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Shannon Layne, DVM and VCA Dunmore Animal Hospital Now Offer Stem Cell Therapy to Pet Patients in Pain

Posted: October 17, 2014 at 6:41 am

Dunmore, Pennsylvania (PRWEB) October 17, 2014

VCA Dunmore Animal Hospital is proud to announce the addition of Shannon Layne, DVM and her interest in stem cell therapy to their team. Credentialed in Regenerative Cell Therapy with Vet-Stem since January of 2011, Dr. Layne has proudly been treating pets with osteoarthritis and ligament injuries in north-east Pennsylvania with stem cell therapy for the last four years.

Dr. Layne graduated from North Carolina State University, College of Veterinary Medicine in 2010 and has taken a special interest in Regenerative Veterinary Medicine and stem cell therapy since. In contrast to widely used drug therapies for pain management, cell-based therapies (like stem cell therapy) can promote healing, reduce inflammation, and decrease pain. Dr Layne also offers traditional Chinese veterinary medicine including acupuncture and Chinese herbs if clients are interested in a more holistic approach.

Stem cells are regenerative cells that can differentiate into many tissue types (reducing pain and inflammation) thus helping to restore range of motion and regenerate tendon, ligament and joint tissues (Vet-Stem.com/science). In a study using Vet-Stem Regenerative Cell Therapy on dogs with osteoarthritis of the hip joint it was found that regenerative cell therapy (adipose-derived stem cells) decreases patient discomfort and increases patient functional ability.

Once Dr. Layne has identified a patient as a good candidate for stem cell therapy the procedure begins with a fatty tissue collection from the patient. The tissue sample is sent overnight to Vet-Stems lab in California for processing. Once processed the stem cells are extracted and fresh, injectable doses of the patients stem cells are sent overnight, back to Dr. Layne at VCA Dunmore Animal Hospital. Within 48hrs of collecting a fat sample from a patient Dr. Layne is able to inject stem cells into (arthritic or injured) affected areas and regeneration and healing can begin.

At VCA Dunmore Animal Hospital Dr. Layne will be practicing in an 8,800 square foot, state of the art facility that includes two extensive surgery suites. For more information on VCA Dunmore Animal Hospital please visit their website at http://www.vcahospitals.com/dunmore.

About Vet-Stem, Inc.

Since its formation in 2002, Vet-Stem, Inc. has endeavored to improve the lives of animals through regenerative medicine. As the first company in the United States to provide an adipose-derived stem cell service to veterinarians for their patients, Vet-Stem pioneered the use of regenerative stem cells for horses, dogs, cats, and some exotics. In 2004 the first horse was treated with Vet-Stem Regenerative Cell Therapy for a tendon injury that would normally have been career ending. Ten years later Vet-Stem celebrated its 10,000th animal treated, and the success of establishing stem cell therapy as a regenerative medicine for certain inflammatory, degenerative, and arthritic diseases. As animal advocates, veterinarians, veterinary technicians, and cell biologists, the team at Vet-Stem tasks themselves with the responsibility of discovering, refining, and bringing to market innovative medical therapies that utilize the bodys own healing and regenerative cells.

For more information about Vet-Stem and Regenerative Veterinary Medicine visit http://www.vet-stem.com or call 858-748-2004.

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Life Issues Institute – Successes of Adult Stem Cell …

Posted: October 16, 2014 at 9:58 pm

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Real-World Successes of Adult Stem Cell Treatments by: Mr. Bradley Richard Hughes Jr.

Summary: Adult stem cell treatments are beginning to show great promise in treating a wide range of illnesses. Yet the debates in the popular media tend to ignore and obscure the medical breakthroughs made by adult stem cell research-success that has conspicuously eluded embryonic stem cell treatments.

With increasing frequency, American citizens and others from around the globe are experiencing newfound freedom from disease, affliction, and infirmity. Individuals' lives are forever changed with the strengthened faith and renewed hope that arise from healed bodies and physical restoration. These seemingly miraculous cures are the result of adult stem cell treatments. Yet the debates in the popular media tend to ignore and obscure the medical breakthroughs made by adult stem cell research--success that has conspicuously eluded embryonic stem cell treatments.[1]

Adult stem cells (or, more accurately, tissue stem cells) are regenerative cells of the human body that possess the characteristic of plasticity--the ability to specialize and develop into other tissues of the body. Beginning in an unspecialized and undeveloped state, they can be coaxed to become heart tissue, neural matter, skin cells, and a host of other tissues. They are found in our own organs and tissues such as fat, bone marrow, umbilical cord blood, placentas, neuronal sources, and olfactory tissue, which resides in the upper nasal cavity.[2] This simple fact has remarkable implications for medicine--diseased or damaged tissue can become healthy and robust through the infusion of such cells. This has consequently commanded the attention of many researchers as well as those suffering from disease.

