OTTAWA — British businessman Peter Sartorius has devised his own treatment regime for prostate cancer. Every day, he drinks a salty cocktail, a mixture of water and an experimental drug called DCA he orders over the Internet.
Jim, a retired handyman in Louisiana, has lung cancer, and has been told that he has only a few months left to live. He swallows homemade capsules filled with DCA that his son, Jason, puts together.
Both men started self-medicating this spring after University of Alberta researchers announced that DCA (dichloroacetate) dramatically shrank tumours in rats without damaging healthy cells. But the Edmonton team was having trouble finding money to see if DCA works in humans.
DCA already has been in use for a long time to treat rare metabolic disorders, so it can't be patented as a new drug. And without a patent even a wonder drug won't make huge profits – major pharmaceutical companies won't invest the hundreds of millions of dollars required to test it and bring it to market.
Dr. Evangelos Michelakis, the principal investigator, has urged patients to wait for valid results, warning that they could poison themselves by taking DCA ordered over the Internet. The University of Alberta so far has raised more than $200,000 toward a small, initial clinical trial.
But from Liverpool to Louisiana, people with cancer say they can't afford to wait.
They are experimenting on themselves, ordering DCA, sharing their results on a website and putting together a database in an attempt to figure out if it is working.
Their do-it-yourself approach illustrates the Internet's growing power to help patients circumvent – and perhaps undermine – traditional medical research on drugs. The Edmonton scientists have warned that by taking it on their own, patients are jeopardizing the chances of a real clinical trial ever taking place: What if anecdotal reports spread that DCA doesn't work, or makes people sicker?
“It's destroying the efforts to do this right,” Dr. Michelakis recently told the science journal Nature.
He is so opposed to patients dosing themselves that he would not be interviewed for this story, because it includes interviews with people who are taking the drug.
The testing of DCA is now proceeding along two very different paths: There is the official scientific approach, which will take years and depends on finding the necessary millions in funding.
Then there are the hundreds of cancer patients, including many in the final stages of the disease, who are experimenting on themselves and sharing their stories on the Internet.
Both camps are seeking the answer to the same question: Is DCA a miracle drug?
The holy grail?
DCA challenges one of the fundamental premises of cancer biology: that mitochondria are permanently damaged by cancer. Mitochondria are energy factories, but their other job is to order cells with damaged DNA to kill themselves.
Cancerous cells don't obey that command; they just keep reproducing, and it's this out-of-control growth that leads to tumours and other forms of the disease.
What DCA seems to do is to reactivate the mitochondria in cancer cells, causing them to commit suicide.
This is different from conventional chemotherapy, which destroys fast-growing cells and can cause serious side effects, including nausea, hair loss and damage to the immune system.
Targeted drugs – which kill only cancerous cells – offer hope of a better way to fight cancer. But the few now on the market or in development work in only one or two kinds of cancer.
Since all cancer cells have damaged mitochrondria, DCA may be effective against many forms of the disease.
“This is the holy grail of cancer therapeutics – how to kill cancer cells and spare normal ones,” Dr. Michelakis said in January.
“One of the big concerns about drugs is that they can harm people, but we already know this drug is safe. It doesn't even affect normal cells,” he said.
DCA's safety record is established from years of its use for unusual inherited metabolic disorders such as lactic acidosis, in which lactic acid builds up in the bloodstream.
But the patent issues were a huge obstacle when it came to getting financial backing. Since the DCA molecule can't be patented, a researcher can claim intellectual ownership only on how a drug is administered to fight a particular disease. “Use” patents are generally considered weaker than standard patents, and are much less valuable to pharmaceutical companies.
When the researchers couldn't interest any drug companies, the University of Alberta started fundraising. Contributions, anywhere from $5 to $1,000, have been steadily flowing in.
A university spokesperson said they are waiting approval from Health Canada to start a small clinical trial on DCA. It will cost $1.5-million, and the researchers will seek the rest of the money from various sources once they get the go-ahead. But much more money will be needed to determine if the drug really works.
No time to wait
Mr. Sartorius was diagnosed with prostate cancer 18 months ago, but doctors told him that surgery wasn't an option because his tumour had spread. Hormone treatments were helping, but he has an independent streak and wanted to take control. He started exercising more and eating better. He read about DCA in January, and in March he ordered it over the Internet.
