One-of-a-Kind ALS drug shows promise in first human test
NCT ID NCT07095712
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This trial tested a custom-made drug called nL-TARD-001 in one person with ALS caused by a specific genetic mutation in TARDBP. The drug is an antisense oligonucleotide designed to target the faulty gene. The study measured changes in muscle function, breathing, and thinking over 12 months, along with safety and biomarker levels.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- nL-TARD-001 (personalized antisense oligonucleotide)
- What this could lead to
- If successful, this could point toward a personalized treatment for ALS caused by TARDBP mutations, potentially slowing disease progression.
- What could go wrong
- This is a very early, single-participant study, so results may not apply to others. The drug is experimental, and safety or effectiveness is not yet proven.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
Locations
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Columbia University, Irving Medical Center
New York, New York, 10032, United States
-
Houston Methodist
Houston, Texas, 77030, United States
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