Category Archives: Cell Medicine

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

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.

Here is the original post:
Study links high-salt diet and cognitive impairment - Cornell Chronicle

Stem cell therapy for animals has seen breakthroughs

Stem cell therapy is increasingly becoming a more mainstream form of medicine. Usually applied to humans, the use of this regenerative treatment is now also being extended to animals including cats and dogs. Regenerative medicine, particularly stem cell treatment has seen many advancements in recent years with some groundbreaking studies coming to light.

Taking the cells from bone marrow, umbilical cords, blood or fat, stem cells can grow to become any kind of cell and the treatment has seen many successes in animals. The regenerative therapy has been useful particularly for treatment of spinal cord and bone injuries as well as problems with tendons, ligaments and joints.

Expanded Potential Stem Cells (EPSCs) have been obtained from pig embryos for the first time. The cells offer groundbreaking potential for studying embryonic development and producing transnational research in genomics and regenerative medicine, biotechnology and agriculture.

The cells have been efficiently derived from pig preimplantation embryos and a new culture medium developed in Hong Kong and Cambridge enabled researchers from the FLI to establish permanent embryonic stem cell lines. The cells have been discovered in a collaboration between research groups from the Institute of Farm Animal Genetics at the Friedrich-Loeffler-Institut (FLI) in Mariensee, Germany, the Wellcome Trust Sanger Institute in Cambridge, UK and the University of Hong Kong, Li Ka Shing Faculty of Medicine, School of Biomedical Sciences.

Embryonic stem cells (ESC) are derived from the inner cells of very early embryos, the so-called blastocysts. Embryonic stem cells are all-rounders and can develop into various cell types of the body in the culture dish. This characteristic is called pluripotency. Previous attempts to establish pluripotent embryonic stem cell lines from farm animals such as pigs or cattle have resulted in cell lines that have not really fulfilled all properties of pluripotency and were therefore called ES-like.

Dr Monika Nowak-Imialek of the FLI said: Our porcine EPSCs isolated from pig embryos are the first well-characterized cell lines worldwide. EPSCs great potential to develop into any type of cell provides important implications for developmental biology, regenerative medicine, organ transplantation, disease modelling and screening for drugs.

The stem cells can renew themselves meaning they can be kept in culture indefinitely, and also show the typical morphology and gene expression patterns of embryonic stem cells. Somatic cells have a limited lifespan, so these new stem cells are much better suited for long selection processes. It has been shown that these porcine stem cell lines can easily be modified with new genome editing techniques such as CRISPR/Cas, which is particularly interesting for the generation of porcine disease models.

The EPSCs have a high capacity to develop not only into numerous cell types of the organism, but also into extraembryonic tissue, the trophoblasts, making them very unique and lending them their name. This capacity could prove valuable for the future promising organoid technology, where organ-like small cell aggregations are grown in 3D aggregates that can be used for research into early embryo development, various disease models and testing of new drugs in petri dishes. In addition, the authors were able to show that trophoblast stem cells can be generated from their porcine stem cells, offering a unique possibility to investigate functions or diseases of the placenta in vitro.

A major hurdle to using neural stem cells derived from genetically different donors to replace damaged or destroyed tissues, such as in a spinal cord injury, has been the persistent rejection of the introduced material (cells), necessitating the use of complex drugs and techniques to suppress the hosts immune response.

Earlier this year, an international team led by scientists at University of California San Diego School of Medicine successfully grafted induced pluripotent stem cell (iPSC)-derived neural precursor cells back into the spinal cords of genetically identical adult pigs with no immunosuppression efforts. The grafted cells survived long-term, displayed differentiated functionality and caused no tumours.

The researchers also demonstrated that the same cells showed similar long-term survival in adult pigs with different genetic backgrounds after only short course use of immunosuppressive treatment once injected into injured spinal cord.

Senior author of the paper Martin Marsala, MD, professor in the Department of Anesthesiology at UC San Diego School of Medicine said: The promise of iPSCs is huge, but so too have been the challenges. In this study, weve demonstrated an alternate approach.

We took skin cells from an adult pig, an animal species with strong similarities to humans in spinal cord and central nervous system anatomy and function, reprogrammed them back to stem cells, then induced them to become neural precursor cells (NPCs), destined to become nerve cells. Because they are syngeneic genetically identical with the cell-graft recipient pig they are immunologically compatible. They grow and differentiate with no immunosuppression required.

