Category Archives: Stem Cells

A virus infection Stephanie Connor acquired during pregnancy put her unborn daughter at significant risk for brain damage and lifelong hearing loss.

"It was traumatic," said Connor, of LaBelle, Fl, after learning about her daughter's condition. "It was like mourning the loss of a child."

At age 1, baby Madeleine was completely deaf in her right ear and her hearing was severely lost in the left, said Connor. While a hearing aid helped to amplify some sounds for Madeleine, it would never fully repair the damage in her ear.

But a simple experimental procedure that Connor enrolled in for Madeleine may have restored her hearing and reversed her condition.

In January 2012, Madeleine, 2, became the first child to undergo an experimental hearing loss treatment through an FDA-approved trial at Memorial Hermann-Texas Medical Center that infused stem cells from her own banked cord blood into her damaged inner ear.

Within the last six months, Connor says she's seen a dramatic improvement in Madeleine's ability to hear.

"Before, when she would hear something she would look all around," Connor said. "But now we notice that she turns in the right direction of the sound."

Madeleine was also able to speak for the first time, Connor said.

For more than two decades, umbilical cord blood transplantation -- either by a baby's own cord blood or another's, depending on the type of procedure -- has been used to treat otherwise fatal diseases including blood disorders, immune diseases, and some types of cancers.

Courtesy Stephanie Connor

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Stem Cells Restore Toddler's Hearing

Any study that claims to overturn long-held dogmas is going to run headlong into controversy. Take, for example, a stream of recent papers which apparently showed that adult ovaries contain stem cells capable of producing eggs.

If thats true, its a really big deal. For decades, the textbooks have said that women are born with their lifetime supply of eggs, which slowly dwindle away and are never replaced. If adult ovaries do indeed have stem cells that can regenerate new eggs, that has big implications for fertility treatments, and when a woman could potentially have children.

But wait! A new study, which used a different technique to isolate these ovarian stem cells in mice, found that they dont divide, and never produce actual eggs. Maybe the textbooks are fine as they are?

But WAIT! This study, far more than many of the others I cover, has divided opinion. Obviously, the authors think that it deals a critical blow to the idea of egg-making adult stem cells. And obviously, one of the people behind the stem cell discovery thinks that the new experiments arent very good. I also contacted four other scientists who work on ovarian biology and their views differ considerably.

Ive written about this story for The Scientist so head over there for the details, and the grounds for the disagreement.

Image from RWJMS IVF Laboratory

Excerpt from:
New paper stirs more debate about whether adult ovaries can make new eggs | Not Exactly Rocket Science

Featured Article Academic Journal Main Category: Stem Cell Research Also Included In: Pregnancy / Obstetrics;Pediatrics / Children's Health;Biology / Biochemistry Article Date: 09 Jul 2012 - 9:00 PDT

Current ratings for: Stem Cells From Amniotic Fluid

Scientists from Imperial College London, and University College London Institute of Child Health, and colleagues, said their discovery means it may be possible to store stem cells from donated amniotic fluid for clinical and research use, offering a much needed alternative to the limited supply of embryonic stem cells.

"These cells have a wide range of potential applications in treatments and in research. We are particularly interested in exploring their use in genetic diseases diagnosed early in life or other diseases such as cerebral palsy," said co-senior author Dr Pascale Guillot, from the Department of Surgery and Cancer at Imperial.

Stem cells hold promise for regenerative medicine because they have the potential to become virtually any cell in the body. The current "gold standard" of human stem cells is the human embryonic stem cell (hESC), which is harvested from human embryos.

However, researchers and clinicians are keen to find alternatives to hESCs because of ethical concerns about using human embryos and also because of their limited availability.

Previous studies have shown it is possible to use other types of cell and, by introducing extra genes, often using viruses as carriers, make them almost as versatile or pluripotent as hESCs. For instance, scientists have reprogrammed human skin cells to behave like embryonic stem cells.

But this way of making induced pluripotent stem cells (iPSCs) is not efficient and there is also a risk that the DNA disruption that occurs (something the authors attribute to "random integration of the reprogramming transgenes into the host genome") will lead to tumors.

This new study is the first to make iPSCs without having to insert foreign genetic material into the cells.

Guillot and colleagues also found the iPSCs they made from amniotic fluid stem cells (AFSCs) showed some of the characteristics normally only seen in embryonic stem cells, that are not present in iPSCs made from adult cells.

See the article here:
Stem Cells From Amniotic Fluid

ScienceDaily (July 9, 2012) Scientists showed in mice that disabling a gene linked to a common pediatric tumor disorder, neurofibromatosis type 1 (NF1), made stem cells from one part of the brain proliferate rapidly. But the same genetic deficit had no effect on stem cells from another brain region.

