(26, October, 2016) “The ‘boy in the bubble’ moved a world he could not touch” is the epitaph on David Phillip Vetter’s gravestone, the American boy who was affected by ADA-SCID, a genetic disease, spent the 12 years of his life inside a sterile nylon bubble so as not to come into contact with lethal viruses and bacteria. David’s story moved the whole world and generated a wave of opinion that pushed towards the investment in research to defeat this disease. Today, the answer is finally here thanks to gene therapy that stemmed from an all-Italian alliance between Telethon Foundation, San Raffaele Hospital and the pharmaceutical company GlaxoSmithKline Italy. Milan is now the destination for all the children affected by this disease. Giuseppe Recchia, Medical&Scientific Director of GlaxoSmithKline is the one who has recalled David’s story during the course of a conference organised in Rome by Farmindustria titled “Advanced Treatments. An Italian Success” (Le terapie avanzate. Un successo made in Italy). One of the great successes- certainly not the only one- of Italian research in the sector is that- among the six advanced therapies approved by the EU- three of them are international achievements of Italian pharmaceutical research. The first drug in Europe consisting of stem cells, the first gene therapy and the first cellular therapy. More broadly, Italy is contributing to a “Renaissance of research which bears the white, red and green [of our flag]. It is an important process. There are 7,000 new drugs to arrive. If we look at the context of biotechnology, it is an extraordinary Renaissance” according to the President of Farmindustria, Massimo Scaccabarozzi. He adds that “research has become highly productive. Today we are talking about advanced treatments that give us access to medicines that are borne from biotechnological and biological processes. Therefore, medicines that derive from living cells and tissues that will allow for a personalised and faster treatment. The right drug for the right person knowing whether it will work or not and with excellent tolerance levels”. Scaccabarozzi further underlines that “more than 202 drugs are already available from biotechnologies and 324 more will arrive in the next few years. This is why we speak of a ‘Renaissance’. Research today is not what it used to be 10 years ago. Today, thanks to new impulses and investments, we are helping the world”. According to Eugenio Aringhieri, President of Gruppo Biotecnologie of Farmindustria, “biotech pharmaceutical research is the bridge between the present and the future. With tissue engineering, cellular therapy and gene therapy; pathologies in the areas such as cardiovascular diseases, haematology, dermatology, inflammatory and autoimmune diseases, muscular-skeletal diseases, neurology, oncology, urology; have the possibility to be treated. Many of the 27 projects under development are in their final stages especially the ones regarding gene and cellular therapy, to the benefit of quality of life of those affected by rare diseases”. (Roc)
