Artificial embryo shows early potential for medical therapies, not babies – CNN

The artificial structure shows promise as a tool for medical research, though it cannot develop into an actual baby.

After an egg is fertilized by a sperm, it begins to divide multiple times. This process generates a small, free-floating ball of stem cells: a blastocyst.

Within a mammalian blastocyst, the cells that will become the body of the embryo (embryonic stem cells) begin to cluster at one end. Two other types of cells, the extra-embryonic trophoblast stem cells and the endoderm stem cells, begin to form patterns that will eventually become a placenta and a yolk sac, respectively.

To develop further, the blastocyst has to implant in the womb, where it transforms into a more complex architecture. However, implantation hides the embryo from view -- and from experimentation.

In the study, Zernicka-Goetz wanted to replicate developing embryonic events using stem cells.

Other scientists who have attempted the same thing have used only embryonic stem cells, but these experiments, though they have yielded embryoid bodies, have not been entirely successful. The artificial bodies never follow the same chain of events found in nature, and they lack the structure of a natural embryo.

Zernicka-Goetz, a professor in Cambridge's Department of Physiology, Development and Neuroscience, hypothesized that the trophoblast stem cells communicate with the embryonic stem cells and guide their development.

She and her colleagues placed embryonic and trophoblast stem cells within an extra-cellular matrix: the non-cell component found in all tissues and organs that provides biochemical support to cells. This formed a scaffold on which the two stem cell types could co-develop.

The embryonic stem cells sent chemical messages to the trophoblast stem cells and vice versa, said Zernicka-Goetz. Essentially, the different stem cells began to "talk to each other," and this helped the embryonic stem cells, she explained.

"They respond by turning on particular developmental gene circuits or by physically changing shape to accomplish some architectural remodeling," she wrote in an email. "This happens in normal embryogenesis and it is what we are trying to recreate in the culture dish."

Ultimately, the cells organized themselves into a structure that not only looked like an embryo, it behaved like one, with anatomically correct regions developing at the right time and in the right place.

"The results were spectacular -- they formed structures that developed in a way strongly resembling embryos in their architecture and expressing specific genes in the right place and at the right time," Zernicka-Goetz wrote.

Despite its resemblance to a real embryo, this artificial embryo will not develop into a healthy fetus, the researchers said. That would require the endoderm stem cells, which "does other things that are most likely necessary for further development," said Zernicka-Goetz.

"Whether adding these to the system would be enough to achieve further development, I don't know," she said.

"Correct placental development" is essential for proper implantation into "either the womb or a substitute for the womb," she said. "To achieve this will be some time off."

According to Dr. Christos Coutifaris, president-elect of the American Society for Reproductive Medicine and a professor at the University of Pennsylvania, the new study is significant because it shows how "the cells that are extra-embryonic -- the ones that are going to give rise to the placenta -- actually play a role" in the development of cells that eventually become the fetus.

"It's not two completely separate entities," Coutifaris said, referring to the embryo and its support structure. Understanding how the two types of cells interact and the chemical signals they exchange is "really, really critical."

Zernicka-Goetz's model has practical applications in research, where it can be used to better understand the conversation between embryonic stem cells and trophoblast stem cells, he said. "You can manipulate these cells molecularly to try to understand these interactions and how early development occurs pre-implantation."

According to Kyle E. Orwig, an associate professor of obstetrics, gynecology and reproductive sciences, and molecular genetics and biochemistry at the University of Pittsburgh, Zernicka-Goetz's model "will enable investigators to investigate the effects of genetic manipulations, environmental toxins, therapeutics and factors on embryo development." Artificial embryos "represent a powerful tool for research that might reduce (but not eliminate) the need for human embryos," Orwig said.

Dr. David Adamson, a reproductive endocrinologist, an adjunct clinical professor at Stanford University and chairman of the International Committee Monitoring Assisted Reproductive Technologies, believes that it's "very important to continue to do basic science research in reproductive medicine."

"How our species reproduces is very important to know," Adamson said. "When you learn about reproduction and learn how cells reproduce and how cells differentiate and what makes things happen normally and what makes thing happen abnormally, then there clearly are a lot of potential therapeutic applications."

Past advances in reproductive medicine have helped scientists prevent genetic-based diseases, he said. Specifically, in vitro fertilization techniques have allowed doctors to biopsy and conduct genetic tests on embryos to prevent inherited illnesses, including Huntington's.

In vitro fertilization is "fundamentally transformative," said Adamson, who sees the new research as adding to the wealth of knowledge about this procedure.

In fact, Zernicka-Goetz works in the same nondescript brick building on the Cambridge campus where Robert Edwards, a reproductive medicine pioneer, once toiled. Edwards developed the Nobel Prize-winning technique of in vitro fertilization, which eventually resulted in the birth of the first "test tube" baby, Louise Brown.

