WASHINGTON, April 2, 2020 /PRNewswire/ -- An article published in Experimental Biology and Medicine (Volume 245, Issue 6, March 2020) (https://journals.sagepub.com/doi/pdf/10.1177/1535370220910690) examines the safety of stem cell therapy for the treatment of colon cancer.The study, led by Dr. J. Liu in the State Key Laboratory of Bioreactor Engineering and Shanghai Key Laboratory of New Drug Design at the East China University of Science and Technology in Shanghai (China), reports that mesenchymal stem cells from a variety of sources promote the growth and metastasis of colon cancer cells in an animal model.

Mesenchymal stem (MSCs), a category of adult stem cells, are being evaluated as therapy for numerous cancers.MSCs are excellent carriers for tumor treatment because they migrate to tumor tissues, can be genetically modified to secrete anticancer molecules and do not elicit immune responses.Clinical trials have shown that MSCs carrying modified genes can be used to treat colon cancer as well as ulcerative colitis. However, some studies have demonstrated MSCs can differentiate into cancer-associated fibroblasts and promote tumor growth.Therefore, additional studies are needed to evaluate the safety of MSCs for targeted treatment of colon cancer.

In the current study, Dr. Liu and colleagues examined the effects of mesenchymal stem cells (MSCs) from three sources (bone marrow, adipose and placenta) on colon cancer cells.MSCs from all three sources promoted tumor growth and metastasis in vivo. In vitro studies demonstrated that MSCs promote colon cancer cell stemness and epithelial to mesenchymal transition, which would enhance tumor growth and metastasis respectively.Finally, the detrimental effects of MSCs could be reversed by blocking IL-8 signaling pathways. Dr. Ma, co-author on the study, said that "Mesenchymal stem cells have a dual role: promoting and/or suppressing cancer. Which effect is dominant depends on the type of tumor cell, the tissue source of the MSC and the interaction between the MSC and the cancer cell. This is the major issue in the clinical application research of MSCs, and additional preclinical experimental data will be needed to evaluate the safety of MSCs for colon cancer treatment."

Dr. Steven R. Goodman, Editor-in-Chief of Experimental Biology & Medicine, said: "Lui and colleagues have performed elegant studies on the impact of mesenchymal stem cells (MSCs), from various sources, upon the proliferation, stemness and metastasis of colon cancer stem cells (CSCs) in vitro and in vivo. They further demonstrate that IL-8 stimulates the interaction between colon CSCs and MSCs, and activates the MAPK signaling pathway in colon CSCs.This provides a basis for the further study of MSCs as a biologic therapy for colon cancer."

Experimental Biology and Medicine is a global journal dedicated to the publication of multidisciplinary and interdisciplinary research in the biomedical sciences. The journal was first established in 1903. Experimental Biology and Medicine is the journal of the Society of Experimental Biology and Medicine. To learn about the benefits of society membership, visit http://www.sebm.org. For anyone interested in publishing in the journal, please visit http://ebm.sagepub.com.

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Stem Cell Therapy for Colon Cancer - Yahoo Finance

Bone Marrow Stem Cells Comments Off on Stem Cell Therapy for Colon Cancer – Yahoo Finance | April 2nd, 2020

Global Bone Marrow Aspirate Concentrates Market was valued US$ XX Bn in 2018 and is expected to reach US$ XX Bn by 2026, at CAGR of 6.5 % during forecast period of 2019 to 2026

Bone marrow concentrate (BMC) uses stem cells that are harvested from your own bone marrow to help the body heal itself. These cells when injected directly into an injury site, prompt a rapid and efficient restoration of the tissue, returning it to a more healthy state by stimulating the bodys natural healing response. It is non-surgical treatment for various orthopedic injuries, including mild to moderate osteoarthritis, disc degeneration and soft tissue injuries.

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Global Bone Marrow Aspirate Concentrates Market Drivers and RestrainsBone marrow-derived stem cell treatment is considered a promising and advanced therapy. It reduces the injury healing time in orthopedic diseases to five to six weeks from four to six months in case of surgery. Reduction in the healing time is a factor likely to fuel the Bone Marrow Aspirate Concentrates market during the forecast period.

Pain associated with the treatment, lack of awareness, and use of alternative treatments are major restraints to the Global Bone Marrow Aspirate Concentrates Market. Furthermore, increased investments in R&D and clinical trials attributed to slow approval processes entailing sunken costs, and marginal returns on investment for manufacturers are factors hindering Global Bone Marrow Aspirate Concentrates Market.

Global Bone Marrow Aspirate Concentrates Market key segmentationBy end-use market is divided into hospitals & clinics, pharmaceutical & biotechnology companies, Contract Research Organizations (CROs) & Contract Manufacturing Organizations (CMOs), and academic & research institutes. The hospitals & clinics segment dominated the bone marrow aspirate concentrates market in 2018 and is expected to maintain its dominance during the forecast period. The hospitals & clinics segmental growth is boosted by the biotechnology & biopharmaceutical companies in terms of revenue during the forecast period. Growth of the segment is attributed to increasing number of biotechnology companies and rising partnerships among the market players to expand globally.

Global Bone Marrow Aspirate Concentrates Market regional analysisBy regional analysis, global bone marrow aspirate concentrates market is divided into major five geographical regions, including North America, Europe, Asia-Pacific, Latin America and Middle East and Africa. North America held largest share of the Global Bone Marrow Aspirate Concentrates market owing to technological advancements and regulatory approval for new devices, rising awareness about stem cell therapy, and number of cosmetic surgical procedures. Furthermore, Asia Pacific orthopedic market is key driver, which led to this massive and augmented growth. The orthopedic market in Asia including bone graft, spine, and bone substitute is anticipated to grow as fast as the overall orthopedic market which will further boost growth of BMAC market in the region during forecast period.

The objective of the report is to present comprehensive analysis of Global Bone Marrow Aspirate Concentrates Market including all the stakeholders of the industry. The past and current status of the industry with forecasted market size and trends are presented in the report with the analysis of complicated data in simple language. The report covers all the aspects of industry with dedicated study of key players that includes market leaders, followers and new entrants by region. PORTER, SVOR, PESTEL analysis with the potential impact of micro-economic factors by region on the market have been presented in the report. External as well as internal factors that are supposed to affect the business positively or negatively have been analyzed, which will give clear futuristic view of the industry to the decision makers.

The report also helps in understanding Global Bone Marrow Aspirate Concentrates Market dynamics, structure by analyzing the market segments, and project the Global Bone Marrow Aspirate Concentrates Market size. Clear representation of competitive analysis of key players by Bone Marrow Aspirate Concentrates Type, price, financial position, product portfolio, growth strategies, and regional presence in the Global Bone Marrow Aspirate Concentrates Market make the report investors guide.

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Global Bone Marrow Aspirate Concentrates Market by product type

Bone Marrow Aspirate Concentrates Systems Bone Marrow Aspirate Concentrates AccessoriesGlobal Bone Marrow Aspirate Concentrates Market Application

Orthopaedic Surgery, Wound Healing, Chronic Pain, Peripheral Vascular Disease, Dermatology;Global Bone Marrow Aspirate Concentrates Market by region

Asia Pacific North America Europe Latin America Middle East AfricaGlobal Bone Marrow Aspirate Concentrates Market by end-user

Hospitals & Clinics Pharmaceutical & Biotechnology Companies Contract Research Organizations (CROs) and Contract Manufacturing Organizations (CMOs) Academic & Research InstitutesKey players operating on Global Bone Marrow Aspirate Concentrates Market

Terumo Corporation (Terumo BCT), Ranfac Corp., Arthrex, Inc., Globus Medical, Inc., Cesca Therapeutics Inc., MK Alliance Inc. (TotipotentSC), and Zimmer Biomet Holdings, Inc Cesca Therapeutics Inc. Stryker Paul Medical Systems LIFELINX SURGIMED PVT. LTD.

MAJOR TOC OF THE REPORT

Chapter One: Bone Marrow Aspirate Concentrates (BMAC) Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Bone Marrow Aspirate Concentrates (BMAC) Market Competition, by Players

Chapter Four: Global Bone Marrow Aspirate Concentrates (BMAC) Market Size by Regions

Chapter Five: North America Bone Marrow Aspirate Concentrates (BMAC) Revenue by Countries

Chapter Six: Europe Bone Marrow Aspirate Concentrates (BMAC) Revenue by Countries

Chapter Seven: Asia-Pacific Bone Marrow Aspirate Concentrates (BMAC) Revenue by Countries

Chapter Eight: South America Bone Marrow Aspirate Concentrates (BMAC) Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Bone Marrow Aspirate Concentrates (BMAC) by Countries

Chapter Ten: Global Bone Marrow Aspirate Concentrates (BMAC) Market Segment by Type

Chapter Eleven: Global Bone Marrow Aspirate Concentrates (BMAC) Market Segment by Application

Chapter Twelve: Global Bone Marrow Aspirate Concentrates (BMAC) Market Size Forecast (2019-2026)

Browse Full Report with Facts and Figures of Bone Marrow Aspirate Concentrates (BMAC) Market Report at: https://www.maximizemarketresearch.com/market-report/global-bone-marrow-aspirate-concentrates-bmac-market/37078/

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Global Bone Marrow Aspirate Concentrates (BMAC) Market: Industry Analysis and forecast (2019 to 2026): By product Type, Application, End Users, and...

Bone Marrow Stem Cells Comments Off on Global Bone Marrow Aspirate Concentrates (BMAC) Market: Industry Analysis and forecast (2019 to 2026): By product Type, Application, End Users, and… | April 2nd, 2020

, which were found to produce high levels of Interleukin-6 (IL-6), was abated in the presence of anti-IL-6 monoclonal antibodies, according to study results intended to be presented at the annual meeting of the American College of Cardiology (ACC.20).

In a diseased state, cardiacmesenchymal stromal cells (cMSCs) remodel and secrete inflammatory cytokines,including IL-6. IL-6 has been shown to be a potent inducer of Ca2+-mediatedarrhythmia substrates in human myocytes. While anti-IL-6 monoclonal antibodies havean established role in the treatment of autoimmune diseases and malignancies, theiruse in the treatment of cardiac disease has not been well studied.

Using extracted device leads and explanted hearts from patients with and without heart failure, investigators isolated cMSCs (failing and non-failing cMSCs, respectively), and quantified IL-6 using an enzyme-linked immunosorbent assay. Myocytes were derived from induced pluripotent stem cells (iPSCs) from individuals without heart failure and cultured in monolayers. Myocytes were treated with exogenous IL-6 or cocultured with failing cMSCs with and without anti-IL-6 monoclonal antibody. Fluorescent indicators were used to detect the presence of Ca2+ alternans during steady state pacing.

The secretion of IL-6 was found tobe 5.6 times higher in failing vs nonfailing cMSCs (n=4; P <.005) and 66 times higher in cMSCs vs iPSC-derived humanmyocytes (n=5; P <.002). Myocytes thatwere cocultured with failing cMSCs or were exposed to exogenous IL-6 had largeincreases in Ca2+ alternans compared with myocytes cultured alone (343%,n=12, P <.001 and 300%, n=5, P <.002, respectively). These Ca2+alternans were reduced to baseline levels in myocyte/cMSC cocultures treated vsnot treated with IL-6 (reduction, 400%; n=18, P <.001).

These results suggest anovel anti-arrhythmic therapeutic strategy in heart failure using anti-IL-6drugs such as tocilizumab, sarilumab, or siltuximab, concluded theresearchers.

Reference

Vasireddi S, Sattayaprasert P,Moravec C, et al. Targeted anti-inflammatory treatment with anti-Il-6monoclonal antibody for calcium-mediated arrhythmia substrates in heartfailure. Intended to be presented at: American College of Cardiologys 69thAnnual Scientific Session; March 28-30, 2020; Chicago, IL. Presentation 915-09.

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Anti-IL-6 Monoclonal Antibodies as Antiarrhythmic Treatment for HF - The Cardiology Advisor

Cardiac Stem Cells Comments Off on Anti-IL-6 Monoclonal Antibodies as Antiarrhythmic Treatment for HF – The Cardiology Advisor | April 2nd, 2020

HAMBURG, Germany and TORONTO, April 2, 2020 /CNW/ - Evotec SE (Frankfurt Stock Exchange: EVT, MDAX/TecDAX, ISIN: DE0005664809) and the innovative biotechnology company panCELLa Inc. announced today that the companies have entered into a licensing and investment agreement.

Under the terms of the agreement, Evotec will receive a non-exclusive licence to access panCELLa's proprietary iPS cell lines "iACT Stealth Cells", which are genetically modified to prevent immune rejection of derived cell therapy products ("cloaking"). Furthermore, Evotec will also have access to a new-generation cloaking technology known as hypoimmunogenic cells. In addition, the "FailSafe" mechanism effectively addresses a key challenge in iPSC-based cell therapy, potential tumour formation by residual undifferentiated cells.

Using the cell lines, Evotec will be able to develop iPSC-based, off-the-shelf cell therapies with long-lasting efficacy that can be safely administered to a broad population of patients without the use of medication to supress the patients' immune system. With a growing portfolio of iPSC-based cell therapy projects at Evotec, access to research as well as GMP-grade iPSC lines modified with one or both of the panCELLa technologies significantly accelerates Evotec's cell therapy discovery and development efforts. Modified iPSC lines will be available for the development of cell therapy approaches across a broad range of indications by Evotec and potential partners. Furthermore, Evotec has made an investment to take a minority stake in panCELLa and has nominated Dr Andreas Scheel to join panCELLa's supervisory board.

Dr Cord Dohrmann, Chief Scientific Officer of Evotec, commented: "Cell therapies hold enormous potential as truly regenerative or curative approaches for a broad range of different diseases with significant medical need. Integrating panCELLa's technology and cell lines into our ongoing proprietary research and development efforts strengthens Evotec's position in cell therapy. It is our goal to provide safe highly-effective cell therapy products to as many patients as possible. In addition to small molecules and biologics, cell therapy will become yet another major pillar of Evotec's multimodality discovery and development platform."

