To date, there are no therapeutic strategies that are able to significantly modify the course of Parkinson’s. What is certain is that the time factor plays a fundamental role: when the first motor symptoms appear (such as slow movement or tremor at rest), in fact, the disease is already at an advanced stage. Some studies have suggested that mutations in the Lrrk2 gene are one of the most common genetic risk factors: starting from here, in the United States, the first phase of testing of a molecule that inhibits the enzyme produced by Lrrk2 has been completed and that it could slow the progression of the disease. The results are published on Science Translational Medicine.
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The increasing prevalence of Parkinson’s
Parkinson’s disease is a progressive neurodegenerative syndrome that today affects 5 million people worldwide, of which about 400,000 are in Italy. It affects about 2% of adults aged 65 and over and its incidence is increasing with the aging of the world population: it is estimated that over the next fifteen years there will be six thousand new cases every year, half of which are of working age. In addition to the known physical symptoms, such as muscle stiffness, tremor at rest, slowness of movement and resistance to passive movements, the disease can also manifest itself with depression and slowness in speech.
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The role of lysosomes
The onset of Parkinson’s has been linked to the increase in the production of an enzyme by the Lrrk2 gene (a kinase), which in turn triggers a dysfunction in some cellular structures, the lysosomes. These can be considered as a center for the disposal and redistribution of “waste” present in all cells, and their malfunction leads to an accumulation of toxic substances that can cause brain cell death and a lack of dopamine: precisely the ingredients of the Parkinson’s.
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“The therapeutic goal of inhibiting Lrrk2 is to improve lysosomal function and slow disease progression,” explains Danna Jennings, director of clinical research at the Denali Therapeutics Institute for Neurodegenerative Disorders in San Francisco, California, to Health. author of the study. “We are not inhibiting the actual gene. Rather, the therapy based on small molecules of Dnl201 inhibits the activity of the kinase (the enzyme) Lrrk2 with the aim of improving the function of the lysosome: this could prevent the accumulation of toxic proteins that lead to neurodegeneration ( death of neurons and other brain cells, ed). We are conducting two advanced-stage clinical trials to test this approach in people with early-stage Parkinson’s disease who do not have a mutation in the Lrrk2 gene and another in patients who have a mutation in the Lrrk2 gene. We believe that improving lysosomal function may be beneficial in both populations. And because Parkinson’s is a progressive disease, we believe there may be a benefit in preserving neuronal function and health even for people who have had it for the longest time. However, this aspect must be tested in other clinical studies “.
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The results of the first tests on the new molecule
The molecules of Dnl201 demonstrated the ability to suppress the activity of the enzyme (and therefore to enhance the function of lysosomes) in rats, and were found to be safe even when administered to macaques for 28 days. Their functionality was then the subject of the first phase of a clinical study which involved 122 healthy volunteers and 28 patients with Parkinson’s disease, in which a reduction in the enzyme associated with Lrrk2 in the blood was noted. “We found improvements in fluid biomarkers associated with lysosomal function in both animals and healthy humans and people with Parkinson’s in our early-stage clinical trials,” Jennings concludes. “However, we need to investigate whether treatment with our Lrrk2 inhibitor can actually slow the progression of Parkinson’s in larger, longer-term studies. We have recently launched an experiment, the Luma study, on people with early-stage Parkinson’s to test this hypothesis “.
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