Telethon presents the results of a year’s work. The comparison with scientific publications in the rest of the world is flattering. Montezemolo “When we want to, we Italians are capable of being competitive".
A record-breaking yearly report was presented today by the Telethon Foundation at the LUMSA University’s Sala Giubileo during the conference entitled “Actions that yield results”, with funds for research up 12 percent, progress made in finding a cure for a constantly increasing number of genetic diseases, and a boom in the scientific output.
«For us these figures are those that any company would describe as profit,” said Telethon’s president Luca di Montezemolo addressing the Foundation’s “shareholders”. “In our case there are no profits or dividends, but all funds are invested to finance the best possible research projects selected by our international scientific committee. This level of excellence is globally acknowledged as also proven by the statistics that see us leading the world rankings, ahead of Europe and the United States. All this once again proves that Italy is capable of being competitive in the world, when it combines the solidarity of its citizens and the talent of its best brains with a transparent, rigorous and excellent organization».
Just a little over three months before closing the year’s balance sheet (the Foundation’s financial year ends on June 30th) and two months before the traditional December TV marathon, Telethon has presented the results of this year’s work. Thanks to the generosity of Italian citizens and companies, 28.5 million euro, 12 percent more than the year before, have been allocated to research on genetic diseases. With these funds, Telethon has financed 40 research projects involving a total of 101 research laboratories all over Italy, as well as the work done in its institutes at the Istituto Tigem in Naples, the Istituto Hsr-Tiget in Milan, the Telethon Dulbecco (DTI) career programme currently active in 20 Italian laboratories in Italy, and at Tecnothon, the Sarcedo (Vicenza) technological research laboratory devoted to creating and developing prototypes of objects capable of improving living standards for the disabled. Telethon’s network of genetic biobanks, a fundamental resource for research and diagnostics, has also grown and now has ten centres.
As far as research is concerned, after the success achieved for ADA SCID immunodeficiency through gene therapy, which, with a single treatment made it possible for 14 children from all over the world to lead a normal life, in the past year important clinical trials were started on Duchenne muscular dystrophy and continued on metachromatic leukodystrophy, Wiskott Aldrich syndrome, Stargardt syndrome and Leber’s congenital amaurosis. Furthermore, laboratory research has reached a turning point for at least another 15 diseases for which clinical trials on patients will begin in the coming years.
The presentation of this new report also coincided with an important anniversary, that of the signing of a historical agreement with the pharmaceutical multinational GlaxoSmithKline to develop and make available to patients all over the world the genetic treatment devised by researchers at the Hsr-Tiget for seven serious genetic diseases. As managing director Francesca Pasinelli said, «This partnership represents the beginning of a new phase in the history of our organization. To fully maintain the promise made from the very start to patients, we must in fact involve all other parties usually involved in the development of therapies, such as, first of all, the pharmaceutical industry and the regulating authorities. It is only in this manner that the therapeutic strategies developed by researchers can reach all those who are ill».
The current situation in the progress made by research was empathised by Luigi Naldini, director of the Hsr-Tiget : «We are currently testing the safety and effectiveness of genetic treatment for two serious genetic diseases, Wiskott-Aldrich syndrome, a rare immunodeficiency, and for metachromatic leukodystrophy, which instead affects the nervous system resulting in the progressive loss of cognitive and motor capabilities. At the moment seven children have received treatment and although it is premature to reach conclusions, and we will only have conclusive results in two years, preliminary data is extremely encouraging. Treatment appears to have no immediate side-effects and is very efficient in terms of transferring the correct gene to the bodies of these patients».
Luc Debruyne, president and CEO of Gsk Italia, ha also expressed his satisfaction:«Since more than 20 years we both work in the rare diseases arena and few perhaps know that from 1983 to 2011 GSK brought 14 treatments to patients for those pathologies. Just one year after combining our excellences in terms of research, innovation and sustainability we are even more confident that this new model of collaborative research can be successful».