What are stem cells?
Stem cells are the body's raw materials — cells from which all other cells with specialized functions are generated. Under the right conditions in the body or a laboratory, stem cells divide to form more cells called daughter cells.
These daughter cells either become new stem cells (self-renewal) or become specialized cells (differentiation) with a more specific function, such as blood cells, brain cells, heart muscle or bone. No other cell in the body has the natural ability to generate new cell types.
Stem cells have the remarkable potential to develop into many different cell types in the body during early life and growth. In addition, in many tissues they serve as a sort of internal repair system, dividing essentially without limit to replenish other cells as long as the person or animal is still alive. When a stem cell divides, each new cell has the potential either to remain a stem cell or become another type of cell with a more specialized function, such as a muscle cell, a red blood cell, or a brain cell.
Stem cells are distinguished from other cell types by two important characteristics. First, they are unspecialized cells capable of renewing themselves through cell division, sometimes after long periods of inactivity. Second, under certain physiologic or experimental conditions, they can be induced to become tissue- or organ-specific cells with special functions. In some organs, such as the gut and bone marrow, stem cells regularly divide to repair and replace worn out or damaged tissues. In other organs, however, such as the pancreas and the heart, stem cells only divide under special conditions.
Stem cells are the body's raw materials — cells from which all other cells with specialized functions are generated. Under the right conditions in the body or a laboratory, stem cells divide to form more cells called daughter cells.
These daughter cells either become new stem cells (self-renewal) or become specialized cells (differentiation) with a more specific function, such as blood cells, brain cells, heart muscle or bone. No other cell in the body has the natural ability to generate new cell types.
Stem cells have the remarkable potential to develop into many different cell types in the body during early life and growth. In addition, in many tissues they serve as a sort of internal repair system, dividing essentially without limit to replenish other cells as long as the person or animal is still alive. When a stem cell divides, each new cell has the potential either to remain a stem cell or become another type of cell with a more specialized function, such as a muscle cell, a red blood cell, or a brain cell.
Stem cells are distinguished from other cell types by two important characteristics. First, they are unspecialized cells capable of renewing themselves through cell division, sometimes after long periods of inactivity. Second, under certain physiologic or experimental conditions, they can be induced to become tissue- or organ-specific cells with special functions. In some organs, such as the gut and bone marrow, stem cells regularly divide to repair and replace worn out or damaged tissues. In other organs, however, such as the pancreas and the heart, stem cells only divide under special conditions.
Stem cells are a renewable source of tissue that can be coaxed to become different cell types of the body. The best-known examples are the embryonic stem (ES) cells found within an early-stage embryo. These cells can generate all the major cell types of the body (they are “pluripotent”). Stem cells have also been isolated from various other tissues, including bone marrow, muscle, heart, gut and even the brain. These “adult” stem cells help with maintenance and repair by becoming specialized cells types of the tissue or organ where they originate. For example, special stem cells in the bone marrow give rise to all the various types of blood cells (similar blood cell-forming stem cells have also been isolated from umbilical cord blood).
Adult vs. embryonic stem cells Because adult stem cells become more committed to a particular tissue type during development, unlike embryonic stem cells, they appear to only develop into a limited number of cell types (they are “multipotent”).
What are induced pluripotent stem cells? In addition to ES cells, induced pluripotent stem (iPS) cells, discovered in 2007, represent an important development in stem cell research to treat diseases like Parkinson’s disease. Essentially, iPS cells are ”man-made” stem cells that share ES cells' ability to become other cell types. IPS cells are created when scientists convert or "reprogram" a mature cell, such as a skin cell, into an embryonic-like state. These cells may have potential both for cell replacement treatment approaches in patients and as disease models that scientists could use in screening new drugs.
IPS cell technology is somewhat related to a previous method called somatic cell nuclear transfer (SCNT) or “therapeutic cloning” (the technology that gave us Dolly the Sheep). Unlike the iPS cell approach, which converts adult cells directly into stem cells, SCNT involves transferring the genetic material of an adult cell into an unfertilized human egg cell, allowing the egg cell to form an early-stage embryo and then collecting its ES cells (which are now genetic “clones” of the person who donated the adult cell). To date, however, this has not been successfully demonstrated with human cells and iPS cell methods may be replacing SCNT as a more viable option.