The results can be explained by differences in the way stem cells from these regions of the brain respond to cancer-causing genetic changes.

NF1 is among the world's most common genetic disorders, occurring in about one of every 3,000 births. It causes a wide range of symptoms, including brain tumors, learning disabilities and attention deficits.

Brain tumors in children with NF1 typically arise in the optic nerve and do not necessarily require treatment. If optic gliomas keep growing, though, they can threaten the child's vision. By learning more about the many factors that contribute to NF1 tumor formation, scientists hope to develop more effective treatments.

"To improve therapy, we need to develop better ways to identify and group tumors based not just on the way they look under the microscope, but also on innate properties of their stem cell progenitors," says David H. Gutmann, MD, PhD, the Donald O. Schnuck Family Professor of Neurology.

The study appears July 9 in Cancer Cell. Gutmann also is the director of the Washington University Neurofibromatosis Center.

In the new study, researchers compared brain stem cells from two primary sources: the third ventricle, located in the midbrain, and the nearby lateral ventricles. Before birth and for a time afterward, both of these areas in the brain are lined with growing stem cells.

First author Da Yong Lee, PhD, a postdoctoral research associate, showed that the cells lining both ventricles are true stem cells capable of becoming nerve and support cells (glia) in the brain. Next, she conducted a detailed analysis of gene expression in both stem cell types.

"There are night-and-day differences between these two groups of stem cells," Gutmann says. "These results show that stem cells are not the same everywhere in the brain, which has real consequences for human neurologic disease."

The third ventricle is close to the optic chiasm, the point where the optic nerves cross and optic gliomas develop in NF1 patients. Lee and Gutmann postulated that stem cells from this ventricle might be the source of progenitor cells that can become gliomas in children with NF1.

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Pediatric brain tumors traced to brain stem cells

Opinion is divided on a new paper showing that adult ovaries do not contain egg-making stem cells, contrary to the findings of two recent studies that themselves appeared to overturn longstanding dogma.

For more than half a century, textbooks have stated that women and other female mammals are born with all the eggs, or oocytes, they will ever have. This supply gradually shrinks with age, and ovaries are incapable of producing more of these reproductive cells.

This dogma has taken a pounding in recent years, however. Starting in 2004, Ji Wu of Shanghai Jiao Tong University in China and Jonathan Tilly of Massachusetts General Hospital isolated stem cells from the ovaries of mice, which could apparently divide to produce fresh oocytes. And earlier this year, Tilly announced that he has found cells with the same qualities, known as oogonial stem cells (OSCs), in the ovaries of middle-aged women.

These discoveries promised to offer new treatments for fertility, allowing women to have babies without worrying about an ageing supply of eggs. But as with all dogma-contradicting discoveries, they remained contentious.

Now, a new study from researchers at the University of Gothenburg is likely to fuel the controversy. Kui Liu and his colleagues used fluorescing proteins to identify the alleged egg-producing stem cells in the ovaries of mice, and found that the cells do not divide into oocytes. They published their results today (July 9) in the Proceedings of the National Academy of Sciences.

The reaction to Lius study is strongly divided. Patricia Hunt, a reproductive biologist from Washington State University, described it as solid and informative. For those of us who have remained skeptical about the existence or role of stem cells in the ovary, this approach is a most welcome addition to the field, she said.

I took a close look at this and the work is fantastic, agreed David Albertini, a physiologist from the University of Kansas. It should put previous discussions into perspective. At least in mice, OSCs do not exist.

But Evelyn Telfer, a cell biologist from the University of Edinburgh, is less impressed, and said that Liu missed the opportunity to obtain robust experimental evidence. Because Liu used completely new methods, it is unclear how the cells he identified compare with those from previous studies. We are left with more questions than answers, said Telfer.

When Liu first saw the papers by Wu and Tilly, he was more excited than sceptical. My first impulse was: I want to repeat this, he said. But he was troubled by the fact that both Wu and Tillys teams fished for their cells using antibodies that recognize DDX4, a protein found in reproductive stem cells. But DDX4 is not a surface protein, Liu said, and is mainly found inside cells. The fishing technique should not have worked.

To avoid this problem, Lius members Hua Zhang and Wenjing Zheng worked with rainbow mice, whose reproductive cells glow green under normal conditions, but change to red, orange, or blue if they switch on the Ddx4 gene. Zhang and Zheng identified several such cells and watched them for 72 hours. They never once divided or produced oocytes. The duo did find some cells that looked like oocytes, but these did not express Ddx4.

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Ovarian Stem Cell Debate

Study finds no evidence of stem cells in mouse ovaries

Web edition : 3:53 pm

Women may indeed be limited to the number of eggs their ovaries contain at birth, a new study finds, directly contradicting recent research that suggests otherwise.