The powder looks like table salt and mixes easily with water. His doctor doesn't know he is taking it, but his family is supportive. “I'd rather be doing this than meekly going to the doctor and saying, ‘What can we do now?' ”
By mid-April, he had developed side effects including nausea, lethargy, anxiety, leg weakness and a mild tremor in his hands. So he cut his initial dose of two grams a day in half, and then reduced it even more.
The side effects now aren't severe, he says. He and his wife continue to take long walks in the Welsh mountains, and he still enjoys time with his five-year-old granddaughter.
He is not in the least emotional about his decision to take DCA. It is as if he did a cost-benefit analysis and decided that the risk was worth the chance of potentially ridding his body of cancer. He doesn't trust the pharmaceutical industry, he says – patients have to look out for themselves.
Mr. Sartorius doesn't accept the argument that by taking DCA now he may be endangering future clinical trials: “The medical profession can't say, ‘We've been using this for years, but you can't use it for cancer.' ”
In a few weeks, he'll get the results of blood tests that will suggest whether his cancer is in retreat – or not. After that, “I'll have to make a decision,” he says.
Susan Hirasawa, a Seattle woman who has advanced breast cancer, is waiting for those results. She is part of the group setting up an unofficial DCA database. They are hoping to get a sense of whether it is working in 200 patients whom they are asking to fill out a questionnaire designed with the help of an oncologist.
They want more than anecdotes about higher energy levels, or tumours that feel smaller through the skin: They are looking for test results on whether a cancer has progressed, retreated or remained the same.
Even then, Ms. Hirasawa knows it will be impossible to say for sure if any progress is due to DCA or to other treatments.
Many of the patients most tempted by DCA are close to death and have run out of other options, which could affect the database as well.
But it's a start. “Right now, people are taking this in the dark.”
Ms. Hirasawa says her volunteer work is self-interested. She was first diagnosed in 2000, and the cancer returned in 2005. “At some point, the chemo is going to run out, and I want to have some research on this.”
The database will be part of a website – TheDCAsite.com – that was established by Jim Tassano, a California man who owns a pest-control company. Mr. Tassano wanted to help a favourite ballroom-dancing instructor, who was dying of cancer.
It has become an online community, its members united by the hope that DCA will save their lives or someone they love.
“We went for the scan results today (5/14/07),” wrote the family of a man named Richard, whose lung cancer did not respond to chemotherapy.
“We were hoping for and expecting a positive report. No such luck. The scan showed significant growth of the tumour. … The oncologist said there were no further options, and sent Richard home to die.”
The news was better from another family, whose father, Terry, has aggressive bladder cancer. He ran out of treatment options and turned to DCA. He started to feel better, gained weight and was able to exercise.
Mr. Tassano has started a second website, BuyDCA.com, where he sells the drugs that he and a chemist friend synthesize themselves, which he says is a simple process. A number of other companies are also selling it over the Internet.
Pharmacists can compound it, Mr. Tassano says, and a number of doctors are willing to prescribe it for their patients.
So far, roughly a thousand people have ordered DCA, including several hundred in Canada, Mr. Tassano says. Many are close to death, and their doctors have told them that nothing can be done; they have a right, he says, to try something that might keep them alive.
A 20-gram bottle costs $20 (U.S.) and a 100-gram bottle is $85. The dosage is calculated based on the “use” patent on DCA filed by Dr. Michelakis, which Mr. Tassano has posted on the DCA website – 10 to 100 milligrams per kilogram of body weight a day.
“The patent was the Rosetta stone,” Mr. Tassano says.
Helping cancer patients treat themselves with DCA clearly wasn't what Dr. Michelakis had in mind when he filed the patent, or when he published his results in January in the medical journal Cancer Cell.
But he may have underestimated the power of the Internet, and the appeal of the DCA story. In January, he gave interviews to reporters from all over the world, but now he doesn't want to talk to the press about DCA.
“Dr. Michelakis is a highly ethical man and feels a great deal of responsibility for his research and the health of patients,” University of Alberta spokeswoman Jo-anne Nugent said in an e-mail.
“We have found that every time we participate in such publicity, it plants a seed of hope and results in more patients taking DCA.”