It is necessary to note that the power of adult stem cells is not nebulously potential, but tangible and real, as it has produced wonderful results in multiple cases. These have been documented in clinical trials, that is, treatments with human patients. With adult stem cells, physicians have successfully treated autoimmune diseases such as lupus, multiple sclerosis, Crohn's disease, and rheumatoid arthritis.[3] Furthermore, adult stem cells have helped to avert corneal degeneration and to restore vision in cases of blindness.[4] They have also restored proper cardiac function to heart attack sufferers[5] and improved movement in spinal cord injury patients.[6]

It is also important to note that all of these successes have come exclusively from adult stem cell research. Embryonic stem cell research, which requires the destruction of early human life to acquire the cells, has not produced any successes in human patients.[7] The breakthroughs demonstrated by adult stem cells are detailed below.

Spinal Cord Injuries

Spinal cord injuries are one of the most severe forms of debilitation known to humanity. Many times they result in different forms of paralysis, including paraplegia and quadriplegia; other times they involve the immediate or imminent death of the patient. Laura Dominguez is an example of the former. Living in San Antonio, Texas, she was a sixteen-year-old girl attending summer school in 2001. On her way back from class, she and her brother encountered an oil spill on the highway that caused their car to careen out of control. The accident left her paralyzed from the neck down with a C6 vertebrae burst fracture. She subsequently entered various hospitals to be emphatically informed that she would never walk again.[8]

After relocating to San Diego, California, Dominguez and her mother checked into a protracted physical therapy program. While there, they consulted with many spinal cord injury specialists and concluded that the most promising option existed in Portugal, where a cutting-edge procedure was being performed.

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Misfolded Proteins Clump Together in a Surprising Place

Posted: October 16, 2014 at 9:58 pm

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Newswise KANSAS CITY, MO Scientists at the Stowers Institute for Medical Research have made a surprising finding about the aggregates of misfolded cellular proteins that have been linked to aging-related disorders such as Parkinsons disease. The researchers report their results in the October 16, 2014 online issue of the journal Cell.

Using 3-D time-lapse movies to track the fate of misfolded proteins in yeast cells, the researchers determined that about 90 percent of aggregates form on the surface of the endoplasmic reticulum (ER), a location of protein synthesis in the cell. It had been thought that misfolded proteins spontaneously clump together in the cytosol, the fluid component of a cells interior.

Our findings have challenged the notion of the aggregation process as a passive consequence of accumulating misfolded proteins, says Stowers Investigator Rong Li, Ph.D., who led the study. Using budding yeast Saccharomyces cerevisae, a frequently used laboratory model in aging research, Stowers scientists experimentally used heat and other forms of stress to induce misfolded proteins to clump together.

Li and collaborators also found that the aggregation of misfolded proteins on the ER surface depends on the active synthesis of proteins by ribosomes. These molecular machines translate the cells recipes for proteins. Guided by the recipe, the ribosome generates a linear polypeptide chain, the initial form of a protein.

The newly synthesized polypeptide folds into a distinctive three-dimensional structure resulting in a protein with a functional shape. Proteins that fail to fold correctly cannot perform their biological functions and are potentially toxic to cells. Thus, the aggregation of misfolded or unfolded proteins may help protect the cell and prevent their transfer to daughter cells during cell division, said Chuankai Zhou, a predoctoral researcher in the Li lab and first author of the paper.

In addition to determining that protein aggregation is regulated and requires active translation, Stowers scientists revealed that the mitochondria, the cells powerhouses, play a key role in the mobility of these protein aggregates. We found the majority of aggregates on the surface of ER were in regions where ER and mitochondria come together, which is surprising but fits well with the view of regulated aggregation, says Zhou.

The current study builds upon previous research, published by the Li lab in 2011 in Cell, that revealed that most aggregates of unfolded proteins are retained by the mother yeast cell during the asymmetric cell division that characterizes this organism as well as stem cells. Budding yeast reproduce when only a small growth out of the mother yeast cell, a bud, becomes a daughter cell.

