Stem cell “Collaboratory” opens in La Jolla

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“Patient advocates: this is our day!” Lorraine Stiehl shouted, rallying the crowed assembled on November 29 to witness the grand opening of the Sanford Consortium for Regenerative Medicine, a new 150,000 square-foot, state-of-the-art research facility located in the Torrey Pines Mesa life science research cluster in La Jolla, a northern coastal area of San Diego, Calif.

Ms. Stiehl is a patient advocate coordinator for the California Institute for Regenerative Medicine (CIRM), the $3 billion stem cell agency created after California voters approved ballot measure Prop 71 in 2004. CIRM, and patient advocates like Ms. Stiehl, have played a huge role in bringing the Sanford Consortium to fruition. CIRM contributed $43 million to the project and patients are the reason that the consortium’s scientists are doing what they do—working to advance our understanding of stem cell biology and ultimately find new treatments for Alzheimer’s disease, diabetes, and many other conditions.

“You see 150,00 square feet of new research space,” Ms. Stiehl continued. “We see 150,000 square feet of hope, 150,000 square feet of empowerment.”

Dedicated to Christopher Reeve: Hot Topics in Stem Cell Biology 2011

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Some of the world’s premier stem cell researchers will engage in a spirited discussion of what’s happening right now in stem cell research on Monday, November 14 at 6:30 p.m. at the Washington, D.C. Convention Center. Sponsored by EMD Millipore, the 8th Annual Christopher Reeve Hot Topics in Stem Cell Biology gathering will be held in conjunction with Neuroscience 2011, the Society for Neuroscience’s annual meeting. Throughout the evening, each researcher will highlight a single research topic followed by a brief discussion. This unique, rapid-fire forum moves beyond the scientific stump speech to showcase the state-of-the-art in stem cell research.

Developments to Watch: New frontier in Alzheimer’s disease

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Medscape, a physician-oriented website run by WebMD, visited Sanford-Burnham’s La Jolla campus this summer to record interviews with researchers from both Orlando and San Diego for a new online video program called Developments to Watch. The talk show-like discussions are hosted by Dr. Evan Snyder, who directs the Stem Cells and Regenerative Biology Program at Sanford-Burnham. The first episode, A New Frontier in Alzheimer’s Disease, is now available. In the video, Dr. Snyder speaks with Dr. Stuart Lipton, director of the Del E. Web Neuroscience, Aging and Stem Cell Research Center, about his work on Alzheimer’s disease. They discuss what new findings—and potential treatments—are on the horizon and how they might impact patients.

A user name and password are required to access Medscape, but the site and content are free. New installments will be added monthly.

Watch the video, then come back here to let us know what you think!

For more about our research on Alzheimer’s disease, check out these blog posts:
Getting to the root of Alzheimer’s disease
Diagnosing Alzheimer’s Earlier
New Partnership Targets Brain Conditions
Safely Treating Alzheimer’s Disease
Saying NO to Alzheimer’s and Parkinson’s Diseases

Coming soon: Medscape’s “Developments to Watch”

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Last week, Sanford-Burnham’s Fishman Auditorium, on the Institute’s La Jolla campus, was transformed into a temporary television studio. It was hardly recognizable under the bright lights and set dressing. Medical website Medscape recorded interviews with three Sanford-Burnham researchers for a new video series called “Developments to Watch.” The talk show-like discussions were hosted by Dr. Evan Snyder, who directs the Stem Cells and Regenerative Biology Program at Sanford-Burnham. Dr. Snyder is both a medical doctor who regularly sees patients and a scientist who conducts research in his own lab – the perfect person to help explain how discoveries made today might one day help patients.

Medscape is part of the network of sites run by WebMD. With this newest video series, Sanford-Burnham scientists will be providing expert commentary and information to help keep Medscape’s audience – primary care physicians, specialists and other health professionals – up-to-date on the latest medical research and what it means for their patients.

Crunching the Proteome

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Every day we gain a better understanding of how cells work. In the past 20 years, new tools to examine gene expression and function have illuminated many different mechanisms that guide all aspects of cellular behavior. However, to fully understand normal cellular functions and how they malfunction in disease, we need more in-depth information about the many proteins our genes produce. Which proteins are being produced? How are they modified? What is each protein’s ultimate function and how do they interact on a system-wide level? New technologies in the proteomics facility at Sanford-Burnham are providing reams of data that could help answer these and many other questions.In a room full of advanced technology, the Thermo LTQ-Orbitrap Velos mass spectrometer system stands apart. The system has been part of the proteomics toolbox for about a year and has proven its value identifying proteins several times over. Dr. Laurence Brill, director of Advanced Proteomics in Sanford-Burnham’s Proteomics Facility, notes that the Velos system is 10 times more sensitive and three times faster than previous machines, but there’s a lot more to the core’s success than the excellent equipment. “We use very stringently applied analytical methods that take years to develop and refine,” says Dr. Brill. “We are thinking very carefully about the goals and biology of each assay and making them reproducible from run to run.”

