Energizing, accelerating and reimagining translational research
Year–End Review by President and CEO David A. Brenner, MD
Our mission is to energize, accelerate and reimagine translational research to create and deliver new therapies, faster and better, that improve human lives, health and well–being.
48+ years of pioneering research
A world–class NCI–designated Cancer Center One of only seven basic research centers in the U.S. (since 1981)
Top 3% of research institutions worldwide based on citations per paper
Top 3 of all independent research institutes ranked by NIH funding
169 peer–reviewed papers published in 2024
Top 9% in the nation: Nature Index of nonprofit/nongovernment institutions in biomedical science
1,444 patents, 37 start–ups from Sanford Burnham Prebys innovations
As of January 2025
60+ drug discovery projects
Over $100M of translational science grants in the last five years
12
Clinical trials underway
• Cancer
• Immune Therapy
• Kidney Disease
• Calcification
• Smoking Cessation
• Opioid Addiction
8
FDA-approved treatments and tests
• Aggrastat®
• Epogen®
• Integrilin®
• Strensiq®
• Targretin®
• Venclexta®
• PSA Test
• Duvyzat™
David A. Brenner, MD President and Chief Executive Officer
Donald Bren Chief Executive Chair
It may not be a reach to say there are 2,024 reasons to celebrate 2024 as one of the most active and notable years in the almost half-century history of Sanford Burnham Prebys.
It has been a busy and productive 12 months. Allow me to enumerate:
1. Funded by T. Denny Sanford’s transformational gift in 2023, we recruited 13 new faculty, both early–career researchers with bold, bright futures and well–established science superstars. Their fields of interest span the enterprise, from cancer and neuroscience to computational biology and advanced imaging technologies.
2. We established our four disease–focused centers in cancer, cardiovascular and muscular diseases, neurologic diseases and metabolic and liver diseases, plus two technology–enabling centers to support our science: the Center for Therapeutics Discovery (which includes the Conrad Prebys Center for Chemical Genomics) and the Center for Data Sciences.
3. We bolstered our education efforts, with a new dean of the Graduate School of Biomedical Sciences and an ambitious plan to attract, encourage and promote excellence across our strategic mission and vision.
4. We’re sharpening our entrepreneurial ethos, with new business development programs to help students and faculty better realize the rewards of their work.
5. We’re telling our story everywhere, from published essays to expansive social media to popular community outreach events, like open houses and the new A Conversation About series.
Tremendous credit and gratitude go to Denny Sanford for his extraordinary and enduring support of our mission and vision, now and over the years. But science—and the support of science— are team efforts. Great achievement requires the unified, focused effort of many.
This report is filled with illuminating and inspiring stories of what can be accomplished with your support. We are a team. We work together to translate science into health. It is an honor and privilege.
David A. Brenner, MD President and Chief Executive Officer
Donald Bren Chief Executive Chair
On December 11, 2024, T. Denny Sanford was honored with the first-ever Erkki Ruoslahti Award for Transformational Leadership at a special event attended by Sanford Burnham Prebys trustees and faculty.
The award recognizes individuals whose visionary leadership has driven positive change, inspired groundbreaking solutions, and contributed to the transformation of industries and society.
Named after Dr. Erkki Ruoslahti, one of the Institute’s early faculty members and former president, the award celebrates his pioneering contributions to cell biology and nanomedicine. Ruoslahti’s work, which includes groundbreaking discoveries in cell adhesion and tumor-targeting peptides, has reshaped biomedical research. His extraordinary career has earned him numerous prestigious honors, including the 2022 Albert Lasker Basic Medical Research Award, known as “America’s Nobel.”
T. Denny Sanford, a distinguished philanthropist and one of the Institute’s key supporters, was praised for his lifelong commitment to advancing biomedical science. “Denny Sanford has been a friend, supporter, and mentor for many years,” said Dr. David Brenner, president and CEO of Sanford Burnham Prebys. “His unwavering support has been critical to our vision and mission. No one has done more to help advance our work, and this award is a heartfelt expression of our gratitude.”
Sanford’s leadership and dedication have not only shaped the growth of Sanford Burnham Prebys but have also played a pivotal role in transforming the landscape of biomedical research and healthcare.
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Deep expertise in disease–focused research bolstered by state–of–the–art technology
Founded in 1981, the NCI–Designated Cancer Center is one of only seven National Cancer Institute designated basic laboratories in the nation. Our investigators collaborate to answer fundamental questions in cancer biology and to address unmet clinical needs by conducting translational studies leading to new and better cancer treatments.
We get to the heart of biological processes in muscle cells and tissue to discover new treatments for millions suffering from debilitating muscular conditions and cardiovascular disease.
Our researchers are working to gain a fundamental and therapeutically actionable understanding of agerelated diseases of the brain and central nervous system that can lead to novel treatment and improved quality of life for all.
