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Thérapie génique : Des dons à la réalité

31 octobre 2016Édith LacroixNon classé

Bonne nouvelle !

La Coalition pour la recherche sur le syndrome de Hunter – Hunter Syndrome Research Coalition (qui regroupe des fondations et des familles d’enfants atteints du syndrome de Hunter ou MPS II, comme la nôtre) a récemment donné 200 000 $US pour appuyer la prochaine phase du programme de thérapie génique appliqué au syndrome de Hunter II piloté par les D. McCarty et D. Fu de l’Hôpital pour enfants Nationwide (Ohio, États-Unis).

Nous sommes incroyablement reconnaissants envers nos donateurs – amis, familles et même étrangers – qui rendent ce travail possible. Les prochaines étapes du financement de la recherche nécessiteront des efforts encore plus concertés : plus de deux millions de dollars sont nécessaires pour amener la recherche à un essai clinique complet chez l’humain. Nous serons bientôt à la recherche d’un soutien pour nos efforts futurs.

Merci beaucoup pour vos dons et vos efforts jusqu’à présent afin d’aider à sauver cette génération de garçons atteints du syndrome de Hunter!

Vous trouverez ci-après notre communiqué de presse (en anglais seulement).

p1090841

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COALITION OF PARENT-LED FOUNDATIONS BRINGS GENE THERAPY TREATMENT CLOSER TO REALITY 

Parent led-fundraising efforts have donated over $500,000 to treat the rare disease Hunter Syndrome (MPS II)

Vancouver, British Columbia – Over the past three years, parent-led efforts have raised half a million dollars to support gene therapy research at Nationwide Children’s Hospital (Columbus, Ohio) to treat the ultra-rare disease Hunter Syndrome (also known as Mucopolysaccharidosis or MPS II). This month alone, Vancouver, Toronto and Montreal based MPS II Research Fund, along with Nashville-based Saving Case & Friends, Ohio-based Help Extinguish Hunter Syndrome, and Texas-based Hunter Syndrome Foundation collectively donated $200,000 to bring the research closer to a human clinical trial.

Together with several other parent-led fundraising efforts – Caring for Carter, Ryan Our Lion, and Hope 4 Hunter, these foundations have supported this research, led by Douglas McCarty, Ph.D. and Haiyan Fu, Ph.D. of Nationwide, for several years.

Hunter Syndrome is a rare, genetic condition that affects approximately 2,000 patients worldwide, almost exclusively young boys. Patients are missing an enzyme, resulting in the accumulation of cellular waste throughout the body. Babies develop normally for the first few years, then begin to experience progressive symptoms like stiff joints, enlarged liver and spleen, behavioral problems, constant ear infections and runny nose, and heart valve complications. The average life span for someone with the most common, severe form of the disease is in the early teens.

There is no cure for the disease, although once diagnosed, patients can begin receiving a weekly infusion of an enzyme replacement therapy. This medication, Elaprase, is one of the most expensive in the world, often $400,000 or more per patient, per year, and only stabilizes some of the physical symptoms of the disease. Because it does not cross the blood-brain-barrier, it does nothing to prevent the progressive brain damage that occurs in most children affected by the disease.

Dr. McCarty notes, “This gene therapy for MPS II is the result of more than a decade of collaborative research efforts of the research team here at Nationwide and Dr. Joseph Muenzer [of the University of North Carolina, Chapel Hill], with support from MPS II patient family foundations. This gene therapy approach targets the root cause of MPS II by delivering the correct gene using a vector that can cross the blood-brain-barrier. Our preclinical data have shown great promise with lifelong benefits in MPS II mice after a single intravenous injection. We believe that we are well positioned to move forward towards a Phase 1/2 clinical trial in patients with MPS II.”

To raise funds, the foundations organized local fundraisers and also promoted an award-winning online campaign called Project Alive (ProjectAlive.org). The campaign received a Telly Award for its powerful and emotional video and an Honorable Mention for its song “Alive,” written by a parent of a child with Hunter Syndrome, from the International Songwriting Competition.

 About The MPS II Research Fund

Started in 2004, the MPS II Research Fund is a leading Hunter Syndrome research and advocacy organization within The Isaac Foundation, raising awareness of Hunter Syndrome globally, funding curative research for the disease, and advocating for patients and families affected by Hunter Syndrome. For more information, visit: www.treypurcell.com or www.theisaacfoundation.com/MPSII/.

Extrait du site Internet du Fonds de recherche MPS II – MPS II Research Fund (http://www.treypurcell.com/blog/recent-blogs/).

Édith Lacroix
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