GAITHERSBURG, Md., Oct. 24, 2019 /PRNewswire/ --MaxCyte, the global cell-based therapies and life sciences company, announces today that, having completed dosing of the second cohort of patients, clinical investigators have initiated dosing in the third cohort of patients of MaxCyte's Phase I clinical trial with the next higher cell dose of MCY-M11. This lead, wholly-owned, non-viral mRNA-based cell therapy candidate from MaxCyte's CARMA platform is a mesothelin-targeting chimeric antigen receptor (CAR) therapy being tested in individuals with relapsed/refractory ovarian cancer and peritoneal mesothelioma.

The dose escalation trial is evaluating the safety and tolerability, as well as preliminary efficacy, of MCY-M11 administered intraperitoneally across a series of ascending dose-level cohorts. In the first two cohorts, the infusion of MCY-M11 has been well tolerated in all patients treated. No dose-limiting toxicities, infusion-related adverse events, on-target or off-target toxicities, or other unwanted events were observed.

"We are making significant progress with our lead CAR therapeutic and our proprietary CARMA autologous cell therapy platform. Furthermore, the on-going trial continues to demonstrate the feasibility of our one-day cell therapy manufacturing process," said Claudio Dansky Ullmann, MD, Chief Medical Officer. "We are very excited about the potential of MCY-M11 as a new, effective therapeutic in solid tumors where the majority of patients still have very limited treatment options."

About the Phase I Clinical TrialThe multi-center, non-randomized, open label, dose-escalation Phase I clinical trial is evaluating the safety and preliminary efficacy of intraperitoneal infusions of MCY-M11 in individuals with platinum-resistant, high-grade, serous adenocarcinoma of the ovary, primary peritoneum or fallopian tube, or individuals with advanced peritoneal mesothelioma with recurrence after prior chemotherapy. MaxCyte anticipates approximately 15 study participants will be enrolled across the two clinical sites participating in the study (the National Cancer Institute (NCI) at the National Institutes of Health (NIH) and Washington University at St. Louis (WUSTL)). More information about the study can be found at ClinicalTrials.gov.

About the CARMA PlatformCARMA is MaxCyte's clinical-stage, non-viral, mRNA-based cell therapy platform that allows for the transfection of mRNA into cells and provides a simple, rapid-to-manufacture, dose-controllable product. CARMA requires less than one day for manufacture therapies for patients, where existing CAR-T therapies require one to two weeks or more to manufacture. MaxCyte's wholly-owned lead CARMA candidate, MCY-M11, is currently being evaluated in a Phase I clinical trial in patients with advanced ovarian cancer and peritoneal mesothelioma. MaxCyte management is evaluating independent sources of financing for CARMA. More information on the CARMA platform and pipeline is available at http://www.maxcyte.com/car/.

About MaxCyte MaxCyte is a clinical-stage global cell-based therapies and life sciences company applying its proprietary cell engineering platform to deliver the advances of cell-based medicine to patients with high unmet medical needs. MaxCyte is developing novel CARMA therapies for its own pipeline, with its first drug candidate in a Phase I clinical trial. CARMA is MaxCyte's mRNA-based proprietary therapeutic platform for autologous cell therapy for the treatment of solid cancers. In addition, through its life sciences business, MaxCyte leverages its Flow Electroporation Technology to enable its biopharmaceutical partners to advance the development of innovative medicines, particularly in cell therapy. MaxCyte has placed its flow electroporation instruments worldwide, including with all of the top ten global biopharmaceutical companies. The Company now has more than 80 partnered programme licenses in cell therapy with more than 45 licensed for clinical use, including six commercial licenses. With its robust delivery technology platform, MaxCyte helps its partners to unlock the full potential of their products. For more information, visit http://www.maxcyte.com.

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MaxCyte Advances Phase I Clinical Trial of Lead CARMA(TM) mRNA-based Cell Therapy to Third Cohort of Patients - Herald-Mail Media

Report Scope: The scope of this report is broad and covers various type of product available in the stem cell and regenerative medicines market and potential application sectors across various industries.

