Science Subp.: The new study details the dynamics of two protein markers of Huntington's disease in patients

December 20, 2020 /--- Huntington's disease is an incurable neurodegenerative disease.
In a new two-year longitudinal study, researchers from research institutions such as University College London revealed how two proteins associated with Huntington's disease change over time in patients and in asymptomatic people who carry the genetic mutations that cause the disease.
by detailing the dynamics of these two disease markers, these results may help clinicians develop tools to predict the progression of Huntington's disease and evaluate new treatments in clinical trials.
study was published in the December 16, 2020 issue of the Journal of Science Translational Medicine under the title "Mutant Huntingtin and neurofilament light have a longitudinal dynamics in Huntington's Disease."
longitudinal dynamics of mHTT and NfL over a 24-month 24-month 24-month 24-month 2020, doi:10.1126/scitranslmed.abc2888.
Though there is no cure for Huntington's disease, scientists theoretically believe that targeting the mutant huntingtin (mHTT) may prevent people with disease-related mutations who have not yet developed symptoms from developing the disease.
testing this strategy requires measuring mHTT or another disease-related protein called neurofilament light, NfL, but it has not been clear how the concentrations of these two proteins change as the disease progresses.
to conduct the study, Filipe Rodriguez and colleagues collected plasma and cerebrospinal fluid samples from 20 controls, 20 mutant carriers and 40 Huntington's disease patients.
the researchers conducted two years of longitudinal measurements of mHTT and NfL and found that the NfL levels of the mutant carriers rose faster and in different patterns than those of the controls.
, patients with the highest baseline levels of mHTT and NfL showed faster disease progression and more severe brain atrophy two years later.
the researchers concluded, "These new insights... Clinical trials for the design and development of disease mitigation will be of direct value, especially as we enter the era of preventive trials, and qualified alternative endpoints will be critical.
" (Bioon.com) Reference: 1. Filipe B. Rodrigues et al. Mutant huntingtin and neurofilament light have distinct longitudinal dynamics in Huntington's disease. Science Translational Medicine, 2020, doi:10.1126/scitranslmed.abc2888.2.Two-year study details dynamics of Huntington's disease markers in patients。