Support pioneering gene therapy research

Aksel is three years old and last year, after a series of persistent colds and infections, he was diagnosed with a range of viruses that went on to trigger Hemophagocytic lymphohistiocytosis (HLH).

HLH is a devastating rare genetic condition of the immune system affecting approximately 1.2 children per million globally each year. In children with HLH, infected or damaged cells are not removed from the body as they should, causing the immune system to become overstimulated. This damages the patient’s healthy tissues and organs such as the bone marrow, the liver and the brain. Sadly many children with HLH die suddenly before their second birthday because the condition goes undiagnosed.

Aksel’s story

Just before Christmas 2014, Aksel’s health deteriorated very quickly and he was rushed to hospital for an emergency operation. His parents were told that he may not survive. For 11 days through the Christmas holidays, with his parents by his side, Aksel fought for his life in the Pediatric Intensive Care Unit at Miami Children’s Hospital. Thankfully, he survived.

Aksel is currently stable, but he is a long way from being able to enjoy the childhood of a healthy three year- old. He is not allowed to go to school or play with his friends, as any small virus could reactivate his HLH and could be fatal. Current treatment for HLH is limited to a bone marrow transplant but this is only possible if a matched donor is found and many children like Aksel struggle to find a suitable match. Better, safer ways of treatment are urgently needed.

Hope for children like Aksel

Through work carried out at the Imagine Institute at Necker-Enfants Malades children’s hospital in Paris and at Great Ormond Street Hospital in London, researchers have discovered the gene defect that can cause a type of HLH. They hope to use gene therapy to introduce a working copy of the gene into the patient’s own cells and are almost ready to trial this treatment in patients. This exciting work could result in a much safer treatment option for these children and bring hope to thousands of families across the world. tr2

The Imagine Institute and Necker-Enfants Malades children’s hospital are seeking funds to bring these therapies to a clinical trial across both Necker Hospital and Great Ormond Street Hospital.


This is a really exciting project and I am delighted to be working in Partnership with my colleague in Paris, Professor Marina Cavazzana. Together, we have worked on gene therapy projects for the last 15 years and have run joint trials of gene therapy for three different conditions affecting the immune system. Patients from Paris have come to London for gene therapy treatment and patients from London have gone to Paris.” Professor   Bobby     Gaspar,   Professor of Paediatrics and Immunology at the UCL Institute of Child Health and Consultant at Great Ormond Street Hospital

This clinical trial is vital to patients like Aksel and we urgently need to raise €1 million (US$1,096,290) to make it happen. Your donation could make all the difference to children with HLH across the world.

Please donate now:

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