New York, Oct. 24, 2019 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Stem Cell and Regenerative Therapy Market" - https://www.reportlinker.com/p05791357/?utm_source=GNW The current report offers a detailed analysis of the stem cell and regenerative medicines market.

The report highlights the current and future market potential of stem cell and regenerative medicines and provides a detailed analysis of the competitive environment, recent development, merger and acquisition, drivers, restraints, and technology background in the market. The report also covers market projections through 2024.

The report details market shares of stem cell and regenerative medicines based on products, application, and geography.Based on product the market is segmented into therapeutic products, cell banking, tools and reagents.

The therapeutics products segments include cell therapy, tissue engineering and gene therapy. By application, the market is segmented into oncology, cardiovascular disorders, dermatology, orthopedic applications, central nervous system disorders, diabetes, others

The market is segmented by geography into the following regions: North America, Europe, Asia-Pacific, South America, and the Middle East and Africa. The report presents detailed analyses of major countries such as the U.S., Canada, Mexico, Germany, the U.K. France, Japan, China and India. For market estimates, data is provided for 2018 as the base year, with forecasts for 2019 through 2024. Estimated values are based on product manufacturers total revenues. Projected and forecasted revenue values are in constant U.S. dollars, unadjusted for inflation.

Report Includes: - 28 data tables - An overview of global markets for stem cell and regenerative medicines - Analyses of global market trends, with data from 2018, estimates for 2019, and projections of compound annual growth rates (CAGRs) through 2024 - Details of historic background and description of embryonic and adult stem cells - Information on stem cell banking and stem cell research - A look at the growing research & development activities in regenerative medicine - Coverage of ethical issues in stem cell research & regulatory constraints on biopharmaceuticals - Comprehensive company profiles of key players in the market, including Aldagen Inc., Caladrius Biosciences Inc., Daiichi Sankyo Co. Ltd., Gamida Cell Ltd. and Novartis AG

Summary The global market for stem cell and regenerative medicines was valued at REDACTED billion in 2018.The market is expected to grow at a compound annual growth rate (CAGR) of REDACTED to reach approximately REDACTED billion by 2024.

Growth of the global market is attributed to the factors such as growingprevalence of cancer, technological advancement in product, growing adoption of novel therapeuticssuch as cell therapy, gene therapy in treatment of chronic diseases and increasing investment fromprivate players in cell-based therapies.

In the global market, North America held the highest market share in 2018.The Asia-Pacific region is anticipated to grow at the highest CAGR during the forecast period.

The growing government funding for regenerative medicines in research institutes along with the growing number of clinical trials based on cell-based therapy and investment in R&D activities is expected to supplement the growth of the stem cell and regenerative market in Asia-Pacific region during the forecast period.

Reasons for Doing This Study Global stem cell and regenerative medicines market comprises of various products for novel therapeutics that are adopted across various applications.New advancement and product launches have influenced the stem cell and regenerative medicines market and it is expected to grow in the near future.

The biopharmaceutical companies are investing significantly in cell-based therapeutics.The government organizations are funding research and development activities related to stem cell research.

These factors are impacting the stem cell and regenerative medicines market positively and augmenting the demand of stem cell and regenerative therapy among different application segments.The market is impacted through adoption of stem cell therapy.

The key players in the market are investing in development of innovative products. The stem cell therapy market is likely to grow during the forecast period owing to growing investment from private companies, increasing in regulatory approval of stem cell-based therapeutics for treatment of chronic diseases and growth in commercial applications of regenerative medicine.

Products based on stem cells do not yet form an established market, but unlike some other potential applications of bioscience, stem cell technology has already produced many significant products in important therapeutic areas. The potential scope of the stem cell market is now becoming clear, and it is appropriate to review the technology, see its current practical applications, evaluate the participating companies and look to its future.

