If you are an expectant parent, it is helpful to know that more than 80 diseases are currently being treated with umbilical cord blood stem cells. Although the first successful stem cell transplant from bone marrow was done almost 60 years ago, the first cord blood transplant wasn’t done until 1988. Matthew Farrow, then a five-year-old boy with Fanconi anemia was the recipient. He is now 30 years old, married, and a father. Founded in 1971, New England Cryogenic Center (NECC), the parent company of New England Cord Blood Bank (NECBB), is considered one of the foremost pioneers in the processing and cryopreservation of human cells and tissue. NECBB continues to generate significant research advancements for cellular therapies.
Currently, clinical trials with cord blood stem cells are being conducted in the US for neurological, cardiovascular, and auto-immune disorders.
What is a clinical trial? Simply put, it is an investigation of some form of treatment. Clinical trials follow strict guidelines to ensure the new drug or procedure is safe and effective. A treatment, drug, or procedure (called an “intervention”) often starts with an animal model, computer model, or cell-based model. To assess how well the intervention works in people, it must be studied in human models. The clinical trial is a multi-step process, broken down into phases. Each phase has a specific purpose and increases the number of participants in succession. First, participants sign and agree to an “informed consent” that covers the scope of the study, expected risks, and potential benefits. Participants may withdraw from the study at any time for any reason.
- Phase 0 and 1 studies are small and have safety as the main goal, such as determining if there are any severe events, and how the drug is metabolized and excreted.
- Phase 2 studies center on determining the drug’s effectiveness for a given condition. Participants are often compared with other test groups receiving a placebo (an inactive substance) or a standard treatment for the condition.
- Phase 3 expands the test population and further ascertains safety and effectiveness, often looking at different dosages and test populations. At this stage, the FDA can approve or reject the intervention.
- Phase 4 studies, if approved, often take place to determine long-term trends of the intervention.
“A Randomized Study of Autologous Umbilical Cord Blood Reinfusion in Children with Cerebral Palsy” at ClinicalTrials.gov is one example of a clinical trial involving cord blood stem cell treatment. To date, more than 30,000 cord blood transplants have been performed around the world. Through clinical trials and the commitment to research by organizations like New England Cord Blood Bank, there is no doubt that this is just the beginning.