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Archive - May 2, 2017


Dr. Zon Alerts to National ALS Advocacy Day May 16; Notes Promising Stem Cell Work

In his latest “Zone in with Zon” blog post, dated May 2, 2017, and published by TriLink BioTechnologies of San Diego, Gerald “Jerry” Zon, PhD, gives advance notice of National ALS Advocacy Day that will take place on Tuesday, May 16, 2017. He notes that ALS (amyotrophic lateral sclerosis) is commonly called “Lou Gehrig’s disease” in the United States after the famous New York Yankee baseball player Lou Gehrig who was struck by the disease and died two years after the dread disease forced him to leave the game in 1939. In his popular blog, Dr. Zon outlines the grim features of this fatal disease for which there is still no cure, but he also highlights promising stem cell work that has taken place recently in Israel. He reported that a company named BrainStorm Cell Therapeutics, Inc. (BCLI) has developed a patented stem cell-based technology that delivers a growth factor that can help neurons live longer at or near the site of injury or damage. More specifically, Dr. Zon noted, “a mesenchymal stem cell isolated from an ALS patient is grown in a cell culture under certain conditions to produce a differentiated phenotype that secretes brain-derived neurotrophic factor (BDNF) at a level at least five-times greater than normal.”….“After these ‘super secreting’ cells are obtained ex vivo, they are reintroduced into the same ALS patient (i.e., an autologous transplant), wherein BDNF acts on neurons of the central nervous system and the peripheral nervous system, helping to support the survival of existing neurons, and encourage the growth and differentiation of new neurons and synapses.” Dr.

Chemical Compound Promotes Running Endurance in Sedentary Mice

Every week, there seems to be another story about the health benefits of running. That's great--but what if you can't run? For the elderly, obese, or otherwise mobility-limited, the rewards of aerobic exercise have long been out of reach. Salk Institute scientists, building on earlier work that identified a gene pathway triggered by running, have discovered how to fully activate that pathway in sedentary mice with a chemical compound, mimicking the beneficial effects of exercise, including increased fat burning and stamina. The study, which appears in Cell Metabolism on May 2, 2017, not only deepens our understanding of aerobic endurance, but also offers people with heart conditions, pulmonary disease, type 2 diabetes, or other health limitations the hope of achieving those benefits pharmacologically. The open-access article is titled “PPARδ Promotes Running Endurance by Preserving Glucose.” "It's well known that people can improve their aerobic endurance through training," says senior author Ronald Evans, Howard Hughes Medical Institute investigator and holder of Salk's March of Dimes Chair in Molecular and Developmental Biology. "The question for us was: how does endurance work? And if we really understand the science, can we replace training with a drug?" Developing endurance means being able to sustain an aerobic activity for longer periods of time. As people become more fit, their muscles shift from burning carbohydrates (glucose) to burning fat. So researchers assumed that endurance is a function of the body's increasing ability to burn fat, though details of the process have been murky.

Scientists Develop Fruit Fly Model of Kidney Cyst Formation; May Be Applicable to Study of Polycystic Kidney Disease (PKD)

According to PKD International, 12.5 million people worldwide are affected by polycystic kidney disease (PKD). There is no known cure. But that may one day change, thanks in part to new research by a Concordia University (Montreal, Canada) biology researcher. In a study, published online on April 13, 2017 in PLOS Genetics, Chiara Gamberi, PhD, and her coauthors developed an innovative fruit-fly-based model of the types of harmful cysts that can form on kidneys. The model has enormous potential for assisting the study of how cells proliferate in PKD and cancer. But what do fruit flies have to do with it? "The human and fly genomes show a surprising level of similarity. In fact, gene relationships, or genetic pathways, are virtually identical between human beings and fruit flies," explains Dr. Gamberi, who is affiliate assistant professor of biology in Concordia's Faculty of Arts and Science. "Most human organs have fly counterparts. That's a great advantage we can leverage to study the functions of disease-associated genes, and also to identify possible methods of combating those diseases." The PLOS Genetics article is titled “Bicaudal C Mutation Causes myc and TOR Pathway Up-Regulation and Polycystic Kidney Disease-Like Phenotypes in Drosophila.” Kidneys are particularly challenging to investigate because of the difficulties of isolating the nephrons -- tiny tubes in the kidney that filter substances from body fluids. The fruit fly equivalent, small though it is, acts as an effective stand-in, with the added advantage of allowing researchers to rapidly assess genetic and chemical influences because of the fruit fly's short lifespan. Dr. Gamberi and her coauthors reported the first example of renal cysts in the fruit fly species Drosophila melanogaster.

