Stem cell facts

Stem cells are cells that have the potential to develop into many different or specialized cell types. Stem cells can be thought of as primitive, "unspecialized" cells that are able to divide and become specialized cells of the body such as liver cells, muscle cells, blood cells, and other cells with specific functions. Stem cells are referred to as "undifferentiated" cells because they have not yet committed to a developmental path that will form a specific tissue or organ. The process of changing into a specific cell type is known as differentiation. In some areas of the body, stem cells divide regularly to renew and repair the existing tissue. The bone marrow and gastrointestinal tract are examples of areas in which stem cells function to renew and repair tissue.

The best and most readily understood example of a stem cell in humans is that of the fertilized egg, or zygote. A zygote is a single cell that is formed by the union of a sperm and ovum. The sperm and the ovum each carry half of the genetic material required to form a new individual. Once that single cell or zygote starts dividing, it is known as an embryo. One cell becomes two, two become four, four become eight, eight become sixteen, and so on, doubling rapidly until it ultimately grows into an entire sophisticated organism composed of many different kinds of specialized cells. That organism, a person, is an immensely complicated structure consisting of many, many, billions of cells with functions as diverse as those of your eyes, your heart, your immune system, the color of your skin, your brain, etc. All of the specialized cells that make up these body systems are descendants of the original zygote, a stem cell with the potential to ultimately develop into all kinds of body cells. The cells of a zygote are totipotent, meaning that they have the capacity to develop into any type of cell in the body.

The process by which stem cells commit to become differentiated, or specialized, cells is complex and involves the regulation of gene expression. Research is ongoing to further understand the molecular events and controls necessary for stem cells to become specialized cell types.

Stem Cells: One of the human body's master cells, with the ability to grow into any one of the body's more than 200 cell types.

All stem cells are unspecialized (undifferentiated) cells that are characteristically of the same family type (lineage). They retain the ability to divide throughout life and give rise to cells that can become highly specialized and take the place of cells that die or are lost.

Stem cells contribute to the body's ability to renew and repair its tissues. Unlike mature cells, which are permanently committed to their fate, stem cells can both renew themselves as well as create new cells of whatever tissue they belong to (and other tissues).

Stem cells represent an exciting area in medicine because of their potential to regenerate and repair damaged tissue. Some current therapies, such as bone marrow transplantation, already make use of stem cells and their potential for regeneration of damaged tissues. Other therapies that are under investigation involve transplanting stem cells into a damaged body part and directing them to grow and differentiate into healthy tissue.

During the early stages of embryonic development the cells remain relatively undifferentiated (immature) and appear to possess the ability to become, or differentiate, into almost any tissue within the body. For example, cells taken from one section of an embryo that might have become part of the eye can be transferred into another section of the embryo and could develop into blood, muscle, nerve, or liver cells.

Cells in the early embryonic stage are totipotent (see above) and can differentiate to become any type of body cell. After about seven days, the zygote forms a structure known as a blastocyst, which contains a mass of cells that eventually become the fetus, as well as trophoblastic tissue that eventually becomes the placenta. If cells are taken from the blastocyst at this stage, they are known as pluripotent, meaning that they have the capacity to become many different types of human cells. Cells at this stage are often referred to as blastocyst embryonic stem cells. When any type of embryonic stem cells is grown in culture in the laboratory, they can divide and grow indefinitely. These cells are then known as embryonic stem cell lines.

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The embryo is referred to as a fetus after the eighth week of development. The fetus contains stem cells that are pluripotent and eventually develop into the different body tissues in the fetus.

Adult stem cells are present in all humans in small numbers. The adult stem cell is one of the class of cells that we have been able to manipulate quite effectively in the bone marrow transplant arena over the past 30 years. These are stem cells that are largely tissue-specific in their location. Rather than typically giving rise to all of the cells of the body, these cells are capable of giving rise only to a few types of cells that develop into a specific tissue or organ. They are therefore known as multipotent stem cells. Adult stem cells are sometimes referred to as somatic stem cells.

The best characterized example of an adult stem cell is the blood stem cell (the hematopoietic stem cell). When we refer to a bone marrow transplant, a stem cell transplant, or a blood transplant, the cell being transplanted is the hematopoietic stem cell, or blood stem cell. This cell is a very rare cell that is found primarily within the bone marrow of the adult.

One of the exciting discoveries of the last years has been the overturning of a long-held scientific belief that an adult stem cell was a completely committed stem cell. It was previously believed that a hematopoietic, or blood-forming stem cell, could only create other blood cells and could never become another type of stem cell. There is now evidence that some of these apparently committed adult stem cells are able to change direction to become a stem cell in a different organ. For example, there are some models of bone marrow transplantation in rats with damaged livers in which the liver partially re-grows with cells that are derived from transplanted bone marrow. Similar studies can be done showing that many different cell types can be derived from each other. It appears that heart cells can be grown from bone marrow stem cells, that bone marrow cells can be grown from stem cells derived from muscle, and that brain stem cells can turn into many types of cells.

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Most blood stem cells are present in the bone marrow, but a few are present in the bloodstream. This means that these so-called peripheral blood stem cells (PBSCs) can be isolated from a drawn blood sample. The blood stem cell is capable of giving rise to a very large number of very different cells that make up the blood and immune system, including red blood cells, platelets, granulocytes, and lymphocytes.

All of these very different cells with very different functions are derived from a common, ancestral, committed blood-forming (hematopoietic), stem cell.

Blood from the umbilical cord contains some stem cells that are genetically identical to the newborn. Like adult stem cells, these are multipotent stem cells that are able to differentiate into certain, but not all, cell types. For this reason, umbilical cord blood is often banked, or stored, for possible future use should the individual require stem cell therapy.

