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Article on Barts - HSCT

http://multiple-sclerosis-research.blogspot.com/2015/01/clinicspeak-are-we-being-too.htm

This blog post has triggered a lively debate and it would appear times are changing.

Hi Humbug,

tried the link bt it says the blog/page doesn’t exist anymore.

Dave.

The article is still there and makes very interesting reading.

Very interesting. Not really sure how I feel about it though. Would need a lot of questions answered first I think.

Have heard that this sort of treatment could help the MS but cause other problems that could be even worse. How right this is I don’t know.

Shazzie xx

What I have found frustrating when it comes to research is that no conclusion ever seems to be reached and research projects fizzle out and get lost.

I thought the survey at the end focused the mind wonderfully. One of the questions was: “What risk of dying are you willing to take?” (I may be paraphrasing slightly, because I’m too lazy to revisit the website) - and a choice of percentages.

For me personally, as things stand at the moment - none whatsoever! I know my views might change if I were a lot, lot worse. But for me, at the moment, there is absolutely no treatment that would justify a risk of dying - that is just too big a stake. If I were in the position it was highly likely the MS would kill me anyway within the next few years (it’s rare, but let’s not pretend it doesn’t happen), then certainly I might be thinking: “My odds don’t look very good anyway, so what have I got to lose?” So I can completely understand it would appeal in some circumstances - it might even appeal to me in some circumstances.

But in my actual circumstances, as they apply today? No, there is nothing that would justify a treatment I might not survive.

Before anybody jumps in - yes, I do know that even commonly prescribed symptom relief does carry a very, very tiny risk of death - you might have a rare allergic reaction, or something like that. So nothing is absolutely risk-free - even an aspirin.

But faced with a table: “Which of these risks of death do you accept?”, and assuming my MS continues much as it has done, my reaction is: “None of them.”

Tina

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You have repeated this several times, but I don’t really understand why you think so.

As far as I can tell, a conclusion is always reached, and the results published - but it might not be in the sort of publication you or I would read every day - or at all. Nevertheless, it’s all available if you really want to find out.

I don’t really know what you mean by “fizzle out” either. A very high proportion of research (I think about 98%, but don’t quote me) does not translate into a successful treatment, because effectiveness was found to be too low, or risks and side effects too great, or both. However, I don’t think that means the research “fizzled out”. It did reach a conclusion, and the conclusion was there’s no mileage in it, because it would be unethical ever to give it to anyone.

Tina

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The early survey results are in and the comments make particularly interesting reading.

I think its great and about time voices were heard. Lets hope some good comes from it.

[quote=Anitra]

You have repeated this several times, but I don’t really understand why you think so.

As far as I can tell, a conclusion is always reached, and the results published - but it might not be in the sort of publication you or I would read every day - or at all. Nevertheless, it’s all available if you really want to find out.

I don’t really know what you mean by “fizzle out” either. A very high proportion of research (I think about 98%, but don’t quote me) does not translate into a successful treatment, because effectiveness was found to be too low, or risks and side effects too great, or both. However, I don’t think that means the research “fizzled out”. It did reach a conclusion, and the conclusion was there’s no mileage in it, because it would be unethical ever to give it to anyone.

Tina

Pick any drug in current use for treating m.s. and give me one concrete fact about it’s efficacy.

Is there any chance of getting this in the UK if you can pay for it? I would go to the US to have it but I don’t think I could live with myself if I went to Russia for it.

Thank you to humbug and whammel for posting about this. I will be trying to find out more.

For krakowian - below I have posted a few concrete results about the efficacy the best MS drugs, just to show that there is plenty of information out there. You can find more of this stuff by going to the section on disease modifying drugs, on this website. There’s plenty about side-effects and risks in the same place. I’m making the point that there is good quality information out there - you don’t even have to look very far to find it.

A two-year study showed that, on average, people taking Tysabri had a 67 per cent reduction in the number of relapses (compared to what would be expected if no treatment was taken).

Tysabri also slowed the accumulation of disability over two years. Only 17 per cent of people taking the drug had worse disability, compared with 29 per cent of people taking a placebo (dummy treatment).

A two-year study comparing Gilenya to a dummy treatment showed that it reduced relapse rates by 54 to 60 per cent, and reduced disability progression by about 30 per cent.

