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The expanding role of the clinical haematologist in the new world of advanced therapy medicinal products

Lowdell, MW; Thomas, A; (2017) The expanding role of the clinical haematologist in the new world of advanced therapy medicinal products. British Journal of Haematology , 176 (1) pp. 9-15. 10.1111/bjh.14384. Green open access

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Abstract

Advanced therapy medicinal products (ATMPs) represent the current pinnacle of 'patient-specific medicines' and will change the nature of medicine in the near future. They fall into three categories; somatic cell-therapy products, gene therapy products and cells or tissues for regenerative medicine, which are termed 'tissue engineered' products. The term also incorporates 'combination products' where a human cell or tissue is combined with a medical device. Plainly, many of these new medicines share similarities with conventional haematological stem cell transplant products and donor lymphocyte infusions as well as solid organ grafts and yet ATMPs are regulated as medicines and their development has remained predominantly in academic settings and within specialist centres. However, with the advent of commercialisation of dendritic cell vaccines, chimeric antigen receptor (CAR)-T cells and genetically modified autologous haematopoietic stem cells to cure single gene-defects in β-thalassaemia and haemophilia, the widespread availability of these therapies needs to be accommodated. Uniquely to ATMPs, the patient or an allogeneic donor is regularly part of the manufacturing process. All of the examples given above require procurement of blood, bone marrow or an apheresate from a patient as a starting material for manufacture. This can only occur in a clinical facility licensed for the procurement of human cells for therapeutic use and this is likely to fall to haematology departments, either as stem cell transplant programmes or as blood transfusion departments, to provide under a contract with the company that will manufacture and supply the final medicine. The resource implications associated with this can impact on all haematology departments, not just stem cell transplant units, and should not be under-estimated.

Type: Article
Title: The expanding role of the clinical haematologist in the new world of advanced therapy medicinal products
Location: England
Open access status: An open access version is available from UCL Discovery
DOI: 10.1111/bjh.14384
Publisher version: http://dx.doi.org/10.1111/bjh.14384
Language: English
Additional information: © 2016 John Wiley & Sons Ltd. This is the peer reviewed version of the following article: Lowdell, MW; Thomas, A; (2017) The expanding role of the clinical haematologist in the new world of advanced therapy medicinal products. British Journal of Haematology, 176 (1) pp. 9-15., which has been published in final form at http://dx.doi.org/10.1111/bjh.14384. This article may be used for non-commercial purposes in accordance with Wiley Terms and Conditions for Self-Archiving.
Keywords: Advanced therapy medicinal product, cell therapy, clinical trial, haematopoietic stem cell transplant
UCL classification: UCL
UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > Research Department of Haematology
URI: https://discovery.ucl.ac.uk/id/eprint/1523353
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