Alzheimer’s and Huntington’s: Using Stem Cells to Understand and Treat Disease

(Part 4 of 7) Mathew Mark Blurton-Jones, a professor at UC-Irvine's Institute for Memory Impairments and Neurological Disorders, spoke at "Synapses Firing: Connections Made", a patient advocacy event hosted by the California stem cell funding agency (CIRM). The 100+ people in attendance heard from patient advocates about living with neurodegenerative disease and from scientists about recent progress in stem cell research that may lead to new treatments

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Alzheimer's and Huntington's: Using Stem Cells to Understand and Treat Disease

Parkinson’s Disease: Spotlight on Stem Cell Research – Jeff Bronstein

(Part 1 of 3) Jeff Bronstein, MD, Ph.D., spoke at the "Spotlight on Parkinson's Disease," an educational event presented at the CIRM Governing Board meeting on May 7, 2008. Bronstein presented an overview of Parkinson's disease and discussed how stem cell research provides hope for finding new Parkinson's disease therapies.

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Parkinson's Disease: Spotlight on Stem Cell Research - Jeff Bronstein

Beauty salon ‘offers’ stem cell therapy

With all the publicity about the miraculous effects of stem cell therapy, the Department of Health (DOH) should prepare itself for the possibility that the new procedure would be performed by unqualified, and completely clueless, people.

I passed a beauty parlor recently and saw a huge poster on its door announcing the arrival of stem cell therapy. I was instantly reminded of botched breast enhancement and nose jobs performed by salon personnel who seemed to think it was as easy to learn complicated surgical procedures as it was to train to cut hair or do manicures and pedicures.

The DOH should start warning the public not to fall for these special offers just because they are available at giveaway rates.

Modern lifestyle problem

Experts have repeatedly talked about problems brought about by modern lifestyles. Changing diets and stress are two of the best known. Dr. Jaime G. Ignacio, section chief of gastroenterology at Veterans Hospital and head of the Digestive Malignancy Council of the Philippine Society of Gastroenterology, said constipation could be one of the consequences of the combination of these two factors.

Speaking at an event hosted by Boehringer Ingelheim, maker of Dulcolax (generic name Bisacodyl), a formulation for constipation relief, Ignacio, who, as a gastroenterologist is a specialist in digestive system disorders, defined the problem as having fewer than three bowel movements in a week (normal ranges from three times a week to three times a day).

He said constipation itself was not a disease but it could sometimes be a symptom of something serious, like colorectal cancer. But he said about 95 percent of cases were acuteoccurring suddenly and lasting for only a short periodresulting from some sudden lifestyle or hormonal changes, the taking of medication, lack of exercise, etc.

Ignacio said acute was easy to treat, with products like Dulcolax to solve the problem. But, if left unattended, acute constipation could lead to a chronic or long-term condition, which was the more worrisome, and would need medical attention.

He said constipation should be treated as soon as the problem had lasted for four or more days.

Constipation is part of modern living. [Like other diseases] prevention is the key. Safe and effective treatment is available [if needed], Ignacio stressed.

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Beauty salon ‘offers’ stem cell therapy

Stem cell model for hereditary disease developed

ScienceDaily (Oct. 15, 2012) A new method of using adult stem cells as a model for the hereditary condition Gaucher disease could help accelerate the discovery of new, more effective therapies for this and other conditions such as Parkinson's, according to new research from the University of Maryland School of Medicine.

Scientists at the University of Maryland School of Medicine reprogrammed stem cells to develop into cells that are genetically similar to and react to drugs in a similar way as cells from patients with Gaucher disease. The stem cells will allow the scientists to test potential new therapies in a dish, accelerating the process toward drug discovery, according to the paper published online in the journal the Proceedings of the National Academy of Sciences (PNAS) on Oct. 15.

"We have created a model for all three types of Gaucher disease, and used stem cell-based tests to evaluate the effectiveness of therapies," says senior author Ricardo Feldman, Ph.D., associate professor of microbiology and immunology at the University of Maryland School of Medicine, and a research scientist at the University of Maryland Center for Stem Cell Biology and Regenerative Medicine. "We are confident that this will allow us to test more drugs faster, more accurately and more safely, bringing us closer to new treatments for patients suffering from Gaucher disease. Our findings have potential to help patients with other neurodegenerative diseases as well. For example, about 10 percent of Parkinson's disease patients carry mutations in the recessive gene for Gaucher disease, making our research possibly significant for Parkinson's disease as well."

Gaucher disease is the most frequent lipid-storage disease. It affects 1 in 50,000 people in the general population. It is most common in Ashkenazi Jews, affecting 1 in 1,000 among that specific population. The disease occurs in three subtypes -- Type 1 is the mildest and most common form of the disease, causing symptoms such as enlarged livers and spleens, anemia and bone disease. Type 2 causes very serious brain abnormalities and is usually fatal before the age of two, while Type 3 affects children and adolescents.

The condition is a recessive genetic disorder, meaning that both parents must be carriers for a child to suffer from Gaucher. However, said Dr. Feldman, studies have found that people with only one copy of a mutated Gaucher gene -- those known as carriers -- are at an increased risk of developing Parkinson's disease.

