Stem Cell Clinic

BlueRock Therapeutics to Present at 2019 Cell & Gene Meeting on the Mesa – BioSpace

CAMBRIDGE, Mass., Sept. 25, 2019 /PRNewswire/ --BlueRock Therapeutics, today announced that Emile Nuwaysir, Ph.D., Chief Executive Officer of BlueRock and Vice Chairman of the Alliance for Regenerative Medicine (ARM), will present at the Cell & Gene Meeting on the Mesa, to be held Oct. 2-4 in Carlsbad, Calif.

The following are specific details regarding BlueRock's participation at the conference:Event: BlueRock Therapeutics Company PresentationDate: Wednesday, Oct. 2Time: 11:45 a.m. - 12:00 p.m. PDTLocation: Cognate Bioservices Ballroom,Park Hyatt Aviara Resort, 7100 Aviara Resort Dr., Carlsbad, CA 92011

Event: Panel: Cell and Gene Therapy for Neurological Product IndicationsDate: Wednesday, Oct. 2Time: 4:00 - 5:00 p.m. PDTLocation: BlueRock Therapeutics Ballroom, Park Hyatt Aviara Resort, 7100 Aviara Resort Dr., Carlsbad, CA 92011

A live video webcast of all company presentations and workshops will be available at: http://www.meetingonthemesa.com/webcast and will also be published on the conference website shortly after the event.

Organized by the Alliance for Regenerative Medicine, the Cell & Gene Meeting on the Mesa is a three-day conference featuring more than 80 dedicated company presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering, and broader regenerative medicine technologies, as well as over 100 panelists and featured speakers.

About BlueRock Therapeutics BlueRock Therapeutics, a wholly-owned and independently operated subsidiary of Bayer AG, is a leading engineered cell therapy company with a mission to develop regenerative medicines for intractable diseases. BlueRock Therapeutics'CELL+GENE platform harnesses the power of cells for new medicines across neurology, cardiology and immunology indications. BlueRock Therapeutics' cell differentiation technology recapitulates the cell's developmental biology to produce authentic cell therapies, which are further engineered for additional function. Utilizing these cell therapies to replace damaged or degenerated tissue brings the potential to restore or regenerate lost function. BlueRock's culture is defined by scientific innovation, highest ethical standards and an urgency to bring transformative treatments to all who would benefit. For more information, visit http://www.bluerocktx.com.

Company Contact:Candice Ellis, 978-879-7273Associate Director, Investor Relations and Corporate Communicationscellis@bluerocktx.com

Media Contacts: David Schull or Maggie BellerRusso Partners, LLC(646) 942-5631Maggie.beller@russopartnersllc.com

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SOURCE BlueRock Therapeutics

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BlueRock Therapeutics to Present at 2019 Cell & Gene Meeting on the Mesa - BioSpace

Novel CAR T-cell therapy for leukaemia and lymphoma targets BAFF-R – Drug Target Review

Scientists in the US have developed a promising new CAR T-cell therapy that targets the BAFF-R protein, which has demonstrated superior cancer destruction to existing FDA-approved CAR-T therapies.

The first CAR T-cell therapy targeting the B cell-activating factor receptor on cancerous cells, eradicated CD19-targeted therapy-resistant human leukaemia and lymphoma cells in animal models, according to research by theCity of Hope.

The new therapy will be used in a clinical trial next year for patients who relapsed after CD19 immunotherapy treatments and may also be potentially used as a first-line of CAR T-cell therapy treatment.

An estimated 20 to 30 percent of leukaemia and lymphoma patients who achieve remission after receiving CD19 CAR-T therapy will relapse after a few years. The effectiveness of the CAR-T cells that target the CD19 protein on cancerous B cells begins to wane and the cancer returns.This new therapy offers a possible solution for such patients.

One major obstacle to current CAR-T therapy is that in up to a third of these patients, the tumour is actually smart and comes back because it has learned how to no longer express the target thats recognised by the original immunotherapy, saidLarry Kwak, MD, PhD, vice president and deputy director of City of Hopes comprehensive cancer center and the studys principal author, who leads the research with Hong Qin, PhD, a research professor in City of Hopes Department of Hematology & Hematopoietic Cell Transplantation.

To combat that, City of Hope research has found a new, and potentially more effective, target for CAR T-cell therapy against B cell leukaemias and lymphomas. We plan to open a clinical trial next year using the BAFF-R CAR T-cell therapy.