A potentially exciting use for iPS cells is the development of cell models of Parkinson’s disease. In theory, scientists could use cells from people living with Parkinson’s disease to create iPS cell models of the disease that have the same intrinsic cellular machinery of a Parkinson’s patient. Researchers could use these cell models to evaluate genetic and environmental factors implicated in Parkinson’s disease.
Stem cells: What they are and what they do. Stem cells and derived products offer great promise for new medical treatments. Learn about stem cell types, current and possible uses, ethical issues, and the state of research and practice.
By Mayo Clinic Staff
You've heard about stem cells in the news, and perhaps you've wondered if they might help you or a loved one with a serious disease. You may wonder what stem cells are, how they're being used to treat disease and injury, and why they're the subject of such vigorous debate.
Here are some answers to frequently asked questions about stem cells.
Why is there such an interest in stem cells?
Researchers and doctors hope stem cell studies can help to:
Where do stem cells come from?
Researchers have discovered several sources of stem cells:
Embryonic stem cells are obtained from early-stage embryos — a group of cells that forms when a woman's egg is fertilized with a man's sperm in an in vitro fertilization clinic. Because human embryonic stem cells are extracted from human embryos, several questions and issues have been raised about the ethics of embryonic stem cell research.
The National Institutes of Health created guidelines for human stem cell research in 2009. Guidelines included defining embryonic stem cells and how they may be used in research and donation guidelines for embryonic stem cells. Also, guidelines stated embryonic stem cells may only be used from embryos created by in vitro fertilization when the embryo is no longer needed.
Where do these embryos come from?
The embryos being used in embryonic stem cell research come from eggs that were fertilized at in vitro fertilization clinics but never implanted in a woman's uterus. The stem cells are donated with informed consent from donors. The stem cells can live and grow in special solutions in test tubes or petri dishes in laboratories.
Why can't researchers use adult stem cells instead?
Although research into adult stem cells is promising, adult stem cells may not be as versatile and durable as are embryonic stem cells. Adult stem cells may not be able to be manipulated to produce all cell types, which limits how adult stem cells can be used to treat diseases.
Adult stem cells also are more likely to contain abnormalities due to environmental hazards, such as toxins, or from errors acquired by the cells during replication. However, researchers have found that adult stem cells are more adaptable than was initially suspected.
What are stem cell lines and why do researchers want to use them?
A stem cell line is a group of cells that all descend from a single original stem cell and is grown in a lab. Cells in a stem cell line keep growing but don't differentiate into specialized cells. Ideally, they remain free of genetic defects and continue to create more stem cells. Clusters of cells can be taken from a stem cell line and frozen for storage or shared with other researchers.
What is stem cell therapy (regenerative medicine), and how does it work?
Stem cell therapy, also known as regenerative medicine, promotes the reparative response of diseased, dysfunctional or injured tissue using stem cells or their derivatives. It is the next chapter of organ transplantation and uses cells instead of donor organs, which are limited in supply.
Researchers grow stem cells in a lab. These stem cells are manipulated to specialize into specific types of cells, such as heart muscle cells, blood cells or nerve cells.
The specialized cells can then be implanted into a person. For example, if the person has heart disease, the cells could be injected into the heart muscle. The healthy transplanted heart cells could then contribute to repairing defective heart muscle.
Researchers have already shown that adult bone marrow cells guided to become heart-like cells can repair heart tissue in people, and more research is ongoing.
Have stem cells already been used to treat diseases?
Yes, doctors have performed stem cell transplants, also known as bone marrow transplants. In stem cell transplants, stem cells replace cells damaged by chemotherapy or disease or as a way for the donor's immune system to fight some types of cancer and blood-related diseases, such as leukemia. These transplants use adult stem cells or umbilical cord blood.
Researchers are testing adult stem cells to treat other conditions, including a number of degenerative diseases such as heart failure.
What are the potential problems with using embryonic stem cells in humans?
To be useful in people, researchers must be certain that stem cells will differentiate into the specific cell types desired.
Adult vs. embryonic stem cells Because adult stem cells become more committed to a particular tissue type during development, unlike embryonic stem cells, they appear to only develop into a limited number of cell types (they are “multipotent”).
