In the latest issue of Discoveries:

🔬 Drug discovery: Sanford Burnham Prebys researchers join a $31.7 million ARPA-H–funded project using advanced computational models to predict drug safety earlier, potentially accelerating development and reducing reliance on animal models.

🧫 Alexander disease: New research reveals how a protein helps maintain mitochondria—the cell’s power plants—offering insight into the neurodegeneration behind this rare genetic disorder.

🧠 Muscle regeneration: Scientists uncover how a key protein supports functional muscle stem cells, pointing to potential therapeutic strategies for age-related muscle loss.

Sanford Burnham Prebys joins consortium to rewrite the realities of drug discovery

On December 4, 2025, the Advanced Research Projects Agency for Health (ARPA-H), part of the U.S. Department of Health and Human Services, announced a multi-institution, $31.7 million contract to fund a project called Pharmacological Research and Evaluation through Digital Integration and Clinical Trial Simulation, or PREDICTS. The goal is to trim years from drug development, reduce the need for laboratory animal models and catch safety problems with new drugs before they are tested in people.

Researchers at Sanford Burnham Prebys will be part of this new, ambitious project to transform the drug discovery process by developing computational models to predict human toxicity, potentially cutting years off development timelines and reducing costs.

Protein puppeteer pulls muscle stem cells’ strings

As we age, the muscles we rely on for daily activities tend to become less reliable. With enough decline, even normal movements such as getting out of bed become risky. Low muscle mass in the elderly—known as sarcopenia—is a major concern for maintaining the quality of life in an aging population.

A new study from scientists in the Sacco lab shows how a protein in the jelly-like substance between muscle cells promotes a thriving community of functional muscle stem cells needed for efficient muscle regeneration. The researchers also found that levels of this protein decrease with age, leading to a decline in muscle stem cells and muscle repair. Restoring appropriate amounts of this protein may be a therapeutic strategy for age-related muscle loss.

Structural support protein takes center stage in maintaining mitochondria

Scientists in the Zhang lab and an international team of collaborators published findings in the Proceedings of the National Academy of Science demonstrating a new role for a ropelike protein serving as one of the structural components that gives shape and structure to a cell and all its constituent parts.

The scientists showed that this protein plays an important part in maintaining the mitochondria serving as the power plants of our cells. The researchers also found that mutations in the gene carrying the code for this protein disrupt the delicate balance of mitochondria splitting and merging to meet the cells’ energy needs. This may provide a fundamental explanation for the neurodegeneration caused by Alexander disease, a rare genetic disorder caused by mutations in this gene.

Season of giving to support scientists

As we close the year, your generosity fuels discovery. Philanthropic support accelerates scientific research, trains our next generation of scientists, sparks innovative ideas and advances new therapeutics that bring hope to patients. Every gift helps transform bold science into real-world impact, improving lives today and shaping a healthier future.

For more information about making a gift, please contact the Sanford Burnham Prebys Philanthropy office at giving@sbpdiscovery.org or call (858) 795-5089.

Sanford Burnham Prebys Medical Discovery Institute is a 501(c)(3) nonprofit organization. Tax ID: 33-0319504.

Institute employee holiday “socktail” party provides thousands of pairs of socks for those in need

Curebound grants to advance therapeutic treatments and cures

Ten scientists at Sanford Burnham Prebys Medical Discovery Institute were awarded eight grants from Curebound, a San Diego–based philanthropic organization that raises and invests funds in promising cancer research to accelerate the development of treatments.

The grants were part of 23 awards totaling $8.5 million.

Opinion: U.S. biomedical research relies on philanthropy, large and small

The National Institutes of Health is the nation’s largest supporter of basic research, but it’s important to remember it is not the only one.

In his latest essay for The San Diego Union-Tribune, Dr. Brenner discusses the particular history and importance of private philanthropy in supporting biomedical research.

In this structured illumination micrograph, a crawling cell is shown with DNA in blue and actin filaments in pink. Image courtesy of Dylan T. Burnette, Vanderbilt University.