What began in 2022, the Foundation has brought forth a particularly prolific two-year period. Not only in terms of scientific results but in the synergy between 46 public and private partners that promise to root new technologies in the research and production system and transfer the results from laboratories to drug development projects.
“Understanding the often very complex molecular bases of diseases means discovering new targets, which often occur unexpectedly. The revolution in the approach to treatment starts with the evolution of knowledge and transforms it into new drugs or gene therapy protocols. Italy cannot afford to watch the advancement of new therapeutic perspectives from afar but rather must participate in the future of medical discoveries, shares the President of the Foundation, Prof Rosario Rizzuto, who is the former Rector of the University of Padua and current Director of its Department of Biomedical Sciences. “One of our main objectives is to create the infrastructure within Italy to bring these drugs to fruition and an area dedicated to technology transfer.”
A concrete example of the innovations resulting from the research work of the National Center lies in a new understanding of the functions of mitochondria towards the development of new drugs. “We have discovered the role of mitochondria in processes beyond their known function, which is to give energy to biological processes” explains Prof. Rosario Rizzuto.
The advantage of drugs composed of non-coding RNAs comes from their ability to adapt to any molecular target, even difficult-to-reach tissues such as those found within the nervous system. These treatments follow the simple rules of base pairing nucleic acids, which makes this technology adaptable to a large number of applications.
According to Prof Stefano Gustincich, Director of the Central RNA Lab of the Italian Institute of Technology in Genoa and Director of the Center for Personalized Preventive and Predictive Medicine in Aosta, “non-coding RNAs is the Yin and Yang of new medicine. The Yin uses the therapeutic molecules to inhibit the expression of malfunctioning genes, for example reducing the production of toxic proteins. The Yang does the opposite, as it administers RNA molecules to stimulate the expression of genes so that they produce a greater quantity of protective proteins, or act on other mechanisms aimed at re-establishing correct cellular homeostasis.”
The National Center research group focused on pre-clinical development, Good Manufacturing Practices (GMP), and clinical trials of Gene Therapy Medicinal Products (GTMP) explains that they have begun the first clinical trials in Europe dealing with acute T lymphoblastic leukemia and neoplasms of the central nervous system. The leader of this particular research group, Prof Franco Locatelli, Director of the Department of Hematology and Pediatric Oncology at the Bambino Gesù Children’s Hospital, shares more, “trials have already begun on autoimmune diseases, such as systemic lupus erythematosus or inflammatory muscle diseases. We have also entered into other fascinating areas including the identification of useful targets in the field of acute myeloid leukemia and the development of alternative cellular platforms to the classic patient-specific T lymphocytes. Regarding treatments of hereditary diseases, we have reached advanced stages of development for hemoglobinopathies (thalassemia and sickle cell anemia), rare lysosomal storage diseases that claim an adverse impact on the survival and quality of life of children.” Prof Locatelli continues by confirming that the clinical application of additional project development in Italy is within our grasp.”
The press conference is an opportunity to reach beyond scientific challenges, as the Foundation concentrates on attracting and developing more relationships within the private sector, most notably financial investors. Entering its third year of activities, the Foundation aims to capitalize on its innovative results while avoiding the dispersion of this unique national collaboration. The knowledge gained and the connections made cannot be squandered. The amount of talent, determination, enthusiasm, and pride that has been achieved thus far must continue.
The National Center research group focused on pre-clinical development, Good Manufacturing Practices (GMP), and clinical trials of Gene Therapy Medicinal Products (GTMP) explains that they have begun the first clinical trials in Europe dealing with acute T lymphoblastic leukemia and neoplasms of the central nervous system. The leader of this particular research group, Prof Franco Locatelli, Director of the Department of Hematology and Pediatric Oncology at the Bambino Gesù Children’s Hospital, shares more, “trials have already begun on autoimmune diseases, such as systemic lupus erythematosus or inflammatory muscle diseases. We have also entered into other fascinating areas including the identification of useful targets in the field of acute myeloid leukemia and the development of alternative cellular platforms to the classic patient-specific T lymphocytes. Regarding treatments of hereditary diseases, we have reached advanced stages of development for hemoglobinopathies (thalassemia and sickle cell anemia), rare lysosomal storage diseases that claim an adverse impact on the survival and quality of life of children.” Prof Locatelli continues by confirming that the clinical application of additional project development in Italy is within our grasp.”
The press conference is an opportunity to reach beyond scientific challenges, as the Foundation concentrates on attracting and developing more relationships within the private sector, most notably financial investors. Entering its third year of activities, the Foundation aims to capitalize on its innovative results while avoiding the dispersion of this unique national collaboration. The knowledge gained and the connections made cannot be squandered. The amount of talent, determination, enthusiasm, and pride that has been achieved thus far must continue.
Foundation staff writer Ellen Jane Corcoran
