At the age of 34, Maria was advised by her doctor to have a bone marrow transplant. Her leukaemia had come back following chemotherapy, and she was scared of the word transplant, which hematopoietic stem cells. Her medical staff told her that the natural repair system of the body is the stem cells- the cells that can repair themselves and change into nearly all kinds of specialised cells. In her situation, such cells would reconstruct her immune system on the basis of the foundations. She had her younger brother as a perfect match, and this is why the stem cells were successfully transplanted. Maria is now cancer-free and this is a demonstration of the life-saving qualities of stem cell therapy.
Stem cells have 2 characteristic properties: self-renewal, which is a property of cell division with all divisions occurring, and differentiation, which is the ability of the cell to be developed into specialised cells like blood, nerve or muscle cells. These are the aspects that make them the pillars of regenerative medicine. In addition to the use of blood cancers using hematopoietic stem cell transplantation (HSCT), researchers are finding ways to use stem cells in the repair of spinal cord injuries, insulin-producing cells in diabetes, and in repairing damaged cartilage.
Stem Cell Applications in Regenerative Medicine
The goal of regenerative medicine is to restore or replace damaged tissues and organs. Stem cells serve as repair kits on demand, and as such, they are known to migrate to the injury site and divide and differentiate depending on the need. The most recognised is HSCT, which is currently in regular application in curing leukaemia, lymphoma and genetic blood disorders. It is being tested in clinical trials for wider uses in neurological, cardiac and autoimmune illnesses.
Types of Stem Cells
Stem cells are different in genesis and strength:
- Embryonic stem cells (ESCs) are a type of pluripotent cell that can differentiate into any type of cell, yet their application is ethically questionable and has a risk of tumour development.
- Multipotent, ethically unproblematic, but with lower differentiation capabilities, are adult stem cells (ASCs) (hematopoietic and mesenchymal stem cells).
- The induced pluripotent stem cells (iPSCs) are cells in adults that have been reprogrammed to act as ESCs without being associated with ethics, yet they are considered to be experimental.
Hematopoietic stem cells, which today are only approved to be used in bone marrow, peripheral blood, or cord blood, are used in routine clinical practice.
Hematopoietic Stem Cell Transplantation (HSCT)
HSCT is used in the treatment of leukaemia, lymphoma, multiple myeloma, sickle cell disease, thalassemia, aplastic anaemia and immunodeficiencies by replacement of the diseased bone marrow with healthy stem cells. The findings depend upon the nature of the disease, the health of the patient and compatibility between the donor and recipient.
Transplantation Autologous vs. Allogeneic
In autologous HSCT, the patient is subjected to the use of her or his cells. It averts the development of graft-versus-host disease (GVHD) and is prevalent with multiple myeloma and certain lymphomas, but is more likely to recur because the transfused cells are likely to include cancer cells.
Allogeneic HSCT involves donor cells and has the advantage of the graft-versus-leukaemia (GVL) effect, wherein the immune cells of the donor assault leftover cancer. Nevertheless, it will present the risk of GVHD and necessitate an HLA-compatible donor.
Donor Matching and Pre-Transplant Evaluation
Applicants are also subjected to rigorous examinations in order to monitor their ability to withstand the process, such as organ scrutiny, infection check-up and psychosocial preparation. Preparation also includes fertility preservation and vaccination review. The donors could be the identical or non-identical brothers, non-identical volunteers, haploidentical relatives, or cord blood units. HLA typing makes it compatible and minimises the risk of GVHD.
Harvesting, Processing and Infusion
The stem cells are obtained by either:
- Bone marrow is anesthetically harvested, or
- Blood stem cell (PBSC), after growth factor (e.g. G-CSF) mobilisation.
To verify sterility and viability, the collectors obtained from the collectors are treated in GMP-compliant labs. The stem cells are then injected through intravenous injection after conditioning chemotherapy or radiation, and move to the bone marrow, where engraftment starts.
Engraftment and Outcomes
The engraftment process is generally two to four weeks, with an increase in the neutrophil and platelet counts. Positional care/supportive care, such as antimicrobials, transfusion and nutritional support, is essential in this susceptible period. The side effects in the long-term are GVHD, infections, infertility, and secondary cancers, yet HSCT in many cases provides the best opportunity of cure.
Next Generation Stem Cell Applications
In addition to HSCT, there is research to use stem cells in stroke recovery, heart failure, diabetes and cartilage repair. Mesenchymal stem cells, iPSCs, and tissue-specific progenitors are under trial and yet these therapies are yet to be investigated. Such treatments should only be sought in controlled clinical trials and not in the commercial stem cell clinics that provide an unproven cure.
Regenerative Medicine Safety of Participation
It is also the responsibility of the patients to ensure that any therapy being suggested is a trial that has been endorsed by an IRB with clear protocols, GMP-manufactured products, and qualified experts. It is necessary to ask about evidence, risks, and alternatives as well as long-term follow-up requirements.
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