Top Stories - Stem Cells

Dr. Robert Wechsler-Reya (left) and Dr. Evan Snyder at the grand opening of the Sanford Consortium for Regenerative Medicine. Both of these stem cell researchers will soon relocate their labs to the new "collaboratory." (Photo credit: Mark Dastrup)
Stem cell “Collaboratory”...

On November 29, scientific and community leaders gathered to celebrate the grand opening of the...

Dr. Evan Y. Snyder (right), with collaborator Dr. Seung U. Kim
A new stem cell enters the mix

The iPS cell approach to regenerative medicine is tantalizing because these cells could be derived...

Brandon Nelson, manager of Sanford-Burnham's Stem Cell Research Center, welcomes visitors.
Stem Cell Awareness Day at...

On October 5, we opened our La Jolla campus to the San Diego community in honor of Stem Cell...

Dr. Stuart Lipton
New stem cell techniques make...

Dr. Stuart Lipton, and colleagues, develop new methods to create neural stem cells.

You’re invited to “get real” for stem cell research

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Many people from all walks of life are excited to see where stem cell research will lead us. Will it one day allow us to cure paralysis, regenerate tissue damaged by heart disease, or replace beta cells in diabetics… or all of the above? You may not be a scientist conducting research in a lab like our Stem Cell Research Center, but now is your opportunity to use your unique talents to help pursue these dreams. That is exactly what Sanford-Burnham’s annual fundraiser, Bring It! invites participants to do.

Sanford-Burnham in San Diego will host its fourth annual Bring It! event, in collaboration with HeadNorth, at the Del Mar Fairgrounds Activity Center on April 27, 2012. This year’s theme, Get Real for Stem Cell Research, asks teams of enthusiastic supporters to showcase their knowledge of reality T.V. and thirst for fame to dominate the competition.

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.

Stem Cell Awareness Day at Sanford-Burnham

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On October 5, we opened our La Jolla campus to the San Diego community in honor of Stem Cell Awareness Day. Despite the rain and wind, a number of people from the San Diego Blood Bank, Juvenile Diabetes Research Foundation, the California Institute for Regenerative Medicine, local schools, and elsewhere dropped by to learn about stem cell research and the promise these special cells hold for discovering the root causes of disease, finding new treatments, and ultimately improving the human condition.

A visit from Congressman Duncan D. Hunter

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Sanford-Burnham often welcomes public figures or community leaders onto its campuses to share the work taking place at the Institute. Congressman Duncan D. Hunter, U.S. Representative for California’s 52nd congressional district, expressed his gratitude at having drug discovery illuminated for him during a recent visit to the Institute’s La Jolla campus.

During his tour, Congressman Hunter met with Brandon Nelson, manager of the Stem Cell Core, one of Sanford-Burnham’s valuable Shared Resources. Nelson presented some recent advances in stem cell biology, including how researchers are using induced pluripotent stem (iPS) cells to generate heart and nerve cells. With this tool, scientists are able to model diseases in a dish and test potential new medicines. Congressman Hunter even took a look at beating cardiomyocytes (heart cells) under a microscope.

From Diabetes to Pancreatic Cancer

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Dr. Pamela Itkin-Ansariis an expert on diabetes – especially type 1 diabetes, the kind that mostly affects children. Type 1 diabetes is caused by a person’s own immune system, as they attack the insulin-producing beta-cells in the pancreas.

But, as it happened, observations made while researching one area of biology can inform another. That’s how Dr. Itkin-Ansari and her team found themselves studying pancreatic cancer.

“During the course of our diabetes studies, we noted that a particular growth stimulus we were investigating pushed pancreatic duct cells into the cell cycle – essentially converting them from quiet, complacent cells to cells that divide and proliferate,” explains Dr. Itkin-Ansari, adjunct assistant professor in Sanford-Burnham’s Development and Aging Program. “And since duct cells are the precursors of pancreatic cancer, we knew immediately that this could have important implications for pancreatic cancer.”

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.”

New stem cell techniques make more neurons, faster

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Stroke, Alzheimer’s, Parkinson’s, ALS—these neurological conditions strip people of their abilities to think, move or both. One of the goals driving regenerative medicine research is to use stem cells to create neural cells to treat these and other neurodegenerative conditions. Two papers recently published in Proceedings of the National Academy of Sciences USA (PNAS) bring us measurably closer to this goal.In the first paper, Dr. Stuart Lipton, Professor and Director of the Del E. Web Center for Neuroscience, Aging and Stem Cell Research, collaborated with lead authors Dr. Sheng Ding of the Gladstone Institutes and Dr. Kang Zhang of the UC San Diego School of Medicine to create large quantities of self-renewing, neural stem cells from human embryonic stem cells. These cells can become many types of brain cells and showed no increased risk of forming tumors, a problem that has plagued other efforts.

