Website Edition: February/March 2009

The Dystonia Medical Research Foundation: Our Mission and Commitment

By Janet Hieshetter, Executive Director, Dystonia Medical Research Foundation
Mahlon DeLong, MD, William Timmie Professor of Neurology, Emory University School of Medicine

Mission & History

For more than thirty years the DMRF has been funding breakthrough research on dystonia, reflecting the foundation's core mission of promoting and supporting basic and translational research. The foundation's broader mission is to promote awareness and education, and to support the needs and well being of affected individuals and families. The foundation plays a major role, as well, in sponsoring scientific meetings and workshops and advocating on behalf of dystonia at the federal and NIH level.

The DMRF was founded in 1976 by Samuel and Frances Belzberg, of Vancouver, BC, after their daughter was diagnosed with dystonia. Dedicated to serving the needs of all persons affected with dystonia and their families, the DMRF has grown from a small family-based foundation to a membership-driven organization of approximately 38,000 persons. The DMRF is governed by a volunteer Board of Directors led by the President.

The Volunteer Network was established 1993, serving as a local conduit for support, awareness, and fundraising. The country is divided into seven regions served by volunteer Regional Coordinators who work with the local support groups and area contacts and with the leadership appointees in the areas of Awareness, Advocacy, Development, Medical Education, and patient programs.

Medical and Scientific Advisory Council

The Medical and Scientific Advisory Council (MSAC) is comprised of pre-eminent scientists and clinician/scientists from multiple disciplines. The members conduct peer-review of research applications for recommendation to the Board for funding, set the directions of research, and both plan and participate in focused workshops.

Cure Dystonia Initiative (CDI)

Recognizing the need for leadership in promoting the development of more effective treatments, the DMRF recently established the Cure Dystonia Initiative (CDI). As other lay organizations have done, the foundation has essentially adopted a business approach to the development of therapies by enlisting the help of individuals from the bio-pharmaceutical industry who possess the talents and expertise to help plan and oversee therapeutic initiatives, by recruiting the necessary experienced professionals, and by modeling efforts on the best practices within the newly emerging world of profit/non-profit partnerships.

The CDI approaches dystonia as a drug discovery and development problem, providing direction, management and seed funding for projects aimed directly at the development of new therapeutics, while helping to create partnerships between academia, federal institutions and biopharmaceutical companies. The Initiative encompasses a number of promising avenues, including novel therapeutics, small molecule pharmaceuticals, as well as the repurposing of existing drugs. Assisting the CDI program is the Advisory Council, a group comprised of leaders who have extensive experience in the areas of venture capital, regulatory affairs, the pharmaceutical industry, drug discovery and dystonia research. These efforts within the CDI and the overall research agenda have been greatly aided by the recruitment of a full-time science officer, Jan Teller, Ph.D.

Accomplishments

Research

  • Awarded over $23.5 million for research grants and contracts (449 projects) including support of the work by Laurie Ozelius and Xandra Breakfield that identified DYT1, the gene responsible for the majority of cases primary generalized dystonia in childhood and adolescence.
  • Worked with the NINDS, through a Letter of Understanding, to provide interim financial support for investigators who, because of funding cutbacks, could not be funded.
  • Co-hosted with NINDS a key international dystonia meeting in 2006 in order to review and identify critical areas of research.
  • Has been a leading voice in organizing other lay organizations interested in different dystonias to work collaboratively on programs to advance research.
  • Partnered with the Dystonia Study Group in developing and supporting clinical trials for dystonia.
  • Hosted scientific meetings and workshops on specific issues that bring scientific and clinician thought leaders together to develop plans for advancing that area of research.

Advocacy

  • Helped to develop and to provide staff support for the Dystonia Advocacy Coalition (DAC), comprised of the Benign Essential Blepharospasm Research Foundation, DySTonia, Inc., the DMRF, the National Spasmodic Dysphonia Association and the National Spasmodic Torticollis Association. The DAC advocates for additional federal funding for NIH and has taken on such issues as Follow-on Biologics, proposed CMS rule changes that would have limited access to DBS, and impacted reimbursement.
  • Received the 2008 Advocacy Award from the American Brain Coalition

Education/Awareness

  • Hosts a website that provides comprehensive, accurate information on dystonia diagnosis, treatment and life strategies (www.dystonia-foundation.org). The website receives over 12 million "hits" and 365,000 visits annually.
  • Has extended its outreach to affected persons, their families and interested parties with a presence on social networks such as MySpace, Facebook and Twitter and launched a cell phone texting program to alert persons to upcoming programs and opportunities.
  • Provides educational meetings, in person and through webinars that provide information on dystonia and dystonia research.
  • Publishes The Dystonia Dialogue, which is distributed to over 38,000 households.
  • Supports the development of documentary films such as the award-winning film "TWISTED" that was broadcast on PBS in 2007 and the new film, "In This Moment" to be released in 2009.
  • Provides updates annually on dystonia research in the Foundation's publication, 'Progress & Promise'.
DMRF Support Programs

Serving the needs of individuals and families affected by dystonia through supportive services is an important element of the DMRF. The DMRF sponsors a host of programs aimed at various age groups and populations to help enhance the lives of people with dystonia. Programs are also aimed at increasing awareness and education about dystonia and its effects among healthcare professionals, affected persons and their families and the public. For more information on any DMRF program, contact the DMRF at 800-377-DYST (3978) or dystonia@dystonia-foundation.org

Dystonia Community Forums

The DMRF holds educational meetings to provide medical information on all forms of dystonia, including diagnosis, treatment options, research updates and local medical resources. Additionally, these forums highlight a patient's perspective to provide strategies for managing dystonia, including strong messages for all who are affected to stay well informed and connected with others.

