Activity monitoring biomarkerUsing novel algorithms to chart daily activities of ambulant and non-ambulant boys
The DRF has awarded £320,000 to Dr Aldo Faisal’s team at Imperial College London to develop a novel biomarker to track the progression of Duchenne muscular dystrophy. As part of the study, both ambulant and non-ambulant Duchenne patients will wear sensors in the form of four Apple watches (one on each wrist and one on each ankle) to monitor their daily activities and movements outside a clinical setting. Dr Faisal’s expertise is in developing a novel algorithm to analyse the data gathered from the sensor devices. On three occasions in the year the participants will then put on a sensor bodysuit just for their clinic appointment, in order to evaluate the link between the data gathered at home and their progress in clinic.
One of the major challenges in Duchenne drug development is linking measureable clinical trial endpoints with actual quality of life benefits. While there is a measurable and predictable decline in function during the course of the disease, each boy declines in a unique way, which can be hard to measure through classic functional tests like the six-minute walk test. This vital research aims to complement or even replace classic clinical endpoints, more sensitively capturing functional changes, and increasing the number of boys and young men eligible for clinical trials. This study will be carried out as a clinical natural history study at the Clinical Research Facility of Great Ormond Street Hospital Trust, overseen by Professor Thomas Voit.
Funded in 2017-18
Learn more about this ground-breaking technology
The development and approval of disease-modifying treatments is often slowed down by the fact that it takes a long time to determine if a treatment works well or not for a group of patients. Many gold-standard methods for tracking disease progression rely on judgements ‘by eye’ in the clinic. This is especially true in ‘slow’ degenerative disorders such as Duchenne. We have now the unique opportunity to translate our artificial intelligence technology to the monitoring of Duchenne progression. We will give Duchenne boys wearable sensors (in the form of Apple watches), which will capture their arm and leg movement where it matters most to them: at home and in school. This movement data is collected discreetly, 24/7, and will be analysed by our artificial intelligence technology. This is a bit like having a personal neurologist looking after you day and night, day after day, who can notice even subtle changes in movement ability. We hope that these novel approaches will significantly improve the accuracy and reduce the time it takes to detect disease progression, potentially reducing the duration of future clinical trials and the cost of treatment development. We are delighted to have a forward looking clinical partner in Great Ormond Street’s Clinical Research Facility, with whom we will be working closely.Dr Aldo Faisal
Why are we supporting this project?
Despite the significant acceleration of therapeutic developments in the field of Duchenne in the last few years, a number of issues remain to be solved and require dedicated support and attention. We need better and more reliable outcome measures for boys with Duchenne, which is an area that has been insufficiently explored in the field so far. Characterisation of better outcome measures will enable the field to monitor disease progression and benefits of any therapeutic strategies in a more coherent and reliable manner. The newly funded projects supported by the Duchenne Research Fund will address those shortcomings and I am confident that the successful completion of those will make seminal contributions to the field of Duchenne.Professor Ronald Cohn
View our other newly funded research projects.
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