A study published in the Proceedings of the National Academy of Sciences identifies Neuraminidase 3 (Neu3) as a key enzyme responsible for colitis, a chronic digestive disease. Inhibiting Neu3 with Relenza breaks the chain of inflammation, and augmenting intestinal alkaline phosphatase appears to be equally beneficial.
Scientists at Sanford Burnham Prebys have gained insight into the process of autophagy, where cells degrade and recycle cellular components. The study reveals that a chemical modification helps direct the transport of autophagosomes to cellular recycling plants, potentially leading to new targets for age-related diseases.
A preclinical study supports AAV8-TNAP-D10 as a single-dose treatment for hypophosphatasia, extending the life span of mice with minimal side effects. The therapy may provide an alternative to current medication injections, benefiting patients with severe skeletal and dental abnormalities.
A study identified a tumor marker that can predict which breast cancer patients will benefit from early HER2 targeted therapy, helping to avoid disease relapse. The research found that low or absent MLH1 and PMS2 gene expression may indicate an increased risk of death from the disease.
Scientists at Sanford Burnham Prebys Medical Discovery Institute identified lipid kinases as essential for balancing cellular metabolism and promoting overall health. The findings support targeting these proteins as a promising approach to treating diseases like cancer.
Victoria Blaho, Ph.D., assistant professor at Sanford Burnham Prebys, received the first-ever Lina M. Obeid Award for her work on sphingolipids and their role in immune function, cancer, neurodegeneration, metabolic disorders, and cardiovascular disease.
Researchers at Sanford Burnham Prebys have gained atomic-level insights into how PLEKHA7 interacts with the cell membrane to regulate intercellular communications. The study identifies hotspots within PLEKHA7 as potential targets for cancer drugs, particularly in advanced colon, breast, and ovarian cancers.
Researchers at Sanford Burnham Prebys have identified a set of human genes that help control SARS-CoV-2 infection, including interferon-stimulated genes (ISGs) that inhibit viral replication. The study provides new insights into the biology of the virus and suggests possible therapeutic options.
Researchers at Sanford Burnham Prebys Medical Discovery Institute have identified two potential drug candidates for a deadly AML subtype in children. JAK inhibitors and Mepron showed promise in slowing human AML cell growth and extending survival in mice with the CALM-AF10 mutation.
Researchers have identified a small molecule drug candidate that targets the uptake of glutamine in cancer cells, slowing the growth of melanoma and other cancers. The study, published in Molecular Cancer Therapeutics, offers an exciting new therapeutic approach for treating tumors addicted to glutamine.
Researchers at Sanford Burnham Prebys found that blocking macropinocytosis in the thick tissue surrounding a pancreatic tumor slows tumor growth. The study suggests that macropinocytosis is an important driver of pancreatic cancer growth and provides a potential therapeutic target.
A recent study published in Nature suggests that the leprosy drug clofazimine exhibits potent antiviral activities against SARS-CoV-2 and prevents exaggerated inflammatory response associated with severe COVID-19. Clofazimine could be a potential at-home treatment for people who test positive for COVID-19 but are not hospitalized.
Researchers have identified two drugs that work synergistically to promote significant anti-leukemia activity when combined, but only weakly effective when used alone. The study found that the combination of MDM2 inhibitors and BET inhibitors unleashes the full anti-cancer activity of p53 in AML patients.
Researchers at Sanford Burnham Prebys Medical Discovery Institute identified MDA-5 as the primary sensor in human lungs that detects SARS-CoV-2. The protein recognizes replicating viruses and activates interferon, a key player in the body's defense against viral invasion.
Fruit flies exposed to microgravity for three weeks showed profound changes to their hearts, including smaller size and reduced contractility. These findings suggest that astronauts may benefit from protective measures to keep their hearts healthy during long-duration space travel.
Scientists at Sanford Burnham Prebys Medical Discovery Institute have created a drug that can lure stem cells to damaged tissue, improving treatment efficacy for neurological disorders. The discovery could expand the use of stem cell therapies to new conditions such as heart disease or arthritis.
