JPAC Joint United Kingdom (UK) Blood Transfusion and Tissue Transplantation Services Professional Advisory Committee

22.8: Component definitions

22.8.1: Definitions

Unmanipulated: HPC as obtained at collection and not subject to any manipulation.

Manipulated: Subjected to an ex vivo process that selectively removes/enriches, expands or functionally alters HPCs.

  • Minimally manipulated: Processing that does not alter the relevant biological characteristics of cells or tissues.
     
  • More than minimally manipulated: Processing that does alter the relevant biological characteristics of cells or tissues.

Investigational medicinal product (IMP): A pharmaceutical form of an active substance or placebo being tested or used as a reference in a clinical trial, including a product with a marketing authorisation when used or assembled in a way different from the authorised form, or when used for an unauthorised indication, or when used to gain further information about the authorised form. These products require a separate IMP manufacturer’s licence from the Medicines and Healthcare products Regulatory Agency (MHRA).

Advanced therapy medicinal product (ATMP): An ATMP is a medicinal product which is:

  • a gene therapy medicinal product as defined in Part IV of Annex 1 to Directive 2001/83/EC; or
     
  • a somatic cell therapy medicinal product as defined in Part IV of Annex 1 to Directive 2001/83/EC; or
     
  • a tissue engineered product as defined in Article 2 1 (b) of the ATMP Regulation.

The MHRA is the Competent Authority for the assessment of applications for clinical trial authorisations and the associated manufacturer’s licence for investigational ATMPs. It is also the Competent Authority for ATMPs which are prepared and used under the hospital exemption scheme (laid down in Article 3 (7) of the ATMP Regulation) and made and supplied under the ‘specials’ scheme.

22.8.2: Products

HPC, apheresis (HPC-A): HPC collected from the peripheral blood using an apheresis technique usually after receiving a haemopoietic growth factor.

HPC marrow (HPC-M): HPC aspirated from the iliac crests, sternum or other bones.

HPC cord blood (HPC-C): HPC from umbilical cord ± placenta at time of delivery.

Therapeutic cells (TC): Cell products harvested or manufactured for the purpose of providing therapeutic benefit.

  • TC, T cells (TC)
     
  • TC, dendritic cells (DC)
     
  • TC, marrow
     
  • TC, whole blood
     
  • TC, apheresis
     
  • TC, T regulatory cells (T-Reg)
     
  • TC, tumour-derived
     
  • TC, mesenchymal stem cell (MSC)
     
  • TC, natural killer (NK)
     
  • TC, cytotoxic lymphocytes (CTL)
     
  • Other therapeutic cells

22.8.3: Product modifications

B cell depleted: Cells processed by negative selection for B lymphocytes.

Buffy coat enriched: Cells remaining after depletion of mature erythrocytes and plasma by sedimentation or centrifugation using devices, supplies and techniques validated for this purpose.

CD34 selected: Enriched cells processed by positive selection for CD34 antigen bearing cells.

Cryopreserved: Cells frozen using devices, supplies and techniques validated to maintain viability.

Density enriched: Primarily mononuclear cells remaining after depletion of mature erythrocytes, polymorphonuclear cells and plasma by separation of the cell on the basis of density. This is achieved using devices or reagents validated for the separation of cells based on density.

Ex vivo expanded: Cells that have been cultured in vitro for the purpose of producing and/or enriching for a specific functional subset. Note: Ex vivo expanded cells may require an IMP or ATMP manufacturer’s licence from the MHRA.

Gene manipulated: Cells that have been processed to alter their own genes or introduce new genetic material.

Plasma and RBC reduced: Cells remaining after depletion of mature erythrocytes and a portion of plasma by sedimentation and/or centrifugation using devices, supplies and techniques validated for this purpose

Plasma reduced: Cells remaining after a portion of plasma has been depleted by sedimentation or centrifugation using devices, supplies and techniques validated for this purpose.

RBC reduced: Cells remaining after depletion of mature erythrocytes by sedimentation and/or centrifugation using devices, supplies and techniques validated for this purpose.

T cell depleted: Cells processed by negative selection for T lymphocytes.

Tumour cell depleted: Cells processed by negative selection for tumour cells.