In the current paper, the scientists identified the quality control mechanism that limits the spreading of the misfolded protein aggregates to the bud and thereby the daughter cell. During the mitosis stage of budding yeasts division, aggregates of abnormal protein are tethered to well-anchored mitochondria in the mother cell. As a result, the mitochondria acquired by the bud are largely free of the abnormal aggregates.

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PROCHYMAL (Human Adult Stem Cells) for the Treatment of …

Posted: October 16, 2014 at 9:58 pm

Primary Outcome Measures: Safety [TimeFrame:Through 2 years] [Designatedassafetyissue:Yes] Secondary Outcome Measures: Pulmonary Function Tests [TimeFrame:Through 1 year] [Designatedassafetyissue:No] Exercise Capability [TimeFrame:Through 1 year] [Designatedassafetyissue:No] Quality of life [TimeFrame:Through 1 year] [Designatedassafetyissue:No]

COPD is currently the fourth leading cause of death in the United States. It is clear that there is a significant unmet medical need for safe and effective therapies to treat moderate to severe COPD. This patient population has a high mortality rate and requires frequent hospitalizations due to disease-related exacerbations. Based on severity distribution estimates, approximately 70% of all current COPD patients have either moderate or severe COPD. COPD has no known cure, thus current therapeutic intervention is aimed at providing relief of symptoms. Oxygen therapy is the only treatment that has been shown to improve survival. Smoking cessation has been shown to slow the rate of FEV1 decline and COPD progression. In general patients are treated with bronchodilators and inhaled corticosteroids, but again, these measures do not provide any significant benefit regarding disease progression or prognosis. The characteristics and biologic activity of PROCHYMAL, along with a good safety profile in human trials to date, suggest that PROCHYMAL may be a good candidate for addressing this unmet medical need.

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Stem Cells in India | Stem Cell Therapy in India | Quality …

Posted: October 16, 2014 at 9:58 pm

There are many serious and debilitating diseases that seem to have no easy treatment or any treatment at all. For several of this type of ailments, the advancements in stem cell therapy could be the answer people are looking for.Unfortunately, stem cell therapy remains controversial, and is yet to be offered in many places. And in most developed countries, the prices for the treatment are beyond the reach of many people. However, a cheaper, more affordable alternative exists in the form of stem cell treatment in India, giving more people a chance to get better.

Youve probably heard of stem cells one time or another its a hot topic when it comes to medicine, after all. But what are stem cells anyway? According the U.S. National Institutes of Health resource for stem cell research, stem cells are cells not specific to any bodily function, and have the capability to divide, and hence multiply even after long periods of inactivity.

Also, they can be induced to transform into cells for tissues or organs a function that makes them ideal for treatment of various diseases. This ability means the stem cells can heal injured human body parts the reason why stem cells have been called the holy grail of medicine before. Scientists have worked with two kinds of stem cells: embryonic stem cells and adult stem cells.

These stem cells are then cultured and grown in laboratories to be used for treatment later on. Stem cell treatment entails introduction of the stem cells into the body of a person. The nature of the cells enables them to divide and heal tissues or organs for treatment.

When it comes to procedures related to stem cells, India is one of the popular destinations. According to Forbes India, the countrys government, recognizing the importance of stem cells in medicine, allocated more than $66 million toward stem cell research in India. The research was geared toward understanding stem cells more, and conducting clinical trials to test a therapys effectiveness.

Like the other services offered by the countrys medical tourism industry, stem cell transplant in India is offered at a price a lot lower than the prices in developed countries. Besides the lower costs of stem cell transplant, India offers some treatments that may not be available in other locations.

There are many diseases and ailments that could possibly be cured by stem cells diseases which were previously thought to be incurable, leading to terminal illnesses that ultimately result in death. A common example would be leukemia. According to the University of Utahs Genetic Science Learning Center, doctors opt for bone marrow transplants whenever chemotherapy alone cannot eliminate all the abnormal leukocytes in a patient with leukemia.

Aside from cancer and leukemia, stem cell treatment in India also addresses various other diseases such as multiple sclerosis and several other degenerative disorders of the brain including metabolic disorders, Parkinsons disease, cerebral palsy, and cerebellar degeneration. Stem cell therapy in India commonly uses adult stem cells such as mesenchymal stem cells, which are cells that can turn into bone cells, cartilage cells, and fat cells.

