Evidence map›Paper›PMID 31740545›Full record

Trial reportNeurology2019

NurOwn, phase 2, randomized, clinical trial in patients with ALS: Safety, clinical, and biomarker results.

James D Berry, Merit E Cudkowicz, Anthony J Windebank, Nathan P Staff, Margaret Owegi, Katherine Nicholson, Diane McKenna-Yasek, Yossef S Levy, Natalie Abramov, Haggai Kaspi and 4 more

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

Trial report in Neurology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05695521 (Phase 1 Safety Run-in Study and Phase 1b Randomized, Double Blinded, Placebo Controlled Trial), which is not on this map. Cited by 97 papers, 7 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
97citing papers in PubMed, 7 pooled it
11.3field-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.

NCT05695521 phase1active not recruitingnot on this mapstarted 2023, after this paper: background citation

Phase 1 Safety Run-in Study and Phase 1b Randomized, Double Blinded, Placebo Controlled Trial

TypeinterventionalSponsorCellenkos, Inc.Ran2023 to 2027Enrolled66ConditionsAmyotrophic Lateral SclerosisArmsCK0803, Excipient
3 · Its place in the literature

Who cites it

97 citing papers in PubMed, 7 syntheses or guidelines pooled it, 155 citations in OpenAlex.

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

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

14 authors at 2 institutions in 1 country.