The report provides the reader with a background on stem cell and regenerative therapy, analyzes the current factors influencing the market, provides decision-makers the tools that inform decisions about expansion and penetration in this market.Read the full report: https://www.reportlinker.com/p05791357/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

__________________________

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Clare: clare@reportlinker.comUS: (339)-368-6001Intl: +1 339-368-6001

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Global Stem Cell and Regenerative Therapy Market - Yahoo Finance

NEW YORK, Oct. 24, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, today announced, Chaim Lebovits, President and CEO, will serve as a Keynote Speaker at Cell Series UK.Cell Series UK, will be held October 29-30, 2019, at London Novotel West, London, UK. The Conference, organized by Oxford Global, is one of the foremost events in Europe focused on regenerative medicine and cellular innovation.

Ralph Kern MD, MHSc, Chief Operating and Chief Medical Officer of Brainstorm, who will also participate at Cell Series UK stated, We are very pleased to have Chaim Lebovits presenting at this prestigious conference where global leaders in stem cell and regenerative medicine will have the opportunity to learn more about NurOwn and the critical research being conducted by the Company. Mr. Lebovits Keynote Address, Stem Cell Therapeutic Approaches For ALS, will be presented to leading members of the scientific and business community including potential partners and investors.

About NurOwnNurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

AboutBrainStorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn Cellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm received U.S. FDA clearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) in December 2018 and has been enrolling clinical trial participants since March 2019. For more information, visit the company's website.

Safe-Harbor Statements Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PR Phone: +1.646.677.1839sean.leous@icrinc.com

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BrainStorm Cell Therapeutics' President and CEO to be Featured as Keynote Speaker at Cell Series UK 2019 - GlobeNewswire

Its Nobel Prize season! In the biological sciences, the prestigious prizes are often awarded for discoveries that unlock lifes most basic mechanisms. Nobel-prize-winning work often invokes concepts so fundamental that most people take them for granted. This years Nobel Prize for Physiology and Medicine is no exception. Awarded to William Kaelin Jr, Sir Peter Ratcliffe, and Gregg L. Semenza, the 2019 prize honors the discovery of how cells adapt to the presence of oxygen.

In a 2007 Science article, one of the winners, Gregg Semenza, describes portions of the groundbreaking work. The major issue, as Semenza describes it, is that while many of lifes essential functions depend on oxygen, the amount available is not always consistent. For example, oxygen availability has not been consistent throughout evolutionary history. Early organisms began utilizing oxygen for energy production in increasing degrees, starting around 2.5 billion years ago, about a billion years after life began. After photosynthetic organisms that produced oxygen as a byproduct emerged, the atmospheric oxygen concentration increased dramatically.

Early life, and the cells of more complicated organisms, had to get used to this newly abundant resource. Non-photosynthetic organisms became dependent on oxidative phosphorylation, a means by which the energy of sugar is unlocked for use by cells. Within the cell, in the face of changing external conditions, the amount of available oxygen must be relatively constant. Too little, and it will be impossible to generate energy; too much, and potentially deadly waste products can build up.

Enter HIF-1, aka Hypoxia Induced Factor 1, a protein known as a transcription factor. Transcription factors control the conditions under which a specific gene or genes is expressed, and HIF-1 controls hundreds, perhaps thousands, of genes. The amount of HIF-1 available varies depending on the available oxygen, increasing or decreasing how often the suite of genes controlled by HIF-1 gets expressed.

Semenza and his colleagues teased out the details of the incredibly complicated mechanism by which HIF-1 functions to maintain constant oxygen levels. There are more than a dozen steps, and related biochemical components, in the process. They have found versions of the HIF-1 process in much simpler animals, such as roundworms and flies, helping them trace the evolutionary origins of the adaptation to oxygen.

