Chair: Gregor K. Wenning
Co-Chairs: Phillip A. Low and Shoji Tsuji
We plan to develop a common data-set, including disease-specific validated rating scales for harmonized data acquisition. Second, we intend to launch a global MSA patient registry. Third, we will launch studies focusing on the discovery of diagnostic and surrogate (bio)markers, and determination of environmental and genetic underpinnings. Finally, we will develop consensus (best-practice) guidelines for the standard of care in MSA (based on the principles of evidence-based medicine).
To this end, we defined the following specific aims:
1. To establish a global patient registry
2. To set up a decentralized biomaterial bank and thereby define standard operating procedures to harmonize blood, CSF and brain tissue sampling.
3. To identify MSA genetic risk loci using large-scale genome-wide association studies
4. To develop, translate and validate additional MSA-specific rating scales
5. To define and validate autonomic progression markers
6. To define and validate MRI and functional imaging surrogate progression markers
7. To develop interventional trial guidelines
8. To develop best-practice (evidence-based) guidelines for pharmacologic as well as non-pharmacologic treatments.
9. To investigate MSA pathophysiological mechanisms and screen candidate compounds in preclinical MSA models
Need for a study group in the field
Multiple system atrophy (MSA) is a rare and relentlessly progressive movement disorder with an estimated prevalence of 4/100,000 people (Schrag 1999). There is currently no treatment available to significantly alleviate motor and autonomic symptoms or to modify the natural course of the disease. In addition, there are no widely accepted guidelines on symptomatic treatment strategies available.
To date, clinical MSA research has been limited by the low prevalence rate preventing individual research sites from studying sufficient patient numbers. Thus, a coordinated effort at an international level is required to advance MSA research in the field of biomarker discovery, early diagnosis, definition of genetic underpinnings and translational drug development. Consequently, an administrative framework for global collaborative MSA research is needed.
Europe: Austria: Werner Poewe, Gregor Wenning; Bulgaria: Latchezar Traykov; Czech Republic: Evzen Ruzicka; Denmark: Karen Østergaard, Kristian Winge; France: Francois Tison, Wassilios Meissner, Olivier Rascol, Anne Pavy-LeTraon; Germany: Thomas Klockgether, Heinz Reichmann, Günther Deuschl, Wolfgang Oertel, Thomas Gasser; Ireland: Tim Lynch, John McKinley; Israel: Ruth Djaldetti, Tanya Gurevich; Italy: Pietro Cortelli, Alberto Albanese, Paolo Barone, Carlo Colosimo, Giuseppe Meco, Ubaldo Bonuccelli, Roberto Ceravolo; Portugal: Joaquim Ferreira, Miguel Coelho; Serbia: Vladimir S. Kostić; Spain: Eduardo Tolosa, Jose Berciano; Sweden: Håkan Widner; The Netherlands: Bastiaan R. Bloem; United Kingdom: Huw Morris, Henry Houlden, Kailash P. Bhatia, Christopher J. Mathias, Alexander Gerhard; Poland: Jaroslaw Slawek
North/South America: Argentina: Emilia M. Gatto; Canada: Anthony E. Lang; Chile: Pedro Chaná; Mexico: Mayela Rodriguez Violante; Peru: Carlos Cosentino; United States: David S. Goldstein, Roy Freeman, Steven Vernino, Joseph Jankovic, Susan Perlman, David Robertson, Laurie Ozelius, Horacio Kaufmann, Phillip A. Low, Eliezer Masliah, Irene Litvan, James Bower, Robert A. Hauser
Asia and Oceania: Australia: Victor Fung; China: Weihong Gu; India: Sjith Ovallath; Japan: Shoji Tsuji, Tetsutaro Ozawa, Atsushi Takeda, Hidenao Sasaki, Masaaki Matushima, Masashi Aoki, Osamu Onodera, Masatoyo Nishizawa, Hidehiro Mizusawa, Kinya Ishikawa, Satoshi Kuwabara, Kimihito Arai, Kazuko Hasegawa, Tatsuhiko Yuasa, Hirohisa Watanabe, Mizuki Ito, Gen Sobue, Fumiaki Tanaka, Kenichi Yasui, Kenji Nakashima, Yuishin Izumi, Takeo Kato, Susumu Kusunoki, Kazumasa Saigoh, Hiroshi Takashima, Hjiri Ito; Korea: Chong Sik Lee, Han-Joon Kim