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Experimental Drug Beneficial in NIH Trial to Treat a Rare Sarcoma

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Key Points

  • In a phase II trial, patients with metastatic, unresectable alveolar soft part sarcoma who were given once-daily cediranib achieved a response rate of 35%, with 15 of 43 patients achieving partial response.
  • Researchers discovered that tumor biopsy specimens obtained after treatment had lower expression of two genes that are important in regulating blood vessel development: ANGPT2 and FLT1.

Patients with advanced alveolar soft part sarcoma achieved some control of their disease using the experimental anticancer drug cediranib. The results from this largest clinical trial on alveolar soft part sarcoma to date were published online ahead of print on April 29, 2013, in the Journal of Clinical Oncology. The phase II trial was funded by the National Cancer Institute (NCI), part of the National Institutes of Health.

Alveolar soft part sarcoma accounts for less than 1% of soft tissue sarcomas and occurs mostly in people ages 15 to 35. Surgical removal of these tumors is the recommended treatment. However, not all patients are candidates for surgical resection based on extent of disease or other conditions. Standard anticancer therapy has not been effective for treating patients with metastatic alveolar soft part sarcoma. The median survival time for patients with unresectable metastatic alveolar soft part sarcoma is 40 months, with a 5-year survival rate of 20%. 

Cediranib is a vascular endothelial growth factor receptor inhibitor that has been tested in clinical trials against other cancers, including non–small cell lung cancer, kidney cancer, and colorectal cancer, with varying degrees of benefit.

Trial Details

The 43 patients with metastatic alveolar soft part sarcoma on the trial were given a 30-mg oral dose of cediranib once daily until either their disease progressed or they developed significant side effects. To date, the objective response rate, which requires a more than 30% reduction in tumor size of target lesions, is 35% (15 of the 43 patients achieved a partial response). An additional 26 patients, or 60%, have stable disease.

“It’s unusual to see such high rates of tumor shrinkage in a cancer that traditionally has not responded to standard chemotherapy treatments used for sarcomas,” said Shivaani Kummar, MD, NCI principal investigator for this trial. “Our ability to bring patients with alveolar soft part sarcoma from across the country to the clinical facility on the NIH campus for treatment has made it possible to rapidly test this new drug in this rare tumor.”

To understand how cediranib works in alveolar soft part sarcoma and to identify abnormalities that may help in the selection of future therapies, tumor biopsy specimens were obtained before treatment and again during the first week of treatment. When the investigators compared gene expression in these two specimen groups, they discovered that the samples taken after treatment had lower expression of two genes that are important in regulating blood vessel development: ANGPT2 and FLT1.

Follow-up Trial Underway

Based on the encouraging responses observed in this trial, the researchers have initiated a follow-up trial comparing the effectiveness of cediranib to another vascular endothelial growth factor receptor drug, sunitinib (Sutent), in patients with metastatic alveolar soft part sarcoma.  

The clinical trials identifier for this study is NCT00942877.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.


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