{"id":3032,"date":"2023-03-14T14:42:37","date_gmt":"2023-03-14T18:42:37","guid":{"rendered":"https:\/\/cqdm.org\/en\/achievement\/funded-projects\/treatment-of-hirschsprung-disease-by-rectal-administration-of-neurotrophic-factors-2\/"},"modified":"2024-09-03T10:20:37","modified_gmt":"2024-09-03T14:20:37","slug":"treatment-of-hirschsprung-disease-by-rectal-administration-of-neurotrophic-factors-2","status":"publish","type":"project","link":"https:\/\/cqdm.org\/en\/achievement\/funded-projects\/treatment-of-hirschsprung-disease-by-rectal-administration-of-neurotrophic-factors-2\/","title":{"rendered":"Treatment of Hirschsprung disease by rectal administration of neurotrophic factors."},"content":{"rendered":"\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<div class=\"wp-block-columns is-layout-flex wp-container-core-columns-is-layout-28f84493 wp-block-columns-is-layout-flex\">\n<div class=\"wp-block-column is-layout-flow wp-block-column-is-layout-flow\">\n<p class=\"is-style-suptitle\"><strong>Principal Investigator:<\/strong><\/p>\n\n\n\n<p><strong><strong>Nicolas Pilon<\/strong><br><\/strong>Universit\u00e9 du Qu\u00e9bec \u00e0 Montr\u00e9al &#8211; UQAM<\/p>\n<\/div>\n\n\n\n<div class=\"wp-block-column is-layout-flow wp-block-column-is-layout-flow\">\n<p class=\"is-style-suptitle\"><strong>Co-investigators<\/strong><\/p>\n\n\n\n<p><strong>Lekha Sleno<\/strong><br>Universit\u00e9 du Qu\u00e9bec \u00e0 Montr\u00e9al &#8211;<br>UQAM<\/p>\n<\/div>\n<\/div>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\"><strong>Project of $1&nbsp;500 000 &nbsp;over 3 years<\/strong><\/h2>\n\n\n\n<ul class=\"is-style-checkmark wp-block-list\">\n<li><strong><strong>Supported by CQDM through:<\/strong><\/strong> Minist\u00e8re de l\u2019\u00c9conomie, de l\u2019Innovation et de l\u2019\u00c9nergie du Qu\u00e9bec (MEIE)<\/li>\n<\/ul>\n\n\n\n<ul class=\"is-style-checkmark wp-block-list\">\n<li><strong><strong>And by a co-funding partner:<\/strong><\/strong> Neurenati<\/li>\n<\/ul>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\"><strong>Challenge<\/strong><\/h2>\n\n\n\n<p>Hirschsprung disease (HSCR) is a deadly congenital malformation where the enteric nervous system (ENS) is missing from the colon. Because the ENS controls gastrointestinal motility, the ENS-devoid segment remains constantly contracted, resulting in obstruction by fecal material (megacolon). The ENS also controls other gastrointestinal functions, explaining why children with HSCR have high risk [MR1]\u00a0of developing fatal systemic infection by gut-resident microbes. Current treatment consists of painful surgical removal of the ENS-devoid segment followed by re-connection of innervated colon to the anus. Unfortunately, many children have<br>post-surgical complications like stool leakage and life-threatening inflammation, often leading to hospital re-admission and repeat surgery.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Solution<\/h2>\n\n\n\n<p>Based on recently published results by Prof. Pilon\u2019s team, a non-surgical regenerative medicine approach might now be possible for children with HSCR. Specifically, they found that a protein called GDNF can induce the formation of a new ENS in the colon of three HSCR mouse models. The induced ENS significantly improves gastrointestinal functions, delaying death of many of these HSCR mice. New data now show that a small<br>molecule normally produced by gut-resident microbes as well as another neurotrophic factor can both enhance the effect of GDNF treatment, not only on the ENS but also on other components of the gastrointestinal ecosystem. Moreover, via a collaboration with the team of Prof. Sleno, they found that specific proteins and metabolites present in the colon wall could serve as biomarkers predictive of a positive response to GDNF treatment.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><strong>Achievements\/Impact<\/strong><\/h2>\n\n\n\n<p>This collaborative research project with a Quebec company, Neurenati, will elucidate mechanism of action of GDNF and molecules that increase the therapeutical potential. Also, the research team will define pharmacokinetic properties of GDNF, which will help to develop an optimal formulation for clinical assays.<br>This project will be led by a multidisciplinary team that will use a wide array of new and innovative technologies and approaches. This research will help propel Quebec as a leader in the field of rare disease research.<\/p>\n","protected":false},"featured_media":3823,"template":"","project-category":[97],"class_list":["post-3032","project","type-project","status-publish","has-post-thumbnail","hentry","project-category-biological-and-therapeutic"],"acf":[],"_links":{"self":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/project\/3032","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/project"}],"about":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/types\/project"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/media\/3823"}],"wp:attachment":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/media?parent=3032"}],"wp:term":[{"taxonomy":"project-category","embeddable":true,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/project-category?post=3032"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}