Patients with ultra-rare bone marrow disease set to benefit from £1.15m grant from LifeArc and The Aplastic Anaemia Trust

Grant will support researchers from King’s College London and King’s College Hospital to test a personalised treatment approach for Aplastic Anaemia patients who have not responded to available therapies

21 October 2019 - LifeArc, a UK-based medical research charity, and the Aplastic Anaemia Trust (AAT) 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 the ultra-rare condition aplastic anaemia (AA). The results of this research could give new hope to people living with a severe, life-limiting form of this condition.

The grant will fund 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. This follows laboratory-based research from the team of scientists where T-reg cells from a patient’s own blood were collected, selected for activity and multiplied. In a test tube, these cells prevented the immune system from attacking the patient’s bone marrow stem cells.[i]

Professor Ghulam Mufti, Department of Haematological Medicine at King’s College London and King’s College Hospital, and lead study investigator said: “For patients with this ultra-rare disease, we’re looking for the first time at a personalised medicine approach where their own immune cells could be used to alter their disease. In AA there is a reduction in the number of T-regs and most of the ones that the AA patients do have are non-functional. We’ve seen success in the laboratory by selecting and bolstering the number of functional T-reg cells. Now, with funding from LifeArc and the AAT, we can investigate the potential of this approach in treating AA patients who currently have very limited treatment options.”
AA is an ultra-rare life-threatening illness caused by the bone marrow failing to make enough of all three types of blood cells—red blood cells, white blood cells and platelets. Only around 100–150 people in UK are diagnosed per year, affecting all ages but most commonly people between the ages of 10 to 20 years old and those over the age of 60 years.

People with the illness are at greater risk of infections, bleeding, and can experience extreme fatigue, which leaves them unable to carry out simple daily tasks that most people take for granted. Around one in three patients with severe AA fail to respond to existing drug treatments and the other option — a bone marrow transplant – is reliant on finding a suitable donor, requires life-long treatment with immunosuppression therapy and is unsuccessful in one in three people.
Dr Catriona Crombie, LifeArc’s Head of Philanthropic Fund explained why the charity had approved the funding: “LifeArc set up the Philanthropic fund to support translational research into rare diseases, where there is less interest from commercial organisations. Patients with AA can have limited treatment options; this opportunity with King’s College London, King’s College Hospital and the AAT has the potential to transform the lives of patients living with a severe form of the disease.”
The trial at King’s College London and King’s College Hospital will run for a duration of three years and aims to recruit nine patients. A blood sample of the patient’s T-reg cells will be extracted, purified and grown in the lab before being given back to the patient in a higher concentration. As patients with AA are more susceptible to infection, this personalised treatment approach is more likely to avoid the risk of severe infection and inflammation.

Grazina Berry, CEO of the AAT said: “AA can severely impact a person’s quality of life. Through AAT’s close work with King’s College London and King’s College Hospital as a specialist centre of clinical care and research in AA, we identified the project with the most potential to directly benefit patients who are currently at a loss for solutions. We are delighted to have partnered with LifeArc and King’s College London and King’s College Hospital to progress this ground-breaking work, which could potentially enable people living with severe AA to once again lead a normal life.”