UCLA Sarcoma Patient-Derived Tumor Organoid Biobank (2024)

Overview

UCLA biobank of 194 sarcoma specimens from 126 patients spanning 24 distinct bone and soft-tissue subtypes (February 2018–May 2022), combined with a high-throughput patient-derived tumor organoid (PDTO) “mini-ring” drug-screening platform returning drug-sensitivity profiles within one week. RNAseq, DNA panel sequencing, and single-agent screening data deposited at Synapse (PDTOSarcoma); panel-sequencing data also shared via cBioPortal as sarcoma_ucla_2024 PMID:39305899.

Composition

Assays / panels (linked)

Papers using this cohort

  • PMID:39305899 — Al Shihabi et al. 2024: Demonstrates PDTO drug-screening platform identifies ≥1 FDA-approved or NCCN-recommended effective regimen for 59% of sarcoma specimens; organoid responses to neoadjuvant MAP tracked post-resection necrosis and PFS in 3 osteosarcoma cases; motivates the prospective PREMOST trial (NCT06064682).

Notable findings derived from this cohort

  • 80.4% (74/92) of specimens screened against ≥10 compounds had ≥1 significant response (top 5% viability score); 59% of specimens and 58% of patients had ≥1 FDA-approved or NCCN-recommended top-five regimen PMID:39305899.
  • Osteosarcoma PDTOs significantly more sensitive than pan-sarcoma average to ceralasertib (p=0.00026), topotecan (p=0.028), cabozantinib (p=0.024), and everolimus (p=0.012) PMID:39305899.
  • Chordoma PDTOs preferentially sensitive to TAK-285 (EGFR/ERBB2 kinase inhibitor, p=0.034) and significantly less sensitive to bortezomib (p=1.8×10⁻⁵) and everolimus (p=0.022) PMID:39305899.
  • PIK3CA H1047L hotspot in SARC0117 (undifferentiated spindle cell sarcoma) drove broad PI3K/mTOR pathway sensitivity (alpelisib, apitolisib, copanlisib, BGT226, vistusertib) PMID:39305899.
  • PDTO-predicted larotrectinib resistance in SARC0127 correctly distinguished high-grade spindle cell/sclerosing RMS (SCSRMS) from infantile fibrosarcoma (ETV6–NTRK3 fusion absent by FISH) within one week versus 18 days for pathology PMID:39305899.
  • Normalized organoid viability correlated with time-to-next-treatment in n=5 patients who received organoid-matched treatment (R²=0.921, p=0.009) PMID:39305899.

Sources

  • cBioPortal study sarcoma_ucla_2024. Raw data also at Synapse (PDTOSarcoma). Al Shihabi A, et al. Personalized chemo-sensitivity profiling of patient-derived sarcoma organoids for treatment guidance. Nature Cancer. 2024. PMID:39305899.

This page was processed by crosslinker on 2026-04-30.