Check out the world of medical research! Sanford-Burnham invites you to a behind-the-scenes tour of our La Jolla campus on Wednesday, June 5, 2013. Tours are free of charge and include an overview of the Institute’s history, followed by a visit to our Stem Cell Research Center and the Conrad Prebys Center for Chemical Genomics. Another highlight of the tour is a look at our ultra-high-throughput/high-content screening facility’s robotic system. Learn how the robotic platform screens chemical compounds by the millions to find the few that could potentially be developed into the medicines of tomorrow.
Watch scientists in our Stem Cell Research Center during an Institute tour on June 5
The winning team, from left to right: Brandon Heess, Kim Renna, Michele Bart, Christina McCabe, Nicole Lomitola, Ryan Hiller and Paul Jacobson in front.
On April 25, Sanford-Burnham hosted its fifth annual Bring It! event, the Institute’s spring fundraiser to benefit stem cell research. More than 200 guests attended the camp-themed affair which took place at the Del Mar Fairgrounds.
Scientific research isn’t always easy to explain—or to understand. Whichever side of the conversation you’re on, you might feel a communication gap. The California Institute for Regenerative Medicine (CIRM) , the state’s stem cell agency, recently set out to bridge that gap with a #SciencePitch Challenge. Their goal was to encourage stem cell researchers to develop their “elevator pitch” — the short overview of their work that they’d give if a fellow elevator passenger asked them what they do and they only had a short ride in which to explain it. In the process, the participants are also demonstrating the importance of stem cell research and generating excitement about their work.
Skeletal myospheres ("mini muscles") generated by adding MyoD and BAF60C to embryonic stem cells
To make “mini muscles” from stem cells, you need the protein BAF60C.
Pier Lorenzo Puri, Ph.D., and his team study what makes a muscle cell just that—a muscle cell. They’re especially interested in applying that information to regenerate new muscle for people with muscular dystrophy.
Last year, the team discovered that two proteins called MyoD and BAF60C work together to mark the DNA of precursor cells, setting them on a course to become muscle cells. When the MyoD/BAF60c complex receives the right signals, it unwinds the cell’s genome and begins the process of producing muscle-specific proteins. This chain of events eventually triggers these precursor cells—those that hang out in our normal muscle tissue—to mature into new muscle cells.
What’s your favorite memory of summer camp? Is it a great friend you made or a game you mastered? We’re giving you a chance to relive those fun, youthful memories with a grown-up purpose: raising money for stem cell research.
You’re invited to our annual Bring It! event at the Del Mar Fairgrounds Activity Center in Del Mar, Calif., on April 25. Former San Diego mayor Jerry Sanders will co-chair with founding chairs Stath and Terry Karras. This year’s theme, Camp Bring It!, will challenge guests with a variety of camp-themed games.
In this study, researchers used an ARVD/C patient's skin cells to make induced pluripotent stem cells. Then they used those stem cells to generate ARVD/C patient-specific heart cells (shown here in green). These heart cells provide a valuable “disease in a dish” model that can be used to study ARVD/C and test new treatments.
Most patients with an inherited heart condition known as arrhythmogenic right ventricular dysplasia/cardiomyopathy (ARVD/C) don’t know they have a problem until they’re in their early 20s. The lack of symptoms at younger ages makes it very difficult for researchers to study how ARVD/C evolves or to develop treatments.
A new stem cell-based technology created by 2012 Nobel Prize winner Shinya Yamanaka, M.D., Ph.D., helps solve this problem. With this technology, researchers can generate heart muscle cells from a patient’s own skin cells. However, these newly made heart cells are mostly immature. That raises questions about whether or not they can be used to mimic a disease that occurs in adulthood.
In a paper published January 27 in Nature, researchers unveil the first maturation-based “disease in a dish” model for ARVD/C. The model was created using Yamanaka’s technology and a new method to mimic maturity by making the cells’ metabolism more like that in adult hearts. For that reason, this model is likely more relevant to human ARVD/C than other models and therefore better suited for studying the disease and testing new treatments.
Sanford-Burnham's Stem Cell Research Center provides resources and expertise to the entire scientific community. They are also building the world's largest collection of human induced pluripotent stem cells (iPSCs).
New collaboration combines Sanford-Burnham’s renowned scientific team and Intrexon’s proprietary discovery platforms to accelerate human induced pluripotent stem cell (iPSC) research
Today, we announced a new collaboration with Intrexon Corporation, a leading synthetic biology company, aimed at accelerating stem cell research. Under the agreement, Sanford-Burnham will gain access to sophisticated proprietary cellular selection and gene regulation technologies that are not currently on the market, including Intrexon’s Laser-Enabled Analysis and Processing (LEAP™) instrument and RheoSwitch Therapeutic System® (RTS®). As part of the agreement, Intrexon may obtain commercial and intellectual property rights resulting from technological advances made under the collaboration.
