Feature Channels: Personalized Medicine

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The first patient in a new Phase 1 gene therapy trial for pediatric brain tumors underwent a leading-edge procedure at Ann & Robert H. Lurie Children’s Hospital of Chicago. During surgery to remove the brain tumor, the patient was injected with an adenovirus, a common cold virus, at the tumor site. The virus was bioengineered not to cause illness but rather deliver a gene that produces human interleukin 12 (hIL-12), a powerful protein to jumpstart the immune system to kill remaining tumor cells. For the next 14 days, the patient is given a pill – veledimex – to activate the gene and control the immune response, so that the inflammation fights the tumor without overwhelming the rest of the body.

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A sharp rise in the number of women seeking BRCA genetic testing to evaluate their risk of developing breast cancer was driven by multiple factors, including celebrity endorsement, according to researchers at the University of Georgia.

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African American women participating in a clinical study on breast cancer had more side effects and poorer survival rates than did women of European ancestry, according to a an Indiana University study that identified ethnicity through genetics--a first in this type of research.

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Imagine being able to quickly and accurately screen for diseases or chemical contaminants in a tiny drop of blood. Berkeley Lab scientist Daojing Wang and others have developed a multinozzle emitter array (MEA), a silicon chip that can dramatically shorten the time it takes to identify proteins, peptides, and other molecular components within small volumes of biological samples. This patented technology is now being commercialized by Newomics Inc., a company Wang launched to further develop the product and build a platform for personalized health care.

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Scientists have discovered for the first time how to accurately predict cellular radiation resistance without actually irradiating cells, instead measuring their internal ‘manganese-complexes’ responsible for resistance. This new broad measure of radiation resistance opens exciting scientific possibilities such as: allowing for more personalized cancer treatments, development of radioprotectors needed for astronauts to get to Mars and back, all while minimizing the need for animals in radiation studies, according to “Across the Tree of Life, Radiation Resistance is Governed by Antioxidant Mn2+, Gauged by Paramagnetic Resonance” published in the October 17 online issue of the Proceedings of the National Academy of Sciences USA.

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Researchers investigating pediatric low-grade gliomas (PLGG), the most common type of brain tumor in children, have discovered key biological differences in how mutated genes combine with other genes to drive this childhood cancer. By shedding light on subtle distinctions in tumor biology, these findings offer clues to designing more effective anticancer treatments to precisely target tumors in individual patients.

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Accurately subtyping glioblastoma tumors could lead to diagnostics and precision therapy

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Advancements in individualized medicine are offering health care providers new tools to quickly and accurately diagnose, treat, predict and, eventually, prevent disease.

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Individualized medicine — the concept of matching medical care precisely to each patient’s genes, lifestyle and environment is no longer just a theory. Experts in individualized medicine — also known as personalized or precision medicine — will be in Rochester Oct. 9-10, presenting the latest ways to apply precision medicine to all patients. They are available for interviews on groundbreaking discoveries at Individualizing Medicine 2017: Advancing Care Through Genomics, which will be held at Mayo Civic Center.

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The University of Michigan has launched a new initiative to harness campus-wide research aimed at finding personalized solutions to improve the health and wellness of individuals and communities.

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By enabling early disease detection and personalized treatment, laboratory tests can save lives as well as billions in medical costs each year. Join AACC and leading experts in laboratory medicine for a discussion of how clinical tests can be leveraged to achieve these goals, as well as the policies needed to support testing’s vital contribution to healthcare.

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Pediatric researchers in the Roberts Collaborative for Genetics and Individualized Medicine at Children's Hospital of Philadelphia have identified common gene variants that raise the risk of developing an aggressive form of the childhood cancer neuroblastoma. The discovery, in the MMP20 gene, may assist doctors in better diagnosing subtypes of neuroblastoma.

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With disease-modifying treatment trials for Alzheimer’s disease (AD) currently unsuccessful and only medications to treat symptoms available, what now? A leading neuroscientist has developed the “Dementia Prevention Initiative,” which abandons generalized ways to research and treat AD. His secret weapon: a novel “N-of-1 design” that tailors medicine down to a single patient. Instead of conducting a conventional trial of 100 people all getting the same treatment, he has switched it around and is conducting 100 single personalized trials.

