Observational Study of Responses to Treatments in Advanced Central Nervous System (CNS) Tumors

Background:

• Primary central nervous system (CNS) tumors are uncommon and are mostly classified as rare diseases. Despite their low incidence, CNS cancers are associated with significant morbidity and mortality across all age groups.
• Treatment options for CNS tumors are limited, especially upon disease progression, largely due to the incomplete understanding of disease biology and the challenges in conducting clinical studies, given the rarity of these diseases.
• The traditional pathway for oncology drug development, involving several phases of clinical trials, can take many years to a decade and comes with a tremendously high financial cost. To overcome this challenge and speed up improving oncology patient care, drug repurposing has emerged as a useful approach.
• Real-time drug screening for rare brain tumors is advancing precision medicine by generating clinical trial concepts aimed at overcoming key challenges in brain tumor management. However, delayed turnaround times remain a significant limitation to their clinical utility.
• The Gujral lab at the Fred Hutchinson Cancer Center has developed SmartMatch, a cutting-edge artificial intelligence (AI)-driven system pharmacology-based platform to address the challenges in precision medicine. The platform allows the generation of drug response data using real-time tissue-based screening using freshly resected tumor or biopsy from a patient. The data collected is used to train a machine learning model, allowing precise prediction to a large panel of Food and Drug Administration (FDA)-approved drugs. This is a test that can be ordered by any physician in the US, and results are available upon request.

Objective:

-To determine the proportion of participants with advanced CNS tumors for whom SmartMatch drug screen analysis results are generated within 21 days from the time of tumor tissue acquisition

Eligibility:

• Participants >=15 years with advanced CNS tumors.
• Participants must have been scheduled for a brain tumor biopsy or resection planned to take place at NIH.

Design:

• Tumor and blood samples will be collected for multi-omic analysis.
• Fresh tumor samples will be used for drug screen analysis by SmartMatch.
• After initial sample collection, participants will be followed remotely every 6 (+/-3) months for 3 years.

• INCLUSION CRITERIA
• Participants must have advanced CNS tumors confirmed by a documented pathology report, including:

• recurrent isocitrate dehydrogenase (IDH)-wild-type high-grade glioma
• recurrent IDH-mutant gliomas
• other recurrent CNS tumors
• Participants must have been scheduled for a brain tumor biopsy or resection. Note: Scheduled brain tumor biopsy or resection must be at least 6 months after any previous radiation therapy, if applicable. All procedures are planned to take place at NIH.
• Age >= 15 years.
• Ability of participant, parent/guardian, or Legally Authorized Representative (LAR) to understand and sign a written informed consent document.

Exclusion criteria

None.