Category Archives: Diseases

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In collaboration with Fibrocell Science, Inc., (OTCBB:FCSC.OB), researchers at the University of California, Los Angeles (UCLA) have identified two rare adult stem cell-like subpopulations in adult human skin, a discovery that may yield further ground-breaking research in the field of personalized medicine for a broad range of diseases. Using technology developed by Fibrocell Science, Inc. the researchers were able to confirm the existence of these two types of cells in human skin cell cultures, potentially providing a source of stem cell-like subpopulations from skin biopsies, which are quicker to perform, relatively painless and less invasive than bone marrow and adipose tissue extractions, which are the current methods for deriving adult stem cells for patient-specific cellular therapies.

The findings, which are reported in the inaugural issue of BioResearch Open Access, pertain to two subtypes of cells: SSEA3-expressing regeneration-associated (SERA) cells, which may play a role in the regeneration of human skin in response to injury and mesenchymal adult stem cells (MSCs), which are under investigation (by many independent researchers) for their ability to differentiate into the three main types of cells: osteoblasts (bone cells), chondrocytes (cartilage cells) and adipocytes (fat cells). Finding these specialized cells within the skin cell cultures is important because rather than undergoing a surgical organ or tissue transplantation to replace diseased or destroyed tissue, patients may one day be able to benefit from procedures by which stem cells are extracted from their skin, reprogrammed to differentiate into specific cell types and reimplanted into their bodies to exert a therapeutic effect. Research in this area is ongoing.

Finding these rare adult stem cell-like subpopulations in human skin is an exciting discovery and provides the first step towards purifying and expanding these cells to clinically relevant numbers for application to a variety of potential personalized cellular therapies for osteoarthritis, bone loss, injury and/or damage to human skin as well as many other diseases, said James A. Byrne, Ph.D., the studys lead author and Assistant Professor of Molecular and Medical Pharmacology at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. In addition to pursuing our own research investigations with Fibrocell Science using this method, we envision a time not too far in the future when we will be able to isolate and produce mesenchymal stem cells and SERA cells on demand from skin samples, which may allow other researchers in need of specialized cells to pursue their own lines of medical and scientific research.

We congratulate the UCLA researchers on the publication of their breakthrough data, which may ultimately lead to new patient-specific, personalized cellular therapies to treat various diseases, said David Pernock, Chairman and CEO of Fibrocell Science, Inc. Fibrocell Science is proud of our role in helping to establish the potential of dermal skin cells for the future of personalized, regenerative medicine. We look forward to continuing our relationship with UCLA and Dr. Byrnes team to advance this research.

Discovering Viable, Regenerative Cells in the Skin

Dr. Byrne and colleagues confirmed previous research identifying a rare population of cells in adult human skin that has a marker called the stage-specific embryonic antigen 3 (SSEA3). Dr. Byrne observed that there was a significant increase in the number of SSEA3 expressing cells following injury to human skin, supporting the hypothesis that the SSEA3 biomarker can be used to facilitate the identification and isolation of these cells with tissue-regenerative properties.

Using Fibrocells proprietary technology, the researchers collected cells from small skin samples, cultured the cells in the lab, and purified them via a technique known as fluorescence-activated cell sorting (FACS). Under FACS, cells in suspension were tagged with fluorescent markers specific for undifferentiated stem cells. This method allowed the researchers to separate the rare cell subpopulations from other types of cells.

Dr. Byrne and colleagues also observed a rare subpopulation of functional MSCs in human skin that existed in addition to the SERA cells.

Being able to identify two sub-populations of rare, viable and functional cells that behave like stem cells from within the skin is an important finding because both cell types have the potential to be investigated for diverse clinical applications, said Dr. Byrne.

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Fibrocell Science Technology Leads to Discovery of Two Rare Adult Stem Cell-Like Subpopulations in Human Skin

Newswise ANN ARBOR, Mich. The University of Michigans second human embryonic stem cell line has just been placed on the U.S. National Institutes of Healths registry, making the cells available for federally-funded research. It is the second of the stem cell lines derived at U-M to be placed on the registry.