Mahendra Rao, MD, PhD, CEO at panCELLa, added: "We welcome the partnership with Evotec. Evotec's widely recognised expertise and existing portfolio of iPSC-related technology platforms will allow panCELLa to rapidly advance its own therapeutic interests in NK cell therapy, pancreatic islet production and iPSC-derived MSC platform, in addition to enabling panCELLa to make its platform technologies widely available. I believe that the investment by Evotec in our company is a strong validation of the leading role of panCELLa in the field of regenerative medicine and in the utility of its platform technologies. We welcome Dr Andreas Scheel to our Board."

No financial details of the agreement were disclosed.

About Evotec and iPSCInduced pluripotent stem cells (also known as iPS cells or iPSCs) are a type of pluripotent stem cell that can be generated directly from adult cells. The iPSC technology was pioneered by Shinya Yamanaka's lab in Kyoto, Japan, who showed in 2006 that the introduction of four specific genes encoding transcription factors could convert adult cells into pluripotent stem cells. He was awarded the 2012 Nobel Prize along with Sir John Gurdon "for the discovery that mature cells can be reprogrammed to become pluripotent". Pluripotent stem cells hold great promise in the field of regenerative medicine. Because they can propagate indefinitely, as well as give rise to every other cell type in the body (such as neurons, heart, pancreatic and liver cells), they represent a single source of cells that could be used to replace those lost to damage or disease.

Evotec has built an industrialised iPSC infrastructure that represents one of the largest and most sophisticated iPSC platforms in the industry. Evotec's iPSC platform has been developed over the last years with the goal to industrialise iPSC-based drug screening in terms of throughput, reproducibility and robustness to reach the highest industrial standards, and to use iPSC-based cells in cell therapy approaches via the Company's proprietary EVOcells platform.

About cell therapy and panCELLa's FailSafe iPSC technologyCell therapy, one of the most promising regenerative medicine approaches, replaces a patient's missing or broken cells with functioning cells from a range of different sources, either from a donor, from the patient's own material, or from stem cells. The advent of induced pluripotent stem cells ("iPSC") has opened up stem cells as an almost unlimited source of consistent-quality material for such cell therapies. At the same time, differentiating cell therapies from a single validated source circumvents critical risks of contamination associated with administering both donor and patient cell material.

However, the patient's immune system will treat such iPSC-based transplant as "foreign" and use the body's immune system to counteract the therapy, thus undermining its long-term efficacy. While organ transplants require an often lifelong regimen of immunosuppressants, iPSC-derived cells used for cell therapies can be cloaked to make them undetectable by the patient's immune system, thus avoiding rejection and enabling effective long-term relief of the patient's symptoms.

To increase the safety of such iPSC-derived cell products, panCELLa's proprietary FailSafe technology is able to inactivate any iPSC-derived proliferating cell before and after transplantation through the use of a readily available anti-infective medication. FailSafe is the only quantifiable "safety switch" on the market which is expected to be critical for regulators, clinicians and patients to make informed decisions when evaluating treatment options.

About panCELLa Inc. Incorporated in August 2015, panCELLa (www.pancella.com) was founded by Dr Andras Nagy and Dr Armand Keating based on Dr Nagy's ground-breaking work in the area of stem cell research. Through panCELLa, Drs Keating and Nagy are seeking to create an effective cell therapy derived from stem cells, which are modified to provide a sufficient and very high level of safety before and after the cells are introduced to the patient. panCELLa serves those companies developing products from stem cells. panCELLa seeks to create universal "off the shelf" FailSafe Cells and to assist pharmaceutical and biotechnology sectors to achieve such with their own cell lines. Targeted medical applications include deadly, debilitating, or aggressive diseases requiring immediate treatment where there is no time to cultivate a customized stem cell treatment from the patient (i.e. cancer, cardiac infarct, diabetes, stroke and spinal cord injury).

About Evotec SEEvotec is a drug discovery alliance and development partnership company focused on rapidly progressing innovative product approaches with leading pharmaceutical and biotechnology companies, academics, patient advocacy groups and venture capitalists. We operate worldwide and our more than 3,000 employees provide the highest quality stand-alone and integrated drug discovery and development solutions. We cover all activities from target-to-clinic to meet the industry's need for innovation and efficiency in drug discovery and development (EVT Execute). The Company has established a unique position by assembling top-class scientific experts and integrating state-of-the-art technologies as well as substantial experience and expertise in key therapeutic areas including neuronal diseases, diabetes and complications of diabetes, pain and inflammation, oncology, infectious diseases, respiratory diseases, fibrosis, rare diseases and women's health. On this basis, Evotec has built a broad and deep pipeline of approx. 100 co-owned product opportunities at clinical, pre-clinical and discovery stages (EVT Innovate). Evotec has established multiple long-term alliances with partners including Bayer, Boehringer Ingelheim, Bristol-Myers Squibb, CHDI, Novartis, Novo Nordisk, Pfizer, Sanofi, Takeda, UCB and others. For additional information please go to http://www.evotec.com and follow us on Twitter @Evotec.

FORWARD LOOKING STATEMENTSInformation set forth in this press release contains forward-looking statements, which involve a number of risks and uncertainties. The forward-looking statements contained herein represent the judgement of Evotec as of the date of this press release. Such forward-looking statements are neither promises nor guarantees, but are subject to a variety of risks and uncertainties, many of which are beyond our control, and which could cause actual results to differ materially from those contemplated in these forward-looking statements. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any such statements to reflect any change in our expectations or any change in events, conditions or circumstances on which any such statement is based.

SOURCE panCELLa Inc.

Company Codes: Frankfurt:EVT, OTC-PINK:EVTCY

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Evotec Expands its iPSC-Based Cell Therapy Platform EVOcells Through Licensing Agreement with panCELLa - BioSpace

IPS Cell Therapy Comments Off on Evotec Expands its iPSC-Based Cell Therapy Platform EVOcells Through Licensing Agreement with panCELLa – BioSpace | April 2nd, 2020

Lonza has announced the launch of the TheraPEAK SfAAV Medium, the first chemically defined, non-animal origin medium designed specifically for the production of Adeno Associated Virus (AAV) in Spodoptera fuigiperda (Sf9) insect cells for gene therapy applications. The new high-performance medium aims to accelerate cell growth, increase productivity, and reduce process variability and costs, expediting time-to-market for safe, scalable, life-saving gene therapies.

Gene therapy holds great promise for the treatment of rare disorders and diseases, and viral vectors are commonly used to facilitate the delivery of the gene of interest into patient cells. AAV has been established as a viral vector of choice, due to not replicating in patients, thus posing a lower health risk. Owing to their ability to be grown at high densities, the Sf9 insect cells are ideal for use as hosts for the production of large AAV quantities. To date, however, translational scientists and researchers have been challenged with the lack of a medium dedicated to AAV production in Sf9 insect cells.

Lonzas TheraPEAK SfAAV Medium has been explicitly designed to address this need, providing a cost and time efficient solution for the production of AAV in Sf9 insect cells. Allowing for rapid cell growth, the TheraPEAK SfAAV Medium can reduce processing time significantly, enabling cell infection one day earlier than similar media available on the market, and boosting laboratory performance. Due to its chemically defined nature, the new hydrolysate free medium produces AAV that requires less purification, further decreasing the overall processing time and minimizing labor requirements. Furthermore, the TheraPEAK SfAAV Medium supports consistent cell growth throughout all phases of the culturing process, considerably reducing process variability.

As a chemically defined, non-animal origin product, the TheraPEAK SfAAV Medium can be considered safer to use than media containing animal or human components, thereby facilitating regulatory compliance. Additionally, the medium is supplied with a U.S. Food and Drug Administration drug master file, alleviating the relevant preparation and submission burden.

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Optimizing the Production of AAV in Sf9 Insect Cells - Technology Networks

IPS Cell Therapy Comments Off on Optimizing the Production of AAV in Sf9 Insect Cells – Technology Networks | April 2nd, 2020

Just before Christmastime, Howard Federoff got a tip from Washington: There was a new virus in China. And this one could be bad.

News report of the virus had not yet appeared. Federoff, a neuroscientist, was briefed because years before, he was vetted as part of a group he didnt give a name for the group to consult for the US government on emerging scientific issues. His day job, though, was CEO of Aspen Neurosciences, a Parkinsons cell therapy startup that days before had come out of stealth mode and gave word to investors they were hoping to raise $70 million. That, Federoff realized, would be difficult if a pandemic shut down the global economy.

I started thinking rather early onThere might be something on the horizon that we dont fully understand, Federoff told Endpoints News. We knew that if something did change, it could change rather quickly.

Operating with insight and knowledge other biotechs lacked access to, Federoff went into overdrive trying to close before Covid-19 hit the US, and he emerged today with $70 million in Series A funding led by OrbiMed. The other investors included Frazier Health Partners, Sam Altman and ARCH Venture Partners, the VC whose leader Robert Nelsen became one of the earliest and most prominent voices calling for change.

Weve had long conversations, Federoff said of him and Nelsen.

With the Series A, Federoff has convinced A-list investors to back one version of a long-sought solution to Parkinsons. Aspen will use stem cells grown from Parkinsons patients own skin tissue to grow dopamine neurons that can be implanted into the brain and hopefully replace the degenerating neurons. The idea has been around for decades, with the first transplant occurring in the 80s, but it was never scaleable. The technology to produce stem cells on demand didnt exist.

The company has a rival in BlueRock, which uses donor stem cells and which Bayer acquired in August at a valuation of $1 billion.

Over the winter, though, the investor hunt became less about pitching the science which Federoff says everyone agreed was promising than about beating the clock and investors rising worries about the economy. He prepared to work fast, turning an early meeting with Frazier at the JP Morgan Healthcare Conference into a pivotal one. As the months passed, he phoned investors multiple times a day to keep funding on track.

They were already in from the standpoint of the science, Federoff said. I could tell that there was a growing weariness about whether all that they had previously considered as part of their own respective portfolios outside of Aspen would all be possible.

The money he secured will help fund their Phase I trial on Parkinsons and a second program that uses a form of gene therapy to implant stem cells that have a genetic marker for Parkinsons edited out. The plan had been to start a trial in 2021, but Federoff knows there are no more guarantees.

At this time its not clear what Covid-19 will do to projections, he said.

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'There was a growing weariness': Rushing against a pandemic clock, Aspen Neurosciences secures $70M Series A - Endpoints News

Skin Stem Cells Comments Off on ‘There was a growing weariness’: Rushing against a pandemic clock, Aspen Neurosciences secures $70M Series A – Endpoints News | April 2nd, 2020

Australian startup Cortical Labs is building computer chips that use biological neurons extracted from mice and humans, Fortune reports.

The goal is to dramatically lower the amount of power current artificial intelligence systems need to operate by mimicking the way the human brain.

According to Cortical Labs announcement, the company is planning to build technology that harnesses the power of synthetic biology and the full potential of the human brain in order to create a new class of AI that could solve societys greatest challenges.

The mouse neurons are extracted from embryos, according to Fortune, but the human ones are created by turning skin cells back into stem cells and then into neurons.

The idea of using biological neurons to power computers isnt new. Cortical Labs announcement comes one week after a group of European researchers managed to turn on a working neural network that allows biological and silicon-based brain cells to communicate with each other over the internet.

Researchers at MIT have also attempted to use bacteria, not neurons, to build a computing system in 2016.

As of right now, Corticals mini-brains have less processing power than a dragonfly brain. The company is looking to get its mouse-neuron-powered chips to be capable of playing a game of Pong, as CEO Hon Weng Chong told Fortune, following the footsteps of AI company DeepMind, which used the game to test the power of its AI algorithms back in 2013.

What we are trying to do is show we can shape the behavior of these neurons, Chong told Fortune.

READ MORE: A startup is building computer chips using human neurons [Fortune]

More on neurons: Artificial and Biological Neurons Just Talked Over the Internet

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This Startup's Computer Chips Are Powered by Human Neurons - Futurism

Skin Stem Cells Comments Off on This Startup’s Computer Chips Are Powered by Human Neurons – Futurism | April 2nd, 2020

Disclosure: Dr Henderson is the president and principal owner of The Synaptic Space, a neuroimaging consulting firm, and owner of Neuro-Luminance Corporation. Please see the listed studies for a full list of disclosures.

During the last 20 years, a large body of research has accumulated on the beneficial effects of infrared light in the range of 600 to 1000 nm. Infrared light can activate mitochondria, which in turn stimulate second messenger systems, DNA transcription, and growth factors.1,2 As a result, new synapses are formed, circuits regrow, and pluripotent stem cells differentiate into neurons.

Animal studies have shown that infrared photobiomodulation (PBM) may reduce the size and severity of brain injury and stroke, as well as diminish damage and physiological symptoms in depression, posttraumatic stress disorder (PTSD), Parkinson disease, and Alzheimer disease.1,3-6 Michael Hamblin, PhD, from the Wellman Center for Photomedicine at Massachusetts General Hospital in Boston, a leader in the field, describes PBM as the use of red or near-infrared light to stimulate, heal, regenerate, and protect tissue that has either been injured, is degenerating, or else is at risk of dying.1

Generally in medicine we shy away from the word heal when referring to the brain, and regenerate stirs vague recollections of Frankenstein. Nevertheless, early findings in mouse models of brain injury and disease have spawned a different sort of monster in the commercial world. The internet is now loaded with companies offering infrared LED helmets or pads for the treatment of traumatic brain injury (TBI) and other brain disorders, often based on exaggerated claims about healing the brain. Exorbitant prices in the thousands of dollars are charged for a device that can be made for less than $30. As a result, the public is misled and the potential scientific benefits of infrared light are sullied.

It is time to separate fact from fiction. Yes, infrared light can induce the cellular events described here, reduce the size of stroke injury or TBI in mouse models, and protect neurons from neurotoxins. But is treating a human with a 0.5-W LED the same as treating a mouse? Certainly not! When it comes to infrared light treatment, it is all a matter of getting there: the infrared light must be able to penetrate all the overlying tissue to reach the brain.

Can Infrared Light Reach the Brain?