"This science is a reflection of the mission of the University of Maryland School of Medicine -- to take new treatments from bench to bedside, from the laboratory to patients, as quickly as possible," says E. Albert Reece, M.D., Ph.D., M.B.A., vice president for medical affairs at the University of Maryland and John Z. and Akiko K. Bowers Distinguished Professor and dean of the University of Maryland School of Medicine. "We are excited to see where this research goes next, bringing new hope to Gaucher patients and their families."

Dr. Feldman and his colleagues used the new reprogramming technology developed by Shinja Yamanaka in Japan, who was recognized with this year's Nobel Prize for Medicine or Physiology. Scientists engineered cells taken from the skin of Gaucher patients, creating human induced pluripotent stem cells, known as hiPSC -- stem cells that are theoretically capable of forming any type of cell in the body. Scientists differentiated the cells to form white blood cells known as macrophages and neuronal cells.

A key function of macrophages in the body is to ingest and eliminate damaged or aged red blood cells. In Gaucher disease, the macrophages are unable to do so -- they can't digest a lipid present in the red blood cell membrane. The macrophages become engorged with lipid and cannot completely clear the ingested red blood cells. This results in blockage of membrane transport pathways in the macrophages lodged in the bone marrow, spleen and liver. The macrophages that the scientists created from the reprogrammed stem cells exhibited this characteristic hallmark of the macrophages taken from Gaucher patients.

To further test the stem cells, the scientists administered a recombinant enzyme that is effective in treating Gaucher patients with Type 1 disease. When the cells were treated with the enzyme, the function of the macrophages was restored -- they completely cleared the red blood cells.

"The creation of these stem cell lines is a lovely piece of stem cell research," said Curt Civin, M.D., professor of pediatrics and physiology, associate dean for research and founding director of the Center for Stem Cell Biology & Regenerative Medicine at the University of Maryland School of Medicine. "Dr. Feldman is already using these Gaucher patient-derived macrophages to better understand the disease fundamentals and to find novel medicines for Gaucher disease treatment. A major goal of our Center for Stem Cell Biology & Regenerative Medicine is to translate our fundamental discoveries into innovative and practical clinical applications that will enhance the understanding, diagnosis, treatment, and prevention of many human diseases. Clinical applications include not only transplantation of stem cells, but also the use of stem cells for drug discovery as Dr. Feldman's studies so beautifully illustrate."

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Stem cell model for hereditary disease developed

Stem Cells Reveal Defect in Parkinson's Cells

The nuclei of brain stem cells in some Parkinson's patients become misshapen with age. The discovery opens up new ways to target the disease.

Nubby nucleus: Brain cells from a deceased Parkinsons patient have deformed nuclei (bottom) compared with normal brain cells from an individual of a similar age. Merce Marti and Juan Carlos Izpisua Belmonte

Stem cells in the brains of some Parkinson's patients are increasingly damaged as they age, an effect that eventually diminishes their ability to replicate and differentiate into mature cell types. Researchers studied neural stem cells created from patients' own skin cells to identify the defects. The findings offer a new focus for therapeutics that target the cellular change.

The report, published today in Nature, takes advantage of the ability to model diseases in cell culture by turning patient's own cells first into so-called induced pluripotent stem cells and then into disease-relevant cell typesin this case, neural stem cells. The basis of these techniques was recognized with a Nobel Prize in medicine last week.

The authors studied cells taken from patients with a heritable form of Parkinson's that stems from mutations in a gene. After growing several generation of neural stem cells derived from patients with that mutation, they saw the cell nuclei start to develop abnormal shapes. Those abnormalities compromise the survival of the neural stem cells, says study coauthor Ignacio Sancho-Martinez of the Salk Institute for Biological Studies in La Jolla, California.

Today's study "brings to light a new avenue for trying to figure out the mechanism of Parkinson's," says Scott Noggle of the New York Stem Cell Foundation. It also provides a new set of therapeutic targets: "Drugs that target or modify the activity [of the gene] could be applicable to Parkinson's patients. This gives you a handle on what to start designing drug screens around."

The strange nuclei were also seen in patients who did not have a known genetic basis for Parkinson's disease. The authors suggest this indicates that dysfunctional neural stem cells could contribute to Parkinson's. While that conclusion is "highly speculative," says Ole Isacson, a neuroscientist at Harvard Medical School, the study demonstrates the "wealth of data and information that we now can gain from iPS cells."

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Stem Cells Reveal Defect in Parkinson's Cells

Stem cell doctor in court: Day 2

COLLIER COUNTY -

A Southwest Florida doctor spent another day in court fighting for his right to practice medicine.

Bonita Springs cardiologist Zannos Grekos' license is currently suspended because two of his patients died after undergoing a controversial stem cell procedure.

Wednesday was day two of what's scheduled to be a four day trial. The courtroom was again filled with patients supporting Grekos and his stem cell treatment.

This hearing focuses only on the first patient that died after getting that treatment. Her family was also in attendance and wants Grekos' medical license revoked.

Dr. Richard Roland spoke about a call he got from Grekos in 2010. He had just performed a stem cell procedure on his patient Domenica Fitgzerald and something went wrong.

Roland was Fitzgerald's ICU doctor.

"I was quite shocked. My first comment was, 'You did what?' His response was, 'Yes. We've been having good luck with these procedures,'" Dr. Roland said. "I had concerns that this was criminal."

