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 »

Lighthouse Project shines a beacon on HERVs and their role in ALS

There is recent evidence to suggest that Human Endogenous Retroviruses (HERVs) may be involved in amyotrophic lateral sclerosis (ALS). HERV-K has been directly linked to motor neurone damage and has been found in the brain tissue of patients with ALS.

The MND Association recently awarded a small grant to fund part of the ‘Lighthouse Project’ which is investigating the safety and any beneficial effects of an antiretroviral drug on ALS symptoms.Read More »

Do retroviruses contribute to the common, sporadic form of MND?

New research from scientists at the American National Institute of Neurological Disorders and Stroke in Bethesda suggest that they might. In a research paper published in Science Translational Medicine yesterday, Li, Nath and colleagues proposed that sporadic MND may be linked to an endogenous retrovirus called ‘HERV-K’. So they conducted a series of experiments to investigate their ideas further.

What are endogenous retroviruses?
These are viruses that are our body’s equivalent of fossil – a left over from our evolution many thousands of years ago. Everyone has them but they are normally in an inactive state.

They are a bit like a family heirloom, lets say a vase. You might walk past the vase every day without really noticing it until one day the cat knocks it off and it smashes onto the floor in front of you.

What did the researchers do?
First, they compared brain tissue of 10 people who died from the sporadic form of MND to brain tissue of 10 people who died from Alzheimer’s Disease. They found proteins made by the virus in MND brain tissue but not in Alzheimer’s Disease brain tissue. Next, studying one of these proteins (called ‘env’) in more detail, they found it was toxic to motor neurones.

Li and colleagues then took a step back and asked ‘what triggered HERV-K to become active in the first place?’. (In other words, going back to my analogy, what caused the vase to fall on to the floor?). They found that the trigger was activation by a protein called ‘TDP-43’ – and this protein is already linked MND.

So what does this really mean?
In a comment article giving a wider perspective on the research study, Professors Bob Brown and MND Association grantee Ammar Al-Chalabi concluded:

The exciting observations of Li, Nath and colleagues will provoke further follow up studies that will illuminate the interplay between the biology of endogenous retroviruses and seemingly impenetrable neurodegenerative disorders like ALS”.

So while this study in itself might not give us the answer, its an exciting step forward in understanding the most common form of MND, that other researchers around the world will build on.

Li et al Human endogenous retrovirus-K contributes to motor neurone disease Sci Transl Med 7 307ra153 (2015)

Brown and Al-Chalabi Endogeneous retroviruses in ALS: a reawakening? Sci Transl Med 7 307fs40 (2015)

‘Dormant viruses may cause MND when awoken’ article in The Guardian