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
3D structure of HNF-4α, a protein mutated in MODY1, a rare, inherited form of diabetes, reveals new pockets that could be targeted with therapeutic drugs
Researchers have determined the complete three-dimensional structure of a protein called HNF-4α. HNF-4α controls gene expression in the liver and pancreas, switching genes on or off as needed. People with mature onset diabetes of the young (MODY1), a rare form of the disease, have inherited mutations in the HNF-4α protein. This first-ever look at HNF-4α’s full structure, published today in Nature, uncovers new information about how it functions. The study also reveals new pockets in the protein that could be targeted with therapeutic drugs aimed at alleviating MODY1.
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)
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.
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:
Sanford-Burnham benefactor Malin Burnham, Sanford-Burnham President, Kristiina Vuori, M.D., Ph.D., Pradeep K. Khosla, and Sanford-Burnham CEO John Reed, M.D., Ph.D.
The location of Sanford-Burnham’s headquarters on the Torrey Pines Mesa of La Jolla, Calif., puts us squarely at the center of a hive of innovation. Part of our innovative spirit means we engage in constant dialogue with other, nearby scientific institutions. So when Pradeep K. Khosla, who took up the mantle of Chancellor at UC San Diego on August 1, expressed a desire to get acquainted with his new neighbors, we welcomed him.
Earlier this week, Khosla travelled just across the street to meet with Sanford-Burnham leadership and to tour the Conrad Prebys Center for Chemical Genomics. He was amazed with the state-of-the-art robotic screening center.
Khosla has expressed his commitment to innovation as a means of bringing income to UC San Diego, and to the region. In fact, a writer for the UT San Diego recently said of Khosla’s arrival in town, “The San Diego entrepreneur community should pay close attention.”
Johnson & Johnson CEO Alex Gorsky spoke about health as a strategic investment at the Lake Nona Impact Forum
More than 100 health care leaders from across the country came together last week for the first Lake Nona Impact Forum. The Forum was initiated by the Lake Nona Institute to create an opportunity for industry leaders to exchange ideas on ways to accelerate the impact of health innovation. Speakers included Alex Gorsky, CEO of Johnson & Johnson (J&J), Senator Bill Frist of Tennessee, John Chambers, CEO of Cisco, and Cavan Redmond, CEO of WebMD, among others.
Sanford-Burnham’s CEO, Dr. John Reed, moderated a panel that discussed the obstacles and opportunities in accelerating health innovation, which included Vicki Seyfert-Margolis, Senior Advisor for Science, Innovation and Policy at the U.S. Food and Drug Administration (FDA), and Margaret Anderson, Executive Director at FasterCures.
A recurrent theme throughout the three-day event was the impact of technology on health care. J&J’s Gorsky spoke about health being a strategic investment for businesses, communities, and our planet.
“Health care and how it is being delivered is the biggest challenge of our generation,” he said. “And technology is an important aspect of that.”
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
Congratulations to John B. Gurdon and Shinya Yamanaka on winning the 2012 Nobel Prize in Physiology or Medicine! They received the award today for their “discovery that mature cells can be reprogrammed to become pluripotent.” In other words, these scientists 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. Read below to learn more about stem cells and how they are revolutionizing medical research.
What are stem cells?
Stem cells are special because each is like a blank slate. Once it’s given the proper instruction, a stem cell can specialize and become any type of cell in the body—brain, heart, muscle, and more. Stem cells also have the ability to reproduce themselves indefinitely, renewing the supply.
Are there different types of stem cells?
Embryonic stem cells only exist during an organism’s development, when it is an embryo. These cells are pluripotent, meaning they have the capacity to become any cell type in the body.
Adult stem cells exist in fully developed organisms. They are more limited than embryonic stem cells—they are multipotent rather than pluripotent. These stem cells usually can only become a few types of specialized cells, based on the tissue from which they originate.
Induced pluripotent stem cells (iPSCs) are pluripotent, much like embryonic stem cells. iPSCs are produced in the laboratory by genetically reprogramming any adult cell, such as a skin cell.
Dr. Fred Levine worked tirelessly to discover a potential therapeutic for diabetes.
