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Capricor Therapeutics Presents Results of Studies of CAP-2003 (Including Exosomes) at Gordon Research Conference on Extracellular Vesicles

On August 21, 2018, it was announced that at the Gordon Research Conference on Extracellular Vesicles in Newry, Maine, Capricor Therapeutics (NASDAQ: CAPR) presented research findings on the mechanism of action and the immunomodulatory capacities of CAP-2003, the company’s investigational therapy comprised of proprietary extracellular vesicles (EVs), including exosomes, which are derived from cardiosphere-derived cells (CDC-EVs). Capricor is developing CAP-2003 as a therapeutic platform for treating diseases involving inflammation and fibrosis. The Gordon Research Conference on Extracellular Vesicles is focused on cutting-edge research on the biogenesis, molecular composition, functions, physio-pathological roles, and potential clinical applications of extracellular vesicles. Gordon Research Conferences are a group of prestigious international scientific conferences that are at the forefront of research in the biological, chemical, and physical sciences, and their related technologies. “The pre-clinical studies presented at the Gordon Research Conference further elucidate Capricor’s progress in developing this exciting new class of therapeutics, the exosomes which comprise our investigational therapy, CAP-2003,” said Linda Marbán, PhD, Capricor CEO. “The studies further demonstrate that exosomes may be the active pharmaceutical ingredient (API) in CAP-1002, our cell therapy product, because these extracellular vesicles serve as cellular-messengers, altering function and physiology to balance inflammation so that cellular repair can be facilitated.” In the first study, Capricor compared CAP-2003 with exosomes made from mesenchymal stem cells (MSCs).

Innovative Research Described at Annual Meeting of American Association of Stem Cell Physicians

The American Association of Stem Cell Physicians (AAOSCP) hosted its weekend conference August 10-12, 2018 in Miami, Florida, at the Mandarin Oriental. The event was sold out. Its huge success was thanks to the participation of some of the influential keynote speakers in research, medicine, and academia. The founders of the AAOSCP are leaders in medicine who are paving cutting-edge advancements in regenerative medicine. At the 2018 annual American Association of Stem Cell Physicians conference, exciting topics were discussed, such as: Johns Hopkins workshop on retina regeneration by Dr. Scheffer Tseng; Miami's own Bascom Palmer research scientist and retina specialist Dr. Alfonso Sabater talked about tissue engineering strategies for corneal and ocular reconstruction; bioplasty pioneer and Iinventor Dr. Carlos Mercado, who discussed low-intensity shock wave acellular treatments; Kimera Labs introduced everyone to Paul, a quadriplegic who has received successful treatments of intrathecally exosomes with positive results; Cornell University's Dr. Sunny Kim spoke about stem cell therapy for arthritis; and New York University Dr. Benjamin Bieber showcased innovative injection techniques to improve regenerative medicine outcomes. Harvard and Duke were among the prominent institutions represented at the conference, discussing topics about tissue regeneration. It was a stellar event. "The conference was a huge success. It was the first conference to host researchers, academia, and clinical all in one room, great minds exchanging vital information and techniques for the progress of medicine. We look forward to the positive impact this will have on health care," said Bernard Lessa-Bastos, AAOSCP Marketing Director.

Pheromone Discovery Can Help Farmers Combat Stink Bugs, Possibly Saving Millions on Pest Control