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Immune-cell based therapies opening a new frontier for cancer treatment carry unique, potentially lethal side effects that provide a new challenge for oncologists, one addressed by a team led by clinicians at The University of Texas MD Anderson Cancer Center with proposed guidelines for systematically dealing with the toxicities of these drugs.

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Cells can be programmed like a computer to fight cancer, influenza, and other serious conditions – thanks to a breakthrough in synthetic biology by the University of Warwick.

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A team of scientists led by the University of Southampton has demonstrated a groundbreaking new method of gene synthesis – a vital research tool with real-world applications in everything from growing transplantable organs to developing treatments for cancer.

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Bethesda, Md. – For the last two decades, researchers worldwide have been using whole genome sequencing to understand what makes cells radiation-resistant, hoping to solve an old biological mystery: why is it that one of the most radiation-resistant organisms, Deinococcus radiodurans, aka “Conan the bacterium,” can survive hundreds of times more DNA damage caused by gamma rays than most other organisms? According to a study published recently in Standards of Genomic Sciences by researchers at the Uniformed Services University (USU), the amount of radiation a Deinococcus cell can survive in fact has little to do with the number and types of its DNA repair proteins.

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Hengbin Wang and colleagues describe a key role for a protein called RSF1 in silencing genes. Besides the molecular biology details, the researchers also showed that disruption of RSF1 expression in the embryos of African clawed frogs caused severe developmental defects in the tadpoles.

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The University of Chicago Medicine is one of a limited number of U.S. sites working to offer a breakthrough gene therapy for pediatric acute lymphoblastic leukemia (ALL), which was just approved by the U.S. Food and Drug Administration.

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Indiana University School of Medicine researchers have developed a more precise way of diagnosing suicide risk, by developing blood tests that work in everybody

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Press can register here to livestream this special session through Newswise Live on Monday, July 31 at 7:30 PM EDT. The winner of the Qualcomm Tricorder XPRIZE competition will present DxtER—a real-life tricorder—at the 69th AACC Annual Scientific Meeting & Clinical Lab Expo in San Diego. This special session will be the first time that the device is presented to researchers at a U.S. scientific conference.

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A research team from the Schepens Eye Research Institute of Massachusetts Eye and Ear has successfully prevented mice from developing angiogenesis of the retina—the sensory tissue at the back of the eye—using gene-editing techniques with CRISPR-Cas9.

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Ten new reviews and original research reports that illustrate how the progression of research assays from qualitative outputs toward increasingly sensitive quantitative outputs is transforming life sciences and biomedical research and diagnostics by improving the ability of researchers and clinicians to detect and quantify increasingly complex assays.

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The Henry Ford Cancer Institute has treated the world’s first cancer patient today with an advanced radiation therapy that uses an FDA-cleared real-time magnetic resonance imaging and linear accelerator delivery for more precise and accurate radiation treatment.

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This new review in SLAS Discovery analyzes the recent literature and impact of unfolded protein response (UPR) in health and disease.

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Mount Sinai has partnered with Theragene Pharmaceuticals, Inc. to advance a novel airway-delivered gene therapy for treating pulmonary hypertension (PH), a form of high blood pressure in blood vessels in the lungs that is linked to heart failure.

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Laboratory studies suggest that an experimental drug already in early clinical trials for a variety of adult cancers might enhance radiation and chemotherapy for two childhood brain cancers that currently are virtually always fatal.

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A new treatment rapidly removes the oral blood thinner dabigatran (PRADAXA®) from circulation within minutes, allowing life-saving clots to form normally

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Supported by a $3.3 million National Institutes of Health (NIH) RO1 grant, Fedak and Barker will use 4D-Flow MRI, a cutting-edge imaging technique that allows visualization of three-dimensional blood flow in real time, and tissue analysis to inform personalized treatment for BAV patients.

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After 12 weeks of taking an anti-asthma drug, a subset of patients with type 2 diabetes showed a clinically significant reduction in blood glucose during a randomized, double blind, placebo-controlled clinical trial, report University of California San Diego School of Medicine and University of Michigan researchers.