Bring It! For Stem Cell Research

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Stem cell therapy holds promise for many different areas of medicine. But, as Dr. Evan Y. Snyder, director of Sanford-Burnham’s program in stem cells and regenerative biology, told the rapt Bring It!audience on April 21, regeneration of damaged spinal cord tissue is one of the most exciting stem cell applications. For many of those in attendance, the hope for a spinal cord injury treatment holds a distinctly personal significance – they, or someone they love, have been impacted by such an injury.Bring It! is a game show-themed fundraising experience now in its third year in San Diego. This year, Sanford-Burnham again partnered with HeadNorth, an organization that supports spinal cord injury patients. Life Technologies, leading supplier of stem cell research products to labs around the world, was the presenting sponsor.

But the Bring It! audience didn’t focus on tragedy. Their passion for stem cell research brought them there to play games and raise money. The fundraiser’s theme, “Rock on for Stem Cell Research,” gave participants the chance to live out their rock star fantasies, while helping stem cell treatments become a reality.

Tracking stem cells by MRI

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Neonatal stroke or related brain injuries can occur when a newborn baby’s blood supply is restricted, often leading to cerebral palsy, epilepsy or mental retardation. Over the past decade, researchers have been using animal models to experiment with neural stem cells to replace or protect the damaged tissue in this type of surprisingly common brain injury. Stem cells hold great therapeutic promise because they can proliferate in a dish (making many cells for transplantation purposes) and then differentiate on command, specializing into a specific cell type like neurons in the brain or even glial cells, which support and protect neurons. Stem cells are also pathotropic, meaning that they are drawn to, or home in on, pathological locations in the brain, including those that can occur from injury (like stroke) or degeneration (such as occurs in Alzheimer’s disease).

But there are risks to stem cell therapy. One worry is that cells will continue proliferating after transplantation, leading to tumor formation. Scientists also need to make sure the stem cells migrate directly to the locations in need of repair or protection and not to unintended locations. These are tough problems to overcome, though, because it’s difficult to track a stem cell’s behavior once it’s inside a host.

“The ability to monitor neural stem cells for a long time is particularly important for newborns, where implantation could cause unanticipated effects in the developing brain long into the future,” says Dr. Evan Y. Snyder, director of Sanford-Burnham’s Stem Cells and Regenerative Biology Program. Dr. Snyder was also the first to demonstrate pathotropism of solid-organ stem cells, as well as the first to demonstrate the use of stem cells to treat stroke, particularly neonatal stroke.

On the Cutting Edge

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On April 12, Dr. Evan Snyder, who directs the Stem Cells and Regenerative Biology program at Sanford-Burnham, was interviewed by Shally Zomorodi of Fox 5 News about recent advances in stem cell research. Dr. Snyder singled out four different areas where researchers are making great progress: diseases in a dish; using stem cells to protect other cells; recreating organs for transplant and using stem cells to treat diseased tissues or cancers (particularly in the brain) with targeted gene therapy. Dr. Snyder noted that all these approaches are fairly advanced.

A word of caution on mesenchymal stem cells

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Stem cells of many varieties hold a lot of promise for regenerative medicine. Their ability to continually self-replicate (produce more stem cells) and differentiate (specialize) into any number of cell types make them an enticing replacement for diseased or damaged tissue or as delivery vehicles for therapeutic molecules.The problem is that we still don’t know enough about many of the existing stem cell types to predict exactly how they will behave when transplanted into a patient. Each of the different types of stem cells has its unique repertoire of behaviors and its own benefits and drawbacks. In an editorial appearing online March 25 in the journal Experimental Neurology, Dr. Evan Y. Snyder hammers home the possible dangers of one very popular and oft-used type of stem cell. He highlights a paper appearing in the same issue of that journal, in which researchers from the Aristotle University of Thessaloniki in Greece and the Medical University of Vienna show that brain tumors develop when a mouse model of multiple sclerosis (MS) is transplanted with mesenchymal stem cells (MSCs) derived from bone marrow.

Stem cells reach the crest

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The neural crest is a versatile population of stem cells found in a developing embryo. In humans, neural crest arises during the third to fourth weeks of pregnancy, and then the cells specialize into a diverse set of cells, including certain types of nerves, skin, bone and muscle. Scientists have long appreciated this crucial event in development – when it goes wrong, a number of skeletal and nervous system disorders can result. But they haven’t really been able to study it properly in the laboratory. That’s because of the transient nature of the neural crest – it typically only exists for about two weeks in humans (with few exceptions). After that, the cells have migrated away and differentiated into other tissue types. Dr. Alexey Terskikh (along with Dr. Marianne Bronner-Fraser at the California Institute of Technology, Sanford-Burnham’s Dr. Evan Y. Snyder, postdoctoral researchers Dr. Carol Curchoe and Dr. Jochen Maurer and others) recently discovered a way to overcome this problem. In a study published recently in the journal PLoS ONE, they developed a new protocol for generating early migratory neural crest cells from human stem cells.

