Current Research

Research for CUP is ongoing. The following advances may still be under investigation in clinical trials and may not be approved or available at this time. Always discuss all diagnostic and treatment options with your doctor.

Gene expression profiling for diagnosis of the primary site. All cancer cells have abnormalities in the expression of certain genes and proteins. In fact, some of these abnormalities are important in making the cell act like a cancer cell, rather than a normal cell. With improved methods of finding and evaluating these gene abnormalities, it appears that cancers starting from different sites have different patterns of gene abnormalities. For example, a cancer starting in the lung has a different profile of abnormal gene expression than does a cancer arising in the colon or elsewhere. It is likely that testing for the gene expression profile in tumor biopsies from patients with CUP will result in the identification of the primary site in some patients. Several such diagnostic tests are currently being evaluated.

For patients who cannot be classified into any identified CUP subgroups, the following types of clinical trials are being conducted in an attempt to identify more effective treatment:

New combinations of chemotherapy drugs. Many of these clinical trials are attempting to apply treatment programs already found effective in other types of cancer. Some of the studies are testing new drugs (for example, drugs not yet approved by the U.S. Food and Drug Administration [FDA]), while others are studying new combinations or new schedules of approved drugs.

Combinations of chemotherapy and targeted therapies. Many of the newer cancer treatments involve drugs targeted against newly discovered abnormalities specific to cancer cells, called targeted therapy. Several of these agents are already FDA-approved for specific cancers, usually in combination with chemotherapy. Two such drugs, bevacizumab (Avastin) and erlotinib (Tarceva), have demonstrated some activity when used together in patients with CUP. These and other new agents are being further studied in clinical trials.

Treatment with new investigational (phase I) agents. Testing of very new cancer agents (phase I clinical trials) is usually performed in patients whose cancers are resistant to standard treatment. The goals of these studies are to identify the toxicities and optimal doses of these drugs, as well as to get a preliminary idea of whether there is effectiveness against cancer.