2 
3 
4 
50-70 Ambulatory, capable of self-care appropriate 
for age. Up and about >50% of time, but 
unable to carry out any physical activities or 
attend school. 
30-50 Limited self-care only. Up and about <50% 
of time. 
<30 Disabled, no self care. Bedridden or confined 
to chair. 
5.11 Patients must have transduced cell line available that is demonstrably producing 
> 150 pg IL-2/10 6 cells/24 hr. 
6.0 TREATMENT PLAN 
This study proposes to transduce a retroviral vector coupled with the human 
interleukin-2 gene into cell lines of the individual patient’s tumor established at original 
diagnosis of neuroblastoma. The transduced cells are frozen and at the time of relapse, 
thawed and allowed to recover for 3 days in culture. Supernatants are then analyzed 
for content of IL-2. Only if > 150 pg IL-2/10 6 cells/24 hr are secreted (determined by 
ELISA) will these cells be used. Relapsed or refractory patients are then treated with 
two injections of their gene-modified tumor cells, separated by one week as a pre- 
chemotherapv "window" . The second injection will contain 10 times more cells than 
the first up to a maximun of 10 8 cells/kg. After a 3-4 week rest they will be completely 
re-evaluated for evidence of toxicity and response. If there is evidence of an objective 
response without excessive toxicity and if more transduced cells are available, the 
patient may receive 2 additional SC injections separated by 1 week, at the higher of 
the two dosage levels they originally received (see Section 6.1.5). At the completion 
of immunotherapy, they will be offered entry into current Phase I or II studies with 
conventional agents. 
6.1 Cell Administration 
6.1.1 Each patient accepted for this Phase I research study will be admitted 
to the dosage schedule under investigation at the time of enrollment.(see 
Section 6.1.5) 
6.1.2 At least 3 patients will be admitted at each dosage level. 
Recombinant DNA Research, Volume 15 
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