Co-author Samuel Pfaff, PhD, professor and Howard Hughes Medical Institute Investigator at Salk Institute for Biological Studies, said: Using RNA sequencing and innovative bioinformatic methods to deconvolute the RNAs species-of-origin, the research team demonstrated that pig iPSC-derived neural precursors safely acquire the genetic characteristics of mature CNS tissue even after transplantation into rat brains.

NPCs were grafted into the spinal cords of syngeneic non-injured pigs with no immunosuppression finding that the cells survived and differentiated into neurons and supporting glial cells at all observed time points. The grafted neurons were detected functioning seven months after transplantation.

Then researchers grafted NPCs into genetically dissimilar pigs with chronic spinal cord injuries, followed by a transient four-week regimen of immunosuppression drugs again finding long-term cell survival and maturation.

Marsala continued: Our current experiments are focusing on generation and testing of clinical grade human iPSCs, which is the ultimate source of cells to be used in future clinical trials for treatment of spinal cord and central nervous system injuries in a syngeneic or allogeneic setting.

Because long-term post-grafting periods between one and two years are required to achieve a full grafted cells-induced treatment effect, the elimination of immunosuppressive treatment will substantially increase our chances in achieving more robust functional improvement in spinal trauma patients receiving iPSC-derived NPCs.

In our current clinical cell-replacement trials, immunosuppression is required to achieve the survival of allogeneic cell grafts. The elimination of immunosuppression requirement by using syngeneic cell grafts would represent a major step forward said co-author Joseph Ciacci, MD, a neurosurgeon at UC San Diego Health and professor of surgery at UC San Diego School of Medicine.

Other recent advancements include the advancement toward having a long-lasting repair caulk for blood vessels. A new method has been for generating endothelial cells, which make up the lining of blood vessels, from human induced pluripotent stem cells. When endothelial cells are surrounded by a supportive gel and implanted into mice with damaged blood vessels, they become part of the animals blood vessels, surviving for more than 10 months.

The research was carried out by stem cell researchers at Emory University School of Medicine and could form the basis of a treatment for peripheral artery disease, derived from a patients own cells.

Young-sup Yoon, MD, PhD, who led the team, said: We tried several different gels before finding the best one. This is the part that is my dream come true: the endothelial cells are really contributing to endogenous vessels.

When cells are implanted on their own, many of them die quickly, and the main therapeutic benefits are from growth factors they secrete. When these endothelial cells are delivered in a gel, they are protected. It takes several weeks for most of them to migrate to vessels and incorporate into them.

Other groups had done this type of thing before, but the main point is that all of the culture components we used would be compatible with clinical applications.

This research is particularly successful as previous attempts to achieve the same effect elsewhere had implanted cells lasting only a few days to weeks, using mostly adult stem cells, such as mesenchymal stem cells or endothelial progenitor cells. The scientists also designed a gel to mimic the supportive effects of the extracellular matrix. When encapsulated by the gel, cells could survive oxidative stress inflicted by hydrogen peroxide that killed unprotected cells. The gel is biodegradable, disappearing over the course of several weeks.

The scientists tested the effects of the encapsulated cells by injecting them into mice with hindlimb ischemia (restricted blood flow in the leg), a model of peripheral artery disease.

After 4 weeks, the density of blood vessels was highest in mice implanted with gel-encapsulated endothelial cells. The mice were nude, meaning genetically immunodeficient, facilitating acceptance of human cells.

The scientists found that implanted cells produce pro-angiogenic and vasculogenic growth factors. In addition, protection by the gel augmented and prolonged the cells ability to contribute directly to blood vessels. To visualise the implanted cells, they were labelled beforehand with a red dye, while functioning blood vessels were labelled by infusing a green dye into living animals. Implanted cells incorporated into vessels, with the highest degree of incorporation occurring at 10 months.

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Stem cell therapy is for animals too - SciTech Europa

Sixteen months ago, Bob Masterson began a Holy Name Medical Center clinical trial that he hoped would grow more arteries to restore blood flow to his legs sothe wounds on his toeswould heal. If it failed, he risked the possibility of amputation.

Masterson had aa 67% chance of getting the FDA-fast-tracked Pluristem Therapy placenta stem cell injections instead of getting a placebo.

In essence what were injecting is going to recruit the development of blood vessels, cells that are involved in the healing of wounds, said Dr. John Rundback, director of the Interventional Institute at the medical center.