Helping families have babies is the most obvious contribution of in vitro fertilization. Today, Adamson said, there have been approximately 6.5 million babies born using in vitro fertilization since the procedure was first developed. An exact number is not known because many countries, including China, do not have registries to count them, explained Adamson.

Meanwhile, Zernicka-Goetz said she will continue her work on embryonic development as she and the members of her lab are "totally driven by a curiosity to understand these fundamental aspects of life."

She plans to use human stem cells to create a similar embryonic model. Then she plans to use that model to learn more about normal embryonic development and understand when it goes wrong without needing to experiment on an actual human embryo.

The work also "continually teaches us about the properties of stem cells," Zernicka-Goetz said. She added that this knowledge is useful for developing "therapies to replace faulty tissues in so-called regenerative medicine."

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Artificial embryo shows early potential for medical therapies, not babies - CNN

Doctors Claim They’ve Cured a Boy of a Painful Blood Disorder Using Gene Therapy – Futurism

Potential Treatment

Gene therapy has been available for quite some time now. Advances in modern medical science, particularly in stem cell research, have made it possible to use DNA to compensate for malfunctioning genes in humans. The therapies haveeven proven effective fortreating rare forms of diseases. Now, a research team in France has shown that gene therapy may be used to cure one of the most common genetic diseases in the world.

The team, led by Marina Cavazzana at the Necker Childrens Hospital in Paris, conducted stem cell treatment on a teenage boy with sickle cell disease. The disease alters theblood through beta-globin mutations, which cause abnormalities in the blood proteinhemoglobin. These abnormalities cause the blood cells (which have an irregular shape, like a sickle, hence their name) to clump together. Patients with sickle cell disease usually need transfusions to clear the blockages their cells cause, and some are able to have bone marrow transplants. About 5 percent of the global population has sickle cell disease,according to the WHO. In the United States alone, the CDC reports that approximately 100,000 people have sickle cell disease.

The patient is now 15 years old and free of all previous medication, Cavazzana saidwhen discussing the outcome of their study. He has been free of pain from blood vessel blockages, and has given up taking opioid painkillers. Their research is published in the the New England Journal of Medicine.

The particular treatment given to the teenage boy at Necker Childrens Hospitalbegan when he was 13 years old. The team took bone marrow stem cells from the boy and added mutated versions of the gene that codes for beta-globin before putting these stem cells back into the boys body. The mutated genes were designed to stop hemoglobin from clumping together and blocking blood vessels the hallmark of sickle cell disease.

Two years later, the boys outcomelooks promising.All the tests we performed on his blood show that hes been cured, but more certainty can only come from long-term follow-up, Cavazzan said. Her team also treated seven other patients who also showed promising progress.

If the method shows success in larger scale clinical trials, it could be a game changer, saidDeborah Gill at the University of Oxford, The fact the team has a patient with real clinical benefit, and biological markers to prove it, is a very big deal.

Other research involving gene therapy is also showing similar promise. One which has already been approved by the FDA is a potential treatment for blindness. Others look at treating Parkinsons disease or evenprolonging human life. What these studies show is that gene therapyand stem cells may be able togive hope to patients with diseases that have long been considered incurable.

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Doctors Claim They've Cured a Boy of a Painful Blood Disorder Using Gene Therapy - Futurism

Embryo Experiments Reveal Earliest Human Development, But Stir Ethical Debate – NPR

Notes This is a seven-day-old embryo that scientists kept alive in a laboratory dish. If it developed further, the clusters in green would become cells that shape the body and the red/purple cells would form the placenta.

Ali Brivanlou slides open a glass door at the Rockefeller University in New York to show off his latest experiments probing the mysteries of the human embryo.

"As you can see, all my lab is glass just to make sure there is nothing that happens in some dark rooms that gives people some weird ideas," says Brivanlou, perhaps only half joking.

Brivanlou knows that some of his research makes some people uncomfortable. That's one reason he's agreed to give me a look at what's going on.

His lab and one other discovered how to keep human embryos alive in lab dishes longer than ever before at least 14 days. That's triggered an international debate about a long-standing convention (one that's legally binding in some countries, though not in the U.S.) that prohibits studying human embryos that have developed beyond the two-week stage.

Ali Brivanlou's research team at Rockefeller University in New York was one of two groups internationally that figured out how to keep human embryos alive in lab dishes beyond the 14-day stage of development. Rob Stein/NPR hide caption

And in other experiments, he's using human stem cells to create entities that resemble certain aspects of primitive embryos. Though Brivanlou doesn't think these "embryoids" would be capable of developing into fully formed embryos, their creation has stirred debate about whether embryoids should be subject to the 14-day rule.

Brivanlou says he welcomes these debates. But he hopes society can reach a consensus to permit his work to continue, so he can answer some of humanity's most fundamental questions.

"If I can provide a glimpse of, 'Where did we come from? What happened to us, for us to get here?' I think that, to me, is a strong enough rationale to continue pushing this," he says.

