Costly research could keep cheap cancer treatment from patients
A new treatment for cancer could cost as little as $2 a dose and be as easy to administer as taking a pill or getting a shot. But scientists fear that their struggles to find financing for further research could keep the treatment from ever reaching the public.
Last month, scientists working at the University of Alberta discovered that dichloroacetate, or DCA, a drug long used to treat rare metabolic diseases, seemed to halt the spread of cancer.
The discovery has been met with cautious optimism by the medical community and with excitement among cancer patients eager to participate in clinical trials. However, because the drug is not patented and can be produced by multiple companies, it is unlikely to be profitable and therefore has yet to appeal to the pharmaceutical companies that researchers typically rely on to finance clinical trials.
“There is no real way a company could invest and get a return on this,” said Dr. Evangelos Michelakis, whose team of scientists conducted the research.
The discovery happened almost accidentally. A research team, led by Michelakis, was seeking to combat heart disease using DCA, but during the testing the scientists realized that the same drug might help with cancer. They applied the drug to human lung, breast and brain cancer tissue, and in every case, the researchers said, the majority of the cells died. When the team later tested DCA on lung cancer in rats, the tumors began shrinking within five minutes.
Simply put, the scientists found that DCA can correct chemical imbalances affecting the metabolism of cancer cells, ultimately causing the cells to die. These imbalances have long been viewed as a side effect of cancer. However, with this discovery, Michelakis has become the first to confirm a growing theory that these imbalances could be used to cure cancer.
“And we did it with a cheap, widely used drug,” Michelakis said. The small price tag is not all that makes DCA appealing: Unlike chemotherapy and other current cancer treatments that kill both sick and healthy cells, causing side effects like nausea and hair loss, DCA seems to leave healthy cells unharmed, Michelakis said.
“The research community should rejoice that we have another pathway to tackle cancer,” said Dr. Chi Dang, vice dean for research at Johns Hopkins University School of Medicine, whose research team has been studying the treatment of chemical imbalances in cancer cells. Now, he added, “We really need to buckle down to see if these work for patients in a clinical setting.”
This is where the story becomes troubled. According to the National Cancer Institute, testing DCA’s effectiveness will require holding clinical trials testing each type of cancer with thousands of patients. These trials could cost anywhere from $1 million to $100 million, depending on the cancer, said a spokesman for the cancer institute. These experiments could begin immediately, because the U.S. Food and Drug Administration and Health Canada granted limited approval for DCA more than 30 years ago for use in fighting rare diseases like congenital lactic acidosis, a metabolic disease that causes organ failure and death in infants.
Getting the money to do that, Michelakis has discovered, is not so easy. DCA’s discovery patent expired several years ago, making it difficult to find a pharmaceutical company willing to finance the trials. When drug companies hold a patent to a medication, they can set the price high because no other company has the right to produce it. Without this patent, any company can produce the drug, forcing the price down; Michelakis estimates that DCA would cost less than $2 a dose.
“It’s a real issue,” said the head of a pharmaceutical company that produces cancer-fighting drugs and who spoke on condition of anonymity. “It would be difficult to imagine who would take on all the expense and risk of developing the drug only to have another company mass-produce a generic version.”
There may be one solution: selling a use patent, which would prohibit other companies from marketing DCA specifically as a cancer treatment. However, Michelakis has tried to attract investment from pharmaceutical companies with the use patent to no avail.
So for now, the scientists are looking to government agencies, nonprofit organizations and individual donors to support further research.
And they are starting small: Michelakis and his team are focusing on raising a few hundred thousand dollars to test a smaller group of fewer than 100 patients. They have created a Web site in response to the bombardment of calls they have received from patients asking to participate in clinical trials. The site explains their findings and warns patients not to self-administer DCA treatments.
The site also requests donations. In its first four days, the site had more than 9,000 hits. While Michelakis is unsure how much money he has raised so far, he is confident that he will be able to start the smaller trials later this year with the support from the site and money from the Canadian government.
He hopes that as limited human trials produce positive results, philanthropists and nonprofit groups will take notice and invest in the larger scale trials required by the FDA and Health Canada.
“It’s a potentially interesting drug, and we have to be skeptical until we see it work in humans,” Michelakis said. “But it will also be interesting to see if work on a drug like this can happen without the support of pharmaceutical companies.”