Evidence mapPaperPMID 32719487Full record

Trial reportNature medicine2020

Assessment of cognitive and neural recovery in survivors of pediatric brain tumors in a pilot clinical trial using metformin.

Ramy Ayoub, Rebecca M Ruddy, Elizabeth Cox, Adeoye Oyefiade, Daniel Derkach, Suzanne Laughlin, Benjamin Ades-Aron, Zahra Shirzadi, Els Fieremans, Bradley J MacIntosh and 6 more

5 registry-linked trialsOpen access · greenAbstract readClinical Trial, Phase IIIRandomized Controlled Trial
In one paragraph

Trial report in Nature medicine, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 5 registered trials, which are not on this map. Cited by 69 papers, 4 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
69citing papers in PubMed, 4 pooled it
9.2field-weighted citation impact, top 2% of its field
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.

NCT02040376 phase3completednot on this map

Placebo Controlled Double Blind Crossover Trial of Metformin for Brain Repair in Children With Cranial-Spinal Radiation for Medulloblastoma

TypeinterventionalSponsorThe Hospital for Sick ChildrenRan2014 to 2017Enrolled24ConditionsBrain Tumor Treated With Cranial or Cranial-Spinal RadiationArmsMetformin, Placebo
NCT05298670 phase2unknown statusnot on this mapstarted 2022, after this paper: background citation

Drug Repurposing Using Metformin for Improving the Therapeutic Outcome in Multiple Sclerosis Patients

TypeinterventionalSponsorGerman University in CairoRan2022 to 2023Enrolled80ConditionsMultiple SclerosisArmsMetFORMIN 1000 Mg Oral Tablet, Interferon beta-1a
NCT05853601 phase1 / phase2active not recruitingnot on this mapstarted 2023, after this paper: background citation

Theophylline Prophylaxis During Hypothermia to Limit Neonatal Nephron Damage

TypeinterventionalSponsorMedical College of WisconsinRan2023 to 2027Enrolled30ConditionsAcute Kidney Injury, HIEArmsSingle Dose Theophylline, Repeat Dose Theophylline
NCT06779487 recruitingnot on this mapstarted 2025, after this paper: background citation

Systemic Biomarkers to Predict Radiation-Induced Neurocognitive Decline in Pediatric and Young Adults With Primary Brain Tumors (BIO-RIN)

TypeobservationalSponsorTata Memorial CentreRan2025 to 2032Enrolled200ConditionsBrain Tumors, Medulloblastoma, Glioma, Ependymoma
NCT07120971 early_phase1recruitingnot on this mapstarted 2025, after this paper: background citation

A Phase 0/Phase 1 Trial of Metformin for Premature Infants With Bronchopulmonary Dysplasia

TypeinterventionalSponsorMedical College of WisconsinRan2025 to 2030Enrolled40ConditionsBronchopulmonary Dysplasia (BPD)ArmsMetformin (open-label)
3 · Its place in the literature

Who cites it

69 citing papers in PubMed, 4 syntheses or guidelines pooled it, 119 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Pooled it
  4. Pooled it
  5. Trial
  6. Trial
  7. Radiomitigators: Breakthroughs in Post-Radiation Recovery.Antioxidants (Basel, Switzerland) · 2026
    Review
  8. Social information processing in survivors of pediatric brain tumors.Journal of cancer survivorship : research and practice · 2026
    Article
  9. Review
  10. Article
  11. Article
  12. Review
  13. Review
  14. Article
  15. Article
  16. Review
  17. Review
  18. Metabolic stress-mediated cell death and autophagy in human lung cancer cells.The Journal of pharmacology and experimental therapeutics · 2025
    Article
  19. Approaches to Reduce Toxicity in Pediatric Brain Tumors.Current oncology (Toronto, Ont.) · 2025
    Review
  20. Article

9 more citing papers are in PubMed but not listed here.

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

16 authors at 4 institutions in 2 countries.

