We announced today that we’ve signed a new collaborative agreement with Mayo Clinic to build a pipeline of therapeutic drugs aimed at a variety of diseases with serious unmet medical needs. Under this agreement, Mayo Clinic scientists will work with researchers in our Conrad Prebys Center for Chemical Genomics (Prebys Center) to conduct early-stage drug discovery, including assay development, high-throughput screening, and lead identification.
Michael Jackson, Ph.D., vice president of drug discovery and development in Sanford-Burnham's Conrad Prebys Center for Chemical Genomics (photo by Mark Dastrup)
San Diego is a powerhouse for cancer research, home to two National Cancer Institute (NCI)-designated centers for basic research—our Cancer Center and the Salk Institute Cancer Center—and the University of California, San Diego Moores Cancer Center, the region’s only NCI-designated comprehensive cancer center.
L to R: Peter Preuss, Peggy Preuss and Peter Preuss, Jr., whose family foundation provides generous support for cancer research and brain cancer seminars in San Diego.
Napoleone Ferrara, Ph.D., was propelled into the national spotlight last week, when he was named one of 11 winners of the first Breakthrough Prizes in Life Sciences. This new prize—awarding a no-strings-attached $3 million to each recipient—was bestowed by Silicon Valley innovators Sergey Brin, Anne Wojcicki, Mark Zuckerberg and Yuri Milner. One goal of the prize is to make household names out of the country’s top scientists.
Drs. Smith (TRI), Izumo (Takeda), and Kelly (Sanford-Burnham; left to right) in traditional Japanese coats at the signing ceremony for the renewed partnership
We held a special signing ceremony today at our Lake Nona, Orlando, campus to renew our research agreement with Florida Hospital and Takeda Pharmaceutical Company Limited. The renewal extends our collaboration to discover and evaluate new therapeutic approaches to obesity. The collaboration uses a research and drug-development model that creates an early feedback loop in the discovery pipeline. We and our partners expect this model will shorten the time to develop new therapeutics.
Interest in the development of obesity treatments remains strong, as the regulatory approval of two new obesity therapeutics in 2012 offered a proven pathway for drug candidates. “As the worldwide obesity crisis continues to escalate, we are seeing a rise in the prevalence of severe obesity—defined by BMI greater than 40—and we know that this subset of the obese population experiences increased mortality and associated diseases, such as heart disease, diabetes and cancer,” said Steven R. Smith, M.D., scientific director of the Florida Hospital – Sanford-Burnham Translational Research Institute for Metabolism and Diabetes (TRI) and president-elect of The Obesity Society. “These statistics are staggering and clearly demonstrate the need to rapidly develop treatment strategies for obesity.”
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.
Potential cancer drug sabutoclax blocks Bcl-2 protein family members that help keep cancer cells alive. This image shows the structure of one Bcl-2 protein, known as Bcl-Xl. (Image courtesy of the Pellecchia laboratory)
Researchers find that certain types of drug-resistant leukemia stem cells are vulnerable to sabutoclax, a novel cancer stem cell-targeting drug based on Sanford-Burnham research.
New experiments show that sabutoclax, a novel cancer stem cell-targeting drug that grew out of research at Sanford-Burnham Medical Research Institute, in combination with other therapies, could effectively treat diseases like chronic myeloid leukemia (CML). Sabutoclax might also lower the chance of relapse.
“The demonstration of sabutoclax’s preclinical activity in mouse models of CML is exciting and encourages further evaluation of this promising drug candidate for aggressive leukemias. We look forward to continuing our collaborative studies of sabutoclax, as we move this drug closer to the clinic,” said John Reed, M.D., Ph.D., professor and Donald Bren Chief Executive Chair at Sanford-Burnham.
Sabutoclax was first discovered as a result of research in the laboratories of Reed and his Sanford-Burnham colleague, Maurizio Pellecchia, Ph.D. The pair is now working with biotechnology company Oncothyreon Inc to develop sabutoclax into a potential anti-cancer drug. This latest study of sabutoclax’s efficacy, published January 17 in the journal Cell Stem Cell, was led by Catriona Jamieson, M.D., Ph.D., at UC San Diego Moores Cancer Center, in collaboration with Reed, Pellecchia and others.
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.
Sanford-Burnham's John Reed (right) with Margaret Anderson of FasterCures (left) and Vicki Seyfert-Margolis of the FDA at the Impact Forum.
In our last blog post about the Lake Nona Impact Forum, we focused on technology as an enabler of the health care of the future. Today’s post will be about the importance of partnerships and collaboration in the quest to make health care more efficient, affordable, and accessible. Already, it seems like there’s an extensive amount of collaboration happening in the field. Pharmaceutical companies partner with nonprofit research institutes like Sanford-Burnham to advance drug discovery, health technology companies collaborate with the U.S. Department of Defense to develop novel prostheses, and medical schools partner with hospitals to better educate the physicians of the future. But as we learned during talks and panel discussions at the Impact Forum, collaboration will become even more important in the near future.
As Alex Gorsky, CEO of Johnson & Johnson, said during his speech, “No one is going to solve the world’s health care problems alone.” We need to work together to innovate health care solutions that make an impact.
