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Stem Cell Treatments

In this article about stem cell treatments, we will explore a number of questions that are posed by people today about the use of stem cells and what treatments currently exist to help with the treatment and cure of many of the diseases that populate our world. This article also includes information on obstacles that need to be overcome so that stem cell therapies can be made available and much more.

Are Stem Cell Treatments Currently Used in Therapies Today ?

Bone marrow transplants which are also known as hematopoietic stem cell transplants are currently used. This is a medical procedure that is used to treat blood conditions such as leukemia, sickle cell anemia and some metabolic conditions. This procedure relies on the hematopoietic (blood) stem cells that are present in the bone marrow. These are the precursors to all blood cells. Doctors have been transferring blood stem cells by bone marrow transplant for more than forty years. Advanced techniques for collection or “harvesting” blood stem cells are now used. Cord blood, like bone marrow, is stored as a source of blood stem cells and is used as an alternative to bone marrow in some transplants.

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Other stem cell applications are the use of skin progenitor cells for burns and the use of limbal stem cells, which can be found in the cornea, for corneal injuries. Despite intensive research, there are no therapies available as yet that use embryonic stem cells, although two clinical trials were approved in 2010. There were trials to treat spinal cord injury and a certain type of blindness.

With the exception of the treatments mentioned above, the use of stem cell therapies still remains at an experimental stage and has not been shown to be safe or effective.

What Obstacles Must Be Overcome So Stem Cells Therapies Can Be Realized ?

Some of the promise of stem cell therapy has already been realized. An example of this is bone marrow transplantation. Even with this, there are still a number of problems that need to be solved. Challenges that stem cell therapies face include :

Adult Stem Cells: Tissue-specific stem cells in adults tend to be rare. Whilst these cells can regenerate themselves in animals and in people, they are very difficult to grow and expand in the laboratory. Because of this, it is difficult to obtain sufficient numbers of many of the different types of adult stem cells for study and clinical use. Hematopoietic stem cells in the bone marrow, for example, only make up one in a hundred thousand cells of the bone marrow. These can be isolated, but can only be expanded a very limited amount in the laboratory. Fortunately, large numbers of whole bone marrow cells can be isolated and administered for the treatment of a number of blood diseases.

Skin stem cells can be expanded and these are used to treat burns. There has been some success achieved in expanding some of the other types of stem cells and these include mesenchymal stem cells. The application of these in animals has however been difficult. One major problem is the mode of administration. Bone marrow cells can be infused in the blood stream and will then find their way to the bone marrow. For other stem cells, such as muscle stem cells, mesenchymal stem cells and neural stem cells, the route of administration in humans in more problematic. It is believed, however, that once healthy stem cells find their niche, they will start repairing the tissue.

In another approach scientists attempt to differentiate stem cells into functional tissue and this is then transplanted.

The final problem is rejection. If stem cells from the patient are used, reject by the immune system is not a problem. If donor stem cells are used, the immune system of the recipient can reject the cells unless the immune system is suppressed by drugs. In the case of bone marrow transplantation there is another problem. The bone marrow contains immune cells from the donor and these can attach the tissues of the patient and this can cause graft-versus-host disease.

Pluripotent Stem Cells: All embryonic stem cell lines are derived from very early stage embryos and will therefore be genetically different from any patient. This means that immune rejection is a major problem. For this reason, induced pluripotent stem cells are a major breakthrough as they won’t be rejected. IPS cells are generated from the cells of the patient through a process of reprogramming. A problem with these is that a lot of iPS cell lines are generated by insertion of genes using viruses and this carries the risk of transformation into cancer cells. Furthermore, undifferentiated embryonic stem cells and iPS cells for tumors when transplanted into mice. This means that cells derived from embryonic stem cells or iPS cells have to be devoid of the original stem cells to avoid tumor formation. This is a major safety concern.

A second challenge is differentiation of pluripotent cells into cells or tissues that are functional in an adult patient and that meet the standards that are required for transplantation grade tissues and cells.

A major advantage to using pluripotent cells is that they can be grown and expanded indefinitely in the laboratory. This means that cells numbers are less of a limiting factor. Another advantage is that given their very broad potential, several cell types that are present in an organ might be generated. Sophisticated tissue engineering approaches are now being developed and this could allow scientists and researchers to reconstruct organs in the laboratory.

The results from animal models are promising but research on stem cells and their applications to treat various human diseases is still at an early stage. As with any medical treatment, a rigorous research and testing process must be followed to ensure long term efficacy and safety.

What is an Experimental Treatment ?

An experimental treatment is one that is still being developed and has not yet been shown in careful clinical trials to be safe and effective. Only a few stem cell treatments, such as bone marrow transplantation to treat blood diseases are widely accepted by the medical community.

What is a Clinical Trial ?

A clinical trial is a research study that has been designed to answer specific questions about a new treatment or a new way of using current treatments. Clinical trials are used to establish whether new treatments are safe and effective. It is very important to understand that the new treatment may not be better than, or even as good as, existing treatments.

Most drugs and treatments that are widely accepted by the medical community have been tested in clinical trials. If a trial is successful and shown to be safer and more effective or cheaper than previously available treatments, it can become a standard treatment.

What the difference between a clinical trial and an experimental procedure ?

The fact that a procedure is experimental does not mean that it is part of a research study or clinical trial. A responsible clinical trial can be characterized by a number of key features. The first of these is preclinical data which is data obtained in the laboratory that was taken from animal models. This data supports the conclusion that the treatment that is being studies is likely to be safe and effective. The next thing that is required is oversight by an independent group. In many countries the trial is assessed and approved by a national regulatory agency, such as the European Medicines Agency (EMA) or the U.S. Food and Drug Administration (FDA).

The trial is designed to answer specific questions about a new treatment or a new way of using current treatments. This is often done with a control group to which the group of people receiving the new treatment is compared. Typically the cost of the new treatment and trial monitoring is defrayed by the company developing the treatment or by local or national government funding.

Responsibly conducted clinical trials are critical in the development of new treatments as they allow us to learn whether these treatments are safe and effective.

Are Cancer Cell Treatments Dangerous or Can Stem Cells Cause Cancer ?

Every medical procedure has risks. The goal of clinical trials is to determine whether the potential benefit of the treatment outweighs the risks. A possible risk of some stem cell treatments may be the development of tumors or cancers. For example, when cells are grown in culture, the cells may lose the normal mechanisms that control growth. A particular danger of pluripotent cells is that if undifferentiated they may form tumors called teratomas. Other possible risks include infection, tissue rejection and complications arising from the procedure itself.

Are Treatments Using My Own Stem Cells (autologous) Safe ?

While your own cells are less likely to be rejected by your immune system, this does not mean that the cells are safe to use as a therapeutic treatment. The methods used to isolate, modify, grow or transplant the cells could alter the cells, cause infection or introduce other unknown risks to the body. Transplanting cells into a different part of the body other than where they originated from may have unforeseen risk, complications or unpredictable outcomes.

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