For the millionth time– Viruses are not just pathogens that make us sick. In the modern world, viruses have been domesticated, and are now used to treat/cure diseases. Example #gazillion: Hemophilia B:
This study is far from perfect, and of course very preliminary, but still very exciting. Hemophilia B is a disease in males caused by point mutations/deletions/etc in the clotting Factor IX gene. If you dont make Factor IX, you wont clot properly, and will have all of the health issues we associate with hemophilia.
The good news is, is that today Hemophilia B is readily treatable with pharmaceutical Factor IX.
The bad news is, patients have to get infusions of Factor IX all the time. Like, several times a week.
The scientists in this paper took six Hemophilia B patients, and treated them with an Adeno-Associated Virus-8 (two at low, medium, and high doses of virus) that contained a functional copy of the Factor IX gene.
Four of them of them bumped up their Factor IX production enough to go off pharmaceutical Factor IX treatment all together (even with one of the dudes being a marathon runner!), and the two others went from needing treatments several times a week, to only needing a treatment once every couple weeks or so.
Those treatments must be IV, so we are talking about a significant improvement in quality of life here, for these guys, and thats not even factoring in the $$$. A lifetime of these treatments is expensive– while this kind of gene therapy is one shot (you literally only have one shot– you will make an antibody response to the virus delivering the functional gene) and thats it. That shot has literally got to cost millions and millions of dollars before it would be as expensive as a lifetime of Factor IX infusions.
In the United States and other developed countries, annual costs for a single adult patient of clotting factors for hemophilia are approximately $150,000 for on-demand therapy and $300,000 for prophylaxis,6 which could incur a lifetime cost of over $20 million.
Since the vector is estimated to cost $30,000 per patient, dramatic cost savings have already been achieved.
You know what I was saying yesterday about the rich having access to treatments that people in the developing world cant even dream of? While $30K is by no means ‘cheap’, it is cheaper, and will hopefully keep getting cheaper upon further refinement. Because this is what happens with Hemophilia B in the developing world:
In developing countries, prophylactic and frequent on-demand therapy is not affordable, and patients still have chronic joint disease and die young.
This treatment is not perfect yet– but its a huge step in a right direction, and only possible because of viruses.