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What's going on with gene therapies?

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Re: What's going on with gene therapies?

#41

Post by JohnMakin »

anon84873628 wrote: post #40
southernplaces7 wrote: post #6

Earlier 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 find this comment to be downright insulting to the parent. You manage to: 1) Call them entitled for feeling angry 2) Remind them that other people have it worse 3) Imply they're stupid for imaging things could work any other way And then try to defend your obviously immature, unproductive, and unsolicited criticism in the "I'm just being logical!" excuse. The parent didn't go on a screed and rage at the world. They…

It is, which is why I'm not really going to engage with it directly. honestly, this attitude is so insanely common and I struggle to understand it personally so I dont really engage with it much. In real life people often struggle to understand, like, if you look at me I "look" fine, yet, I am mobility limited and have to use a cane a lot. They'll be casually dismissive about it like "oh, there's gene therapies now, someday they will cure you." Like, no, and I still have to live with it until and if that day comes (it probably will not). Saying things like that diminishes the very real struggles I deal with for pretty much no reason at all and is really callous, even though people are often unaware of how it sounds.

I'm also being purposely vague here to protect my privacy but I think most people if they had a condition that (and this is all true):

- causes severe pain and limits mobility to the use of a wheelchair over a period of time. No ability to exercise or have sex after it has progressed enough;

- Limits your ability to procreate because of the above bullet point but also because of the high risk of passing it off to offspring

- A cure exists but society hasn't deemed you special enough to put time into it

That.. wouldn't make you angry a little bit? I don't have to even blame anyone or anything to be allowed that. It's enormously frustrating. I'm not being entitled by being frustrated and angered by it, that idea is of course perfectly absurd. As is the idea that the anger needs to be "directed" anywhere. I guess I maybe drink a little too much and that's where it goes? honestly, who cares? And if I may borrow the parlance of the GP comment, their doesn't seem very "logical" from my POV. As is the downvoting swarms that usually accompany any kind of sentiment against the Almighty Altar of Capitalism. I'm not even making a critique of it, or offering an alternative, because I don't have one, but I don't need to provide one. It does suck, albeit for a small amount of people. But I guarantee you if you added up the sum of rare but probably treatable/curable diseases that we havent spent time on because of the rarity, the sum of those people is probably a significant chunk of the population.

Re: What's going on with gene therapies?

#42

Post by JohnMakin »

southernplaces7 wrote: post #6
JohnMakin wrote: post #2

I 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…

Besides how insulting this comment is, which I'm going to ignore, I'd just point out a common theme in this thread is "limited resources" yet those limited resources are currently being incinerated on things like AI sex bots. Seems like a bit of a fallacy or cope to me. Research for some of these things costs essentially a rounding error. The limited resource you're mentioning here is "profit," not actual resources. it's not like society can't decide to invest in these things instead of the enrichment of a very small group of people.

Re: What's going on with gene therapies?

#43

Post by thechao »

folli wrote: post #39
l0new0lf-G wrote: post #27

Could it be just that gene therapies should be publicly funded? Taxpayer money has paid for a substantial percentage of research around the world, and it doesn't seem necessary that we rely on profit-driven private companies for the actual therapies when they could be done at publicly funded hospitals too.

This argument is not limited to gene therapies, but would apply to pretty much every pharmaceutical product. One issue is however that the actual costs are not so much in early R&D (what the publicly funded universities and hospitals are doing), but in the later stage (clinical trials) which needs deep pockets and appetite for risk, which only big pharma has, because they see a potential big payout.

Right. Because your average citizen has a few hundred thousand dollars for their rare congenital disease. My bet — just wild eyed speculation — is that most of this stuff is paid for by the govt through sone sort of insurance-like subsidy. That means the risk is really borne by the tax payer.

Let's just cut out the middle man.

Re: What's going on with gene therapies?

