In the field of adult motor neuron diseases (primarily amyotrophic lateral sclerosis, or ALS), clinical research is focused on two main objectives:  

Gain a better understanding of the mechanisms underlying the disease.

To this end, various types of patient data are used: clinical, biological, genetic, imaging, neurophysiological, and/or histological data. In ongoing studies, the collection and analysis of this data help address several key issues: better understanding the heterogeneity of ALS, developing new diagnostic biomarkers, identifying imaging markers in asymptomatic individuals carrying a genetic mutation that puts them at risk of developing the disease, and improving our understanding of the role of certain signaling pathways in maintaining motor function.  

Development of new treatments through clinical trials.

ALS is a disease for which there is currently no cure, but a better understanding of its pathophysiological mechanisms is enabling the development of new drugs that target various mechanisms of action. These trials, which are most often international and conducted in partnership with pharmaceutical companies, allow for the study of the safety and efficacy of new treatments. The treatments currently being tested aim to reduce neuronal death (through mechanisms involving oxidative stress, cellular or protein energy metabolism, iron metabolism, neuroinflammation, etc.) or to slow the decline in motor function (through a molecule that activates a protein promoting muscle contractility). Another innovative therapeutic approach involves gene therapies targeting genes responsible for ALS. A recent study demonstrated the beneficial effect on the progression of ALS of a treatment administered via intrathecal injection in patients carrying a mutation in the SOD1. Thanks to these results, some patients whose ALS is linked to a mutation in the SOD1 can now access this treatment through compassionate use, and an ongoing study aims to determine whether its effect in asymptomatic carriers of the mutation could delay the onset of disease symptoms.  

To learn more about amyotrophic lateral sclerosis, click here https://institutducerveau-icm.org/fr/maladie-de-charcot/   

In the field of adult motor neuron diseases (primarily amyotrophic lateral sclerosis, or ALS), clinical research is focused on two main objectives:  

Gain a better understanding of the mechanisms underlying the disease.

To this end, various types of patient data are used: clinical, biological, genetic, imaging, neurophysiological, and/or histological data. In ongoing studies, the collection and analysis of this data help address several key issues: better understanding the heterogeneity of ALS, developing new diagnostic biomarkers, identifying imaging markers in asymptomatic individuals carrying a genetic mutation that puts them at risk of developing the disease, and improving our understanding of the role of certain signaling pathways in maintaining motor function.  

Development of new treatments through clinical trials.

ALS is a disease for which there is currently no cure, but a better understanding of its pathophysiological mechanisms is enabling the development of new drugs that target various mechanisms of action. These trials, which are most often international and conducted in partnership with pharmaceutical companies, allow for the study of the safety and efficacy of new treatments. The treatments currently being tested aim to reduce neuronal death (through mechanisms involving oxidative stress, cellular or protein energy metabolism, iron metabolism, neuroinflammation, etc.) or to slow the decline in motor function (through a molecule that activates a protein promoting muscle contractility). Another innovative therapeutic approach involves gene therapies targeting genes responsible for ALS. A recent study demonstrated the beneficial effect on the progression of ALS of a treatment administered via intrathecal injection in patients carrying a mutation in the SOD1. Thanks to these results, some patients whose ALS is linked to a mutation in the SOD1 can now access this treatment through compassionate use, and an ongoing study aims to determine whether its effect in asymptomatic carriers of the mutation could delay the onset of disease symptoms.  

To learn more about amyotrophic lateral sclerosis, click here https://institutducerveau-icm.org/fr/maladie-de-charcot/