John Milne PhD
Training Director, NIBRT
Cell and gene therapy (CGT) aims to treat, prevent, or potentially cure diseases and has the potential to alleviate the underlying cause of genetic and acquired diseases.
A look at therapies already approved or currently in development in the CGT area clearly demonstrates the interest in these treatments by the wider biopharmaceutical industry.
Cell therapy has been used for many years, for example in bone marrow transplantation. However in the context of CGT, cell therapy refers to cells that have been expanded or modified through exposure to growth conditions outside the body. Cell therapies can be classified as autologous (where a patient is treated with their own cells) or allogeneic (where a patient is treated with cells from another individual).
Gene therapy involves the administration of genetic material to modify or manipulate the expression of a gene product. Genes provide the specific information to produce proteins crucial for life. Gene therapy can replace a gene that causes a medical problem with a healthy copy of the gene, add genes to help the body to fight or treat disease or turn off genes that are causing disease.
Gene therapy involves the administration of genetic material to modify or manipulate the expression of a gene product.
Modifying genes for targeted therapy
Gene modified cell therapy combines both cell and gene therapies where cells are genetically modified outside the body and infused into the patient. A good example is CAR-T cell treatments, where T-cells (crucial cells in the immune system) are harvested from cancer patients, re-programmed with a specific gene delivered by a viral vector, expanded, tested and infused back into the patient. The gene modification allows the modified T-cells to detect and kill the cancer cells and have the advantage that they can persist in the patient after the cancer is effectively treated.
Opportunity for Ireland
Given Ireland’s tremendous success over recent years in positioning itself as a major international hub for biopharmaceutical manufacturing, there is now great potential to embrace the opportunity that CGT will undoubtedly bring.
Collaboration between stakeholders drawn from government agencies, higher education institutes and the wider industry to provide the necessary workforce of the future, is underway and will be crucial to our success in this area.