A clinical trial to investigate a novel CAR T-cell therapy, designed to target cancer cells more quickly and cause less side effects, has shown very promising results for children with previously incurable acute lymphoblastic leukaemia (ALL).
Researchers at the UCL Great Ormond Street Institute of Child Health (ICH) and Great Ormond Street Hospital (GOSH) are to lead a new clinical trial for Kawasaki syndrome (KD-CAAP), a rare immune system condition that causes swelling of the blood vessels of the heart and can lead to severe fever and risk of heart problems such as aneurysms.
A two-year old patient at the Royal Manchester Children’s Hospital (RMCH) is the first in the world to receive a pioneering new gene therapy treatment for the rare and life-limiting genetic condition, Mucopolysaccharidosis IIIA (MPSIIIA). The treatment was manufactured at Great Ormond Street Hospital (GOSH) and the UCL Great Ormond Street Institute of Child Health (ICH) after being developed by RMCH and the University of Manchester.
To mark International Clinical Trials Day this May, we took a visit to the Clinical Research Facility (CRF) to meet with GOSH mum Flavia, whose son Matei has been on a clinical trial at the hospital for the last couple of years.
25 young people came to Great Ormond Street Hospital’s (GOSH) new digital hub DRIVE last week as part of an event to help digital researchers and industry engage with a diverse group of young people and hear how their products could be improved to better meet young people's needs.