Successful Preliminary Trials Bring New Hope to Huntington’s Patients
- PULSE MedTech
- Apr 8
- 4 min read
When Tammy Stewart found out she had Huntington’s disease, it changed the way she viewed the rest of her life. Following the diagnosis, she became a self-procclaimed “Negative Nancy”, and struggled with the harsh reality of her future with Huntington’s.
In a 2019 interview with STAT, she explains how it is difficult to feel hopeful when faced with what she calls a “monster disease” and likens to having Alzheimer’s disease, Parkinson’s disease, and amyotrophic lateral sclerosis (ALS) all at once. Tammy explains that, “[e]ventually, your muscles give away and you can’t swallow, or walk, or feel yourself. You get incontinent and stuff like that.”
Tammy has already witnessed the disease massively affect her family. After experiencing her father and sister’s death from the same disease, she is now the primary caretaker for her brother who is in the late stages of Huntington’s disease (HD). Her brother, Mike, has lost the balance to walk on his own, needs someone to prepare every meal for him, and has limited communication ability. As his caretaker, Tammy is reminded daily of the rapid progression she too will have to face. She already has begun to develop a tremor and experiences periods of depression, both of which are associated with the disease, and worries about her future if a treatment is not found.
“If I’m at the beginning stages of it now, my quality of life with stress and stuff will go down fast,” she said. “And I don’t want that. I don’t want to live like that. I don’t.”
The Stewart family, along with roughly 41,000 Americans, face this harrowing reality. Huntington’s Disease is a fatal neurological disease that is characterized by deterioration of the ability to control body movement, loss of memory and cognitive abilities, personality or mood changes, and more. It can affect people in different ways, and symptoms can begin at any age. These symptoms worsen over time, and most patients live for about 10 to 25 years after the symptoms begin.
Huntington’s is caused by a gene mutation, meaning that there is an error in the patterning of a gene which causes other cellular processes to not function properly. In this case, the symptoms are from a mutation in a gene called HTT, which is passed on from parents to their children. If a child inherits the gene mutation, they will develop Huntington’s Disease. This mutation causes a protein called Huntington to be produced incorrectly and accumulate in the brain, leading to the death of brain cells and causing the range of HD symptoms.
There are currently no cures for Huntington’s, and treatment is mostly limited to managing symptoms through means such as physical, speech, and psychological therapy. While these can be helpful, they can pose a financial strain and only manage symptoms, not slow the progression of the disease. Because of the current lack of a cure or sufficient treatment, a Huntington’s diagnosis is equivalent to a death sentence.
At the time of Tammy’s interview, she was part of a trial for a drug called Tominersen, which has since been discontinued due to lack of improvement in patient outcomes and concerns of worsening symptoms associated with higher doses of the drug. While this was incredibly disheartening to many, it has not stopped the search for a successful cure.
A new treatment developed by Dutch biotechnology company UniQure brings new hope for those affected by Huntington’s. Their treatment, named AMT-130, utilizes gene therapy techniques to administer a one-time treatment via a 8 to 10 hour-long brain surgery. This process uses a small modified virus called AAV5 to carry microRNA into the cells of areas of the brain associated with HD. This microRNA is designed specifically to limit the “activation” of the Huntingtin gene, preventing it from producing the proteins which are associated with HD and in turn slowing the progression of the disease.
So far, this treatment has had shocking results, slowing the rate of disease progression by up to 75% and providing some increases in motor and cognitive function. Although it has only been tested on 12 people so far, AMT-130 may bring some much needed hope for people impacted by Huntington’s. Unlike previous treatments that only attempted to manage symptoms, AMT-130 targets the underlying genetic cause of the disease.
Larger studies are still necessary to confirm that this treatment is both safe and effective, but this still represents an important step forward in Huntington’s research. Even if AMT-130 may not be the long-term solution, it brings us closer to slowing and maybe even stopping Huntington’s.
Since Tammy’s interview with STAT, her brother has passed away and the clinical trial she was enrolled in failed. The hope that AMT-130 brings is needed now more than ever for people like Tammy and her family. While treatments are still years away from widespread availability, the progress being made by researchers suggests that the future of Huntington’s disease may not always look the way it does today. As more experiential treatments emerge, there persists new hope that Huntington’s disease may become more manageable, and maybe even curable in the future. †
Written by Editor and Staff Writer Audrey Gayou (agayou@ucsd.edu)
Works Cited
“Living with Huntington’s Disease.” YouTube, STAT, 2019,
Dolgin, Elie.“‘Giant Step Forward’ for Huntington’s - the Scientist behind the First Gene Therapy.”Nature News, Nature Publishing Group, 8 Dec. 2025,
Gallagher, James. “Huntington’s Disease Successfully Treated for First Time.”BBC News,
BBC, 24 Sept. 2025, www.bbc.com/news/articles/cevz13xkxpro.
“Huntington’s Disease Society of America.” Huntington’s Disease Society of America
Family Is Everything, 2 July 2015, hdsa.org/.
Promising Huntington Treatment Tominersen Has Generation HD1 Study Discontinued
Neurologylive - Clinical Neurology News and Neurology Expert Insights,
hd1-discontinued. Accessed 30 Mar. 2026.
uniQure. “Huntington’s Disease: Programs & Pipeline.” uniQure,
www.uniqure.com/programs-pipeline/huntingtons-disease. Accessed 29 Mar. 2026.
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