Can 0.5-W LEDs penetrate human scalp and skull to reach the brain? The answer is No.2 My colleague, Larry Morries, DC, and I showed that these LEDs did not even penetrate 2 mm of human skin. In contrast, our laser device, which emits infrared light in the range of 10 to 15 W, was able to effectively penetrate human tissue. We found that 33% of our 10-W infrared laser energy penetrated 2 mm of human skin and delivered from 1.2% to 2.4% of the energy from our device 3 cm into the brain. These data were replicated in a study by Juanita Anders, PhD, and colleagues at the Uniformed Services University of Health Sciences.7

The human scalp and skull provide a significant barrier. Infrared light energy needs to be in the range of 0.9 to 15 J/cm2 at the target tissue to activate mitochondria and other cellular events.2-3,8-9 Even if a 0.5-W LED only had to penetrate the skull to reach the surface of the brain, it could only deliver 0.0064 J/cm2, or 1/140th of the minimum energy necessary to induce PBM.10 No energy would be expected to reach the depths of the brain needed to treat stroke, Parkinson disease, Alzheimer disease, or many brain injuries. Although more than 40% of the incident light from a light source may penetrate mouse skull, only 4.2% penetrates human skull.8,10

There is a hairier problem facing LED devices: human hair blocks infrared light. More than 98% of infrared light can be blocked by 2 mm of hair (ie, 9.764 W of a 10-W beam of 810 nm infrared light is absorbed by human hair).11 If 98% of the energy from a 0.5-W LED is absorbed by hair, 80% to 90% is absorbed by 2 mm of skin, and 96% of incident energy is attenuated by skull, then claims of neurophysiological benefits of LED-based devices become highly questionable.

Another misconception propagated by companies selling LED-based devices is that multiple LEDs somehow increase light penetration, even though each LED projects light on its own path. For example, 100 0.5-W LEDs do not generate 50 W on the brain, they generate 0.5 W on 100 spots.11 The argument that light scattering in the brain provides the cumulative value of multiple LEDs also falls apart if nothing can get through the overlying tissues.

Given that a small percentage (<1%) of incident infrared light gets through human scalp and skull, we must question the results of human trials of LEDs. Studies demonstrated small yet almost insignificant positive effects, and the benefits are generally transient.12 In contrast, our protocol yields persistent and robust clinical changes in patients with TBI, PTSD, and depression.

Treating TBI, PTSD, and Depression with Infrared Light

Our patented multi-Watt Neuro-Luminance approach involves transcranial infrared laser treatment (NILT), and in 2015 we published an initial open-label trial of 10 subjects with mild to moderate TBI.13 After a course of 10 NILT treatments (20 treatments in a subset of 4 patients), all patients experienced significant clinical improvement of symptoms, including headaches, cognitive problems, sleep disturbances, irritability, and depression. In telephone interviews every 6 months after treatment, patients report sustained improvements.12

An open-label clinical trial (n=39) of multi-Watt Neuro-Luminance demonstrated effectiveness for depression.4 Overall, 92% of patients responded and 82% remitted, which is notably better than the response rate for oral antidepressants. Patients saw benefits within 4 treatments, and some achieved resolution of depressive symptoms within 8 treatments. In follow-up telephone interviews, patients report sustained improvements. Similarly, in our unpublished data, using a protocol of 20 treatments, each lasting 24 minutes, over the course of 9 weeks, 20 patients with PTSD treated with multi-Watt NILT experienced reduced hyperarousal, anxiety, sleep disturbance, and nightmares.

LED Photobiomodulation in Comparison

Naeser and colleagues15 treated 2 patients with TBI daily for approximately 1 hour by applying 3 separate LED cluster heads (2 head; 1 foot). The first patient, who was 7 years post-TBI and had significant postconcussive symptoms, received weekly treatments over the course of 7 months and then daily treatments at home for more than 6 years. The patient experienced transient benefits, and if treatment was stopped, symptoms returned within 2 weeks.15 The second patient received daily treatments, and in 4 months, most symptoms improved, allowing her to return to work. This patient also noted that symptoms returned if treatments were stopped for more than 1 week.15

In an open-label study,16 11 patients with TBI and persistent cognitive dysfunction were treated for 18 sessions, each lasting 20 minutes, over the course of 6 weeks. At follow-up, there had been a significant effect on attention, inhibition, verbal learning and memory, and long-delay free recall.16 The LED treatment led to mild improvement in 3 of 5 cases of depression.

In 12 patients with TBI treated with 220 0.5-W LEDs for 18 sessions, each lasting 20 minutes, over the course of 6 weeks, there was significant improvement in psychological testing results (P =.45).17 However, the study did not correct for multiple comparisons, instead using parallel paired t-tests, which could exaggerate findings.18 PTSD has received considerably less attention.19,20

Cassano and colleagues21 described a 5-W laser treatment of 4 patients with depression. In a double-blind, sham-controlled extension of their initial findings, subjects in the treatment group received 16 treatments, each lasting 30 minutes, over the course of 8 weeks.22 In 13 completers, Hamilton-D-17 scores separated the treatment group from sham controls (mean score, 15.74.41 vs 6.17.86; P =.031). In contrast, in our open-label trial of a 13-W laser, the mean Hamilton-D-17 score decreased from baseline (mean score, 21.485.24 to 6.05.12; P =6.4510-13).23

Table. Case series, open-label, and double-blind studies of infrared light therapy for TBI, PTSD, and depression

Alternative Explanation for Clinical Response to LED Brain Treatments

Researchers, along with the human PBM field, need to reconsider the potential mechanisms underlying the meager improvements derived from LED-based devices. The light from LED devices may not penetrate beyond the skin, but could induce central nervous system benefits via a remote or systemic effect in irradiated skin, dubbed remote photobiomodulation.24

Infrared irradiation can have remote or indirect effects on tissue that has not been irradiated. For example, Braverman and colleagues25 demonstrated this indirect effect by creating matching skin lesions on the left and right dorsum of a rabbit, treating 1 side with infrared light. Both lesions showed accelerated healing relative to nonirradiated controls. Rochkind and colleagues26 demonstrated that remote PBM could occur in the peripheral nervous system and the central nervous system. After bilateral sciatic nerve crush, 1 side was irradiated with infrared light and the other side was not. Nerves on both sides showed enhanced recovery of function, and the number of anterior horn motor neurons was greater on both sides compared with nonirradiated controls.

Ganeshan and colleagues27 irradiated the dorsum and hind limbs of a rat with infrared light (670 nm) before injection of a neurotoxin (MPTP) and demonstrated reduced loss of dopaminergic neurons in rodents treated with indirect PBM to the skin compared with untreated controls. Given the overwhelming evidence that low-power LEDs do not penetrate the brain, it is more likely that the benefits of LED-based devices result from an effect mediated by the skin, where most, if not all, of the infrared energy is absorbed. In other words, LED-based devices may be working by remote PBM.

Conclusions

The excitement about the potential of infrared light therapy is not merely that it does not involve taking a pill. There is considerable enthusiasm about its potential to treat conditions such as TBI, dementia, and Parkinson disease. In our excitement, we must not overlook the unique physical limitations of light. Similarly, we must not imbue infrared light with magical powers. Infrared light can only work if it reaches target tissue.

Thus, a sharp divide can be drawn between LED-based treatment technologies, which offer minimal results and may not even reach the brain, and multi-Watt technologies that demonstrably reach the brain and offer lasting clinical benefit. Potentially, infrared light may prove to be effective for numerous neuropsychiatric conditions. However, for infrared light to work on the brain, it must be able to reach the brain.

References

1. Hamblin MR. Shining light on the head: Photobiomodulation for brain disorders. BBA Clin. 2016;6:113-124.

2. Henderson TA, Morries, LD. Near-infrared photonic energy penetration: can infrared phototherapy effectively reach the human brain? Neuropsychiatr Dis Treat. 2015;11:2191-2208.

3. Chung H, Dai T, Sharma SK, Huang YY, Carroll JD, Hamblin MR. The nuts and bolts of low-level laser (light) therapy. Ann Biomed Eng. 2012;40(2):516-533.

4. Henderson TA, Morries LD. Multi-Watt near-infrared phototherapy for the treatment of comorbid depression: an open-label single-arm study. Front Psychiatry. 2017;8:187.

5. Johnstone DM, Moro C, Stone J, Benabid AL, Mitrofanis J. Turning on lights to stop neurodegeneration: the potential of near infrared light therapy in Alzheimers and Parkinsons disease. Front Neurosci. 2016;11;9:500.

6. Hamblin MR. Photobiomodulation for Alzheimers disease: has the light dawned? Photonics. 2019;6(3):77.

7. Tedford CE, DeLapp S, Jacques S, Anders J. Quantitative analysis of transcranial and intraparenchymal light penetration in human cadaver brain tissue. Lasers Surg Med. 2015;47(4):312-322.

8. Ando T, Xuan W, Xu T, et al. Comparison of therapeutic effects between pulsed and continuous wave 810-nm wavelength laser irradiation for traumatic brain injury in mice. PLoS One. 2011;6(10):e26212.

9. Yip KK, Lo SC, Leung MC, So SK, Tang CY, Poon DM. The effect of low-energy laser irradiation on apoptotic factors following experimentally induced transient cerebral ischemia. Neuroscience. 2011;190:301-306.

10. Lapchak PA, Boitano PD, Butte PV, et al. Transcranial near-infrared laser transmission (NILT) profiles (800 nm): systematic comparison in four common research species. PLoS One. 2015;3;10(6):e0127580.

11. Henderson TA, Morries LD. Near-infrared photonic energy penetration principles and practice. In: Hamblin, MR and Huang YY, eds. Photobiomodulation and the Brain: Low-level Laser (Light) Therapy in Neurology and Neuroscience. London: Academic Press; 2019.

12. Morries LD, Henderson TA. Treatment of traumatic brain injury with near-infrared light. In: Hamblin, MR and Huang YY, eds. Photobiomodulation and the Brain: Low-level Laser (Light) Therapy in Neurology and Neuroscience. London: Academic Press; 2019.

13. Morries LD, Cassano P, Henderson TA. Treatments for traumatic brain injury with emphasis on transcranial near-infrared laser phototherapy. Neuropsychiatr Dis Treat. 2015;11:2159-75.

14. Connolly KR, Thase ME. If at first you dont succeed: a review of the evidence for antidepressant augmentation, combination and switching strategies. Drugs. 2011;71(1):43-64.

15. Naeser MA, Saltmarche A, Krengel MA, Hamblin MR, Knight JA. Improved cognitive function after transcranial, light-emitting diode treatments in chronic, traumatic brain injury: two case reports. Photomed Laser Surg. 2011;29(5):351-358.

16. Naeser MA, Zafonte R, Krengel MH, et al. Significant improvements in cognitive performance post-transcranial, red/near-infrared light-emitting diode treatments in chronic, mild traumatic brain injury: open-protocol study. J Neurotrauma. 2014;31(11):1008-1017.

17. Hipskind SG, Grover FL Jr, Fort TR, et al. Pulsed transcranial red/near-infrared light therapy using light-emitting diodes improves cerebral blood flow and cognitive function in veterans with chronic traumatic brain injury: a case series. Photobiomodul Photomed Laser Surg. 2019;37(2):77-84.

18. Henderson TA, Morries LD. Infrared light cannot be doing what you think it is doing (re: DOI: 10.1089/photob.2018.4489). Photobiomodul Photomed Laser Surg. 2019;37(2):124-125.

19. Schiffer F, Johnston AL, Ravichandran C, et al. Psychological benefits 2 and 4 weeks after a single treatment with near infrared light to the forehead: a pilot study of 10 patients with major depression and anxiety. Behav Brain Funct. 2009;5:46.

20. LED light therapy to improve cognitive & psychosocial function in TBI-PTSD veterans. ClinicalTrials.gov. NCT02356861. https://clinicaltrials.gov/ct2/show/NCT02356861. Accessed February 29, 2020.

21. Cassano P, Cusin C, Mischoulon D, et al. Near-infrared transcranial radiation for major depressive disorder: proof of concept study. Psychiatry J. 2015;2015:352979.

22. Cassano P, Petrie SR, Mischoulon D, et al. Transcranial photobiomodulation for the treatment of major depressive disorder. The ELATED-2 Pilot Trial. Photomed Laser Surg. 2018;36(12):634-646.

23. Henderson TA, Morries LD. Multi-Watt near-infrared phototherapy for the treatment of comorbid depression: an open-label single-arm study. Front Psychiatry. 2017;8:187.

24. Gordon LC, Johnstone DM. Remote photobiomodulation: an emerging strategy for neuroprotection. Neural Regen Res. 2019;14(12):2086-2087.

25. Braverman B, McCarthy RJ, Ivankovich AD, Forde DE, Overfield M, Bapna MS. Effect of helium-neon and infrared laser irradiation on wound healing in rabbits. Lasers Surg Med. 1989;9(1):50-58.

26. Rochkind S, Rousso M, Nissan M, Villarreal M, Barr-Nea L, Rees DG. Systemic effects of low-power laser irradiation on the peripheral and central nervous system, cutaneous wounds, and burns. Lasers Surg Med. 1989;9(2):174-182.

27. Ganeshan V, Skladnev NV, Kim JY, Mitrofanis J, Stone J, Johnstone DM. Pre-conditioning with remote photobiomodulation modulates the brain transcriptome and protects against MPTP insult in mice. Neuroscience. 2019;400:85-97.

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Infrared Laser Treatment of TBI, PTSD, and Depression: An Expert Perspective - Psychiatry Advisor

Skin Stem Cells Comments Off on Infrared Laser Treatment of TBI, PTSD, and Depression: An Expert Perspective – Psychiatry Advisor | April 2nd, 2020

BOTHELL, Wash.--(BUSINESS WIRE)-- Seattle Genetics, Inc.. (Nasdaq:SGEN) today provided an update on the phase 1b/2 multicohort EV-103 trial (also known as KEYNOTE-869) of PADCEVTM (enfortumab vedotin-ejfv) in combination with anti-PD-1 therapy pembrolizumab for the treatment of patients with unresectable locally advanced or metastatic urothelial cancer who are unable to receive cisplatin-based chemotherapy in the first-line setting. Based on recent discussions with the U.S. Food and Drug Administration (FDA), data from the randomized cohort K, along with other data from the EV-103 trial evaluating PADCEV combined with pembrolizumab as first-line therapy for cisplatin-ineligible patients, could potentially support registration under accelerated approval regulations in the United States. PADCEV is a first-in-class antibody-drug conjugate (ADC) that is directed against Nectin-4, a protein located on the surface of cells and highly expressed in bladder cancer.1

We are excited that EV-103 provides PADCEV with a potential pathway for U.S. accelerated approval in first-line metastatic urothelial cancer, said Roger Dansey, M.D., Chief Medical Officer at Seattle Genetics. Our initial data on the combination of PADCEV and pembrolizumab in previously untreated patients who could not receive cisplatin are encouraging.

