New Tool Set to Transform Huntington’s Disease Patient Assessments

MARYLAND, USA — A new patient-reported outcome measure aims to transform how Huntington’s disease (HD) is assessed. Developed by researchers Matthew W. Roché, PhD, and Neha Sinha, PhD, the Functional Rating Scale 2.0 (FuRST 2.0) focuses on capturing subtle changes in patients’ daily lives.

Since her diagnosis in 2018, Jill Briceño has faced the challenges of HD, which has affected her family significantly. ‘Unlike traditional methods that overlook what patients experience at home, FuRST 2.0 puts us at the center of evaluation,’ said Jill’s husband, Carlos Briceño. This approach seeks to monitor early signs of progression that standard assessments often miss.

The FuRST 2.0 allows patients to report difficulties with everyday tasks, like making phone calls or driving, which can be critical for understanding their condition. ‘It’s like trying to measure a whisper with equipment meant for loud music; the nuances often go unnoticed,’ added Carlos.

Recognizing that many current clinical tools are not sensitive enough during the early stages of HD, Roché and Sinha’s team utilized input from focus groups consisting of HD families. Their ongoing research is being conducted with informal guidance from the U.S. Food and Drug Administration, aiming to meet regulatory standards for clinical trials.

As Carlos explained, ‘Every response in our study contributes to validating a tool that could change how HD is managed and treated.’ This online study is anonymous, takes about 20 minutes, and is critical in seeking early interventions that may slow the progression of HD.

Families struggling with HD are encouraged to participate because the results could lead to improved assessments and treatments in the future. ‘Contributing to this research is not just about us; it’s about fighting back against the disease that has impacted our lives,’ Carlos emphasized.

The FOCUS-HD Online study aims to develop instruments that truly reflect patient experiences, providing hope for better clinical assessments. For further inquiry about taking part in this important research, reach out to the study coordinators.