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Patient-derived xenografts for individualized care in advanced sarcoma
Justin Stebbing1, Keren Paz, Gary K Schwartz
1Department of Oncology, Imperial College and Imperial Healthcare National Health Service Trust, Hammersmith Hospital, London, United Kingdom.
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.
Insights
Patient-derived xenografts (PDX) successfully predicted effective sarcoma treatments. This personalized approach showed a strong correlation between PDX testing and patient outcomes, offering hope for rare cancers.
Area of Science:
- Oncology
- Translational Research
- Precision Medicine
Background:
- Advanced, metastatic sarcoma presents a poor prognosis with limited therapeutic options.
- The rarity and heterogeneity of sarcoma subtypes complicate clinical trial design.
- Previous work demonstrated the utility of patient-derived xenografts (PDX) in predicting treatment response for common solid tumors.
Observation:
- Tumors from 29 sarcoma patients were implanted into immunodeficient mice (TumorGrafts).
- Drug sensitivity testing was performed on these PDX models to guide personalized treatment.
- Successful engraftment was achieved in 76% (22/29) of implanted tumors.
Findings:
- A correlation between PDX results and clinical outcomes was observed in 81% (13/16) of evaluable patients.
- No patients receiving PDX-guided therapy experienced disease progression.
- The TumorGraft model demonstrated its potential in identifying effective therapeutic regimens for individual sarcoma patients.
Implications:
- The personalized TumorGraft model shows promise as a platform for therapeutic decision-making in rare tumors like sarcoma.
- These findings support the validation of PDX models in clinical practice for guiding cancer treatment.
- A randomized phase 3 trial comparing PDX-guided therapy to physician's choice is warranted to confirm these results.
