Experimental drug beneficial in NIH trial to treat a rare sarcoma

May 7, 2013
Experimental drug beneficial in NIH trial to treat a rare sarcoma
CT scan from 25 year-old patient with newly diagnosed metastatic ASPS; top scan is cross-section of the chest at the start of the trial and bottom scan is post-treatment, with significant tumor shrinkage.

(Medical Xpress)—Patients with advanced alveolar soft part sarcoma (ASPS), a rare cancer, achieved some control of their disease using an experimental anti-cancer drug called cediranib. The results from this largest clinical trial on ASPS 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.

ASPS accounts for less than 1 percent of soft tissue sarcomas, cancers that originate in connective and supportive tissue, and occurs mostly in people ages 15 to 35. The tumor relies on the growth of new blood vessels to obtain oxygen and nutrients and to facilitate its spread to the lungs, brain, and bones. 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 ASPS. The median survival time for patients with unresectable metastatic ASPS is 40 months, with a 5-year survival rate of 20 percent.

Cediranib is a drug that inhibits a class of proteins called vascular endothelial which regulate . The drug, developed by AstraZeneca, Wilmington, Del., has been tested in clinical trials against other cancers, including non-small cell lung cancer, , and colorectal cancer, with varying degrees of benefit.

The 43 patients with metastatic ASPS on the trial were given a 30 milligram 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 percent reduction in of target lesions, is 35 percent (15 of the 43 patients achieved a partial response). An additional 26 patients, or 60 percent, 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, M.D., NCI principal investigator for this trial. "Our ability to bring patients with ASPS 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 ASPS 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.

Based on the encouraging responses observed in this trial, the researchers have initiated a follow-up trial that is comparing the effectiveness of cediranib to another vascular endothelial growth factor receptor drug, called sunitinib, in patients with metastatic ASPS.

Explore further: Drug improves survival of colorectal cancer patients, trial results show

More information: The clinical trials identifier for this study is: NCT00942877.

Related Stories

Drug improves survival of colorectal cancer patients, trial results show

January 18, 2012
An investigational drug called regorafenib slowed the progression of tumors and lengthened the lives of patients with metastatic colorectal cancer, an international phase III clinical trial found. The findings were presented ...

Targeted therapy with pazopanib prolongs progression-free survival in advanced soft-tissue sarcoma

May 15, 2012
For patients with metastatic soft-tissue sarcoma whose disease has progressed following standard chemotherapy, treatment with pazopanib (a drug that targets the growth of new cancer-related blood vessels) nearly tripled progression-free ...

Bevacizumab significantly improves survival for patients with recurrent and metastatic cervical cancer

February 8, 2013
(Medical Xpress)—Patients with advanced, recurrent, or persistent cervical cancer that was not curable with standard treatment who received the drug bevacizumab (Avastin) lived 3.7 months longer than patients who did not ...

Study to treat deadly form of thyroid cancer shows promise, researchers say

April 19, 2013
(Medical Xpress)—A combination of therapies may prove to be a promising advance for the treatment of anaplastic thyroid cancer based on results of a phase I clinical trial, say researchers at Mayo Clinic in Florida.

Recommended for you

Zebrafish larvae could be used as 'avatars' to optimize personalized treatment of cancer

August 21, 2017
Portuguese scientists have for the first time shown that the larvae of a tiny fish could one day become the preferred model for predicting, in advance, the response of human malignant tumors to the various therapeutic drugs ...

Searching for the 'signature' causes of BRCAness in breast cancer

August 21, 2017
Breast cancer cells with defects in the DNA damage repair-genes BRCA1 and BRCA2 have a mutational signature (a pattern of base swaps—e.g., Ts for Gs, Cs for As—throughout a genome) known in cancer genomics as "Signature ...

Scientists discover vitamin C regulates stem cell function, curbs leukemia development

August 21, 2017
Not much is known about stem cell metabolism, but a new study from the Children's Medical Center Research Institute at UT Southwestern (CRI) has found that stem cells take up unusually high levels of vitamin C, which then ...

How a non-coding RNA encourages cancer growth and metastasis

August 21, 2017
A mechanism that pushes a certain gene to produce a non-coding form of RNA instead of its protein-coding alternative can promote the growth of cancer, report researchers at the Medical University of South Carolina (MUSC) ...

Spaser can detect, kill circulating tumor cells to prevent cancer metastases, study finds

August 21, 2017
A nanolaser known as the spaser can serve as a super-bright, water-soluble, biocompatible probe capable of finding metastasized cancer cells in the blood stream and then killing these cells, according to a new research study.

Comprehensive genomic analysis offers insights into causes of Wilms tumor development

August 21, 2017
A comprehensive genomic analysis of Wilms tumor - the most common kidney cancer in children - found genetic mutations involving a large number of genes that fall into two major categories. These categories involve cellular ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.