Margot Martini's family launch awareness day

The family of little Margot Martini has announced the launch of a day to promote the need for potential stem cell donors to join UK and worldwide registries.

A year ago today the brave youngster was diagnosed with both acute lymphoblastic leukaemia (ALL) and acute myeloid leukaemia (AML). She was just 14 months old at the time.

A worldwide hunt was launched to find a perfect stem cell match, leading to more than 50,000 people coming forward to be tested under the Swab4Margot campaign.

Finally in February a suitable donor was found and she underwent a bone marrow transplant. The following month doctors declared she was 99 per cent certain to be disease free, but tests in June revealed a small amount of ALL had returned.

Margot's parents Yaser and Vicki made the difficult decision in July to stop her treatment after medical experts told them her chances of being able to live a normal life after further treatment were slim.

Now two-year-old, Margot is living out the rest of her days at home with her loving family, but throughout her brave fight Team Margot - the group formed to support her battle - has remained committed to promoting awareness around the need for more potential stem cell donors to come forward. As a result of the group's efforts in the UK, more than 500 people will now receive potentially life saving bone marrow transplants.

Booked to take place on October 7, 2015, the inaugural Team Margot Stem Cell and Bone Marrow Awareness Day will give particular encouragement to those of mixed race to register, as this is an under-represented stem bank.

There are 37,000 people worldwide who are desperately seeking a stem cell match and only 60 per cent of transplant recipients currently find a perfect donor.

Of those from a black, Asian or ethnic minority background, a little more than 20 per cent of transplant recipients ever find their perfect match.

Yaser Martini said: "Team Margot is committed to raising global awareness around the need for more stem cell donor recruits, hence the launch of this annual international awareness day to help promote greater understanding and to encourage more people to register as potential stem cell /bone marrow donors.

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Margot Martini's family launch awareness day

Stem cells discovered in the esophagus

Despite previous indications to the contrary, the esophagus does have its own pool of stem cells, said researchers from the University of Pittsburgh School of Medicine in an animal study published online today in Cell Reports. The findings could lead to new insights into the development and treatment of esophageal cancer and the precancerous condition known as Barrett's esophagus.

According to the American Cancer Society, more than 18,000 people will be diagnosed with esophageal cancer in the U.S. in 2014 and almost 15,500 people will die from it. In Barrett's esophagus, the lining of the esophagus changes for unknown reasons to resemble that of the intestine, though gastro-esophageal reflux disease or GERD is a risk factor for its development.

"The esophageal lining must renew regularly as cells slough off into the gastrointestinal tract," said senior investigator Eric Lagasse, Pharm.D., Ph.D., associate professor of pathology, Pitt School of Medicine, and director of the Cancer Stem Cell Center at the McGowan Institute for Regenerative Medicine. "To do that, cells in the deeper layers of the esophagus divide about twice a week to produce daughter cells that become the specialized cells of the lining. Until now, we haven't been able to determine whether all the cells in the deeper layers are the same or if there is a subpopulation of stem cells there."

The research team grew pieces or "organoids" of esophageal tissue from mouse samples, and then conducted experiments to identify and track the different cells in the basal layer of the tissue. They found a small population of cells that divide more slowly, are more primitive, can generate specialized or differentiated cells, and have the ability to self-renew, which is a defining trait of stem cells.

"It was thought that there were no stem cells in the esophagus because all the cells were dividing rather than resting or quiescent, which is more typical of stem cells," Dr. Lagasse noted. "Our findings reveal that there indeed are esophageal stem cells, and rather than being quiescent, they divide slowly compared to the rest of the deeper layer cells."

In future work, the researchers will examine human esophageal tissues for evidence of stem cell dysfunction in Barrett's esophagus disease.

"Some scientists have speculated that abnormalities of esophageal stem cells could be the origin of the tissue changes that occur in Barrett's disease," Dr. Lagasse said. "Our current and future studies could make it possible to test this long-standing hypothesis."

