DISCOVERIES

November 2024

Stem cell pioneer Su-Chun Zhang joins Sanford Burnham Prebys

Acclaimed stem cell scientist Su-Chun Zhang, MD, PhD, whose work has profoundly changed both understanding of how brain cells work and led to novel approaches for repairing or replacing them in diseases like Alzheimer’s and Parkinson’s, is joining the faculty at Sanford Burnham Prebys.

“During his entire career, Dr. Zhang has embodied the best of both basic and translational science,” said David Brenner, MD, president and chief executive officer at Sanford Burnham Prebys. “His research has foundationally advanced our understanding of how brain cells are born, develop and function, often breaking new ground, and he has then turned those discoveries into new tools and potential therapies for truly difficult and daunting neurological diseases like Alzheimer’s and Parkinson’s.”

Zhang comes from the Waisman Center at the University of Wisconsin-Madison, long acclaimed for its stem cell research. Zhang, who held the title of Steenbock Professor of Behavioral and Neural Sciences, worked at the Waisman Center for 23 years, officially retiring in August 2024.

Decoding and debugging biological programs for a healthier future

New Sanford Burnham Prebys scientist Alessandro Vasciaveo aims to leverage computational expertise to deepen understanding of diseases and develop innovative solutions for improving health by integrating experimental biology with advanced machine learning.

Protein superfamily crucial to the immune system experiences Broadway-style revival

More than 25 years after targeting a member of this superfamily of proteins led to groundbreaking treatments for several autoimmune diseases including rheumatoid arthritis and Crohn’s disease, San Diego scientists note a resurgence of interest in research to find related new drug candidates.

The implastic nature of plastic culture

There is an art (and science) to creating cell culture models that reflect the complexities of disease. Such models have long been indispensable to parsing the underlying mechanisms of pathology and to preclinical drug discovery.

But art, writes Kevin Tharp, PhD, assistant professor in the Cancer Metabolism and Microenvironment Program, doesn’t always imitate life — at least not when it comes to finding effective cancer therapeutics.

“Just like a machine-learning algorithm trained on irrelevant datasets, efforts to discover anticancer therapeutics are limited by the models we use,” Tharp writes in the British Journal of Pharmacology. “Our drug discovery pipeline works incredibly well but is applied to models that poorly recapitulate in vivo physiology. This may be why drug discovery approaches efficiently identify drugs that work in the context tested and yet often fail to translate into clinical success.”

Philanthropy

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Community

Decades of dedication led to FDA approval of a new treatment for Duchenne Muscular Dystrophy

Nearly 30 years of discoveries by a Sanford Burnham Prebys scientist and collaborators lead to federal approval of the first non-steroidal drug to treat Duchenne muscular dystrophy.

For one San Diegan scientist at Sanford Burnham Prebys, the March 2024 federal approval of a new drug to treat Duchenne muscular dystrophy (DMD) marked a milestone in three decades of studying muscle regeneration and muscle-wasting diseases.

The compound, called Givinostat and marketed as DUVYZAT™, is a histone deacetylase inhibitor (HDACi) and was approved by the FDA for the treatment of boys with DMD.

"I have been working from the very beginning of my research career to translate early, basic discoveries into a treatment for DMD,” said Pier Lorenzo Puri, MD, director and professor in the Development, Aging and Regeneration Program at Sanford Burnham Prebys. “The lack of effective treatments for boys with DMD has left families and patients hopeless since the discovery of this disease. Witnessing the progression of such a disease without any option to counter its progression is cruel and I felt the urgency to help these people.”

Seven questions for FDA advisor Evan Snyder

Sanford Burnham Prebys physician-scientist advises the FDA on cell-based therapeutics, tissue engineering and gene therapies.

Sanford Burnham Prebys physician-scientist Evan Y. Snyder, MD, PhD, was reappointed to the Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) in the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA). He is serving a four-year term from August 30, 2024, to March 31, 2028.

We sat down with Snyder to learn more about the committee, his advisory role and the importance of safeguards for new therapies.

Honoring Hud Freeze and colleagues for literally writing the book on glycobiology

Glycobiology is the study of the structure, biosynthesis and biology of glycans — carbohydrates or sugars that cover the surfaces of all cells and many proteins.

In recent years, understanding and recognition of the importance of glycans has grown dramatically.

They are, among other things, critical to the maintenance of cell and tissue structure and integrity. Their dysfunction is linked to a growing list of human diseases. They have played surprising roles in human evolution.

Beginning in 1999, a cohort of researchers working in the emerging field of glycobiology created the first textbook on the subject: “Essentials of Glycobiology.” The fourth edition was published in 2022.

The Society for Glycobiology recently honored the editors for their ongoing work with its 2024 Distinguished Service Award. Among the honorees, Hudson Freeze, PhD, director of the Sanford Children’s Health Research Center at Sanford Burnham Prebys and a renowned expert in congenital disorders of glycosylation, a group of rare metabolic disorders that primarily afflict children.

Demystifying Día de los Muertos at Sanford Burnham Prebys

The Institute honored the holiday with a special event that connected participants with the tradition’s customs and cuisine.

On Wednesday, November 6, 2024, Chairmen’s Hall on the Sanford Burnham Prebys campus was transformed by the sights, smells and sounds of Día de los Muertos.

As attendees snacked on sweet breads and sipped Mexican hot chocolate and spiced coffee, they were encouraged to learn more about the holiday through a gallery exhibit featuring posters discussing the tradition’s origin and symbolism.

In the News

Jamey Marth interviewed by The Scientist

The Sanford Burnham Prebys scientist discussed the Cre-loxP recombination system, which continues to be a mainstay genetic engineering technology.

“Techniques come and go with new technology, it’s just like night and day,” said Jamey Marth, PhD, professor and director of the Immunity and Pathogenesis Program at Sanford Burnham Prebys during his interview with The Scientist regarding his lab’s contributions to a genetic engineering technique that has stood the test of time.

“So, when you have a technique that’s lasted 30 years with no replacement technology, I think that’s kind of remarkable.”

Science in Pictures

A chromosome from Drosophila melanogaster (fruit fly) salivary glands, using Brightfield microscopy. Fruit flies are model organisms, sharing 75% of the genes that cause disease in humans.

Image courtesy of Earl Nishiguchi, Kauai Community College and Nikon Small World.