Induced pluripotent stem cells (iPSCs) were first created from human cells in 2007. These are adult cells that have been genetically converted to an embryonic stem celllike state. In animal studies, iPSCs have been shown to possess characteristics of pluripotent stem cells. Human iPSCs can differentiate and become multiple different fetal cell types. iPSCs are valuable aids in the study of disease development and drug treatment, and they may have future uses in transplantation medicine. Further research is needed regarding the development and use of these cells.

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Embryonic stem cells and embryonic stem cell lines have received much public attention concerning the ethics of their use or non-use. Clearly, there is hope that a large number of treatment advances could occur as a result of growing and differentiating these embryonic stem cells in the laboratory. It is equally clear that each embryonic stem cell line has been derived from a human embryo created through in-vitro fertilization (IVF) or through cloning technologies, with all the attendant ethical, religious, and philosophical problems, depending upon one's perspective.

Routine use of stem cells in therapy has been limited to blood-forming stem cells (hematopoietic stem cells) derived from bone marrow, peripheral blood, or umbilical cord blood. Bone marrow transplantation is the most familiar form of stem cell therapy and the only instance of stem cell therapy in common use. It is used to treat cancers of the blood cells (leukemias) and other disorders of the blood and bone marrow.

In bone marrow transplantation, the patient's existing white blood cells and bone marrow are destroyed using chemotherapy and radiation therapy. Then, a sample of bone marrow (containing stem cells) from a healthy, immunologically matched donor is injected into the patient. The transplanted stem cells populate the recipient's bone marrow and begin producing new, healthy blood cells.

Umbilical cord blood stem cells and peripheral blood stem cells can also be used instead of bone marrow samples to repopulate the bone marrow in the process of bone marrow transplantation.

In 2009, the California-based company Geron received clearance from the U. S. Food and Drug Administration (FDA) to begin the first human clinical trial of cells derived from human embryonic stem cells in the treatment of patients with acute spinal cord injury.

Stem cell therapy is an exciting and active field of biomedical research. Scientists and physicians are investigating the use of stem cells in therapies to treat a wide variety of diseases and injuries. For a stem cell therapy to be successful, a number of factors must be considered. The appropriate type of stem cell must be chosen, and the stem cells must be matched to the recipient so that they are not destroyed by the recipient's immune system. It is also critical to develop a system for effective delivery of the stem cells to the desired location in the body. Finally, devising methods to "switch on" and control the differentiation of stem cells and ensure that they develop into the desired tissue type is critical for the success of any stem cell therapy.

Researchers are currently examining the use of stem cells to regenerate damaged or diseased tissue in many conditions, including those listed below.

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Medically Reviewed on 9/8/2016

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"Stem Cell Information." National Institutes of Health.

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There may not be a greater debate in the medical community right now than that of embryonic stem cell research. Initially banned by the Federal government, these stem cells often originate from human embryos that were created for couples with reproductive issues and would be discarded. These stem cells are thought to be the key that will unlock the cure to many diseases, from Alzheimers to rare immune and even genetic disorders. On the other side of the issue, some see the destruction of an embryo as the murder of an unborn child.

The primary benefit of this research is the enormous amount of potential that it holds. Embryonic stem cells have the ability to create new organs, tissues, and systems within the human body. With a little guidance from scientists, these stem cells have shown that they can become new organs, new blood vessels, and even new ligaments for those with ACL tears. By culturing stem cells and them implanting them, recovery times could be halved for many serious injuries, illnesses, and diseases.

Because nearly one-third of the population could benefit from treatments and therapies that could originate from embryonic stem cell research, many scientists believe that this field could alleviate as much human suffering as the development of antibiotics was able to do. Because funding was restricted on embryonic stem cell lines for several years, however, the chances of any therapies being viable in the near future are slim.

The primary argument against this research is a moral one. Some people see the creation of an embryo as the creation of life, so to terminate that life would equate to murder. This primarily originates from a point of view where life as we define it begins at conception, which would mean that any medical advancement from this research would be at best unethical.

Those against this research argue that since the creation of this research field in the early 1980s, there have been no advancements in it whatsoever. Because of this lack of advancement, it could mean decades of additional research, thousands of embryos destroyed to further that research, and that is morally unacceptable for some.

The debate about embryonic stem cell research isnt in the potential benefits that this field of study could produce. It is in the ethics and morality of how embryonic stem cells are created. There often is no in-between view in this area: you either define life at some part of the physical development of the human body during the pregnancy or you define it at conception. This view then tends to lead each person to one side of this debate. Where do you stand?

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Studies on diseases like ALS, Alzheimer's, Parkinson's, and Huntington's jeopardized if GOP-controlled Congress cuts funding for embryonic stem cell research.

In 2010, when renowned stem cell scientist Lawrence Goldstein, PhD, published his groundbreaking book Stem Cells for Dummies, with co-author Meg Schneider, the forecast for human embryonic stem cell research had just begun to brighten.

In 2001, former President George W. Bush cast a cloud over this field of science by barring the National Institutes of Health (NIH) from funding research that used embryonic stem cells beyond the 60 cell lines that already existed.

But in 2009, then-President Barack Obama signed an executive order repealing Bushs policy.

Obamas decision enabled researchers like Goldstein, director of the UC San Diego Stem Cell Program, and Sanford Stem Cell Clinical Center, to make real progress, inching closer to human clinical trials.

Goldsteins work focuses on discovering clinical applications for human embryonic stem cells, also known as ESC.

His work looks specifically at clinical applications for neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), and Alzheimers, Parkinsons, and Huntingtons diseases.

