New Treatments & Clinical Trials for Huntington's Disease

Huntington's disease is a fatal inherited brain disorder caused by a single genetic mutation. About 30,000 people in the United States have symptomatic Huntington's, with another 200,000 at risk of inheriting it. No treatments slow disease progression yet, but dozens of trials are testing gene-silencing therapies and other approaches to delay or prevent symptoms.

What's actually going on in research

Trials are testing antisense oligonucleotides and other gene-silencing methods to reduce the mutant huntingtin protein. Researchers are also studying drugs that protect brain cells from damage, treatments for movement and psychiatric symptoms, and ways to measure disease progression before symptoms appear. Some trials now enroll people who carry the mutation but have no symptoms yet.

What to know before you search

Eligibility depends on genetic testing confirming the Huntington's mutation, disease stage (presymptomatic versus symptomatic), and functional ability.

What types of trials are currently open

• Gene-silencing trials — Testing drugs that reduce production of the mutant huntingtin protein, delivered by spinal injection or gene therapy. These aim to slow or stop disease progression.
• Neuroprotection trials — Testing drugs that may protect brain cells from damage caused by mutant huntingtin, even without lowering the protein itself.
• Symptom trials — Testing treatments for movement problems, psychiatric symptoms like depression and irritability, or cognitive decline.
• Presymptomatic trials — Studies in people who carry the Huntington's mutation but have no symptoms, testing whether early intervention can delay disease onset.
• Natural history studies — Following people with Huntington's over time to understand how the disease progresses and develop better outcome measures for trials.