Tight junctions are multi-protein complexes that serve as barriers in epithelial tissues such as the skin or lining of the gut. Loss of a specific tight junction barrier protein, claudin 18, occurs in the majority of gastric cancer patients and is correlated with poor prognosis in patients with advanced gastric cancer. Understanding how claudin 18 loss occurs and what pathways it regulates may provide new strategies to inhibit neoplastic progression
Collaboration with SRI Provides QBioMed Expertise in Formulation Development and Preclinical Work in Autism, as well as CMC and Manufacturing capability Q BioMed Inc., a biotechnology accelerationcompany, announced a collaborative agreement with SRIInternational to provide formulation development, preclinical development, andearly clinical manufacturing of QBM-001, in support of Q BioMed’s AutisticSpectrum Disorder (ASD) drug development program for non-verbal or minimallyverbal autistic children. SRI has broad expertise in solving formulation, drug delivery,
Scientists who recently identified the molecular start of Alzheimer’s disease have used that finding to determine that it should be possible to forecast which type of dementia will develop over time – a form of personalized medicine for neurodegenerative diseases. A new study from UT Southwestern shows that single toxic tau proteins that stick together and spread degeneration across the brains of dementia patients have different shapes. The folds of these
A scientific strategy that explores therapeutic targets based on the biology of aging is gaining ground as an effective approach to prevent and treat Alzheimer's disease, accordingdd to research published in the December 7, 2018 online issue of Neurology®. A comprehensive review of the clinical trial landscape, including current agents being studied for the prevention and treatment of Alzheimer's disease (and other dementias), points to the need to develop and test
Researchers at Johns Hopkins Medicine have identified in live human brains new radioactive “tracer” molecules that bind to and “light up” tau tangles, a protein associated with a number of neurodegenerative diseases including Alzheimer’s disease and other related dementias. Two studies will be published back-to-back in the December issue of Journal of Nuclear Medicine—one as featured article of the month—describing testing of three candidate molecules in patients with Alzheimer’s disease, as
After over a decade of preclinical research and development, a new gene therapy treatment for Sickle Cell Anemia (SCA) is reversing disease symptoms in two adults and showing early potential for transportability to resource-challenged parts of the world where SCA is most common. Preliminary data from a pilot Phase 1-2 clinical trial testing the gene-addition therapy were presented Dec. 3 at the American Society of Hematology’s (ASH) annual meeting in San
New results from clinical trials of immunotherapy and experimental targeted agents for patients with leukemia and lymphoma are being presented by Dana-Farber Cancer Institute researchers at the American Society of Hematology (ASH) Annual Meeting Dec. 1-4. Here are summaries of three presentations, including one that compared outcomes of CAR T-cell therapy in patients in clinical trials with outcomes in the “real world” of clinical practice: CAR T-cell treatment provides durable
Scientists have discovered the signals that determine the fate of immature cells in the pancreas. The research shows that they are very mobile and that their destiny is strongly influenced by their immediate environment. This breakthrough published in the journal Nature will facilitate the manufacturing of pancreatic islet cells from stem cells and might help combating type 1 diabetes. Prof. Dr. Henrik Semb who led the study recently joined Helmholtz
In a proof-of-principle study, researchers at Johns Hopkins revealed that certain immune system cells found in the human liver, called liver macrophages, contain only inert HIV and aren’t likely to reproduce infection on their own in HIV-infected people on long-term antiretroviral therapy (ART). ART is a regimen containing combinations of HIV-targeting drugs that prevents the growth of the virus but does not eradicate it. The report on the findings, published
Researchers have developed a way to grow human platelets in the laboratory from stem cells derived from fat tissue. The achievement, reported today in the journal Blood, suggests manufactured platelets could eventually reduce the reliance on donated platelets to help patients with cancer and other disorders. Platelets are a component of blood that helps with clotting. Platelet transfusions can be life-saving for patients dealing with cancer or the effects of chemotherapy, infections,
IntraBio Inc., a late-stage biopharmaceutical company developing novel therapies for common and rare (“orphan”) neurodegenerative diseases, announced results for its lead compound series (IB1000s) for the treatment of dementia, including positive clinical results from compassionate-use studies in patients with Lewy Body Dementia and Fronto-Temporal Dementia, and from pre-clinical studies in Alzheimer’s Disease. Recent compassionate-use studies with IB1000s in patients with Lewy Body Dementia and Fronto-Temporal Dementia showed meaningful improvement in quality
Researchers at Johns Hopkins have identified two patients with HIV whose immune cells behave differently than others with the virus and actually appear to help control viral load even years after infection. Moreover, both patients carry large amounts of virus in infected cells, but show no viral load in blood tests. While based on small numbers, the data suggest that long-term viral remission might be possible for more people. A
Researchers at the Johns Hopkins Kimmel Cancer Center say they have added to evidence that measuring and monitoring tumor DNA that naturally circulates in the blood of melanoma patients can not only reliably help reveal the early stages of cancer growth and spread but also uncover new treatment options that tumor genetic analysis alone may not. “For some patients in our study, ctDNA (circulating tumor DNA) levels measured in a relatively simple
The U.S. Food and Drug Administration today approved Gamifant (emapalumab-lzsg) for the treatment of pediatric (newborn and above) and adult patients with primary hemophagocytic lymphohistiocytosis (HLH) who have refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. This FDA approval is the first for a drug specifically for HLH. "Primary HLH is a rare and life-threatening condition typically affecting children and this approval fills an unmet medical need
UT Southwestern researchers have found that an enzyme on the surface of some lung cancer cells helps feed the cancer, making it a tempting treatment target. The enzyme, transmembrane serine protease 11B (TMPRSS11B) is described in a report published today in the journal Cell Reports. In addition to being found in squamous cell lung cancer and prostate cancer, the enzyme also has been identified in squamous cell head, neck, and cervical cancers,
Cancer develops as a result of the accumulation of mutations in our cells. These mutations are not distributed evenly in our chromosomes, so some regions hold more than others. A study headed by the Institute for Research in Biomedicine (IRB Barcelona) and published in the journal Nature Cell Biologyexamined whether the opening of chromatin (a complex formed by DNA bound to proteins) is the factor that determines the accumulation of more
A new study by University of Minnesota biomedical engineers shows how they stopped cancer cells from moving and spreading, even when the cells changed their movements. The discovery could have a major impact on millions of people undergoing therapies to prevent the spread of cancer within the body. The research is published today in Nature Communications, a leading research journal. Researchers have known for years that tumors have patterns that are like
A DNA vaccine tested in mice reduces accumulation of both types of toxic proteins associated with Alzheimer’s disease, according to research that scientists say may pave the way to a clinical trial. A new study by UT Southwestern’s Peter O’Donnell Jr. Brain Institute shows that a vaccine delivered to the skin prompts an immune response that reduces buildup of harmful tau and beta-amyloid – without triggering severe brain swelling that earlier antibody
As part of an ongoing commitment to battle opioid addiction, the Rockefeller Neuroscience Institute at West Virginia University today marked a major milestone, enrolling the first patient in a randomized clinical trial that will test the effectiveness of an injectable non-opioid, non-steroid micropellet to treat sciatica. The phase III clinical trial utilizes a clonidine micropellet, which is half the size of a grain of rice and is placed in a
Anti-malaria drugs known as chloroquines have been repurposed to treat cancer for decades, but until now no one knew exactly what the chloroquines were targeting when they attack a tumor. Now, researchers from the Abramson Cancer Center of the University of Pennsylvania say they have identified that target – an enzyme called PPT1 – opening up a new pathway for potential cancer treatments. The team also used CRISPR/Cas9 gene editing
