First UK recruit for clinical trial treating rare disorder

Evelina London Children's Hospital has recruited the first child in the UK to take part in a clinical research study looking at whether an existing treatment used in adults living with a rare inflammatory condition called neuromyelitis optica spectrum disorder is safe and effective for use in children.

The treatment being studied, Inebilizumab, is a monoclonal antibody (a type of protein) that attaches to immune cells called B-cells and eliminates them.

While this monoclonal antibody treatment is already used in adults, further research is needed before it can be licensed for use in children. 

13-year-old Elisha joined the study, taking place in 1 of 2 dedicated children's clinical research facilities in Evelina London, in October 2023. It is an 'open label' study, which means that all the children taking part receive the treatment. She received her first infusion of Inebilizumab in the autumn and returns to Wolf clinical research facility for regular follow up visits where she is supported by a specialist children's research team.

Before joining the study, Elisha was receiving the standard treatment available for her condition at her local hospital but made the decision with her family to come to Evelina London to join this study.  Elisha and her mum, Deborah, explained what it means to them to be involved.

Deborah said: "We had heard good things about this treatment but it's not currently licensed for use in children. Being involved in this study is really important to us, as not only does it give my daughter access to a treatment that could help her, but that also could help other children in future."

Elisha said:

The research team in Wolf are really lovely and friendly and I feel at ease here. When I come here I forget I'm in a hospital.

Dr Thomas Rossor, paediatric neurology consultant who leads the brain and spine inflammation service at Evelina London, said: "New treatments are often first studied and licensed for use in adults. So, it's vitally important that organisations like ours carry out research studies that could expand the available treatment options for children.  It's always so pleasing to hear what taking part in research studies like this one means to children and their families."

Evelina London has 2 dedicated children's research facilities, a delivery unit of the National Institute of Health Research (NIHR) funded clinical research facility at Guy's and St Thomas' NHS Foundation Trust.