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Blood and Bone Marrow Transplant – NHLBI, NIH

By NEVAGiles23

When the healthy stem cells come from you, the procedure is called an autologous transplant. When the stem cells come from another person, called a donor, it is an allogeneic transplant. Blood or bone marrow transplants most commonly are used to treat blood cancers or other kinds of blood diseases that decrease the number of healthy blood cells in the body. These transplants also may be used to treat other disorders.

For allogeneic transplants, your doctor will try to find a donor whose blood cells are the best match for you. Your doctor will consider using cells from your close family members, from people who are not related to you and who have registered with the National Marrow Donor Program, or from publicly stored umbilical cord blood. Although it is best to find a donor who is an exact match to you, new transplant procedures are making it possible to use donors who are not an exact match.

Blood or bone marrow transplants are usually performed in a hospital. Often, you must stay in the hospital for one to two weeks before the transplant to prepare. During this time, you will have a narrow tube placed in one of your large veins. You may be given medicine to make you sleepy for this procedure. You also will receive special medicines and possibly radiation to destroy your abnormal stem cells and to weaken your immune system so that it wont reject the donor cells after the transplant.

On the day of the transplant, you will be awake and may get medicine to relax you during the procedure. The stem cells will be given to you through the narrow tube in your vein. The stem cells will travel through your blood to your bone marrow, where they will begin making new healthy blood cells.

After the transplant, your doctor will check your blood counts every day to see if new blood cells have started to grow in your bone marrow. Depending on the type of transplant, you may be able to leave, but stay near the hospital, or you may need to remain in the hospital for weeks or months. The length of time will depend on how your immune system is recovering and whether or not the transplanted cells stay in your body. Before you leave the hospital, the doctors will give you detailed instructions that you must follow to prevent infection and other complications. Your doctor will keep monitoring your recovery, possibly for up to oneyear.

Although blood or bone marrow transplant is an effective treatment for some conditions, the procedure can cause early or late complications. The required medicines and radiation can cause nausea, vomiting, diarrhea, tiredness, mouth sores, skin rashes, hair loss, or liver damage. These treatments also can weaken your immune system and increase your risk for infection. Some people may experience a serious complication called graft-versus-host disease if the donated stem cells attack the body. Other people may reject the donor stem cells after the transplant, which can be an extremely serious complication.

VisitBlood-Forming Stem Cell Transplantsfor more information about this topic.

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Regeneration of the entire human skin using transgenic …

By Sykes24Tracey

Epidermolysis bullosais is rare, but the charity DEBRA, which campaigns for EB patients, estimates half a million people are affected around the world.

There are different forms of epidermolysis bullosa, including simplex, dystrophic and, as in this case, junctional.

Each is caused by different genetic faults leading to different building blocks of skin being missing.

Prof Michele De Luca, from the University of Modena and Reggio Emilia, told the BBC: The gene is different, the protein is different and the outcome may be different [for each form of EB] so we need formal clinical trials.

But if they can make it work, it could be a therapy that lasts a lifetime.

An analysis of the structure of the skin of the first patient to get 80% of his replaced has discovered a group of long-lived stem cells are that constantly renewing his genetically modified skin.

Genetically modified skin cells were grown to make skin grafts totalling 0.85 sq m (9 sq ft). It took three operations over that winter to cover 80% of the childs body in the new skin. But 21 months later, the skin is functioning normally with no sign of blistering.

Nature Regeneration of the entire human epidermis using transgenic stem cells

Junctional epidermolysis bullosa (JEB) is a severe and often lethal genetic disease caused by mutations in genes encoding the basement membrane component laminin-332. Surviving patients with JEB develop chronic wounds to the skin and mucosa, which impair their quality of life and lead to skin cancer. Here we show that autologous transgenic keratinocyte cultures regenerated an entire, fully functional epidermis on a seven-year-old child suffering from a devastating, life-threatening form of JEB. The proviral integration pattern was maintained in vivo and epidermal renewal did not cause any clonal selection. Clonal tracing showed that the human epidermis is sustained not by equipotent progenitors, but by a limited number of long-lived stem cells, detected as holoclones, that can extensively self-renew in vitro and in vivo and produce progenitors that replenish terminally differentiated keratinocytes. This study provides a blueprint that can be applied to other stem cell-mediated combined ex vivo cell and gene therapies

SOURCES BBC News, Nature

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Fully Functional Skin Grown From Stem Cells Could Double …

By Sykes24Tracey

If theres one thing skin can do well, its grow. Each month our body replaces its skin,nearly 19 million skin cells per inch a feat thats been far less successful in the lab. However, the days of lab-grown skin may not be too far off:Recently, a team of Japanese scientists not only grew fully functional skin tissue, but also transplanted it successfully onto living organisms.

Though the technique has only been tested on mice so far, the team predicts it could one day revolutionize treatments for burn victims, or other patients that have suffered catastrophic skin damage. On a less gruesome note, the team says it may also be useful in treating a more common condition: baldness.

The study, published online in Science Advances, involved researchers from the Riken Center for Developmental Biology and Tokyo University of Science, among other Japanese institutions. The researchers first step was to transform cells from the gums of mice into induced pluripotent stem cells, or adult cells that have been genetically reprogrammed back into an embryonic stem cell state. This is done by forcing the cells to express genes associated with embryonic stem cells. Once transformed into stem cells, they can then be manipulated to become any type of cell in the body.

Next, the team placed the stem cells into a petri dish, where they added the molecule Wnt10b, which coaxed the stem cells to form into clusters that resembled a developing embryo. These clusters were then transplanted into mice bred without a fully functional immune system, which ensured that their bodies did not reject the transplant. Here, they underwent cell differentiation, the process by which unspecialized cells become specialized. In this case, they were becoming skin cells, and once the process had begun, the cells were transplanted again onto the skin of new mice, where they made normal connections with surrounding nerve and muscle tissue to become fully functional skin.

Skin is one of the largest and most important organs in the human body, yet its also one of the most difficult to treat when its damaged. Current treatment options involve painful skin grafts or barely functional artificial skin. According to the new study, however, being able to grow skin in the lab will account for more than just skin's use in protecting our inner bodies. The lab-grown skin also showed the ability to develop hair follicles and sweat glands, which play a role in controlling body temperature and keeping the skin moisturized it's in these areas that skin repair has often fallen short.

"Up until now, artificial skin development has been hampered by the fact that the skin lacked the important organs, such as hair follicles and exocrine glands, lead researcher, Takashi Tsuji of the RIKEN Center for Developmental Biology,said in a recent statement. With this new technique, we have successfully grown skin that replicates the function of normal tissue.

In addition to revolutionizing skin repair, the technique may also help those with certain types of hair loss. The study noted that using Wnet10b on the stem cells resulted in the production of a higher number of hair follicles than previous attempts at growing skin. Within two weeks of receiving the transplanted skin, the mice began to grow hair. Dr. Seth Orlow, chair of dermatology at NYU School of Medicine in New York City, told U.S. News Health that this feature of the lab-grown skin could be manipulated to help patients with both alopecia and pattern baldness.

In theory, we may eventually be able to create structures like hair follicles and other skin glands that could be transplanted back to people who need them, Orlow told U.S. Health News.

According to The Washington Post, the technique is still about five to 10 years away from being safe and effective enough to be used on humans. But with about 95 percent of men and 50 percent of women experiencing some degree of baldness over the course of their lives, its a safe bet that there will be no shortage of eager customers ready to get their hair back when the treatment is approved for use in doctors offices.

Source: Takagi R, Ishimaru J, Sugawara A, et al. Bioengineering a 3D integumentary organ system from iPS cells using an in vivo transplantation model. Science Advances . 2016

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"Latest Stem Cells News" – news from the world about stem …

By NEVAGiles23

To meet the industry needs and to benefit students and research scholars, Nitte University has set up the a centre for stem cell research at K S Hedge Medical Academy (Kshema).

The Nitte University Centre for Stem Cell Research and Regenerative Medicine (NUCSReM), has been established to further advance the understanding of stem cell biology and to facilitate clinical application of stem cells to treat patients with various ailments, says N Vinaya Hegde, chancellor, Nitte University.

Gianvito Martino, the head of the Neurosciences division at the Institute of San Raffaele in Milan in a speech at Multiple Sclerosis Week, which took place from May 23-31, warned against trips of hope to clinics that promise effective treatments using stem cells.

According to Martino, who coordinated a Consensus Conference on last Tuesday in London on the neurodegenerative disease, where the guidelines for pre-clinical studies and clinical treatments with stem cells were defined, hundreds of Italian patients each year go on these trips due to cures that are promised. In the best-case scenario, these patients return in the Read More

Scientists have claimed they would serve the worlds first test tube hamburger this October.

A team, led by Prof Mark Post of Maastricht University in the Netherlands, says it has already grown artificial meat in the laboratory, and now aims to create a hamburger, identical to a real stuff, by generating strips of meat from stem cells.

The finished product is expected to cost nearly 220,000 pounds, The Daily Telegraph reported.

Prof Post said his team has successfully replicated the process with cow cells and calf serum, bringing the first artificial burger a step closer.

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Studies begun by Harvard Stem Cell Institute (HSCI) scientists eight years ago have led to a report published today that may be amount to a major step in developing treatments for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrigs disease.

The findings by Kevin Eggan, a professor in Harvards Department of Stem Cell and Regenerative Biology (HSCRB), and colleagues also has produced functionally identical results in human motor neurons in a laboratory dish and in a mouse model of the disease, demonstrating that modeling the human disease with customized stem cells in the laboratory could relatively soon eliminate some Read More

Frank LaFerla, left, Mathew Blurton-Jones and colleagues found that neural stem cells could be a potential treatment for advanced Alzheimer's disease

UC Irvine scientists have shown for the first time that neural stem cells can rescue memory in mice with advanced Alzheimers disease, raising hopes of a potential treatment for the leading cause of elderly dementia that afflicts 5.3 million people in the U.S.

Mice genetically engineered to have Alzheimers performed markedly better on memory tests a month after mouse neural stem cells were injected into their brains. The stem cells secreted a protein that created more neural connections, improving Read More

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What are stem cells and how will they be used to treat the …

By Sykes24Tracey

Stem cell research is often controversial but it has also led to incredible medical progress in recent years.

Stem cell research is at defining moment. Although it can be controversial and does raise a lot of important ethical issues, this area of medical science has been characterised by a number of important advances, ever since the first embryonic stem cells were isolated from mice in the 1980s. In the near future, it could reshape the way we treat some of the worlds most debilitating diseases.

Stem cells have already been used as treatment for a number of years think bone marrow transplant and they have the potential to help with many other medical conditions. They could also prove crucial for scientists wishing to understand more about human biology and development.

Studies using stem cells have benefited from important media coverage in recent years and many of them hailed as breakthroughs. However, the reality is often more complex, and a number of scientific and ethical challenges often stand in the way of successes in animal models being replicated in humans.

IBTimes UK takes a look at what stem cell research is, what it is used for and what the future looks like.

Stem cells could be defined as building block cells that have not yet differentiated into one cell type and could develop into many different cell types. Stem cells can continue to divide almost indenitely.

There are two main types of stem cells: embryonic stem cells and adult stem cells.

Embryonic stem cells were first isolated in mice in the early 1980s at the University of Cambridge. All developing embryos contains a number of stem cells that can go on to develop into different cell types. In humans, these cells can be isolated from around five days after the egg has been fertilised around 50 to 100 stem cells are present at that stage.

These cells are isolated from embryos that have been donated by couples who have been through IVF and have extra embryos left which were not used during the treatment.

Stem cells are also found in adults, particularly in the bone marrow, the blood, the eyes, the brain and the muscles. They are also known as somatic stem cells.

They can also differentiate into other cells, but into a much more limited number than embryonic stem cells. They range from cells that are able to form different kinds of tissues to more specialised cells that form just some of the cells of a particular tissue or organ. They also have the ability to divide and reproduce indefinitely.

19th Place: Dr Gist F Croft, Lauren Pietilla, Stephanie Tse, Dr. Szilvia Galgoczi, Maria Fenner, Dr Ali H. Brivanlou, Rockefeller University, Brivanlou Laboratory New York, New York, USA: Human neural rosette primordial brain cells, differentiated from embryonic stem cells Confocal, 10x (Dr Gist F Croft, Lauren Pietilla, Stephanie Tse, Dr. Szilvia Galgoczi, Maria Fenner, Dr Ali H. Brivanlou)

Scientists have also found a way to make induced pluripotent stem cells cells taken from any adult tissue and genetically modified to behave like an embryonic stem cell (and thus able to differentiate into any cell type). The term pluripotant refers to the fact that the stem cells can produce almost all of the cells in the body.

To create these induced pluripotent stem cells, researchershave learnt to reprogramme the genes of human adult cells. A major 2007 US study, found that introducing 14 genes could reprogramme the cells to become stem cells, and the researchers then narrowed this down to four genes. Subsequent studies have built on this knowledge to find new, safer ways to turn adult cells into pluripotant stem cells.

Stem cells are already used to help a number of patients around the world. For nearly 50 years, they have been used in the form of bone marrow transplants.

Indeed, bone marrow contains stem cells that can produce many different blood cells. A bone marrow transplant can be used to treat people with blood cancers or genetic blood disorders, such as sickle cell anaemia. The stem cell turn into healthy blood cells that can help the patient. Some hospitals also use stem cells to grow skin grafts for patients with life-threatening burns. It is also possible to receive a stem cell therapy based on limbal stem cells (in the eye) to repair damaged corneas.

Stem cells are also very useful for scientists conducting basic research on diseases, as they can be used to model a large number of conditions. Recent studies have used stem cells to model the nerve cells that are lost in Alzheimers disease or to model deafness or Autism Spectrum disorder.

