A Compassionate Use/Expanded Access Protocol Using 131I-MIBG Therapy for Patients With Refractory Neuroblastoma and Metastatic Pheochromocytoma

Inclusion Criteria:

- Diagnosis: Relapsed/Refractory neuroblastoma, with an original diagnosis made
histologically or from elevated urine catecholamines with abnormal tumor cells in bone
marrow OR relapsed/refractory pheochromocytoma.

- Disease status: Progressive disease at any time (defined as any new lesion or an
increase in size by >25% of pre-existing disease), or a failure to respond to standard
therapy. Patients must have evidence of MIBG uptake into tumor at ≥ one site within 6
weeks prior to entry on study and subsequent to any intervening therapy

- Prior therapy: A minimum of two weeks since should have elapsed since any chemotherapy
causing myelosuppression. It must be a minimum of three months since receiving
radiation to any of the following fields: craniospinal, total abdominal, whole lung,
total body irradiation. For any other sites of radiation, at least 2 weeks should have
elapsed. For patients who received radiation to the only site of MIBG-avid disease
within two months of study entry, biopsy confirmation of residual active disease is
required, with positive bone marrow being sufficient. At least 7 days should have
elapsed since completion of therapy with a biologic agent and at least 3 half-lives
should have elapsed since therapy with a monoclonal antibody. No cytokine therapy may
be given within 24 hours of receiving 131I-MIBG. Patients may have received prior MIBG
therapy, provided they demonstrated a response or stable disease initially, with
progressive disease occurring at least 35 days following treatment.

- Organ function: ANC >500/uL, platelets >20,000/uL with transfusion allowed. Bilirubin
≤2x ULN, AST/ALT ≤10x ULN.Serum Creatinine ≤2x ULN OR 24-hour creatinine clearance OR
GFR ≥60ml/min/1.73m2. Normal lung function demonstrated by no dyspnea, exercise
intolerance or oxygen requirement. Oxygen saturation ≥94% on room air. No clinically
significant cardiac dysfunction and ejection fraction ≥45% on echocardiogram.

- Stem cells: Patients must have a minimum of 2.0 x106/kg viable CD34+ peripheral blood
stem cells for re-infusion following 131I-MIBG. An additional back-up of 2.0x106/kg
CD34+ cells is recommended but not required.

- Life expectancy longer than 8 weeks,Karnofsky or Lansky performance status of ≥ 50%

Exclusion Criteria:

- Pregnant or lactating patients

- Disease of any organ system that would compromise the patient's ability to participate
in the study, including hemodialysis. Significant organ impairment should be discussed
with the Principal Investigator prior to study entry

- Patients with active grade 3-4 infection, as defined by the NCI CTCAE V4.0.

- Patients with known MBIG-avid brain parenchymal disease (leptomeningeal or skull based
metastases are eligible).

- In patients with metastatic pheochromocytoma, a urinalysis must be preformed prior to
study enrollment. If proteinuria is present, a 24 hour urine must be collected and
total protein determined. If the 24-hour urine protein is above the institutional
upper limit of normal, the patient is excluded.