There's an article today in the Boston Globe that I wanted to bring to your attention. It is about several things--a sick kid, a costly medication, poor countries, and technology.
One girl's hope, a nation's dilemma
Here's the part about Tania, the sick kid:
....As a toddler, Tania had been a bright girl with a vivid smile and a penchant for dancing. But by age 8 she was in a strange and frightening decline. She struggled on frail limbs to carry her swollen abdomen. As other children rode their bikes on the tiny fishing village's dirt roads, Tania lay on the sofa in her orange cinderblock house, inert.
No one understood what was wrong. Specialists had run tests on Tania to rule out common diseases, then unusual ones. When Jose's phone finally rang with an answer, the doctor told him Tania had a genetic defect so rare that it strikes only a tiny scattering of people around the world....
The diagnosis was Gaucher Disease. This is a rare disease, and often research and drug development for rare diseases is unappealing from a corporate perspective, and impossible to complete from an academic perspective because academics are not drug developers and distributors.
One biotech company did take it on--Genzyme. However, the business model for this is probably shocking to hear about. They aren't cutting any deals on this drug--they charge full price to nearly everyone who needs it. That makes it wildly expensive--but usually governments are paying the costs, not individual families.
From the Globe article:
....Genzyme's solution, elegant in its way, was to set a price high enough to earn a substantial profit no matter how small its pool of patients. Then the company surprised the medical world - and its investors on Wall Street - by showing that American health insurers could be persuaded to pay the six-figure price tag. And with the only effective treatment for Gaucher disease, Genzyme never needed to lower the price, even as production efficiencies raised profit margins on the drug to as much as 90 percent....
People find this horrifying, understandably. On the other hand, without the opportunity to make back the money for research costs and ongoing production this drug may never have been developed and patients with Gaucher disease would suffer and die.
In a single-payer and public option world questions about the value of the treatment for the cost are going to arise. Poor countries are already facing this issue today as you can read in the Globe story. Personally I think we should cover the costs for rare genetic diseases. It is a complete crapshoot what your genes may bring, for good and for bad. And for anyone's child to suffer and die from a preventable cause is unacceptable to me. And I'm willing to pay for your kid's or your grandkid's treatment for this.
So there is one dilemma: who gets care and where do we use resources for the most good?
But there's actually a second dilemma: What if there was a less costly treatment? Would that be worth pursuing?
There is actually a less costly manufacturing strategy that is being developed. A company in Israel called Protalixis working on this new method. They got it running and they published a peer-reviewed paper with the results of the Phase I clinical trials (it is open access, you can go read the whole thing). As this is merely Phase I we don't yet know if it will actually work and replace the Genzyme product. Ongoing trials will have to determine that.
But here's the kicker: that new version of the drug is being developed in a genetically modified plant--that's a GMO.
The advantages of using plants for this drug are several fold: 1) Mammalian cell culture is expensive and difficult. 2) Although some drugs can be produced by E. coli (such as insulin), more highly processed drugs with other post-translational modifications are not always produced correctly in simpler systems. 3) Unwanted modifications or interactions that might occur in mammalian cells may be avoided.
There are people who want to prevent research and use of GMOs. Like other technologies I think they need to be monitored and regulated. However, for some there is no middle ground. They are working to prevent this technology from being funded, studied, and used.
Another issue arises around poor countries: is it fair to prevent them from education and research around these technologies? What if local scientists know of a need that will never be served by the Genzymes of the world. Should they be prevented from learning and accessing these technologies?
You may hear about legislation that permits (but does not mandate) funding for research around this topic, called S.384.
As I stated the other day:
You may be asked soon to call your representatives to protest this legislation--please consider the full story before you do that. And you may decide that you don't want any biotech science supported--that is your call. I would ask you to consider whether you really want that. Many of us were pleased to see science returning to it's proper place in an administration.
Not all GMOs are from Monsanto. And not all research in this area is related to them. Please consider the larger picture.