#44

Post by edge17 »

bashmelek wrote: post #28
condiment wrote: post #11

To summarize, the problem with gene therapy is simply that it's extremely expensive and the list of qualified clients is extremely small. That's because these therapies have been necessarily targeted towards ultra-rare and assuredly fatal diseases. That approach developed the technologies, but what if a mass market approach is what is required to create the economies of scale we need to actually benefit from this sci…

I have been curious if it would be possible to create a business around pet-genetics as a means to improve the underlying technology without having to be overly concerned about the human factor…granted there will still be ethical restraints of a sort

A lot of the technology is regulated by FDA requirements, which apply to humans but not animals. Some of these requirements are challenging engineering problems - like how to do certain type of liquid transfers without exposing the contents of a container to the atmosphere, etc. There are lots of tools for doing liquid transfers that mimic what a human would do, but FDA places a different bar when it comes to developing a controlled manufacturing process.

There's also the question of, who writes the rules. Often it's industry experts that are already working on the process, and have incentives like keeping others out or licensing their own IP.

Re: What's going on with gene therapies?

#45

Post by southernplaces7 »

anon84873628 wrote: post #40
southernplaces7 wrote: post #6

Earlier 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 find this comment to be downright insulting to the parent. You manage to: 1) Call them entitled for feeling angry 2) Remind them that other people have it worse 3) Imply they're stupid for imaging things could work any other way And then try to defend your obviously immature, unproductive, and unsolicited criticism in the "I'm just being logical!" excuse. The parent didn't go on a screed and rage at the world. They…

I see nothing insulting in my comment, unless you think that just so much as questioning any of the premises of an emotional response in someone suffering is automatically an insult. In that case, you're just wrong and irrationally so.

Being sick doesn't make a person free of possibly feeling entitled or coming to unreasonable conclusions, nor does it make it wrong to question their feelings. I did so fairly politely. In my comment above, I was honestly asking for them to clarify who they think is worth being angry at, and why. I also explained reasonable reasons for why it's misdirected to claim as unfair a very general thing that's removed from you specifically only because it hasn't yet gotten around to creating relief for a problem you have, in a complex world full of problems that can't all be solved all at once or in just the way any one person desires.

This is a reality that all of us live with, so how the hell is it insulting if someone points it out?

>The parent didn't go on a screed and rage at the world. They didn't try to claim their anger is righteous. And they certainly didn't ask you to play two bit therapist

No, but they commented about their problem in a comment thread where people comment back and debate randomly. I don't give shit one about being anyone's therapist, but if you mention something personal, in a comment thread of all things, expect the possibility of someone responding with questions or opinion, like you yourself are doing. Should I feel insulted too?

I too have a long-term, very tedious and problematic health condition I need to deal with day by day, and it has no real medical remedy yet. I try to temper my frustration though. I understand that some things just aren't easy to fix.

Re: What's going on with gene therapies?

#46

Post by edge17 »

pedro_caetano wrote: post #12

From what I understand the situation is quite the opposite, there is a huge pipeline of investment both in startups as well as very large corps on the development and application of gene therapy. As anecdata, Luxturna costs north of a quarter of a million per vial, it treats a fairly rare disease (Leber congenital amaurosis). Roche still poured millions into development. Bio-tech, specially in a Medical field, is a v…

The other thing no one wants to say is the technical software/hardware talent in biotech is not as good as broader tech, and the people in charge of these projects are usually coming from a science background first and a technology background second.

Re: What's going on with gene therapies?

#47

Post by outworlder »

mandevil wrote: post #31
michaelbrave wrote: post #30

Earlier quoted context omitted.

I think a lot of our diseases if looked at genetically instead of symptoms-wise that we will probably find out that it's actually multiple conditions that we just group together for manifesting in similar ways. I've felt this in my own life with ADHD things where it seems to me that there are at least 3-4 different types of ADHD and that they respond to treatments/medications differently, and this makes me think that…

This is such a deep hole of complexity. (My wife is a pharmacist, I make computers do what I tell them to, and she has convinced me that her field is far more complex than us software people can imagine.) To pick one story my wife has told me, take the example of Multiple Sclerosis. In the 1970's and 1980's, thanks to the MRI machine, there was finally a good diagnosis tool for MS: can you see the lesions in the scan…

There are multiple brain conditions that are increasingly being suspected of being caused by viruses. They are the ultimate DNA editors. And some can remain dormant for decades before reactivating.

