Neuroendocrine Tumor Clinical Trials

• Written, informed consent obtained prior to any study-specific procedures.

• Diagnosis of neurofibromatosis, or schwannomatosis

• The results of immune staining of the patient's cancer specimens positive for any one or more of a list of tumor-associated antigens.

• Age ≥ 1 years

• At least one volumetrically measurable and ≥ 0.5 cc NF-related tumor (schwannoma, ependymoma, meningioma - histological confirmation not required) with radiographic evidence of progression (either as unequivocal progression on conventional MRI, or a \>10% volume increase by 3D volumetrics) over the past ≤12 months, designated as the primary target tumor OR Volumetrically measurable and ≥ 0.5 cc VS with ipsilateral progressive hearing loss over the past ≤12 months, designated as the primary target tumor.

• Progressive Hearing Loss Criteria for Enrollment: Audiogram showing drop in pure tone average (PTA) of 10dB HL at ≥ 2 nonconsecutive or consecutive frequencies or drop in speech discrimination score (SDS) below the 95% critical difference threshold, compared to previous audiogram ≤ 1 year prior.

• Karnofsky/Lansky performance status (PS) 50-100%. Note: Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.

• Any neurologic deficits must be stable for ≥ 1 week.

• Adequate bone marrow reserve with

‣ absolute neutrophil count (ANC) ≥ 1000/mm3.

⁃ Platelets ≥100,000/mm3.

⁃ Adequate renal and hepatic function with

∙ Serum creatinine ≤ 2 x upper limit of normal (ULN).

‣ Serum bilirubin ≤ 2 x ULN.

‣ aspartate aminotransferase (AST)/ALT ≤ 2 x ULN.

‣ Alkaline phosphatase ≤ 5 x ULN.

‣ Serum bilirubin 2.0 is acceptable in the setting of known Gilbert's syndrome.