Evidence map›Paper›PMID 40696909›Full record

ReviewJournal of child neurology2026

Inflammation and Immunomodulation in Cerebral X-linked Adrenoleukodystrophy: Review of Pathology and Interventions.

Melissa A Wright, Courtney Demmitt-Rice, Keith P Van Haren, Troy C Lund, Florian Eichler, Joshua L Bonkowsky

Abstract readReview
In one paragraph

Review in Journal of child neurology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from 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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Article
  2. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors.

Melissa A WrightDivision of Pediatric Neurology, Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT, USA.ORCID 0000-0002-7804-9627
Courtney Demmitt-RiceDivision of Pediatric Neurology, Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT, USA.
Keith P Van HarenDepartment of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA, USA.
Troy C LundDivision of Pediatric Blood and Marrow Transplantation & Cellular Therapy, Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Florian EichlerDepartment of Neurology, Massachusetts General Hospital, Boston, MA, USA.
Joshua L BonkowskyDivision of Pediatric Neurology, Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, UT, USA.ORCID 0000-0001-8775-147X

Funding

Validation of the GMFC-MLDU54NS115052 · NINDS · CHILDREN'S HOSP OF PHILADELPHIA · PI Laura Ann Adang, FLORIAN S EICHLER · 2019 to 2026
$14.3M
The Utah Regional Network for Excellence in Neuroscience Clinical Trials (UR-NEXT)U24NS107156 · NINDS · UTAH STATE HIGHER EDUCATION SYSTEM--UNIVERSITY OF UTAH · PI Joshua Leitch Bonkowsky, John Robinson Singleton · 2018 to 2026
$3.2M
NINDS NIH HHS U24 NS107156NINDS NIH HHS U54 NS115052
6 · The paper itself

Abstract

ObjectivesMore than half of males with X-linked adrenoleukodystrophy (ALD) develop progressive, inflammatory cerebral demyelination (cerebral adrenoleukodystrophy). Treatment for cerebral adrenoleukodystrophy is limited with no standard therapies for advanced cerebral adrenoleukodystrophy. We reviewed cerebral adrenoleukodystrophy literature and expert opinion, compiling immunopathology, biomarkers, and therapies tested.MethodsWe reviewed published literature from January 1, 1970, through November 1, 2024, and surveyed expert clinicians worldwide caring for cerebral adrenoleukodystrophy patients for unpublished agent use.ResultsWe identified 20 publications with primary data on human cerebral adrenoleukodystrophy immunopathology. Seventeen publications reported cerebral adrenoleukodystrophy biomarkers. We identified 14 publications reporting use of 7 different agents; unpublished clinician reports identified use of 9 different agents.ConclusionsCerebral adrenoleukodystrophy immunopathology represents complex dysregulation of cytokines, macrophages, T cells, astrocytes, oligodendrocytes, and microglia. Partial responses to cerebral adrenoleukodystrophy were noted with intravenous immunoglobulin, sirolimus, leriglitazone, and mycophenolate. Our findings suggest consideration for a randomized platform trial of immunomodulatory agents for advanced cerebral adrenoleukodystrophy.

Indexed as

AdrenoleukodystrophyImmunomodulationInflammationHumansALDcerebral ALDimmunopathologyleukodystrophyX-linked adrenoleukodystrophy

Identifiers

PMID40696909
PMCPMC12313269

What Socratic holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

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 Socratic graph.