A recent study led by researchers at University College London (UCL) and Roche has revealed that utilizing a smartphone app for digital tests could significantly improve the detection of disease progression in Huntington's disease. These tests could also enhance the efficiency of clinical trials.
The study, published in Brain, demonstrated how digitizing tests designed to measure motor symptom progression in individuals with Huntington's disease can offer a sensitive and reliable method to monitor changes in patient function. By completing five simple movement control tests, including assessments of balance, finger tapping, and involuntary movements, a Huntington's Disease Digital Motor Score (HDDMS) is generated.
The HDDMS value indicates motor function status, with lower values representing better function and higher values indicating worse function. Regularly conducting these tests in daily life allows doctors and researchers to track disease progression using the HDDMS.
Researchers believe that incorporating the HDDMS in clinical trials could help detect even minor symptom changes, making it easier to assess the effectiveness of new treatments. This could lead to shorter study durations and expedite the development of new therapies for Huntington's disease.
Professor Ed Wild from UCL Huntington's Disease Centre emphasized the significance of the HDDMS in clinical trials, stating that it could provide clearer insights into treatment efficacy with fewer participants and shorter lead times compared to traditional measures. The convenience of evaluating the HDDMS in a five-minute home assessment makes it a more meaningful approach than in-clinic motor impairment assessments.
Huntington's disease is a hereditary neurodegenerative disorder that results in progressive loss of movement control, cognitive function, and behavior. While there is no cure for the condition, ongoing clinical trials are exploring new therapies. The development of the HDDMS and smartphone tests is crucial for improving the accuracy and efficiency of these trials.
Development of HDDMS and Smartphone Tests
The HDDMS was created by UCL and Roche researchers using data from 1,048 individuals in observational and interventional studies. By analyzing various digital tasks through a custom smartphone app, the researchers identified the most predictive measurements of disease progression, leading to the HDDMS battery.
Comparing the HDDMS to the Unified Huntington's Disease Rating Scale, the researchers found that the former was twice as sensitive in detecting real changes in motor function. Professor Wild highlighted the importance of sensitive tools in rare diseases like Huntington's, where minimizing trial burden and size is crucial for research advancement.
The study emphasizes the role of digital biomarkers in neurological research and showcases the potential for technology-driven advancements in disease monitoring and drug development. The HDDMS is now accessible to academic and industry entities involved in Huntington's disease research through Roche Diagnostics.
Study Limitations
While the research demonstrates promising results, there are limitations to consider. Data collection was limited to individuals in specific clinical trials, excluding those before symptom onset or with advanced HD. Further research is needed to validate the HDDMS's ability to predict long-term functional decline.
Source: News-Medical