T-reg project - Quarter 1, 2020 update What are the T-regs? T regulatory cells (Tregs) are a type of immune-modulatory cells within the immune system, that bring acceptance of self-antigens, and prevent self-directed attacks of the immune system against our own tissues and organs. They are pivotal at preventing auto-immune diseases. Often, a lot of auto-immune conditions have either dysfunctional or deficient Tregs, at the heart of it. Aplastic anaemia is a quintessential auto-immune disorder, where the immune system clears the stem cells (the queen bee cell within the bone marrow making all the different type of blood cells) within the bone marrow. This results in a deficiency of the blood making cells and the consequent downstream effects of low blood counts, in all types of blood cells. Immune suppression through immuno-suppressive treatment (IST) have been the cornerstone of treatments in aplastic anaemia, to stall and prevent the immune attack on the stem cells. Research studies from King's have previously shown that Tregs are not only deficient, but also dysfunctional in aplastic anaemia. This important finding, raises the spectre of harnessing the usefulness of these Tregs at suppressing the immune system and restoring the balance in the blood making factory within the bone marrow. Allogeneic Tregs (Tregs isolated and procured from other healthy donors) have been previously deployed at treating other ‘immune mediated conditions’ such as GvHD (graft versus host disease) and inflammatory bowel disease, as part of a research study, where they have been safely administered and tolerated. Project update, January 2020 On 11 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 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. Here are the headlines from the most recent progress report: The project has made a strong start, with the appointment of a highly experienced research lead, who has been working in one of the best T-reg laboratories in the world under Prof Giovanna Lombardi. The early stage expansion of T-regs in a laboratory has begun. The research lead has been able to get up to a 13-fold increase in T-reg numbers in the laboratory with normal cells under Good Manufacturing Practice (GMP) conditions, which we hope to replicate in patients in the subsequent clinical trial. This positive early progress is welcome news and will be taken further when the new Project Managers starts at the end of April 2020. Please look out for more updates later in the spring/ early summer.