For decades, scientists thought the longest an embryo could survive outside the womb was only about a week. But Brivanlou's lab, and one in Britain, announced last year in the journals Nature and Nature Cell Biology that they had kept human embryos alive for two weeks for the first time.

That enabled the scientists to study living human embryos at a crucial point in their development, a time when they're usually hidden in a woman's womb.

"Women don't even know they are pregnant at that stage. So it has always been a big black box," Brivanlou says.

Gist Croft, a stem cell biologist in Brivanlou's lab, shows me some samples, starting with one that's 12 days old.

"So you can see this with the naked eye," Croft says, pointing to a dish. "In the middle of this well, if you look down, there's a little white speck it looks like a grain of sand or a piece of dust."

Under a microscope, the embryo looks like a fragile ball of overlapping bubbles shimmering in a silvery light with thin hair-like structures extending from all sides.

Croft and Brivanlou explain that those willowy structures are what embryos would normally extend at this stage to search for a place to implant inside the uterus. Scientists used to think embryos could only do that if they were receiving instructions from the mother's body.

"The amazing thing is that it's doing its thing without any information from mom," Brivanlou says. "It just has all the information already in it. That was mind-blowing to me."

The embryos they managed to keep alive in in the lab dish beyond seven days of development have also started secreting hormones and organizing themselves to form the cells needed to create all the tissues and organs in the human body.

The two scientists think studying embryos at this and later stages could lead to discoveries that might point to new ways to stop miscarriages, treat infertility and prevent birth defects.

"The only way to understand what goes wrong is to understand what happens normally, or as normally as we can, so we can prevent all of this," Brivanlou says.

The 14-day cutoff

But Brivanlou isn't keeping these embryos alive longer than 14 days because of the rule.

A long-standing rule prohibits scientists from keeping human embryos alive more than two weeks, after which the central nervous system starts to develop. The 14-day rule, was developed decades ago to avoid raising too many ethical questions about experimenting on human embryos. It's a law in some countries, and just a guideline in the U.S.

"The decision about pulling the plug was probably the toughest decision I've made in my scientific career," he says. "It was sad for me."

The 14-day rule was developed decades ago to avoid raising too many ethical questions about experimenting on human embryos.

Two weeks is usually the moment when the central nervous system starts to appear in the embryo in a structure known as the "primitive streak."

It's also roughly the stage at which an embryo can no longer split into twins. The idea behind the rule is, that's when an embryo becomes a unique individual.

But the rule was initiated when no one thought it would ever be possible to keep embryos growing in a lab beyond two weeks. Brivanlou thinks it's time to re-think the 14-day rule.

"This is the moment," he says.

Scientists, bioethicists and others are debating the issue in the U.S., Britain and other countries. The rule is law in Britain and other countries and incorporated into widely followed guidelines in the United States.

Insoo Hyun, a bioethicist at Case Western Reserve University, advocates revisiting the rule. It would allow more research to be done on embryos that are destined to be destroyed anyway, he says embryos donated by couples who have finished infertility treatment.

"Given that it has to be destroyed," Hyun says, "some would argue that it's best to get as much information as possible scientifically from it before you destroy it."

But others find it morally repugnant to use human embryos for research at any stage of their development and argue that lifting the 14-day rule would make matters worse.

"Pushing it beyond 14 days only aggravates what is the primary problem, which is using human life in its earliest stages solely for experimental purposes," says Dr. Daniel Sulmasy, a Georgetown University bioethicist.

The idea of extending the 14-day rule even makes some people who support embryo research queasy, especially without first finding another clear stopping point.

Hank Greely, a Stanford University bioethicist, worries that going beyond 14 days could "really draws into question whether we're using humans or things that are well along the path to humans purely as guinea pigs and purely as experimental animals."

Embryo alternative: 'Embryoids'

So as that debate continues, Brivanlou and his colleagues are trying to develop another approach. The scientists are attempting to coax human embryonic stem cells to organize themselves into entities that resemble human embryos. They are also using induced pluripotent stem (iPS) cells, which are cells that behave like embryonic stem cells, but can be made from any cell in the body.

Embryoids like this one are created from stem cells and resemble very primitive human embryos. Scientist hope to use them to learn more about basic human biology and development. Courtesy of Rockefeller University hide caption

Embryoids like this one are created from stem cells and resemble very primitive human embryos. Scientist hope to use them to learn more about basic human biology and development.

Brivanlou's lab has already shown that these "embryo-like structures" or "embryoids" can create the three fundamental cell types in the human body.

But the scientists have only been able to go so far using flat lab dishes. So the researchers are now trying to grow these embryonic-like structures in three dimensions by placing stem cells in a gel.

"Essentially, we're trying to, in a way, to recreate a human embryo in a dish starting from stem cells," says Mijo Simunovic, another of Brivanlou's colleagues.

