{"id":2565,"date":"2024-03-14T14:42:37","date_gmt":"2024-03-14T18:42:37","guid":{"rendered":"https:\/\/cqdm.org\/en\/news-and-events\/treatment-of-hirschsprung-disease-by-rectal-administration-of-neurotrophic-factors\/"},"modified":"2024-09-25T16:24:16","modified_gmt":"2024-09-25T20:24:16","slug":"treatment-of-hirschsprung-disease-by-rectal-administration-of-neurotrophic-factors","status":"publish","type":"post","link":"https:\/\/cqdm.org\/en\/news-and-events\/treatment-of-hirschsprung-disease-by-rectal-administration-of-neurotrophic-factors\/","title":{"rendered":"Treatment of Hirschsprung disease by rectal administration of neurotrophic factors."},"content":{"rendered":"

Principal Investigator: <\/o_p><\/strong><\/p>\n

Nicolas Pilon \/<\/strong> <\/strong>Universit\u00e9 du Qu\u00e9bec \u00e0 Montr\u00e9al – UQAM<\/o_p><\/strong><\/p>\n

Co-Investigators : <\/o_p><\/strong><\/p>\n

Lekha Sleno \/ <\/b>Universit\u00e9 du Qu\u00e9bec \u00e0 Montr\u00e9al –
\nUQAM<\/o_p><\/strong><\/p>\n

Project of $1 500 000  over 3 years<\/o_p><\/strong><\/p>\n

Supported by CQDM through : <\/o_p><\/strong><\/p>\n

Minist\u00e8re de l\u2019\u00c9conomie, de l\u2019Innovation et de
\nl\u2019\u00c9nergie du Qu\u00e9bec (MEIE)<\/o_p><\/p>\n

And by co-funding partners:<\/o_p><\/strong><\/p>\n

Neurenati<\/o_p><\/strong><\/p>\n

The project in details : <\/o_p><\/strong><\/p>\n

Challenge:<\/strong>  Hirschsprung disease (HSCR) is a deadly
\ncongenital malformation where the enteric nervous system (ENS) is missing from
\nthe colon. Because the ENS controls gastrointestinal motility, the ENS-devoid
\nsegment remains constantly contracted, resulting in obstruction by fecal
\nmaterial (megacolon). The ENS also controls other gastrointestinal functions,
\nexplaining why children with HSCR have high risk <\/a>[MR1]<\/a> of developing
\nfatal systemic infection by gut-resident microbes. Current treatment consists
\nof painful surgical removal of the ENS-devoid segment followed by re-connection
\nof innervated colon to the anus. Unfortunately, many children have
\npost-surgical complications like stool leakage and life-threatening
\ninflammation, often leading to hospital re-admission and repeat surgery.<\/o_p><\/p>\n

Solution
\n:<\/strong> Based on recently published results by Prof.
\nPilon\u2019s team, a non-surgical regenerative medicine approach might now be
\npossible for children with HSCR. Specifically, they found that a protein called
\nGDNF can induce the formation of a new ENS in the colon of three HSCR mouse
\nmodels. The induced ENS significantly improves gastrointestinal functions,
\ndelaying death of many of these HSCR mice. New data now show that a small
\nmolecule normally produced by gut-resident microbes as well as another neurotrophic
\nfactor can both enhance the effect of GDNF treatment, not only on the ENS but
\nalso on other components of the gastrointestinal ecosystem. Moreover, via a
\ncollaboration with the team of Prof. Sleno, they found that specific proteins
\nand metabolites present in the colon wall could serve as biomarkers predictive
\nof a positive response to GDNF treatment.<\/o_p><\/p>\n

Expected
\nAchievements \/Impacts :<\/strong>  – This
\ncollaborative research project with a Quebec company, Neurenati, will elucidate
\nmechanism of action of GDNF and molecules that increase the therapeutical
\npotential. Also, the research team will define pharmacokinetic properties of
\nGDNF, which will help to develop an optimal formulation for clinical assays.
\nThis project will be led by a multidisciplinary team that will use a wide array
\nof new and innovative technologies and approaches. This research will help
\npropel Quebec as a leader in the field of rare disease research.<\/p>\n

<\/p>\n

<\/p>","protected":false},"excerpt":{"rendered":"

Principal Investigator: Nicolas Pilon \/ Universit\u00e9 du Qu\u00e9bec \u00e0 Montr\u00e9al – UQAM Co-Investigators : Lekha Sleno \/ Universit\u00e9 du Qu\u00e9bec \u00e0 Montr\u00e9al – UQAM Project of $1 500 000  over 3 years Supported by CQDM through : Minist\u00e8re de l\u2019\u00c9conomie, de l\u2019Innovation et de l\u2019\u00c9nergie du Qu\u00e9bec (MEIE) And by co-funding partners: Neurenati The project in details : Challenge: …<\/p>\n","protected":false},"author":2,"featured_media":1709,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[1],"tags":[],"class_list":["post-2565","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-our-news"],"acf":[],"_links":{"self":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/posts\/2565","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/comments?post=2565"}],"version-history":[{"count":1,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/posts\/2565\/revisions"}],"predecessor-version":[{"id":4727,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/posts\/2565\/revisions\/4727"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/media\/1709"}],"wp:attachment":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/media?parent=2565"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/categories?post=2565"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/tags?post=2565"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}