EV-103 is a multi-cohort, open-label, multicenter phase 1b/2 trial of PADCEV alone or in combination, evaluating safety, tolerability and efficacy in muscle invasive urothelial cancer, and in locally advanced or metastatic urothelial cancer in first- or second-line settings. Cohort K from EV-103 is intended to enroll 150 patients randomized 1:1 to PADCEV monotherapy or PADCEV in combination with pembrolizumab in locally advanced or metastatic urothelial cancer patients who are ineligible for cisplatin-based chemotherapy. The primary outcome measure is objective response rate (ORR) per blinded independent central review (BICR) using RECIST 1.1 and duration of response (DoR).

In addition to EV-103, the recently initiated EV-302 phase 3 randomized clinical trial is intended to support global registrations and potentially serve as a confirmatory trial if accelerated approval is granted based on EV-103. The EV-302 trial is evaluating the combination of PADCEV and pembrolizumab with or without chemotherapy versus chemotherapy alone in patients with previously untreated locally advanced or metastatic urothelial cancer. Importantly, EV-302 includes metastatic urothelial cancer patients that are either eligible or ineligible for cisplatin-based chemotherapy. The trial is expected to enroll 1,095 patients and has dual primary endpoints of progression-free survival and overall survival. Both the EV-103 and EV-302 trials are being conducted in collaboration with Astellas and Merck.

FDA recently granted Breakthrough Therapy designation for PADCEV in combination with pembrolizumab for the treatment of patients with unresectable locally advanced or metastatic urothelial cancer who are unable to receive cisplatin-based chemotherapy in the first-line setting based on initial results from the EV-103 trial.

PADCEV (enfortumab vedotin-ejfv) was approved by the FDA in December 2019 and is indicated for the treatment of adult patients with locally advanced or metastatic urothelial cancer who have previously received a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and a platinum-containing chemotherapy before (neoadjuvant) or after (adjuvant) surgery or in a locally advanced or metastatic setting. PADCEV was approved under the FDAs Accelerated Approval Program based on tumor response rate. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.2

About Bladder and Urothelial Cancer

It is estimated that approximately 81,000 people in the U.S. will be diagnosed with bladder cancer in 2020.3 Urothelial cancer accounts for 90 percent of all bladder cancers and can also be found in the renal pelvis, ureter and urethra.4 Globally, approximately 549,000 people were diagnosed with bladder cancer in 2018, and there were approximately 200,000 deaths worldwide.5

About PADCEV

PADCEV is a first-in-class antibody-drug conjugate (ADC) that is directed against Nectin-4, a protein located on the surface of cells and highly expressed in bladder cancer.6,7 Nonclinical data suggest the anticancer activity of PADCEV is due to its binding to Nectin-4 expressing cells followed by the internalization and release of the anti-tumor agent monomethyl auristatin E (MMAE) into the cell, which result in the cell not reproducing (cell cycle arrest) and in programmed cell death (apoptosis).8 PADCEV is co-developed by Astellas and Seattle Genetics.

Important Safety Information

Warnings and Precautions

Adverse Reactions

Serious adverse reactions occurred in 46% of patients treated with PADCEV. The most common serious adverse reactions (3%) were urinary tract infection (6%), cellulitis (5%), febrile neutropenia (4%), diarrhea (4%), sepsis (3%), acute kidney injury (3%), dyspnea (3%), and rash (3%). Fatal adverse reactions occurred in 3.2% of patients, including acute respiratory failure, aspiration pneumonia, cardiac disorder, and sepsis (each 0.8%).

Adverse reactions leading to discontinuation occurred in 16% of patients; the most common adverse reaction leading to discontinuation was peripheral neuropathy (6%). Adverse reactions leading to dose interruption occurred in 64% of patients; the most common adverse reactions leading to dose interruption were peripheral neuropathy (18%), rash (9%) and fatigue (6%). Adverse reactions leading to dose reduction occurred in 34% of patients; the most common adverse reactions leading to dose reduction were peripheral neuropathy (12%), rash (6%) and fatigue (4%).

The most common adverse reactions (20%) were fatigue (56%), peripheral neuropathy (56%), decreased appetite (52%), rash (52%), alopecia (50%), nausea (45%), dysgeusia (42%), diarrhea (42%), dry eye (40%), pruritus (26%) and dry skin (26%). The most common Grade 3 adverse reactions (5%) were rash (13%), diarrhea (6%) and fatigue (6%).

Lab Abnormalities

In one clinical trial, Grade 3-4 laboratory abnormalities reported in 5% were: lymphocytes decreased, hemoglobin decreased, phosphate decreased, lipase increased, sodium decreased, glucose increased, urate increased, neutrophils decreased.

Drug Interactions

Specific Populations

For more information, please see the full Prescribing Information for PADCEV here.

About Seattle Genetics

Seattle Genetics, Inc. is a global biotechnology company that discovers, develops and commercializes transformative medicines targeting cancer to make a meaningful difference in peoples lives. The company is headquartered in Bothell, Washington, and has offices in California, Switzerland and the European Union. For more information on our robust pipeline, visit https://www.seattlegenetics.com and follow @SeattleGenetics on Twitter. For information on our response to the COVID-19 pandemic, please visit our website.

About the Astellas and Seattle Genetics Collaboration

Seattle Genetics and Astellas are co-developing PADCEV under a collaboration that was entered into in 2007 and expanded in 2009. Under the collaboration, the companies are sharing costs and profits on a 50:50 basis worldwide.

About the Seattle Genetics, Astellas and Merck Collaboration

Seattle Genetics and Astellas entered a clinical collaboration agreement with Merck to evaluate the combination of Seattle Genetics and Astellas PADCEV and Mercks KEYTRUDA (pembrolizumab), in patients with previously untreated metastatic urothelial cancer. KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

Seattle Genetics Forward-Looking Statements

Certain statements made in this press release are forward looking, such as those, among others, relating to the potential of data from the EV-103 clinical trial to support accelerated approval in the U.S. of PADCEV in combination with pembrolizumab for the treatment of patients with unresectable locally advanced or metastatic urothelial cancer who are unable to receive cisplatin-based chemotherapy in the first-line setting; the possibility of using data from the EV-302 clinical trial to obtain global regulatory approval or confirm accelerated approval of PADCEV in the referenced first line setting; clinical development plans relating to PADCEV; the therapeutic potential of PADCEV; and its possible safety, efficacy, and therapeutic uses, including in the first-line setting. Actual results or developments may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include the possibility that ongoing and subsequent clinical trials of PADCEV may fail to produce data sufficient to support regulatory approvals; the fact that FDA has not made a final determination regarding whether the data from the EV-103 clinical trial will be sufficient to support accelerated approval in the U.S.; the risk that the COVID-19 pandemic could delay our ability to conduct the EV-103 clinical trial and delay FDAs regulatory timelines, including with respect to any potential accelerated approval; the fact that adverse events or safety signals may occur and that adverse regulatory actions or other setbacks could occur as PADCEV advances in clinical trials even after promising results in earlier clinical trials. More information about the risks and uncertainties faced by Seattle Genetics is contained under the caption Risk Factors included in the companys Annual Report on Form 10-K for the year ended December 31, 2019 filed with the Securities and Exchange Commission. Seattle Genetics disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

1Challita-Eid P, Satpayev D, Yang P, et al. Enfortumab Vedotin Antibody-Drug Conjugate Targeting Nectin-4 Is a Highly Potent Therapeutic Agent in Multiple Preclinical Cancer Models. Cancer Res 2016;76(10):3003-13.2 PADCEV [package insert]. Northbrook, IL: Astellas, Inc.3 American Cancer Society. Cancer Facts & Figures 2020. https://www.cancer.org/content/dam/cancer-org/research/cancer-facts-and-statistics/annual-cancer-facts-and-figures/2020/cancer-facts-and-figures-2020.pdf. Accessed 02-20-2020.4 American Society of Clinical Oncology. Bladder cancer: introduction (10-2017). https://www.cancer.net/cancer-types/bladder-cancer/introduction. Accessed 05-09-2019.5 International Agency for Research on Cancer. Cancer Tomorrow: Bladder. http://gco.iarc.fr/tomorrow.6 Challita-Eid P, Satpayev D, Yang P, et al. Enfortumab Vedotin Antibody-Drug Conjugate Targeting Nectin-4 Is a Highly Potent Therapeutic Agent in Multiple Preclinical Cancer Models. Cancer Res 2016;76(10):3003-13.7 PADCEV [package insert]. Northbrook, IL: Astellas, Inc.8 PADCEV [package insert]. Northbrook, IL: Astellas, Inc.

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Seattle Genetics Announces Potential Accelerated Approval Pathway in the US for PADCEV (enfortumab vedotin-ejfv) in Combination with Immune Therapy...

Skin Stem Cells Comments Off on Seattle Genetics Announces Potential Accelerated Approval Pathway in the US for PADCEV (enfortumab vedotin-ejfv) in Combination with Immune Therapy… | April 2nd, 2020

news, national

Nearly a decade after doctors told him he had up to six years to live, Ian Fox's blood cancer is in remission. He was part of an early trial of Revlimid, one of a handful of drugs added to the Pharmaceutical Benefits Scheme on Wednesday. "It was a bit scary because right from the start they said, 'Look, there's not a cure for this'," the 65-year-old told AAP. He was placed on a trial for the drug in 2012 after he got stem cell treatment and he's happy he could help researchers, with doctors telling him the cancer was in remission in 2015. "I'm rapt ... I'm on a maintenance program now, still taking a minimum dose," he said. Before his diagnosis, Mr Fox noticed cooking smells were making him nauseous. But it wasn't until he donated blood that he was referred to a GP, with doctors finally diagnosing him with myeloma. Mr Fox said he couldn't have gotten through the past 10 years without his wife Lesley and his kids Cameron and Maddy. Now the retired web designer is working on his vintage cars, having had to give up his motorcycle collection due to peripheral neuropathy in his feet - damage to the nerve cells that carry messages to and from the brain and spinal cord - a side-effect of myeloma treatment. "That's why I'm sort of directing my energies into the classic cars, because they're a bit more stable," Mr Fox said. Revlimid targets blood cancer cells while boosting the immune system. Its addition to the PBS is expected to save Australians $194,000 in out-of-pocket costs over the course of their treatment. More than 1000 people each year are expected to benefit from the change, with about 18,000 Australians living with myeloma. The government has also added Kadcyla, a breast cancer treatment; Symtuza, a treatment for people living with HIV; and Briviact, an epilepsy drug. Health Minister Greg Hunt said the drugs would give patients a better quality of life and boost their chances of recovery. Australian Associated Press

https://nnimgt-a.akamaihd.net/transform/v1/crop/frm/silverstone-feed-data/5926065c-ff01-4d99-b2de-a02d418090f4.jpg/r0_74_800_526_w1200_h678_fmax.jpg

Nearly a decade after doctors told him he had up to six years to live, Ian Fox's blood cancer is in remission.

He was part of an early trial of Revlimid, one of a handful of drugs added to the Pharmaceutical Benefits Scheme on Wednesday.

"It was a bit scary because right from the start they said, 'Look, there's not a cure for this'," the 65-year-old told AAP.

He was placed on a trial for the drug in 2012 after he got stem cell treatment and he's happy he could help researchers, with doctors telling him the cancer was in remission in 2015.

"I'm rapt ... I'm on a maintenance program now, still taking a minimum dose," he said.

Before his diagnosis, Mr Fox noticed cooking smells were making him nauseous.

But it wasn't until he donated blood that he was referred to a GP, with doctors finally diagnosing him with myeloma.

Mr Fox said he couldn't have gotten through the past 10 years without his wife Lesley and his kids Cameron and Maddy.

Now the retired web designer is working on his vintage cars, having had to give up his motorcycle collection due to peripheral neuropathy in his feet - damage to the nerve cells that carry messages to and from the brain and spinal cord - a side-effect of myeloma treatment.

"That's why I'm sort of directing my energies into the classic cars, because they're a bit more stable," Mr Fox said.

Revlimid targets blood cancer cells while boosting the immune system.

Its addition to the PBS is expected to save Australians $194,000 in out-of-pocket costs over the course of their treatment.

More than 1000 people each year are expected to benefit from the change, with about 18,000 Australians living with myeloma.

The government has also added Kadcyla, a breast cancer treatment; Symtuza, a treatment for people living with HIV; and Briviact, an epilepsy drug.

Health Minister Greg Hunt said the drugs would give patients a better quality of life and boost their chances of recovery.

Australian Associated Press

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Blood cancer treatment added to PBS - Newcastle Herald

Spinal Cord Stem Cells Comments Off on Blood cancer treatment added to PBS – Newcastle Herald | April 1st, 2020

- Novellus's patented mRNA-based cell-reprogramming technology creates unique mesenchymal stem cells (MSCs) with superior immunomodulatory properties and manufacturing advantages over primary adult donor-derived MSCs - much greater supply and faster scale-up

- MSCs prevent and suppress cytokine storm believed to be the cause of the severe inflammation of ARDS and now seen in COVID-19 patients

CRANFORD, N.J., April 1, 2020 /PRNewswire/ -- Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a specialty pharmaceutical company focused on developing and commercializing critical care drug products, today signed an exclusive six-month option agreement to in-license a stem-cell therapy for acute respiratory distress syndrome (ARDS) from a subsidiary of Novellus, Inc., a preclinical-stage biotechnology company based in Cambridge, MA.

