Researchers used gene editing to restore hearing in adult mice with a form of inherited deafness called autosomal dominant deafness-50 (DFNA50). The approach involved shutting down a damaged copy of the microRNA (miRNA) gene, which enabled the animals to regain hearing. This method may eventually lead to potential treatments for inheri...
Researchers have shown that a single normal copy of a defective gene can prevent cysts in polycystic kidney disease. Additionally, they found that a type of drug called a glycoside can sidestep the effects of the defective gene and prevent cyst formation.
Researchers used 3-D models to show that gene activity differs based on a cell's location within a tumor, influencing its role in cancer biology. The study could lead to more precise therapies by targeting specific areas of tumors.
Researchers found an enzyme in liver cancer cells that can convert a group of compounds into anticancer drugs, killing cells and reducing disease in animals. The enzyme SULT1A1 activates compounds like YC-1, making them toxic to tumor cells.
A new approach combining organ-in-a-dish and organ-on-a-chip technologies reveals the crucial role of glucose in forming polycystic kidney disease (PKD) cysts. Researchers found that PKD cysts absorb glucose, leading to rapid expansion and potentially life-threatening complications.
Researchers analyzed de-identified EHR data in the National COVID Cohort Collaborative to find patterns that can help identify individuals with long COVID. The study identified over 200,000 likely cases as of May 2022 and found common features such as new medications, doctor visits, and symptoms.
Scientists have created a 3D model of two rare neuromuscular diseases using a tissue chip platform. The model mimics the biology of chronic inflammatory demyelinating polyneuropathy and multifocal motor neuropathy, allowing researchers to study how a drug could potentially treat these devastating conditions.
A new study by NIH's NCATS suggests that people with rare diseases face significantly higher healthcare costs, often three to five times those without a rare disease. The study estimates total yearly direct medical costs for approximately 25-30 million individuals in the US could be around $400 billion.
Researchers at NIH have devised a four-part small-molecule cocktail, CEPT, that protects induced pluripotent stem cells from stress and maintains normal stem cell structure and function. The cocktail enhances the potential therapeutic uses of stem cells in treating diseases like diabetes, Parkinson's disease, and spinal cord injury.
A large randomized Phase 3 clinical trial will test existing prescription and over-the-counter medications for treating mild-to-moderate COVID-19 symptoms. The ACTIV-6 trial aims to provide evidence-based treatment options for adult patients with mild-to-moderate symptoms, reducing the need for hospitalization.
Researchers identified a novel drug duo that exploits a weakness in SCLC's reproduction process, increasing replication stress and damaging DNA. The berzosertib-topotecan combination helped improve symptoms in over one-third of patients in a clinical trial.
Researchers developed a new method called biological activity-based modeling (BABM) to identify promising drug compounds. BABM uses a compound's activity patterns to predict its effectiveness against a new target or in a new drug assay.
The National Institutes of Health has awarded grants to support researchers' efforts in using tiny, bioengineered models of human tissues and organ systems to study diseases and test drugs. The goal is to develop ways to predict which patients are most likely to benefit from an investigational therapy prior to initiating clinical trials.
The National Institutes of Health (NIH) is expanding two randomized, placebo-controlled clinical trials to evaluate the effectiveness and safety of convalescent plasma as a treatment for patients hospitalized with COVID-19. The trials aim to assess clinical improvement measures and resource needs, such as ventilators.
A team of scientists has created a fluorescent nanoparticle probe that mimics how SARS-CoV-2 infects cells, allowing for rapid testing of potential therapeutic agents. The probe's ability to track viral attachment and effects on cells in real-time makes it a powerful tool for drug discovery.
The National Institutes of Health has launched a centralized analytics platform to study COVID-19 patient data, aiming to develop effective treatments and improve clinical care. The platform will analyze health risk factors, identify potentially effective treatments, and enable researchers to explore connections and patterns in the data.