“I’m looking forward to merging and melding our expertise,” said Evan Y. Snyder, M.D., Ph.D., professor and director of Sanford-Burnham’s Stem Cell Research Center and Stem Cell and Regenerative Biology Program. “We’ll bring our iPSC and gene therapy expertise to the table. Likewise, our colleagues at Intrexon will share their knowledge of how best to use the technologies. We envision we’ll be meeting with them frequently and sharing insights to further advance the platforms for stem cell applications.”
Sanford-Burnham is currently building the world’s largest collection of human iPSCs generated from individual patients and healthy volunteers. The Stem Cell Research Center’s expertise and resources are available to all Sanford-Burnham scientists, as well as other researchers at nonprofit and for-profit research organizations around the world.
Stuart A. Lipton, M.D., Ph.D., director of Sanford-Burnham’s Del E. Webb Center for Neuroscience, Aging, and Stem Cell Research and a clinical neurologist
Neurons made from stem cells drive brain activity after transplantation in laboratory model
Originally published November 15, 2012
Researchers and patients look forward to the day when stem cells might be used to replace dying brain cells in Alzheimer’s disease and other neurodegenerative conditions. Scientists are currently able to make neurons and other brain cells from stem cells, but getting these neurons to properly function when transplanted to the host has proven to be more difficult. Now, researchers at Sanford-Burnham Medical Research Institute have found a way to stimulate stem cell-derived neurons to direct cognitive function after transplantation to an existing neural network. The study was published November 7 in the Journal of Neuroscience.
A consortium of researchers around the U.S. used transplanted neural stem cells (shown here) to treat a mouse model of ALS.
In 11 independent studies, a consortium of ALS researchers shows that transplanting neural stem cells into the spinal cord of an ALS mouse model slows disease onset and progression, improves motor function, and significantly prolongs survival.
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is untreatable and fatal. Nerve cells in the spinal cord die, eventually taking away a person’s ability to move or even breathe. A consortium of ALS researchers at multiple institutions, including Sanford-Burnham Medical Research Institute, Brigham and Women’s Hospital, and the University of Massachusetts Medical School, tested transplanted neural stem cells as a treatment for the disease. In 11 independent studies, they found that transplanting neural stem cells into the spinal cord of a mouse model of ALS slows disease onset and progression. This treatment also improves host motor function and significantly prolongs survival.
Surprisingly, the transplanted neural stem cells did not benefit ALS mice by replacing deteriorating nerve cells. Instead, neural stem cells help by producing factors that preserve the health and function of the host’s remaining nerve cells. They also reduce inflammation and suppress the number of disease-causing cells in the host’s spinal cord. These findings, published December 19 in Science Translational Medicine, demonstrate the potential neural stem cells hold for treating ALS and other nervous system disorders.
“While not a cure for human ALS, we believe that the careful transplantation of neural stem cells, particularly into areas that can best sustain life—respiratory control centers, for example—may be ready for clinical trials,” Evan Y. Snyder, M.D., Ph.D., director of Sanford-Burnham’s Stem Cell and Regenerative Biology Program and senior author of the study.
The latest episode of Developments to Watch, our collaborative video series produced by Medscape, is now available online: Disease in a Dish: The Ultimate Personalized Medicine.
In the video, Sanford-Burnham CEO John Reed, M.D., Ph.D., talks to Michael Jackson, Ph.D., vice president of drug discovery and development, about the Institute’s work on creating personalized “disease in a dish” models using stem cells derived from patients. They also talk about drug repurposing—finding new applications for existing therapeutic drugs in order to get treatments to patients faster.
Here’s an excerpt:
Stuart A. Lipton, M.D., Ph.D., director of Sanford-Burnham’s Del E. Webb Center for Neuroscience, Aging, and Stem Cell Research and a clinical neurologist
Sanford-Burnham researchers convince transplanted stem cell-derived neurons to direct cognitive function—getting us a step closer to using these cells to treat Alzheimer’s disease and other neurodegenerative conditions.
Researchers and patients look forward to the day when stem cells might be used to replace dying brain cells in Alzheimer’s disease and other neurodegenerative conditions. Scientists are currently able to make neurons and other brain cells from stem cells, but getting these neurons to properly function when transplanted to the host has proven to be more difficult. Now, researchers at Sanford-Burnham Medical Research Institute have found a way to stimulate stem cell-derived neurons to direct cognitive function after transplantation to an existing neural network. The study was published November 7 in the Journal of Neuroscience.
Symposium attendees await the announcement of Best Talk and Best Poster awards (Photo by Karolina Kucharova)
Each year, the Sanford-Burnham Science Network, our organization of postdoctoral researchers and graduate students, holds a symposium for young scientists to practice presenting their work and gain valuable feedback from their peers and our faculty members. This year, the La Jolla group’s event was held at the Sanford Consortium for Regenerative Medicine.
