StemCell Therapy

A womans genetic background can powerfully modifiy her cancer risk from a BRCA1 mutation.

By Jocelyn KaiserDec. 17, 2019 , 4:00 PM

Women who learn that they have a mutation in the breast cancer gene BRCA1 face a wrenching decision. Their doctor or genetic counselor will likely tell them that women with such mutations have, on average, a 72% lifetime risk of breast cancer and a 44% risk of ovarian cancer. Given that, up to half decide to have prophylactic mastectomies, and many have ovaries removed, too.

But recent studies show a woman could receive a more individualized, accurate cancer risk estimate by factoring in other gene variants. A preprint posted last month finds that a person's "polygenic" background influences not only the disease risk conferred by a BRCA1 defect, but also risks from single gene mutations linked to colorectal cancer and heart disease. Some individuals were very likely to develop cancer or heart disease by age 75, the analysis showed, whereas in others the risk was not much greater than in a person without the high-risk mutation.

"It's pretty striking," says cardiologist and geneticist Amit Khera of Massachusetts General Hospital (MGH) in Boston, leader of the study, which is on the medRxiv preprint server. "It's become clear that there are both monogenic and polygenic [disease] drivers. The future is to assess both."

"The message is a very important one for patients and clinicians," says Teri Manolio of the National Human Genome Research Institute in Bethesda, Maryland. "Carriers of BRCA1 mutations or other pathogenic variants don't invariably develop disease, and genomics can be used to help parse carriers who are at lower risk." Others caution, however, that risk scores summing how dozens to thousands of other genetic variants interact with a single major disease gene aren't yet accurate enough to use in the clinic. The new paper "is teasing at the possibility, but there's a lot of work to be done," says Harvard University epidemiologist Peter Kraft.

MGH cardiology fellow Akl Fahed and others in Khera's group explored polygenic influences on the three important single-gene disorders in the United States: familial hypercholesterolemia, which leads to sky-high cholesterol levels and dramatically elevates risk of heart disease; Lynch syndrome, a flaw in DNA repair that brings a lifetime risk of colorectal cancer of about 60%; and inherited breast cancer, caused by variants in BRCA1 or BRCA2. They took advantage of databases that combine medical and genomic information from thousands of people, enabling researchers to tally how the many genetic variants with subtle effects modify disease risks and complex traits such as height.

Drawing on some 50,000 participants in the UK Biobank and 19,000 women tested for BRCA genes by the company Color Genomics, the team found that polygenic background strongly modified the risk of carrying a mutation in the key genes for the three disorders. For a small proportion of major disease gene carriers, other genetic variants boosted their overall risk of cancer or heart disease to about 80%, well above the average of 30% to 40% that Khera's group estimated for its study populations based on just the single disease gene mutations. (The team's monogenic disease risk predictions are lower than many other estimates for several possible reasons, Khera notes, including that the UK Biobank participants are healthier than the general population.) At the other extreme, the polygenic analysis suggested that a few people with those mutations have much lower risks than predicted by their single mutation alone, as low as 11% for colon cancer, 13% for breast cancer, and 17% for heart diseasenot much higher than other people in general.

Khera's group says adding polygenic data to single-gene tests could help people decide whether to take aggressive steps to head off diseasemastectomy or removal of the ovaries for women carrying BRCA mutations or frequent colonoscopies for people with Lynch syndrome. But the new study does not include enough data for clinical decisions, says genetic epidemiologist Antonis Antoniou of the University of Cambridge in the United Kingdom. Only 116 women in the UK Biobank sample had BRCA mutations, which he notes "is an extremely small number to make inferences about risks."

Two years ago, Antoniou led a study that reported on how polygenic scores influence risks in 25,000 carriers of BRCA mutations and found nearly as wide a range of overall cancer risks. His team has incorporated those data into a breast cancer risk estimator along with factors such as family history.

The MGH study is "an important and exciting paper" that complements other work, says David Ledbetter, chief scientific officer for the Geisinger Health System in Danville, Pennsylvania. His team recently looked at 92,000 participants in an ongoing genomic medicine study called MyCode, focusing on those who carried mutations predisposing them to 11 rare disorders that affect traits such as height, weight, and cholesterol levels. Incorporating polygenic scores helped predict those traits, the group reported on 25 October in Nature Communications.

It may be a while before physicians are comfortable telling patients how genetic backgrounds modify the risk posed by a major disease gene mutation. But some companies already offer polygenic scores for cancer and other diseases, and tests that combine both kinds of information are imminent. Before insurance companies agree to pay for such tests, Ledbetter cautions, "They're going to want to see much more clinical validation"including for minorities, because current polygenic analyses draw on data primarily from people of European ancestry.

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'Polygenic' profile could better predict disease risk for those with cancer mutations - Science Magazine

UNC Police is asking the public for help in their investigation of a series of credit card thefts fromdifferent medical research buildings on south campus earlier this month.

The department tweeted out photos on Tuesday morning, asking for helping identifying two people of interest in relation to the investigation.

According to an Alert Carolina post made on December 5, the thefts occurred during business hours on Wednesday December 4 inMacNider Hall, Beard Hall, the Bioinformatics Building and the Genetic Medicine Research Building among others. The post says credit cards were taken from unsecured offices and cubicles throughout the buildings.

Anyone seeing any suspicious activity anywhere on campus is reminded to call 911 immediately. UNC Police also encourage people to use smart security practices while in a work environment, like putting away visible valuables, keeping a record of all keys that have been issued and no admitting strangers into places of work.

If you have any information about the individuals, call the UNC Police Department at (919) 962-8100.

Related

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UNC Police Investigating Series of Credit Card Thefts on South Campus - Chapelboro.com

Researchers at Baylor College of Medicine report today in the journal Neuron evidence that refutes the link between increased levels of herpes virus and Alzheimers disease. In addition, the researchers provide a new statistical and computational framework for the analysis of large-scale sequencing data.

About 50 million people worldwide are affected by Alzheimers disease, a type of progressive dementia that results in the loss of memory, cognitive abilities and verbal skills, and the numbers are growing rapidly. Currently available medications temporarily ease the symptoms or slow the rate of decline, which maximizes the time patients can live and function independently. However, there are no treatments to halt progression of Alzheimers disease.

Like all types of dementia, Alzheimers disease is characterized by massive death of brain cells, the neurons. Identifying the reason why neurons begin and continue to die in the brains of Alzheimers disease patients is an active area of research, said corresponding author Dr. Zhandong Liu, associate professor of pediatrics at Baylor and the Jan and Dan Duncan Neurological Research Institute at Texas Childrens Hospital.

One theory that has gained traction in the past year is that certain microbial infections, such as those caused by viruses, can trigger Alzheimers disease. A 2018 study reported increased levels of human herpesvirus 6A (HHV-6A) and human herpesvirus 7 (HHV-7) in the postmortem brain tissues of more than 1,000 patients with Alzheimers disease when compared to the brain tissues of healthy-aging subjects or those suffering from a different neurodegenerative condition.

Presence of elevated levels of genetic material of herpes viruses indicated active infections, which were linked to Alzheimers disease. In less than a year, this study generated a flurry of excitement and led to the initiation of several studies to better understand the link between viral infections and Alzheimers disease.

Surprisingly, when co-author Dr. Hyun-Hwan Jeong, a postdoctoral fellow in Dr. Lius group and others, reanalyzed the data sets from the 2018 study using the identical statistical methods with rigorous filtering, as well as four commonly used statistical tools, they were unable to produce the same results.

The team was motivated to reanalyze the data from the previous study because they observed that while the p-values (a statistical parameter that predicts the probability of obtaining the observed results of a test, assuming that other conditions are correct) were highly significant, they were being ascribed to data in which the differences were not visually appreciable.

Moreover, the p-values did not fit with simple logistic regression a statistical analysis that predicts the outcome of the data as one of two defined states. In fact, after several types of rigorous statistical tests, they found no link between the abundance of herpes viral DNA or RNA and likelihood of Alzheimers disease in this cohort.

As high-throughput omics technologies, which include those for genomics, proteomics, metabolomics and others, become affordable and easily available, there is a rising trend toward big data in basic biomedical research. In these situations, given the massive amounts of data that have to be mined and extracted in a short time, researchers may be tempted to rely solely on p-values to interpret results and arrive at conclusions, Liu said.

Our study highlights one of the potential pitfalls of over-reliance on p-values. While p-values are a very valuable statistical parameter, they cannot be used as a stand-alone measure of statistical correlation data sets from high-throughput procedures still need to be carefully plotted to visualize the spread of the data, Jeong said. Data sets also have to be used in conjunction with accurately calculated p-values to make gene-disease associations that are statistically correct and biologically meaningful.

Our goal in pursuing and publishing this study was to generate tools and guidelines for big data analysis, so the scientific community can identify treatment strategies that will likely benefit patients, Liu said.

This study was funded by the Huffington Foundation.

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Link between herpes virus infections, Alzheimer's refuted - Baylor College of Medicine News

Drug Target Review lists its 10 most popular news stories from 2019, summarising the drug targets that you wanted to read about.

Drug Target Review has published a wide range of news stories this year, from the identification of novel drug targets to improvements in toxicology studies and developments in screening.

As the year draws to a close, we reflect on the biggest and most popular stories from 2019. To read the full pieces, click on the title of each news story.

A genetic analysis study revealed that variants of hundreds of genes work together in contributing to the development of Tourettes syndrome, in our tenth most popular story this year.

According to the researchers, from the Massachusetts General Hospital (MGH) and collaborators, their findings confirm that the underlying basis for Tourettes syndrome is polygenic, meaning that hundreds of small DNA changes cause the condition, rather than one inactive gene.

