ArchAngel MLD Trust

ArchAngel MLD Trust
ArchAngel MLD Trust supports medical teams around the world who are working to help people with the rare genetic illness MLD. ArchAngel also awards private grants to UK families with MLD affected children and is leading the work required to have MLD added to the UK newborn bloodspot test, to ensure that future MLD affected babies have the chance of receiving life-saving treatment.

Metachromatic Leukodystrophy is a rare genetically inherited illness which affects around 1 in every 100,000 people. An inherent error in the patients metabolic system causes progressive damage to the nervous system, resulting in life-long disability or death. It is classified as both a ‘demyelinating illness’ (leukodystrophy) and a ‘lysosomal storage disorder’ (LSD).

ArchAngel MLD Trust was inspired by a little girl named Ava, who was diagnosed with MLD at the age of 6 in 2013. Ava’s 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 told them to go home and make some memories with Ava while they still had the chance.

Through research however, her parents discovered the San Raffaele Telethon Institute 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 high mortality rate which are associated with using donor stem cells. Fortunately, the doctors at San Raffaele chose MLD to test the theory, giving Ava’s parents new cause for hope.

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 changing the predicted course of the disease. Following highly favourable trial results, the therapy 'Libmeldy' received EMA approval in 2020 and approval for use by NHS England in February 2022, other Eurpoean countries ahve subsequnetly approved Libmeldy and many children have already been successfuly treated.

Every child counts, no matter how rare their condition. Thank you for your support.
©2024 Copyright ArchAngel MLD Trust
Website by Modern Websites