After 10 years, weve seen a variety of projects that use embryonic stem cells moving closer to clinical applications and in clinical trials, Goldstein told Healthline.

Although its taken some time, were getting closer to seeing the most promising approach for treatment of different neurologic disorders that have no suitable treatment alternative.

Read more: Stem cells therapy offers hope for MS remission

But now use of human embryonic stem cells are once again under fire from conservative and pro-life groups.

Contrary to popular belief, human embryonic stem cells do not come from aborted fetuses.

All the human embryonic stem cell lines currently in use are derived from unused embryos developed for in vitro fertilization and donated for research.

They are cells that would have only been discarded.

Nevertheless, their use in research is opposed by many in the pro-life movement, including a vocal coalition in Congress.

Last month, 41 conservatives in the House urged President Trump to fire Dr. Francis Collins, director of the NIH, the worlds largest agency funding biomedical research, because Collins supports embryonic stem cell research.

However, Trump announced last week he was reappointing Collins, a widely respected physician-geneticist.

Several Republican leaders in Congress had reportedly urged Trump to retain him, calling Collins the right person, at the right time, to continue to lead the worlds premiere biomedical research agency.

But Trumps decision didnt sit well with many in his voting base and his own cabinet.

Vice President Mike Pence, and Health and Human Services Secretary, Tom Price, have both spoken out against the use of embryonic stem cells on moral grounds.

Just a few weeks ago, Pence got a standing ovation at the National Catholic Prayer Breakfast when he reminded the audience that he was the one who cast the tie-breaking vote in the United States Senate that allowed states to defund Planned Parenthood.

The Congressional conservatives who called on Trump to fire Collins are voicing their anger over Trumps decision to retain Collins.

Rep. Jim Banks, R-Ind., told LifeNews, a pro-life publication, that he was disappointed in the Trump Administrations decision.

Dr. Collins support of embryonic stem cell research, along with his comments that cloned embryos do not deserve the same moral protections as naturally generated embryos, make him a less than an ideal fit for a pro-life administration, Banks said. I am hopeful that Dr. Collins will turn away from embryo-killing research as he continues his tenure as NIH director.

Trumps election has resulted in a new and unprecedentedly tenuous era for the NIH. Its funding has typically had bipartisan support.

Despite Trumps seemingly pro-science pivot, the NIH still faces a potential $5.8 billion cut about 18 prevent in the presidents fiscal 2018 budget.

And this plank from the 2016 GOP platform remains in place:

We oppose embryonic stem cell research. We oppose federal funding of embryonic stem cell research. We support adult stem cell research and urge the restoration of the national placental stem cell bank created by President George H.W. Bush but abolished by his Democratic successor, President Bill Clinton.

Goldstein and several other scientists interviewed for this story said that while Trumps decision to retain Collins is a positive, theres still no guarantee that embryonic stem cell research will continue getting support from the federal government in this increasingly hostile and volatile political climate.

While human embryonic stem cells are just one of several types of stem cells being studied for their innate, but complex, abilities to treat diseases, Goldstein explained, they are an important weapon in a growing arsenal.

It takes a great deal of time, money, and patience to develop these therapies, he noted.

It would be a shame to go back to the dark age solution that we had under the Bush administration, said Goldstein, who is currently focused largely on ALS, also known as Lou Gehrigs disease, named for the legendary New York Yankee.

Gehrig died from the disease at age 37.

Goldstein said a lot of individuals and institutions are working to find treatments for ALS, which has enjoyed a boost in awareness and funding thanks to the recent Ice Bucket Challenge that caught on nationwide.

Its important that we develop an aggressive set of cell therapy programs so that we have multiple shots on goal, Goldstein said. We need to attack the disease from as many angles as possible.

Read more: Stem cell therapy a possible treatment for RA

For the last 25 years, Frances Saldaa has been on a mission to increase awareness of Huntingtons disease (HD), a debilitating, incurable, and often inherited disease.

Its become my mission in life to advocate for support of HD research and for excellence in patient care, said Saldaa, whose husband, Hector Portillo, didnt tell her he had the disease.

Three of their children inherited HD from their father. Both of Saldaas daughters have died, and her son is not doing well.

My son Michael is fighting for his life every single day, but time is running out for him too, she said. The suffering endured at the end of life for HD patients is unimaginable. My daughter, Margie, and her husband did not have the money to go through IVF when they started their family. They had two beautiful children. I live in fear that my two grandchildren, now 19 and 21, are also at risk of inheriting HD.

When Saldaa learned that members of Congress were urging President Trump to fire Collins, her heart sank.

To have the door close on this research because they have this belief would be tragic, she said. In my opinion, an embryonic egg is not life until its attached to the placenta.

Saldaa said that if she had the opportunity, she would ask people who oppose this research, Have they ever seen their own children dying devastating deaths? Have they ever seen their own children lose the ability to talk, to swallow? Have they ever had their own child die in their arms, and yet know that there is hope, that there is a chance with this research to find a cure?

Ive dedicated my life to supporting this research, from Team Hope walks to bake sales, everything and anything to find a cure, she said.

Leslie Thompson, PhD, a professor of psychiatry and human behavior, and professor of neurobiology and behavior, at the University of California Irvine, has devoted her entire career to unlocking the mysteries of Huntingtons disease and finding treatments.

Thompson, who said many of her patients with HD are like family, said human embryonic stem cells present great hope for finding a treatment for HD.

And after decades of painstaking study, she told Healthline that her work could lead to human clinical trials for people with HD as soon as two or three years from now.

Thompson keeps a picture of Saldaas children in her office to remind her of what her research is really all about.

Im deeply concerned how this could move the field backwards, she said, but added that she is hopeful her work and that of others will be allowed to continue.

