Trials to start of daily pill for MND that could reverse condition

Kaz

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Human trials will begin next month of a new treatment scientists believe may reverse devastating motor neurone disease. It may also hold hope for Alzheimer's.


Australian motor neurone disease patients will be the first in the world to trial a “novel” treatment that scientists hope could not only slow — but actually reverse — the condition.

If successful, the US biotech firm Spinogenix drug would be the first regenerative treatment for the devastating and rapid neurodegenerative disease — which kills two Australians a day — and may also help fight Alzheimer's and schizophrenia.

The first MND human trial will begin next month, thanks to a successful safety trial in Melbourne, and patients could take the daily pill for up to a year after the regulator approved access for 11 months post-trial even if patients received a placebo initially.

The trial is open to 24 MND patients with Lou Gehrig’s disease (ALS).

The drug was effective in animal trials and, while these do no guarantee success in people, Spinogenix founder Dr Stella Sarraf told the Herald Sun she was “super excited”.


She said their first-of-a-kind drug targeted the connections between our neurons in a bid to regrow those lost to MND, Alzheimer's or schizophrenia.

“[It] restores synapses, these are connections between the neurons that enable communication,” she said.

“We believe this will show benefits in motor, respiratory and cognition; so walk, breathe and remember.

“We really hope this improves the lives of Australian patients.”

She said MND was complex, so needed multiple types of treatments, but it would mean everything if their drug, SPG302, worked.

“I know what it’s like to fight the battle (of a neurodegenerative disease),” she said.
“I’ve lost both parents.

“If I make an impact with a drug that helps people win that battle … it’s my dream.

“We’re hoping our drug will not only fight the disease, but destroy it.”

She said groups like MND Australia and Neale Daniher’s Fight MND had been instrumental in raising awareness, without which Spinogenix would not be to “move as quickly as we are” in Australia.

Melbourne dad-of-two Todd Johnson, 48, said living with an incurable disease like MND was just as much of a mental fight as a physical one.
He said it was a “battle trying to stay positive” but clinical trials “give you hope” and “helps you feel part of something … a way to contribute to finding that cure”.

“The psychological impact of … who knows maybe this could be the drug,” he said.


MND Australia research executive director Dr Gethin Thomas said while this was early research, every new trial helped locals access new therapies and it would be “brilliant” if it progressed to stage two.

“We need as many different therapies as possible … an arsenal of weapons,” he said.

Any eligible Australian patient can apply for the trial — which will run in three hospitals in Sydney, Brisbane and Adelaide — at clinicaltrials.gov.


 

Mr 95%

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Human trials will begin next month of a new treatment scientists believe may reverse devastating motor neurone disease. It may also hold hope for Alzheimer's.


Australian motor neurone disease patients will be the first in the world to trial a “novel” treatment that scientists hope could not only slow — but actually reverse — the condition.

If successful, the US biotech firm Spinogenix drug would be the first regenerative treatment for the devastating and rapid neurodegenerative disease — which kills two Australians a day — and may also help fight Alzheimer's and schizophrenia.

The first MND human trial will begin next month, thanks to a successful safety trial in Melbourne, and patients could take the daily pill for up to a year after the regulator approved access for 11 months post-trial even if patients received a placebo initially.

The trial is open to 24 MND patients with Lou Gehrig’s disease (ALS).

The drug was effective in animal trials and, while these do no guarantee success in people, Spinogenix founder Dr Stella Sarraf told the Herald Sun she was “super excited”.


She said their first-of-a-kind drug targeted the connections between our neurons in a bid to regrow those lost to MND, Alzheimer's or schizophrenia.

“[It] restores synapses, these are connections between the neurons that enable communication,” she said.

“We believe this will show benefits in motor, respiratory and cognition; so walk, breathe and remember.

“We really hope this improves the lives of Australian patients.”

She said MND was complex, so needed multiple types of treatments, but it would mean everything if their drug, SPG302, worked.

“I know what it’s like to fight the battle (of a neurodegenerative disease),” she said.
“I’ve lost both parents.

“If I make an impact with a drug that helps people win that battle … it’s my dream.

“We’re hoping our drug will not only fight the disease, but destroy it.”

She said groups like MND Australia and Neale Daniher’s Fight MND had been instrumental in raising awareness, without which Spinogenix would not be to “move as quickly as we are” in Australia.

