Our salute to you

DMRF Founders Fran and Sam Belzberg in Vancouver with the Olympic torch. Sam Belzberg was an official flame carrier and is pictured here in the uniform of those who carried the torch for these games.

Athletes prepare for years to qualify for the Olympic competition. They work tirelessly on conditioning, skill building and strategy – all with an eye toward the dream of winning a gold, silver or bronze medal someday. Persons living with dystonia are all gold medal winners in our eyes. They too work tirelessly to develop the skills it takes to manage life with dystonia, creating strategies for how to overcome the limitations dystonia has imposed and the effort to do this is equal or more than that of an Olympiad. We salute those struggling to overcome the burdens of dystonia and go into our annual meeting, research reviews and planning for 2010, inspired by your stories, your courage and strength.

DMRF Update

Pictured above are Jan Teller (standing) and Mahlon DeLong.

The DMRF has been working to prepare for the annual meeting of our Medical and Scientific Advisory Council (MSAC) and Board of Directors meetings scheduled for February 25 to 27th. This is a busy but exciting time for the Foundation. Our wonderful MSAC members will review the grant and fellowship applications – discussing the scientific merit and the applications relevancy.

I wish everyone could witness this process as these reviews are thorough and thoughtful. Our MSAC members work with an understanding of the urgency in the community and the need for us to invest in those projects and investigators that are most likely to produce another piece of this dystonia puzzle – leading us to have a fuller scientific picture. Leading this effort is Mahlon DeLong, the Foundation’s Scientific Director and Jan Teller, the Foundation’s Science Officer – we are fortunate to have the benefit of these two dedicated individuals and are grateful to them for all they do for the DMRF and the dystonia community.

Joining us this year for this meeting will be the President and Executive Director of the European Dystonia Federation (EDF). We know that solving the dystonia mystery is going to take a global effort and we are so pleased that they are joining us for this meeting and discussions on how we can continue our collaboration going into 2010 and beyond.

The DMRF Board will also be discussing a number of new and exciting program initiatives. We look forward to sharing those with all of you following the meeting. Stay tuned for research and program announcements in March.

Happy New Year from the DMRF

On behalf of the DMRF I want to wish everyone a Happy New Year. We are looking forward to continuing the momentum that the events of 2009 generated into 2010. There is much to be grateful for as we look back at the milestones from last year.

• The wonderful research projects supported by the DMRF in 2009. We are beginning to receive progress reports from the work funded by the Foundation last year and it is exciting to see results. We are always pleased with our fellowships – funding young investigators is so important.

• Working in partnership with the Foundation for Dystonia Research, the DMRF launched a $1.8 million dollar research contract to identify and validate novel molecular targets for dystonia drug discovery and development. This project is possible because of the many years of research the DMRF has supported – including the DMRF funded discovery of the DYT1 gene in 1997. The first phase of this enormous scientific project was completed in 2009.

• Additionally, the DMRF is contracting for the development of a new animal model for dystonia. This is also important for drug discovery/development.

• DMRF continues to hold discussions with pharma to engage them in dystonia. In 2009 a significant meetings were held and we are optimistic about the interest expressed in dystonia – developing into additional, new treatments for dystonia.

• DMRF was pleased to join with Tyler’s Hope in launching a patient registry for DYT1 dystonia – another important element in getting new treatments on the market.

• Our scientific meetings continue to be important. Last year, the DMRF hosted a meeting for young investigators, a meeting on myclonus dystonia and a number of other meetings to help support the development of the NIH/ORD Dystonia Coalition program.

• The Children & Family Symposium was a major success – with many new families learning that they are not alone. This combined with our ongoing educational efforts and the fabulous support/education efforts of our support groups has provided accurate information to thousands of people.

• The Dystonia Coalition – the clinical research program approved by NIH is a major step forward for the community. The DMRF is proud to serve as an administrative center for this important program – providing administrative support for meetings, payments to the clinical sites, pilot projects and this past year, to have co-sponsored the Career Development Award.

• Last – but certainly not least – we are grateful to join with our advocacy partners the Benign Essential Blepharospasm Research Foundation, DySTonia, Inc., the National Spasmodic Dysphonia Association and the National Spasmodic Torticollis Assocition in celebrating the addition of dystonia to the list of eligible diseases for funding through the Congressionally Directed Medical Research Program. This was a wonderful way to end the year.

2010 looks to be a year of continued progress. The research grant applications recently submitted look good – these will be reviewed in February when our Medical & Scientific Advisory Council will be meeting. We will be launching new scientific exploration through our contract program and look forward to sponsoring and participating in key scientific meetings throughout the year.

We also look forward to continuing our community education programs, webinars, a western regional meeting and another successful Advocacy Day this spring.

Achieving our goal of a cure for all forms of dystonia can’t be done alone. We appreciate the partnerships we have globally and thank all of you for your support of the DMRF. We look forward to sharing with you the progress of the coming year and to hearing from you about how we can help you until that wonderful day when our work is finished.

Courage and Persistence

The two most powerful weapons are courage and persistence. Nothing can stop a person armed with both. - Scott Sorrel

Armed with courage and persistence the advocates of the Dystonia Advocacy Coalition (DAC) have worked tirelessly these past few years and finally achieved our goal of having dystonia included on the list to be eligible for funding through the Congressionally Directed Medical Research Program. This means, because of the efforts of our dystonia advocates, our researchers will have another source of potential funding – getting more dystonia research going! Dystonia advocates have walked the halls of Congress – meeting with Members and their key staff; met multiple times with Congressional Staff in local offices; and have written letters and made countless calls to make this happen – AND THEY DID IT!

