5 ms·

I've had MS for 20 years. The procedure for MS is HSCT. It's erroneously called "transplant," as it actually is stem-cell rescue. The basic idea is to extract

I've had MS for 20 years. The procedure for MS is HSCT. It's erroneously called "transplant," as it actually is stem-cell rescue. The basic idea is to extract your own stem cells, kill of most (not all) of your existing immune system, and then re-infuse your stem cells to "reinstall" your immune system. Hopefully before you get an infection while you don't have any defenses. This treatment has been shown to be effective for the most mild form of MS, Relapsing Remitting (RRMS), and only in the early stages of the disease. It's relatively risky, and if we were to do the procedure for everyone diagnosed with RRMS today, hundreds (if not thousands) of people would die from the treatment. Either from complications from the procedure, or from increased instances of cancer years down the road, since the poison part of the treatment is known to be carcinogenic. Meanwhile, there is Rituximab, which is a monoclonal antibody targeting the CD20 protein that has had its patent recently expire. This means it can be manufactured as a generic. Clinical trials recently run in England have shown that not only is it extremely effective at controlling the disease in the most mild form (RRMS), but CD20-targeting monoclonal antibodies are the only drugs known to be in any (meaningful) way effective for the more serious forms of the disease, Secondary Progressive (SPMS) and Primary Progressive (PPMS). Given the choice between HSCT or Rituximab, in terms of effectiveness, safety and cost, Rituximab wins as a treatment for MS, hands-down. For those stuck in the United States, the FDA will let you get Ocrelizumab (Ocrevus), which is basically Rituximab tweaked enough so that Genentech could get patent protection for it. With that patent protection, they got the financial incentive to run it through the FDA approval process so they can now sell it to you for the bargain price of about $80,000 per year. It's just about as effective as Rituximab and maybe only a little more dangerous.

pcnix 8y ago
It sounds like the more complicated version of the aggressive bone marrow translpants for cancer back in the 80s, and like those, causes increased risk for cancer down the line due to the carcinogens used. I've been looking into MS drugs recently for someone I know, and Rituximab definitely sounds like the better option.
ascorbic 8y ago
And for those wondering, in the UK Rituximab costs the NHS £349.50 for a course. https://bnf.nice.org.uk/medicinal-forms/rituximab.html https://bnf.nice.org.uk/medicinal-forms/rituximab.html
- isostatic 8y ago
  You’re clearly wrong. It’s only America where people are cured. The NHS has death panels to get rid of people they don’t like. /sarcasm
- pedrocr 8y ago
  That link only works if you are in the UK.
  - SamuelAdams 8y ago
    https://en.wikipedia.org/wiki/Rituximab https://en.wikipedia.org/wiki/Rituximab > In the United Kingdom this amount cost the NHS approximately £182 in 2015.[9] The average wholesale price in the United States of a typical treatment for rheumatoid arthritis (1,000 mg IV dose, 2 weeks apart) would have been $14,100 a month in 2014 ($705 per 100 mg)[10] but the patent expired in 2016.[
adrianratnapala 8y ago
> For those stuck in the United States, the FDA will let you get Ocrelizumab, which is basically Rituximab tweaked enough so that Genentech could get patent protection for it. So Rituximab was never approved by the FDA? I assume it too had once been under patent, and its manufacturer would have tried to get it approved.
- steelframe 8y ago
  In the U.S., the FDA approves a drug for a particular use. Rituximab would have to undergo a complete set of clinical trials to show its safety and efficacy for MS in particular. Otherwise doctors can only prescribe it off-label, and the system (i.e., liability) is set up to dissuade doctors from prescribing drugs off-label if there exists a drug approved by the FDA for the particular purpose.

pcnix 8y ago

It sounds like the more complicated version of the aggressive bone marrow translpants for cancer back in the 80s, and like those, causes increased risk for cancer down the line due to the carcinogens used. I've been looking into MS drugs recently for someone I know, and Rituximab definitely sounds like the better option.

ascorbic 8y ago

And for those wondering, in the UK Rituximab costs the NHS £349.50 for a course. https://bnf.nice.org.uk/medicinal-forms/rituximab.html https://bnf.nice.org.uk/medicinal-forms/rituximab.html

isostatic 8y ago
You’re clearly wrong. It’s only America where people are cured. The NHS has death panels to get rid of people they don’t like. /sarcasm
pedrocr 8y ago
That link only works if you are in the UK.
- SamuelAdams 8y ago
  https://en.wikipedia.org/wiki/Rituximab https://en.wikipedia.org/wiki/Rituximab > In the United Kingdom this amount cost the NHS approximately £182 in 2015.[9] The average wholesale price in the United States of a typical treatment for rheumatoid arthritis (1,000 mg IV dose, 2 weeks apart) would have been $14,100 a month in 2014 ($705 per 100 mg)[10] but the patent expired in 2016.[

adrianratnapala 8y ago

> For those stuck in the United States, the FDA will let you get Ocrelizumab, which is basically Rituximab tweaked enough so that Genentech could get patent protection for it. So Rituximab was never approved by the FDA? I assume it too had once been under patent, and its manufacturer would have tried to get it approved.

steelframe 8y ago
In the U.S., the FDA approves a drug for a particular use. Rituximab would have to undergo a complete set of clinical trials to show its safety and efficacy for MS in particular. Otherwise doctors can only prescribe it off-label, and the system (i.e., liability) is set up to dissuade doctors from prescribing drugs off-label if there exists a drug approved by the FDA for the particular purpose.