Category Archives: Diseases

Stem cells have assumed near-mythical status in the popular imagination as a possible cure for every disease under the sun. But while public attention has focused on their potential in regenerative medicine, stem cells have quietly gained a foothold in drug development a move that may hail a huge but unheralded shake-up of the biological sciences.

I think there are tremendous parallels to the early days of recombinant DNA in this field, says James Thomson, director of regenerative biology at the Morgridge Institute for Research in Madison, Wisconsin, and one of the founders of Cellular Dynamics International, also in Madison. I dont think people appreciated what a broad-ranging tool recombinant DNA was in the middle '70s." At the same time, he says, they underestimated the difficulty of using it in treatments.

Now stem cells are in a similar situation, he says, and although therapeutic use is likely to come to fruition eventually, people underappreciate how broadly enabling a research tool it is, he says.

Laboratory-grown stem cells hold much promise for regenerative medicine, but are being increasingly used in drug testing.

MASSIMO BREGA, THE LIGHTHOUSE/SCIENCE PHOTO LIBRARY

Drug companies began dipping a tentative toe into the stem-cell waters about two years ago (see 'Testing time for stem cells'). Now, the pharmaceutical industry is increasingly adopting stem cells for testing the toxicity of drugs and identifying potential new therapies, say those in the field.

Cellular Dynamics sells human heart cells called cardiomyocytes, which are derived from induced pluripotent stem (iPS) cells. Thomson says that essentially all the major pharma companies have bought some. The company also produces brain cells and cells that line blood vessels, and is about to release a line of human liver cells.

Yet Cellular Dynamics is just one of the companies in the field. Three years ago, stem-cell biologist Stephen Minger left his job in UK academia to head GE Healthcares push into stem cells (see 'Top scientist's industry move heralds stem-cell shift'). The medical-technology company, headquartered in Chalfont St. Giles, UK, has been selling human heart cells made from embryonic stem (ES) cells for well over a year, and is due to start selling liver cells soon.

Minger and his team at GE Healthcare assessed the heart cells in a blind trial against a set of unnamed drug compounds to see if the cells would reveal which compounds were toxic. When the compounds were unmasked, Minger says, they found that the cells had been affected by the known toxic compounds. But, crucially, in a number of cases, the cells identified a problem that had only been discovered after the drugs had reached the market and after they had been approved by agencies such as the US Food and Drug Administration (FDA).

These are compounds which went all the way through animal testing, then went through phase I, II, III and then were licensed in many cases by the FDA, says Minger.

See the original post:
Stem cells take root in drug development

RICHMOND, Calif., May 21, 2012 /PRNewswire/ --Sangamo BioSciences, Inc. (SGMO) announced today that data from clinical, preclinical and research-stage programs focused on the development of ZFP Therapeutics for HIV/AIDS, monogenic diseases and stem cell applications, were described in twelve presentations given by Sangamo scientists and collaborators at the 15th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). The meeting was held in Philadelphia from May 15-19, 2012.

"Sangamo's zinc finger DNA-binding protein (ZFP) technology is enabling development of new and improved gene and cell therapy approaches," said Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president, research and development. "Our ZFP Nuclease (ZFN) technology provides an extremely efficient and precise process for editing any DNA sequence. This enables us to disrupt specific genes or to precisely add DNA sequences that allow a patient's own gene to be corrected and its proper function restored while preserving the natural regulation of the gene.

Sangamo has also developed technology that allows a therapeutic gene to be inserted into a specific 'safe harbor' site. Our ability to target changes to precise locations rather than randomly into the genome, avoids the challenges of traditional gene-addition approaches that can result in unintended mutations. The increased number of related presentations at this meeting demonstrates the growing adoption of ZFN-based gene editing by the field."

Presentations from Sangamo included preliminary clinical data from ongoing Phase 1 clinical trials in HIV/AIDS as well as data from preclinical and research-stage human therapeutic programs. Therapeutic areas included ZFP-based approaches for monogenic diseases such as hemophilia, hemoglobinopathies and Huntington's disease as well as adoptive T-cell therapies for oncology.

"Visibility of ZFPs in the scientific agenda at the ASGCT meeting illustrates the broad range of potential applications for ZFP Therapeutics," said Edward Lanphier, Sangamo's president and CEO. "Our technology can be used to modify any gene with singular specificity and high efficiency. As our technology functions at the DNA level, it can potentially be applied to any disease-related gene making it a versatile platform for the generation of novel therapeutic approaches for the treatment of unmet medical needs."

ZFP Therapeutics Featured at ASGCT Meeting

All abstracts for the meeting are available online at 2012 ASGCT Meeting Abstracts.