Ramy Ayoub *Neurosciences and Mental Health Program, Research Institute, Hospital for Sick Children, Toronto, Ontario, Canada.ORCID http://orcid.org/0000-0001-7099-9284
Rebecca M Ruddy *Institute of Medical Science, University of Toronto, Toronto, Ontario, Canada.
Elizabeth CoxNeurosciences and Mental Health Program, Research Institute, Hospital for Sick Children, Toronto, Ontario, Canada.
Adeoye OyefiadeNeurosciences and Mental Health Program, Research Institute, Hospital for Sick Children, Toronto, Ontario, Canada.
Daniel DerkachInstitute of Medical Science, University of Toronto, Toronto, Ontario, Canada.ORCID http://orcid.org/0000-0002-8515-9795
Suzanne LaughlinDiagnostic Imaging, Hospital for Sick Children, Toronto, Ontario, Canada.
Benjamin Ades-AronCenter for Biomedical Imaging, Department of Radiology, New York University Grossman School of Medicine, New York, NY, USA.ORCID http://orcid.org/0000-0001-5077-3341
Zahra ShirzadiDepartment of Medical Biophysics, University of Toronto, Toronto, Ontario, Canada.
Els FieremansCenter for Biomedical Imaging, Department of Radiology, New York University Grossman School of Medicine, New York, NY, USA.
Bradley J MacIntoshDepartment of Medical Biophysics, University of Toronto, Toronto, Ontario, Canada.ORCID http://orcid.org/0000-0001-7300-2355
Cynthia B de MedeirosNeurosciences and Mental Health Program, Research Institute, Hospital for Sick Children, Toronto, Ontario, Canada.ORCID http://orcid.org/0000-0003-4997-1994
Jovanka SkocicNeurosciences and Mental Health Program, Research Institute, Hospital for Sick Children, Toronto, Ontario, Canada.
Eric BouffetDivision of Haematology/Oncology, Hospital for Sick Children, Toronto, Ontario, Canada.
Freda D MillerNeurosciences and Mental Health Program, Research Institute, Hospital for Sick Children, Toronto, Ontario, Canada.
Cindi M MorsheadInstitute of Medical Science, University of Toronto, Toronto, Ontario, Canada. cindi.morshead@utoronto.ca.
Donald J MabbottNeurosciences and Mental Health Program, Research Institute, Hospital for Sick Children, Toronto, Ontario, Canada. donald.mabbott@sickkids.ca.ORCID http://orcid.org/0000-0001-6161-5066
University of Toronto · CAHospital for Sick Children · CANew York University · USSunnybrook Health Science Centre · CA

Funding

TR&D 3: Revealing Microstructure: Biophysical Modeling and Validation for Discovery and Clinical CareP41EB017183 · NEW YORK UNIVERSITY SCHOOL OF MEDICINE · 2025 to 2025
$1.3M
NIBIB NIH HHS P41 EB017183
6 · The paper itself

Abstract

We asked whether pharmacological stimulation of endogenous neural precursor cells (NPCs) may promote cognitive recovery and brain repair, focusing on the drug metformin, in parallel rodent and human studies of radiation injury. In the rodent cranial radiation model, we found that metformin enhanced the recovery of NPCs in the dentate gyrus, with sex-dependent effects on neurogenesis and cognition. A pilot double-blind, placebo-controlled crossover trial was conducted (ClinicalTrials.gov, NCT02040376) in survivors of pediatric brain tumors who had been treated with cranial radiation. Safety, feasibility, cognitive tests and MRI measures of white matter and the hippocampus were evaluated as endpoints. Twenty-four participants consented and were randomly assigned to complete 12-week cycles of metformin (A) and placebo (B) in either an AB or BA sequence with a 10-week washout period at crossover. Blood draws were conducted to monitor safety. Feasibility was assessed as recruitment rate, medication adherence and procedural adherence. Linear mixed modeling was used to examine cognitive and MRI outcomes as a function of cycle, sequence and treatment. We found no clinically relevant safety concerns and no serious adverse events associated with metformin. Sequence effects were observed for all cognitive outcomes in our linear mixed models. For the subset of participants with complete data in cycle 1, metformin was associated with better performance than placebo on tests of declarative and working memory. We present evidence that a clinical trial examining the effects of metformin on cognition and brain structure is feasible in long-term survivors of pediatric brain tumors and that metformin is safe to use and tolerable in this population. This pilot trial was not intended to test the efficacy of metformin for cognitive recovery and brain growth, but the preliminary results are encouraging and warrant further investigation in a large multicenter phase 3 trial.

Indexed as

AdolescentAdultBrainBrain NeoplasmsCancer SurvivorsChildChild, PreschoolCognitionCognitive DysfunctionDouble-Blind MethodFemaleHumansMagnetic Resonance ImagingMaleMetforminNeurogenesisMetformin

Identifiers

PMID32719487
PMCPMC8176964
OpenAlexW3044625941

What Socratic holds

Textmetadata
LicenceTDM
Read underepoch 390

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 Socratic graph.