The work was doubly significant. Not only did the team come to a detailed understanding of one of lifes key processes, the discovery has great potential in medicine as well. Some of the most common cardiovascular diseases, such as stroke or heart attack, restrict the oxygen supply to cells. If a medication or procedure can be devised to control how cells respond to the drop in oxygen, it may be possible to prevent or even reverse the damage from cardiovascular disease. The mechanism may even be manipulated to limit the growth of tumors. Congratulations to doctors Kaelin, Ratcliffe, and Semenza!

JSTOR is a digital library for scholars, researchers, and students. JSTOR Daily readers can access the original research behind our articles for free on JSTOR.

By: Gregg L. Semenza

Science, New Series, Vol. 318, No. 5847 (Oct. 5, 2007), pp. 62-64

American Association for the Advancement of Science

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What Did the Winners of the Nobel Prize in Medicine Discover? - JSTOR Daily

News Release

Thursday, October 24, 2019

Researchers have identified 57 genetic variations ofagenestrongly associated withdeclinesinbloodoxygen levelsduring sleep. Low oxygen levels during sleep are a clinical indicator of the severity of sleep apnea, a disorder that increases the risk of heart disease, dementia, and death. The study, published today in theAmerican Journal of Human Genetics, was funded by the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health.

A persons average blood oxygen levels during sleep are hereditary, and relatively easy to measure, said study author Susan Redline, M.D., senior physician in the Division of Sleep and Circadian Disorders at Brigham and Womens Hospital, and professor at Harvard Medical School, Boston. Studying the genetic basis of this trait can help explain why some people are more susceptible to sleep disordered breathing and its related morbidities.

When we sleep, the oxygen level in our blood drops, due to interruptions in breathing. Lung and sleep disorders tend to decrease those levels further, and dangerously so. But the range of those levels during sleep varies widely between individuals and, researchers suspect, is greatly influenced by genetics.

Despite the key roleblood oxygen levelsplayin health outcomes,theinfluenceof genetics on theirvariabilityremains understudied. The current findings contribute toa betterunderstanding, particularly because researcherslookedat overnight measurementsof oxygen levels. Thoseprovide more variability than daytime levelsdue to the stressesassociated withdisordered breathing occurring during sleep.

The researchers analyzed whole genome sequence data from the NHLBIs Trans-Omics for Precision Medicine (TOPMed) program. Tostrengthenthe data,they incorporated results of family-basedlinkage analysis, a method for mapping genes that carry hereditary traits to their location in the genome. Themethod usesdata fromfamilies with several members affected by aparticulardisorder.

This study highlights theadvantage of using family data in searching for rare variants, which is often missed in genome-wide association studies, said James Kiley, Ph.D., director of the Division of Lung Diseases at NHLBI. It showed that, when guided by family linkage data, whole genome sequence analysis can identify rare variants that signal disease risks, even with a small sample. In this case, the initial discovery was done with fewer than 500 samples.

The newly identified 57 variants of the DLC1 gene were clearly associated with the fluctuation in oxygen levels during sleep. In fact, they explained almost 1% ofthevariability in the oxygen levels in European Americans, which is relatively high for complex genetic phenotypes, or traits, that are influenced by myriad variants.

Notably,51 of the 57genetic variantsinfluence and regulate human lung fibroblast cells, a type of cell producing scar tissue in the lungs, according to study author XiaofengZhu, Ph.D., professor at the Case Western Reserve University School of Medicine, Cleveland.

This is important becauseMendelian Randomization analysis, a statistical approach for testing causal relationship between an exposure and an outcome, shows a potential causal relationship between how the DLC1 gene modifies fibroblasts cells andthechanges in oxygen levels during sleep, he said.

Thisrelationship,Kileyadded,suggests thata shared molecular pathway, or a common mechanism,may beinfluencing a persons susceptibility to the lack of oxygen caused by sleep disordered breathingand other lung illnesses such as emphysema.

The project was jointly led by Zhu and Redline, who also directs the National Sleep Research Resource, supported by NHLBI.

