Derek Lowe on this use of CAR-T: https://www.science.org/content/blog-post/car-t-autoimmune-d...
And on the apparent long term risks of CAR-T: https://www.science.org/content/blog-post/downside-car-t-the...
For the sake of my question let’s assume this is legit and magically works for a wide range of autoimmune conditions. How many years away are we from being able to sign up for this treatment? 10 years? 20? 30?
It would be nice to dream of it happening sometime soon.
Maybe not long at all. Scroll down to the section that says "Our Approach : Building a Designer Immune System" on this site[0]
amen. I've tried 3 tnf inhibitors that have not worked (and had negative side effects). Cosentyx (il 17a inhibitor) kinda scares me. I always like to keep my hopes that some better autoimmune treatments could come soon
In the US, my "back of the napkin" estimate tends to be 8-15 years, if it passes trials.
Is there a way to speed up trials without compromising quality?
Possible to use surrogate endpoints, which is used for slower evolving diseases. But then the claims need to be associated with the new endpoints.
FDA can fast track certain drugs, but it really needs to show amazing efficacy.
Not really, the process gets shortcut when the benefits of moving fast are dramatic enough.
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.
Depends. Do you want to be a patient, or a trial test subject?
In Mexico, maybe a year?
(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)
While the promise is great, let's be real. All of these personalised therapies get loads of funding because they almost inherently will be very expensive since they cannot scale, meaning drug manufactures supplying these can make a killing even compared to already expensive but more commonly used therapies. I guess this is more a comment on car-t usage in oncology and if this leads to seriously improved health outxomes, then of course, all the power to them.
Edit: i was mistaken, see below.
> very expensive since they cannot scale
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.
I understand this therapy is inherently more complex, but there are plenty of medical treatments that have helped millions of people without scaling like a drug: IVF, cognitive behavior therapy, casts for broken bones, etc.
Maybe I’m too optimistic but I’m sure the overall cost can be reduced dramatically over time even with individualization.
That’s the whole point here, it’s not personalized. It comes from a donor so it’s somewhat standardized.
That sounded almost too good to be true, especially with autoimmune stuff which seems even trickier to tackle than many cancers. One hell of achievement if it pans out long term.
I suffer from psoriasis. This seems like another 'solution' that really only addresses how the diseases manifest but not the root cause. I can go get a shot today that blunts my immune system, and my psoriasis will calm down for a year but I'll probably get a couple wicked chest colds.
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.
> This seems like another 'solution' that really only addresses how the diseases manifest but not the root cause.
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.
Does this new donor-cell therapy potentially include the top-10 autoimmune diseases, such as rheumatoid arthritis, lupus (checked), and/or Hashimoto's thyroiditis?
Can I ask how “top 10” is defined? Is it the most common, or perhaps the most destructive to the individual?
I sorted by the most common, not the most destructive, but the key point is how 'easily' this could be extrapolated to other autoimmune diseases.
In the future a pill with donor cells will be prescribed 1x a day for 3 days.
Depends. If it's up to bigPharma, it will be 1 pill 3x a day, forever. Chronic patients are the best to the bottom line
Keep an eye on sickle cell anemia cure then.
I thoroughly agree with your grayed comment. It doesn't necessarily imply that it's all greed and evil, but there's enough of it to dignify your statement, without applying my own experience.