Doddie Weir

[billy106]

Doddie Weir was on the Scottish News tonight , Saying what most of with MND have been saying for years, That he finds it frustrating and unbelievable that with the Billions of pounds that have went into MND research and trying to find a treatment has Produced absolutely nothing. He also said that within months they can find a Vaccine for Covid19 does not add up.
My personal opinion its not justifiable for MND charities to throw Big Grants into Research or career funding . Smart seems to have turned into nothing , It started with a bang and flag waving , but in the end as usual they need more money....
Reminds me of Panorama did a programme into certain UK charities who were making recommendations on treatments for diseases but not the best ones . The reason was Big Pharma Companies were making massive donations to them on the basis they recommended their drugs. Google the worse ones ... Blindness Charity and Big Pharma Cash. also Drug Firms Fund British MPs

[Banned]

Doddie is correct and it has been going on for all of the years I have been in the MNDA and Stuart of Build-UK MND forum, the predecessor to this forum was an arch critic of our MNDA too.

Don't get me wrong, as many of us are, I want to see progress on new treatments and new technology and processes that help, not hinder.

The MNDA management team is weak.

There is no overall strategy to help us, rather just short term projects that get done and on with the next.

As Deborah Meadon says, "You have to have skin in it!".

We will see if Lee will make a difference but he is up against it.

[richard]

Doddie has done amazing things to bring MND to the fore front of the UK public’s knowledge. Link that with the BBCs wonderful publicity with Doddie, Rob Burrows and Stephen Derby we are getting the publicity. We now need dedicated targeted funding and one day we shall be there and see the end of this horrible disease.

Richard

[shelly21]

richard I so agree. I'm sharing lots on social media just to educate people.

I decided not to take part in trials as it just wasn't for me. I think its just a serious complex disease. Maybe there just isn't a cure.

I know that sounds pessimistic but somethings are just out of human hands.

[Ellie]

Maybe there just isn't a cure.

For now at least, many, including me, agree with you shelly21 The most likely scenario is that sporadic motor neuron disorders become more of a chronic condition, rather than a fatal one, before a 'cure' is available - whether that translates into a tangible improvement/benefit to the person living with the chronic condition might be in the eye of the beholder...

Familial ALS might indeed be stopped from developing in the first place.

[matthew55]

It's lovely to think that future generations l may not face what we have. But meanwhile we are set on our course with no deviation. ☹️😉😁🤗xx

[Onein300]

All,

As you are aware, Doddie Weir, Rob Burrow and Stephen Darby are actively working with patients, leading researchers and the 3 main charities on #United2endmnd.

The coalition was formed at a time when research into MND had reached a translational time. By translational we mean, time to rapidly move forward in light of many base neurodegenerative MND science advances that now underpin our understanding of the disease.

You could liken the time to all the technologies for building a car have now been prototyped and we are ready to build a car or more precisely a whole load of cars, in a factory.

To move forward, our researchers need a solid infrastructure in the U.K. to trial treatments, continue base science and discover drug candidates.

The coalition is exactly that. A coalition of all the key players in U.K. MND science.

Our spending review bid for the budget this week, which we submitted in July is well worth a read. It is very detailed but only 26 pages long and quite a straight forward document.

We are asking for a £50m government investment to build a new MND virtual Insitute which will have its own advisory science board and governance. The intention is that it would be funded directly by government, charities and industry. It will NOT be managed by any one charity alone, and the charities would still continue their vital work including care, campaigning, research as well as investing into the institute.

For an overview of the proposed governance, please see pages 19 to 24 of the proposal.

https://www.mndassociation.org/app/u...sion-final.pdf

To support the proposal submission the coalition has been working behind the scenes extensively. We have active discussions in motion with all parties, government Ministers, MPs, Civil service and Industry players, which to my knowledge and life with MND, are the most comprehensive that ever been held.

Along with recent promising developments in research, all of this activity, which has seen patients, researchers and different charities unite for the first time to ask for enhanced government funding, serves to underline current optimism for breakthroughs in MND research.

The awareness that has been raised, not least with the help of Doddie, Rob and Stephen has taken MND from a lesser known neurodegenerative disease to one that it is at the forefront, both in Parliament and in the public eye.

Like this disease we live with, we need to be relentless in campaigning for research funding and we are on the road for the long term.