Novellus's patented process uses its exclusive non-immunogenic synthetic messenger ribonucleic acid (mRNA) molecules to create induced pluripotent stem cells (iPSCs) that, in turn, generate mesenchymal stem cells (MSCs) with superior immunomodulatory properties. MSCs have been shown to be safe in over 900 clinical trials and to be safe and effective in treating a number of inflammatory diseases, including ARDS.

"ARDS is the most common cause of respiratory failure and mortality in COVID-19 patients. Currently, there is no proven treatment for ARDS. Literature supports the use of counter-inflammatory MSCs for ARDS, and papers published in China have shown that at least seven COVID-19 patients with ARDS responded to MSC therapy. Clearly this is an avenue that shows promise and should be pursued as a potential treatment for ARDS. We believe Novellus is at the forefront of creating allogeneic, iPSC-derived MSCs. These cells have the potential to overcome the limitations of MSCs derived from adult donors, which are telomere shortened and introduce variability into the manufacturing process," said Citius Chief Executive Officer Myron Holubiak.

Novellus Chief Science Officer Matt Angel, PhD, stated, "Using our mRNA-based cell-reprogramming technology, Novellus can provide a near-unlimited supply of MSCs for treating patients with ARDS, including those critically ill from COVID-19. These will be allogeneic ('off-the-shelf') cells that in vitro have demonstrated much greater expansion potential and much higher immunomodulatory protein expression than donor-derived MSCs. We are excited to employ our technology to such an urgent medical crisis and believe that our MSCs represent an ideal source of cells to be used in this extremely important development effort."

Holubiak added, "No effective pharmacotherapy for ARDS exists, and ARDS-related morbidity and mortality are high. MSCs have been studied in the treatment of lung injury, and we aim to build upon this work with Novellus's iPSC-derived MSCs to improve the immunomodulatory response in humans. We have assembled a team of experts who are dedicated to advancing this project to an Investigational New Drug (IND) application as quickly as possible."

About ARDSAcute respiratory distress syndrome (ARDS) is a type of respiratory failure characterized by rapid onset of widespread inflammation in the lungs. ARDS is a rapidly progressive disease that occurs in critically ill patients most notably now in those diagnosed with COVID-19. ARDS affects approximately 200,000 patients per year in the U.S., exclusive of the current COVID-19 pandemic, and has a 30% to 50% mortality rate. ARDS is sometimes initially diagnosed as pneumonia or pulmonary edema (fluid in the lungs from heart disease). Symptoms of ARDS include shortness of breath, rapid breathing and heart rate, chest pain, particularly while inhaling, and bluish skin coloration. Among those who survive ARDS, a decreased quality of life is relatively common.

About Citius Pharmaceuticals, Inc.Citius is a late-stage specialty pharmaceutical company dedicated to the development and commercialization of critical care products, with a focus on anti-infectives and cancer care. For more information, please visit http://www.citiuspharma.com.

About Novellus, Inc.Novellus is a pre-clinical stage biotechnology company developing engineered cellular medicines using its non-immunogenic mRNA, nucleic-acid delivery, gene editing, and cell reprogramming technologies. Novellus is privately held and is headquartered in Cambridge, MA. For more information, please visit http://www.novellus-inc.com.

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Safe HarborThis press release may contain "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements are made based on our expectations and beliefs concerning future events impacting Citius. You can identify these statements by the fact that they use words such as "will," "anticipate," "estimate," "expect," "should," and "may" and other words and terms of similar meaning or use of future dates. Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition, and stock price. Factors that could cause actual results to differ materially from those currently anticipated are: the risk of successfully negotiating a license agreement with Novellus within the option period; our need for substantial additional funds; the estimated markets for our product candidates, including those for ARDS, and the acceptance thereof by any market; risks associated with conducting trials for our product candidates, including those expected to be required for any treatment for ARDS and our Phase III trial for Mino-Lok; risks relating to the results of research and development activities; risks associated with developing our product candidates, including any licensed from Novellus, including that preclinical results may not be predictive of clinical results and our ability to file an IND for such candidates; uncertainties relating to preclinical and clinical testing; the early stage of products under development; risks related to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; our ability to identify, acquire, close, and integrate product candidates and companies successfully and on a timely basis; our ability to attract, integrate, and retain key personnel; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions, or circumstances on which any such statement is based, except as required by law.

Contact:Andrew ScottVice President, Corporate Development(O) 908-967-6677ascott@citiuspharma.com

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Citius Signs Exclusive Option with Novellus to License Novel Stem-Cell Therapy for Acute Respiratory Distress Syndrome (ARDS) Associated with COVID-19...

Skin Stem Cells Comments Off on Citius Signs Exclusive Option with Novellus to License Novel Stem-Cell Therapy for Acute Respiratory Distress Syndrome (ARDS) Associated with COVID-19… | April 1st, 2020

Dublin, April 01, 2020 (GLOBE NEWSWIRE) -- The "Graft-Versus-Host Disease: Epidemiology Forecast in Asia-Pacific Markets to 2028" report has been added to ResearchAndMarkets.com's offering.

GvHD is a common complication of allogeneic HSCT that occurs when the donated (graft) cells are rejected and attack the host's cells as foreign. GvHD can progress from mild to severe forms as either aGvHD or cGvHD. Both aGvHD and cGvHD commonly affect organs such as the skin, gastrointestinal (GI) tract, liver, oral mucosa, and eyes. The global distribution of GvHD is directly dependent on transplantation-related factors, including the donor type, the age of the donor and the recipient, the sex parity between the recipient and the donor, the pre-transplantation conditioning regimen, and the use of GvHD prophylaxis pre- and/or post-transplantation.

The publisher epidemiologists utilized historical HSCT data available through country-wide registry reports in the 5GM to the best extent possible to arrive at a meaningful in-depth analysis and forecast for GvHD. In this analysis, The publisher epidemiologists provided detailed, clinically relevant segmentations for the diagnosed aGvHD and cGvHD incident cases. Further, The publisher epidemiologists used country-specific estimates using valid diagnostic criteria to present aGvHD and cGvHD prevalent, grades and mortality cases.

The following data describes epidemiology of GvHD cases. In 2018, the 5GM had 8,794 diagnosed incident cases of GvHD (aGvHD and cGvHD). This is expected to increase to 13,673 diagnosed incident cases by 2028, at an Annual Growth Rate (AGR) of 5.55%. This increase is partly attributed to the moderately rising trend in incidence in transplantation in the 5GM. In the 5GM, the diagnosed incident cases of aGvHD will increase from 4,650 cases in 2018 to 7,212 cases in 2028, at an Annual Growth Rate (AGR) of 5.51% per year, and the diagnosed incident cases of cGvHD will increase from 4,144 cases in 2018 to 6,461 cases in 2028, at an AGR of 5.59% per year.

Scope

Reasons to Buy

Key Topics Covered:

1 Table of Contents1.1 List of Tables1.2 List of Figures

2 Graft-Versus-Host Disease: Executive Summary2.1 Related Reports2.2 Upcoming Reports

3 Epidemiology3.1 Disease Background3.2 Risk Factors and Comorbidities3.3 Global and Historical Trends3.3.1 Australia3.3.2 China3.3.3 India3.3.4 Japan3.3.5 South Korea3.4 Forecast Methodology3.4.1 Sources Used3.4.2 Sources Not Used3.4.3 Forecast Assumptions and Methods3.5 Epidemiological Forecast for GvHD (2018-2028)3.5.1 Incident Cases of First Allogeneic HSCT3.5.2 Incident Cases of aGvHD in First Allogeneic HSCT3.5.3 Incident Cases of cGvHD in First Allogeneic HSCT3.5.4 Age-Specific Incident Cases of aGvHD and cGvHD3.5.5 Diagnosed Incident Cases of aGvHD by Grade3.5.6 Diagnosed Incident Cases of cGvHD by Severity3.5.7 100-Day Mortality in Diagnosed Incident Cases of aGvHD3.5.8 One-Year Mortality in Diagnosed Incident Cases of cGvHD3.5.9 Three-Year Diagnosed Prevalent Cases of aGvHD3.5.10 Three-Year Diagnosed Prevalent Cases of cGvHD3.6 Discussion3.6.1 Epidemiological Forecast Insight3.6.2 Limitations of Analysis3.6.3 Strengths of Analysis

4 Appendix4.1 Bibliography4.2 About the Authors4.2.1 Epidemiologist4.2.2 Reviewers4.2.3 Global Director of Therapy Analysis and Epidemiology4.2.4 Global Head and EVP of Healthcare Operations and Strategy4.3 About the publisher4.4 Contact Information4.5 Disclaimer

For more information about this report visit https://www.researchandmarkets.com/r/8yhcjq

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Insights into the Asia-Pacific Graft-Versus-Host Disease Industry to 2028 - Develop Business Strategies by Understanding the Trends Shaping and...

Skin Stem Cells Comments Off on Insights into the Asia-Pacific Graft-Versus-Host Disease Industry to 2028 – Develop Business Strategies by Understanding the Trends Shaping and… | April 1st, 2020

TORONTO, March 31, 2020 /CNW/ - The Gairdner Foundation is pleased to announce the 2020 Canada Gairdner Award laureates, recognizing some of the world's most significant biomedical research and discoveries. During these challenging times, we believe it is important to celebrate scientists and innovators from around the world and commend them for their tireless efforts to conduct research that impacts human health.

2020 Canada Gairdner International AwardThe five 2020 Canada Gairdner International Award laureates are recognized for seminal discoveries or contributions to biomedical science:

Dr. Masatoshi TakeichiSenior Visiting Scientist, RIKEN Center for Biosystems Dynamics Research, Kobe, Japan; Professor Emeritus, Kyoto University, Kyoto, Japan

Dr. Rolf KemlerEmeritus Member and Director, Max Planck Institute of Immunobiology and Epigenetics, Freiburg, Germany

Awarded "For their discovery, characterization and biology of cadherins and associated proteins in animal cell adhesion and signalling."

Dr. Takeichi

The Work: The animal body is made up of numerous cells. Dr. Takeichi was investigatinghow animal cells stick together to form tissues and organs, and identified a key protein which he named 'cadherin'.Cadherin is present on the surface of a cell and binds to the same cadherin protein on the surface of another cell through like-like interaction, thereby binding the cells together. Without cadherin, cell to cell adhesion becomes weakened and leads to the disorganization of tissues. Dr. Takeichi found that there are multiple kinds of cadherin within the body, each of which are made by different cell types, such as epithelial and neuronal cells. Cells with the same cadherins tend to cluster together, explaining the mechanism of how different cells are sorted out and organized to form functional organs.

Further studies by Dr. Takeichi's group showed that cadherin function is supported by a number of cytoplasmic proteins, includingcatenins, and their cooperation is essential for shaping of tissues. His studies also revealed that the cadherin-dependent adhesion mechanism is involved in synaptic connections between neurons, which are important for brain wiring.

Dr. Kemler

The Work: Dr. Kemler, using an immunological approach, developed antibodies directed against surface antigens of early mouse embryos. These antibodies were shown to prevent compaction of the mouse embryo and interfered with subsequent development. Both Dr. Kemler and Dr. Takeichi went on to clone and sequence the gene encoding E-cadherin and demonstrate that it was governing homophilic cell adhesion.

Dr. Kemler also discovered the other proteins that interact with the cadherins, especially the catenins, to generate the machinery involved in animal cell-to-cell adhesion. This provided the first evidence of their importance in normal development and diseases such as cancer. It has been discovered that cadherins and catenins are correlated to the formation and growth of some cancers and how tumors continue to grow. Beta catenin is linked to cell adhesion through interaction with cadherins but is also a key component of the Wnt signalling pathway that is involved in normal development and cancer. There are approximately 100 types of cadherins, known as the cadherin superfamily.

Dr. Takeichi

The Impact: The discovery of cadherins, which are found in all multicellular animalspecies, has allowed us to interpret how multicellular systems are generated and regulated. Loss of cadherin function has been implicated as the cause of certain cancers, as well as in invasiveness of many cancers. Mutations in special types of cadherin result in neurological disorders, such as epilepsy and hearing loss. The knowledge of cadherin function is expected to contribute to the development of effective treatments against such diseases.

Dr. Kemler

The Impact: Human tumors are often of epithelial origin. Given the role of E-cadherin for the integrity of an epithelial cell layer, the protein can be considered as a suppressor of tumor growth. The research on the cadherin superfamily has had great impact on fields as diverse as developmental biology, cell biology, oncology, immunology and neuroscience. Mutations in cadherins/catenins are frequently found in tumors. Various screens are being used to identify small molecules that might restore cell adhesion as a potential cancer therapy.

Dr. Roel NusseProfessor & Chair, Department of Developmental Biology; Member, Institute for StemCell Biology andRegenerativeMedicine, Stanford University, School of Medicine.Virginia and Daniel K. Ludwig Professor of Cancer Research. Investigator, Howard Hughes Medical Institute

Awarded"For pioneering work on the Wnt signaling pathway and its importance in development, cancer and stem cells"

The Work: Dr. Nusse's research has elucidated the mechanism and role of Wnt signaling, one of the most important signaling systems in development. There is now abundant evidence that Wnt signaling is active in cancer and in control of proliferation versus differentiation of adult stem cells, making the Wnt pathway one of the paradigms for the fundamental connections between normal development and cancer.

Among Dr. Nusse's contributions is the original discovery of the first Wnt gene (together with Harold Varmus) as an oncogene in mouse breast cancer. Afterwards Dr. Nusse identified the Drosophila Wnt homolog as a key developmental gene, Wingless. This led to the general realization of the remarkable links between normal development and cancer, now one of the main themes in cancer research. Using Drosophila genetics, he established the function of beta-catenin as a mediator of Wnt signaling and the Frizzleds as Wnt receptors (with Jeremy Nathans), thereby establishing core elements of what is now called the Wnt pathway. A major later accomplishment of his group was the first successful purification of active Wnt proteins, showing that they are lipid-modified and act as stem cell growth factors.

The Impact: Wnt signaling is implicated in the growth of human embryos and the maintenance of tissues. Consequently, elucidating the Wnt pathway is leading to deeper insights into degenerative diseases and the development of new therapeutics. The widespread role of Wnt signaling in cancer is significant for the treatment of the disease as well. Isolating active Wnt proteins has led to the use of Wnts by researchers world-wide as stem cell growth factors and the expansion of stem cells into organ-like structures (organoids).