This new treatment could change the face of leukaemia and lymphoma treatment in the US and worldwide, added Kwak, the Dr Michael Friedman Professor in Translational Medicine.

CAR T-cell therapy involves taking a patients T cells from the bloodstream. The immune cells are then genetically engineered in a laboratory to recognise and attack a specific cancer-associated protein, such as BAFF-R, before being reintroduced into the patients bloodstream, whereupon they begin destroying tumour cells.

For this study, animal models with CD19 therapy-resistant human-tumours (including Burkitt, mantel cell, and other non-Hodgkins lymphoma subtypes and acute lymphoblastic leukaemia) received BAFF-R CAR-T therapy. Remarkable tumour regression and prolonged survival were observed after treatment with these CAR-T cells. In animal models with human Burkitt lymphoma, BAFF-R CAR-T therapy achieved a cure (complete tumour regression with 100 percent long-term survival) after a single treatment.

Tumour samples from patients who relapsed after receiving CD19-targeted immunotherapy (blinatumomab) were also investigated. The study demonstrated that BAFF-R CAR-T cells were consistently active against these tumours, whereas CD19 CAR-T cells had greatly diminished responses to each patients relapse tumour compared to the pre-therapy samples.

We did a head-to-head comparison of two new therapies and our data showed that our BAFF-R CAR-T therapy actually did a better job than FDA-approved CD19 CAR-T therapies, Qinsaid. If these results continue, we will pursue BAFF-R CAR-T therapy as a kind of frontline CAR-T therapy treatment for lymphoma and leukaemia patients.

City of Hope plans to open a clinical trial next year using the BAFF-R CAR T-cell therapy for B cell leukaemia and lymphoma patients who have relapsed after receiving CD19 CAR T-cell therapies or blinatumomab.

This research was published inScience Translational Medicine.

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Novel CAR T-cell therapy for leukaemia and lymphoma targets BAFF-R - Drug Target Review

PolarityTE Selected to Present at 2019 Cell & Gene Meeting on the Mesa – Business Wire

SALT LAKE CITY--(BUSINESS WIRE)--PolarityTE, Inc. (Nasdaq: PTE), a biotechnology company developing and commercializing regenerative tissue products and biomaterials, today announced that Nikolai Sopko, MD, PhD, Chief Scientific Officer of PolarityTE, was selected to present at the annual Cell & Gene Meeting on the Mesa, which will be held from October 2-4, 2019 in Carlsbad, California. Dr. Sopko will discuss the Companys R&D pipeline and highlight the progress made thus far with the Companys first clinically available product, SkinTE, an autologous, homologous human cellular and tissue-based product designed to regenerate full-thickness, functional skin for use in wounds, traumas, and burns.

PolarityTE is scheduled to present on October 3rd from 9:45-10am PST in the Cognate Bioservices Ballroom, Park Hyatt Aviara Resort.

A live video webcast of all company presentations will be available at: http://www.meetingonthemesa.com/webcast and will also be published on the conference website shortly after the event. Please visit http://www.meetingonthemesa.com for full information on the conference, including registration.

Organized by the Alliance for Regenerative Medicine, the Cell & Gene Meeting on the Mesa is a three-day conference featuring more than 80 dedicated company presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering, and broader regenerative medicine technologies, as well as over 100 panelists and featured speakers.

About PolarityTE

PolarityTE is focused on transforming the lives of patients by discovering, designing and developing a range of regenerative tissue products and biomaterials for the fields of medicine, biomedical engineering and material sciences. Rather than manufacturing with synthetic and foreign materials within artificially engineered environments, PolarityTE manufactures products from the patient's own tissue and uses the patient's own body to support the regenerative process. From a small piece of healthy autologous tissue, the company creates an easily deployable, dynamic and self-propagating product designed to regenerate the target tissues. PolarityTE's innovative method is intended to promote and accelerate growth of the patient's tissues to undergo a form of effective regenerative healing. Learn more at http://www.PolarityTE.com Welcome to the Shift.

About SkinTE

SkinTE is a human cellular and tissue-based product derived from a patient's own skin intended for the repair, reconstruction, and replacement of skin tissue. SkinTE has been proven to regrow skin over exposed bone, muscle, joint and tendon and has been used to treat a variety of skin defects, including burns, wounds, traumatic injuries, surgical reconstruction, scars, and failed skin grafts or conventional treatments for wounds and burns.

