26.09.11 2° AriSLA meeting
ALS Research STATE-OF-THE-ART The 2nd National Conference of AriSLA "New Research Perspectives. For a future without ALS!" which was held in Milan on September 27th, has offer the opportunity to present to the scientific community the winning projects of the "AriSLA 2010 Call for Projects" and to discuss about two of the most relevant and debated themes of the research on Amyotrophic Lateral Sclerosis: genetic studies and stem cells. The event has been attended by over 800 people, about 500 connected via web streaming: this method has allowed international researchers and numerous ALS patients to follow the updates of todays and future research priorities. The event was opened by Prof. Melazzini, AriSLA President, who welcomed the guests and presented what the Foundation has done this past year and what it is going to do to support the activities of researchers: the President has in fact announced the launch of a new service facility for animal models, in collaboration with the ASL 1 Milano and the Institute for Pharmacological Research "Mario Negri", thanks to a contribution of Regione Lombardia. Greetings from the President were accompanied by the attendance of the AriSLA founding members: Giuseppe Guzzetti for Cariplo Foundation, Francesca Pasinelli, delegated by Luca Cordero di Montezemolo for Telethon Foundation and Massimo Mauro for Vialli e Mauro Foundation. The intervention of Dr. Formigoni, President of Regione Lombardia and the Councillor for Social Policies and Health Services of the City of Milan, Pierfrancesco Majorino, has testified the will to maintain an open dialogue with AriSLA in order to create synergies that promote effective research. One of the main topic of the discussion was the recent discovery of mutations in the gene C9orf72 detected in almost 40% of patients analyzed by a group of international researchers, which includes numerous Italian researchers, coordinated by prof. Adriano Chiò. This research opens up a lot of possibilities for the studies on the causes of both the familiar and sporadic form of the disease, but requires even greater efforts from the scientific community to understand its relevance for ALS patients. The second round table on stem cells and their possible use in ALS has seen the intervention of Nicholas Maragakis - Associate Professor of Neurology at Johns Hopkins University in Baltimore, USA - and Prof. Letizia Mazzini, neurologist at the "Ospedale Maggiore della Carità" of Novara and head of the ALS Center at the University of Eastern Piedmont. Dr. Maragakis introduced the new challenges opened by stem cells in the study and treatment of ALS and showed some of his results on the transplantation of glial progenitors in transgenic mice for mutant SOD1. Stem cells in fact can be helpful to understand the mechanisms underlying the disease and for disease modeling and a valuable tool for drug screening and cell therapy. Dr. Mazzini has presented some of the results obtained over the past 10 years by her research group regarding the transplantation of mesenchymal stem cells. The use of stem cells for therapy - she said- requires the ability to easily reach the target tissue where exert the therapeutic effect, favoring the diffusion of trophic factors and immune modulators to motor neurons and surrounding cells. She also explained how a clinical trial is planned, how patients are selected to be enrolled, and presented the first results of phase I trial in ALS patients performed by her group. The focus of the comparison was the confirmation of the therapeutic potential of stem cells, and the in order to be able to better evaluate the possible benefits and results. Researchers has also continued to discuss in the afternoon session, with the presentation of the winning projects of the "Arisla Call for Projects 2010": a brief "overview" of these 8 scientific projects - 3 pilot and 5 full grants - that have been judged by the AriSLA International Scientific Committee, according to the granting system of peer review. The event was closed by the AriSLA Scientific Director - Giulio Pompilio - and the Secretary General of the Foundation - Renato Pocaterra - who highlighted that this 2nd Conference has been a precious moment of scientific debate and an opportunity to learn and contribute to the achievement of the Foundation that is serving the research and the researchers work to "defeat the disease". Dr. Pocaterra, reaffirming the will of the Foundation to keep always open the dialogue with the scientific community through other opportunities of discussion during the year, he renewed the appointment to the 3rd AriSLA Conference. The Conference intervention are available on www.alscience.it. 2nd National Conference of AriSLA "New Research Perspectives. For a future without ALS!"
The first roundtable was attended by two researchers of international relevance: prof. John Landers, University of Massachusetts Medical School - Department of Neurology and prof. Wim Robberecht, head of the Department of Neurology and Neuroscience and Section of Neurology, University of Leuven, Medical School, Belgium. They discussed about the benefits and limitations of the new technologies available for genetic studies on ALS, as the "exons capture" and "Next Generation Sequencing". These technologies helped to identified several causative mutations in ALS and are now affordable for most laboratories. At the same time, the sequencing of candidate genes can be very risky because, as pointed out by Dr. Landers, each individual can bring ~ 300-500 new alterations of the genome not recorded yet in any database and almost always non-pathogenic. Dr. Robberecht also showed that gathering all these information is not always positive. In fact only very few genes found altered in ALS has today a known biological function.
