As you look back on 2011, what are you grateful for? What are your hopes for 2012? Help us make the new year a year of advances in medical research. Here are five reasons to make a tax-deductible gift by December 31.
Researchers envision a brighter future for people suffering from disease. You can help.
A group from The Atlantic Meets the Pacific conference tours the Stem Cell Research Center at Sanford-Burnham's San Diego campus. (Photo by Mark Dastrup)
When The Atlantic magazine was looking for a location to host its first West Coast symposium, the Torrey Pines Mesa area of San Diego sprang to their attention. As Atlantic Vice President Elizabeth Baker Keffer wrote in her welcome, “The research hub of San Diego makes this the perfect location to experience the interdisciplinary experiments occurring on the front line of discovery.” They partnered with UC San Diego to present The Atlantic Meets the Pacific, October 17-19.
Sanford-Burnham joined The Scripps Research Institute, the Salk Institute, Scripps Institution of Oceanography, and UC San Diego’s Calit2 and Moores Cancer Center in representing the powerhouse of scientific ideas that resides here overlooking the Pacific Ocean.
Left: Brandon Nelson, manager of Sanford-Burnham's Stem Cell Research Center, pulls stem cells out of cryogenic storage (Photo by Nadia Borowski Scott) Right: Cluster of neurons (pink) derived from stem cells.
Wednesday, October 5, 2011 is Stem Cell Awareness Day, sponsored by the California Institute for Regenerative Medicine. To mark the occasion at Sanford-Burnham, we are opening our doors to anyone who wants to learn about the latest in stem cell research and the therapeutic potential stem cells hold for Alzheimer’s disease, heart disease, joint diseases, spinal cord injury, diabetes, and more.
- What: Stem Cell Awareness Day
Public tours of Sanford-Burnham’s stem cell research facility and drug discovery center - When: Wednesday, October 5, 2011, 3:00-4:00 p.m.
- Where: Sanford-Burnham’s La Jolla, California campus
10901 North Torrey Pines Road
La Jolla, California 92037
See map here and follow event signs to parking. Tours will begin outside Building 7. - Who: students, teachers, patients, advocates, press, and other members of the community are invited
- RSVP: If you plan to attend or have any questions, please contact Heather Buschman at
hbuschman@sanfordburnham.org or 858-610-3808.
Attendees will tour our Stem Cell Research Center and meet scientists who are turning stem cells into other cell types that can be used to replace diseased or damaged tissue in heart disease, Alzheimer’s disease, and more. Visitors will have the chance to look through a microscope at cardiomyocytes (heart muscle cells) that are able to beat even in a laboratory dish. We will also take visitors to the Conrad Prebys Center for Chemical Genomics, where stem cells and robots are being used to discover new medicines by modeling diseases in a dish.
Check out more Stem Cell Awareness Day events taking place throughout California, the U.S., and the world or read more about Sanford-Burnham stem cell research on our blog.
CEO Dr. John Reed (left) and Congressman Duncan D. Hunter
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.
Scientists in Sanford-Burnham's Conrad Prebys Center for Chemical Genomics use robotic arms like this one to search for compounds that alter cellular behavior—precursors to new medicines.
Yesterday, we introduced a study in which scientists in Sanford-Burnham’s NCI-Designated Cancer Center and Conrad Prebys Center for Chemical Genomics were looking for compounds that regulate invadopodia, cellular projections that allow cancer cells to invade and metastasize. They used robotic technology and automated microscopy to screen a library of pharmacologically active compounds—compounds already known to influence cellular function. In the course of the study, the researchers found some compounds that inhibit invadopodia and some that promote their formation. One of the latter was paclitaxel. Paclitaxel, also known by the brand name Taxol, is an FDA-approved drug currently used to treat several different kinds of cancer. The drug’s anti-tumor activity is based on its ability to bind and stabilize microtubules, one component of the cellular cytoskeleton, thereby halting cell division and inducing cellular suicide (a good thing, for cancer).