The scientists said their next step is to expand their sample size to around 12,000 patients, made possible with a potential international collaboration.

The study was published in the American Journal of Psychiatry.

A group of researchers identified new genetic targets on which BRCA2-driven cancer cells are dependent upon, providing a potential avenue for drug development.

The study, conducted at Brigham and Womens Hospital, used CRISPR and short-hairpin RNAs (shRNAs) to test 380 genes with a known or suspected role in DNA-damage response. This allowed the team to narrow in on the most promising genes: APEX2 and FEN1, two novel targets for breast cancer.

The results were published in Molecular Cell.

Immunotherapy treatment could reduce the persistence of HIV in patients receiving triple therapy, found a group of researchers.

The researchers, from the University of Montreal Hospital Research Centre, discovered that these therapies expose the virus to the immune system. Three proteins PD-1, LAG-3 and TIGIT were uncovered by the scientists as frequently expressed on the surface of HIV-hiding cells; these proteins are also cancer targets.

According to the team, their study could lead to the development of new HIV therapies based on cancer immunotherapies.

The study was published in Nature Communications.

Researchers at the Indiana University School of Medicine developed a blood test to measure pain and improve diagnosis. The team analysed hundreds of patient samples to reveal biomarkers in their blood, which could be used as a scale to determine pain.

According to the researchers, the biomarkers act like a signature that can be matched against a prescription database. This could allow medical professionals to select the appropriate compound and reduce pain for the patient.

The study was published in Molecular Psychiatry.

A team of scientists revealed that immune cells could be key in causing endometriosis, a pelvic pain experienced by women, through an investigation into macrophages. The study was led by researchers from Warwick Medical School and the University of Warwick.

Macrophages can adapt their function according to local signals from their surroundings and so become modified by disease. This led the researchers to add modified macrophages to a cell culture, which resulted in the production of higher levels of insulin-like growth factor-1 (IGF-1).

The team conclude that macrophages therefore present a drug target for endometriosis.

The results can be found in The FASEB Journal.

Scientists from the University of Pennsylvania imaged a molecule that induces inflammation and leads to lupus, in our fifth most popular story of 2019. The researchers discovered that the molecule is comprised of two sections: SHMT2 and BRISC, a cluster of proteins. When these two sections bind to each other, they cause inflammation.

When mice models lacking BRISC were tested, they were resistant to lupus. This led the team to conclude that a molecule which blocks BRISC and SHMT2 could be a drug target for lupus.

The findings were published in Nature.

A team of researchers reported that a CRISPR-Cas9 gene therapy which specifically reduces fat tissue and obesity-related metabolic disease was successful in mice.

The scientists, from Hanyang University, argue that their technique could be used as a way to combat type 2 diabetes and other obesity-related diseases.

Targeting Fabp4, a fatty acid metabolism gene, the researchers observed a 20 percent reduction of body weight in obese mice. It also resulted in improved insulin resistance after only six weeks of treatment.

The findings were published in Genome Research.

A compound that promotes the rebuilding of the protective sheath around nerve cells has been developed by researchers at the Oregon Health & Science University (OHSU).

The team found that the S3 compound reverses the effect of hyaluronic acid (HA) in mice. HA has been found to accumulate in the brain of patients with multiple sclerosis, and accumulation of HA

has also been linked to maturity failure of cells called oligodendrocytes, which generate myelin, the protective layer of axons.

The team therefore believe that the S3 compound could provide a therapeutic strategy for treating nervous system disorders.

The study can be found in Glia.

A group of researchers formed a complex view of the functional dysbiosis in the gut microbiome during inflammatory bowel disease (IBD), to reveal new targets for treatments.

The scientists, from theBroad InstituteofMITandHarvard University, observed microbial changes and human gene regulatory shifts from stool and blood samples of patients.

This multi-omic study enabled the team to discover that during periods of disease activity, IBD patients had higher levels of polyunsaturated fatty acids in both the blood and stool. They also identified other varying levels of nutrients and vitamins, presenting several potential drug targets.

The findings were published in Nature.

In our most popular news piece this year, researchers found that the small molecule PJ34 reduces the number of human pancreatic cancer cells in transplanted tumours by 90 percent.

The team, from Tel Aviv University, built on previous research to treat xenografts with their small molecule. It is permeable in the cell membrane, but affects human cancer cells exclusively, making it an attractive compound for development.

The scientists found that PJ34 causes a rapid cell death and in one mouse, the tumour completely disappeared. They concluded that the molecule could be a potent therapeutic against pancreatic cancer.

The results were published in Oncotarget.

Related organisationsBrigham and Women's Hospital, Hanyang University, Harvard University, Indiana University School of Medicine, Massachusetts General Hospital (MGH), MIT, Oregon Health & Science University (OHSU), Pennsylvania University, Tel Aviv University, University of Montreal Hospital Research Centre, Warwick Medical School, Warwick University

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DTR's news round-up 2019: the stories that defined the year - Drug Target Review

Spark will continue its operations in Philadelphia as an independent company within the Roche Group

Roche and Spark Therapeutics, Inc. have announced the completion of the acquisition following the receipt of regulatory approval from all government authorities required by the merger agreement.

Commenting on this important step forward, Severin Schwan, CEO of Roche, said, We are excited about this important milestone because we believe that together, Roche and Spark will be able to significantly improve the lives of patients through innovative gene therapies.This acquisition supports our long-lasting commitment to bringing transformational therapies and innovative approaches to people around the world with serious diseases.

Spark Therapeutics, based in Philadelphia, Pennsylvania, is a fully integrated, commercial company committed to discovering, developing and delivering gene therapies for genetic diseases, including blindness, haemophilia, lysosomal storage disorders and neurodegenerative diseases. Spark Therapeutics will continue to operate as an independent company within the Roche Group.

Today ushers in a new and promising era in the development of genetic medicines for patients and families living with inherited diseases and beyond, said Jeffrey D. Marrazzo, co-founder and CEO of Spark Therapeutics. Spark and Roche share an ethos of imagining the unimaginable. Together, we have the potential to change the future of medicine and deliver the medicines of tomorrow today. We couldnt be more thrilled about whats next.

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Roche acquires Spark Therapeutics to strengthen presence in gene therapy - BSI bureau

DUBLIN--(BUSINESS WIRE)--The "Innovations Transforming the Global Healthcare IT, Biomarker, Biologics, and Small Molecule Landscape" report has been added to ResearchAndMarkets.com's offering.

This edition of the Life Science, Health & Wellness TechVision Opportunity Engine (TOE) provides technological insights across 26 global healthcare innovations.

The technologies analyzed include advances in digital health, biologics, small molecules, medical imaging, dental caries and precision oncology platforms. The TOE also covers application and megatrends impact, apart from exclusive analyst insights for each innovation.

The Life Science, Health & Wellness TOE will feature disruptive technology advances in the global life sciences industry. The technologies and innovations profiled will encompass developments across genetic engineering, drug discovery and development, biomarkers, tissue engineering, synthetic biology, microbiome, disease management, as well as health and wellness among several other platforms.

The Health & Wellness cluster tracks developments in a myriad of areas including genetic engineering, regenerative medicine, drug discovery and development, nanomedicine, nutrition, cosmetic procedures, pain and disease management and therapies, drug delivery, personalized medicine, and smart healthcare.

Companies featured:

For more information about this report visit https://www.researchandmarkets.com/r/98ey4z

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Innovations Transforming the Global Healthcare IT, Biomarker, Biologics, and Small Molecule Landscape, 2019 Research Report - ResearchAndMarkets.com -...

Church was born in England and grew up in post-war London. As a child, she took to the water early.

I was a swimmer, Church said. I had a chance to train for the 1940 Olympic team but the war came along and they cancelled everything so that was the end of that.

She worked at Cambridge University during the second world war as a stenographer and met her future husband Harry, a Canadian soldier.

Harry returned to Canada in 1944, while Jessica followed two years later. The pair built a life in Thunder Bay Ont, before moving out west to Vancouver, Victoria and finally Parksville.

(Thunder Bay) was too darn cold so I said to my husband that we have to move down the coastits like English weather, Church said.

The two raised five children, a daughter and four sons. Church also worked for the Canadian government in a security and investigative role.

Church said the world has immeasurably changed in her time and was at wonder about the development of technology.

When we were in England when I was growing up, all we had was a radio, nothing else. No TV, no computers, no nothingjust a radio, Church said.

Church is a 17-year resident of Buttertubs Place and spends her time painting flowers and gardening.

