Clinical trials suitable for non MYCN amplified daughter

Treatment / Research
1

Hi,
I am just wondering if anyone here can help me with a question I have about clinical trials after frontline therapy. My daughter has stage 4 high risk neuroblastoma which had spread to her bone marrow and bones. She has completed 8 rounds of chemo and has had her surgery. She still has to go through high dose chemo, radiotherapy and immunotherapy.
I am hoping all this goes to plan and am trying to research anything we can do after frontline therapy.
Am I correct in saying that there are 2 clinical trials at present - DFMO and the vaccine in New York?
Can I ask if these trials are just suitable for kids who are MYCN amplified?
My daughter’s tumour is not MYCN amplified, but she does have 11q loss and 17q gain. I just wonder if either or these trials could help her.
Thanks for taking the time to read this.

2

Hi Claire, I’m not intimately familiar with the vaccine trial, and recommend you check in with Sloan on that.

With DFMO, MYCN doesn’t really matter - it seems to work the same either way. Where are you being treated? If you’re close to a Beat Childhood Cancer hospital (the consortium that does the DFMO trials), you wouldn’t have to travel as much.

3

Thanks for the reply Kyle.We are in the UK so there would be travelling involved with either option! That is good to know that at least one of the trials could be an option for her.

4

Hi Claire,

As far as I know, all the frontline trials that are available to MYCN stage 4 are also available to non MYCN stage 4. They make very little amendments to most protocols here unless you are on one of the genetic trials where they try to cater drugs to particular genes they find.

There is very little that changes in the treatment plan of stage 4 based on MYCN alone, in general Oncologists will get more aggressive and consider more trials if the tumor is refractory and does not respond to treatment.

The gamut of treatment that is available at MSK for high risk is a possibility, including

  • Skipping high dose chemo and going direct to radiotherapy followed by hu3F8 for immunotherapy

  • Vaccine treatment post treatment

Hope this helps and wishing you and your daughter all the best.