By Annie Wagner ‘27
In the United States alone, over 41,000 people battle a debilitating and incurable disease that costs them their ability to perform everyday tasks, live on their own, and eventually their lives. This condition, known as Huntington’s disease, is tied to the HTT gene, which produces the protein huntingtin. Entirely genetic, children have a 50% chance of acquiring the disease if a parent has it, and multiple members of families often suffer from the same terminal illness . Recently, a treatment called AMT-130 has been developed to slow the progression of symptoms through the infusion of healthy copies of the HTT gene directly into the brain via surgery. Despite its novelty, invasive nature, and lack of a cure, AMT-130 is still a viable and even worthwhile option for many people with Huntington’s disease.
Not yet out of clinical trials, AMT-130 could be the newest treatment out there, but for people with a disease that gets worse every year, the risk could be worth the reward. Many people are hesitant to adopt a treatment that has not been widely tested, as potential side effects or even drug efficacy are not yet known. However, the British Medical Journal studied the risks associated with participating in stage-1 clinical trials and determined that only 0.31% of participants experienced serious adverse effects during their respective studies. This includes adverse effects unrelated to the study and effects experienced by people given a placebo. Additionally, AMT-130 is the first treatment of its kind: despite many attempts to develop similar drugs over the years, AMT-130 is the only treatment available that addresses the root cause of Huntington’s disease and not just the symptoms. Because of this, its novelty should not be a significant barrier to widespread use of the treatment: as the adage goes, “beggars can’t be choosers”, and people will be amenable to the treatment since it is their only option to slow the progression of their terminal illness.
Brain surgery is not something that many people are willing to take on lightly, and costs, especially in countries that do not have free healthcare, can be a significant barrier to accessing treatment. Nevertheless, millions of people undergo cosmetic surgery every year purely to augment their looks, so one could argue that surgeries are not only feasible but a good option. For the same reason that people choose cosmetic surgery, AMT-130 has the ability to greatly bolster quality of life for people with Huntington’s disease, with some study participants even seeing a reversal in progression after 1.5 years that leaves them not only stable in terms of symptom progression, but actually better than before they got the treatment.
Unfortunately, this treatment still does not provide a cure for Huntington’s disease. It cannot stop the disease progression, but only slow it to a certain degree. Despite this, it provides hope for a group that has been largely without hope for as long as the disease has existed, and is a valuable jumping off point for new treatments to be developed and improved upon. For many, each additional week or even day that they can continue to spend with loved ones is worth the world, and AMT-130 provides an avenue to extend the life expectancy of people with Huntington’s disease, and make each year leading up to the end more enjoyable.
Overall, despite its novelty, invasive nature, and lack of a cure, AMT-130 is still a viable and even worthwhile option for many people with Huntington’s disease. From the point of view of someone personally unaffected by the disease, it is easy to conclude that the drawbacks of the treatment are simply too much to bear. When the time comes, however, that we or someone we know is diagnosed with a terminal illness, potential side effects pale in comparison to the reality of a disease that gets worse and worse as time goes on. While considering the scientific and societal implications for each treatment, we must also put ourselves in the shoes of those who are affected by the disease and would benefit from the treatment, because only they get to decide whether AMT-130 is beneficial or harmful to them. In the end, the treatment provides something invaluable and new to a long-suffering community 41,000 strong: hope.
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