She added the simplest advice she can give on living life to its fullest was to take it easy, take things in stride and dont get upset.

alex.rawnsley@jpbg.ca

On Twitter: @alexrawnsley

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100-years-young: Nanaimo woman reflects on lifetime of change - Nanaimo News NOW

For any farmer, judging their livestock is a key part to success. For Lydia Gosney, not only is it a skill that she has been working on since she was seven-years-old but it is also she is a part of the best team in the nation, and those two elements will send her to Scotland for international competition. She is a member of Kentuckys first 4-H Livestock Judging team to win the top spot at the North American International Livestock Exposition. Her teammates include Chevy Vaske (Grant County), Will Banks (Harrison County), and Kasey Johnson (Mason County). When Kentucky was called as National Champions, we jumped out of our seats, tears flowing and hugged each other so tight, recalled Gosney of that moment. I still get chills thinking about it and all of the raw emotion. While the team was having unparalled success, she was receiving individual honors, too. After placing in many areas, it came down to the overall awards. As the announcer got closer and closer to first place, he paused. I vividly remember the sound of his voice and the silence as he announced the top two individuals were tied. She was named Reserve National Champion. I was blessed and immediately started crying, not because I was upset about getting second, but because I had put my whole heart and soul into this for the past seven years, she explained. As a livestock judge, they evaluate animals and their genetics to determine their longevity in the herd and/or their carcass merit for slaughter. At the North American International contest, her team are to judge 11 classes of either sheep, goats, cattle or hogs. They answer 30 questions on three of those classes, as well. My favorite and most competitive category is Reasons, Gosney stated in explaining the competition process. This is where they defend their decisions on the four classes they placed earlier in the day. They write four sets of speeches and, with minimal preparation, they deliver the speeches in front of the judges. Pendleton County 4-H Agent Shelly Meyer has watched Gosney grow into the national champion. Lydia is a prodigy of the Pendleton County Livestock Judging program, she said of the young lady who started in the program as a nine-year-old and came back stronger each year. Gosney has teamed with Zach Wyatt, and the duo have been the team to beat in Kentucky, according to Meyer. Beyond their success in competition, Meyer said she saw even more in them. I turned the county judging team over to them to coach and train for this past years competition, she said. It was truly remarkable to watch two young people share their knowledge and expertise with their fellow 4-Hers.

It was an incredible way to give back. Gosney credits Meyer for her success. Shelley Meyer started this journey. She judged livestock in college and decided to put together a competitive team of local kids. She has traveled the country, competing and winning competitions, but in June, her team will travel to Scotland and Ireland to represent Kentucky.

The journey was not easy though as Gosney told Falmouth Outlook that each weekend since August her teammates loaded up in a University of Kentucky van to travel and practice nonstop. I dont think people really understand all the blood, sweat and tears put into this. The bond between my team is unbelievably strong, she said. I am humbled and honored to be a part of this team. As in all champions, the hard work, dedication and commitment from the individual is directly related to their success, there is a support system that assists and guides them along the way. Coach Steve Austin saw potential in her and has pushed her to be the best she can be. My parents, Martha and Brent Gosney, are the best support system I could ask for, she said. She credits mom for keeping her confidence levels up but reminding her to stay humble and thanking the Lord for everything He has given her. Her dad pushes her to be the best version of herself she can be but never fails to remind her that she is a champion whether she wins or not.

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Gosney travels the country to become a national champion - The Falmouth Outlook

Glenn Ellis

By Glenn Ellis

(TriceEdneyWire.com) It saddens me (as I grow older myself) how many people are struggling with accepting old age. Much of our society is filled with folks, young and old, who see aging as a negative aspect of life. Our society discards those who have reached old age, and we are inundated with promises and potions that have us fixated on staying young.

We are programmed to die;agingis the outcome of this programming, and it is no secret that the human body changes over time. Both genetics and lifestyle play a huge role in how we age.

We all age (if we live long enough), but seldom do we think about the process that takes place that takes us from the vibrant, youthful, and energetic creatures we used to be, to the sedentary and limited beings we become in old age.

There are those in the scientific and medical community who are advocating thatagingshould be treated as adisease. Aging is defined as the progressive accumulation of damage to your cells, tissues and organs, leading to disease and death. According to one study, this dreadful process starts at 24 years of age, at least for the brain; it could be a bit later.

Aging is nothing more than the natural wear and tear of the bodys component parts. Its inevitable, and endlessly intriguing. While many age-related changes cannot be prevented, a lifestyle that includes exercise and a well-balanced diet will slow or minimize many problems related to aging.

As we age, our bodys organs and other systems make changes. These changes alter our susceptibility to various diseases. Researchers are just beginning to understand the processes that cause changes over time in our body systems. Understanding these processes is important because many of the effects of aging are first noticed in our body systems.

No single process can explain all the changes of aging. Aging is a complex process that varies as to how it affects different people and even different organs. Most Gerontologists (people who study aging) feel that aging is due to the interaction of many lifelong influences. These influences include heredity, environment, culture, diet, exercise and leisure, past illnesses, and many other factors.

Since 1900, average U.S. life expectancy has risen from 47 to 79. A lot of those gains come from a lower infant-mortality rate: A century ago, 1-in-10 babies born in the U.S. died before age 1, while today that figure is 1-in-170. But longevity gains in later years have also been substantial.

Most people are scared, indeed, terrified of old age because they feel that aging is characterized by a progressive loss of essential body functions that they have learnt to take for granted over the years; for instance, loss of vision, hearing, teeth, memory, intelligence, sexual drive, muscle strength and vigor. However, it needs to be emphasized that you can become old healthily; remember that old age does not necessarily mean progressive deterioration or susceptibility to a plethora of ailments!

Fortunately, aging doesnt have to be a downhill slide. Older people have the reputation of being more mature, experienced and thoughtful. Whether or not you become wiser as you grow older, you are likely to become farsighted for sure! Farsightedness, one example of aging, is a change in vision thats a normal part of aging. It is caused by a gradual hardening of the eyes lens, which impairs your ability to see up close. Your optometrist may recommend a pair of non-prescription reading glasses or prescribe bifocals for you.

Never think ofageas being anything but just a number. There are some things in life we have no control over, such as when we were born. Age is no more than a circumstantial detail, like the color of your eyes, or the names of your parents; it doesnotdefine who you are. Aging is inevitable, growing old is avoidable. Expressed differently, one is never too young to be old or die, but one is never so aged as to become old.

If we live long enough, we will age. Just like the flowers, trees, bees, and all other living species on earth. It is up to us to accept aging as another stage of the life cycle nothing more. Too many of us become despondent, depressed, and feel worthless. Just make the best of whatever you have to work with in old age. Remember, healthy aging is not just about preventing problems. Its also about spotting them and addressing them before they get worse or drag down the rest of your health and independence.

Dying is not just an event that happens to us at the close of our lives. It is our purpose for being. We begin to die the very day we are born and live all our life towards death. Sure, we may get sick as we age, but we can get sick at any stage of life. Illness and aging need not go hand in hand. If you take good care of your body in the morning, it will take good care of you in the evening of your life.

Remember, Im not a doctor. I just sound like one. Take good care of yourself and live the best life possible!

The information included in this column is for educational purposes only. It is not intended nor implied to be a substitute for professional medical advice.Glenn Ellis, is Research Bioethics Fellow at Harvard Medical School and author of Which Doctor?, and Information is the Best Medicine. Ellis is an active media contributor on Health Equity and Medical Ethics. For more good health information visit: http://www.glennellis.com

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How and Why We Age - charlestonchronicle.net

Theres no way to spend half an hour talking with Jenny Tung and not come away with the unmistakable sense that youve encountered one of the smartest people youll ever meet. Shell speak quickly, words forming in a rapid-fire progression, explaining what are, to her, rudimentary concepts, but, to youif youre a journalist whose academic background is in social scienceare strange and foreign ideas.

Yet you grasp the gravity of what shes saying. Its not so much the granular details that matter. What matters is the fact that the work Tung and her colleagues are doing could fundamentally change how we understand societies and health and longevity.

In September, the John D. and Catherine T. MacArthur Foundation named Tung, a thirty-seven-year-old evolutionary anthropologist at Duke University, one of twenty-six 2019 fellows, an honor that comes with an unrestricted $625,000 Genius grant. (Tung was one of two North Carolinians to become a fellow this year; the other is an artist in Yancey County.)

The foundation said it awarded Tung the fellowship because her research has important implications for human health. While associations between socially induced stress and negative health outcomes have long been observed in humans, her findings suggest there is a causal link between social and environmental adversity and poor health.

To explain: We know that, on average, wealthier people live longer than poor people. There are a lot of potential causes: They have better health care. They smoke less. Theyre more likely to exercise and have access to more nutritious foods. Theyre less likely to live in environmentally hazardous neighborhoods.

Tungs work flips our notions of causality on their heads. Sure, those factors matter. But, in studies of baboons living in the wild in Kenya and rhesus monkeys in captivity, she and her team have shown that povertydiminished access to resourcesand lower social status actually affect us on the genomic and cellular level.

In essence, the research indicates that those on lower rungs of the socioeconomic ladder tend to have poorer immune systems, rendering them more susceptible to adverse health conditions and early death. And research with captive monkeys suggests that the health effects can be reversed by assigning the lower-ranking monkeys to a higher status.

Thats part of the reason why the MacArthur Foundation sees so much promise in her work.

Tung started out as an undergraduate at Duke in 1999. She planned to be a doctor, but those plans were derailed early on, when she took a course on evolution and social behavior. She was drawn to genetics and their role in quality-of-life determinants.

These things were often studied from the perspective of social sciences, she thought. Why not study them through the lens of life sciences?

If there are direct relationships between social conditions and how our organs and tissues and cells function, then thats a biological function, Tung says. Thats the framework. The how and the why.

Unlike social sciences, life sciences allowed for experimentation and the manipulation of social environments (albeit not with humans). Here, baboons proved especially useful. They are social animals that dont live nearly as long as humansabout eighteen years, on average. But thats long enough to track changes in lifespan. And the baboon group in Kenya has been monitored by scientists for decades, which made it ideal for this kind of generational research.

What shes discovered is that baboons born into early-life adversityduring droughts, or whose mothers died, or who were socially isolatedtend to live ten years less than their peers. In most cases, the cause of death isnt clear, nor is it clear whether the baboons died for the same reason.

But what is clear, Tung says, is that social adversity is toxic to all kinds of systems.

The rhesus monkeys might provide a hint as to whats going on. In lower-status and socially isolated monkeys, genes that are involved in the defense against viruses crank up, leading to molecular inflammation and eventually obliterating cellsa defense mechanism gone wild, as Tung puts it.

