I was more excited about the technological implications until I looked up how bone marrow transplants work. It is quite frightening. From what I can gather, you have to "make space" for new...
I was more excited about the technological implications until I looked up how bone marrow transplants work. It is quite frightening.
From what I can gather, you have to "make space" for new marrow. That's a euphemism for killing your existing bone marrow with drugs and/or radiation. Then you receive the donor stem cells via blood transfusion. The process leaves you vulnerable to infection.
The improvement this offers is that there should be no complications arising from incompatibility, since you are your own donor.
This just makes me wonder how long it will take before CRISPR on human embryos becomes practical. It would be better to prevent genetic diseases from the start.
CRISPR on human embryos is practical already. It has been for a while. There are three Chinese babies born around 2018 using CRISPR to choose genetic traits, and last I heard they were alive and...
CRISPR on human embryos is practical already. It has been for a while. There are three Chinese babies born around 2018 using CRISPR to choose genetic traits, and last I heard they were alive and well. The doctor has been disgraced by the medical establishment for doing it. It is illegal in every country, as far as I am aware.
https://sl.bing.net/hE1iMM34ckC Note: the US researcher I read about is not implanting embryos. I think this is the one you referred to, where they modified the embryos to reduce the risk of HIV—a...
The legal status of using CRISPR on human embryos varies by country. In the United States, such clinical trials are effectively banned by a rule that prevents the Food and Drug Administration from reviewing applications for any procedure that would introduce heritable changes in human embryos. However, some researchers have used private funds to conduct gene editing experiments on human embryos for basic research purposes.
Note: the US researcher I read about is not implanting embryos.
In China, there are no clear policies on gene editing of human embryos, and some scientists have claimed to create the first gene-edited babies using CRISPR.
I think this is the one you referred to, where they modified the embryos to reduce the risk of HIV—a not very useful modification considering the risk involved.
This has raised ethical questions and calls for a global ban on germline editing. Other countries have different legal regimes that may allow or restrict gene editing of human embryos depending on the purpose, the stage of development, and the consent of the donors.
Seems like it's not entirely a legal question at this point, but rather an ethics and PR one.
I'll try and find a source but I remember reading at the time that a side effect of adding HIV resistance genes was an increase in intelligence as well
they modified the embryos to reduce the risk of HIV
I'll try and find a source but I remember reading at the time that a side effect of adding HIV resistance genes was an increase in intelligence as well
I'm hopeful this will lead to long term results but I sort of despair that it'll get cheap enough to be widespread enough to make larger impact. Obviously it's great to save lives but it's so...
I'm hopeful this will lead to long term results but I sort of despair that it'll get cheap enough to be widespread enough to make larger impact. Obviously it's great to save lives but it's so disheartening to see treatments out of reach for so many
Almost all the world's currently most expensive drugs are gene therapy drugs, but it's worth keeping in mind that's largely because it's still an emerging technology. So I don't think it's...
Almost all the world's currently most expensive drugs are gene therapy drugs, but it's worth keeping in mind that's largely because it's still an emerging technology. So I don't think it's unrealistic to expect the prices to drop significantly as the field develops more, initial R&D costs get recuperated, and competition arrives.
And I think it's also worth remembering that gene therapy drugs are, for the most part, legitimate cures, so they're generally one-time or short-term costs. And the current costs of traditional treatments for the same diseases often run into similar price ranges, or even significantly higher, but just spread out over a lifetime.
E.g. The current treatment for hemophilia requires a lifetime of regular injections of clotting factor replacements, amongst other things. So while Hemgenix (hemophilia B gene therapy drug) costs $3.5m, and Roctavian (hemophilia A gene therapy drug) costs $2.9m, even at those prices they're still considered economically cost-effective, since the lifetime costs for traditional treatment of hemophilia are estimated to be in the $20m (for HB) to $100m (for HA) range.
Sure. But the doctors are talking about the treatment not reaching African countries where the need is the most severe. Comparing those costs to hemophilia care in Africa and the same concerns are...
Sure. But the doctors are talking about the treatment not reaching African countries where the need is the most severe.
Comparing those costs to hemophilia care in Africa and the same concerns are raised - not enough diagnostic screening or education sure, but ultimately older treatments
Another issue of Haemophilia care is access to novel therapies for low-income countries [21]. These novel therapies have shown to greatly improve the management of Hemophilia, and will become the gold standard for hemophilia treatment. ...
Gene therapy is also in the pipeline and there are now very promising results, and the hope is that market authorization will be obtained in the very near future. It is feared that the availability of these new treatments will deepen the gap already existing between high and low-income countries, because of a number of barriers to access. Currently, initiatives coordinated by the WFH, in partnership with private laboratories, have provided donation of EHL CFC and Emicizumab for selected patients in resource-limited countries. ...
In summary, 75% of Hemophilia patients live in developing world and do not have access to routine care due to many access barriers. Challenges of access to African countries remain, and should be tackled by a participative approach, taking actions to ensure more accessibility, education and linkage with other prevalent disease.
About 79% of new diagnoses of sickle cell are in Africa alone if my napkin math and an infographic are correct.
I'm aware of how all this works, and likely the price will drop some, but as someone taking a pill that Merck got approved in 2006, and may be off the generic market until 2029 depending on their lawsuits (20 years plus up to 30 months with lawsuits, plus multiple patents are involved I guess? ) I'm not optimistic that self-donated bone marrow transplant is going to be that cheap or widespread.
I'm happy it's gonna save lives. Just tired of the people that need things the most being the last to get it.