A one-year study showed that it reduced relapse rates by 53 per cent compared to beta interferon-1a.

In the DEFINE trial, Tecfidera was shown to reduce relapse rates by between 48 and 53 per cent, and disability progression by between 34 and 38 percent compared to placebo.

In the CONFIRM trial, showed that Tecfidera reduced relapse rates by between 44 and 51 per cent, compared to a 29 per cent reduction in those taking Copaxone. It also reduced disability progression by between 21 and 24 per cent, compared to 7 per cent for Copaxone.

MRI scans in both trials also showed a significant reduction in new or newly enlarging brain lesions (damage caused by MS) after two years in those treated with Tecfidera compared with those treated with either placebo or Copaxone.

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Nothing available in the UK for MS and need a trial first, although apparently, HSCT has been used to treat other conditions since the sixties. The cheapest place is Moscow at USD40,000 and there is a three year waiting list, so don’t expect the price to come down anytime soon.

There is a closed facebook group with plenty of information on file and quite a few anecdotal reports, in case you are interested.

The link doesn’t seem to work, but this is the group name - Hematopioetic Stem Cell Transplant - MS & Autoimmune Diseases

Thanks again.

Here we go - Sewing Chick’s already given you loads, but to take one at random (and because it’s early in the alphabet), Avonex: http://www.mstrust.org.uk/atoz/avonex.jsp

I quote:

Studies have shown that on average Avonex reduces the relapse rate in people with relapsing remitting MS by about a third and also reduces the severity of those relapses that do occur.

Are you suggesting a reputable organisation like the MSTrust is making this stuff up?

Note that “on average” doesn’t mean: “No concrete evidence”. The fact there is no guarantee it will be effective for a specific individual does not mean there is no evidence of overall effectiveness.

And “about a third” also does not mean: “No evidence”. OK, they haven’t quoted the exact figure to two decimal places. If I had all afternoon with nothing better to do, I could probably find it for you. But “about a third” is sufficiently clear to most people who might consider taking it.

For the record, I’ve absolutely no axe to grind about DMDs themselves - I chose not to take them. But I do dispute either that there’s “no evidence”, or that research fizzles out and gets lost.

I do accept there is no unambiguous evidence of long-term benefits. Projections of reduction in disability have been based on the assumption that most of it results from relapses, therefore if you can reduce relapses, you will reduce or postpone disability. But it is not known for sure that one follows from the other. Then again, even the oldest drugs are still relatively new (there were no DMDs at all before the 1990s), so we do not yet have a body of patients who’ve been followed into old age, to see how they did. The only thing that solves that is time. Eventually, we will know the answer, but until we have enough patients who’ve been on it a lifetime, we can’t know how much difference it makes. That will always be an issue with any new drug - we can’t tell how patients fared after 20, 30, 40 years.

Tina

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This study is probably one to keep an eye on. Stem Cell Therapy for Patients With Multiple Sclerosis Failing Alternate Approved Therapy- A Randomized Study - Full Text View - ClinicalTrials.gov

Last one, I promise.

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There is a wealth of info on the facebook site and elsewhere on the net.

Its something I’ve been following for a while.

thanks Anitra and Sewing Chick.

You quote ‘Avonex reduces relapse rate by about a third’ ‘Tysabri 67% reduction in relapse rate’ ‘Gilenya reduces relapse rate by 54-60%’

But what do these figures actually mean -

Will I as someone with m.s. have a 67% reduction in relapse rate if i take Tysabri - what is a relapse rate - what does 67% reduction mean? 67% of what?

Powerful drugs with equally powerful side effects are being prescribed to pwms without empirical evidence that they will help that individual. That is not right.

A trial reports the average outcome - there is no way of knowing how something will effect every individual.

Looking back at your posts it sounds like you are being deliberately obtuse but I will answer your question.

Relapse rate = how many times you relapse over a certain period.

This is a massive simplification but trials take two groups of people, one getting treatment (group a) and the other not (group b). They count how many relapses each group has over the period. If group b had 6 relapses and group a 2, this suggests that this 67% reduction is down to the treatment. The numbers they use are obviously much larger to avoid any difference between the groups being down to chance.

Some people will find the treatment works better than expected and some worse. We’re taking our chances but I’d rather that than just letting MS do its thing!

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