Revolutionary Treatments for the Devastating Huntington’s Disease

Authored by: Emma Robinson

Art by: Joyce Wang

Anyone who is familiar with Huntington’s Disease has likely heard of its life-altering effects. It is a particularly destructive neurodegenerative disease that leads to cognitive and motor deficits in those impacted [1]. The disease is relatively rare with only 2.7 per 100,000 individuals diagnosed, but has gained attention due to its severity [1]. While the age of onset differs depending on the type from young childhood to later adulthood, it leads to brain shrinkage, neuron degeneration, and eventual death [2]. Those with the disease tend to exhibit physical symptoms such as involuntary movements and weakness, as well as behavioral inconsistencies, memory loss, and even psychosis. Most individuals have adult onset in their forties or fifties, but those with juvenile onset develop symptoms before they are twenty years old with some even becoming symptomatic under ten years of age [3]. 

The genetic mutation causing the disease has been well-known for many years. It is caused by CAG (cytosine, guanine, adenine) repeats in the gene coding for the Huntington protein located on chromosome four. Patients with more of these repeats tend to have earlier disease onset [1]. The exact molecular and cellular mechanisms behind Huntington’s Disease progression are still being uncovered [4]. In its fully functional form, the Huntington protein has many roles such as the development of synapses and axonal transport [3]. In diseased patients, this protein is misfolded and accumulates in the patient’s neuronal cells [4]. However, due to the complex roles of the protein itself, it is difficult to determine all the manners in which the misfolded protein impacts the brain. Preclinical drug research has primarily been aimed at elucidating the mechanisms of neuronal injury. 

Due to the severity of the disease, there have been substantial efforts focused on minimizing or reversing disease progression. The most common treatments are aimed at treating the symptoms of the disease including Tetrabenazine to reduce involuntary movements [5]. Other drugs like Pridopidine are under development for the purpose of inducing biological protective proteins to minimize the impact of diseased Huntington proteins. One interesting new therapy from Brazil called Cellavita HD attempts to use stem cells from teeth to repair the damaged brain tissue [5]. Over the past few years, most new drugs such as Tominersen aim to target the mRNA for the toxic Huntington protein [6]. By targeting mRNA, these treatments should prevent protein from misfolding in the first place [6]. However, there are issues with many of these drugs in that they have not been able to be officially approved for widespread use due to side effects or inconclusive results in clinical trials. 

A recent and very promising breakthrough has been made with the AMT-130 treatment. This is a drug that also targets the Huntington protein mRNA but is more directly administered [7]. It works by injecting viruses that code for specific microRNAs into affected brain regions. These microRNAs sequester the mRNA for the diseased Huntington protein and prevent it from being translated into active proteins [8]. While still in the process of being FDA-approved, there have been some promising clinical study results. 

Twenty-nine Huntington Disease patients with Huntington’s Disease were given a one-time dose of AMT-130 directly to the brain through surgeries ranging from 8 to 10 hours. Patients were assigned to a high dose group or a low dose group in order to elucidate the dose-dependent effects [8]. In the seventeen individuals that were given the high dose, there was a 75% decrease in disease progression compared to individuals with the disease who did not receive the treatment in the first three years post-surgery [7]. Further supporting the treatment’s success and verifying its effect on a molecular level, researchers also saw a decrease in the misfolded protein in the spinal fluid of the treated individuals [8]. If this treatment continues to prove effective and is FDA-approved, it could drastically change outcomes for individuals with this devastating disease. Although the treatment development process is long and difficult, such revolutionary science allows for diseases like Huntington’s Disease to become a thing of the past. 

Works Cited

1. Ajitkumar, Anitha, and Orlando De Jesus. “Huntington Disease.” PubMed, StatPearls Publishing, 2023, www.ncbi.nlm.nih.gov/books/NBK559166/.

2. Frank, Samuel. “Treatment of Huntington’s Disease.” Neurotherapeutics, vol. 11, no. 1, 24 Dec. 2014, pp. 153–160, www.ncbi.nlm.nih.gov/pmc/articles/PMC3899480/, https://doi.org/10.1007/s13311-013-0244-z.

3.  Gatto, Emilia M., et al. “Huntington Disease: Advances in the Understanding of Its Mechanisms.” Clinical Parkinsonism & Related Disorders, vol. 3, no. 2590-1125, 2020, p. 100056, https://doi.org/10.1016/j.prdoa.2020.100056.

4. Rojas, Natalia Gonzalez, et al. “Review of Huntington’s Disease: From Basics to Advances in Diagnosis and Treatment.” Journal of Neurology Research, vol. 12, no. 3, 22 Oct. 2022, pp. 93–113, www.neurores.org/index.php/neurores/article/view/721/701#s6, https://doi.org/10.14740/jnr.v12i3.721.

5. Huntington's Disease Society of America. “Therapies in Pipeline | Huntington’s Disease Society of America.” Hdsa.org, 2017, hdsa.org/hd-research/therapies-in-pipeline/.

6. Singh, Kuldeep, et al. “Emerging Pharmacological Approaches for Huntington’s Disease.” European Journal of Pharmacology, vol. 980, 1 Oct. 2024, pp. 176873–176873, https://doi.org/10.1016/j.ejphar.2024.176873.

7. Gaither, Katherine. “Breakthrough in Huntington’s Disease Treatment Shows Unprecedented Results for Patients - Heersink School of Medicine News.” Uab.edu, 3 Oct. 2025, www.uab.edu/medicine/news/neurology/breakthrough-in-huntingtons-disease-treatment-shows-unprecedented-results-for-patients.

8. Dolgin, Elie. “Huntington’s Disease Treated for First Time Using Gene Therapy.” Nature, 25 Sept. 2025, www.nature.com/articles/d41586-025-03139-9, https://doi.org/10.1038/d41586-025-03139-9.