What's going on with gene therapies?
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What's going on with gene therapies?
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Re: What's going on with gene therapies?
#2I have an extremely rare autosomal dominant subtype of a certain genetic disorder that affects maybe less than a few thousand individuals at any one time in the USA. Much common versions of the disorder that affect children have been successfully treated/cured with gene therapy. However gene treatment unlikely to ever reach people like me because so few are affected by it, it's not viable commercially. It's extremely frustrating and I struggle with tremendous feelings of anger about it, but I can't really do anything short of become a billionaire and fund my own treatment. Such is the state of medicine for profit, I guess.
Re: What's going on with gene therapies?
#3The fact that for profit medicine is holding up some of the most advanced medical treatments in history is really disturbing. As a society, there will be a significant benefit, especially once it's cost optimized. But we'll never get there if beancounters hold it up, preferring short term profits above all else.
I'd love to know if anyone knows what a solution to this problem might look like.
Re: What's going on with gene therapies?
#4I have an extremely rare autosomal dominant subtype of a certain genetic disorder that affects maybe less than a few thousand individuals at any one time in the USA. Much common versions of the disorder that affect children have been successfully treated/cured with gene therapy. However gene treatment unlikely to ever reach people like me because so few are affected by it, it's not viable commercially. It's extremely…
Wouldn't the same problem occur with any system that has to prioritize resources (that is, any realistic system)?
Re: What's going on with gene therapies?
#5I have an extremely rare autosomal dominant subtype of a certain genetic disorder that affects maybe less than a few thousand individuals at any one time in the USA. Much common versions of the disorder that affect children have been successfully treated/cured with gene therapy. However gene treatment unlikely to ever reach people like me because so few are affected by it, it's not viable commercially. It's extremely…
One of the sort of strange things about gene therapy is how cheap a custom solution really is. You can arbitrarily genetically modify a plant or bacteria for a few hundred dollars to the level of arbitrarily picking the letters of a gene sequence (or you know, doing some research and doing something real) and getting the gene modifying tools manufactured very very cheaply.
If you have a slightly different form of a much more common ailment, there probably is good hope that eventually patient specific gene therapy for you will be something of a routine. You may be taking extra risks because of the lack of available research, but I'm sure any patient can become well informed enough to make the choice for themselves. I'm not saying it's commonplace or anything yet, but the tools are there or almost there to fix a slightly incorrect single gene issue (like a single point deletion or letter swap, etc).
Like just look
https://www.idtdna.com/pages/products/crispr-genome-editing/...
obviously this is for research and not in vivo human treatment which takes quite a bit more care and specifics to target whatever tissues / organs / whatever is broken in you...
but the reagents for doing custom sequence edits are priced for research in the hundreds of dollars. sure once an established research program finished and got approved they'd spend tens or hundreds of millions and charge each patient millions
but it's not insane to imagine a biohacker curing you in their garage for $5,000 while only maybe killing you
Given that, I think there's a lot of hope that custom gene therapy will become commonplace and relatively affordable.
Re: What's going on with gene therapies?
#6I have an extremely rare autosomal dominant subtype of a certain genetic disorder that affects maybe less than a few thousand individuals at any one time in the USA. Much common versions of the disorder that affect children have been successfully treated/cured with gene therapy. However gene treatment unlikely to ever reach people like me because so few are affected by it, it's not viable commercially. It's extremely…
But in all seriousness, who would you direct your anger at in this case? For one thing, being angry in a general sense implies that a fortuitous blend of circumstances is owed to you by the world in a broad sense so that your specific, completely accidental (of nature) misfortune can be remedied. Secondly, many people suffer from much more common conditions that also have no remedy so far (i'm an example of such), and this doesn't mean that it's the fault of any system or person in particular. It's more the case that there are so many multitudes of types of suffering that could be made better in our world, and only limited, incremental resources for going after any number of them over time. Given that, it's obvious that many causes of suffering, including illnesses, will be left wanting at any given time without it necessarily being something that you could fairly fault human agency for..
