Evidence map›Paper›PMID 37217980›Full record

ArticleTranslational neurodegeneration2023

An open-label multiyear study of sargramostim-treated Parkinson's disease patients examining drug safety, tolerability, and immune biomarkers from limited case numbers.

Katherine E Olson, Mai M Abdelmoaty, Krista L Namminga, Yaman Lu, Helen Obaro, Pamela Santamaria, R Lee Mosley, Howard E Gendelman

Registry-linked trialOpen access · goldAbstract read
In one paragraph

Article in Translational neurodegeneration, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03790670 (Safety, Tolerability and Biomarker Assessments of Leukine), which is not on this map. Cited by 18 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
18citing papers in PubMed, 1 pooled it
6.4field-weighted citation impact, top 3% 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.

NCT03790670 phase1completednot on this map

Safety, Tolerability and Biomarker Assessments of Leukine (Sargramostim) During Extended Timed Treatment for Parkinson's Disease: A Phase I Pilot Study

TypeinterventionalSponsorUniversity of NebraskaRan2019 to 2024Enrolled7ConditionsParkinson DiseaseArmssargramostim
3 · Its place in the literature

Who cites it

18 citing papers in PubMed, 1 synthesis or guideline pooled it, 31 citations in OpenAlex.

  1. Pooled it
  2. Trial
  3. Article
  4. Immune-mediated excitotoxicity in brain disorders.Nature reviews. Immunology · 2026
    Review
  5. Review
  6. The Progress of Active Immunotherapy for Parkinson's Disease.International journal of molecular sciences · 2026
    Review
  7. Article
  8. Article
  9. Review
  10. Review
  11. Neuroprotection in Parkinson Disease.Neurology and therapy · 2025
    Review
  12. Review
  13. Article
  14. Review
  15. Article
  16. Review
  17. Immune senescence in aged APP/PS1 mice.NeuroImmune pharmacology and therapeutics · 2023
    Article
  18. 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

8 authors at 2 institutions in 1 country.

Katherine E Olson *Department of Pharmacology and Experimental Neuroscience, Center for Neurodegenerative Disorders, University of Nebraska Medical Center, Omaha, NE, 68198, USA.
Mai M Abdelmoaty *Department of Pharmacology and Experimental Neuroscience, Center for Neurodegenerative Disorders, University of Nebraska Medical Center, Omaha, NE, 68198, USA.
Krista L NammingaDepartment of Pharmacology and Experimental Neuroscience, Center for Neurodegenerative Disorders, University of Nebraska Medical Center, Omaha, NE, 68198, USA.
Yaman LuDepartment of Pharmacology and Experimental Neuroscience, Center for Neurodegenerative Disorders, University of Nebraska Medical Center, Omaha, NE, 68198, USA.
Helen ObaroGreat Plains Center for Clinical and Translational Research, Nebraska Medicine, Omaha, NE, USA.
Pamela SantamariaNeurology Consultants of Nebraska, PC and Nebraska Medicine, Omaha, NE, USA.
R Lee MosleyDepartment of Pharmacology and Experimental Neuroscience, Center for Neurodegenerative Disorders, University of Nebraska Medical Center, Omaha, NE, 68198, USA.
Howard E GendelmanDepartment of Pharmacology and Experimental Neuroscience, Center for Neurodegenerative Disorders, University of Nebraska Medical Center, Omaha, NE, 68198, USA. hegendel@unmc.edu.
University of Nebraska Medical Center · USNebraska Medical Center · US

Funding

UNMC Structural Biology CoreP20GM103427 · NIGMS · UNIVERSITY OF NEBRASKA MEDICAL CENTER · PI PAUL L SORGEN · 2012 to 2026
$59.2M
Neuroprotective Immunity and HIV DementiaR01NS034239 · NINDS · UNIVERSITY OF NEBRASKA MEDICAL CENTER · PI Howard E Gendelman, R Lee Mosley · 1995 to 2026
$12.3M
Targeted lipid nanoparticles for gene therapeutics delivery approach to eradicating HIV reservoirsR01MH121402 · NIMH · UNIVERSITY OF NEBRASKA MEDICAL CENTER · PI Benson Edagwa, Howard E Gendelman · 2019 to 2026
$4.9M
NIGMS NIH HHS P20 GM103427NIMH NIH HHS R01 MH121402NINDS NIH HHS R01 NS034239
6 · The paper itself

Abstract

backgroundThe clinical utility and safety of sargramostim has previously been reported in cancer, acute radiation syndrome, autoimmune disease, inflammatory conditions, and Alzheimer's disease. The safety, tolerability, and mechanisms of action in Parkinson's disease (PD) during extended use has not been evaluated.

methodsAs a primary goal, safety and tolerability was assessed in five PD patients treated with sargramostim (Leukine

resultsSargramostim-associated adverse events included injection-site reactions, elevated total white cell counts, and bone pain. On drug, blood analyses and metabolic panels revealed no untoward side effects linked to long-term treatment. Unified Parkinson's Disease Rating Scale scores remained stable throughout the study while regulatory T cell number and function were increased. In the initial 6 months of treatment, transcriptomic and proteomic monocyte tests demonstrated autophagy and sirtuin signaling. This finding paralleled anti-inflammatory and antioxidant activities within both the adaptive and innate immune profile arms.

conclusionsTaken together, the data affirmed long-term safety as well as immune and anti-inflammatory responses reflecting clinical stability in PD under the sargramostim treatment. Confirmation in larger patient populations is planned in a future phase II evaluation.

trial registrationClinicalTrials.gov: NCT03790670, Date of Registration: 01/02/2019, URL: https://clinicaltrials.gov/ct2/show/NCT03790670?cond=leukine+parkinson%27s&draw=2&rank=2 .

Indexed as

Granulocyte-Macrophage Colony-Stimulating FactorParkinson DiseaseBiomarkersHumansProteomicsRecombinant ProteinsBiomarkersGranulocyte-Macrophage Colony-Stimulating FactorRecombinant ProteinssargramostimGranulocyte–macrophage colony-stimulating factorNeuroprotectionParkinson’s diseaseRegulatory T cellsSargramostim therapyUnified Parkinson’s Disease Rating Scale

Identifiers

PMID37217980
PMCPMC10201023
OpenAlexW4377293982

What OpenQuestion holds

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LicenceCC BY
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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.