Huntington's disease successfully treated for first time

 

Yes — very recent reports indicate a major breakthrough: a gene therapy called AMT-130 appears to have for the first time slowed progression of Huntington’s disease in human patients. (Sky News)

Here are the key details, and also the caveats:

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✅ What seems to have happened

• In a clinical trial (involving 29 patients), those who received a high dose of AMT-130 showed about 75% slower progression of the disease after 36 months (3 years) compared to matched external controls. (CNBC)

• There was also a roughly 60% slower decline in functional abilities as a secondary outcome. (CNBC)

• The treatment is a one-time procedure: delivered via neurosurgery (injection of a viral vector into parts of the brain), after which cells produce microRNA to block mutant huntingtin protein (the toxic form that causes neuronal degeneration). (Українські Національні Новини (УНН))

• The treated patients had fewer markers of neuronal death (e.g. neurofilaments) than would be expected. (Medical Daily)

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⚠️ Important caveats

• “Slowing” disease progression is not the same as fully “curing” the disease. The therapy delays or reduces decline, but doesn’t reverse all damage. (CNBC)

• It appears this result is from a high dose group; the magnitude of benefit might depend significantly on dose and perhaps stage of disease. (CNBC)

• The surgical procedure is complex (12-to-20-hour brain surgery) and carries risks, costs, and logistic challenges. (mint)

• Long-term safety, durability of effect beyond 3 years, wider applicability (different genetic variants, stages of disease, etc.) are not yet fully known.

• Access may be an issue: cost, expertise (neurosurgery and specialized care), regulatory approvals needed.

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If you like, I can pull up the full paper (or press release) so we can see exactly who was treated, at what disease stage, and what the outcomes looked like in detail. Do you want me to do that?...READ MORE