In early experiments, Simonovic says, he's been able to get stem cells to "spontaneously" form a ball with a "cavity in its center." That's significant because that's what early human embryos do in the uterus.

Simunovic says it's unclear how close these structures could become to human embryos entities that have the capability to develop into babies.

"At the moment, we don't know. That's something that's very hot for us right now to try to understand," Simunovic says.

Simunovic argues the scientists are not "ethically limited to studying these cells and studying these structures" by the 14-day rule.

There's a debate about that, however.

"At what point is your model of an embryo basically an embryo?" asks Hyun, especially when the model seems to have "almost like this inner, budding life."

"Are we creating life that, in the right circumstances, if you were to transfer this to the womb it would continue its journey?" he asks.

Dr. George Daley, the dean of the Harvard Medical School and a leading stem cell researcher, says scientists have been preparing for the day when stem-cell research might raise such questions.

"I think what prospects people are concerned about are the kinds of dystopian worlds that were written about by Aldous Huxley in Brave New World," Daley says. "Where human reproduction is done on a highly mechanized scale in a petri dish."

Daley stresses scientists are nowhere near that, and may never get there. But science moves quickly. So Daley says it's important scientists move carefully with close ethical scrutiny.

The latest guidelines issued by the International Society for Stem Cell Research call for intensive ethical review, Daley notes.

Brivanlou acknowledges that some of his experiments have produced early signs of the primitive streak. But that's a very long way from being able to develop a spinal cord, or flesh and bones, let alone a brain. He dismisses the notion that the research on embryoids would ever lead to scientists creating humans in a lab dish.

"They will not get up start walking around. I can assure you that," he says, noting that full human embryonic development is a highly complex process that requires just the right mix of the biology, physics, geometry and other factors.

Nevertheless, Brivanlou says all of his experiments go through many layers of review. And he's convinced the research should continue.

"It would be a travesty," he says, "to decide that, somehow, ignorance is bliss."

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Embryo Experiments Reveal Earliest Human Development, But Stir Ethical Debate - NPR

ISSCR 2017 – Drug Target Review

event

Date: 14 June 2017 - 17 June 2017

Location: Boston Convention and Exhibition Center 415 Summer Street Boston 02210 United States

Website: ISSCR2017.org

Email: [emailprotected]

Telephone: +1 224-592-5700

The International Society for Stem Cell Research (ISSCR) 2017 annual meeting will be held 14-17 June in Boston, Mass., U.S., at the Boston Convention and Exhibition Center. The meeting brings together 4000 stem cell researchers and clinicians from around the world to share the latest developments in stem cell research and regenerative medicine. In a series of lectures, workshops, poster presentations, and a dynamic exhibition floor, researchers focus on recent findings, technological advances, trends, and innovations that are realizing progress in using stem cells in the discovery and validation of novel treatments.

In 2017, the ISSCR is expanding its translational and clinical programming with two half-day, pre-meeting educational sessions geared toward bringing new therapies to the clinic. The Workshop on Clinical Translation (WCT) and the Clinical Advances in Stem Cell Research (CASC) programs are designed for scientists and physicians interested in learning more about the process of developing stem cell-based therapies and advances in stem cell applications in the clinic.

The Presidential Symposium recognizes a decade of progress in iPS cell research and application with a distinguished lineup of speakers including Shinya Yamanaka, discoverer of iPSCs. Additional plenary presentations include distinguished speakers from around the world focusing on organoids and organogenesis, the making of tissues and organs; stem cells and cancer; chromatin and RNA biology; stress, senescence and aging; tissue regeneration and homeostasis; and the frontiers of cell therapy.

Concurrent sessions feature new and innovative developments across the breadth of the field, and incorporate more than 100 abstract-selected speakers. Disease modelling, tissue engineering, stem cell niches, epigenetics, hematopoietic stem cells, and gene modification and gene editing are just a few of the 28 topic areas presented.

Other meeting offerings include career development sessions and networking opportunities. A full listing of the ISSCR 2017 meeting programming can be found at ISSCR2017.org.

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ISSCR 2017 - Drug Target Review

Findings reveal effect of embryonic neural stem cell development on later nerve regeneration capacity – Medical Xpress

March 1, 2017 Neural progenitor cells (green) in the lateral ganglionic eminence (LGE), the region in the developing brain that produces the majority of adult neural stem cells. Credit: Sven Falk

Neurodegenerative diseases such as Alzheimer's or Parkinson's, but also strokes or other types of traumatic brain damage, result in the death of nerve cells in the brain. Since the mammalian brain is capable of replacing nerve cells only in certain restricted regions, such nerve-cell loss is in most cases permanent. Similarly, the capacity to form new nerve cells in the mature brain is limited to specific areas. The cells responsible for neurogenesis in the mature brain are called adult neural stem cells, but little is known about their developmental origins. Now an international research collaboration led by Magdalena Gtz, Professor of Physiological Genomics at LMU's Biomedical Center and Director of the Institute for Stem Cell Research at the Helmholtz Zentrum Munich, has demonstrated that the mode of division of stem cells has a profound influence on the numbers of adult neural stem cells formed during embryonic development.

