The cell and gene therapy have a much simpler principle than what is perceived. As a field, the cell and gene therapy gained a lot of attention due to several factors such as (1) the list of applications that keep growing, (2) the state-of-the-art facilities, and (3) the complexity of some processes. This spike in attention brought with it tremendous funding and support, and helped increase the pace of cell and gene therapy development. This heightened pace also led and still is to more products in testing. However, this attention also raised the bar of standards of manufacturing in a way that may seem too high for new comers. In this article, I will explain what cell and gene therapy principle is, and how the bar is actually at a reasonable height.
What is cell therapy?
In its principle, cell therapy is the use of cells to prevent, correct, or treat a specific condition. Cell therapy has been described in different format to highlight a specific specialty such as stem cell therapy, cell and gene therapy, or regenerative medicine and cellular therapies. The change in format at times also reflects the cell type that was used, e.g., stem cell, other times the format reflects the use of some sort of gene modification such as cell and gene therapy. Regardless of changes in format, the principle of this therapy is to use a cell-based therapy.
Classification of cell therapy
To simplify this field, I’ll describe the product classifications and what they mean.
Origin: In general, the cell-based product may originate from the patient themselves (autologous), or may originate from a different person (allogeneic).
Manipulation: Autologous or allogeneic products may be exposed to a brief processing that don’t alter the product characteristics (minimally manipulated). Alternatively, the product may be exposed to a manipulation that alter the original characteristics or functions of the product (more that minimal manipulation). Each level of manipulation has a specific set of standards to ensure the safety of the final product. You may also refer to my previous articlefor details on manipulation.
Components of cell therapy
The cell therapy system has 3 major components, the clinic, collection, and manufacturing. Generally, the cell therapy works in the following order:
Patient being diagnosed at the clinic, and a cell therapy plan is devised.
Based on the therapy plan, either the patient or the donor gets their cells collected at the collection facility.
The collected product is sent to the manufacturing facility with prescription to define the required manipulation. At this facility, the personnel would follow the prescription for minimal manipulation such as volume reduction or RBC depletion. The personnel would also follow the prescription for more than minimal manipulation such as transduction or gene editing.
Once the manufacturing is complete, the approved cell therapy products are sent to the clinic to be administered to the recipient.
Therapies like the cellular ones are mostly being administered through clinical trials because they are still experimental. And although experimental, many cell therapies are the only hope for so many patients. Unlike pharmaceuticals, cell therapies do not go through terminal sterilization, which makes it necessary to protect the product throughout manufacturing. To ensure such clean production facility, regulatory standards were set to control every step of the clinical production.
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About the author
Naseem Almezel, earned his MSc degree in Cellular Therapies in 2010, since then his career focus is to support Bone Marrow Transplant and Oncology programs. Naseem likes to work in the lab doing translational research, or in the cleanroom doing GMP production. When he is not working, Naseem likes to read and to spend time outdoors. Find more about Naseem here
