To 5 years of gene therapy by stem cells?

Photo courtesy
Gene therapy by stem cells do cure not Alexis Verville-Tailleur, who is suffering from leukodystrophy of form 4H, but not to hinder the development of their illness.

Valerie Bidégaré

Monday, 16 October 2017 00:00

UPDATE
Monday, 16 October 2017 00:00

Look at this article

Hope to see the birth of a stem cell treatment to halt the disease, a genetic degenerative of their teenager is revived for a couple of Quebec. The first samples that will help launch the research in the laboratory has finally been completed.

“It’s been 11 years that one picks up under the with the foundation and that one passes all the preliminary steps prior to treatment and then, the day has come, it is almost a treatment in the next 2 to 5 years “, confides with emotions Marjolaine Verville, in an interview with the Newspaper.

The mother of Alexis Verville-Tailleur has launched the Foundation Leuco Dystrophy in order to find a treatment for this genetic disease, degenerative whose symptoms resemble those of multiple sclerosis. His boy, 17 years old, is suffering from leukodystrophy of form 4H since he was three years old, as published by the Journal in January, leaving glimpses of the spectre of death in early adulthood. The 20 June, after a battery of tests, the first samples of stem cells have been carried out on the teenager.

“The levies were to be made in Montreal, but my boy has had pneumonia so we instead took the direction of the CHUL,” Mrs. Verville. “The blood is only good for four hours and had to go to Montreal then my boyfriend went in Orléans Express and sent the samples by bus. It was a race against the clock. “

Correct the anomalies

The couple will know in a few weeks if enough stem cells have been collected to allow a team of researchers from the University of Montreal in an attempt to develop a treatment consisting of a gene therapy by stem cells. “They take the stem cells specific to the patient to transform them into the cell of the brain. They will correct the diseased cells with transformed cells. It remyélinise the brain because of the problem with leukodystrophy, it is a lack of myelin in the brain “, illustrates Ms. Verville, who argues that in a realistic world, the treatment could be ready in 5 years.

“This would be the grand finale. We will not be able to go back even if it is what we would like. Alexis will remain disabled, but at least his condition will remain stable so it will be a better quality and a better hope of life “, does he / she share.

The mother intends to then fight so that this expensive treatment, the price of which would vary between $ 50,000 and $ 100,000 per patient, is accessible to the greatest number of children possible.

“If they do not have enough stem cells for Alexis, it does not change the general picture because they have started the tests with one subject in march. Once the lab tests are completed, they will need to collect new stem cells to inject the treatment in the spiciness in four places in the brain because they can not take the old cells, ” she explains.

“It happens finally something concrete. It is close to the goal in the hope that all goes well because every day counts. “

Gene therapy by stem cells

• Collect a patient’s stem cells
• Turn them into brain cells
• Remyelinate the brain by injecting the cells transformed in the brain of the patient in order to replace the defective cells
• The treatment could halt the progression of leukodystrophy
• He could see the light of day in 2 to 5 years
• It would be a first
• The price of the injections would vary between $ 50,000 and $ 100,000 per patient