The work were contributing to helps clarify a lot about how social interactions could be causal to the outcomes we care about, she says.

There are many questions still to be answered, and the practical implications of Tungs work still need to be developed.

A utopian future in which there are no social stressors seems unlikely, Tung says, but improving childrens social environments could have a significant effect on their long-term health. Shes also looking at the UKs recent decision to add a Minister of Loneliness, aimed at giving isolated elderly people someone to talk to.

And she hopes her research might eventually help explain why some people seem more vulnerable to adverse conditions than others.

As for what she plans to do with the Genius money?

My immediate plan is to try to finish this semester without drowning, she told the INDY earlier this month. [The grant] comes with this onus to do something. I need to think about it.

Contact editor in chief Jeffrey C. Billman at jbillman@indyweek.com.

Support independent local journalism.Join the INDY Press Clubto help us keep fearless watchdog reporting and essential arts and culture coverage viable in the Triangle.

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19 People of 2019: Jenny Tung - INDY Week

Global Cell Culture Media Market insights relating to crucial aspects of the Cell Culture Media industry influencing its growth during the forecast period to 2024. It shows the sales of various Cell Culture Media forms of product increasing or declining in various regions and countries. The regional Cell Culture Media analysis provided within the report includes an excellent assessment of geographical niches by all factor-like CAGR and market share, production, and consumption. The Cell Culture Media market is divided into quite a detail for a transparent clarification of key growth areas with leading players that can make the foremost of it.

The Cell Culture Media industry research report additionally ensures the geographical division of this market. The proposed forecast typically includes global market size, share, product demand and supply, market trends, consumer tendencies, profitability, revenue outcomes and also company profiles of the key participants operating in the global Cell Culture Media market. For long-lasting business growth and strategic management, every manufacturer/company peruses intact forecast analysis that drives their business ahead of the curve.significant players of this Cell Culture Media Market are Atlanta Biologicals, Thermo Fisher, Merck Millipore, Zenbio, Sigma-Aldrich, PromoCell, BD, CellGenix, Corning (Cellgro), Life Technologies, GE Healthcare, Lonza, Takara and HiMedia

At the company level, this report focuses on the application, classification, and forecasts pertaining to volume and value, and future predictions for each manufacturer covered in this report. The top-down approaches have been used to counter-validate the Global Cell Culture Media Market numbers, while bottom-up approaches have been used to evaluate the market numbers for each element. The data has been authenticated by using the triangulation method in this exclusive report.

Key Players Of Cell Culture Media Market:

Atlanta Biologicals, Thermo Fisher, Merck Millipore, Zenbio, Sigma-Aldrich, PromoCell, BD, CellGenix, Corning (Cellgro), Life Technologies, GE Healthcare, Lonza, Takara and HiMedia

Product Types Of Cell Culture Media Market:

Serum-free Media, Stem Cell Media and Classical Media & Salts

Cell Culture Media Market isolation based on:

Tissue Culture & Engineering, Gene Therapy, Biopharmaceutical Manufacturing and Cytogenetic

Key regions that operate Cell Culture Media market includes Latin America (Argentina, Brazil, and Colombia), North America (Canada, The United States, and Mexico), Asia-Pacific (China, Japan, Korea, India, South-East Asia, and Thailand), Cell Culture Media market in Europe (Russia, Italy, United Kingdom, Germany, and Italy), The Middle East and Africa (South Africa, UAE, Nigeria, Saudi Arabia, and Egypt). Alongside, consumption value, market share, Cell Culture Media market value, import/export details, price/cost, Cell Culture Media market gross margin analysis and SWOT analysis.

1. Product overview and scope of Cell Culture Media market

2. Revenue and sales of Cell Culture Media by type and application (20192024)

3. Major players in the Cell Culture Media industry

4. Cell Culture Media players/suppliers Profiles and Sales data

5. Marketing strategy analysis and Cell Culture Media development trends

6. Worldwide Cell Culture Media Market effect factor analysis

7. Emerging niche segments and regional Cell Culture Media markets

8. A complete framework analysis, including an assessment of the parent Cell Culture Media industry

9. An empirical assessment of the trajectory of the Cell Culture Media market

1o. Major changes in Cell Culture Media market dynamics

11. Historical, present, and prospective size of the worldwide Cell Culture Media industry from the perspective of both value and volume

12. Understanding The Current Cell Culture Media Market

In conclusion, it is an in-depth research report on Global Cell Culture Media industry. Here, we express our thanks for the support and assistance from industry chain related technical experts and Cell Culture Media marketing engineers during Research Teams survey and interviews.

We have a too many categories research reports like Consumer Goods & Retailing, Agriculture, Food & Beverage, Food Services, Energy & Resources, Manufacturing & Construction, Chemicals & Materials, Transportation & Shipping, Biotechnology, Medical Devices, Pharmaceuticals & Healthcare, Business Services & Administration, IT & Telecom, Textiles, Automobile, Electrical & Electronic Device, Ship Manufacturing, Hotel and Tourism, Petroleum Industry, Trading Industry, Technology, Aerospace & Defense, Entertainment, etc.

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Global Cell Culture Media Market 2019 Analysis Sigma-Aldrich, Corning (Cellgro), Life Technologies and Thermo Fisher - Business Broker

LONDON, Dec. 18, 2019 /PRNewswire/ --GSK today announced positive headline results for intravenous (IV) Benlysta (belimumab) in the largest controlled phase 3 study in active lupus nephritis (LN), an inflammation of the kidneys caused by systemic lupus erythematosus (SLE) which can lead to end-stage kidney disease.

The Efficacy and Safety of Belimumab in Patients with Active Lupus Nephritis (BLISS-LN) study, involving 448 patients, met its primary endpoint demonstrating that a statistically significant greater number of patients achieved Primary Efficacy Renal Response (PERR) over two years when treated with belimumab plus standard therapy compared to placebo plus standard therapy in adults with active LN (43% vs 32%, odds ratio (95% CI) 1.55 (1.04, 2.32), p=0.0311).

Dr Hal Barron, Chief Scientific Officer and President R&D, GSK said: "Lupus nephritis is one of the most common and serious complications of SLE, occurring in up to 60% of adult patients. The results of the BLISS-LN study show that Benlysta could make a clinically meaningful improvement to the lives of these patients who currently have limited treatment options."

Dr Richard Furie,Chief of the Division of Rheumatology and Professor at the Feinstein Institutes atNorthwell Health and Lead Investigator of BLISS-LN said: "My journey with Benlysta began nearly twenty years ago when we performed the very first clinical research trial in lupus patients. To see it culminate in a successful phase 3 lupus nephritis study is a key achievement as the inadequate response of our patients with kidney disease to conventional treatment has long been an area in need of major improvement."

Belimumab also demonstrated statistical significance compared to placebo across all four major secondary endpoints: Complete Renal Response (CRR) after two years (the most stringent measure of renal response), Ordinal Renal Response (ORR) after two years, PERR after one year, and the time to death or renal-related event. In BLISS-LN, safety results for patients treated with belimumab were generally comparable to patients treated with placebo plus standard therapy. The safety results are consistent with the known profile of belimumab.

Benlysta is currently not recommended for use in severe active lupus nephritis anywhere in the world because it has not been previously evaluated in these patients. Based on these positive phase 3 data, GSK plans to progress regulatory submissions in the first half of 2020 to seek an update to the prescribing information.

The full results will be submitted for future presentation at upcoming scientific meetings and in peer-reviewed publications.

About lupus nephritisSystemic lupus erythematosus (SLE), the most common form of lupus, is a chronic, incurable, autoimmune disease associated with a range of symptoms that can fluctuate over time including painful or swollen joints, extreme fatigue, unexplained fever, skin rashes and organ damage. In lupus nephritis (LN), SLE causes kidney inflammation, which can lead to end-stage kidney disease. Despite improvements in both diagnosis and treatment over the last few decades, LN remains an indicator of poor prognosis.1,2 Manifestations of LN include proteinuria, elevations in serum creatinine, and the presence of urinary sediment.

About BLISS-LNBLISS-LN,which enrolled 448 adult patients, was a phase 3, 104-week, randomised, double-blind, placebo-controlled post-approval commitment study to evaluate the efficacy and safety of IV belimumab 10 mg/kg plus standard therapy (mycophenolate mofentil for induction and maintenance, or cyclophosphamide for induction followed by azathioprine for maintenance, plus steroids) compared to placebo plus standard therapy in adult patients with active lupus nephritis. Active lupus nephritis was confirmed by kidney biopsy during screening visit using the 2003 International Society of Nephrology/Renal Pathology Society (ISN/RPS) criteria, and clinically active kidney disease.

The primary endpoint PERR was defined as estimated Glomerular Filtration Rate (eGFR) 60 mL/min/1.73m2 or no decrease in eGFR from pre-flare of > 20%; and urinary protein:creatinine ratio (uPCR) 0.7; and not a treatment failure. The most stringent secondary endpoint CRR was defined as eGFR is no more than 10% below the pre-flare value or within normal range; and uPCR < 0.5; and not a treatment failure. ORR was defined as complete, partial or no response.

About Benlysta (belimumab)Benlysta, a BLyS-specific inhibitor, is a human monoclonal antibody that binds to soluble BLyS. Benlysta does not bind B cells directly. By binding BLyS, Benlysta inhibits the survival of B cells, including autoreactive B cells, and reduces the differentiation of B cells into immunoglobulin-producing plasma cells.