The new findings appear in the journal Neuron.

Neural cells develop from progenitors called neural stem cells, which are produced in large numbers during embryonic development. However, in the mature mammalian brain, very few of these progenitors survive as so-called adult neural stem cells capable of generating new nerve cells. In order to determine what enables these cells to retain their stem-cell character into adulthood, Gtz and her colleagues took a closer look at neural stem cells in the developing mouse embryo called radial glia cells (RGCs). RGCs form long processes that span the apicobasal axis of the neuroepithelium and their nuclei come to lie close to the apical surface which faces a fluid-filled cavity known as the ventricle. When RGCs divide, some of the daughter cells again are RGCs, i.e. the RGC self-renews. These cells that retain the self-renewing capacity, a characteristic of stem cells, are the source of the adult neural stem cells found in a specific region of the developing brain called the lateral ganglionic eminence, which forms the lateral wall of the ventricle in the adult brain. The nerve cells derived from the adult neural stem cells subsequently migrate into the olfactory bulb, one of the regions in which new nerve cells are integrated in the mature brain.

"We have now shown that the orientation of the plane of division of embryonic progenitor cells has a major impact on the production of adult neural stem cells," Gtz says. The plane of cleavage during cell division determines which parts of the cytoplasm are inherited by the two daughter cells. Most of the RGCs in the lateral ganglionic eminence were found to divide along a plane that is approximately vertical (at an angle of 60-90) to the apical cell surface. However, when the researchers genetically randomized the orientation of the cleavage plane such that the frequency of oblique or horizontal divisions was increased the number of adult neural stem cells generated was significantly reduced. Hence the orientation of the cleavage plane of RGCs is a crucial factor that affects the number of adult stem cells. However, timing also plays a crucial role. Adult neural stem cells are produced only during a specific, temporally and regionally restricted phase in embryonic development. Genetic randomization of the cleavage plane progenitor cells in the post-natal mouse brain proved to have no effect on the number of adult stem cells.

The total number of adult neural stem cells produced is a crucial determinant of the brain's capacity for repair and regeneration, because each of these cells can generate only a limited number of new nerve cells. "With a better understanding of how the formation of adult neural stem cells is regulated, we could look for ways of ensuring that other embryonic neural stem cells maintain this capacity, and perhaps even persuade other cell types to do so. Our new results represent an important step toward this goal," says Sven Falk, first author of the new study. The researchers hope that their findings will open up new approaches to the therapy of neurodegenerative diseases.

Explore further: Specific roles of adult neural stem cells may be determined before birth

More information: Sven Falk et al. Time-Specific Effects of Spindle Positioning on Embryonic Progenitor Pool Composition and Adult Neural Stem Cell Seeding, Neuron (2017). DOI: 10.1016/j.neuron.2017.02.009

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Findings reveal effect of embryonic neural stem cell development on later nerve regeneration capacity - Medical Xpress

Stem cell treatment changed the life of one guest at Trump’s speech – CNN

She credits an experimental stem cell treatment with giving her new hope for her health and her future -- a newfound hope that also caught attention of Republican Rep. Pete Olson.

"She is the face of the 21st Century Cures Act because of what she's gone through in her life," he said Tuesday.

"It became pretty clear to me that ... I (have) got to tell her story," he said. "That's why she's here: She's awesome."

Immediately after the House vote, Hughes said, Olson called her at home to invite her to be his guest of honor.

"I still cannot believe I will be in the same room as our President and lawmakers," she said before attending Trump's speech.

It took Crowley's father, John, to launch the New Jersey biotechnology company Amicus Therapeutics to identify a drug treatment that would save her life, Trump said.

"If we slash the restraints, not just at the FDA but across our Government, then we will be blessed with far more miracles like Megan," he said. "In fact, our children will grow up in a Nation of miracles."

Hughes spent most of her adolescence hospitalized, as she became so sick that she could barely walk and suffered immense pain. Her body was evaluated, treated and studied at the National Institutes of Health in collaboration with her doctors from the University of Texas Health Science Center at Houston.

Yet relief came in 2014, when Hughes received a high-dose adult stem cell treatment that was not approved in the United States.

For the procedure, Hughes had her own healthy stem cells cultured at the FDA-registered biotechnology company Celltex Therapeutics in Houston. Then she traveled to Cancun, Mexico, to have the cells infused back into her body.

Each infusion involved about 200 million stem cells, and Hughes received some 22 infusions over almost two years. The cells could help normalize her immune system, which was overactive due to her autoimmune disease.

Before the stem cell therapy, Hughes said, she was taking 23 medications a day. Now, she is on eight medications at lower doses.