What are induced pluripotent stem cells? In addition to ES cells, induced pluripotent stem (iPS) cells, discovered in 2007, represent an important development in stem cell research to treat diseases like Parkinson’s disease. Essentially, iPS cells are ”man-made” stem cells that share ES cells' ability to become other cell types. IPS cells are created when scientists convert or "reprogram" a mature cell, such as a skin cell, into an embryonic-like state. These cells may have potential both for cell replacement treatment approaches in patients and as disease models that scientists could use in screening new drugs.
IPS cell technology is somewhat related to a previous method called somatic cell nuclear transfer (SCNT) or “therapeutic cloning” (the technology that gave us Dolly the Sheep). Unlike the iPS cell approach, which converts adult cells directly into stem cells, SCNT involves transferring the genetic material of an adult cell into an unfertilized human egg cell, allowing the egg cell to form an early-stage embryo and then collecting its ES cells (which are now genetic “clones” of the person who donated the adult cell). To date, however, this has not been successfully demonstrated with human cells and iPS cell methods may be replacing SCNT as a more viable option.
A potentially exciting use for iPS cells is the development of cell models of Parkinson’s disease. In theory, scientists could use cells from people living with Parkinson’s disease to create iPS cell models of the disease that have the same intrinsic cellular machinery of a Parkinson’s patient. Researchers could use these cell models to evaluate genetic and environmental factors implicated in Parkinson’s disease.
Stem cells: What they are and what they do. Stem cells and derived products offer great promise for new medical treatments. Learn about stem cell types, current and possible uses, ethical issues, and the state of research and practice.
By Mayo Clinic Staff
You've heard about stem cells in the news, and perhaps you've wondered if they might help you or a loved one with a serious disease. You may wonder what stem cells are, how they're being used to treat disease and injury, and why they're the subject of such vigorous debate.
Here are some answers to frequently asked questions about stem cells.
Why is there such an interest in stem cells?
Researchers and doctors hope stem cell studies can help to:
- Increase understanding of how diseases occur. By watching stem cells mature into cells in bones, heart muscle, nerves, and other organs and tissue, researchers and doctors may better understand how diseases and conditions develop.
- Generate healthy cells to replace diseased cells (regenerative medicine). Stem cells can be guided into becoming specific cells that can be used to regenerate and repair diseased or damaged tissues in people.
People who might benefit from stem cell therapies include those with spinal cord injuries, type 1 diabetes, Parkinson's disease, Alzheimer's disease, heart disease, stroke, burns, cancer and osteoarthritis.
Stem cells may have the potential to be grown to become new tissue for use in transplant and regenerative medicine. Researchers continue to advance the knowledge on stem cells and their applications in transplant and regenerative medicine. - Test new drugs for safety and effectiveness. Before using new drugs in people, some types of stem cells are useful to test the safety and quality of investigational drugs. This type of testing will most likely first have a direct impact on drug development for cardiac toxicity testing.
New areas of study include the effectiveness of using human stem cells that have been programmed into tissue-specific cells to test new drugs. For testing of new drugs to be accurate, the cells must be programmed to acquire properties of the type of cells to be tested. Techniques to program cells into specific cells continue to be studied.
For instance, nerve cells could be generated to test a new drug for a nerve disease. Tests could show whether the new drug had any effect on the cells and whether the cells were harmed.
Where do stem cells come from?
Researchers have discovered several sources of stem cells:
- Embryonic stem cells. These stem cells come from embryos that are three to five days old. At this stage, an embryo is called a blastocyst and has about 150 cells.
These are pluripotent (ploo-RIP-uh-tunt) stem cells, meaning they can divide into more stem cells or can become any type of cell in the body. This versatility allows embryonic stem cells to be used to regenerate or repair diseased tissue and organs, although their use in people has been to date limited to eye-related disorders such as macular degeneration. - Adult stem cells. These stem cells are found in small numbers in most adult tissues, such as bone marrow or fat. Compared with embryonic stem cells, adult stem cells have a more limited ability to give rise to various cells of the body.
Until recently, researchers thought adult stem cells could create only similar types of cells. For instance, researchers thought that stem cells residing in the bone marrow could give rise only to blood cells.