The DMRF has proudly worked with advocates from the Benign Essential Blepharospasm Research Foundation, DySTonia, Inc., the National Spasmodic Dysphonia Association and the National Spasmodic Torticollis Association for over four years to address the legislative and policy needs of the dystonia community. Congratulations to everyone who had a part in this. You have demonstrated that our working together – collaborating on this critical effort – makes a difference and we all have something to celebrate.

Please plan on joining us as we continue our fight for dystonia. The DAC Advocacy Day is scheduled to take place in Washington, DC on May 5 with a training and orientation session planned for May 4th. We look forward to having you there.

Register for Advocacy Day by visiting the following link:
http://www.rsvpbook.com/event.php?470398

Myoclonus Dystonia Meeting

Pictured above from left to right: Thomas Gasser, MD, Marie Vidailhet, MD and Susan Bressman, MD

This past weekend the DMRF hosted a meeting on myoclonus dystonia, a form of dystonia with symptoms that include rapid jerky movements, alone or in combination with the sustained muscular contractions and postures. We were fortunate to have the leadership of Drs. Susan Bressman and Marie Vidailhet in developing the agenda and bringing together the best faculty in the world to discuss the status of myoclonus dystonia research, clinical and basic, and to develop a plan for keeping the momentum of progress moving forward. Joining the DMRF for this incredible meeting was esteemed researchers from France, Germany, Italy, the Netherlands, the United Kingdom, Canada and the USA. The agenda included a review of genetics, imaging and neurophysiology, therapeutics, and animal models with a discussion on future directions.

In summarizing the meeting, Dr. Bressman said this was the best workshop she had been involved in for many years. We felt the same way – the energy, the enthusiasm and the spirit of collaboration was amazing and we look forward to implementing the plan for attacking myclonus dystonia.

Having these kinds of meetings is an important part of the DMRF’s scientific strategy – implemented and operated by our full time Science Officer, Jan Teller, PhD, and our Medical and Scientific Director, Mahlon DeLong, MD of Emory University. Supporting research, awarding fellowships and contracting for specific scientific work is very important but so too are these meetings when we are able to bring great minds together to discuss a specific topic and develop next steps.

We are so thankful to all who participated in the meeting and to everyone in the dystonia research community for their dedication and commitment to ending all forms of dystonia.

The DMRF: A Critical Piece of the Dystonia Puzzle

Starting a scientific career is a daunting proposition, especially for a physician-scientist that has clinical as well as lab responsibilities. Yet, it is physicians who witness first hand the struggles of patients, and this knowledge can be a powerful motivator for scientific discovery -- even when the underlying science is unknown and therefore “off the radar” of basic investigators.

The DMRF has provided essential support to my scientific career, both at the critical early stage, as well as at later stages. Shortly after the gene encoding torsinA was discovered, I received a grant from the DMRF to generate torsinA mutant animals. This grant - to a young and untested physician-scientist – was helpful in supporting the work but, perhaps as importantly, gave me an early track record of securing funding and was therefore helpful in me getting other grants, both from the Howard Hughes Medical Institute as well as the NIH. The ongoing support of the DMRF also enabled me to hire and support Rose Goodchild, an outstanding young scientist who now has an independent lab of her own (studying torsinA). Moreover, the DMRF has consistently organized and supported basic science and clinical workshops that have been invaluable in helping to integrate and focus the dystonia research community. I have found these meetings invaluable, both scientifically and as a place where trainees in my laboratory (and others) can begin to present their work and develop confidence as scientists. The DMRF has consistently focused on identifying and supporting the best science, and I’m convinced that we’d be far, far further from our goal of improving the treatment and hopefully cure of dystonia without the many valuable DMRF-supported activities.

Guest Blogger:

William Dauer M.D.
Elinor Levine Associate Professor
Neurology & Cell and Developmental Biology University of Michigan Medical School

Would you like to know more about DMRF grant funding opportunities?
http://www.dystonia-foundation.org/pages/funding_opportunities/142.php

The deadline for all applications is December 15, 2009. Visit http://www.dystoniagrants.org to view the application form.

Dystonia Researchers

Pictured above are Rose Goodchild, PhD, University of Tennessee and Cris Bragg, PhD, Massachusetts General Hospital

Attracting and keeping smart, dedicated young researchers is really important. Recently the DMRF hosted a meeting of young investigators, bringing together some of the brightest young dystonia researchers to talk about dystonia research and how best to support their work. We wanted to hear from them as to the challenges and opportunities they are experiencing – and how they thought the Foundation could best help young dystonia scientists. We also wanted their ideas on new, perhaps innovative ideas for DMRF research support. It was a very productive meeting with a great exchange of ideas for how we can move forward into 2010 and beyond.



This week the Society for Neuroscience is meeting in Chicago. This is an amazing meeting and in the past few years – dystonia has been present in poster sessions and talks. Having a dystonia presence at this meeting is an accomplishment for all to celebrate. DMRF, along with NSDA, had a booth at the meeting – giving us the opportunity to further promote dystonia at this meeting.



Last evening the DMRF hosted an event where we brought together many members of the dystonia research community – senior level investigators with younger investigators – all talking about what is new in dystonia research, what steps we should look at next and the progress that has been made in the last few years. It was wonderful to see the dialogue between these generations of dystonia researchers, to witness the established investigators encouraging the thoughts and ideas of those investigators beginning their careers.