"If not for the help of high-dose autologous mesenchymal stem cell therapy, I would not be here today," Hughes said at the hearing.

"I was running out of time, but I was willing to put my life at risk to get on an airplane. My quality of life had become so dismal, even one small improvement from my own stem cells would have been enough for me," she said in her speech. "What happened in the days, weeks and years following my first infusion has changed my outlook. It's hard to believe, in my sick body, I had a wealth of healthy adult stem cells with the ability to so significantly improve my quality of life."

"My hope is that our new President will spend time looking at how to help all Americans have access to new therapies like the one I had," she said.

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Stem cell treatment changed the life of one guest at Trump's speech - CNN

Johns Hopkins Medicine, Maryland Stem Cell Research Fund and BioCardia Announce First Patient Treated with … – Business Wire (press release)

SAN CARLOS, Calif. & BALTIMORE--(BUSINESS WIRE)--Johns Hopkins Medicine, the Maryland Stem Cell Research Fund (MSCRF) and BioCardia, Inc. (OTC:BCDA) today announced that the first patient has been treated in the pivotal Phase III CardiAMP clinical trial of a cell-based therapy for the treatment of ischemic heart failure that develops after a heart attack. The first patient was treated at Johns Hopkins Hospital by a team led by Peter Johnston, MD, a faculty member in the Department of Medicine and Division of Cardiology, and principal investigator of the trial at Johns Hopkins.

The investigational CardiAMP therapy is designed to deliver a high dose of a patients own bone marrow cells directly to the point of cardiac dysfunction, potentially stimulating the bodys natural healing mechanism after a heart attack.

The patient experience with CardiAMP therapy begins with a pre-procedural cell potency screening test. If a patient qualifies for therapy, they are scheduled for a bone marrow aspiration. A point of care cell processing platform is then utilized to concentrate the autologous bone marrow cells, which are subsequently delivered in a minimally-invasive procedure directly to the damaged regions in a patients heart.

This cell-based therapy offers great potential for heart failure patients, said Carl Pepine, MD, professor and former chief of cardiovascular medicine at the University of Florida, Gainesville and national co-principal investigator of the CardiAMP trial. We look forward to validating the impact of the therapy on patients quality of life and functional capacity in this important study.

In addition to Dr. Johnston, the CardiAMP research team at Johns Hopkins includes Gary Gerstenblith, MD, Jeffrey Brinker, MD, Ivan Borrello, MD, Judi Willhide, Katherine Laws, Audrey Dudek, Michele Fisher and John Texter, as well as the nurses and technicians of the Johns Hopkins Cardiovascular Interventional Laboratory.

Funding the clinical trial of this cell therapy, which could be the first cardiac cell therapy approved in the United States, is an important step towards treatments, said Dan Gincel, PhD., executive director of the MSCRF at TEDCO. Through our clinical program, we are advancing cures and improving healthcare in the State of Maryland.

The CardiAMP Heart Failure Trial is a phase III, multi-center, randomized, double-blinded, sham-controlled study of up to 260 patients at up to 40 centers nationwide, which includes an optional 10-patient roll-in cohort. The primary endpoint for the trial is a significant improvement in Six Minute Walk distance at 12 months post-treatment. Study subjects must be diagnosed with New York Heart Association (NYHA) Class II or III heart failure as a result of a previous heart attack. The national co-principal investigators are Dr. Pepine and Amish Raval, MD, of the University of Wisconsin.

For information about eligibility or enrollment in the trial, please visit http://www.clinicaltrials.gov or ask your cardiologist.

About BioCardia BioCardia, Inc., headquartered in San Carlos, CA, is developing regenerative biologic therapies to treat cardiovascular disease. CardiAMP and CardiALLO cell therapies are the companys biotherapeutic product candidates in clinical development. For more information, visit http://www.BioCardia.com.

About Johns Hopkins Medicine Johns Hopkins Medicine (JHM), headquartered in Baltimore, Maryland, is one of the leading health care systems in the United States. Johns Hopkins Medicine unites physicians and scientists of the Johns Hopkins University School of Medicine with the organizations, health professionals and facilities of The Johns Hopkins Hospital and Health System. For more information, visit http://www.hopkinsmedicine.org.

About Maryland Stem Cell Research Fund The Maryland Stem Cell Research Act of 2006was established by the Governor and the Maryland General Assembly during the 2006 legislative session and created the Maryland Stem Cell Research Fund. This fund is continued through an appropriation in the Governor's annual budget. The purpose of the Fund is to promote state-funded stem cell research and cures through grants and loans to public and private entities in the State. For more information, visit http://www.MSCRF.org.

