human stem cell derived neurons

Clinical Benefit of Using Molecular Profiling to Determine an Individualized Treatment Plan for Patients With High Grade Glioma (PNOC008)



This pilot trial will evaluate an individualized treatment approach for children and young adults with high-grade glioma (HGG), using proteomic, transcriptomic, and genomic profiling to analyze the tumor tissue and provide a personalized therapy plan that is unique to the patient’s tumor biology.  

Patients with newly diagnosed high grade glioma (including midline HGG but excluding DIPG) who have undergone surgery as part of their standard of care and can provide the necessary tumor tissue for analysis will be eligible for this study. The tissue will be sent for detailed profiling: 

  • Whole exome sequencing
  • Whole genome sequencing
  • Targeted panel approach (UCSF 500 gene panel)
  • Quantitative proteomics, using mass spectrometry 
  • RNA sequencing

After the surgery, patients will be treated with focal irradiation per institutional guidelines. After completion of radiation therapy, patients will follow the specialized tumor board recommendation. Genetic alterations found during tumor analysis may be matched with existing targeted therapies or investigational new drug study agents that block the growth of cancer by interfering with the molecular targets that drive progression. The treatment will be based on each child’s individual tumor profile and will include up to four FDA-approved medications and in special circumstances, IND study agents. Depending on the individual treatment plan, the tumor board will also recommend required study observations to assure safety for the patient.

The objective of this pilot trial is to test the efficacy of this new treatment approach which uses detailed molecular profiling to determine an individualized treatment recommendation for each patient. 

Read news article about the trial > 


Inclusion Criteria
  1. Patients with newly diagnosed HGG (including midline HGG but excluding DIPG), who undergo tissue collection as part of standard of care. Patients with disseminated disease are not eligible, and MRI of the spine must be performed if disseminated disease is suspected by the treating physician. Primary spinal cord tumors are eligible.
  2. Enrollment within 3 weeks of the start of radiation therapy.
  3. Start of radiation therapy within 6 weeks from initial tissue diagnosis.
  4. Age ≤ 21 years
  5. Karnofsky score ≥ 50 for patients ≥ 16 years of age and Lansky score ≥ 50 for patients ≤15 years of age. 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
  6. Adequate tissue for molecular profiling (>25% tumor content).
  7. The effects of the current treatment paradigm on the developing human fetus are unknown. For this reason, females of child-bearing potential and males 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, and 30 days after completion of study drug administration. Should a female become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Males treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 30 days after completion of study drug administration.
  8. Adequate neurologic function defined as: Patients with seizure disorder may be enrolled if seizures are well controlled.
  9. Ability by patient or parent/legal guardian to understand a written informed consent document, and the willingness to sign it.

For the most up-to-date list of criteria, please visit

Exclusion Criteria
  1. Patients who are currently enrolled on another therapeutic clinical trial. Individual cases should be discussed with the study chair.
  2. Patients who are currently taking any anti-cancer directed therapy. Steroids are not considered anti-cancer therapy. The use of temozolomide during radiation therapy is allowed at standard dosing (maximum 75 to 90 mg/m^2 daily for a total of 42 days). Any other schedule(s) need to be discussed with the study chair.
  3. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
  4. Female patients of childbearing potential must not be pregnant or breast-feeding. Female patients of childbearing potential must have a negative serum or urine pregnancy test prior to the start of therapy (as clinically indicated).
  5. Patients with inability to return for follow-up visits or obtain follow-up studies required to assess toxicity to therapy.

For the most up-to-date list of criteria, please visit


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