about preventive trial, but still DFMO:

(barbara) #70

That is very interesting for me, as my son (high risk NBL, mycn amplified, NED) has an ultra rare genetic condition called Snyder Robinson Syndrome (SRS), which is characterised by a lack of polyamines. He responded very well to the treatment and is NED since 2 years. This article strengthens my opinion that there must be a correlation between the good outcome and SRS.
Any thoughts from anyone? :wink:

(Sumera Hassan) #71

Hi Daniel,
Could you please tell me which country you are based in? You mentioned your doctor will talk to colleagues in the UK so I am curious if you are living in the UK? If so, will the scans your doctor will do over the two years be coveted by the nhs or privately funded?
Look forward to your reply. Thanks in advance.


(Daniel Witt Corell) #72

Hi Sumera

We are from Denmark.

Our Doctor has workrd with neuroblastoma in UK for two years, but are back in Denmark now.

We are waiting for answer if we can get it covered by Danish healt Care - will have an answer by the end of the week.

Our doctor has approved the trial, after talking with Dr. Sholler.

(Jelena Magic) #73

Hi all,
Just and update - had a chat with an oncology consultant at the Royal Children’s in Melbourne, Dr Martin Campbell. They are looking at bringing DFMO to Australia, he’s said it will likely be available in 4-6 months’ time at the RCH. Will keep you posted if anyone is interested in exploring options in this part of the world.