However, emerging evidence suggests that adult stem cells may be able to create unrelated types of cells. For instance, bone marrow stem cells may be able to create bone or heart muscle cells. This research has led to early-stage clinical trials to test usefulness and safety in people. For example, adult stem cells are currently being tested in people with neurological or heart disease. - Adult cells altered to have properties of embryonic stem cells (induced pluripotent stem cells). Scientists have successfully transformed regular adult cells into stem cells using genetic reprogramming. By altering the genes in the adult cells, researchers can reprogram the cells to act similarly to embryonic stem cells.This new technique may allow researchers to use these reprogrammed cells instead of embryonic stem cells and prevent immune system rejection of the new stem cells. However, scientists don't yet know if altering adult cells will cause adverse effects in humans.
Researchers have been able to take regular connective tissue cells and reprogram them to become functional heart cells. In studies, animals with heart failure that were injected with new heart cells experienced improved heart function and survival time. - Perinatal stem cells. Researchers have discovered stem cells in amniotic fluid in addition to umbilical cord blood stem cells. These stem cells also have the ability to change into specialized cells.
Amniotic fluid fills the sac that surrounds and protects a developing fetus in the uterus. Researchers have identified stem cells in samples of amniotic fluid drawn from pregnant women during a procedure called amniocentesis, a test conducted to test for abnormalities.
More study of amniotic fluid stem cells is needed to understand their potential.
Embryonic stem cells are obtained from early-stage embryos — a group of cells that forms when a woman's egg is fertilized with a man's sperm in an in vitro fertilization clinic. Because human embryonic stem cells are extracted from human embryos, several questions and issues have been raised about the ethics of embryonic stem cell research.
The National Institutes of Health created guidelines for human stem cell research in 2009. Guidelines included defining embryonic stem cells and how they may be used in research and donation guidelines for embryonic stem cells. Also, guidelines stated embryonic stem cells may only be used from embryos created by in vitro fertilization when the embryo is no longer needed.
Where do these embryos come from?
The embryos being used in embryonic stem cell research come from eggs that were fertilized at in vitro fertilization clinics but never implanted in a woman's uterus. The stem cells are donated with informed consent from donors. The stem cells can live and grow in special solutions in test tubes or petri dishes in laboratories.
Why can't researchers use adult stem cells instead?
Although research into adult stem cells is promising, adult stem cells may not be as versatile and durable as are embryonic stem cells. Adult stem cells may not be able to be manipulated to produce all cell types, which limits how adult stem cells can be used to treat diseases.
Adult stem cells also are more likely to contain abnormalities due to environmental hazards, such as toxins, or from errors acquired by the cells during replication. However, researchers have found that adult stem cells are more adaptable than was initially suspected.
What are stem cell lines and why do researchers want to use them?
A stem cell line is a group of cells that all descend from a single original stem cell and is grown in a lab. Cells in a stem cell line keep growing but don't differentiate into specialized cells. Ideally, they remain free of genetic defects and continue to create more stem cells. Clusters of cells can be taken from a stem cell line and frozen for storage or shared with other researchers.
What is stem cell therapy (regenerative medicine), and how does it work?
Stem cell therapy, also known as regenerative medicine, promotes the reparative response of diseased, dysfunctional or injured tissue using stem cells or their derivatives. It is the next chapter of organ transplantation and uses cells instead of donor organs, which are limited in supply.
Researchers grow stem cells in a lab. These stem cells are manipulated to specialize into specific types of cells, such as heart muscle cells, blood cells or nerve cells.
The specialized cells can then be implanted into a person. For example, if the person has heart disease, the cells could be injected into the heart muscle. The healthy transplanted heart cells could then contribute to repairing defective heart muscle.
Researchers have already shown that adult bone marrow cells guided to become heart-like cells can repair heart tissue in people, and more research is ongoing.
Have stem cells already been used to treat diseases?
Yes, doctors have performed stem cell transplants, also known as bone marrow transplants. In stem cell transplants, stem cells replace cells damaged by chemotherapy or disease or as a way for the donor's immune system to fight some types of cancer and blood-related diseases, such as leukemia. These transplants use adult stem cells or umbilical cord blood.
Researchers are testing adult stem cells to treat other conditions, including a number of degenerative diseases such as heart failure.
What are the potential problems with using embryonic stem cells in humans?
To be useful in people, researchers must be certain that stem cells will differentiate into the specific cell types desired.
