What to do next? Stage 4 10year old battling for 6 years

1

I am a friend trying to help out a young girl supposedly near the end of her battle. This is what she has endured:
Busulfan/melphalan
Vincristine/topotecan
Stem cell transplant
Radiation
3f8
Humanized 3f8
MiGB therapy
Ch 14.18
Vaccine at mskcc

If there is any information you can offer, please do and thank you.

2

Hi @dasw,

I am afraid I can not help with any specific recommendations here, being treated at MSK means you are at the cutting edge of treatment. I am heartbroken to hear about your daughter. Wishing you the best.

3

Hi @dasw ,

It’s true that MSK is state of the art for this disease, however since you have already been through all of the “common” therapies, I’d recommend investigating one of the following (I also have them in the back of my head in case we get no response in our kid from the standard treatment):

  1. CAR T-Cell Therapy: this looks very promising and I thinkg it already has been used with success for leukaimia cases. There seem to be a couple of open Phase 1 studies, you could consult your doctors and contact them to see if they are still recruiting and if the child is eligible to participate:

https://www.smartpatients.com/trials/NCT03721068

  1. CBD Oil usage at home with large doses: There are no human trials, nor official results on this, but you have nothing to lose giving that a try since there are no side-effects. There has been some proof in isolated cells that CBD can act against neuroblastoma tumors:
    In vitro and in vivo efficacy of non-psychoactive cannabidiol in neuroblastoma - PubMed

Keep us posted on your progress; I also wish you the best for the little girl, please don’t give up.

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Dear @dasw

It might be worth contacting Andras Heczey about his NKT CAR trial – he has presented on first couple children treated. Rapidly progressive disease not eligible – stubborn but stable disease probably best suited – see eligibility/exclusion criteria and contact info – GD2 Specific CAR and Interleukin-15 Expressing Autologous NKT Cells to Treat Children With Neuroblastoma - Full Text View - ClinicalTrials.gov

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Thank you everyone for your help. I have forwarded this information to this young girl’s Mother. The current situation is: The disease has worsened in her legs, which is giving her pain and making it difficult to walk. She is being sent back to radiation. The Doctors at Sloan will be giving her a drug called Lutathera, which should slow the growth of the disease. Their insurance is giving them a hard time but they are staying positive.

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It appears the Lutathera is helping and will slow the growth and further spread…unfortunately chances of it stopping it completely are small. The doctors have found that the cancer had metastasized to her lungs. The time remaining is now projected to be less than they anticipated. :frowning_face:

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So sorry for this. Did the parents contact anyone about any of the available/suggested trials for relapsed/refractory disease? If lutathera is stabilizing spread then she may be eligible for some of them

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Last I heard, She was doing well on Lutathera and will be going for scans soon to see what’s going on in her body. I will try and keep this topic updated.

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Her latest scan’s on 2/14 showed improvement while on Lutathera! A few new spots were seen but may be pneumonia. She has been admitted to Sloan and the next dose of Lutathera is currently on hold.

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:broken_heart:
Angelina Rose passed away this morning. 8/12/09 - 2/21/20
We thank you for your support.

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So sorry for your loss, heartbreaking

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I’m so sorry, no words :frowning:

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I’m so so sorry. As my daughter is newly diagnosed too…