(DGIwire) – The year 2016 has been a lucky one for a 27-year-old man from San Diego: although his acute lymphoblastic leukemia (ALL) returned for the fourth time in his life, it is now in remission. But his treatment involved no radiation or chemo; instead, the credit goes to a cutting-edge form of immunotherapy. As reported by the University of California San Diego, the patient underwent the treatment—known as CAR T-cell therapy—as part of a Phase 1/2 clinical study at UC San Diego Health, sponsored by Kite Pharma.
According to UC San Diego, in this novel cellular immunotherapy, the patient’s own T-cells—part of his immune system—were collected through a blood draw. In a lab, his T-cells were genetically modified to produce special receptors on their surfaces, called chimeric antigen receptors (CARs). CARs allow the T-cells to recognize a specific protein on cancer cells. After infusion back into the patient’s body, the CAR T-cells trained his immune system to recognize and kill his leukemia cells because they harbored that specific protein.
“Being able to genetically engineer a patient’s own T-cells to recognize and attack cancer cells represents a great leap forward in the treatment of various cancers,” says Mike Rice, President and CEO of BioLife Solutions, a public Seattle-area company supplying biopreservation tools to the cell therapy industry. “One of the challenges that must be overcome for CAR T-cell therapies to succeed in treating and curing various blood cancers and solid tumors is ensuring that a patient’s collected blood, including the precious T cells, remains viable during transport to the lab and that the T cell therapy manufactured from the blood draw remains viable during transport back to the clinic for infusion into the patient.”
Traditional methods of storing and transporting human cells, tissues and organs at low temperatures often fall short. For example, healthy cell samples are often so stressed after freezing and thawing that they can die or degrade, rendering them therapeutically useless. Additionally, cell, tissue and organ samples are often stored and transported in containers that are scarcely more sophisticated than Styrofoam coolers, with no way of ensuring the time- and temperature-sensitive biologic material remains within an acceptable temperature range at every point during the trip.
BioLife Solutions is meeting this challenge on two fronts. The company’s CryoStor® and HypoThermosol® biopreservation media enable a significant reduction in the levels of cell death (improved survival or yield) compared to traditional biopreservation media formulations. The CryoStor family of products are intended for the cryopreservation of biologics (T cells, cord blood cells, etc.) at -70°C to -196°C. Meanwhile, the HypoThermosol family of products are optimized for storage and shipping of cells and tissues at 2°C to 8°C. To date, the company has built up a large customer base that uses its products in more than 220 validations and clinical trials of cellular therapies. These customers include bluebird bio, Adaptimmune, TC Biopharm, Bellicum Pharmaceuticals and Kite Pharma, which has incorporated BioLife’s CryoStor freeze media into its manufacturing process for a CAR T-cell therapy currently in four clinical studies of various cancers—including the one in San Diego that helped the 27-year-old patient.
On the other front, BioLife Solutions, with its joint venture partner SAVSU Technologies, has developed a cloud-based cold chain logistics system branded as biologistex™. The heart of this system is the evo™ Smart Shipper, a leading edge, durable and reusable “smart box.” The biologistex SaaS application receives live data streams transmitted from the evo while in transit. Critical payload information—such as location, payload temperature, pressure, humidity and tilt—is provided to users through this cloud application. Users can set up email and text alerts to be sent to destination contacts informing them that the evo is approaching the destination, when it arrives, when it is opened and how much shelf life remains, to ensure time-sensitive, life-saving cellular therapies are administered to the patient within a validated stability profile.
“A genetically modified cell sample or other tissue or organ isn’t just an object that needs to be protected as it is stored or transported—on the contrary, it literally may be a life-improving treatment or even a life-saving cure for the patient,” adds Rice. “We owe it to that patient to ensure that the biologic-based medicines are kept as healthy as possible. CryoStor, HypoThermosol, evo and biologistex represent best cold chain practices for preservation and distribution of time- and temperature-sensitive biologic materials.”