Since its first successful use more than three decades ago, umbilical cord blood has become an increasingly important source of stem cells for transplants for bone marrow failures, leukemia, lymphoma, sickle cell anemia, and other life-threatening diseases. Over 40,000 cord blood transplants have been performed worldwide for a wide variety of malignant and non-malignant disorders. The first successful cord blood stem cell transplant was performed in 1988 as a treatment for a 5-year-old boy with Fanconi Anemia; his newborn sister’s umbilical cord blood saved his life. Today, more than 80 diseases have FDA-approved cord blood stem cell transplant treatments.
In recent years, cord blood has extended its reach beyond the realm of transplant medicine, delving into the promising domains of regenerative medicine and clinical research trials. This paradigm shift involves a distinct approach compared to traditional transplant medicine, where cord blood is explored for its potential to rejuvenate or facilitate healing by tapping into the body’s innate regenerative capabilities.
In regenerative medicine, both cord blood and cord tissue stem cells are harnessed in clinical trials aimed at addressing currently untreatable conditions. Approximately 83.7% of revenues generated from cord blood and cord tissue in the global market stem from their use in stem cell transplantation, whereas the remaining 16.3% is attributed to regenerative medicine applications.
Traditionally, cord blood has predominantly served as a source for hematopoietic stem cell (HSC) transplantation, primarily for blood cancers. However, a paradigm shift is underway as these resources are increasingly employed in novel applications targeting non-hematopoietic diseases and as agents for cellular regenerative therapy and immune modulation. Today, novel cell-based products are derived from umbilical cord blood cells, including mesenchymal stromal cells, endothelial progenitors, and neural progenitors.
Forward-looking companies like Nohla Therapeutics, Gamida Cells, Angiocrine Bioscience, and BioIntegrate LLC have been at the forefront, dedicating their efforts to developing innovative cord blood and cord tissue-based cellular therapies. These therapies hold promise for treating conditions such as spinal cord injury, stroke, traumatic brain injury, diabetes mellitus, gastrointestinal diseases, and dermatologic ailments.
Off-the-shelf cord blood units (CBUs) have emerged as a rich source of cells for the advancement of allogeneic cell and gene therapies. Additionally, cryopreserved cord tissue is gaining traction as a readily accessible source of allogeneic (donor-derived) mesenchymal stem cells (MSCs). Cord Blood Registry, a prominent entity in the field, plays a pivotal role by releasing nearly 80% of its stored cord blood for clinical research purposes.
The journey of umbilical cord blood from its early triumphs in transplant medicine to its current role in pioneering regenerative therapies represents a remarkable evolution in the field of medical science. With each passing day, the potential applications of cord blood and cord tissue continue to expand, offering newfound hope to patients.