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Scientists at Sanford Burnham Prebys Medical Discovery Institute and Radboud University Medical Center in the Netherlands have identified mutations in a gene called CNOT1 that affect brain development and impair memory and learning. The research, published in The American Journal of Human Genetics, also revealed that CNOT1 interacts with several known autism spectrum disorder (ASD) genes, opening new research avenues for the condition.

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Scientists at Sanford Burnham Prebys Medical Discovery Institute have created a next-generation drug called Ciapavir (SBI-0953294) that is effective at reactivating dormant human immunodeficiency virus (HIV). The research, published in Cell Reports Medicine, aims to create a functional HIV cure by activating and then eliminating all pockets of dormant HIV—an approach called “shock and kill.”

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Scientists at Sanford Burnham Prebys Medical Discovery Institute have shown that pancreatic cancer metastasis—when tumor cells gain the deadly ability to migrate to new parts of the body—can be suppressed by inhibiting a protein called Slug that regulates cell movement. The study, published in the Journal of Experimental Medicine, also revealed two druggable targets that interact with Slug and hold promise as treatments that may stop the spread of pancreatic cancer.

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Scientists at Sanford Burnham Prebys Medical Discovery Institute and the Centro de Biología Molecular Severo Ochoa in Spain have created a test that determines which children with CAD deficiency—a rare metabolic disease—are likely to benefit from receiving uridine, a nutritional supplement that has dramatically improved the lives of other children with the condition. The study was published in Genetics in Medicine.

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Scientists at Sanford Burnham Prebys Medical Discovery Institute have mapped for the first time the vast network of proteins that interact with proinsulin, the protein the body normally processes into insulin. The study, published in Diabetes, also revealed one protein—called PRDX4—that may be essential for proinsulin folding and insulin production. The research suggests that boosting PRDX4 levels may be a novel therapeutic approach to improving the health of people with diabetes.

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Scientists at Sanford Burnham Prebys have discovered that combining immunotherapy with a drug called tumor necrosis factor (TNF) eradicated a deadly type of pediatric brain tumor in mice. The discovery, published in Nature Neuroscience, is expected to lead to a clinical trial to test the benefits of the treatment in patients. The findings also hold implications for other cancers that do not respond to immunotherapy.

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Scientists at Sanford Burnham Prebys Medical Discovery Institute and Loma Linda University Health have demonstrated the promise of applying magnetic resonance imaging (MRI) to predict the efficacy of using human neural stem cells to treat a brain injury—a first-ever “biomarker” for regenerative medicine that could help personalize stem cell treatments for neurological disorders and improve efficacy. The study was published in Cell Reports.

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Sanford Burnham Prebys Medical Discovery Institute is pleased to announce that Sumit Chanda, Ph.D., has received a $10.2 million, four-year grant from the Department of Defense to develop and advance broad-spectrum antivirals for respiratory diseases. The award aims to provide U.S. military forces and the nation with safe, effective and innovative therapies that combat multiple types of respiratory viruses.

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Scientists at Sanford Burnham Prebys Medical Discovery Institute, the University of Hong Kong, Scripps Research, UC San Diego School of Medicine, the Icahn School of Medicine at Mount Sinai and UCLA have identified 30 existing drugs that stop the replication of SARS-CoV-2, the virus that causes COVID-19. The study was placed on bioRxiv (pronounced “bio-Archive”), an open-access distribution service for preprints of life science research.

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Scientists at Sanford Burnham Prebys have found a new way to kill pancreatic cancer cells by disrupting their pH equilibrium. The study, published in Cancer Discovery, reports how depleting an ion transport protein lowers the pH to a point that compromises pancreatic cancer cell growth.

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Sanford Burnham Prebys Medical Discovery Institute today announced that Karen Haynes, Ph.D., president emerita at California State University San Marcos, and Donald Kearns, M.D., president emeritus at Rady Children’s Hospital-San Diego, have been appointed to the Institute’s Board of Trustees.

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Scientists at Sanford Burnham Prebys Medical Discovery Institute and Harvard University have discovered that mitochondria trigger senescence, the sleep-like state of aged cells, through communication with the cell’s nucleus—and identified an FDA-approved drug that helped suppress the damaging effects of the condition in cells and mice. The discovery, published in Genes & Development, could lead to treatments that promote healthy aging or prevent age-associated diseases such as cancer, Alzheimer’s disease, heart disease and more.

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Scientists at Sanford Burnham Prebys Medical Discovery Institute have shown that two prebiotics, mucin and inulin, slowed the growth of melanoma in mice by boosting the immune system’s ability to fight cancer. The study, published today in Cell Reports, provides further evidence that gut microbes have a role in shaping the immune response to cancer, and supports efforts to target the gut microbiome to enhance the efficacy of cancer therapy.

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Scientists at Sanford Burnham Prebys Medical Discovery Institute have shown that cells from children with NGLY1 deficiency—a rare disorder first described in 2012—lack sufficient water channel proteins called aquaporins. The discovery was published in Cell Reports and may help explain the disorder’s wide-ranging symptoms—including the inability to produce tears, seizures and developmental delays—and opens new avenues to find therapies to treat the disorder.

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Scientists at Sanford Burnham Prebys have identified a new way to boost the immune system’s ability to fight cancer. The study used a mouse model to identify the importance of the Siah2 protein in the control of immune cells called T regulatory cells (Tregs), which limit the effectiveness of currently used immunotherapies. The research, which offers a new avenue to pursue immunotherapy in cases where the treatment fails, was published today in Nature Communications.

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Scientists at Sanford Burnham Prebys Medical Discovery Institute have shown that worms live longer lives if they produce excess levels of a protein, p62, which recognizes toxic cell proteins that are tagged for destruction. The discovery, published in Nature Communications, could help uncover treatments for age-related conditions, such as Alzheimer’s disease, which are often caused by accumulation of misfolded proteins.

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Scientists at Sanford Burnham Prebys Medical Discovery Institute have identified a combination of two anti-cancer compounds that shrank pancreatic tumors in mice—supporting the immediate evaluation of the drugs in a clinical trial. U.S. Food and Drug Administration (FDA)–approved versions of the compounds are used today to treat certain leukemias and solid tumors, including melanoma. The study was published in Nature Cell Biology.

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The National Institute on Drug Abuse (NIDA), part of the National Institutes of Health (NIH), awarded a $3.58 million grant to Sanford Burnham Prebys scientist Anthony Pinkerton, Ph.D., to advance a potential treatment for opioid-use disorders, called SBI-553.

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Scientists at Sanford Burnham Prebys Medical Discovery Institute have developed a machine-learning algorithm that gleans information from microscope images—allowing for high-throughput epigenetic drug screens that could unlock new treatments for cancer, heart disease, mental illness and more. The study was published in eLife.

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Premature babies have delicate brain tissue that is prone to bleeding and can result in post-hemorrhagic hydrocephalus, a dangerous condition that leads to excess fluid accumulation and brain dysfunction. Now, scientists from Sanford Burnham Prebys Medical Discovery Institute have identified a new disease mechanism and potential molecular drug target that may protect premature newborns from developing the brain disorder. The study was published in Science Advances.