Identifying viable regulatory and innovation pathways for regenerative medicine: a case study of cultured red blood cells

Mittra, James; Tait, Joyce; Mastroeni, Michele; Turner, Marc; Mountford, Joanne C.; Bruce, Kevin

doi:10.1016/j.nbt.2014.07.008

Cited by 29 publications

(36 citation statements)

References 37 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…57 Alternative methods for the derivation of hematopoietic populations are now starting to emerge with direct reprogramming as a forerunner in this fast expanding area of research. Although still in its infancy, direct reprogramming explores the instructive conversion of one somatic lineage into another mediated by the ectopic expression of TFs.…”

Section: European Research Contributionsmentioning

confidence: 99%

The European Hematology Association Roadmap for European Hematology Research: a consensus document

et al. 2016

View full text Add to dashboard Cite

T he European Hematology Association (EHA) Roadmap for European Hematology Research highlights major achievements in diagnosis and treatment of blood disorders and identifies the greatest unmet clinical and scientific needs in those areas to enable better funded, more focused European hematology research. Initiated by the EHA, around 300 experts contributed to the consensus document, which will help European policy makers, research funders, research organizations, researchers, and patient groups make better informed decisions on hematology research. It also aims to raise public awareness of the burden of blood disorders on European society, which purely in economic terms is estimated at €23 billion per year, a level of cost that is not matched in current European hematology research funding. In recent decades, hematology research has improved our fundamental understanding of the biology of blood disorders, and has improved diagnostics and treatments, sometimes in revolutionary ways. This progress highlights the potential of focused basic research programs such as this EHA Roadmap. The EHA Roadmap identifies nine 'sections' in hematology: normal hematopoiesis, malignant lymphoid and myeloid diseases, anemias and related diseases, platelet disorders, blood coagulation and hemostatic disorders, transfusion medicine, infections in hematology, and hematopoietic stem cell transplantation. These sections span 60 smaller groups of diseases or disorders. The EHA Roadmap identifies priorities and needs across the field of hematology, including those to develop targeted therapies based on genomic profiling and chemical biology, to eradicate minimal residual malignant disease, and to develop cellular immunotherapies, combination treatments, gene therapies, hematopoietic stem cell treatments, and treatments that are better tolerated by elderly patients.

show abstract

Section: European Research Contributionsmentioning

confidence: 99%

The European Hematology Association Roadmap for European Hematology Research: a consensus document

et al. 2016

View full text Add to dashboard Cite

show abstract

“…Current efforts are focusing on the development of novel 3D-culture supports for the large-scale culture of inducible HSCs (iHSCs) (126). However, none of these iHSCs have so far been approved for medical use, and a new trial for blood cell generation from iPSCs should start in 2017 (127).…”

Section: Induced Pluripotent Stem Cells (Ipscs)mentioning

confidence: 99%

Stem cell manipulation, gene therapy and the risk of cancer stem cell emergence

Clément¹,

Grockowiak²,

Zylbersztejn³

et al. 2017

Stem Cell Investig.

View full text Add to dashboard Cite

Stem cells (SCs) have been extensively studied in the context of regenerative medicine.Human hematopoietic stem cell (HSC)-based therapies have been applied to treat leukemic patients for decades. Handling of mesenchymal stem cells (MSCs) has also raised hopes and concerns in the field of tissue engineering. Lately, discovery of cell reprogramming by Yamanaka's team has profoundly modified research strategies and approaches in this domain. As we gain further insight into cell fate mechanisms and identification of key actors and parameters, this also raises issues as to the manipulation of SCs. These include the engraftment of manipulated cells and the potential predisposition of those cells to develop cancer.As a unique and pioneer model, the use of HSCs to provide new perspectives in the field of regenerative and curative medicine will be reviewed. We will also discuss the potential use of various SCs from embryonic to adult stem cells (ASCs), including induced pluripotent stem cells (iPSCs) as well as MSCs. Furthermore, to sensitize clinicians and researchers to unresolved issues in these new therapeutic approaches, we will highlight the risks associated with the manipulation of human SCs from embryonic or adult origins for each strategy presented.

show abstract

“…in 2010. Gatekeepers also appear willing to explore innovations in regulatory science that would enable good quality in vitro data to play a greater role in early stage proof of concept and safety (Mittra et al, 2015). Other CAT initiatives were to consider whether to: 'Extend incentives for SMEs to academia, hospitals, trusts and small research groups?…”

Section: Gatekeeping the Marketplacementioning

confidence: 99%

Special Treatment? Flexibilities in the Politics of Regenerative Medicine’s Gatekeeping Regimes in the UK

Faulkner

2017

Science as Culture

View full text Add to dashboard Cite

Emerging flexibilities are apparent in gatekeeping regimes applicable to regenerative medicine products, raising issues about the extent to which and forms in which such flexibilities might promote emerging products as a sector warranting special treatment, in the context of recent policy developments in the UK and wider European Union. Concepts of 'gatekeeping', 'gatekeeping regimes' and 'gateways' can point to the ways in which regulatory institutions, health technology assessment organisations, and national planners and purchasers of health services together define and control entry to the medical product marketplace and the adoption of products into the public healthcare system. Flexibilities in existing regimes and new gateways are a way of maintaining 'connection' between gatekeeping regimes and technoscientific innovation in order to steer innovation pathways. The gateways concept has affinity with that of Callon's 'obligatory passage points'. A wide set of recent policy documents show that the measures promoted exhibit a range of alternative gateways that are being constructed around central, legal, restrictive gatekeeping regimes. However, it would be easy to overestimate the significance of these developments as relaxations that would favour innovative producers and their products on a large scale with wide public health impacts. The concepts of gatekeeping regimes and gateways enable understanding of hybrid developments of exceptions and exemptions to dominant regimes which bridge across the arenas of market regulation, health technology assessment and healthcare system planning. These arenas are being drawn closer together as a means of politically managing stakeholders' aims in the UK, EU and other innovating biomedical health systems globally.

show abstract

Identifying viable regulatory and innovation pathways for regenerative medicine: a case study of cultured red blood cells

Cited by 29 publications

References 37 publications

The European Hematology Association Roadmap for European Hematology Research: a consensus document

The European Hematology Association Roadmap for European Hematology Research: a consensus document

Stem cell manipulation, gene therapy and the risk of cancer stem cell emergence

Special Treatment? Flexibilities in the Politics of Regenerative Medicine’s Gatekeeping Regimes in the UK

Contact Info

Product

Resources

About