Global trial launches pioneering study for Huntington’s disease

The journey towards an effective treatment for Huntington’s Disease (HD) takes a significant step forward as the first patient has been recruited into a global trial for the investigational medication ALN-HTT02. This study, a collaborative effort led in the UK by Professor Sarah Tabrizi from the UCL Queen Square Institute of Neurology and UCLH, is set to explore the safety and impact of ALN-HTT02 on the disease.

Understanding the study

The primary objective of this trial is to assess the safety of ALN-HTT02 in HD patients. Additionally, researchers aim to investigate its ability to reduce levels of HTT protein in the brain. This faulty protein, produced by a mutated HTT gene, is known to damage nerve cells and drive the progression of HD.

Participants in the study will undergo a series of specialised tests and procedures at the NIHR UCLH Clinical Research Facility, based in the National Hospital for Neurology and Neurosurgery. In addition to the UK, clinical sites in Canada, the United States, and Germany are expected to participate, targeting enrolment of up to 54 individuals.

What is ALN-HTT02?

ALN-HTT02, developed by Alnylam Pharmaceuticals in collaboration with Regeneron Pharmaceuticals, uses RNA interference (RNAi) technology to silence the faulty HTT gene. By stopping the body’s production of HTT protein, this innovative medication could potentially slow or halt the progression of HD. Administered through an injection into the spinal fluid, ALN-HTT02 targets exon 1 of the HTT gene, which has been identified as a critical factor in the disease’s toxicity.

Trial design

Participants in the study will initially enter a six-month double-blind period, during which they will be randomly assigned to receive either ALN-HTT02 or a placebo. After this period, participants who received the placebo will have the option to receive the active medication.

A step towards hope

Professor Sarah Tabrizi:

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 HD and make a meaningful difference for patients and families affected by this devastating condition.

Kevin Sloan, Vice President, Development Programs at Alnylam, praised the collaboration with UCLH:

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.

About Huntington’s disease

Huntington’s Disease is an inherited brain disorder that progressively affects an individual’s ability to walk, talk, think, and communicate. With no current treatments to halt the disease’s progression, this study represents a beacon of hope for the approximately 7,000 people living with HD in the UK and many more worldwide.

Looking ahead

This trial not only highlights the innovative potential of RNAi technology but also underscores the dedication of researchers, clinicians, and pharmaceutical collaborators to finding a solution for this devastating disease. The successful progression of ALN-HTT02 through this and future trials could mark a turning point in the fight against Huntington’s Disease.