Huntington’s disease (HD) specialists and scientists are cautiously optimistic about a clinical trial in which a gene therapy (known as AMT-130) slowed the disease’s progression by 75% in patient participants over three years. Preliminary results were announced in September 2025.

How AMT-130 Gene Therapy Works

HD is caused by a change (mutation) in the gene for a protein called huntingtin. AMT-130 gene therapy uses a harmless virus to carry special genetic instructions into the brain during an extensive surgical procedure. These instructions help reduce the amount of mutant huntingtin protein, which is what leads to HD symptoms.

A small group of 29 people with HD took part in the study, and only a few received the higher dose of AMT-130. That higher-dose group seemed to show the strongest benefits, including better overall function and improvements in motor and cognitive tests.

Dr. Bega, however, cautions that bringing the gene therapy to patients will be a slow process. Although it is a gene therapy, it is regulated like a drug and must gain the approval of the U.S. Food & Drug Administration (FDA) before reaching patients. It also requires more research. “Turning this into real-world application is another step that will require some patience,” he says. 

“Still, this is a great step forward and brings a lot of hope to all of us. This is the first time that any drug for HD has shown this type of finding on measures that the FDA would consider valuable,” Dr. Bega adds. 

Understanding Huntington’s Disease

Huntington’s disease is a rare, hereditary neurodegenerative disorder of the central nervous system, which affects movement, behavior and thinking. It progressively breaks down nerve cells and currently has no cure.

HD typically begins around age 40 and progresses over 10 to 20 years. About 41,000 Americans have symptoms of Huntington’s disease, and about 200,000 are at risk of inheriting it. If a parent has HD, their child has a 50% chance of developing it too.

Because it is genetically passed down, a blood test can detect the genetic mutation. “We look for a particular stretch of DNA that repeats itself too many times,” says Dr. Bega. Additional testing can include a computed tomography (CT) scan and magnetic resonance imaging (MRI).

Early symptoms include:

• Jerking movements/fidgeting
• Clumsiness
• Changes in gait
• Problems with complex tasks
• Depression
• Mood swings
• Involuntary muscle movements

“This type of involuntary muscle movement is known as chorea and becomes more severe as the disease progresses,” explains Dr. Bega.

Supporting Quality of Life Today

While the headlines focus on slowing disease progression, Dr. Bega emphasizes that caring for patients today remains the priority.

“We still need to focus on their mental health, physical health and current symptoms — these must be managed for people living with the disease today,” he says.

A Team Approach to Living With HD

Dr. Bega is the director of one of the few designated Huntington’s Disease Society of America (HDSA) Centers of Excellence in the United States, based at Northwestern University in conjunction with Northwestern Medicine.

He works with a network of specialists — from genetic counselors and social workers to neuropsychiatric experts — to manage HD symptoms and improve patients’ quality of life. This includes providing education and support to patients, family members and caretakers.

“We really discuss all aspects of the disease with the patient,” Dr. Bega explains. “The first visit is often anxiety-provoking, but it’s rewarding to see improvement in how someone feels over time.”

This commitment to comprehensive care and compassion is what drove Dr. Bega to specialize in movement disorders. “You’re dealing with intimate things that affect the quality of their life, and that’s important to me,” he says. “We’re able to make a difference, whether it’s improving walking, mood or simply providing education and support.”

The Future of Huntington’s Disease Treatment

Currently, there are no approved treatments to slow the progression of Huntington’s disease.

“This [AMT-130 clinical trial] is the first time we’re seeing not only a reduction in mutant huntington protein but also favorable clinical outcomes — the same measures used in everyday practice and reviewed by the FDA,” says Dr. Bega.

Dr. Bega believes meaningful advances are within reach.

“Because we know exactly what causes this disease, I actually believe we’re going to make a meaningful impact in my lifetime,” he says.

Learn more about this breakthrough in Huntington’s disease.