Optimal Time to Treat Huntington’s Disease ID’d; Abnormally High Levels of Neurofilament Light (NfL) Biomarker Detectable 24 Years Before Onset of Clinical Symptoms; Marker Could Be Used to Monitor & Gauge Effectiveness of Future Treatments

The earliest brain changes due to Huntington’s disease can be detected 24 years before clinical symptoms show, according to a new University College London (UCL)-led study. The researchers say their findings, published in the June 1, 2020 issue of The Lancet Neurology (https://www.thelancet.com/journals/laneur/article/PIIS1474-4422(20)30143-5/fulltext), could help with clinical trials by pinpointing the optimal time to begin treating the disease. The open-access article is titled “Biological and Clinical Characteristics of Gene Carriers Far from Predicted Onset in the Huntington's Disease Young Adult Study (HD-YAS): A Cross-Sectional Analysis.” There is currently no cure for Huntington’s, a hereditary neurodegenerative disease, but recent advances in genetic therapies hold great promise. Researchers would ultimately like to treat people before the genetic mutation has caused any functional impairment. However, until now, it was unknown when the first signs of damage emerge--but because there is a genetic test for Huntington’s susceptibility, researchers have a unique opportunity to study the disease before symptoms appear. Professor Sarah Tabrizi (photo), MD, PhD, (https://en.wikipedia.org/wiki/Sarah_Tabrizi) (UCL Huntington’s Disease Centre, UCL Queen Square Institute of Neurology), the study lead, said: “Ultimately, our goal is to deliver the right drug at the right time to effectively treat this disease – ideally we would like to delay or prevent neurodegeneration while function is still intact, giving gene carriers many more years of life without impairment.” “As the field makes great strides with the drug development, these findings provide vital new insights informing the best time to initiate treatments in the future, and represent a significant advance in our understanding of early Huntington’s.”
Login Or Register To Read Full Story