A novel framework that assesses the progression of Huntington’s disease, has been developed by scientists including Prof Sarah Tabrizi (UK DRI at UCL), as part of an international consortium. The system groups people with the condition according to their underlying biological, clinical, and functional characteristics, and will pave the way for clinical drug trials targeting the earlier stages of disease.
Published in The Lancet Neurology today, the new evidence-based research framework for Huntington’s, called the Huntington’s Disease Integrated Staging System (HD-ISS), includes criteria to biologically define the disease as well as a staging system that encompasses the whole lifespan of disease progression from birth. It is the first time a staging system has been developed for a genetic neurological condition.
The HD-ISS defines groups of people with Huntington’s who have similar prognostic characteristics, aiding research facilitating data comparison across different trials and studies. The researchers hope this will accelerate drug development, and aid in communication between different stakeholders, from families affected by the disease to health policy professionals.
Importantly, this new biological research definition of Huntington’s will allow evaluation of new therapeutic approaches in people during the very early stages of disease, before they start to experience clinical signs. This will likely provide the best chance of substantially slowing disease progression and offer the greatest clinical benefit.