Experts in the field of Huntington’s disease research from across the globe gathered at the World Forum in The Hague, Netherlands, between 16 and 18 September for the European Huntington’s Disease Network (EHDN) Plenary Meeting.
Attendees heard from eminent speakers from scientific and research backgrounds who delivered presentations on progress in current research, alongside updates on ongoing clinical trials and findings from clinical trials that have recently completed.
For non-scientists, there were lay presentations and posters on display in a more easily understandable format, meaning those without specific scientific knowledge were able to gain an insight into recent advancements in HD research.
Bill Crowder, Head of Advisory Service at HDA, said: “It was very encouraging to hear the progress being made in all areas of HD research. There were also useful science sessions which included clinical practice in Enroll-HD, and psychiatric and psychological support, which gave further insight into new developments.”
Bill outlines some of the key highlights from the conference presentations below:
Research
- Historically, genetic scientists have been focusing on CAG repeats, but recently there has been a focus on RAN genes – with the aim if removing RNA and RAN proteins.
- An interesting piece of research was referenced which saw how Parkin is being used fruit flies with the result of significant life extension, which is the result of research undertaken into the treatment of Parkinson’s disease.
- Another advancement has been with zinc finger technology that works by targeting DNA – this technology does not affect normal CAG repeats but has been seen to repress repeats that are greater than 39 – a significant technique for Huntington’s disease research.
- A number of the pharmaceutical companies present talked with optimism of Huntingtin lowering working in humans. We were told to keep an eye on the companies’ websites for the latest progress as they are working hard to determine how early in the disease they would need to start therapeutic intervention, which could be early manifest or pre-symptomatic stage. The companies involved include: Ionis, Biomaris, Spark, UniQure, Voyager and Meditronic.
- HD Clarity, a research study using cerebrospinal fluid, is investigating biomarkers and pathways with the aim of developing novel treatments. They have announced they need 600 people to take part (both HD and non-HD patients) – check out HDClarity.net to find out more .
- Researchers re-confirmed that exercise has been shown to alter disease progression, improving mobility and balance, which has the knock on effect of improving cognition and social mobility, therefore giving a better quality of life to the patient and carer.
Highlights of completed clinical trials
- SD 809 – The Deutetrabenazine trial of 90 participants has demonstrated a small but significant weight gain. The Arc HD trial is for those already on Tetrabenazine but who have transferred to Deutetrabenazine. The trial has also shown much improvement in chorea and these are similar results from the two trials that have been completed.
- Action HD – This trial of non-pharmaceutical therapy, investigated apathy in people with HD. This successfully demonstrated an improvement in apathy of 50 patients participating over 3 years.
Update on ongoing trials
- Ionis Huntingtin lowering – The trial is showing to be safe and tolerable, which now allows progression to the next dosage stage. Find out more at hdresearch.ucl.ac.uk
- Amaryllis – This trial is looking to change motor function in HD and is on schedule to complete in October 2016, with a further phase study to follow. Find out more at hdresearch.ucl.ac.uk
- Legato – This drug has been developed for application in Multiple Sclerosis as it is aimed at neuroinflammatory, but there are signs that this could have effect in Huntington’s patients. Find out more at hdresearch.ucl.ac.uk
- Stair – This investigation is in the second phase of a 12 week study looking at irritability in people with HD. Find out more at clinicaltrials.gov
- Trihep 3 – This study is looking at fatty acid in the liver and caudate atrophy to see if there is a link. Find out more at inserm.fr