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Chrisp
24th November 2018, 17:42
Hello
There are lots of discussions about Riluzole, Rch4, Edaravone,stem cells and gene therapy etc. Where is the evidence that some of the treatments work or do not work?
I know that there is a lot of research into a cure for MND,but there is little evidence of progress.
Chris

Ellie
24th November 2018, 19:41
Any licensed medicine must first undergo several clinical tests to establish efficacy and safety, so both Riluzole & Edaravone have been through that process.

Brainstorm is currently in Phase 3 of NurOwn stem cell treatment in the US.

Results of clinical trials are available online.

RCH4 is not a licensed drug.

Chrisp
25th November 2018, 10:11
Hello

There are good reports on Edaravone, so why is not prescribed by the NHS?
Chris

Kayleigh
25th November 2018, 12:29
Hello Chris,
It looks like from the letter you received from Tim Farron (in your thread titled Edaravone started on 11 Sept) that this drug is not yet licensed for use in this country because it is still being considered by the European Medicines Agency. It may be argued by the Government that the drug needs to go through more clinical trials and tests etc before it could be considered for being licensed for use here. There is also the issue that the Government may not want the cost of the drug to be covered by the NHS if it does not deem it to be 'effective enough'. In America the drug is covered by people's private health insurance. Surely they would not cover the cost of Edaravone if it was dangerous to use or if it had no medical benefit. If I lived in America and I had the chance to take Edaravone then I would take it. It may sound simplistic to say, but if it is good enough for MND patients in America then it is good enough for me! I am not a scientist and am not very good at analysing scientific data but all I know is that 'red tape' is stopping us from having the opportunity to access Edaravone which must have some chance of extending the lives of people with MND, otherwise it would not be licensed for use in countries including the USA. The Government here has the luxury of taking as much time as it deems necessary before making a decision concerning Edaravone - but time is limited for us!
(Just my thoughts as an ordinary mum and wife who has MND)

Best wishes
Kayleigh x

I had originally put in this post that it would be illegal to import Edaravone for personal use - but since then Ellie has very helpfully pointed out that it is NOT illegal to import an unlicensed drug but does need the support of a GP (as in her 2nd post below) I apologise for my error and apologise if I mislead anyone. Still annoying that it is not available here on the NHS!

Ellie
25th November 2018, 12:56
Edaravone is not currently licensed in the EU.

The manufacturers of Edaravone have applied to the EMA for marketing authorisation and is under evaluation.

There is no indication of timescale but, barring an agreement, it remains to be seen what happens when the UK leaves the EU.

Edaravone is available to buy through a Dutch website an is available to those with ALS in Italy simply because they've a law specifically allowing for use of unlicensed drugs in certain circumstances.

Even if it gets approval, countries which cover all or part of drug costs through public funds will do a cost v benefit analysis before deciding whether or not to provide it.

Ellie
25th November 2018, 13:05
Kayleigh,

It's not illegal to import Edaravone into the UK for personal use with a Doctor's involvement - same goes for many other drugs which are licensed in other jurisdictions.

Love Ellie.

Kayleigh
25th November 2018, 13:22
My mistake Ellie - many thanks for pointing this out! I will add a note to my post amending this. It is just so frustrating that a decision about it being licensed here and whether it will be available on the NHS is taking so long!
Best wishes
Kayleigh x

Catsparkle
26th November 2018, 18:02
My consultant has pretty much told me not to bother with riluzole, as did the care centre. I guess on a quality of life v side effects v effectiveness?

Kayleigh
26th November 2018, 18:37
Hello,

I thought that it was left to each individual who has MND to decide whether they take Riluzole but maybe there are factors (such as certain medical conditions) that do not make Riluzole suitable for everyone with MND.

I was told about Riluzole by my consultant and there is also some useful information about it on the MNDA website. There are side effects but not everyone who takes Riluzole has the side effects. The decision about whether or not to take Riluzole was left to me and I researched it before I made my decision. If someone decides to start taking Riluzole, they do not have to continue taking it .... they can decide to stop taking it at any time.

What needs to be taken into account though is that if someone with MND wants to be part of a clinical trial then they must NOT have taken Riluzole - if they have taken Riluzole then they will not to be considered for the trial (that is what a I have read concerning clinical trials but this info would have to be checked out according to which trial it was).

Best wishes,

Kayleigh x

Kayleigh
26th November 2018, 18:49
Further to my previous post, this is a link to MNDA factsheet about Riluzole
https://www.mndassociation.org/wp-content/uploads/2015/07/05a-riluzole.pdf

Ellie
26th November 2018, 20:27
Hi Lisa,

It's YOUR decision whether or not to take Riluzole - the only drug approved in the UK for ALS - not someone's who isn't living with ALS...

Most people on the Forum take it, some don't due to adverse side effects, but whatever you decide, do it for the right reason.

Love Ellie.

Catsparkle
26th November 2018, 22:37
HI Ellie, how do you mean the right reason? I tend to experience side effects from drugs quite strongly. The fact sheet says there's little effect on progression? Hmm,I'm continuing to read around it anyway..I do appreciate there are no other drugs licensed for mnd in the UK x
https://www.thelancet.com/journals/laneur/article/PIIS1474-4422(18)30091-7/fulltext#
I should clarify too, he did make it clear from the start that he's happy to prescribe it if I'd like to.

Chrisp
27th November 2018, 13:01
Hello
So it is the cost of Edaravone and the procedure for the hospitals. In real terms I would cost nothing compared to hospital admissions.
Chris

Ellie
27th November 2018, 13:47
Hi Lisa,

By "for the right reason" I mean it being your decision and not that of your Neurologist.

