archangel MLD trust
The reason for the ArchAngel Trust: A lovely nine year old named Ava.
In 2013 Ava was diagnosed with Metachromatic Leukodystrophy (MLD), a terminal illness, affecting around 1 in every one hundred thousand people. Her family found themselves in very unfamiliar territory with little help and information due to the rareness of MLD. London’s children’s hospitals and specialists said there was nothing they could do and sent them away with a few website links and little hope.
Through research, her parents discovered the San Raffaele Hospital in Milan, Italy, where a ‘Gene Therapy’ research study was underway. Gene Therapy is a ground-breaking treatment which aims to correct a person’s faulty DNA. In this case, it is applied via a bone marrow transplant using the patient’s own stem cells. This removes all of the risks and 50% mortality rate which are associated with using donor stem cells. Thankfully, the doctors at San Raffaele chose MLD to test the theory.
In January 2014 Ava was accepted as the 12th child in the world to participate in the trial and her transplant was successful. Her DNA has been corrected and her new cells are actively halting progression of the disease. Following the publication of favourable trial results, the therapy has been submitted for a European-wide license. It is hoped that once this therapy is approved, it can be applied to numerous other genetic disorders, changing the lives of many children.
The ArchAngel Trust was established to support medical teams around the world who are working to help people with MLD, including the team at San Raffaele Hospital where Ava received her treatment, and the ‘Foundation Telethon’ who funded the initial research at San Raffaele.
ArchAngel also helps families with MLD affected children in the UK and are spearheading a campaign to get MLD added on to the newborn heel-prick blood test in the UK.
The name ArchAngel is inspired by the eight metre statue of Archangel Raphael which towers over the hospital.