Melbourne dad-of-two Todd Johnson, 48, said living with an incurable disease like MND was just as much of a mental fight as a physical one.
He said it was a “battle trying to stay positive” but clinical trials “give you hope” and “helps you feel part of something … a way to contribute to finding that cure”.

“The psychological impact of … who knows maybe this could be the drug,” he said.


MND Australia research executive director Dr Gethin Thomas said while this was early research, every new trial helped locals access new therapies and it would be “brilliant” if it progressed to stage two.

“We need as many different therapies as possible … an arsenal of weapons,” he said.

Any eligible Australian patient can apply for the trial — which will run in three hospitals in Sydney, Brisbane and Adelaide — at clinicaltrials.gov.


We are living in a grand age. Such people like Dr Stella Sarraf, and collaborators and such visionaries, always remind me of the words of Robert F. Kennedy,

”Some men see things as they are and ask, Why?
I dream of things that never were and ask, Why not?”

A cure for MND, and other diseases mentioned, will come one day..Why not now?!
 

Memberberries

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I'm pretty sure my mum has MND and is in the early stages?

She no longer understands simple explanations.
Even if you drew what you were explaining on a board she still wouldn't get it.

It's strange how it works.
She seems fine throughout the day?
Just early morning and late evening she seems disorientated.

If you call her out on it she gets very upset and defensive!
 

Robbothedestroyer

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Wish the trial every success.
We have some extraordinary and talented medical minds in our country they just need the appropriate funding to allow them to achieve amazing things.
Shame the government doesn’t offer more financial support. Can find hundreds of millions $ for a referendum no one wanted. Sad reality funding these initiatives gets people out of hospitals and care facilities with sizeable healthcare savings and benefits but doesn’t win enough votes.
 

Kaz

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Who knows?
She needs to get diagnosed by a competent doctor and not a stupid quack who will tell her what she wants to hear!
Can you take her to a Dr, but not tell her what it is for.
 

Bob dog

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Finally some hope for the Alzheimer outbreak that has dragged on for ten years.
Australia leads the world.
 

Alan79

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Who knows?
She needs to get diagnosed by a competent doctor and not a stupid quack who will tell her what she wants to hear!
You might be able to get a home consultation. I think with these type of tests it might be a repeat thing to establish if they've regressed.

I can't help with more advice. But I've spoken with a few residents within the last three years who were worried about seeing friends there lose their memory who have regressed to the point they don't know me or where they are. If you are her primary carer, might be a good idea to get the tests done to plan ahead. It can seem like a sudden thing if she forgets who her loved ones are (might happen some days not others). But having a plan to deal with it if it happens is helpful.
 

Memberberries

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You might be able to get a home consultation. I think with these type of tests it might be a repeat thing to establish if they've regressed.

I can't help with more advice. But I've spoken with a few residents within the last three years who were worried about seeing friends there lose their memory who have regressed to the point they don't know me or where they are. If you are her primary carer, might be a good idea to get the tests done to plan ahead. It can seem like a sudden thing if she forgets who her loved ones are (might happen some days not others). But having a plan to deal with it if it happens is helpful.
That's a good idea.
I'll arrange a home visit.
 

Trinfly

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I'm pretty sure my mum has MND and is in the early stages?

She no longer understands simple explanations.
Even if you drew what you were explaining on a board she still wouldn't get it.

It's strange how it works.
She seems fine throughout the day?
Just early morning and late evening she seems disorientated.

If you call her out on it she gets very upset and defensive!
Would be constant worry for you sorry to hear that bro,
 

The DoggFather

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I'm pretty sure my mum has MND and is in the early stages?

She no longer understands simple explanations.
Even if you drew what you were explaining on a board she still wouldn't get it.

It's strange how it works.
She seems fine throughout the day?
Just early morning and late evening she seems disorientated.

If you call her out on it she gets very upset and defensive!
I wish and pray for nothing but the best her and your family my bro
 

Mitch Connor

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I'm pretty sure my mum has MND and is in the early stages?

She no longer understands simple explanations.
Even if you drew what you were explaining on a board she still wouldn't get it.

It's strange how it works.
She seems fine throughout the day?
Just early morning and late evening she seems disorientated.

If you call her out on it she gets very upset and defensive!
That’s dementia mate. Not MND.
 
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