Fitzgerald's family says the breast cancer survivor was looking for a cure for nerve damage caused by chemotherapy.

They say Grekos gave her false hope.

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Stem cell doctor in court: Day 2

Microscopic packets of stem cell factors could be key to preventing lung disease in babies

ScienceDaily (Oct. 31, 2012) Researchers at Boston Children's Hospital have found that microscopic particles containing proteins and nucleic acids called exosomes could potentially protect the fragile lungs of premature babies from serious lung diseases and chronic lung injury caused by inflammation.

The findings explain earlier research suggesting that while transplanting a kind of stem cell called mesenchymal stem cells (MSCs) could help reduce lung injury and prevent inflammation in a mouse model, the fluid in which the cells were grown was more effective than the cells themselves.

The research team -- led by Stella Kourembanas, MD, and S. Alex Mitsialis, PhD, and spearheaded by led by Changjin Lee, PhD, all of the Division of Newborn Medicine at Boston Children's -- published their findings online on October 31 in the journal Circulation.

Premature babies often struggle to get enough oxygen into their underdeveloped lungs, resulting in hypoxia and the need for ventilator assistance to breathe. Their lungs are particularly susceptible to inflammation, which can lead to poor lung growth and chronic lung disease. Inflammation is also often associated with pulmonary hypertension (PH) -- dangerously high blood pressure in the pulmonary artery (the vessel that carries blood from the heart to the lungs), which can have both short- and long-term consequences.

"PH is a complex disease fueled by diverse, intertwined cellular and molecular pathways," according to Kourembanas, who chairs Boston Children's Newborn Medicine division. "We have treatments that improve symptoms but no cure, largely because of this complexity. We need to be able to target more than one pathway at a time."

In 2009, Kourembanas, Mitsialis and others showed that injection of MSCs could prevent PH and chronic lung injury in a newborn mouse model of the disease. The results were puzzling, though, because the team found that few of the injected stem cells actually engrafted within the lungs. They also found that they could achieve better results by injecting just conditioned media -- the fluid the cells had been grown in -- than by injecting the cells themselves.

"We knew, then, that the significant anti-inflammatory and protective effects we saw had to be caused by something released by the MSCs," Kourembanas explained. "The question was, what?"

To answer that question, the research team grew mouse MSCs in the laboratory and searched the conditioned media for any secreted factors. They came upon exosomes, which many cell types, including MSCs, produce and release as a kind of communication vehicle.

The team found that injecting just purified exosomes from MSCs reduced lung inflammation and prevented the occurrence of PH in their animal model of PH. In contrast, neither MSC-conditioned media depleted of exosomes nor exosomes purified from other cell types had any effect on inflammation or PH in the model, indicating that something unique to the MSC-produced exosomes is required for their protective effect.

"We are actively working to figure out what exactly within the MSC-produced exosomes causes these anti-inflammatory and protective effects," Kourembanas said. "But we know that these exosomes contain microRNAs as well as other nucleic acids. They also induce expression of specific microRNAs in the recipient lung."

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Microscopic packets of stem cell factors could be key to preventing lung disease in babies

No stem cell act at the moment – Liow

KUALA LUMPUR, Oct 29 (Bernama) -- The health ministry did not have plans to introduce a stem cell act at the moment, said the minister, Datuk Seri Liow Tiong Lai.

He said, the ministry's four guidelines on stem cell research sufficiently served as standards to which practitioners and scientists involved in stem cell research and therapy should adhere to, ensured patients were out of harm's way.

The guidelines would provide a framework for researchers, clinicians and companies involved in research, clinical trials and manufacture of stem cells, he noted.

"There is no stem cell act in this country. But the guidelines alone are sufficient to provide the grounds and ethical environment to carry out their work," Liow told reporters after launching the 1st National Stem Cell Congress here today.

The four guidelines are National Standards For Haemopoietic Stem Cell Therapy, National Standards For Cord Blood Banking and Transplantation, National Standards For Stem Cell Transplantation and Guidelines On Stem Cell Research and Therapy.

Liow said: "Before we came up with the guidelines, we formed a committee to discuss the details of the research. The committee also included Jakim and religious officials for their views."

He said the use of cell-based therapies should be done strictly under clinical trials. Prior to the clinical trials, there must be sufficient evidence to show safety, quality and efficacy.

Meanwhile, Liow said stem cell therapy in Malaysia was developing well in government, as well as university hospitals, noting that the number of patients receiving bone marrow and stem cell transplantation for leukaemia and solid tumours was on the rise.

He said a total of 213 haemopoietic stem cell transplants were performed and registered in the country, the majority of which centred on malignant disorders, namely leukaemia and lymphoma.

Currently, the minister disclosed, there were 11 haemopoietic transplant centres performing haemopoietic stem cell transplants in the country, including Ampang Puteri Specialist Hospital, Haematology Department (Ampang Hospital), Haemopoietic Stem Cell Transplant Unit (Universiti Sains Malaysia Hospital) and Paediatric BMT Unit, Institute of Paediatrics Kuala Lumpur Hospital.

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No stem cell act at the moment - Liow

Nobel Laureates Yamanaka and Gurdon Meet on UCSF Campus for Stem Cell Symposium

Shinya Yamanaka, MD, PhD, left, and John Gurdon, PhD, speak at an Oct. 24 press briefing during the ISSCR-Roddenberry International Symposium on Cellular Reprogramming held at UCSF's Mission Bay campus.