Dr. Mina J. Bissell Distinguished Senior Scientist, Biological Systems and Engineering Division, Lawrence Berkeley National Laboratory; Faculty; Graduate Groups in Comparative Biochemistry, Endocrinology, Molecular Toxicology and Bioengineering, University of California Berkeley, Berkeley, CA, USA

Awarded "For characterizing "Dynamic Reciprocity" and the significant role that extracellular matrix (ECM) signaling and microenvironment play in gene regulation in normal and malignant cells, revolutionizing the fields of oncology and tissue homeostasis."

The Work: Dr. Mina Bissell's career has been driven by challenging established paradigms in cellular and developmental biology. Through her research, Dr. Bissell showed that tissue architecture plays a dominant role in determining cell and tissue phenotype and proposed the model of 'dynamic reciprocity' (DR) between the extracellular matrix (ECM) and chromatin within the cell nucleus. Dynamic reciprocity refers to the ongoing, bidirectional interaction between cells and their microenvironment. She demonstrated that the ECM could regulate gene expression just as gene expression could regulate ECM, and that these two phenomena could occur concurrently in normal or diseased tissue.

She also developed 3D culture systems to study the interaction of the microenvironment and tissue organization and growth, using the mammary gland as a model.

The Impact:Dr. Bissell's model of dynamic reciprocity has been proven and thoroughly established since its proposal three decades ago and the implications have permeated every area of cell and cancer biology, with significant implications for current and future therapies. Dr. Bissell's work has generated a fundamental and translationally crucial paradigm shift in our understanding of both normal and malignant tissues.

Her findings have had profound implications for cancer therapy by demonstrating that tumor cells can be influenced by their environment and are not just the product of their genetic mutations. For example, cells from the mammary glands grown in two-dimensional tissue cultures rapidly lose their identity, but once placed in proper three-dimensional microenvironments, they regain mammary form and function. This work presages the current excitement about generation of 3D tissue organoids and demonstrates Dr. Bissell's creative and innovative approach to science.

Dr. Elaine FuchsHoward Hughes Medical Institute Investigator and Rebecca C. Lancefield Professor and Head of the Robin Chemers Neustein Laboratory of Mammalian Cell Biology and Cell Biology; The Rockefeller University, New York, NY, USA

Awarded"For her studies elucidating the role of tissue stem cells in homeostasis, wound repair, inflammation and cancer."

The Work: Dr. Fuchs has used skin to study how the tissues of our body are able to replace dying cells and repair wounds. The skin must replenish itself constantly to protect against dehydration and harmful microbes. In her research, Fuchs showed that this is accomplished by a resident population of adult stem cells that continually generates a shell of indestructible cells that cover our body surface.

In her early research, Fuchs identified the proteins---keratinsthat produce the iron framework of the skin's building blocks, and showed that mutations in keratins are responsible for a group of blistering diseases in humans. In her later work, Fuchs identified the signals that prompt skin stem cells to make tissue and when to stop. In studying these processes, Fuchs learned that cancers hijack the fundamental mechanisms that tissue stem cells use to repair wounds. Her team pursued this parallel and isolated and characterized the malignant stem cells that are responsible for propagating a type of cancer called "squamous cell carcinoma." In her most recent work, she showed that these cells can be resistant to chemotherapies and immunotherapies and lead to tumor relapse.

The Impact: All tissues of our body must be able to replace dying cells and repair local wounds. Skin is particularly adept at performing these tasks. The identification and characterization of the resident skin stem cells that make and replenish the epidermis, sweat glands and hair provide important insights into this fountain of youth process and hold promise for regenerative medicine and aging. In normal tissues, the self-renewing ability of stem cells to proliferate is held in check by local inhibitory signals coming from the stem cells' neighbours. In injury, stimulatory signals mobilize the stem cells to proliferate and repair the wound. In aging, these normal balancing cues are tipped in favour of quiescence. In inflammatory disorders, stem cells become hyperactivated. In cancers, the wound mechanisms to mobilize stem cells are hijacked, leading to uncontrolled tissue growth. Understanding the basic mechanisms controlling stem cells in their native tissue is providing new strategies for searching out refractory tumor cells in cancer and for restoring normalcy in inflammatory conditions.

2020 John Dirks Canada Gairdner Global Health AwardThe 2020 John Dirks Canada Gairdner Global Health Award laureate is recognized for outstanding achievements in global health research:

Professor Salim S. Abdool KarimDirector of CAPRISA (Centre for the AIDS Program of Research in South Africa), the CAPRISA Professor in Global Health at Columbia University, New York and Pro Vice-Chancellor (Research) at the University of KwaZulu-Natal, Durban, South Africa

Professor Quarraisha Abdool KarimAssociate Scientific Director of CAPRISA, Professor in Clinical Epidemiology, Columbia University, New York and Professor in Public Health at the Nelson Mandela Medical School and Pro Vice-Chancellor (African Health) at the University of KwaZulu-Natal, Durban, South Africa

Awarded"For their discovery that antiretrovirals prevent sexual transmission of HIV, which laid the foundations for pre-exposure prophylaxis (PrEP), the HIV prevention strategy that is contributing to the reduction of HIV infection in Africa and around the world."

The Work: UNAIDS estimates that 37 million people were living with HIV and 1.8 million people acquired HIV in 2017. In Africa, which has over two thirds of all people with HIV, adolescent girls and young women have the highest rates of new HIV infections. ABC (Abstinence, Be faithful, and use Condoms) prevention messages have had little impact - due to gender power imbalances, young women are often unable to successfully negotiate condom use, insist on mutual monogamy, or convince their male partners to have an HIV test.

In responding to this crisis, Salim and Quarraisha Abdool Karim started investigating new HIV prevention technologies for women about 30 years ago. After two unsuccessful decades, their perseverance paid off when they provided proof-of-concept that antiretrovirals prevent sexually acquired HIV infection in women. Their ground-breaking CAPRISA 004 trial showed that tenofovir gel prevents both HIV infection and genital herpes. The finding was ranked inthe "Top 10 Scientific Breakthroughs of 2010" by the journal, Science. The finding was heralded by UNAIDS and the World Health Organization (WHO) as one of the most significant scientific breakthroughs in AIDS and provided the first evidence for what is today known as HIV pre-exposure prophylaxis (PrEP).

The Abdool Karims have also elucidated the evolving nature of the HIV epidemic in Africa, characterising the key social, behavioural and biological risk factors responsible for the disproportionately high HIV burden in young women. Their identification of the "Cycle of HIV Transmission", where teenage girls acquire HIV from men about 10 years older on average, has shaped UNAIDS policies on HIV prevention in Africa.

The impact: CAPRISA 004 and several clinical trials of oral tenofovir led tothe WHO recommending a daily tenofovir-containing pill for PrEP as a standard HIV prevention tool for all those at high risk a few years later. Several African countries are among the 68 countries across all continents that are currently making PrEP available for HIV prevention. The research undertaken in Africa by this South African couple has played a key role in shaping the local and global response to the HIV epidemic.

2020 Canada Gairdner Wightman AwardThe 2020 Canada Gairdner Wightman Award laureate is a Canadian scientist recognized for outstanding leadership in medicine and medical science throughout their career:

Dr. Guy Rouleau Director of the Montreal Neurological Institute-Hospital (The Neuro); Professor & Chair of the Department of Neurology and Neurosurgery, McGill University; Director of the Department of Neuroscience, McGill University Health Center

Awarded "For identifying and elucidating the genetic architecture of neurological and psychiatric diseases, including ALS, autism and schizophrenia, and his leadership in the field of Open Science."

The Work: Dr. Rouleau has identified over 20 genetic risk factors predisposing to a range of brain disorders, both neurological and psychiatric, involving either neurodevelopmental processes or degenerative events. He has defined a novel disease mechanism for diseases related to repeat expansions that are at play in some of the most severe neurodegenerative conditions. He has significantly contributed to the understanding of the role of de novo variants in autism and schizophrenia. In addition, he has made important advances for various neuropathies, in particular for amyotrophic lateral sclerosis (ALS) where he was involved in the identification of the most prevalent genetic risk factors -which in turn are now the core of innumerable ALS studies worldwide.

Dr. Rouleau has also played a pioneering role in the practice of Open Science (OS), transforming the Montreal Neurological Institute-Hospital (The Neuro) into the first OS institution in the world. The Neuro now uses OS principles to transform research and careand accelerate the development of new treatments for patients through Open Access, Open Data, Open Biobanking, Open Early Drug Discovery and non-restrictive intellectual property.

The Impact: The identification of genetic risk factors has a number of significant consequences. First, allowing for more accurate genetic counselling, which reduces the burden of disease to affected individuals, parents and society. A revealing case is Andermann syndrome, a severe neurodevelopmental and neurodegenerative condition that was once relatively common in the Saguenay-Lac-St-Jean region of Quebec. Now this disease has almost disappeared from that population. Second, identifying the causative gene allows the development of treatments. For instance, his earlier work on a form of ALS linked to the superoxide dismutase-1 gene (SOD1) opened up studies which are now the focal point of phase 2 clinical studies showing great promise.

Byactingasalivinglabforthelast coupleofyears,TheNeuroisspearheading the practice of OpenScience (OS).TheNeurois alsoengagingstakeholdersacross Canadawiththegoal of formalizinganational OSallianceforthe neurosciences.Dr.Rouleau'sworkinOScontributesfundamentallytothetransformationoftheveryecosystemofsciencebystimulatingnewthinkingandfosteringcommunitiesofsharing.InspiredbyTheNeuro'svision,theglobalsciencecommunityisreflecting oncurrentresearchconventionsandcollaborativeprojects,andthemomentumforOSisgainingafootholdinorganizationsandinstitutionsinallcornersoftheearth.

About the Gairdner Foundation:

The Gairdner Foundation was established in 1957 by Toronto stockbroker, James Gairdner to award annual prizes to scientists whose discoveries have had major impact on scientific progress and on human health. Since 1959 when the first awards were granted, 387scientists have received a Canada Gairdner Award and 92 to date have gone on to receive the Nobel Prize.The Canada Gairdner Awards promote a stronger culture of research and innovation across the country through our Outreach Programs including lectures and research symposia. The programs bring current and past laureates to a minimum of 15 universities across Canada to speak with faculty, trainees and high school students to inspire the next generation of researchers. Annual research symposia and public lectures are organized across Canada to provide Canadians access to leading science through Gairdner's convening power.

http://www.gairdner.org

SOURCE Gairdner Foundation

View original content to download multimedia: http://www.newswire.ca/en/releases/archive/March2020/31/c7291.html

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2020 Canada Gairdner Awards Recognize World-renowned Scientists for Transformative Contributions to Research That Impact Human Health - Benzinga

Skin Stem Cells Comments Off on 2020 Canada Gairdner Awards Recognize World-renowned Scientists for Transformative Contributions to Research That Impact Human Health – Benzinga | April 1st, 2020

Researchers at the Institute of Oral Biology of the University of Zurich have released 2 studies that examine how stem cells promote neuronal growth in tissue regeneration and in cancer progression.

Their findings demonstrate that different stem cell populations are innervated in distinct ways and that innervation may be crucial for proper tissue regeneration, according to the study. They also demonstrate that cancer steam cells likewise establish contacts with nerves.

Stem cells can generate a variety of specific tissues that are increasingly being used for clinical application, such as the replacement of bone or cartilage, but are present in cancerous tissues and are involved in cancer progression and metastasis. Nerves are therefore fundamental for regulating the physiological and regenerative processes involving stem cells.

Using organ-on-a-chip technology, which relies on small 3-dimensional devices mimicking the basic function of human organs and tissues, the researchers demonstrated that both types of stem cells promoted neuronal growth. The dental pulp stem cells, however, yielded better results compared with bone marrow stem cells. They induced more elongated neurons, formed dense neuronal networks, and established close contacts with nerves.

Dental stem cells produce specific molecules that are fundamental for the growth and attraction of neurons. Therefore, stem cells are abundantly innervated, according to the study authors. The formation of such extended networks and the establishment of numerous contacts suggest that dental stem cells create functional connections with nerves of the face. Therefore, these cells could represent an attractive choice for the regeneration of functional, properly innervated facial tissue.

In the second study, the researchers examined the interaction between nerves and cancer stem cells found in ameloblastoma, an aggressive tumor of the mouth. They first demonstrated that ameloblastomas have stem cell properties and are innervated by facial neurons. When ameloblastoma cells were isolated and placed in the organ-on-a-chip devices, they retained not only their stem cell properties, but also attracted nerves and established contact with them.

Nerves appear to be fundamental for the survival and function of cancer stem cells. These results create new possibilities for cancer treatment using drugs that modify the communication between neurons and cancer stem cells. The researchers hope this opens unforeseen paths toward effective therapies against cancer.

The combination of advanced molecular and imaging tools and organ-on-a-chip technology offers an opportunity to reveal the hidden functions of neurons and their interactions with various stem cell types, in both healthy and pathological conditions.

Reference

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Stem Cells, Nerves Found to Interact in Cancer Progression - Pharmacy Times

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Dr. Jim Surrell, Journal columnist

The overall function of our human immune system is to prevent or limit infection. The primary job of our immune system is to distinguish between our normal, healthy cells and possible other dangerous cells, such as viruses and bacteria that may come into our blood stream. Our immune system is always on duty to look for and recognize these possible infectious viruses and bacteria.

The immune system looks closely at these potentially infectious cells to do all it can to prevent us from getting an infection.

Know that we rely on our immune system every day to help us fight off infections and keep us healthy. Our immune system contains numerous cell types that either circulate throughout the body or reside in our particular body tissues. Each cell type plays a unique role, with different ways of performing their function to fight off infections. Further, our great medical and scientific researchers continuously work to optimize our immune responses to confront specific potential infectious issues, such as is being done at this time with regard to the coronavirus (COVID-19).