SkinTE is intended to be used by physicians or other appropriate healthcare providers for homologous uses of skin tissues/integument. Patients who have suffered from an event, disease, process or acquired deficit that results in the functional loss or void of skin/integument systems can receive SkinTE as an adjunct and/or in place of split-thickness skin grafting, full-thickness grafting, temporizing skin coverage and/or skin substitute products. SkinTE is for autologous use only. Aseptic technique during harvest and deployment of SkinTE is mandatory. SkinTE is marketed as an HCT/P regulated by the FDA solely under Section 361 of the Public Health Service Act and 21 CFR 1271.

Forward Looking Statements

Certain statements contained in this release are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. They are generally identified by words such as "believes," "may," "expects," "anticipates," "intend," "plan," "will," "would," "should" and similar expressions. Readers should not place undue reliance on such forward-looking statements, which are based upon the Company's beliefs and assumptions as of the date of this release. The Company's actual results could differ materially due to risk factors and other items described in more detail in the "Risk Factors" section of the Company's Annual Reports and other filings with the SEC (copies of which may be obtained at http://www.sec.gov). Subsequent events and developments may cause these forward-looking statements to change. The Company specifically disclaims any obligation or intention to update or revise these forward-looking statements as a result of changed events or circumstances that occur after the date of this release, except as required by applicable law.

POLARITYTE, the POLARITYTE logo, WHERE SELF REGENERATES SELF, WELCOME TO THE SHIFT, and SKINTE are trademarks or registered trademarks of PolarityTE, Inc.

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PolarityTE Selected to Present at 2019 Cell & Gene Meeting on the Mesa - Business Wire

Natera Sees Foundation Medicine Deal as "Additive" to Other Cancer Monitoring Opportunities – GenomeWeb

NEW YORK Natera's recent deal with Foundation Medicine to develop personalized cancer monitoring assays will allow it to piggyback on the Roche subsidiary's dominant market share in tumor genomic profiling to establish its Signatera test for use in advanced cancer patients, while continuing to pursue other opportunities for the test on its own.

Foundation Medicine, on the other hand, is banking on Natera's technology and expertise in circulating tumor DNA analysis to bring monitoring assays to patients more quickly than it could on its own.

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Natera Sees Foundation Medicine Deal as "Additive" to Other Cancer Monitoring Opportunities - GenomeWeb

Don’t Be Shy About Asking to Remain in the Hospital – Sickle Cell Anemia News

One of the best feelings in the world is cutting a hospital bracelet.

Cutting a hospital bracelet can symbolize a fresh start. Youve been discharged from the hospital. You are feeling like the best version of yourself happy, relaxed, optimistic, and ready to prove that sickle cell cant keep you down.

However, sometimes I feel like hospital bracelets are cut prematurely.

I was admitted to the hospital Sept. 13 because of a vaso-occlusive crisis. I had pain in my arms, my back, and my legs. I was taking oral morphine, dihydrocodeine, and acetaminophen every four hours. I wouldve been arrested if I had been caught driving in that state.

It was frustrating, because every time the medications wore off, the pain was just as intense as it was before I was admitted. The pain wasnt getting better. In fact, the pain was getting worse, which meant the medicine just kept coming.

Days passed. It got to the point where every doctor, nurse, and consultant suggested I leave the hospital, as I could use the same painkillers at home. I declined each time.

I hate going to the hospital so much that I treat it as a last resort. You know its serious if I take myself to the emergency room. I need help that I cant sufficiently give myself.

My floor was full of elderly patients with cancer-related complications. There were three other women in my room who looked unwell. I was uncomfortable speaking to them and seeing the type of treatments they needed. The environment didnt promote positive thoughts, but I insisted on staying.

I sat in the emergency room for hours and was admitted because I was feeling sick. I didnt intend on leaving until I felt better, yet the constant suggestions made me feel like I was wasting National Health Service resources. I couldnt help but wonder if someone needed my bed more than me.

I was discharged Sept. 17, and I honestly regret not staying in the hospital. The pain is just as intense and Ive seriously considered going back.

This is just to encourage all you sickle cell warriors to stay put. If youre not feeling well enough to be discharged, you have every right to stay. You know your body best. If you feel awkward about advocating for yourself, speak to a friend or family member who can effectively relay the importance of staying in the hospital to the appropriate healthcare professionals.

***

Note: Sickle Cell Anemia News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Sickle Cell Anemia News or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to sickle cell anemia.

