Feature Channels: Pharmaceuticals

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While breakthrough treatments have emerged for several cancers over the last two decades, driving striking improvements in survival and other clinical outcomes, too little is known about the risk of therapy-related hematologic cancers following targeted and immunotherapeutic approaches. In a study to be presented at the American Society of Clinical Oncology (ASCO) 2020 virtual meeting, a Roswell Park Comprehensive Cancer Center team reports that in many cases, these newer treatment approaches may reduce the risk of therapy-related myelodysplastic syndrome or acute myeloid leukemia (tMDS/AML) compared to chemotherapy-based treatment strategies.

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By analyzing tumors from patients treated with immunotherapy for advanced kidney cancer in three clinical trials, Dana-Farber Cancer Institute scientists have identified several features of the tumors that influence their response to immune checkpoint inhibitor drugs.

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Despite improvements in their performance over the past decade, electronic health records (EHRs) commonly used in hospitals nationwide fail to detect up to one in three potentially harmful drug interactions and other medication errors, according to scientists at University of Utah Health, Harvard University, and Brigham and Women’s Hospital in Boston.

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Patients with advanced non-small cell lung cancer (NSCLC) and the MET exon 14 (METex14) skipping mutation had a 46.5% objective response rate to the targeted therapy drug tepotinib, as shown in a study published today in the New England Journal of Medicine and presented at the 2020 American Society of Clinical Oncology (ASCO) Annual Meeting (Abstract 9556 – Poster 322) by researchers from The University of Texas MD Anderson Cancer Center.

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The targeted therapy pralsetinib appears to have high response rates and durable activity in patients with a broad variety of tumors harboring RET gene fusions, according to results from the international Phase I/II ARROW trial, led by researchers at The University of Texas MD Anderson Cancer Center.

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The adoptive T-cell therapy ADP-A2M4, which is engineered to express a T-cell receptor (TCR) directed against the MAGE-A4 cancer antigen, achieved responses in patients with multiple solid tumor types, including synovial sarcoma, head and neck cancer and lung cancer, according to results from a Phase I clinical trial led by researchers at The University of Texas MD Anderson Cancer Center.

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Results of a Phase I clinical trial conducted by researchers at the Yale Cancer Center have shown that ARV-110, an androgen receptor PROTAC® protein degrader, demonstrates anti-tumor responses in patients with metastatic castration-resistant prostate cancer.

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A Georgetown University Medical Center clinical trial investigating the cancer drug nilotinib in people with Alzheimer’s disease finds that it is safe and well-tolerated, and researchers say the drug should be tested in a larger study to further determine its safety and efficacy as a potential disease-modifying strategy.

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During a plenary session of the 2020 American Society of Clinical Oncology (ASCO) Virtual Scientific Program, findings will be presented from the global phase III JAVELIN Bladder 100 randomized study conducted by investigators from Seattle Cancer Care Alliance, Fred Hutchinson Cancer Research Center and University of Washington Medicine along with several other research centers. The results demonstrated significantly prolonged overall survival of patients with locally advanced (unresectable) or metastatic urothelial cancer when treated with first-line immunotherapy avelumab plus best supportive care (BSC) compared to BSC alone (following disease control on induction chemotherapy).

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Dr. Alistar has pioneered the use of CPI-613® (devimistat), a unique first-in-class treatment for locally advanced or metastatic pancreatic cancer, in combination with two chemotherapy drugs. Devimistat uniquely targets enzymes involved in cancer cell metabolism, against pancreatic cancer. The study’s efficacy data suggest devimistat has synergy with chemotherapy.The current study was funded by the drug’s manufacturer, New Jersey-based Rafael Pharmaceuticals and by Atlantic Health System.

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According to findings led by researchers at Yale Cancer Center, treatment with the targeted therapy osimertinib following surgery significantly improves disease-free survival (DFS) in patients with early-stage, non-small cell lung cancer (NSCLC) with EGFR gene mutations.

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During the COVID-19 pandemic, Johns Hopkins Medicine Media Relations is focused on disseminating current, accurate and useful information to the public via the media. As part of that effort, we are distributing our “COVID-19 Tip Sheet: Story Ideas from Johns Hopkins” every Tuesday throughout the duration of the outbreak.

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University of Colorado Cancer Center team uses liquid biopsy to predict multiple myeloma drug resistance and sensitivity.

