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Generic drug shows promise for brain tumour treatment, study shows

She was young, only 30, and had one of the deadliest forms of cancer - an aggressive type of brain tumour that kills many patients within 15 months of their diagnosis.

Her medical team at the University of Alberta Hospital offered her dichloroacetate, or DCA, a drug that hundreds of desperate cancer patients have ordered through the Internet in hopes of self-medicating themselves into remission. In 2007, a team at the U of A discovered that the generic drug, used for years to treat rare metabolic disorders, also had a remarkable ability to shrink tumours in rats.

"Patient 5" was part of the first official experiment to see if DCA could fight cancer in humans. She took the compound in combination with conventional treatment - surgery, radiation and chemotherapy - and after 15 months her tumour had disappeared. She had no symptoms. Three others who took the drug also had their tumours shrink significantly or stop growing. In all, five patients with glioblastoma were given the drug - one man died three months into the trial.

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Those results, released Wednesday, seem likely to fuel an increase in demand for DCA, despite the pleas of researchers and other experts that patients not take it until more studies are done. The DCA phenomenon is a sign of the Internet's growing power to help patients circumvent traditional medical research on drugs, to run their own, unofficial clinical trials. This trend can lead to an increase in online scams, as well: On Tuesday, 22-year-old Hazim Gaber of Edmonton pleaded guilty in a Phoenix courtroom of selling a white powder he claimed was DCA to 65 people in the United States, Canada and several other countries. It was little more than corn starch.

One of the reasons some patients have turned to the Internet for DCA is that the drug has been around for years and can't be patented. No pharmaceutical company was willing to invest the money required to bring it to market as a cancer treatment.

The University of Alberta researchers raised more than $200,000 for their experiment, resulting in the small sample size of five patients. As another facet of the experiment, lead scientist Evangelos Michelakis and his colleagues also injected the drug into pieces of brain tumour taken from 49 patients during surgery. They found the drug worked the same way in those human tumour cells as it had in earlier experiments on cancerous animal cells.

Dr. Michelakis and his colleagues say this study is a critical step, but that more work needs to be done with much larger samples. They are urging patients not to take the drug on their own. "It is possible they will get hurt," Dr. Michelakis said. Consumers can't be sure they are getting pure DCA, he says, as illustrated in the Phoenix case. Dangerous impurities could also be in the drug, he says, as it is often sold in a highly acidic form that could cause "catastrophic" complications. It also might interact with other cancer drugs.

The Canadian Cancer Society is also advising patients to wait.

"We have not yet had the opportunity to review the findings from this study. However, as for any clinical trial, it is important to ensure the treatment is safe and effective. Until these clinical trials are finished, we can't advise cancer patients in the general population to use the agent," Gillian Bromfield, senior manager, cancer control policy, said Wednesday.

Dr. Michelakis adds that patients shouldn't go off standard therapy to go on DCA.

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The preliminary results suggest that DCA may work best in combination with other treatments, said neurosurgeon Kenneth Petruk, part of the U of A team.

The team would like to test that hypothesis, and follow the four patients, but first they need to raise more money. Meanwhile, another study is under way at the Cross Cancer Institute in Alberta to determine at what dose the drug becomes toxic and causes nerve damage. A team at the University of California is also starting a trial with money from a donor, Dr. Michelakis said.

But many people who are dying from cancer feel they can't wait for more studies.

That was the case of Jason, a Louisiana man who ordered the drug on the Internet for his father in 2007. His dad has smoked since he was 12, and had lung cancer. He had declined chemotherapy because he didn't think the suffering was worth the chance of living a few extra months.

At first the drug seemed to work, but then his father's condition deteriorated and he died. Jason, who asked that his last name not be published, said he has no regrets - he said he did what he could to save his father's life.

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About the Author

Anne McIlroy has been a journalist for more than 25 years. She joined the Globe in 1996, and has been the science reporter as well as the parliamentary bureau chief. She studied journalism at Carleton University in Ottawa. More

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