Through the Conrad Prebys Center for Chemical Genomics, our scientists take new knowledge and shape it into novel therapeutics that can positively impact the health of patients around the world. Using the latest technologies, our researchers identify and develop the next generation of drugs.
The rising prevalence of metabolic disorders is alarming, especially those conditions that affect the liver–central to metabolic health. We aim to identify and interpret the underlying causes of, and drivers of, metabolic and liver diseases.
No new drug can be created without a solid foundation of knowledge. In science, knowledge often takes the form of data. The Center for Data Sciences supports the entire research enterprise, deciphering and translating vast repositories of information into knowledge.
Jianhua Zhao, PhD
Assistant Professor
Cancer Metabolism and Microenvironment Program
Kevin Tharp, PhD
Assistant Professor Cancer Metabolism and Microenvironment Program
Kelly Kersten, PhD
Assistant
Professor
Cancer Metabolism and Microenvironment Program
Scientists at Sanford Burnham Prebys, led by Jianhua Zhao, PhD, have used cryo-electron microscopy to develop a sharper picture of how crucial machinery in the human cell’s recycling process for obsolete and misshapen proteins— known as proteasomes—are formed. Issues with proteasomes can contribute to certain cancers and are associated with age-related diseases.
Learning more about the assembly of proteasomes may provide opportunities to either target certain cancers or improve outcomes linked to aging, and potentially design better versions that may lead to new treatments for many cancer and other conditions linked to aging.
While immunotherapies that boost the immune system have revolutionized the way we treat cancer, some patients do not respond, and the mechanisms of resistance remain unclear.
Recruited in 2023, Kelly Kersten, PhD, and Kevin Tharp, PhD, both assistant professors in the Cancer Metabolism and Microenvironment Program, study the hypothesis that part of the reason why this occurs is that these therapies fail due to tumor-associated fibrosis—a thick layer of scar-forming collagen.
They found that tumor-associated macrophages, a type of immune cell found abundantly in the tumor microenvironment, respond to fibrosis by suppressing the anti-tumor functions of other immune cells. Tharp and Kelly hope that their findings will aid in the development of novel strategies to boost immunotherapies used to treat cancer patients.
Mudra Choudhury, PhD Postdoctoral Associate Sanju Sinha Lab
Mudra Choudhury had loved computer programming growing up, but she noticed her interest fading as an undergraduate computer science major at Oregon State University in Corvallis.
“I just wasn’t excited anymore by the questions that were driving my coursework,” said Choudhury. “I looked around to see how I could apply computer science to things I care about, and computational biology turned out to be a great fit with fascinating research opportunities focused on improving health.”
Choudhury earned her undergraduate degree in BioResource Research focused on Bioinformatics and Genomics at Oregon State University. She went on to complete her doctoral studies in Bioinformatics at the University of California Los Angeles, where she studied RNA editing dysregulation in neuropsychiatric diseases. After earning her PhD, Choudhury joined Sanford Burnham Prebys in October 2023 as a computational biologist and postdoctoral associate in the lab of Sanju Sinha, PhD, an assistant professor in the Cancer Molecular Therapeutics Program.
“I chose to join Sanford Burnham Prebys because it felt like a tight-knit community with very collaborative principal investigators, and I appreciated the Institute’s efforts to grow its computational research portfolio,” said Choudhury. “I also was excited to work with Sanju, as I knew his expertise aligned with my desire to continue growing my skills in computational methods development and machine learning.”
Since joining the Institute, Choudhury has studied circular extra-chromosomal DNA elements (ecDNA), pieces of DNA that have broken off normal chromosomes and then been stitched together by DNA repair mechanisms. This phenomenon leads to circular DNA elements floating around in a cancer cell, which is known to be a marker of poor patient outcomes.
Choudhury, Sinha and a team of collaborators are testing the use of deep-learning AI algorithms to identify tumors with ecDNA by analyzing the biopsy slides that are routinely created by pathologists to diagnose brain cancer.
“Our group is currently developing and testing a tool called ecPath that is able to predict the presence of ecDNA in tumors and forecast patient outcomes,” said Choudhury. This new technique may lead to improved diagnostic methods and the development of precision therapies for patients suffering from tumors with ecDNA.
We are proud to support groundbreaking research at Sanford Burnham Prebys that aligns with our foundation’s mission to advance innovative solutions for diabetes and related conditions. Martha’s passion for improving lives through science continues to guide our work. The discovery of LARP6’s role in liver fibrosis offers remarkable promise, and we are honored to help fund this vital research. Together, we aim to transform these scientific insights into treatments that can change the course of liver disease and improve the lives of millions impacted by diabetes and its complications.
More than 38 million Americans live with diabetes, yet many remain unaware of a silent but life-threatening danger: Metabolic Associated Steatohepatitis (MASH).
