Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.

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Latest news

An early role for the Huntington's disease gene – but don't believe all the headlines

An early role for the Huntington's disease gene – but don't believe all the headlines

Dr Jeff Carroll on February 06, 2018

New hints are emerging about the normal role of the gene that causes Huntington's disease. A recent report uses cutting edge techniques to study this question in cells growing in the lab. We'll help separate the fascinating new science from some scary-sounding headlines.

New interest in an old target

New interest in an old target

Dr Jeff Carroll on January 30, 2018

A recent mouse study of a drug known as CTEP suggests the drug is surprisingly helpful for HD-like symptoms in mice. This is a welcome surprise, because it suggests a well-understood brain process might be a useful drug target for future HD research.

Ask the expert: Q&A on the huntingtin-lowering trial program

Ask the expert: Q&A on the huntingtin-lowering trial program

Dr Ed Wild on December 18, 2017

The research news, announced on 11 December 2017, that a research team from the Huntington's Disease Centre at University College London have made significant step towards a possible treatment for Huntington's disease, raised many questions for the Huntington's disease community. Dr Ed Wild answered some of these questions on behalf of the UK HD Association, helping to give some context to what this news means for people affected by Huntington's now and in the future.

Success! ASO drug reduces levels of mutant protein in Huntington's disease patients

Success! ASO drug reduces levels of mutant protein in Huntington's disease patients

Dr Jeff Carroll on December 11, 2017

In an announcement likely to stand as one of the biggest breakthroughs in Huntington's disease since the discovery of the HD gene in 1993, Ionis and Roche today announced that the first human trial of a huntingtin-lowering drug, IONIS-HTTRx, demonstrates that it reduces mutant huntingtin in the nervous system, and is safe and well-tolerated.

Switch off the genome editor when you’re done

Switch off the genome editor when you’re done

Dr Michael Flower on November 02, 2017

Genome editing is a much-discussed frontier in medical science at the moment, with ‘DNA surgery’ having the potential to treat or cure genetic diseases like Huntington’s. Here we look at what this technology can currently do and discuss the challenges that still lie in the way. We’ll also discuss how a team of Swiss scientists have recently developed a way to switch off the genome-editing machinery after it’s done its job.

A new way of thinking about trials to prevent Huntington's disease

A new way of thinking about trials to prevent Huntington's disease

Dr Jeff Carroll on October 27, 2017

A new analysis of clinical data from the TRACK-HD and COHORT studies proposes a way to design of clinical trials designed to delay the onset of HD, rather than treating symptoms after they occur.

Update confirms Huntington's disease 'gene silencing' trial on track

Update confirms Huntington's disease 'gene silencing' trial on track

Dr Jeff Carroll on October 15, 2017

Ionis Pharmaceuticals launched the first ever trial of a huntingtin-lowering drug – sometimes called a 'gene silencing drug' – in late 2015. In a significant update, the company has announced two important milestones: the trial is now fully recruited, and an 'open-label extension' will be activated for the volunteers in the current trial. While nothing is guaranteed, this bodes well for the future of this important program.

New roles for huntingtin: removing a healthy protein to understand its function

New roles for huntingtin: removing a healthy protein to understand its function

Leora Fox on September 26, 2017

Some techniques aimed at lowering mutant huntingtin can also affect the normal form of the protein. With clinical trials underway, it’s all the more important to understand the role of normal huntingtin in the adult brain. Researchers recently inactivated the huntingtin gene in healthy adult mice of different ages. They found that this could cause neurological and behavioral problems. Mice aren’t perfect for modelling human brains, and no huntingtin-lowering drug would remove the protein completely - but this research supports the need for continued caution as we test drugs that lower normal huntingtin.

Precision huntingtin-lowering drug trials target the mutant protein

Precision huntingtin-lowering drug trials target the mutant protein

Dr Michael Flower on August 25, 2017

A new exciting chapter in Huntington’s disease (HD) treatment is just beginning – WAVE Life Sciences have announced PRECISION-HD1 and 2, clinical trials of two new drugs that lower the mutant Huntington’s disease protein. We’re excited about this novel approach to huntingtin lowering therapies, but these are early days and we’ve got a long way to go to show they’re safe and effective in people.