You might have heard about this in the news (again!): Scientists close to a cure for MS.
To be honest, I heard this on the radio this morning whilst waiting for a blood test in my GP surgery but disregarded it. I zoned out, not just because waiting for a blood test is far from my favourite pastime ever (I’m past literally jumping out of the chair when the needle approaches, hell I can even go on my own! No Brave Girl sticker though sadly), but because anything stating ‘cure’ in the news is just going to be plain wrong – the follow-ups for any treatments at the moment are not yet long enough to know whether the MS comes back. Whatever caused the MS in the first place could happen again and tip the immune system again. The media Love HSCT(hematopoietic stem cell transplant, or in other words stem cell reboot) because some people get amazing improvements. It’s something of a ‘miracle’ – a great story. However, it is currently still pretty dangerous (up to a 1/20 chance of dying in some centers), is in no way guaranteed – some do not see improvement and continue to progress – and it is unknown as to how long the effects last. So it’s not a treatment to be taken lightly. It’s a last hope.
HOWEVER this research IS very interesting! – they appear to have worked out how to stop the MS coming back post-treatment. Their trial showed no MS progression for at least 5 years since treatment, and also excitingly showed an absence of inflammatory markers in the blood. Very interesting.
The lead researcher has done a guest post for the St Barts neuro blog (a blog that I wholeheardedly recommend – run by some of the top and most engaged neuros arguably in the world). Take a squizz.
Out of 24 patients, tragically someone died. However, the lead researcher (Prof Mark Freedman) believes this was because they didn’t control the dosage carefully enough of the highly-potent (nasty!) drug that kills off your immune system. Basically the poison worked too well. Let’s see what the neuro community has to say and wait for repeated trials in other institutions.
The other interesting finding I’d like to point out is that the results indicate that any damage that had begun before the immune system was rebooted continued even after the immune system was all shiny and new, and for 2 years. This is a magic number. I’m going to be stressing this a lot in future posts, but to just briefly cover this: it has been observed by many neuros that the high-efficacy Disease Modifying Treatments (DMT’s) do not stop damage that has already started. It’s like a one-way street, taking up to 2 years before the damage stops. So if you have started a DMT you may well have symptoms worsening for up to 2 years but that does not necessarily mean that the DMT is not working. Your DMT may well be stopping other parts of your body from succombing to MS damage, and this saving will become more evident as time goes on, because your disease will stabilise up to the 2 year mark. It’s worth knowing this so you can make an informed judgement call if you do start a high-efficacy DMT (of which I mean Lemtuzumab, Tysabri and Gilenya).
General info: Prof Giavannoni – the first commenter on that blog post – is the well-respected prof of the St barts team. His comments are always well worth listening to. Other commenters make good points – how to fund something that is so dangerous at the moment (and stops Pharma gaining any money vs. the requirement of regular drugs – who will fund Phase 3 trials?) and whether we can truly consider the follow up to be long enough to show MS will not return. Perhaps the participants have just not yet been exposed to whatever the trigger(s) for MS are.
We’ve some way to go before this treatment becomes a viable option for many, but the findings don’t just show a potential future treatment making good steps – they also help to understand what the underlying mechanisms of MS are, which we still don’t know. And that is perhaps something to watch.
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1 thought on “Research Update: Yet another media frenzy over a Stem Cell ‘cure’? Or is this interesting?”
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Some people have been followed now for 13 years, so we are starting to get long-term data on this. The long timescales of MS are always a difficult problem when doing studies. I think it is exciting but certainly far from a cure as you mention. Though I have a followup on the two years timeline. I think that may vary a lot treatment to treatment. Because this treatment takes much longer to complete, that is part of the timeline. Most treatments see improvements within several months, say 3-6 months. Copaxone is 12 months. Alemtuzamab is 2 years.