This condition develops when fatty liver disease progresses, causing fibrotic scarring that can lead to liver dysfunction and, ultimately, failure. With no effective drugs currently available to treat liver fibrosis, the need for innovative solutions has never been more urgent.
Thanks to the vision and generosity of the Martha Proctor Mack Foundation, hope is on the horizon. Dr. David A. Brenner, president and CEO of Sanford Burnham Prebys, is leading pioneering research to develop lifechanging treatments for MASH and other fibrotic diseases.
Brenner’s team has made a groundbreaking discovery about the role of the protein LARP6 in driving liver fibrosis. LARP6 stabilizes collagen mRNA, fueling the excessive production of collagen—the hallmark of fibrosis. By targeting the interaction between LARP6 and collagen mRNA, researchers have identified a promising pathway for developing the first effective drug to halt fibrosis before it causes irreversible liver damage.
“This breakthrough would not have been possible without the vision and generosity of the Martha Proctor Mack Foundation,” said Brenner.
With their support, this scientific discovery is transforming into a real-world solution that may change lives for generations to come. Together, we are closer than ever to developing a treatment that could one day cure MASH, providing relief and restoring health for those who need it most.
3D bioprinting and patient samples help advance models of liver disease
David A. Brenner, MD
Metabolic dysfunction–associated steatohepatitis, or MASH, is an inflammatory, liver-scarring disease that has reached epidemic proportions. There is only one FDA-approved pharmacological therapy, in part due to a lack of adequate preclinical models for study and testing.
Scientists at Sanford Burnham Prebys, led by Brenner, developed a 3D bioprinted liver tissue model employing liver cells from healthy or MASH-diseased donors. The tissues display all of the characteristics of MASH, including fibrosis, and offered a peek at the disease’s underlying pathology. This new model represents a fully human system with the potential to detect clinically active targets and therapies for patients currently lacking effective treatment options.
Ancient signaling pathway may lead to modern cancer therapy
Brooke Emerling, PhD Professor Cancer Metabolism and Microenvironment Program Director and Associate Professor Cancer Metabolism and Microenvironment Program
Scientists at Sanford Burnham Prebys demonstrated for the first time that the activity of a group of lipid enzymes is connected to the regulation of an ancient cell signaling system called the hippo pathway, which is found in a wide variety of organisms and is known to help human organs grow and control their size.
The hippo pathway is often dysregulated in many cancers—including liver cancer—and has proven difficult to target with potential treatments. This new finding shows it may work to correct wayward hippo pathways in cancer cells through drugs that instead target the linked lipid enzymes.
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Jerold Chun, MD, PhD
Most previous research had indicated that the messenger RNA (mRNA) in brain bubbles carrying plans for proteins were chopped into too many shorter fragments to allow recipient cells to change their construction patterns. Scientists in the Chun lab at Sanford Burnham Prebys found the opposite to be true.
The research team, led by Jerold Chun, MD, PhD, identified more than 10,000 full-length mRNAs in brain vesicles traveling within the brain that had the potential to modify the behavior of recipient cells. Scientists said that vesicles in brain samples from patients with Alzheimer’s disease carried different mRNA that reflected genetic changes caused by the condition. It may be possible to leverage the bubbles as a targeted delivery system for new treatments.
Anne Bang, PhD Professor Degenerative Diseases Program Director, Cell Biology
Conrad Prebys Center for Chemical Genomics
Scientists have linked neuropsychiatric disorders, such as schizophrenia and autism, to changes in many genes involved in early brain development. However, more research is needed to understand how these gene variants influence the biological mechanisms that underlie these disorders.
Anne Bang, PhD, director of Cell Biology at the Institute’s Conrad Prebys Center for Chemical Genomics, is part of an interdisciplinary team of scientists and clinicians working to establish clearer connections between implicated genes and their effects on brain function and mental health. The group was awarded a $12.7 million grant from the California Institute for Regenerative Medicine (CIRM) to create a new research center to support this work.
Sanford Burnham Prebys scientists and their San Diego academic and industry collaborators posted results from the Phase 1 clinical testing of a nicotine addiction drug candidate called SBP-9330 in April 2024. The research team demonstrated that the drug candidate is safe and well tolerated in healthy smoking and nonsmoking human participants in the clinical trial.
The Conrad Prebys Center for Chemical Genomics has trained its team’s drug discovery expertise on the national epidemic of opioid addiction.
In 2021, fentanyl and other synthetic opioids accounted for nearly 71,000 of 107,000 fatal drug overdoses in the U.S. By comparison, in 1999, drug-involved overdose deaths totaled fewer than 20,000 among all ages and genders.
Like other addictive substances, opioids are intimately related to the brain’s dopamine-based reward system. Dopamine is a neurotransmitter that serves critical roles in memory, movement, mood and attention. Michael Jackson, PhD, director of the Center for Therapeutics Discovery and senior vice president of Drug Discovery and Development at the Prebys Center, and co-principal investigator Lawrence Barak, MD, PhD, at Duke University, have been developing a completely new class of drugs that works by targeting a receptor on neurons called neurotensin 1 receptor, or NTSR1, which regulates dopamine release.
