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Blood cancer drug has "life-changing" results in study

The drug had a swift effect in most patients and showed "significant" improvement when used in one hard-to-treat form of leukemia.

By Stephen Feller

LEICESTER, England, Nov. 11 (UPI) -- Leukemia patients in a clinical trial for the drug ONO/GS-4059, which inhibits a protein important to the growth of cancer cells, experienced what researchers are calling a dramatic effect in response to the experimental treatment.

Researchers at the University of Leicester report the patients' cancer responded to the drug in nearly two-thirds of evaluable patients in the trial, with 75 percent of adverse health events associated with the treatment being minor.

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"These patients were confronted with a cruel reality: they had failed multiple chemotherapy lines and there were no other treatment options available for them," said Dr. Harriet Walter, a researcher at the University of Leicester, in a press release. "This drug has changed their lives; from desperate and tired they are now leading a normal and really active life."

The researchers started with 90 patients in January 2012 at medical centers in England and France, all of whom had one of three types of leukemia: chronic lymphocytic leukemia, or CLL; Mantle Cell Lymphoma, or MCL; and diffuse large B-cell lymphoma, or DLBCL.

CLL patients showed the best response, with 96 percent responding to the drug during a median treatment period of 80 weeks. The researchers said CLL also responded quickly to ONO/GS-4059, which 21 patients continue to receive.

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Of patients with MCL, 92 percent responded during a median treatment period of 40 weeks and 8 patients are still being treated with the drug.

Just 35 percent of patients with DLBCL responded to the drug during a median treatment period of 12 weeks, which was cut short for many patients because their cancer continued to develop. The researchers regard this response, however, as significant because of the difficulty treating DLBCL, writing in the study that the drug had "significant activity" without major drug-related toxicity.

Most also patients did not experience serious side effects during treatment with ONO/GS-4059, as researchers reported 75 percent of all health events were minor and most others were recovered from spontaneously during treatment.

Researchers said further studies will be conducted that combine the drug with others to improve the overall response of disease during treatment, in addition to continuing research into how the drug works and ways to avoid potential tumor resistance to it.

The study is published in the journal Blood.

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