Targeting Molecular Pathway That Causes Pulmonary Arterial Hypertension

Dr. Thistlethwaite

Pulmonary arterial hypertension (PAH) is a type of high blood pressure in the lungs, in which blood vessels are narrowed, blocked or destroyed, causing the heart to work harder and, in time, results in cardiac weakness and failure. The disease is relatively rare, but affects an estimated 100,000 persons in the United States, and results in 20,000 deaths annually. There is no cure. In a study published May 4, 2022 in Science Translational Medicine, researchers at the University of California San Diego (UCSD) School of Medicine describe the underlying signaling pathway that results in PAH–and a novel monoclonal antibody therapy that blocks the abnormal blood vessel formation characterizing the disease. The article is titled “JAGGED-NOTCH3 Signaling in Vascular Remodeling in Pulmonary Arterial Hypertension.”

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Retinal Cell Map Could Advance Precise Therapies for Blinding Diseases; NEI Discovery Sheds Light on Tissue Targeted by Age-Related Macular Degeneration and Other Diseases

Researchers have identified distinct differences among the cells comprising a tissue in the retina that is vital to human visual perception. The scientists from the National Eye Institute (NEI) discovered five subpopulations of retinal pigment epithelium (RPE)—a layer of tissue that nourishes and supports the retina’s light-sensing photoreceptors. Using artificial intelligence, the researchers analyzed images of RPE at single-cell resolution to create a reference map that locates each subpopulation within the eye. A report on the research was published on May 6, 2022 in PNAS. Th article is titled ““Single Cell-Resolution Map of Human Retinal Pigment Epithelium Helps Discover Subpopulations with Differential Disease Sensitivity.”

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Cutting Calories and Eating at Right Time of Day Leads to Longer Life in Mice in Takahashi-Led Study

One recipe for longevity is simple, if not easy to follow: eat less. Studies in a variety of animals have shown that restricting calories can lead to a longer, healthier life. Now, new research suggests that the body’s daily rhythms play a big part in this longevity effect. Eating only during their most active time of day substantially extended the lifespan of mice on a reduced-calorie diet, Howard Hughes Medical Institute Investigator Joseph Takahashi (photo), PhD, and colleagues reported on May 5, 2022, in Science. The article is titled “Circadian Alignment of Early Onset Caloric Restriction Promotes Longevity in Male C57BL/6J Mice.” In his team’s study of hundreds of mice over four years, a reduced-calorie diet alone extended the animals’ lives by 10 percent. But feeding mice the diet only at nighttime, when mice are most active, extended life by 35 percent. That combination–a reduced-calorie diet plus a nighttime eating schedule–tacked on an extra nine months to the animals’ typical two-year median lifespan. For people, an analogous plan would restrict eating to daytime hours.

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Tracing a Cancer’s Family Tree to Its Roots Reveals How Tumors Grow

Family trees of lung cancer cells, built using CRISPR-based lineage tracing, reveal in depth how cancer evolves from its earliest stages to become aggressive and capable of spreading throughout the body. Over time, cancer cells can evolve to become resistant to treatment, more aggressive, and metastatic—capable of spreading to additional sites in the body and forming new tumors. The more of these traits that a cancer evolves, the deadlier it becomes. Researchers want to understand how cancers evolve these traits in order to prevent and treat deadly cancers, but by the time cancer is discovered in a patient, it has typically existed for years or even decades. The key evolutionary moments have come and gone unobserved.

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Researchers Find Link Between Parkinson’s Gene (Alpha-Synclein) and Vocal Issues That Could Lead to Earlier Diagnosis; Experiment in Zebra Finch Animal Model Proves Key

Parkinson’s disease is perhaps best known for its movement-related symptoms, particularly tremors and stiffness. But the disease is also known to hinder vocal production, giving those with Parkinson’s a soft monotonous voice. Those symptoms, research has suggested, often appear much earlier in the disease’s development–sometimes decades before movement-related symptoms. New research by University of Arizona neuroscientists suggests that a specific gene commonly associated with Parkinson’s may be behind those vocal-related issues–a finding that could help lead to earlier diagnoses and treatments for Parkinson’s patients. The research was conducted in the lab of Julie E. Miller, PhD, an Assistant Professor of Neuroscience and of Speech, Language, and Hearing Sciences in the College of Science. “We have this big gap here–we don’t know how this disease impacts the brain regions for vocal production, and this is really an opportunity to intervene early and come up with better treatments,” said Dr. Miller, who also has joint appointments in the Department of Neurology and the Graduate Interdisciplinary Program in Neuroscience, and is a member of the U Arizona BIO5 Institute.