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The majority of Americans over age 50 take two or more prescription medicines to prevent or treat health problems, and many of them say the cost weighs on their budget, a new poll finds. But many older adults aren’t getting – or asking for – as much help as they could from their doctors and pharmacists to find lower-cost options, the new data reveal.

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Medicinal payload could be delivered by engineered RNAs that can be controlled by a billion year-old “genetic fossil” found in all cells

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Researchers have developed micro-cubes that can sponge up a hydrophobic anti-cancer drug and deliver it to cancer cells. Tissue culture tests show these tiny, porous cubes, loaded with the hydrophobic drug, are more potent against liver cancer cells and less harmful to normal liver cells.

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Using a new skin cell model, researchers have overcome a barrier that previously prevented the study of living tissue from people at risk for early heart disease and stroke. This research could lead to a new understanding of disease progression in aortic aneurysm – ballooning of the large artery in the chest that carries blood from the heart to the body.

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Antibiotics were the wonder drug of the 20th century, but bacteria evolved resistance. According to the CDC, more than 2 million people in the U.S. develop MDR infections every year. Researchers at Univ. of Utah developed a rapid screen to pair existing FDA-approved drugs to combat MDR infections.

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The nuclear receptor REV-ERB plays a key role in muscle regeneration, suggesting the receptor may be a good target for new drugs to treat a variety of muscle disorders and injuries.

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Fungi are rich sources of natural molecules for drug discovery, but numerous challenges have pushed pharmaceutical companies away from tapping into this bounty. Now scientists at Northwestern University, the University of Wisconsin-Madison and the biotech company Intact Genomics have developed technology that uses genomics and data analytics to efficiently screen for molecules produced by molds to find new drug leads -- maybe even the next penicillin. From three diverse fungal species, the research team discovered 17 new natural products.

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As the pace of drug approvals accelerates and the U.S. Food and Drug Administration (FDA) faces potential budget cuts, a new research design from Perelman School of Medicine scientists offers a new way to successfully assess safety of newly approved drugs, as well as drugs that have been on the market for a long time and have had a marked rise in their use. The study, published in the July issue of the journal Epidemiology, offers benefits over typically used randomized clinical trials, as such studies are often too small to identify rare side effects or may be performed in a group of patients who do not take other types of medications or have other conditions that could skew the drug's effect in a broader group following approval. Also first-in-class drugs may not have an applicable comparator drug, and traditional follow-up studies may give inaccurate results if those who take a new drug are different from those who took the comparator drug.

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At a meeting in Leuven, Belgium, a coherent picture began to emerge for how fluid pockets of proteins and RNAs contribute to health and disease.

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A new Iowa State University study may have identified the link that explains years of conflicting research over a mitochondrial gene and the risk for Alzheimer’s disease.

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The health of children’s bones could be determined before they are born, a new University of Southampton study has shown.

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Pressures on primary care doctors to move away from opioid pain management are increasing, but practitioners need practical, evidence-based information on how to employ multidisciplinary pain care successfully in everyday clinical practice. A senior investigator for Kaiser Permanente, speaking at the American Pain Society Annual Scientific Conference, believes wider use of practical clinical trials and more emphasis on patient self-management are key solutions for achieving wider use of multidisciplinary pain care to improve patient function and help lower use and misuse of opioids.

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Dr. Ayelet Erez, a doctor and geneticist at the Weizmann Institute of Science, has found that while a lack of the protein citrin slows children’s growth, blocking it in cancer slows tumor growth

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Research findings to be published about new blood test technology that will greatly reduce errors in labwork and improve care in public health and infectious disease. Press briefing scheduled for May 16, reserve press access to live virtual event now.

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In a small and preliminary clinical trial, Johns Hopkins researchers and their collaborators have shown that an experimental gene therapy that uses viruses to introduce a therapeutic gene into the eye is safe and that it may be effective in preserving the vision of people with wet age-related macular degeneration (AMD).

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Scientists at the University of Birmingham have developed a type of eye drop which could potentially revolutionise the treatment of one of the leading causes of blindness in the UK.

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Metformin, the most widely used drug to treat type 2 diabetes, could potentially be used to treat symptoms of Fragile X syndrome, an inherited form of intellectual disability and a cause of some forms of autism.

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