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Don't Be Shy About Asking to Remain in the Hospital - Sickle Cell Anemia News

Silencing of TAZ inhibits the motility of hepatocellular carcinoma cel | CMAR – Dove Medical Press

Wei Zhou,1,2,* Jiachun Weng,1,* Keyan Wu,1,2,* Xiao Xu,1,* Hui Wang,1,* Jing Zhang,3,* Chengxue Zhao,1 Jie Yang,1 Yu Zhang,1,4,5 Weigan Shen1,4,5

1Department of Cell Biology, School of Medicine of Yangzhou University, Yangzhou, Peoples Republic of China; 2Department of Internal Medicine, Affiliated Hospital of Yangzhou University, Yangzhou, Peoples Republic of China; 3Department of Internal Medicine, Northern Jiangsu Peoples Hospital Affiliated to Yangzhou University, Yangzhou, Peoples Republic of China; 4Jiangsu Co-Innovation Center for Prevention and Control of Important Animal Infectious Diseases and Zoonoses, Yangzhou University, Yangzhou, Peoples Republic of China; 5Jiangsu Key Laboratory of Integrated Traditional Chinese and Western Medicine for Prevention and Treatment of Senile Diseases, Yangzhou University, Yangzhou, Peoples Republic of China

*These authors contributed equally to this work

Correspondence: Yu Zhang; Weigan ShenDepartment of Cell Biology, School of Medicine of Yangzhou University, No. 136 Middle Jiangyang Street, Yangzhou 225009, Jiangsu, Peoples Republic of ChinaTel +86 05 148 797 8875Fax +86 05 148 734 1733Email yzzy10182001@aliyun.com; shenweigan@hotmail.com

Purpose: The aim of the present study was to investigate the effect of knockdown and knockout of the transcriptional co-activator with PDZ-binding motif (TAZ) on the migration, invasion and autophagy of the hepatocellular carcinoma (HCC) cell lines, as well as the functional connection between the autophagy and cell migratory processes induced by loss of TAZ in HCC cell lines.Methods: HCC cell lines SMMC-7721 and SK-HEP1 stably knockdown and knockout of TAZ were established by the lentiviral-mediated TAZ knockdown and knockout approaches. Reverse transcription-quantitative real-time polymerase chain reaction and Western blotting were performed to examine the expression of TAZ and indicated genes in downstream pathways in HCC cell lines. Transwell assay and autophagic flux assay were used to evaluate the effect of TAZ knockdown and knockout on the motility and the autophagy of HCC cell lines.Results: We initially found that TAZ exhibited highly abundant and was expressed predominantly in HCC cell lines with different spontaneous metastatic potential. Through performing loss-of-function assays, we demonstrated that both TAZ knockdown and knockout promoted HCC cell autophagy and reduced HCC cell migration, invasion and epithelial-to-mesenchymal transition. In addition, autophagy inhibition in TAZ knockdown and knockout SMMC-7721 and SK-HEP1 cells in the presence of 3-methyladenine or chloroquine partially abrogated the migratory and invasive ability induced by TAZ knockdown and knockout.Conclusion: Our findings indicated that loss of TAZ in HCC cells suppressed cell motility probably via altering the autophagy, suggesting that TAZ emerges as an important target in regulating cell motility and autophagy in HCC cells, and blocking TAZ may be a novel therapeutic strategy against HCC.

Keywords: TAZ, autophagy, migration, invasion, hepatocellular carcinoma

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Silencing of TAZ inhibits the motility of hepatocellular carcinoma cel | CMAR - Dove Medical Press

Calithera to Host Webcast Conference Call for Analysts and Investors During European Society for Medical Oncology (ESMO) Congress 2019 – GlobeNewswire

SOUTH SAN FRANCISCO, Calif., Sept. 26, 2019 (GLOBE NEWSWIRE) -- Calithera Biosciences, Inc. (Nasdaq: CALA), a clinical-stage biotechnology company focused on discovering and developing novel small molecule drugs for the treatment of cancer and other life-threatening diseases, today announced it will host a conference call and audio webcast on Monday, September 30 at 5:30 a.m. Pacific Time (8:30 a.m. ET) to review data from the Calithera clinical program presented at the European Society for Medical Oncology (ESMO) Congress 2019. Calithera management will review data from the ENTRATA study evaluating the glutaminase inhibitor telaglenastat and data presented on INCB001158, an oral arginase inhibitor.

