ArchAngel MLD Trust: Reason for being
The reason for the ArchAngel Trust: A lovely seven year old named Ava.
Ava was recently 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. Miraculously, the doctors at San Raffaele had chosen MLD to test the theory.
Ten children participated in the first phase of the trial, which completed late last year. All are doing well!
A long and tense wait for news on Phase II of the trial followed, whilst Ava’s parents endeavoured to keep her condition stable. In January 2014 Ava was accepted as the 12th child to participate in the trial and her treatment began on 23rd March 2014.
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 has been established to support the team at San Raffaele Hospital in Milan who are working to cure Ava from MLD and to raise funds for ‘Foundation Telethon’ who have funded the research at San Raffaele.
The name ArchAngel is inspired by the eight metre statue of Archangel Raphael which towers over the hospital.
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