All the time we hear about it: the “race for a cure” or a “walk” for this or that illness. When it is explained why the event is being held, the phrase “raise awareness” is always included. Money is always raised, too; it’s never entirely clear what the money is used for. Perhaps it is used to purchase awareness from those who do not give it away.
What makes me think of this is a fairly startling scientific study, the results of which were released a couple of weeks ago in Science magazine. It carried the non-captivating title, “Metabolic Modulation of Glioblastoma with Dichloroacetate,” but its content was exciting: A substance, dichloroacetate, shows great promise in the treatment of solid tumors — yes, these are cancerous — and certain aggressive and invariably fatal brain tumors in particular.
It turns out that dichloroacetate is what is called an “orphan drug.” This is a class of pharmaceuticals that either provide cures for relatively rare diseases or that cannot be patented — DCA is among the latter.
Anyone who has used prescription drugs has probably complained about their price. The drug companies get blamed for these prices, by people who do not know why they’re so expensive. It costs a huge amount of money to bring a drug to market. Typically, the research plus the costs of jumping through regulatory hoops in order to gain approval for a single new pharmaceutical product are in the neighborhood of a half-billion dollars. When thinking of this, bear in mind that not all drugs make the grade — some are abandoned for one reason or another, many millions of dollars into the process. (There are liability issues as well. Remember Vioxx? This popular drug was highly effective in relieving the crippling pain of arthritis, but over time it emerged that it slightly increased the likelihood that users would have heart attacks or strokes. It was withdrawn. Many users, including a good friend of mine, said they would willingly take the risk because no other drug provided relief. But the choice was not theirs to make. Vioxx disappeared in a cloud of lawyers and television class-action lawsuit ads.)
When a drug is an “orphan,” pharmaceutical companies cannot hope to recoup those tremendous costs. So the product never becomes available.
Which brings us to DCA. Scientists at the University of Alberta in Canada used the substance on five — that’s not a misprint, it was only five — patients suffering from very aggressive brain tumors. Over 18 months, according to a university press release, “the team showed that DCA works in these tumors exactly as was predicted by test tube experiments… . Importantly, in some patients there was also evidence for clinical benefit, with the tumors either regressing in size or not growing further during the 18 month study.” Actually, “some” is an understatement: four of the five got better. What’s more, there were no terrible side effects in dosages that were sufficient to work on the tumors.
But a study involving only five test subjects won’t get anything approved for use. That would require a much, much bigger experiment. That kind of research is very expensive. And there’s more: if money were found to study a thousand brain tumor patients, the drug might get approved — but only for brain tumors. It might be as effective as all get out on other solid tumors, but it would not be legal to use it for treatment of them.
Because DCA is in the public domain — no one can patent it — no pharmaceutical company will undertake the research. A company could gain approval for it, but anyone then could manufacture it and the company would have to swallow the cost. No company stays in business doing things like that. DCA, by the way, is cheap to manufacture.
Which brings me back to the races, walks, and other activities that are supposed to “raise awareness.” The problem isn’t a lack of awareness. The problem is the lack of cures.
DCA strikes me as a perfect example of the kind of thing that money publicly raised ought to be financing. There are all kinds of problems in getting the government to pay for research, not the least being that the costs rise exponentially and factors other than the safety and effectiveness of the drug enter the mix. No, unless money is raised and put directly to use on studies of products such as DCA — I’m sure there are others as well — that research will simply not get done.
So when I read that a very promising drug has undergone a clinical trial on a paltry five people, it makes me wonder where all that money is going.
We have the walking and running and awareness parts taken care of. Time now for the other side of the equation: the cure.
Otherwise, all those fine activities make the good and sincere participants feel as if they’ve done something, when maybe they haven’t.
No comments:
Post a Comment