The scientists were awarded $9 million from the NIH and NIDA to support toxicology studies and drug manufacturing efforts to prepare for a Phase II clinical trial. Preclinical research suggests future opportunities to explore the drug’s potential to treat other forms of addiction. Read More >>
The researchers received a $6.3 million award from NIH and the National Institute on Drug Abuse (NIDA) in 2023 to advance their addiction drug candidate, called SBI-810, to the clinic. SBI-810 is an improved version of SBI-533, which previously had been shown to modulate NTSR1 signaling and demonstrated robust efficacy in mouse models of addiction without adverse side effects.
The funding from the NIH and NIDA will be used to complete preclinical studies and initiate a Phase 1 clinical trial to evaluate safety in humans.
“The novel mechanism of action and broad efficacy of SBI-810 in preclinical models hold the promise of a truly new, first-in-class treatment for patients affected by addictive behaviors,” said Jackson.
Karen Ocorr, PhD
Discovering
Excessive thickening of the heart muscle—known as cardiac hypertrophy—is often the result of the heart trying to maintain proper blood flow while adapting to changes caused by other heart diseases, such as hypertension or heart valve malfunction. Hypertrophy in the heart’s left ventricle can lead to heart attacks, strokes and sudden cardiac death.
Scientists at Sanford Burnham Prebys, led by Karen Ocorr, PhD, showed that a protein called CREB-regulated transcription co-activator (CRTC) is likely one of the underlying causes of cardiac hypertrophy. Their findings open new possibilities for learning more about this protein and its related signaling molecules to find new targets for drugs to treat heart conditions. The researchers also noted links to muscular dystrophy and highlighted opportunities to expand the work beyond heart disease.
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Maximiliano D’Angelo, PhD
Assistant Professor Development, Aging and Regeneration Program Associate Professor Cancer Metabolism and Microenvironment Program
Nuclear pore complexes (NPCs) are channels composed of multiple proteins that ferry molecules in and out of the nucleus. By facilitating the nucleus’s imports and exports, NPCs regulate many critical cellular functions. A team of researchers at Sanford Burnham Prebys, led by Maximilano D'Angelo, PhD, uncovered Nup358, one of roughly 30 proteins that form the NPCs, as an early player in the development of myeloid cells.
Myeloid stem cells originate in bone marrow and mature into various adult blood cells. When these cells are not formed or working properly, myeloid disorders such as leukemias can occur. The researchers found that when they eliminated Nup358 in a mouse model, the animals experienced a severe loss of mature myeloid cells responsible for fighting pathogens and eliminating cancer.
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Nearly 30 years of discoveries led to federal approval of the first nonsteroidal drug to treat Duchenne muscular dystrophy.
Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy, affecting approximately one in 3,500 male births. DMD is linked to the X chromosome, which harbors the gene coding for the protein dystrophin, which protects muscles from degeneration after they contract. In boys with DMD, dystrophin-deficient muscles undergo progressive replacement of contractile myofibers with fibrotic scars and fatty infiltration.
“ ”I have been working from the very beginning of my research career to translate early, basic discoveries into a treatment for Duchenne muscular dystrophy and other muscle-wasting diseases.
Pier Lorenzo Puri, MD Director and Professor Development, Aging and Regeneration Program
There is currently no cure for DMD. For decades, steroids have been the only treatment, although their use is based on empirical evidence rather than targeting the underlying mechanisms of disease development. Longterm use of steroids also comes with side effects, such as weight gain, weakened bones, high blood pressure, and behavioral changes.
Seminal discoveries from Pier Lorenzo Puri, MD, revealed that targeting the balance of two enzymes with opposing activities—histone acetyltransferases and histone deacetylases—can regulate skeletal muscle growth and regeneration, potentially correcting the main pathological consequence of dystrophin deficiency: the replacement of contractile myofibers with fibrotic scars and fatty infiltration.
Puri’s group demonstrated that histone deacetylase inhibitors (HDACi) can prevent the formation of fibrotic scars and abnormal fatty deposits while reducing inflammation, promoting the formation of regenerating myofibers, and restoring functional parameters in preclinical models of DMD.
Puri’s group has focused on the preclinical and clinical development of the HDACi Givinostat, the first FDAapproved non-steroidal pharmacological treatment for DMD, now available on the market as DUVYZAT.
“Although Givinostat has shown promising therapeutic effects in boys with DMD, this compound does not fully realize the therapeutic potential of HDACi, which can be significantly improved,” said Puri. “Unfortunately, Givinostat is not well tolerated by boys with DMD at doses that could achieve optimal therapeutic effects, and therefore, it has been used at suboptimal doses,” he continued. “Moreover, Givinostat has been used in combination with steroids, and we observed in preclinical studies that this combination reduces the therapeutic efficacy of HDACi for DMD.”
