Cell & Gene Therapy Apheresis 101
What Is Apheresis?
Apheresis is a medical procedure during which the donor’s or patient’s blood flows through an apheresis device, within a closed sterile kit, in order to remove a specific blood component (e.g., plasma, red blood cells, white blood cells and platelets) while returning the other components back to the patient or donor.
A small amount of whole blood is continuously removed through an intravenous line into the kit, the device uses centrifugation to separate the blood components, the target component is siphoned off into a collection or waste bag, and the remaining blood components are returned to the donor or patient through a second intravenous line. Depending on the procedure type, the typical time that the donor or patient is connected to the device is about 1-4 hours.
What Is Apheresis Used For?
Cellular Therapy Apheresis
Cellular therapy apheresis refers to apheresis procedures with the goal of collecting an adequate number of cells which are the actual treatment. The cells collected are further processed for standard bone marrow transplantation or for novel cell and gene therapies (CGT). Cellular therapy products are created either from a patient’s own “autologous cells” or from a donor’s “allogeneic cells” and include a variety of starting cell types such as hematopoietic stem cells or various white blood cell types such as lymphocytes or monocytes.
Some researchers and clinicians choose to outsource this process to ensure regulatory compliance, data quality, safe and effective product storage and cellular therapy product manufacturing without taking on the cost burden of equipment, supplies and highly trained and experienced professionals.
Citing a marked increase in CGT related applications, the FDA announced in early 2019 its intention to hire 50 additional clinical reviewers to oversee the investigational new drug applications (IND) for CGTs. FDA IND approval is required to implement CGTs. Recognizing the growth and potential in this field, the FDA has developed guidance documents and assistance to researchers and also provided an accelerated approval pathway for certain circumstances.
Therapeutic apheresis (TA) refers to apheresis procedures which in and of themselves constitute the actual treatment. The medical condition (e.g. thrombotic thrombocytopenic purpura, sickle cell disease) determines what blood component needs to be removed, and with certain procedure types, blood-based replacement fluid is also required.
The American Society for Apheresis (ASFA) issued evidence-based guidelines for TA in 2019 containing over 80 conditions for which TA can be used. For certain conditions, therapeutic apheresis is the first-line treatment, and for other conditions, it is an important adjunctive treatment.
Why Is Apheresis Important for the Advancement of Cellular Therapy?
Cell and gene therapy products hold the promise of advancing the field of medicine by providing potential treatments and cures for previously intractable cancers and genetic disorders. Development of these new therapies depends on the availability of various blood cell types to manufacture these products in the laboratory. Apheresis provides a more simple and safe method for collecting these cells compared to direct bone marrow harvest. Companies that have expertise and are available to provide these services in diverse locations have the potential to bring these therapies to all patients, even those treated in small community-based hospitals.
Comprehensive Cell Solutions’ Apheresis Expertise
An industry leader, New York Blood Center’s Comprehensive Cell Solutions provides expertise in apheresis collections and cellular therapy product manufacturing and is equipped to deliver the highest quality services. Services include procurement of cells by apheresis from patients or donors, transport under temperature-controlled conditions, cellular therapy product manufacturing, temperature controlled storage, and distribution of the resulting product.
fill out an inquiry form or Call us at (212) 570-3021 to learn how we can support your organization.