Pre-IDE Submission: Clinical Protocol 
Neuroblastoma Bone Marrow Purging System 
BAXTER HEALTHCARE CORPORATION, HYLAND DIVISION 
GENE MARKING 
You (your child) has received all the drugs available that could possibly cure you (your child’s) 
neuroblastoma. Because the disease is no longer responding to these drugs you have agreed to 
enter a research study that involves treatment with very high doses of chemotherapy followed 
by a bone marrow transplant using your (your child’s) own bone marrow. This is necessary 
because the chemotherapy also kills normal cells in the marrow. 
Even with this treatment, the neuroblastoma may come back. We do not know why this 
happens. We would therefore like you (your child) to receive marrow that has been marked. 
If the disease returns or progresses we may be able to learn if the malignant cells came from the 
marrow we put back. 
The benefits of this marker study would be a better understanding of neuroblastoma and of 
marrow transplants. This would help us plan better ways of treating neuroblastoma. If you 
(your child) needed another transplant in the future, these changes might help you (your child). 
Otherwise, what we learn might lead to better treatment for other patients with your (your 
child’s) disease. 
To do this part of the study, we will insert two different "genetic markers" into some of your 
(your child’s) marrow cells before we return them to you (your child). Part of the marrow will 
be stored as soon as it has been marked. Another part will also be marked, but in a slightly 
different way, and will then be given treatment (purging) which may help to remove cancer 
cells. If there were neuroblastoma cells in the marrow when we stored it we may be able to 
mark the cells and if you (your child) relapsed we could tell if the neuroblastoma had come back 
from the marrow we gave you (your child). We might also be able to tell if the marrow 
"purging" helped remove the cancer. Each step of the treatment plan is given below. 
Recombinant DNA Research, Volume 16 