Metastatic cancer cells form invadopodia, shown here as bright red spots. (Image by Begoña Díaz)
Metastasis—the spread of cancer from the place where it first started to another place in the body—is the most common reason that cancer treatments fail. To metastasize, some types of cancer cells rely on invadopodia, cellular membrane projections that act like feet, helping them “walk” away from the primary tumor and invade surrounding tissues. To determine how cells control invadopodia formation, Sanford-Burnham scientists screened a collection of pharmacologically active compounds to identify those that either promote or inhibit the process. They turned up several invadopodia inhibitors that target a family of enzymes called cyclin-dependent kinases (Cdks), revealing a previously unrecognized role for Cdks in invadopodia formation. These findings appeared online July 26 in Science Signaling.
“Previous studies by our group and others have demonstrated that we might be able to target invadopodia to prevent cancer cell invasiveness,” says Dr. Sara Courtneidge, professor and director of the Tumor Microenvironment Program in Sanford-Burnham’s NCI-Designated Cancer Center and senior author of the study. “In this study, we established a cell-based screening assay to help us identify regulators of invadopodia formation.”
Dr. Courtneidge’s group has been studying invadopodia for a number of years with the goal of unraveling how they regulate tumor cell invasion. Here, her team, led by postdoctoral researcher Dr. Manuela Quintavalle, joined forces with scientists in Sanford-Burnham’s Conrad Prebys Center for Chemical Genomics (Prebys Center). This collaboration provided the Courtneidge lab with extra expertise in chemical genomics, the robotic technology necessary to rapidly and reproducibly screen more than 1,000 compounds with known pharmacological activity in cell-based assays, and automated microscopy capable of detecting and measuring invadopodia formation.
The students in this year's Preuss School UCSD intership program, in Bradley Plaza on the La Jolla campus.
A group of 12 San Diego high school students has been waking up early all week and making the most of their summer break. If you look at their Facebook pages you might see updates saying things things like, “OMG, silencing DAF2 gene in C. elegans–amazing.” An internship program taking place at Sanford-Burnham demonstrates that the right educational opportunities have the power to get young people excited about science and perhaps change their lives.
That was the case for Tony Chau, who completed the Sanford-Burnham/Preuss School UCSD Summer Internship program in 2009. This fall he heads to Duke University, complete with a scholarship, to double major in Biomedical Engineering and Economics. “The internship program helped me greatly through the rest of high school and in applying to college,” he recalls. “The people I met and the experiences I had will stay with me throughout my career.”
Dr. Anthony Tether, Dr. Michael Jackson, Duane Roth and Greg Lucier
What will the future look like? On June 5, four research experts and around 200 guests gathered at the Hilton La Jolla Torrey Pines to answer this question. The event, Sanford-Burnham’s annual President’s Circle reception, brought together Dr. Anthony Tether, former director of the Defense Advanced Research Projects Agency (DARPA), Greg Lucier, chairman of both Life Technologies and the Sanford-Burnham board of trustees and Dr. Michael Jackson, vice president of Drug Discovery at Sanford-Burnham and was moderated by Duane Roth, CEO of CONNECT. Together, they shared their thoughts on how research will impact human health in coming years.
DARPA has been a key part of the United States’ technological success for 50 years. The agency was created after the first Sputnik launch, an event that shocked the American public and led to new approaches to research. “DARPA was initiated to create technological surprise,” said Dr. Tether.
You might have thought Conrad Prebys had been given 50 yard-line seats to the Super Bowl—that’s how thrilled he was to visit Sanford-Burnham. Two years ago, Prebys made a $10 million gift to support chemical genomics at the Institute. He stopped by on February 10 to learn how the research at the Conrad Prebys Center for Chemical Genomics is progressing and to tour one of our labs.
First the tour. Dr. Hudson Freeze studies congenital disorders of glycosolation (CDG), a family of rare diseases in which defective enzymes fail to add necessary sugar chains to proteins, creating multiple medical issues. CDG is fatal in 20 percent of affected children. While visiting the Freeze lab, Prebys met several researchers and spoke via Skype with a CDG patient, a young woman in the Netherlands who has spent a significant portion of her life in hospitals. Researchers in the Freeze lab are looking for creative ways to fix the enzyme deficiencies that cause these conditions.
Robots in the Prebys Center screen hundreds of thousands of compounds to find the handful that could become new medicines.
