EDMONTON – Edmonton doctors have successfully finished the first clinical trial in cancer patients for DCA, an inexpensive drug whose tumour-shrinking abilities were heralded as a long-sought miracle by patients around the world when the effects were first highlighted in rats.
Recent human tests were performed on a small sample – just five patients with brain tumours and a life expectancy of only seven months – but the results were enough to confirm the drug has the ability to stop and even reverse the growth.
At the end of the 15-month study, four of the five patients were still alive and several were cancer free.
“Keep in mind, these are people who had very big tumours,” said Dr. Evangelos Michelakis, who led the team of researchers from the University of Alberta. “Their expected survival was less than seven months.”
His studies prove the drug can have an effect on human tumours, not just animal tumours, which isn’t always the case. But beyond that, the sample size is too small to “permit any definitive conclusion.”
The results were published in today’s edition of Science Translational Medicine, a journal of the American Association of the Advancement of Science.
DCA, or dichloroacetate, affects the metabolism of the fast-growing cancer cells, the way the cells take up energy. Michelakis originally published the results of his lab tests on cancer cells in animals in January 2007, and excitement spread around the world.
That excitement turned to frustration when patients were told that, because DCA is a common chemical and can’t be patented, drug companies would not provide funding to bring the drug through the extensive clinical trials needed to make it a viable treatment option.
But in the months that followed, members of the public stepped forward with their own fundraising efforts. One girl sold homemade coasters and sent in a $75 cheque. Others launched a radio campaign in northern Alberta, and a Tim Hortons donated proceeds from coffee and doughnut sales.
All those efforts combined raised $800,000, which was topped off by the Hecht Foundation and the federal government to fund the $1.5-million trial. The University of Alberta and Alberta Health Services gave access to their equipment, which meant dozens of MRIs and PET tests.
“Without them, we wouldn’t be sitting here thinking about Step 2. We would be sitting at Step 0, which happens all the time,” said Michelakis. He hopes their success with developing a generic drug will also inspire others to believe it can be done.
Michelakis and his team started the clinical trial in September 2007. The first step was to verify the drug actually worked on human cancer cells. They took samples from 49 brain tumours removed from patients during surgery and watched the effect of DCA in test tubes. The results of that process gave them confidence to move on to treating patients.
Because brain tumours advance so aggressively, doctors were approved to start with high doses of the drug, so high many of the patients experienced numbness in their fingers and lower limbs. Doctors then reduced the dose to a level where no side-effects were found.
In two patients, their tumour regressed. One patient’s tumour stopped growing. A fourth patient underwent a second surgery to remove a residual part of the tumour, which then never regrew. The fifth patient died within the first three months.
Now, doctors believe DCA isn’t very effective during the first three months a patient starts taking it, because the drug is metabolized and broken down quickly by the liver.
That’s one of the reasons it’s important to take the drug only under supervision, Michelakis said. Each patient breaks the drug down at a different rate. Someone needs to monitor drug levels in the patient’s blood and watch the tumour grow or regress with the aid of an MRI. The symptoms of overdose from the drug can be very similar to the symptoms caused by the tumour itself.
After news broke of DCA’s potential and the lack of funding for tests, many desperate patients tried to self-medicate before the drug was approved for use. Several Internet-based companies offered the chemical for sale. An Edmonton man pleaded guilty Tuesday in a Phoenix court to selling a cornstarch-like substance online and pretending it was DCA.
A second test is ongoing at the Cross Cancer Institute for patients with other types of cancers. More long-term tests, and tests with patients in multiple hospitals in multiple jurisdictions are still needed before the drug is approved for use.
How fast that happens partially depends on continued fundraising efforts, and on interest from other doctors and researchers around the world. But it’s a unique process for a drug, because the drug companies aren’t behind it, Michelakis said. “This is the work of the people, for the people.”
Many patented cancer drugs cost between $60,000 and $80,000 a year and some don’t prolong life more than one month, Michelakis said. “That’s how bad it is in oncology.”
If approved, he said, DCA, a chemical with a very simple structure, could be produced by a facility like the University of Alberta for less than $100 a year.
More to come …
estolte@thejournal.canwest.com
Comments
Want to discuss? Please read our Commenting Policy first.