About Sangamo

Sangamo BioSciences, Inc. is focused on research and development of novel DNA-binding proteins for therapeutic gene regulation and genome editing. It has ongoing Phase 2 and Phase 1/2 clinical trials to evaluate the safety and efficacy of a novel ZFP Therapeutic for the treatment of HIV/AIDS.Sangamo's other therapeutic programs are focused on monogenic diseases, including hemophilia and hemoglobinopathies such as sickle cell anemia and beta-thalassemia, and a program in Parkinson's disease. Sangamo's core competencies enable the engineering of a class of DNA-binding proteins known as zinc finger DNA-binding proteins (ZFPs). Engineering of ZFPs that recognize a specific DNA sequence enables the creation of sequence-specific ZFP Nucleases (ZFNs) for gene modification and ZFP transcription factors (ZFP TFs) that can control gene expression and, consequently, cell function. Sangamo has entered into a strategic collaboration with Shire to develop therapeutics for hemophilia and other monogenic diseases and has established strategic partnerships with companies in non-therapeutic applications of its technology including Dow AgroSciences and Sigma-Aldrich Corporation. For more information about Sangamo, visit the company's website at http://www.sangamo.com.

ZFP Therapeutic is a registered trademark of Sangamo BioSciences, Inc. CompoZr is a registered trademark of Sigma-Aldrich Corporation.

Excerpt from:
Sangamo BioSciences And Collaborators Highlight Widening Applications Of ZFP Therapeutics® In Presentations At Major ...

Tim and Padma Vellaichamy of Parramatta have had their new born child's umbilical cord stored cryogenically for future treatment. Pictured with their as yet unnamed three week old daughter. Picture: Adam Ward Source: The Daily Telegraph

IT'S current preservation for the future regeneration - and now umbilical cord tissue is going on ice in Australia for the first time.

Usually discarded after birth, umbilical tissue from newborn babies is being collected and cryogenically frozen to be used one day for regenerative and stem cell medicine. And it doesn't just have potential for the babies involved, either. Experts say stem cells could also be used for family members who are genetically compatible.

It is hoped the cells will eventually be able to be used to repair damaged tissues and organs, with researchers investigating its uses for treating diseases like multiple sclerosis, cerebral palsy and diabetes, as well as for bone and cartilage repair.

Although cord blood storage has been available for many years, Cell Care Australia has added cord tissue storage in anticipation of new discoveries in the regenerative medicine field.

Cell Care Australia medical director associate professor Mark Kirkland said the storage process - already popular in the US, Europe and Southeast Asia - was long overdue for Australian shores.

"The science is developing around the world and we're really behind the rest of the world in providing parents the option to store these cells and we thought it was about time it was brought here," he said.

"It's finding a way to take what would otherwise be waste tissue and turning it into something of potential future value for not only your child but also potentially for other family members.'

Parramatta couple Tim and Padma Vellaichamy are among the first to use the service in Australia.

Mr Vellaichamy, 31, said he heard of the technology while working as a dentist in India and decided to store their daughter's cord cell tissue after birth three weeks ago.

Originally posted here:
Stem cell medicine thrown umbilical rope

Connie K. Ho for RedOrbit.com

A new study by researchers at the University of California, Los Angeles (UCLA) has discovered two adult stem cell-like subpopulations in adult human skin.

The findings allow for further research to be done in the area of personalized medicine and patient-specific cellular therapies.

The study, using technology from Fibrocell Science, allowed the researchers to identify and confirm two types of cells in human skin cell cultures; the possible source of stem cell-like subpopulations from skin biopsies would be faster to perform, painless, and less invasive than current extractions from adipose tissues and bone marrow.

The research, featured in the inaugural issue of BioResearch Open Access, discusses two subtypes of cells. BioResearch Open Access is a bimonthly, peer-reviewed journal. It features scientific topics like biochemistry, bioengineering, gene therapy, genetics, microbiology, neuroscience, regenerative medicine, stem cells, systems biology, tissue engineering and biomaterials, and virology.

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, commented Dr. James A. Bryne, lead author of the report.

Brynes research, first at Stanford University then at UCLA, focused on reprogramming beginnings of cells from animals and then humans. A graduate of Cambridge University, Bryne studied the intra- and inter-species of epigenetic reprogramming. His work also highlighted how primate embryonic stem cells could be derived from somatic cell nuclear transfers.

The study published in BioResearch Open Access confirmed previous research that identified a rare population of cells in adult human skin that had a marker called stage-specific embryonic antigen 3 (SSEA3). Bryne and his colleagues found that there was an increase in the amount of SSEA3 expressing cells after injury to the human skin. It showed that the SSEA3 biomarker could be used to help identify and isolate cells with tissue-regenerative traits.

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, remarked Bryne, an Assistant Professor of Molecular and Medical Pharmacology at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

Bryne and his team used Fibrocell technology to collect cells from skin samples, cultured the cells in the lab, and purified them by fluorescence-activated cell sorting (FACS). The FACS tagged suspended cells with fluorescent markers for undifferentiated stem cells. The researchers were able to separate the rare cell subpopulations from other kinds of cells.

Link:
Study Identifies Cell Subtypes For Potential Personalized Cellular Therapies

At its founding eight years ago, the Harvard Stem Cell Institute had fewer than ten principal faculty members, according to Benjamin D. Humphreys, co-director of the HSCI Kidney Program. Today, that number has ballooned to more than 80.