Puri’s group is currently working on optimizing HDACibased treatment for DMD by testing next-generation HDACi in combination with new molecules and synergistic conditions identified in recent studies from his lab. The Puri lab is also exploring potential synergistic effects of HDACi with gene therapy and has recently discovered a new strategy to enhance the therapeutic potential of HDACi using extracellular vesicles as an alternative delivery approach.
“The goal of my lab’s current and future studies is to optimize HDACi-based treatments for DMD and potentially extend them to other muscular dystrophies by developing new compounds and combinations that achieve the highest therapeutic effect in patients.”
Thanks to a transformational investment from T. Denny Sanford, we recruited 13 new faculty scientists over the last 24 months, a rare and significant achievement.
Su-Chun
Zhang, MD, PhD
They come from the best basic research and academic institutions in the country, with broad and diverse research interests including computational biology, cancer, stem cells, aging, congenital heart defects, and next-generation imaging technologies.
Four of these new faculty members were recruited in 2024. We are pleased to introduce:
“Sanford Burnham Prebys offers a perfect environment for me to continue my venture in basic and translational neuroscience/ regenerative medicine research. I’m especially excited about the opportunities to turn laboratory discovery into treatments benefiting patients through close interaction with industries, which is my lifelong goal.”
Zhang joined Sanford Burnham Prebys in November 2024. He is an acclaimed stem cell scientist whose work has profoundly changed our understanding of how brain cells work and led to novel approaches for repairing or replacing neural cells in diseases like Alzheimer’s and Parkinson’s. Earlier this year, Zhang’s lab described producing the first 3D-printed brain tissue that can grow and function like typical brain tissue.
At Sanford Burnham Prebys, Zhang will continue his stem cell research as director of the Institute’s Center for Neurologic Diseases and in close proximity to BrainXell Therapeutics (pronounced brain cell ), a San Diego–based company he co-founded in 2022 to translate his research into personalized regenerative cell therapies for disorders of the nervous system.
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Debanjan Dhar, PhD Director and Professor Center for Neurologic Diseases
Jeanne And Gary Herberger Leadership Chair In Neuroscience Associate Professor Cancer Genome and Epigenetics Program
“During my postdoctoral fellowship, I was supported by the American Liver Foundation and had a chance to meet with cancer survivors. It was really inspirational, as I could see the real-world effect of scientific discoveries on people’s lives. I truly want to see what I work on reach patients who need new diagnostics and better treatments.”
Dhar joined Sanford Burnham Prebys in April 2024. He focuses on how lifestyle factors and genetic predispositions can lead to problematic changes in the liver, heart and kidneys. Specifically in the liver, growing deposits of fat in the tissue can lead over time to chronic inflammation and the accumulation of thickened scar tissue, a condition known as metabolic-associated steatohepatitis (MASH).
MASH is the second-leading cause of liver transplantation and a major risk factor for liver cancer. By studying the conversation among the liver, the immune system, heart and kidneys, Dhar hopes to discover signals that could be used to detect MASH and liver cancer much earlier, when they’re easier to treat.
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Douglas Sheffler, PhD
“One of my major projects involves a drug in clinical trials that targets a neuronal signaling pathway that underlies addictive behaviors, including tobacco use. If it is ultimately approved for the healthcare market, it would be a first-in-class oral therapeutic to help people quit smoking.”
Sheffler joined the faculty in August 2024 after serving as a research assistant professor in the lab of Nicholas Cosford, PhD. In his work, Sheffler has studied many facets of addiction, including addiction to nicotine. Smoking continues to be the leading cause of preventable deaths in the United States.
Cosford and Sheffler, in collaboration with colleagues at UC San Diego and Camino Pharma LLC, a San Diego–based biotechnology company Cosford co-founded, recently received a $9 million award from the National Institutes of Health (NIH) and National Institute on Drug Abuse (NIDA) to advance an investigational drug called SBP-9330 to Phase II clinical trials.
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Alessandro Vasciaveo, PhD Associate Professor Cancer Molecular Therapeutics Program Assistant Professor NCI-Designated Cancer Center
“I see my lab as a hub for innovation, where we’re working to deepen our understanding of biology and contribute to advancements in healthcare. We’re creating opportunities for scientists who want to be part of this exciting journey where computation meets biology to solve real-world problems.”
Vasciaveo joined Sanford Burnham Prebys in October 2024. He uses his training and experience as a scientist and engineer to advance knowledge of human biology through research. He has learned how to apply his expertise in computer science and computational biology toward the identification of novel treatments and cures for diseases.
Prior to joining the Institute, Vasciaveo studied conditions ranging from cancer to neurodegenerative diseases as an associate research scientist at Columbia University in New York City. He will largely focus on cancer research at Sanford Burnham Prebys, drawing on his experience with numerous projects in the field, including one that demonstrated how a computer algorithm may be able to identify treatments for individual patients affected by cancer.