Stem cell-derived cardiomyocytes (heart muscle cells) expressing a green fluorescent protein
For years, scientists have been looking for a good source of heart cells that can be used to study cardiac function in the lab, or perhaps even to replace diseased or damaged tissue in heart disease patients. To do this, many are looking to stem cells. Researchers at Sanford-Burnham Medical Research Institute, the Human BioMolecular Research Institute, and ChemRegen, Inc. have been searching for molecules that convert stem cells to heart cells for about eight years—and now they’ve found one. Writing in the August 3 issue of Cell Stem Cell, the team describes how they sifted through a large collection of drug-like chemicals and uncovered ITD-1, a molecule that can be used to generate unlimited numbers of new heart cells from stem cells.
“Heart disease is the leading cause of death in this country. Because we can’t replace lost cardiac muscle, the condition irreversibly leads to a decline in heart function and ultimately death. The only way to effectively replace lost heart muscle cells—called cardiomyocytes—is to transplant the entire heart,” said Mark Mercola, Ph.D., director of Sanford-Burnham’s Muscle Development and Regeneration Program and senior author of the study. “Using a drug to create new heart muscle from stem cells would be far more appealing than heart transplantation.”
Fresno State faculty member Jason Bush and one of his former students, Shana Morshedian, now a research assistant in John Reed's lab.
Last week, faculty from California State University, Fresno (Fresno State) and Sanford-Burnham met to define the next steps in the Collaborative Project in Cancer Health Disparities Research.
Sanford-Burnham, Fresno State, and the Central Valley Health Policy Institute share this collaborative project, funded by the National Cancer Institute. Their mission is to train undergraduate and graduate students for future cancer research careers and enhance cancer research potential at Fresno State (a minority-serving institution). The three-year grant gives Fresno State minority students the opportunity to spend a summer in Sanford-Burnham laboratories, where they become more familiar with biomedical research.
Rongsheng Jin, Ph.D (second from the right) and researchers in his laboratory
provided by Georgia Health Sciences University
Some unusual alliances are necessary for you to wiggle your fingers, researchers report.
Understanding those relationships should enable better treatment of neuromuscular diseases, such as myasthenia gravis, which prevent muscles from taking orders from your brain, said Lin Mei, Ph.D., director of the Institute of Molecular Medicine and Genetics at Georgia Health Sciences University.
During development, neurons in the spinal cord reach out to muscle fibers to form a direct line of communication called the neuromuscular junction. Once complete, motor neurons send chemical messengers, called acetylcholine, via that junction so you can text, walk, or breathe.
The new San Diego Skeletal Muscle Research Center will be made up of three core facilities shared by five local institutions.
The National Institutes of Health (NIH) recently awarded a new grant to establish the San Diego Skeletal Muscle Research Center. This new center, led by UC San Diego’s Rick Lieber, Ph.D., Sanford-Burnham’s Mark Mercola, Ph.D., and The Scripps Research Institute’s Velia Fowler, Ph.D., will allow 21 scientists at five different research institutions to combine their expertise and state-of-the-art methods to accelerate research that advances our understanding of skeletal muscles and the diseases that affect them.
insulin-producing beta-cells in a mouse pancreas (Image courtesy of Edward Monosov)
Dr. Barbara Ranscht and her lab are working to better understand how T-cadherin—a protein found on the surface of neurons, muscle, and other cells—regulates communication between cells during development and disease. The best way to go about this is to see what happens when the protein is missing. To do this, her lab developed a mouse model that lacks the protein altogether. Using these animals, Dr. Ranscht’s group has revealed that T-cadherin protects the stressed heart and is necessary for new blood vessel growth in injury models.
One day, Dr. Ranscht found herself discussing possible roles for T-cadherin in metabolism with Sanford-Burnham colleagues Dr. Björn Tyrberg and Dr. Fred Levine. The researchers especially wondered about T-cadherin’s role in the pancreas (Drs. Tyrberg’s and Levine’s organ of expertise).
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)
“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.”
Photo by Rob Lee, www.flickr.com/photos/roblee/
In science we know that little things add up to big things. The human body contains trillions of cells, and within each of those cells, billions of proteins do the work that keeps those cells, and in turn, the body, functioning. And each one of those cells has a purpose.
As a not-for-profit medical research institute, Sanford-Burnham relies on donations from individuals and foundations—in addition to grant funding—to continue its scientific progress day after day. Big donations make news. But even little ones make a difference.
Today, on 11/11/11, we want to remind supporters of medical research that every ONE counts. Dollar by dollar, your tax-deductible gifts make an impact. You may not think of yourself as a philanthropist, but you can play a part. With any contribution, you are supporting medical research—which has the potential to touch millions of lives.
Give $1. Give $11. Give $111, or as much as you are inspired to! Visit this link to make a gift. And there is another way you can make a difference. Help spread the word about our 11/11/11 Every ONE Counts fundraising effort on Twitter, Facebook, and LinkedIn.
Thank you for your support!
Meet some of our other supporters here.