In the past decade, Harvard has increasingly poured resources into groundbreaking research in one of the largest collections of stem cell research labs in the country.

According to HSCI co-director Douglas A. Melton, a professor in the stem cell and regenerative biology department, there are more than 800 Harvard affiliates in stem cell science scattered throughout roughly 80 laboratories. The largest concentration of stem cell researchers are located in Harvards Sherman Fairchild Building, which reopened in August of 2011 after it underwent a two-year demolition and reconstruction project to accommodate the stem cell and regenerative biology department.

In the past decade, Harvard has focused on centralizing this research with the creation of HSCI and the stem cell and regenerative biology department.

HSCI consists of scientists and practitioners interested in stem cell research from all over the Harvard community, including the Faculty of Arts and Sciences, the medical school, and 11 teaching hospitals and research institutions including the Childrens Hospital Boston and the Massachusetts General Hospital.

So far, HSCI has given out more than $100 million to its researchers, according to Humphreys.

"[Harvard has] definitely made a tangible commitment to stem cell research," Humphreys said. "The results are that we are leaders in certain areascertainly I can speak of the kidneynot even just in the U.S., but worldwide in terms of stem cell research in the kidney."

With important potential applications such as the generation of cells and tissues that could be used for cell-based therapies, stem cells are at the forefront of scientific research. Stem cells, which can differentiate into specific cell types, offer the possibility of a renewable source of replacement cells and tissues to treat some of the most serious diseases.

"What were doing at the HSCI Kidney Group is working collaboratively to identify new therapeutic strategies that will help slow disease progression," said Humphreys.

Still, Humphreys added that much more research is necessary before scientists can use stem cells to their fullest potential.

More here:
Harvard Stem Cell Institute Sees Growth

CARLSBAD, Calif.--(BUSINESS WIRE)--

International Stem Cell Corporation (OTCBB: ISCO.OB - News) http://www.internationalstemcell.com today announced that the Company has developed new technologies to commercialize the use of human parthenogenetic stem cells (hpSC) to treat human diseases. The methods announced today are capable of producing populations of stem cells and their therapeutically valuable derivatives not only to a higher level of purity but also at a cost that is approximately several times lower than previously reported techniques.

ISCOs research team has developed a new method to derive high-purity populations of neural stem cells (NSC) from hpSC and further differentiate them into dopaminergic neurons. This method is capable of generating sufficient quantities of neuronal cells for ISCOs pre-clinical and clinical studies and is highly efficient as it requires substantially less time and labor in addition to using fewer costly materials than traditional methods. ISCOs technologies make possible the creation of billions of neuronal cells necessary for conducting such studies from a small batch of stem cells.

ISCO has also announced today that it has developed a new high-throughput cell culture method for growing human parthenogenetic stem cells (hpSC) in large quantities. This new technique is easily scalable and can produce the quantities of cGMP grade hpSC necessary for commercial and therapeutic applications.

One of the most challenging issues in commercializing stem cell based treatments is creating high-purity populations of stem cell derivatives at a reasonable cost. I believe the new methods we have developed solve this important problem and help position us for future clinical studies, says Dr. Ruslan Semechkin, Vice President, R&D.

About International Stem Cell Corporation

International Stem Cell Corporation is focused on the therapeutic applications of human parthenogenetic stem cells (hpSCs) and the development and commercialization of cell-based research and cosmetic products. ISCO's core technology, parthenogenesis, results in the creation of pluripotent human stem cells from unfertilized oocytes (eggs). hpSCs avoid ethical issues associated with the use or destruction of viable human embryos. ISCO scientists have created the first parthenogenic, homozygous stem cell line that can be a source of therapeutic cells for hundreds of millions of individuals of differing genders, ages and racial background with minimal immune rejection after transplantation. hpSCs offer the potential to create the first true stem cell bank, UniStemCell. ISCO also produces and markets specialized cells and growth media for therapeutic research worldwide through its subsidiary Lifeline Cell Technology, and stem cell-based skin care products through its subsidiary Lifeline Skin Care (www.lifelineskincare.com). More information is available at http://www.internationalstemcell.com or follow us on Twitter @intlstemcell.

To receive ongoing corporate communications, please click on the following link: http://www.b2i.us/irpass.asp?BzID=1468&to=ea&s=0.

Forward-looking Statements

Statements pertaining to anticipated developments, the potential benefits of research programs and new manufacturing technologies, and other opportunities for the company and its subsidiaries, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates,") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products and technologies regulatory approvals, need and ability to obtain future capital, application of capital resources among competing uses, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the company's business, particularly those mentioned in the cautionary statements found in the company's Securities and Exchange Commission filings. The company disclaims any intent or obligation to update forward-looking statements.

Read more from the original source:
International Stem Cell Corporation Announces New Stem Cell Manufacturing Technologies to Support its Therapeutic ...