Evidence map›Paper›PMID 36064599›Full record

Trial reportNature medicine2022

Transplantation of human neural progenitor cells secreting GDNF into the spinal cord of patients with ALS: a phase 1/2a trial.

Robert H Baloh, J Patrick Johnson, Pablo Avalos, Peggy Allred, Soshana Svendsen, Genevieve Gowing, Kristina Roxas, Amanda Wu, Becky Donahue, Sheryl Osborne and 28 more

Registry-linked trialOpen access · hybridAbstract readClinical Trial, Phase IClinical Trial, Phase II
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Nature medicine, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02943850 (Human Neural Progenitor Cells Secreting Glial Cell Line-Derived Neurotrophic Factor), which is not on this map. Cited by 93 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
93citing papers in PubMed, 1 pooled it
16.8field-weighted citation impact, top 1% 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.

NCT02943850 phase1completednot on this map

Human Neural Progenitor Cells Secreting Glial Cell Line-Derived Neurotrophic Factor (CNS10-NPC-GDNF) for the Treatment of Amyotrophic Lateral Sclerosis

TypeinterventionalSponsorCedars-Sinai Medical CenterRan2017 to 2019Enrolled18ConditionsAmyotrophic Lateral SclerosisArmsStem cell (HPC) implantation, Stereotactic surgical device
3 · Its place in the literature

Who cites it

93 citing papers in PubMed, 1 synthesis or guideline pooled it, 146 citations in OpenAlex.

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

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

38 authors at 3 institutions in 3 countries.

Robert H Baloh *Board of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
J Patrick Johnson *Cedars-Sinai Spine Center, Los Angeles, CA, USA.
Pablo Avalos *Board of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Peggy AllredDepartment of Neurology, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Soshana SvendsenBoard of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Genevieve GowingBoard of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Kristina RoxasBoard of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Amanda WuBoard of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.ORCID http://orcid.org/0000-0001-9546-1472
Becky DonahueBoard of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Sheryl OsborneBoard of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
George LawlessBoard of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Brandon ShelleyBoard of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Koral WheelerDepartment of Neurology, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Carolyn PrinaDepartment of Neurology, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Dana FineDepartment of Neurology, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Tami Kendra-RomitoDepartment of Neurology, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Haniah StokesDepartment of Neurology, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Vicki ManoukianDepartment of Neurology, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Abirami MuthukumaranDepartment of Neurology, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Leslie GarciaBoard of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Maria G BañuelosBoard of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Marlesa GodoyBoard of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA.ORCID http://orcid.org/0000-0002-7012-1612
Catherine BreseeBiostatistics Core, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Hong YuDepartment of Neurology, Massachusetts General Hospital, Boston, MA, USA.
Doniel DrazinDepartment of Neurosurgery, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Lindsey RossDepartment of Neurosurgery, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Robert NaruseDepartment of Anesthesiology, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Harish BabuDepartment of Neurosurgery, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Eric A MacklinBiostatistics Center, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Ashley VoCedars-Sinai Comprehensive Transplant Center, Los Angeles, CA, USA.
Ashraf ElsayeghDepartment of Pulmonary and Critical Care, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Warren TourtellotteDepartment of Neurology, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Marcel MayaDepartment of Imaging, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Matthew BurfordDepartment of Neurology, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Frank DiazDepartment of Neurology, Cedars-Sinai Medical Center, Los Angeles, CA, USA.ORCID http://orcid.org/0000-0003-3526-1852
Chirag G PatilDepartment of Neurosurgery, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Richard A LewisDepartment of Neurology, Cedars-Sinai Medical Center, Los Angeles, CA, USA.
Clive N SvendsenBoard of Governors Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA, USA. Clive.svendsen@cshs.org.ORCID http://orcid.org/0000-0001-8696-3446
Cedars-Sinai Medical Center · USHarvard University · USMassachusetts General Hospital · US

Funding

Elp1 function in Familial DysautonomiaR01NS095894 · NINDS · NORTHWESTERN UNIVERSITY AT CHICAGO · PI TOURTELLOTTE, WARREN G · 2016 to 2019
$1.6M
Mentoring and Research in Mouse PathobiologyK26OD010945 · OD · NORTHWESTERN UNIVERSITY AT CHICAGO · PI TOURTELLOTTE, WARREN G · 2012 to 2014
$471k
Mentoring and Research in Mouse PathobiologyK26RR026099 · NCRR · NORTHWESTERN UNIVERSITY AT CHICAGO · PI TOURTELLOTTE, WARREN G · 2010 to 2011
$317k
NCRR NIH HHS K26 RR026099NIH HHS K26 OD010945NINDS NIH HHS R01 NS095894
6 · The paper itself

Abstract

Amyotrophic lateral sclerosis (ALS) involves progressive motor neuron loss, leading to paralysis and death typically within 3-5 years of diagnosis. Dysfunctional astrocytes may contribute to disease and glial cell line-derived neurotrophic factor (GDNF) can be protective. Here we show that human neural progenitor cells transduced with GDNF (CNS10-NPC-GDNF) differentiated to astrocytes protected spinal motor neurons and were safe in animal models. CNS10-NPC-GDNF were transplanted unilaterally into the lumbar spinal cord of 18 ALS participants in a phase 1/2a study (NCT02943850). The primary endpoint of safety at 1 year was met, with no negative effect of the transplant on motor function in the treated leg compared with the untreated leg. Tissue analysis of 13 participants who died of disease progression showed graft survival and GDNF production. Benign neuromas near delivery sites were common incidental findings at post-mortem. This study shows that one administration of engineered neural progenitors can provide new support cells and GDNF delivery to the ALS patient spinal cord for up to 42 months post-transplantation.

Indexed as

Amyotrophic Lateral SclerosisNeural Stem CellsAnimalsDisease Models, AnimalGlial Cell Line-Derived Neurotrophic FactorHumansSpinal CordSuperoxide DismutaseGlial Cell Line-Derived Neurotrophic FactorSuperoxide Dismutase

Identifiers

PMID36064599
PMCPMC9499868
OpenAlexW4294636879

What OpenQuestion holds

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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.