Stem Cell Technology: Preserving Your Future Today
Stem cells are the master cells for every tissue and organ of our bodies. They can be considered a personal and precious cell reservoir for each individual — the building blocks of our body. During our life, we continue to rely on our stem cells to regenerate injured tissues and to replace cells that every day are damaged or lost. They possess the peculiar property of self-renewal: that is, they divide in such a way that they generate identical copies of themselves or differentiate into any kind of specialized cells in our body, depending on the microenvironment and the stimuli to which they are subjected. Despite the great potential of these kinds of cells and their fundamental role in preserving our body, stem cells can mutate during life, becoming malignant, causing severe diseases, or simply losing their potential. Just the fact that they are subjected to aging causes a reduction in the number and efficacy of stem cells during our life. For these reasons, harvesting and banking stem cells when they are young and healthy is very important.
In this sense, umbilical cord blood, a potent source of hematopoietic stem cells, represents the much easier and safer way to collect the youngest stem cells. The procedure consists of harvesting cord blood from the umbilical vein at birth, the process of which poses no risks for mother or baby. Typically, the sample is then shipped to the responsible laboratory in order to separate and store cord stem cells for future medical applications.
The first successful cord blood transplant was performed in 1988 under the direction of American scientist Dr. Hal Broxmeyer. A six-year-old boy affected by Fanconi’s anemia was treated and cured using his sister’s stem cells. That young patient is now in his early 30s and leads a healthy and normal life. In the early 90s, researcher Pablo Rubinstein from the New York Blood Center established the first public cord blood bank in the US. Shortly afterwards, the first cord blood transplant from an unrelated donor was performed on a four-year-old boy with T-cell leukemia. In the following years, cord blood was utilized as an alternative to bone marrow to treat different disorders such as hematological malignancies, genetic immunodeficiency syndromes, bone marrow failure, hemoglobinopathies, and metabolic diseases. To date, more than 400,000 transplants have been performed. Currently, about 800,000 unrelated stem cell samples are stored in public banks while five million are stored in private ones.
The first successful cord blood transplant was performed in 1988 under the direction of American scientist Dr. Hal Broxmeyer. A six-year-old boy affected by Fanconi’s anemia was treated and cured using his sister’s stem cells.
The main clinical limitation of cord blood samples is the reduced number of stem cells that can be harvested, thus influencing the transplant outcome. Wagner and colleagues partially mitigated this issue by using two umbilical cord blood units for each transplant, and, more recently, many groups have been developing multiple methods of cord blood stem cells expansion to obtain a large quantity of cells for transplant.
In addition to hematopoietic stem cells, another group of cells that is rising in importance is mesenchymal stem cells. These cells can be easily isolated both from Wharton’s jelly, a cord tissue jelly localized around umbilical cord blood vessels, and from adipose tissue, which can be collected easily through liposuction also in adults.
The ability to differentiate into bone, cartilage, and fat has created a great interest in using mesenchymal stem cells in orthopedics, ophthalmology, neurosurgery, and cardiology. Thanks to their immunoregulatory properties, these kinds of cells are also under clinical study to treat immune-related disorders such as graft-versus-host disease following bone marrow transplant and autoimmune diseases such as multiple sclerosis, type I diabetes, and scleroderma. Mesenchymal stem cells can also be co-transplanted with hematopoietic stem cells to ameliorate the engraftment and increase transplant success in hematological malignancies. At present, almost 5,000 clinical trials worldwide are testing stem cells for different pathologies as reported on the ClinicalTrials.gov registry.
Numerous studies have indicated that stem cells isolated from older donors are neither as prevalent (in terms of the number of cells in the sample) nor as potent as those isolated from younger, healthier donors. Stem cells collected from older or disease-afflicted donors seem less able to differentiate into different cell types needed for tissue repairing, have a reduced capacity to proliferate and expand into numbers of cells that would allow for multiple treatments, and are more prone to dying during culture and use. In light of this, it is important to preserve cells as soon as possible during one’s life. Nevertheless, how these cells are collected, processed, and preserved is also very important. All the steps of this important process have to be performed by well-trained people, using validated and approved procedures and materials.
It is important to preserve cells as soon as possible during one’s life
Starting from collection through to sample transportation, manipulation, and quality controls in place, everything needs to be validated and controlled each time. Quality assurance plays an important role in the development of products and services in pharma. In fact, a laboratory that processes and stores stem cells is considered a pharmaceutical company, and international law recognizes stem cells as a drug.
Quality assurance refers to the step-by-step process of examining whether goods and services meet specific quality, efficacy, and safety requirements, as per their intended use. Some national and international standards are used as guidelines to ensure that a company’s quality assurance system is both effective and ready to be implemented. For a cord blood bank, for example, the most important and worldwide-recognized standard is issued by FACT-NetCord, which was established in 1999. A facility’s ability to store stem cells in a FACT-NetCord-accredited cord blood bank is assurance that the facility strives to achieve the highest quality care for cord blood bank programs. This is particularly important for physicians, as they are guaranteed that the samples coming from the facility are safe and approved for a transplant.
In addition to international accreditations, every laboratory that processes and stores stem cells has to be authorized at national level. In Switzerland, the surveillance authority for medicines is the Swiss Agency for Therapeutic Products (Swissmedic), which inspects facilities every two years to ensure compliance with current standards.
Stem cell research is rapidly evolving, holding great therapeutic potential for the treatment of many diseases that were once considered incurable. Research on stem cells has given rise to a great number of discoveries; the improvement and reconstruction of damaged cells or tissues by stem cells are the major findings reported in scientific literature. More impressively, gene therapy or organ development using stem cells is a promising future medical perspective.
On this basis, stem cell banking becomes more and more important, which means a greater focus must be placed on the ethics surrounding it. Crucially, informed consent should be obtained before stem cell collection, all medical benefits and/or clinical trials status must be clearly explained to patients, and the quality, traceability, and safety of the cryopreserved samples must be guaranteed. In this regard, a FACT-NetCord accredited bank makes all the difference.