Annual ISEV Meeting on Extracellular Vesicles (Including Exosomes) in Toronto May 18-21

The annual meeting of the International Society for Extracellular Vesicles (ISEV 2017) (, will take place from May 18-21 (plus the May 17 Education Day) in Toronto, Canada, and will offer an unparalleled opportunity to network with, and learn from, the preeminent leaders in extracellular vesicle (EV) research. To register for this meeting, please click here ( The scope and quality of the anticipated scientific exchange make ISEV 2017 the largest and the premier meeting in EV research in the world. This event features four days of the best in vesicle science covering all aspects of basic, clinical, and translational research. The research theme includes diverse areas of science encompassing rare and neglected diseases, infectious disease, coagulation, cancer, neuroscience, cardiovascular studies, immunology, regenerative medicine, virology, parasitology, and more. The overall theme of ISEV 2017 is “Diversity of EV Composition and Function in Disease Diagnosis and Therapeutics.” Amidst growing interest in the promise of EVs in disease detection and treatment, ISEV 2017 will bring scientists and clinicians in medical and biotechnology communities together to translate their research. No other meeting in the world offers the scope, participation level, and thematic focus of ISEV 2017 concentrating and cross-pollinating scientific investigations in the field of disease biomarkers and therapeutic tools by disseminating cutting-edge developments in EV research. Among the plenary speakers scheduled to address the meeting are Clotilde Thery, Ph.D. (Research Director, Institut Curie), Philip Stahl, Ph.D. (Professor Emeritus of Cell Biology and Physiology, Washington University School of Medicine), Thomas Thum M.D., Ph.D.

Stereotactic Radiation Highly Effective for Metastatic Kidney Cancer, Study Indicates

Kidney cancer patients may soon have more treatment choices that provide a higher quality of life, thanks to research completed by physician scientists at the University of Texas (UT) Southwestern Medical Center. Their recent study showed that treating metastatic kidney cancer with an advanced and focused form of radiation called stereotactic ablative radiation therapy achieves more than 90 percent control of local tumors, and offers the possibility of safely delaying systemic therapy. “This study shows that stereotactic radiation provides a good noninvasive alternative to conventional treatment such as surgery, and that it effectively controls the disease,” said Raquibul Hannan (photo), MD, PhD, Assistant Professor of Radiation Oncology and Co-Leader of the Kidney Cancer Program of the Harold C. Simmons Comprehensive Cancer Center and senior author of the study. “It may also offer an alternative to patients who are not candidates for surgery. Often due to the number and location of the metastases and sometimes due to other conditions, patients are not candidates for surgery.” The standard of care for metastatic renal cell carcinoma is systemic therapy, which can be associated with significant side effects like tiredness, fatigue, high blood pressure, and rash. These side effects can be significant and debilitating. According to Dr. Hannan, the new study shows that patients with metastatic kidney cancer can be treated with stereotactic radiation therapy with the goal of being cured, or to delay systemic therapy allowing patients to enjoy a better quality of life without the side effects of the drugs.

Study Opens New Line of Attack on Spinal Muscular Atrophy

Though spinal muscular atrophy (SMA) in its most severe form remains incurable and fatal in early childhood, researchers are sustaining a multipronged counterattack for patients and their families. The first treatment for the disease gained U.S. market approval in December. Now a new discovery led by Brown University scientists deepens the basic understanding of how the genetic mutation that causes SMA appears to undermine the communication between motor neurons and the muscles they control. "We are making progress," said Anne Hart (photo), PhD, Professor of Neuroscience at Brown and senior author of the new study published online on May 2, 2017 in eLife. The open-access article is titled “Decreased MicroRNA Levels Lead to Deleterious Increases in Neuronal M2 Muscarinic Receptors in Spinal Muscular Atrophy Models.” About one in 8,000 children is born with some form of SMA in which mutations in both copies of the gene that code for the survival motor neuron (SMN) protein cripple its production. The end result, which the new study helps to explain, is dysfunction of motor neurons that control muscle along with muscle atrophy and weakness. In the most acute form, Type I, children die by age two as even functions as fundamental as breathing become compromised. With other SMA types, patients can live much longer, but they still suffer significant muscle weakness. It's a positive sign that spinal cord injections of nusinersen, the newly approved drug, restore some motor function and prolong survival by improving SMN production, Dr. Hart said, but researchers can make further and perhaps more lasting headway by understanding how the lack of SMN ultimately undermines muscle function.

MicroRNA-210 Reduces Stroke Risk from Rupture in Carotid Artery

The molecule microRNA-210 stabilizes deposits in the carotid artery and can thus prevent them from tearing, and prevent dangerous blood clots from forming. This is what scientists headed by Professor Lars Mägdefessel, Professor of Vascular Biology at the Technical University of Munich (TUM) and head of a junior scientist group in the German Centre for Cardiovascular Research (DZHK) have discovered. Their results open up new treatment approaches to reduce stroke risk in patients with carotid arteries at risk of rupturing. Their results were published in the February 17, 2017 issue of Circulation Research. The article is titled “MicroRNA-210 Enhances Fibrous Cap Stability in Advanced Atherosclerotic Lesions.” The most common cause for the narrowing of the carotid artery and thus the major risk factor for strokes is atherosclerosis, where so-called plaques build up on the vessel walls. If a plaque ruptures, blood clots can form that either further occlude the site that is already narrowed, or are carried away by the blood flow, which could lead to vascular occlusion at a different site. If this happens in the carotid artery, it could lead to a stroke. How easily a plaque ruptures depends on how thick the tissue layer surrounding its core is. The thicker this so-called fibrous cap, the more stable and thus more harmless the vessel deposit. “New imaging procedures enable us to detect dangerous plaques with increasing precision; but the therapies currently available for removing these unstable plaques and thus preventing a stroke entail a certain amount of risk that the plaques will rupture during the procedure,” explains Professor Mägdefessel.