Approval of Italian stem cell gene therapy a world first

(AGI) Rome, June 27 - Five children born with the "wrong" gene and forced to live in a sterile environment to avoid risk of death from simple infections have been cured at Milan's San Raffaele Institute with a revolutionary gene therapy that relies on stem cells. Named "Strimvelis", it is the first stem cell gene therapy to be approved in the world. It was developed thanks to the joint efforts of three institutions: the Telethon Foundation, the San Raffaele Hospital, and the pharmaceutical industry GSK. The therapy was presented to the Presidency of the Italian Council of Ministers, which was attended by: the Undersecretary Claudio De Vincenti; the head of the San Raffaele Telethon Institute's clinical department, Alessandrio Aiuti; the managing director of Irccs Ospedale San Raffaele, Nicola Bedin; the director of the San Raffaele Telethon Institute, Luigi Naldini; the director general of the Italian Medicines Agency (AIFA), Luca Pani; and sir Andrew Witty, CEO of GSK. The illness cured by Strimvelis, adenosine deaminase severe combined immunodeficiency (ADA-SCID), is extremely rare, with only 15 people a year suffering from it in Europe and 350 in the world. Children born with ADA-SCID don't develop a healthy immune system, making them unable to combat common infections, resulting in a severe and potentially deadly illness. Without rapid treatment, it is often fatal within the first year of life. Experimentation with the gene therapy began 15 years ago at San Raffaele hospital and its success took many years of effort. Strimvelis is administered only once and does not require a donor, meaning there's no risk of immunologic incompatibility, a common side effect of bone marrow transplants. By utilising the patients' own stem cells, doctors and researchers replaced the diseased gene through a weakened viral agent that acted as a Trojan Horse. Strimvelis marks the shift from an experimental therapy to a drug available to all, wherever they are born or live. The authorisation to place Strimvelis on the market is based on data from 18 children cured by the experimental treatment. The survival rate was 100 percent for the 12 children who first started taking part in the clinical study three years ago and for the six others treated in the meantime. The three institutions behind the therapy signed an agreement on Monday with the aim of making their know-how available to develop and market seven other therapies for similarly rare diseases. The Italian government expressed great satisfaction towards the accomplishment. "Strimvelis is an important achievement by Italy's research and industrial activities," underlined Undersecretary De Vincenti. "And it's not an isolated success, because our country is producing important results in the pharmaceutical sector. Our objective is to increasingly develop precision medicine that allows us to tackle rare diseases." (AGI). .