Cell/Gene Autologous Therapy

Autologous Stem Cell Therapy

Defined as the implantation, transplantation, infusion or transfer of human cells or tissue back into the individual from whom the cells or tissue were recovered.

Autologous Cell Therapy involves extracting one’s cells, manipulating them and returning them to one’s body. It has several advantages compared to allogeneic cell therapy, including immediate donor availability and no HLA matching required, which significantly reduces the risks to the patient.

Manipulating the extracted cells involves processes before it is being injected again to one’s body. Since you cannot extract a large volume of blood- forming cells, the extracted cells will undergo a cell production under certain laboratory conditions to reach the required volume specification. The performing laboratory is required to comply sterile standards of the procedure room, the mandated equipment like the tide motion bioreactors and cell therapy aseptic isolator to be used during cell production, and the required PPE of the trained personnel during preparation.

Advantages of Autologous Products
  • The donor is immediately available.
  • No Human Leukocyte Antigen (HLA) matching is required
    • Reduced rejection
    • Reduced graft-versus-host-disease but no graft-versus-disease effect (alloreactivity)
  • No immunosuppression required
Types of Autologous Products
• Hematopoietic progenitor cells

— Blood cells are produced by the proliferation and differentiation of a very small population of pluripotent hematopoietic stem cells (HSCs) that also have the ability to replenish themselves by self-renewal. During differentiation, the progeny of HSCs progress through various intermediate maturational stages, generating multi-potential and lineage-committed progenitor cells prior to reaching maturity. Bone marrow (BM) is the major site of hematopoiesis in humans and, under normal conditions, only small numbers of hematopoietic stem and progenitor cells (HSPCs) can be found in the peripheral blood (PB). Treatment with cytokines (in particular granulocyte colony-stimulating factor; G-CSF), some myelosuppressive drugs used in cancer treatment, and compounds that disrupt the interaction between hematopoietic and BM stromal cells can rapidly mobilize large numbers of stem and progenitors into the circulation.

• Non hematopoietic progenitor cells

— Also called as the bone marrow stromal stem cells (also called mesenchymal stem cells, or skeletal stem cells by some). These non-hematopoietic stem cells make up a small proportion of the stromal cell population in the bone marrow and can generate bone, cartilage, and fat cells that support the formation of blood and fibrous connective tissue.

• Cytotoxic Cells

Cytotoxic T lymphocytes represent a crucial component of the adaptive immune system with particular importance in the control of intracellular pathogens. Effector CTL have the capacity to promote the apoptotic death of carefully chosen target cells, using a combination of granule (perforin/granzyme)- and receptor (Fas/tumour necrosis factor)-mediated mechanisms. While natural killer cells also promote cell death, CTL are distinguished by their exquisite specificity for antigen, which they recognise using a clonally unique T-cell receptor (TCR). Target cells are ‘flagged’ for the attention of CTL when they present antigen-derived peptide fragments on the cell surface, inserted into the groove of class I major histocompatibility (MHC) molecules.

Regenerative Cells
• Antigen Presenting Cells

— These are heterogeneous group of immune cells that mediate the cellular response by processing and presenting antigens for recognition by certain lymphocytes such as T cells. Classical APCs include dendritic cells, macrophages, Langerhans cells and B cells.

• Tumor Vaccines

A therapeutic agent produced by isolating tumor cells from an individual and processing these tumor cells into a vaccine formulation in vitro; the vaccine is then administered to the individual from whom the tumor cells were isolated. Typically combined with an adjuvant immune stimulant, an autologous cell vaccine may elicit a cytotoxic T-lymphocytic immune response to cell surface-expressed tumor-associated antigens (TAAs), resulting in tumor cell death.