Forward Looking Statements This press release contains forward-looking statements as that term is defined under the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, among other things, references to the enrollment of our Phase 3 trial, commercialization and efficacy of our products and therapies, the product development timelines of our competitors. Actual results could differ from those projected in any forward-looking statements due to numerous factors. Such factors include, among others, the inherent uncertainties associated with developing new products or technologies, unexpected expenditures, the ability to raise the additional funding needed to continue to pursue BioCardias business and product development plans, competition in the industry in which BioCardia operates and overall market conditions, and whether the combined funds will support BioCardias operations and enable BioCardia to advance its pivotal Phase 3 CardiAMP cell therapy program. These forward-looking statements are made as of the date of this press release, and BioCardia assumes no obligation to update the forward-looking statements.

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Johns Hopkins Medicine, Maryland Stem Cell Research Fund and BioCardia Announce First Patient Treated with ... - Business Wire (press release)

Cerebral organoids enter the fold – Nature.com

Cerebral organoids enter the fold Nature.com Organoids are stem-cell-derived, three-dimensional cultures that self organize to some extent and include multiple cell types and features of a particular organ. Kriegstein, with Marina Bershteyn, Tomas Nowakowski and their colleagues, generated ...

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Cerebral organoids enter the fold - Nature.com

Canadian Pacific makes a $1 million gift to fund stem cell research at the CHU Sainte-Justine – New hope for … – Canada NewsWire (press release)

From left to right: Dr. Fabrice Brunet, The Honorable Michael M Fortier, Mr. Keith Creel, President and CEO of Canadian Pacific, Dr. Gregor Andelfinger, Ms. Maud Cohen, Ms. Janice Pierson, Mr. Richard Lanoue, Mher Mike Stepanian, Samuel Gauthier, Mariama Hawa Barry, Samy Touati, Tyler Lanoue and Olivier Boissonneault. (CNW Group/CHU Sainte-Justine Foundation)

MONTRAL, Feb. 27, 2017 /CNW Telbec/ -An extraordinary $1 million commitment from Canadian Pacific (CP) towards stem cell research will allow the CHU Sainte-Justine to lead the way in developing new treatments to transform the lives of children suffering from complex congenital heart defects. Currently, there is no treatment available to provide a permanent means of repairing the heart. Today, patients and cardiac experts gathered to recognize the major impact of such strong support for research at the CHU Sainte-Justine, as well as the national importance of research in the development of innovative new stem cell technologies.

Thanks to this exceptional gift, CP is making possible the creation of Quebec's first platform for stem cell research and pediatric regenerative medicine. "These funds will allow us to purchase new equipment and recruit an additional researcher, which will significantly accelerate essential research, namely the identification of the mechanisms that form the heart and the types of intervention that can halt the progression of cardiac illnesses in children," stated Dr.Gregor Andelfinger, pediatric cardiologist at the CHU Sainte-Justine and associate research professor in the Department of Pediatrics at the Universit de Montral. "Our aim is to put in place biological factory, capable of producing cardiac tissues from stem cells," he added.

Research remains the best means of understanding, improving the treatment of, and curing congenital heart defects, which are the most commonly occurring birth defects in the world. They affect one in 80 children in Canada every year, many of whom eventually develop fatal heart failure.

"For over a decade, knowledge and understanding about heart defects have grown considerably at the CHU Sainte-Justine, along with the development of new tools for the genetic analysis of families where several family members suffer from a heart defect. Thanks to its team of experts specializing in pediatrics, cardiology, and congenital malformations, the CHU Sainte-Justine is a leader in providing better diagnoses and better targeted therapies to treat congenital heart defects," stated Mr. Fabrice Brunet, CEO of the CHUM-CHU Sainte-Justine.

Ms. Maud Cohen, CEO of the CHU Sainte-Justine Foundation, expressed gratitude for CP's generous support, which provides the hope of regenerating cardiac tissue in babies affected by congenital heart defects. "I am thrilled that the CHU Sainte-Justine is showing such leadership in pediatric regenerative medicine in Quebec, while also increasing our national and international outreach. The CHU Sainte-Justine Foundation is very proud to have the support of CP as a major donor to the Healing More Better campaign. Not only does this remarkable $1 million gift allow for the development of new cures to help save the lives of thousands of children suffering from cardiovascular diseases, but it will also serve as a driver for future funding. This support will enable Dr. Andelfinger's team to quickly undertake activities that show promising early results," she said.

"Since 2014, through our CP Has Heart program, we have been committed to making communities stronger and healthier thanks to research, treatment and prevention. With today's announcement, we have now donated nearly $10 million to this important cause" said Mr. Keith Creel, CP's President and CEO. "When we learned that the CHU Sainte-Justine was seeking to accelerate stem cell research, an extremely promising avenue for the repair of congenital heart defects, we immediately felt that it was an initiative we wanted to support. We firmly believe that a partnership with such a renowned institution as the CHU Sainte-Justine to create the first pediatric research platform in Quebec will significantly improve upon current treatments. This will ensure that the thousands of babies born with heart defects every year will have a chance to grow up with healthy hearts and live healthy lives," Mr. Creel concluded.

