The problem with DCA
Posted: May 18th, 2010 | Author: Innovation Law Blog | Filed under: Featured, Intellectual Property, Patent | No Comments »[By Patric Senson]
You would think that a potential cure for a wide range of cancers would have pharmaceutical companies lining up for the chance to get a piece of the action. But what happens when the drug in question is already out there on the market and freely available for use?
That’s the apparent story with Dichloroacetate (DCA). A study published this week in Science Translational Medicine, appears to show that DCA can at least slow down growth, if not stop growth of cancerous brain tumours. If this small study, which tested tumours from 49 patients, can be replicated, this may be a far less invasive approach to treating brain cancer than anything currently available.
The case for DCA has been building over the last few years. In 2007, the same team of researchers from the University of Alberta published a study looking at cancer cells in a test-tube and showed that growth was slowed when DCA was added. This prompted international interest in the drug, and prompted online sales of DCA, lucrative enough to cause some to sell fake DCA.
Yet all this interest has yet to generate the capital necessary to get full scale clinical trials of DCA as used for cancer underway. According to the CBC, the current work was largely funded by private donations, and that no large pharmaceutical was interested in getting behind the work. Apparently their feeling that they would be unable to patent a product scared them off. Without the backing of drug companies the work is unlikely to progress much further. The CBC story echoes the idea that lack of patentability limits commercial interest.
This lack of patentability and the resulting lack of commercial interest seems to support the underlying premise of why we need patent law. Patents are supposed to protect an innovator’s investment. By providing them with exclusive rights to sell the subject of their patent, they have enough time to recoup their costs and make a decent profit before others can get involved. But in this situation, a potentially valuable drug may never see the light of day, all because any pharmaceutical company thinking of becoming involved in testing DCA has no guarantee it will get back its investment.
Not that there aren’t some protections designed to encourage companies to invest in new drug development even when patenting isn’t an option. Rebecca Eisenberg’s article, “The Role of the FDA in Innovation Policy” (PDF), explains that the Food and Drug Administration in the United States gives a limited protection to companies that invest in R&D for products that cannot be patented. However, as she points out in her article, this does not prevent other companies from producing the drug for their own different uses. With a situation like that found with DCA this would immediately severely restrict any benefit a company could receive from its license. Cancers come in many different forms, and each one would require its own licensing process. For instance, the current tests have shown efficacy for a particular type of brain cancer. Nothing would stop another pharmaceutical company from sweeping in and sponsoring tests for lung or liver cancers. The return from each particular license would be very limited. Compare this to the benefit a patent would give. A patent could be much broader, since the owner could license the process of how the drug actually works, which seems to be common across cancers (based on the preliminary work). This would offer a much wider protection and a greater return on investment. Unfortunately patenting seems not to be an option for this drug.
This problem of licensing would be further compounded if off-label prescribing is also considered. When a drug like DCA is widely available, there is nothing to stop doctors from prescribing off-label. In theory if enough doctors did so, over time a body of evidence would build showing that DCA is effective for treating various cancers. While this might eventually lead to an effective treatment, gathering the data would be problematic, since there would be no controls on the ‘experiments,’ and doctors may be unwilling to give up information that they have been prescribing off-label. Again, a patent protecting the use would make this off-label prescription illegal.
But does that have to be the case that DCA could never have been patented? Comparing the story of DCA to AZT, a drug that was originally developed for treating cancer, there are some possible parallels. As laid out in the facts of Apotex Inc v. Wellcome Foundation Ltd., 2002 SCC 77, [2002] 4 S.C.R. 153, the drug was synthesized decades before it was finally patented and tested for treating HIV/AIDS.
Like AZT, DCA could have potentially passed most of the tests for patentability. The use was non-obvious and novel until the original study was published. In fact, when this study was published in 2007 it created a stir for its use in a way no one had previously anticipated. It passes the utility test, since both the 2007 and the current study together demonstrate a sound prediction of future usability. Again, we need look no further than the example of AZT, which in early level trials showed potential effectiveness against HIV, in the case of DCA, the mode of action against cancer cells is clearly established.
The failure for DCA would come today in terms of novelty. Too much time has passed since the original paper for a patent to be issued now. Why was it not patented back in 2007 when the original paper was published? The widespread availability of DCA was probably a factor, as discussed above. Also, the age of the drug seemed to be discouraging investment (see New Scientist).
If DCA, though, is not patentable , or if patentable, unlikely to be protectable, thanks to off-label prescribing, this raises an important policy consideration. DCA, if it works, could save thousands, if not millions of lives (whether DCA kills cancers other than brain cancer is not yet confirmed). Yet the formation of patent laws mean it is not eligible for the kind of strict protection it needs, and hence no one is willing to put forward the money for clinical trials. Without large-scale trials the drug won’t be able to receive approval from Health Canada, or, more importantly the FDA in the United States.
Not that DCA is a sure thing. For one, it’s known to be toxic in large doses. These small scale trials do not show whether doses for treatment of cancer would be small enough to be safe for consumption. The current desperate trends towards self-medication with DCA are very worrying, given the dangers of the drug. If for no reasons other than safety, we need good clinical trials of the compound.
This is a prime example of a time when governments should be willing to step in and run clinical trials themselves. Universities and hospitals have the infrastructure to perform safety and efficacy trials, all that is missing is the cash that usually comes from the drug companies. When public health is at risk, our governments should take an active interest in these issues, although in the past they have tended to maintain an arms-length position.
In the long run, DCA is not going to the last compound we see in this category of orphan chemicals. Other compounds, particularly those from developing countries are (quite reasonably) unpatentable. But is it time for some kind of system that provides some incentive for companies to invest in the research and development needed to prove that they’re safe and effective as medicines? While certain protections exist in the United States through the FDA, they are generally based on a paradigm of drugs that have been formally covered by patents that are now coming to the end, and for which secondary uses have been found. What is missing is protection for this new type of drug- drugs that come from either easy to produce sources, or simply natural products, that need extensive testing to ensure efficacy and safety. These are hard to protect, since they are already widely distributed, but this wide distribution may itself justify the further testing. Maybe governments need to take a more active interest in paying for the testing of these products, or creating a system that more effectively compensates those who invest in their development.
And more generally, does this problem reflect a weakness in the patent system. As Eisenberg puts it, the patent system is a ‘one size fits all’ approach that was never designed with biopharmaceuticals in mind. As more questions arise about patenting of genes, drugs and medical processes, maybe it is time to reevaluate how well the system works for biological molecules at all.
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