MTM-CNM Family Connection
  • Home
  • About
    • About MTM/CNM
    • About Us
    • Contact
    • Newsletters
    • Traveling Turtle
  • Family Conference
    • 2019
    • Previous Conferences >
      • 2017
      • 2015
      • 2013
      • 2011
      • 2009
  • Resources
  • Research
    • Research Studies
    • Drug Approval Overview
    • Selected Publications
  • Fundraising
  • Blog


Our Blog

Community Conversations: Tamoxifen for MTM

7/20/2020

2 Comments

 
Our "Community Conversations" series, co-hosted by MTM-CNM Family Connection and Where There's A Will There's A Cure, continued on July 13th 2020, with an excellent presentation and Q&A from Dr. Jim Dowling of the Hospital for Sick Children in Toronto, Ontario.  There are several therapeutic approaches being studied for X-Linked Myotubular Myopathy, all borne out of years of hard work and research by many people, but this one also came about with a bit of "serendipity".  Through an incidental and unexpected outcome in the laboratory while working with a mouse model of MTM, Dr. Dowling's team discovered that the anti-cancer drug tamoxifen seems to have a positive effect on the disease.  This finding was also published by another team of researchers working completely independently.  While the exact mechanism of how tamoxifen effects the disease processes in MTM is still unknown, the preclinical data from the mice is compelling.  Dr. Dowling is now preparing to launch a human clinical trial to test the safety and efficacy of tamoxifen as a treatment for XLMTM.  Watch the replay of the presentation (recorded live on Zoom) here:
2 Comments
James
10/10/2020 08:51:14 am

This isn’t true. 3 kids have died since I’ve choose to put my child in this treatment.

Reply
MTM-CNM Family Connection
10/10/2020 09:30:56 am

James, you are confusing this trial (repurposing Tamoxifen) with a completely different trial for XLMTM (gene therapy). It is true that, tragically, there have been three patients in the gene therapy trial that have passed away over the last few months and that program is currently on hold pending investigation. https://www.audentestx.com/press_release/audentes-therapeutics-provides-update-on-the-aspiro-clinical-trial-evaluating-at132-in-patients-with-x-linked-myotubular-myopathy/

Dr. Dowling's trial discussed in this video does not involve gene therapy at all, but rather uses an existing medicine in a new way. It is hoped - but not yet proven - that this medicine will be safe for patients with MTM. As of this writing, the Tamoxifen trial has not yet begun in the United States.

If you would like to discuss in more detail, please feel free to send an email to info@mtm-cnm.org. We hope you and your family are doing well!

Reply

Your comment will be posted after it is approved.


Leave a Reply.

    MTM-CNM Family Connection's Blog

    Sharing information, resources, updates, and thoughts on various topics of interest to the MTM-CNM community.

    Archives

    July 2020
    November 2019
    October 2019
    July 2019
    June 2019
    May 2019
    February 2019
    January 2019
    November 2018
    October 2018
    July 2017
    March 2017
    November 2016
    October 2016

    Categories

    All
    Patient Advocacy
    Patient Care
    RARE Disease Community
    Research News

    RSS Feed

Picture
Copyright 2014-2022 MTM-CNM Family Connection, Inc 
Admin
  • Home
  • About
    • About MTM/CNM
    • About Us
    • Contact
    • Newsletters
    • Traveling Turtle
  • Family Conference
    • 2019
    • Previous Conferences >
      • 2017
      • 2015
      • 2013
      • 2011
      • 2009
  • Resources
  • Research
    • Research Studies
    • Drug Approval Overview
    • Selected Publications
  • Fundraising
  • Blog