Which is why it was so puzzling to see the response during the last pandemic. More so with people concerned about mRNA vaccines and conflating that with 'DNA changes'. If one is concerned about their DNA, they should avoid viruses and do whatever they can to help their immune system fight them as quickly as possible.

The odds of a virus giving us something beneficial like a placenta are minimal, the drawbacks are just enormous.

Re: What's going on with gene therapies?

#48

Post by outworlder »

baggy_trough wrote: post #4
JohnMakin wrote: post #2

I 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)?

Maybe not. Because such as system, by design, would have to create a lot of common, shared infrastructure.

Once you have centralized the ability to edit genes, most of that machinery can be repurposed for less 'profitable' endeavors.

A very crude comparison would be time sharing a radiotelescope. If you want to build one to run school experiments, it's prohibitive. But once you have one, you can use any spare capacity to allow for observations that would be impossible to justify economically.

Re: What's going on with gene therapies?

#49

Post by southernplaces7 »

JohnMakin wrote: post #41
anon84873628 wrote: post #40

Earlier quoted context omitted.

I find this comment to be downright insulting to the parent. You manage to: 1) Call them entitled for feeling angry 2) Remind them that other people have it worse 3) Imply they're stupid for imaging things could work any other way And then try to defend your obviously immature, unproductive, and unsolicited criticism in the "I'm just being logical!" excuse. The parent didn't go on a screed and rage at the world. They…

It is, which is why I'm not really going to engage with it directly. honestly, this attitude is so insanely common and I struggle to understand it personally so I dont really engage with it much. In real life people often struggle to understand, like, if you look at me I "look" fine, yet, I am mobility limited and have to use a cane a lot. They'll be casually dismissive about it like "oh, there's gene therapies now,…

For what it's worth, I actually sympathize a lot with your frustration and have felt the same, but was honestly questioning if you were arguing from some sort of real claim that it's unfair for you to not have a solution now, or just from general emotional frustration.

For the first one, my arguments above, for the second one, fair enough, it happens to anyone suffering enough from something, and though I've tried to calm and redirect it when I feel that way, it's hard to pull off.

You also didn't explain, and i'm honestly curious: is this something that has no remedy for someone who's an adult, or something that has a treatment, but it's too expensive for you?

>But I guarantee you if you added up the sum of rare but probably treatable/curable diseases that we havent spent time on because of the rarity, the sum of those people is probably a significant chunk of the population.

Well yes, but there are also many much more widely devastating problems that we can also treat, but which haven't been fixed yet, and the reasons for that are way too complex to blame any one thing, or capitalism. Governments and all their immense resources are around too, yet they also don't quite seem to get around to fixing so many things that have solutions. Again, comparing the real world with your desires is never going to work. The world needs to be compared with its previous states for a fair sense of perspective.

Re: What's going on with gene therapies?

#50

Post by colechristensen »

mandevil wrote: post #26
colechristensen wrote: post #24

Earlier quoted context omitted.

If you were a grad student with RP, you could replicate the LUMEOS study drug for $400 + your time for a garage level safety self treatment. I can certainly see someone being willing to take that risk themselves for a 40% chance of improvement to reverse or prevent their own blindness. There are many guardrails for safety which are quite expensive but the actual materials are absolutely not expensive at all.

The experience of patients who received Luxterna was that the single most important difference in how large the improvement was and how many complications they had was the quality of the surgeon who delivered the actual therapy. The best surgeons saw much better results and far fewer complications, and these surgeons are way beyond the capability of some grad student in a lab. The best surgeons were way better than o…

There is a point there but those are also a couple of the most extreme examples. The eyes are delicate complicated organs very isolated from the rest of the body and changing blood characteristics requires sterilizing and replacing your bone marrow which is crazy complex and dangerous. There are many more mundane targets for gene therapy.

Also one of the benefits of cheap to produce unique gene therapies is once you figure out one treatment, similar treatments for slightly different genetic anomalies affecting the same tissues will become much easier to apply. In other words once you have a delivery mechanism down targeting something specific, the gene payload can be swapped at much lower incremental cost.

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