‘Gene silencing’ – a promising investigational treatment for Huntington’s Disease

By October 30, 2015Blog Posts, Pharma Beat


A new approach to the treatment of Huntington’s disease (HD) made notable progress this week. Patients enrolled in the phase 1/2 clinical trial received their first dose of ISIS-HTTRx, a drug designed to target the disease at its genetic roots, with no immediate complications reported.

Huntington’s disease (HD) is a genetic brain disease caused by a mutated gene that produces a toxic protein called huntingtin. This protein slowly kills brain cells causing uncontrolled movements, behaviour changes and poor cognition. Current medication treats the symptoms, with no mechanism to slow or prevent progressive brain damage.

ISIS-HTTRx is an experimental medicine, known as a “gene silencer”. Messenger RNA (mRNA) provides a blueprint for creating proteins, and ISIS-HTTRx binds with mRNA from the HD gene, stopping production of the huntingtin protein. This type of treatment is known as an ‘antisense’ drug and is considered the most promising therapeutic strategy currently under investigation.

The study is being lead by University College London, with sites in Canada and Germany. Ongoing safety and levels of the mutant huntingtin protein will be monitored as the study progresses.