Stumbling blocks
If it seems hard to believe that a promising and inexpensive treatment for cancer would never get tested, listen to Judes Poirier, director for the Centre for Studies in Aging at McGill University.
“I have been there before,” he says. “This is a recurring story. It happens in many different fields.”
Researchers are increasingly going back to look at older drugs to see if they might be good at treating more than one disease, he says.
In 1996, Dr. Poirier found that a heart-disease drug called Probucol showed promise in keeping Alzheimer's disease at bay. It worked in lab animals, and a small human trial conducted in the late 1990s was also encouraging.
But there is no patent for Probucol – like DCA, it has been around too long. Dr. Poirier filed a use patent and naively hoped that Probucol would be tested soon in a large clinical trial because it already had been proved safe in heart patients.
He couldn't interest any pharmaceutical companies. They wanted him to change the drug's chemical formula just enough to get a new patent, but that would mean time-consuming tests to prove that the new version was safe.
He turned to a generic drug company, one that sells cheaper versions of name-brand drugs once their patents have expired.
Too risky, he was told. Their business approach didn't involve trying an old drug against a new disease.
In the case of DCA, however, the Canadian Institutes of Health Research might be able to help, says Phil Branton.
The CIHR is the federal agency that funds medical research in Canada, and Dr. Branton is scientific director of its Institute of Cancer Research. “If there are stumbling blocks, I'm going to help it along,” he says.
Determining if a drug is safe and works in humans can cost $20-million to $50-million. Additional studies are required to compare it to other treatments, and determine the correct dosage.
The total price tag, according to the pharmaceutical industry, is $1-billion.
Dr. Branton worries about the divide between the hopes of patients and the reality of getting a cancer drug to market.
“I know [Dr. Michelakis] believes he has something here that he believes is going to be of enormous use for treating cancer. But there is an enormous amount of work left to do.”
Many potential cancer drugs show promise in animals, but most never make it out of the lab. They either don't work in humans, or prove to be too dangerous.
So Dr. Branton is concerned about all the people taking DCA on their own.
“It's pretty scary,” he says.
‘Catastrophic' risks
What could go wrong?
Dr. Michelakis and his colleagues have warned that DCA ordered over the Internet may contain dangerous impurities. It is often sold in a highly acidic form that could cause “catastrophic” complications, they explain on the university's own DCA website, www.depmed.ualberta.ca/dca.
It might interact with other anti-cancer drugs, he has said, and may cause severe nerve damage. People might lose their ability to walk, or speak.
The Canadian Cancer Society is urging people not to take it.
So far, a number of users have said they had to stop taking the drug because they couldn't tolerate its side effects, which include reversible nerve damage in the arms and legs, nausea and drowsiness.
Jim, the 62-year-old Louisiana man with advanced lung cancer, hasn't been feeling any serious side effects. But his son, Jason, 35, was nervous when he first ordered it for him.
(They spoke on condition that their last names not be used.)
Jason waited until his father declined chemotherapy before bringing up DCA as an option. His father has seen a number of friends go through chemo and didn't think the suffering was worth the chance of living a few extra months.
Jason told his dad, who has smoked since he was 12 years old, not to get his hopes up.
“I started out by saying, ‘Hey, look, this isn't going to work. There is no chance it is going to work. I don't want you to believe that.' ”
His father says he was eager to give it a shot.
The first few days he took the drug, in mid-March, he felt pain near his collar bone and in his chest, at the sites of his three tumours.
But then he started feeling great, and was able to cut his grass regularly and pressure-wash his house.
“I'm feeling good. I've got 90 days left to live – I'm thinking I should be feeling something,” he said last month.
But by this week, he was feeling much worse. He had no appetite, and was having difficulty breathing. He spends most of the day resting and watching television.
“I can't tell if it is working,” he said. “I was real optimistic at first. But I'm a little doubtful.”
Both father and son have considered Dr. Michelakis's argument that patients taking DCA now may be undermining chances for a clinical trial.
But Jim says, “If I wait and I die, and they find out down the road that it is a cure, or it will put these tumours into remission, what do I gain by it?”
Jason says he respects Dr. Michelakis's opinion, but wants to do everything he can to save his father.
“I in no way want to endanger any possible good that might come for the masses out of this.
“But I only have one dad.”
Anne McIlroy is The Globe and Mail's science reporter.
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