Jeffrey Schweitzer, MD, PhD, neurosurgeon, discusses his interest in movement disorder research using stem cells to restore normal function in patients with movement disorders such as tremor, Parkinson's and dystonia. Current technologies can develop particular cell types from stem cells, which can be delivered to the brain. Dr. Schweitzer is interested in improving technology to deliver into precise locations in the brain, allowing these cells to survive and restore normal function.
SUBSCRIBE TO THE LATEST UPDATES FROM NEUROSCIENCE ADVANCES IN MOTION
Movement disorder surgery is designed to address patient quality of life. Specifically, there are three main areas of movement disorder that are potential candidates for surgery: tremor, Parkinson's disease and dystonia. These are surgeries that are reserved for people who don't tolerate medication or for whom medication is not sufficiently effective.
Over the last few decades, there's been an interest in going beyond creating either anatomical or physiological holes in the nervous system, blocking things or using white noise to affect the improvement in movement control that we want to see in movement disorder patients. The interest is in really finding cures that will restore normal function, and with particular respect to Parkinson's disease where we have a good scientific basis and knowledge of what underlies at least the motor symptoms, attention has turned to implanting tissue into the brain to improve function in these areas.
And although there's a popular misconception that stem cells are put into the brain to restore the cells that are missing, while that's not quite true, we do indeed now have the technology to use stem cells in the laboratory to develop particular cell types that we do want to put back into the brain to restore function. This technology is progressing very rapidly at a number of laboratories around the world, including here at Harvard Medical School and McLean Laboratory. I've worked with them on a project to develop this type of technology for use in Parkinson's disease, which we expect to go into clinical investigation, clinical trials, within the near future.
Our particular interest, my laboratory interest, is in improving technology to allow the delivery of this tissue into precise locations, and to ensure that when it gets there, it survives and does what we want it to do. With the development of better stem cell technology, we will have in the next 10 years a menu of options to offer patients involving closed loop stimulation for symptoms, particularly for things like tremor or dystonia where there's no obvious cell restorative solution, but also for Parkinson's disease, more precisely developed and engineered cell products to offer them. These will include things made from standard cell lines that may require people to be on immunosuppression. It may involve products developed from patient's own body cells, first program to become stem cells, and then programmed to the precise type of cell that we want. So we're going to see an explosion of a variety of options.
I think it's too early to tell which one of these things is going to be the standard. I suspect that there will be more than one, but it's very exciting to live at a time and be at the forefront, and in a place where this technology is developing. We're going to see a revolution in this field that I think will rival that which occurred in 1968 when Levodopa therapy was developed for Parkinson's disease. That will offer a range of options that have never before been possible for quality of life in an ever larger number of patients.
SUBSCRIBE TO THE LATEST UPDATES FROM NEUROSCIENCE ADVANCES IN MOTION
Movement disorder surgery is designed to address patient quality of life. Specifically, there are three main areas of movement disorder that are potential candidates for surgery: tremor, Parkinson's disease and dystonia. These are surgeries that are reserved for people who don't tolerate medication or for whom medication is not sufficiently effective.
Over the last few decades, there's been an interest in going beyond creating either anatomical or physiological holes in the nervous system, blocking things or using white noise to affect the improvement in movement control that we want to see in movement disorder patients. The interest is in really finding cures that will restore normal function, and with particular respect to Parkinson's disease where we have a good scientific basis and knowledge of what underlies at least the motor symptoms, attention has turned to implanting tissue into the brain to improve function in these areas.
And although there's a popular misconception that stem cells are put into the brain to restore the cells that are missing, while that's not quite true, we do indeed now have the technology to use stem cells in the laboratory to develop particular cell types that we do want to put back into the brain to restore function. This technology is progressing very rapidly at a number of laboratories around the world, including here at Harvard Medical School and McLean Laboratory. I've worked with them on a project to develop this type of technology for use in Parkinson's disease, which we expect to go into clinical investigation, clinical trials, within the near future.
