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Scientists give emotional celebration as Huntington’s disease successfully treated for first time

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In a massive medical breakthrough, a research team has found a way to slow the progression of Huntington’s disease for the very first time using gene therapy.

Huntington’s disease is a genetically inherited, fatal neurological disorder that progressively damages nerve cells in the brain, causing problems with movement, cognition, and changes in behavior.

First classified in 1872 by American physician George Huntington, there has never been a cure for the condition. Existing treatments only aim to manage symptoms of Huntington’s, which tend to crop up in patients who are around 30 – 40 years of age.

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Now, more than a century later, scientists have discovered a way to successfully hinder its progress, leaving the medical community stunned and hopeful.

Unsplash.com: robina weermeijerHuntington’s disease is a fatal disorder that degrades brain cells over time.

Researchers slow Huntington’s disease by 75% with gene therapy

On September 24, 2025, a team of medical researchers shared the results of a trial they had conducted over the last three years, which found that a specific type of gene therapy can help slow the progression of Huntington’s disease.

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The condition is caused by a mutation of the huntingtin protein in the brain, transforming it into a toxin that attacks and kills other brain cells over time.

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Using a combination of gene therapy and gene splicing techniques, scientists were able to infuse a non-threatening ‘virus,’ a vehicle for the gene therapy that had been altered to contain a specific sequence of DNA, into several parts deep within patients’ brains.

Unsplash.com: National Cancer InstituteScientists have found a way to slow the spread of Huntington’s disease by 75% using gene therapy.

After insertion, the DNA then activates, attaching itself to messenger RNA and disrupting the process by which the huntingtin gene’s code is translated into proteins.

The delicate surgical operation takes anywhere from 12-18 hours to complete. Surgeons use a catheter and MRI imaging to inject the therapy, making it equal parts grueling for everyone involved and likely incredibly expensive.

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uniQure, a leading company in gene therapy, published the study’s results on September 24, showing that patients experienced an average 75% slowing of Huntington’s three years after undergoing treatment.

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Speaking to the BBC, the director of the University College London Huntington’s Disease Centre, Prof Tabrizi, gushed over the trial’s “spectacular” results.

“We never in our wildest dreams would have expected a 75% slowing of clinical progression,” she told the outlet.

Prof Ed Wild, a consultant neurologist at the National Hospital for Neurology and Neurosurgery at UCLH, said he got a “bit teary” at the news.

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“There was every chance that we would never see a result like this, so to be living in a world where we know this is not only possible, but the actual magnitude of the effect is breathtaking, it’s very difficult to fully encapsulate the emotion,” he said.



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Climate Change Is Bringing Legionnaire’s Disease to a Town Near You
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Climate Change Is Bringing Legionnaire’s Disease to a Town Near You

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This story originally appeared on Vox and is part of the Climate Desk collaboration.

Air conditioners have been working overtime this hot summer, from those tiny window units to the massive AC towers that serve the tightly packed apartment buildings in major cities. And while they bring the relief of cool air, these contraptions also create the conditions for dangerous bacteria to multiply and spread.

One particularly nasty bacteria-borne illness is currently spreading in New York City using those enormous cooling units as its vector: Legionnaire’s disease. The bacterial pneumonia, which usually recurs each summer in the US’s largest city, has sickened more than 100 people and killed five in a growing outbreak.

If you don’t live in New York City or the Northeast, you may never have heard of Legionnaire’s, but this niche public health threat may not be niche for much longer.

Climate change is helping to make Legionnaire’s disease both more plentiful in the places where it already exists and creating the potential for it to move to new places where the population may not be accustomed to it. Cities in the Northeast and Midwest, where hotter weather meets older infrastructure, have reported more cases in recent years. Recently, Legionella bacteria was discovered in a nursing home’s water system in Dearborn, Michigan—one of the states, along with Ohio, Pennsylvania, Illinois, and Wisconsin, that have seen more activity in the past few years.

Anyone can contract Legionnaire’s disease by inhaling tiny drops containing the bacteria, and the symptoms—fever, headache, shortness of breath—appear within days. It can cause a severe lung infection, with a death rate of around 10 percent.

While healthier people often experience few symptoms, the more vulnerable—young children, the elderly, pregnant people, and those with compromised immune systems—face serious danger from the illness. Around 5,000 people die every year in the United States from Legionnaire’s disease, many of them living in low-income housing with outdated cooling equipment where the bacteria can more readily grow and spread.

Legionnaire’s disease is a microcosm of climate change’s impact on low-income communities. As warmer temperatures facilitate the spread of disease, the most socially vulnerable populations are going to pay the steepest price.

The Collision of Legionnaire’s Disease, Climate Change, and Economic Disparities

Legionnaire’s disease was first documented after an unusually aggressive pneumonia outbreak during an American Legion conference in Philadelphia in 1976. Soon, Centers for Disease Control and Prevention scientists confirmed the cause of the mysterious illness: a previously unknown bacteria that was accordingly named Legionella. Legionella, unfortunately, is everywhere—in streams, lakes, and water pipes across the country.

But usually, it occurs in such low concentrations and is so remote that it doesn’t pose a threat to humans. Usually.

Now, city health officials have found the bacteria in the large cooling tanks that serve massive apartment buildings across New York City, particularly in Harlem. Cooling tanks are ideal places for Legionnaire’s to grow and spread. They’re filled with stagnant, warm water that is more hospitable to bacterial growth. Like an evaporative cooler, the systems convert warm stagnant water into cool air for apartment dwellers. They can spray mists laden with the bacteria into the open air, dispersing it across the surrounding air, where it can enter a person’s lungs when they inhale. According to the Environmental Protection Agency, 80 percent of Legionnaire’s cases are linked to potable water systems.



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