Cediranib for metastatic alveolar soft part sarcoma

Area of Science:

• Oncology
• Vascular Biology
• Medical Imaging

Background:

• Alveolar soft part sarcoma (ASPS) is a rare, highly vascular tumor lacking effective systemic treatments for unresectable cases.
• Cediranib is an oral small-molecule inhibitor targeting vascular endothelial growth factor receptors (VEGFRs).

Purpose of the Study:

• To evaluate the objective response rate (ORR) of cediranib in patients with metastatic, unresectable ASPS.
• To assess the impact of cediranib on tumor gene expression, proliferation, and angiogenesis.

Main Methods:

• A phase II trial administered cediranib (30 mg daily) in 28-day cycles to patients with metastatic ASPS.
• Objective response rate (ORR) was determined, alongside gene expression profiling and imaging studies (PET, DCE-MRI) to evaluate treatment effects.

Main Results:

• The ORR was 35% (15/43 partial responses); 60% (26/43) had stable disease, yielding an 84% disease control rate at 24 weeks.
• Gene expression analysis revealed downregulation of vasculogenesis-related genes following cediranib treatment.
• Imaging correlated with molecular changes, indicating reduced tumor proliferation and angiogenesis.

Conclusions:

• Cediranib demonstrated significant single-agent activity in metastatic ASPS, with a 35% ORR and 84% disease control rate at 24 weeks.
• These findings support cediranib as a potential treatment for unresectable ASPS.
• A randomized phase II trial comparing cediranib with sunitinib is ongoing for metastatic ASPS.