Scientists have gained a better understanding of blood stem cells (Alden Chadwick/Flickr)

A number of treatment using stem cells has been tested by researchers around the world. A type of patients that could be helped by stems cells are those suffering from spinal cord injuries. Stem cell therapy for spinal cord repair could be used to promote the growth of nerve cells directly or to transplant cells that protect the nerves and help them function.

One of most important studies in this area was published in October 2010. tested the used embryonic stem cells on patients in the US who had sustained a spinal cord injury in the previous 14 days. Preliminary findings were encouraging.

Studies have also been conducted to assess the safety and efficacy of stem cells in helping patients who suffered a stroke. The idea is that stem cells could help in rehabilitation after a persons brain has been damaged by the stroke. Stem cells have also been investigated to treat diseases such as MS, diabetes and to reverse ageing.

Beyond clinical trials, which still remain limited in number, many of the preliminary research opens up a number of very interesting perspectives. One of the main area of interest is growing organs in the lab from tissues created from stem cells. These organs may one day be used for transplantation in humans.

Recently, stem cells have been shown to present an interest to improve fertility treatments with the creation of a new technique in mice in-vitro gametogenesis. The idea is to create eggs and sperm using pluripotant stem cells.

By La Surugue

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Glossary of Terms | Aplastic Anemia and MDS International …

By JoanneRUSSELL25

acute myelogenous leukemia

(uh-KYOOT my-uh-LAH-juh-nuss loo-KEE-mee-uh) A cancer of the blood cells. It happens when very young white blood cells (blasts) in the bone marrow fail to mature. The blast cells stay in the bone marrow and become to numerous. This slows production of red blood cells and platelets. Some cases of MDS become AML. But most do not. Also called AML, acute myeloblastic leukemia, acute myelocytic leukemia, acute myeloid leukemia.

A procedure where bone marrow stem cells are taken from a genetically matched donor (a brother, sister, or unrelated donor) and given to the patient through an intravenous (IV) line. In time, donated stem cells start making new, healthy blood cells.

See complementary and alternative medicine.

(an-uh-fuh-LAK-suss) A very severe allergic reaction to a foreign protein, as in a bee sting, or to a medicine. This reaction causes the blood pressure to drop and trouble breathing. Before a patient receives ATG, a treatment for aplastic anemia, a skin test is given to find out if they are likely to develop anaphylaxis. Also known as anaphylactic shock.

An approach to treating bone marrow failure using natural male hormones. Androgen therapy can help the bone marrow make more blood cells. This is an older treatment for bone marrow failure that is rarely used because of the side effects. Scientists are studying these medicines to try to better understand why they work in some cases of acquired and genetic bone marrow failure.

(uh-NEE-mee-uh) A condition in which there is a shortage of red blood cells in the bloodstream. This causes a low red blood cell count. Symptoms of anemia are fatigue and tiredness.

(an-tee-by-AH-tik) A medicine that fights bacterial infections. When a person with bone marrow failure does not have enough neutrophils, the white blood cells that fight infection, antibiotics may help to prevent and fight infection.

(ant-i-ko-AG-yuh-lunt) See blood thinner.

(ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells: red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most scientists believe that aplastic anemia happens when the immune system attacks the bone marrow stem cells. Aplastic anemia can be acquired (begin any time in life) or can be hereditary (less common, passed down from parent to child).

Programmed cell death.

(uh-SITE-eez) Extra fluid and swelling in the belly area (abdomen). Also called hydroperitoneum.

Any condition that happens when the immune system attacks the body's own normal tissues by mistake.

A procedure in which some of the patient's own bone marrow stem cells are removed, frozen, and then returned to the through an intravenous (IV) line. In time, the stem cells start making new, healthy blood cells.

Describes one of several ways that a trait or disorder can be inherited, or passed down through families. "Autosomal" means that the mutated, or abnormal, gene is located on one of the numbered, or non-sex, chromosomes. "Dominant" means that only one copy of the mutated gene is enough to cause the disease. Dyskeratosis congenita is a rare cause of bone marrow failure disease. It may have an autosomal dominant, autosomal recessive or x-linked pattern of inheritance.

Describes one of several ways that a trait or disorder can be inherited, or passed down through families. "Autosomal" means that the mutated, or abnormal, gene is located on one of the numbered, or non-sex, chromosomes. "Recessive" means that two copies of a mutated gene must be present to cause the disease. Dyskeratosis congenita is a rare cause of bone marrow failure. It may have an autosomal dominant, autosomal recessive or x-linked pattern of inheritance.

The study of a subject to increase knowledge and understanding about it. The goal of basic research in medicine is to better understand disease. In the laboratory, basic research scientists study changes in cells and molecules linked to disease. Basic research helps lead to better ways of diagnosing, treating, and preventing disease. Also called basic science research.

A type of white blood cell that plays a role in allergic reactions.

A chemical that is widely used by the chemical industry in the United States to make plastics, resins, nylon and synthetic fibers. Benzene is found in tobacco smoke, vehicle emissions, and gasoline fumes. Exposure to benzene may increase the risk of developing a bone marrow failure disease. Benzene can affect human health by causing bone marrow stem cells not to work correctly.

(bil-i-ROO-bun) A reddish yellow substance formed when red blood cells break apart. It is found in the bile and in the blood. Yellowing of the skin and eyes can occur with high levels of bilirubin. Also called total bilirubin.

A substance made from a living system, such as a virus, and used to prevent or treat disease. Biological drugs include antibodies, globulin, interleukins, serum, and vaccines. Also called a biologic or biological drug.

A young white blood cell. The number of blast cells in the bone marrow helps define how severe MDS is in a person. When 20 out of 100 cells in the bone marrow are blasts, this is considered acute myeloid leukemia.

See Blast Cells.

A mass of blood that forms when platelets stick together. Harmful blood clots are more likely to happen in PNH. The term thrombus describes a blood clot that develops and attaches to a blood vessel. The term embolus describes a blood clot or other foreign matter that gets into the bloodstream and gets stuck in a blood vessel.

A medicine used to stop blood clots from forming. Blood thinners can be used to treat or prevent clots. Some common blood thinners are enoxaprin (Lovenox), heparin (Calciparine or Liquaemin), and warfarin (Coumadin). Also called and anticoagulant or thrombopoiesis inhibitor.

A procedure in which whole blood or one of its components is given to a person through an intravenous (IV) line into the bloodstream. A red blood cell transfusion or a platelet transfuson can help some patients with low blood counts.

The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.

A medical procedure to remove of a small amount of liquid bone marrow through a needle inserted into the back of the hip. The liquid bone marrow is examined for abnormalities in cell size, shape, or look. Tests may also be run on the bone marrow cells to look for any genetic abnormalities.

A medical procedure to remove a small piece of solid bone marrow using a needle that goes into the marrow of the hip bone. The solid bone marrow is examined for cell abnormalities, the number of different cells and checked for scaring of the bone marrow.

A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be hereditary (less common, passed down from parent to child).

A procedure where bone marrow stem cells are collected from marrow inside the donor's hipbone and given to the patient through an intravenous (IV) line. In time, donated stem cells start making new, healthy blood cells.

(bud-kee-AR-ee SIN-drome) A blood clot in the major vein that leaves the liver (hepatic vein). The liver and the spleen may become enlarged. Budd-Chiari syndrome can occur in PNH.

How much of the bone marrow volume is occupied by various types of blood cells.

(kee-moe-THER-uh-pee) The use of medicines that kill cells (cytotoxic agents). People with high-risk or intermediate-2 risk myelodysplastic syndrome (MDS) may be given chemotherapy to kill bone marrow cells that have an abnormal size, shape, or look. Chemotherapy hurts healthy cells along with abnormal cells. If chemotherapy works in controlling abnormal cells, then relatively normal blood cells will start to grow again. Low-dose chemotherapy agents include: cytarabine (Ara-C) and hydroxyurea (Hydrea). High-dose chemotherapy agents include: daunorubicin (Cerubidine), idarubicin (Idamycin), and mitoxanrone (Novantrone).

The part of the cell that contains our DNA or genetic code.

A medical condition that lasts a long time. A chronic illness can affect a person's lifestyle, ability to work, physical abilities and independence.

A person who gives advice, or counsel, to people who are coping with long-term illness. A chronic illness counselor helps people understand their abilities and limitations, cope with the stress, pain, and fatigue associated with long-term illness. A chronic illness counselor can often be located by contacting a local hospital.

A type of research that involves individual persons or a group of people. There are three types of clinical research. Patient-oriented research includes clinical trials which test how a drug, medical device, or treatment approach works in people. Epidemiology or behavioral studies look at the patterns and causes of disease in groups of people. Outcomes and health services research seeks to find the most effective treatments and health services.

A type of research study that tests how a drug, medical device, or treatment approach works in people. There are several types of clinical trials. Treatment trials test new treatment options. Diagnostic trials test new ways to diagnose a disease. Screening trials test the best way to detect a disease or health problem. Quality of life (supportive care) trials study ways to improve the comfort of people with chronic illness. Prevention trials look for better ways to prevent disease in people who have never had the disease.

Trials are in four phases: Phase I tests a new drug or treatment in a small group to see if it is safe. Phase II expands the study to a larger group of people to find out if it works. Phase III expands the study to an even larger group of people to compare it to the standard treatment for the disease; and Phase IV takes place after the drug or treatment has been licensed and marketed to find out the long-term impact of the new treatment.

To make copies. Bone marrow stem cells clone themselves all the time. The cloned stem cells eventually become mature blood cells that leave the bone marrow and enter the bloodstream.

To thicken. Normal blood platelets cause the blood to coagulate and stop bleeding.

A group of proteins that move freely in the bloodstream. These proteins support (complement) the work of white blood cells by fighting infections.

A medical approach that is not currently part of standard practice. Complementary medicine is used along with standard medicine. Alternative medicine is used in place of standard medicine. Example of CAM therapies are acupuncture, chiropractic, homeopathic, and herbal medicines. There is no complementary or alternative therapy that effectively treats bone marrow failure. Some CAM therapies may even hinder the effectiveness of standard medical care. Patients should talk with their doctor if they are currently using or considering using a complementary or alternative therapy.

A group of tests performed on a small amount of blood. The CBC measures the number of each blood cell type, the size of the red blood cells, the total amount of hemoglobin, and the fraction of the blood made up of red blood cells. Also called a CBC.

A procedure where umbillical cord stem cells are given to the patient through an intravenous (IV) line. Stem cells are collected from an umbilical cord right after the birth of a baby. They are kept frozen until needed. In time, donated stem cells given to the patient begin making new, healthy blood cells.

An imaging technique using x-ray technology and computerization to create a three-dimentional image of a body part. Also called a CT scan, it can be used to locate a blood clot in the body.

A response to treatment indicating that no sign of abnormal chromosomes are found. When a test is done on a patient with 5q deletion MDS, and there are no signs of an abnormal chromosome 5, then that patient has achieved a cytogenetic remission. Also called cytegenetic response.

(sie-toe-juh-NEH-tiks) The study of chromosomes (DNA), the part of the cell that contains genetic information. Some cytogenetic abnormalities are linked to different forms of myelodysplastic syndromes (MDS).

(sie-tuh-PEE-nee-uh) A shortage of one or more blood cell types. Also called a low blood count.

(sie-tuh-TOK-sik) A medicine that kills certain cells. Chemotherapy for MDS patients often involves the use of cytotoxic agents.

A test that helps doctors find out if a person has a problem with blood clotting.

(di-NO-vo) Brand new, referring to the first time something occurs. MDS that is untreated or that has no known cause is called de novo MDS.

The death of part of the intestine. This can happen if the blood supply in the intestine is cut off, for example, from a blood clot in the abdomen. Also called intestinal necrosis, ischemic bowel, dead gut.

A rare form of pure red cell aplasia that can be passed down from parent to child. Diamond-Blackfan anemia (DBA) is characterized by low red blood cell counts detected in the first year of life. Some people with DBA have physical abnormalities such as small head size, low frontal hairline, wide-set eyes, low-set ears. Genetic testing is used to diagnose DBA.

Vitamins, minerals, herbs and other substances meant to improve your nutritional intake. Dietary supplements are taken by mouth in the form of a pill, capsule, tablet or liquid.

To become distinct or specialized. In the bone marrow, young parent cells (stem cells) develop, or differentiate, into specific types of blood cells (red cells, white cells, platelets).

The gene that always expresses itself over a recessive gene. A person with a dominant gene for a disease has the symptoms of the disease. They can pass the disease on to children.

An inherited disease that may lead to bone marrow failure.

Refers to how well a graft (donor cells) is accepted by the host (the patient) after a bone marrow or stem cell transplant. Several factors contribute to better engraftment: physical condition of the patient, how severe the disease is, type of donor available, age of patient. Successful engraftment results in new bone marrow that produces healthy blood cells.

A type of white blood cell that kills parasites and plays a role in allergic reactions.

The study of patterns and causes of disease in groups of people. Epidemiology researchers study how many people have a disease, how many new cases are diagnosed each year, where patients are located, and environmental or other factors that influence disease.

(i-RITH-ruh-site) See red blood cell.

(i-rith-row-POY-uh-tun) A protein made by the kidneys. Erythropoietin, also called EPO, is created in response to low oxygen levels in the body (anemia). EPO causes the bone marrow to make more red blood cells. A shortage of EPO can also cause anemia.

A medicine used to help the bone marrow make more red blood cells. Epoetin alfa (Epogen, Procrit) and darbepoetin alfa (Aranesp) are erythropoietin-stimulating agents that can help boost the red blood cell count of some bone marrow failure patients. Also called red blood cell growth factor.

A form of estrogen, it is the most potent female hormone. It is also present in males. Estradiol is involved in many body functions beyond the reproductive system. Researchers are investigating the role of estradiol in the treatment of genetic bone marrow failure.

