Brain gene therapy shows promise for rare childhood disease
NCT ID NCT01801709
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested a gene therapy for children with early-onset metachromatic leukodystrophy (MLD), a rare and severe brain disease. Five children aged 6 months to 5 years received injections of a harmless virus carrying a working copy of the ARSA gene directly into their brains. The goal was to see if the treatment is safe and can slow the disease's progression.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
gene therapy (AAVrh.10cuARSA)
What this could lead to
If successful, this could slow or stop the progression of metachromatic leukodystrophy in young children, potentially preserving motor and cognitive function.
What could go wrong
This is a very early, small trial (only 5 children) with no control group. The treatment involves direct brain injections, which carry risks like infection or brain injury. It may not work or could have unknown side effects.
Disclaimer
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Bicêtre Hospital - Paris Sud
Le Kremlin-Bicêtre, France