Still No Treatment or Future Cures

[Graham]

While cancer is very obvious in many cases, the better and modern way to treat cancer is to understand how the cancer forms using genetics, rather than poisoning the entire body and hoping that the cancer dies first.

The 100,000 genome project that I was allowed to join was primarily about studying the genetics of cancer. However, it also was able to identify a gene in me that probably caused my MND.

[Olivia H]

I wonder, do people have to get gene tested to benefit from cancer treatments? I'm not party to the inner workings of what research is going on, and why only into some forms of mnd. If this is the hereditary form, then that won't be most of us. Everything about the research, how it will translate into actual treatments/therapies and techniques, and that it should be prescribed to pretty much everyone with any mnd, as soon as it is approved, regardless of whether they do or don't submit genetic info, should be a given, open and transparent! There are thousands of other serious illnesses we treat daily without making sufferers feel they are asking for some sort of special dispensation to be informed and treated.

[Graham]

Hi Guys,

I badly wanted Brainstorm to succeed despite my reservations about their approach. The main point was that the markets thought that they had failed and Brainstorm lost over $100M of equity. Had they had a successful trial, Brainstorm would be worth well over $1Bn.

What I find baffling and telling is that the NHS still have difficulty in telling us what sort of MND we have. From a cancer analogy, do you have lung cancer or brain cancer??

There are many ways that our neurones fail and the b'stards are molecular sized and deep down in us.

[billy106]

Its a bit deflating when there is a flurry of excitement on the research etc coming out of Edinburgh. if you go back on early posts in this forum , you will see the same excitement but as usual nothing sees the light of day. again still only Riluzol out there, and an ever greater demand for grants . or as I say , career furthering grants and for back slapping events. Last 40 yrs Millions of pounds donated and still not as much as an aspirin. Do you get the impression there must be another road. ie getting into bed with big Pharma. I await the replies from the usual bunch , we must keep trying this trusted road zzzzz

[nunhead_man]

Good morning all

With Brainstorm failing last week

Mike Henson in the US disagrees with you – the USA folk have picked up the improvements in the trial created in the later stages of MND as something that should be promoted to help people with MND survive longer even if it is not creating a complete reversal and are trying to get the FDA to approve it as a treatment.

I could post some of the 1 hour videos that Mike produces to get his group to badger USA legislators to get this stuff done if anybody wants to see them.

[Onein300]

Hi so can i please ask, why are researchers researching for a treatment for inherited MND when that is only 10% of all MND sufferers, surely they should be trying to find a treatment for the majority 90%??

They are. However, as this disease is so varied we have to target. The best target is those with a distinct cause and where we can attack the fault.

These patients provide the best test for any precision treatments because we can both measure any effects and because sadly we can predict their disease progress more accurately.

Unless we start small we will never make any headway.

The most likely scenario is that we develop a slowing therapy for certain genetic forms. This will probably happen in the near future and gene therapy is the most advanced treatment of any trials globally.

Once we find really a effective treatment for 1 form, we will have proven MND is treatable. This will be a game changing moment and research money will flood in from industry for the wider forms.

If we scattergun now we will make no progress ever. History is proof of that.

[Wooley]

Hi so can i please ask, why are researchers researching for a treatment for inherited MND when that is only 10% of all MND sufferers, surely they should be trying to find a treatment for the majority 90%??

[Onein300]

Onein300 I hear what your saying and understand by the sounds of it you are quite clued up on things. But the basic mechanics of the way things are being done in finding any treatment for MND are tried and trusted. That is the problem , there must be something or new avenues to take.
Here is an example why we are no further forward. I have been trying to get Sativex for years now . through GPs , Consultants, and Health boards. and the weak and pathetic reason I keep getting is , Its not been tested on MND. but keep taking Gabapentin , Diazepan, Brufen etc. So what chance does any treatment have if these walls are still there.

Being frank, you are talking about, at best symptom dampening drugs. Sativex is hardly a game changing treatment. Don’t waste your time.