The cause or origin of a disease.

A criteria used for classifying different types of myelodysplastic syndromes (MDS). The FAB (French, American, British) Classification System was developed by a group of French, American and British scientists. This system is based on 2 main factors: the percentage of blast cells in bone marrow, and the percentage of blast cells in the bloodstream. The FAB system is somewhat outdated, but is still used by some doctors today. The World Health Organization (WHO) Classification System has largely replaced the FAB Classification System.

A rare inherited disorder that happens when the bone marrow does not make enough blood cells: red cells, white cells, and platelets. Fanconi anemia is diagnosed early in life. People with Fanconi anemia have a high likelihood of developing cancer. Genetic testing is used to diagnose Fanconi anemia.

(FER-i-tin) A protein inside of cells that stores iron for later use by your body. Sometimes ferritin is released into the blood. The ferritin level in the blood is called serum ferritin.

(FER-i-tin) A blood test used to monitor how much iron the body is storing for later use.

(fie-BRO-suss) Scarring of tissue. Fibrosis of the bone marrow is an feature seen in some types of unclassified myeldysplastic syndrome (MDS).

See fluorescence in situ hybridization.

(sy-TOM-uh-tree) A laboratory test that gives information about cells, such as size, shape, and percentage of live cells. Flow cytometry is the test doctors use to see if there are any proteins missing from the surface of blood cells. It is the standard test for confirming a diagnosis of paroxysmal nocturnal hemoglobinuria (PNH).

(flor-EH-sense in SIT-tyoo hy-bru-duh-ZAY-shun) An important laboratory test used to help doctors look for chromosomal abnormalities and other genetic mutations. Fluorescence in situ hybridization, also called FISH, directs colored light under a microscope at parts of chromosomes or genes. Missing or rearranged chromosomes are identified using FISH.

(FOE-late) A B-vitamin that is found in fresh or lightly cooked green vegetables. It helps the bone marrow make normal blood cells. Most people get enough folate in their diet. Doctors may have people with paroxysmal nocturnal hemaglobinuria (PNH) take a man-made form of folate called folic acid.

See folate.

A laboratory test that looks at the whether red blood cells break apart too easily when they are placed in mild acid. This test has been used in the past to diagnose paroxysmal nocturnal hemoglobinuria (PNH). Most doctors now use flow cytometry, a more accurate method of testing for PNH. Ham Test is also called acid hemolysin test.

(hi-MA-tuh-crit) A blood test that measures the percentage of the blood made up of red blood cells. This measurement depends on the number of red blood cells and their size. Hematocrit is part of a complete blood count. Also called HCT, packed cell volume, PCV.

(hee-muh-TOL-uh-jist) A doctor who specializes in treating blood diseases and disorders of blood producing organs.

(hi-mat-uh-poy-EE-suss) The process of making blood cells in the bone marrow.

A condition that occurs when the body absorbs and stores too much iron. This leads to a condition called iron overload. In the United States, hemochromatosis is usually caused by a genetic disorder. Organ damage can occur if iron overload is not treated.

A protein in the red blood cells. Hemoglobin picks up oxygen in the lungs and brings it to cells in all parts of the body.

(hee-muh-gloe-buh-NYOOR-ee-uh) The presence of hemoglobin in the urine.

(hi-MOL-uh-suss) The destruction of red blood cells.

See human leukocyte antigen.

A part of the endocrine system that serves as the body's chemical messengers. Hormones move through the bloodstream to transfer information and instruction from one set of cells to another.

(LEW-kuh-site ANT-i-jun) One of a group of proteins found on the surface of white blood cells and other cells. These antigens differ from person to person. A human leukocyte antigen test is done before a stem cell transplant to closely match a donor and a recipient. Also called HLA.

A condition in which there are too many cells, for example, within the bone marrow. Patients with leukemia have hypercellular bone marrow filled with to many immature white blood cells.

A condition in which there are too few cells, for example, within the bone marrow. Patients with aplastic anemia have hypocellular bone marrow.

Usually refers to any condition with no known cause.

(i-myoo-no-KOM-pruh-mized) Occurs when the immune system is not functioning properly, leaving the patient open to infection. A person can be immunocompromised due to low white blood cell count or due to some medicines. Also called immune compromised.

(i-myoo-no-suh-PREH-siv) Drugs that lower the body's immune response and allow the bone marrow stem cells to grow and make new blood cells. ATG (antithymocyte globulin) or ALG (antilymphocyte globulin) with cyclosporine are used to treat bone marrow failure in aplastic anemia. Immunosuppressive drugs may help some patients with myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH).

A committee that makes sure a clinical trial is safe for patients in the study. Each medical center, hospital, or research facility doing clinical trials must have an active Institutional Review Board (IRB). Each IRB is made up of a diverse group of doctors, faculty, staff and students at a specific institution.

A system that turns patient data into a score. The score tells how quickly a myelodysplastic syndrome (MDS) case is progressing and helps predict what may happen with the patient's MDS in the future. Also called IPSS.

A method of getting fluids or medicines directly into the bloodstream over a period of time. Also called IV infusion.

A new drug, antibiotic drug, or biological drug that is used in a clinical trial. It also includes a biological product used in the laboratory for diagnostic purposes. Also called IND.

(kee-LAY-shun) A drug therapy to remove extra iron from the body. Patients with high blood iron (ferritin) levels may receive iron chelation therapy. The U.S. Food and Drug Administratin (FDA) has approved two iron chelators to treat iron overload in the U.S.: deferasirox, an oral iron chelator, and deferoxamine, a liquid given by injection.

A condition that occurs when too much iron accumulates in the body. Bone marrow failure disease patients who need regular red blood cell transfusions are at risk for iron overload. Organ damage can occur if iron overload is not treated.

(iss-KEE-mee-uh) Occurs when the blood supply to specific organ or part of the body is cut off, causing a localized lack of oxygen.

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Stem Cell-Based Therapy for Cartilage Regeneration and …

By Sykes24Tracey

Our initial application established the goals of our project and the reasons for our study. Arthritis is the result of degeneration of cartilage (the tissue lining the joints) and leads to pain and limitation of function. Arthritis and other rheumatic diseases are among the most common of all health conditions and are the number one cause of disability in the United States. The annual economic impact of arthritis in the U.S. is estimated at over $120 billion, representing more than 2% of the gross domestic product. The prevalence of arthritic conditions is also expected to increase as the population increases and ages in the coming decades. Current treatment options for osteoarthritis are limited to pain reduction and joint replacement surgery. Stem cells have tremendous potential for treating disease and replacing or regenerating the diseased tissue. In this project our objective is to use cells derived from stems cells to treat arthritis. We have completed our experiments as per our proposed timeline and have met milestones outlined in our grant submission. We have established conditions for converting stem cells into cartilage tissue cells that can repair bone and cartilage defects in laboratory models. We have identified several cell lines with the highest potential for tissue repair. We optimized culture conditions to generate the highest quality of tissue. In our initial experiments we found no evidence of cell rejection response in vivo. We have testing efficacy of the most promising cell lines in regenerating healthy repair tissue in cartilage defects and have selected a preclinical candidate.The next step is to plan safety and efficacy studies for the preclinical phase, identify collaborators with the facilities to obtain, process, and provide cell-based therapies, and identify clinical collaborators in anticipation of clinical trials. If necessary we will also identify commercialization partners. We also anticipate that stem cells implanted in arthritic cartilage will treat the arthritis in addition to producing tissue to heal the defect in the cartilage. An approach that heals cartilage defects as well as treats the underlying arthritis would be very valuable. If our research is successful, this could lead to first treatment of osteoarthritis that alters the progression of the disease. This treatment would have a huge impact on the large numbers of patients who suffer from arthritis as well as in reducing the significant economic burden created by arthritis.

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Genetically modified skin grown from stem cells saved a 7 …

By NEVAGiles23

Scientists reported Wednesday that they genetically modified stem cells to grow skin that they successfully grafted over nearly all of a child's body - a remarkable achievement that could revolutionize treatment of burn victims and people with skin diseases.

The research, published in the journal Nature, involved a 7-year-old boy who suffers from a genetic disease known as junctional epidermolysis bullosa (JEB) that makes skin so fragile that minor friction such as rubbing causes the skin to blister or come apart.

By the time the boy arrived at Children's Hospital of Ruhr-University in Germany in 2015, he was gravely ill. Doctors noted that he had "complete epidural loss" on about 60 percent of his body surface area, was in so much pain that he was on morphine, and fighting off a systemic staph infection. The doctors tried everything they could think of: Antibiotics, changing dressings, grafting skin donated by his father. But nothing worked, and they told his parents to prepare for the worst.

"We had a lot of problems in the first days keeping this kid alive," Tobias Hirsch, one of the treating physicians, recalled in a conference call with reporters this week.

Hirsch and his colleague Tobias Rothoeft began to scour the medical literature for anything that might help and came across an article describing a highly experimental procedure to genetically engineer skin cells. They contacted the author, Michele De Luca, of the Center for Regenerative Medicine University of Modena and Reggio Emilia in Italy. De Luca flew out right away.

Using a technique he had used only twice before and even then only on small parts of the body, De Luca harvested cells from a four-square-centimeter patch of skin on an unaffected part of the boy's body and brought them into the lab. There, he genetically modified them so that they no longer contained the mutated form of a gene known to cause the disease and grew the cells into patches of genetically modified epidermis. They discovered, the researchers reported, that "the human epidermis is sustained by a limited number of long-lived stem cells which are able to extensively self-renew."

In three surgeries, the child's doctors took that lab-grown skin and used it to cover nearly 80 percent of the boy's body - mostly on the limbs and on his back, which had suffered the most damage. The procedure was permitted under a "compassionate use" exception that allows researchers under certain dire circumstances to make a treatment available even though it is not approved by regulators for general use. Then, over the course of the next eight months while the child was in the intensive care unit, they watched and waited.

The boy's recovery was stunning.

The regenerated epidermis "firmly adhered to the underlying dermis," the researchers reported. Hair follicles grew out of some areas. And even bumps and bruises healed normally. Unlike traditional skin grafts that require ointment once or twice a day to remain functional, the boy's new skin was fine with the normal amount of washing and moisturizing.

"The epidermis looks basically normal. There is no big difference," De Luca said. He said he expects the skin to last "basically the life of the patient."

In an analysis accompanying the main article in Nature, Mariacelest Aragona and Cedric Blanpain wrote that this therapy appears to be one of the few examples of truly effective stem-cell therapies. The study "demonstrates the feasibility and safety of replacing the entire epidermis using combined stem-cell and gene therapy," and also provides important insights into how different types of cells work together to help our skin renews itself.

They said there are still many other lingering questions, including whether such procedures might work better in children than adults and whether there would be longer-term adverse consequences, such as the development of cancer.

There are also many challenges to translating this research to treating wounds sustained in fires or other violent ways. In the skin disease that was treated in the boy, the epidermis is damaged but the layer beneath it, the dermis, is intact. The dermis is what the researchers called an ideal receiving bed for the lab-grown skin. But if deeper layers of the skin are burned or torn off, it's possible that the artificial skin would not adhere as well.

"No matter how you prepare, it's a bad situation," De Luca said. For the time being, he says he's continuing to study the procedure in two clinical trials that involve genetic diseases.

Meanwhile, Hirsch and Rothoeft report that the boy is continuing to do well and is not on any medication for the first time in many years. Doctors are carefully monitoring the child for any signs that there may be some cells that were not corrected and that the disease may re-emerge, but right now that does not appear to be happening in the transplanted areas. However, the child does have some blistering in about 2 to 3 percent of his body in non-grafted areas and they are considering whether to replace that skin as well.

But for now, they are giving the boy time to be a boy, Rothoeft said: "The kid is now back to school and plays soccer and spends other days with the children."

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‘Extraordinary’ tale: Stem cells heal a young boy’s lethal …

By raymumme

T

he complications of the little boys genetic skin disease grew as he did. Tiny blisters had covered his back as a newborn. Then came the chronic skin wounds that extended from his buttocks down to his legs.

By June 2015, at age 7, the boy had lost nearly two-thirds of his skin due to an infection related to the genetic disorder junctional epidermolysis bullosa, which causes the skin to become extremely fragile. Theres no cure for the disease, and it is often fatal for kids. At the burn unit at Childrens Hospital in Bochum, Germany, doctors offered him constant morphine and bandaged much of his body, but nothing not even his fathers offer to donate his skin worked to heal his wounds.

We were absolutely sure we could do nothing for this kid, Dr. Tobias Rothoeft, a pediatrician with Childrens Hospital in Bochum, which is affiliated with Ruhr University. [We thought] that he would die.

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As a last-ditch effort, the boys father asked if there were any available experimental treatments. The German doctors reached out to Dr. Michele De Luca, an Italian stem cell expert who heads the Center for Regenerative Medicine at the University of Modena and Reggio Emilia, to see if a transplant of genetically modified skin cells might be possible. De Luca knew the odds were against them such a transplant had only been performed twice in the past, and never on such a large portion of the body. But he said yes.

The doctors were ultimately able to reconstruct fully functional skin for 80 percent of the boys body by grafting on genetically modified cells taken from the boys healthy skin. The researchers say the results of this single-person clinical trial, published on Wednesday in Nature, show that transgenic stem cells can regenerate an entire tissue. De Luca told reporters the procedure not only offers hope to the 500,000 epidermolysis bullosa patients worldwide but also could offer a blueprint for using genetically modified stem cells to treat a variety of other diseases.

To cultivate replacement skin, the medical team took a biopsy the size of a matchbook from the boys healthy skin and sent it to De Lucas team in Italy. There, researchers cloned the skin cells and genetically modified them to have a healthy version of the gene LAMB3, responsible for making the protein laminin-332. They grew the corrected cultures into sheets, which they sent back to Germany. Then, over a series of three operations between October 2015 and January 2016, the surgical team attached the sheets on different parts of the boys body.

