Lievo said:
I agree with you, bobze, but for the part where you minimize the side effect of getting a cancer. To me it was not an incident. It was a hugh failure in need to be fixed.
Well again, I think its a matter of "cost-benefit". You can't control where a viral vector inserts genes, ergo it is possible it will insert genes into places that may not beneficial.
In the case of "bubble-boy", he had SCID. A disease in which you have literally no immune system--None. People with this disease, without transplants or gene therapy live to the ripe old age of "a few years old". When they underwent the first gene therapy trials, some of these children (a decade later) developed leukemias. I think for the time being, the trade-off of leukemia (something much more treatable than a total lack of an immune system) is worth the risk. At least for this disease, maybe not others.
The point was though, that this severely set back gene therapy treatments available to other people with the disease, because of an over-reaction (IMO). Given the choice between "imminent" death and dealing with leukemia (though it should be noted that only 4 or the 10 patients developed leukemia) a decade down the road (a decade of life you
wouldn't have)--I'll take my chances with the leukemia any day.
I think it is incredibly wrong to not put the choice into those families hands if given informed consent. I know as a parent, if my child had SCID--I'd again, take the chances with leukemia down the road.
I'm not saying the situation is ideal, but there are a lot of disease (and families) that gene therapy could be helping where the pay off (not imminent death) is worth the current risk, IMO. And I'm not minimizing the potential side of effects of current gene therapy--Of course, this is something we need to be putting lots and lots of research dollars into, such that we can learn to control the position of gene insertion, but the point I maintain is that the decision for current gene therapy should be in the hands of individuals and families afflicted by diseases where the option of "no therapy" is imminent death. Is the situation (at current) ideal? Certainly not, but when you have nothing else to loose--You have nothing else to loose. Know what I mean?