Details of the ESMO 2019 oral late-breaking presentation for telaglenastat are as follows:

Date:Saturday, September 28, 2019, 8:30 a.m. CET, Barcelona AuditoriumSession Title: Proffered Paper 1-Genitourinary tumors, non-prostateAbstract Title: "ENTRATA: Randomized, double-blind, phase 2 study of telaglenastat (CB-839) + everolimus vs. placebo + everolimus in patients with advanced/metastatic renal cell carcinoma (RCC)."R. Motzer, et al.Abstract:LBA54Presenter: Chung-Han Lee, M.D., PhD of the Memorial Sloan Kettering Cancer Center

Details of the oral presentation for INCB001158 are as follows:

Date:Sunday, September 29, 2019, 4:30 p.m. CET, Malaga AuditoriumSession Title: Proffered Paper Developmental therapeuticsAbstract Title: "Phase 1 study of the arginase inhibitor INCB001158 (1158) alone and in combination with pembrolizumab in patients with advanced/metastatic solid tumors."A. Naing, et al.Abstract:440O Presenter: Aung Naing, M.D., FACP, Associate Professor, Department of Investigational Cancer Therapeutics, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center

Link: Abstract is available at https://www.esmo.org/Conferences/ESMO-Congress-2019

To participate in the Calithera ESMO 2019 Call, please dial (855) 783-2599 (domestic) or (631) 485-4877 (international) five minutes prior to the start of the call and provide the conference ID 1469186. To access the live audio webcast or the subsequent archived recording, visit the Investors section of the Calithera website at http://www.calithera.com. The webcast will be recorded and available for replay on Calitheras website for 30 days.

About Telaglenastat

Telaglenastat is an investigational first-in-class, novel glutaminase inhibitor specifically designed to block glutamine consumption in tumor cells. RCC tumors commonly exhibit metabolic alterations that increase their dependence on glutamine. In preclinical studies, telaglenastat produced synergistic antitumor effects when used in combination with standard-of-care RCC therapies. On June 17, 2019, Calithera announced that a randomized Phase 2 trial of telaglenastat plus everolimus versus everolimus plus placebo (ENTRATA) met its primary endpoint of improving progression free survival, demonstrating proof of concept for telaglenastat in patients with advanced RCC. The ongoing CANTATA trial is a global, randomized, double-blind trial designed to evaluate the safety and efficacy of telaglenastat plus cabozantinib versus placebo plus cabozantinib in patients with advanced or metastatic RCC.

About INCB001158 (CB-1158)

INCB001158 (CB-1158) is an investigational first-in-class, novel small molecule arginase inhibitor. Arginase is an enzyme that suppresses the immune-mediated destruction of tumors by depleting levels of a key amino acid, L-arginine, from the tumor microenvironment. A number of cell types in the tumor microenvironment, including myeloid-derived suppressor cells, macrophages, and neutrophils, can secrete arginase. L-arginine deprivation can act via nutrient sensor pathways to exert several suppressive effects on T-cell function, inhibiting proliferation, decreasing cytokine production, and diminishing expression of the T-cell receptor CD3 chain. Arginase activity may thus impair T-cell mediated anti-tumor responses. INCB001158 is being developed in a global collaboration with Incyte Corporation.

About Calithera

Calithera Biosciences is a clinical-stage biopharmaceutical company pioneering the discovery and development of targeted therapies that disrupt cellular metabolic pathways to preferentially block tumor cells and enhance immune-cell activity. Driven by a commitment to rigorous science and a passion for improving the lives of people impacted by cancer and other life-threatening diseases, Calithera is advancing a pipeline of first-in-clinic, oral therapeutics to meaningfully expand treatment options available to patients. Calithera is headquartered in South San Francisco, California. For more information about Calithera, please visit http://www.calithera.com.

Forward Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "anticipate," "estimate," "intend," "poised" and similar expressions (as well as other words or expressions referencing future events, conditions, or circumstances) are intended to identify forward-looking statements. These statements include those related to the safety, tolerability and efficacy of Calitheras product candidates, the overall advancement of Calitheras product candidates in clinical trials, the unmet need in the treatment of patients with advanced disease, and Calitheras plans to continue development of its product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. The product candidates that Calithera develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all. In addition, clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release. Such product candidates may not be beneficial to patients or successfully commercialized. The failure to meet expectations with respect to any of the foregoing matters may have a negative effect on Calithera's stock price. Additional information concerning these and other risk factors affecting Calithera's business can be found in Calithera's most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission, and other periodic filings with the Securities and Exchange Commission at http://www.sec.gov. These forward-looking statements are not guarantees of future performance and speak only as of the date hereof, and, except as required by law, Calithera disclaims any obligation to update these forward-looking statements to reflect future events or circumstances.