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A new study from researchers at the UCLA Jonsson Comprehensive Cancer Center helps explain how disruptions in genes can lead to the resistance to one of the leading immunotherapies, PD-1 blockade, and how new drug combinations could help overcome resistance to the anti-PD-1 therapy in a mechanistically-based way.

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Early exposure to anesthetics may make adolescents more susceptible to developing alcohol use disorder (AUD), according to new research from Binghamton University, State University of New York.

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Penn State Health Milton S. Hershey Medical Center has enrolled its first patients in the continuation of a clinical trial using the investigational antiviral drug remdesivir for treatment of COVID-19.

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Since the outbreak of the COVID-19 pandemic and its rapid spread, the scientific community has been working on developing an effective treatment for the virus responsible for the disease.

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In this issue, Guest Editor Veli-Pekka Jaakola, Ph.D., (Confo Therapeutics, Belgium) highlights a series of articles focused on new screening tools and assays that find new chemical matter for medically relevant membrane protein targets. In addition, an overview of a new and emerging protein-lipid reconstitution methodology utilizing styrene maleic acid (SMA) polymers is featured.

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While scientists around the world search for a coronavirus vaccine, an equivalent effort should be made to develop drugs that would mitigate the virus’s effects on patients, says Johns Hopkins Carey Business School Senior Lecturer Bonnie Robeson, who previously served as a principal investigator in drug discovery and development at the National Cancer Institute.

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New research from the Perelman School of Medicine at the University of Pennsylvania catalogued every use documented in medical literature so far and found physicians have reported on the use of more than 100 different off-label and experimental treatments

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For patients with diabetes – especially those with lower incomes – preventive drug lists (PDLs) of essential medications available with no deductible can reduce out-of-pocket costs while increasing use of important treatments, reports a study in a June supplement to Medical Care. The journal is published in the Lippincott portfolio by Wolters Kluwer.

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• In an analysis of information on US adults initiating hemodialysis, 16% of patients were dispensed a short-acting benzodiazepine, and approximately one-quarter of these patients were also dispensed opioids. • Among patients with an opioid prescription, being dispensed a short-acting benzodiazepine had a 1.9-fold higher risk of dying over a median follow-up of 16 months compared with patients without a short-acting benzodiazepine.

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A new landmark study by researchers at the University of Maryland School of Medicine (UMSOM) found that patients with a vascular condition, called abdominal aortic aneurysm, received no benefits from taking a common antibiotic drug to reduce inflammation.

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The investigational antiviral remdesivir is superior to the standard of care for the treatment of COVID-19, according to a report published today in The New England Journal of Medicine.

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When seizures last longer than about 5 minutes--a condition called status epilepticus--emergency treatment is required. About two-thirds of people respond to initial treatment with benzodiazepines, but the others need a second drug. Which drug to choose is a matter of some debate.

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Montefiore Health System and Albert Einstein College of Medicine have begun the next stage of the Adaptive COVID-19 Treatment Trial (ACTT), to evaluate treatment options for people hospitalized with severe COVID-19 infection. The new iteration of the trial, known as ACTT 2, is sponsored by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health.

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A new report, published in Nature Reviews Drug Discovery, reveals that the cellular immunotherapy landscape continues to expand, with 472 more cellular therapies in development now than one year ago

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Developing frog embryo brains damaged by nicotine exposure can be repaired by treatment with ionoceutical drugs that restore bioelectric patterns in the embryo, followed by repair of normal anatomy and brain function. The research suggests therapeutic drugs may be used to help repair birth defects.

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Access to healthcare has been at the forefront of social and political debate for decades. Reliable and equitable access to provider prescribed medications is tantamount to the delivery of appropriate healthcare, and the lifecycle of medication manufacturing, distribution, pricing and procurement has been shrouded in an incomprehensible array of transactions and involved stakeholders. Among the middlemen interspersed between pharmaceutical manufacturers and patients are pharmacy benefit managers (PBM). Initially tasked with administering drug plans for health insurers,1 the role of PBMs has expanded over time. They currently function in a lightly regulated area,2,3 with few requirements for business transparency. Three PBMs, CVS Caremark, Optum RX, and Express Scripts, control distribution of nearly ¾ of the medications in the United States.

