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NZ clinical trial for symptomatic treatment

New Zealand may be a tiny country, but when it comes to Huntington’s disease research we are a big force for progress. The main driving forces in HD research at the moment are 1) understanding the disease, and 2) using that understanding to try to treat the disease.

The main challenge standing in our way is the brain itself; it is incredibly complex and is affected in a number of different ways by Huntington’s disease. In order to treat a symptom you have to know which changes to the brain are causing that symptom, and how you could go about returning it to its normal way of behaving. It’s not a straight-forward process, and can take a frustrating amount of time to make progress: but progress is being made. We are getting closer.

One of the researchers currently working in New Zealand to treat Huntington’s disease is Dr Ailsa McGregor. Ailsa and her team are currently testing a drug that may be able to ease the symptoms of Huntington’s disease in the early stages. I say ‘may’ because this possible treatment is still very new, and while it looks very promising it’s important to remember that science takes time and things don’t always go as expected. With that in mind, the progress Ailsa and her team have made is nothing short of incredible, with tests demonstrating that this drug may improve the motor and cognitive symptoms of early Huntington’s disease.

In order to explain how the drug works we first have to understand a few things about the brain. The cells in your brain communicate by sending chemical signals. There are different signals depending on where you are in the brain, and each one does something a little (or a lot!) different from the rest. For those with Huntington’s disease there is a drop in a chemical called acetylecholine, which is important for motor function and also for things like memory. Basically, what Ailsa and her team did was find a drug to replace that lost acetylcholine and they found that those functions that were suffering, e.g. motor function and memory, significantly improved.

 This simplified diagram shows a zoom in of the 'receiving' end (top) and the 'giving' end (bottom) of a brain cell. In HD there is less acetylcholine to activate its receptors, which means the cell is less activated, and there is less dopamine (brain happy chemical) released from the cell. The drug that Ailsa and team are trialing is able to mimic acetylcholine and activate its receptors, meaning the brain cell becomes more activated and releases more dopamine! 

This simplified diagram shows a zoom in of the 'receiving' end (top) and the 'giving' end (bottom) of a brain cell. In HD there is less acetylcholine to activate its receptors, which means the cell is less activated, and there is less dopamine (brain happy chemical) released from the cell. The drug that Ailsa and team are trialing is able to mimic acetylcholine and activate its receptors, meaning the brain cell becomes more activated and releases more dopamine! 

Participants in the study reported being able to think clearer, remember more, maintain control of their emotions, and more easily identify emotions in the faces of others. There were also improvements in their fine motor control and walking, however the cognitive changes were much more apparent than the motor changes.

The drug these researchers chose was in no way random. It was chosen because it slots into the same receptors as acetylcholine, so can trigger the same effects, and because it is currently available for the treatment of another condition. Once a drug is accepted as a safe treatment for one condition it is much easier and faster to get it approved to treat another condition. Using this drug over one specifically designed for Huntington’s disease means that, if it works, the treatment could be available in a year or so instead of a decade.

For the moment, things look really good for this potential treatment becoming a reality for those with Huntington’s disease. New Zealand based researchers are making a real difference, and their determination to find treatments is strong. There may not be an available treatment yet, there may not be a cure yet, but our scientists and scientists around the world will not stop until there is.

Read Ailsa’s scientific publications here:

https://www.ncbi.nlm.nih.gov/pubmed/28025093 - study on drugs effectiveness in HD mice

https://www.ncbi.nlm.nih.gov/pubmed/27695336 - HD human clinical trial

 

 

About the Author of this blog

Blaise.jpg
Blaise is a science writer for the Brain Health Research Centre at the University of Otago, and a certified member of the Australasian Medical Writers Association. She has studied psychology and neuroscience, and her aim is to raise awareness for neurological and psychological illnesses. Blaise is driven by a desire to make scientific information accessible and understandable to both the general public and to those impacted by these illnesses.