Every year several patients and their families from all around the world come to the SR-TIGET (San Raffaele Telethon Institute for Gene Therapy) in Milan for the gene therapy developed by researchers here. The Fondazione Telethon “Just like home” programme was set up for these people, to facilitate their access to therapy for certain rare diseases. Gene therapies include the approved drug for ADA-SCID (Strimvelis), as well as experimental treatments for diseases such as metachromatic leukodystrophy, Wiskott-Aldrich syndrome and beta thalassemia.
The “Just like home” team support the family before, during and after their stay in Milan, helping to make conditions easier for them and so contributing to the success of the treatment and reducing the negative emotional and logistical impacts on the family’s life. The therapy often involves a long journey, sometimes from faraway countries, and a long stay in Milan, several months away from home as well as further periodical check-ups during subsequent years.
The programme provides on-site assistance, welcoming the family and creating a community that can offer emotional and practical support during their stay in Milan and throughout the treatment.
Specifically, staff at Fondazione Telethon provide a range of services to support families who come to Italy:
Families come from all over the world to the SR-TIGET (up until now we have welcomed patients from more than 30 different countries). The distance from home and, in some cases, cultural differences, can make it difficult for them to settle. Staff at Telethon provide practical support before the journey even starts, giving the family detailed information about the therapy, helping the parents to plan their journey and find accommodation. Fondazione Telethon may also cover certain expenses (this is reviewed on a case by case basis).
Many of the patients who have to undergo gene therapy are children. They have to spend about one and a half months in a sterile room, while their parents take it in turns to stay with them. This situation can really put even the closest of families to the test, particularly those with other children and who have come from faraway countries. That is why families are always offered the support of a psychologist during the period of treatment. Caregivers, professional instructors and teachers are also available to help parents care for their children (in and out of the sterile room) during the therapy.
Language support and cultural mediation
Language is the main barrier for families coming to Milan from abroad. Telethon staff work closely with a team of language and cultural mediators able to welcome and guide the patients and their parents during the therapy and within their new community.
A key figure: the care coordinator
For families coming to Milan, the Telethon care coordinator is a point of reference before, during and after the therapy. The care coordinator works closely with a multidisciplinary team that includes psychologists, cultural mediators and, if necessary, professionals able to provide ad hoc services (e.g. teachers, caregivers, professional instructors, etc.). While research nurses provide information on the therapy and its various stages, the care coordinator provides assistance and information on travel, accommodation, services, logistical aspects, etc. The care coordinator also coordinates the network of volunteers who support the families by helping them meet their daily practical needs, sharing their fears but also their joy, making them feel welcome, and never alone.
My job is mainly about listening, and trying to understand the needs of the families, whether or not these are explicitly stated. We work on finding an answer to these needs, trying to engage the people who can best help the families. Often it is me who solves the problems, but the thing that makes me happiest is being able to give families the means to handle difficult situations, and when I see them gain confidence and overcome difficulties with a new strength and in the knowledge that they are never alone.