Haematopoietic stem cell function in sickle cell disease
Research type
Research Study
Full title
Study of haematopoietic stem cell health and function in sickle cell disease and other haematological diseases
IRAS ID
314005
Contact name
Paul Telfer
Contact email
Sponsor organisation
Queen Mary University of London
Clinicaltrials.gov Identifier
150586, EDGE
Duration of Study in the UK
2 years, 11 months, 30 days
Research summary
Sickle cell disease (SCD) is an inherited disorder of the oxygen carrying protein
haemoglobin. It is the commonest inherited condition in England, and predominant
affects the black ethnic minority population. SCD patients suffer a variety of
complications, and have reduced life expectancy. Over 1000 people living with sickle
cell disorders in East London and Essex and are managed at Bart’s Health and
network hospitals. There are only a few treatment options at present, but good
prospects for new drugs and curative approaches with gene therapy. Gene therapy
would involve genetic correction of blood stem cells (haematopoietic stem cells, HSC's), but early studies show
that HSC’s are under stress and not easily manipulated. Possible late effects of HCS
damage include emergence of cancerous cells. It is therefore crucial that we obtain a
better understanding of HSC health in SCD.
We plan to undertake a programme of laboratory based studies to elucidate the nature of HSC deficits, and their causes and consequences. We wish to study HSC's from patients representing a range of age, disease severity and treatment history. We will also investigate the potential of different potential therapies to improve HSC function and health in vitro. These studies are anticipated to provide information on potential long-term risk to patients managed with standard care, as well as means of optimising gene therapy protocols and identifying new treatments.
In order to undertake this work, we will be studying cells purified from blood and bone marrow from patients and controls. Bone marrow sampling is a safe procedure, but is uncomfortable. We will be taking samples from consented SCD patients and control subjects (age >5) managed at Barts Health NHS Trust who are undergoing planned surgery under general anaesthetic.REC name
London - Brighton & Sussex Research Ethics Committee
REC reference
22/PR/0991
Date of REC Opinion
19 Oct 2022
REC opinion
Further Information Favourable Opinion