Best wishes
Lee (aka Onein300 on Twitter and blog)

[matthew55]

You make them out as some kind of Saint but if they hadn't been professional rugby players who would have listened to them? Just saying.

[Deb]

I guess they have the platform to raise awareness and therefore funds because they are famous matthew55.
When I watched the Rob Burrow documentary I was full of admiration that he allowed himself to be filmed during some very private moments. He did this to get publicity in order to raise money. 😘🤗x

[matthew55]

You're not a cynic like me then. 😉😁✌️🙏xx

[Deb]

You're far too lovely to be a cynic.. we all know you ! X 😘🤗

[Gordan1111]

Thanks for update Lee

[Banned]

Thanks for the update Lee.

Let's start with the recent Tofersen failure. Please ask BioGen: -

1. In lay man terms, did the drug totally fail to work as anticipated?

2. Have 'lessons learned' been shared with other pharmas?

3. What are the main 'anti-sense' treatments in the pipeline for MND?

[matthew55]

More drugs. I already feel like I live in a chemists. 😉😁🤘🤗xx

[Onein300]

Thanks for the update Lee.

Let's start with the recent Tofersen failure. Please ask BioGen: -

1. In lay man terms, did the drug totally fail to work as anticipated?

2. Have 'lessons learned' been shared with other pharmas?

3. What are the main 'anti-sense' treatments in the pipeline for MND?

Afternoon Graham. Biogen are a Pharmaceutical company who are clearly hugely committed to our disease. Just in the time I have been diagnosed it’s evident that they are in it for the long haul. They are a beacon for MND research. I don't believe Biogen will be shy in coming forward ASAP with further information on the Tofersen trial results. But I will, for what it's worth, give you my opinion on your first two points.

In one sentence, Tofersen did not completely fail and might still become an effective treatment for MND.

Yes, the unfortunate headline from the trial results was that it was a ‘failure’ in a strict sense. Within the 6 month placebo controlled trial, the end point of significant alsfrs-r slope reduction was missed. But there is far more to it than that, although I must admit on the first reading of the data I did miss the promising aspects.

The data published publicly so far shows positive reductions in both the emerging biomarker NFL and sod1 protein. Along with this, there is a ‘hint’ of disease slow down with early diagnosed and/or slower progressors.

What is evident, as a stand out to me, is that Biogen will have a lot of long term data from the Open label extension (OLE) that will stretch out to years. The data so far released is 80 weeks, but there appears to be much more in the pipeline.

We await further analysis from Biogen. As mentioned, I am confident they will not be hesitant on bringing further conclusions to our community.

On the subject of lessons learned from this trial and others

Learning from every trial is key. This hasn’t been a strong characteristic over the years in mnd trials for a number of reasons. These include the state of the art of the science and, of course, the brutal lack of funding in the UK. We certainly may have not have not learnt enough as we need to from failures.

Lessons can be broadly categorised in 2 areas; first those from the trial protocols and secondly those of disease pathology/learning.

The former is easier to comment on now, and as a person living with mnd I feel I would like to air my views.

Our scientific community is caring, dedicated and has listened to our patient community. In some cases the listening has perhaps even been thrust upon them. For example, there has been a constant, clearly understandable, very vocal pressure to reduce placebo involvement because of our severe prognosis. In my opinion, however, 6 months is just too short given the current status of biomarker technology, disease precession etc. If the tofersen trial, for example, had run for 12 months we might have been in a very different position earlier, if not only for regulatory rules.

It’s perhaps a 'careful what you wish for moment’. I think the pressure to reduce, although well intentioned, might have backfired and may just have delayed the arrival of a potential treatment. We are very fortunate that Biogen are investing heavily in a funded Expanded Access Program which could provide early access to SOD1 patients.

Secondly on the general subject of lessons learned in any trial with regards to disease learning etc. Data and data gathering is vital. Part of the #united2endmnd proposal to the UK government for the MND Translational Research Institute, is to build infrastructure that would enable greater learning from every trial as well as an on-demand trial platform. Currently Pharma have to build, rip down and rebuild trial platforms for every trial. This is clearly not efficient.

Finally, the current Biogen MND/ALS pipeline can be seen here.

https://www.biogen.com/en_us/pipeline.html

Lee (aka Onein300)