When getting the procedure last year,Mastersonsays he had no idea whether he was getting the placebo or the real thing, but the results from regular visits to the hospital seem to indicate it was the latter.

Doctors want to improve National Institutes of Healthnumbers, whichfind half of all amputees die within five years, with new therapies that could come from the clinical trial of 246 patients at six hospitals.

Masterson says hes grateful for the results but doesnt consider the apparent reversal of his condition a miracle.

But, I see it as a progress in medicine,he said. Cause over my years, Ive seen a lot of people, especially with us who have diabetes, losing their limbs like it was nothing. And this gives them hope.

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Holy Name clinical trial tests the healing power of stem cells - NJTV News

Oct. 21, 2019 05:00 UTC

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the Phase III IMbrave150 study, evaluating Tecentriq (atezolizumab) in combination with Avastin (bevacizumab) as a treatment for people with unresectable hepatocellular carcinoma (HCC) who have not received prior systemic therapy, met both of its co-primary endpoints demonstrating statistically significant and clinically meaningful improvements in overall survival (OS) and progression-free survival (PFS) compared with standard-of-care sorafenib.

Safety for the combination of Tecentriq and Avastin was consistent with the known safety profiles of the individual medicines, with no new safety signals identified. Data from the IMbrave150 study will be presented at an upcoming medical meeting.

We are very pleased with the results of our study testing the combination of Tecentriq and Avastin, which marks the first treatment in more than a decade to improve overall survival in people with unresectable hepatocellular carcinoma who have not received prior systemic therapy, said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. HCC is a major cause of death globally and particularly in Asia, making this study an important step in our mission of addressing unmet medical needs for patients around the world. We will submit these data to global health authorities as soon as possible. Our hope is to bring a new treatment to people with this aggressive disease who currently have limited options.

In July 2018, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) for Tecentriq in combination with Avastin in HCC based on data from an ongoing Phase Ib trial.

Genentech has an extensive development program for Tecentriq, including multiple ongoing and planned Phase III studies, across lung, genitourinary, skin, breast, gastrointestinal, gynecological and head and neck cancers. This includes studies evaluating Tecentriq both alone and in combination with other medicines.

About the IMbrave150 study

IMbrave150 is a global Phase III, multicenter, open-label study of 501 people with unresectable HCC who have not received prior systemic therapy. People were randomized 2:1 to receive the combination of Tecentriq and Avastin or sorafenib. Tecentriq was administered intravenously, 1200 mg on day 1 of each 21-day cycle, and Avastin was administered intravenously, 15 mg/kg on day 1 of each 21-day cycle. Sorafenib was administered by mouth, 400 mg twice per day, on days 1-21 of each 21-day cycle. People received the combination or the control arm treatment until unacceptable toxicity or loss of clinical benefit as determined by the investigator. Co-primary endpoints were OS and PFS by independent review facility (IRF) per Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1). Secondary efficacy endpoints included overall response rate (ORR), time to progression (TTP) and duration of response (DoR), as measured by RECIST v1.1 (investigator-assessed [INV] and IRF) and HCC mRECIST (IRF), as well as patient-reported outcomes (PROs), safety and pharmacokinetics.

About hepatocellular carcinoma

According to the American Cancer Society, it is estimated that more than 42,000 Americans will be diagnosed with liver cancer in 2019. Liver cancer incidence has more than tripled since 1980 and HCC accounts for approximately 75% of all liver cancer cases in the United States. HCC develops predominantly in people with cirrhosis due to chronic hepatitis (B and C) or alcohol consumption, and typically presents at an advanced stage where there are limited treatment options.

About the Tecentriq and Avastin combination

There is a strong scientific rationale to support further investigation of Tecentriq plus Avastin in combination. Avastin, in addition to its anti-angiogenic effects, may further enhance Tecentriqs ability to restore anti-cancer immunity by inhibiting vascular endothelial growth factor (VEGF)-related immunosuppression, promoting T-cell tumor infiltration and enabling priming and activation of T-cell responses against tumor antigens.

About Tecentriq (atezolizumab)

Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1. Tecentriq is designed to bind to PD-L1 expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the re-activation of T cells. Tecentriq may also affect normal cells.