Let us now take a look at the many components of our human immune system and how our body works to fight off infections. All of our numerous immune cells come from basic immune cells in our bone marrow and develop into mature cells through a series of changes that can occur in different parts of the body. Following is a brief look at our various body components that make up our immune system.

Skin: Our skin is usually the first line of defense against infectious organisms. Skin cells produce and secrete important antimicrobial proteins, and immune cells can be found in specific layers of our skin.

Bone marrow: Our bone marrow contains stem cells that can develop into a variety of cell types. These stem cells in our bone marrow develop our many various types of immune cells that are very important first-line responders to infection. These stem cells create our essential infection-fighting cells, called B cells and T cells. These B cells and T cells are responsible for mounting a response to specific microbes that may cause infections. We also have natural niller (NK) immune cells that also provide defenses to fight off infections. These immune system B cells, T cells, and NK cells are also called lymphocytes.

Bloodstream: Immune cells constantly circulate throughout the bloodstream, patrolling for problems. When blood tests are used to monitor white blood cells, another term for immune cells, a snapshot of the immune system is taken. If our white blood cells are too few, or overabundant in the bloodstream, this may reflect a problem that should be addressed by a professional health care provider.

Thymus Gland: Immune system T cells mature in our small thymus gland, located in the upper chest.

Lymphatic system: The lymphatic system is a network of vessels and tissues composed of lymph, an extracellular fluid, and our lymph nodes. The lymphatic system is the essential part of our immune system that provides communication between our various body tissues and our bloodstream. Immune cells are carried through the lymphatic system and converge in lymph nodes. Lymph nodes are a communication hub where immune cells sample information brought in from the body. Thus, doctors may check patients for swollen lymph nodes, which may indicate an active immune response.

Spleen: The spleen is an organ located behind the stomach. Immune cells are found in the spleen and if there are any blood-borne infectious organisms, these immune cells activate and respond accordingly.

Lastly, be aware that our human immune system regenerates and repairs itself every night when we sleep. Studies show that people who dont get quality sleep or enough sleep are more likely to get sick after being exposed to an infectious virus or bacteria. Lack of sleep can also affect how fast you recover if you do get sick. To keep you and your immune system healthy, get the recommended seven to eight hours of sleep each and every night. And yes, these are Doctors Orders!

EDITORS NOTE: Dr. Jim Surrell is the author of The ABCs For Success In All We Do and the SOS (Stop Only Sugar) Diet books. Requests for health topics for this column are encouraged. Contact Dr. Surrell by email at sosdietdoc@gmail.com.

At the time I am writing to you, scientists are hunched over test tubes and Petri dishes in a desperate race ...

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Talk with the Doc | News, Sports, Jobs - Marquette Mining Journal

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VIVA! Communications

Australians living with an incurable blood cancer set to receive new reimbursed treatment option

Australians living with the incurable blood cancer, multiple myeloma1 are set to gain access to a new treatment option, with the listing of REVLIMID(lenalidomide) for maintenance treatment on the Pharmaceutical Benefits Scheme (PBS) from Wednesday April 1, 2020.2

REVLIMID(lenalidomide) represents Australias first and only maintenance treatment specifically indicated and reimbursed for those newly diagnosed with multiple myeloma (NDMM) who have undergone an autologous stem cell transplant (ASCT). 2, 3

This announcement coincides with an article just published in Medical Journal of Australias MJA Insight, calling for timely access to effective treatments for those living with multiple myeloma.4

According to article author, Professor Miles Prince, Clinical Haematologist and Director of Cancer Immunology and Molecular Oncology (Epworth Healthcare, Melbourne), continuing to broaden access to multiple myeloma treatments is critical to improving patient quality of life.4

Currently, people living with myeloma have a median survival rate of more than seven years which is significant in comparison to the median survival rate of just three years in the early 2000s.4

For survival rates to continue to improve however, patients must receive timely access to the most effective treatments, said Prof. Prince.

The PBS listing of maintenance for multiple myeloma will provide newly diagnosed patients with an additional treatment option for their disease.

Not to be confused with the skin cancer, melanoma, multiple myeloma is an incurable blood cancer that develops from plasma cells, a type of white blood cell found in the bone marrow.5, 6

Representing Australias third most common blood cancer (after lymphoma and leukaemia),7, 8 approximately 18,000 Australians are living with multiple myeloma at any given time,1 only half of whom will survive five years post- diagnosis.9

Myeloma Australia CEO, Steve Roach, today welcomed the availability of a new treatment option for the incurable disease.

The multiple myeloma patient journey involves a pattern of Response, Remission and Relapse, with individuals responding differently to certain treatments due to the complex nature of the devastating disease.

Additional treatment options are required throughout the patient journey, for both the newly diagnosed, and those who have already commenced therapy. Although incurable, we hope that multiple myeloma will one day be treated as a chronic, rather than a terminal disease, Mr Roach said.

The incurable nature of the disease and the likelihood of relapse, may have a psychological impact on patients, who can continue to live in fear even during periods of remission.10, 11

With studies revealing more than half (52%) of those living with multiple myeloma experience symptoms of anxiety or depression,12 improving access to treatment, and extending time spent in remission, may help to improve psychological wellbeing.

Wife and mother-to-two, Maria (53) was diagnosed with multiple myeloma in December 2018 and found her initial diagnosis very overwhelming.

When my husband Danny and I first heard the diagnosis, we were completely overwhelmed. We didnt know what Myeloma was and we didnt know what this meant short or long term. I didnt know if I was going to die in a month, a year or 10 years. How on earth was I going to tell everyone I had cancer, said Maria.

During my journey I blogged about my experience with multiple myeloma and posted to my Facebook daily to keep the calls and fears of my family and friends at bay. I have since accepted that myeloma is now a part of my life. I have no anger or fear and instead just live in the moment and take one day at a time.

Its very exciting to see new treatment options for multiple myeloma being funded by the government, and I hope to keep raising awareness, to ensure the myeloma community continues to receive access to the best treatment options available, Maria said.

About multiple myeloma

Multiple myeloma is a cancer that develops from abnormal plasma cells. A plasma cell is a type of white blood cell found in the bone marrow, that forms part of the immune system and helps to protect against infection.4, 7 The abnormal plasma cells crowd the bone marrow and make it difficult to produce enough normal blood cells.7

Multiple myeloma can be challenging to diagnose due to its wide range of symptoms, including high blood calcium levels, anaemia, fatigue, kidney failure, recurrent infections and bone pain.6

Current treatment for multiple myeloma includes a continuous approach, often comprising initial therapy, consolidation maintenance, and salvage therapy.13 Treatment options for multiple myeloma include chemotherapy, corticosteroids, autologous stem cell transplant (ASCT), immunomodulating drugs (IMiDs) monoclonal antibodies therapy, and proteasome inhibitors.14, 15

About REVLIMID (lenalidomide)

Representing an oral medication approved for the treatment of relapsed myeloma over 10 years ago,16 REVLIMID is an immunomodulating agent (IMiD) that slows the growth of multiple myeloma plasma tumour cells and proteins know to play a key role multiple myeloma, delays the development of new blood vessels, and enhances immune function.3

The most commonly reported side-effects of REVLIMID include diarrhoea, constipation, nausea, vomiting, stomach pain, indigestion, dehydration, dry mouth, mouth ulcer, sore mouth, increase or decrease in weight, increase or decrease in appetite, loss of taste, itchiness, rash, redness of the skin, dry skin, bruising, excessive sweating, dizziness, fainting, headache, shaking or tremors, unusual weakness, night sweats, reduced sense of touch, difficulty sleeping, depression, anxiety, feeling of confusion, back pain, muscle spasms, muscle and/or joint pain, swollen joints, bone pain, muscular weakness, pain in the extremities, feeling tired, falling, swelling of hands, ankles or feet.17

Other possible side effects of REVLIMID include heart palpitations or fast heartbeat, chest pains, dizziness or fainting, shortness of breath, weakness or reduced ability to exercise, bleeding (including nose-bleeds), bruising more than usual, numbness, tingling, blurred vision or difficulty seeing, passing large amounts of urine, excessive thirst, and having a dry mouth and skin, abnormal eye movements, convulsions, mood changes, irregular heart rhythms or tender swollen lymph nodes. 17

All patients receiving REVLIMID must be registered on, and abide by the requirements of, i-access risk management program to avoid exposure to unborn babies, due to the potential for birth defects. It is important to note that a small number of patients with multiple myeloma may develop additional types of cancer, regardless of their type of therapy. At this stage, it cannot be excluded that this risk may be slightly increased with REVLIMID maintenance treatment.17

Disclosure

Celgene supports disclosure and transparency on interactions between the healthcare industry and healthcare professionals to ensure public trust and confidence. No expert spokespeople have been offered compensation for their involvement in this media campaign. All expert spokespeople have been briefed on the approved use of these products and their obligations with regard to promotion to the general public. Prof Miles Prince and Myeloma Australia have received funding from Celgene for projects unrelated to this announcement. All opinions expressed are their own.

About Celgene

Celgene, a Bristol-Myers Squibb Company, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialisation of innovative therapies for the treatment of cancer and inflammatory diseases, through next-generation solutions in protein homeostasis, immuno-oncology, epigenetics, immunology and neuro-inflammation.

/Public Release.

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Australians living with an incurable blood cancer set to receive new reimbursed treatment option - Mirage News

Bone Marrow Stem Cells Comments Off on Australians living with an incurable blood cancer set to receive new reimbursed treatment option – Mirage News | April 1st, 2020

VANCOUVER, Washington, March 30, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn or the Company), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced today that an additional three critically ill COVID-19 patients have been treated with leronlimab. These additional patients increase the total to 10 patients receiving leronlimab treatment under an Emergency Investigational New Drug (EIND) granted by the U.S. Food and Drug Administration (FDA).

The treatment with leronlimab is targeted as a therapy for patients who experience respiratory complications as a result of contracting SARS-CoV-2 causing the Coronavirus Disease 2019 (COVID-19). Leronlimab is believed to provide therapeutic benefit by enhancing the immune response while mitigating the cytokine storm that leads to morbidity and mortality in these patients. The laboratory evaluation of the first four patients treated with leronlimab revealed that the immune profile in these patients approached normal levels and the levels of cytokines involved in the cytokine storm (including IL-6 and TNF alpha) were much improved. The results of the three additional patients are expected this week.

Jacob Lalezari, M.D., Interim Chief Medical Officer of CytoDyn, commented, The preliminary results observed in patients who were severely ill with COVID-19 and treated with leronlimab are encouraging. Although the data set is still small, we saw fairly rapid and positive laboratory responses in all 4 patients treated, and in three of the 4 patients these laboratory results were associated with a favorable clinical outcome. We eagerly await the results of additional patients treated under the FDAs emergency IND program, as well as the results of several randomized clinical trials about to start.

Nader Pourhassan, Ph.D., President and Chief Executive Officer of CytoDyn, added, We remain encouraged and hopeful that leronlimab will help patients from this devastating and relentless disease. We will aggressively pursue treatment for COVID-19 patients, and to explore leronlimabs role in helping to alleviate the impending burden of supply chain and institutional capacity issues.

About Coronavirus Disease 2019SARS-CoV-2 was identified as the cause of an outbreak of respiratory illness first detected in Wuhan, China. The origin of SARS-CoV-2 causing the COVID-19 disease is uncertain, and the virus is highly contagious. COVID-19 typically transmits person to person through respiratory droplets, commonly resulting from coughing, sneezing, and close personal contact. Coronaviruses are a large family of viruses, some causing illness in people and others that circulate among animals. For confirmed COVID-19 infections, symptoms have included fever, cough, and shortness of breath. The symptoms of COVID-19 may appear in as few as two days or as long as 14 days after exposure. Clinical manifestations in patients have ranged from non-existent to severe and fatal. At this time, there are minimal treatment options for COVID-19.

About Leronlimab (PRO 140) The FDA has granted a Fast Track designation to CytoDyn for two potential indications of leronlimab for deadly diseases. The first as a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer. Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including NASH. Leronlimab has completed nine clinical trials in over 800 people, including meeting its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab could significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

In the setting of cancer, research has shown that CCR5 may play a role in tumor invasion, metastases, and tumor microenvironment control. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is, therefore, conducting a Phase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to support further the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD, blocking the CCR5 receptor from recognizing specific immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted orphan drug designation to leronlimab for the prevention of GvHD.

About CytoDyn CytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a critical role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as GvHD and NASH. CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. CytoDyn plans to seek FDA approval for leronlimab in combination therapy and plans to complete the filing of a Biologics License Application (BLA) in April of 2020 for that indication. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication. If successful, it could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs). Moreover, a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients; some patients on leronlimab monotherapy have remained virally suppressed for more than five years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is at http://www.cytodyn.com.

Forward-Looking Statements This press release contains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as believes, hopes, intends, estimates, expects, projects, plans, anticipates and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. The Companys forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i) the sufficiency of the Companys cash position, (ii) the Companys ability to raise additional capital to fund its operations, (iii) the Companys ability to meet its debt obligations, if any, (iv) the Companys ability to enter into partnership or licensing arrangements with third parties, (v) the Companys ability to identify patients to enroll in its clinical trials in a timely fashion, (vi) the Companys ability to achieve approval of a marketable product, (vii) the design, implementation and conduct of the Companys clinical trials, (viii) the results of the Companys clinical trials, including the possibility of unfavorable clinical trial results, (ix) the market for, and marketability of, any product that is approved, (x) the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companys products, (xi) regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii) general economic and business conditions, (xiii) changes in foreign, political, and social conditions, and (xiv) various other matters, many of which are beyond the Companys control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form 10-K, and any risk factors or cautionary statements included in any subsequent Form 10-Q or Form 8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CYTODYN CONTACTSInvestors: Dave Gentry, CEO RedChip Companies Office: 1.800.RED.CHIP (733.2447) Cell: 407.491.4498 dave@redchip.com

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Three Additional Patients with Severe COVID-19 Treated with Leronlimab in New York Medical Center Bringing the Total to 10 Patients - Associated Press

Bone Marrow Stem Cells Comments Off on Three Additional Patients with Severe COVID-19 Treated with Leronlimab in New York Medical Center Bringing the Total to 10 Patients – Associated Press | March 31st, 2020

Dailymail.co.uk

Adam Castillejo (pictured), 40, was known only as the London Patient when doctors revealed his success story last March after a stem cell transplant to treat his cancer.

