A Big Step Forward Against Parkinson's Disease Through Michael J. Fox | Forbes France

Cellular Waste



The mutation in the gene that makes LRRK2 is the most common genetic cause of Parkinson’s disease

This variation was discovered 15 years ago. Sergey Brin, co-founder of Google , carries a copy, and his company 23andMe, a start-up of genetics for the general public, studied it. Pfizer, Merck and Genentech have all been working on drugs targeting this gene, but it has been discovered that they appear to cause disturbing lung changes in animals and all studies have been halted. It seemed at that time that this genetic index possibly containing the key to a cure for Parkinson’s disease could never be studied.

It was then that the Fox Foundation


joined the party, organizing meetings at which all companies involved in the study of treatments acting on the LRRK2 put their results together and tried to understand the origin of the problem. The Genentech team (now Denali’s) participated in these meetings and subsequently established a company and patented the Genentech molecule. It is this treatment whose data has been presented these days.

The current study does not include any patients with Parkinson’s disease, but it answers two questions: is the LRRK2 molecule able to block it enough to have an effect on Parkinson’s patients? And can she do it without serious side effects, especially in the lungs or blood pressure? It seems that the answer is yes.

There was a slight numerical difference between the placebo group and the groups receiving different doses of Denali’s drug, but the effects appear to have not worsened as doses increased, were not statistically significant, and did not seem to be of clinical importance. “The differences between the different assays are not clinically significant, and therefore not indicative of a dose-related effect,” says Carole Ho, Denali’s chief medical officer. Slight changes in blood pressure have been observed, but without researchers being able to match the drug.

Finally, genetic studies of individuals with mutations in the gene responsible for the synthesis of LRRK2 revealed no pulmonary problems.


Denali molecule 

The next step will be to test the Denali molecule in patients with Parkinson’s disease, both in those whose disease is caused by a mutation in the gene responsible for the production of LRRK2 and in those with Parkinson’s disease. is not related to this enzyme. Indeed, for researchers, such a drug could perhaps also have a positive effect on them. According to Ho, Denali intends to begin testing on the first patient of these studies by the end of the year. One of the biggest challenges will be finding people with Parkinson’s disease who have LRRK2.

Denali has partnered with Centagene, a genetic testing company, to find them more easily. GlaxoSmithKline, which also prepares an inhibitor of LRRK2, has partnered with 23andMe and will communicate with patients in the 23andMe database who carry the mutation.

” We find it both very important and promising to see the testing of an LRRK2 inhibitor begin in humans, ” said Brian Fiske, Vice President of the Fox Foundation. ” Of course, this is only the first stage of the trials, but it is for us one day to mark a milestone. “


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