About Avastin (bevacizumab)

Avastin is a prescription-only medicine that is a solution for intravenous infusion. It is a biologic antibody designed to specifically bind to a protein called VEGF that plays an important role throughout the lifecycle of the tumor to develop and maintain blood vessels, a process known as angiogenesis. Avastin is designed to interfere with the tumor blood supply by directly binding to the VEGF protein to prevent interactions with receptors on blood vessel cells. The tumor blood supply is thought to be critical to a tumors ability to grow and spread in the body (metastasize).

Tecentriq U.S. Indications

Tecentriq is a prescription medicine used to treat adults with:

A type of bladder and urinary tract cancer called urothelial carcinoma. Tecentriq may be used when your bladder cancer:

The approval of Tecentriq in these patients is based on a study that measured response rate and duration of response. Continued approval for this use may depend on the results of an ongoing study to confirm benefit.

A type of lung cancer called non-small cell lung cancer (NSCLC).

A type of breast cancer called triple-negative breast cancer (TNBC).

Tecentriq may be used with the medicine paclitaxel protein-bound when your breast cancer:

The approval of Tecentriq in these patients is based on a study that measured the amount of time until patients disease worsened. Continued approval for this use may depend on results of an ongoing study to confirm benefit.

A type of lung cancer called small cell lung cancer (SCLC).

It is not known if Tecentriq is safe and effective in children.

Important Safety Information

What is the most important information about Tecentriq?

Tecentriq can cause the immune system to attack normal organs and tissues and can affect the way they work. These problems can sometimes become serious or life threatening and can lead to death.

Patients should call or see their healthcare provider right away if they get any symptoms of the following problems or these symptoms get worse.

Tecentriq can cause serious side effects, including:

Getting medical treatment right away may help keep these problems from becoming more serious. A healthcare provider may treat patients with corticosteroid or hormone replacement medicines. A healthcare provider may delay or completely stop treatment with Tecentriq if patients have severe side effects.

Before receiving Tecentriq, patients should tell their healthcare provider about all of their medical conditions, including if they:

Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of Tecentriq when used alone include:

The most common side effects of Tecentriq when used in lung cancer with other anti-cancer medicines include:

The most common side effects of Tecentriq when used in triple-negative breast cancer with paclitaxel protein-bound include:

Tecentriq may cause fertility problems in females, which may affect the ability to have children. Patients should talk to their healthcare provider if they have concerns about fertility.

These are not all the possible side effects of Tecentriq. Patients should ask their healthcare provider or pharmacist for more information. Patients should call their doctor for medical advice about side effects.

Report side effects to the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch.

Report side effects to Genentech at 1-888-835-2555.

Please visit http://www.Tecentriq.com for the Tecentriq full Prescribing Information for additional Important Safety Information.

Avastin is approved for:

Avastin in combination with paclitaxel, pegylated liposomal doxorubicin or topotecan, is approved to treat platinum-resistant recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer (prOC) in women who received no more than two prior chemotherapy treatments.

Avastin, either in combination with carboplatin and paclitaxel or with carboplatin and gemcitabine, followed by Avastin alone, is approved for the treatment of patients with platinum-sensitive recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer (psOC)

Possible serious side effects

Everyone reacts differently to Avastin therapy. So, its important to know what the side effects are. Although some people may have a life-threatening side effect, most do not. Their doctor will stop treatment if any serious side effects occur. Patients should contact their health care team if there are any signs of these side effects.

Side effects seen most often

In clinical studies across different types of cancer, some patients experienced the following side effects:

Avastin is not for everyone

Patients should talk to their doctor if they are:

Patients should talk with their doctor if they have any questions about their condition or treatment.

Report side effects to the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch.

Report side effects to Genentech at 1-888-835-2555.

For full Prescribing Information and Boxed WARNINGS on Avastin please visit http://www.avastin.com.

About Genentech in personalized cancer immunotherapy

For more than 30 years, Genentech has been developing medicines with the goal to redefine treatment in oncology. Today, were investing more than ever to bring personalized cancer immunotherapy (PCI) to people with cancer. The goal of PCI is to provide each person with a treatment tailored to harness his or her own immune system to fight cancer. Genentech is currently studying more than 10 cancer immunotherapy medicines across 70 clinical trials alone or in combination with other medicines. In every study we are evaluating biomarkers to identify which people may be appropriate candidates for our medicines. For more information visit http://www.gene.com/cancer-immunotherapy.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.

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Genentech's Tecentriq in Combination With Avastin Increased Overall Survival and Progression-free Survival in People With Unresectable Hepatocellular...