Home Aplastic anaemia research The Aplastic Anaemia Trust enables research into aplastic anaemia and other rare bone marrow failures. We encourage and support research that is truly transformative and can accelerate access to better treatments and cures. Guided by our Patient Support Group and governed by our peer-led Research and Clinical Advisory Panel our research strategy, launched in 2018, focuses on both types of aplastic anaemia - acquired and inherited, in adult and child populations. While the primary focus is on aplastic anaemia, recognising the links with other rare bone marrow failures, we will consider research into dyskeratosis congenita, fanconi anaemia, PNH and others. Our research priorities To improve the understanding of rare bone marrow failures in child and adult populations To improve the understanding of the diseases by molecular/ genetic research into the damaged cells and their environment To identify risk factors in aplastic anaemia patients that may later lead to MDS (Myelodysplastic Syndrome) or AML (Acute Myeloid Leukaemia) To improve treatments, identifying alternative cures, enabling experimental therapies and ultimately eradicate the disease Apply for funding Current and recent research projects Mutation Timing and Clonal Trajectories in Aplastic Anaemia The Aplastic Anaemia Trust are funding a new research project, investigating genetic mutations in patients with aplastic anaemia or PNH. By developing our understanding of these mutations and their timings in different patients, researchers believe we could develop ways to better predict a patient’s likely response to treatment, and their risk of developing blood cancer. Read more TIARA trial into the potential for T-reg cells to fight aplastic anaemia LifeArc, a UK-based medical research charity, and the Aplastic Anaemia Trust have jointly awarded a £1.15m research grant to King’s College London and King’s College Hospital to investigate the potential of a novel type of “personalised cellular therapy” to reverse aplastic anaemia. The grant is funding a clinical trial to investigate the safety and efficacy of using a patient’s own T-reg cells to restore the blood-making function of the bone marrow. Read more Novel somatic mutations in aplastic anaemia patients The AAT has funded an extended mutational screen in 291 genes currently known to be mutated in MDS, CHIP and aplastic anaemia, in a subset of RACE trial patients in response to treatment. This has extended the current panel of 31 genes (most commonly mutated in these diseases) that has been funded by Cancer Research UK to examine for novel abnormal clones longitudinally. Read more Paediatric Bone Marrow Registry The Aplastic Anaemia Trust has funded a research proposal from Great Ormond Street Hospital team and colleagues from York and Manchester Universities to 'Establish a UK Paediatric bone marrow failure registry and biobank'. Over a period of three years, researchers will aim to obtain laboratory, clinical and quality of life data of all children diagnosed with bone marrow failure within a specified time, to determine how many children are being diagnosed, with what specific bone marrow failure diseases, what treatments they’re receiving and the quality of life following treatment. The biobank will enable future research into paediatric aplastic anaemia. Read more Research study into the Covid-19 vaccination response in patients with aplastic anaemia and/or PNH This research will investigate whether the Covid-19 vaccinations are effective in patients with aplastic anaemia and PNH, and will look at all current Covid-19 vaccines available. It will look at differences that can be measured in the immune system response between patients with these conditions and healthy volunteers. The PNH National Service will also collect clinical diagnostic data and record vaccination outcome. For the first time, we can build a picture of how patients respond to the vaccine and any changes to the course of the patient’s disease that might be attributed to it. Read more Registry for aplastic anaemia and rare bone marrow failure patients treated at King’s. This database was set up in 2012 and finished recruiting in March 2017. It provided an invaluable insight into the incident rates of acquired and inherited Aplastic Anaemia, while also determining the prevalence of MDS/AA overlap for these patients. It’s enabled King’s College experts to plan service requirements for the future. Read more Read all the latest news updates about our research Manage Cookie Preferences