Scientists have long thought that female mammals, including humans, are born with all the egg cells they will ever have. But a few papers, culminating with a study published earlier this year in Nature Medicine, have suggested that ovaries contain rare stem cells that can replenish egg supplies (SN: 4/7/12, p. 8). These egg-producing stem cells could lead to new treatments for fertility problems, ways to delay menopause and advancements in the basic understanding of human egg cells.

Not so fast, says Kui Liu, a molecular reproductive biologist at the University of Gothenburg in Sweden. Working with mice, Liu and his colleagues used a technique to identify egg cells and their precursors in ovaries. The team found no evidence of the stem cells in ovaries that reproductive biologist Jonathan Tilly of Massachusetts General Hospital and colleagues recently described in Nature Medicine.

To look for egg-making stem cells in mouse ovaries, Liu and his colleagues genetically engineered mice so that every cell glows green with a fluorescent protein, except for eggs, sperm and cells destined to become gametes. Those cells glow yellow, blue or red. The scientists found cells glowing red purported gamete precursors in the ovary, but those cells did not divide the way stem cells would and did not produce new eggs, leading the researchers to conclude that stem cells dont exist in the ovary. The researchers report their work online July 9 in the Proceedings of the National Academy of Sciences.

The new finding in no way disproves the existence of egg-making stem cells in the ovary, says Evelyn Telfer, a reproductive biologist at the University of Edinburgh. For one thing, these cells are absolutely not the same as the ones Tillys got, she says. The cells Liu found are much larger and are probably already nondividing egg cells.

Liu says his point in publishing the paper is not to say that Tilly is wrong, but to urge scientists to take a hard look at the evidence for and against stem cells in the ovary.

The rest is here:
Egg production after birth questioned

CAMBRIDGE, Mass.--(BUSINESS WIRE)--

Verastem, Inc., (VSTM) a biopharmaceutical company focused on discovering and developing drugs to treat breast and other cancers by targeting cancer stem cells, announced that the Company will present at the JMP Securities Healthcare Conference 2012 on July 13, 2012, in New York City.

The Verastem presentation will be at 9:00am ET on Friday, July 13, 2012, in the Tribeca Room at the Peninsula Hotel in New York, NY.

A webcast of the presentation can be accessed by visiting the investors section of the Companys website at http://www.verastem.com. A replay of the webcast will be archived on the Verastem website for two weeks following the presentation date.

About Verastem, Inc.

Verastem, Inc. (VSTM) is a biopharmaceutical company focused on discovering and developing drugs to treat breast and other cancers by targeting cancer stem cells. Cancer stem cells are an underlying cause of tumor recurrence and metastasis. For more information please visit http://www.verastem.com.

Forward-looking statements:

Any statements in this press release about future expectations, plans and prospects for the Company constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements. The Company anticipates that subsequent events and developments will cause the Companys views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

Link:
Verastem to Present at the JMP Securities Healthcare Conference 2012

LOS ANGELES--(BUSINESS WIRE)--Capricor, Inc., a privately held biotechnology company focused on regenerative medicine, today announced that the U.S. Food and Drug Administration has approved initiation of its Investigational New Drug (IND) application for the ALLSTAR study, which will use allogeneic cardiac-derived stem cells (CDCs) to treat patients following large myocardial infarctions (MI).

IND approval for ALLSTAR is another major milestone for Capricor as we continue to develop cardiac-derived stem cells for the treatment of heart disease

ALLSTAR will study the use of CAP-1002 delivered directly into a coronary artery from thirty days to one year following a heart attack. The trial will have a 14 patient lead in phase and is planned as a 260 patient, twenty center randomized controlled trial. ALLSTAR will study a variety of safety and effectiveness endpoints with the goal of demonstrating sufficiently strong data to permit an eventual Phase III trial as a path to commercialization of CAP-1002.

ALLSTAR is predicated on the positive results of the landmark CADUCEUS trial that showed approximately 50 percent reduction of scar size and 50 percent more viable muscle in the infarction zones of patients studied one year after a heart attack. ALLSTAR will use donor cells whereas CADUCEUS used each patients own CDCs. The shift from autologous to allogeneic cells is supported by extensive pre-clinical evidence of safety and effectiveness and is expected to expand the market opportunity as well as to reduce the costs for treatment.

"IND approval for ALLSTAR is another major milestone for Capricor as we continue to develop cardiac-derived stem cells for the treatment of heart disease," said Linda Marbn, Ph.D., CEO of Capricor. "There are greater than 6 million people in the US living with heart failure, and that number continues to rise as heart disease remains the number one killer. Capricors CDCs represent a novel treatment to repair the heart after muscle loss following large heart attacks through the regeneration of heart muscle and the shrinking of scar tissue. Our ultimate goal will be to demonstrate that muscle regeneration in these patients will result in clinically meaningful improvements to their lives."

