MILAN--(BUSINESS WIRE)--Pancreatic islets, containing insulin-producing cells, are isolated from a donor's pancreas and implanted in the liver of the recipient through a simple infusion procedure. Once in place, the transplanted islets begin to produce insulin.
This is the mechanism of action of pancreatic islet transplantation, now a reality for those suffering from type 1 diabetes and in particular from the so-called "brittle diabetes", or unstable diabetes, an uncontrolled disease despite medications. To ensure the results of this innovative approach, however, it is necessary to protect the pancreatic islets that are attacked by the immune system of the recipient.
This is now possible thanks to Reparixin, a drug developed by the investigators of the biopharmaceutical company Dompé; the compound is currently undergoing Phase III clinical trials, the last step before its commercialization. The clinical trial involves 5 countries and 8 centers in Europe and the U.S. and is expected to enroll approximately 50 patients, or about half of those who receive yearly, in the above mentioned regions, this innovative therapeutic procedure.
Islet cell transplantation is one of the topics addressed by experts gathered in Milan for the twelfth edition of the International Conference of the CTS, the World Congress on Cell Therapy. "The islet transplantation has proven to be effective, but we still need to improve the clinical outcomes of treatment - explains Lorenzo Piemonti, San Raffaele Diabetes Research Institute Deputy Director and Director islet transplantation program - Some factors, like the isolation of the islets themselves, can in fact gradually reduce the functionality of the transplanted islets. The research currently focuses on the inflammatory response that develops in the patient immediately after the infusion of islet cells; such response has a dramatic influence on the survival of the islets themselves, reducing their function by 50 percent during the first seven days. Reparixin, a potent and selective inhibitor of interleukin-8 (a chemokine), was identified in the Italian laboratories of Dompé and is now being investigated to assess to what extent it is able to specifically inhibit the inflammatory response, thus preserving the islet function and, therefore, improving the efficacy of this procedure".
Innovation in research, then, starts right from Italy. And, more specifically, from the Polo Dompé of L'Aquila, where the compound is manufactured. A Phase III randomized, multicenter, double-blind trial aims at assessing the drug's efficacy in improving the efficiency of islet transplantation, protecting the function and survival of transplanted cells, and increasing the proportion of patients who reach insulin independence thanks to therapy.
"In the fight against type 1 diabetes, which affects 20 million people worldwide, research plays a fundamental role and islet cell transplantation is a prospective, effective treatment, especially if, through the use of reparixin, we can further improve the results of this approach - says Camillo Ricordi, Director Diabetes Research Institute, Miami. The goal must be clear: to achieve a definitive cure that can free patients from the need for insulin treatment, which today, however, is essential to ensure a good metabolic control."
Italy is a world leader in this research area. According to the latest data available, in our country the islet transplantation procedure is performed at San Raffaele Hospital and Niguarda in Milan and at ISMETT of Palermo; 18-22 allografts (cells harvested from a donor) and 10-14 autologous grafts (from the same subject) are performed yearly. As to Europe, in the United Kingdom - where NICE (National Institute for Health and Care Excellence) has already given green light to this therapeutic approach - 15 to 25 allogeneic transplants, and about 10 autologous transplantation procedures are performed every year. In France, an average of 20 allogeneic transplants is performed every year whereas autologous transplants are performed only occasionally (1-2 a year); the same pattern is observed in Germany where, however, they perform about 10 allogeneic transplants a year. The goal of investigators, especially if the current Reparixin trial confirms the possibility of freeing patients from insulin treatment, is to make this innovation available to more patients. And even health economics experts agree with this approach, in view of future developments.
"The need to cut costs on a short-term horizon must not conflict with the development of a country and should not prevent a health care system from promoting and supporting research and innovation – states Mario Del Vecchio, Director OCPS (Observatory on Private Consumption in the Health Care System), SDA Bocconi, Milan. We need to understand how to safeguard the future while respecting the constraints of the present, through a more careful analysis and with the help of explicit mechanisms along the path from research (companies and healthcare organizations of excellence) to the introduction of innovation, to its consolidation in the clinical practice. "
Innovative research is therefore a key driver for the future development of the country, both from a scientific and an economic and social development perspective. And it is in this context that Dompé launches its challenge to health. The Group, in fact, is implementing its "strategic revolution" focused on their distinctive capabilities.
"As demonstrated by Reparixin, the focus of our commitment to research is represented by rare, often orphan diseases, to which we devote a large share of our skills and financial resources - said Eugenio Aringhieri, CEO Dompé Group. The area of Transplantation along with Diabetes and Ophthalmology, represent the competitive arena in which we have accepted the challenge, aware of the health expectations of patients and of the difficult development path of our projects. But we are also very confident that the route taken to date and the results obtained together with a national and international scientific community of the highest quality, as shown by Reparixin, provide a strong foundation that supports and encourages us to continue with ever greater determination along this direction."