The new system assesses the progression of Huntington’s disease. As part of an international consortium, researchers from the University College London (UCL) have developed a novel staging framework that evaluates the progression of Huntington's disease (HD), similar to how cancer is staged from 0
The researchers believe that the new framework will allow for the early study of Huntington’s disease.As part of an international consortium, researchers from thehave developed a novel staging framework that evaluates the progression of Huntington’s disease , similar to how cancer is staged from 0 to 4. This ‘significant’ step opens the door for drug clinical trials in the early stages of the disease.
The HD-ISS staging criteria span the entire disease spectrum, starting at Stage 0 , progressing to Stage 1 , Stage 2, and then Stage 3 .First author, Professor Sarah Tabrizi said: “HD families have long known that signs and symptoms begin decades before the classical motor onset diagnosis that occurs late in the disease course.
“Ultimately, our goal is to deliver therapeutics at the right time to effectively treat this disease. Ideally, we’d like to delay or prevent neurodegeneration while function is still intact, giving people with HD many more years without impairment. The HD-ISS is an important tool that will allow us to do this as therapeutics emerge.’’
Cristina Sampaio, MD, Ph.D., Chief Clinical Officer at CHDI Management/CHDI Foundation, said: “As a validated staging system, the HD-ISS has the potential to transform how HD clinical research is conducted, enabling the study of the earliest disease phases and planning of preventive clinical trials, as well as facilitating data aggregation and sharing.
The disease phase prior to this current diagnostic stage has been variously described as ‘presymptomatic’, ‘premanifest’, or ‘prodromal’, poorly defined terms that make it difficult to compare data and findings across trials and studies. The HD-ISS was developed as part of the HD-RSC in partnership with CHDI Foundation and C-Path and involved academic researchers, clinician scientists, and representatives from pharmaceutical and biotech companies that have clinical HD programs.
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