Our proud history - back in the day

The Aplastic Anaemia Trust emerged as a charity from the Marrow Environment Fund (MEF), and Aplastic Anaemia Patient Support network in the late 90s. The change was designed to highlight the main disease the Charity was formed to help, aplastic anaemia (AA). The MEF was started in 1985 by relatives and friends of patients who had suffered from aplastic anaemia and allied conditions of bone marrow failure, including constitutional or inherited disorders. At that time the outlook for patients was very poor.

Bone marrow transplantation had been introduced to Hammersmith Hospital, where the MEF originally directed its efforts under the dedicated leadership of Professor Ted Gordon-Smith, in 1972, but only a minority of AA patients at that time were suitable. An innovative treatment to suppress the immune system, antilymphocyte globulin (ALG), had not long been available and trials of its efficacy were just starting.

In 1987 Professor Ted moved to St George’s Hospital Medical School to set up a specialist unit devoted to the study and treatment of AA and other causes of marrow failure.

Our unwavering focus on research

Over the decades a key aim of the AAT has been the development of research and treatment in a mutually dependent way. The AAT raised funds for a number of research posts including leading scientists at St George’s to help set up the programme.

With a huge supporter backing and fundraising effort, we provided funds for state-of-the-art equipment for the research team to study the nature and functioning of the damaged AA stem cells and the environment. We funded a special in-patient unit – the Ruth Myles Unit for patients with bone marrow failure, particularly but not exclusively for aplastic anaemia. The team was joined by Professor Judith Marsh who had worked at Hammersmith Hospital in the original clinical team, and acquired further research skills in Manchester and Canada, at the world-renowned stem cell research establishments.

St George’s became the main UK centre for aplastic anaemia with an international reputation thanks to the continued support of the AAT for research, equipment, clinical support staff and finally a complete refurbishment of the day care facilities of the Ruth Myles Unit – to this day operating effectively in the treatment of bone marrow failures. The AAT invested nearly £2m in our work with the St George’s team.

Following the retirement of Professor Ted and Professor Marsh’s move to King’s College Hospital, this became a national centre of excellence for rare bone marrow failure patients.

Our recent research work

In more recent years, the AAT has funded ground-breaking clinical research into acquired AA at King’s College Hospital, enabling to predict responses to treatment, published in Blood 2016.
 
We have provided funds for a clinical nurse specialist at King’s, to co-ordinate vital care and enable patients to participate in clinical trials with nearly 400 patients supported.
 
We funded the establishment of aplastic anaemia and bone marrow failures registry, enabling the study of epidemiology and planning services for the future. Nearly 500 patients have been recruited, with different types of bone marrow failure, of whom 43% have acquired AA and with 110 people diagnosed with either severe or very severe AA. A break-through finding emerged of higher than expected incidence of inherited AA. This study has enabled a joined project with the National Institute of Health (NIH), USA, to produce the largest study of inherited heterozygous RTEL1 mutations in patients with AA and MDS, published in Blood Advances, in 2017. We were the first to show an association between acquired somatic mutations in AA and risk of later developing MDS – reported in the Blood Journal, 2014