This is a big deal. Stem cells have the enticing potential to heal injuries and treat disease, but it has been difficult to produce enough stable cells for clinical use. The ability to create large quantities of neural stem cells brings us a step closer to treatments.

Support Spinal Cord Injury Research

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by Paula Baldin

How can you help support spinal cord injury research?
Write a letter of support
for renewal of the Roman Reed Spinal Cord Injury Research Act.
Let your Representative know
that you support federal funding for stem cell research.

During a college football game in 1994, young NFL hopeful Roman Reed suffered a debilitating spinal cord injury that left him paralyzed. As a result of this tragedy, Roman and his father, Don C. Reed, created the Roman Reed Foundation to increase awareness of paralysis. The foundation supports studies of both the causes and potential therapies for neurological disorders, especially those aimed at mitigating spinal cord injury through regenerative medicine. The Reeds lobby tirelessly to promote and fund research in this field.

In 2000, the foundation’s work led to California’s Roman Reed Spinal Cord Injury Research Act, which directed $14.6 million in state funds towards spinal cord injury research. Over the past decade, these funds have grown to $63.8 million through donations and other sources, including the National Institutes of Health (NIH).

The Roman Reed Act is now up for renewal and needs your support.

On the road from stem cell to neuron

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Early in embryonic development, the neural crest – a transient group of stem cells – gives rise to parts of the nervous system and several other tissues. But what determines which cells become neurons and which become other cell types? It turns out a gene called SOX2acts as a stem cell gatekeeper – only cells expressing it have the potential to become neurons.The SOX2 gene encodes a transcription factor, a type of protein that switches other genes on or off. SOX2 is one of two key genes researchers use to generate induced pluripotent stem cells (iPSCs), which are capable of differentiating into all cell types for research and potential therapeutic applications.

In a paper published May 5 in the journal Cell Stem Cell, Drs. Alexey Terskikh, Flavio Cimadamore and colleagues show that SOX2 maintains the potential for neural crest stem cells to become neurons in the peripheral nervous system, where they interface with muscles and other organs. These results could help better inform therapies aimed at neurocristopathies, diseases caused by defects in the neural crest, which include microphthalmia and CHARGE syndrome.

According to Dr. Cimadamore, post-doctoral researcher and first author of the study, “Neural crest cells are notoriously difficult to study in humans because of their very early and transient nature – a woman is usually not even yet aware of her pregnancy when they start to migrate and differentiate. So here we took advantage of an embryonic stem cell-based model of human neural crest previously developed in our lab to get a better understanding of the molecular pathways that control the differentiation potential of such cells in humans.”

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.

Doing the Math

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Dr. Igor Katkov is a cryobiologist. (No, he isn’t trying to freeze human beings for future revival – that’s the pseudoscience cryonics, which Dr. Katkov and the overwhelming majority of his colleagues consider fraudulent.) He studies the effects of low temperatures on living things. More specifically, Dr. Katkov tries to find better ways to freeze cells for storage. This is especially important in our Stem Cell Research Center, where special stem cell lines must be carefully preserved. This is a lot harder than you might think.

“There are five main cellular factors to achieve optimal freezing,” Dr. Katkov explains. “These are the size of the cell, the surface area, the permeability of the membranes to water and solutes and the osmotically inactive volume of the cell (the non-water part of the cell).”

Personalized Medicine 101

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In 2003, the completion of the human genome project gave us an unprecedented amount of genetic information. From this, a new clinical concept is emerging: personalized medicine.Conventional medical care generalizes treatment to all patients with a particular disease. But since a disease is as individual as the person who has it, casting a wide therapeutic net has its limitations. For one, patients with a certain genetic makeup might not respond to a particular drug as well as patients with different genetics, or they might experience different side effects. As personalized medicine becomes a reality, it could rectify these less-than-ideal situations.

From the diagnostic point-of-view, personalized medicine is a shift from reactive to proactive. Based on a person’s health, genetic, and environmental profiles, doctors practicing personalized medicine could assess a patient’s risk for acquiring a genetic disease before any symptoms develop. This might allow them to target the specific genes that account for illness (the BRCA1/BRCA2 genes that predispose a woman to breast cancer, for example), incorporate a prevention strategy, and monitor those genes over time. When it comes to treatment, personalized drugs could be prescribed based on an individual’s molecular “build” and targeting treatment where it will do the most good and the least harm.

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.