More than two weeks after winning the 2012 Nobel Prize for Physiology or Medicine Shinya Yamanaka, MD, PhD, and John Gurdon, PhD, were able to celebrate their monumental achievement together for stem cell discoveries made half a century and half a world apart.

The pair appropriately met for the International Symposium on Cellular Reprogramming, held in San Francisco last week at UCSFs Mission Bay campus by the International Society for Stem Cell Research (ISSCR) and the Roddenberry Center for Stem Cell Biology and Medicine at the Gladstone Institutes.

During an Oct. 24 press briefing, Yamanaka president of the ISSCR, a Gladstone senior investigator and a UCSF professor of anatomy urged his home country of Japan to invest more in science, while Gurdon, of the Gurdon Institute in Cambridge, England, advocated for allowing well-informed patients to assume risks of clinical trials that might benefit them, without a burdensome amount of regulation.

The work for which they won the Nobel Prize, to be awarded in Stockholm in December, began in 1958 before Yamanaka was born when Gurdon cloned a normal tadpole by putting the DNA-enveloping nucleus of a skin cell from a frog into a de-nucleated frog egg. The experiment showed that the DNA from a fully mature cell body called a somatic cell still contains the genetic information needed to program the orderly development of a complete organism.

This knowledge inspired Yamanaka in his own work decades later.

Shinya Yamanaka, president of the International Society for Stem Cell Research (ISSCR), presented at the ISSCR-Roddenberry International Symposium on Cellular Reprogramming last week.

In 2007, Yamanaka showed that the manipulation of just four genes within a cell can induce a specialized skin cell to become a pluripotent stem cell one capable of spinning off virtually any cell type in the body. This achievement was one few thought possible, and it has inspired thousands of scientists and raised hopes for new therapies to replace damaged organs and tissues.

In the long term, the induced pluripotent stem (iPS) cells first developed by Yamanaka may offer some advantages over embryonic stem cells in the study of disease and in the development of novel tissue transplants for damaged organs. Embryonic stem cells still are regarded as the gold standard of pluripotency, Yamanaka said, but iPS researchers are making rapid progress.

My hope is that one day we can use iPS cells instead of embryonic stem cells, Yamanaka said at the briefing.

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Nobel Laureates Yamanaka and Gurdon Meet on UCSF Campus for Stem Cell Symposium

Cytomedix to Present at Upcoming Stem Cell Conferences

GAITHERSBURG, MD--(Marketwire - Oct 23, 2012) - Cytomedix, Inc. ( OTCQX : CMXI ) (the "Company"), a leading developer of biologically active regenerative therapies, today announced that it will present at the First Annual Houston Stem Cell Summit on October 26th and at the 2012 Stem Cell Meeting on the Mesa Conference, October 29th - 30th.The presentations will be given by Edward L. Field, Chief Operating Officer of Cytomedix.

Houston Stem Cell Summit Houston, TX, Oct 26 - 27

Presentation Time: 9:50 AM, Friday, October 26th, 2012 Location: The Houstonian Hotel, 111 North Post Oak Lane, Houston, TX

The Houston Stem Cell Summit will bring together, for the first time; the many and varied stem cell research and commercialization activities in Texas with the leading global researchers and entrepreneurs. The latest therapeutic research regarding the use of adult stem and progenitor cell therapies will be featured.The Summit will also provide a forum for entrepreneurs to discuss their latest efforts to commercialize stem cell therapies, and to debate and discuss FDA and other legal and regulatory issues impacting stem cell research and commercialization. Participating Texas institutions include: Baylor College of Medicine, Texas Heart Institute, Texas A&M, Rice University, and MD Anderson.The organizers expect more than 35 speakers, 300 attendees, and participation from a dozen of the leading commercial stem cell companies.

2012 Stem Cell Meeting on the Mesa Investor and Partnering Forum La Jolla, CA, Oct 29 - 30

Presentation Time: 1:30 PM, Monday October 29th, 2012 Location: Sanford Consortium, 2880 Torrey Pines Scenic Drive, La Jolla, CA

Edward Field will also be a panelist in a discussion "Getting to Market: Keys to Regulatory Success" on October 30th at 2:00pm. This session will examine the regulatory challenges facing regenerative medicine companies and the strategies they employ to reach the market.

The 2012 Stem Cell Meeting on the Mesa is aimed at bringing together senior members of the business development and scientific research communities in regenerative medicine to advance stem cell science into cures for debilitating diseases and injuries. The meeting features a nationally recognized Scientific Symposium attended by more than 600 leading scientists and researchers alongside the regenerative medicine industry's premier annual Investor and Partnering Forum designed to facilitate a bridge between academia and industry through one-on-one meetings and further the translation of clinical research.