About theNational Heart, Lung, and Blood Institute (NHLBI): NHLBI is the global leader in conducting and supporting research in heart, lung, and blood diseases and sleep disorders that advances scientific knowledge, improves public health, and saves lives. For more information, visithttps://www.nhlbi.nih.gov.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

Sequencing analysis at 8p23 identifies multiple rare variants in DLC1 associated with sleep related oxyhemoglobin saturation level.

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More:
Researchers identify genetic variations linked to oxygen drops during sleep - National Institutes of Health

A post shared on Facebook makes a disturbing claim that many popular food and drink companies use "aborted baby fetus cells" to enhance the flavor of their products.

The post features text around a collage of popular food and drink items such as Pepsi, Doritos, Lays, Fritos, Aunt Jemima and Gatorade. It says:

"If only sheeple knew .. that theres a flavor enhancement company called senomyx that puts aborted baby fetus cells in their food and drinks."

The post also has a lengthy caption that appears to be taken entirely from a 2015 blog post by Rich Swier, in which Swier includes this excerpt from a Conservative Post story:

"Kraft, PepsiCo, Nestle, work with Semonyx, a California-based [company] that uses aborted embryonic cells to test fake flavoring chemicals. The aborted human fetal cell line is known as HEK-293, and it is used to see how the human palate will react to synthetic flavors. Since most of todays processed food lacks flavor, companies like Semonyx are hired to develop flavors on their own..."

Swier, according to the blog, holds a doctorate of education from the University of Southern California.

The post was flagged as part of Facebooks efforts to combat false news and misinformation on its News Feed. (Read more about our partnership with Facebook.)

Lets just start off by saying that no neither Kraft nor Pepsi nor any other U.S. food company is selling items to the public that contain "aborted baby fetus cells."

The Conservative Post story that Swiers blog and the Facebook post reference no longer exists on the website, but we traced the controversial claim back to a 2011 dispute involving Senomyx, a San Diego-based biotechnology company, and a pro-life group called Children of God for Life.

According to an archived version of a March 29, 2011, press release from Children of God for Life, the group called for a boycott of food companies that contract with Senomyx. The organization pointed to an April 2002 report by Senomyx researchers as proof that the company was adding HEK 293 Human Embryonic Kidney 293 cells in its research and development to enhance flavor.

HEK 293 is a line of cells originally derived from human embryo kidney cells and that were grown in a tissue culture. The first source of the cells was a fetus that was legally aborted in the Netherlands in the 1970s. The cell line has been widely used in biological and medical inquiry, especially for cancer research.

Fetal stem cell research has been used in cell biology for over 30 years. But no company is manufacturing food or other products intended for human consumption that contain aborted human fetuses.

Senomyx has used the HEK 293 cell line in its flavor research to function as the mouths taste receptor cells, allowing the company to test hundreds of substances. But these cells are not in any of the actual food products that consumers would find on the market. CBS News wrote about this in 2011:

"To non-scientists this may sound a bit strange, but the reality is that HEK 293 cells are widely used in pharmaceutical research, helping scientists create vaccines as well as drugs like those for rheumatoid arthritis. The difference here is that Senomyx's work for Pepsi is one of the first times the cells have (potentially) been used to create a food or beverage. (And it's important to note that no part of a human kidney cell are ever a part of Senomyx's taste enhancers or any finished food products.)"

Gwen Rosenberg, vice president of investor relations and corporate communications for Senomyx, described the process to the Miami New Times during the 2011 controversy and said the process is "basically a robotic tasting system":

"(Rosenberg) depicted rows of little plastic square dishes with hundreds of tiny indentations in each dish. A protein is placed in each indentation, then a flavor. If the protein reacts to the flavor, the results are charted. If the new flavor (of which the company has more than 800,000) is successful with the protein test, the company then conducts taste tests with (live) adult humans."

Science and medicine writer Matthew Herper also broke down the process in a 2012 Forbes article:

"This is 35-year-old technology. And it is widely used in cell biology. And there is no way you'll consume them or that the cells would cause any health problems.