SOURCE:Calithera Biosciences, Incorporated

CONTACT:

Jennifer McNealeyir@Calithera.com650-870-1071

MEDIA CONTACT:Hannah Hurdlehannahhurdle@sambrown.com 805-338-4752

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Calithera to Host Webcast Conference Call for Analysts and Investors During European Society for Medical Oncology (ESMO) Congress 2019 - GlobeNewswire

UCI School of Medicine researchers lead an international team for $4.3M NIH BRAIN Initiative award for new brain mapping tool – Newswise

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Newswise Irvine, CA - September 26, 2019 Professors Xiangmin Xu and Rozanne Sandri-Goldin, in collaboration with Professors Bert Semler and Todd Holmes at the University of California, Irvine, School of Medicine, were awarded a three-year, $4.3 million Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative grant. Working together with a dream team of US and Chinese scientists, they will lead the development of a new brain mapping tool for neuroscience research.

The mapping tool is based on genetically engineered herpesviruses. Herpes simplex virus (HSV) type 1, strain 129 (H129) will be harnessed as an anterograde, monosynaptic viral tracer with high labeling efficacy and low toxicity for neural circuit analysis.

Current versions of genetically modified H129 herpesviruses are limited primarily by high virulence and toxicity, said Xu. We have a comprehensive plan to reduce the toxicity and also to enhance signal outputs and generate variants carrying different functional payloads. Ultimately, we will be able to create a new set of safe, effective and validated anterograde-directed viral vectors.

Expected to have broad impact, the new tool will be made available through the UCI Center for Virus Research where it can be disseminated to the entire neuroscience community.

"The development of trans-synaptic viral tracers is an important component of the BRAIN Initiative, said Xu.At present, the lack of viral-based anterograde, monosynaptic tracing tools with high signal strength and low toxicity is a critical gap in neuroscience, preventing researchers from gaining a full understanding of how the brain works.

The team is excited about the prospect that new recombinant H129 viral vectors will lead to transformative tools for neuroscience research, and in the more distant future, clinical applications for human diseases such as Alzheimer's disease, Parkinson's disease and epilepsy. Xu and Sandri-Goldin have assembled a collaborative, interdisciplinary team composed of virologists and systems neuroscientists to develop the new neural analysis tool. Other principal investigators include Professor Gregory D. Horwitz from the University of Washington and Professor Min-Hua Luo from the Wuhan Institute of Virology, Chinese Academy of Sciences.

Their research proposal, "Genetically engineered anterograde monosynaptic viral tracers for multi-species neural circuit analysis, was submitted in response to a special RFA as part of the BRAIN Initiative: Development and Validation of Novel Tools to Probe Cell-Specific and Circuit-Specific Processes in the Brain.

About the NIH BRAIN Initiative

The Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative is aimed at revolutionizing our understanding of the human brain. By accelerating the development and application of innovative technologies, researchers will be able to produce a revolutionary new dynamic picture of the brain that, for the first time, shows how individual cells and complex neural circuits interact in both time and space. Long desired by researchers seeking new ways to treat, cure, and even prevent brain disorders, this picture will fill major gaps in our current knowledge and provide unprecedented opportunities for exploring exactly how the brain enables the human body to record, process, utilize, store, and retrieve vast quantities of information, all at the speed of thought. For more information, visit: https://www.braininitiative.nih.gov/

About the UCI School of Medicine

Each year, the UCI School of Medicine educates more than 400 medical students, as well as 200 doctoral and masters students. More than 600 residents and fellows are trained at UC Irvine Medical Center and affiliated institutions. The School of Medicine offers an MD; a dual MD/PhD medical scientist training program; and PhDs and masters degrees in anatomy and neurobiology, biomedical sciences, genetic counseling, epidemiology, environmental health sciences, pathology, pharmacology, physiology and biophysics, and translational sciences. Medical students also may pursue an MD/MBA, an MD/masters in public health, or an MD/masters degree through one of three mission-based programs: the Health Education to Advance Leaders in Integrative Medicine (HEAL-IM), the Leadership Education to Advance Diversity-African, Black and Caribbean (LEAD-ABC), and the Program in Medical Education for the Latino Community (PRIME-LC). The UCI School of Medicine is accredited by the Liaison Committee on Medical Accreditation and ranks among the top 50 nationwide for research. For more information, visit som.uci.edu.

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UCI School of Medicine researchers lead an international team for $4.3M NIH BRAIN Initiative award for new brain mapping tool - Newswise

Navigating the Clinical Trial Maze – Curetoday.com

Cutting-edge treatment is not out of reach, thanks to a little guidance from the Cancer Research Institute.

