Gene and cell therapy are the essence of the popular buzzwords personalized medication, patient-specific therapies or targeted medicine. Gene and cell therapy both aim at the direct cause of a disease by replacing or repairing defective genes or cells. These therapies are around for some time, but only in the past two decades have they been applied in a wider range and haven been taken on to an advanced therapy stage. When gene and cell therapies undergo clinical trials, they provide unprecedented challenges for the supply chain – especially for cold chain logistics.
Gene and cell therapy defined
Both cell and gene therapy are fields of biomedical research. Yet, their procedures slightly differ from one another:
- Cell therapy: This approach implies the injection of whole live cells into a patient. This has been applied broadly in bone marrow transplantation and in oncology. A patient´s defective bone marrow cells are being destroyed by radiation or chemotherapy. Then donor cells, usually of a closed relative, are infused so that they can populate in the bone marrow and replicate. The repertoire of cell types apt for cell therapy is growing.
- Gene therapy: This approach is designed to insert gene material into cells to compensate for defective genes or to produce an advantageous protein. Since directly inserting a gene does not work, the gene is delivered by a carrier into the cell. Modified viruses are usually used as carriers since they “infect” the cell and thus insert the engineered gene. The modified virus is either injected or administered intravenously as an infusion. That depends on where the defective gene that is to be replaced is located. As an alternative form of delivery, a sample of a patient´s cells can be extracted and then be treated with the carrier virus in a laboratory setting. Then it is returned to the patient.
Why the bar for cold chain logistics is rising
Even though gene and cell therapy have been around for a bit, only lately are they applied more often to rare diseases, neurological, cardiovascular, metabolic or infectious diseases. Research is advancing rapidly as gene and cell therapy is a promising field – aiming at the cause of an illness not merely at its symptoms. This comes to no wonder that more and more clinical trials focus on cell and gene therapies. The number of gen-based clinical trials alone has increased about 500 % in the past 20 years.
Clinical trial logistics procedures in gene and cell therapies are complex and demand a lot of all stakeholder involved in the supply chain. These therapies entail taking up starting cell material from the patients (e.g. stem cells, whole blood etc.) and delivering living cells or viral vectors which have a limited lifespan. This needs strict temperature-management and fast delivery. Even more so since medication is targeted at a single, specific patient which fragments the supply chain.
- Keeping up a seamless cold chain: All the way from and to the patient
- Keeping a strict temperature range: According to World Courier most common is 2-8°C, but there are also frozen or cryogenic transports
- Sticking to tight delivery times: Living cells must be administered swiftly
- Managing cross-border shipments: There are trials that are carried out in 39 sites all over the world
- Handling various regulations: Cross-border shipments need to take into account the different regulations for the handling of living cells in the different countries
Gene and cell therapy clinical trials require safe and 100 % reliable cold chain packaging and logistics providers that are flexible and dependable. The bar for cold chain delivery is raising.