Rch4

[nunhead_man]

Good afternoon Doblett

I see you are now back on the forum after being thrown off after you declared information about me that I shared here outside this forum against the rules thereof.

If I find you been sharing any more information about me outside this forum, I will find some way of throwing the book at you.

[noow17]

Hi, Our mum was diagnosed in Nov 2017 and begun using RCH4 in Jan 2018. She hasn't used any other ALS drugs. Her condition has stabilised since commencing RCH4 and there have been no side effects.
We as a family are extremely grateful to the charity that supplies RCH4 free of charge along with ongoing support.
Our hope is that the charity gains the recognition they deserve, to bring this treatment option to the market and help other PALs dealing with this devastating disease.

Below are her latest ALS scores.ALSFRS-R 09-19.png

[Terry]

Hi Noow17,

Pleased to hear that your mum is still getting the drug OK because I was led to believe that Dobett couldn't get it.

Love Terry

[Sportingmac]

Hi all,

Neater Robo-arms are out of stock - sadly for me it will be a 4~5 week wait.

In other news: I have always doubted the veracity of ALSFR-S scales since a final number is used to assess the progress of ALS. I was doodling along with my usual research on ALS/MND when I came across a more detailed variant that breaks the final number down to specific area - respiratory et etc.

https://www.mdcalc.com/revised-amyot...rs-r#use-cases

Wondering if any other on the forum think it more useful to see the breakdown and improve a prognosis - note the original research document is from 1999.

I think it a much better scale than an extract from a spreadsheet..

What does everyone think?

Kind regards

Chas

[Terry]

Hi Chas,

Sorry to hear about the arms being out of stock. There was one advertised but it was adapted for a small person. I doubt that it is still available but I could check if you have short arms.

Love Terry

[Ellie]

Chas, I may have misread your post, but that ALSFRS-R scale is the scale being used, not only by the posters on this thread, but in MND Clinics and by other healthcare professionals too. (ALSFRS-R = ALS Functional Rating Scale Revised)

As someone who has completed many, many ALSFRS-R sheets over the years, my issue with the ALSFRS-R is that it is a subjective, self-reported system, with no scope for nuance in the blunt scoring system.

That said, there has to be some standardised way of, for example, having inclusion/exclusion criteria for clinical trial participation, so the ALSFRS-R is often one of the criteria used for enrolment.

Love Ellie.

[Sportingmac]

Thanks for that Terry. However I am - was - 6ft - and longish arms.

I will make enquiries though about modifications.

Kind regards

Chas

[Sportingmac]

Chas, I may have misread your post, but that ALSFRS-R scale is the scale being used, not only by the posters on this thread, but in MND Clinics and by other healthcare professionals too. (ALSFRS-R = ALS Functional Rating Scale Revised)

As someone who has completed many, many ALSFRS-R sheets over the years, my issue with the ALSFRS-R is that it is a subjective, self-reported system, with no scope for nuance in the blunt scoring system.

That said, there has to be some standardised way of, for example, having inclusion/exclusion criteria for clinical trial participation, so the ALSFRS-R is often one of the criteria used for enrolment.

Love Ellie.

Exactly my views too Ellie. I also checked with my neurologist in Sheffield - they still use the version I tagged.

Kind regards

Chas

[originalthought]

Sportingmac said

I have always doubted the veracity of ALSFR-S scales since a final number is used to assess the progress of ALS. I was doodling along with my usual research on ALS/MND when I came across a more detailed variant that breaks the final number down to specific area - respiratory et etc.

He then correctly directs us to the more modern ALSFRS-R scale

https://www.mdcalc.com/revised-amyot...rs-r#use-cases

which is highly accurate in consideration of the fact that it is the only validated system. There is no validated biomarker ("test") to measure MND.
Basically this is exactly the scale that the RCH4 charity uses for everyone's monthly monitoring report. Actually they have added further datum points to their form which is also dynamic in that it also encompasses predictive analysis etc. which builds over time.