I mean, do you really believe that in some realistic alternative system of organizing society, your specific, extremely rare condition would have already been addressed, or have had more of a chance of being addressed than under what exists now in the world?
Sorry for elaborating so much in this comment, but the complaint you make is one i've frequently seen and for the reasons above it has never seemed entirely logical to me.
Re: What's going on with gene therapies?
#7The fact that for profit medicine is holding up some of the most advanced medical treatments in history is really disturbing. As a society, there will be a significant benefit, especially once it's cost optimized. But we'll never get there if beancounters hold it up, preferring short term profits above all else. I'd love to know if anyone knows what a solution to this problem might look like.
Solution: public funding of research which appropriately gives intellectual property ownership to the people preventing patents and driven by reasonable boards giving away grants to researchers and practitioners to find and practice new therapies
We're kind of going in the right direction forcing open access of publicly funded research, but there's a lot more to do. And a big step backward with the insanity of the current administration which we can only hope will end soon enough.
Re: What's going on with gene therapies?
#8The fact that for profit medicine is holding up some of the most advanced medical treatments in history is really disturbing. As a society, there will be a significant benefit, especially once it's cost optimized. But we'll never get there if beancounters hold it up, preferring short term profits above all else. I'd love to know if anyone knows what a solution to this problem might look like.
I think now is a good time to see that privatizing essential services was a pretty dumb idea.
The only thing that could work is either have a natural public monopoly on the health sector (for essentials) with direct democratic control measures (otherwise the "beancounters" will corrupt it) or just pour public money on it and hope it sticks. It's pretty obvious by now that the market won't regulate itself and will not care if people die. If it's not profitable enough they won't care (and by they I mean the abstraction of individuals who would care but are on distance by capital/stakeholders or psychopaths).
Re: What's going on with gene therapies?
#9I have an extremely rare autosomal dominant subtype of a certain genetic disorder that affects maybe less than a few thousand individuals at any one time in the USA. Much common versions of the disorder that affect children have been successfully treated/cured with gene therapy. However gene treatment unlikely to ever reach people like me because so few are affected by it, it's not viable commercially. It's extremely…
But in all seriousness, who would you direct your anger at in this case? For one thing, being angry in a general sense implies that a fortuitous blend of circumstances is owed to you by the world in a broad sense so that your specific, completely accidental (of nature) misfortune can be remedied. Secondly, many people suffer from much more common conditions that also have no remedy so far (i'm an example of such), an…
I think they are angry because a solution is available but not accessible.
People are gatekeeping resources that would enable access to treatment because for big companies, spending $1 to earn $1 isn’t a sustainable business model.
Re: What's going on with gene therapies?
#10Earlier quoted context omitted.
But in all seriousness, who would you direct your anger at in this case? For one thing, being angry in a general sense implies that a fortuitous blend of circumstances is owed to you by the world in a broad sense so that your specific, completely accidental (of nature) misfortune can be remedied. Secondly, many people suffer from much more common conditions that also have no remedy so far (i'm an example of such), an…
I think they are angry because a solution is available but not accessible. People are gatekeeping resources that would enable access to treatment because for big companies, spending $1 to earn $1 isn’t a sustainable business model.
My understanding of the comment wasn't that. It was that a solution for his adult version hasn't yet been developed because it's not commercially viable to do so due to its rarity. This of course brings me right back to the points I made in my own comment above. Resources are scarce, and commercial viability, or at least resource viability for all the effort of development, is a factor under any system you choose to look at. There are just so many priorities out there and we all know this. We can't compare the world of our ideal vision of how it should be with what's the case. We need to compare what's the case with what's realistic, and with was previously the case, and how it has changed, either for better or worse.
If on the other hand, a solution for his problem does exist and is simply not available, I don't see how that makes sense. Why wouldn't it be sold if it were available?