What are cell and gene therapies and how do they work?
Ground-breaking cell and gene therapies have the potential to transform the way we treat and prevent many diseases, providing hope to those with conditions that were previously untreatable. These therapies offer a range of potential benefits such as drastically improved treatment outcomes, improved quality of life, cost savings, and reduced side effects.
Here, we look at exactly what cell and gene therapies are and how they work.
What is gene therapy?
The use of genetic material in the treatment or prevention of disease, gene therapy works by introducing healthy genes into cells in place of missing or malfunctioning genes. The healthy gene can then produce a functional protein which can help the cell work properly.
Gene therapy is used to treat a wide range of diseases, including cancer, neurological conditions, and immune disorders.
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What is cell therapy?
Cell therapy is the therapeutic use of living cells or cell products to treat or prevent a disease. It is a type of regenerative medicine that involves the transfer of healthy cells, tissues, or organs from a donor to a recipient in order to replace or repair damaged or dysfunctional cells, tissues, or organs.
It can be used in a wide range of diseases, including cancer, autoimmune diseases, and genetic disorders.
What are the main differences between the two?
Cell therapy works by cultivating or modifying cells outside of the body before injecting them into the patient. These cells can either come from the patient themselves (autologous cells) or from a donor (allogeneic cells).
Gene therapy, on the other hand, involves replacing, inactivating, or introducing genes into cells - either inside or outside of the body. Some therapies are considered both cell and gene therapies, as they involve altering genes in specific types of cells and inserting them into the body.
How many different types of therapy are there?
There are two main types of cell and gene therapy: autologous and allogeneic. Autologous cells are those taken from the same individual, while allogeneic cells are those derived from a donor.
Additionally, there are a variety of techniques used to deliver cells and genes to a patient, such as viral vectors, direct injection, and ex vivo engineered cells.
More than 1,000 different types of gene therapy are in clinical trials, while almost any gene in the human genome can be targeted, meaning the potential for new therapies is enormous.
Has treatment been approved?
Yes! Cell and gene therapy have been approved in certain cases. In the United States, the Food and Drug Administration (FDA) has approved several cell and gene therapies, including CAR-T (chimeric antigen receptor) therapy and gene therapy for certain types of cancer, as well as gene therapies for certain inherited diseases.
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In the United Kingdom, the Medicines and Healthcare Products Regulatory Agency (MHRA) has approved several cell and gene therapies, including CAR-T therapy for certain types of blood cancer and gene therapy for certain inherited diseases.
Under guidance from the National Institute for Health and Care Excellence (NICE), children with a rare, fatal genetic disorder called metachromatic leukodystrophy will be able to undergo a one-off treatment known as atidarsagene. It costs around £2.8million at list price and is the most expensive drug NICE has ever examined.