NurOwn, phase 2, randomized, clinical trial in patients with ALS
Safety, clinical, and biomarker results
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Article Information
- Received January 30, 2019
- Accepted in final form July 30, 2019
- First Published November 18, 2019.
Article Versions
- Previous version (November 18, 2019 - 12:45).
- You are viewing the most recent version of this article.
Author Disclosures
- James D. Berry, MD,
- Merit E. Cudkowicz, MD,
- Anthony J. Windebank, MD,
- Nathan P. Staff, MD,
- Margaret Owegi, MD,
- Katherine Nicholson, MD,
- Diane McKenna-Yasek,
- Yossef S. Levy, PhD,
- Natalie Abramov, MSc,
- Haggai Kaspi, PhD,
- Munish Mehra, PhD,
- Revital Aricha, PhD,
- Yael Gothelf, PhD and
- Robert H. Brown, DPhil, MD
- James D. Berry, MD,
(1) MT Pharma (commercial entity) - attended one-day scientific advisory meeting (2) Denali Therapeutics (commercial entity) - attended one-day scientific advisory meeting (3) Orion Corporation (commercial entity) - attended one- day scientific advisory meeting (4) Maxim Group (commercial entity) - one lecture about ALS research (5) Anelixis Therapeutics (commercial entity) - advisor to company (6) Mayo Clinic (non-profit entity) - DSMB member for ALS stem cell trial
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(1) ALS Association (non-profit entity) - reimbursed for travel to ALS patient symposium (2) MDA (non-profit entity) - reimbursed for travel to scientific conference (3) ALS One (non-profit entity) - reimbursed for travel to ALS advocacy meeting (4) ALS Finding a Cure (non-profit entity) - reimbursed for travel to ALS research meetings
Wife (Ann Shinn) - Neuropsychopharmacology - Editorial Board Member
MicroRNAs in neurodegenerative disorders (WO2013055865)
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(1) Neuraltus Pharmaceuticals - Commercial entity - Site Investigator for Trial (2) Cytokinetics - Commercial entity - Site Investigator for Trial (3) Brainstorm Cell Therapeutics - Commercial entity - Site Investigator for Trial (4) Amylyx Pharmaceuticals - Commercial entity - Site Investigator for Trial (5) MT Pharma - Commercial entity - Principle Investigator for study (6) Biogen - Commercial entity - Principle Investigator for study (7) Genentech - Commercial entity - Site Investigator for Trial
(1) NIH/NINDS (PI: Steven M. Greenberg) Co-Investigator, 2011-2018 (2) NIH R01DC013547 (PI: Jordan Green) Co-PI, 2014-2019 (3) NIH/NINDS R21 NS101673 (PI: Butovsky) Co-PI, 2017-2019 (4) NIH/NINDS R21 NS104609 (PI: Butovsky) Co-PI, 2018-2020 (5) NIH-R01 DC017291 (PIs: Green and Yunusova) Co-I, 2018- 2023
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(1) Muscular Dystrophy Association (2) ALS Finding a Cure (3) ALS Association (4) ALS One
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MicroRNAs in neurodegenerative disorders (WO2013055865) - Licensing royalty
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- Merit E. Cudkowicz, MD,
Lilly DSMB
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Neurotherapeutics editorial board JAMA Neurology editorial board
Metabolomics in ALS - patent MIR 155 inhibitor - patent
UptoDate MND chapter
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Cytokinetics, Biogen, Orion, ImmunityPharm, Biohaven, Avexis, Revalesio, MT Pharma
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NINDS - U01 NeuroNEXT
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Muscular dystrophy association, als association, als finding a cure foundation, ALS One
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- Anthony J. Windebank, MD,
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Paracelsus Medical University, Salzburg Austria (Scientific Advisory Board) (travel support only) National University of Ireland Galway, Ireland (CURAM) (Scientific Advisory Board) (travel support only)
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US Patent #7,163,545 Spinal cord surgical implant
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(1) BrainStorm Therapeutics - Clinical trial for the use of neurotrophically enhanced autologous stem cells in patients with ALS. Support is only for patient care and investigator effort dedicated to the trial.
CSCR15IRG002 / FR00081599 Windebank (PI) 5/28/2016 Â 5/28/2018 New Jersey Commission on Spinal Cord Research A cell-assembled matrix template to guide nerve regeneration in the spinal cord UL1TR 000135-10 Khosla (PI) 09/01/17 Â 08/31/21 NCRR Mayo Clinic Center for Clinical and Translational Research Goal: The goal of the program is to provide infrastructure and education programs to accelerate clinical and translational science. Role: Coinvestigator UL1TR 000135-10 09/01/17 Â 08/31/21 NCRR Mayo Clinic Center for Clinical and Translational Research Goal: The goal of this project is to provide education programs for PhD and medical students in clinical and translational science. Role: Principal Investigator W81XWH-14-2-003 Windebank (PI) 01/01/14 Â 12/31/18 Department of Defense AFIRM II: Tissue Engineering for repair of critical nerve gaps Goal: This project is to design and test a combination device (scaffolds and cells) for repair of peripheral nerves. Role: Principal Investigator K08CA 169443 (Mentor for Nathan Staff, MD, PhD) 07/01/2012 Â 06/30/2017 National Cancer Institute Mechanisms of Bortezomib-induced Peripheral Neuropathy Goal: This project studies the cellular mechanism of Bortezomib-induced peripheral neuropathy. Role: Mentor 2T32EB005583 Kohn (PI) 7/1/2012 Â 6/30/2017 NIH/NIBIB Postdoctoral Training in Translational Research in Regenerative Medicine Goal: This NRSA postdoctoral training program offers collaborative research experiences built on core disciplines of biomaterials science, cell biology and pharmaceutical science. A geographically dispersed training community of eight trainees and 15 faculty mentors will engage in translational research to develop regenerative therapies based on tissue engineering, stem cells and drug delivery approaches. Role: Co-Principal Investigator
Princeton University Wang (PI) 8/1/2016 Â 7/31/2017 Effect of extracellular matrix on promoting nerve regeneration across conduit repaired nerve defects Princeton University Wang (PI) 5/16/2016 Â 4/30/2017 Effect of extracellular matrix on promoting nerve regeneration across conduit repaired nerve defects
Bowen Family Foundation for spinal cord injury research
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- Nathan P. Staff, MD,
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Dr. Staff is a co-investigator for the ALS clinical trial from BrainStorm Cell Therapeutics.
