Therapeutic Targeting of Sex Differences in Pediatric Brain Tumor Glycolysis

Inclusion Criteria:

- Diagnosis of a recurrent primary brain tumor with no curative therapy available.

- Measurable disease using pediatric Response Assessment in Neuro-Oncology Criteria
(RANO) criteria.

- Life expectancy > 12 weeks

- Prior treatment with radiation alone, chemotherapy alone or combined radiation and
chemotherapy is allowed.

- No more than 21 years of age.

- Normal bone marrow and organ function as defined below:

- Leukocytes ≥ 3,000/mcL

- Absolute neutrophil count ≥ 1,500/mcl

- Platelets ≥ 100,000/mcl

- Total bilirubin ≤ 1.5 x IULN

- AST(SGOT)/ALT(SGPT) ≤ 3.0 x IULN

- Creatinine ≤ IULN OR creatinine clearance ≥ 60 mL/min/1.73 m2 for patients with
creatinine levels above institutional normal

- Normal room air oxygenation must be documented. If room air oxygen saturation is
less than 97%, a diffusion capacity of carbon monoxide (DLCO) of greater than
80%, must be demonstrated.

- Karnofsky performance status of ≥ 60

- Patients of childbearing potential must agree to use adequate contraception (hormonal
or barrier method of birth control, abstinence) prior to study entry and for the
duration of study participation. Should a female patient become pregnant or suspect
she is pregnant while participating in this study, she must inform her treating
physician immediately.

- Ability to understand and willingness to sign an IRB approved written informed consent
document (or that of legally authorized representative, if applicable).

Exclusion Criteria:

- Contraindicated for ketogenic diet.

- Primary and secondary carnitine deficiency

- Carnitine palmitoyltransferase I or II deficiency

- Carnitine translocase deficiency

- Mitochondrial β-oxidation defects

- Pyruvate carboxylase deficiency

- Glycogen storage diseases

- Ketolysis defects

- Ketogenesis defects

- Porphyria

- Prolonged QT syndrome

- Liver insufficiency

- Renal insufficiency

- Pancreatic insufficiency

- Pulmonary insufficiency

- Hyper insulinism

- Pregnant and/or breastfeeding. Female patients of childbearing potential must have a
negative pregnancy test within 14 days of study entry.