Were in an exciting, unprecedented time of opportunity to use stem cells for treatments, she said.

When former President George W. Bush decided to halt new human embryonic stem cell research, Saldaa recalled, We all jumped and went toward getting Prop 71 passed.

The California Stem Cell Research and Cures Initiative, which was passed by a 60-40 margin, was drafted by the California Institute for Regenerative Medicine. It has provided millions of dollars in stem cell research in the state, including embryonic stem cell research.

The public vote on this initiative has helped California become the national leader in stem cell research.

There is also solidarity in the stem cell community. Even scientists who dont use human embryonic stem cells still support their colleagues who do.

Jeanne Loring, PhD, a professor of developmental neurobiology, and director of the Center for Regenerative Medicine at the Scripps Research Institute in La Jolla, Calif., made the shift about a decade ago from human embryonic stem cells to induced pluripotent stem cells (IPS), which she makes from cells cultured from skin biopsies.

There are certain advantages to IPS, she said, but added that she still fully supports embryonic stem cell research.

Its hard to predict what President Trump will do, said Loring, whose lab is working on finding treatments for Parkinsons disease, discovering the cause of autism, ways to treat it, and more.

Loring notes that there are great misunderstandings about embryonic stem cell research and where the cells come from.

There is always an undercurrent of misunderstanding about sources of human stem cells. People think they are associated with abortion but they are not. she said.

Read more: Using stem cells to heal broken bones

What lies ahead for human embryonic stem cell research is anyones guess.

When Obama signed the order to lift Bushs ban on new embryonic stem cell research, he said, In recent years, when it comes to stem cell research, rather than furthering discovery, our government has forced what I believe is a false choice between sound science and moral values.

In this case, I believe the two are not inconsistent. As a person of faith, I believe we are called to care for each other and work to ease human suffering. I believe we have been given the capacity and will to pursue this research and the humanity and conscience to do so responsibly.

Goldstein said he hopes Trump, too, will embrace the importance of this research that seeks to find treatments for deadly diseases.

Its early in this administration, there is still time for them to staff up with people who will give the President the appropriate scientific advice, Goldstein said. There are many challenges facing us that are technological in nature. You cant have a humming economy without robust investment in science. It drives the development of new technologies and devices. An investment in science pays far more than what you put in.

Goldstein said investing heavily in science has helped give the United States the quality of life that Americans now enjoy.

Our investments in science and technology are likely the reason for winning World War II, he said. And our investment in biotech has revolutionized medicine and has been invaluable to providing jobs in many states.

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September 18, 2017| Stem Cell Orthopedic Institute of Texas Team | STEM CELL THERAPY

If you have any interest in stem cells, you may be familiar with the debate on embryonic stem cells. Stem cells have been researched since the mid-1900s. While adult stem cells have proven to be safe and effective, embryonic stem cells are banned for human use in the United States.

As such, at the Stem Cell Orthopedic Institute, we exclusively use adult stem cells. For a complete description of our process, please visit our procedure overview.

While we do not use embryonic stem cells in America, it is important to remember how we got to this point to inform what choices we make going forward. Heres the truth about embryonic stem cells:

Just like the name sounds, embryonic stem cells are derived from embryos. Most embryonic stem cells come from embryos that develop from eggs that were fertilized in vitro (in an in vitro fertilization clinic) and then donated for research purposes with the donors consent. These stem cells are not derived from the eggs that have been fertilized in a womans body.

Fertilization usually occurs in the oviduct. The few days after the embryo travels down the oviduct and into the uterus, a series of cleavage divisions will occur. The embryo is a ball of around 100 cells at this point (called a blastocyst). The outer layer of cells forms the placenta and is called the trophectoderm. The cells during this stage are undifferentiated, which means that they do not look or act like the specialized adult cells, and they are not yet committed to becoming any specific type of differentiated cell.

The first differentiation event occurs at around five days of development which is when an outer layer of cells separates from the inner cell mass (ICM). The ICM cells have the potential to generate any cell type, but once they have been implanted, they are quickly depleted as they differentiate into other cell types with limited developmental potential. However, the ICM cells can continue to multiply and endlessly replicate themselves, while maintaining the developmental potential to form any cell only if the ICM is removed from its normal embryonic environment and cultured under proper conditions.

Embryonic stem cells have a never-ending lifespan with an almost unlimited developmental potential. Embryonic stem cells are pluripotent, which means that can grow into any one of the three primary germ layers: ectoderm, endoderm, and mesoderm. These cells have the potential to form any type of cell in the body, from muscle to nerve to blood. Embryonic stem cells provide endless possibilities for researchers.

In 2001, President George W. Bush allowed federal funding for limited embryonic stem cell research. However, President Barack Obama revoked that statement in 2009 and released Executive Order 13505 to remove the restrictions on federal funding for stem cell research. This allowed the National Institutes of Health (NIH) to fully fund research with embryonic stem cells. The NIH issued guidelines to establish the policy. These guidelines were created to help ensure that all NIH-funded research on human stem cells is morally responsible and scientifically relevant.

Embryonic stem cells are used for many purposes, from basic research to transplantation therapies for various diseases like heart disease, Parkinsons disease, leukemia, and more. Since it is illegal to use human embryonic stem cells, researchers rely on mice and other animals for these cells. Stem cells have to potential to grow new cells to replace damaged organs or tissues, correct portions of organs that work improperly, research causes of genetic defects in cells, research how diseases occur or why certain cells develop into cancer cells and test new drugs for safety and effectiveness.

Research with embryonic stem cells (ESCs) is highly debated and many people have strong opinions about their stance on the issue. Many of the discussions lie around moral and ethical issues. The dilemma forces us to choose between two moral principles: the duty to prevent and alleviate suffering or the duty to respect the value of human life.

