José Lopes, PhD, managing science editor —

The upcoming Phase 3 trial of RG6042 will test whether lowering the levels of a mutant form of huntingtin (mHTT) translates into functional, motor and cognitive benefits for people with Huntington’s disease. Potential benefits could also help advance gene-silencing approaches in other neurodegenerative diseases. “Deep down, we all…

A method called quantitative electroencephalography (qEEG) enables the identification of Huntington’s gene carriers and could become a disease biomarker, according to a pilot study. The research, “EEG may serve as a biomarker in Huntington’s disease using machine learning automatic classification,” appeared in the journal…

Although well-tolerated, the investigational treatment Huntexil (pridopidine) failed to improve motor functions in a year-long Phase 2 clinical trial of Huntington’s patients. The research, “Safety and efficacy of pridopidine in patients with Huntington’s disease (PRIDE-HD): a phase 2, randomised, placebo-controlled, multicentre, dose-ranging study,” appeared in…

Measuring concentrations of mutant huntingtin (mHTT) protein and another protein, called neurofilament light protein (NfL), can provide early and specific indications of Huntington’s-related changes, according to a new study. The findings suggest the potential of these two markers in both clinical practice and clinical research. The study, “…

A Georgetown University Medical Center (GUMC) Phase 1b clinical trial is recruiting patients to test the safety and tolerability of a leukemia treatment called nilotinib in Huntington’s disease patients. The research team plans to initially enroll 10 patients with early to moderate Huntington’s. Patients will receive 150mg of…

Two new clinical studies of Roche’s investigational therapy RG6042 for Huntington’s disease (HD) are planned to start by the end of 2018 and will begin enrolling participants by early 2019. These studies will help researchers to understand Huntington’s progression and the therapeutic effectiveness of RG6042, which may “potentially be the…

Immunotherapy candidate laquinimod failed to meet its primary objective of improving motor function in Huntington’s disease patients after 12 months of treatment in a Phase 2 clinical trial. However, it did meet its secondary goal of reducing brain atrophy, according to a press release from Active Biotech. Laquinimod, developed…

Targeting specific genes involved in inflammation and cellular structure may be a therapeutic strategy to reduce protein clumps in patients with Huntington’s disease, according to researchers. Their study, “High-Throughput Functional Analysis Distinguishes Pathogenic, Nonpathogenic, and Compensatory Transcriptional Changes in Neurodegeneration,” appeared in the journal Cell…

A key protein in Huntington’s disease has damaging effects on heart function beyond the disease’s devastating neurological impact, according to researchers. Their study, “Cardiac mTORC1 Dysregulation Impacts Stress Adaptation and Survival in Huntington’s Disease,” was published in the journal Cell Reports. Huntington’s disease is…

Treatment with investigational medication VX15/2503 halts disease-related loss of brain volume and improves its metabolic activity in Huntington’s disease patients, according to a Phase 2 clinical study. The research, “Clinical Development of VX15/2503 Anti-Semaphorin 4D Antibody as a Potential Treatment for Huntington’s Disease,” was presented by Elizabeth Evans, PhD,…