Doctors from India could get these mesenchymal stem cells from a patients iliac crest, which is a part of the human pelvis. Extraction of these cells do not require full surgery a minimally invasive procedure the entails the usage of needles is utilized. Other options exist such as bone grafting and the usage of bone morphogenic protein, but the former is painful and the latter is extremely expensive. Mesenchymal stem cells used in India would therefore seem a more appealing and more financially feasible option.

Many Indian doctors doing stem cell therapy have extensive experience in the field, some having trained in the United States for years. These doctors have been doing the procedure for long stretches of time already. Besides the doctors themselves, there are Indian hospitals offering stem cell treatment that have NLAB-accredit laboratories. NLAB or National Laboratories Accreditation Bureau is an organization that recognizes the technical competence of laboratories. All procedures offered to the patients have been duly analyzed and tested on pre-clinical studies.

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Sangamo BioSciences ZFP Therapeutic Program in HIV/AIDS Featured at Three Major Scientific Conferences in October 2014

Posted: October 16, 2014 at 9:56 pm

RICHMOND, Calif., Oct. 16, 2014 /PRNewswire/ --Sangamo BioSciences, Inc. (NASDAQ: SGMO) announced that Company scientists and clinicians, as well as academic collaborators, were invited speakers at three major scientific meetings in Europe and the United States. Their presentations covered a number of therapeutic uses of Sangamo's novel zinc finger protein (ZFP) technology, but were primarily focused on reviewing the progress of the Company's ZFP Therapeutic program in HIV/AIDS. Presentations included a review of current clinical data with SB-728-T as well as the design of Sangamo's ongoing Phase 2 clinical trial (SB-728-mR-1401) and the preclinical rationale for targeting CCR5 in hematopoietic stem cells, which is expected to enter clinical testing in late 2014.

"Participation in these key scientific forumsthe NIH 'Strategies for an HIV Cure 2014' meeting, the European Society of Gene and Cell Therapy (ESGCT) Annual Meeting, and most recently, the British HIV Association (BHIVA) Autumn Conferenceunderscores the tremendous interest from the international scientific and clinical communities in Sangamo's progresstoward developing a ZFP Therapeutic that may replicate natural, durable resistance to HIV infection," said Edward Lanphier, Sangamo's president and CEO. "Sangamo's ongoing Phase 2 trial incorporates all that we have learned from previous trials about the potential mechanism of this novel therapeutic clinical approach, and we believe the data produced will provide a clear path to pivotal studies. We expect to accrue all subjects onto the clinical trial by the end of 2014 and to present data in 2015."

This week, October 15-17, at the "Strategies for an HIV Cure 2014" conference organized by the National Institute for Allergies and Infectious Diseases at the NIH, Philip Gregory, D.Phil., Sangamo's senior vice president, research, and CSO, will present an overview of the SB-728-T program, along with Sangamo collaborators Paula Cannon, Ph.D., Associate Professor Molecular Microbiology & Immunology, Pediatrics, Biochemistry & Molecular Biology, Keck School of Medicine, University of Southern California; Pablo Tebas, M.D., Professor of Medicine at the Hospital of the University of Pennsylvania; and Hans-Peter Kiem, M.D., Jose Carreras/E. Donnall Thomas Endowed Chair for Cancer Research at the Fred Hutchinson Cancer Research Center.

Dr. Gregory will also be presenting an overview of the SB-728-T clinical program at a "bench to bedside" discussion forum of the annual ESGCT meeting, October 23-26, which will beheld in The Hague. Dr. Gregory and Sangamo collaborator Luigi Naldini, M.D., Ph.D., Director, San Raffaele Telethon Institute for Gene Therapy (TIGET) will discuss the larger field of genome editing, utilizing the company's HIV studies as a model. Sangamo collaborators will also be discussing the use of Sangamo zinc finger nucleases in preclinical and research studies of SCID-X1, cancer and Wiskott-Aldrich Syndrome.

Earlier in October, Geoffrey Nichol, M.B., Ch.B., Sangamo's executive vice president, research and development, was invited to deliver the Foundation Lecture, reviewing recent clinical data from the SB-728-T program at the Autumn Conference of the British HIV Association which was held in London, UK.

