{"id":1830,"date":"2023-04-13T17:43:13","date_gmt":"2023-04-13T15:43:13","guid":{"rendered":"https:\/\/demoucelle.sites-stag.agency\/uncategorized\/the-demoucelle-parkinson-charity-supports-research-to-find-a-molecule-that-can-clean-up-the-brain\/"},"modified":"2023-04-13T17:43:13","modified_gmt":"2023-04-13T15:43:13","slug":"the-demoucelle-parkinson-charity-supports-research-to-find-a-molecule-that-can-clean-up-the-brain","status":"publish","type":"post","link":"https:\/\/demoucelle.sites-stag.agency\/fr\/uncategorized\/the-demoucelle-parkinson-charity-supports-research-to-find-a-molecule-that-can-clean-up-the-brain\/","title":{"rendered":"The Demoucelle Parkinson Charity supports research to find a molecule that can \u2018clean up\u2019 the brain"},"content":{"rendered":"

<\/a>The Demoucelle Parkinson Charity has decided to support a research project that aims to improve the ability of brain cells to deal with \u2018trash\u2019 i.e. old and damaged molecules. It is hoped that by reducing the amount of waste buildup in the brain, it will be possible to slow the progression of Parkinson\u2019s disease.<\/p>\n\n\n\n

The Demoucelle Parkinson Charity is working together with the Michael J. Fox Foundation for Parkinson\u2019s Research to support this study.<\/p>\n\n\n\n

For more information about the research, please click on this link or read the text below.<\/p>\n\n\n\n

https:\/\/www.michaeljfox.org\/grant\/testing-small-molecules-activate-removal-cellular-debris-treatment-parkinsons-disease<\/a><\/p>\n\n\n\n

\u2014\u2014\u2014\u2014\u2014<\/strong><\/p>\n\n\n\n

Testing Small M<\/strong>olecules that Activate Removal of Cellular Debris for the Treatment of Parkinson\u2019s Disease<\/strong><\/p>\n\n\n\n

Study Rationale:<\/strong><\/em>\nPeople with Parkinson\u2019s disease (PD) exhibit a diminished ability to remove cellular trash \u2014 including old and damaged molecules and organelles \u2014 from the brain. This impairment is caused by a reduction in trash collection and delivery (a process called macroautophagy) and a decline in the number and effectiveness of trash cans (compartments called lysosomes). Activation of TRPML1, a channel that resides on the lysosome, has been shown to enhance macroautophagy and to increase lysosome function and number in cells. Therapeutics that boost TRPML1 activity could therefore alleviate the symptoms and progression of PD.<\/em><\/p>\n\n\n\n

Hypothesis:<\/strong>\u00a0<\/em>\nWe hypothesize that developing small compounds that can activate TRPML1 should also enhance macroautophagy and increase lysosome function in animals and potentially in humans.<\/em><\/p>\n\n\n\n

Study Design:\u00a0<\/strong><\/em>\nIn this project, we seek to demonstrate that activation of TRPML1 increases macroautophagy and lysosome growth signaling in animals, and that this activation and its subsequent effects are dose dependent. We will then determine whether and how much activation of TRPML1 is needed to show beneficial effects in animal models of PD. Lastly, we seek to lay the foundation for development of an assay or suite of assays that will allow us to measure activation of TRPML1 and its subsequent activation of macroautophagy and lysosome growth signaling in future clinical trials.<\/em><\/p>\n\n\n\n

Impact on Diagnosis\/Treatment of Parkinson\u2019s Disease:<\/strong><\/em>\nEstablishing that activation of TRPML1 increases macroautophagy and lysosome growth signaling in animals, and that these effects can be monitored in potential future clinical trials, would provide a strong body of evidence supporting the development of small compounds that can activate TRPML1 for the treatment of PD.<\/em><\/p>\n\n\n\n

Next Steps for Development:<\/strong><\/em>\nSuccess in this grant would provide strong support for the continued advancement of Libra\u2019s small compound TRPML1 activators into animal toxicology studies, which are required for advancement into clinical safety evaluation in people with PD and healthy volunteers.<\/em><\/p>\n\n\n\n

Photo by Michael Longmire on Unsplash<\/p>","protected":false},"excerpt":{"rendered":"

Find out more about the 13th project supported by DPC<\/p>","protected":false},"author":1,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1],"tags":[],"class_list":["post-1830","post","type-post","status-publish","format-standard","hentry","category-uncategorized"],"_links":{"self":[{"href":"https:\/\/demoucelle.sites-stag.agency\/fr\/wp-json\/wp\/v2\/posts\/1830","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/demoucelle.sites-stag.agency\/fr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/demoucelle.sites-stag.agency\/fr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/demoucelle.sites-stag.agency\/fr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/demoucelle.sites-stag.agency\/fr\/wp-json\/wp\/v2\/comments?post=1830"}],"version-history":[{"count":0,"href":"https:\/\/demoucelle.sites-stag.agency\/fr\/wp-json\/wp\/v2\/posts\/1830\/revisions"}],"wp:attachment":[{"href":"https:\/\/demoucelle.sites-stag.agency\/fr\/wp-json\/wp\/v2\/media?parent=1830"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/demoucelle.sites-stag.agency\/fr\/wp-json\/wp\/v2\/categories?post=1830"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/demoucelle.sites-stag.agency\/fr\/wp-json\/wp\/v2\/tags?post=1830"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}