Gene Therapy Slows Huntington’s Disease in Early Trial
A brutal genetic disease passed down in families — and at last, a treatment that doesn’t just mask symptoms, but slows its advance.
A Patient Story
She was in her mid-50s, suffering from Huntington’s chorea. By the time I met her, she had no control over her limbs or bowels. I was called in to place a new feeding tube, so she could at least get nutrition directly into her stomach.
Before this devastating disease had taken hold, she had been a successful professional — well-regarded in her field, the kind of person colleagues respected and friends admired. She had raised two wonderful kids, both in college, neither of whom would be afflicted by Huntington’s because they didn’t carry the gene.
But those kids had to watch their mom change. They watched her body betray her, her movements become violent and uncontrollable, her personality blur. Huntington’s disease can strip away the person you once knew, leaving behind someone you barely recognize.
What Huntington’s Disease Is
Huntington’s disease (HD) is a genetic disorder inherited in an autosomal dominant pattern. That means if only one parent has the defective gene, each child has a 50% chance of inheriting it.
Some key facts:
The gene is located on chromosome 4 — not a sex chromosome, so men and women are equally affected.
If you inherit the gene, you will eventually develop the disease.
If you don’t inherit the gene, you are in the clear — and you can’t pass it to your kids.
Unlike many genetic conditions, having two copies of the gene doesn’t worsen the disease or come on earlier. One copy is enough.
Symptoms usually start between 35 and 55. What begins with subtle forgetfulness or clumsy movements progresses to involuntary jerks, mood swings, memory loss, and finally the total loss of independence.
Why Today’s News Matters
For decades, we’ve had no way to slow Huntington’s. All we could do was treat symptoms. In the case of my patient, keep her comfortable and feed her through a tube. The disease is devastating. But now, there’s a glimpse of hope.
A new gene therapy trial from uniQure shows that in a small group of patients, disease progression slowed by 75% over three years compared with controls. Not stopped, not cured — but slowed. In the world of Huntington’s, that’s groundbreaking.
The treatment uses a harmless virus to deliver a microRNA into brain cells. Think of it as a molecular muzzle that silences the faulty Huntington gene. Fewer toxic proteins are produced, neurons live longer, and patients hold on to independence for more years.
The Caveats
This was a tiny study — just 29 people. The procedure is not minor: it requires MRI-guided brain surgery, drilling into the skull, and infusing the therapy directly into the striatum. And the likely cost will be over $1 million per person.
So while this is the clearest signal yet that we can actually alter Huntington’s progression, it’s far from ready for everyone.
The Bigger Picture
Other companies, like Spark Therapeutics (owned by Roche), are also racing to test similar approaches. CRISPR-based strategies may follow. For the first time, instead of asking if we can change Huntington’s course, researchers are asking how well, how safely, and how affordably.
For families who’ve lived with the shadow of Huntington’s, that shift alone is monumental.
Huntington’s in the Public Eye
When we talk about Huntington’s disease, it’s easy to feel like an abstract diagnosis, something rare that happens to “other people.” But history and culture remind us otherwise. Some of the most familiar names in music, film, and science have lived with HD — or worked tirelessly to fight it.
Woody Guthrie and his family
The folk singer-songwriter Woody Guthrie — best known for “This Land Is Your Land” — had Huntington’s. Like so many in his era, his symptoms were first mistaken for alcoholism or mental illness, and he was institutionalized multiple times before his death from complications of the disease in 1967. Guthrie had inherited HD from his mother, Nora Belle Guthrie. His son, Arlo Guthrie, not only carried on the music tradition, but also became a passionate advocate for HD research, co-founding the Huntington’s Disease Society of America (HDSA).
Other notable voices
Charles Sabine — the former NBC News foreign correspondent — learned in 1994 that his father had HD, and later discovered he too carried the gene. He has since become one of the most visible international advocates, working to destigmatize HD.
Marianna Palka — actress and filmmaker, best known from the Netflix series GLOW. She tested positive for the HD gene and made the award-winning documentary The Lion’s Mouth Opens about her decision to be tested and her family’s story.
Scott Porter — actor from Friday Night Lights and Ginny & Georgia. While not gene-positive himself, he has spoken openly about HD’s impact on his family and his ongoing fight for awareness.
George Rainsford — British actor and patron of the Huntington’s Disease Association, who became an advocate after portraying a character with HD on the TV series Casualty.
Nancy Wexler — a scientist whose mother died of HD. Wexler’s landmark research with Venezuelan families ultimately led to the discovery of the HD gene itself — a turning point in genetics and in our understanding of inherited disease.
These individuals have used their voices — in music, media, and science — to shed light on a disease that too often leaves families feeling isolated. Their stories remind us that Huntington’s is not just a genetic code on chromosome 4. It is mothers, fathers, sons, and daughters. It is human.
Good News in Medicine
Closing Thoughts
I’m going to keep bringing you these bits of good news from medicine — stories where science is pushing the frontier and giving hope where there once was none.
A special thank-you to the paid members who make this work possible. Your support helps me share these updates and keep them accessible.
And let’s not forget: breakthroughs like this don’t happen by accident. They come from decades of basic research, often funded quietly and consistently. We need to keep investing — not just in rare diseases like Huntington’s, but also in the more common ones that affect millions. Because when we fund science, we fund hope.