MONTREAL – A therapeutic approach “revolutionary” developed in Montreal could change the lives of children with Hirschsprung’s disease.
This rare genetic disease affecting approximately one newborn baby out of 5000 is characterized by a lack of enteric neurons in the wall of the colon.
Researchers of the Centre of excellence in research on orphan diseases – Foundation Courtois (CERMO-FC) of the Université du Québec à Montréal (UQAM) have been working on this advanced. They have obtained the collaboration of colleagues from the CHU Sainte-Justine and the Children’s Hospital of Philadelphia.
In his recently published study in the journal Gastroentorology,” professor Pilon and his colleague Rodolphe Soret, associate professor in the Department of biological sciences, tell of how they have been able to generate new enteric neurons in children with Hirschsprung’s disease. This approach could avoid the need for recourse to an operation.
“Although this surgery saves the lives of most affected children, many develop serious long-term sequelae, such as fecal incontinence and increased susceptibility to entérocolites. These effects can be fatal,” explained the professor Pilot “News UQAM”.
Tests on mice
To carry out their work, researchers have had to resort to the mouse. The rectal administration of the neurotrophic factor derived from glial cells can induce the formation of enteric neurons in the colon, which enabled the mice to live longer, written in “Actualités UQAM”.
The enteric neurons were absent during the launch of the tests, but they are then “multiplied”.
“In just a few days, there was already more than half of the neurons that are normally found in a colon health”, said the researcher, who believes that clinical trials in humans could begin within two years”.
“Unlike other groups around the world, our team has made the bet that it was possible to stimulate stem cells present locally within the affected area,” said the professor Pilon.
Pushing further their work, researchers conducted tests on samples of colons in post-operative children are living with Hirschsprung’s disease. “A similar response has been obtained, which leaves us to believe that the transfer could be possible in humans,” said Mr. Pilon.