Huntington’s Disease Cure and what that Means for Patients

By: Jadelyn Law

Huntington’s Disease⸺named after the doctor, George Huntington, who first diagnosed it⸺is fatally degenerative. It slowly robs the affected person of their ability to control their movements, impairs their cognitive skills, may cause emotional distress, and creates a number of other issues relating to the decline of their physical and mental capacities. First recognized and recorded in 1842 by Charles Oscar Waters in the publication of Practice of Medicine, Huntington’s Disease has affected over 41,000 people in the United States alone and over 200,000 people who are susceptible to inheriting it.

It is not a communicable disease, but rather one that is inherited from either parent. There is a 50% chance that a parent carrying the gene for Huntington’s Disease will pass it to their offspring. However, many people carrying it unknowingly pass it before learning of their chances of transference. Although symptoms may be apparent from a young age, obvious signs of Huntington’s Disease might not appear until a person is in their thirties or forties. By the time they start showing these symptoms, based on the speed of their cognitive and physical decline, they may live another five to twenty-five years.

Until now, the only available solutions to the deadly disease have been treatments for specific symptoms like chorea⸻involuntary jerks or movements that may impair a person’s ability to control their body. While there have been discussions of using gene therapy to repair the HTT gene that carries Huntington’s Disease, Professor Sarah Tabrizi announced on the 25th of September that profound new technology had been found to reduce the presence of the HTT gene by 75%. 

Professor Sarah Tabrizi is not only the co-founder and director of the University College of London (UCL) Huntington’s Disease Research Center, but also the Joint Head of the UCL Queen Square Institute of Neurology. She has dedicated her life to academia, specifically pertaining to neuroscience and has specialized in the treatment of Huntington’s Disease (along with many other specialized fields, possessing 5 degrees and 10 awards and recognitions). Professor Tabrizi and her team hope that the Food and Drug Administration (FDA) will be able to distribute their latest research and medicines in 2026, stating in her interview with UCL: “We are eager to discuss the data with the FDA at our pre-Biologics License Application meeting expected later this year, with the goal of submitting a BLA in the first quarter of 2026.”

The above technology includes a combination of gene therapy treatment and neurosurgery. The first event that the administrations would require would be the infusion of a “safe virus” that carries a sequence of DNA that, when activated by the neurons, would send a signal to begin the production of microRNA to interrupt, and hopefully, destroy the cells that code for the HTT gene. It is important to note that, whilst this is most definitely ground-breaking technology and is the closest we have come to curing people of the disease, it is not actually curing the disease. Instead, it is slowing and blocking cells from being able to code for the gene and reducing the toxic proteins created by Huntington’s. This radically improves the quality of life and may be the gateway to an official cure.

This is some of the most important and innovative technology of our age, but it is important to recognize that this will not be easily accessible nor realistic for most patients. The neurosurgery imperative to the treatment is complex and will be expensive, especially during its immediate release, and people who desperately need it may not be able to afford it. This is not to belittle the ingenious work of the scientists and professors behind it, but simply a recognition of the broken healthcare system that plagues both the United States and the United Kingdom (where the research and development is taking place). This is not anything new; it has been a known side effect of great technology for years now, but the American government shutdown and national lack of funding surrounding several healthcare insurances only brings the issues of this country further to light. Over half of all Americans rely on these insurances to cover them and may only be able to seek healthcare (no matter how serious the ailment may be) if they are covered by said insurances.

If this treatment is to go into public use (which seems probable), then it must be affordable or else it will be virtually useless. If no one can actually afford the treatment, then there is no reason to even create such a remedy because no one benefits from it and no one is actually treated by it. America may have some of the best healthcare globally, with hospitals such as the Cleveland Clinic and the Mayo Clinic treating over 16 million patients annually, but if such hospitable centers can not aid anyone without breaking the bank, they are rendered futile.

The research led by Professor Tabrizi, along with the countless other scientists and doctors, has improved the lives and health of an immeasurable number of people and has contributed to science in ways that may not even be fully realized today. As we celebrate the founding of new technology, we must recognize the people who have helped pave the way here without forgetting the ongoing obstacles that interrupt the healthcare system and the people who are affected.

Citations

Durbach, N, M R Hayden. “George Huntington: the man behind the eponym.” Journal of Medical Genetics. Accessed October 21, 2025. https://pmc.ncbi.nlm.nih.gov/articles/PMC1016378/

Gallagher, James. “Huntington’s Disease successfully treated for the first time.” Last modified September 24, 2025. Accessed October 21, 2025. https://www.bbc.com/news/articles/cevz13xkxpro

“Huntington’s Disease.” Mayo Clinic. Last modified April 25, 2024. Accessed October 21, 2025. https://www.mayoclinic.org/diseases-conditions/huntingtons-disease/symptoms-causes/syc-20356117

Mandal, Ananya. “Huntington’s Disease History.” Last modified February 26, 2019. Accessed October 21, 2025. https://www.news-medical.net/health/Huntingtons-Disease-History.aspx#:~:text=The%20first%20recorded%20mention%20of,acknowledgement%20as%20a%20medical%20condition.

Estofan, Leonel, Giordano Farmer, Jill M. “Huntington’s Disease: A Therapeutic Update.” Pratricalneurology.com. Last modified November 26, 2017. Accessed October 21, 2025. https://practicalneurology.com/diseases-diagnoses/movement-disorders/huntingtons-disease-a-therapeutic-update/30308/

Lane, Chris. “Gene therapy appears to slow Huntington’s disease progression.” Last modified September 24, 2025. Accessed October 21, 2025. https://www.ucl.ac.uk/news/2025/sep/gene-therapy-appears-slow-huntingtons-disease-progression

“Professor Sarah Tabrizi.” Accessed October 21, 2025. https://profiles.ucl.ac.uk/6907-sarah-tabrizi

Lovelace Jr., Berkeley. “What government shutdown means for Medicare, Medicaid and other health programs.” Last modified October 3, 2025. Accessed October 21, 2025. https://www.nbcnews.com/health/health-care/government-shutdown-medicare-medicaid-health-programs-rcna235052

“State of the Clinic: In 2024, Cleveland Clinic Serves Most Patients in its History.” Last modified January 27, 2025. Accessed October 21, 2025. https://newsroom.clevelandclinic.org/2025/01/27/state-of-the-clinic-in-2024-cleveland-clinic-serves-most-patients-in-its-history