by Chiara Bidoli
The «Multi-round» tender launches a new tranche of funding that will allow the realization of 24 projects
Telethon Foundation announces the winners of the second phase of the «Multi round» call which allows 4.36 million euros to be assigned to 24 projects that will allow the study of the mechanisms of various genetic diseases and potential therapeutic approaches. Among the winning projects there are studies concerning pathologies which, although rare, are known as beta thalassemia, Kennedy’s disease, SMA, Duchenne muscular dystrophy, Huntington’s disease and cystic fibrosis, but the tender also rewards work on diseases which, due to the number of patients involved, can be defined as ultra-rare such as Niemann-Pick, Dravet syndrome, and dyskeratosis congenita.
The «Multi-round» call
«There are at least 6 thousand rare diseases and some of these affect a number of patients that can be counted on one hand, others affect hundreds if not thousands of patients. As Telethon we try not to leave anyone behind, as much as possible we try to address the spectrum of rare diseases”, explains Celeste Scotti, Telethon’s new Director of Research and Development.
The “Multi-round” call, launched in 2022, allows researchers working in Italy to present – in four tranches over three years – research projects on rare genetic diseases and, in case of approval, access funding.
«As Telethon we have several calls for tenders that cover a broad spectrum of research funding needs – explains Scotti -. This is called “Multi round” because 4 phases of access to the financing call have been foreseen. We are now working on the “next round”: by October 31st researchers will be able to present their projects and participate in the edition that will give access to funding in 2024.” With what criteria are the winning projects chosen? «The selection and review of projects is based on peer review: we receive funding proposals which are carefully and rigorously reviewed by a pool of international scientists – continues Telethon’s Director of Research and Development -. Projects receive a score based on the scientific rationale and solidity of the foundations, but also on the presence of preliminary data that allow us to tackle a rare disease that needs a cure. In particular, this call has two application fronts: one for “basic” research, which allows us to discover mechanisms of action, molecular details of certain diseases and another more applicative, closer to the clinic, which allows us to provide a proof of principle for a potential cure. This year we have financed 24 projects involving 12 Italian research institutes for a total of 4.3 million in funding. An important commitment that makes us proud of the scientific level achieved.”
The evolution of the Telethon foundation
There are between 2.1-3.5 million people affected by a rare disease in Italy and in the majority of cases these are children. Telethon is committed not only to funding research, but also to transforming the results into therapies accessible to all patients who must be able to count on a certain diagnosis and treatment. «Over the last 30 years the Telethon Foundation has moved with coherence and determination in financing research aimed at finding cures and treatments for rare diseases that pharmaceutical companies struggle to deal with. We are also the first non-profit organization in the world to take on the responsibility of commercializing a gene therapy for the immunodeficiency ADA-SCID, and we hope that others will decide to do as we do. We firmly believe that there can and must be a sustainable model for healthcare systems, for those who manage the therapy and for those who produce the medicines”, points out Scotti.
«In light of our choice to be an active part in the market for therapies for rare genetic diseases, we have chosen to expand our scientific staff with two key figures. In fact, the next few years will be increasingly challenging and it is necessary to add specific skills. Celeste Scotti (new director of research and development) and Stefano Vavassori (research manager) will be fundamental in allowing us to grow further, always remaining faithful to our goal of leaving no one behind”, explains Francesca Pasinelli, General Director of Telethon.
Research after Covid
« What Covid has taught us is that there is no research that is not useful. Let’s think about the two 2023 Nobel Prizes for medicine that were awarded to those (Katalin Karikó and Drew Weissman for the mRNA vaccines against Covid) who developed a technology that proved crucial in the pandemic, opening up a range of opportunities for a great number of diseases. In fact, when new technologies are developed, the applications can be very broad and, again, Covid has taught us that we can do great things quickly when we create a critical mass of people, technologies, skills and funding. And this also applies when it comes to rare diseases. However, a new communication approach is needed: rare diseases exist, they can be diagnosed and with rigorous and high-level research it is possible to find cures”, concludes Scotti.
October 18, 2023 (modified October 18, 2023 | 11:17)
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