Stratification of sporadic Alzheimer’s disease for drug discovery II
Research type
Research Study
Full title
Stratification of sporadic Alzheimer’s disease for an intelligent drug discovery approach using patient derived induced pluripotent stem cells: Improving therapeutic intervention, diagnosis and prognosis of sporadic Alzheimer’s disease using in vitro neuronal models of the disease.
IRAS ID
268793
Contact name
Chris Ward
Contact email
Sponsor organisation
StrataStem Ltd
Duration of Study in the UK
3 years, 0 months, 1 days
Research summary
There is no cure for Alzheimer’s disease (AD). As part of our previous research, we have created induced pluripotent stem cells (iPSCs) from blood samples from those with familial Alzheimer disease (fAD). We have used these to generate neural stem cells. These fAD neural stem cells can be differentiated to functional neurons and are similar to those seen within typical patient’s brains at post-mortem. We have used these iPSC-derived neurons to identify several drug candidates and learn more about the disease. However, fAD represents only a small proportion of AD patients (<5%). So, this research study will examine the make-up of neurons from the more common sporadic version of AD (sAD). We will recruit patients with a clinical diagnosis of late-onset sporadic AD to donate a blood sample to allow us to make ‘neurons in a dish’ to model the disease. This model will aid in the development of biomarkers, therapeutics and diagnostics for the disease. In addition, we will develop a platform for personalised drug testing. This will help predict individual drug response prior to treatment.
Otherwise healthy male and female patients over 65 years of age will be eligible for this study if they have a clinical diagnosis of late-onset/sporadic AD and are willing and able to provide a blood sample of 20ml and complete lifestyle and clinical questionnaires. Patient recruitment and sampling will be undertaken via Greater Manchester Mental Health NHS Foundation Trust Memory Assessment Services (MAS) and clinical trial participants already recruited and phenotyped by the Greater Manchester Dementia Research Centre (GMDRC) who have consented to further research involvement. Blood sampling and questionnaires will be completed at the Memory Assessment Services clinic. Alternatively, there may be opportunities to perform home visits if participants are unable to visit the hospital. Healthy donor samples will be requested from spouses and/or relatives at time of patient contact by MAS and collected during the patient appointment. In the event that insufficient healthy controls are recruited from spouses/relatives we may access healthy volunteers via Join Dementia Research (as part of the NIHR CRN Portfolio). The appointment will last approximately 1 hour. The laboratory-based research for the will take approximately 18 months to complete and a further 18 months to collate and analyse the data. A summary of the data obtained from the study will be published at www.stratastem.com/technology.php.
REC name
North West - Greater Manchester East Research Ethics Committee
REC reference
19/NW/0656
Date of REC Opinion
2 Jan 2020
REC opinion
Further Information Favourable Opinion