For Patients
This section looks in depth at how stem cells reveal the secrets of some of the most baffling diseases and how the special properties of stem cells might be harnessed to treat and even cure disease.
The therapeutic potential of stem cells has so profoundly changed the landscape of medical research that it has spawned an entirely new field – regenerative medicine. Particularly exciting developments have been the discovery of stem cells in virtually every organ in the body and the discovery that skin cells can be reprogrammed to a pluripotent state. Both of these discoveries open new possibilities for therapeutic treatment.
There is indeed a great deal of promise, but also a tendency to think that a cure is just around the corner. The path from research to the clinic is often long and complicated, with many small advances. Our aim is to present an objective view of the most important advances, even though these are being overtaken continuously by new research. These articles will give the reader a foundational knowledge against which to read and evaluate new research as it appears in the media.
Generally, it is helpful to understand four different approaches to using stem cells for clinical benefit, in addition to their still critical role in understanding the disease processes:
Stem cell transplantation
Stem cell transplantation exploits the special characteristics of stem cells to multiply indefinitely and to become other cells required by the body. For example, stem cells that reside in the bone marrow or are plentiful in cord blood are removed and used to generate new blood cells. These are then transplanted back into the body, thereby restoring a patient’s immune system and capacity to fight disease.
Cellular therapy
Cellular therapy relies on stem cells’ ability to regenerate indefinitely and to evolve from one kind of cell to another based on elaborate codes and cues. Stem cells residing in our own tissues may be re-programmed, or new ones manufactured artificially through therapeutic cloning, to replace the cells damaged by disease or injury. Then, these can be generated in large enough quantities for transplantation.
Tissue regeneration
While stem cells help scientists to understand the mechanisms and pathways that initiate and perpetuate disease, it is now evident that they can also be mobilized to reverse this process. By manipulating growth factors that enhance stem cell activity and silencing the genes that drive disease, scientists are already demonstrating that stem cells will be able to regenerate tissue and restore lost function.
Drug discovery
By manipulating stem cells, scientists are now able to re-create human disease in animal models. Furthermore, they can isolate and multiply the cells that are responsible for the disease process and decode the critical messages. Not only is this leading to the discovery of new drugs, but new technology allows scientists to generate large quantities of patient-specific cells on which to test them.
The articles go into considerable detail about the most promising approaches for each disease. You may wish to consult the section on stem cell basics to gain familiarity with the concepts and terminology that are used in presenting the latest research specific to each disease.
Although there is occasional reference to clinical trials that are envisioned or underway, we neither endorse nor evaluate these trials. You may wish to refer to the FAQ section for more information on clinical trials or consult the Patient Handbook on Stem Cell Therapies, published by the International Society of Stem Cell Research.