The gene-repaired skin took, and spread. Within just a month the wounds were islands within intact skin. The boy was sent home from the hospital in February 2016, and over the next 21 months, researchers said his skin healed normally. Unlike burn patients whose skin grafts arent created from genetically modified cells the boy wont need ointment for his skin and can regrow his hair.

And unlike simple grafts of skin from one body part to another, we had the opportunity to reproduce as much as those cells as we want, said plastic surgeon Dr. Tobias Hirsch, one of the studys authors. You can have double the whole body surface or even more. Thats a fantastic option for a surgeon to treat this child.

Dr. John Wagner, the director of the University of Minnesota Masonic Childrens Hospitals blood and marrow transplant program, told STAT the findings have extraordinary potential because, until now, the only stem cell transplants proven to work in humans was of hematopoietic stem cells those in blood and bone marrow.

Theyve proven that a stem cell is engraftable, Wagner said. In humans, what we have to demonstrate is that a parent cell is able to reproduce or self-renew, and differentiate into certain cell populations for that particular organ. This is the first indication that theres another stem cell population [beyond hematopoietic stem cells] thats able to do that.

The researchers said the aggressive treatment outlined in the study necessary in the case of the 7-year-old patient could eventually help other patients in less critical condition. One possibility, they noted in the paper, was to bank skin samples from infants with JEB before they develop symptoms. These could then be used to treat skin lesions as they develop rather than after they become life-threatening.

The treatment might be more effective in children, whose stem cells have higher renewal potential and who have less total skin to replace, than in adults, Mariaceleste Aragona and Cdric Blanpain, stem cell researchers with the Free University of Brussels, wrote in an accompanying commentary for Nature.

But De Luca said more research must be conducted to see if the methods could be applied beyond this specific genetic disease. His group is currently running a pair of clinical trials in Austria using genetically modified skin stem cells to treat another 12 patients with two different kinds of epidermolysis bullosa, including JEB.

For the 7-year-old boy, life has become more normal now that it ever was before, the researchers said. Hes off pain meds. While he has some small blisters in areas that didnt receive a transplant, they havent stopped him from going to school, playing soccer, or behaving like a healthy child.

The kid is doing quite well. If he gets bruises like small kids [do], they just heal as normal skin heals, Rothoeft said. Hes quite healthy.

Southern Correspondent

Max covers hospitals and health care.

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A boy with a rare disease gets new skin, thanks to gene …

By daniellenierenberg

A new therapy could restore healthy and protective skin to patients with a rare genetic disease.

iStock.com/Andrey Prokhorov

By Kelly ServickNov. 8, 2017 , 1:00 PM

A 7-year-old who lost most of his skin to a rare genetic disease has made a dramatic recovery after receiving an experimental gene therapy, researchers announced today. The treatmenta whole-body graft of genetically modified stem cellsis the most ambitious attempt yet to treat a severe form of epidermolysis bullosa (EB), an often-fatal group of conditions that cause skin to blister and tear off at the slightest touch.

The new approach can address only a subset of the genetic mutations that cause EB. But the boys impressive recoveryhes now back inschool and is even playing soccercould yield insights that help researchers use stem cells to treat other genetic skin conditions.

It is very unusual that we would see a publication with a single case study anymore, but this one is a little different, says Jakub Tolar, a bone marrow transplant physician at the Masonic Cancer Center, University of Minnesotain Minneapolis who is developing therapies for EB. This is one of these [studies] that can determine where the future of the field is going to go.

EB results from mutations to any of several genes that encode proteins crucial for anchoring the outer layer of skin, the epidermis, to the tissue below. The missing or defective protein can cause skin to slough off from minor damage, creating chronic injuries prone to infection. Some forms of EB can be lethal in infancy, and some predispose patients to an aggressive and deadly skin cancer. The only treatment involves painfully dressing and redressing wounds daily. Bandage costs can approach $100,000 a year, says Peter Marinkovich, a dermatologist at Stanford University in Palo Alto, California, who treats EB patients. Theyre like walking burn victims, he says.

In fact, the new approach is similar to an established treatment for severe burns, in which sheets of healthy skin are grown from a patients own cells and grafted over wounds. But stem cell biologist and physician Michele De Luca of the University of Modena and Reggio Emilia in Italy and his colleagues have been developing a way to counteract an EB-causing mutation by inserting a new gene into the cells used for grafts. His group has already treated two EB patients with this approach. They publishedencouraging resultsfrom their first attemptwith small patches of gene-corrected skin on a patients legsin 2006.

In 2015, De Lucas team got a desperate request from doctors in Germany. Their young patient had a severe form of the disease known as junctional EB, caused by a mutation in a gene encoding part of the protein laminin 332, which makes up a thin membrane just below the epidermis. It was the same gene De Lucas team was targeting in an ongoing clinical trial, but this case was especially dire: Lacking most of his skin, the boy had contracted multiple infections and was in a life-threatening septic state. The emergency treatment would be the first test of their gene therapy approach over such a large and severely damaged area.

De Lucas team used a patch of skin a little bigger than a U.S. postage stamp from an unblistered part of the boys groin to culture epidermal cells, which include stem cells that periodically regenerate the skin. They infected those cells with a retrovirus bearing healthy copies of the needed gene,LAMB3, and grew them into sheets ranging from 50 to 150 square centimeters. In two surgeries, a team at Ruhr University in Bochum, Germany, covered the boys arms, legs, back, and some of his chest in the new skin.

After a month,most of the new skin had begun to regenerate, covering 80% of the boys body in strong and elastic epidermis, the researchers report online today inNature. Whats more, hes developed no blisters in the grafted areas in the 2 years since the surgery.

Other researchers have long been concerned that using a retrovirus to insert genes at random points in cells genomes might cause cancer. (In the early 2000s, five children who participated in a retrovirus-based gene therapy trial for severe combined immunodeficiencydeveloped leukemia.) But the current study found no evidence that the insertion affected cancer genes.

De Luca and colleagues were also able to track which grafted cells regenerated the skin over time by using the different locations of the genetic insert as markers for individual cells and their progeny. They found that most cells from the graft disappeared after a few months, but a small population of long-lived cells called holoclones formed colonies that renewed the epidermis.

Epidermal stem cells known as holoclones (shown in pink) were responsible for regenerating the young epidermolysis bullosapatients skin, whileother cell types disappeared over time.

News & Views/Nature; adapted by E. Petersen/Science

Thats an important lesson, Tolar says; it suggests that future attempts to correct genetic skin diseases should focus on culture conditions that nourish these stem cells, and potentially even target them for modification. If you have a gene correction strategy, he says, youd better have these primitive epidermal stem cells in mind.

The current results could benefit several thousand EB patients across the world, Marinkovich says, but it wont work for all of them. More than half have a form of the disease called EB simplex, which is causednot by a missing protein, but by mutations that produce an active but dysfunctional protein. For these errors, correction with a gene-editing tool like CRISPR makes more sense, De Luca says.

The grafts also cant repair damage to internal surfaces such as the esophagus, Tolar notes, which occurs in some EB cases. Fortunately, that wasnt an issue for the boy in this study. The treatment is a good step in the right direction, he says, but its not curative.

Both De Luca and Marinkovichs teams are exploring a similar gene therapy for another major form of the disease, called dystrophic EB, caused by a different genetic error affecting a larger protein. Biotech companies are working with each group to test the approach in larger clinical trials.

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Doctors replace boys skin using breakthrough gene therapy …

By raymumme

In a breakthrough treatment, researchers at a burn unit in Europe found a way to replace 80 percent of a boys skin using a combination of gene therapy and stem cells. The grafted skin attached to his body has continued to replace itself, even months later.

The patient - a boy who was 7 years old at the time of the treatment - was born with a rare skin condition called junctional epidermolysis bullosa. The condition causes the outer layer of the skin to peel away easily from the lower skin layers, making it incredibly fragile and prone to injury.

This is a very severe, devastating disease, where kids suffer a lot, said Dr. Michele De Luca, one of the authors of the research.

Experts not involved in the research have said this successful grafting treatment is a big step for those suffering from genetic skin conditions like this one.

This is really quite exciting, to have this translation for these patients, said Dr. Dennis Orgill, medical director of the Brigham and Womens Hospital Wound Center in Boston, who was not involved with the study. "That they can do these genetic manipulations and then have a long term result, which theyve demonstrated here, is a major breakthrough."

In this case, the treatment may have been lifesaving. The patient arrived at the hospital with a life-threatening bacterial skin infection spread over much of his body. Over the following weeks, his doctors tried everything they could to treat him without success.

Out of options, his treatment team was preparing to start end-of-life care when his parents pleaded with them to try an experimental therapy.

Surgeons in Germany took a sample of the boys skin, less than one square inch in size, that was unharmed by the bacterial infection. In a lab, researchers infected the skin biopsy with a virus specially designed to alter the genetic code within the skin cells, correcting the mutation responsible for his fragile skin. The researchers "grew" the skin and used it to surgically replace the patients blistered and destroyed skin.

After 21 months, the new skin is regenerating itself without problems and has been resilient; it can hold up to normal wear much better than his original skin.

While this result only applies to one rare skin disorder right now, experts said the approach could be used more widely for other diseases in the future.

We are running other clinical trials on other kinds of junctional epidermolysis bullosa," De Luca said. "In the future, it could be applied to other genetic diseases of the skin.

Researchers hope that it could help other people with seriously damaged skin in the future, too.

This technology could be extended into other patients with genetic conditions, or patients with extensive burns, Orgill said.

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Scientists replace skin using genetically modified stem cells

By LizaAVILA

Related content

(CNN) - For the first time, doctors were able to treat a child who had a life-threatening rare genetic skin disease through a transplant of skin grown using genetically modified stem cells.

The grafts replaced 80% of the boy's skin.

The skin of his arms, legs, back and flanks, and some of the skin on his stomach, neck and face was missing or severely affected due to epidermolysis bullosa.

The compassionate-use experimental treatment is detailed in a case study published in the journal Nature on Wednesday.

Skin as fragile as a butterfly's wings -- that's how children with epidermolysis bullosa are described and why they're often called butterfly children.

The disease, of which there are five major types and at least 31 subtypes, is incurable. People with the condition have a defect in the protein-forming genes necessary for skin regeneration.

About 500,000 people worldwide are affected by forms of the disease. More than 40% of patients die before reaching adolescence.

Their skin can blister and erode due to something as simple as bumping into something or even the light friction of clothing, according to an email from Dr. Jouni Uitto, a professor and chairman of the Department of Dermatology and Cutaneous Biology at the Sidney Kimmel Medical College in Philadelphia. Uitto was not involved with this study.

Epidermolysis bullosa makes the skin incredibly susceptible to infections, and in the case of 7-year-old Hassan, whose treatment was detailed in Nature, those infections can be life-threatening.

A week after he was born in Syria, Hassan had a blister on his back, his father said through an interpreter in an interview provided by the hospital in Germany where the boy was treated.

Hassan's last name, as well as the first names of his family members, are not being disclosed to protect the privacy of the family.

In his first few weeks of life, Hassan was immediately diagnosed with epidermolysis bullosa, and their doctor in Syria told Hassan's family that there was no cure or therapy.

Over the years, their efforts to find help for their son's disease led the family to the Muenster University hospital in Germany in 2015, when Hassan was 7. His condition worsened, and he struggled with severe sepsis and a high fever. He weighed just over 37 pounds.

They didn't think he would make it, and doctors at Muenster decided in summer 2015 to transfer Hassan to the Ruhr-Universitt Bochum's University Hospitals, including the burn center -- one of the oldest in the country.

By the time Hassan arrived at Bochum, he had lost two-thirds of his surface skin.

"We had a lot of problems in first days just keeping him alive," said Dr. Tobias Rothoeft, consultant at the University Children's Hospital at Katholisches Klinikum Bochum.

Doctors tried to promote healing by changing his dressings and treating him with antibiotics, as well as putting him on an aggressive nutrition schedule, but nothing helped. They even tried transplanting skin from Hassan's father.

"By that time, he had lost 60% of his epidermis, the upper skin layer, and had 60% open wounds all over his body," said Dr. Maximilian Kueckelhaus of the Department of Plastic Surgery at Bochum's Burn Center.

Every approach failed, so the doctors prepared Hassan's family for what end-of-life care would entail. But the parents pleaded, asking the doctors to consult studies and research for experimental treatments that might help.

They found Dr. Michele De Luca at the University of Modena's Center for Regenerative Medicine in Italy. His publications described an experimental treatment transplanting genetically modified epidermal stem cells that healed small, non-life-threatening wounds in adults.

The medical team reached out to De Luca, asking whether he could help them replicate the procedure on a larger scale to help Hassan, and he agreed. De Luca told Hassan's parents that he believed there was a 50% chance of the treatment being successful.

They were more than willing to accept the risk, to do anything to help their son have a chance at a normal life.

Hassan "was in severe pain and was asking a lot of questions: 'Why do I suffer from this disease? Why do I have to live this life? All children can run around and play. Why am I not allowed to play soccer?' I couldn't answer these questions," his father said. "It was a tough decision for us, but we wanted to try for Hassan."

To obtain the skin's stem cells, the doctors took a small biopsy -- only accounting for 1 square inches -- from an unaffected part of Hassan's skin. The stem cells were processed by De Luca in Italy. A healthy version of the gene that is normally defective in epidermolysis bullosa patients was added to the cells, along with retroviral vectors: virus particles that assist the gene transfer.

This genetic transfer would essentially "correct" the cells.