For stink bugs to attract a mate or to communicate that they have found food, they use their own chemical language: pheromones. Virginia Tech researchers have discovered insights into this chemical language, which can be used to develop alternative pest controls. "We have gained a deeper understanding of how stink bugs synthesize pheromones, and this knowledge may allow us to produce pheromones in expendable food crops - also called 'trap crops' - to lure the bugs away from cash crops," said Dr. Dorothea Tholl, a Professor of Biological Sciences in the College of Science and a Fralin Life Science Institute affiliate. These new environmentally friendly and sustainable alternatives to insecticides could save farmers millions of dollars. In Virginia, crops such as grapes, sweet corn, and apples, have been under attack by the invasive brown marmorated stink bug (image) since 2004; cabbage has also been affected, but by the harlequin stink bug. A relative, the southern green stinkbug is also a severe pest worldwide and attacks many different crops including beans and soybeans. Dr. Tholl is interested in the chemical communication of organisms and studies how this chemical language has evolved in insects. With support by a grant from the USDA National Institute of Food and Agriculture, her lab investigates the enzymes that produce stinkbug pheromones in an interdisciplinary collaboration with colleagues at Virginia Tech and national and international institutions. Her team's research has recently been published in PNAS. "Our recent paper provides valuable insight into our understanding of how insects synthesize complex sesquiterpene compounds that are typically used as pheromones.

New Scale for Prioritizing Tumor DNA Mutations Will Simplify & Standardize Choices for Targeted Cancer Treatments

A new scale for tumor DNA mutations which will simplify and standardize choices for targeted cancer treatment has been agreed upon by leading cancer specialists in Europe and North America. The scale, called ESCAT (ESMO Scale for Clinical Actionability of Molecular Targets), was published online on August 21, 2018 in the Annals of Oncology. The ESMO is the European Society for Medical Oncology. The new scale aims to optimize patient care by making it easier to identify patients with cancer who are likely to respond to precision medicines, and help make treatment more cost-effective. "Doctors receive a growing amount of information about the genetic make-up of each patient's cancer, but this can be difficult to interpret for making optimal treatment choices," explains Professor Fabrice André, Chair of the ESMO Translational Research and Precision Medicine Working Group who initiated this project. "The new scale will help us distinguish between alterations in tumor DNA that are important for decisions about targeted medicines or access to clinical trials, and those which aren't relevant."The new grading system classes alterations in tumor DNA according to their relevance as markers for selecting patients for targeted treatment, based on the strength of clinical evidence supporting them (Tier I-V, Table 1). It is the first time that a classification has been developed that is relevant to all potential targeted cancer medicines, not just those that have been approved for use by national regulatory bodies. The classification also enables mutations to be upgraded or downgraded in response to newly available data.

Maple Leaf Extract May Prevent Wrinkles, According to Presentation at ACS Meeting in Boston

Maple trees are best known for their maple syrup and lovely fall foliage. But it turns out that the beauty of those leaves could be skin-deep -- and that's a good thing. Today, scientists report that an extract from the leaves may prevent wrinkles. The researchers are presenting their results at the 256th National Meeting & Exposition of the American Chemical Society (ACS). ACS, the world's largest scientific society, is holding the meeting in Boston August 19-August 23, 2018. The ACS meeting features more than 10,000 presentations on a wide range of science topics. The maple leaf scientists had previously studied the chemistry and health benefits of sap and syrup obtained from sugar maple and red maple trees. Historical records suggested that other parts of the trees could also be useful, according to Navindra P. Seeram, PhD, the project's principal investigator. "Native Americans used leaves from red maple trees in their traditional system of medicine," he notes, "so why should we ignore the leaves?" Skin elasticity is maintained by proteins such as elastin. Wrinkles form when the enzyme elastase breaks down elastin in the skin as part of the aging process. "We wanted to see whether leaf extracts from red maple trees could block the activity of elastase," says Hang Ma, PhD, who is presenting the work at the ACS meeting and is a research associate in Dr. Seeram's lab. The researchers, who are at the University of Rhode Island, zeroed in on phenolic compounds in the leaves known as glucitol-core-containing gallotannins (GCGs) and examined each compound's ability to inhibit elastase activity in a test tube.

