AYA Preclinical Models

Establishment of preclinical models to develop therapies against glioblastoma in adolescent and young adult (AYA) patients


Cancer in Adolescents and Young Adults (AYA)

Ranging in age from 15-39, the adolescent and young adult cohort has 70,000 cancer diagnoses each year, six times that of children. Research shows that AYA tumors are molecularly different than those of older patients (>50 years of age), yet relatively little research has been published on AYA tumors. Click here to learn more about cancer in adolescents and young adults.

Glioblastoma in adolescents and young adults is subject to many of the same problems. GBM in AYA is approximately five times as common as GBM in children, yet there are very few preclinical models or clinical research studying this cancer, despite the fact that GBM in AYA is different from GBM in older individuals. Click here to see our research in AYA GBM.


Glioblastoma in adolescent and young adults (AYA), an age group spanning 15 to 39 years, is molecularly different than glioblastoma in older adults over 50 years of age. Therefore, to develop an effective therapy against GBM in AYA patients, we need to develop preclinical models (glioblastoma stem cells [GSCs] and patient-derived glioblastoma xenografts [PDGX]) from AYA patients for personalized drug development.

The AYA group will be subdivided into five groups (A to E) based on age.

A: 15-19 years; B: 20-24 years; C: 25-29 years; D: 30-34 years; E: 35-39 years


Recent AYA victims of GBM: Beau Biden, age 39, son of former Vice-President Joe Biden;  Brittany Maynard, age 29, an advocate of right to die; and David Pearson, age 16, son of cancer research advocate Amanda Haddock.



For each age group, we will develop preclinical models from at least ten patients to capture inter tumor heterogeneity.

Tumor samples will be collected from multiple medical centers, within a 200 mile radius of Duke University, to allow fresh tumor samples to be brought to the laboratory for preclinical model (GSC and PDGX) development and molecular analyses. Fresh tumor samples will be divided into three parts:

  1. The first part will be used for molecular analyses.
  2. The second part will be implanted sub-cutaneously in immunocompromised for PDGX development.
  3. The third part will be used to isolate GSCs.


Molecular Analyses

Primary tumor, PDGX, and GSC from each patient will be subjected to the following analyses:

  • Exome sequencing
  • DNA methylation
  • RNAseq
  • Proteomics
  • Metabolomics



Development of preclinical models and molecular analyses would cost about $40,000/patient (National Brain Tumor Society estimate)

Total project cost:  $2 million ($40,000 x 50).

Project duration: 3 years (or less depending on the availability of samples)

Funds needed to start the project: $100,000 for hiring a research assistant/program coordinator to recruit investigators at various centers, to start the IRB process, and for buying of research supplies



Ostrom QT et al. American Brain Tumor Association Adolescent and Young Adult Primary Brain and Central Nervous System Tumors Diagnosed in the United States in 2008-2012. Neuro Oncol. 2016 Jan;18 Suppl 1:i1-i50. doi: 10.1093/neuonc/nov297.