Fast Track Your Cell Therapy Research

After years of cell and gene therapy research work to develop a new cellular product or a new technique, researchers provide a proof of concept of their innovation. The new product is safe in preclinical studies, and is also effective; in short, it works!

I have previously discussed how a new cell therapy process may be suitable for clinic in a separate post, and showed that the new process need to be Repeatable, Scalable, and Compliant. In this post I’ll discuss the “Compliance” requirement a little further. 

As researchers move on with their product to be translated to clinic, they are faced by the regulatory requirements that need to be met for the product to be approved for clinical production. Researchers may find some of the regulatory requirements hard to meet, and may take them back to research. In other words, researchers are moving backward instead of forward.

To understand why researchers may be faced by regulations, we first need to know the purpose of the regulations. Regulatory standards are set to protect the patient, and all involved in the cell therapy cycle. This include the environment, facility, personnel, etc. Regulatory agencies may accept alternatives that satisfy the purpose of the regulations, but if a gap exists between regulations and practice, the practice will not be approved.

For research that is intended to be translated to clinic, researchers need to not loose sight of “clinical production” requirements. This may be too much to ask from researchers, therefore, translational research centers would benefit from employing a team member with quality assurance focus.

As a researcher, you need to be aware of this phase that every researcher will arrive at to translate their work to clinic. This knowledge allows you to prepare by developing a process that is as compliant as possible.

• Know the regulations: you need to know what regulation is demanding from developers of a product type like yours. As mentioned above, these requirements will define the environmental, reagent, material, equipment, facility, and personnel requirements. Using this information, you can tweak your process only to match the requirements. As you proceed with your research, you also implement the material grade required, or at least make sure you can easily switch to them. For instance, some reagent need special preparation, such details need to be part of your process early on and not something you switch to for production. You may switch grade of material for implementation, but the material need to be part of your process.
• Know the facility: unlike the other requirement, having the right facility for clinical production is usually the bottle neck for cell therapy production. Knowing early on the required type of facility allows you to plan for any needed modification, or even constructing a whole new facility; a process that is costly and time consuming, and may halt your progress if not planned properly.

Reach out to regulatory agencies to help you identify the requirement, reach out to them early on in your process for many aspects of your research may be impacted to be compliant. Similarly, reach out to companies of cleanroom facility construction and find out how YOUR process may be adopted and what facility type suits your products/regulatory requirements the best.

This type of experience is cumulative, the first time you do it may be challenging, but it certainly paves the way for your following inventions, and makes the translational research a breeze.

Let us know if we missed anything, or if you would like a specific subject to be discussed, write to us. 

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