Lets take a look at both stances. In order to obtain embryonic stem cells, you must destroy the embryo also meaning that you are destroying a potential human life. On the other hand, embryonic stem cell research could help us find new medical treatments that could help alleviate or end those suffering from many disorders and diseases. The biggest question: which moral principle should have the upper hand? The answer ultimately depends on how you view the human embryo.

Embryonic stem cells also have a high oncogenic potential meaning it is potentially cancerous. Since these cells have the possibility to transform into practically any cell, there is a possibility they can form tumors in patients.

At the Stem Cell Orthopedic Institute of Texas, we never use embryonic stem cells only adult stem cells. Our patients health and safety is our priority. Rest assured, our doctors will give you the personalized attention you deserve. To learn more about our stem cell treatments or to schedule an appointment, call us at 210-293-3136.

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Dr. Francis Collins has not shown any pro-life leadership at the National Institutes of Health (NIH). In fact, in an interview, Dr. Collins response to a congressional letter outlining pro-life members concerns dripped with condescension, implying that the group of 41 congressmen understood neither the science nor the ethics of embryo and stem cell experiments. Dr. Collins owes us an apology. We know the science, use the scientifically accurate terms and know the ethical facts. Dr. Collins positions at NIH have not been pro-life.

His lack of pro-life leadership might have been expected when he served under the previous administration, which was the antithesis of pro-life. However, now Dr. Collins has agreed to work for President Trump, who campaigned on a pro-life agenda. Will Dr. Collins change his positions and adjust his agenda? When will we have a pro-life NIH Director who reflects the policy of our president?

As one example of the void in pro-life leadership, Dr. Collins designed and oversees the NIH registry of human embryonic stem cell lines, a listing of cells created by destroying young human embryos that are eligible for hundreds of millions in federal taxpayer dollars. Dr. Collins continuously approves cells for this registry, and did so most recently in March and again in June of this year.

The registry has created a cottage industry for those who want to destroy human embryos and then reap taxpayer dollars for their efforts. The establishment of the registry created an incentive for further destruction of young human embryos, under the guise of expanding scientific research and providing more experimental material. Dr. Collins called it important, life-saving research, despite the fact that embryonic stem cells have to this day not saved a single human life nor proven to have any near-term success in patients.

Eight years after its inception, the registry is nothing more than an embryonic charnel house. The stem cell lines sit as names and numbers on the registry, memorial markers to the lives of the human embryos destroyed in the name of science.

Moreover, scientific leaders admit that human embryonic stem cells now serve primarily as references to compare with nonembryonic stem cells in the laboratory. Induced pluripotent stem (iPS) cells, which show the same characteristics as embryonic stem cells, have largely replaced embryonic stem cells. The Nobel-prize-winning iPS cells can be made from virtually any person or tissue, healthy or diseased, more cheaply and efficiently than embryonic stem cells, without destroying the donor of the cells.

After consuming a decade and a half of federal funding, amounting to well over a billion taxpayer dollars, embryonic stem cell research has produced no help for patients. The stem cell registry at NIH and federal funding for it should be foreclosed, and the funds should be redirected to research that shows real hope for patients.

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In previous interviews, Dr. Collins has ignored the gold standard of stem cells for patients: adult stem cell research. Adult stem cells have now treated well over 1 million patients around the globe, including tens of thousands of children. Adult stem cells are the only stem cell option to show authenticated, life-saving success in patients, validated by hundreds of scientific publications.

Yet in a 2009 interview, Dr. Collins touted the one clinical trial approved by the FDA at the time a single trial that showed no benefit to any patient from embryonic stem cells, even to today and ignored over 2,000 adult stem cell clinical trials ongoing at the time (the number of adult stem cell trials now exceeds 3,000). Adult stem cell research is providing real innovation for patients now, and it could use the funding that now goes to dogmatic support for antiquated science. Will Dr. Collins voice his support for adult stem cell research and redirect funding toward patient-focused science.

The House of Representatives has shown tangible support for this idea with the introduction of H.R. 2918, the Patients First Act of 2017. The bill would direct HHS to prioritize adult stem cell research that has the best chance of producing near-term benefits in patients without the creation, destruction, or risk of injury to human embryos. Furthermore, the bill advocates for the ethical approach without authorizing any additional spending.

In addition to refusing to acknowledge the potential of adult stem cell research, Dr. Collins has also supported human cloning to create embryos for experiments. In this scenario, a cloned human embryo would not be allowed to survive and develop, but rather be torn apart for the use of its cells in laboratory tests. Cloning (technically termed somatic cell nuclear transfer) requires the transfer of a cell nucleus into an egg that has had its own nucleus removed. This is the way Dolly the cloned sheep and other cloned animals all began, as cloned embryos.

Yet Dr. Collins takes the unscientific view that a cloned embryo is not really an embryo, because, he says, it was not produced by a sperm and egg coming together. Even the NIH states that the cloning process produces an embryo.

The NIH can be a world leader in successful, ethical science and medicine. But this shift requires a pro-life leader at the helm.

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Doubts have surfaced about a landmark paper claiming that human embryos were cleared of a deadly mutation using genome editing. In an article1 posted to the bioRxiv preprint server on 28 August, a team of prominent stem-cell scientists and geneticists question whether the mutation was actually fixed.

The 2 August Nature paper2, led by reproductive biologist Shoukhrat Mitalipov at the Oregon Health and Science University in Portland, described experiments in dozens of embryos to correct a mutation that causes a heart condition called hypertrophic cardiomyopathy.

