Trial reportNature communications2019
First-in-human trial of blood-brain barrier opening in amyotrophic lateral sclerosis using MR-guided focused ultrasound.
Trial report in Nature communications, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 229 papers, 3 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
229 citing papers in PubMed, 3 syntheses or guidelines pooled it, 473 citations in OpenAlex.
- Neuromodulation effects of low-intensity transcranial focused ultrasound in human, a systematic review focusing on motor and sensory functions.Journal of neuroengineering and rehabilitation · 2025Pooled it
- Focused ultrasound-mediated enhancement of blood-brain barrier permeability for brain tumor treatment: a systematic review of clinical trials.Journal of neuro-oncology · 2024Pooled it
- MRI Guided Focused Ultrasound-Mediated Delivery of Therapeutic Cells to the Brain: A Review of the State-of-the-Art Methodology and Future Applications.Frontiers in neurology · 2021Pooled it
- Pilot study of repeated blood-brain barrier disruption in patients with mild Alzheimer's disease with an implantable ultrasound device.Alzheimer's research & therapy · 2022Trial
- A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Trial
- Microbubble-free mechanical sonoporation for MR-guided drug delivery in solid tumours: a proof-of-concept study.Drug delivery and translational research · 2026Article
- Recent Advancements in Drug Delivery Across the Blood-Brain Barrier in Amyotrophic Lateral Sclerosis (ALS).CNS neuroscience & therapeutics · 2026Review
- Noninvasive focal gene transfer of chemogenetic proteins in the primate brain.Cell reports methods · 2026Article
- Focused ultrasound in pediatric neuro-oncology: Current applications and future directions.Neuro-oncology advances · 2026Review
- Noninvasive enrichment of circulating tumor biomarkers in a mouse model of diffuse midline glioma using focused ultrasound.Neuro-oncology advances · 2026Review
- Applications of transcranial focused ultrasound for primary brain tumors.Neuro-oncology advances · 2026Review
- Intranasal vs. Device-Assisted Drug Delivery: Advantages and Limitations for the Delivery of Biopharmaceuticals to the CNS.Pharmaceutics · 2026Review
- Safety and neuromodulation effect of transcranial focused ultrasound for motor recovery in patients with subacute stroke.Journal of neuroengineering and rehabilitation · 2026Article
- Crossing the blood-brain barrier: nanoparticle-based strategies for neurodegenerative disease therapy.Drug delivery and translational research · 2026Review
- Rigid Hollow Microparticles for Enhanced Focused Ultrasound Treatment Under Optoacoustic Guidance.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Edaravone: Advances on cytoprotective effects, pharmacological properties, and mechanisms of action.Pharmacological reviews · 2026Review
- Focused ultrasound-enhanced nose-to-brain delivery of a therapeutic antibody in a large-animal model.Theranostics · 2026Article
- From Ablation to Neuromodulation Platform: The Evolving Role of Magnetic Resonance-Guided Focused Ultrasound in Functional Neurosurgery.Journal of clinical neurology (Seoul, Korea) · 2026Review
- Triple-negative complementary metamaterial for ultrasound transmission through elastic barrier.Scientific reports · 2025Article
- Computational Fluid Dynamic Model Prediction of Enhanced Glymphatic Clearance in Response to Focused Ultrasound-Mediated Blood-Brain Barrier Opening.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Article
169 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
12 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
MR-guided focused ultrasound (MRgFUS) is an emerging technology that can accurately and transiently permeabilize the blood-brain barrier (BBB) for targeted drug delivery to the central nervous system. We conducted a single-arm, first-in-human trial to investigate the safety and feasibility of MRgFUS-induced BBB opening in eloquent primary motor cortex in four volunteers with amyotrophic lateral sclerosis (ALS). Here, we show successful BBB opening using MRgFUS as demonstrated by gadolinium leakage at the target site immediately after sonication in all subjects, which normalized 24 hours later. The procedure was well-tolerated with no serious clinical, radiologic or electroencephalographic adverse events. This study demonstrates that non-invasive BBB permeabilization over the motor cortex using MRgFUS is safe, feasible, and reversible in ALS subjects. In future, MRgFUS can be coupled with promising therapeutics providing a targeted delivery platform in ALS.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.