Here are five random things we learned last week at the 11th annual symposium:
Left: Medulloblastoma tumor (green) from untreated mouse. Right: Corresponding tissue from mouse treated with bFGF lacks tumor growth.
Brain tumors arising from different cell types might require different—and more personalized—treatment approaches.
Cancers arise when a normal cell acquires a mutation in a gene that regulates cellular growth or survival. But the particular cell this mutation happens in—the cell of origin—can have an enormous impact on the behavior of the tumor, and on the strategies used to treat it.
Robert Wechsler-Reya, Ph.D., professor and director of the Tumor Development Program in Sanford-Burnham’s NCI-designated Cancer Center, and his team study medulloblastoma, the most common malignant brain cancer in children. A few years ago, they made an important discovery: medulloblastoma can originate from one of two cell types: 1) stem cells, which can make all the different cell types in the brain or 2) neuronal progenitor cells, which can only make neurons.
Stem cells and progenitor cells are regulated by different growth factors. So, Wechsler-Reya thought, maybe the tumors arising from these cells respond differently to different therapies…
Conventional therapies don't hit tumors in the right place - at the cancer stem cells - so they keep coming back
The scientific symposium portion of San Diego’s annual Stem Cell Meeting on the Mesa fell on Halloween this year—good timing for a discussion about the dark side of stem cells: cancer stem cells.
Robert Wechsler-Reya, Ph.D., director of Sanford-Burnham’s Tumor Development Program, once said, “Current cancer therapies are like trying to kill a zombie by kicking it in the shins.”
Everyone knows you can only kill a zombie with a shot to the brains—anywhere else might slow it down temporarily, but only a very targeted hit to the head will get rid of it for good. (See the CDC’s Zombie Preparedness Guide.) So what Wechsler-Reya means is that the current methods for destroying or removing tumor cells are not aimed at what may, in some cases, be the actual “brains” of the problem—cancer stem cells.
Like other types of stem cells, cancer stem cells can self-renew, producing more cells. They also differentiate, specializing into other cell types. Those are very useful features when scientists are using stem cells to repair or replace diseased or damaged tissue (rebuilding heart muscle tissue after a heart attack, for example). However, cellular proliferation is also a hallmark of cancer.
In some cancers, stem cells may be the initial source of the problem, giving rise to tumors. They might also be the reason some tumors are resistant to standard cancer therapies such as chemotherapy or radiation therapy. What’s more, cancer stem cells can allow tumors to recur—even if the bulk of a tumor is removed, a few remaining cancer stem cells rise up to rebuild a new tumor. Like zombies, they are hard to get rid of.
Scientists are now trying to learn how stem cells turn to the dark side in cancer so that they can figure out how to better detect, prevent, and treat tumor growth—targeting the zombie’s brains, not just its shins.
Meet the four cancer stem cell (zombie)-fighting scientists who spoke at the 2012 Stem Cell Meeting on the Mesa:
Symposium speakers Christine Mummery, Ph.D. (Leiden University Medical Center) and Mark Mercola, Ph.D. (Sanford-Burnham) both discussed their work on generating new heart muscle tissue from stem cells
Scientists from all over San Diego—and beyond—gathered last Friday for Sanford-Burnham’s 34th annual symposium. This year’s theme: Frontiers in Stem Cell Biology for Drug Discovery. The topic was timely, given the recently announced 2012 Nobel Prize in Physiology or Medicine, awarded to John B. Gurdon and Shinya Yamanaka for their “discovery that mature cells can be reprogrammed to become pluripotent.” Yamanaka figured out how to turn a normal adult cell, such as a skin cell, into a stem cell that has the potential to become any other type of cell in the body. These special, laboratory-made stem cells are called induced pluripotent stem cells (iPSCs).
The symposium’s discussions centered on the idea that stem cells –especially iPSCs—can be used to model an individual’s own unique disease in a laboratory dish. These human cell-based models can then be used to test new and existing drugs for their toxicity and efficacy against disease.
Speakers came from Sanford-Burnham, Harvard, UT Southwestern, Mass General Hospital, UC San Diego, Stanford, and more. They talked about using stem cells to study and develop new therapies for conditions such as motor neuron disease, heart disease, autism, brain injury, Huntington’s disease, and spinal muscular atrophy.
We live-tweeted the event. For a snapshot of the day, including interesting tidbits, pictures, quotes, and links for more information, check out the Storify version of our tweets below. Then join the discussion on Twitter — look for us at @SanfordBurnham and #SBsymposium.
For more on stem cells and Sanford-Burnham’s work in the field, see:
Stem Cells 101
What is “Disease in a Dish”?
More stem cell blog posts
California Institute for Regenerative Medicine