From the `Net
Measuring the progression: The ALSFRS-R score

As the ALSFRS-R score is a subjective observation, it will likely be scored differently by any two different people. It is said that the PALS must be scored by a specialist medical professional - but this is not the case.
See the evaluation study in the accuracy of on-line self reporting https://www.tandfonline.com/doi/full...68.2011.633268 (Journal Amyotrophic Lateral Sclerosis Volume 13, 2012 - Issue 2)


In fact the precise ALSFRS-R score is not particularly relevant. What matters is the CHANGE in the score over exactly the same time periods.
Provided that the SAME person (e.g., the patient or a care giver) does the scoring every month over a long time, the submitted change in data is very accurate. Further, scoring by the same person over extended time ensures elimination of patient or carer bias as the submitted data would rapidly exceed the envelope of credibility.

Again from the `Net, the patients own reporting is very consistent. In this case for the RCH4 drug which was given to everyone for free but the charity has no money now.
als-2-year-117 chart.jpg

The ALSFRS-R score does not reflect loss of neurons or other subtle changes, but someone rightly said that if the ALSFRS-R score never changed then we would all die from extreme old age or something else. Speaking as a medical doctor, I must accept that is true.

[Sportingmac]

Hi Originalthought,

Thank you for point. However - it remains a subjective method.

Also- the results depend largely on the type of motor neurone disease - ALS versus PLS versus Bulbar onset versus limb onset. Each would have a different trajectory.

There has been research into objective testing. The research used two different methodologies: MRI scans and EMG results. The EMG determines the number of neurones used to move a selected muscle and the size of the neurones . Using these two objective tests it was shown how much degradation was occurring in the patient.

I was part of that research.

Harvard University picked up the ongoing research - sadly I have yet to see its outcome - I declined my participation through my lack of mobility to get to Sheffield. Ongoing research for me.

Hope that helps explain my position.

Meantime I wish you all the best in your research.

Kind regards

Chas

[originalthought]

Hi Chas,
Thanks for your best wishes for my research. Sorry there is a misunderstanding. I am not smart enough to be a scientist or researcher! Just a retired GP dealing with this awful condition.

My “research” only went so far as to learn that Riluzole did nothing to slow the progression and RCH4 definitely does in my case. My concern is how long the RCH4 charity, who supply it for free, will last as they have no funding.
We all know that MND is a most heterogeneous condition so outcomes will vary a lot. The charity http://www.rch4.com say it is ineffective in 16% of the treated population. However I came on this interesting table from PLM and also see that it has the highest votes on ALS Untangled (our American cousins call it ALS).
PLM Efficacy Table.png

To address your point about the ALSFRS-R score. You are absolutely right that it is subjective, but it has been validated as the gold standard for measuring progression in the absence of any biomarker being available. As I posted before, what the actual score is for each patient is not in fact relevant. What matters is the CHANGE IN THE SCORE OVER TIME.

Even if an accurate objective protocol or biomarker were developed tomorrow, it would still be academic as there is no “cure”. All an accurate biomarker would do is enable earlier definite diagnosis. Sadly, that’s little help to us.

[noow17]

Oct 2019.png

Mums latest ALSFRS-R chart. She's still doing really well.

[Terry]

Hi all,

Neater Robo-arms are out of stock - sadly for me it will be a 4~5 week wait.

Chas

Hi Chas;

Any new on the arms.

Love Terry

[Terry]

Hi Noow17,

Pleased to hear that your mum is still getting the drug OK.

I was led to believe that Dobett couldn't get it and that was the reason for his anger.

Love Terry

[Sportingmac]

Hi Chas;

Any new on the arms.

Love Terry

Ongoing saga with NHS. NHS say NO. MNDA need reason for no before offering assistance. Have written to CCG asking for their help. Also have investigation by Neurologist in Rehab Team into reasons too.

Meantime I now have a GridPad - Yeahhhhh Robo-Arms would be good to reach the screen. Eyegaze if no robo-arms.