The single cells were grown and cultivated on plastic and fibrin substrate, which is used to treat large skin burns, to form a large piece of epidermis. This method enabled the researchers to grow as much skin as they needed. The whole process took three to four weeks, Kueckelhaus said.

Once the sheets were ready, they were transferred from Italy to Germany and transplanted onto the well-cleaned wounds right away during two surgeries. The first procedure in October 2015 applied the sheets to Hassan's arms and legs. The second surgery, in November, grafted the sheets to Hassan's entire back and the other affected areas.

Hassan began to improve immediately. The researchers noticed that the grafts were not rejected; they bound to all of the areas they were transplanted.

"For everyone that was involved, taking off the bandages and seeing for the first time that this is working out, that the transplants are actually attached to the patient and growing skin, that's an incredible moment," Kueckelhaus said.

Hassan was discharged from the hospital in February 2016.

After steady followups over 21 months, the researchers found that Hassan's new skin healed normally, didn't blister anymore, and was resistant to stress. It was even growing hair. Unlike some skin graft patients, he doesn't require any ointment to keep his skin smooth and hydrated. And like any growing kid, he bruises and recovers normally.

They also learned that only a few stem cells contribute to the long-term maintenance of the epidermis, shedding light on cellular hierarchy in this regard.

"The investigators removed of small piece of patient's skin, isolated cells with stem cell potential for growth, introduced a normal copy of the mutated gene to the cells, propagated a large number of these cells in culture and then grafted them back to the skin," Uitto said. "This concept is not new, but what is remarkable here is that they were able to change essentially the entire skin of the patient with normal cells."

Hassan's family is currently living in Germany. Hassan, now 9, is able to go to school and play sports, but he maintains a schedule of frequent monitoring at the hospital to ensure that the initial success of the treatment continues. The area of his skin that was not treated sometimes shows small blisters, and if it worsens, he may receive transplants there as well.

"Seeing him 18 months after the initial surgery with an intact skin is incredible because he has been in the ICU for so long," Kueckelhaus said. "He had bandages all over his body except his hands, feet and face. He was on extremely strong pain medication. So the quality of life was really, really bad for him. Seeing him play soccer, play sports, play with other kids, that is just amazing because that's something he couldn't do before."

"It felt like a dream for us," the boy's father said. "Hassan feels like a normal person now. He plays. He's being active. He loves life."

Everything points to a good long-term outcome for Hassan.

The researchers will continue to monitor him for complications. Sometimes, genetic modifications can cause malignancies in cells.

"That is of course one thing we really have to be aware of," Kueckelhaus said. "However, analyzing the integration profile of that gene into the boy's DNA, which we did, we saw that it's mostly in areas that don't cause too much concern about developing malignancies."

Epidermolysis bullosa patients can be at a very high risk of developing skin cancer simply because of the disease. Because Hassan now has intact skin and intact DNA, this risk might even decrease, but that will have to be proved through follow-up, Kueckelhaus said.

Given that this was one successful outcome for one patient, the experimental treatment can't be applied for other patients just yet. De Luca is conducting clinical trials using the treatment.

"This is one case with a distinct type of EB, and further studies will show whether this approach is applicable to other forms of EB as well," Uitto said. "It should be noted that in some severe forms of EB, the patients also suffer from fragility of the gastrointestinal and vesico-urinary tract, and some forms are associated with the development of muscular dystrophy. Obviously, gene therapy of the skin cannot correct them, and these issues have to be addressed in further studies."

Hassan's treatment also cost hundreds of thousands of dollars. Although the process could be optimized, doctors would still have to individually grow transplants for each patient, which could get very expensive.

But for patients' families, epidermolysis bullosa is already expensive.

"Standard maintenance treatment of patients with EB, including daily bandaging, antibiotics and special moisturizer, as well as frequent hospitalizations, can be extremely costly, and gene correction as described in this paper may well be cost-effective over the lifetime of these patients," Uitto noted.

Brett Kopelan, executive director of the Dystrophic Epidermolysis Bullosa Research Association of America, has a 10-year-old daughter, Rafi, with recessive dystrophic EB. Between January and August, $751,1778 for wound/burn dressings was charged to Kopelan's insurance company, he says. That doesn't account for drugs or hospital visits and surgeries.

Kopelan's nonprofit sends free supplies and bandages to families. The nonprofit can provide its employees with insurance that covers the medical equipment, but that isn't the case for everyone impacted by the condition, he said.

Kopelan is hopeful about the results of the study. The baths and bandage changes that are necessary for epidermolysis bullosa patients to stave off life-threatening infections can last hours and feel torturous.

"Do you remember the last time you got a paper cut and put Purell on it? It burned, right? Now think of 60% of body being an open wound, and opioids don't really work for this kind of pain," Kopelan wrote in an email. "This is what make EB kids and adults the strongest people on Earth."

The study "confirms our hopes that gene therapy is potentially the most efficacious path forward to providing a significant treatment option for those with epidermolysis bullosa," Kopelan said. "While it's important to remember that this is only one patient and more work needs to be done to demonstrate how effective this gene therapy platform may prove to be, I am very enthused."

"I wish that all children with the same disease could be treated in this way," Hassan's father said.

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Hematopoietic cell transplantation (bone marrow …

By JoanneRUSSELL25

HEMATOPOIETIC CELL TRANSPLANTATION OVERVIEW

Hematopoietic cell transplantation (also called bone marrow transplantation or stem cell transplant), is a type of treatment for cancer (and a few other conditions as well). A review of the normal function of the bone marrow will help in the understanding of stem cell transplantation.

Hematopoietic stem cell functionBone marrow is the soft, spongy area in the center of some of the larger bones of the body. The marrow produces all of the different cells that make up the blood, such as red blood cells, white blood cells (of many different types), and platelets. All of the cells of the immune system are also made in the bone marrow. All of these cells develop from a type of precursor cell found in the bone marrow, called a "hematopoietic stem cell."

The body is able to direct hematopoietic stem cells to develop into the blood components needed at any given moment. This is a very active process, with the bone marrow producing millions of different cells every hour. Most of the stem cells stay in the marrow until they are transformed into the various blood components, which are then released into the blood stream. Small numbers of stem cells, however, can be found in the circulating blood, which allows them to be collected under certain circumstances. Various strategies can be employed to increase the number of hematopoietic stem cells in the blood prior to collection. (See 'Peripheral blood' below.)

Hematopoietic cell transplantationSome of the most effective treatments for cancer, such as chemotherapy and radiation, are toxic to the bone marrow. In general, the higher the dose, the more toxic the effects on the bone marrow.

In hematopoietic cell transplantation, you are given very high doses of chemotherapy or radiation therapy, which is intended to more effectively kill cancer cells and unfortunately also destroy all the normal cells developing in the bone marrow, including the critical stem cells. After the treatment, you must have a healthy supply of stem cells reintroduced, or transplanted. The transplanted cells then reestablish the blood cell production process in the bone marrow. Reduced doses of radiation or chemotherapy that do not completely destroy the bone marrow may be used in some settings. (See 'Non-myeloablative transplant' below.)

The cells that will be transplanted can be taken from the bone marrow (called a bone marrow transplant), from the bloodstream (called a peripheral blood stem cell transplant, which requires that you take medication to boost the number of hematopoietic stem cells in the blood), or occasionally from blood obtained from the umbilical cord at the time of birth of a normal newborn (called an umbilical cord blood transplant).

TYPES OF HEMATOPOIETIC CELL TRANSPLANTATION

There are two main types of hematopoietic cell transplantation: autologous and allogeneic.

Autologous transplantIn autologous transplantation, your own hematopoietic stem cells are removed before the high dose chemotherapy or radiation is given, and they are then frozen for storage and later use. After your chemotherapy or radiation is complete, the harvested cells are thawed and returned to you.

Allogeneic transplantIn allogeneic transplantation, the hematopoietic stem cells come from a donor, ideally a brother or sister with a similar genetic makeup. If you do not have a suitably matched sibling, an unrelated person with a similar genetic makeup may be used. Under some circumstances, a parent or child who is only half-matched can also be used; this is termed a haploidentical transplant.

Myeloablative transplantA myeloablative transplant uses very high doses of chemotherapy or radiation prior to transplantation with autologous or allogeneic hematopoietic stem cells.

Non-myeloablative transplantA non-myeloablative transplant, sometimes referred to as a "mini" or reduced intensity transplant, allows you to have less intensive chemotherapy before transplantation with allogeneic hematopoietic stem cells. This approach may be recommended for a variety of reasons including your age, type of disease, other medical issues, or prior therapies.

Which type of transplant is best?Your physician will determine whether allogeneic or autologous transplantation is best, based on many factors including the type of cancer, your age and overall health, and the availability of a suitable donor. As a general rule, autologous transplantation is associated with fewer serious side effects, since you are given cells from your own body. However, an autologous transplant may be less effective than an allogeneic transplant in treating certain kinds of cancer.

In an allogeneic transplant, the donor's immune system, which is generated from the transplanted hematopoietic stem cells, recognize your cells, including the tumor cells, as foreign and rejects them. This beneficial reaction is called the graft-versus-tumor effect. In many cancers, the immune response caused by the transplanted cells improves the overall effectiveness of the treatment. This immune response helps kill off any residual cancer cells remaining in your body.

A major concern is that you will have an immune response against normal tissues as well, called graft-versus-host disease. (See 'Graft-versus-host disease' below.)

In a non-myeloablative transplant, it is hoped that the graft-versus-tumor effect, rather than the high-dose chemotherapy, will help eradicate the cancer, although graft-versus-host disease is a concern (see 'Graft-versus-host disease' below).

CHOOSING A DONOR

There are many possible choices for an allogeneic hematopoietic stem cell donor. These are described below. (See "Donor selection for hematopoietic cell transplantation".)

Matched donorTo help minimize the problems that can be caused by the expected immune response, a donor who has similar genetic makeup to you is preferred. Your cells will seem "less foreign" to the transplanted donor cells. Siblings (ie, brothers and sisters who share the same parents as you) are typically the only members of your family that are tested for being a donor because they have a one in four chance of sharing genetic characteristics with you; these characteristics are critical for your body to accept the graft. In general, parents, children, and relatives are not suitable donors since they do not share the same parents, and therefore do not have the same genetic material.

An exception is called a haploidentical transplant, which may be considered under certain circumstances.

Matched unrelated donorIf no siblings are available, or if testing the blood of the siblings does not reveal a match, a matched unrelated donor may be used. The search for an appropriate donor can be accomplished using transplant registries throughout the world.

Mismatched related donor or umbilical cord blood donorSome patients are offered treatment with cells from a partially matched family member (called mismatched related donor). The hematopoietic stem cell product may be specially prepared to minimize the immune response in the patient. Another alternative is to use umbilical cord blood, collected from a healthy newborn infant at the time of delivery; this blood is a rich source of hematopoietic stem cells.

PRE-HEMATOPOIETIC CELL TRANSPLANTATION PROCEDURES

Stem cell transplantation regimens vary from one patient to another, and depend upon the type of cancer, the treatment program used by the medical center, the clinical trial protocol (if the patient is enrolled in a clinical trial), as well as other factors. The most common components of the hematopoietic cell transplantation procedure are outlined here. You should talk with your transplant team about specific details of their program. (See "Preparative regimens for hematopoietic cell transplantation".)

Health evaluationBefore undergoing hematopoietic cell transplantation, you will have a complete evaluation of your health. Your complete health history is reviewed by the transplant team. Most patients also have a number of tests.

Your mental health is reviewed because of the stress and demands of stem cell transplantation; some patients meet with a mental health counselor to discuss concerns and to plan coping strategies.

You will also meet with a transplant coordinator or nurse to discuss the transplant process. Because patients who receive donor bone marrow are hospitalized for several weeks to months, it is important that you have a clear understanding of what will happen and what services are available. Some patients prefer to have a friend or family member accompany them, tape record the conversation with the transplant physician, or have this information in writing so that they can review it later.

In many cases, patients undergo hematopoietic cell transplantation while they are in remission from their underlying disease. You may feel well going into treatment, but you should be prepared to feel poorly for a period of time. You must understand that you will require intensive treatment and monitoring, but that there are long-term benefits from the treatment.

Life planningPatients who will be in the hospital for several weeks or months need to make plans regarding their family, home, finances, pets, and employment. The National Marrow Donor Program has excellent information about these and other stem cell transplantation related topics.

During the pre-transplant planning process, you should consider completing an advanced directive. This is a legal document that describes the type of care you want in case you are unable to communicate. Advance directives include a living will, durable power of attorney, and healthcare proxy; a social worker or attorney can provide guidance about what documents are needed. The laws surrounding these documents vary from one state to another, so it is important to be sure the correct guidelines are used.

Central line placementA number of medications will be required before, during, and after hematopoietic cell transplantation. To avoid the need for multiple intravenous lines and needle sticks, most patients will have a central line placed before treatment begins. This requires a short surgical procedure to insert a thin, flexible plastic tube into a large vein in the chest, above the heart. The line usually has two or three ports, which can be used to infuse medications or blood products (including the hematopoietic stem cell product), as well as to withdraw blood samples.

After the central line is placed, you must keep the area clean and watch for signs and symptoms of infection (pain, redness, swelling, or fluid drainage from the site, fever or chills).

Harvesting hematopoietic stem cellsIf you are having an autologous transplant, hematopoietic stem cells will be removed from your body before intensive chemotherapy or radiation begins. The most common sources for hematopoietic stem cells are bone marrow and blood.

Bone marrowIf your bone marrow has been invaded with cancer cells, hematopoietic stem cell removal may be preceded by one or more courses of chemotherapy. Removal (called harvest) of bone marrow stem cells is done while you are under general or epidural anesthesia. The harvest is done by using a long needle to repeatedly remove a sample of bone marrow fluid from multiple areas in your pelvic and hip bones.

