‘There are very few advanced therapy platforms in the world like the one at the SJD Barcelona Children’s Hospital’

A team of medical professionals from the l’Hôpital Necker in Paris, headed by professor and physician Alain Fischer and accompanied by Dr Jean-Roch Fabreguettes, Dr Laure Boquet and Dr Florence Baguet, have been able to experience the Advanced Therapies Platform at the SJD Barcelona Children's Hospital first hand. This facility houses staff who are researching the genetic origins of rare diseases in order to better understand them and develop new treatments that are still out of reach today.

Dr Fischer, a leading expert in the field of Hematology and Pediatric Immunology, was invited to the SJD Barcelona Children's Hospital by Dr Alessandra Magnani, Head of the Advanced Therapies Platform. The visit included a tour of the facilities, which were described as ‘interesting and promising’. They added: ‘The platform at the SJD Barcelona Children's Hospital is a marvelous example of the kind of facilities needed to develop new therapies. There are very few in the world like this one.’

The team of French professionals were also able to learn about the Únicas project, which aims to facilitate the diagnosis of rare diseases. It was the Chief Executive Officer of the SJD Barcelona Children's Hospital, Dr Manel del Castillo, who was able to tell them more about this particular challenge. ‘A pediatric hospital that aims to develop innovative medical treatments for rare diseases must have the necessary infrastructure in place to be able to implement services like gene therapy, for example. Here, you have that infrastructure’, remarks Fischer.

The Advanced Therapies Platform boasts a specialised team who are prepared for the needs of all of the different departments in the hospital. On that note, Dr Fischer and his colleagues were able to visit the Pediatric Cancer Center Barcelona, the Clinical Command Center—a data monitoring centre within the hospital that ensures optimal efficiency in patient management—and they were also able to exchange ideas with research staff in the hospital.

To end their visit, Dr Alain Fischer offered an open-invitation seminar to all SJD Barcelona Children's Hospital staff on gene therapy and his findings after a long career working in the field. In it, he highlighted the importance of innovative new treatments: ‘It has been demonstrated that gene therapy works in treating several rare diseases. There are many diseases out there that we still have not identified and it is our responsibility to develop new therapies that can help remedy this situation’.

‘It is a long process that requires willing patients, prepared staff and money. We are only beginning to get started, but we are on the right path. We must follow your example and, most importantly, work together to create a European cooperation network to share the fruits of our labour’, concludes Dr Fischer.