With regards to “disease altering drugs” the trial for Tofersen for SOD1 genetic linked MND is the sort of trial that is most promising right now. It’s targeted and measurable unlike many other current drugs.

Yes it’s not the majority of MND but if we can break one form, the dam will break for both scientific but more importantly financial reasons. We would have shown that MND is treatable and industry money will flood into the area.

Keep optimistic.

[billy106]

Onein300 I hear what your saying and understand by the sounds of it you are quite clued up on things. But the basic mechanics of the way things are being done in finding any treatment for MND are tried and trusted. That is the problem , there must be something or new avenues to take.
Here is an example why we are no further forward. I have been trying to get Sativex for years now . through GPs , Consultants, and Health boards. and the weak and pathetic reason I keep getting is , Its not been tested on MND. but keep taking Gabapentin , Diazepan, Brufen etc. So what chance does any treatment have if these walls are still there.

[Onein300]

Hi Lee,

I can agree with the notion that the researchers know more than ever before and should be able to offer a treatment "soon", after the success of the vaccine rollout. It is all protein engineering and the fastest supercomputers are able to model our neurones failing, in the various ways they do. So all I am asking is: Why is it taking so long?

The production of a vaccine is easy compared to a systemic, self perpetuating central nervous system disease.

We have a good idea of some of the processes involved but not how they contribute to neuronal death in mnd. Combine this with the lack of of biomarkers and we have a perfect storm.

Computer power helps with models we humans have come up with, but contrary to public belief a computer can only speed up Hunan developed models.

I give you an example.

Why was the c9orf72 gene causation found 25 years after sod1 and yet it affects far more people than sod1?

Its complicated, but it took a logical leap of mathematics and more so an innovative view of genetics to come up with the hypothesis that was then modelled and then proven by computer analysis.

We are very close, but we need more investment to push over the line. What the uk researchers have achieved on barely a 1/10 of the usa investment is staggering.

We also probably need to attack the disease pre-symptoms, rather like we do with statins for heart disease.

[billy106]

Why is 80% of any research grant given usually to cover Salaries and expenses. It goes back to what everybody has been saying. badly Audited funding for freeloaders, career furthering and back slapping events. This must have an impact on the reason MND is no further forward in any treatment considering the millions if not billions poured into it over the years. I wish the way big Pharma and the Govt got around to fast track a vaccine for covid , could be done for MND. But the impression is the spongers would be against that . No more money .

[Graham]

Hi Lee,

I can agree with the notion that the researchers know more than ever before and should be able to offer a treatment "soon", after the success of the vaccine rollout. It is all protein engineering and the fastest supercomputers are able to model our neurones failing, in the various ways they do. So all I am asking is: Why is it taking so long?

[Onein300]

If there is no focus on expediting the best treatments that are currently in trial, then it is the same old, same old, of back slapping and high-fives with no end result.

This is a brand new approach centred around a more efficient trials platform and a conveyor belt of new drug discovery. Here in the UK we have world leading trial capabilities and just need the right funding. If we can get the ask for the required initiative it would represent a tripling of yearly government funding! This would in turn attract pharma investment.

With regards to expediting current promising drugs in trials. By definition they have to run full length and above all show proven, data driven and targeted effectiveness. Nothing can speed up those trials in action aside a time machine.

Advocacy for any drug is only truly worthwhile, and efficient, once shown scientifically to be effective and not by anecdotal stories. Sadly we keep seeing such pressures. I am not aware of any drugs that have yet shown such efficacy. There will be, but we can’t say when yet.

We are making genuine progress now, at a pace not seen before.

[Graham]

If there is no focus on expediting the best treatments that are currently in trial, then it is the same old, same old, of back slapping and high-fives with no end result.

[billy106]

.MND Research liked
MND Association
@mndassoc
Launching soon Microscope #United2EndMND.

This will bring together people with MND, researchers and charities. We're asking the Government for £50 million over 5 years to fund a virtual centre of excellence for MND research.

This is going in the right direction at last.....