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Investigating Cancer Drug Toxicity Leads to Critical Discovery; Researchers Uncover New Strategy to Avoid Cancer Immunotherapy Side Effects

It’s not often that a failed clinical trial leads to a scientific breakthrough. When patients in the UK started showing adverse side effects during a cancer immunotherapy trial, researchers at La Jolla Institute for Immunology (LJI) Center for Cancer Immunotherapy and the University of Liverpool went back through the data and worked with patient samples to see what went wrong. Their findings, published on May 4, 2022 in Nature, provide critical clues to why many immunotherapies trigger dangerous side effects—and point to a better strategy for treating patients with solid tumors. “This work shows the importance of learning from early-stage clinical trials,” says La Jolla Institute for Immunology (LJI) Professor Pandurangan Vijayanand, MD, PhD, who co-led the new research with Christian H. Ottensmeier, MD, PhD, FRCP, a professor with  the University of Liverpool, The Clatterbridge Cancer Centre NHS Foundation Trust, and adjunct professor at LJI.

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Low-Cost Handheld Device Could Painlessly Identify Skin Cancers in Seconds; Researchers at Stevens Institute of Technology Use Millimeter-Wave Imaging to Potentially Slash Rate of Unnecessary Biopsies

Skin biopsies are no fun: doctors carve away small lumps of tissue for laboratory testing, leaving patients with painful wounds that can take weeks to heal. That’s a price worth paying if it enables early cancer treatment. However, in recent years, aggressive diagnostic efforts have seen the number of biopsies grow approximately four times faster than the number of cancers detected, with about 30 benign lesions now biopsied for every case of skin cancer that’s found. Researchers at Stevens Institute of Technology are now developing a low-cost handheld device that could cut the rate of unnecessary biopsies in half and give dermatologists and other frontline physicians easy access to laboratory-grade cancer diagnostics. “We aren’t trying to get rid of biopsies,” said Negar Tavassolian, PhD, Associate Professor, Director of the Bio-Electromagnetics Laboratory at Stevens. “But we do want to give doctors additional tools and help them to make better decisions.”

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BrainStorm Announces Upcoming Scientific Conference Presentations: Data on MSC-NTF Exosome Technology Will Be Described at ISCT 2022 and ISEV2022 Conferences; NurOwn® CSF Biomarker Analysis to Be Presented at ALS Drug Development Summit

On May 3, 2022, BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, announced presentations at upcoming scientific conferences in May 2022. New data on the company’s proprietary MSC-NTF exosome technology will be presented at  ISCT 2022 (May 4 to 7 in San Francisco, California) and at the ISEV 2022 Annual Meeting (May 25 to 29 in Lyon, France).  An analysis of CSF biomarkers from the NurOwn® Phase 3 trial will be presented at the ALS Drug Development Summit (May 24 to 26 in Boston, Massachusetts). [Editor’s Note: MSC-NTF is mesenchymal stem cells-neurotrophic factors; CSF is cerebrospinal fluid.]

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BrainStorm Announces Presentation of NurOwn® Exosome Preclinical Data at ISCT 2022 San Francisco Meeting; NurOwn-Derived Exosomes Showed More Potent Anti-Inflammatory Effects in Vitro Compared to Naïve Mesenchymal Stem Cell-Derived Exosomes

On May 4, 2022, BrainStorm Cell Therapeutics, Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, announced the presentation of a poster titled, “MSC-NTF Derived Small Extracellular Vesicles Display Superior Macrophage Immunomodulation Compared with Vesicles Derived from Naïve MSCs,” at the International Society of  Cell & Gene Therapy ISCT 2022 Meeting, being held from May 4-7, 2022 in San Francisco, California. The poster will be presented by Kim Thacker, MD, Senior Vice President, Medical Affairs and Clinical Innovation, BrainStorm Cell Therapeutics.

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Octopus and Squid Genome Studies Reveal How Cephalopods’ Unique Traits Evolved

Squid, octopus, and cuttlefish–even to scientists who study them–are wonderfully weird creatures. Known as the soft-bodied or coleoid cephalopods, they have the largest nervous system of any invertebrate, complex behaviors such as instantaneous camouflage, arms studded with dexterous suckers, and other evolutionarily unique traits. Now, scientists have dug into the cephalopod genome to understand how these unusual animals came to be. Along the way, the researchers discovered cephalopod genomes are as weird as the animals are. Scientists from the Marine Biological Laboratory (MBL) in Woods Hole, Massachusetts; the University of Vienna; the University of Chicago; the Okinawa Institute of Science and Technology; and the University of California, Berkeley, reported their findings in two new studies in Nature Communications.  “Large and elaborate brains have evolved a couple of times,” said co-lead author Caroline Albertin, PhD, Hibbitt Fellow at the MBL. “One famous example is the vertebrates. Another is the soft-bodied cephalopods, which serve as a separate example for how a large and complicated nervous system can be put together. By understanding the cephalopod genome, we can gain insight into the genes that are important in setting up the nervous system, as well as into neuronal function.”

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