Top 10 Huntington’s stories of 2024
Therapy trials, rising optimism, and HDSA talks caught readers' interest
Throughout 2024, the team at Huntington’s Disease News covered the latest developments in research and news related to Huntington’s disease. Here we’ve compiled a list of the top 10 most read stories, each with a brief summary. We look forward to continuing to serve the Huntington’s community throughout the new year.
No. 10 – Smoking, alcoholic beverages may affect Huntington’s progression
A study of more than 2,600 people with presymptomatic Huntington’s, meaning they carry disease-causing mutations but are not yet showing Huntington’s symptoms, found that those who smoked cigarettes tended to have faster disease progression than nonsmokers. Those consuming low-to-moderate amounts of alcohol had slower progression than people who didn’t drink alcohol at all. Higher levels of education and frequently drinking coffee also were identified as factors that slow Huntington’s progression in its presymptomatic stages.
No. 9 – uniQure, FDA discussing steps for Huntington’s gene therapy AMT-130
uniQure announced in May that it was on track to start talks with the U.S. Food and Drug Administration (FDA) concerning future steps for AMT-130, the company’s experimental gene therapy for Huntington’s. AMT-130 is designed to suppress the production of the mutant form of the huntingtin protein that causes Huntington’s. A month later, AMT-130 became the first potential disease treatment to be given regenerative medicine advanced therapy status by the FDA, which is meant to help accelerate its development and regulatory review. The therapy has shown a potential to slow early Huntington’s progression in two ongoing Phase 1/2 clinical trials, whose final data may be sufficient to file a request for approval, the FDA agreed in late 2024.
No. 8 – Doctors optimistic about potential for new Huntington’s meds
A Spherix Global Insights’ survey of neurologists in the U.S. with expertise in the care of Huntington’s found many optimistic about the potential for new disease treatments to be proven effective and approved in the near future. Clinicians stressed a need for treatments that can slow nerve damage before symptoms kick in, with many expressing frustration that, currently, no treatments are able to meaningfully slow a patient’s cognitive decline.
No. 7 – Two Huntington’s trials highlighted at HDSA annual conference
Two placebo-controlled Phase 2 trials, one still recruiting participants, into two potential Huntington’s treatments were showcased at last year’s Huntington’s Disease Society of America (HDSA) annual convention. One is testing tominersen, a therapy given by injection into the spine that aims to target Huntington’s underlying cause, in up to 300 adults with early stage disease, who are being recruited at sites worldwide. The other trial was testing dalzanemdor, an experimental oral treatment designed to boost cognitive function in Huntington’s patients. However, the dalzanemdor trial failed to show any cognitive benefits with the therapy relative to a placebo, and its clinical development was ultimately discontinued.
No. 6 – PTC518 showing potential in interim analysis of Phase 2 trial
Interim data from a Phase 2a study showed that PTC518, PTC Therapeutics’ experimental oral treatment, is able to reduce levels of mutant huntingtin in the blood and fluid surrounding the brain and spinal cord of people with early or intermediate Huntington’s disease. Available data found that PTC518 was well tolerated overall, and hinted that the therapy might be slowing Huntington’s progression.
No. 5 – Results anticipated for trial of Huntington’s treatment WVE-003
Wave Life Sciences announced in January that final data from a Phase 1/2 trial of its experimental medicine WVE-003 in 47 early Huntington’s patients would be presented later in the year. The therapy is designed to specifically target the mutant huntingtin protein, while leaving the healthy version unaffected. Results announced over the summer showed WVE-003 was reducing mutant protein levels as intended, with trends toward slower disease progression. Wave now is planning next steps in the development of WVE-003.
No. 4 – ‘Never been a more optimistic time,’ HDSA CEO says
In a speech at the opening ceremony of last year’s HDSA annual convention, Louise Vetter, HDSA’s president and CEO at the time, said there has never been a more optimistic time for the Huntington’s community. People affected by Huntington’s, Vetter said, have access to more and better resources than ever before, including medications that can better manage certain symptoms. Regulatory agencies also are paying more attention to the needs of Huntington’s patients, and there are dozens of potential treatments in development, including those targeting the disease’s underlying cause.
No. 3 – Huntington’s families need psychological support, study finds
At the HDSA meeting, Nicolo Zarotti, a clinical psychologist, presented results from an interview-based study emphasizing that people with Huntington’s and their families need better psychological support to help cope with the challenges of the disease across all its stages. Many interviewed participants felt they were not getting enough psychological support, and many considered mental health providers often ill-equipped for the task. But while clinicians may not be helpful, Zarotti noted that advocacy groups like the HDSA offer a variety of resources to support people affected by Huntington’s.
No. 2 – Huntington’s patients may benefit from psychotherapy
Spencer Diehl, a therapist who works with Huntington’s patients, gave a talk about psychotherapy at the HDSA meeting, noting that there are many types of psychotherapy that may help in navigating life with the condition and coping with its associated challenges. He also emphasized that patients should feel empowered to seek support whenever they feel they need it, and they should not “settle” when it comes to therapists, as finding a good therapist is a highly individualized process.
No. 1 – Good sleep hygiene can help Huntington’s patients sleep well at night
During another HDSA presentation, the neurologist Victor Sung shared advice for how to get a good night’s sleep, which can be challenging for people with Huntington’s. The advice focused mainly on practicing good sleep hygiene, which includes things like going to bed at the same time each night, avoiding stressful situations and bright screens before bedtime or in bed, and maintaining a quiet, cozy space for sleep. Taking small steps like establishing routines and practicing relaxation techniques are among the ways Huntington’s patients can work toward good sleep hygiene.
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We hope these stories, and all of our reporting here at Huntington’s Disease News, has been informative, and we look forward to continuing to serve as a resource for the Huntington’s community. We wish all our readers a bright 2025!
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