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Multidisciplinary study of the COVID-19 pandemic and its wide-ranging impact has become an urgent endeavor worldwide. To further and deepen global understanding of the crisis, the Harvard Data Science Review (an open access platform of the Harvard Data Science Initiative) is publishing a special issue examining the novel coronavirus and its impact through the lens of data science.

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The Arthur C. Clarke Center for Human Imagination at UC San Diego organized the collaborative Psychedelics and Health Research Initiative, which explores the potential for psychedelics to address chronic pain conditions.

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A clinical trial in people with the new coronavirus is testing a drug that may halt an overactive immune response before it ramps up.

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As dentists and their teams across America get back to their regular schedules after a sharp COVID-19-related reduction, a new study shows a key opportunity to reduce the use of opioid painkillers by their patients. The analysis shows that those who had dental procedures on a Friday or a day before a holiday were much more likely to fill a prescription for an opioid than other patients.

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In recent years, genome-wide association studies (GWAS) have identified many different genetic variants associated with schizophrenia. These genetic discoveries raise the promise of developing urgently needed new treatments targeting the underlying biology and pathophysiology of schizophrenia, according to a special article in the Journal of Clinical Psychopharmacology. The journal is published in the Lippincott portfolio by Wolters Kluwer.

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Tip sheet with latest Johns Hopkins research news NOT related to COVID-19. Stories: more women & seniors needed in cholesterol drug trials, improving medical care quality for homebound seniors & 2020 JHM Science Writers Boot Camp goes virtual.

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Midlands Medictech company Medherant has just this month (May 2020) signed a partnership agreement with Cambridge based Cycle Pharmaceuticals to develop multiple new products using Medherant technology developed by University of Warwick chemistry researchers.

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In response to the COVID-19 pandemic, researchers from the Massachusetts Institute of Technology have published a pair of studies in a COVID-19 special issue of the Harvard Data Science Review, freely available via open access, describing new methods for accelerating drug approvals during pandemics and for providing more accurate measures of the probabilities of success for clinical trials of vaccines and other anti-infective therapies.

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Birth control pills are an effective and safe form of birth control; however, they are associated with a number of side effects, including mood alterations.

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A new UT Southwestern study shows how an effective but largely abandoned treatment for Type 2 diabetes could be used again in combination with another drug to eliminate problematic side effects.

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A team of scientists led by Abhishek Singharoy at Arizona State University used the Summit supercomputer at the Oak Ridge Leadership Computing Facility to simulate the structure of a possible drug target for the bacterium that causes rabbit fever.

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For decades, scientists have explored the use of liposomes –– hollow spheres made of lipid bilayers –– to deliver chemotherapy drugs to tumor cells. Now, researchers report in ACS’ Nano Letters a way to stabilize liposomes by embedding a stiff nanobowl in their inner cavity.

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Scientists at Dana-Farber Cancer Institute have launched a clinical test of a blood cancer drug in patients infected with the COVID-19 virus.

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Researchers in the Ludwig Center at the Johns Hopkins Kimmel Cancer Center report they have identified a drug treatment that could—if given early enough—potentially reduce the risk of death from the most serious complication of Coronavirus disease 2019 (COVID-19), also known as SARS-CoV-2 i

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Researchers at the University of Nebraska Medical Center have identified a key cell signaling pathway that drives the devastating muscle loss, or cachexia, suffered by many cancer patients. The study, which will be published May 22 in the Journal of Experimental Medicine, suggests that targeting this pathway with a drug already in phase 2 clinical trials for diabetes could prevent this syndrome.

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How can researchers and oncologists glean meaningful information from mounds of data to help guide cancer research and patient care? A new analytic tool developed by University of Michigan Rogel Cancer Center researchers combines multiple data sets to help sift the signal from the noise.

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A new paper published online in the Annals of the American Thoracic Society describes a nimble, pragmatic and rigorous multicenter clinical trial design to meet urgent community needs in the face of the COVID-19 pandemic.

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MTX110 is a new formulation of panobinostat, a chemotherapy drug that has shown promise in laboratory models of medulloblastoma, the most common malignant brain tumor in children. Now, MTX110 is the focus of a novel trial that places the therapy directly into the fourth ventricle of the brain to treat patients with recurrent medulloblastoma.

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Three drugs that are already approved by the Food and Drug Administration (FDA) or other international agencies can block the production of the novel coronavirus that causes COVID-19 in human cells, according to computational and pharmaceutical studies performed by UT Southwestern scientists.