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July 10, 2018; 91 (2 Supplement 1) ArticleOpen Access

Considerations for development of therapies for cutaneous neurofibroma

Sharad K. Verma, Vincent M. Riccardi, Scott R. Plotkin, Hubert Weinberg, R. Rox Anderson, Jaishri O. Blakeley, Kurt Jarnagin, James Lee
First published July 9, 2018, DOI: https://doi.org/10.1212/WNL.0000000000005791
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Article Information

vol. 91 no. 2 Supplement 1 S21-S30
DOI: 
https://doi.org/10.1212/WNL.0000000000005791
PubMed: 
29987132
Published By: 
Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology
Print ISSN: 
0028-3878
Online ISSN: 
1526-632X
History: 
  • Received November 1, 2017
  • Accepted in final form April 23, 2018
  • First Published July 9, 2018.
Copyright & Usage: 
© 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 License 4.0 (CC BY), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

Author Disclosures

    1. Sharad K. Verma, PhD,
    2. Vincent M. Riccardi, MD, MBA,
    3. Scott R. Plotkin, MD, PhD,
    4. Hubert Weinberg, MD,
    5. R. Rox Anderson, MD,
    6. Jaishri O. Blakeley, MD,
    7. Kurt Jarnagin, PhD and
    8. James Lee, MD, PhD
  1. Sharad K. Verma, PhD,
    2. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for Travel or Speaker Honoraria:

    1. NONE

    Editorial Boards:

    1. (1) Translational Genetics and Genomics, Editorial Advisory board member (non-compensated), 2016 - present.

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. (1) Genentech (2) Bristol-Myers-Squibb

    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. (1) National Comprehensive Cancer Network Stock/Stock Options, Medical Equipment & Materials: (1) GlaxoSmithKline (2008 - present) (2) Epizyme (2016 - present)

    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. Vincent M. Riccardi, MD, MBA,
    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. Scott R. Plotkin, MD, PhD,
    4. Scientific Advisory Boards:

    1. NONE

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Dr. Plotkin has been reimbursed by the American Academy of Neurology and the Children?s Tumor Foundation for travel for educational activities.

    Editorial Boards:

    1. Journal of Neuro-oncology, editorial board, started 7/1/2014. Unpaid

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. Commercial entity. NFlection Therapeutics (a company focused on developing therapies for NF1)

    Speakers' Bureaus:

    1. NONE

    Other activities:

    1. Founder of NFlection Therapeutics (with other individuals)

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. NONE

    Research Support, Government Entities:

    1. Dr. Plotkin has received research support from the NIH (1R13NS084619-01, PI 2013-2014; 1 R01 CA201130-01A1; co-PI), Department of Defense (W81XWH-12-1-0155 and W81XWH-16-1-010)

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. Dr. Plotkin has received research support from an anonymous foundation.

    Stock/Stock Options/Board of Directors Compensation:

    1. Stock for commercial entity NFlection Therapeutics (not on board of directors but as founder) Non-financial disclosures: As noted above, I am involved with the company NFlection therapeutics which is involved in developing treatments for NF1-related tumors.

    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. Hubert Weinberg, 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. R. Rox Anderson, 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. 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

  6. Jaishri O. Blakeley, MD,
    7. Scientific Advisory Boards:

    1. I served in an uncompensated role on a Scientific Advisory Board for Novartis in 2010. I served as a paid consultant for a medical advisory board for Abbvie June 2015 and as a paid consultant for Abbvie for clinical trial design for a glioblastoma quality of life study in 2016. I currently sit on the Medical Advisory Council for the Children's Tumor Foundation

    Gifts:

    1. NONE

    Funding for travel or speaker honoraria:

    1. Travel to a scientific conference in Novemebr 2017 during which I participated in a panel discussion about the drug selumetinib for brain tumors and plexiform neurofibromas. Travel expenses were paid for by AstraZenica.

    Editorial Boards:

    1. Guest editor, Experimental Neurology, January 2018

    Patents:

    1. NONE

    Publishing Royalties:

    1. NONE

    Employment, Commercial Entity:

    1. NONE

    Consultancies:

    1. I served as a compensated consultant to Abbvie in June 2015 for glioblastoma study design and as a paid consultant for Abbvie for clinical trial design for glioblastoma quality of life in 2016.

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. Glaxo Smith Kline non-salary research support for vestibular schwannoma clinical trial with lapatinib; Sanofi non-salary research support for glioma clinical trial; Lily non-salary research support for glioma clinical trial

    Research Support, Government Entities:

    1. NIH/NIBIB, R01EB009731; Co-investogator, 07/01/09- 04/30/13; Department of Defense Neurofibromatosis Clinical Trial Consortium site PI

    Research Support, Academic Entities:

    1. NONE

    Research Support, Foundations and Societies:

    1. Children's Tumor Fundation; Brain Tumor Funders Collaborative; American Society of Clinical Oncology

    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

  7. Kurt Jarnagin, PhD and
    8. 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. BioPharm Tech a consulting organization 2017-present -- no relationship to this work Pfizer -- 2016-2017 -- no relationship to this work Anacor Pharmaceuticals -- 2008-2016 -- no relationship to this work

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. Jarnagin servers as a general consultant to BioPharma industry. Those activities cover many areas, but none within the topic areas of the papers at issue here.

    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. Anacor was purchased by Pfizer in 2016. As such Jarnagin received stock option-based compensation in 2016. No relationship to this work.

    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. James Lee, MD, PhD
    9. 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. I am an employee of Dermavant Sciences

    Consultancies:

    1. NONE

    Speakers' Bureaus:

    1. NONE

    Other Activities:

    1. NONE

    Clinical Procedures or Imaging Studies:

    1. NONE

    Research Support, Commercial Entities:

    1. I am an employee of Dermavant Sciences

    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. I receive stock options as an employee of Dermavant Sciences

    Stock/Stock Options, Medical Equipment & Materials:

    1. NONE

    Legal Proceedings:

    1. NONE

  1. From the Department of Neurology (S.K.V., J.O.B.), The Johns Hopkins University School of Medicine, The Neurofibromatosis Therapeutic Acceleration Program, Baltimore, MD; The NF Institute (V.M.R.), La Crescenta, CA; Department of Neurology and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston; Division of Plastic Surgery (H.W.), Mount Sinai Hospital, Icahn School of Medicine at Mount Sinai, New York, NY; Department of Dermatology (R.R.A.), Harvard Medical School, Boston, MA; BioPharm Tech (K.J.), San Mateo, CA; and Dermavant Sciences (J.L.), Durham, NC.
  1. Correspondence
    Dr. Verma sverma20{at}jhmi.edu
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