Sangamo's SB-728-mR-1401 trial is an open-label, multi-center study designed primarily to evaluate safety and tolerability and the effect of repeat doses of SB-728-T following optimal cyclophosphamide (Cytoxan) pre-conditioning, on engraftment, viral load and total CD4 counts in peripheral blood. Electroporation of mRNA is being used to deliver the zinc finger nucleases to a subject's T-cellsto generate the modified autologous T-cell product (SB-728-T). mRNA delivery is more efficient than the previous adenoviral delivery method used and enables treatment of subjects with multiple doses of CCR5-modified cells. Up to nine subjects will be enrolled into two cohorts. Each subject will receive a total of up to 40 billion ZFN modified T-cells. The first cohort will receive this dose divided into infusions of two equal doses of SB-728mR-T 14 days apart after a cyclophosphamide (1 g/m2) preconditioning treatment two days prior to the first SB-728mR-T infusion, and the second cohort will receive three doses of cells. Dividing the total cell dose and administering sequentially in this manner is thought to maximize overall cell engraftment. Four weeks after the last SB-728-mR infusion, subjects with CD4 cell counts 500 cells/mm3 will undergo a 16 week treatment interruption (TI) during which time their anti-retroviral therapy will be discontinued.

About SB-728-TSangamo's therapy, SB-728-T, is generated by ZFN-mediated modification of the gene encoding CCR5 in a patient's own T-cells.ZFN modification disrupts the expression of this key co-receptor for HIV entry and renders cells resistant to HIV infection. The approach is based on the observation that a naturally occurring mutation in the CCR5 gene, CCR5 delta-32, provides protection from HIV infection. Individuals in whom both copies of the CCR5 gene carry the delta-32 mutation are generally not susceptible to the most common strain of HIV.

About SangamoSangamo BioSciences, Inc. is focused on Engineering Genetic Cures for monogenic and infectious diseases by deploying its novel DNA-binding protein technology platform in therapeutic gene regulation and genome editing. The Company has ongoing Phase 2 clinical trials to evaluate the safety and efficacy of a novel ZFP Therapeutic for the treatment of HIV/AIDS (SB-728-T) and NGF-AAV for Alzheimer's disease (CERE-110). Sangamo's other therapeutic programs are focused on monogenic and rare diseases. The Company has formed a strategic collaboration with Shire International GmbH to develop therapeutics for hemophilia, Huntington's disease and other monogenic diseases, and with Biogen Idec for hemoglobinopathies, such as sickle cell disease and beta-thalassemia. It has also 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.

This press release may contain forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation, references relating to research and development of novel ZFP TFs and ZFNs and therapeutic applications of Sangamo's ZFP technology platform for the treatment of HIV/AIDS, including a potential functional cure or control for HIV/AIDS, the ability of a ZFP Therapeutic to control HIV infection, the initiation of clinical studies and enrollment of aPhase 2 clinical trial for HIV/AIDS, projected timing of release of SB-728-T clinical data, clinical data from new HIV/AIDS trials to support pivotal studies, the efficiency of the mRNA delivery system, and the use of Sangamo's technology in potential preclinical and research studies. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to the initiation and completion of stages of our clinical trials, whether the clinical trials will validate and support the tolerability and efficacy of ZFNs, technological challenges, Sangamo's ability to develop commercially viable products and technological developments by our competitors. For a more detailed discussion of these and other risks, please see Sangamo's public filings with the Securities and Exchange Commissio
n, including the risk factors described in its Annual Report on Form 10-K and its most recent Quarterly Report on Form 10-Q. Sangamo assumes no obligation to update the forward-looking information contained in this press release.

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$25M gift prompts University pediatric hospital name change

Posted: October 16, 2014 at 9:54 pm

Doctors at the University of Minnesotas childrens hospital traded in their white coats for ones embroidered with a new name.

With its $25 million donation, the Minnesota Masonic Charities became the Universitys largest donor, and in honor of the gift, the campuss pediatric hospital was renamed Tuesday as the University of Minnesota Masonic Childrens Hospital.

The donation will primarily go toward finding cures and treatments for childhood diseases, said Eric Neetenbeek, Minnesota Masonic Charities president and CEO.

The gift will specifically enhance patient and family experiences, as well as advance pediatric research on neurobehavioral development, rare and infectious disease, and stem cell therapy.