Dr. Fred Levine, director of our Sanford Children’s Health Research Center, started searching for potential diabetes drugs in 2005. Back then, Sanford-Burnham didn’t have a high-throughput drug screening center. It didn’t even have a children’s health research center.
One day, Dr. Levine was conferring with his colleague Dr. Mark Mercola, a heart researcher. Dr. Mercola was using some modest drug screening equipment set up in a converted office down the hall from his laboratory. He was screening chemical compounds with the hope of finding a few they could further explore as potential drugs for treating heart disease. Dr. Levine thought the same technique might lead to potential treatments for type 1 (juvenile) diabetes.
Ring forms of the Plasmodium falciparum (malaria) parasite, inside red blood cells (Image by Michael Zahniser)
An international team of scientists, including researchers at the University of California, San Diego (UCSD) and Sanford-Burnham Medical Research Institute, have identified the first reported inhibitors of a key enzyme involved in survival of the parasite responsible for malaria. Their findings, which may provide the basis for anti-malarial drug development, were published July 19 in the Journal of Medicinal Chemistry.
According to the World Health Organization, there were 216 million cases of malaria worldwide in 2010. Severe forms of the disease are mainly caused by the parasite Plasmodium falciparum, transmitted to humans by female Anopheles mosquitoes. Malaria eradication has not been possible due to the lack of vaccines and the parasite’s ability to develop resistance to most drugs.
Mustafa Malik observes a laboratory demonstration along with some of his classmates
This week, Sanford-Burnham is proud to welcome 11 students from Newsweek’s #1 Transformative High School in the nation to our La Jolla campus. They will spend four days in different laboratories, receiving mentoring from faculty and postdoctoral researchers. Opportunities like this one are what prepare students at The Preuss School UCSD for successful academic futures and later, careers.
Mustafa Malik took part in the program—which is now in its fifth year—during the summer of 2010. He is heading to Yale University this fall. An exceptional young man with myriad possibilities before him, he has not settled on a major. “I definitely have had an interest in science my whole life, but I am not sure where exactly I will end up career-wise,” he says. “I think going into college too sure of what you want can possibly be harmful because it can hold you back from exploring new and exciting things that you might never have tried.”
Wherever he ends up, Mustafa knows that his experience at Sanford-Burnham has had an impact.
High school interns at Sanford-Burnham’s Lake Nona campus, with their mentors Greg Roth (right), John Shelley (center), Sheila Collins (front row), and Darrin Kuystermans (left)
As summer begins, books get shelved and schools close, but for many teens and young adults, leaving the classroom doesn’t mean learning stops. On the contrary, they pursue internships that allow them to advance their education in a hands-on way, often taking steps toward a career.
Sanford-Burnham opens its doors to a large number of interns, at various stages of their education, throughout the year, but summer is an especially busy time. The enthusiastic voices of young people, some making their own scientific discoveries for the first time, emanate through the hallways, reminding everyone how exciting science can be.
Last week, Sanford-Burnham at Lake Nona in Orlando welcomed five students from area high schools including Trinity Preparatory School and Seminole High School. Forty students participating in a Junior Achievement summer program visited the institute to learn about medical research career paths. Each one is paired with a mentor in a research laboratory or the Conrad Prebys Center for Chemical Genomics for two to four weeks. Eight college interns will soon be arriving as well. Throughout the summer, a number of aspiring scientists will be scattered throughout the Institute’s La Jolla laboratories for anywhere from one week to the entire summer.
The Collaborative Research Grant program will provide Florida scientists with access to our screening center
Last week was a great one for medical researchers across the state of Florida. The state legislature and governor approved funding for the Collaborative Research Grant program between the Florida Department of Health and Sanford-Burnham Medical Research Institute. Starting in July, the program will provide scientists at universities and non-profit institutes throughout Florida with access to Sanford-Burnham scientists and our state-of-the-art technologies for drug discovery. This includes access to the Institute’s Conrad Prebys Center for Chemical Genomics.
Together with the Florida Department of Health, Sanford-Burnham will develop a competitive grant program, based on peer-review that will provide funds for collaborative projects between Florida-based research scientists and Sanford-Burnham’s fully operational, state-of-the-art drug discovery technology center based at Lake Nona.