“Liquid Biopsy” (ctDNA) Predicts Lymphoma Therapy Success Within Days, Stanford-Led Study Finds

A blood test can predict which patients with a type of cancer called diffuse large B cell lymphoma are likely to respond positively to initial therapy and which are likely to need more aggressive treatment, according to a multicenter study led by researchers at the Stanford University School of Medicine. The study validates the clinical usefulness of tracking the rise and fall of circulating tumor DNA (ctDNA) in the blood of patients before and after therapy. It suggests that clinicians may soon be able to determine how a patient is responding to treatment within days or weeks of starting therapy rather than waiting until therapy is completed five to six months later. "Although conventional therapy can cure the majority of patients with even advanced B cell lymphomas, some don't respond to initial treatment," said Associate Professor of Medicine Ash Alizadeh, MD, PhD. "But we don't know which ones until several months have passed. Now, we can predict nonresponders within 21 days after the initiation of treatment by tracking the levels of ctDNA in a patient's blood. We can look earlier and make a reliable prediction about outcome." The study was published online on August 20, 2018 in the Journal of Clinical Oncology. The article is titled “Circulating Tumor DNA Measurements As Early Outcome Predictors in Diffuse Large B-Cell Lymphoma.” Dr. Alizadeh shares senior authorship with Associate Professor of Radiation Oncology Maximilian Diehn, MD, PhD. Instructor of Medicine David Kurtz, MD, PhD, and postdoctoral scholar Florian Scherer, MD, are the lead authors. Diffuse large B cell lymphoma, a blood cancer, is the most common type of non-Hodgkin lymphoma.

Hydrogel Technique for Delivering Muscle Stem Cells to Injured, Aging Muscle Tissue Is Successful in Model System; Technique May Ultimately Be Applicable to Duchenne Muscular Dystrophy

A car accident leaves an aging patient with severe muscle injuries that won’t heal. Treatment with muscle stem cells from a donor might restore damaged tissue, but doctors are unable to deliver them effectively. A new method may help change this. Researchers at the Georgia Institute of Technology (Georgia Tech) engineered a molecular matrix, a hydrogel, to deliver muscle stem cells called muscle satellite cells (MuSCs) directly to injured muscle tissue in patients whose muscles don’t regenerate well. In lab experiments on mice, the hydrogel successfully delivered MuSCs to injured, aged muscle tissue to boost the healing process while protecting the stem cells from harsh immune reactions. The method was also successful in mice with a muscle tissue deficiency that emulated Duchene muscular dystrophy, and if research progresses, the new hydrogel therapy could one day save the lives of people suffering from the disease. Simply injecting additional muscle satellite cells into damaged, inflamed tissue has proven inefficient, in part because the stem cells encounter an immune system that opposes them. “Any muscle injury is going to attract immune cells. Typically, this would help muscle stem cells repair damage. But in aged or dystrophic muscles, immune cells lead to the release a lot of toxic chemicals like cytokines and free radicals that kill the new stem cells,” said Dr. Young Jang, an Assistant Professor in Georgia Tech’s School of Biological Sciences and one of the study’s principal investigators. Only between 1 and 20 percent of injected MuSCs make it to damaged tissue, and those that do, arrive there weakened. Also, some tissue damage makes any injection unfeasible, thus the need for new delivery strategies. “Our new hydrogel protects the stem cells, which multiply and thrive inside the matrix.

Cellect’s Stem Cell Selection Technology Patent Receives Notice of Allowance in Korea

On August 20, 2018, it was announced that, in Korea, Cellect recently signed a collaboration agreement with Cell2in to improve stem cell selection and expansion. The agreement covers use of the ApoTainer™ device, as well as methods employing the device in stem cell selection for conditions including graft-versus-host disease (GvHD). On August 20, 2018 in Tel Aviv, Israel, Cellect Biotechnology Ltd., a developer of a novel stem cell production technology and headquartered in Israel, announced that it has received a Notice of Allowance from the Korean Intellectual Property Office for its patent titled, "Devices and Methods for Selecting Apoptosis-Signaling Resistant Cells, and Uses Thereof.” This patent, recently granted to Cellect in Europe, addresses the Company's ApoTainer™ device which is used in conjunction with its platform ApoGraft™ technology. "As we expand the number of stem cell industry collaborations for Cellect worldwide, our international patent assets and protections become increasingly important. In the past six weeks alone, we entered into collaborations with companies in Germany, Switzerland, and Korea. Working with industry partners to improve the safety and efficacy of stem cells and expanding regenerative medicine's wide scale-availability and affordability are cornerstones of Cellect's strategy. Our growing IP estate supports this purpose," stated Cellect CEO Dr. Shai Yarkoni. The patent addresses Cellect's devices and methods for specifically selecting desired stem cells from a heterogeneous cell population for use in a range of medical indications. Through negative selection, Cellect's technology identifies mature cells that can be harmful to the recipient and selectively eliminates those cells through apoptosis (cell death).