Peripheral bloodThe harvest of peripheral blood stem cells is similar to the process of platelet donation and is more frequently used than a bone marrow harvest. It uses an apparatus, called an apheresis device, which removes hematopoietic stem cells and other cells from blood by a filtration process. Blood is removed from a vein in one location, filtered, and then returned to a vein in another location. The process does not require anesthesia.

In order for there to be sufficient numbers of hematopoietic stem cells in the blood, you (or the donor) must first be treated with either chemotherapy or a growth factor that stimulates the production and release of hematopoietic stem cells into the blood. Healthy donors only receive growth factor; patients with cancer may receive growth factor alone or chemotherapy plus growth factor. The most commonly used growth factor is granulocyte colony-stimulating factor (G-CSF or Neupogen).

Allogeneic bone marrow harvestPeople who donate their bone marrow will undergo harvest the day of transplant or one day prior. The donor is usually given general anesthesia to prevent pain.

Following the procedure, pain in the donor is usually relatively minor and can be treated with pain medications such as acetaminophen. The donor may be hospitalized overnight following the procedure, and generally returns to his or her prior state of health within the following one to two weeks.

Myeloablative therapyAs noted above, many patients receiving hematopoietic cell transplantation will undergo myeloablative therapy, which destroys bone marrow function as part of the intensive treatment for the patient's underlying cancer. The purpose of this treatment is to reduce the amount of cancer in the body and also to suppress the immune system adequately so that the graft will not be rejected. Depending upon the underlying disease and other factors, this phase of treatment may involve intensive chemotherapy, total body irradiation (radiation therapy), or both.

Preventing infectionWhen bone marrow function is destroyed, you are at risk for developing life-threatening infections because you have temporarily lost your ability to produce white blood cells (the infection-fighting cells in the blood). You are also at risk for excessive bleeding due to the reduced number of platelets in the blood. (See "Prevention of infections in hematopoietic cell transplant recipients".)

It is important to minimize your exposure to bacteria, viruses, and fungi after myeloablative therapy because even a small number of organisms (that are usually encountered every day) can cause serious infection.

Patients who undergo allogeneic transplant are often placed in protective isolation in a private room. The room's air is filtered and air from the room is forced out when the door is opened (called a positive-pressure room). This isolation, combined with feeling poorly, can be challenging to some people who may feel depressed and/or anxious. Discussing these issues with your health care team is very important.

Special precautions are required for all persons who enter the room to reduce the chance of infection. Hand washing is one of the most important precautions, and has been shown to significantly reduce the chance of transmitting infection. Visitors should NOT bring fresh fruit, plants, or flowers into your room because these can harbor microorganisms that are dangerous.

Other measures may be taken to reduce the chance of infection. For example, antibiotics, antifungal, and/or antiparasitic medications may be given to prevent infections, and your diet may be restricted to exclude items that contain potentially infectious organisms. For example, all foods should be cooked until hot, raw fruits and vegetables should be avoided, and drinking water should be sterilized.

Most patients can shower. There has been a concern that showers can aerosolize fungal spores, and some centers prefer that patients take a tub or sponge bath. You can wear a hospital gown or your own clean clothing.

Different transplant centers use different precautions and your health care team will discuss the precautions and procedures that they expect.

Blood product transfusionsDuring the time that the marrow is not functioning, you will likely require transfusion of blood products, such as red cells, which carry oxygen to the tissues, or platelets, which help prevent bleeding. These blood products have no white blood cells and are irradiated to reduce the risk of an immune response.

HEMATOPOIETIC CELL TRANSPLANTATION PROCEDURE

When the intensive chemotherapy and/or radiation are complete, you will be given an infusion of the harvested bone marrow or peripheral blood stem cells. The infusion is given through an intravenous (IV) line, usually the central line. The infusion usually takes about an hour, and usually causes no pain.

The cells find their way to the bone marrow, where they will reestablish normal production of blood cells; this process is called engraftment. Determining when engraftment has occurred is important because it is used to determine when it is safe for you to go home and/or reduce isolation procedures. Medications that stimulate the bone marrow to produce white and red cells may be used when engraftment is slower than expected. (See "Hematopoietic support after hematopoietic cell transplantation".)

Engraftment is measured by performing daily blood cell counts. Neutrophils are a type of white blood cell that are a marker of engraftment; the absolute neutrophil count (ANC) must be at least 500 for three days in a row to say that engraftment has occurred. This can occur as soon as 10 days after transplant, although 15 to 20 days is common for patients who are given bone marrow or peripheral blood cells. Umbilical cord blood recipients usually require between 21 and 35 days for neutrophil engraftment.

Platelet counts are also used to determine when engraftment has occurred. The platelet count must be between 20,000 and 50,000 (without a recent platelet transfusion). This usually occurs at the same time or soon after neutrophil engraftment, but can take as long as eight weeks and even longer in some instances for people who are given umbilical cord blood.

HEMATOPOIETIC CELL TRANSPLANTATION SIDE EFFECTS

The high-dose chemotherapy and total body irradiation required for hematopoietic cell transplantation can have serious side effects. You should discuss the expected side effects, toxicities, and risks associated with stem cell transplant before deciding to undergo the procedure. You will be asked to sign a consent form indicating that you have received verbal and written information to understand the risks and benefits of the proposed treatment, possible treatment alternatives, and that all your questions have been answered.

Common side effectsSome of the most common side effects include:

Mucositis(mouth sores) and diarrhea Mucositis and diarrhea are caused by the damage done to rapidly dividing cells (such as skin cells in the mouth and digestive tract) by chemotherapy and radiation. If mucositis is severe and affects your ability to eat, intravenous nutrition (called TPN, total parenteral nutrition) may be given. Pain medications are usually given as well.

Nausea and vomiting Nausea and vomiting can be prevented and treated with a combination of medications, usually including a 5-HT3 receptor antagonist (dolasetron, granisetron, ondansetron, tropisetron, or palonosetron), an NK1 receptor antagonist (aprepitant [Emend]), and a steroid (dexamethasone).

Loss of hair Loss of hair is temporary, and generally includes hair on the head, face, and body. After high-dose chemotherapy and radiation are completed, hair begins to regrow. No treatment is available to prevent hair loss or speed its regrowth.

Infertility The risk of permanent infertility after stem cell transplant depends upon the treatments used (high-dose chemotherapy versus total body irradiation, ablative versus non-ablative regimen) and dosage given. If you are of reproductive age, you should speak with your healthcare provider about options for lowering the risks of infertility and the option of donating eggs or sperm before treatment begins. (See "Fertility preservation in patients undergoing gonadotoxic treatment or gonadal resection".)

Organ toxicity The lungs, liver, and bones are at greatest risk of damage as a result of treatments used with stem cell transplantation. People who have total body irradiation can develop cataracts in the eyes, although this complication is less common with current methods of delivering radiation treatment.

Secondary cancers There is a small risk of a second cancer developing in patients who undergo stem cell transplantation, probably as a result of the treatments used for the first cancer as well as the treatments required for transplant. The second cancer usually develops several years (typically three to five) after stem cell transplantation. (See "Malignancy after hematopoietic cell transplantation".)

Graft-versus-host diseaseBetween 10 and 50 percent of patients who receive an allogeneic transplant experience a side effect known as graft-versus-host disease (GVHD). Graft-versus-host disease is separated into acute and chronic phases due to timing and clinical presentation. This problem does not occur following autologous transplantation (when the patient is the donor). (See "Prevention of acute graft-versus-host disease".)

The "graft" refers to the transplanted hematopoietic stem cells; the "host" refers to the patient. Thus, graft-versus-host disease refers to a condition in which the donor's immune cells attack some of your organs. GVHD is the biggest single threat, other than the underlying disease, to the success of a stem cell transplant.

Treatments are given to help prevent GVHD, and generally include immunosuppressive medications, antibiotics, and sometimes steroids. If GVHD develops, additional treatment with high-dose steroids may lessen its severity. Symptoms can include skin rash, diarrhea, liver damage, or other problems, depending upon the organ that is affected. (See "Treatment of chronic graft-versus-host disease".)

Graft failureFailure of engraftment is a rare complication that occurs in approximately one percent of cases following hematopoietic cell transplantation. The risk of graft failure can be higher depending upon the type of transplant and the source of hematopoietic stem cells. Discuss these risks with the transplant team prior to treatment. (See "Immunotherapy for the prevention and treatment of relapse following hematopoietic cell transplantation".)

Risk of deathHematopoietic cell transplantation carries a risk of treatment-related death. The risk of death depends upon your age, the nature of the underlying disease, the type of transplant (autologous or allogeneic), and other factors, including the skill and expertise of the institution where treatment is offered. Your risk, as well as the potential benefits of hematopoietic cell transplantation, should be discussed with the treatment team before any decision is made about undergoing a transplant procedure.

POST-HEMATOPOIETIC CELL TRANSPLANTATION CARE

After engraftment occurs, blood cell counts continue to rise and the immune system becomes stronger. You will usually be cared for by the transplant team and monitored closely for complications.

Non-myeloablative transplants may be done on an outpatient basis, allowing you to sleep at home. Other types of transplantation require you to stay in the hospital for three to four weeks following transplantation. In all cases, frequent visits to the healthcare provider's office are needed following discharge. If you live a distance from your provider, you should arrange to live in a place within reasonable driving distance to the treatment center until at least 100 days have passed since the transplant.

Patients who undergo hematopoietic cell transplantation are at an increased risk of infection for many months following transplantation. You should be aware of these risks and monitor yourself for symptoms of infection, including fever (temperature greater than 100.4F or 38C), pain, or chills. You may be given antibiotics to prevent infections.

Studies have shown that most patients who undergo transplant and remain free of cancer have a good quality of life. Most patients are able to be active, employed, and in reasonably good health. Quality of life usually continues to improve in the months following transplant.

CLINICAL TRIALS

A clinical trial is a carefully controlled way to study the effectiveness of new treatments or new combinations of known therapies, and patients who will undergo hematopoietic cell transplantation may be asked to participate. Ask a healthcare provider for more information about clinical trials, or read further at the following web sites.

http://www.cancer.gov/clinicaltrials/

http://clinicaltrials.gov/

Videos addressing common questions about clinical trials are available from the American Society of Clinical Oncology (http://www.cancer.net/pre-act).

SUMMARY

Hematopoietic cell transplantation (also called bone marrow transplantation or stem cell transplant) is a treatment used in some types of cancer particularly malignancies of the blood.

Bone marrow is the soft, spongy area in the center of some of the larger bones of the body. The marrow produces all of the different cells that make up the blood, such as red blood cells, white blood cells, and platelets. All of these cells develop from a type of basic cell found in the bone marrow, called a stem cell.

In hematopoietic cell transplantation, the patient is given very high doses of chemotherapy or radiation therapy, which kills cancer cells and destroys all the normal cells developing in the bone marrow, including the critical stem cells. After the treatment, the patient must have a healthy supply of hematopoietic stem cells reintroduced, or transplanted.

There are two types of hematopoietic cell transplantation, autologous and allogeneic. An autologous hematopoietic cell transplant uses a patient's own bone marrow or blood. An allogeneic hematopoietic cell transplant uses a donor's bone marrow or blood. The donor is usually a relative of the patient (eg, sister), although unrelated donors are sometimes used.

Most patients who have hematopoietic cell transplantation must remain in the hospital for several days or weeks during their treatment and recovery. It is important to understand and follow the hospital's stem cell transplantation treatment plan to minimize the risk of complications (eg, infection) and to know what to expect in advance.

The treatments required before and during hematopoietic cell transplantation can have serious side effects. Patients should be aware of the most common side effects (eg, diarrhea, nausea, vomiting, mouth sores) as well as the types of treatments that are available to improve comfort.

Following hematopoietic cell transplantation, most people stay in the hospital for several weeks. However, even after going home, frequent visits with a doctor or nurse are needed for three to six months.

Clinical trials are carefully controlled studies of new treatments or new combinations of current treatment. Clinical trials help researchers to learn the best way to treat specific conditions. Some patients who have stem cell transplantation will be asked to participate in a clinical trial.

WHERE TO GET MORE INFORMATION

Your healthcare provider is the best source of information for questions and concerns related to your medical problem.

This article will be updated as needed on our website (www.uptodate.com/patients). Related topics for patients, as well as selected articles written for healthcare professionals, are also available. Some of the most relevant are listed below.

Patient level informationUpToDate offers two types of patient education materials.

The BasicsThe Basics patient education pieces answer the four or five key questions a patient might have about a given condition. These articles are best for patients who want a general overview and who prefer short, easy-to-read materials.

Patient education: Bone marrow transplant (The Basics)Patient education: Donating bone marrow or blood stem cells (The Basics)Patient education: Leukemia in adults (The Basics)Patient education: Leukemia in children (The Basics)Patient education: Lymphoma (The Basics)Patient education: Acute lymphoblastic leukemia (ALL) (The Basics)Patient education: Acute myeloid leukemia (AML) (The Basics)Patient education: Chronic lymphocytic leukemia (CLL) (The Basics)Patient education: Chronic myeloid leukemia (CML) (The Basics)Patient education: Diffuse large B cell lymphoma (The Basics)Patient education: Follicular lymphoma (The Basics)Patient education: Hodgkin lymphoma in adults (The Basics)Patient education: Hodgkin lymphoma in children (The Basics)Patient education: Myelodysplastic syndromes (MDS) (The Basics)Patient education: Sickle cell disease (The Basics)Patient education: Immune thrombocytopenia (ITP) (The Basics)Patient education: Beta thalassemia major (The Basics)Patient education: Chronic granulomatous disease (The Basics)Patient education: Invasive aspergillosis (The Basics)Patient education: When your child has sickle cell disease (The Basics)Patient education: Neutropenia and fever in people being treated for cancer (The Basics)

Beyond the BasicsBeyond the Basics patient education pieces are longer, more sophisticated, and more detailed. These articles are best for patients who want in-depth information and are comfortable with some medical jargon.