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At Sanford Burnham Prebys, our mission extends beyond advancing the boundaries of biomedical research. We are also committed to fostering the next generation of scientific leaders.
Through our educational pathway, Sanford Burnham Prebys is creating a lasting impact in biomedical research and education. By providing hands–on learning opportunities and fostering a culture of inclusion, we are ensuring that the next generation of researchers is equipped to tackle the challenges of tomorrow’s biomedical landscape.
In addition to our Graduate School for Biomedical Studies and our renowned postdoctoral training program, the Institute’s education pathway has many entry points, providing critical training for high school students, undergraduate students, and post–baccalaureates.
Almost
150 postdocs and students mentored per year
70 graduates from the Graduate School for Biomedical Studies to date
More than 100 high school students have participated in Sanford Burnham Prebys summer programs
We are dedicated to preparing the next generation of scientists to lead groundbreaking discoveries.
Building on the Institute’s longstanding reputation for excellence in biomedical research, the program offers students a robust foundation in the sciences while fostering hands-on experience
through collaborative projects with faculty across various disciplines, including biology, chemistry, bioinformatics, and engineering. The program emphasizes innovation, personalized learning, and the use of cutting-edge technologies to accelerate scientific breakthroughs.
After five years in the pharmaceutical industry, Michael Alcaraz decided to pursue a graduate degree to transition into leading his own research group. Alcaraz, a fourthyear graduate student in the Sanford Burnham Prebys Graduate School of Biomedical Sciences, conducts research in the laboratory of Peter D. Adams, PhD, the director of the Cancer Genome and Epigenetics Program, with a focus on the mechanisms of aging.
Michael Alcaraz
Graduate Student
Peter Adams Lab
Alcaraz investigates the fundamental connections between aging and the increased risk of Alzheimer’s Disease (AD), the most common cause of dementia. His project is focused on the role of nicotinamide adenine dinucleotide (NAD+), an essential metabolite and building block for enzymes. NAD+ levels decrease with age in several tissues, including in the brains of humans and mouse models of AD. The decline of this important metabolite is associated with insufficient energy metabolism that is a major hallmark of AD.
As a Prebys fellow, Alcaraz had the opportunity to greater leverage the cutting–edge technologies and closely work with mentors to accelerate his knowledge and experience.
“In collaboration with the Conrad Prebys Center for Chemical Genomics, we will test a potential drug to promote production of NAD+ in the brain by activating a key enzyme involved in NAD+ biosynthesis, hopefully leading to increased brain metabolism and function in our mouse models,” said Alcaraz.
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The establishment of the Don Barach Memorial Fishman Award reflects our commitment to recognizing and empowering the next generation of scientific innovators. We believe that by supporting their work, we can help advance biomedical research and ultimately improve lives around the world.
Reena Horowitz Co-founder Fishman Fund
On September 17, six excellent postdoctoral researchers at Sanford Burnham Prebys were celebrated at the 23rd annual Fishman Awards ceremony for their groundbreaking biomedical research.
The Fishman Awards Fund, established in 2001 by Mary Bradley and Reena Horowitz to honor the Institute’s founders, supports early-career researchers with awards that include stipends, fellowships, and opportunities for professional development.
This year’s award ceremony was extra special, as two new awards were established in 2024: the Cynthia Schwartz Shenkman Research Excellence Award, given to a Sanford Burnham Prebys postdoc for their outstanding biomedical research contributions and demonstrated track record of research excellence; and the Don Barach Memorial Fishman Fund Award, to provide an outstanding researcher with funds for professional development opportunities.
The Don Barach Memorial Fishman Award was established by Sam and Reena Horowitz in honor of their beloved brother. Don was a caring and generous individual, always ready to lend a hand and offer support to those in need. His unwavering dedication to others left a lasting impact on all who knew him.
The 2024 awardees, whose research spans diverse fields, received recognition for their innovative contributions to science. These awardees exemplify the spirit of scientific innovation and collaboration fostered by the Fishman Award Fund, enabling the next generation of scientists to make strides in addressing some of the most pressing health challenges.
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“Dr. Nicoletti’s groundbreaking research and her commitment to improving patients' lives exemplifies the type of work we are proud to support. We are excited to see where her journey will lead, as she inspires the next generation of researchers and continues to make a difference.
Aaron and Cynthia Shenkman
Chiara Nicoletti, PhD, a postdoctoral associate at Sanford Burnham Prebys, was selected as the first recipient of the Cynthia Schwartz Shenkman Research Excellence Award.
This Fishman Award recognizes and rewards experienced postdoctoral fellows who have achieved a track record of research excellence. It is designed to help offset the cost of living in the region, and to benefit recipients as they prepare for the next step in their careers.