Advance in Research on Treatment for Retinitis Pigmentosa—Knockdown & Replacement of Rhodopsin Gene in Dog Model

The last year has seen milestones in the gene therapy field, with FDA approvals to treat cancer and an inherited blinding disorder. New findings from a team led by University of Pennsylvania vision scientists, who have in the past taken gene therapies into clinical trials, are proving successful, this time treating a form of retinitis pigmentosa, a disease that progressively robs people of their night and peripheral vision before blindness develops. The researchers, from Penn's School of Veterinary Medicine and Perelman School of Medicine, in collaboration with University of Florida scientists, developed a therapy that effectively eliminates the abnormal copy of rhodopsin, a light-sensing molecule, and then restores it with a healthy copy of the protein. This knockdown and replacement approach preserved the retina's light-sensing photoreceptor cells in affected dogs, which can develop a very similar disease to affected humans. What's more, the scientists accomplished this using a single viral vector (AAV) to co-deliver the genetic material needed to achieve both the knockdown and replacement. Though more than 150 different mutations in rhodopsin have been identified to cause retinitis pigmentosa, this approach is intended to work regardless of the mutation or the mechanism by which rod photoreceptor cells, those responsible for vision in dim light, die. That means that a large percentage of patients with rhodopsin autosomal dominant retinitis pigmentosa could benefit if the therapy is found to be safe and effective in people. "It's a one treatment fits all," says Dr. William A. Beltran, Professor of Ophthalmology and Director of the Division of Experimental Retinal Therapies at Penn Vet and co-lead author of the study, which was published online on August 20, 2018 in PNAS.

Perinatal Hypoxia Associated with Long-Term Cerebellar Learning Deficits and Purkinje Cell Misfiring

Oxygen deprivation associated with preterm birth leaves telltale signs on the brains of newborns in the form of alterations to cerebellar white matter at the cellular and the physiological levels. Now, an experimental model of this chronic hypoxia reveals that those cellular alterations have behavioral consequences. Chronic sublethal hypoxia is associated with locomotor miscoordination and long-term cerebellar learning deficits in a clinically relevant model of neonatal brain injury, according to a study led by Children's National Health System researchers published online August 13, 2018 in Nature Communications. The open-access article is titled “Neonatal Brain Injury Causes Cerebellar Learning Deficits and Purkinje Cell Dysfunction.” Using high-tech optical and physiological methods that allow researchers to turn neurons on and off and an advanced behavioral tool, the research team finds that Purkinje cells fire significantly less often after injury due to perinatal hypoxia. However, an off-the-shelf medicine now used to treat epilepsy enables those specialized brain cells to regain their ability to fire, improving locomotor performance. Step out of the car onto the pavement, hop up to the level of the curb, stride to the entrance, and climb a flight of stairs. Or, play a round of tennis. The cerebellum coordinates such locomotor performance and muscle memory, guiding people of all ages as they adapt to a changing environment. "Most of us successfully coordinate our movements to navigate the three-dimensional spaces we encounter daily," says Vittorio Gallo, PhD, Children's Chief Research Officer and the study's senior author. "After children start walking, they also have to learn how to navigate the environment and the spaces around them." These essential tasks, Dr.

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