(1)NIH/NCI,(K08)CA169443, PI, 5 years total (completed July 2017) (2)NIH/NCATS, UL1TR 000135-10, co-investigator, 5 years total (in last year) (3)NIH/NCI, (R01)CA215973, co-investigator, 2 years total (4)State of Minnesota, RMM 11215 CT002, PI, 2 years total (in last year)
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(1) Target ALS
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- Margaret Owegi, MD,
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- Katherine Nicholson, MD,
(1) Scientific advisory board for Biohaven Pharmaceuticals (2) Scientific advisory board for Avanir Pharmaceuticals
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(1) Brainstorm Therapeutics (for role as Co-Investigator)
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(1) ALS Finding a Cure (2) ALS Association (3) Muscular Dystrophy Association (4) Target ALS (5) Salah Foundation
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- Diane McKenna-Yasek,
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RO1-NS050641-04 salary support for D. McKenna-Yasek
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- Yossef S. Levy, PhD,
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Methods of Generating Mesenchymal Stem Cells which secrete Neurotrophic Factors Method of Qualifying cells
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Brainstorm Cell Therapeutics, VP Cell Production, 14 years
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Brainstorm Cell Therapeutics Employee
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- Natalie Abramov, MSc,
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Methods for treating Amyotrophic Lateral Sclerosis
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Brainstorm Cell Therapeutics Research Associate 9 years
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Brainstorm Cell Therapeutics Employee
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- Haggai Kaspi, PhD,
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Methods for treating amyotrophic lateral sclerosis (als)
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Brainstorm Cell Therapeutics, R&D associate, 3
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- Munish Mehra, PhD,
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Funding for travel for meetings with Brainstorm cell therapeutics team members
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Tigermed, Director, 3.5 years
Brainstorm Cell Therapeutics
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Brainstorm Cell Therapeutics
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- Revital Aricha, PhD,
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METHODS FOR TREATING AMYOTROPHIC LATERAL SCLEROSIS (ALS)
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Brainstorm Cell Therapeutics employee. VP R&D 4 years
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- Yael Gothelf, PhD and
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1) Methods of Generating Mesenchymal Stem Cells which secrete Neurotrophic factors 2) Method of qualifying cells 3) Methods for treating Amyotrophic Lateral Sclerosis
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Brainstorm Cell Therapeutics Vice President Scientific and Regulatory Affairs 10 years
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Brainstorm Cell Therapeutics - employee
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- Robert H. Brown, DPhil, MD
Kirac Foundation ALS Research Laboratory, 2009-2019 Above and Beyond review panel, 2018 Servier - Scientific Advisory Board 2019 Project ALS 2000- ALS - Finding a Cure 2016- NIH - NINDS Board of Scientific Counsellors 2016- Warren Alpert Foundation - Board Member 2018- APIC-Bio Scientific Co-founder and consultant 2018 -
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Above and Beyond 2018 Kyoto meeting, FP Pharmaceuticals 2018 Servier, Inc 2019
ALS and Other Motor Neuron Disorders, reviewer, 2008-2015
patent on aspects of superoxide dismutase in ALS, filed 1993
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Consultant - Voyager Therapeutics - ∼ 2015-2017 APIC-BIO, 2018-2019 Servier - 2019
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Wave Lifesciences 2016-2018
NINDS, RO1, ALS genetics, (R. Brown, PI) NINDS, UO1, ALS therapies (R. Brown, PI)
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ALS Therapy Alliance: SAB and grantee, 2002-2015 Angel Fund for ALS Research: SAB and grantee, 1997-2019 Pierre L. deBourgknecht ALS Research Fund: grantee, 1984- 2019 Day Neuromuscular Research Foundation: grantee, 1984-2019 CureALS, 2014-2015 Project ALS, 1998-2016 ALS-Finding A Cure ∼ 2015-2019 MAPP-One ALS ∼2016-2019 Target ALS - 2016-2019 ALSOne -2-15-2019
Voyager Therapeutics - ∼ 2015 Imstar Therapeutics 2017- Amylyx Therapeutics 2017- Warren Alpert Foundation - board of trustees - 2019
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Royalty fees from Mass General Hospital, for licensing fees for use of transgenic ALS (SOD1) mice
Voyager Therapeutics
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- From 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.
- Correspondence
Dr. Brown robert.brown{at}umassmed.edu
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