Our particular interest, my laboratory interest, is in improving technology to allow the delivery of this tissue into precise locations, and to ensure that when it gets there, it survives and does what we want it to do. With the development of better stem cell technology, we will have in the next 10 years a menu of options to offer patients involving closed loop stimulation for symptoms, particularly for things like tremor or dystonia where there's no obvious cell restorative solution, but also for Parkinson's disease, more precisely developed and engineered cell products to offer them. These will include things made from standard cell lines that may require people to be on immunosuppression. It may involve products developed from patient's own body cells, first program to become stem cells, and then programmed to the precise type of cell that we want. So we're going to see an explosion of a variety of options.
I think it's too early to tell which one of these things is going to be the standard. I suspect that there will be more than one, but it's very exciting to live at a time and be at the forefront, and in a place where this technology is developing. We're going to see a revolution in this field that I think will rival that which occurred in 1968 when Levodopa therapy was developed for Parkinson's disease. That will offer a range of options that have never before been possible for quality of life in an ever larger number of patients.
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https://advances.massgeneral.org/neuro/video.aspx?id=1084
Overactive Brain Waves Trigger Essential Tremor January 15, 2020
In Brief
The source of essential tremor—a movement disorder that causes involuntary trembling of the hands, arms, and head—has been enigmatic, impeding the development of effective treatments for a condition that affects 4% of people over 40.
Now a new study from the Vagelos College of Physicians and Surgeons at Columbia University Irving Medical Center and NewYork-Presbyterian suggests the tremors are caused by overactive brain waves at the base of the brain, raising the possibility of treating the disorder with neuromodulation to calm the oscillations.
“Past studies have identified changes in brain structure in people with essential tremor, but we didn’t know how those changes caused tremors,” says Sheng-Han Kuo, MD, the study’s senior author and assistant professor of neurology at Columbia University Vagelos College of Physicians and Surgeons.
“This study pins down how those structural changes affect brain activity to drive tremor.”
The study was published online today in Science Translational Medicine.
Read More
In Brief
The source of essential tremor—a movement disorder that causes involuntary trembling of the hands, arms, and head—has been enigmatic, impeding the development of effective treatments for a condition that affects 4% of people over 40.
Now a new study from the Vagelos College of Physicians and Surgeons at Columbia University Irving Medical Center and NewYork-Presbyterian suggests the tremors are caused by overactive brain waves at the base of the brain, raising the possibility of treating the disorder with neuromodulation to calm the oscillations.
“Past studies have identified changes in brain structure in people with essential tremor, but we didn’t know how those changes caused tremors,” says Sheng-Han Kuo, MD, the study’s senior author and assistant professor of neurology at Columbia University Vagelos College of Physicians and Surgeons.
“This study pins down how those structural changes affect brain activity to drive tremor.”
The study was published online today in Science Translational Medicine.
Read More
Stem Cells and Regenerative Medicine Research
There is widespread interest in the use of stem cells for cell replacement therapies in human neurological disease and stroke; however, we have only begun to appreciate the cell and molecular biology of these cells that hold great promise for transplantation or other therapeutics relying on the use of different stem/progenitor cell populations or biogenic factors associated with their growth for many repair or cancer treatment approaches.
Five concurrently run projects aim to advance our understanding and use of neural stem cell therapies:
There is widespread interest in the use of stem cells for cell replacement therapies in human neurological disease and stroke; however, we have only begun to appreciate the cell and molecular biology of these cells that hold great promise for transplantation or other therapeutics relying on the use of different stem/progenitor cell populations or biogenic factors associated with their growth for many repair or cancer treatment approaches.
Five concurrently run projects aim to advance our understanding and use of neural stem cell therapies:
- The development and refinement of new in vitro methodologies to selectively expand particular embryonic and adult, including iPSC, stem or progenitor cell populations, and also control their differentiation into particular types of neurons and glia;
- The discovery of genes and factors involved in stem/progenitor cell growth and differentiation as a model for reactive neurogenesis, by way of creating cell and molecular libraries from normal and neurological disease brain tissues;
- Use of animal models and in vitro bridge bioassays of neurodegenerative disease (e.g. Parkinson’s, Huntington’s) and Dystonia by a dedicated cell culture and transplant group in the lab that is refining methods of integrating grafted stem/progenitor cells into at-risk brain circuitries;
- Stem cell plasticity and homing in a variety of tissues; and
- Studying distinct stem/progenitor cell populations as a potential source of primary tumors, and understanding their molecular biographies in order to devise new chemotherapeutic and immunological approaches for their control.
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