New promising trial started

[Marc from London]

Hello

maybe this has been shared already.

[shelly21]

Yes I read this. Think its 50 Oxford patients to do pre trials then if that goes well to begin a larger trial.

I'm wondering if it might be added to the mnd smart trial.

[Cinderella]

Thanks Marc from London for posting that info. I have just requested the drug from the consultant as if it is already available on prescription then they need to get involved, I have already been prescribed finesteride in the past due to alopecia - but not sure if this is the same drug although it reads the same. When there is so little to help us then it's time to make a noise.

[billy106]

Cindrella your pills for alopecia .helping MND . Eh give me a break mate.

[Ellie]

Some examples of repurposed drugs being successfully used to also treat unreleated illnesses/conditions, including for alopecia.

Drug name	Original indication	Disease name
Aspirin	Analgesia	Colorectal cancer
Azathioprine	Rheumatoid arthritis	Renal transplant
Cycloserine	Urinary tract infection	Tuberculosis
Duloxetine	Depression	Stress urinary incontinence
Galantamine	Polio and paralysis	Alzheimer
Gemcitabine	Antiviral	Cancer
Finasteride	Benign prostatic hyperplasia	Hair loss
Imatinib	Chronic myelogenous leukaemia	Gastrointestinal tumours
Topiramate	Epilepsy	Obesity
Minoxidil	Hypertension	Hair loss
Phentolamine	Dermal necrosis (hypertension)	Autism
Raloxifene	Osteoporosis	Breast cancer
Sildenafil	Angina	Erectile dysfunction
Sunitinib	GIST, renal cell carcinoma	Pancreatic tumors
Thalidomide	Nausea	Leprosy and multiple myeloma
Zidovudine	Cancer	AIDS

[Banned]

How do plateaux of symptoms work Ellie?

[Banned]

While there have been rare occasions where treatments have been repurposed, it seems highly unlikely that it is likely to happen for MND.

For starters, the drug must penetrate the blood brain barrier to access the neurones. Very few are able to do this.

In many cases we are told "You most likely have MND". "What MND??" should be the next question.

Unless you have a known genetic diagnosis, the neurologist will have no idea.

And so without knowing what even which MND you have in the first instance, how on earth can this be anything other than a cruel mind game or CRAP as Billy politely puts it.

Elsie, you owe Billy an apology.

[Ellie]

For starters, the drug must penetrate the blood brain barrier to access the neurones. Very few are able to do this

Terazosin, the drug referenced in the topic of this thread, does of course cross the blood-brain barrier, as do Memantine and Trazodone, the two MND Smart trial drugs ridiculed on the thread created by the user cited by you in your post above.

I don’t know if you have any interest in how Terazosin works but I’m sure other members have, this is an albeit science-speak explanation and explains why repurposing this known, safe, approved drug is causing people to get excited and to hope …

"Terazosin treatment extended survival, improved motor phenotypes and increased motor neuron number in Thy1-hTDP-43 mice. In ESC-derived motor neurons expressing TDP-43^M337V, terazosin protected against oxidative stress-induced cell death and increased basal glycolysis rates, while rescuing stress granule assembly.
Our data demonstrate that terazosin protects motor neurons via multiple pathways, including upregulating glycolysis and rescuing stress granule formation."

[Banned]

I don't even know what kind of MND I have Elsie. The best I know is that it MAY be caused by a faulty gene DCTN1. My lead neurologist emphasised that it may only be the cause and I should not take it as the root cause. This will be the same for the vast majority of us.

In the early stages of my misdiagnosis I was provided with many 'speculative' drugs and they all caused me problems, so much so that I would not take any more. Any drug introduces toxicity into the system.

Further, I have not been tested for TDP-43^M337V and so I would not know if that is a problem.

I do share the utter frustration of Billy however in the glacial progress of the diagnosis process let alone any treatment. It is a cruel mind game that is being played on us for the lack of any meaningful diagnosis or treatment. Your apology to Billy is awaited.

[colinmac]

I discussed this with my neurologist a few weeks ago and subsequently with my GP who was happy to switch me from my normal antihypertensive to this drug. Although it is rarely prescribed for high blood pressure he agreed with me that I might as well be on this as it showed some promise over a broad range of motoneuron disease types. I just felt that I had little to lose as to be honest, I'd lost interest in taking the previous blood pressure medication due to the nature of this illness. The trouble is, I'm not sure how long I should go on with it before deciding if there is any improvement and if there is an improvement how will I know that it's due to this drug of what would have happened anyway?

[Ellie]

A warm welcome to the forum Colin, sorry you've had to join our motley crew...

What an enlightened GP you have.

... if there is an improvement how will I know that it's due to this drug of what would have happened anyway?

