Patient-derived xenografts for individualized care in advanced sarcoma

Justin Stebbing, Keren Paz, Gary K. Schwartz, Leonard H. Wexler, Robert Maki, Raphael E. Pollock, Ronnie Morris, Richard Cohen, Arjun Shankar, Glen Blackman, Victoria Harding, David Vasquez, Jonathan Krell, Daniel Ciznadija, Amanda Katz, David Sidransky

Research output: Contribution to journalArticlepeer-review

104 Scopus citations


BACKGROUND Patients with advanced, metastatic sarcoma have a poor prognosis, and the overall benefit from the few standard-of-care therapeutics available is small. The rarity of this tumor, combined with the wide range of subtypes, leads to difficulties in conducting clinical trials. The authors previously reported the outcome of patients with a variety of common solid tumors who received treatment with drug regimens that were first tested in patient-derived xenografts using a proprietary method ("TumorGrafts"). METHODS Tumors resected from 29 patients with sarcoma were implanted into immunodeficient mice to identify drug targets and drugs for clinical use. The results of drug sensitivity testing in the TumorGrafts were used to personalize cancer treatment. RESULTS Of 29 implanted tumors, 22 (76%) successfully engrafted, permitting the identification of treatment regimens for these patients. Although 6 patients died before the completion of TumorGraft testing, a correlation between TumorGraft results and clinical outcome was observed in 13 of 16 (81%) of the remaining individuals. No patients progressed during the TumorGraft-predicted therapy. CONCLUSIONS The current data support the use of the personalized TumorGraft model as an investigational platform for therapeutic decision-making that can guide treatment for rare tumors such as sarcomas. A randomized phase 3 trial versus physician's choice is warranted. Cancer 2014;120:2006-2015.

Original languageEnglish (US)
Pages (from-to)2006-2015
Number of pages10
Issue number13
StatePublished - Jul 1 2014


  • TumorGraft
  • biomarker
  • chemotherapy
  • mice
  • prediction
  • response
  • sarcoma
  • trial

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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