Slow down the disease by changing the activity of the genes that cause Amyotrophic Lateral Sclerosis (ALS) with a specific drug. The administration on a young patient took place in recent days, for the first time in Sicily, at the UOSD of Neurodegenerative Diseases with High Assistance Complexity of the AOU “G. Martin” by Messinadirected by Prof.ssa Sonia Messina. It is a latest generation drug capable of correcting the activity of the gene (S0D1) responsible for about 2% of cases of ALS, a pathology that causes progressive paralysis of the muscles. A therapy, infused with lumbar puncture, which – as scientifically demonstrated – initially produces a benefit on a biological level, reducing the values of that protein that causes cell deterioration. About 200 patients affected by ALS are followed up at the neurodegenerative diseases centre, patients who are diagnosed and treated taking into account the genetic picture so that the most appropriate treatment can be proposed.
“In about 20% of patients with ALS, the cause lies in an alteration in the functioning of four genes – explains Dr. Massimo Russo, neurologist. Today thanks to small molecules formed by DNA or RNA (antisense oligonucleotides) in some cases we are able to correct the level of activity of genes that function in a pathological way. The drug we are using has proved to be effective for the SOD1 gene; we hope that new therapies will soon become available to treat even those patients with forms of ALS linked to the involvement of other genes or not on a genetic basis”.
A treatment, the one possible today within the University Hospital of Messina, which over the last few months has increasingly consolidated an innovative organizational model for a public health system, the only example from Naples downwards. The UOSD of Neurodegenerative Diseases with High Care Complexity – to which all patients from Sicily and Calabria refer – sees at work not only neurologists, but also pulmonologists, speech therapists, physiotherapists and psychologists: a staff dedicated both to patients hospitalized in department, and to those who come to the center on an outpatient and day service basis.
“Our organization – underlines Prof. Sonia Messina – is aimed at promoting a global management of the person affected by a neurodegenerative disease. Being able to count on the presence of professionals with different specialties within the UOSD, in addition to collaborations with the other operating units of the AOU – and in particular with the UOC of Neurology and Neuromuscular Diseases, the UOSD of Neurophysiopathology and Movement Disorders, the COU of Pathology and TIN and the COU of Resuscitation with Intensive Care – allows us to develop a multidisciplinary management of complex patients with a high care burden. We have therefore developed a synergistic, constructive management, aimed at offering a clinical response consistent with international guidelines and new therapeutic approaches”.