A clinical trial is planned to evaluate personalized drug screens for medulloblastoma, a deadly form of pediatric brain cancer. The approach uses tumor cells from biopsies and has been shown to identify effective therapies that cannot be predicted using other methods.
Faculty members Peter Adams and Jerold Chun receive prestigious awards for groundbreaking research on human aging and Alzheimer's disease. The grants will support innovative approaches to healthy aging, prevent degenerative disease and cancer.
Researchers at Sanford Burnham Prebys Medical Discovery Institute have discovered a way to selectively kill aggressive cancer cells by blocking the construction of nuclear pore complexes. This breakthrough could lead to new treatments for deadly tumors such as melanoma, leukemia, and colorectal cancer.
A $11.4 million NIH grant has funded the advancement of a novel drug candidate, SBP-9330, targeting a neuronal signaling pathway underlying addictive behaviors to treat nicotine addiction. The drug is expected to be effective in reducing nicotine self-administration and may broaden its indication to other types of addiction.
Researchers have found a way to restore muscle regeneration and prevent scarring in Duchenne muscular dystrophy by correcting the content of extracellular vesicles. This novel approach uses pharmacologically corrected vesicles to deliver HDAC inhibitors directly to muscles, overcoming systemic adverse effects.
Researchers at Sanford Burnham Prebys aim to develop new therapies for schizophrenia, depression, anxiety and substance abuse by identifying potential drug targets in 'orphan' receptors. The team has created an innovative platform called LIFT to screen small molecules and validate these receptors as bonafide drug targets.
Researchers at Sanford Burnham Prebys Medical Discovery Institute have discovered a novel drug target, PPP1R1B, that stops the spread of pancreatic cancer in mice. Increased levels of this protein have been found in tumor samples from people with metastatic pancreatic cancer, suggesting it has therapeutic potential.
A Nature study identified 21 existing drugs that stop SARS-CoV-2 replication, including four that work synergistically with remdesivir. These compounds have potential for COVID-19 treatment and could provide affordable alternatives if the virus becomes drug-resistant.
Researchers will test two existing drugs against SARS-CoV-2-infected mini lungs in a dish to find solutions for COVID-19. The grant aims to advance promising drug candidates and improve patient care.
Researchers at Sanford Burnham Prebys Medical Discovery Institute have identified vitronectin, a blood protein, as a promising drug target for dry age-related macular degeneration. The study's findings suggest that vitronectin drives the formation of abnormal deposits that cause progressive vision loss.
Scientists at Sanford Burnham Prebys discovered that PRMT5 inhibitors can sensitize unresponsive melanoma to immune checkpoint therapy, enhancing antigen presentation and innate immunity. The study suggests that these inhibitors may also be effective in tumors of other types, providing a potential breakthrough for cancer treatment.
Brooke Emerling receives a four-year grant to study targeting tumors with p53 gene mutations, which are present in most human cancers. Her research aims to develop new approaches to eliminating cancer as a major health problem.
Researchers identified mutations in the CNOT1 gene that affect brain development and impair memory and learning, suggesting potential therapeutic benefits. The study also revealed interactions between CNOT1 and known autism spectrum disorder genes, paving the way for further research.
Scientists at Sanford Burnham Prebys have developed a new drug called Ciapavir that effectively reactivates dormant human immunodeficiency virus (HIV) without overactivating the immune system. This approach, called 'shock and kill,' aims to create a functional HIV cure by eliminating all pockets of dormant HIV.
Scientists at Sanford Burnham Prebys Medical Discovery Institute have shown that a protein called Slug regulates cell movement and can be inhibited to suppress pancreatic cancer metastasis. The study also identified two druggable targets, ERK and eIF2 alpha, which hold promise as treatments for the deadly disease.
Scientists have mapped proinsulin's vast network of interacting proteins, revealing a key player in proper folding and insulin production. Boosting PRDX4 levels may offer a novel therapeutic approach to improving type 2 diabetes treatment.
Researchers at Sanford Burnham Prebys identified a promising immunotherapy combination that eradicates medulloblastoma in mice. By adding tumor necrosis factor (TNF), the treatment removes the 'invisibility cloak' from p53-mutant tumors, allowing them to be detected and destroyed by the immune system.
