Potential cancer applications
Cancer cells generally use glycolysis rather than oxidation for energy (the Warburg effect), as a result ofhypoxia that exists in tumors and damaged mitochondria. The body often kills damaged cells by apoptosis, a mechanism of self-destruction that involves mitochondria, but this mechanism fails in cancer cells. According to the Warburg hypothesis of cancer growth, cancer is caused by the metabolic changes in mitochondria, although it is now known that cancer is caused by mutations in the genome of the cells.
A study published in January 2007 by researchers at the University of Alberta, testing DCA on in vitrocancer cell lines and a rat model, found that DCA restored mitochondrial function, thus restoring apoptosis, killing cancer cells in vitro, and shrinking the tumors in the rats.
These results received extensive media attention, beginning with an article in New Scientist titled "Cheap, Safe Drug Kills Most Cancers". Subsequently, the American Cancer Society and other medical organizations have received a large volume of public interest and questions regarding DCA. Reports have since pointed out that although the study results are promising, no formal clinical trials in humans with cancer have yet been conducted, emphasizing the need for caution in interpreting the preliminary results, though some doctors are treating patients with DCA "off-label,"  and under a cloud of controversy. Medicor Cancer Centres, a private clinic in Toronto, run by the husband-and-wife team of Drs. Humaira and Akbar Khan, is since March 2007 using DCA off-label for the treatment of several cancers and said on their web site that some patients "are showing varied positive responses to DCA including tumour shrinkage, reduction in tumour markers, symptom control, and improvement in lab tests." However, they have not published their results nor reported it at medical conferences. Dr. Terry Polevoy, of Kitchener, Ontario, called on the College of Physicians and Surgeons of Ontario to take away the Khans' licences for offering a compound that hasn't been proven to shrink tumours in humans. "They are not oncologists. They should not be making these decisions. I think they should be disciplined for using this stuff. That, to me, is unethical, to use something that has never been proved to do anything." But the College said that it was not their role to say which therapies a doctor can use.
The New Scientist later editorialized, "The drug may yet live up to its promise as an anti-cancer agent - clinical trials are expected to start soon. It may even spawn an entirely new class of anti-cancer drugs. For now, however, it remains experimental, never yet properly tested in a person with cancer. People who self-administer the drug are taking a very long shot and, unlikely as it may sound, could even make their health worse."
More than 90% of drugs entering phase I trials are found unacceptable. The FDA approves 8-11% of drugs entering Phase I testing. DCA has been used historically to treat patients with lactic acidosis, and therefore could arguably enter phase 2 trials in patients with cancer.
DCA is non-patentable as a compound, though a patent has been filed for its use in cancer treatment.Research by Dr. Evangelos Michelakis has received no support from the pharmaceutical industry. Concerns have been raised that without strong intellectual property protection, the financial incentive for pharmaceutical industry interest is reduced, and therefore clinical trials of DCA may not be funded. However, other sources of funding exist; previous studies of DCA have been funded by government organizations such as theNational Institutes of Health, the Food and Drug Administration, the Canadian Institutes of Health Researchand by private charities (e.g. the Muscular Dystrophy Association). Recognizing anticipated funding challenges, Dr Michelakis's lab took the unorthodox step of directly soliciting online donations to fund the research.After 6 months, his lab had raised over $800,000, enough to fund a small Clinical Phase 2 study. Dr. Michelakis and Dr. Archer have applied for a patent on the use of DCA in the treatment of cancer.
On 24th September 2007, the Department of Medicine of Alberta University reported that after the trial funding was secured, both the Alberta local ethics committee and Health Canada approved the first DCA Clinical Trial in Cancer. This initial trial will be relatively small (enrollment- up to 50 patients in the following 18 months). The patients will originate from the Edmonton area and be available for scheduled assessments for the duration of the trial.