SOURCE: Cell Gene Therapeutics

Cell Gene Therapeutics

April 12, 2013 11:24 ET

Hope for 560 Million Patients Suffering From Rare Diseases

The Creation of Gene Therapy International Consortium Aims to Speed Up Results That Will Change the Lives of These Patients 

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QUEBEC--(Marketwired - Apr 12, 2013) - Due to the progress in genomics, we now know the cause of over 10,000 inherited diseases attributed to the malfunction of a single gene. The next step is to develop gene therapies. "If we have adequate financial support for research on gene therapy for rare diseases, you can develop effective treatments for most of them during the next ten years," says Professor Jacques Tremblay, Professor of the Molecular Medicine department of Laval University (Canada), President of the Quebec Association of Gene Therapy, President of Cell Gene Therapeutics Quebec, a company located in Quebec city, Canada and the founder of the International Consortium of Gene Therapy.

The main problem at present is that there is a considerable decrease in funding for research in this area. "Especially when it comes to research focused on certain rare diseases such as Friedrich's Ataxia and Duchenne Muscular Dystrophy, which most pharmaceutical companies would not be interested in attributing resources because the financial return is very low. Therefore, these disorders are said to be orphan diseases. Essentially, the funding for such research comes from small foundations set up by relatives of patients," says the expert and member of the Research Center of the Centre Hospitalier Universitaire de Québec, Canada. 

What you may not yet realize is that, when put together, rare diseases affect a huge portion of the world population, i.e., eight percent of humanity. "These are staggering numbers and what is important is that when you do a research to correct a particular monogenetic disease, the knowledge acquired may be applied to many other hereditary diseases. Therefore a consortium for gene therapy will permit to accelerate the research, the obtention of results and the commercialisation of the treatments. Note that these treatments would benefit to approximately 560 million peoples," commented the scientist.

To change the scenario, Professor Tremblay mobilized 50 other renowned researchers who have published along with him, in the latest issue of the journal Molecular Therapy, a letter of intent for the creation of the International Consortium of Gene Therapy that aims to overcome these difficulties of the development of treatments. "The creation of the Consortium will be equivalent in the medical field, as was Project Apollo to the space race. Our hope is that, in a short time, we could improve the health of patients. Likewise, we expect that the technologies developed during this huge project will become economically viable for biotechnology companies. Accordingly, our hope is that the resulting treatments will be available for the whole world population."

Gene therapy has already begun to show significant results, he assures. Patients with hemophilia and hereditary blindness were cured after the application of this type of therapy. The same success was achieved with children suffering from hereditary immune deficiency and needing to live within bubbles, to avoid dying following an infection.

Some of these treatments are currently performed by removing some stem cells from the patient himself, the cells are then genetically corrected in culture and transplanted back to the same patient. Other techniques are also being developed in order to repair the defective gene.

Currently, we cannot expect investment by pharmaceutical companies, the governments of developed countries need to support such research so that the project could become more attractive to biotechnology companies. Professor Tremblay suggests. "Canada should increase funding targeting gene therapy research, as it already does since a few years to stimulate the development of the Genome Project aiming to sequence the genome of different species and identify mutations."

The authors of the intent letter and other researchers interested in this cause, will meet next May 17th in Salt Lake City, United States. There will be a workshop during the annual meeting of the American Society of Gene and Cell Therapy. This event will set the foundation of this Consortium.

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