The current US and EU indication for Benlysta are summarised below:

In the US, "Benlysta is indicated for the treatment of patients aged 5 years and older with active, autoantibody-positive, systemic lupus erythematosus (SLE) who are receiving standard therapy. Limitations of Use: The efficacy of Benlysta has not been evaluated in patients with severe active lupus nephritis or severe active central nervous system lupus. Benlysta has not been studied in combination with other biologics or intravenous cyclophosphamide. Use of Benlysta is not recommended in these situations."

Full US prescribing information including Medication Guide is available at: https://www.gsksource.com/pharma/content/dam/GlaxoSmithKline/US/en/Prescribing_Information/Benlysta/pdf/BENLYSTA-PI-MG.PDF

In the EU, "Benlysta is indicated as "add-on therapy in patients aged 5 years and older with active, autoantibody-positive systemic lupus erythematosus (SLE) with a high degree of disease activity (e.g., positive anti-dsDNA and low complement) despite standard therapy."

The Precaution and Warnings for Benlysta includes information that "Benlysta has not been studied in the following adult and paediatric patient groups, and is not recommended: severe active central nervous system lupus; severe active lupus nephritis; HIV; a history of, or current, hepatitis B or C; hypogammaglobulinaenia (IgG < 400mg/dl) or IgA deficiency (IgA < 10 mg/dl); a history of major organ transplant or hematopoietic stem cell/marrow transplant or renal transplant."

The EU Summary of Product Characteristics for Benlysta is available on: http://www.ema.europa.eu

Benlysta is available as an intravenous and a subcutaneous formulation. The Benlysta subcutaneous formulation is not approved for use in children.

GSK's commitment to immunologyGSK is focused on the research and development of medicines for immune-mediated diseases, such as lupus and rheumatoid arthritis, that are responsible for a significant health burden to patients and society. Our world-leading scientists are focusing research on the biology of the immune system with the aim to develop immunological-based medicines that have the potential to alter the course of inflammatory disease. As the only company with a biological treatment approved for adult and paediatric lupus, GSK is leading the way to help patients and their families manage this chronic, inflammatory autoimmune disease. Our aim is to develop transformational medicines that can alter the course of inflammatory disease to help people live their best day, every day.

Important Safety Information for belimumabPlease consult the full Prescribing Information for all the labelled safety information for Benlysta (belimumab)

Contraindications:Previous anaphylaxis with BENLYSTA.

Warnings and precautions: Not recommended in adult and paediatric groups with severe active central nervous system lupus, severe active lupus nephritis, HIV, history of/current hepatitis B or C, hypogammaglobulinaemia (IgG <400 mg/dl) or IgA deficiency (IgA <10 mg/dl) and patients with a history of major organ transplant or hematopoietic stem/cell/marrow transplant or renal transplant.

Mortality:In adult intravenous (IV) clinical trials, death occurred in 0.8% of patients treated with BENLYSTA and in 0.4% of patients receiving placebo; etiologies included infection, cardiovascular disease, and suicide. In the adult SC clinical trial, death occurred in 0.5% of patients receiving BENLYSTA and in 0.7% of patients receiving placebo; infection was the most common cause of death.

Serious Infections:Serious and sometimes fatal infections have been reported in patients receiving immunosuppressive agents, including BENLYSTA. The most frequent serious infections in adults treated with BENLYSTA IV included pneumonia, urinary tract infection, cellulitis, and bronchitis. Use caution in patients with severe or chronic infections, and consider interrupting therapy in patients with a new infection.

Progressive Multifocal Leukoencephalopathy (PML):Cases of JC virus-associated PML resulting in neurological deficits, including fatal cases, have been reported in patients with SLE receiving immunosuppressants, including BENLYSTA. If PML is confirmed, consider stopping immunosuppressant therapy, including BENLYSTA.

Hypersensitivity Reactions (Including Anaphylaxis):Acute hypersensitivity reactions, including anaphylaxis (eg, hypotension, angioedema, urticaria or other rash, pruritus, and dyspnea) and death, have been reported, including in patients who have previously tolerated BENLYSTA. Generally, reactions occurred within hours of the infusion but may occur later. Non-acute hypersensitivity reactions (eg, rash, nausea, fatigue, myalgia, headache, and facial edema) typically occurred up to a week after infusion. Patients with a history of multiple drug allergies or significant hypersensitivity may be at increased risk. With BENLYSTA SC, systemic hypersensitivity reactions were similar to those in IV trials.

Healthcare providers (HCPs) should monitor patients during and after IV administration and be prepared to manage anaphylaxis; discontinue immediately in the event of a serious reaction. Premedication may mitigate or mask a hypersensitivity response. Advise patients about hypersensitivity symptoms and instruct them to seek immediate medical care if a reaction occurs.

Infusion Reactions:Serious infusion reactions (eg, bradycardia, myalgia, headache, rash, urticaria, and hypotension) were reported in adults. HCPs should monitor patients and manage reactions if they occur. Premedication may mitigate or mask a reaction. If an infusion reaction develops, slow or interrupt the infusion.

Depression and Suicidality:In clinical trials, psychiatric disorders (depression, suicidal ideation and behavior) were reported more frequently in patients receiving BENLYSTA than placebo. In adult trials, psychiatric events reported more frequently with BENLYSTA IV related primarily to depression-related events, insomnia, and anxiety; serious psychiatric events included serious depression and suicidality, including 2 completed suicides. No serious depression-related events or suicides were reported in the BENLYSTA SC trial. Before adding BENLYSTA, physicians should assess patients' risk of depression and suicide and monitor them during treatment. Instruct patients to contact their HCP if they experience new/worsening depression, suicidal thoughts, or other mood changes.

Malignancy:The impact of BENLYSTA on the development of malignancies is unknown; its mechanism of action could increase the risk for malignancies.

Immunization: Live vaccines should not be given for 30 days before or concurrently with BENLYSTA as clinical safety has not been established.

Use With Biologic Therapies or IV Cyclophosphamide:BENLYSTA has not been studied and is not recommended in combination with other biologic therapies, including B-cell targeted therapies, or IV cyclophosphamide.

Adverse Reactions:The most common serious adverse reactions in adults were serious infections: BENLYSTA IV 6.0% (placebo 5.2%), some of which were fatal. Adverse reactions occurring in 3% of adults and 1% more than placebo: nausea 15% (12%); diarrhea 12% (9%); pyrexia 10% (8%); nasopharyngitis 9% (7%); bronchitis 9% (5%); insomnia 7% (5%); pain in extremity 6% (4%); depression 5% (4%); migraine 5% (4%); pharyngitis 5% (3%); cystitis 4% (3%); leukopenia 4% (2%); viral gastroenteritis 3% (1%).

Adverse reactions in pediatric patients aged 5 years receiving BENLYSTA IV were consistent with those observed in adults.

The safety profile observed for BENLYSTA SC in adults was consistent with the known safety profile of BENLYSTA IV with the exception of local injection site reactions.

Pregnancy and lactation:Pregnancy: There are insufficient data in pregnant women to establish whether there is drug-associated risk for major birth defects or miscarriage. After a risk/benefit assessment, if prevention is warranted, women of childbearing potential should use contraception during treatment and for 4 months after the final treatment.

Lactation:No information is available on the presence of belimumab in human milk, the effects on the breastfed infant, or the effects on milk production. Consider developmental and health benefits of breastfeeding with the mother's clinical need for BENLYSTA and any potential adverse effects on the breastfed child or from the underlying maternal condition.

Pediatric Use:The safety and effectiveness have not been established for BENLYSTA IV in patients <5 years of age and for BENLYSTA SC in patients <18 years of age.

Black/African American Patients:In clinical trials there have been mixed results regarding how well BENLYSTA works in this patient population. Consider risks and benefits when prescribing BENLYSTA.

About GSK GSK is a science-led global healthcare company with a special purpose: to help people do more, feel better, live longer. For further information please visit http://www.gsk.com.

Trademarks are owned by or licensed to the GSK group of companies.

References

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Cautionary statement regarding forward-looking statements

GSK cautions investors that any forward-looking statements or projections made by GSK, including those made in this announcement, are subject to risks and uncertainties that may cause actual results to differ materially from those projected. Such factors include, but are not limited to, those described under Item 3.D 'Principal risks and uncertainties' in the company's Annual Report on Form 20-F for 2018.

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GSK announces positive headline results in phase 3 study of Benlysta in patients with lupus nephritis - BioSpace

Gene therapy is fighting to enter mainstream medicine. With sickle cell disease, the fight is heating up.

Roughly two years ago, the FDA made the historic decision to approve the first gene therapy in the US, finally realizing the therapeutic potential of hacking our biological base code after decades of cycles of hope and despair. Other approvals soon followed, including Luxturna to target inherited blindness and Zolgensma, a single injection that could save children with a degenerative disease from their muscles wasting away and dying before the age of two.

Yet despite their transformative potential, gene therapy has only targeted relatively rareand often fataldisorders. Thats about to change.

This year, a handful of companies deployed gene therapy against sickle-cell anemia, a condition that affects over 20 million people worldwide and 100,000 Americans. With over a dozen therapies in the run, sickle-cell disease could be the indication that allows gene therapy to enter the mainstream. Yet because of its unique nature, sickle-cell could also be the indication that shines an unflinching spotlight on challenges to the nascent breakthrough, both ethically and technologically.

You see, sickle-cell anemia, while being one of the worlds best-known genetic diseases, and one of the best understood, also predominantly affects third-world countries and marginalized people of color in the US. So far, gene therapy has come with a hefty bill exceeding millions; few people afflicted by the condition can carry that amount. The potential treatments are enormously complex, further upping costs to include lengthy hospital stays, and increasing potential side effects. To muddy the waters even more, the disorder, though causing tremendous pain and risk of stroke, already has approved pharmaceutical treatments and isnt necessarily considered life-threatening.