Story Source:

The above story is based on materials provided by University of Pittsburgh Schools of the Health Sciences. Note: Materials may be edited for content and length.

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Stem cells discovered in the esophagus

Pitt/McGowan Institute team discovers stem cells in the esophagus

PUBLIC RELEASE DATE:

16-Oct-2014

Contact: Anita Srikameswaran SrikamAV@upmc.edu 412-578-9193 University of Pittsburgh Schools of the Health Sciences @UPMCnews

PITTSBURGH, Oct. 16, 2014 Despite previous indications to the contrary, the esophagus does have its own pool of stem cells, said researchers from the University of Pittsburgh School of Medicine in an animal study published online today in Cell Reports. The findings could lead to new insights into the development and treatment of esophageal cancer and the precancerous condition known as Barrett's esophagus.

According to the American Cancer Society, more than 18,000 people will be diagnosed with esophageal cancer in the U.S. in 2014 and almost 15,500 people will die from it. In Barrett's esophagus, the lining of the esophagus changes for unknown reasons to resemble that of the intestine, though gastro-esophageal reflux disease or GERD is a risk factor for its development.

"The esophageal lining must renew regularly as cells slough off into the gastrointestinal tract," said senior investigator Eric Lagasse, Pharm.D., Ph.D., associate professor of pathology, Pitt School of Medicine, and director of the Cancer Stem Cell Center at the McGowan Institute for Regenerative Medicine. "To do that, cells in the deeper layers of the esophagus divide about twice a week to produce daughter cells that become the specialized cells of the lining. Until now, we haven't been able to determine whether all the cells in the deeper layers are the same or if there is a subpopulation of stem cells there."

The research team grew pieces or "organoids" of esophageal tissue from mouse samples, and then conducted experiments to identify and track the different cells in the basal layer of the tissue. They found a small population of cells that divide more slowly, are more primitive, can generate specialized or differentiated cells, and have the ability to self-renew, which is a defining trait of stem cells.

"It was thought that there were no stem cells in the esophagus because all the cells were dividing rather than resting or quiescent, which is more typical of stem cells," Dr. Lagasse noted. "Our findings reveal that there indeed are esophageal stem cells, and rather than being quiescent, they divide slowly compared to the rest of the deeper layer cells."

In future work, the researchers will examine human esophageal tissues for evidence of stem cell dysfunction in Barrett's esophagus disease.

"Some scientists have speculated that abnormalities of esophageal stem cells could be the origin of the tissue changes that occur in Barrett's disease," Dr. Lagasse said. "Our current and future studies could make it possible to test this long-standing hypothesis."

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Pitt/McGowan Institute team discovers stem cells in the esophagus

Embryonic Stem Cell Therapy Shows Long-Term Effectiveness, Safety

Posted: Tuesday, October 14, 2014, 7:00 PM

TUESDAY, Oct. 14, 2014 (HealthDay News) -- A new study is the first to show the long-term safety of embryonic stem cell transplants to treat human disease.

The research involved 18 people who received the transplants to treat forms of macular degeneration, a leading cause of vision loss.

The transplants, which restored some sight in more than half of the patients, appeared safe up to three years after the procedure.

The study, funded by a U.S.-based company called Advanced Cell Technology, was published Oct. 14 in The Lancet.

"Embryonic stem cells have the potential to become any cell type in the body, but transplantation has been complicated by problems," lead author Dr. Robert Lanza, chief scientific officer at Advanced Cell Technology, said in a journal news release. Those problems include the rejection of the transplanted cells by the patient's immune system, as well as the danger that the cells might spur certain types of cancers called teratomas.

A teratoma is a type of cancer that occurs when stem cells develop into multiple types of cells and form incompatible tissues that can include teeth and hair.

As Lanza explained, because of these issues, scientists interested in embryonic stem cell therapy have tended to focused on sites in the body that typically do not produce a strong immune response. The eye is one such spot.

