Canadian ALS Research Network formation To address the primary problem of too few clinical trials available for Canadian ALS patients, a meeting of the leaders of all 15 Canadian academic ALS clinics was arranged in December 2007 at the International Motor Neuron Disease Symposium. Its mission is to promote multicenter ALS research studies in Canada, which include investigator- and industry-initiated clinical trials, companion epidemiological studies and translational studies in collaboration with ALS basic scientists. CALS is recognized by the ALS Society of Canada as the official clinical trials network of Canada and CALS is the official ALS affiliate organization of the Canadian Neurological Sciences Federation. Conditions of membership include a scientific and/or clinical research interest in ALS, attendance at the CALS annual general meeting conducted at the research forum held by the ALS Society of Canada and participation in expedited reviews of industryproposed projects when requested. In addition to attracting and conducting clinical trials, epidemiological, genetic and quality of life studies in ALS can be facilitated by a cross-Canada network of ALS researchers. These studies require multicenter and often multinational participation given the low incidence and prevalence rates of ALS. CALS can form international collaborations with other ALS Consortia and researchers to pool resources and share data. In cooperation with the ALS Society of Canada, CALS can promote public and primary care education campaigns to increase ALS awareness. The study represented the first Canada-wide investigator-initiated clinical trial in patients with ALS and the first ALS Canada and NIH-sponsored investigator-initiated trial conducted in Canada. CALS members were coprinciple future science group Developing a consortium for ALS clinical research: the Canadian ALS Research Network Research Update Canadian ALS Research Network Figure 1. Upon future science group study completion and publication, the trial was noted for its innovative design and for being the first study to dismiss lithium carbonate as a treatment for ALS. Given the success of the first collaboration between the consortia, CALS was invited to participate in a second NIH-supported Phase III study testing ceftriaxone in ALS subjects. 3(12) 1115 Research Update Zinman, Genge & Figlewicz registry , a Public Health Agency of Canada-supported study to determine Canadian ALS incidence and prevalence rates, a multinational ALS quality of life measure validation study, the development of Home Ventilation national guidelines for ALS and the development of Canadian best practice guidelines for patients with ALS. CALS has also been used to assist in the sharing of blood and tissue samples from ALS subjects to help determine disease pathophysiology and for the identification of novel genes underlying familial forms of ALS. A large grant was recently obtained for a collaborative imaging study in ALS and will use CALS infrastructure and investigators. In the truest form of translational research, CALS investigators are preparing to conduct a Phase II clinical trial testing a compound found by Canadian ALS scientists to slow disease progression in two ALS animal models.
Neuromuscular ALS Research
The Carolinas Neuromuscular/ALS MDA Center, part of the Carolinas HealthCare System Neurosciences Institute-Neurology, is an internationally recognized program of clinical care, research and education for degenerative neuromuscular diseases, such as Duchenne and limb girdle muscular dystrophy, and amyotrophic lateral sclerosis. The center’s goal is to discover, evaluate and implement treatments for ALS and neuromuscular diseases including environmentally-caused, genetic and idiopathic neurodegenerative diseases with central and peripheral involvement having neuromuscular consequences. The Carolinas ALS endowment was established in 1998 to provide the funds for treatment, patient support, research and education programs conducted by the center. The Carolinas Neuromuscular/ALS-MDA Center is now one of the most comprehensive ALS and muscular dystrophy facilities in the United States, and the Southeast’s major comprehensive facility for the study and treatment of degenerative neuromuscular diseases. Muscular Dystrophy Association and other community-based organizations support the center’s clinical and research programs in neuromuscular diseases and ALS. The center has two translational research laboratories: the Carolinas Neuromuscular/ALS Research Laboratory and the McColl-Lockwood Laboratory for Muscular Dystrophy Research. The Carolinas Neuromuscular/ALS Research Laboratory maintains a biospecimen and tissue repository to provide samples for research. The laboratory is a source of basic and clinical research training and education for undergraduate, graduate and medical students, post-doctoral fellows, residents and visiting faculty to further advance this mission. The Carolinas Neuromuscular/ALS Research Laboratory examines areas of therapy development, motor neuron cell biology and diagnostic test development for neuromuscular diseases, particularly ALS. The laboratory was established in 2000 via a $1 million challenge gift from Crandall and Erskine Bowles together with other philanthropic donations to the ALS Research Fund in order to advance the diagnosis and treatment of ALS and neuromuscular diseases. At the time of the gift, Crandall Bowles said, “ALS has impacted our family, and we want to do all that we can to combat it. Through this challenge gift, we hope to inspire others to support a project right here in our community that may have worldwide impact for all families affected by this disease.” All research is conducted under the direction of the Medical Director of the Carolinas Neuromuscular/ALS – MDA Center, Benjamin Rix Brooks, MD. The laboratory has three translational research groups in the Cannon Research Center: motor neuron cell biology, stem cell biology/nanotechnology/bioinformatics, and mitochondrial biology. The individual research groups are directed by Richelle Hemendinger, PhD, Jean-Luc Mougeot, PhD, and Alexander Panov, MD, PhD. Mohamed Sanjak, PhD directs clinical studies and clinical trials research programs and coordinates with the translational research staff. All group directors direct externally and internally funded research efforts.