Stem cells have the potential to treat a wide range of diseases. Find out here why these cells are such a powerful tool for treating diseases and what obstacles experts face before new therapies reach patients. Stem cells are a type of cell that can develop in different ways to form every organ in the body, from bones, kidneys, and liver to blood and brain. Specialized types of stem cells have the ability to stop immune responses.
Therefore, stem cells can be very useful as therapy for diseases in which organs are damaged or where the immune system is too active. Some types of stem cells are already used for therapy, such as hematopoietic (blood) stem cells, which are used to treat bone marrow cancer. The use of other types of stem cells is currently being studied in the laboratory and in experimental therapies. Researchers are trying to find the best way to give stem cells to patients, where do cells go in the body and how long they survive in the patient.
We hope that many more stem cell therapies will be available in the future. Researchers hope that stem cells will one day be effective in treating many medical conditions and diseases. However, treatments with unproven stem cells can be unsafe, so be aware of all the facts if you are considering treatment. We offer diagnostic and treatment options for common and complex medical conditions.
At Mass General, the brightest minds in medicine collaborate on behalf of our patients to unite the science of innovation with cutting-edge clinical medicine. The Center for Regenerative Medicine is dedicated to understanding how tissues form and can be repaired. Our main goal is to develop novel therapies to regenerate damaged tissues and overcome debilitating chronic diseases. Adult stem cells are thought to exist in all types of tissue in the body.
However, to date, the isolation of many types of adult stem cells has been limited. Hematopoietic (blood) stem cells are readily available by bone marrow aspiration. However, stem cells from solid organs such as the liver or brain have been shown to be more difficult to identify and derive. The hope is that hESCs can be used to derive all types of adult stem cells in the body and allow research that is currently not possible.
At this point, the promise is enormous, but research on hESC is still in its early stages. Research on human embryonic stem cells (hESCs) only began in 1998, when a group led by Dr. James Thomson, from the University of Wisconsin, developed a technique to isolate and grow cells. Induced pluripotent stem cells (iPS cells) are cells that began as normal adult cells (for example, a skin cell) and that scientists designed (“induced”) to become pluripotent, that is, capable of forming all types of cells in the body.
This process is often referred to as “rescheduling”. Scientists are currently exploring whether they differ in clinically significant ways. The technology used to generate iPS cells holds great promise for creating patient- and disease-specific cell lines for research purposes. These cells are already useful tools for drug development and scientists hope to use them in transplant medicine.
However, additional research is needed before reprogramming techniques can be used to generate stem cells suitable for safe and effective therapies. Somatic cell nuclear transfer (SCNT) is a technique in which the nucleus of a somatic cell (any cell in the body except sperm and eggs) is injected or transplanted into an egg that has had its nucleus removed. The SCNT product has the same genetic material as the somatic cell donor. SCNT is a cloning technique.
The product of the SCNT is almost genetically identical to the somatic cell used in the process. It should be noted that the product of SCNT is not technically 100% identical, since the cytoplasm of the oocyte includes mitochondrial DNA. The following FAQs address these differences. Reproductive cloning includes placing the SCNT product in the uterus for the purpose of giving birth.
The resulting organism would, in theory, be the genetic copy of the somatic cell donor. Reproductive cloning has been carried out on animals for many years and is burdened by many technical and biological problems. Only about 1 percent of all eggs that receive donor DNA can become normal surviving clones. Therapeutic cloning uses SCNT for the sole purpose of deriving cells for research and potentially, in the future, for therapy.
In therapeutic cloning, the SCNT product is not placed in the uterus and, therefore, a live birth is never possible. Therapeutic cloning provides two potential benefits. Research on human embryonic stem cells at the Center for Regenerative Medicine has been supported in part by private philanthropic donations. These grants allowed us to support a wide range of research activities that could not have been supported by other sources, such as NIH funding.
In the future, we hope to receive support from NIH and other funding agencies for eligible activities. The Center for Regenerative Medicine is dedicated to understanding how tissues form and how they can be repaired in injury environments. Integrated into the Mass General Hospital, the main objective of the Center is to develop novel therapies to regenerate damaged tissues and, therefore, overcome chronic debilitating disease. The success of this effort requires a cohesive team of scientists and doctors with diverse areas of expertise, but with a shared mission and dedication to the broader goal.
Adult stem cells are already used in the treatment of genetic diseases. Bone marrow transplants and the hematopoietic stem cells it contains are used to treat genetic and acquired diseases of the blood and immune system. New developments include genetic engineering of hematopoietic stem cells to cure some genetic diseases. Other adult stem cells are being explored in preclinical animal models for transplant therapy in a variety of genetic diseases, for which the development of cellular models is an important potential application.
Stem cells can be obtained from patients with a known diagnosis and indicated genetic mutation. With these, and the cells derived from them, the genetic and biochemical pathways altered by the disease can be investigated and compared with cells from healthy controls. When stem cell products are used in unapproved ways or when processed in ways that are more than minimally manipulated, which relates to the nature and degree of processing, the FDA can (and has already taken) a variety of administrative and judicial actions, including criminal enforcement, according to the violations involved. Some clinics may also falsely announce that there is no need for FDA review and approval of stem cell therapy.
In addition, retinal regeneration with stem cells isolated from the eyes may lead to a possible cure for damaged or diseased eyes and may one day help to reverse blindness. Immune rejection is unlikely when adult stem cells are derived from the same patient (an autologous procedure). The standard technique for creating iPS cells uses viruses to transfer reprogramming genes to skin cells. Blood and bone marrow stem cells are currently the main type of stem cells commonly used for therapy.
Because each cell line has its own genetic fingerprint, researchers are often interested in using the same cell line for a series of related experiments. The only stem cell-based products that are FDA-approved for use in the United States consist of blood-forming stem cells (hematopoietic progenitor cells) derived from cord blood. Learn how stem cell research funded by CIRM could lead to treatments for many chronic diseases and injuries. In recent years, stem cells have been used as a powerful tool to establish models of patient-derived diseases, both to understand the molecular basis of disorders and to use them in drug development (on a plate).
As mentioned above, embryonic stem cells are present in very young embryos one week old, to be exact. A research team led by University of Central Florida professor Kiminobu Sugaya found that treating bone marrow cells in laboratory cultures with bromodeoxyuridine, a compound that becomes part of DNA, increases the likelihood that adult human stem cells will develop as cells after implantation into the brains of adult rats. At the moment, there are still doubts about the safety of such treatments, but some initial tests have been carried out in humans to repair damaged eyes with cells derived from embryonic stem cells. .