Scientists at Sanford Burnham Prebys 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. The researchers found that rats with larger penumbra areas surrounding brain injury, which received human neur...
The Department of Defense has awarded $10M to Sanford Burnham Prebys to develop broad-spectrum antivirals that can combat multiple respiratory viruses. The research aims to provide safe and effective therapies for U.S. military forces and the nation, reducing secondary infections and spread.
Scientists at Sanford Burnham Prebys have found a new way to kill pancreatic cancer cells by disrupting their pH equilibrium. Suppressing NHE7 lowers the cell's cytoplasmic pH, triggering cell death in both human and mouse tumors. This approach could provide a new therapeutic avenue for treating pancreatic cancer.
Researchers at Sanford Burnham Prebys Medical Discovery Institute and Harvard University discovered that mitochondria trigger senescence in cells by communicating with the cell's nucleus. They identified an FDA-approved drug that helped suppress the damaging effects of senescence in cells and mice, providing a potential treatment for a...
Researchers at Sanford Burnham Prebys Medical Discovery Institute found that prebiotics mucin and inulin slowed melanoma growth in mice by enhancing anti-tumor immunity. The study suggests a potential benefit of prebiotics in treating cancer or augmenting current therapies.
Researchers found that cells from children with NGLY1 deficiency lack sufficient water channel proteins called aquaporins, leading to inability to produce tears and other wide-ranging symptoms. The discovery opens new avenues for finding therapies to treat the disorder.
Scientists at Sanford Burnham Prebys have discovered a new way to boost the immune system's ability to fight cancer. The study found that a PD-1 inhibitor can be used to treat tumors that currently do not respond to this therapy, when administered in mice lacking the Siah2 gene.
Scientists at Sanford Burnham Prebys Medical Discovery Institute found that excess levels of a protein, p62 or SQSTM1, increase lifespan in worms. This discovery could help uncover treatments for age-related conditions like Alzheimer's disease.
Scientists at Sanford Burnham Prebys Medical Discovery Institute 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. The study provides rationale for an immediate clinical trial evaluating the 2 therapies
Researchers aim to develop a new compound, SBI-553, to treat opioid-use disorders with limited effectiveness. The compound has shown promise in preclinical testing, reducing addictive behaviors and improving safety profiles.
Scientists developed a machine-learning algorithm to automate high-throughput screens of epigenetic medicines, identifying potential treatments for glioblastoma and other diseases. The approach, called Microscopic Imaging of Epigenetic Landscapes (MIEL), detects active drugs and spots epigenetic changes across multiple cell lines.
Researchers have identified a new disease mechanism and potential molecular drug target to protect premature newborns from developing post-hemorrhagic hydrocephalus. Treating mice with compounds blocking the LPA receptor prevented excess fluid accumulation in the brain.
The CIRM grant will enable pre-investigational new drug studies for neural stem cells as a potential treatment for perinatal hypoxic-ischemic brain injury, which can cause cerebral palsy and other neurological disorders. The project aims to protect at-risk brain cells and potentially restore balance to the injured region.
Researchers have identified a signaling pathway regulating macropinocytosis in pancreatic tumors, which could lead to personalized treatments. The study reveals that tumors can dynamically adjust their nutrient uptake, with some 'dialing up' or 'dialing down' macropinocytosis based on glutamine availability.
Researchers have developed a method to generate natural-looking hair that grows through the skin using human induced pluripotent stem cells. This technology has the potential to treat hair loss in millions of people worldwide.
Scientists from Sanford Burnham Prebys have revealed a new mechanism for ALS' pathogenesis and suggest that modulating membralin has potential in ALS therapy. A membralin-boosting gene therapy extended the survival of mice with ALS-like symptoms, providing an important new perspective into the disease.
Scientists have identified a promising compound, PHT-7.3, that shrinks KRAS-driven tumors in mice by targeting the protein's partner, Cnksr1. The study provides a potential treatment for KRAS-positive cancers, which currently have no effective therapy.