About Cytomedix, Inc. Cytomedix, Inc. is a fully integrated regenerative medicine company commercializing and developing innovative platelet and adult stem cell separation products that enhance the body's natural healing processes. The Company's advanced autologous technologies offer clinicians a new treatment paradigm for wound and tissue repair. The Company's patient-derived PRP systems are marketed by Cytomedix in the U.S. and distributed internationally. Our commercial products include the AutoloGel System, cleared by the FDA for wound care and the Angel Whole Blood Separation System. The Company is developing novel regenerative therapies using our proprietary ALDH Bright Cell ("ALDHbr") technology to isolate a unique, biologically active population of a patient's own stem cells. A Phase 2 trial evaluating the use of ALDHbr for the treatment of ischemic stroke is underway. For additional information please visit http://www.cytomedix.com.

Safe Harbor Statement Statements contained in this press release not relating to historical facts are forward-looking statements that are intended to fall within the safe harbor rule for such statements under the Private Securities Litigation Reform Act of 1995. The information contained in the forward-looking statements is inherently uncertain, and Cytomedix' actual results may differ materially due to a number of factors, many of which are beyond Cytomedix' ability to predict or control, including among many others, risks and uncertainties related to the Company's ability to successfully integrate the Aldagen acquisition, to successfully manage contemplated clinical trials, to manage and address the capital needs, human resource, management, compliance and other challenges of a larger, more complex and integrated business enterprise, viability and effectiveness of the Company's sales approach and overall marketing strategies, commercial success or acceptance by the medical community, competitive responses, the Company's ability to raise additional capital and to continue as a going concern, and Cytomedix's ability to execute on its strategy to market the AutoloGel System as contemplated. To the extent that any statements made here are not historical, these statements are essentially forward-looking. The Company uses words and phrases such as "believes", "forecasted," "projects," "is expected," "remain confident," "will" and/or similar expressions to identify forward-looking statements in this press release. Undue reliance should not be placed on forward-looking information. These forward-looking statements are subject to known and unknown risks and uncertainties that could cause actual events to differ from the forward-looking statements. More information about some of these risks and uncertainties may be found in the reports filed with the Securities and Exchange Commission by Cytomedix, Inc. Cytomedix operates in a highly competitive and rapidly changing business and regulatory environment, thus new or unforeseen risks may arise. Accordingly, investors should not place any reliance on forward-looking statements as a prediction of actual results. Except as is expressly required by the federal securities laws, Cytomedix undertakes no obligation to update or revise any forward-looking statements, whether as a result of new information, changed circumstances or future events or for any other reason. Additional risks that could affect our future operating results are more fully described in our U.S. Securities and Exchange Commission filings, including our Annual Report for the year ended December 31, 2011 and other subsequent filings. These filings are available at http://www.sec.gov.

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Cytomedix to Present at Upcoming Stem Cell Conferences

Stem cell therapy a cure-all? Not so fast

MANILA, Philippines Its supposed to cure various illnesses such as cancer, spinal cord injury and Parkinsons disease. Is stem cell therapy the cure-all that it is touted to be?

Health Undersecretary Teodoro Herbosa said it is important to note that stem cell treatments are still at the experimental stage.

"The advisory is very clear. This is still an investigative form of therapy. Anecdotal reports are not enough evidence to say there is treatment, he said in an interview on ANC's Talkback with Tina Palma.

He said there are only two standard stem cell therapies considered effective and acceptable to the medical community.

"To date, I can only name two cases that are considered standard therapy. That is bone marrow transplantation--one for severe cancer, blood cancer and the other one is bone marrow transplantation after chemotherapy for any type of cancer, he said.

Herbosa said the Department of Health cannot confirm yet if stem cell treatment is indeed effective against certain diseases.

Dr. Tranquilino Elicao Jr., an oncologist who availed of the treatment in April in Frankfurt, Germany, said stem cell therapy cured his high blood pressure, sugar, cholesterol and uric acid.

He had 12 injections of cells, which came from lambs.

After a month, I had my blood tests. Everything went down to normal, Elicao said.

Elicao also said he is not taking medication anymore because he has regained his health.

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Stem cell therapy a cure-all? Not so fast

Stem cell therapies for multiple sclerosis, other myelin disorders expected soon

ScienceDaily (Oct. 25, 2012) When the era of regenerative medicine dawned more than three decades ago, the potential to replenish populations of cells destroyed by disease was seen by many as the next medical revolution. However, what followed turned out not to be a sprint to the clinic, but rather a long tedious slog carried out in labs across the globe required to master the complexity of stem cells and then pair their capabilities and attributes with specific diseases.

In a review article appearing October 25 in the journal Science, University of Rochester Medical Center scientists Steve Goldman, M.D., Ph.D., Maiken Nedergaard, Ph.D., and Martha Windrem, Ph.D., contend that researchers are now on the threshold of human application of stem cell therapies for a class of neurological diseases known as myelin disorders -- a long list of diseases that include conditions such as multiple sclerosis, white matter stroke, cerebral palsy, certain dementias, and rare but fatal childhood disorders called pediatric leukodystrophies.

"Stem cell biology has progressed in many ways over the last decade, and many potential opportunities for clinical translation have arisen," said Goldman. "In particular, for diseases of the central nervous system, which have proven difficult to treat because of the brain's great cellular complexity, we postulated that the simplest cell types might provide us the best opportunities for cell therapy."

The common factor in myelin disorders is a cell called the oligodendrocyte. These cells arise, or are created, by another cell found in the central nervous system called the glial progenitor cell. Both oligodendrocytes and their "sister cells" -- called astrocytes -- share this same parent and serve critical support functions in the central nervous systems.

