Gene therapies have revolutionized medicine. They are a functional cure for diseases by replacing mutated copies of genes in the patient’s cells with a functional copy. To date, only three gene therapies have been approved in the US: Luxturna, Zolgensma and now Hemgenix. With each proceeding gene therapy, the price has escalated. Luxturna costs $425k per eye. Zolgensma was the most expensive medicine in the world, at $2.125 million until Hemgenix came along a few weeks at an insane $3.5 million to treat Hemophilia. As the majority of people cannot afford these prices even with a viral GoFundMe campaign, insurance companies now have the godlike ability to determine who lives or dies.
This is a Stat worthy piece with detailed information and some pithy conclusions. I can only hope that future therapies set a new downward trend on pricing but as Dr. Zayner points out the opposite trend is pretty strongly evident.
We need control of this science, we need to all learn it so well we don’t need big pharma. We need to implement this in schools now- and for everyone- and FREE.
My good friend and his wife "hacked" her own aggressive cancer using new (and old) techniques in immunotherapy. I will send you a copy (for the rest of yall it is https://www.amazon.com/Curing-Cancer-Immunotherapy-happened-attempted/dp/0997820012) . The book is amazing because it not only nicely explains the treatment of cancer on a scientific basis but their own experimentation with immunotherapy. Not to mention the non-scientific issues with trials and why certain simple cancer cures like Coley's toxins (it's just strep throat bacteria!) are banned in the US. It is a great compliment to your own journey!
I feel like The Odin can provide people with education and materials to at least develop their own cures but as you point out, the biggest problem is providing supervision and some way to test. The Chees were able to work with the help of a couple of trusted physicians and it didn't hurt that the wife has a PhD in biology. I wonder if you can develop a network of physicians to act as these trusted advisors for experimenters in general?
One of the easiest ways to minimize risk is to experiment on a substitute, but it is impossible to replicate a human because of the biological complexities. Not sure if there are ways to "prove" that if a gene therapy works on a subset like a culture of cells, then you can safely assume it will work in a more complex biological environment. I know Dr. Sofia discovered a cure for Hepatitis C that has very little side effects, not sure if you can "lift" that research to work for the general case -- you'd know more about that than me :)
I suffer from a genetic disease myself and I think it's sick that companies generate such high earnings at the expense of people like me but regarding Zolgensma you don't take into account the fact that not every country conducts newborn screening for SMA, not every newborn is suitable for therapy if only because there are children in who the AAV virus can be destroyed in the body, so theoretically, a very expensive drug will not work, the things I have mentioned mean that despite 500 children with SMA in the US alone, the drug cannot be given to everyone every year and there are a few other conditions, sorry for my english but I'm writing with a translator :)
The amount of unnecessary suffering in this world infuriates me. I've seen it over and over again as a mental health professional and it continuously breaks my heart.
Glad someone with a background in bio hacking is bringing this topic to the surface. There are quite a few interesting, rare and evil diseases that can be cured with modern science instead we use surgical intervention until patients can no longer tolerate it or by some chance get a cure. Some way, some how we need to move this along
It's good to air our grievances ... but, how do we make a road map to freedom from the mighty boot-jack?
Is there a collaborative planning platform that can be used to make a plan for an open source version of these technologies?
If such a planning effort is successful, how do we defend the results against an assured legal pounding and thereby force the establishment to draw a clear line between technology and the right to own our own biology?
This is a Stat worthy piece with detailed information and some pithy conclusions. I can only hope that future therapies set a new downward trend on pricing but as Dr. Zayner points out the opposite trend is pretty strongly evident.
We need control of this science, we need to all learn it so well we don’t need big pharma. We need to implement this in schools now- and for everyone- and FREE.
Thank you for writing this!
My good friend and his wife "hacked" her own aggressive cancer using new (and old) techniques in immunotherapy. I will send you a copy (for the rest of yall it is https://www.amazon.com/Curing-Cancer-Immunotherapy-happened-attempted/dp/0997820012) . The book is amazing because it not only nicely explains the treatment of cancer on a scientific basis but their own experimentation with immunotherapy. Not to mention the non-scientific issues with trials and why certain simple cancer cures like Coley's toxins (it's just strep throat bacteria!) are banned in the US. It is a great compliment to your own journey!
I feel like The Odin can provide people with education and materials to at least develop their own cures but as you point out, the biggest problem is providing supervision and some way to test. The Chees were able to work with the help of a couple of trusted physicians and it didn't hurt that the wife has a PhD in biology. I wonder if you can develop a network of physicians to act as these trusted advisors for experimenters in general?
One of the easiest ways to minimize risk is to experiment on a substitute, but it is impossible to replicate a human because of the biological complexities. Not sure if there are ways to "prove" that if a gene therapy works on a subset like a culture of cells, then you can safely assume it will work in a more complex biological environment. I know Dr. Sofia discovered a cure for Hepatitis C that has very little side effects, not sure if you can "lift" that research to work for the general case -- you'd know more about that than me :)
Its a combination of greed and regulatory capture.
I suffer from a genetic disease myself and I think it's sick that companies generate such high earnings at the expense of people like me but regarding Zolgensma you don't take into account the fact that not every country conducts newborn screening for SMA, not every newborn is suitable for therapy if only because there are children in who the AAV virus can be destroyed in the body, so theoretically, a very expensive drug will not work, the things I have mentioned mean that despite 500 children with SMA in the US alone, the drug cannot be given to everyone every year and there are a few other conditions, sorry for my english but I'm writing with a translator :)
Maybe we need an open source platform for gene therapy? It worked in computing to introduce real competition, and lower prices.
The amount of unnecessary suffering in this world infuriates me. I've seen it over and over again as a mental health professional and it continuously breaks my heart.
Glad someone with a background in bio hacking is bringing this topic to the surface. There are quite a few interesting, rare and evil diseases that can be cured with modern science instead we use surgical intervention until patients can no longer tolerate it or by some chance get a cure. Some way, some how we need to move this along
There is a coming in this area, fresh off the heels of open-source software and open-source hardware. I will be the martyr! :-D Cheers Senior Zayner!
I know, but ... how else will they ever boot jack us in the neck and dominate our existence?
https://static.wikia.nocookie.net/harrypotter/images/9/9f/Magic_is_might.jpg
On a sincere note:
I thought I felt oppressed before... instead ...
I'd rather discuss how to FIX this??!!??
It's good to air our grievances ... but, how do we make a road map to freedom from the mighty boot-jack?
Is there a collaborative planning platform that can be used to make a plan for an open source version of these technologies?
If such a planning effort is successful, how do we defend the results against an assured legal pounding and thereby force the establishment to draw a clear line between technology and the right to own our own biology?
This is a fascinating topic. I wish I understood it better. Thanks for your work!