Yeah, I hear you, and feel the same. :( With such ridiculously expensive to develop and manufacture drugs, I can at least understand the reasoning behind that. But even when cost isn't the major...
Just tired of the people that need things the most being the last to get it.
Yeah, I hear you, and feel the same. :( With such ridiculously expensive to develop and manufacture drugs, I can at least understand the reasoning behind that. But even when cost isn't the major issue, the people most in need often still get the short end of the stick. E.g. Leprosy can be treated with standard antibiotics costing only ~$2 up to ~$30 for severe cases. And yet despite that there are supposedly still hundreds of thousands of new, largely left untreated cases worldwide every year. :(
Honestly I actually disagree in that I don't understand the reasoning behind not saving more lives and that not being a fundamental motivation for the people behind these decisions. I understand...
Honestly I actually disagree in that I don't understand the reasoning behind not saving more lives and that not being a fundamental motivation for the people behind these decisions. I understand that it's not their motivation but it doesn't make sense and I can't understand it at a fundamental level. I have to make judgement calls I don't love but they're never me falling on the "death" side of a "life or death" call.
But if John Green's advocacy on the tuberculosis issue has taught me anything is that they only care about money, and the next cure would be something as common as dirt and cost literal pennies to refine millions of doses and they'd still charge $1.50 a pill.
I may have woken up a grouchy this morning. This isn't directed at you by any means
I was more excited about the technological implications until I looked up how bone marrow transplants work. It is quite frightening.
From what I can gather, you have to "make space" for new marrow. That's a euphemism for killing your existing bone marrow with drugs and/or radiation. Then you receive the donor stem cells via blood transfusion. The process leaves you vulnerable to infection.
The improvement this offers is that there should be no complications arising from incompatibility, since you are your own donor.
This just makes me wonder how long it will take before CRISPR on human embryos becomes practical. It would be better to prevent genetic diseases from the start.
CRISPR on human embryos is practical already. It has been for a while. There are three Chinese babies born around 2018 using CRISPR to choose genetic traits, and last I heard they were alive and well. The doctor has been disgraced by the medical establishment for doing it. It is illegal in every country, as far as I am aware.
https://sl.bing.net/hE1iMM34ckC
Note: the US researcher I read about is not implanting embryos.
I think this is the one you referred to, where they modified the embryos to reduce the risk of HIV—a not very useful modification considering the risk involved.
Seems like it's not entirely a legal question at this point, but rather an ethics and PR one.
I'll try and find a source but I remember reading at the time that a side effect of adding HIV resistance genes was an increase in intelligence as well
I'm hopeful this will lead to long term results but I sort of despair that it'll get cheap enough to be widespread enough to make larger impact. Obviously it's great to save lives but it's so disheartening to see treatments out of reach for so many
Almost all the world's currently most expensive drugs are gene therapy drugs, but it's worth keeping in mind that's largely because it's still an emerging technology. So I don't think it's unrealistic to expect the prices to drop significantly as the field develops more, initial R&D costs get recuperated, and competition arrives.
And I think it's also worth remembering that gene therapy drugs are, for the most part, legitimate cures, so they're generally one-time or short-term costs. And the current costs of traditional treatments for the same diseases often run into similar price ranges, or even significantly higher, but just spread out over a lifetime.
E.g. The current treatment for hemophilia requires a lifetime of regular injections of clotting factor replacements, amongst other things. So while Hemgenix (hemophilia B gene therapy drug) costs $3.5m, and Roctavian (hemophilia A gene therapy drug) costs $2.9m, even at those prices they're still considered economically cost-effective, since the lifetime costs for traditional treatment of hemophilia are estimated to be in the $20m (for HB) to $100m (for HA) range.
Sure. But the doctors are talking about the treatment not reaching African countries where the need is the most severe.
Comparing those costs to hemophilia care in Africa and the same concerns are raised - not enough diagnostic screening or education sure, but ultimately older treatments
About 79% of new diagnoses of sickle cell are in Africa alone if my napkin math and an infographic are correct.
I'm aware of how all this works, and likely the price will drop some, but as someone taking a pill that Merck got approved in 2006, and may be off the generic market until 2029 depending on their lawsuits (20 years plus up to 30 months with lawsuits, plus multiple patents are involved I guess? ) I'm not optimistic that self-donated bone marrow transplant is going to be that cheap or widespread.
I'm happy it's gonna save lives. Just tired of the people that need things the most being the last to get it.
Yeah, I hear you, and feel the same. :( With such ridiculously expensive to develop and manufacture drugs, I can at least understand the reasoning behind that. But even when cost isn't the major issue, the people most in need often still get the short end of the stick. E.g. Leprosy can be treated with standard antibiotics costing only ~$2 up to ~$30 for severe cases. And yet despite that there are supposedly still hundreds of thousands of new, largely left untreated cases worldwide every year. :(
Honestly I actually disagree in that I don't understand the reasoning behind not saving more lives and that not being a fundamental motivation for the people behind these decisions. I understand that it's not their motivation but it doesn't make sense and I can't understand it at a fundamental level. I have to make judgement calls I don't love but they're never me falling on the "death" side of a "life or death" call.
But if John Green's advocacy on the tuberculosis issue has taught me anything is that they only care about money, and the next cure would be something as common as dirt and cost literal pennies to refine millions of doses and they'd still charge $1.50 a pill.
I may have woken up a grouchy this morning. This isn't directed at you by any means
No worries. I totally understand the need to vent about the unfairness of it all, and the greed that often lies behind that unfairness. :(