Oligodendrocytes produce myelin, a fatty substance that insulates the fibrous connections between nerve cells that are responsible for transmitting signals throughout the body. When myelin-producing cells are lost or damaged in conditions such as multiple sclerosis and spinal cord injury, signals traveling between nerves are weakened or even lost. Astrocytes also play an essential role in the brain. Long overlooked and underappreciated, it is now understood that astrocytes are critical to the health and signaling function of oligodendrocytes as well as neurons.

Glial progenitor cells and their offspring represent a promising target for stem cell therapies, because -- unlike other cells in the central nervous system -- they are relatively homogeneous and more readily manipulated and transplanted. In the case of oligodendrocytes, multiple animal studies have shown that, once transplanted, these cells will disperse and begin to repair or "remyelinate" damaged areas.

"Glial cell dysfunction accounts for a broad spectrum of diseases, some of which -- like the white matter degeneration of aging -- are far more prevalent than we previously realized," said Goldman. "Yet glial progenitor cells are relatively easy to work with, especially since we don't have to worry about re-establishing precise point to point connections as we must with neurons. This gives us hope that we may begin to treat diseases of glia by direct transplantation of competent progenitor cells."

Scientists have reached this point, according to the authors, because of a number of key advances. Better imaging technologies -- namely advanced MRI scanners -- now provide greater insight and clarity into the specific damage caused in the central nervous system by myelin disorders. These technologies also enable scientists to precisely follow the results of their work.

Even more importantly, researchers have overcome numerous obstacles and made significant strides in their ability to manipulate and handle these cells. Goldman's lab in particular has been a pioneer in understanding the precise chemical signals necessary to coax stem cells into making glial progenitor cells, as well as those needed to "instruct" these cells to make oligodendrocytes or astrocytes. His lab has been able to produce these cells from a number of different sources -- including "reprogramming" skin cells, a technology that has the advantage of genetically matching transplanted cells to the donor. They have also developed techniques to sort these cells based on unique identifying markers, a critical step that ensures the purity of the cells used in transplantation, lowering the risk for tumor formation.

Nedergaard's lab has studied the integration of these cells into existing neural networks, and well as in imaging their structure and function in the adult nervous system. Together, the two labs have developed models of both human neural activity and disease based on animals transplanted with glial progenitor cells, which will enable human neural cells to be evaluated in the context of the live adult brain -- as opposed to a test tube. This work has already opened new avenues in both modeling and potentially treating human glial disease.

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Stem cell therapies for multiple sclerosis, other myelin disorders expected soon

Pedersen creates stem cell drug powerhouse

A Cambridge University technology team that developed a way to generate liver cells from skin cells has formed a new company to supply stem cell products to the drug discovery and regenerative medicine sectors.

DefiniGEN is based on the research of Dr Ludovic Vallier, Dr Tamir Rashid and global stem cell guru Professor Roger Pedersen of the Anne McLaren Laboratory of Regenerative Medicine.

DefiniGEN is based in Cambridge and has been funded by a group led by Cambridge Enterprise, the Universitys commercialisation arm, along with members of Cambridge Angels and Cambridge Capital Group.

Prof Pedersen is one of the coveted brains of stem cell research. He moved to Cambridge to escape a puritan US approach to stem cell research and has resisted attempts to woo him back across the Atlantic.

The potential therapeutic applications of stem cells such as regenerating damaged tissues or organs have generated a great deal of interest over the past decade. While these types of applications are exciting, it is a long journey from lab to clinic.

The most immediate impact of stem cells on human health will most likely come from their use in the development of new drugs. The ability to generate stem cells by reprogramming cells from patients skin has revolutionised human stem cell research.

These cells, known as human induced pluripotent stem cells (hIPSC), can be differentiated into almost any cell type, allowing the opportunity to have a ready source of human cells for testing new therapies.

DefiniGEN has developed the technology to generate human liver cells (hepatocytes) in a highly reproducible and scalable manner for commercial use. This represents a major breakthrough in the costly and time-consuming process of developing new therapies.

The technology has also been used to effectively model a diverse range of inherited liver diseases and has the potential to accelerate the development of new therapies for these conditions.

The liver is the key organ for metabolising drugs and removing toxins from the body. Consequently, it is often affected by toxic compounds.

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Pedersen creates stem cell drug powerhouse

New lab space for stem cell research

SANTA BARBARA Biomedical research at UC Santa Barbara has catapulted to a position of leadership in the arena of stem cell biology, offering progress toward cures for vision diseases such as macular degeneration. Stem cell research has the potential to transform the practice of medicine, by replacing diseased tissue with healthy new cells. Interdisciplinary teams of UC Santa Barbara researchers including world-renowned faculty members recruited from the U.S. and Britain are leading the charge. The university's newly renovated lab space is critical to the mission.

The latest research holds the promise of developing stem cells from skin, called induced pluripotent stem cells, which could eventually replace the use of human embryonic stem cells. The hope is to find cures for diseases including macular degeneration, diabetic retinopathy and Alzheimer's.

