ALSUntangled – untangling the mysteries of alternative and off-label treatments

There are literally thousands of websites on the internet that make claims about amazing alternative or off-label treatments (AOTs) and even cures for MND but with little or no scientific evidence to back these claims up. This presents a real problem for people living with the disease who may want to try them. Are they safe? Will they help? Or are they going to cause more problems than they solve?

Bedlack, Dr Rick

 

In 2009 Dr Richard Bedlack, Professor of Neurology at Duke University in the USA, founded ALSUntangled to develop a system of review for some of these treatments using the available evidence, to make it easier for people with MND and their families to make more informed decisions about them.Read More »

MND Engage – collaboration, engagement and communication

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Martina, Lydia, Mandy, Nick and Kirsten from the MND Association

MND Engage was a one-day collaborative event, bringing together MND researchers and people affected by MND. The inaugural event was held at the Francis Crick Institute in London on 23 July. The event, organised by researchers Jasmine Harley, Giulia Tyzack and Helen Divine, and supported by the MND Association’s Research Development and Communications teams, brought together MND researchers from several UK laboratories working on MND in order to explore ways in which public engagement in MND research could be improved.

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Introducing Riddhi – Research Information Co-ordinator

Hi all! I’m Riddhi and I’ve just joined the MND Association as a ‘Research Information Co-ordinator’ in the Research Development Team 😊

Riddhi
Riddhi Naik

You’ll probably see me across the blog, from writing posts on exciting updates in MND research to replying to your comments, so I thought it would be nice to introduce myself and my background (including how I used ladybirds in neurodegenerative diseases). I’m thrilled to be part of the organisation that continuously strives towards helping people living with and affected by MND through funding research, campaigning and providing care and support. I look forward to exploring all the studies that contribute to finding an effective treatment or cure and then communicating these to you.Read More »

New gene therapy targeting C9orf72-ALS begins Phase 1 clinical trial in the UK

This article was written by Dr Keith Mayl and Dr Ahmad Al Khleifat of King’s College London.

Researchers at King’s College Hospital, led by Professor Christopher Shaw, have embarked on the first gene therapy clinical trial for patients affected by a specific genetic form of ALS, the most common type of MND.

ALS is a progressive disease in which the nerves controlling muscle movement, known as motor neurons, degenerate resulting in muscle wasting and weakness. In about 10% of people the cause is a mutation in the C9orf72 gene. This mutation results in the formation of toxic products which are harmful to motor neurons. People with the mutation typically develop symptoms in their 50s, starting with speech and swallowing problems, followed by weakness of the arms, legs and breathing. It is also linked to problems with language and behaviour and is the most common genetic cause of frontotemporal dementia.Read More »

Kennedy’s Disease vs ALS: How muscle patterns can aid diagnosis and perform as a novel biomarker

Researchers from University College London led by Dr Pietro Fratta and Dr John Thornton found that muscle imaging can help distinguish Amyotrophic Lateral Sclerosis (ALS) from Kennedy’s Disease based on the way specific muscle groups deteriorate in each condition. The method can also help assess the severity of the disease.

ALS is a rapidly progressing condition which affects both upper and lower motor neurones, leading to inability to move limbs and failure of breathing muscles at the later stages of the disease. The complex cause of this condition is not yet fully understood but is thought to be a combination of genetic and environmental factors. Kennedy’s Disease on the other hand is much slower in progression and severity and is primarily caused by a gene mutation.

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Picture of face and eye

Do the eyes have it? Could Resistant Nerves See Our Way to a Treatment?

The Science Show on ABC Radio National in Australia, features an interview with Professor Justin Yerbury on Saturday 17th August.

The article containing the interview titled ‘Resistant nerves could lead to treatment for neurodegenerative disease‘ is a fascinating insight into Prof Yerbury’s work on the  delicate balance of proteins in solution within our nerves and how this is interrupted in MND.

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Focusing on mitochondria – a potential target for early MND treatment?

Dr Arpan Mehta, one of our Lady Edith Wolfson Clinical Fellows, and his team at the Euan MacDonald Centre at the University of Edinburgh have recently carried out a systematic review and meta-analysis of the pre-clinical literature (studies using animal models) to assess the therapeutic potential of targeting mitochondrial dysfunction in MND, examining if these interventions significantly affect survival in animal models of the disease, and determining the most effective time to begin treatment.

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Could MND be treated by HIV drugs?

Recently published results from the open-label Lighthouse trial investigating safety of the drug Triumeq in people with MND revealed the treatment was safe and ready to progress to a larger Phase 3 clinical trial.

The trial was held in Australia and recruited 40 people with MND who all received the active drug; this was because the aim of the trial was to see whether Triumeq, which is already licensed to treat HIV, has a potential as a treatment for MND.

Why HIV drugs?

One of the possible triggers of MND has been suggested to be a group of ‘fossil’ viruses that, over many millions of years of evolution, have left traces of their DNA within our genome. When activated, these ‘retroviruses’ have the ability to merge into our cells, by copying their DNA into our genome, which leads to incorporation of the two DNAs into one. When the affected cell then creates new proteins, partial copies of the virus are produced with it.

Retroviruses have been linked to MND because of findings of a particular retrovirus, called human endogenous retrovirus (HERV-K), in the brains and motor neurons of people with the disease. Although some studies failed to confirm this finding, researchers deemed this to be a promising area of therapeutic focus.Read More »

How animals are helping to improve our understanding of MND

‘From antibiotics and insulin to blood transfusions and treatments for cancer or HIV, virtually every medical achievement in the past century has depended directly or indirectly on research using animals’ – from the Royal Society’s position statement on the use of animals in research.

We know that talking about using animals in research is an emotive topic. We appreciate that some people will never accept that using animals in research is necessary, and we understand that it is not our place to try and influence anyone’s opinion on the use of animals in research. The purpose of this blog is to explore how using animal models of MND can further our understanding of this devastating disease, and how animals make it possible for potential new treatments for the disease to move forward into clinical trials in people.Read More »

FaTHoM 2: UK-leading MND clinicians on inherited MND

After its successful premiere in 2017, the University of Oxford organised another meeting of people affected by inherited MND, called ‘Families for the Treatment of Hereditary MND (FaTHoM)’. This turned out to be yet another excellent day where MND clinicians-researchers presented on topics such as genetics of MND, genetic testing and gene therapies. Below you can find out more about what was presented on the day and links to the videos of recorded talks.

Understanding familial MND

Introducing the rationale of the meeting, Prof Martin Turner set the scene by explaining the great difficulty in understanding the disease due to its many possible causes. Being such long cells, many things can go wrong in the motor neurones and in the vast amount of their support cells (such as astrocytes or microglia). But one factor can help us understand the disease better – genes.

Around 5-10% of MND is considered familial. That is, around 1 in 15 people have had someone in their family affected by this disease in the past, making them more likely to develop the disease themselves. Specifically, if we consider the ‘multistep hypothesis’ of MND which assumes that six steps have to happen in our lifetime for the disease to develop, a mistake in a specific gene may reduce the number of the necessary steps to four or even two (read more about the impact of genetic on the multistep hypothesis here).Read More »