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Updated: Jun 23, 2026

Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
Published on: July 25, 2020
Expanding the boundaries of clinical practice: building on experience with targeted therapies
1Department of Medical Oncology, San Camillo Forlanini Hospital, Rome, Italy. cstern@mclink.it
Background:
Advances in understanding of the mechanisms underlying cancers such as renal cell carcinoma (RCC) and gastrointestinal stromal tumour (GIST) have enabled the identification of therapeutic targets. Targeted agents have considerably improved the prognosis for patients with these cancers.
Design:
This supplement reviews approaches to the management of metastatic RCC (mRCC) and advanced GIST using targeted agents and examines clinical evidence supporting these strategies. Future developments in RCC and GIST treatment are also considered.
Results:
In a phase III trial in 750 treatment-naive mRCC patients, the multitargeted tyrosine kinase inhibitor sunitinib conferred median progression-free survival more than double that seen with interferon-alfa and median overall survival exceeding 2 years. Sunitinib is now considered the reference standard of care for first-line treatment of mRCC, and bevacizumab plus interferon-alfa is also recommended in this setting. As a result, the treatment algorithm has evolved to reflect the role of sunitinib and other targeted agents for mRCC. For advanced GIST, sunitinib has been shown to extend survival in patients with imatinib-resistant/-intolerant GIST and is licensed multinationally in this setting.
Conclusions:
Developments with targeted agents may enhance the treatment of patients with advanced RCC and GIST and offer benefits in other settings, such as adjuvant and neoadjuvant therapies.
Insights
Targeted agents like sunitinib significantly improve survival for advanced renal cell carcinoma (RCC) and gastrointestinal stromal tumour (GIST). These therapies are becoming standard care, offering new hope for patients with these difficult-to-treat cancers.
Area of Science:
- Oncology
- Pharmacology
- Clinical Medicine
Background:
- Understanding cancer mechanisms in renal cell carcinoma (RCC) and gastrointestinal stromal tumour (GIST) has identified key therapeutic targets.
- Targeted therapies have significantly improved patient outcomes for RCC and GIST.
Purpose of the Study:
- To review management strategies for metastatic RCC (mRCC) and advanced GIST using targeted agents.
- To examine clinical evidence for these targeted therapies.
- To consider future directions in RCC and GIST treatment.
Main Methods:
- Review of clinical evidence for targeted agents in mRCC and advanced GIST.
- Analysis of phase III trial data for sunitinib in treatment-naive mRCC patients.
- Examination of sunitinib efficacy in imatinib-resistant/intolerant advanced GIST.
Main Results:
- Sunitinib more than doubled progression-free survival and exceeded 2-year overall survival in treatment-naive mRCC patients compared to interferon-alfa.
- Sunitinib is now a standard of care for first-line mRCC treatment; bevacizumab plus interferon-alfa is also recommended.
- Sunitinib extends survival in advanced GIST patients resistant or intolerant to imatinib and is approved globally for this indication.
Conclusions:
- Targeted agents represent a significant advancement in treating advanced RCC and GIST.
- These developments may also benefit patients in adjuvant and neoadjuvant settings.
- Future research will likely focus on expanding the role of targeted therapies in various cancer stages.
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