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This site is dedicated to scientific community working on ALS. Our aim is to optimize researchers time and efforts by providing updated, well organized information on novel findings, available resources and research support.
AriSLA - The Foundation for research on ALS - has been set up to make ALS research investments more effective and efficient, to speed up the clinical research impact e and to provide patients with better care, improved conditions and life expectancy. Its aim is to boost Italian excellencies in basic, clinical and technological research. The Foundation founders are Fondazione Cariplo, Fondazione Telethon, Fondazione Vialli and Mauro and AISLA.

 

 

From Houston, a new hope to slow down the progression of ALS. The study is made by Prof. Appel, member of AriSLA advisory Board and invited speaker of the next Focus on ALS

New findings to counter ALS thanks to the results obtained from recent research conducted at the Houston Methodist Neurological Institute. The study, in fact, laid the basis for a more in-depth study with a new immunotherapy, which proved to be safe for the 3 ALS patients involved, and also found that it is possible to slow down the progression of the disease.

 

 

 

Published on Neuroimmunology & Neuroinflammation, the official journal of the American Academy of Neurology, the research is signed by the team of Professor Stanley Appel, professor of neurology and co-director of the Houston Methodist Neurological Institute, and member of the AriSLA Foundation Advisory Board.

Professor Appel explains "Our preclinical data had documented that mSOD1 ALS mice crossed with mice lacking CD4 lymphocytes had markedly shortened survival; transplants of Tregs significantly prolonged survival. In ALS patients we found Tregs to be dysfunctional, with impaired suppression of both T effector lymphocytes and pro-inflammatory macrophages, and lower levels of Tregs were associated with increased mortality. Expansion of the dysfunctional Tregs ex vivo restored their suppressive functions, and prompted our study of autologous infusions of expanded Tregs in 3 ALS patients at early stages (4 doses over 2 months) and later stages (4 doses over 4 months) of disease. These infusions were safe and well tolerated in all 3 subjects in both phases of disease. During the treatment period the disease progressed at a slower rate in all patients, and measures of maximal inspiratory pressure stabilized. However, during the several months between the early and late sets of Treg infusions and following the 2nd set of infusions, significant clinical progression was noted; the clinical benefit of Treg infusions did not last. The major limitation is that only 3 ALS patients were studied, and the trial did not control for a placebo effect. Nevertheless throughout the clinical course, disease progression correlated with ex vivo measurements of Treg suppressive function."

"Overall, our study showed it was safe to increase ALS patient Treg levels, and progression of ALS can be slowed. Our Phase 2 Treg infusion study scheduled to begin in the fall is to determine whether progression can be slowed for prolonged periods by repeated administrations of Tregs. Our goal is to give our patients hope for the future. We may not  cure a patient's disease, but we can make a difference."

The study was funded by ALS Association e ALS Finding a Cure.

Professor Appel has often presented his research at the conferences organized by AriSLA and, in particular, at the first National ALS Symposium, which took place in Naples in 2015 and was promoted by AISLA and Arisla.

In a few months Prof. Appel will be in Italy again as speaker of the 'Focus SLA', which will be held in Genoa on 27, 28 and 29 September and promoted by AISLA, AriSLA and MND - MOTOR NEURON DISEASES: Molecular and Cellular Basis of Vulnerability.

The 2018 scientific event, which will be open to all those wishing to deal with ALS experts, will present an update on the progress of the research, providing new hypotheses to indicate promising ways to treat the disease. Among the many topics there will be discussed the vulnerability of motor neurons with respect to genetic hereditary mutations, the key role played by the immune and inflammatory systems and the  role of muscles in the pathogenetic mechanisms that can influence the fate of the motor neuron.

For the full article http://nn.neurology.org/content/5/4/e465

 

 




     
     
     
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