Campus leaders, dignitaries, scientists, and engineers gathered at UC Santa Barbara on Friday (Oct. 26) to celebrate the completion of a dream: 10,000 square feet of renovated laboratory space devoted to stem cell research. This was the grand opening ceremony and ribbon-cutting in celebration of UC Santa Barbara's Center for Stem Cell Biology and Engineering, part of the Neuroscience Research Institute. The California Institute for Regenerative Medicine (CIRM) contributed to the renovation with a $3.2 million grant that was matched by $3.2 million from the university. The top-flight new laboratories are a magnet for attracting new talent.

Chancellor Henry T. Yang commented: "We think UCSB can make important contributions to stem cell research at the interface of biology, neuroscience, and engineering. The future is very bright as we continue to grow and add more top-notch researchers and faculty to this area. These new laboratories will make this possible."

Kenneth S. Kosik, physician and Harriman Chair in the Department of Molecular, Cellular, and Developmental Biology, and co-director of the Neuroscience Research Institute, remarked on this watershed moment in UCSB's stem cell research. "This is a momentous event," said Kosik. "It's a big milestone for UCSB to have come from a premier engineering institute to a place where we are doing world-class work in another entire area, in stem cell biology. With that triumph, that success, we are moving into world-class ranks here."

Leading stem cell biologist Dennis Clegg, center executive director of strategy, planning and operations, and co-principal director of the California Project to Cure Blindness, has directed the center since its inception, and also directed strategy for UC Santa Barbara's specific contribution to stem cell biology.

"We realized that we have a tremendous collection of expertise in bioengineering, basic molecular biology, and neuroscience," said Clegg. "Those groups together, working together collaborating across normal interdisciplinary lines make for a powerful combination of expertise. And we've been able to address some of the key problems in stem cell research, like how to deliver the cells, how to get the cells pure, how to monitor the cells after transplantation, using this combination of bioengineering and molecular biology.

"This has been a long process to complete this renovation for the stem cell center," Clegg said. "It started back in 2007, with our application to CIRM for a major facilities grant. It involved a large number of people on campus scientists, design and construction, administrators.

"We spent many hours figuring out how to come up with matching funds and how to raise money for the stem cell center. It really is a realization of our dream to expand stem cell research on campus. We think we have a lot more to offer. The trajectory is upward; we are really making progress. This will allow us to hire new people, new faculty, bring them in and collaborate with the group of stellar scientists that we've already assembled."

One of the new center directors, Peter Coffey, was hired with a CIRM recruitment grant earlier this year. Coffey, who holds the Garland Chair and is the center's executive director of translation, is considered a star in the world of stem cell science. While at the University College London, he started a group called the London Project to Cure Blindness, which will begin clinical trials early next year. Coffey hopes to bring a part of this project to California.

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New lab space for stem cell research

FDA issues warning letter to local stem cell company

The Food and Drug Administration has informed the Sugar Land company involved in Gov. Rick Perry's adult stem-cell procedure that it is illegally marketing an unlicensed drug.

In a warning letter, the FDA gave Celltex Therapeutics Corp. 15 business days to submit a plan to address the agency's concerns, including correcting previously cited manufacturing problems. The letter said that failure to respond promptly could result in seizure or injunction by the FDA.

"Based on (our) information, your product violates the Federal Food, Drug, and Cosmetic Act and the Public Health Service Act," says the letter, sent on Sept. 24 and publicly posted Tuesday.

The letter comes about six months after the FDA made a 10-day inspection of the facilities where Celltex banks and grows stem cells taken from prospective patients. The agency subsequently filed a report, obtained by the Chronicle in June, detailing dozens of manufacturing deficiencies, from incorrectly labeled products to failed sterility tests.

The new warning letter reiterates those problems and asks for more information about them.

David Eller, Celltex's CEO, was unavailable Tuesday, but a public relations official said the company on Wednesday would make available a redacted copy of its letter to the FDA.

In a previous news release, Eller said Celltex "respectfully but firmly" disagreed with the FDA's position that its process causes the cells to be considered biological drugs and thus subject to the federal agency's regulations. Biological drugs involve living human cells, as opposed to chemically synthesized drugs.

"We are considering all options as we work with the agency toward a resolution," Eller said in the release.

Adult stem cells multiply to replenish dying cells. Long used to treat leukemia and other cancers, they have recently shown promise for tissue repair in other diseases, though most scientists in the field consider them not ready for mainstream use.

Treatment, at a price

Read more:
FDA issues warning letter to local stem cell company

Harvard Ophthalmologist Dr. Ula Jurkunas Introduces Stem Cell Transplant for Eyes

Grants Pass, OR (PRWEB) October 16, 2012

Harvard Ophthalmologist and Corneal Stem Cell Researcher Ula Jurkunas, MD, has announced an important new stem cell transplant procedure for the eyes.

Speaking on the Sharon Kleyne Hour Power of Water radio show, Dr. Jurkunas, predicted that the procedure will offer a significant benefit to patients with certain corneal diseases, and corneal injuries such as chemical and thermal burns (The cornea is the eyes clear portion).

Stem cell research has been in the news because the 2012 Nobel Prize for Medicine was awarded for stem cell research.

Dr. Jurkunas explained to host Sharon Kleyne that the human eye produces its own adult (non-embryonic) stem cells. These are found between the limbus (where the clear cornea meets the white of the eye) and the conjunctiva (the red meaty tissue in the eyes inner corner). Their function is to replenish corneal cells to keep the cornea clear and healthy.