Currently, 4,000 active clinical trials involve immunotherapy drugs, which are examining T cell targets, cell therapy, vaccines and other immunomodulators. CRI works to help fund and advance this research.

Founded in 1953 by Helen Coley Nauts, the daughterof Dr. William B. Coley more commonly known as the Father of Cancer Immunotherapy and her friend Oliver R. Grace Sr., CRI has conducted research in this space for 65 years. Immunotherapy treats cancer by using the bodys immune system to prevent, target, control and eliminate the disease.

Decades of research have led to breakthrough treatments that mobilize our own immune systems to hunt down and eliminate cancer cells, wherever they are in the body, Jill ODonnell-Tormey, who holds a doctorate in philosophy, cell biology and anatomy, and serves as CEO and director of scientific affairs at CRI, said in an interview with CURE. Its a more sophisticated, dynamic and durable approach than other cancer treatments and may prove to be the best way to keep pace with cancers that evolve over time. Immunotherapies have also been proven to synergize with other forms of cancer treatment, increasing response rates while minimizing negative side effects and reducing risk of recurrence in some cancers.

The Food and Drug Administration has approved seven checkpoint inhibitors, a specific type of immunotherapy, to treat different cancers: Bavencio (avelumab), Imfinzi (durvalumab), Keytruda (pembrolizumab), Libtayo (cemiplimab), Opdivo (nivolumab), Tecentriq (atezoli- zumab) and Yervoy (ipilimumab).

A lot of research must still be done, according to ODonnell-Tormey. She encourages patients and their caregivers to be open to clinical trials, which give patients an opportunity to receive cutting-edge treatments under the watchful care of an expert team.

Many patients who participate in clinical trials describe the experience as very positive, often feeling part of the research team while cognizant that they are contributing to science and potentially helping future patients with cancer, she said. They can expect to receive lots of information about the study and the rationale supporting it, along with around-the-clock access to a clinical team that can answer any questions patients might have. While the treatments being tested are investigational, its important for patients to understand that new drugs or drug combinations undergo extensive laboratory testing before they are given to humans.

Often, the most challenging part of the process is finding a clinical trial. Not all health care providers discuss these studies with patients, and navigating some websitescan be difficult. CRI offers a Clinical Trial Finder to help patients learn about the basics and what to consider before enrolling, as well as connect with navigators who will walk them through the process.

Patients are asked to fill out a disease-specific questionnaire. The navigator needs to know the primary diagnosis, including tumor type; stage or extent of disease, such as if it has spread; and any treatment history. Then the navigator searches a national database of immunotherapy trials to find the best match for the patient.

Beyond locating trials and connecting to trial sites, our navigation is really customized education that varies by patient, said Caroline Melendez, director of client services at EmergingMed, the company that runs CRIs Clinical Trial Finder. Some people are very familiar with their diagnosis and treatment history, but those who have very little information are offered guidance about what to ask their health care team. Once a patient has identified trial matches, the navigator can help explain the differences between the types of treatment modalities and phases of trials being offered in their match results.

Enrollment time varies by trial and site, and patientsgo through an informed consent process, Melendez said. Next, they learn about the specific trial and undergo an examination to determine eligibility. The process can take several days, a few weeks or longer, she said.

We encourage every patient to ask this question: Is there an interesting clinical trial available to me today? Melendez said. The decision to enroll in a clinical trial is entirely dependent upon whether a compelling new therapy is in development. Everyones situation is unique, and patient interest in clinical trials should be informed by discussions with their medical team each time they have to make a treatment decision.

Several myths surround clinical trials, such as that they are a last resort or that patients are treated like guinea pigs. Another common misconception is that being on a trial means theres a chance youll receive placebo rather than treatment, ODonnell-Tormey said. This is not true except in cases where there are no existing treatments proven to improve patient outcomes. Generally, patients will receive some form of therapy, whether its standard of care, a new drug or a combination of the two.

Patients should investigate all options, educate them- selves and then make treatment decisions. Trials open and close all the time, but generally speaking, there are trialsfor patients at almost every phase of the journey, newly diagnosed to relapsed, Melendez said. Its never too early to get educated and connected to a clinical trial navigator, but it can be too late.

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Navigating the Clinical Trial Maze - Curetoday.com

The nanoengineer working to mend broken hearts – UNSW Newsroom

When Hossein Tavassoli first told his mother that he was researching heart disease, she responded with But, youre an engineer...

