TARGET: A precision medicine approach to target myeloid stem cells
Research type
Research Study
Full title
TARGET: Developing a precision medicine approach to target stem cells in myeloid disorders
IRAS ID
329310
Contact name
Anjum Khan
Contact email
Sponsor organisation
University of York
Duration of Study in the UK
4 years, 11 months, 30 days
Research summary
Acute myeloid leukemia (AML) is a devastating blood cancer with a very poor prognosis, particularly in the older age-groups who are predominantly affected by the disease. Standard treatment with intensive chemotherapy is toxic, risky and poorly tolerated by elderly people and those with comorbidities. More effective and safer treatments are desperately needed.
The majority of poor outcomes are caused by relapse, which is driven by unique populations of leukaemic stem cells. These cells, located in the bone marrow, are resistant to conventional chemotherapy due to poorly understood reasons.
Although scientific knowledge about the genetic changes seen in AML and its precursor MDS (myelodysplastic syndrome) is improving, there is very limited understanding about how changes to the cellular building blocks (proteins) enables leukaemic stem cells to resist chemotherapy so effectively. Combining the study of protein changes in AML (proteomics) with new information on genetics has been shown to be effective in delivering breakthroughs in AML care. Indeed the major recent change in AML treatment for elderly people has been a fruitful less toxic approach based upon targeting an important survival protein, however this approach is not curative for the majority of people.
In this study, funded by the Medical Research Council, we will study the whole range of protein changes seen in leukaemic stem cells in AML & MDS. The work will be carried out by experts in the study of proteins and blood stem cell biology, drawing on unique experimental facilities at the University of York. We will use surplus clinical samples from adult patients with AML/MDS wishing to participate in the study, taken during routine clinical tests so no extra invasive procedures are required. We will use this new knowledge to develop safer targeted treatments. By specifically focusing on stem cells, we aim to prevent relapse and develop truly curative therapies.REC name
South Central - Oxford C Research Ethics Committee
REC reference
24/SC/0271
Date of REC Opinion
19 Aug 2024
REC opinion
Favourable Opinion