It’s getting cold out there! And while winter temperatures may have us yearning for a warm spot by the fireside, cold is a good thing for cell and gene therapies—in many cases, the colder the better. Logistics dictate that international shipping of cellular therapeutics often means freezing them first. Cryopreservation protects fragile cellular material from loss of viability or functionality during transit and storage, by halting chemical and biological degradation processes. To understand how important that is, it is worth noting that two of the hottest cell therapy drugs on the market this year (Yescarta® and Kymriah®) are both cryopreserved prior to infusion into the patient. But how cold does it really need to be to protect the efficacy of these cellular therapeutics? We decided to put that question into perspective.
Cryopreservation of therapeutic cells allows the long-term storage of functional cells and prevents degradative processes. Fresh cells are also used for cellular therapy approaches; however, there are many circumstances that make the availability of cryopreserved cells a must for repeated and on-hand use of cells. Cell cryopreservation provides a reliable stock of cells, which reduces the variability of cell therapy product. This translates to a more standardized product that can be used on a larger scale and without constraints on optimal availability for clinical use.
The leading cause of death for U.S. citizens is cardiovascular disease, which affects nearly a third of the U.S. population. There is an active and dedicated search for effective therapies to address this serious medical issue. The use of immunotherapy approaches for a number of diseases and conditions is continuously gaining momentum. Included in this is the use of CD34+ stem cells from a patients’ own blood (autologously derived) to treat cardiovascular disease. CD34+ is derived from bone marrow and other tissue types. However, CD34+ stems cells are more widely known for their hematopoietic origin.
Leukapheresis is a procedure to separate white bloods cells (including dendritic and progenitor cells) from a blood sample. Products obtained from leukapharesis procedures, leukopaks, are regularly used in the research setting. They are also used for cell therapy process development and clinically for certain treatment procedures for blood disorders. Another way to obtain white blood cells and platelets is to obtain them from the buffy coat.
Nearly 7 million people in the U.S. (about 2% of the total U.S. population) suffer some level of stroke-related brain damage and related health problems. Studies in animals suggest that cell-based therapies can improve post-stroke outcomes. To determine the safety of cell-therapy approaches in humans, researchers from Stanford University conducted a clinical trial to study the safety of a procedure to transplant donor stem cells in the brains of patients with chronic stroke.