In the new study, human embryonic stem cells were first prompted to develop into eye cells called retinal pigment epithelial cells. They were then transplanted into nine people with Stargardt's macular dystrophy, and another nine with dry atrophic age-related macular degeneration.

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Embryonic Stem Cell Therapy Shows Long-Term Effectiveness, Safety

Stem Cell Eye Treatment May Restore Vision

Antonio Regalado for MIT Technology Review 2014-10-15 19:15:44 UTC

When stem cells were first culled from human embryos sixteen years ago, scientists imagined they would soon be treating diabetes, heart disease, stroke, and many other diseases with cells manufactured in the lab.

It's all taken longer than they thought. But now, a Massachusetts biotech firm has reported results from the largest, and longest, human test of a treatment based on embryonic stem cells, saying it appears safe and may have partly restored vision to patients going blind from degenerative diseases.

Results of three-year study were described Tuesday in the Lancet by Advanced Cell Technology and collaborating eye specialists at the Jules Stein Eye Institute in Los Angeles who transplanted lab-grown cells into the eyes of nine people with macular degeneration and nine with Stargardt's macular dystrophy.

The idea behind Advanced Cell's treatment is to replace retinal pigment epithelium cells, known as RPE cells, a type of caretaker tissue without which a person's photoreceptors also die, with supplies grown in laboratory. It uses embryonic stem cells as a starting point, coaxing them to generate millions of specialized retina cells. In the study, each patient received a transplant of between 50,000 and 150,000 of those cells into one eye.

The main objective of the study was to prove the cells were safe. Beyond seeing no worrisome side effects, the researchers also noted some improvements in the patients. According to the researchers half of them improved enough to read two to three extra lines on an eye exam chart, results Robert Lanza, chief scientific officer of Advanced Cell, called remarkable.

"We have people saying things no one would make up, like 'Oh I can see the pattern on my furniture, or now I drive to the airport," he says. "Clearly there is something going on here."

Lanza stressed the need for a larger study, which he said the company hoped to launch later this year in Stargardt's patients. But if the vision results seen so far continue, Lanza says "this would be a therapy."

Some eye specialists said it's too soon to say whether the vision improvements were real. The patients weren't examined by independent specialists, they said, and eyesight in patients with low vision is notoriously difficult to measure. That leaves plenty of room for placebo effects or unconscious bias on the part of doctors.

"When someone gets a treatment, they try really hard to read the eye chart," says Stephen Tsang, a doctor at Columbia University who sees patients losing their vision to both diseases. It's common for patients to show quick improvements, he says, although typically not as large as what Advanced Cell is reporting.

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Stem Cell Eye Treatment May Restore Vision

Stem cell treatment may harbor blindness cure, says study

A new study has revealed that stem cell treatment may be helpful in treating blindness.According to the study, a pioneering treatment for progressive blindness has been proved safe three years after patients were injected with stem cells derived from human embryos.

The researchers said that more than half of the patients with macular degeneration where the eye's light-sensitive cells are progressively destroyed experienced a significant improvement in their eyesight, but none showed any adverse effects due directly to the transplant of the embryonic cells.

Doctors injected the stem cells into the eyes of 18 patients nine with Stargardt's macular dystrophy and nine with dry, age-related macular degeneration with the ultimate aim of repairing damaged photoreceptors in the retina at the back of the eye.

It was found that about half of the patients had an improvement in visual acuity of three lines or more, which corresponds to a doubling of the visual angle, and is generally accepted as clinically significant.Follow-up testing found that 10 out of the 18 patients experienced substantial improvements in how well they could see.

The study was published in the journal The Lancet.