How we handle gene therapies for sickle-cell could inform many other similar therapies to come. With nearly 400 clinical trials in the making and two dozen nearing approval, theres no doubt that hacking our genes will become one of the most transformative medical wonders of the new decade. The question is: will it ever be available for everyone in need?

Even those uninterested in biology have likely heard of the disorder. Sickle-cell anemia holds the crown as the first genetic disorder to be traced to its molecular roots nearly a hundred years ago.

The root of the disorder is a single genetic mutation that drastically changes the structure of the oxygen-carrying protein, beta-globin, in red blood cells. The result is that the cells, rather than forming their usual slick disc-shape, turn into jagged, sickle-shaped daggers that damage blood vessels or block them altogether. The symptoms arent always uniform; rather, they come in crisis episodes during which the pain becomes nearly intolerable.

Kids with sickle-cell disorder usually die before the age of five; those who survive suffer a lifetime of debilitating pain and increased risk of stroke and infection. The symptoms can be managed to a degree with a cocktail of drugsantibiotics, painkillers, and a drug that reduces crisis episodes but ups infection risksand frequent blood transfusions or bone marrow transplants. More recently, the FDA approved a drug that helps prevent sickled-shaped cells from forming clumps in the vessels to further combat the disorder.

To Dr. David Williams at Boston Childrens Hospital in Massachusetts, the availability of these treatmentshowever inadequatesuggests that gene therapy remains too risky for sickle-cell disease. Its not an immediately lethal diseaseit wouldnt be ethical to treat those patients with a highly risky experimental approach, he said to Nature.

Others disagree. Freeing patients from a lifetime of risks and pain seems worthy, regardless of the price tag. Inspired by recent FDA approvals, companies have jumped onto three different treatments in a bitter fight to be the first to win approval.

The complexity of sickle-cell disease also opens the door to competing ideas about how to best treat it.

The most direct approach, backed by Bluebird Bio in Cambridge, Massachusetts, uses a virus to insert a functional copy of the broken beta-globin gene into blood cells. This approach seems to be on track for winning the first FDA approval for the disorder.

The second idea is to add a beneficial oxygen-carrying protein, rather than fixing the broken one. Here, viruses carry gamma-globin, which is a variant mostly present in fetal blood cells, but shuts off production soon after birth. Gamma-globin acts as a repellent that prevents clotting, a main trigger for strokes and other dangerous vascular diseases.

Yet another idea also focuses on gamma-globin, the good guy oxygen-carrier. Here, rather than inserting genes to produce the protein, the key is to remove the breaks that halt its production after birth. Both Bluebird Bio and Sangamo Therapeutics, based in Richmond, California, are pursing this approach. The rise of CRISPR-oriented companies is especially giving the idea new promise, in which CRISPR can theoretically shut off the break without too many side effects.

But there are complications. All three approaches also tap into cell therapy: blood-producing cells are removed from the body through chemotherapy, genetically edited, and re-infused into the bone marrow to reconstruct the entire blood system.

Its a risky, costly, and lengthy solution. Nevertheless, there have already been signs of success in the US. One person in a Bluebird Bio trial remained symptom-free for a year; another, using a CRISPR-based approach, hasnt experienced a crisis in four months since leaving the hospital. For about a year, Bluebird Bio has monitored a dozen treated patients. So far, according to the company, none has reported episodes of severe pain.

Despite these early successes, advocates worry about the actual impact of a genetic approach to sickle-cell disease.

Similar to other gene therapies, the treatment is considered a last-line, hail Mary solution for the most difficult cases of sickle cell disease because of its inherent risks and costly nature. Yet end-of-the-line patients often suffer from kidney, liver, and heart damages that make chemotherapy far too dangerous.

Then theres the problem of global access. Some developing countries, where sickle-cell disease is more prevalent, dont even have consistent access to safe blood transfusions, not to mention the laboratory equipment needed for altering blood-producing stem cells. Recent efforts in education, early screening, and prevention have also allowed people to live longer and reduce the stigma of the disorder.

Is a $1 million price tag ever attainable? To combat exhorbitant costs, Bluebird Bio is offering an installment payment plan for five years, which can be terminated anytime the treatment stops working. Yet for patients in South Africa, India, or Cambodia, the costs far exceed the $3 per month price tag for standard treatment. Even hydroxyurea, the newly-approved FDA drug to reduce crisis pain episodes, is just a fraction of the price tag that comes with gene therapy.

As gene therapy technologies are further refined and their base cost reduced, its possible that overall costs will drop. Yet whether these treatments will be affordable in the long run remains questionable. Even as scientists focus on efficacy rather than price tag, NIH director Dr. Francis Collins believes not thinking about global access is almost unethical. There are historical examples for optimism: vaccines, once rather fringe, now touch almost every corner of our world with the help of scientific knowledge, advocacy groups, andfundamentallyproven efficacy.

With the rise of gene therapy, were now in an age of personalized medicine beyond imagination. Its true that perhaps sickle-cell disease genetic therapies arent quite there yet in terms of safety and efficacy; but without tackling access issues, the therapy will be stymied in its impact for global good. As genetic editing tools become more powerful, gene therapy has the potential to save even more livesif its made accessible to those who need it most.

Image Credit: Image by Narupon Promvichai from Pixabay

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Gene Therapy for Sickle-Cell Anemia Looks Promisingbut It's Riddled With Controversy - Singularity Hub

SAN DIEGO, Dec. 12, 2019 /PRNewswire/ -- Aspen Neuroscience, Inc. today announced its launch following a $6.5 million seed round led by Domain Associates and Axon Ventures and including Alexandria Venture Investments,Arch Venture Partners,OrbiMedand Section 32 to develop the first autologous cell therapies for Parkinson's disease. Aspen's proprietary approach was developed by the company's co-founders, Jeanne F. Loring, Ph.D., Professor Emeritus and founding director of the Center for Regenerative Medicine at The Scripps Research Institute, and Andres Bratt-Leal, Ph.D., a former post-doctoral researcher in Dr. Loring's lab. The company was initially supported by Summit for Stem Cell, a founding partner and non-profit organization which provides a variety of services for people with Parkinson's disease. Aspen is led by industry veteran Howard J. Federoff, M.D., Ph.D., as Chief Executive Officer.

Parkinson's disease is characterized by the loss of specific brain cells that make the chemical dopamine. Without dopamine, nerve cells cannot communicate with muscles and people are left with debilitating motor problems. Aspen is focusing on human pluripotent stem cells, cultured cells that can become any cell type in the human body. The company's research is specific to induced pluripotent stem cells (iPSCs), which it develops by taking a skin biopsy from a person with Parkinson's disease and turning the tissue into pluripotent stem cells using genetic engineering. Aspen then differentiates the pluripotent stem cells into dopamine-releasing neurons that can be transplanted into that same person (autologous), thereby restoring the types of neurons lost in Parkinson's disease.

As an autologous cell therapy for Parkinson's disease, Aspen's treatment would eliminate the need for immunosuppression because the neurons are transplanted back into the same patient from which they were generated. The use of immunosuppression is necessary with currently available cell therapies for Parkinson's disease and when transplanting cells from one patient to another (allogeneic) to prevent rejection but can pre-dispose the patient to life-threatening complications including infection and add cost to the patient and health system. Aspen is the only company in the world offering an autologous neuron replacement therapy for Parkinson's disease.

Aspen encompasses a powerful executive leadership team including Dr. Federoff who, in addition to his leadership roles at the UC Irvine Health System, was the Executive Vice President for Health Sciences and the Executive Dean of Medicine at Georgetown University. Dr. Federoff also has significant biotech industry experience including co-founding MedGenesis Therapeutix and Brain Neurotherapy Bio, as well as leading the U.S. Parkinson's Disease Gene Therapy Study Group. The company is also proud to announce the addition of several experienced and well-known members to its leadership team including Edward Wirth, M.D., Ph.D., as Chief Medical Officer.

Dr. Wirth currently serves as the Chief Medical Ofcer for Lineage Cell Therapeutics where he oversees clinical development of its two therapeutic programs for spinal cord injuries and lung cancer. He received his M.D. and Ph.D. from the University of Florida in 1994 and remained to conduct postdoctoral research including leading the University of Florida team that performed the rst human embryonic spinal cord transplant in the U.S. Dr. Wirth went on to serve as the Medical Director for Regenerative Medicine at Geron Corporation where the world's rst clinical trial of human embryonic stem cell (hESC)-derived product occurred which demonstrated initial clinical safety.

Drs. Federoff and Wirth are joined by Dr. Loring, as Chief Scientific Officer; Jay Sial, as Chief Financial Officer; Andres Bratt-Leal, Ph.D., as Vice President of Research and Development; Thorsten Gorba, Ph.D., as Senior Director of Manufacturing and Naveen M. Krishnan, M.D., M.Phil., as Senior Director of Corporate Development.

"Aspen is developing a restorative, disease modifying autologous neuron therapy for people suffering from Parkinson's disease," said Dr. Federoff. "We are fortunate to have such a high-caliber scientific and medical leadership team to make our treatments a reality. Our cell replacement therapy, which originated in the laboratory of Dr. Jeanne Loring and was later supported by Summit for Stem Cell and its President, Ms. Jenifer Raub, has the potential to release dopamine and reconstruct neural networks where no disease-modifying therapies exist."