In contrast to previous human-embryo editing studies, Mitalipovs team reported a high success rate at correcting a disease-causing mutation in a gene. The team claimed that the CRISPRCas9 genome editing tool was able to replace a mutant version of the MYBPC3 gene carried by sperm with a normal copy from the egg cell, yielding an embryo with two normal copies. Mitalipovs team also introduced a healthy version of the gene along with the CRISPR machinery, but they found that the corrected embryos had shunned it for the maternal version.

But there is reason to doubt whether this really occurred, reports a team led by Dieter Egli, a stem-cell scientist at Columbia University in New York City, and Maria Jasin, a developmental biologist at Memorial Sloan Kettering Cancer Center in New York City. George Church, a geneticist at Harvard Medical School in Boston, Massachusetts, is another co-author.

In their bioRxiv paper, Egli and Jasin and their co-authors say that there is no plausible biological mechanism to explain how a genetic mutation in sperm could be corrected based on the eggs version of the gene. More likely, they say, Mitalipovs team failed to actually fix the mutation and were misled into thinking they had by using an inadequate genetics assay. Egli and Jasin declined to comment because they say they have submitted their article to Nature.

The critique levelled by Egli et al. offers no new results but instead relies on alternative explanations of our results based on pure speculation, Mitalipov said in a statement.

But other scientists contacted by Nature's news team shared the Egli team's concerns. (Natures news team is editorially independent of its journal team.) Reproductive biologist Anthony Perry at the University of Bath, UK, says that after fertilization, the genomes of the egg and sperm reside at opposite ends of the egg cell, and each is enshrouded in a membrane for several hours. This fact, Perry says, would make it difficult for CRISPR-Cas9 to fix the sperms mutation based on the eggs version of the gene, using a process called homologous recombination. Its very difficult to conceive how recombination can occur between parental genomes across these huge cellular distances, he says.

Egli and Jasin raise that issue in their paper. They suggest that Mitalipovs team was misled into believing that they had corrected the mutation by relying on a genetic assay that was unable to detect a far likelier outcome of the genome-editing experiment: that CRISPR had instead introduced a large deletion in the paternal gene that was not picked up by their genetic assay. The Cas9 enzyme breaks DNA strands, and cells can attempt to repair the damage by haphazardly stitching the genome together, often resulting in missing or extra DNA letters.

That explanation makes sense, says Gatan Burgio, a geneticist at the Australian National University in Canberra. In my view Egli et al. convincingly provided a series of compelling arguments explaining that the correction of the deleterious mutation by self repair is unlikely to have occurred.

Another possibility Eglis team raise is that the embryos were produced without a genetic contribution from sperm, a process known as parthenogenesis. Mitalipovs team showed that the paternal genome was present in only 2 out of the 6 embryonic stem cell lines they made from gene-edited embryos.

Robin Lovell-Badge, a developmental biologist at the Francis Crick Institute in London, says that it is possible that there is a novel or unsuspected biological mechanism at work in the very early human embryo that could explain how Mitalipovs team corrected the embryos genomes in the manner claimed. He would first like to hear from Mitalipov before passing judgement. It simply says that we need to know more, not that the work is unimportant, Lovell-Badge says of Egli and Jasins paper.

In the statement, Mitalipovs said his team stands by their results. We will respond to their critiques point by point in the form of a formal peer-reviewed response in a matter of weeks.

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Doubts raised about CRISPR gene-editing study in human embryos - Nature.com

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| Marlowe Hood |

PARIS (AFP) Lab monkeys with Parkinsons symptoms regained significant mobility after neurons made from human stem cells were inserted into their brains, researchers reported Wednesday in a study hailed as groundbreaking.

The promising results were presented as the last step before human clinical trials, perhaps as early as next year, the studys senior author, Jun Takahashi, a professor at Kyoto University, told AFP.

Parkinsons is a degenerative disease that erodes motor functions. Typical symptoms include shaking, rigidity and difficulty walking. In advanced stages, depression, anxiety and dementia are also common.

Worldwide, about 10 million people are afflicted with the disease, according to the Parkinsons Disease Foundation.

Earlier experiments had shown improvements in patients treated with stem cells taken from human foetal tissue and likewise coaxed into the dopamine-producing brain cells that are attacked by Parkinsons.

Dopamine is a naturally occurring chemical that plays several key roles in the brain and body.

But the use of foetal tissue is fraught with practical and ethical problems.

So Takahashi and his colleagues, in a medical first, substituted so-called induced pluripotent stem cells (iPSCs), which can be easily made from human skin or blood. Within a year, some monkeys who had could barely stand up gradually recovered mobility.

They became more active, moving more rapidly and more smoothly, Takahashi said by email. Animals that had taken to just sitting start walking around in the cage.

These findings are strong evidence that human iPSC-derived dopaminergic neurons can be clinically applicable to treat Parkinsons patients, he said.

Experts not involved in the research described the results as encouraging.

The treatment, if proven viable, has the potential to reverse Parkinsons by replacing the dopamine cells that have been lost a groundbreaking feat, said David Dexter, deputy research director at Parkinsons UK.

Not only did the new cells survive but they also integrated with the existing neuronal network, he said.

Neurons made from foetal tissue grafted into brains have been known to survive for more than a decade, and the researchers said they expected those derived from iPSCs to last just as long.

Tilo Kunath, Parkinsons Senior Research Fellow at the University of Edinburgh, said the outcome was extremely promising, and highlighted the advantage of avoiding stem cells extracted from human foetal tissue.

It means that this therapy can be used in any country worldwide, including Ireland and most of South America, where medical use of human embryonic stem cells is banned.

The results, reported in the journal Nature, were not the same for the dozen monkeys in the experiment, each of which received donor neurons from a different person.