The line, known as UM11-1PGD, was derived from a cluster of about 30 cells removed from a donated five-day-old embryo roughly the size of the period at the end of this sentence. That embryo was created for reproductive purposes, tested and found to be affected with a genetic disorder, deemed not suitable for implantation, and would therefore have otherwise been discarded when it was donated in 2011.

It carries the gene defect responsible for Charcot-Marie-Tooth disease, a hereditary neurological disorder characterized by a slowly progressive degeneration of the muscles in the foot, lower leg and hand. CMT, as it is known, is one of the most common inherited neurological disorders, affecting one in 2,500 people in the United States. People with CMT usually begin to experience symptoms in adolescence or early adulthood.

The embryo used to create the cell line was never frozen, but rather was transported from another IVF laboratory in the state of Michigan to the U-M in a special container. This may mean that these stem cells will have unique characteristics and utilities in understanding CMT disease progression or screening therapies in comparison to other human embryonic stem cells.

We are proud to provide this cell line to the scientific community, in hopes that it may aid the search for new treatments and even a cure for CMT, says Gary Smith, Ph.D., who derived the line and also is co-director of the U-M Consortium for Stem Cell Therapies, part of the A. Alfred Taubman Medical Research Institute. Once again, the acceptance of these cells to the registry demonstrates our attention to details of proper oversight, consenting, and following of NIH guidelines.

U-M is one of only four institutions including two other universities and one private company to have disease-specific stem cell lines listed in the national registry. U-M has several other disease-specific hESC lines submitted to NIH and awaiting approval, says Smith, who is a professor in the Department of Obstetrics and Gynecology at the University of Michigan Medical School. The first line, a genetically normal one, was accepted to the registry in February.

Stem cell lines that carry genetic traits linked to specific diseases are a model system to investigate what causes these diseases and come up with treatments, says Sue OShea, Ph.D., professor of Cell and Developmental Biology at the U-M Medical School, and co-director of the Consortium for Stem Cell Therapies.

Each line is the culmination of years of preparation and cooperation between U-M and Genesis Genetics, a Michigan-based genetic diagnostic company. This work was made possible by Michigan voters' November 2008 approval of a state constitutional amendment permitting scientists to derive embryonic stem cell lines using surplus embryos from fertility clinics or embryos with genetic abnormalities and not suitable for implantation.

The amendment also made possible an unusual collaboration that has blossomed between the University of Michigan and molecular research scientists at Genesis Genetics, a company that has grown in only eight years to become the leading global provider of pre-implantation genetic diagnosis (PGD) testing. PGD is a testing method used to identify days-old embryos carrying the genetic mutations responsible for serious inherited diseases. During a PGD test, a single cell is removed from an eight-celled embryo. The other seven cells continue to multiply and on the fifth day form a cluster of roughly 100 cells known as a blastocyst.

Genesis Genetics performs nearly 7,500 PGD tests annually. Under the arrangement between the company and U-M, patients with embryos that test positive for a genetic disease now have the option of donating those embryos to U-M if they have decided not to use them for reproductive purposes and the embryos would otherwise be discarded.

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Second U-M Stem Cell Line Now Publicly Available to Help Researchers Find Treatments for Nerve Condition

Public release date: 26-Apr-2012 [ | E-mail | Share ]

Contact: Beth Dunham bethdunh@usc.edu 213-740-4279 University of Southern California

A new study, appearing in Cell Stem Cell and led by researchers at the University of Southern California, outlines the specifics of how autoimmune disorders can be controlled by infusions of mesenchymal stem cells.

Mesenchymal stem cells (MSC) are highly versatile stem cells that originate from the mesoderm, or middle layer of tissue, in a developing embryo. MSC can be isolated from many different kinds of human tissue, including bone marrow and the umbilical cord.

Principal investigator Songtao Shi, professor at the Ostrow School of Dentistry of USC Center for Craniofacial Molecular Biology, said that recent studies have shown the benefits of administering MSC to patients with immune-related disorders such as graft versus host disease, systemic lupus erythematosus, rheumatoid arthritis, and more.

These studies showed that infusions of MSC appeared to quell the production and function of overactive immune cells, including T- and B-lymphocytes. However, the specific mechanism behind how MSC get the immune cells under control hasn't been fully understood.