"... The kidney cells were forced to take up bits of DNA using a technique invented in 1973 that used a calcium solution. The resulting cells don't act much like human cells at all, but they are very easy to work with and have become workhorses of cellular biology. That's why they're used in the development of drugs and vaccines. (Here's the original paper on the creation of the HEK cells.) No new fetal tissue has been used to keep the cell culture going; the use of this cell line isn't leading to new abortion."

Our ruling

A Facebook post claims several popular food companies add "aborted baby fetus cells in their food and drinks" for flavor enhancement.

The post mischaracterizes the use fetal stem cell research by biotechnology companies such as Senomyx. HEK 293 cells, a cell line from an aborted fetus from the 1970s, has been widely used in cell biology research for over 30 years in multiple areas, including food and pharmaceutical development. But none of these cells are found in the food products available to consumers.

So, while there is a back-story associated with this post, the claim itself is too inaccurate to rate it anything but False.

Read more here:
No, food companies are not selling products that contain 'aborted fetus cells' - PolitiFact

LONDON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced an access agreement with NHS England for all currently licensed Vertex cystic fibrosis (CF) medicines and any future indications of these medicines.

This means that within 30 days patients with CF in England ages 2 years and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene can be prescribed ORKAMBI (lumacaftor/ivacaftor) by their doctor and CF patients ages 12 years and older who either have two copies of the F508del mutation or one copy of the F508del mutation and a copy of one of the other 14 licensed mutations can be prescribed SYMKEVI (tezacaftor/ivacaftor) in combination with ivacaftor.

The agreement also offers expanded access to KALYDECO (ivacaftor) to include people ages 18 years and older who have the R117H mutation and those patients ages 12 months and older who have one of the nine licensed gating mutations.

Today is a significant day for the cystic fibrosis community in England. This important agreement, reached in collaboration and partnership with NHS England and NICE, will allow more than 5,000 eligible cystic fibrosis patients in England to have access to CFTR modulators to treat the underlying cause of their disease, said Ludovic Fenaux, Senior Vice President, Vertex International.

In addition to England, reimbursement agreements have also recently been announced in Scotland, Australia and Spain.

About CF in the UKOver 10,000 people in the UK have CF the second highest number in the world. Over 8,000 people in England have CF. CF is a debilitating, life-shortening inherited condition that causes progressive damage to organs across the body from birth. Currently, there is no cure for CF and half of people in the UK with CF die before they are 32. The daily impact of treatment is significant. It can take up to four or more hours involving, nebulizers, physiotherapy and up to 70 tablets a day. CF accounts for 9,500 hospital admissions and over 100,000 hospital bed days a year. A third of these are used by children under 15.

About ORKAMBI (lumacaftor/ivacaftor) and the F508del mutationIn people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface. Patients with two copies of the F508del mutation are easily identified by a simple genetic test.

Lumacaftor/ivacaftor is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface.

For complete product information, please see the Summary of Product Characteristics that can be found on http://www.ema.europa.eu.

About SYMKEVI (tezacaftor/ivacaftor) in combination with ivacaftorSome mutations result in CFTR protein that is not processed or folded normally within the cell, and that generally does not reach the cell surface. Tezacaftor is designed to address the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface and ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface.

SYMKEVI is indicated for people with CF ages 12 and older who either have two copies of the F508del mutation or one copy of the F508del mutation and have one of the following 14 mutations in which the CFTR protein shows residual function: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3AG, S945L, S977F, R1070W, D1152H, 2789+5GA, 3272-26AG, or 3849+10kbCT.

For complete product information, please see the Summary of Product Characteristics that can be found on http://www.ema.europa.eu.

About KALYDECO (ivacaftor)KALYDECO (ivacaftor) is the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.

KALYDECO is indicated in people ages 12 months and older who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. KALYDECO is also indicated for the treatment of patients with CF ages 18 years and older who have an R117H mutation in the CFTR gene.