This topic currently has no corresponding Beyond the Basics content.

Professional level informationProfessional level articles are designed to keep doctors and other health professionals up-to-date on the latest medical findings. These articles are thorough, long, and complex, and they contain multiple references to the research on which they are based. Professional level articles are best for people who are comfortable with a lot of medical terminology and who want to read the same materials their doctors are reading.

Donor selection for hematopoietic cell transplantationHematopoietic support after hematopoietic cell transplantationImmunotherapy for the prevention and treatment of relapse following hematopoietic cell transplantationPreparative regimens for hematopoietic cell transplantationPrevention of acute graft-versus-host diseaseSources of hematopoietic stem cellsTreatment of chronic graft-versus-host diseasePrevention of infections in hematopoietic cell transplant recipientsFertility preservation in patients undergoing gonadotoxic treatment or gonadal resectionMalignancy after hematopoietic cell transplantation

The following organizations also provide reliable health information.

National Library of Medicine

(www.nlm.nih.gov/medlineplus/healthtopics.html)

National Marrow Donor Program

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Hematopoietic cell transplantation (bone marrow ...

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bone marrow/stem cell transplant – verywell.com

By JoanneRUSSELL25

If you or a loved one will be having a bone marrow transplant or donating stem cells, what does it entail? What are the different types of bone marrow transplants and what is the experience like for both the donor and recipient?

A bone marrow transplant is a procedure in which when special cells (called stem cells) are removed from the bone marrow or peripheral blood, filtered and given back either to the same person or to another person.

Since we now derive most stem cells needed from the blood rather than the bone marrow, a bone marrow transplant is now more commonly referred to as stem cell transplant.

Bone marrow is found in larger bones in the body such as the pelvic bones. This bone marrow is the manufacturing site for stem cells. Stem cells are "pluripotential" meaning that the cells are the precursor cells which can evolve into the different types of blood cells, such as white blood cells, red blood cells, and platelets.

If something is wrong with the bone marrow or the production of blood cells is decreased, a person can become very ill or die. In conditions such as aplastic anemia, the bone marrow stops producing blood cells needed for the body. In diseases such as leukemia, the bone marrow produces abnormal blood cells.

The purpose of a bone marrow transplant is thus to replace cells not being produced or replace unhealthy stem cells with healthy ones.

This can be used to treat or even cure the disease.

In addition for leukemias, lymphomas, and aplastic anemia, stem cell transplants are being evaluated for many disorders, ranging from solid tumors to other non-malignant disorders of the bone marrow, to multiple sclerosis.

There are two primary types of bone marrow transplants, autologous and allogeneic transplants.

The Greek prefix "auto" means "self." In an autologous transplant, the donor is the person who will also receive the transplant. This procedure, also known as a "rescue transplant" involves removing your stem cells and freezing them. You then receive high dose chemotherapy followed by infusion of the thawed out frozen stem cells. It may be used to treat leukemias, lymphomas, or multiple myeloma.

The Greek prefix "allo" means "different" or "other." In an allogeneic bone marrow transplant, the donor is another person who has a genetic tissue type similar to the person needing the transplant. Because tissue types are inherited, similar to hair color or eye color, it is more likely that you will find a suitable donor in a family member, especially a sibling. Unfortunately, this occurs only 25 to 30 percent of the time.

If a family member does not match the recipient, the National Marrow Donor Program Registry database can be searched for an unrelated individual whose tissue type is a close match. It is more likely that a donor who comes from the same racial or ethnic group as the recipient will have the same tissue traits.

Learn more about finding a donor for a stem cell transplant.

Bone marrow cells can be obtained in three primary ways. These include:

The majority of stem cell transplants are done using PBSC collected by apheresis (peripheral blood stem cell transplants.) This method appears to provide better results for both the donor and recipient. There still may be situations in which a traditional bone marrow harvest is done.

Donating stem cells or bone marrow is fairly easy. In most cases, a donation is made using circulating stem cells (PBSC) collected by apheresis. First, the donor receives injections of a medication for several days that causes stem cells to move out of the bone marrow and into the blood. For the stem cell collection, the donor is connected to a machine by a needle inserted in the vein (like for donating blood.) Blood is taken from the vein, filtered by the machine to collect the stem cells, then returned back to the donor through a needle in the other arm. There is almost no need for a recovery time with this procedure.

If stem cells are collected by bone marrow harvest (much less likely,) the donor will go to the operating room and while asleep under anesthesia, a needle will be inserted into either the hip or the breastbone to take out some bone marrow. After awakening, there may be some pain where the needle was inserted.

A bone marrow transplant can be a very challenging procedure for the recipient.

The first step is usually receiving high doses of chemotherapy and/or radiation to eliminate whatever bone marrow is present. For example, with leukemia, it is first important to remove all of the abnormal bone marrow cells.

Once a person's original bone marrow is destroyed, the new stem cells are injected intravenously, similar to a blood transfusion. The stem cells then find their way to the bone and start to grow and produce more cells (called engraftment.)

There are many potential complications. The most critical time is usually when the bone marrow is destroyed so that few blood cells remain. Destruction of the bone marrow results in greatly reduced numbers of all of the types of blood cells (pancytopenia.) Without white blood cells there is a serious risk of infection, and infection precautions are used in the hospital (isolation.) Low levels of red blood cells (anemia) often require blood transfusions while waiting for the new stem cells to begin growing. Low levels of platelets (thrombocytopenia) in the blood can lead to internal bleeding.

A common complication affecting 40 to 80 percent of recipients is graft versus host disease. This occurs when white blood cells (T cells) in the donated cells (graft) attack tissues in the recipient (the host,) and can be life-threatening.

An alternative approach referred to as a non-myeloablative bone marrow transplant or "mini-bone marrow transplant" is somewhat different. In this procedure, lower doses of chemotherapy are given that do not completely wipe out or "ablate" the bone marrow as in a typical bone marrow transplant. This approach may be used for someone who is older or otherwise might not tolerate the traditional procedure. In this case, the transplant works differently to treat the disease as well. Instead of replacing the bone marrow, the donated marrow can attack cancerous cells left in the body in a process referred to as "graft versus malignancy."

If you'd like to become a volunteer donor, the process is straightforward and simple. Anyone between the ages of 18 and 60 and in good health can become a donor. There is a form to fill out and a blood sample to give; you can find all the information you need at the National Marrow Donor Program Web site. You can join a donor drive in your area or go to a local Donor Center to have the blood test done.

When a person volunteers to be a donor, his or her particular blood tissue traits, as determined by a special blood test (histocompatibility antigen test,) are recorded in the Registry. This "tissue typing" is different from a person's A, B, or O blood type. The Registry record also contains contact information for the donor, should a tissue type match be made.

Bone marrow transplants can be either autologous (from yourself) or allogeneic (from another person.) Stem cells are obtained either from peripheral blood, a bone marrow harvest or from cord blood that is saved at birth.

For a donor, the process is relatively easy. For the recipient, it can be a long and difficult process, especially when high doses of chemotherapy are needed to eliminate bone marrow. Complications are common and can include infections, bleeding, and graft versus host disease among others.

That said, bone marrow transplants can treat and even cure some diseases which had previously been almost uniformly fatal. While finding a donor was more challenging in the past, the National Marrow Donor Program has expanded such that many people without a compatible family member are now able to have a bone marrow/stem cell transplant.

Sources:

American Society of Clinical Oncology. Cancer.Net. What is a Stem Cell Transplant (Bone Marrow Transplant)? Updated 01/16. http://www.cancer.net/navigating-cancer-care/how-cancer-treated/bone-marrowstem-cell-transplantation/what-stem-cell-transplant-bone-marrow-transplant

U.S. National Library of Medicine. MedlinePlus. Bone Marrow Transplant. Updated 10/03/17. https://medlineplus.gov/ency/article/003009.htm

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Cell Replacement Therapy For Parkinsons Disease And The …

By LizaAVILA

The following was written withProf. Gerold Riempp, a professor of information systems who was diagnosed with Parkinsons disease 16 years ago at age 36. He is co-founder of a charitable organization in Germany that supports the development of therapies that aim to cure PD.

The idea behind cell replacement therapy(CRT) for PD is pretty simple: lack of mobility in PD is the result of the dysfunction and death of a specific kind of cell in the midbrain. While there are a few other things that go wrong in PD, the progressive loss of motor skills is the biggest problem most diagnosed face. Since we are reasonably sure that this lack of mobility results from the impairment and death of dopamine producing cells in an area of the midbrain called the substantia nigra,why not try to replace those cells?

A group of iPS cells grown from human skin tissue at Osaka University

Replacing those cells is one of three core problems that each person diagnosed with PD needs to address. They are:

1. Keeping remaining cells healthyOnce diagnosed, most people have already lost production of 50-80% of dopamine in their midbrain. The problem then is to stop further disease progression by figuring out how to get rid of everything that might be harming the remaining 20-50% of cells while giving their body everything it needs to keep those cells alive and active.

2. Clearing clogged cellsOf those 50-80% of non-dopamine producing cells, a portion are still alive, they are just not doing their job, producing dopamine. This impairment is a result of a range of interrelated factors that harm the cells and eventually lead to their death. Most researchers believe the problem can be boiled down to the clumping of a misfolded protein called alpha-synuclein. Many different methods are being tried in labs around the world to clear these clumps and stop more from accumulating. But this might only be part of the story since a wide variety of other factors also lead to cell death.

3. Replacing dead cellsThen we come to what to do about all of those dead cells. A couple of different options are being considered to get the brain tostimulate the production of new neurons orreplace the function of dead ones. However, the most promising therapy being developed is stem cell therapy, now commonly referred to as cell replacement therapy. It works by placing new dopamine producing neurons into the part of the brain where the dead neurons used to release dopamine.

If a patient manages to address problems one and two they might have no need for CRT. The reason for this is that he or she can likely rescue a considerable portion of the damaged but still living cells and thereby bring dopamine production back to a level that allows for normal movement. CRT will generally be for people who have had PD for a longer time and whose remaining healthy cells plus the rescued ones together are not capable of providing enough dopamine.

The late 80s and 90s saw a number of CRT trials for Parkinsons disease with mixed results. But we nowhave a much better understanding of what kind of cells to use, how to culture and store those cells, how to implant them, and who this therapy would be best for.

We also now have iPS cells (induced pluripotent stem cells). Discovered in 2006, these are cells that have been chemically reprogrammed, usually from adult skin tissue, back into pluripotent stem cells. (Pluripotent means they are capable of becoming almost any cell in the body). Using these cells for transplantation has two major advantages. One, it eliminates the need for potentially harmful immuno-suppressors. Two, it has none of the ethical issues that come with using fetal stem cells. But iPS cells are much more expensive and technically difficult to produce.

Despite all the progress made, cell replacement therapy is still very controversial and fraught with all sorts of technical issues. Luckily, CRT for PD is one of the only fields of medical science where the top labs around the world are cooperating with each other. An international consortium of labs has come together under a name that sounds like it was ripped out of a Marvel comic, the GForce-PD. Each lab in the GForce-PD aims to bring CRT for PD to clinical trial within the next few years.

Infographic made by PhD neuroscientist Kayleen Schreiber at kayleenschreiber.com

The GForce-PD

New York City Run by Dr. Lorenz Studer out of the Rockefeller research labs in New York City. Dr. Studer pioneered many of the reprogramming techniques being used around the world to convert pluripotent stem cells into dopamine producing neurons. His lab wasrecently announced to be part of a huge funding initiative from Bayer Pharmaceuticals to help speed up development of CRT. Studers lab is aiming to start transplantation of embryonic stem cells in human trials in early 2018.

Kyoto, Japan Dr. Jun Takahashis lab in Kyoto is working on producing several iPS lines for the Japanese population. One advantage they have is the relative homogeneity of Japanese people allows them to use a dozen or so iPS lines for almost everyone in the country. The lab recently made headlines with results from monkey trials that showed human iPS cells graft safely, with no signs of malignant growth, two years after transplantation.

Cambridge, England Dr. Roger Barkers lab has been working on cell replacement therapy for Parkinsons disease for a number of years through the Transeuro project. His lab is pushing forward with more embryonic stem cell transplantations expected to begin in 2020. They also work very closely with the team in Sweden.

Lund, Sweden The lab in Lund has been working on CRT for PD since the 80s and has been part of a number of human trials. The lab is now run by Dr. Malin Parmar whose team has also pioneered many of the techniques used in direct programming that will one day allow researchers to skip the stem cell phase all together and produce dopamine cells directly in the brain.

San Diego, California The team is moving rapidly towards iPS cell transplantation under Dr. Jeanne Loring at the Scripps research center. They are the only lab that uses patients own cells for transplantation. Another unique feature of this lab is that it has been a community funded initiative under theSummit For Stem Cellsfoundation.

(Dr. Roger Barker talking about CRT for PD)

Though there is a lot of excitement building around cell replacement therapy, we need to proceed carefully. The field has potential for setbacks from some of the less rigorous trials being conducted in places like Australia and China where regulatory standards are more lax. Researchers in these areas are already going ahead with trials that do not meet the standards set by the GForce-PD. These have the potential to put a black-eye on all cell replacement therapies.

Also, producing pure batches of dopamine neurons is still a highly technical process that only a few labs in the world are capable of doing safely and effectively. Thankfully a few other labs around the world are joining the efforts of the GForce-PD, such as Dr. Tilo Kunaths lab in Edinburgh, which is working on techniques to better differentiate and characterize the cell lines used for transplantation.

