The centre – located in Ranica, in the province of Bergamo – was founded in 1992 to promote independent clinical research on rare diseases. The very first commitment was to establish an information service to the patients affected by rare diseases. In the early ’90 rare diseases were in general poorly studied entities and knowledge on epidemiology, pathophysiology and treatment was scarce for a great number of them. Patients diagnosed with a rare disease – it was the reasoning that suggested the importance of an information service - need, primarily, to learn as much as they can about their condition, where they can find doctors expert in diagnosis and treatment, how to cope with the disease in daily life.
Over the years, the Centre has expanded its actions and initiatives to help in shaping health policies in Italy and Europe in favour of patients with rare diseases, in the form of collaborations with ministries, regional councils, local health agencies and health professionals. Since December 2001, the Centre has been established as Coordinating Centre for the Network of Rare Diseases in Lombardy (Regional Government Decree N° VII/7328,11/12/2001).
While developing action in the area of public health, the Centre has increasingly focused the effort in becoming a health facility dedicated to designing and conducting clinical trials in the field of rare diseases and kidney diseases.
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Today, the centre’s commitment to serving patients is expressed through multidisciplinary projects ranging from basic research to clinical and epidemiological research, along with outreach and educational activities.
The centre is a unique example of a facility that can accommodate and develop every phase of a research project in one place. Physicians, nurses, biologists, pharmacologists, bioengineers, laboratory technicians, and experts in statistics, information technology and health economics all work together closely to assess patients’ health and collect and process data in order to determine the effectiveness of new diagnostic and therapeutic strategies.
Over the years, pharmacological clinical trials have been designed and conducted to study new treatments for rare kidney diseases. These studies are important for finding treatments for not only rare diseases but also more common ones. The centre carries outstudy that focus on diabetic nephropathy, diabetic and non-diabetic proteinuric chronic kidney diseases, thrombotic microangiopathies, diseases caused by alteration of the complement system, non-proteinuric kidney diseases such as polycystic kidney disease. A number of studies also aim to investigate strategies to minimise the need for immunosuppressive therapy in kidney transplantation and induce immunological tolerance to transplanted organs using mesenchymal stromal cell therapy.
In March 2009, the Daccò Centre was accredited by the Lombardy Region and has since been performing diagnostics within the Italian National Health Service (Servizio Sanitario Nazionale - SSN) for a number of rare diseases.
The centre’s research receives crucial support from the Fondazione ARMR (Aiuti alla Ricerca sulle Malattie Rare), which has been at the Mario Negri's side since 1993. The funds raised each year are used to establish annual fellowships for both Italian and international researchers.
Il Centro di Ricerche Cliniche per le Malattie Rare “Aldo e Cele Daccò” di Ranica, in provincia di Bergamo, è nato nel 1992 con l’intento di creare una struttura esclusivamente dedicata allo studio delle malattie rare, ambito in cui il Centro ha svolto un ruolo pionieristico, quando in Italia non esisteva pressoché nulla.
All'inizio della propria attività il Centro offriva soprattutto informazioni per i pazienti affetti da malattie rare che soffrivano della scarsità di notizie e di cultura medica. Sapere di più della propria condizione è, infatti, l'esigenza più urgente che ha chi soffre di una malattia rara.
Nel corso degli anni, accanto a questa attività di servizio per i pazienti, il Centro Daccò grazie all'accrescersi della banca dati contenente notizie relative a numerose malattie rare, è andato configurandosi come un importante centro di raccolta di dati utili nella programmazione degli studi clinici.
Parallelamente è aumentata l'importanza dell'attività istituzionale del Centro, che si è concretizzata nelle collaborazioni con ministero, assessorati regionali, aziende sanitarie locali e singoli medici, contribuendo a dare forma alla politica sanitaria sulle malattie rare in Italia. Nel tempo il Centro ha mantenuto rapporti di collaborazione con ricercatori e istituzioni a livello internazionale, specie con la Commissione Europea.
Dal Dicembre 2001 il Centro di Ranica è stato riconosciuto come Centro di Coordinamento della Rete Regionale Malattie Rare (DGR N° VII/7328 del 11/12/2001).
Un fondamentale sostegno alle attività di ricerca del Centro deriva dal supporto della Fondazione ARMR (Aiuti alla Ricerca sulle Malattie Rare), che dal 1993 è a fianco del Mario Negri. I fondi raccolti ogni anno sono destinati a istituire borse di studio annuali da assegnare a Ricercatori, sia italiani che stranieri.
Non solo malattie rare
L'attività di ricerca del Centro riguarda non solo le malattie rare, ma anche le patologie renali croniche le complicanze del diabete e il trapianto d'organo. Per questo sono state rafforzate competenze diagnostiche di livello avanzato in alcune aree come la caratterizzazione di microangiopatie trombotiche, di malattie renali rare con particolare riferimento a malattie cistiche ereditarie, malattie glomerulari e forme sindromiche con coinvolgimento del tratto urinario, giungendo alla identificazione di nuovi geni malattia, al chiarimento di meccanismi patogenetici e allo studio di potenziali trattamenti.
Over the years, the growth of our database containing information about numerous rare diseases has become a real asset that has also been enriched by the availability of biological specimens/samples. This important and rich database has made it possible to develop several Disease Registries that can help to answer complex questions. The following registries have been developed:
- MPGN/C3G - Membranoproliferative Glomerulonephritis/C3 glomerulopathies
- HUS/TTP - Haemolytic-Uremic Syndrome/Thrombotic Thrombocytopenic Purpura
- SRNS - Steroid-Resistant Nephrotic Syndrome
Today, the Ranica centre has several biobanks, which are integrated into a Biological Resource Centre (CRB)and support research with the goal of developing new therapeutic approaches.
Since being appointed as the Coordinating Centre for the Regional Rare Diseases Network in Lombardy in 2001, the centre has added new and important tasks and responsibilities to its mandate, such as:
Intercheck web: web application aimed at health professionals to support the proper prescription of drugs in the elderly.
Information service for pregnant and nursing mothers on the appropriate use of medicines.
Information service for health professionals and patients on treatment plans and rare diseases.
Information service for health professionals and patients on kidney diseases and transplants.
Information services aimed at health professionals and the general public on the correct use of drugs, side effects and interactions in the elderly.
