The first patient has begun treatment on a global trial of a novel medication for Huntington's disease, led within the UK by a UCL and UCLH academic.
Professor Sarah Tabrizi, Director of the Huntington's Disease Centre, UCL Queen Square Institute of Neurology, and a consultant at UCLH, is leading the UK arm of a world-first trial of an investigational (experimental) medication for adults with Huntington's disease.
The study will investigate whether the medication - called ALN-HTT02 - is safe when given to patients with Huntington's. It will also seek to understand ALN-HTT02's effects in the body, and whether it can reduce levels of HTT protein in the brain.
A faulty version of the HTT gene causes Huntington's disease by damaging nerve cells in the brain.
Patients on the study at UCLH will have the study-specific tests and procedures and receive the study medication at the NIHR UCLH Clinical Research Facility site based in the National Hospital for Neurology and Neurosurgery. Multiple other sites are expected to participate in study across the UK, Canada, United States and Germany.
Huntington's disease is an inherited brain condition. It is a progressive disease and gradually reduces a person's ability to walk, talk, think, and communicate.
Around 7,000 people are living with Huntington's disease in the UK. Treatments and support are available to help reduce some of the symptoms associated with the disease such as physiotherapy to help with movement and balance and speech and language therapy for feeding and communication problems.
There are currently no treatments, however, to prevent the disease from progressing.
The sponsor of this first-in-human trial, Alnylam Pharmaceuticals, has co-developed the investigational medication under its collaboration with Regeneron Pharmaceuticals. The medication is designed to use RNA interference (RNAi) - a natural process that cells use to control which genes are active and how much of a specific protein is made.
It is hoped that this will silence the faulty HTT gene and temporarily stop the body's production of HTT protein.
Professor Sarah Tabrizi said: "We are very excited about this trial as the drug, ALN-HTT02, targets a specific region of the HTT gene called exon 1, which we now think is a key toxicity driver of the damage seen in Huntington's disease.
"I am so pleased we have begun this important and innovative trial, which we hope will bring us closer to an effective treatment for Huntington's disease and make a meaningful difference for patients and families affected by this devastating condition. I'm looking forward to the smooth running of the study and, hopefully, seeing ALN-HTT02 progress to further clinical development."
ALN-HTT02 is administered through an injection into the fluid around the spinal cord.
The goal is to enrol up to 54 participants in the study. Participants initially enter the 'double-blind period' of the study for the first six months, during which they are randomly assigned to receive a single injection of either ALN-HTT02 or placebo.
The placebo injection arm of the study contains no active ingredients.
Following the double-blind period, participants randomised to receive the placebo can then choose whether or not to receive ALN-HTT02.
Kevin Sloan, Vice President, Development Programs at Alnylam who leads the ALN-HTT02 programme, said: "Professor Tabrizi and UCLH are widely recognised for their expertise in HD and their patient-centric approach to clinical research.
"We're thrilled to be collaborating with established leaders in the space to advance an investigational RNAi therapeutic that we believe has the potential to alter the course of this devastating disease."