(The pictures above show human embryonic stem cells being differentiated into dopamine cells at days 2, 4 and 7. Courtesy of Dr. Tilo Kunaths lab at the University of Edinburgh)

The Future of Cell Replacement Therapy

These therapies being developed for Parkinsons disease will, in essence, be version 1.0 of CRT. Clinical trials are set to begin next year and the therapy is expected to be widely available to people diagnosed with Parkinsons disease within the next 5-10 years.

Version 2.0 will be CRISPR-modified, disease resistant grafts, with genetic switches to modulate dopamine production and graft size.

Version 3.0 will make use of an emerging field called in vivo direct programming where viruses are inserted into the brain and transform other existing cells into dopamine producing cells.

(Edit: Credit to Dr. Tilo Kunath for correcting versions 2.0 and 3.0)

Dopamine neurons grown from iPS cells at 40 times magnification, from the Gladstone Institute

CRT for PD is one of the most exciting areas of research on the planet. It is a powerful demonstration of the progress we as a species have made in our attempt to gain mastery over the forces of biology.It has the potential to improve the lives of the millions living with PD, and the millions yet to be diagnosed. Once the transplanted cells have connected with their surroundings and start delivering dopamine to the right places, it should allow patients to gradually reduce their medication. Being able to move normally and not deal with the side effects of all the drugs and other therapies is what PD patients around the world are dreaming of.

Click here for more information on the future of cell replacement therapy for Parkinsons disease and the work of the GForce-PD.

And if you want to be part of bringing CRT to the clinic you can do so by supporting organizations like Summit For Stem Cells.

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Stuck on You: Nanogel Capsule Helps Cardiac Stem Cells …

By JoanneRUSSELL25

Stem cells (magenta with green nuclei) encapsulated in nanogel (yellow) for heart repair. Credit: MolGraphics.com

Cardiac stem cell therapy is a promising treatment for damaged hearts. However, researchers are still working on two major issues with the therapy how to keep the stem cells in place and how to prevent rejection when the stem cells are not from the patients own body. A new approach from NC State researcher Ke Cheng and a team of international collaborators may solve both of these problems.

The heart is a powerful muscle. Thats great if youre running a marathon, but if youre trying to inject stem cells into the heart with the hopes that theyll stay put, its a problem. One of the major drawbacks of cardiac stem cell therapy is simply that the cells do not stick to the injured heart tissue.

Enter a thermosensitive nanogel that is liquid at room temperature but becomes a thick, sticky gel as it warms. Cheng, associate professor of molecular biomedical sciences at NC States College of Veterinary Medicine and associate professor in the NC State/UNC Joint Department of Biomedical Engineering, partnered with chemical engineers from the University of Adelaide and cardiac specialists from the University of Zhengzhou and UNC-Chapel Hill to test the nanogel as a delivery mechanism for cardiac stem cells.

The nanogel, poly(N-isopropylacrylamine-co-acrylic acid), or P(NIPAM-AA) for short, had another property that made it appealing for use: in its thickened state it had porous openings large enough for a stem cells healing factors to escape, but not large enough for immune cells to enter. And it could be adjusted to slowly degrade over time, giving stem cells enough time to repair a damaged heart before dissolving away.

Autologous stem cells grown from a patients own cells are ideal to use in therapies, but that isnt always practical, Cheng says. For one thing, growing the cells takes time, which a patient may not have. For another, the heart cells themselves may be affected by disease, so stem cells taken from that source would not be useful.

Thats why were working on allogeneic stem cell therapies, but whenever you introduce cells from an outside source into the body, the immune system will attack them. The nanogel delivery method keeps the cells in place, protects them from the bodys immune response, and allows the regenerative factors released by the stem cells to reach the heart.

Cheng and his collaborators tested the nanogel delivery system in mice and pigs with hearts damaged by a heart attack. Without the nanogel, only about one percent of injected stem cells stayed in the heart. With the gel, up to 15 percent of the stem cells stayed put. They also found that in both animal models heart function improved three to four weeks after treatment. Mice showed a greater improvement than pigs, but in both models heart function was maintained and did not decrease.

We are pleased with these results, Cheng says. The nanogel is a safe, cost-effective way to deliver the cells directly to the affected area, and the large animal (pig) data is promising, which may lead to a human clinical trial in the future.

Chengs work appears in the journal ACS Nano, and was supported by the NIH and by NC States Chancellors Faculty Excellence Program and Chancellors Innovation Fund.

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Stem cell and bone marrow transplants – NHS Choices

By daniellenierenberg

A stem cell or bone marrow transplant replaces damaged blood cells with healthy ones. It can be used to treat conditions affecting the blood cells, such as leukaemia and lymphoma.

Stem cells arespecial cells produced bybone marrow (aspongytissue found in the centre of some bones) that can turn into different types of blood cells.

The three maintypes of blood cellthey can become are:

A stem cell transplant involves destroying any unhealthy blood cells and replacing them with stem cells removed from the blood or bone marrow.

Stem cell transplants are used to treat conditions in which the bone marrow is damaged and is no longer able to produce healthy blood cells.

Transplants can also be carried out to replace blood cells that are damaged or destroyed as a result of intensive cancer treatment.

Conditions that stem cell transplants can be used to treat include:

A stem cell transplant will usually only be carried out if other treatments haven't helped, the potential benefits of a transplant outweigh the risks and you're in relatively good health, despite your underlying condition.

A stem cell transplant can involve taking healthy stem cells from the blood or bone marrow of one person ideally a close family member with the same or similar tissue type (see below) and transferring them to another person. This is called an allogeneic transplant.

It's also possible to remove stem cells from your own body and transplant them later, after any damaged or diseased cells have been removed. This is called an autologous transplant.

Astem celltransplant has five main stages. These are:

Having a stem cell transplant can be an intensive and challenging experience. You'll usually need to stay in hospital fora month or more until the transplant starts to take effect and itcan takea year or two to fully recover.

Read more about what happens during a stem cell transplant.

Stem celltransplants arecomplicated procedures with significant risks. It's important that you're aware of both the risks and possible benefits before treatment begins.

Possible problems that can occur during or after the transplant process include:

Read more about the risks of having a stem cell transplant.

Ifit isn't possible to use your own stem cells for the transplant (see above), stem cells will need to come from a donor.

To improve the chances ofthetransplant being successful, donated stem cells need tocarry a special genetic marker known as a human leukocyte antigen (HLA) that'sidentical or very similar to that of the person receiving the transplant.

The best chance of getting a match is from a brother or sister, or sometimes another close family member. If there are no matches in your close family,a search of theBritish Bone Marrow Registry will be carried out.

Most peoplewill eventually find a donor in the registry,although a small number of people may find it very hard or impossibleto find a suitable match.

The NHS Blood and Transplant website has more information about stem cell and bone marrow donation.

Page last reviewed: 08/10/2015

Next review due: 01/10/2018

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Heart Failure and Transplant Program New Jersey

By Sykes24Tracey

New Jersey's Premier Heart Transplant Program

The Barnabas Health Heart Failure and Transplant program is the most experienced and comprehensive center in New Jersey. It has been at the forefront of highly specialized care for more than twenty years. Our cardiac surgeons and heart specialists' expertise has made RWJBarnabas Health a regional and national training center for physicians, nurses and emergency services technicians.

Dr. Joseph Clemente, host of MHC-TV, interviews Mark J. Zucker, M.D., J.D., Director, Heart Failure Treatment and Transplant Program at Newark Beth Israel Medical Center. They discuss getting a heart transplant, other treatments such as the Left Ventricular Assist Device (LVAD), and about stem cell research ongoing today.

Learn about CardioMEMS, a new treatment for heart failure

Barnabas Health Heart Centers Earn Recognition from American Heart Association in Heart Failure Treatment

The Barnabas Health Heart Centers provide progressive heart failure management that optimizes the patient's transition to home and empowers them to improve their heart health. Advanced practice nurses contact patients within days of discharge and facilitate daily medical monitoring, education, and counseling. Patient-centered programs bring together all the medical resources necessary to combat heart failure and the reduction in hospital readmission rates has been significant. Heart failure clinics are based at all Barnabas Health hospitals.

Clinical trials provide patients with access for breakthrough medications, devices, and therapies, while being continually monitored and evaluated. Our participation in new cardiac stem cell research for patients with refractory angina and chronic myocardial ischemia may someday help the heart heal itself. Improved technology has made positive outcomes with ECMO more likely both at our center and nationally, and has resulted in increased referrals for this critical care.

The Newark Beth Israel Medical Center heart transplant program is one of the top ten heart transplant centers in the United States with survival rates that consistently meet or exceed national benchmarks. The center is at the forefront of improving the quality of life for transplant candidates and recipients, as well as increasing access to transplant. Our unique work in establishing successful protocols for discontinuing steroid medications for immunosuppression is improving the medical management and survival rates for transplant recipients worldwide. Noninvasive gene expression testing offers an alternative technique for assessing immunosuppression and predicting rejection. The center is also part of groundbreaking research that is exploring innovating methods for preserving donor organs for transplant.

Implantable VADs are placed for myocardial recovery, bridge to transplant, and destination therapy. Because the VAD program is totally integrated with heart failure and transplant services, patients are thoroughly and continually evaluated for all treatment options. The heart transplant center's experience has made it a principal site for clinical research trials of the latest generation of mechanical assist devices and a regional VAD transplantation training site. With virtually all FDA-approved and investigational implantable devices available, patients receive the device that meets their individual needs.

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Skin Stem Cells – Methods and Protocols | Kursad Turksen …

By Sykes24Tracey

During the last decade, an increased interest in somatic stem cells has led to a flurry of research on one of the most accessible tissues of the body: skin. Much effort has focused on such topics as understanding the heterogeneity of stem cell pools within the epidermis and dermis, and their comparative utility in regenerative medicine applications. In Skin Stem Cells: Methods and Protocols, expert researchers in the field detail many of the methods which are now commonly used to study skin stem cells. These include methods and techniques for the isolation, maintenance and characterization of stem cell populations from skin. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and key tips on troubleshooting and avoiding known pitfalls.

Authoritative and practical, Skin Stem Cells: Methods and Protocols seeks to aid scientists in the further understanding of these diverse cell types and the translation of their biological potential to the in vivo setting.

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Stem Cells Repair Heart in First-Ever Study – webmd.com

By LizaAVILA

Nov. 14, 2011 -- The first use of heart stem cells in humans looks like a major breakthrough for people suffering heart failure after heart attacks.

It's early -- results are in for only the first 16 patients -- but the results already are drawing praise from experts not easily impressed by first reports.

"This is a groundbreaking study of extreme importance," Joshua Hare, MD, director of the University of Miami's Interdisciplinary Stem Cell Institute, tells WebMD via email. Hare was not involved in the study.

"The reported benefits are of an unexpected magnitude," writes Gerd Heusch, MD, PhD, chair of the Institute of Pathophysiology at the University of Essen, Germany, in an editorial in the Nov. 14 online issue of The Lancet.

Study researcher John H. Loughran, MD, of the University of Louisville, Ky., could barely contain his excitement in an interview with WebMD.

"The improvement we have seen in patients is quite encouraging," he says. "Michael Jones, our first patient, could barely walk 30 feet [before treatment]. I saw him this morning. He says he plays basketball with his granddaughter, works on his farm, and gets on the treadmill for 30 minutes three times a week. It is stories like that that makes these results really encouraging."

The breakthrough comes just as researchers were becoming discouraged by studies in which bone-marrow stem cells failed to heal damaged hearts.

Instead of getting stem cells from the bone marrow, the new technique harvests stem cells taken from the patients' own hearts during bypass surgery. Just 1 gram of heart tissue -- about 3.5 hundredths of an ounce -- is taken.

Using a technique invented by Brigham & Women's Hospital researchers Piero Anversa, MD, and colleagues, heart stem cells are taken from the tissue and grown in the lab. These adult stem cells already are committed to becoming heart cells, but they can transform into any of the three different kinds of heart tissues.

It's the first time tissue-specific stem cells, other than bone-marrow cells, have been tested in humans, Hare says.

In the study, about a million of the cells were infused into each patient's heart with a catheter. Calculations suggest that each of these infused cells could generate 4 trillion new heart cells.

The study was designed to show whether the technique was safe. It was: No harmful effects have been seen. But to the researchers' surprise, the relatively small number of cells infused into patients had a major effect.

Of the 14 patients analyzed so far, heart function improved dramatically. And in the eight patients seen one year after treatment, improvement appears to have continued. Moreover, the scars on patients hearts -- areas of dead tissue killed during their heart attacks -- are healing.

And patients aren't just doing better on measures of heart function. Like Michael Jones, they report vastly improved quality of life and ability to perform daily tasks.

"Now this is an open-label trial, so patients know they are treated. This means we have to take what they say with a grain of salt," Loughran says. "But we see these patients not only are feeling better but doing more."

The only downside of this early success is that the ongoing study already has enrolled all 20 of the patients who will be treated. The experimental treatment simply will not be available to other patients in the near future. A larger, phase II study is planned.

"All the patients that call in to us, and there are quite a few interested, we encourage them to maintain close contact with their doctors," Loughran says. "Lifestyle changes and medical management are the most important things for them right now. We will be working very hard to get new trials under way."

The findings were reported at the American Heart Associations Scientific Sessions meeting in Orlando, Fla., and in the Nov. 14 online edition of The Lancet.

SOURCES:

John H. Loughran, MD, fellow in cardiovascular medicine, University of Louisville, Ky.

Joshua Hare, MD, director, Interdisciplinary Stem Cell Institute, University of Miami.

Bolli, R. The Lancet, published online Nov. 14, 2011.

Heusch, G. The Lancet, published online Nov. 14, 2011.

Traverse, J.H. Journal of the American Medical Association, published online Nov. 14, 2011.

Hare, J. Journal of the American Medical Association, published online Nov. 14, 2011.

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