StemCell Therapy

Grant will support researchers from Kings College London and Kings 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 Kings College London and Kings 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 patients 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 patients own blood were collected, selected for activity and multiplied. In a test tube, these cells prevented the immune system from attacking the patients bone marrow stem cells.[i]

Professor Ghulam Mufti, Department of Haematological Medicine at Kings College London and Kings College Hospital, and lead study investigator said: For patients with this ultra-rare disease, were 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. Weve 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 cellsred blood cells, white blood cells and platelets. Only around 100150 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, LifeArcs 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 Kings College London, Kings 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 Kings College London and Kings College Hospital will run for a duration of three years and aims to recruit nine patients. A blood sample of the patients 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 persons quality of life. Through AATs close work with Kings College London and Kings 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 Kings College London and Kings College Hospital to progress this ground-breaking work, which could potentially enable people living with severe AA to once again lead a normal life.

[i] Kordasti S, Costantini B, Seidl T, Perez-Abellan P, Llordella MM, McLornan D, Diggins KE, Kulasekararaj A, Benfatto C, Feng X, Smith A, Mian SA, Melchiotti R, de Rinadis E, Heck S, Ellis R, Petrov N, Povoleri GAM, Chung SS, Thomas NSB, FarzanehF, Irish JM, Young NS, Marsh JCW, Mufti GJ. Deep-phenotyping of Tregs identifies an immune signature for idiopathic aplastic anemia and predicts response to treatment. Blood. 2016 Sep 1;128(9):1193-20

About Aplastic Anaemia:

Aplastic Anaemia (AA) is a rare andlife-threatening illnesscaused by the bone marrow failing to make enough of all three types of blood cells - red blood cells, white blood cells and platelets.It is estimated that between 100 and 150 people will be diagnosed in UK alone every year. That is around 2 people for every 1,000,000 of population, making AA a very rare disease. In most cases of AA, the immune system attacks the bone marrow, thinking it is faulty. This makesthebone marrow function slow down, leading to the under-production ofall types ofblood cells.

The most common symptoms of AA are anaemia - caused by a lack of red blood cells - with the associated feeling of fatigue, shortness of breath, headaches and, occasionally, chest pains, and increased risk of infections caused by low white blood cells. Low platelets cause people to bleed easily. Being more susceptible to severe and life-threatening infection and bleeding complications makes AA a highly dangerous disease. Patients will visit hospital regularly to receive blood transfusions and treatments for infection; many will be admitted to hospital for weeks at a time while they receive treatment, and those who recover must take a lot of time regaining their strength, avoiding places where they could easily pick up infections.

There are currently two standard first-line treatments for AA:immune-suppressive therapy (IST), which uses medicines (antithymocyteglobulin (ATG) and ciclosporin) to dampen down abnormal immune responses, or bone marrowstem cell transplantation, the only known curative AA treatment to date, but suitable onlyfor a proportion of patients. A bone marrow transplant is an intense procedure that requires an immunological matched donor and may require lifelong immunosuppression therapy, which can lead to further debilitating side effects. Both bone marrow transplants and the combination of ATG and ciclosporin work in only around 2 in 3 of AA patients.

About the Aplastic Anaemia Trust

The Aplastic Anaemia Trust is the only charity in the UK dedicated to research into aplastic anaemia and alliedrare bone marrow failures and supportingeveryone affected nationally. We have builtproductive working partnershipswith major research and treatment centres of excellence in England, providing us with direct access to world-class experts, state-of-the-art labs and excellent patient care.Access to this medical and scientific expertise puts us in a strong position to identify areas of need, raisefunds for research, and engage with the expertcommunity in the UK and internationally. Our vision isa world free from aplastic anaemia and alliedrare bone marrow failures. Our mission is to enable vital research into the causes of aplastic anaemia and other rare bone marrow failures that ultimately leads to finding a cure, and to support everyone affected by them, so they can lead healthy and fulfilling lives.

Our strategic objectives are

Find out more about our work on http://www.theaat.org.uk

About LifeArc

LifeArc is a self-funded medical research charity. Our mission is to advance translation of early science into health care treatments or diagnostics that can be taken through to full development and made available to patients. We have been doing this for more than 25 years and our work has resulted in a diagnostic for antibiotic resistance and four licensed medicines.

Our success allows us to explore new approaches to stimulate and fund translation. We have our own drug discovery and diagnostics development facilities, supported by experts in technology transfer and intellectual property who also provide services to other organisations. Our model is built on collaboration, and we partner with a broad range of groups including medical research charities, research organisations, industry and academic scientists. We are motivated by patient need and scientific opportunity.

Two funds help us to invest in external projects for the benefit of patients: our Philanthropic Fund provides grants to support medical research projects focused on the translation of rare diseases research and our Seed Fund is aimed at start-up companies focussed on developing new therapeutics and biological modalities.

Find out more about our work on http://www.lifearc.org or follow us on LinkedIn or Twitter.

Kings College London

King's College Londonis one of the top 10 UK universities in the world (QS World University Rankings, 2018/19) and among the oldest in England. Kings has more than 31,000 students (including more than 12,800 postgraduates) from some 150 countries worldwide, and some 8,500 staff.

King's has an outstanding reputation for world-class teaching and cutting-edge research. In the 2014 Research Excellence Framework (REF), eighty-four per cent of research at Kings was deemed world-leading or internationally excellent (3* and 4*).

Since our foundation, Kings students and staff have dedicated themselves in the service of society. Kings will continue to focus on world-leading education, research and service, and will have an increasingly proactive role to play in a more interconnected, complex world.Visit our websiteto find out more about Vision 2029, Kings strategic vision for the next 12 years to 2029, which will be the 200th anniversary of the founding of the university. World-changing ideas. Life-changing impact:https://www.kcl.ac.uk/news/headlines.aspx

About Kings College HospitalKings College Hospital is a leading national and international centre for the diagnosis and treatment of blood cancers.

The hospital is home to the largest bone marrow transplant programme in the UK and performs more than 160 transplants a year. Kings College Hospital is also an international centre for research into and the treatment of myeloid leukaemias, lymphomas and myeloma, and have the first immune gene therapy programme for leukaemia approved by the Gene Therapy Advisory Committee (GTAC). It is the first hospital in the UK to treat adult lymphoma patients with CAR T therapy, and it has an active CAR programme in lymphoma, leukaemia and myeloma. It is also a centre of excellence for myelodysplasia and expertise in matched and unrelated transplants for the treatment of myelodysplastic syndrome (MDS).

The haemato-oncology service:

For more information about the haematology service at Kings College Hospital visit https://www.kch.nhs.uk/service/a-z/haemato-oncology

Read more from the original source:
Patients with ultra-rare bone marrow disease set to benefit from 1.15m grant from LifeArc and The Aplastic Anaemia Trust - PharmiWeb.com

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