Some were made with cells from healthy donors, while others were made from Parkinsons disease patients, said lead author Tetsuhiro Kikuchi, also from Kyoto University.

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Study shows human stem cells restore mobility in Parkinson's monkeys - Borneo Bulletin Online

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Sofa Vergaras embryos are citizens of California.

Christopher Polk/Getty Images for TNT

After years of battling ex-fiance Sofa Vergara in court for custody of a pair of frozen embryos they once made together, condiment entrepreneur Nick Loeb might finally be out of luck. Last week, a federal judge in Louisiana dismissed Loebs suit, saying that the embryos are citizens of California, where Vergara and Loeb conceived and froze them. Thus, the judge ruled, embryos have no legal standing to sue in Louisiana, the only state that gives embryos the right to sue and be sued.

Christina Cauterucci is a Slate staff writer.

Embryos are frozen when they are just clumps of a few dozen cells, equivalent to a vaginally inseminated egg that would still take another week to become embedded in the uterine wall. Louisiana law deems these cells juridical personsnot quite human beings, but deserving of legal rights. In Louisiana, embryos are not merely property of the two people who made them, so any legal disputes must meet the best interest of the in vitro fertilized ovum.

Thats the most likely reason why Loeb sued Vergara in Louisiana, despite the fact that neither party maintains a residence there.* (Loeb says he chose the state because the couple broke up there; he dropped an earlier California suit because he didnt want to name his previous girlfriends whod had abortions.) Actually, Loeb didnt exactly sue Vergarathe embryos, Emma and Isabella, did. Plaintiff EMMA is a female human being at the embryonic stage of life, five days old developmentally, the right to live suit read, claiming that Vergara had effectively abandoned and chronically neglected her children by keeping them frozen in a medical tank since 2013. Though Vergara and Loeb had signed a contract when they were together agreeing that the embryos would never be implanted anywhere without both parties consent, Loeb wanted to nullify the agreement and implant them in a surrogate.

Over the past couple of years, the Vergara-Loeb embryo battle has become a proxy fight for anti-abortion advocates who think frozen embryos should be treated like people. Anti-abortion groups have funded or filed amicus briefs in the embryo disputes of split-up couples, including the case of a Missouri woman named Jalesia McQueen. In the middle of her own court battle over two frozen embryos (Noah and Genesis) with an ex-husband who wanted to dispose of them, McQueen founded an organization called Embryo Defense to advocate for all excess embryos in legal limbo. She aggregates news on the Vergara-Loeb case and uses their photos in images made for sharing on social media. The graphics say things like Sofia says its selfish to let the embryos be born without both parents being in a loving relationship. Shouldnt both parents just love the child? and Please pray for Sofia Vergara and those she called her frozen babies, that shed open her heart so they could be a blessing in her life. One pairs a picture of Loebs face with the question what about a fathers right to choose?

The concept of a fathers right to procreate without input from the woman whose egg created the embryo is a favorite rallying cry of the embryo-protector set. Loeb himself made this argument in a 2015 New York Times op-ed with the magnificent headline Sofa Vergaras Ex-Fianc: Our Frozen Embryos Have a Right to Live. A woman is entitled to bring a pregnancy to term even if the man objects, Loeb wrote. Shouldnt a man who is willing to take on all parental responsibilities be similarly entitled to bring his embryos to term even if the woman objects?

The federal judges decision against Loeb is only the latest in a string of disappointing court losses for those who believe embryos should be treated like people. When Barack Obama lifted a ban on federal funding for embryonic stem cell research in 2009, he was hit with a lawsuit from one Mary Scott Doe, a frozen embryo symbolizing all existing frozen embryos. A federal appeals court ruled that Doe had no standing as an amorphous class that could not prove any actual harm. In 2015, a California Superior Court judge ruled that a contract a divorced couple signed at the medical center where they created the embryo prevented either party from taking unilateral custody of the embryos unless one of the parties died. Even McQueen, the founder of Embryo Defense, lost her suit late last year when a St. Louis court ruled that embryos are marital property of a special character, not human beings with unalienable rights.

That St. Louis case might be the most promising decision yet for those who believe that no person should be able to incubate an embryo without the consent of the other person whose genetic material it carries. The others, which rest on the tenets of contract law, legal standing, and jurisdiction, say more about how the suits were filed than what rights adults have to the embryos they create. Still, Loebs loss could set a welcome precedent in these kinds of cases: Jurisdiction-shopping embryo protectors might find Louisiana to be a less hospitable home for a lawsuit than theyd imagined.

*Correction, August 31, 2017: This piece originally referred to "the most likely reason why Loeb sued Vergara in California, despite the fact that neither party maintains a residence there." It should have said Louisiana.

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Sofa Vergara's ex might finally be out of luck in his battle for custody ... - Slate Magazine (blog)

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Cardiac predecessor cells appear to rejuvenate the hearts of older animals, according to a recent study from Cedars-Sinai Heart Institute that may lead to tests in humans.

Signs of rejuvenation in rats included a 20 percent increase in exercise capacity, faster regrowth of hair, and lengthening of the protective caps of chromosomes.

The study used cardiosphere-derived cells, or CDCs, which are like stem cells, but can only develop into heart cells. These cells are already being used in a human clinical trial to repair damage from heart attacks. The trial is being conducted by Beverly Hills-based Capricor in several hospitals, including Scripps La Jolla.

Since these cells have already been found to be safe, it should be fairly straightforward to extend testing from repairing heart damage in people to rejuvenation, said study leader Dr. Eduardo Marbn. Hes director of the Los Angeles Institute, part of Cedars-Sinai Medical Center. Marbn is also a co-founder of Capricor, publicly traded on Nasdaq.