The second person to be cured of HIV has revealed how he was more fearful of dying from cancer than Aids and considered ending his life at Dignitas.Adam Castillejo, 40, was known only as the London Patient when doctors revealed his success story last March after a stem cell transplant to treat his cancer.He remained anonymous until he decided he wanted to be seen as an ambassador of hope after struggling with his health for almost two decades.Mr Castillejo, who was born in Venezuela and moved to London in 2002, was diagnosed with blood cancer in 2012, having already lived with HIV since 2003.His last hope of cancer survival was a bone marrow transplant from a donor with HIV-resistant genes that could wipe out his cancer and virus in one fell swoop.But in a powerful interview with The Sunday Times,Mr Castillejo admitted that he was more fearful of dying from stage 4 Hodgkins lymphoma than Aids.Calling the second diagnosis another death sentence, the sou-chef revealed that he panicked because cancer can kill you faster than HIV.Adam Castillejo, 40, was known only as the London Patient when doctors revealed his success story last March after a stem cell transplant to treat his cancerMr Castillejo embarked upon a gruelling treatment regime that left him physically emaciated and pushed the Venezuelan to the mental edge.Both illnesses became one because you had to deal with the anti-retroviral medications not interfering with thechemotherapy regime and vice versa, he said.By the end of 2014, he said that he had given up on battling the two illnesses, and had made up his mind to end it all at Dignitas in Switzerland.Around this time,Mr Castillejo disappeared, and was found four days later outside London psychologically broken. He does not remember this period.Doctors gave him six months to live, before a switch flicked.At that time I accepted straight away, because what choice have I got? I would rather die fighting, he explained.Within days, he met with Dr Ian Gabriel at the Chelsea and Westminster Hospital, who advised that he could attempt a bone marrow transplant.The procedure in May 2016 meantMr Castillejo was cleared of both cancer and HIV.But he lost five stone and took 60 pills a day, revealing: I told my doctors there werent enough hours in the day to take all the medication I needed.Mr Castillejo, who was born in Venezuela and moved to London in 2002, was diagnosed with blood cancer in 2012, having already lived with HIV since 2003An American man treated in Germany 12 years ago called Timothy Ray Brown (pictured) the so-called Berlin Patient also survived the transplantHe also developed mouth ulcers which inhibited his ability to eat, and his anti-retroviral medication had to be crushed and washed down.Mr Castillejo also claimed that he felt victimised and guilty when he told people that he was suffering from HIV, saying: This is a punishment for you.The Venezuelan chef is the second person to have survived the life-threatening technique and come out the other side HIV-free.An American man treated in Germany 12 years ago called Timothy Ray Brown the so-called Berlin Patient also survived the transplant.He was put into an induced coma for six months, however.Experts have hailed the treatment as a milestone in the fight against HIV, but are urging caution when calling it a cure so early on.In the context of HIV infection, the term cure means there are no virus-carrying cells left.Anti-retroviral therapy is very effective at reducing the viral load in the blood of infected individuals so that it cannot be transmitted to others.Unfortunately, the Berlin and London Patients cases do not change the reality much for 37 million HIV patients.The treatment is unlikely to have potential on a wider scale because both Mr Castillejo and Mr Ray Brown were given stem cells to treat cancer, not HIV.Stem cell and bone marrow transplants are life-threatening operations with huge risks. Patients can suffer a fatal reaction if substitute immune cells dont take.In his private life, Mr Castillejo likes to walk the streets of Shoreditch and travel.Kat Smithson, director of policy at National AIDS Trust, said: We applaud the London Patient Adam Castillejo for sharing his unique experience of having his HIV cured following a bone-marrow transplant to treat cancer. Mr Castillejo has been through a long and extremely challenging journey with his health, within which HIV is just one part.His decision to speak about his experience without anonymity can only enrich our understanding of his experience on a human level, and we thank him for this.Theres still a great deal of stigma around HIV which can make it harder for people to access the services and support they need and for people to talk openly about HIV.His story helps raise much-needed awareness of HIV, but broader than that its a story about incredible resilience, determination and hope.How a stem cell transplant cured the Berlin and London Patients and how it can go badly wrongUsually, HIV patients expect to stay on daily pills for life to suppress the virus. When drugs are stopped, the virus roars back, usually in two to three weeksThe vast majority of humans carry the gene CCR5.In many ways, it is incredibly unhelpful. It affects our odds of surviving and recovering from a stroke, according to recent research.And it is the main access point for HIV to overtake our immune systems.But some people carry a mutations that prevents CCR5 from expressing itself, effectively blocking or eliminating the gene.Those few people in the world are called elite controllers by HIV experts. They are naturally resistant to HIV.If the virus ever entered their body, they would naturally control the virus as if they were taking the virus-suppressing drugs that HIV patients require.Both the Berlin patient and the London patient received stem cells donated from people with that crucial mutation.WHY HAS IT NEVER WORKED BEFORE?There are many reasons this hasnt worked, Dr Janet Siliciano, at the Johns Hopkins University School of Medicine, told DailyMail.com.1. FINDING DONORSIts incredibly difficult to find HLA-matched bone marrow [i.e. someone with the same proteins in their blood as you], Dr Siliciano said.Its even more difficult to find the CCR5 mutation.2. INEFFECTIVE TRANSPLANT LEADS TO CANCER RELAPSESecond, there is a risk that the bone marrow wont take.Sometimes you dont become fully chimeric, meaning you still have a lot of your own cells.This means they will not defeat the cancer if it returns again.3. THE OLD IMMUNE SYSTEM ATTACKS THE NEW ONEThe other most common reason this approach has failed is graft-versus-host disease: whenthe patients immune system tries to attack the incoming, replacement immune system, causing a fatal reaction in most.4. UNKNOWN QUANTITIESInterestingly, both the Berlin patient and the London patient experienced complications that are normally lethal in most other cases.And experts believe that those complications helped their cases.Timothy Ray Brown, the Berlin patient, had both his cancer returned and he developed graft-versus-host disease, putting him in a coma and requiring a second bone marrow transplant.The London patient had one: he suffered graft-versus-host disease.Against the odds, they both survived, HIV-free.Some believe that, ironically, graft-versus-host disease might have helped both of them to further obliterate their HIV.But there is no way to control or replicate that safely.

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Adam Castillejo 'feared dying of cancer more than Aids and considered ending it all at Dignitas' Daily Mail - westofthepond.com

Bone Marrow Stem Cells Comments Off on Adam Castillejo ‘feared dying of cancer more than Aids and considered ending it all at Dignitas’ Daily Mail – westofthepond.com | March 31st, 2020

Researchers at Stanford University report that they can rejuvenate human cells by reprogramming them back to a youthful state. They hope that the technique will help in the treatment of diseases, such as osteoarthritis and muscle wasting, that are caused by the aging of tissue cells.

A major cause of aging is thought to be the errors that accumulate in the epigenome, the system of proteins that packages the DNA and controls access to its genes. The Stanford team, led by Tapash Jay Sarkar, Dr. Thomas A. Rando and Vittorio Sebastiano, say their method, designed to reverse these errors and walk back the cells to their youthful state, does indeed restore the cells vigor and eliminate signs of aging.

In their report, published on Tuesday in Nature Communications, they described their technique as a significant step toward the goal of reversing cellular aging and could produce therapies for aging and aging-related diseases.

Leonard P. Guarente, an expert on aging at M.I.T., said the method was one of the most promising areas of aging research but that it would take a long time to develop drugs based on RNA, the required chemical.

The Stanford approach utilizes powerful agents known as Yamanaka factors, which reprogram a cells epigenome to its time zero, or embryonic state.

Embryonic cells, derived from the fertilized egg, can develop into any of the specialized cell types of the body. Their fate, whether to become a skin or eye or liver cell, is determined by chemical groups, or marks, that are tagged on to their epigenome.

In each type of cell, these marks make accessible only the genes that the cell type needs, while locking down all other genes in the DNAs. The pattern of marks thus establishes each cells identity.

As the cell ages, it accumulates errors in the marking system, which degrade the cells efficiency at switching on and off the genes needed for its operations.

In 2006 Dr. Shinya Yamanaka, a stem-cell researcher at Kyoto University, amazed biologists by showing that a cells fate could be reversed with a set of four transcription factors agents that activate genes that he had identified. A cell dosed with the Yamanaka factors erases the marks on the epigenome, so the cell loses its identity and reverts to the embryonic state. Erroneous marks gathered during aging are also lost in the process, restoring the cell to its state of youth. Dr. Yamanaka shared the 2012 Nobel Prize in medicine for the work.

But the Yamanaka factors are no simple panacea. Applied to whole mice, the factors made cells lose their functions and primed them for rapid growth, usually cancerous; the mice all died.

In 2016, Juan Carlos Izpisua Belmonte, of the Salk Institute for Biological Studies in San Diego, found that the two effects of the Yamanaka factors erasing cell identity and reversing aging could be separated, with a lower dose securing just age reversal. But he achieved this by genetically engineering mice, a technique not usable in people.

In their paper on Tuesday, the Stanford team described a feasible way to deliver Yamanaka factors to cells taken from patients, by dosing cells kept in cultures with small amounts of the factors.

If dosed for a short enough time, the team reported, the cells retained their identity but returned to a youthful state, as judged by several measures of cell vigor.

Dr. Sebastiano said the Yamanaka factors appeared to operate in two stages, as if they were raising the epigenomes energy to one level, at which the marks of aging were lost, and then to a higher level at which cell identity was erased.

The Stanford team extracted aged cartilage cells from patients with osteoarthritis and found that after a low dosage of Yamanaka factors the cells no longer secreted the inflammatory factors that provoke the disease. The team also found that human muscle stem cells, which are impaired in a muscle-wasting disease, could be restored to youth. Members of the Stanford team have formed a company, Turn Biotechnologies, to develop therapies for osteoarthritis and other diseases.

The study is definitively a step forward in the goal of reversing cellular aging, Dr. Izpisua Belmonte said.

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Turning Back the Clock on Aging Cells - The New York Times

Skin Stem Cells Comments Off on Turning Back the Clock on Aging Cells – The New York Times | March 31st, 2020

Guy Rouleau, shown at middle in 2010, is director of the Montreal Neurological Institute and Hospital and one of the recipients of this year's Gairdner Awards. The others are Mina Bissell, top left; Salim and Quarraisha Abdool Karim, middle left; Elaine Fuchs, bottom left; Rolf Kemier, top right; Masatoshi Takeichi, middle right; and Roeland Nusse, bottom right.

John Morstad/The Globe and Mail, handouts

A diverse group of eight scientists whose work has offered insight into how cells interact with each other and their environment, the genetic underpinnings of neurological disease and the transmission of the virus that causes AIDS, have been named this years winners of the Gairdner Awards the countrys most prestigious biomedical research prizes.

Coming in the midst of the COVID-19 pandemic, this years set of awards highlights the importance of basic science to understanding the fundamental processes of life and how those processes relate to human health around the world.

When you build up a scientific environment and a scientific community, you have people who are prepared to do whatever it takes [to address a global health crisis], said Janet Rossant, president and scientific director of the Toronto-based Gairdner Foundation, which announced the award winners on Tuesday. You never know whats going to give you insight into disease, Dr. Rossant said.

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Guy Rouleau works in a Montreal lab in 2017.

Paul Chiasson/The Canadian Press

Among the winners is Guy Rouleau, director of the Montreal Neurological Institute and Hospital, who is this years recipient of the Gairdner Wightman Award, which recognizes scientific leadership in Canada.

In addition to his work linking various rare genes that occur in the French Canadian population to disorders such as ALS (Amyotrophic Lateral Sclerosis), Dr. Rouleau is known for his efforts to make scientific research more accessible.

Starting in 2016, he placed his institute at the forefront of the open science movement by allowing the free flow of data, tools and research results without restrictions related to who will profit from the knowledge.

Reached at his home in Montreal, Dr. Rouleau said the initiative was spurred by a lack of new development in neurological disease, where few treatment options are available for those dealing with brain disorders including Alzheimers disease. We thought that by sharing openly and by breaking down barriers, this would accelerate things, Dr. Rouleau said.

A human T-cell, in blue, comes under attack by HIV, in yellow, the virus that causes AIDS.

Seth Pincus, Elizabeth Fischer, Austin Athman/National Institute of Allergy and Infectious Diseases/NIH via AP

The husband and wife team of Quarraisha and Salim Abdool Karim at the Centre for the AIDS Programme of Research in South Africa were named the joint winners of the John Dirks Gairdner Globe Health award for their work tackling HIV in Africa.

In 1990, the pair described the transmission of the virus that causes AIDS through the African population, which frequently involves the infection of teenage girls by older men. Their work laid the foundations for successful HIV prevention programs focused on women and womens health.

Previous winners of the global health award include Anthony Fauci of the National Institutes of Health in Bethesda, Md., who has lately become a prominent figure for helping to steer the U.S. response to COVID-19 and for repeatedly clarifying or correcting misleading statements by U.S. President Donald Trump.

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Past Gairdner recipient Dr. Anthony Fauci walks past U.S. President Donald Trump at a March 29 news conference at the White House.

Al Drago/Reuters

The five researchers named as recipients of this years Canada Gairdner International Award a prize that often portends a future Nobel win have all done groundbreaking work related to some aspect of the field known as cell signalling.

They include:

In previous years, Gairdner award winners have travelled across Canada giving lectures and meeting with students ahead of a fall symposium and award ceremony in Toronto.

Dr. Rossant said the Foundation is still assessing how this years activities will proceed in light of COVID-19.

Sign up for the Coronavirus Update newsletter to read the days essential coronavirus news, features and explainers written by Globe reporters.

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In a coronavirus crisis, Gairdner Awards honour eight explorers of how cells, genes and viruses work - The Globe and Mail

Skin Stem Cells Comments Off on In a coronavirus crisis, Gairdner Awards honour eight explorers of how cells, genes and viruses work – The Globe and Mail | March 31st, 2020