Advancing Cell and Gene Therapy on International Childhood Cancer Day
Enabling the Outcome™ for Pediatric Patients
Every year on February 15th, International Childhood Cancer Day (ICCD) serves as a global platform to raise awareness about childhood cancer, advocate for improved access to care, and highlight the need for continued research and innovation. Pediatric cancer remains one of the leading causes of death among children worldwide, with approximately 400,000 new cases diagnosed each year. While significant progress has been made in conventional treatments such as chemotherapy, radiation, and surgery, the emergence of cell and gene therapies offers new hope for children battling these devastating diseases.
At Cryoport Systems, we are dedicated to supporting the pioneers behind this next-gen approach to Advanced Therapy Medicinal Products (ATMPs). Our fully integrated, end-to-end approach to temperature-controlled supply chain management plays a crucial role in ensuring the safe, reliable, and timely handling of life-saving treatments, helping to bridge the gap between scientific breakthroughs and real-world patient impact. As we recognize ICCD, we reaffirm our commitment to enabling the next generation of pediatric oncology treatments through innovation, excellence, and unwavering support for our partners in the cell and gene therapy industry.
The Evolution of Pediatric Cancer Treatments: From Traditional to Next-Gen Therapies
The advent of cell and gene therapies (CGTs) represents a paradigm shift, offering the potential for more targeted, less toxic, and highly effective treatment options for childhood cancers.
Cell Therapy Advances: One of the most promising developments in pediatric oncology is CAR-T cell therapy, which involves reengineering a patient’s T cells to recognize and attack cancer cells. This personalized approach has yielded remarkable success in treating pediatric acute lymphoblastic leukemia (ALL), achieving remission rates exceeding 80% in some cases.
Gene Therapy Breakthroughs: Gene-editing technologies, such as CRISPR and viral vector-based therapies, have opened new avenues for correcting genetic mutations that contribute to childhood cancers. By directly modifying a patient’s genetic material, these therapies offer the potential for long-term, durable remissions.
While these therapies hold immense promise, they also present complex challenges. The transportation of patient-derived cells and genetically modified products demands precise temperature control, regulatory compliance, and seamless coordination across global supply chains. This is where Cryoport Systems plays a pivotal role.
The Cryoport Systems Difference: Ensuring Integrity in the Supply Chain
The success of cell and gene therapies hinges on the ability to maintain product integrity from collection to administration. A single deviation in temperature, handling, or transit time can compromise the efficacy of these fragile treatments, ultimately impacting patient outcomes. At Cryoport Systems, we have developed industry-leading solutions to mitigate these risks and ensure the reliable delivery of pediatric oncology therapies while establishing an integrated, end-to-end approach to the temperature-controlled supply chain in support of evolving industry needs and the growing need for efficient, scalable solutions.
Comprehensive Temperature-Controlled Logistics: Our advanced shipping systems encompass proprietary technology to maintain ultra-low temperatures, preserving the viability of sensitive biologics throughout transit. Our Smartpak II® condition monitoring system together with the Cryoportal® provide near real-time data about a shipment’s internal temperature, external temperature, orientation/tilt, location, and other metrics. This proprietary and enhanced logistics management system is validated to demonstrate compliance with 21 CFR Part 11 (Code of Federal Regulations) and ISPE GAMP (International Society for Pharmaceutical Engineering, Good Automated Manufacturing Practices).
Chain of Compliance®: Unlike conventional logistics solutions, Cryoport Systems offers unparalleled traceability, ensuring the integrity and custody of critical materials throughout their journey. In full alignment with the most stringent industry regulations like ISO 21973, our Chain of Compliance® integrates validated requalification procedures, segregates human- and animal-derived products, and maintains a complete history of equipment performance and location. By leveraging extensive data collection and management capabilities, we mitigate risks and implement preventative measures to ensure the safe delivery of invaluable materials.
Cryopreservation and Standardization with IntegriCell™: As cell therapies become more widely used, manufacturability constraints create bottlenecks. IntegriCell™ cryopreservation services optimize leukapheresis-derived cell therapies by enhancing the safety, quality, and viability of manufacture-ready cryopreserved leukopaks. Our state-of-the-art cryopreservation process, integrated with our end-to-end global temperature-controlled supply chain platform, provides unmatched reliability, efficiency, and scope.
Global Infrastructure: With our global network of facilities and services, Cryoport Systems is a strategic partner of choice for temperature-controlled supply chain management of critical materials. Our patented, state-of-the-art shippers, integrated global BioServices and biostorage capabilities, continuous cloud-based monitoring, and efficient local pickup and delivery services combined with tailored advisory and consulting services maximize the safety and quality of your life-saving therapies and critical materials. Our growing footprint is Enabling the Outcome™ for biopharmaceutical research and development, connecting additional services and capacity around the world.
By leveraging these capabilities, we are Enabling the Outcome™, allowing cell and gene therapy developers to focus on what matters most: advancing the science and delivering life-saving treatments to children in need.
Looking Ahead: The Future of Pediatric Oncology
As cell and gene therapies continue to evolve, the future of pediatric cancer treatment is increasingly promising. Research is advancing toward next-generation CAR-T therapies with improved persistence and durability, gene-editing techniques that offer one-time cures, and combination therapies designed to enhance efficacy while minimizing toxicity. However, realizing these advancements requires a robust, scalable, and highly controlled supply chain infrastructure.
Cryoport Systems is committed to staying at the forefront of this transformation. Our ongoing investments in technology, process optimization, and regulatory expertise position us as a trusted partner for the cell and gene therapy community. We understand that behind every shipment and stage in the supply chain, there is a child, a family, and a medical team holding onto hope for a cure. That is why we will continue to push the boundaries of supply chain excellence, ensuring that these therapies reach the patients who need them most.
Our Commitment on International Childhood Cancer Day
On this International Childhood Cancer Day, we stand in solidarity with patients, families, researchers, and healthcare professionals dedicated to the fight against pediatric cancer. We recognize the urgency of developing and delivering innovative treatments, and we are proud to play a role in enabling the outcome for children around the world.
Together, we can turn scientific breakthroughs into life-changing realities and give more children the chance to live healthy, cancer-free lives. At Cryoport Systems, our mission is clear: to enable the future of medicine, one successful therapy at a time.