Top US NB treatment facilities/protocols


(Aaron Basting) #1

My 2 year old daughter has stage 3 high risk Neuroblastoma and is currently being treated using the COG protocol. She’s been through 5 rounds of chemo, surgery, and has just started her final round of chemo before we are on to the stem cell transplant.

My first question is whether or not we go through with the transplant. We know MSKCC’s protocol doesn’t do the transplant and instead offers 3F8. What is everyone’s opinions on the advantages or disadvantages of this? My daughter’s tumor has responded well to chemotherapy so far. The surgeon was able to remove the entire tumor which had shrunk considerably from the chemo and all of the tumor that was removed was dead cancer cells. I wonder whether we are on the correct protocol with COG or if we should be going to MSK to follow their protocol for our daughter.

Another complication our little one has is her tumor had wrapped around and compressed her spine, paralyzing her from the chest down. Where we are currently being treated there is neurosurgery - they performed her laminectomy to decompress her spine, but there is no spinal surgeons, PMNR, or inpatient rehab for spinal injuries. So right now we are treating her cancer and her spinal injury/rehab is being pushed back. I don’t believe Memorial Sloan could manage her spinal injury either so I am curious if anyone has any experience with a Neuroblastoma treatment center that can also manage a spinal injury.

If you can’t shed some light on the above, please share your experience with where your child was or is being treated. Your insight is greatly appreciated to help make sure our daughter is getting the best treatment we are aware of for condition she is in - thank you!


(Alex Chen) #2

Dear Aaron,
Based on the Stage 3 high risk group NB, the tumor normally responds well on chemo and patient will have high chance to reach completed remission based on PETCT scan and bone marrow biopsy. However, the treatment is not stop here. Based on COG standard procedure on high risk group, Bone Marrow Transplant (BMT) and Radiotherapy (RT) are necessary to prevent the NB come back again within the first few years, this has been proven by statistics date in the past years. Also the recent CH14.18 immunotherapy can further increase the 5 year survival rate to about 60-70% (compared to the old 40-50%) if the patient is complete remission before the immunotherapy.

Hu3F8 is definitely a good news for all NB patients. However, it is still under clinical trial with limited data in the past few years. We don’t have right or wrong answer if you choose BMT + Ch14.18 or Hu3F8, any treatment for our kid is a gamble and we can only cross our finger to pray it works to our kids.

As for the Spinal injury, it’s really difficult to predict. I saw one patient also parlaying after the surgery of removing Adrenal tumor (no even close to spin). but I think the Doctor is correct, we should focus on the NB treatment first then we focus on the side effect.


(Sam Saffron) #3

Hi Aaron,

So sorry about your daughters spinal injury but very happy to hear that the tumor responded well.

As Alex says, nobody has the perfect answer here. But there are some interesting data points.

MSKCC skip transplant and treat with humanized 3f8, hu3f8 has had a very successful phase 1 trial, they found the MTD (maximum tolerated dose) to be so high that they basically stopped trying to go any higher. As a result kids being treated with hu3F8 are able to do this without any overnight stay and get much more antibody than MSKCC were previously able to give the kids with non humanized 3F8 (ch14:18 on COG is also not humanized, only st. Judes had a humanized version). They have about 3 or so years of data with hu3F8 and my understanding is that they are very happy with the results.

This hu3F8 trial is extending to a whole bunch of centers around the US, but I am not sure if it will be available to you fast enough except for MSKCC.

There is a large amount of controversy around one transplant, two transplant vs no transplant. However MSKCC have an unfair advantage in that they have a more intense immunotherapy drug and vaccine so a lot of the comparisons in literature are never able to look at a complete protocol by MSKCC.

I would recommend contacting MSK if you can and at least getting some idea about what they could offer you, they may also be able to manage spinal surgeries and since immunotherapy would happen both earlier and less intensely it is possible you could deal with that earlier under their protocol.

Anyway, I wish you all the best and lots of good luck

Sam