"Mesenchymal-Stem-Cell-Induced Immunoregulation Involves FAS-Ligand-/FAS-Mediated T Cell Apoptosis" shines light on how infused MSCs target and defeat overactive immune cells. Examining the effects of MSC infusion in mice with systemic sclerosis (SS)-like immune disorders, Shi and his colleagues discovered that a specific cellular mechanism known as the FAS/FAS-ligand pathway was the key to the remarkable immune system benefits.

Specifically, in mice with SS-like disorders, infusions of MSC caused T-lymphocyte death with FASL/FAS signaling and lessened symptoms of the immune disorder. However, MSC deficient in FAS-ligand failed to treat immune disorders in SS-afflicted mice.

With the hopeful results of the animal model study in mind, Shi's colleagues in China performed a pilot study with patients suffering from systemic sclerosis. Infusions of MSCs provided similar clinical benefits to patients, and experimental analysis revealed that the FASL/FAS pathway was also at work in humans with SS.

The identification of the cellular workings responsible for the stem cell treatments' success may eventually help doctors find optimal cell-based treatment for some immune diseases, Shi said.

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How stem cell therapy can keep the immune system under control

Public release date: 26-Apr-2012 [ | E-mail | Share ]

Contact: Professor Palle Serup palle.serup@sund.ku.dk 01-145-402-20026 University of Copenhagen

Scientists from The Danish Stem Cell Center (DanStem) at the University of Copenhagen and Hagedorn Research Institute have gained new insight into the signaling paths that control the body's insulin production. This is important knowledge with respect to their final goal: the conversion of stem cells into insulin-producing beta cells that can be implanted into patients who need them. The research results have just been published in the well-respected journal PNAS.

Insulin is a hormone produced by beta cells in the pancreas. If these beta cells are defective, the body develops diabetes. Insulin is vital to life and therefore today the people who cannot produce their own in sufficient quantities, or at all, receive carefully measured doses often via several daily injections. Scientists hope that in the not-so-distant future it will be possible to treat diabetes more effectively and prevent secondary diseases such as cardiac disease, blindness and nerve and kidney complications by offering diabetes patients implants of new, well-functioning, stem-cell-based beta cells.

"In order to get stem cells to develop into insulin-producing beta cells, it is necessary to know what signaling mechanisms normally control the creation of beta cells during fetal development. This is what our new research results can contribute," explains Professor Palle Serup from DanStem.

"When we know the signaling paths, we can copy them in test tubes and thus in time convert stem cells to beta cells," says Professor Serup.

The new research results were obtained in a cooperative effort between DanStem, the Danish Hagedorn Research Institute and international partners in Japan, Germany, Korea and the USA. The scientific paper has just been published in the well-respected international journal PNAS (Proceedings of the National Academy of Sciences of the United States of America) entitled Mind bomb 1 is required for pancreatic -cell formation.

Better control of stem cells

The signaling mechanism that controls the first steps of the development from stem cells to beta cells has long been known.

"Our research contributes knowledge about the next step in development and the signaling involved in the communication between cells an area that has not been extensively described. This new knowledge about the ability of the so-called Notch signaling first to inhibit and then to stimulate the creation of hormone-producing cells is crucially important to being able to control stem cells better when working with them in test tubes," explains Professor Palle Serup .

Excerpt from:
Stem cell researchers map new knowledge about insulin production

ANN ARBOR The University of Michigans second human embryonic stem cell line has just been placed on the U.S. National Institutes of Healths registry, making the cells available for federally funded research. It is the second of the stem cell lines derived at UM to be placed on the registry.

The line, known as UM11-1PGD, was derived from a cluster of about 30 cells removed from a donated five-day-old embryo roughly the size of the period at the end of this sentence. That embryo was created for reproductive purposes, tested and found to be affected with a genetic disorder, deemed not suitable for implantation, and would therefore have otherwise been discarded when it was donated in 2011.

It carries the gene defect responsible for Charcot-Marie-Tooth disease, a hereditary neurological disorder characterized by a slowly progressive degeneration of the muscles in the foot, lower leg and hand. CMT, as it is known, is one of the most common inherited neurological disorders, affecting one in 2,500 people in the United States. People with CMT usually begin to experience symptoms in adolescence or early adulthood.