“For me, this award provides recognition of my hard work and that of my colleagues,” said Nicoletti. “It also acknowledges my mentor at Sanford Burnham Prebys and how he supports members of his laboratory.”
Nicoletti studies epigenetic patterns in skeletal muscle development and disease in the laboratory of Pier Lorenzo Puri, MD, director of the Development, Aging and Regeneration Program.
Nicoletti's accomplishments since joining Sanford Burnham Prebys and the Puri lab in 2018 include her work on dysferlinopathy, a rare form of muscular dystrophy. In collaboration with the laboratory of Jyoti K. Jaiswal, MSc, PhD, at Children’s National Research Institute in Washington, D.C., Nicoletti developed an atlas mapping how the disease causes changes in the transcription of genes in skeletal muscle and tracking how the disease progresses at the single-cell level. Dysferlinopathy patients suffer from progressive muscle loss in which muscle cells are replaced by fat and scar tissue. Understanding how this process takes place may accelerate research to find new drugs to improve muscle function.
When Nicoletti completes her postdoctoral fellowship, she would like to remain in academia as an independent principal investigator.
“As a bioinformatician with experience in bench research, I hope to bring different labs together to build interdisciplinary collaborations focused on how diverse biological systems and organs interact. I also would like to teach, as I feel it is our fundamental duty as scientists to train the next generation.”
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The LEAP (Lab Experience As Pathway ) program is a transformative initiative designed to address the systemic barriers that have historically kept students from underserved communities out of advanced STEM fields.
By providing hands-on research experience, professional development, mentorship, and access to essential networks, LEAP is helping to create a more inclusive and diverse future for STEM.
Launched in 2024 thanks to funding from the Prebys Foundation, the LEAP program serves as a one-year, paid, mentored postbaccalaureate opportunity. Its mission is clear: to level the playing field for recent college graduates, particularly those from underserved communities, by providing targeted preparation that enhances their competitiveness for prestigious STEM programs.
The impact of the LEAP program is already evident in the success stories of its participants. Sarina Safavi, a current participant, shared, “LEAP has been invaluable in helping me expand
my research experience and providing me with the professional development I need to apply for PhD programs. The support and guidance I’ve received have been instrumental in my journey toward a career in cancer research.”
Rachel Khoury, another participant, expressed her gratitude for the program’s impact: “The LEAP program has been an incredible opportunity to engage in cutting-edge research and develop critical thinking and project management skills. It’s preparing me for a future in neuropsychiatric disease research and equipping me with the knowledge and confidence I need for graduate school.”
By addressing the challenges faced by students from underserved communities and providing them with the preparation they need to succeed, LEAP is helping to create a more diverse, innovative, and equitable STEM landscape. As the program continues to grow, it will empower future generations of scientists and researchers to make meaningful contributions to the advancement of science and technology.
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The Institute unveiled its newly renovated auditorium in Building 12 during a special Town Hall and ribbon-cutting ceremony in August 2024.
The enhanced meeting space was renamed the Victor E. LaFave III Memorial Auditorium, or “the Vic,” in recognition of the generous support of the LaFave family.
The newly remodeled auditorium features a special symbol, a hidden golden turtle above the auditorium’s entrance, to represent Victor LaFave and his deep love for his family. His beloved wife, Sue LaFave, shared, “The tortoise and the turtle are universally seen as symbols of wisdom and knowledge, so it is quite fitting for representing the scientific discovery, education and learning that will take place in the Victor E. LaFave III Memorial Auditorium.”
The auditorium provides a prominent space for community engagement through new and ongoing events. The meeting
space will help the Institute connect with community members, alumni and research collaborators throughout the region and state, and will be a pivotal space for collaboration and exchanging ideas.
A new community engagement program called A Conversation About launched in the Vic on October 9, 2024, with more than
100 guests at the inaugural program. Taking inspiration from the Group of 12 and Friends, A Conversation About features expert panelists from the scientific and medical communities in dynamic discussions about the future of research and healthcare.
By bringing together community collaborators and clinicians with Sanford Burnham Prebys researchers, A Conversation About offers a unique perspective on how clinical research and practice can be used to inform fundamental and translational science.
“
What inspires us the most is the incredible impact that comes from supporting Sanford Burnham Prebys; making a difference in translating research into new and better treatments, benefiting humanity and the world.
Malin Burnham
”
If you let Sanford Burnham Prebys know about your intended bequest or other planned gift, we want to be able to thank you and recognize you as a member of the Legacy Circle, which honors those who have made a planned gift to Sanford Burnham Prebys.
Through legacy giving, you can support biomedical research, ensuring that your commitment continues to make a positive impact in perpetuity,
Roberta and Malin Burnham were among the first members of the Sanford Burnham Prebys Legacy Circle. In 1996, the La Jolla Cancer Research Foundation was renamed The Burnham Institute after Burnham joined with an anonymous donor to contribute $10 million.
