New Treatments & Clinical Trials for Huntington's Disease

Huntington's disease is a fatal inherited brain disorder caused by a single gene mutation. It affects about 1 in 10,000 people in the U.S., causing progressive movement problems, thinking difficulties, and psychiatric symptoms. No treatment yet slows the disease, so current care focuses on managing symptoms as they appear.

What's actually going on in research

Trials are testing drugs that lower huntingtin protein levels in the brain, including antisense oligonucleotides and gene therapies. Researchers are also studying drugs that might protect brain cells from damage, treatments for chorea and other movement symptoms, and ways to delay symptom onset in people who carry the gene but haven't yet developed symptoms.

What to know before you search

Eligibility typically depends on genetic test confirmation, disease stage (early, middle, late, or presymptomatic), and functional abilities measured by standard Huntington's rating scales.

What types of trials are currently open

• Huntingtin-lowering trials — Testing drugs given by spinal injection or brain infusion to reduce levels of the harmful huntingtin protein. These may require regular dosing over many months.
• Neuroprotection trials — Testing drugs that might protect brain cells from damage without directly targeting huntingtin. These are often pills taken daily.
• Symptom management trials — Testing treatments for chorea, psychiatric symptoms, thinking problems, and other disease manifestations.
• Presymptomatic trials — Studies in people who carry the gene but have no symptoms yet, testing whether early intervention can delay disease onset.
• Observational studies — Following people with Huntington's over time to understand how the disease progresses and what predicts faster or slower decline.