Dr. Joseph Neglia, the hospitals physician-in-chief, said he hopes the gift will create stronger relationships with pediatrics researchers across the University.

Really, to build new bridges is one big part of what Id like us to do, Neglia said. These gifts are vitally important for the hospital.

With the donation, Neglia said he also hopes to expand the hospitals existing research, like its work on correcting genetic defects in human cells and its pediatric medicine international programs in Kenya and Uganda.

Neetenbeek said the Masons donations, which total $125 million over the last 60 years, have been essential at a time when new health care research struggles to receive competitive funding from larger organizations like the National Institutes of Health.

There arent too many venture capitalists willing to go with untried businesses [and] ideas, he said. The same is true when youre looking at research into health care problems.

In the near future, the Masons will meet physicians at the childrens hospital to discuss which promising research projects to allocate the money toward, Neetenbeek said.

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New front in war on Alzheimers, other protein-folding diseases

Posted: October 16, 2014 at 9:54 pm

BERKELEY

A surprise discovery that overturns decades of thinking about how the body fixes proteins that come unraveled greatly expands opportunities for therapies to prevent diseases such as Alzheimers and Parkinsons, which have been linked to the accumulation of improperly folded proteins in the brain.

This finding provides a whole other outlook on protein-folding diseases; a new way to go after them, said Andrew Dillin, the Thomas and Stacey Siebel Distinguished Chair of Stem Cell Research in the Department of Molecular and Cell Biology and Howard Hughes Medical Institute investigator at the University of California, Berkeley.

Dillin, UC Berkeley postdoctoral fellows Nathan A. Baird and Peter M. Douglas and their colleagues at the University of Michigan, The Scripps Research Institute and Genentech Inc., will publish their results in the Oct. 17 issue of the journal Science.

Cells put a lot of effort into preventing proteins which are like a string of beads arranged in a precise three-dimensional shape from unraveling, since a proteins activity as an enzyme or structural component depends on being properly shaped and folded. There are at least 350 separate molecular chaperones constantly patrolling the cell to refold misfolded proteins. Heat is one of the major threats to proteins, as can be demonstrated when frying an egg the clear white albumen turns opaque as the proteins unfold and then tangle like spaghetti.

Heat shock

For 35 years, researchers have worked under the assumption that when cells undergo heat shock, as with a fever, they produce a protein that triggers a cascade of events that field even more chaperones to refold unraveling proteins that could kill the cell. The protein, HSF-1 (heat shock factor-1), does this by binding to promoters upstream of the 350-plus chaperone genes, upping the genes activity and launching the army of chaperones, which originally were called heat shock proteins.

Injecting animals with HSF-1 has been shown not only to increase their tolerance of heat stress, but to increase lifespan.

Because an accumulation of misfolded proteins has been implicated in aging and in neurodegenerative diseases such as Alzheimers, Parkinsons and Huntingtons diseases, scientists have sought ways to artificially boost HSF-1 in order to reduce the protein plaques and tangles that eventually kill brain cells. To date, such boosters have extended lifespan in lab animals, including mice, but greatly increased the incidence of cancer.

Dillins team found in experiments on the nematode worm C. elegans that HSF-1 does a whole lot more than trigger release of chaperones. An equal if not more important function is to stabilize the cells cytoskeleton, which is the highway that transports essential supplies healing chaperones included around the cell.

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New front in war on Alzheimer's, other protein-linked brain diseases

Posted: October 16, 2014 at 9:54 pm

A surprise discovery that overturns decades of thinking about how the body fixes proteins that come unraveled greatly expands opportunities for therapies to prevent diseases such as Alzheimer's and Parkinson's, which have been linked to the accumulation of improperly folded proteins in the brain.

"This finding provides a whole other outlook on protein-folding diseases; a new way to go after them," said Andrew Dillin, the Thomas and Stacey Siebel Distinguished Chair of Stem Cell Research in the Department of Molecular and Cell Biology and Howard Hughes Medical Institute investigator at the University of California, Berkeley.

Dillin, UC Berkeley postdoctoral fellows Nathan A. Baird and Peter M. Douglas and their colleagues at the University of Michigan, The Scripps Research Institute and Genentech Inc., will publish their results in the Oct. 17 issue of the journal Science.