“My philanthropic involvement with Sanford Burnham Prebys is among the most important work I’ve ever had the opportunity to be a part of. I value David Brenner’s leadership and vision in his role as president and CEO of Sanford Burnham Prebys. I think this is the most exciting and momentous time for the Institute, especially in the area of discovering new drugs and therapeutic treatments for patients and their families.
“'Community Before Self' has been the guiding principle of my life, and I am incredibly proud to see our family name associated with Sanford Burnham Prebys. As one of only seven NCI–Designated Basic Laboratory Cancer Centers in the nation, this Institute plays a vital role in the future of medical breakthroughs. Our region—and indeed our entire country—depends on its dedication to fundamental and translational research. Without this work, the discovery of new treatments and cures would not be possible.”
sliarakos@sbpdiscovery.org Join the Sanford Burnham Prebys Legacy Circle
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To learn more about the Sanford Burnham Prebys Legacy Circle, please call, email or write us to inform us of your planned gift. We would like to thank you for your generosity and include you as a member of the Legacy Circle.
For more information, please contact:
Sandy Liarakos, CFRE, CSPG Vice President of Philanthropy (858) 795-5035
Sanford Burnham Prebys has long punched above its weight. You can see and sense it in the tremendous progress made this past year in defining and refining our ambitious vision and mission, in new faculty recruitments, in community outreach, in published research, and in the growth of our graduate school and postdoctoral programs.
As chair of the Board of Trustees, it is my honor to support these efforts, to work with fellow trustees and the extraordinarily talented and dedicated faculty and staff who make Sanford Burnham Prebys what it is—a biomedical research powerhouse.
The purpose of medicine is to improve patients’ lives and well-being. As a physician, it means using the most effective treatments and cures. Places like Sanford Burnham Prebys are where it begins, with brilliant scientists making foundational discoveries that translate into new drugs and therapies. Your investment drives that process. It’s an investment in change, hope, lives improved and saved, and a healthier future for all of us.
Thank you for your support and partnership.
Donald Kearns, MD Board Chair
Sanford Burnham Prebys Board of Trustees
Our researchers, faculty, and trainees rely on people like you, who believe that advanced science will lead to new frontiers of human well-being.
Nine out of every 10 dollars invested at Sanford Burnham Prebys goes directly to support research, from developing new technologies and inspiring innovative thought to continuing a legacy of expertise that leads to real-world cures and a healthier future for all. With Sanford Burnham Prebys, a better world begins with you.
Through philanthropy, we translate science into health.
Invest in a healthier future
Your contribution to this effort will move us forward on our collective journey toward conquering the greatest threats to human health. Every gift is more than a donation; it invests in a healthier future. Make your gift online or contact the Office of Philanthropy at giving@sbpdiscovery.org or (858) 795-5070. Please visit sbpdiscovery.org/philanthropy.
Office of Philanthropy
10901 North Torrey Pines Road La Jolla, CA 92037 (858) 795-5070 giving@sbpdiscovery.org
Sanford Burnham Prebys’ Federal Tax Identification Number is 51-0197108. We are a tax-deductible 501(c)(3) nonprofit organization.
Get the latest news about our achievements in Discoveries—our monthly newsletter. Subscribers will also receive invitations to special events and programs, including the A Conversation About series.
Scan the code to subscribe, or visit sbpdiscovery.org/subscribe.
Donald Kearns, MD Chair President Emeritus, Rady Children’s Hospital-San Diego
Lori Moore Vice Chair Owner, Selma Investments Chair, Sharp Memorial Board of Trustees Sharp Healthcare Board of Directors
David A. Brenner, MD Professor, Cancer Metabolism and Microenvironment Program President and Chief Executive Officer Donald Bren Chief Executive Chair
Malin Burnham Honorary Trustee Former Chairman, The Burnham Companies
Katherine Chapin Co-Founder and General Partner, Chapin Ventures
Nicholas Cosford , PhD Professor, Cancer Molecular Therapeutics Program
Michael R. Cunningham, PhD Managing Director, Threshold Ventures
David W. Down Managing Partner, KPMG LLP – Retired
Daniel J. Epstein Founder, The ConAm Group
William Gerhart CEO, Kinevant Sciences
Douglas H. Obenshain Partner and Senior Vice President, Lockton Commercial Insurance
William A. Roper President, Roper Capital Company
Ted Roth Vice Chairman, ROTH Capital Partners CEO, ROTH Canada
T. Denny Sanford Honorary Trustee Chairman and CEO, United National Corp.
Lonna J. Williams Stolper Director, Rady Children’s Institute for Genomic Medicine
David L. Szekeres President, Connect Biopharma
Stuart Tanz CEO, United Income Properties Inc.
Scott LaFee Vice President, Communications
Sandy Liarakos, CFRE, CSPG Vice President, Philanthropy
Monika Wert-Parkinson Senior Officer, Stewardship and Donor Relations