James D BerryFrom Massachusetts General Hospital Neurological Clinical Research Institute and Harvard Medical School (J.D.B., M.E.C., K.N.), Boston; Mayo Clinic (A.J.W., N.P.S.), Rochester, MN; University of Massachusetts (M.O., D.M.-Y., R.H.B.), Worcester; Brainstorm Cell Therapeutics (Y.S.L., N.A., H.K., R.A., Y.G.), Petach Tikva, Israel; and Tigermed USA (M.M.), Somerset, NJ.
Merit E CudkowiczFrom Massachusetts General Hospital Neurological Clinical Research Institute and Harvard Medical School (J.D.B., M.E.C., K.N.), Boston; Mayo Clinic (A.J.W., N.P.S.), Rochester, MN; University of Massachusetts (M.O., D.M.-Y., R.H.B.), Worcester; Brainstorm Cell Therapeutics (Y.S.L., N.A., H.K., R.A., Y.G.), Petach Tikva, Israel; and Tigermed USA (M.M.), Somerset, NJ.
Anthony J WindebankFrom Massachusetts General Hospital Neurological Clinical Research Institute and Harvard Medical School (J.D.B., M.E.C., K.N.), Boston; Mayo Clinic (A.J.W., N.P.S.), Rochester, MN; University of Massachusetts (M.O., D.M.-Y., R.H.B.), Worcester; Brainstorm Cell Therapeutics (Y.S.L., N.A., H.K., R.A., Y.G.), Petach Tikva, Israel; and Tigermed USA (M.M.), Somerset, NJ.
Nathan P StaffFrom Massachusetts General Hospital Neurological Clinical Research Institute and Harvard Medical School (J.D.B., M.E.C., K.N.), Boston; Mayo Clinic (A.J.W., N.P.S.), Rochester, MN; University of Massachusetts (M.O., D.M.-Y., R.H.B.), Worcester; Brainstorm Cell Therapeutics (Y.S.L., N.A., H.K., R.A., Y.G.), Petach Tikva, Israel; and Tigermed USA (M.M.), Somerset, NJ.
Margaret OwegiFrom Massachusetts General Hospital Neurological Clinical Research Institute and Harvard Medical School (J.D.B., M.E.C., K.N.), Boston; Mayo Clinic (A.J.W., N.P.S.), Rochester, MN; University of Massachusetts (M.O., D.M.-Y., R.H.B.), Worcester; Brainstorm Cell Therapeutics (Y.S.L., N.A., H.K., R.A., Y.G.), Petach Tikva, Israel; and Tigermed USA (M.M.), Somerset, NJ.
Katherine NicholsonFrom Massachusetts General Hospital Neurological Clinical Research Institute and Harvard Medical School (J.D.B., M.E.C., K.N.), Boston; Mayo Clinic (A.J.W., N.P.S.), Rochester, MN; University of Massachusetts (M.O., D.M.-Y., R.H.B.), Worcester; Brainstorm Cell Therapeutics (Y.S.L., N.A., H.K., R.A., Y.G.), Petach Tikva, Israel; and Tigermed USA (M.M.), Somerset, NJ.
Diane McKenna-YasekFrom Massachusetts General Hospital Neurological Clinical Research Institute and Harvard Medical School (J.D.B., M.E.C., K.N.), Boston; Mayo Clinic (A.J.W., N.P.S.), Rochester, MN; University of Massachusetts (M.O., D.M.-Y., R.H.B.), Worcester; Brainstorm Cell Therapeutics (Y.S.L., N.A., H.K., R.A., Y.G.), Petach Tikva, Israel; and Tigermed USA (M.M.), Somerset, NJ.
Yossef S LevyFrom Massachusetts General Hospital Neurological Clinical Research Institute and Harvard Medical School (J.D.B., M.E.C., K.N.), Boston; Mayo Clinic (A.J.W., N.P.S.), Rochester, MN; University of Massachusetts (M.O., D.M.-Y., R.H.B.), Worcester; Brainstorm Cell Therapeutics (Y.S.L., N.A., H.K., R.A., Y.G.), Petach Tikva, Israel; and Tigermed USA (M.M.), Somerset, NJ.
Natalie AbramovFrom Massachusetts General Hospital Neurological Clinical Research Institute and Harvard Medical School (J.D.B., M.E.C., K.N.), Boston; Mayo Clinic (A.J.W., N.P.S.), Rochester, MN; University of Massachusetts (M.O., D.M.-Y., R.H.B.), Worcester; Brainstorm Cell Therapeutics (Y.S.L., N.A., H.K., R.A., Y.G.), Petach Tikva, Israel; and Tigermed USA (M.M.), Somerset, NJ.
Haggai KaspiFrom Massachusetts General Hospital Neurological Clinical Research Institute and Harvard Medical School (J.D.B., M.E.C., K.N.), Boston; Mayo Clinic (A.J.W., N.P.S.), Rochester, MN; University of Massachusetts (M.O., D.M.-Y., R.H.B.), Worcester; Brainstorm Cell Therapeutics (Y.S.L., N.A., H.K., R.A., Y.G.), Petach Tikva, Israel; and Tigermed USA (M.M.), Somerset, NJ.
Munish MehraFrom Massachusetts General Hospital Neurological Clinical Research Institute and Harvard Medical School (J.D.B., M.E.C., K.N.), Boston; Mayo Clinic (A.J.W., N.P.S.), Rochester, MN; University of Massachusetts (M.O., D.M.-Y., R.H.B.), Worcester; Brainstorm Cell Therapeutics (Y.S.L., N.A., H.K., R.A., Y.G.), Petach Tikva, Israel; and Tigermed USA (M.M.), Somerset, NJ.
Revital ArichaFrom Massachusetts General Hospital Neurological Clinical Research Institute and Harvard Medical School (J.D.B., M.E.C., K.N.), Boston; Mayo Clinic (A.J.W., N.P.S.), Rochester, MN; University of Massachusetts (M.O., D.M.-Y., R.H.B.), Worcester; Brainstorm Cell Therapeutics (Y.S.L., N.A., H.K., R.A., Y.G.), Petach Tikva, Israel; and Tigermed USA (M.M.), Somerset, NJ.
Yael GothelfFrom Massachusetts General Hospital Neurological Clinical Research Institute and Harvard Medical School (J.D.B., M.E.C., K.N.), Boston; Mayo Clinic (A.J.W., N.P.S.), Rochester, MN; University of Massachusetts (M.O., D.M.-Y., R.H.B.), Worcester; Brainstorm Cell Therapeutics (Y.S.L., N.A., H.K., R.A., Y.G.), Petach Tikva, Israel; and Tigermed USA (M.M.), Somerset, NJ.
Robert H BrownFrom Massachusetts General Hospital Neurological Clinical Research Institute and Harvard Medical School (J.D.B., M.E.C., K.N.), Boston; Mayo Clinic (A.J.W., N.P.S.), Rochester, MN; University of Massachusetts (M.O., D.M.-Y., R.H.B.), Worcester; Brainstorm Cell Therapeutics (Y.S.L., N.A., H.K., R.A., Y.G.), Petach Tikva, Israel; and Tigermed USA (M.M.), Somerset, NJ. robert.brown@umassmed.edu.
University of Massachusetts Chan Medical School · USMayo Clinic in Arizona · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectiveTo determine the safety and efficacy of mesenchymal stem cell (MSC)-neurotrophic factor (NTF) cells (NurOwn®, autologous bone marrow-derived MSCs, induced to secrete NTFs) delivered by combined intrathecal and intramuscular administration to participants with amyotrophic lateral sclerosis (ALS) in a phase 2 randomized controlled trial.

methodsThe study enrolled 48 participants randomized 3:1 (treatment: placebo). After a 3-month pretransplant period, participants received 1 dose of MSC-NTF cells (n = 36) or placebo (n = 12) and were followed for 6 months. CSF was collected before and 2 weeks after transplantation.

resultsThe study met its primary safety endpoint. The rate of disease progression (Revised ALS Functional Rating Scale [ALSFRS-R] slope change) in the overall study population was similar in treated and placebo participants. In a prespecified rapid progressor subgroup (n = 21), rate of disease progression was improved at early time points (

conclusionA single-dose transplantation of MSC-NTF cells is safe and demonstrated early promising signs of efficacy. This establishes a clear path forward for a multidose randomized clinical trial of intrathecal autologous MSC-NTF cell transplantation in ALS. CLASSIFICATION OF EVIDENCE: This phase II study provides Class I evidence.

Indexed as

AdultAgedAmyotrophic Lateral SclerosisDouble-Blind MethodFemaleHumansMaleMesenchymal Stem Cell TransplantationMiddle AgedNerve Growth FactorsTransplantation, AutologousNerve Growth Factors

Identifiers

PMID31740545
PMCPMC6937497
OpenAlexW2987229198

What OpenQuestion holds

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LicenceCC BY-NC-ND
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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.