However, a researcher not involved in the study said that while it was well done, the history of stem cell treatments indicates that proving efficacy in people promises to be far more difficult.

The study used cells taken from newborn rats, injected into the hearts of older, senescent rats. It was published Aug. 14 in the European Heart Journal.

The study is exceptional in both its scope and breadth, said Dr. Richard Schatz, a Scripps Clinic cardiologist involved in the Capricor trial at Scripps La Jolla.

It examines an extraordinary number of variables rarely seen in such studies to ask the question of the impact of CDC (specialized stem cells) on cardiac aging in rats, Schatz said by email. Every parameter of how aging might be studied moved in the right direction, meaning there might be a biologic effect of their cells throughout the body.

Schatz cautioned that scientific excellence doesnt equal clinical success.

The technologys muscle-improving effectiveness could also help patients with Duchenne muscular dystrophy, Marbn said. That use is in clinical testing by Capricor. Early results in patients have been promising enough that more studies are planned.

Capricor clinical trial information is available at http://capricor.com/clinical-trials.

Marbn said the study adds to growing evidence that progenitor cells exert their healing power by secreting chemicals that stimulate repair, not by permanently incorporating themselves into the body. The chemicals are enclosed in tiny vesicles called exosomes that the cells shed.

Until fairly recently, exosomes were dismissed as cellular debris, but are now being appreciated for their role in cell signaling, Marbn said.

There's a staggering number, something like 100 billion to a trillion exosomes per drop of blood, per drop of cerebrospinal fluid, Marbn said. They are plentiful in breast milk. The only thing we know right now is that there is a complex signaling system.

These exosomes travel far beyond the heart to reach skeletal muscle, which is weakened in Duchenne muscular dystrophy, he said.

Schatz said the study provides evidence that the cells exert many different effects beyond those in a single target organ, through the exosomes, seen in humans as well.

This is very good news if you are a rat, but the obvious limitation is how will this play out in humans, Schatz said.

Previous clinical trials of stem cells have been successful in Phase 1 and 2, Schatz said, but fail in Phase 3. So the researchers face a daunting road ahead to demonstrate usefulness in people.

This does not take away from the brilliant science behind this exceptional group of investigators, Schatz said. They should be congratulated for a very thoughtful and expansive look at a fascinating subject, the clinical relevance of which remains to be seen.

The rejuvenation effects to some degree resemble cells created when adult cells are reprogrammed back to being stem cells, Marbn said.

Certain factors are turned on that regress the cells to act like embryonic stem cells. These are called induced pluripotent stem cells, because they can become nearly any cell in the body, a property called pluripotency.

Something like this might be happening through exosome-mediated reprogramming.

We have a suspicion that even though we didn't go about trying to activate those factors, some of them may in fact be turned on by the therapy, Marbn said.

Understanding precisely what is going on will take much more work to sort out, he said. For example, lengthening the protective caps of chromosomes, or telomeres, is presumably caused by production of telomerase, an enzyme that makes them longer. But how?

Knowing the exosomes are involved doesnt narrow it down very much, he said.

We think that there's thousands and thousands of different bioactive molecules within exosomes. And so I can't right now point to, let's say, these five RNAs and say, they're the ones that we think are doing the trick, Marbn said. But somewhere in the genetic instructions in the exosomes are signals that cause telomerase to be activated and elongation of the telomeres.

Even without understanding the precise mechanism, the demonstrated results have been promising enough for Capricor to continue clinical testing in Duchenne muscular dystrophy, Marbn said, even though its outside the companys initial focus on heart disease.

The heart attack research gave mixed messages, he said. Capricor isnt abandoning it, but has given priority to the muscular dystrophy program.

Duchenne muscular dystrophy patients and their parents are more interested in increasing skeletal muscle function than heart function, he said. The disease virtually exclusively affects males, and they often die when quite young.

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Young cardiac cells rejuvenate heart in animal study - The San Diego Union-Tribune

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A human embryo kept alive in the lab for 12 days begins to show signs of early development. The green cells seen here in the center would go on to form the body. This embryo is in the process of twinning, forming two small spheres out of one. Courtesy of Gist Croft, Cecilia Pellegrini, Ali Brivanlou/Rockefeller University hide caption

Four sheep cloned from the same genetic material as Dolly roam the paddocks in Nottingham, England. The University of Nottingham hide caption

Ken (left) and Henry were created using DNA plucked from a skin cell of Melvin, the beloved pet of Paula and Phillip Dupont of Lafayette, La. Edmund D. Fountain for NPR hide caption

In 1954, Dr. Frederick C. Robbins, then chief of pediatrics and contagious diseases at Cleveland Metropolitan General Hospital, was one of three winners of that year's Nobel Prize in medicine. The scientists' work, which led to a vaccine against polio, was performed in human fetal cells. AP hide caption

Ryoji Noyori, a Nobel Prize-winning chemist and president of Japan's prestigious RIKEN research institute, bows at a news conference in Tokyo Tuesday to apologize for the scientific misconduct of a RIKEN colleague. Eugene Hoshiko/AP hide caption

A mouse embryo grows from stem cells made by stressing blood cells with acid. The blood cells are tagged with a protein that creates green light. Courtesy of Haruko Obokata hide caption

After President Obama overturned Bush-era policy restricting federal funding of embryonic stem cell research in 2009, Nebraska Right to Life led a protest of the research outside the University of Nebraska regents' meeting. Nati Harnik/AP hide caption

Human embryos grow in a petri dish two days after scientists in Oregon cloned them from a donor's skin cell. http://www.flickr.com/photos/ohsunews/8726915230/in/photostream//Courtesy of OHSU Photos hide caption

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embryonic stem cells : NPR

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