Cells put a lot of effort into preventing proteins -- which are like a string of beads arranged in a precise three-dimensional shape -- from unraveling, since a protein's activity as an enzyme or structural component depends on being properly shaped and folded. There are at least 350 separate molecular chaperones constantly patrolling the cell to refold misfolded proteins. Heat is one of the major threats to proteins, as can be demonstrated when frying an egg -- the clear white albumen turns opaque as the proteins unfold and then tangle like spaghetti.

For 35 years, researchers have worked under the assumption that when cells undergo heat shock, as with a fever, they produce a protein that triggers a cascade of events that field even more chaperones to refold unraveling proteins that could kill the cell. The protein, HSF-1 (heat shock factor-1), does this by binding to promoters upstream of the 350-plus chaperone genes, upping the genes' activity and launching the army of chaperones, which originally were called "heat shock proteins."

Injecting animals with HSF-1 has been shown not only to increase their tolerance of heat stress, but to increase lifespan.

Because an accumulation of misfolded proteins has been implicated in aging and in neurodegenerative diseases such as Alzheimer's, Parkinson's and Huntington's diseases, scientists have sought ways to artificially boost HSF-1 in order to reduce the protein plaques and tangles that eventually kill brain cells. To date, such boosters have extended lifespan in lab animals, including mice, but greatly increased the incidence of cancer.

Dillin's team found in experiments on the nematode worm C. elegans that HSF-1 does a whole lot more than trigger release of chaperones. An equal if not more important function is to stabilize the cell's cytoskeleton, which is the highway that transports essential supplies -- healing chaperones included -- around the cell.

"We are suggesting that, rather than making more of HSF-1 to prevent diseases like Huntington's, we should be looking for ways to make the actin cytoskeleton better," Dillin said. Such tactics might avoid the carcinogenic side effects of upping HSF-1.

Dillin is codirector of the Paul F. Glenn Center for Aging Research, a new collaboration between UC Berkeley and UC San Francisco supported by the Glenn Foundation for Medical Research. Center investigators will study the many ways that proteins malfunction within cells, ideally paving the way for novel treatments for neurodegenerative diseases.

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Stem Cell Eye Treatment May Restore Vision

Posted: October 16, 2014 at 9:53 pm

Antonio Regalado for MIT Technology Review 2014-10-15 19:15:44 UTC

When stem cells were first culled from human embryos sixteen years ago, scientists imagined they would soon be treating diabetes, heart disease, stroke, and many other diseases with cells manufactured in the lab.

It's all taken longer than they thought. But now, a Massachusetts biotech firm has reported results from the largest, and longest, human test of a treatment based on embryonic stem cells, saying it appears safe and may have partly restored vision to patients going blind from degenerative diseases.

Results of three-year study were described Tuesday in the Lancet by Advanced Cell Technology and collaborating eye specialists at the Jules Stein Eye Institute in Los Angeles who transplanted lab-grown cells into the eyes of nine people with macular degeneration and nine with Stargardt's macular dystrophy.

The idea behind Advanced Cell's treatment is to replace retinal pigment epithelium cells, known as RPE cells, a type of caretaker tissue without which a person's photoreceptors also die, with supplies grown in laboratory. It uses embryonic stem cells as a starting point, coaxing them to generate millions of specialized retina cells. In the study, each patient received a transplant of between 50,000 and 150,000 of those cells into one eye.

The main objective of the study was to prove the cells were safe. Beyond seeing no worrisome side effects, the researchers also noted some improvements in the patients. According to the researchers half of them improved enough to read two to three extra lines on an eye exam chart, results Robert Lanza, chief scientific officer of Advanced Cell, called remarkable.

"We have people saying things no one would make up, like 'Oh I can see the pattern on my furniture, or now I drive to the airport," he says. "Clearly there is something going on here."

Lanza stressed the need for a larger study, which he said the company hoped to launch later this year in Stargardt's patients. But if the vision results seen so far continue, Lanza says "this would be a therapy."

Some eye specialists said it's too soon to say whether the vision improvements were real. The patients weren't examined by independent specialists, they said, and eyesight in patients with low vision is notoriously difficult to measure. That leaves plenty of room for placebo effects or unconscious bias on the part of doctors.

"When someone gets a treatment, they try really hard to read the eye chart," says Stephen Tsang, a doctor at Columbia University who sees patients losing their vision to both diseases. It's common for patients to show quick improvements, he says, although typically not as large as what Advanced Cell is reporting.

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