World-first therapy using donor cells sends autoimmune diseases into remission
nature.com
nature.com
And on the apparent long term risks of CAR-T: https://www.science.org/content/blog-post/downside-car-t-the...
I think that sounds pretty fucking good considering the other option is definitely just being dead. That's as close to a miracle treatment as I believe we will get.
Autoimmune diseases in no small part ruined my life. Forgive me if I seem rude, but I don't trust pharma to solve jack or shit without the correct financial incentives, and those proper financial incentives just can't organically exist for chronic sufferers of any novel disease.
If you read the article, you'd see that one of the features is that in the vast majority of cases, the B-cells return, but not the B-cells that were causing the immunodeficiency
quote: "Once injected into the hosts, the CAR T cells got to work. They multiplied and targeted and destroyed all the B cells — including pathogenic cells linked to the autoimmune conditions. The bioengineered T cells survived for weeks in the recipients before largely vanishing. Eventually, new healthy B cells returned, but no pathogenic ones did. A similar response has been observed in people with autoimmune conditions who received CAR T cells derived from their own cells."
My point being that the immune system appears to remains functional with this treatment, differentiating it from the clinical therapies that are available to you currently.
Speaking as someone who has also been let down by the medical system, but also who wouldn't be alive without it: I agree that Big Pharma is a problem (anyone disagreeing with that is arguing against the scientific evidence -- Ben Goldacre's book Bad Pharma is a good introduction to the problems with the industry), and that in the gross case they are lacking financial incentives. At the same time, this does not mean that the clinical therapies we have or are developing, are utterly ineffective.
It would be nice to dream of it happening sometime soon.
So sure we could speed things up by killing more people undergoing medical experiments. But the current approach of validating safety in humans then efficacy in humans is inherently serial. Further a lot more stuff is going into the pipeline than actually ends up working.
FDA can fast track certain drugs, but it really needs to show amazing efficacy.
There is also a breakthrough therapy designation where the clinical evidence is solid enough to release the drug while they finish full trials but that’s only in exceptional cases.
(Please don’t downvote me because you don’t like the tone of that. Europe banned artificial food colors and Japan is using self-replicating RNA vaccines. There is a wide spectrum of risk tolerance and healthcare does not revolve around the USA)
Every few years, there is an scandal for illegal medicine, but I think most of the case it's about using industrial silicone in human cosmetic surgery or something as stupid like that, not cutting edge new drugs. Perhaps it's possible to get tourist-illegal-medicine, but I strongly advice against that.
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> Japan is using self-replicating RNA vaccines
Do you have a source for that? I can try to take a look. I suspect it's very bad journalism reporting.
Self-replicating RNA are just https://en.wikipedia.org/wiki/Viroid but they only survive in plants, not animals. If they make their own coating, they are retrovirus. Writing a retrovirus from scratch is too difficult, some vaccines use edited virus, where they add the interesting part but also remove another part to the virus can't reproduce outside the lab. And there are vaccines with "live" attenuated virus, like the oral polio vaccine, but they are more difficult to make.
Your characterization is inaccurate, of both the reporting and the technology
> The ARCT-154 study vaccine consists of sa-mRNA encapsulated in lipid nanoparticles. The RNA comprises a replicon based upon Venezuela equine encephalitis virus (VEEV) in which RNA coding for the VEEV structural proteins has been replaced with RNA coding for the full-length spike (S) glycoprotein of the SARS-CoV-2 D614G variant.
IIUC the RNA makes copies of itself inside the cell, but it does not get a capsule of proteins to travel and invade other cells. Is this correct?
(A few more details in https://en.wikipedia.org/wiki/MRNA_vaccine#Amplification but not enough for my curiosity level.)
My uncle has developed an autoimmune respiratory disease over twenty years ago, and I know he would give a whole lot to improve his condition.
For context, I had been on Taltz before for psoriasis, and it worked very well. It seems like il17 inhibitors specifically are extremely targeted in what they affect.
In comparison, I was also on otezla at one point (tnf-a inhibitor) and it didnt work for me either, and the side effects were terrible.
Which was not the case with otezla. But tnfa blockers - yeah those are bad times.
In any case, good luck with your treatment!
Imagine if the billions of dollars being blown on boiling the oceans for a slightly better autocorrect were being used on basic and applied biological research instead. I have no doubt many afflictions would be a thing of the past (at least for those who embrace science instead of superstition).
The NIH already gets $47 billion per year, do you think they should get more?
There's nothing preventing pharma companies from just charging the equivalent of a lifetime supply for a one-off treatment. In fact, this is how cancer treatments are priced at the same time being the single largest source of revenue for pharma:
https://www.statista.com/chart/18311/sales-revenues-of-drug-...
Personally, I'd rather explore Elysium than give the entirety of my already insufficient and all prospective funds to what I consider pharmaceutical pimps. Poverty in America is, in my experience, worse than much of the 3rd world, where the practice is often more salubrious, less stigmatized and better performed, depending on one's values.
My friend would have long been dead of hep-c had he remained in the US. Although the US is my home and I'll go nowhere else, he took advantage of his Aussie ancestry and obtained citizenship, relocated and was treated for free. Here it would have been at least 250,000.
On the subject of cancer treatment, notably in the US, I'll not argue the subject, but will directly offer my perspective, which views it as highly exploitative and financially motivated. One example is the absurd reluctance to embrace or iin some cases, explore, the subject of enzymatic therapies and other 'foreign' procedures.
I'm an incorrigible cynic here and not worth trying to educate.
These companies are very much profit-driven and in terms of pricing as cold-hearted as they come.
It's just that if they could get your money now at once, they would do it. There's no scheme to keep you paying again and again - they're just not able to provide a different solution.
Meanwhile if a treatment isn't explored by them it's usually due to one of the following reasons:
-Not patentable.
-Not broadly applicable (e.g. only effective in people with specific, rare genes).
-Not effective.
Edit: i was mistaken, see below.
Maybe I’m too optimistic but I’m sure the overall cost can be reduced dramatically over time even with individualization.
These therapies are very individualized and will probably be very expensive.
How is CBT and casting a broken bone in any way like that? Those two things aren't reliant on medicine at all. Please explain, because I feel kind of stupid right now.
This approach does scale, but has new safety risks.
They aren't genetically modifying each patient's own cells, one patient at a time, but are trying to create a line of generic T-cells that will attack only the B-cells of any patient.
The risk is that genetically modified T-cells that are not derived from your own cells might attack all the cells of your body, and not just your misbehaving B-cells.
They have made additional modifications to the CAR-T T-cells that they believe will prevent them from going rogue, but more safely testing is needed.
From what I've read, this same more generic approach is being attempted with CAR-T cancer treatment as well.