"This is terrific news, says Ellen Feigal, M.D., Senior Vice President for Research and Development at Californias stem cell agency, CIRM. This is the first time a Disease Team funded by CIRM has been given an Investigational New Drug (IND) approval from the FDA, a critical step in testing promising therapies in patients. Its a reflection of the progress being made in turning promising therapies into real-world treatments.

Capricor has asked CIRM to assist in the funding of a portion of ALLSTAR. Capricor was founded Baltimore and moved to California almost five years ago in part because of the environment that CIRM has created to foster stem cell research in this state. We are grateful to have received the seed support from CIRM that has funded a portion of our research. Our mission is to develop meaningful treatments for patients suffering from heart disease and to grow Capricor into a major California biotechnology company, said Linda Marbn.

About CAP-1002

CAP-1002, Capricor's lead candidate, is a proprietary allogeneic adult stem cell product for the treatment of myocardial infarction. The product contains multiple progenitor cells and is derived from donor heart tissue. The cells are multiplied in the laboratory using a specialized process, and then introduced directly into a patients heart via infusion in a coronary artery at the time of standard cardiac catherization.

About Capricor, Inc.

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Capricor Announces FDA Approval To Initiate ALLSTAR Trial of Allogeneic Stem Cell Therapy In Patients Following Heart ...

Washington, July 6 : In a new study, researchers including one of an Indian origin have described how human epidermal progenitor cells and stem cells control transcription factors to avoid premature differentiation, preserving their ability to produce new skin cells throughout life.

The findings provide new insights into the role and importance of exosomes and their targeted gene transcripts, and may help point the way to new drugs or therapies for not just skin diseases, but other disorders in which stem and progenitor cell populations are affected.

Stem cells, of course, are specialized cells capable of endlessly replicating to become any type of cell needed, a process known as differentiation. Progenitor cells are more limited, typically differentiating into a specific type of cell and able to divide only a fixed number of times.

Throughout life, human skin self-renews. Progenitor and stem cells deep in the epidermis constantly produce new skin cells called keratinocytes that gradually rise to the surface where they will be sloughed off. One of the ways that stem and progenitor cells maintain internal health during their lives is through the exosome - a collection of approximately 11 proteins responsible for degrading and recycling different RNA elements, such as messenger RNA that wear out or that contain errors resulting in the translation of dysfunctional proteins which could potentially be deleterious to the cell.

"In short," said George L. Sen, PhD, assistant professor of medicine and cellular and molecular medicine at the University of California, San Diego School of Medicine, "the exosome functions as a surveillance system in cells to regulate the normal turnover of RNAs as well as to destroy RNAs with errors in them."

Sen and colleagues Devendra S. Mistry, PhD, a postdoctoral research fellow, and staff scientist Yifang Chen, MD, PhD, discovered that in the epidermis the exosome functions to target and destroy mRNAs that encode for transcription factors that induce differentiation.

Specifically, they found that the exosome degrades a transcription factor called GRHL3 in epidermal progenitor cells, keeping the latter undifferentiated. Upon receiving differentiation inducing signals, the progenitor cells lose expression of certain subunits of the exosome which leads to higher levels of GRHL3 protein. This increase in GRHL3 levels promotes the differentiation of the progenitor cells.

"Without a functioning exosome in progenitor cells," said Sen, "the progenitor cells prematurely differentiate due to increased levels of GRHL3 resulting in loss of epidermal tissue over time."

Sen said the findings could have particular relevance if future research determines that mutations in exosome genes are linked to skin disorders or other diseases.

"Recently there was a study showing that recessive mutations in a subunit of the exosome complex can lead to pontocerebellar hypoplasia, a rare neurological disorder characterized by impaired development or atrophy of parts of the brain," said Sen.

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Key proteins to aid self-renewing skin identified

by JANET ST. JAMES

WFAA

Posted on July 3, 2012 at 5:15 PM

Updated today at 5:24 PM

DALLAS Six weeks ago, 64-year-old Larry Rayes could only walk a few steps without resting.

"I could only walk about 20 yards and I got very lightheaded, very dizzy," Rayes said.

Rayes suffers from congestive heart failure, a condition caused by a heart so weak it can't effectively pump blood through his body.

He needs a heart transplant, but until he gets one, Rayes is part of an experiment to see if his heart can heal itself.

In May, at the same time doctors implanted a heart pump in Rayes' chest, his weakened heart also received a series of 20 stem cell injections.

"We're using these stem cells to regenerate the muscle of the heart, so that their hearts can beat more effectively," explained cardiothoracic surgeon Dr. Todd Dewey.

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North Texas man among first to try stem cells for heart failure