Study raises hopes among advocates of generic drug

EDMONTON – Cancer Digest – It is rare that a substance that kills cancer cells in a test tube produces the same effect in a human patient, but that appears to be the case in a tiny new study of a deadly form of brain cancer. Researchers at the University of Alberta, have reported evidence that the orphan generic drug dichloroacetate (DCA) may hold promise as potential therapy for a type of brain cancer called glioblastoma.

Led by Drs. Evangelos Michelakis and Kenn Petruk, the research team reported its finding the journal Science Translational Medicine, a journal of the American Association of the Advancement of Science.
“This work is one of the first studies in humans to support the emerging idea of altering the metabolism of tumors as a new direction of the treatment of cancer,” Michelakis said in a prepared statement.

The team has been studying DCA for several years. Over that time they had taken glioblastoma tumor tissue excised from 49 patients undergoing surgery for their cancer and exposing the tissues to DCA in a test tube. They showed that the tumors responded by changing their metabolism. They found that the drug altered the function of the mitochondria, a part of the cell that converts nutrients into energy.

"It reverses this glucose addiction that cancer cells have," Dr. John Mackey, who helped oversee the trials, told the Edmonton Journal.

For the current study, the researchers tested the drug in five patients three of whom had exhausted all other treatment options and were given an average of seven months to live. Two patients had been recently diagnosed and had been given 14 months of life expectancy.
DCA is administered orally with water. Results showed that the DCA took three months to reach blood levels high enough to alter tumor metabolism. In the 18-month study, some of the five patients’ tumors either shrank or stopped.
There were no significant adverse effects. Higher levels of the drug caused some nerve malfunction, such as numbing of the fingers and toes.
DCA has a long history of promise among patients desperate for hope of a cure for all types of cancer. According to background information on the University of Alberta web site, DCA is not owned by any pharmaceutical company and it has been difficult to secure funding for the research. As a result much of the funding for Michelakis’ team has come from private donations.

Despite the promising results, the researchers caution that these results stem from a very small study and a clinical trial involving many more patients is needed to determine if the effects shown in this study are real or coincidence. Michelakis warns that use of DCA as a cancer therapy without close clinical observation by experienced medical teams in monitored research trials is not only inappropriate, but also dangerous.

To that end, the next phase of their research will include a larger number of brain-cancer patients and patients from other academic health science centers. The researchers plan to test DCA in combination with standard chemotherapies. They also wish to expand their research to include breast- and lung-cancer patients.

SOURCE: adapted from press materials provided by the University of Alberta.