For complete product information, please see the Summary of Product Characteristics that can be found on http://www.ema.europa.eu.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has four approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational medicines in other serious diseases where it has deep insight into causal human biology, such as sickle cell disease, beta thalassemia, pain, alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy and APOL1-mediated kidney diseases.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including nine consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes.

Special Note Regarding Forward-looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the statements regarding our expectations for the patient populations that will be able to access Vertexs medicines and the timing of such access. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

Read more:
Vertex Announces Agreement with NHS England for Access to All Licensed Cystic Fibrosis Medicines - Business Wire

A high-salt diet may negatively affect cognitive function by causing a deficiency of the compound nitric oxide, which is vital for maintaining vascular health in the brain, according to a new study in mice fromWeill Cornell Medicineresearchers.

When nitric oxide levels are too low, chemical changes to the protein tau occur in the brain, contributing to dementia. In the study,published Oct. 23 in Nature, the investigators sought to understand the series of events that occur between salt consumption and poor cognition and concluded that lowering salt intake and maintaining healthy blood vessels in the brain may stave off dementia.

Accumulation of tau deposits has been implicated in the development of Alzheimers disease in humans.

Our study proposes a new mechanism by which salt mediates cognitive impairment and also provides further evidence of a link between dietary habits and cognitive function, said lead study authorDr. Giuseppe Faraco, an assistant professor of research in neuroscience in the Feil Family Brain and Mind Research Institute at Weill Cornell Medicine.

The new study builds upon research published last year inNature Neuroscienceby Faraco and senior authorDr. Costantino Iadecola, director of theFeil Family Brain and MindResearch Institute and the Anne Parrish Titzell Professor of Neurologyat Weill Cornell Medicine.

The 2018 study found that a high-salt diet caused dementia in mice. The rodents became unable to complete daily living tasks such as building their nests, and had problems passing memory tests. The research team determined that the high-salt diet was causing cells in the small intestine to release the molecule interleukin-17 (IL-17), which promotes inflammation as part of the bodys immune response.

IL-17 then entered the bloodstream and prevented the cells in the walls of blood vessels feeding the brain from producing nitric oxide. This compound works by relaxing and widening the blood vessels, allowing blood to flow. Conversely, a shortage of nitric oxide can restrict blood flow.

Based on these findings, Iadecola, Faraco and their colleagues theorized that salt likely caused dementia in mice because it contributed to restricted blood flow to the brain, essentially starving it. However, as they continued their research, they realized that the restricted blood flow in mice was not severe enough to prevent the brain from functioning properly.

We thought maybe there was something else going on here, Iadecola said.

In their new study, the investigators found that decreased nitric oxide production in blood vessels affects the stability of tau proteins in neurons. Tau provides structure for the scaffolding of neurons. This scaffolding, also called the cytoskeleton, helps to transport materials and nutrients across neurons to support their function and health.

Tau becoming unstable and coming off the cytoskeleton causes trouble, Iadecola said, adding that tau is not supposed to be free in the cell. Once tau detaches from the cytoskeleton, the protein can accumulate in the brain, causing cognitive problems.

The researchers determined that healthy levels of nitric oxide keep tau in check. It puts the brakes on activity caused by a series of enzymes that leads to tau disease pathology, Iadecola said.

To further explore the importance of tau in dementia, the researchers gave mice with a high-salt diet and restricted blood flow to the brain an antibody to promote tau stability. Despite restricted blood flow, researchers observed normal cognition in these mice.

This demonstrated that's whats really causing the dementia was tau and not lack of blood flow, Dr. Iadecola said.

Although research on salt intake and cognition in humans is needed, the current mouse study is a reminder for people to regulate salt consumption, Iadecola said.

And the stuff that is bad for us doesnt come from a salt shaker it comes from processed food and restaurant food, he said. Weve got to keep salt in check. It can alter the blood vessels of the brain and do so in vicious way.

Heather Lindsey is a freelance writer for Weill Cornell Medicine.

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Study links high-salt diet and cognitive impairment - Cornell Chronicle