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ALS: knowing the disease to maximize the effectiveness of clinical trials

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ALS: knowing the disease to maximize the effectiveness of clinical trials

Pathology experts met to train local professionals on the new research challenges

Milan – The innovative approaches in amyotrophic lateral sclerosis (ALS) clinical trials; attention to new biomarkers for sickness; early identification of symptoms are among the topics addressed in workshop “Criticalities in ALS. From disease characterization to clinical trial design”. The training event, dedicated to researchers and health professionals and promoted by NeMO Clinical Centers, took place at the Policlinico Gemelli, the Roman office of the national network expert in the treatment of neurodegenerative and neuromuscular diseases. In the last year alone, the NeMO network has activated 36 clinical trials on ALS and taken care of over 2,500 people with the disease.

The issues addressed by the second stage of the training course, which brought together five expert clinicians from the NeMO network, grasp the challenges facing the scientific community and patients today. And where research opens up new scenarios, the network work alliance becomes the answer which puts the person at the center of his life project.

And it is on this ability that the contribution of the Prof. Valeria Sansone, clinical-scientific director of the NeMO Center in Milan and full professor at the University of Milan, who opened the meeting. Indeed, the value of the alliance becomes an opportunity for translating the experience built on other neuromuscular pathologies, to better approach the complexity of ALSin a historical moment in which there is an increasing number of clinical pharmacological studies on the disease, based on new scientific rationales.

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The urgency and necessity is theearly identification of symptomsdeepened by Dr. Federica Cerrineurologist and ALS area contact person at the NeMO Center in Milan. Timely intervention is important not only in the stage of disease diagnosis. In fact, scientific evidence shows that targeted and early management is essential in preventing clinical worsening, with a concrete impact in improving quality of life and survival. Evidence that opens up new research scenarios in understanding ALS as a “biological process”, which begins with a presymptomatic phase – defined as Mild Motor Impairment – and that it is increasingly necessary to learn to identify and interpret early to be effective even in treatments of care. And precisely the targeted management process must consider every functional aspect – breathing, nutrition, movement and communication – also as a useful indicator for monitoring the disease, to contribute from a scientific point of view to compose the pieces of the ALS puzzle. Between these, the role of cognitive-behavioral disorders in the evolution of the diseasefaced by dr. Emanuele Costantini, neurologist at the NeMO Ancona Center. Often considered belatedly from a clinical point of view, the correlations of cognitive function with the course of ALS are supported by a large scientific literature. The challenge will be to understand how to make the impact of these symptoms on diagnosis and new treatments increasingly measurable.

And inevitably the reference goes to tofersen drug for those with the gene mutation SOD1approved a few weeks ago by the American Regulatory Agency (FDA) and to the scientific reasons that place the dosage of neurofilaments as a possible surrogate biomarker of disease. Topic addressed by prof. Mario Sabatelli, clinical director of NeMO Rome, adult area, and president of the medical-scientific commission of AISLA Onlus. To date, science and clinical practice show that in people with ALS there is an increase in neurofilaments, proteins that make up a sort of skeleton of the nerve fibers. Following the degeneration of motor neurons, neurofilaments are released into the serum and cerebrospinal fluid of the sick person. The measurement of neurofilaments in serum can make an important contribution to early diagnosis and, as in the case of tofersen, a very useful support for evaluating the response to drugs.

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In this context, the first message that emerges is the priority of place the person and his or her right to self-determination at the center of the care relationship. An overview presented by Dr. Stefania Bastianello, technical director of AISLA onlus, on scientific approaches and on European and national legislation, starting from Law 219/2017 regarding the Shared Care Planning (PCC). Tools in support of the doctor-patient alliance that allow the scientific community and patients to build new strategies to cope with the complexity of the disease.

New management strategies, therefore, that change the paradigm of quality of life. This is the topic addressed by dr. Richard Zuccarino, clinical director of the NeMO Trento Center, who closes the works. In fact, ALS forces the person to reconstruct new life contents in the light of the experience of the disease; the clinical response must therefore necessarily start from the relationship and give answers that rethink every time rehabilitative interventions, adapted to the specific needs of each one. It is looking at the detail of everyday life with a creative thought, to find solutions oriented towards the search for everyone’s well-being, such as the simple gesture of readjusting the fork to be independently brought to the mouth.

Under the patronage of AISLA Onlusas part of the celebrations of its 40 years of activity, by Catholic University of the Sacred Heart of Rome and the A. Gemelli Foundation University Hospitalthe training saw the experts share the value of continuity between research and treatment to understand and change the natural history of this serious neurodegenerative disease.

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