Aspen's lead product (ANPD001) is currently undergoing investigational new drug (IND)-enabling studies for the treatment of sporadic Parkinson's disease. Aspen is also developing a gene-edited autologous neuron therapy (ANPD002) that is in the research stage and targeted toward familial forms of Parkinson's disease beginning with the most common genetic variant in the gene encoding glucocerebrosidase (GBA). Aspen leverages proprietary machine-learning tools and artificial intelligence to ensure quality control during manufacturing and to deliver a safe and reproducible product for each cell line.

"Aspen's financial backing, combined with its experienced and proven leadership team, positions it well for future success," said Kim P. Kamdar, Ph.D., Partner at Domain Associates, one of Aspen's seed investors. "Domain prides itself on investing in companies that can translate scientific research into innovative medicines and therapies that make a difference in people's lives. We clearly see Aspen as fitting into that category, as it is the only company using a patient's own cells for replacement therapy in Parkinson's disease."

About Aspen Neuroscience

Aspen Neuroscience Inc. is a development stage, private biotechnology company that uses innovative genomic approaches combined with stem cell biology to deliver patient-specific, restorative cell therapies that modify the course of Parkinson's disease. Aspen's therapies are based upon the scientific work of world-renowned stem cell scientist, Dr. Jeanne Loring, who has developed a novel method for autologous neuron replacement. For more information and important updates, please visithttp://www.aspenneuroscience.com.

CONTACT: Jennifer Viera, AspenNeuroscience@TeamSeismic.com

SOURCE Aspen Neuroscience

http://www.aspenneuroscience.com

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Aspen Neuroscience Launches With $6.5 Million Seed Funding to Advance First-of-its-Kind Personalized Cell Therapy for Parkinson's Disease - PRNewswire

The ODAC discussions were based on the supplemental Biologics License Application (sBLA), currently under priority review at the FDA, seeking approval of KEYTRUDA monotherapy for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, NMIBC with carcinoma in-situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy (removal of bladder). This application is based on results from the Phase 2 KEYNOTE-057 trial.

The positive vote from todays ODAC meeting supports the potential for KEYTRUDA in certain patients with high-risk, non-muscle invasive bladder cancer, who currently have limited non-surgical treatment options approved by the FDA, said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories. We are encouraged by todays productive discussion and look forward to working with the FDA as they continue their review of our supplemental application for KEYTRUDA in this patient population.

The ODAC provides the FDA with independent, expert advice and recommendations on marketed and investigational medicines for use in the treatment of cancer. The FDA is not bound by the committees guidance but takes its advice into consideration. Merck anticipates a Prescription Drug User Fee Act (PDUFA), or target action date, in January 2020, based on priority review.

About Bladder Cancer

Bladder cancer begins when cells in the urinary bladder start to grow uncontrollably. As more cancer cells develop, they can form a tumor and spread to other areas of the body. Bladder cancers are described based on how far they have invaded into the wall of the bladder. NMIBC occurs when the cancer has not grown into the main muscle layer of the bladder. It is estimated that more than 80,000 new cases of bladder cancer will be diagnosed in 2019 in the United States. Approximately 75% of patients with bladder cancer are diagnosed with non-muscle invasive bladder cancer (NMIBC). For high-risk NMIBC patients who are BCG-unresponsive with persistent or recurrent disease, treatment guidelines recommend radical cystectomy, a surgery to remove the entire bladder that often requires removal of other surrounding organs and tissues. In men, removal of the prostate is common, and in women, surgeons may also remove the uterus, fallopian tubes, ovaries and cervix, and occasionally a portion of the vagina.

About KEYNOTE-057

The filing was based on data from KEYNOTE-057 (NCT02625961), a Phase 2, multicenter, open-label, single-arm trial in 102 patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in-situ (CIS) with or without papillary tumors who were ineligible for or had elected not to undergo cystectomy (Cohort A). In this study, BCG-unresponsive high-risk NMIBC is defined as persistent disease despite adequate BCG therapy, disease recurrence after an initial tumor-free state following adequate BCG therapy, or T1 disease following a single induction course of BCG. Patients received KEYTRUDA 200 mg every three weeks until unacceptable toxicity, persistent or recurrent high-risk NMIBC or progressive disease. Assessment of tumor status was performed every 12 weeks, and patients without disease progression could be treated for up to 24 months. The major efficacy outcome measures were complete response (as defined by negative results for cystoscopy [with transurethral resection of bladder tumor (TURBT)/biopsies as applicable], urine cytology, and computed tomography urography [CTU] imaging) and duration of response.

About KEYTRUDA (pembrolizumab) Injection, 100mg

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,000 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patients likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) Indications

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung Cancer

KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Small Cell Lung Cancer

KEYTRUDA is indicated for the treatment of patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy and at least one other prior line of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Head and Neck Squamous Cell Cancer

KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) 1] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum-containing chemotherapy.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory classical Hodgkin lymphoma (cHL), or who have relapsed after 3 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Primary Mediastinal Large B-Cell Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 [combined positive score (CPS) 10] as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

Microsatellite Instability-High (MSI-H) Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

Gastric Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Esophageal Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test, with disease progression after one or more prior lines of systemic therapy.

Cervical Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Hepatocellular Carcinoma

KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Merkel Cell Carcinoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Renal Cell Carcinoma

KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

Selected Important Safety Information for KEYTRUDA

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis, including fatal cases. Pneumonitis occurred in 3.4% (94/2799) of patients with various cancers receiving KEYTRUDA, including Grade 1 (0.8%), 2 (1.3%), 3 (0.9%), 4 (0.3%), and 5 (0.1%). Pneumonitis occurred in 8.2% (65/790) of NSCLC patients receiving KEYTRUDA as a single agent, including Grades 3-4 in 3.2% of patients, and occurred more frequently in patients with a history of prior thoracic radiation (17%) compared to those without (7.7%). Pneumonitis occurred in 6% (18/300) of HNSCC patients receiving KEYTRUDA as a single agent, including Grades 3-5 in 1.6% of patients, and occurred in 5.4% (15/276) of patients receiving KEYTRUDA in combination with platinum and FU as first-line therapy for advanced disease, including Grades 3-5 in 1.5% of patients.

Monitor patients for signs and symptoms of pneumonitis. Evaluate suspected pneumonitis with radiographic imaging. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 or recurrent Grade 2 pneumonitis.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis. Colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 2 (0.4%), 3 (1.1%), and 4 (<0.1%). Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold KEYTRUDA for Grade 2 or 3; permanently discontinue KEYTRUDA for Grade 4 colitis.

Immune-Mediated Hepatitis (KEYTRUDA) and Hepatotoxicity (KEYTRUDA in Combination With Axitinib)

Immune-Mediated Hepatitis

KEYTRUDA can cause immune-mediated hepatitis. Hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.4%), and 4 (<0.1%). Monitor patients for changes in liver function. Administer corticosteroids for Grade 2 or greater hepatitis and, based on severity of liver enzyme elevations, withhold or discontinue KEYTRUDA.

Hepatotoxicity in Combination With Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity with higher than expected frequencies of Grades 3 and 4 ALT and AST elevations compared to KEYTRUDA alone. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased ALT (20%) and increased AST (13%) were seen. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider more frequent monitoring of liver enzymes as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed.

Immune-Mediated Endocrinopathies

KEYTRUDA can cause hypophysitis, thyroid disorders, and type 1 diabetes mellitus. Hypophysitis occurred in 0.6% (17/2799) of patients, including Grade 2 (0.2%), 3 (0.3%), and 4 (<0.1%). Hypothyroidism occurred in 8.5% (237/2799) of patients, including Grade 2 (6.2%) and 3 (0.1%). The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC (16%) receiving KEYTRUDA, as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. Hyperthyroidism occurred in 3.4% (96/2799) of patients, including Grade 2 (0.8%) and 3 (0.1%), and thyroiditis occurred in 0.6% (16/2799) of patients, including Grade 2 (0.3%). Type 1 diabetes mellitus, including diabetic ketoacidosis, occurred in 0.2% (6/2799) of patients.

Monitor patients for signs and symptoms of hypophysitis (including hypopituitarism and adrenal insufficiency), thyroid function (prior to and periodically during treatment), and hyperglycemia. For hypophysitis, administer corticosteroids and hormone replacement as clinically indicated. Withhold KEYTRUDA for Grade 2 and withhold or discontinue for Grade 3 or 4 hypophysitis. Administer hormone replacement for hypothyroidism and manage hyperthyroidism with thionamides and beta-blockers as appropriate. Withhold or discontinue KEYTRUDA for Grade 3 or 4 hyperthyroidism. Administer insulin for type 1 diabetes, and withhold KEYTRUDA and administer antihyperglycemics in patients with severe hyperglycemia.

Immune-Mediated Nephritis and Renal Dysfunction

KEYTRUDA can cause immune-mediated nephritis. Nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.1%), and 4 (<0.1%) nephritis. Nephritis occurred in 1.7% (7/405) of patients receiving KEYTRUDA in combination with pemetrexed and platinum chemotherapy. Monitor patients for changes in renal function. Administer corticosteroids for Grade 2 or greater nephritis. Withhold KEYTRUDA for Grade 2; permanently discontinue for Grade 3 or 4 nephritis.

Immune-Mediated Skin Reactions

Immune-mediated rashes, including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN) (some cases with fatal outcome), exfoliative dermatitis, and bullous pemphigoid, can occur. Monitor patients for suspected severe skin reactions and based on the severity of the adverse reaction, withhold or permanently discontinue KEYTRUDA and administer corticosteroids. For signs or symptoms of SJS or TEN, withhold KEYTRUDA and refer the patient for specialized care for assessment and treatment. If SJS or TEN is confirmed, permanently discontinue KEYTRUDA.

