Davey S.,NHS Blood and Transplant
International Journal of Immunogenetics | Year: 2014
This review article discusses some of the ethical challenges posed by next generation sequencing (NGS), both in the clinical and research setting. Concerns such as how to deal with unexpected results apply equally to conventional techniques. However, these incidental findings are far more likely with the use of NGS and whole genome sequencing. Whilst the lines of responsibility are better defined in the clinical environment, disclosure of such findings in the research setting is less clear. Recruitment of volunteers for public biobank research, in particular, also raises questions regarding consent and confidentiality. © 2014 John Wiley & Sons Ltd. Source
Estcourt L.J.,NHS Blood and Transplant
Transfusion medicine (Oxford, England) | Year: 2014
Platelet transfusions are used in clinical practice to prevent and treat haemorrhage in thrombocytopenic patients or patients with severe platelet dysfunction. In the UK, and abroad there has been a recent rise in platelet component demand. The three largest patient groups that use platelet components are patients with haematological malignancies (up to 67%), patients receiving cardiac surgery (up to 10%) and patients receiving intensive care (up to 8%). This review has explored some of the factors that may explain this recent trend within these three main groups. These factors include a rise in the general population, an ageing population, an increase in the incidence and prevalence of haematological malignancies, and changes in the management of patients with haematological malignancies. However, the only data available that can be correlated directly with national component data are the size of the total population. There is no evidence to support the premise that use of platelet components in patients receiving cardiac surgery or intensive care treatment is rising over and above the general rise in the population, but the data are sparse. © 2014 British Blood Transfusion Society. Source
McDonald C.P.,NHS Blood and Transplant
Transfusion Medicine and Hemotherapy | Year: 2011
Background: Bacterial contamination remains a significant problem in transfusion medicine. A National Health Service Blood and Transplant (NHSBT) study and surveillance data indicated skin commensals derived from the skin of the donor are the major contaminants of blood components. NHSBT therefore explored two interventions: improved donor arm disinfection and diversion. Methods: Improved donor arm disinfection: Commercial and in-house methods of disinfection were evaluated. Swabs at the venepuncture site were taken before and after disinfection and the reduction in bioburden determined. Diversion: Special collection bags were manufactured to allow the initial volume of blood to flow into a pouch, representing the diversion pouch and then the next flow of blood into another pouch representing the collection bag. Pouches were screened for the presence of bacteria. The reduction in bacterial contamination was then determined. Results: A two-step commercial procedure (Donor Prep Kit; DPK) consisting of 70% isopropyl alcohol followed by tincture of iodine was shown to be a best practice procedure (2-min procedure). A 99.79% reduction was obtained, and this method was 10 times more effective than current practice at that time. The DPK was shown in a field trial to increase donor waiting time. A second study was initiated to find a more rapid procedure. ChloraPrep®, consisting of 2% chlorhexidine gluconate and 70% isopropyl alcohol, was shown to have equivalent disinfection efficiency as the DPK, but only took 1 min to perform. In 2006, ChloraPrep was introduced as the national method of donor arm disinfection. Diversion was shown to give a 47% reduction in contamination and was introduced nationally in 2002. Conclusion: Improved donor arm disinfection and diversion are effective, low-cost interventions, but do not eliminate all bacterial transmissions. In 2011, bacterial screening of platelet components was introduced by NHSBT to further increase the safety of the blood supply. © 2011 S. Karger AG, Basel. Source
Agency: Cordis | Branch: H2020 | Program: RIA | Phase: PHC-13-2014 | Award Amount: 5.99M | Year: 2015
Type 2 diabetes will affect >500 million adults by 2040 and its secondary complications will generate enormous socioeconomic costs - in particular, diabetic kidney disease (DKD), which is already the most common cause of chronic kidney disease. DKD is associated with greatly increased mortality and frequently progresses to end stage renal failure. Pharmacotherapy, dialysis and transplantation represent the mainstay treatments for DKD but are costly and provide only limited protection against adverse outcomes. Mesenchymal Stromal Cell (MSC) therapy is a promising approach to halting the progression of DKD toward end-stage renal failure and may also have ancillary benefits in Type 2 diabetes. In preliminary research, we have demonstrated that a single dose of MSC simultaneously improves kidney function (glomerular filtration rate and albuminuria) as well as hyperglycaemia in animals with DKD. NEPHSTROM will conduct a multi-centre, placebo-controlled clinical trial of a novel MSC therapy for stabilization of progressive DKD, leading to superior clinical outcomes and long-term socioeconomic benefit. A key enabler for this trial is a novel MSC population (CD362\MSC, trade name Cyndacel-M) which delivers higher purity and improved characterisation compared to conventional plastic-adherent MSC. The NEPHSTROM Phase 1b/2a clinical trial will investigate the safety, tolerability and preliminary efficacy of a single intravenous infusion of allogeneic Cyndacel-M versus placebo in adults with progressive DKD. NEPHSTROM investigators will also determine the bio-distribution, mechanisms of action, immunological effects and economic impacts associated with Cyndacel-M therapy for DKD. This research will critically inform the optimal design of subsequent Phase 3 trials of Cyndacel-M. Stabilising progressive DKD through NEPHSTROMs next-generation MSC therapy will reduce the high all-cause mortality and end-stage renal failure risk in people with this chronic non-communicable disease.
Agency: Cordis | Branch: H2020 | Program: RIA | Phase: PHC-15-2015 | Award Amount: 6.83M | Year: 2016
CLINICAL PROBLEM AND UNMET NEED There are 11,827 patients with severe structural airway disease in Europe. Even with the current standard of care, when hospitalised this group of patients has a 22% risk of dying. Patients are currently subjected to repeated surgical interventions (stent insertion) which have a high failure rate. Other therapeutic strategies under development include synthetic tracheal scaffolds seeded with patients own stem cells. Preliminary data show that these scaffolds are poorly integrated and are susceptible to infection. TETRA PROJECT Our SME-led project will address the limitations of standard clinical care and competitor products under development and will: - Build on our successful compassionate use experience using autologous stem cell seeded scaffold-tracheal transplants in 48 patients - Follow on from our Phase I 4 patient INSPIRE clinical trial which will improve on the clinical prototype used in compassionate use cases - Conduct a 48 patient Phase II pivotal clinical trial to provide robust, quality data with validated GMP manufacturing processes to support an accelerated route to market for commercial exploitation in this orphan indication - Prepare a dossier for MAA submission BENEFITS Our product, an ATMP, aims to eliminate the need for repeated surgical interventions of high risk and limited efficacy, reduce deaths and improve the quality of life for surviving patients. If treating 20% of the patients with severe structural airway disease, we estimate that in Europe our technology will improve the quality and length of patient lives and result in savings of 517 million per year. We plan to further develop our platform technology to generate other complex tissues/organs such as bowel and liver replacements for clinical applications which will impact the lives of tens of thousands of patient in the EU with bowel and liver diseases.