Production of corneal stem cells, according to Dr. Jurkunas, can become impaired due to a disease entity such as an infection, severe allergy, severe dry eye, immunological disorder or chronic inflammation; or due to injury such as a chemical or thermal burn. These traumas can cause the cornea to become cloudy and ulcerated. Prior to the present corneal stem cell research, there had been no reliable, non-invasive treatment for these conditions.

Corneal stem cell transplantation, Dr. Jurkunas explains, has the advantage of utilizing the patients own tissue as donor cells. Stem cells may be taken either from healthy tissue elsewhere in the diseased eye, from the patients other eye, or from the patients inner cheek (which has many similarities to eye tissue and also produces adult stem cells). Donor stem cells are then isolated and grown in culture. The final step is to transfer them to the affected cornea using a stem cell bandage.

The procedure, says Dr. Jurkunas, has resulted in dramatic corneal clearing and sight restoration. Although research is ongoing and the procedure remains experimental, corneal stem cell therapy is available in clinical trials. Widespread applications of the procedure, including routine testing for corneal stem cell deficiency, are anticipated. Stem cell therapy, according to Dr. Jurkunas, could eventually be used for macular degeneration, glaucoma and other eye diseases.

Dr. Jurkunas stressed the importance of water and hydration in maintaining a healthy tear film and cornea. The tear film covering the cornea is 99% water and is essential to the light refraction that enables vision. Dry eye and related eye infections, according to Dr. Jurkunas, can damage both the cornea and adjacent stem cell producing tissues that enable the cornea to repair itself. Water in the tear film stimulates the healthy production of stem cells. Water is also critical to keeping stem cells viable during transplantation.

Mrs. Kleyne and Dr. Jurkunas agree that non-invasive therapies using the bodys own tissues, such as corneal stem cell transplantation, could eventually prove indispensable in combating the worldwide health effects of global drying and dehydration.

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Harvard Ophthalmologist Dr. Ula Jurkunas Introduces Stem Cell Transplant for Eyes

Realizing the potential of stem cell therapy: Studies report progress in developing treatments for diseases and injuries

ScienceDaily (Oct. 15, 2012) New animal studies provide additional support for investigating stem cell treatments for Parkinson's disease, head trauma, and dangerous heart problems that accompany spinal cord injury, according to research findings released today.

The work, presented at Neuroscience 2012, the annual meeting of the Society for Neuroscience and the world's largest source of emerging news about brain science and health, shows scientists making progress toward using stem cell therapies to repair neurological damage.

The studies focused on using stem cells to produce neurons -- essential, message-carrying cells in the brain and spinal cord. The loss of neurons and the connections they make for controlling critical bodily functions are the chief hallmarks of brain and spinal cord injuries and of neurodegenerative afflictions such as Parkinson's disease and ALS (amyotrophic lateral sclerosis), also known as Lou Gehrig's disease.

Today's new findings show that:

Other recent findings discussed show that:

"As the fields of developmental and regenerative neuroscience mature, important progress is being made to begin to translate the promise of stem cell therapy into meaningful treatments for a range of well-defined neurological problems," said press conference moderator Jeffrey Macklis, MD, of Harvard University and the Harvard Stem Cell Institute, an expert on development and regeneration of the mammalian central nervous system. "Solid, rigorous, and well-defined pre-clinical work in animals can set the stage toward human clinical trials and effective future therapies."

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The above story is reprinted from materials provided by Society for Neuroscience (SfN), via AlphaGalileo.

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Realizing the potential of stem cell therapy: Studies report progress in developing treatments for diseases and injuries

FDA issues warning to local stem cell company

The Food and Drug Administration has informed the Sugar Land company involved in Gov. Rick Perry's adult stem-cell procedure that it is illegally marketing an unlicensed drug.

In a warning letter, the FDA gave Celltex Therapeutics Corp. 15 business days to submit a plan to address the agency's concerns, including correcting previously cited manufacturing problems. The letter said that failure to respond promptly could result in seizure or injunction by the FDA.

"Based on (our) information, your product violates the Federal Food, Drug, and Cosmetic Act and the Public Health Service Act," says the letter, sent on Sept. 24 and publicly posted Tuesday.

The letter comes about six months after the FDA made a 10-day inspection of the facilities where Celltex banks and grows stem cells taken from prospective patients. The agency subsequently filed a report, obtained by the Chronicle in June, detailing dozens of manufacturing deficiencies, from incorrectly labeled products to failed sterility tests.

The new warning letter reiterates those problems and asks for more information about them.

David Eller, Celltex's CEO, was unavailable Tuesday, but a public relations official said the company on Wednesday would make available a redacted copy of its letter to the FDA.

In a previous news release, Eller said Celltex "respectfully but firmly" disagreed with the FDA's position that its process causes the cells to be considered biological drugs and thus subject to the federal agency's regulations. Biological drugs involve living human cells, as opposed to chemically synthesized drugs.

"We are considering all options as we work with the agency toward a resolution," Eller said in the release.

Adult stem cells multiply to replenish dying cells. Long used to treat leukemia and other cancers, they have recently shown promise for tissue repair in other diseases, though most scientists in the field consider them not ready for mainstream use.

Treatment, at a price

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FDA issues warning to local stem cell company