“This new system allows us to dissect what happens during human development – something that is not accessible in any other way,” says Dr. Terskikh, associate professor in Sanford-Burnham’s Development and Aging Program.

Bring It! with Sanford-Burnham and HeadNorth

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On April 21, Sanford-Burnham will partner with the HeadNorth Foundation for the third time to present Bring It!, a game show-style event that challenges teams to compete in a wide range of challenges. This year’s theme, “Rock on for Stem Cell Research” promises a full evening of networking and fun for a great cause, held at the Del Mar Fairgrounds Activity Center. HeadNorth is a San Diego-based nonprofit dedicated to providing help and hope for spinal cord injury survivors. It was founded in 2006 by Eric Northbrook after a motorcycle accident severed his spinal cord.

A new stem cell enters the mix

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Stem cells have the unique ability to self-renew (make more stem cells) and differentiate (specialize into a number of different cell types). There are three main types of stem cells already on the scene: embryonic stem cells, adult stem cells and induced pluripotent stem (iPS) cells. iPS cells are engineered by reprogramming fully differentiated adult cells, often skin cells, back to a primitive state. Like their embryonic cousins, iPS cells can form all cell types. Researchers are currently working to harness the flexibility of stem cells to replace damaged tissue and treat conditions like diabetes and heart disease.

The iPS cell approach to regenerative medicine is tantalizing because these cells could be derived from a patient’s own cells and are therefore less likely to face immune rejection. In the past few weeks, however, a slew of papers have indicated that the therapeutic potential of iPS cells might be limited by reprogramming errors and genomic instability. Given these problems, researchers from Sanford-Burnham, Chung-Ang University in Korea, University of British Columbia, Harvard Medical School and elsewhere wondered if there might be a better way to regenerate lost tissue to treat conditions like heart disease and stroke. Writing March 4 in the Proceedings of the National Academy of Sciences, they outline a method to obtain a new kind of stem cell they call induced conditional self-renewing progenitor (ICSP) cells.

What is “Disease in a Dish?”

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“Disease in a dish” is a cutting-edge, stem cell-based strategy that allows researchers to study an individual patient’s cells in a laboratory dish. Traditionally, scientists interested in a particular disease have used a standard cell line that has been grown in the lab for years or a mouse model (if one exists) that has been engineered to mimic the disease. Although extremely valuable, these techniques have obvious limitations. Animal models never entirely reflect the actual human condition – they don’t capture the complicated interplay between an individual patient’s genetics and the environmental factors that might influence the development of the disease or that patient’s response to a new therapy.

Read below to find out how diseases in a dish are made, how they’re being used to study and treat disease and how Sanford-Burnham researchers are applying the technique.

Stem Cells and Brain Tumors

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As a young scientist in the 1990s, Dr. Evan Snyder, now director of Sanford-Burnham’s Stem Cells and Regenerative Biology Program, had never worked on cancer. But when his close friend Dr. James Galambos died from a glioblastoma, he promised the family he would do everything he could to find a cure. In 2000, Dr. Snyder, Dr. Karen Aboody, now at City of Hope, and colleagues published a paper that described how stem cells could be used to treat cancer. A recent article on the California Institute for Regenerative Medicine(CIRM) website highlighted this breakthrough:

Neural stem cells, it revealed, are attracted to tumors like moths to a light. If they could be made to carry a chemotherapeutic payload, they could serve as weapons against the wanton spread of metastatic brain cancer.

CIRM committed $37 million to successfully move this promising research into clinical trials. To learn more about how stem cells can be used to target tumors, read Manipulated Medicine.

The City of Hope trial has also been featured on CBS News.

Stem Cell Blitz

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Some of the world’s premier stem cell scientists met recently in San Diego at Neuroscience 2010, the Society for Neuroscience’s annual meeting, to discuss the most current stem cell research. The 7th Annual Christopher Reeve Hot Topics in Stem Cell Biology, organized by Dr. Evan Snyder, director of the Stem Cells and Regenerative Biology Program at Sanford-Burnham, featured a series of 15-minute “data blitzes,” during which junior researchers highlighted a single aspect of their lab’s stem cell research. More than 1,000 people attended and the roster read like a who’s who of stem cell research: Dr. Fred “Rusty” Gage, chair for Research on Age-Related Neurodegenerative Diseases at the Salk Institute for Biological Studies; Dr. Ole Isacson, director of the Center for Neuroregeneration Research at Harvard Medical School; Dr. Arnold Kriegstein, director of the Stem Cell Biology Program at UC San Francisco and many more.