Data from real world experience shows a much greater added survival, particularly when Riluzole is started early, and real world experience trumps clinical trial data for me! My clinic is pro Riluzole because of its own data but obvs we have a choice.

If your interested, see https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6053101/

The more balanced info you have should make for a more informed decision.

Love Ellie.

Ellie
27th November 2018, 17:09
Hello
So it is the cost of Edaravone and the procedure for the hospitals. In real terms I would cost nothing compared to hospital admissions.
Chris


Chris, apart from health services not seeing the bigger picture...

Hospital admissions for those with ALS tend to be mainly for feeding tubes and pneumonia, which Edaravone even at its best, wouldn't eliminate.

The data on Edaravone suggests fewer than 10% would derive actual benefit from it but the cohort of newly diagnosed people with ALS is small, so that's a plus.

In the US where it is available, paid for by insurance companies, those companies impose criteria on eligibility e.g. date since diagnosis, breathing scores etc. before approving Edaravone for their client.

Love Ellie.

TIANDB
27th November 2018, 20:48
Hi Lisa,

By "for the right reason" I mean it being your decision and not that of your Neurologist.

Data from real world experience shows a much greater added survival, particularly when Riluzole is started early, and real world experience trumps clinical trial data for me! My clinic is pro Riluzole because of its own data but obvs we have a choice.

If your interested, see https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6053101/

The more balanced info you have should make for a more informed decision.

Love Ellie.

Hope you are not trying to say Riluzole is some kind of miracle drug. It is not and benefits are slight it is known and stated to possibly extend time to tracheostomy by 90 days maybe it seems to work better than that to some. PLM reports are usually right, no one sings and dances about Riluzole or even Edaravone ( Radicava) and how they even got through trials as General ALS treatments is beyond me.
Independent patient reports on PLM by May 2017:

Evaluations of efficacy → Major Moderate Slight None Cannot tell
Riluzole 3% 6% 8% 15% 68% So 81% Slight or no Benefit.
Edaravone 0% 17% 18% 20% 45% So 83% Slight or no benefit.

https://www.patientslikeme.com/treatment_evaluations/browse?brand=false&condition_id=9&id=11

https://www.patientslikeme.com/treatment_evaluations/browse?brand=false&condition_id=9&id=25456

Ellie
27th November 2018, 21:22
No Kevin, I am not trying to say anything of the sort.

Skipton
28th November 2018, 13:27
Thanks Ellie. Totally agree that real world info is very useful

Catsparkle
28th November 2018, 14:09
Thankyou Ellie, I'll have a good look into that. My uncle is a research professor into MS, so I'll get him to help me scan through the published research as well. The article you've linked to is the first I've seen that's positive about riluzole, so that's really helpful. xx

Kayleigh
28th November 2018, 15:34
Hello Ellie and Lisa,

Ellie - thank you for sharing the real world article about Riluzole. I had not seen this article before and found it very interesting.

Lisa - It is a very personal decision about whether you take Riluzole or not. I am glad that you are taking as much time as you need to research Riluzole so that you can make an informed decision - whatever you decide, you will have peace of mind that you are doing what is right for you.

Love and best wishes
Kayleigh x

Catsparkle
30th November 2018, 09:16
Thankyou Kayleigh x Yes, I'm aware that one of the very few benefits of the disease for me so far is that although I'm losing function and get crazy tired, I don't feel ill in myself. So to take something that could make me feel ill would have to be a careful cost/benefit analysis. My uncle said it it benefited some, and it could be worth trying to see how well I tolerated it. Lisa x

Doug Carpenter
30th November 2018, 11:44
Hi Catsparkle

You're absolutely right to take time to come to an informed decision.

I think the paper Ellie showed us, which I didn't know about, is particularly relevant.

All I can offer is anecdotal evidence of no side effects after taking it for 18 months. But we are all different!

Doug

Kayleigh
30th November 2018, 15:15
Hi Lisa,

It is good that you have your uncle to discuss Riluzole with because, being a Research Professor, he must be great at helping you analyse all the information available - there is so much to read on the Internet that sometimes we can get information overload!

I am with you about MND causing tiredness. Have you been prescribed any nutrition drinks such as Complan or Fortsip? If not, you may wish to speak to your Dietitian (if you have one) or your GP about getting some prescribed. These may give you more energy as they will boost your intake of calories and vitamins/minerals.

Love and best wishes,
Kayleigh x

Dan
30th November 2018, 20:10
I questioned taking riluzole when I was first diagnosed. My neurologist explained that the often quoted 3 months more was the result of a 18 months trial so 3 months longer life for every 18 months seems worth it to me.
Also I agree that some trials do not allow anyone who takes riluzole to take part but not all trials! I am on a trial and as long as I stay on riluzole for the duration!
Let’s hope that in the future riluzole with become a drug of the past....Dani

Kayleigh
1st December 2018, 13:33
Hello Dani,

I was interested to read your post and to learn that you are taking part in a clinical trial. I hope that everything is going well for you.

The only other person I have read about on this forum who is doing a clinical trial is Marigold, who is on the MIROCALS trial.

I read on the MNDA website that if someone wants to be considered for the MIROCALS trial then they must not have taken Rilozole yet (from reading Marigold's posts, Rilozole is taken during the trial after some baseline tests have been carried out).

I hope you are having an enjoyable weekend.

Love and best wishes to you (and also to Marigold if she reads this post).

Kayleigh x

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