The embryo used to create the cell line was never frozen, but rather was transported from another IVF laboratory in the state of Michigan to the UM in a special container. This may mean that these stem cells will have unique characteristics and utilities in understanding CMT disease progression or screening therapies in comparison to other human embryonic stem cells.

We are proud to provide this cell line to the scientific community, in hopes that it may aid the search for new treatments and even a cure for CMT, says Gary Smith, Ph.D., who derived the line and also is co-director of the UM Consortium for Stem Cell Therapies, part of the A. Alfred Taubman Medical Research Institute. Once again, the acceptance of these cells to the registry demonstrates our attention to details of proper oversight, consenting, and following of NIH guidelines.

UM is one of only four institutions including two other universities and one private company to have disease-specific stem cell lines listed in the national registry. UM has several other disease-specific hESClines submitted to NIH and awaiting approval, says Smith, who is a professor in the Department of Obstetrics and Gynecology at the University of Michigan Medical School. The first line, a genetically normal one, was accepted to the registry in February.

Stem cell lines that carry genetic traits linked to specific diseases are a model system to investigate what causes these diseases and come up with treatments, says Sue OShea, professor of cell and developmental biology at the UM Medical School, and co-director of the Consortium for Stem Cell Therapies.

Each line is the culmination of years of preparation and cooperation between UM and Genesis Genetics, a Michigan-based genetic diagnostic company. This work was made possible by Michigan voters November 2008 approval of a state constitutional amendment permitting scientists to derive embryonic stem cell lines using surplus embryos from fertility clinics or embryos with genetic abnormalities and not suitable for implantation.

The amendment also made possible an unusual collaboration that has blossomed between the University of Michigan and molecular research scientists at Genesis Genetics, a company that has grown in only eight years to become the leading global provider of pre-implantation genetic diagnosis (PGD) testing. PGDis a testing method used to identify days-old embryos carrying the genetic mutations responsible for serious inherited diseases. During a PGD test, a single cell is removed from an eight-celled embryo. The other seven cells continue to multiply and on the fifth day form a cluster of roughly 100 cells known as a blastocyst.

Genesis Genetics performs nearly 7,500 PGD tests annually. Under the arrangement between the company and UM, patients with embryos that test positive for a genetic disease now have the option of donating those embryos to UM if they have decided not to use them for reproductive purposes and the embryos would otherwise be discarded.

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Second UM Stem Cell Line Now Available To Help Cure Nerve Condition

Washington, April 24 : Scientists have discovered a new stem cell in the adult brain and they hope to put the discovery to use to develop methods that can repair diseases and injury to the brain.

These cells can proliferate and form several different cell types - most importantly, they can form new brain cells.

Analysing brain tissue from biopsies, the researchers at the Lund University for the first time found stem cells located around small blood vessels in the brain.

The cell's specific function is still unclear, but its plastic properties suggest great potential. A similar cell type has been identified in several other organs where it can promote regeneration of muscle, bone, cartilage and adipose tissue.

In other organs, researchers have shown clear evidence that these types of cells contribute to repair and wound healing.

Scientists suggested that the curative properties might also apply to the brain. The next step is to try to control and enhance stem cell self-healing properties with the aim of carrying out therapies targeted to a specific area of the brain.

"Our findings show that the cell capacity is much larger than we originally thought, and that these cells are very versatile," said Gesine Paul-Visse, Ph.D., Associate Professor of Neuroscience at Lund University.

"Most interesting is their ability to form neuronal cells, but they can also be developed for other cell types. The results contribute to better understanding of how brain cell plasticity works and opens up new opportunities to exploit these very features," Paul-Visse added.

The study is of interest to a broad spectrum of brain research. Future possible therapeutic targets range from neurodegenerative diseases to stroke.

"We hope that our findings may lead to a new and better understanding of the brain's own repair mechanisms. Ultimately the goal is to strengthen these mechanisms and develop new treatments that can repair the diseased brain," said Dr. Paul-Visse.

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Stem cell that may aid healing and repair found in brain