For CP, this generous support for stem cell research is a way to pursue its mission to improve heart health throughout North America, and is a natural fit with a cause so close to the company's heart.

The CHU Sainte-Justine Foundation is grateful for CP's invaluable contribution, which will allow the teams at the CHU Sainte-Justine to continue to heal more children, better.

About the CHU Sainte-Justine FoundationThe CHU Sainte-Justine Foundation's mission is to engage the community and support the CHU Sainte-Justine in its pursuit of excellence and its commitment to providing children and mothers with one of the highest levels of healthcare in the world, now and in the future. fondation-sainte-justine.org/en/

About the CHU Sainte-JustineThe Sainte-Justine university hospital centre (CHU Sainte-Justine) is the largest mother-child centre in Canada and the second largest pediatric hospital in North America. A member of the Universit de Montral extended network of excellence in health (RUIS), Sainte-Justine has 5,664 employees, including 1,578 nurses and nursing assistants; 1,117 other healthcare professionals; 502 physicians, dentists and pharmacists; 822 residents and over 200 researchers; 300 volunteers; and 3,400 interns and students in a wide range of disciplines. Sainte-Justine has 484 beds, including 35 at the Centre de radaptation Marie Enfant (CRME), the only exclusively pediatric rehabilitation centre in Quebec. The World Health Organization has recognized CHU Sainte-Justine as a "health promoting hospital." chusj.org

About Canadian PacificCanadian Pacific (TSX:CP)(NYSE: CP) is a transcontinental railway in Canada and the United States with direct links to eight major ports, including Vancouver and Montreal, providing North American customers a competitive rail service with access to key markets in every corner of the globe. CP is growing with its customers, offering a suite of freight transportation services, logistics solutions and supply chain expertise. Visit cpr.ca to see the rail advantages of CP.

About CP Has HeartAt CP, we know that a railroad may serve as the arteries of a nation, but at its heart is community. That's why, through CP Has Heart, we've already committed nearly $10 million to help improve the heart health of men, women and children across North America. And along the way, we're showing heart whenever we can. Find out more on http://www.cpr.ca or @CPhasHeart.

SOURCE CHU Sainte-Justine Foundation

For further information: CHU Sainte-Justine Foundation, Delphine Brodeur, Director, Communication, public relations and donor relations, 514 345-4931, ext. 4356, dbrodeur@fondationSainteJustine.org

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Canadian Pacific makes a $1 million gift to fund stem cell research at the CHU Sainte-Justine - New hope for ... - Canada NewsWire (press release)

Poorly Jack Christmas, 8, needs 20k to get vital stem cell research – Hull Daily Mail

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A little boy with a rare genetic condition which left him unable to walk or talk could be one of just five people to contribute to vital research.

But Jack Christmas' family need to raise 20,000 to send the eight-year-old to America for the stem cell research in June.

Jack, of Gainford Grove, east Hull, was diagnosed with Mowat Wilson Syndrome in April 2012, and is one of only 171 children across the world to have the condition, caused by a gene deficiency or mutation.

Jack's mum, Dawn, said while the little boy is totally dependent on adults, he is making good progress.

"Jack doesn't talk, has to have all food pureed, can't feed or dress himself, is doubly incontinent, in fact he is totally dependent on adults," she said.

"All this said and done and he can now crawl, clap his hands and waves hello and goodbye as well as signing money and bird. Jack can also drink out of a feeder cup and on Christmas Eve 2015 Jack took his first unsteady independent steps."

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As well as Mowat Wilson Syndrome, the Tweendykes School pupil has also been diagnosed with Global Development Delays, Gross Motor Disorder and Brain Atrophy. He also suffers from life-threatening seizures.

But now his family hope Jack can help doctors work towards finding a treatment for the condition by taking part in the research.

Dad Tony said: "For Jack, the gene that's affected is mutated, it is there, so there might be something doctors can do to manipulate it is.

"They might not be able to do anything and even if they can it might not be for 10 years, but Jack taking part in the research might help them find the answers.

"It also means if they do manage to find something, he will be able to have the treatment."

Tony said even if doctors were able to find a treatment, it probably wouldn't completely cure Jack's condition. He said: "He will always have Mowat Wilson Syndrome.

"But they might be able to find ways to make it easier for him."

The family have already raised 8,000 to fund the costs of travelling to the USA, but still need to raise 12,000 to foot the bill. And as part of their fundraising, they are hosting a fundraiser for the Life For A Kid Foundation a Hull charity supporting the family.

Dawn said the evening at Wawne Ferry pub, in Bransholme, would be a "night of singing, dancing, bingo, yes BINGO and a raffle."

Tickets for the 'Get ya dabbers ready' event, from 7.30pm on March 11, cost 5. You can also donate to Jack's cause on the family's Virgin Money Giving page.

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Poorly Jack Christmas, 8, needs 20k to get vital stem cell research - Hull Daily Mail