Sanford-Burnham has been conducting cutting-edge research on neurological and neuropsychiatric conditions for many years and recently that research received a big boost. The Institute announced a collaboration with Ortho-McNeil-Janssen Pharmaceuticals, Inc., (OMJPI), a division of Johnson & Johnson, to discover new drugs to treat Alzheimer’s diseaseand major psychiatric disorders.Under the agreement, Sanford-Burnham will look for new therapeutic targets for Alzheimer’s disease and neuropsychiatric conditions. Then, the Conrad Prebys Center for Chemical Genomics (Prebys Center) will identify chemical compounds that therapeutically alter those targets. These compounds will then be optimized and directed into OMJPI’s drug pipeline.
"Disease in a dish" has great potential to accelerate drug discovery.
“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.
Philanthropist and businessman Arthur Brody has pledged $1 million to Sanford-Burnham to help move basic research findings out of the laboratory and into the clinic. The gift creates the Art Brody Innovation Fund, which will support promising research that might otherwise be stalled due to lack of funding. Most early stage biomedical research is funded by the National Institutes of Health, while clinical trials are usually funded by pharmaceutical and biotechnology companies. Unfortunately, this leaves a large gap in which funding is scarce, the so-called “valley of death” for new treatments. Sanford-Burnham has made numerous investments to help fill this gap, such as creating the Conrad Prebys Center for Chemical Genomics.
A few weeks ago, Sanford-Burnham received a visit from Wesley Overton, age 7, and his mother Heather Atkins. Wesley is a pretty typical kid in most respects—he likes crystals and robots. He has also survived a stage IV Wilm’s tumor (a form of kidney cancer that strikes mostly children) that had metastasized to his lungs.
In January 2009, Heather was cuddling with her son when she noticed the lump. They went to the doctor, and by early evening he was checked into Rady Children’s Hospital. Two days later he had surgery.
“Your entire world changes in a 24-hour period,” says Heather.
Let’s say you are a scientist studying Protein X, a protein that normally tells cells to divide but, when malfunctioning, causes unchecked cell division that leads to a tumor. You think blocking this deviant Protein X might stop cancer. So you take Protein X to your colleagues in Sanford-Burnham’s Conrad Prebys Center for Chemical Genomics (Prebys Center), where robotic systems can screen hundreds of thousands of chemical compounds to find that one needle-in-a-haystack (or handful of needles) that inhibit Protein X. From there, you continue developing these winning compounds, hopefully into a new anti-cancer drug.
To boost the Prebys Center’s drug discovery efforts, scientists in Dr. Nicholas Cosford’s laboratory, part of Sanford-Burnham Cancer Center’s Apoptosis and Cell Death Program, are putting on their 3D glasses. They recently teamed up with French company MEDIT SA to use and enhance a new software platform built around a computer program called MED-SuMo. This platform breaks down 3D images of known protein structures to find chemical fragments that might bind and inhibit Protein X (or other interesting proteins) in real life.
Most Sanford-Burnham scientists are basic researchers, meaning they study the most fundamental aspects of cellular and molecular function. In the course of this research, scientists at Sanford-Burnham and other academic research institutions often discover clues to the underlying causes of human disease. Sometimes, they also uncover promising new drug targets that could be manipulated to treat those diseases. Unfortunately, it can be difficult to interest the pharmaceutical industry in advancing these early findings into new drugs. That’s why some academic institutions are beginning to develop their own drug discovery platforms, like that provided by Sanford-Burnham’s Conrad Prebys Center for Chemical Genomics(Prebys Center). There, scientists use robotic technology to screen chemical compounds by the millions to find the few that could potentially be developed into new medicines.This month, the journal Nature Methods highlights the Prebys Center and several other large-scale academic screening centers around the country. In the article, Project Manager Dr. Thomas Chung discusses how academic drug discovery efforts help advance experiments (also called “assays”) that can’t be done in the pharmaceutical industry:
Industry also shies away from assays that take a long time to optimize, says Thomas ‘TC’ Chung of the screening center at the Sanford-Burnham Medical Research Institute, who, like many academic screeners, spent years in industry. “In pharma, our job was to reject assays that didn’t fit our format,” he says. “Now our job is to reformat assays and make them work.”
For more, see “Academic screening goes high-throughput” in the October 2010 issue of Nature Methods.