Development and feasibility randomised controlled pilot study of HD-DRUM - a novel motor sequence training app for people with Huntington's disease

Huntington’s Disease (HD) is an inherited disease that causes cell loss in brain regions important for learning and planning movements and doing two things at once (multitasking). HD leads to a worsening of these abilities. There is no cure for HD and there are currently no NHS services that can help movement and thinking changes. Also, the current COVID-19 pandemic has highlighted the urgent need for support that can be accessed at home when clinic visits are not possible.

Here, I plan to develop and test a new movement and rhythm training tool that people with HD can use at home. This involves learning drumming sequences that gradually increase in difficulty. Drumming requires key abilities of concentration, planning and making movements and multi-tasking (e.g. listening and drumming). HD affects all of these abilities as they rely on brain regions impaired by the disease. The drumming tool will train these key abilities.

The training is based on many studies in HD mice showing that movement symptoms can be delayed when mice have access to an enriched environment with more for them to do and think about. Similarly, the benefits of transplant therapy, i.e., replacing damaged brain cells with healthy ones, in HD mice can be increased if they are in an enriched environment. Furthermore, movement and thinking training was shown to help people with Alzheimer’s and Parkinson’s disease and can make brain changes including the strengthening of connections. Importantly, I found that two months of drumming improved concentration and multi-tasking abilities in people with HD and strengthened their brain connections.

However, these pilot findings were just in a small number of people and need to be tested in larger groups. As a next step, I need to develop a digital version of the training, where people always practise at the level that is best for them, i.e., neither too easy nor too hard. The tool would also allow to record training improvements, to see if people are training when they should be, and to make it available to everyone interested in using it.

I plan to work with members of HD-Voice, a panel of people with HD, their families and carers, and will instruct Kinneir Dufort, a digital software company, to develop a new digital drumming tool (HD-DRUM). HD-Voice members have already been involved in shaping this proposal and have agreed to help develop HD-DRUM. I will then see how easy it is for people who have not yet developed movement symptoms or have only mild-moderate changes to use HD-DRUM at home for three months (15 min per day, 5 days a week). Half of them will be using HD-DRUM while continuing their standard care and the other half will receive standard care only so that I can compare the two groups.

Everybody will have some movement and thinking tests and brain scans at the beginning and end of the study. These tests will let me work out the number of participants I would need for a future bigger trial for studying the effects of HD-DRUM on movement and thinking and the brain. I am also planning to look at combining HD-DRUM with transplant therapy by asking people with HD who have undergone this therapy to try out HD-DRUM.

In the future, HD-DRUM may be able to provide a remotely accessible training tool to help improve movement and thinking in HD without the risk of harmful side-effects. Even a small delay in symptoms starting due to the strengthening of brain connections and function would have direct and significant benefits for the quality of life of people with HD and their families.