Researchers identified a panobinostat-marizomib combination that effectively kills childhood brain cancer cells and counters genetic mutations. The study uncovered a previously unrecognized vulnerability in cancer cells, which may lead to new strategies against the disease.
The NIH has awarded $31 million in grants to support consortia studying rare diseases, with a focus on clinical research and collaboration. The funding aims to develop improved approaches for diagnosis and treatment, ultimately moving potential treatments closer to the clinic.
Scientists have identified a cellular pathway that allows acute myeloid leukemia cells to evade the effects of drugs. They then engineered a compound that targets this pathway, blocking a mutant protein and halting the cancer cells' ability to sidestep the compound's effects.
Scientists at NCATS and NIDCR report a new strategy to alleviate chronic itch by blocking a receptor found on spinal cord neurons. They identified approximately 1,400 compounds worth examining more closely, with 15 showing promise in halting both human and mouse versions of the receptor.
A set of immune system tissue chips is scheduled to launch on SpaceX's 16th commercial resupply mission to the International Space Station, where they will simulate the aging process of the immune system. The goal is to gain new insights into the molecular basis for many human conditions and develop novel therapies.
Researchers identified metarrestin as a compound that stops tumor metastasis in animal models. The compound effectively blocks the spread of pancreatic and other cancers, with mice treated with metarrestin having fewer tumors and living longer.
The National Institutes of Health has awarded $15 million to support the development of 3-D microphysiological system platforms, called 'tissue chips,' that model human disease. These platforms will enable scientists to better understand disease mechanisms and predict how patients respond to specific drugs.
Scientists have discovered how a compound activates an enzyme to reduce cholesterol and other accumulated fats in the brains and livers of Niemann-Pick type C1 patients. The study may lead to new treatments for NPC1 and other neurodegenerative diseases.
A team of researchers has discovered a potential treatment for Duchenne muscular dystrophy by repurposing a compound originally developed to treat cancer. The compound, SU9516, works by boosting muscle repair and regeneration in affected muscles.
Research teams identified two cyclic peptides that can shut down an enzyme long thought to be undruggable for fighting disease-causing parasites and bacteria. The finding could lead to the development of new antimicrobial drugs.
Researchers identified compounds that can inhibit Zika virus replication and reduce brain cell death, including emricasan and niclosamide. The compounds were found using NCATS' drug repurposing screening robots and will be studied by the broader research community to combat the Zika public health crisis.
Researchers are developing treatments for Niemann-Pick disease type C using cyclodextrin and delta-tocopherol, which have shown promise in studies of patient skin cells and animal models. The goal is to reduce lipid accumulation and improve symptoms in patients with this devastating rare disease.
The NIH has awarded $29 million to expand the Rare Diseases Clinical Research Network, a collaborative effort of physician scientists and patient advocacy groups. This funding will support clinical research and investigation of new treatments for patients with rare diseases, with a focus on improving diagnosis and treatment outcomes.
The National Institutes of Health will award $17 million to support the development of human tissue chip technology, which could revolutionize predicting drug failure or success. Integrating chips mimicking human organ functions into a full body system will enable real-time measurement of drug effects and improve biomedical research.
A drug candidate developed to treat sickle cell disease has been acquired by Baxter International, advancing its clinical development activities. Aes-103 significantly reduces patients' pain in Phase II clinical trials, offering a potential breakthrough treatment.
The NIH Bridging Interventional Development Gaps (BrIDGs) program aims to advance treatments for acute radiation syndrome, brain injury from cardiac arrest, and beta thalassemia. BrIDGs supports expert contractors to perform pre-clinical services, with seven compounds licensed during or after development through the program.
The NIH has made large-scale information on small interfering RNA (siRNA) molecules publicly available, increasing the potential for finding new treatments for diseases. Researchers can now access siRNA sequences targeting over 20,000 human genes, facilitating studies of gene function and disease progression.
