New signs of hope emerge from the clinical study on ALS conducted by Nemo Clinical Centers. After the recent approval of tofersen by EMA (the European Medicines Agency), concrete confirmation of the clinical efficacy of this drug comes from the first all-Italian study which analyzed the highest number of people with ALS with the Sod-1 mutation in our country and for the longest period of time.
“This field study, in the reality of clinical practice, provides important and convincing data – he states Mario Sabatelliclinical director of the Nemo Center Rome, at the Policlinico Gemelli Foundation and president of the medical-scientific commission of Aisla Italian Association of Amyotrophic Lateral Sclerosis – Thanks to the clinical experience on ALS of the network of Nemo Centers and their ability to collaborate it was possible to monitor the group of patients for a very long period of time, a fundamental element for understanding the impact of the drug in relation to the evolution of the disease”.
For a period of at least one year after the initial administration of tofersen – reports a note – the Nemo Centers in Rome, Milan, Brescia, Trento and Ancona collected and analyzed data from 17 patients. This group of patients represents a portion of the 27 who were able to access the drug starting in 2021 through the early access program. This period was combined with 12 months of clinical monitoring prior to the arrival of the drug, for a total of approximately 2 years of evaluation. Precisely the large number of the sample and the long period of clinical observation are to be considered extremely significant, given the rarity of this specific mutation (2-3% of people with ALS in Italy, around 150 in total) and the very complexity of the disease in its entirety. clinical course.
“The value of the results achieved is given by the possibility of comparing the clinical data of the study with the same data collected in daily clinical practice in the period before taking the experimental drug – confirms Federica Cerri, neurologist in charge of the ALS area of the Nemo Center in Milan and also among the experts of the medical-scientific commission of Aisla, which continues – This continuity in taking care of the person, in fact, allows us to conduct a detailed analysis of the history of the disease, tracing clearly two trajectories of its progress, i.e. before and after treatment with tofersen”.
The results of the study, just published in the Journal of Neurology, the official journal of the European Society of Neurology, show a stabilization or even a slight clinical improvement for a significant number of patients involved (53% of the study group). Each patient was monitored every 12 weeks with standardized clinical rating scales (Alsfrs-R, Fvc, Mrc) to verify general functionality, respiratory capacity and muscle strength in the limbs. Furthermore, research has shown that the drug has a positive effect on a biological level in the process of degeneration of motor neurons, as confirmed by the significant reduction in the dosage of neurofilaments, proteins indicative of this process. In the study, a marked reduction of these neurofilaments was observed in 82% of patients. This confirms the positive impact on tofersen disease in at least a subgroup of patients.
Fulvia Massimellinational president of Aisla expresses great satisfaction: “This result renews our hope in research, we now await the rapid approval of Aifa. I want to express on behalf of the community of people with ALS our gratitude to the Nemo Centres, for all of us a reference in care, research and assistance on ALS and neuromuscular diseases. Concrete signs of hope, therefore, “in research on ALS” which confirm how the strength of clinical experience on the disease can be translated in a concrete way into the quality of scientific research. ” he concludes.
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