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Stem cell treatment may harbor blindness cure, says study

Stem cell discovery challenges dogma on how fetus develops; holds insights for liver cancer and reg

PUBLIC RELEASE DATE:

14-Oct-2014

Contact: Greg Williams newsmedia@mssm.edu 212-241-9200 The Mount Sinai Hospital / Mount Sinai School of Medicine @mountsinainyc

A Mount Sinai-led research team has discovered a new kind of stem cell that can become either a liver cell or a cell that lines liver blood vessels, according to a study published today in the journal Stem Cell Reports. The existence of such a cell type contradicts current theory on how organs arise from cell layers in the embryo, and may hold clues to origins of, and future treatment for, liver cancer.

Thanks to stem cells, humans develop from a single cell into a complex being made up of more than 200 cell types. The original, single human stem cell, the fertilized embryo, has the potential to develop into every kind of human cell. Stem cells multiply (proliferate) and specialize (differentiate) until millions of functional cells result, including liver cells (hepatocytes), blood vessel cells (endothelial cells), muscle cells, bone cells, etc.

In the womb, the human embryo early on becomes three "germ" layers of stem cells the endoderm, mesoderm and ectoderm. The long-held consensus was that the endoderm goes on to form the liver and other gut organs; the mesoderm the heart, muscles and blood cells; and the ectoderm the brain and skin. Researchers have sought to determine the germ layer that yields each organ because these origins hold clues to healthy function and disease mechanisms in adults.

"We found a stem cell that can become either a liver cell, which is thought to originate in the endoderm, or an endothelial cell that helps to from a blood vessel, which was thought to derive from the mesoderm," said Valerie Gouon-Evans, PhD, Assistant Professor in the Department of Developmental and Regenerative Biology and Black Family Stem Cell Institute, Icahn School of Medicine at Mount Sinai, and lead author for the study. "Our results go against traditional germ layer theory, which holds that a stem cell can only go on to become cell types in line with the germ layer that stem cell came from. Endothelial cells may arise from both the endoderm and mesoderm."

Cell Growth Plusses and Minuses

Beyond the womb, many human organs contain pools of partially differentiated stem cells, which are ready to differentiate into specific replacement cells as needed. Among these are stem cells that "know" they are liver cells, but have enough "stemness" to become more than one cell type.

By advancing the understanding of stem cell processes in the liver, the study offers insights into mechanisms that drive liver cancer. The rapid growth seen in cells as the fetal liver develops is similar in some ways to the growth seen in tumors. Among the factors that make both possible is the building of blood vessels that supply nutrients and oxygen.

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Stem cell discovery challenges dogma on how fetus develops; holds insights for liver cancer and reg

Could stem cell jab help elderly blind see again?

Elderly people who received treatment had their vision improved, study says Children who suffer from common form of blindness in young also benefited Some can now do things like read their watch and also work on a computer Expert said even small improvements are 'huge difference to quality of life' Critics say it's wrong to plunder unborn child for spare parts for science

By Fiona Macrae Science Correspondent

Published: 18:28 EST, 14 October 2014 | Updated: 02:30 EST, 15 October 2014

A revolutionary stem cell jab has restored the gift of sight, research suggests.

Men and women with severe age-related macular degeneration, the most common form of blindness in the elderly, are able to see better after having tens of thousands of embryonic stem cells injected into the back of their eye.

Children with Stargardts disease, the main cause of blindness in the young, have also benefited.

Researcher Robert Lanza said that one patient who underwent the trial even 'went to the mall for the first time' (file photo)

Some can now do things most of us take for granted like reading their watch or working on a computer. But one man is able to ride horses again and one of the patients has gone to a shopping mall for the first time.

Researcher Robert Lanza, a world-leading stem cell expert, said that even seemingly small improvements have made a huge difference to quality of life. Others described his work as a major accomplishment.

All of those who took part in the landmark trial had advanced eye disease and were blind in one eye. However, Dr Lanzas goal is to treat people early in the disease process to stop them from ever going blind.

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Could stem cell jab help elderly blind see again?

