2023-05-30 13:45:03
Experts and local operators discussed the treatment of amyotrophic lateral sclerosis (ALS) in the workshop ‘Criticalities in Als. From disease characterization to clinical trial design’. The training appointment promoted by the Nemo Clinical Centers took place at the Gemelli Polyclinic, the Rome branch 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 studies on ALS and taken charge of over 2,500 people with the pathology.
Among the topics addressed in the interventions of the 5 expert clinicians of the Nemo network – explains Nemo in a note – the following stand out: innovative approaches in clinical studies on ALS; new biomarkers for disease; early identification of symptoms. The event has the patronage of the Italian Amyotrophic Lateral Sclerosis Association (Aisla) Onlus, as part of the celebrations of its 40 years of activity, of the Catholic University of the Sacred Heart of Rome and the Gemelli Foundation University Hospital. An alliance is needed, a network “to translate the experience built on other neuromuscular pathologies, to better approach the complexity of ALS, in a historical moment in which there is an increasing number of clinical pharmacological studies on the disease, based on new rationales sciences”, says Valeria Sansone, clinical-scientific director of the Nemo Center in Milan and full professor at the University of Milan.
“Intervening promptly is important not only in the disease diagnosis phase – underlines Federica Cerri, neurologist and ALS area contact person at the Nemo Center in Milan – Scientific evidence, in fact, shows how a targeted and early management is essential in preventing clinical worsening, with a concrete impact in improving quality of life and survival”. Recent evidence opens up new research scenarios in considering ALS as a biological process, which begins with a presymptomatic phase (defined as Mild Motor Impairment) and which it is increasingly necessary to learn to identify and interpret early to be effective even in treatment . The targeted management process – continues the note – must consider every functional aspect (breathing, nutrition, movement and communication) also as a useful indicator for monitoring the disease. Among these, the cognitive-behavioral disorders in the evolution of the pathology, addressed by Emanuele Costantini, neurologist at the Nemo Center in Ancona, are often considered late from the clinical point of view, but the correlations of cognitive function with the course of ALS are supported from 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.
The speech by Mario focused on Tofersen, approved a few weeks ago by the American regulatory agency FDA for those with the Sod1 gene mutation, and on the scientific reasons that put the dosage of neurofilaments in the foreground as a possible surrogate biomarker of disease Sabatelli, clinical director of the Nemo Rome Center, adult area, and president of the medical-scientific commission of the Italian Amyotrophic Lateral Sclerosis Association (Aisla) Onlus. In people with ALS – Nemo explains – there is an increase in neurofilaments, proteins that make up a sort of skeleton of the nerve fibers. As a result of motor neuron degeneration, neurofilaments are released into the serum and cerebrospinal fluid of the diseased person. The dosage of neurofilaments in serum can therefore provide an important contribution in early diagnosis and, as in the case of Tofersen, a very useful support for evaluating the response to drugs.
In this context, the first message that emerges is the priority of placing the person and their right to self-determination at the center of the care relationship. In this regard, Stefania Bastianello, technical director of Aisla onlus, presented an overview of scientific approaches and European and national legislation, starting from Law 219/2017 regarding shared care planning.
Finally, Riccardo Zuccarino, clinical director of the Centro Nemo Trento spoke on the new strategies for taking charge that change the paradigm of quality of life. In fact, ALS forces the person to reconstruct new life contents in the light of the experience of the disease, for this reason the clinical response must necessarily start from the relationship and give answers that each time rethink rehabilitation interventions, adapted to the specific needs of each one. In daily research it is a priority to be oriented towards everyone’s well-being – concludes the note – such as the simple gesture of readjusting the fork to be brought to the mouth independently.
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