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July 2018; 5 (4) ArticleOpen Access

Expanded autologous regulatory T-lymphocyte infusions in ALS

A phase I, first-in-human study

Jason R. Thonhoff, David R. Beers, Weihua Zhao, Milvia Pleitez, Ericka P. Simpson, James D. Berry, Merit E. Cudkowicz, Stanley H. Appel
First published May 18, 2018, DOI: https://doi.org/10.1212/NXI.0000000000000465
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Article Information

vol. 5 no. 4 e465
DOI: 
https://doi.org/10.1212/NXI.0000000000000465
PubMed: 
29845093
Published By: 
Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology
Online ISSN: 
2332-7812
History: 
  • Received February 6, 2018
  • Accepted in final form April 11, 2018
  • First Published May 18, 2018.
Copyright & Usage: 
Copyright © 2018 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology. This is an open access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives License 4.0 (CC BY-NC-ND), which permits downloading and sharing the work provided it is properly cited. The work cannot be changed in any way or used commercially without permission from the journal.

Author Disclosures

    1. Jason R. Thonhoff, MD, PhD*,
    2. David R. Beers, PhD*,
    3. Weihua Zhao, MD, PhD,
    4. Milvia Pleitez, MD,
    5. Ericka P. Simpson, MD,
    6. James D. Berry, MD,
    7. Merit E. Cudkowicz, MD and
    8. Stanley H. Appel, MD
  1. Jason R. Thonhoff, MD, PhD*,
    2. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  2. David R. Beers, PhD*,
    3. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  3. Weihua Zhao, MD, PhD,
    4. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  4. Milvia Pleitez, MD,
    5. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  5. Ericka P. Simpson, MD,
    6. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. Co-editor for Case files for Neurology McGraw Hill publication

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. Speaker for Alexion Pharmaceuticals, Inc on INdications for Soliris in Myasthenia Gravis Contracted Speaker for Spinraza in ALS

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. Endowed Chair support from philanthropic donations not related to publication

    Research Support, Foundations and Societies:

    1. NONE

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  6. James D. Berry, MD,
    7. Scientific Advisory Boards:

    1. (1) MT Pharma (commercial entity) - funding for attending a one-day scientific advisory meeting (2) Denali Therapeutics (commercial entity) - funding for attending a one- day scientific advisory meeting (3) Orion Corporation (commercial entity) - funding for attending a one- day scientific advisory meeting

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. (1) ALS Association (non-profit entity) - reimbursed travel to present research at Patient symposium in anew York (2) MDA (non-profit entity) - reimbursed to travel to conference to present ALS research (3) ALS One (non-profit entity) - reimbursed to travel to a meeting about ALS advocacy

    Editorial Boards:

    1. Wife (Ann Shinn) - Neuropsychopharmacology - Editorial Board Member

    Patents:

    1. MicroRNAs in neurodegenerative disorders (WO2013055865)

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. Neurologist Department of Neurology Neurological Clinical Research Institute Massachusetts General Hospital

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. (1) Voyager Therapeutics - Commercial Entity - Fellowship Funding (2) Neuraltus Pharmaceuticals - Commercial entity - Site Investigator for Trial (3) Cytokinetics - Commercial entity - Site Investigator for Trial (4) Brainstorm Cell Therapeutics - Commercial entity - Site Investigator for Trial (5) Amylyx Pharmaceuticals - Commercial entity - Site Investigator for Trial

    Research Support, Government Entities:

    1. (1) NIH/NINDS (PI: Steven M. Greenberg) Co-Investigator, 2011-2018 (2) NIH R01DC013547 (PI: Jordan Green) Co-PI, 2014-2019 (3) NIH/NINDS R21NS101673 (PI: Butovsky) Co-PI, 2017-2019

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. (1) Muscular Dystrophy Association (2) ALS Finding a Cure (3) ALS Association (4) ALS One

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. MicroRNAs in neurodegenerative disorders (WO2013055865) - Licensing royalty

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  7. Merit E. Cudkowicz, MD and
    8. Scientific Advisory Boards:

    1. Commercial, Cytokinetics SAB, Lilly DSMB

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. Neurotherapeutics editorial board JAMA Neurology editorial board

    Patents:

    1. Metabolomics in ALS - patent

    Publishing Royalties:

    1. UptoDate MND chapter

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. Cytokinetics, Biogen-Idec, Lilly, Orion, ImmunityPharm, Biohaven, Avexis, Revalesio

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NINDS - U01 NeuroNEXT

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. Muscular dystrophy association, als association, als finding a cure foundation

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  8. Stanley H. Appel, MD
    9. Scientific Advisory Boards:

    1. (1) Neuraltus,Commercial,Scientific Advisory Board (2) Mitsubishi Pharma,Commercial,Scientific Advisory Board

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Speaker's honorarium for Avanir, Inc

    Editorial Boards:

    1. NONE

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. Avanir, Inc

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. NONE

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. (1) ALS Association - 2016-2017 (2) ALS Finding a Cure (ALSFAC)- Lee Rizzuto Foundation,2016-2017.

    Stock/Stock Options/Board of Directors Compensation:

    1. NONE

    License Fee Payments, Technology or Inventions:

    1. NONE

    Royalty Payments, Technology or Inventions:

    1. NONE

    Stock/Stock Options, Research Sponsor:

    1. NONE

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. Expert consultant- legal procedings in ALS case-2016

  1. From the Houston Methodist Neurological Institute (J.R.T., D.R.B., W.Z., M.P., E.P.S., S.H.A.), Houston Methodist Hospital Research Institute, Stanley H. Appel Department of Neurology, Houston, TX; and Neurological Clinical Research Institute (J.D.B., M.E.C.), Massachusetts General Hospital, Boston, MA.
  1. Correspondence
    Dr. Appel sappel{at}houstonmethodist.org
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