As a rule, MND symptoms don't 'improve' so, if you do improve, then you've won more than the lottery.

If on the other hand, Terazosin 'slows down your progression', then yes, you won't know if your progression would have been slower, faster or the same, had you not taken the drug.

It will take the clinical trials, plus longer term real world evidence, to see what effect, if any, Terazosin has on the general ALS population or subsets of.

Best wishes to you.

Love Ellie xx

[Banned]

Do you know what kind of MND you have Colin?

[Aadam]

colinmac Would you be able to update us with any news since taking Terazosin please?

[Midlander]

Hi All, I'm 69 and was diagnosed with MND in September - though, in my amateur opinion, it was pretty obvious that I'd got it in July. This leads me to a few observations about how our treatment could/must be improved. You'll have to forgive my directness.

1) In some other fields, clinicians believe that even before a formal diagnosis, if you suspect a patient has a disease (in the quoted case it was MS) it can be hugely beneficial and "could spare them a lifetime of debilitating symptoms" to prescribe the drugs without delay. In my case, my original consultant would not confirm his diagnosis until after my second EMG test. Forgive my directness, but I was effectively left to die for two months without medication so he could 'dot the i's and cross the t's'. In my opinion, consultants should be more instinctive and dynamic - prescribing riluzole, at least, as soon as MND is suspected. Maybe NHS rules forbid this? Change the rules.

2) My first two consultants, one private and one NHS, were eminent, caring, kindly medics, but their strategy was simply to reassure me of the ways that medical devices could ease my downward spiral. That's it.
I was then moved for geographical reasons to a new consultant who is active in research and trials. We immediately discussed ways of trying possible new treatments (not, of course, promising miracles). But instead of trying to help me die, he's trying to help me live. I think this mindset is absolutely crucial and this cultural change needs to be implemented throughout MND treatment.

3) There are nearly 1,000 trials worldwide of possible treatments for MND, (and several in the UK). It's impossible to get your hands on CuATSM (I've tried) but many drugs are being repurposed and already in use and proven to be safe - Terazosin is one of them, commonly used for hypertension or Parkinson's.
I asked my earlier consultant to get me on Terazosin fast, and he said "I can't prescribe it as it's not approved for MND in the UK.". It took me to say "Well it IS approved for hypertension , and I've got that - so now can I get onto Terazosin?! He then contacted my GP, and I lined up our local pharmacist to get the tablets in, ready for the prescription from my GP. I've been on Terazosin 10mg for two months now. I also take TUDCA (which is being trialled in Europe and UK - and available on Amazon) and Theracurmin (trialled in the US and also available on Amazon). No sign yet that any of them work - but here's the point: if Riluzole only slows down the disease, what have we got to lose by trying safe, cheap, available drugs that some eminent clinicians believe might well work? I fervently believe that this, too is a culture change which we need throughout the NHS.

4) I'm taking these drugs BUT I DON'T KNOW WHAT I'M SUFFERING FROM. Yes, it's MND, but is which, of the many kinds, is it? I might be wasting my time with Terazosin. I might be missing purposeful and narrowly targeted drugs. In September my earlier consultant sent my blood off for genetic analysis, but said it might take 6 - 9 months before any result as the NHS had replaced multiple testing centres with a single hub, for efficiency......and it was causing huge delays (!). My new consultant asked me to write to Prof Dame Sue Hill at NHS Genomics about this. Despite an automated answer promising a response within three days, I never got a reply. This ain't a rheumatic hip. You and I can't wait 9 months to find out exactly what we've got. I have no problem with the expertise and good intentions of NHS Genomics, but it's unforgiveable that they fail on service delivery, and MND patients will die waiting to hear what type they've got. Amazon can do it - why not NHS Genomics? I've written to my MP, Can you do the same?

I recognise I'm lucky with my current consultant, GP, pharmacist and MP, but if you agree with the points I've made and feel well enough, please do what you can to make these changes happen.

[Midlander]

In short,

Sorry guys, it occurred to me that my first post was very verbose, so I'm just going to summarise what I believe we need from the Government and NHS - just my opinion, of course.

# Prescribe Riluzole AS SOON AS MND IS EVEN SUSPECTED
# Provide a genetic testing service which can DELIVER GENETIC RESULTS WITHIN 6 WEEKS
# Shift the clinical mindset from working to ease our death to WORKING TO HELP US LIVE
# Find ways for patients to quickly TRY THE MOST PROMISING TRIALLED BUT UNPROVEN DRUGS from those known to be safe, affordable and available.

This does not imply ingratitude to our eminent NHS MND teams, nor disrespect to NHS heads and politicians, but from our own unique viewpoint, I think that those of us who believe this have a right to politely lobby for this.