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ALSA’s Science Director Says ALS Research Advances Give Investigators New Tools To Test Important Questions
July 7, 2004
The following article written by Lisa Beaton is reprinted with the permission of the ALS Society of Canada.
It has been more than a century since the first complete description of ALS by Dr. Jean-Martin Charcot. For most of those 130 years, researchers who took on the study of ALS have known little success and much frustration. But recent advances are showing exciting new promise in the field.
“The past decade, beginning in 1990, was dramatically different from any of the past decades combined,” announced Dr. Christopher Shaw, Senior Lecturer in Neurogenetics at King’s College London, UK, at the 14th International Symposium of ALS/MND in Milan , Italy , last Fall. Shaw sited advances in drug development, patient care, stem cell and gene therapy, animal models and an increased collaboration among researchers as some of the reasons for the accelerated pace in the field. “Many exciting vantage points steadily unravel the enigma of ALS, and new ideas are rapidly translated from laboratory to clinic. After many long dark years, we finally see a bright light on the horizon.”
“I do think things are accelerating,” says Dr. Neil Cashman, researcher at the University of Toronto ’s Centre for Research in Neurodegenerative Diseases. Currently establishing clinical trials using stem cells to treat ALS, Cashman is on the front lines of change in the field. “More people are working on ALS than ever before, and more funding is targeted to the disease. There has been a ‘confluence’ of novel technologies including proteomics, high-throughput genomics, and neuroscience methods that have definitely increased the rate of discovery.”
Technology is often sited as the main reason for the new rate of progress. McGill University researcher in neurogenetics, Dr. Guy Rouleau, who with colleagues discovered two of the genes for familial ALS says, “Technology allows us to do things we weren’t able to do before including cloning and manipulating the genes. A key element is the investigation of genetics, but that is the result of technology.” Rouleau also sites advances in computer tools, databases, and communication as helping speed up the pace.
Along with better communication has come increased collaboration in the scientific community, especially among the neurodegenerative diseases, where disease mechanisms can be very similar. Every advance affects not just one disease field, but many. “All science has a dynamic that feeds off success,” says Dr. Nigel Leigh, Director of the Motor Neuron Disease Centre at King’s College London, UK. Leigh established the clinical trials of Riluzole, the only drug licensed for use in ALS as an agent that modifies disease progression.
The new tools and recent progress are attracting young researchers, eager to work in the field of ALS, says the ALS Association’s Science Director Dr. Lucie Bruijn . “This is a very exciting time for those of us working on ALS. We now have the tools to test the important questions.” Bruijn lists the discovery of new genes, new disease models, and more human drug testing on the imminent horizon, adding, “Investigators are thinking of applying stem cells in a more innovative way, not just to replace dying neurons, but also to replace healthy neighbouring cells.”
Of course, new technologies and the current rate of progress can only continue with reliable funding. “There is more need for funding now,” says Dr. Guy Rouleau, “and the need is for constant funding. We’ve been plagued by government changes in funding decisions. The disease associations such as ALSA, MDA and ALS Society of Canada have really saved us in that way. They have actively encouraged the research and brought new researchers into the field.”
Both researchers and financial donors are deciding ALS is a disease worth investing in. “This disease is fascinating and yet so devastating,” says Dr. Bruijn. “The need to make an impact is absolutely compelling.”
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