The NIH's Therapeutics for Rare and Neglected Diseases program aims to develop treatments for rare diseases affecting fewer than 200,000 Americans. Four new pre-clinical projects target retinitis pigmentosa, hypoparathyroidism and LEOPARD syndrome, marking the first use of stem cells and collaboration with Eli Lilly.
The NIH awards $17 million for research projects exploring the emerging field of extracellular RNA communication. Scientists will investigate exRNA's role in diseases, develop biomarkers, and create new treatments. The goal is to unlock exRNA's potential for diagnostics and therapeutics.
The NIH is funding collaborations between academia and industry to explore new uses for existing compounds in eight disease areas, including Alzheimer's disease and schizophrenia. The initiative aims to accelerate the development of new treatments by leveraging existing research and development.
A clinical trial to evaluate cyclodextrin as a potential treatment for Niemann-Pick disease type C1 has begun at the NIH Clinical Center. The disease, caused by cholesterol accumulation in cells, leads to progressive impairment of motor and intellectual function.
A clinical trial has begun to evaluate DEX-M74 as a treatment for hereditary inclusion body myopathy, a rare degenerative muscle disease. The study aims to assess the safety and effectiveness of DEX-M74 in patients with progressive muscle weakness.
Researchers have identified molecular compounds that activate a key enzyme, PKM2, which governs how cancer cells use glucose and its metabolites. These compounds delay the formation of tumors in mice by correcting the altered metabolic state of cancer cells, providing a potential therapeutic strategy for cancer treatment.
The NIH's Therapeutics Discovery program has partnered with eight pharmaceutical companies to make nearly 60 compounds available for research, accelerating the development of new treatments. Researchers can explore these compounds in disease areas where they haven't been previously explored.
The NIH and Lilly are collaborating to create a publicly available resource profiling thousands of approved and investigational medicines. This comprehensive database will enable researchers to better predict treatment outcomes and improve drug development.
The National Institutes of Health has awarded $200 million over five years to five health research centers to speed scientific discoveries into treatments for patients. The grants support the innovation and partnerships necessary to bridge basic research and medical practice.
The NIH Recovery Act has awarded $300 million in shared instrumentation grants to support biomedical research. The grants will enable researchers to access powerful devices such as high-powered electron microscopes and supercomputers to accelerate discoveries in disease prevention, treatment, and cure.
The NIH has awarded $255 million to nine health research centers to develop ways to reduce the time it takes for clinical research to become treatments for patients. The funding will support the Clinical and Translational Science Awards (CTSA) program, which aims to transform discoveries into preventions, treatments, and cures.
The National Institutes of Health (NIH) has awarded $1 billion in grants to construct, repair, and renovate scientific research laboratories across the US. These grants are expected to create or sustain jobs nationwide while fostering scientific advances that may lead to improved human health.
The NIH has awarded $8.5 million to support research on pharmaceuticals for children, with a focus on determining outcome measures to facilitate future studies. The grants will support studies in pediatric cardiology, neonatology, and pediatric neurology, aiming to improve the quality of pediatric research.
The National Institutes of Health is awarding up to $152 million over five years to fund Institutional Development Award (IDeA) Networks of Biomedical Research Excellence (INBRE) in nine IDeA-eligible states. These awards aim to expand research opportunities and increase the number of competitive investigators, promoting the developmen...
The National Institutes of Health has expanded its national consortium for transforming clinical and translational research, awarding $171 million to seven institutions over five years. The consortium aims to accelerate laboratory discoveries into treatments for patients and engage communities in clinical research.
The National Institutes of Health has awarded $122 million to fund Institutional Development Award (IDeA) Networks of Biomedical Research Excellence (INBRE) in seven states. These grants aim to expand research opportunities, increase the number of competitive investigators, and enhance biomedical research capacity.
The NIH Clinical and Translational Science Consortium has grown to 39 members, including the University of Cincinnati, which will receive $22.7 million over five years. The new Center for Clinical and Translational Science and Training will focus on pediatric research and increase outreach into local communities.