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  • Writer's pictureSaeed Anwar Anuj

Stem cell therapies: a brief overview

Updated: Feb 23, 2023

Stem cell therapy is a rapidly evolving medical field that can potentially treat a wide range of diseases and injuries. Stem cells are special cells that have the unique ability to develop into many different types of cells in the body, and they can also self-renew and divide, making them an attractive option for repairing damaged tissue.

There are several types of stem cells, but the most commonly used in therapy are adult stem cells, embryonic stem cells, perinatal stem cells, and induced pluripotent stem cells (iPSCs). Adult stem cells, generally referred to as bone marrow cells, are found in various tissues in the body and can be harvested from a patient's bone marrow, fat, or blood. These are multipotent (potentially becoming any of several cell types), meaning they can differentiate into a limited number of cell types. On the other hand, embryonic stem cells are derived from human embryos and are highly versatile, as they can potentially become any cell type in the body. However, they present some ethical issues due to where they come from. In addition, we have perinatal stem cells, also known as cord blood cells, which can be harvested from the umbilical cord and placenta after a baby has been born and the cord has been cut. They are more 'powerful' than multipotent stem cells as they are pluripotent, meaning they can form all of the cell types that make up the body. Further to these three types, artificially made stem cells are also there. iPSCs are a special type of stem cell that scientists can create in the lab by taking ordinary cells from your body, like skin cells or blood cells, and reprogramming them to become stem cells. These new stem cells are called "pluripotent" because they can turn into many different types of cells in the body, like muscle cells, nerve cells, or heart cells. iPSCs are essential because they can be used to create new cells that can replace damaged or diseased cells in the body, and because they can be made from a patient's own cells, they can potentially be used to create personalized treatments for diseases.

Stem cell therapy has shown tremendous promise in treating various medical conditions, from spinal cord injuries and diabetes to cancer and heart disease. They may also be effective in treating genetic disorders like thalassemia, progeria, and muscular dystrophy, as well as neurological conditions such as Alzheimer's disease, multiple sclerosis, amyotrophic lateral sclerosis, and Parkinson's disease. Some stem cell therapies are already being used to treat various medical conditions, such as bone marrow transplants that use hematopoietic stem cells to treat blood and immune system disorders and CAR T-cell therapy that utilizes genetically modified T-cells to treat some types of cancer.

The potential of stem cell therapy in treating genetic diseases like Duchenne muscular dystrophy (DMD) and progeria is exciting. DMD is a rare genetic disorder that causes progressive muscle weakness and loss of muscle mass, primarily in boys. DMD occurs due to a lack of dystrophin, a protein that maintains the integrity of muscle cells. Stem cell therapy offers a potential solution for DMD patients by using a type of stem cell called myoblasts. In this strategy, researchers genetically engineer myoblasts to express dystrophin. These engineered cells are then transplanted into a patient's body, where they are expected to differentiate into functional muscle cells (myotubes). In studies using animal models of DMD, this approach was found effective in replacing damaged muscle cells and improving muscle function. iPSCs, on the other hand, have also shown potential in treating DMD. These stem cells can differentiate into muscle cells and can be used to replace damaged muscle cells in individuals with DMD. Moreover, the cells can be derived from the patient's body, reducing the risk of rejection by the immune system. Researchers are also investigating CRISPR/Cas9 gene editing technology to correct the genetic mutation that causes DMD in iPSCs before they are used for transplantation. Although myoblasts have shown promise in replacing damaged muscle cells in animal models of DMD, and iPSCs have been used to create healthy muscle cells for transplantation in DMD patients, more research is needed to determine the safety and effectiveness of these approaches before they become widely available as treatments for DMD.

Progeria is another rare genetic disorder that causes premature aging in children. This disorder results from a mutation in the LMNA gene, which affects the production of lamin A, a protein that leads to the buildup of abnormal proteins in the nucleus of cells. Researchers have been able to create iPSCs from the skin cells of a progeria patient and differentiate them into smooth muscle cells, which were transplanted into the patient's blood vessels. This approach can address several symptoms of progeria, including bone loss and stiffness, and improve blood flow. Additionally, mesenchymal stem cells (MSCs) can be harvested from a healthy donor's bone marrow and transplanted into the patient. MSCs have anti-inflammatory and immunomodulatory effects and can promote tissue repair and regeneration. While iPSCs have shown success in producing healthy cells for transplantation in progeria patients, and MSCs have shown promise in promoting tissue repair and regeneration, more research is needed to determine the safety and effectiveness of these approaches before they can be widely used as treatments for progeria.

Stem cell therapy can be administered in various ways depending on the nature of the stem cells used and the disease being treated. One common method involves directly injecting stem cells into the affected area, allowing them to act locally and regenerate damaged or diseased tissue. Alternatively, stem cells can be injected into the bloodstream, allowing them to travel throughout the body to the site of damage. In some cases, stem cells are grown in the lab under specific conditions to produce many cells before being transplanted into the patient, providing a concentrated source of cells for maximum therapeutic benefit. These different modes of administration enable stem cell therapy to be tailored to the needs of each patient and the specific disease or condition being treated.

Despite its potential, stem cell therapy is not without risks. Some stem cell therapies have been associated with the development of tumors and other unwanted side effects, particularly in cases where embryonic stem cells have been used. These risks highlight the need for continued research and careful evaluation of the safety and effectiveness of stem cell therapies. In addition to the potential risks, the cost of stem cell therapy can also be a significant barrier to its widespread use, making it inaccessible to many patients. The high cost of stem cell therapy is often due to the complex and time-consuming process involved in deriving, growing, and administering the cells, as well as the need for specialized equipment and highly trained medical professionals. As a result, many patients who could benefit from stem cell therapy may not have access to it.

Ensuring the safety and efficacy of stem cell therapy is crucial, and only competent medical professionals in regulated settings should carry out the therapy. In the United States, the Food and Drug Administration (FDA) closely supervises stem cell therapies. It imposes stringent regulations to guarantee their safety and efficacy. Similarly, the European Medicines Agency (EMA) has also put in place regulations governing the use of stem cells in medical treatment, requiring developers to demonstrate the safety and efficacy of their treatments before they can be approved for use. In India, the government has established guidelines for the practice of stem cell therapy, which require practitioners to register and adhere to safety standards. Stem cell therapy is also an active area of research and development in other Asian countries, including China, Japan, and South Korea. In all these countries, regulations are in place to safeguard patients and ensure that they receive the best possible care. It is critical that patients only receive stem cell therapy from reliable healthcare institutions with qualified medical professionals who can guide them through the process and answer any questions they may have. By adhering to these guidelines, patients can benefit from the potential of stem cell therapy while minimizing the risks. While regulations and guidelines may vary across regions, there is a growing recognition of the need for standards and oversight to ensure the safety and efficacy of stem cell therapy.

The field of stem cell research is constantly evolving, with new advances and discoveries being made regularly. Researchers are exploring new applications for stem cell therapy and developing new techniques for improving the safety and effectiveness of the therapy. While there is still much to be learned, the potential of stem cell therapy is significant and continues to be an active area of research.

15 August 2022

Saeed Anwar

Edmonton, Canada

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