Cell Discovery Challenges Dogma on How Fetus Develops; Holds Insights for Liver Cancer and Regeneration

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Newswise (New York, NY Oct. 14, 2014) A Mount Sinai-led research team has discovered a new kind of stem cell that can become either a liver cell or a cell that lines liver blood vessels, according to a study published today in the journal Stem Cell Reports. The existence of such a cell type contradicts current theory on how organs arise from cell layers in the embryo, and may hold clues to origins of, and future treatment for, liver cancer.

Thanks to stem cells, humans develop from a single cell into a complex being made up of more than 200 cell types. The original, single human stem cell, the fertilized embryo, has the potential to develop into every kind of human cell. Stem cells multiply (proliferate) and specialize (differentiate) until millions of functional cells result, including liver cells (hepatocytes), blood vessel cells (endothelial cells), muscle cells, bone cells, etc.

In the womb, the human embryo early on becomes three germ layers of stem cells the endoderm, mesoderm and ectoderm. The long-held consensus was that the endoderm goes on to form the liver and other gut organs; the mesoderm the heart, muscles and blood cells; and the ectoderm the brain and skin. Researchers have sought to determine the germ layer that yields each organ because these origins hold clues to healthy function and disease mechanisms in adults.

We found a stem cell that can become either a liver cell, which is thought to originate in the endoderm, or an endothelial cell that helps to from a blood vessel, which was thought to derive from the mesoderm, said Valerie Gouon-Evans, PhD, Assistant Professor in the Department of Developmental and Regenerative Biology and Black Family Stem Cell Institute, Icahn School of Medicine at Mount Sinai, and lead author for the study. Our results go against traditional germ layer theory, which holds that a stem cell can only go on to become cell types in line with the germ layer that stem cell came from. Endothelial cells may arise from both the endoderm and mesoderm.

Cell Growth Plusses and Minuses Beyond the womb, many human organs contain pools of partially differentiated stem cells, which are ready to differentiate into specific replacement cells as needed. Among these are stem cells that know they are liver cells, but have enough stemness to become more than one cell type.

By advancing the understanding of stem cell processes in the liver, the study offers insights into mechanisms that drive liver cancer. The rapid growth seen in cells as the fetal liver develops is similar in some ways to the growth seen in tumors. Among the factors that make both possible is the building of blood vessels that supply nutrients and oxygen.

The research teams newfound, liver-based stem cell type has the ability to become part of newly formed blood vessels. Thus, a detailed understanding of it may have a decisive impact on understanding liver cancer progression, said Dr. Gouon-Evans. If similar bi-potential progenitor cells are found in liver cancers, they may be ideal targets for drugs that eradicate not only their descendant liver cancer cells but also the formation of blood vessels that feed tumors.

The new study also has implications for the field of liver regeneration. Many labs seek to understand how the liver repairs itself when damaged, and many clinical trials to determine whether injecting healthy liver cells into damaged livers can repair them.

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Cell Discovery Challenges Dogma on How Fetus Develops; Holds Insights for Liver Cancer and Regeneration

Stem cells successfully treat blindness

Published: 1:48PM Wednesday October 15, 2014 Source: AP

An experimental treatment for blindness that uses embryonic stem cells appears to be safe, and it improved vision in more than half of the patients who got it.

Researchers followed 18 patients for up to three years after treatment.

The studies are the first to show safety of an embryonic stem cell treatment in humans for such a long period.

"It's a wonderful first step but it doesn't prove that (stem cells) work," said Chris Mason, chair of regenerative medicine at University College London, who was not part of the research.

He said it was encouraging the studies proved the treatment is safe and dispelled fears about stem cells promoting tumour growth.

Embryonic stem cells, which are recovered from embryos, can become any cell in the body.

They are considered controversial by some because they involve destroying an embryo and some critics say adult stem cells, which are derived from tissue samples, should be used instead.

Scientists have long thought about transforming them into specific types of cells to help treat various diseases.

In the new research, scientists turned stem cells into retinal cells to treat people with macular degeneration or Stargardt's macular dystrophy, the leading causes of blindness in adults and children.

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Stem cells successfully treat blindness