Brain Donation Program

The brains of deceased dystonia-affected persons (with all forms of dystonia) and their family members are a precious and scarce resource that researchers value deeply in the pursuit of a cure. The DMRF is working in conjunction with the National Institute of Child Health and Human Development (NICHD) Brain and Tissue Bank for Developmental Disorders at the University of Maryland, along with dedicated researchers to obtain critical information about the pathologic basis and mechanisms of dystonia.

Dystonia Awareness

Dystonia Awareness Week occurs every spring, but dystonia awareness is a year-round campaign. Support groups and volunteers are active throughout the year with unique fundraising events and educational endeavors. The DMRF distributes a variety of awareness materials, and has an extensive collection of educational publications as well as an educational video entitled Dystonia: A Movement Disorder and a children's video entitled "I Am Not Dystonia."

Legislative Advocacy Program

Legislative advocacy is an important component of dystonia awareness and support. The DMRF, working with the Benign Essential Blepharospasm Research Foundation, DySTonia, Inc., the National Spasmodic Dysphonia Association and the National Torticollis Association to form the Dystonia Advocacy Coalition (DAC), encourages the congress to allocate more federal funding to dystonia research and to uphold the advances made in the Individuals with Disabilities Education Act of 1975, the American Disabilities Act of 1990, and other legislation that supports the needs of the dystonia community.

Children & Family Symposia

The DMRF sponsors educational symposia designed specifically for children and families who are dealing with dystonia. The programs focus on education about dystonia, coping strategies, family dynamics, schools and educational policies, and how best to manage the hurdles of everyday living with dystonia. These symposia aid in the development of valuable support networks among children and families.

Junior Advisory Council

The Junior Advisory Council is a network of young professionals and young adults between the ages of 18 and 40 who are working to encourage awareness and raise money for dystonia research.

Oromandibular Network

A forward thinking group of individuals who are affected by oromandibular dystonia (OMD) created a specific support network for persons with this form of dystonia. The network provides guidance, support, and one-on-one help to others with OMD via computer, phone, and mail.

Musicians With Dystonia

Musicians With Dystonia was founded under the auspices of the Dystonia Medical Research Foundation (DMRF) in 2000 by professional French Horn player Glen Estrin and Steven Frucht, MD, a neurologist at Columbia-Presbyterian Medical Center in New York. The group is dedicated to serving the special needs of musicians affected by task-specific focal dystonia, particularly hand and embouchure dystonias and provides input to the MSAC on issues related to task-specific dystonia.

Network Leadership

To help individuals and families overcome the everyday obstacles and challenges of living with dystonia, the DMRF has established a Regional Network of volunteer leaders. Support groups are active throughout the United States and Canada. Most support groups offer an inspiring blend of self-help and community involvement through awareness and/or fundraising. Volunteers of all backgrounds and abilities play a vital role in the dystonia community. Regional coordinators serve as liaisons between support group leaders and the headquarters office.

Staff

The Headquarters staff administers the daily operations for the DMRF. Each staff person is committed to providing professional service to meet the needs of the dystonia community.

Janet Hieshetter, Executive Director
Kathleen Behner, Director of Operations
Jessica Feeley, Editor and Special Projects
Julie Mack, Administrative Coordinator
Martha Murphy, Brain Bank Liaison
Emma Pinto, Development Coordinator
Tammy Reed, Associate Director of Development
Jody Roosevelt, Science and Technology Manager
Jan Teller, PhD, Science Officer
Germaine J. Mulhern, CPADELET, Financial Consultant


Janet HieshetterAbout Janet Hieshetter
Janet Hieshetter has served as Executive Director of the Dystonia Medial Research Foundation since 2004. Ms. Hieshetter has over 20 years experience in association and healthcare-related settings. Previous positions include Director of Community Resource Development at TRIOLOGY, Inc. and Senior Director/Interim Executive Director of the National Osteoporosis Foundation. She earned a BA in Health Sciences from Kalamazoo College in Kalamazoo, Michigan.

Mahlon DeLongAbout Mahlon DeLong
Dr. DeLong is the William Timmie Professor of Neurology. Dr. DeLong was Chairman of the Department of Neurology at Emory University School of Medicine from 1990-2003. He has played a major role in clarifying the functional organization of the basal ganglia and the role of these structures in movement and the pathophysiology of movement disorders such as Parkinson's disease. His studies have led to the development of new and highly effective surgical approaches for the treatment of movement disorders. Dr. DeLong has carried out numerous controlled clinical trials for the surgical treatment of movement disorders. Among numerous awards, Dr. DeLong received a Javitz Neuroscience Investigator Award from the NINDS for his research, and the Alfred E. Springer Award by the APDA and was elected to membership in the Johns Hopkins Society of Scholars and the Institute of Medicine. He was recently selected as the first recipient of the Deans Distinguished Faculty Award and Lecturer at Emory. Dr. DeLong is Chair of the Society for Neuroscience, Government and Public Affairs Committee. He served on the Advisory Council of the NINDS from 1996-2000. He serves on the Editorial Board of several scientific journals and government and private foundation advisory panels.