She was right, of course Hosseins undergraduate degree was in material engineering but at the time, he found it difficult to explain the intersections between biology and engineering. Now, not only is he a nanoengineer working in a tissue regeneration lab, but he is also an avid science communicator.

Its very interdisciplinary, says Hossein, who is completing the biology component of his PhD at UNSW Medicine and the engineering component at Swinburne University of Technology.

Hossein began working with the heart several years ago after finding himself interested in the heart-blood development and regeneration research of UNSW biologists Dr Vashe Chandrakanthan and Professor John Pimanda. Fortunately, they were intrigued by the technology Hossein was working on, and a collaboration began.

The heart is the most important organ, with heart disease the number one killer in the world, Hossein says.

My goal is to find a way for mending broken hearts or, in other words, to find a solution for cardiac problems.

During their first seven days of life, baby mice can self-repair broken heart tissue.

They develop this ability while in the embryo, which although impossible for scientists to view under a microscope could hold the clue to repairing heart damage in humans.

We cannot put a mouse embryo under the microscope, but what I do is model what is going on in the embryonic heart in a microchip, at a microscale.

The microchip that Hossein nanoengineered has been embedded with mice heart cells. It was built to mimic the embryonic heart cells natural environment, enabling the researchers to study how the regenerative cells communicate with each other. Knowledge of how this works could be applied to repairing human heart tissue, although Hossein speculates this technology is at least a decade away. In order to expand their research, additional project funding is needed.

Hossein sees many benefits to using nanotechnology in biomedicine.

First of all, its cheap. You dont need to have lots of plastic, petri dishes, culture media all those things we need for the cell culture. Secondly, you dont need too many animals or cells or patients. Instead of 10-100 million cells, what I need in that tiny family is like 100 or 1000 cells.

Or, in a simple way, instead of needing a hundred mice for each series of experiments, I just need one or two.

Hossein observes the cells interacting via a microscope connected to a supercomputer. Visually, it looks like a microchip. It has channels, it has valves it is like a simplified model of a heart.

When the cells interact in just the right way, it starts to pulse just like a heartbeat.

Hossein is lost for words when remembering the first time he saw the cells start beating.

His work, he explains, differs from many other PhD students. Instead of working from 9-5, he needs to visit the lab at all hours sometimes as late as midnight to feed the cells. One particular lab visit stands out to him.

I came to the lab on Friday to add culture media, which is food for the cells. The day after, I was at Coogee Beach with some friends and said, I should go, I need to check on my cells.

His friends were confused, but it was normal routine for Hossein. It was then he realised that the cells were effectively his babies.

Only when he arrived, they werent beating.

I thought, Oh no, something is wrong! He quickly added the culture media, put them in the incubator, and waited.

A few hours later, they started beating again.

It was incredible, Hossein remembers.

For Hossein, when the cells are beating, they are a symbol of hope and life.

"Everything in our world starts with that beating. And everything finishes with that beating. When that line becomes straight I've seen this in my life, with my family you see that everything is gone. As long as there is beating, there is hope."

Everything in our world starts with that beating. And everything finishes with that beating. When that line becomes straight I've seen this in my life, with my family you see that everything is gone.

As long as there is beating, there is hope.

Hossein knew that his family and friends were proud of his achievements but was frustrated at his inability to properly explain his research to them.

He remembers the specific moment when that changed.

Hossein was taking an Uber to university one night (to feed the cells, he recalls) when the driver asked what he studied. Knowing that the answer was convoluted, Hossein kept his response simple: Im an engineer, he said.

An engineer? the driver responded. Then why am I dropping you off at the medicine building?

Hossein then had a realisation: if he could explain his work to an Uber driver, then perhaps he could also explain it to his mum.

From that moment, he tried to articulate his research to any driver who asked. He tested new ways of describing his work and kept the ones that worked best.

Soon, something surprising happened Hossein developed a love for science communication.

I realised that it is important to explain what I'm doing, but in simple words, so that anyone can understand.

The practice paid off. Earlier this year, Hossein was a finalist in FameLab Australia (a live annual science communication competition), a shortlisted speaker at TEDx Melbournes Open Mic, and last year was a finalist in AMP Amplify Ignite (a national PhD pitch competition). He now shares his research via Twitter.

Science communication is an art, he says. For me, informing and educating is more important than doing just research all the time.

And, he adds, I now get five-star ratings on Uber!

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The nanoengineer working to mend broken hearts - UNSW Newsroom