Québec media have recently covered the story of Jayden, bubble boy, the first Québec patient successfully treated using gene therapy.

Jayden Duquette, five months old, was a patient of Dr. Élie Haddad, a pediatric immunologist at Ste-Justine children’s hospital in Montreal. He was born with an immune system deficiency caused by a genetic disorder which left him vulnerable to infections of all kinds. Jayden’s parents were given the opportunity to have him undergo treatment at the University of California, Los Angeles (UCLA), the only real hope for their son’s survival. In a few days, gene therapy was administered to Jayden by Dr. Donald B. Kohn, a pediatric onco-hematologist at UCLA. It was a complete success and he was immediately in remission. For Jayden’s parents, gene therapy was a miracle. Note that this treatment is also available in France and England.

Some genetic diseases for which there was no hope just a few years ago can now be treated with gene therapy. France, the USA and the UK are the leaders in this field. Healthcare research in this field of science will pave the way for a future industrial complex. In France, there is a move to industrialize innovative therapies to make them accessible to the medical world and to patients. Huge financial investments and substantial human resources are committed to this endeavor in advanced nations.

The hopes for patients and the parents of sick children are enormous. When we talk about genetic diseases, we are often discussing degenerative diseases that cause severe disabilities that last a lifetime. A huge loss for the people living with them and for society as a whole.

For a disease like Friedreich’s ataxia, work in gene therapy done by Professor Jacques P. Tremblay of the CHU in Québec City are fueling the hopes of people affected. Many monogenetic diseases (where a single mutation causes the disorder) could soon benefit from gene therapy and innovative therapies, more broadly.

Will Québec and Canada join the frontrunners? We have the people and the financial resources to do so. What are we waiting for?

References:

http://ici.radio-canada.ca/tele/decouverte/2015-2016/episodes/360692/bulle-papillon-heredite-bleu-canelle?isAutoPlay=1 [report in French]

http://www.institutimagine.org/en/