Cells, each of which contain our full genetic information, are the building blocks for complex life. Genes carry information in the form of DNA which, not only determine our physical characteristics, but also govern the day-to-day functioning of our biological processes and ultimately maintain our health.
A genetic disorder or disease occurs when a segment of our DNA is damaged, missing, duplicated, or substituted, causing our body to function in an unexpected way. This can be seen in conditions like cancer where a dividing cell mutates and develops a genetic abnormality, that causes it to begin to reproduce rapidly developing into a tumour. Other clinical conditions associated with genetic disorders are often described as rare and complex diseases.
As these diseases are caused on a genetic level, they were traditionally understood poorly and largely considered incurable. Genetic diseases remain prevalent, with many having few, if any, treatments to alleviate symptoms. This is especially true of numerous cancers. With the advent of cell and gene therapies, there are now emerging therapies that offer hope for effective treatments or even a cure for these life-threatening conditions.
While they are related fields that sometimes overlap, cell therapy and gene therapy are different.
Cell therapy uses cultivated cells which have been genetically modified to address a specific genetic condition. Cells cultivated under carefully controlled conditions are introduced to the patient and contain vital genetic information to induce the required therapeutic effect.
Cells can be acquired from an individual patient whose cells are processed and re-administered into the patient, known as autologous cell therapy. In some instances, the cells may have been adapted from donor cells. This is known as allogeneic cell therapy.
Gene therapy is a new-age treatment, it works by changing a patient’s DNA on a cellular level either with treatments inside the body (in vivo) or outside the body (ex vivo). Many of these treatments use therapeutic viruses, which are engineered to induce changes in a patient’s cells.
Research into Cell and Gene Therapy becomes real for patients when breakthroughs translate into the manufacture of Advanced Therapy Medicinal Products (ATMPs). These new medicines treat genetic diseases at the source by affecting changes in their tissue, cells, or DNA with the potential to provide an effective ‘once only’ treatment to alleviate severe disease.