Other Immune-Mediated Adverse Reactions

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue in patients receiving KEYTRUDA and may also occur after discontinuation of treatment. For suspected immune-mediated adverse reactions, ensure adequate evaluation to confirm etiology or exclude other causes. Based on the severity of the adverse reaction, withhold KEYTRUDA and administer corticosteroids. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Based on limited data from clinical studies in patients whose immune-related adverse reactions could not be controlled with corticosteroid use, administration of other systemic immunosuppressants can be considered. Resume KEYTRUDA when the adverse reaction remains at Grade 1 or less following corticosteroid taper. Permanently discontinue KEYTRUDA for any Grade 3 immune-mediated adverse reaction that recurs and for any life-threatening immune-mediated adverse reaction.

The following clinically significant immune-mediated adverse reactions occurred in less than 1% (unless otherwise indicated) of 2799 patients: arthritis (1.5%), uveitis, myositis, Guillain-Barr syndrome, myasthenia gravis, vasculitis, pancreatitis, hemolytic anemia, sarcoidosis, and encephalitis. In addition, myelitis and myocarditis were reported in other clinical trials, including classical Hodgkin lymphoma, and postmarketing use.

Treatment with KEYTRUDA may increase the risk of rejection in solid organ transplant recipients. Consider the benefit of treatment vs the risk of possible organ rejection in these patients.

Infusion-Related Reactions

KEYTRUDA can cause severe or life-threatening infusion-related reactions, including hypersensitivity and anaphylaxis, which have been reported in 0.2% (6/2799) of patients. Monitor patients for signs and symptoms of infusion-related reactions. For Grade 3 or 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

Complications of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Immune-mediated complications, including fatal events, occurred in patients who underwent allogeneic HSCT after treatment with KEYTRUDA. Of 23 patients with cHL who proceeded to allogeneic HSCT after KEYTRUDA, 6 (26%) developed graft-versus-host disease (GVHD) (1 fatal case) and 2 (9%) developed severe hepatic veno-occlusive disease (VOD) after reduced-intensity conditioning (1 fatal case). Cases of fatal hyperacute GVHD after allogeneic HSCT have also been reported in patients with lymphoma who received a PD-1 receptorblocking antibody before transplantation. Follow patients closely for early evidence of transplant-related complications such as hyperacute graft-versus-host disease (GVHD), Grade 3 to 4 acute GVHD, steroid-requiring febrile syndrome, hepatic veno-occlusive disease (VOD), and other immune-mediated adverse reactions.

In patients with a history of allogeneic HSCT, acute GVHD (including fatal GVHD) has been reported after treatment with KEYTRUDA. Patients who experienced GVHD after their transplant procedure may be at increased risk for GVHD after KEYTRUDA. Consider the benefit of KEYTRUDA vs the risk of GVHD in these patients.

Increased Mortality in Patients With Multiple Myeloma

In trials in patients with multiple myeloma, the addition of KEYTRUDA to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of these patients with a PD-1 or PD-L1 blocking antibody in this combination is not recommended outside of controlled trials.

Embryofetal Toxicity

Based on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. Advise women of this potential risk. In females of reproductive potential, verify pregnancy status prior to initiating KEYTRUDA and advise them to use effective contraception during treatment and for 4 months after the last dose.

Adverse Reactions

In KEYNOTE-006, KEYTRUDA was discontinued due to adverse reactions in 9% of 555 patients with advanced melanoma; adverse reactions leading to permanent discontinuation in more than one patient were colitis (1.4%), autoimmune hepatitis (0.7%), allergic reaction (0.4%), polyneuropathy (0.4%), and cardiac failure (0.4%). The most common adverse reactions (20%) with KEYTRUDA were fatigue (28%), diarrhea (26%), rash (24%), and nausea (21%).

In KEYNOTE-002, KEYTRUDA was permanently discontinued due to adverse reactions in 12% of 357 patients with advanced melanoma; the most common (1%) were general physical health deterioration (1%), asthenia (1%), dyspnea (1%), pneumonitis (1%), and generalized edema (1%). The most common adverse reactions were fatigue (43%), pruritus (28%), rash (24%), constipation (22%), nausea (22%), diarrhea (20%), and decreased appetite (20%).

In KEYNOTE-054, KEYTRUDA was permanently discontinued due to adverse reactions in 14% of 509 patients; the most common (1%) were pneumonitis (1.4%), colitis (1.2%), and diarrhea (1%). Serious adverse reactions occurred in 25% of patients receiving KEYTRUDA. The most common adverse reaction (20%) with KEYTRUDA was diarrhea (28%).

In KEYNOTE-189, when KEYTRUDA was administered with pemetrexed and platinum chemotherapy in metastatic nonsquamous NSCLC, KEYTRUDA was discontinued due to adverse reactions in 20% of 405 patients. The most common adverse reactions resulting in permanent discontinuation of KEYTRUDA were pneumonitis (3%) and acute kidney injury (2%). The most common adverse reactions (20%) with KEYTRUDA were nausea (56%), fatigue (56%), constipation (35%), diarrhea (31%), decreased appetite (28%), rash (25%), vomiting (24%), cough (21%), dyspnea (21%), and pyrexia (20%).

In KEYNOTE-407, when KEYTRUDA was administered with carboplatin and either paclitaxel or paclitaxel protein-bound in metastatic squamous NSCLC, KEYTRUDA was discontinued due to adverse reactions in 15% of 101 patients. The most frequent serious adverse reactions reported in at least 2% of patients were febrile neutropenia, pneumonia, and urinary tract infection. Adverse reactions observed in KEYNOTE-407 were similar to those observed in KEYNOTE-189 with the exception that increased incidences of alopecia (47% vs 36%) and peripheral neuropathy (31% vs 25%) were observed in the KEYTRUDA and chemotherapy arm compared to the placebo and chemotherapy arm in KEYNOTE-407.

In KEYNOTE-042, KEYTRUDA was discontinued due to adverse reactions in 19% of 636 patients; the most common were pneumonitis (3%), death due to unknown cause (1.6%), and pneumonia (1.4%). The most frequent serious adverse reactions reported in at least 2% of patients were pneumonia (7%), pneumonitis (3.9%), pulmonary embolism (2.4%), and pleural effusion (2.2%). The most common adverse reaction (20%) was fatigue (25%).

In KEYNOTE-010, KEYTRUDA monotherapy was discontinued due to adverse reactions in 8% of 682 patients with metastatic NSCLC; the most common was pneumonitis (1.8%). The most common adverse reactions (20%) were decreased appetite (25%), fatigue (25%), dyspnea (23%), and nausea (20%).

Adverse reactions occurring in patients with SCLC were similar to those occurring in patients with other solid tumors who received KEYTRUDA as a single agent.

In KEYNOTE-048, KEYTRUDA monotherapy was discontinued due to adverse events in 12% of 300 patients with HNSCC; the most common adverse reactions leading to permanent discontinuation were sepsis (1.7%) and pneumonia (1.3%). The most common adverse reactions (20%) were fatigue (33%), constipation (20%), and rash (20%).

In KEYNOTE-048, when KEYTRUDA was administered in combination with platinum (cisplatin or carboplatin) and FU chemotherapy, KEYTRUDA was discontinued due to adverse reactions in 16% of 276 patients with HNSCC. The most common adverse reactions resulting in permanent discontinuation of KEYTRUDA were pneumonia (2.5%), pneumonitis (1.8%), and septic shock (1.4%). The most common adverse reactions (20%) were nausea (51%), fatigue (49%), constipation (37%), vomiting (32%), mucosal inflammation (31%), diarrhea (29%), decreased appetite (29%), stomatitis (26%), and cough (22%).

In KEYNOTE-012, KEYTRUDA was discontinued due to adverse reactions in 17% of 192 patients with HNSCC. Serious adverse reactions occurred in 45% of patients. The most frequent serious adverse reactions reported in at least 2% of patients were pneumonia, dyspnea, confusional state, vomiting, pleural effusion, and respiratory failure. The most common adverse reactions (20%) were fatigue, decreased appetite, and dyspnea. Adverse reactions occurring in patients with HNSCC were generally similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy, with the exception of increased incidences of facial edema and new or worsening hypothyroidism.

In KEYNOTE-087, KEYTRUDA was discontinued due to adverse reactions in 5% of 210 patients with cHL. Serious adverse reactions occurred in 16% of patients; those 1% included pneumonia, pneumonitis, pyrexia, dyspnea, GVHD, and herpes zoster. Two patients died from causes other than disease progression; 1 from GVHD after subsequent allogeneic HSCT and 1 from septic shock. The most common adverse reactions (20%) were fatigue (26%), pyrexia (24%), cough (24%), musculoskeletal pain (21%), diarrhea (20%), and rash (20%).

In KEYNOTE-170, KEYTRUDA was discontinued due to adverse reactions in 8% of 53 patients with PMBCL. Serious adverse reactions occurred in 26% of patients and included arrhythmia (4%), cardiac tamponade (2%), myocardial infarction (2%), pericardial effusion (2%), and pericarditis (2%). Six (11%) patients died within 30 days of start of treatment. The most common adverse reactions (20%) were musculoskeletal pain (30%), upper respiratory tract infection and pyrexia (28% each), cough (26%), fatigue (23%), and dyspnea (21%).

In KEYNOTE-052, KEYTRUDA was discontinued due to adverse reactions in 11% of 370 patients with locally advanced or metastatic urothelial carcinoma. Serious adverse reactions occurred in 42% of patients; those 2% were urinary tract infection, hematuria, acute kidney injury, pneumonia, and urosepsis. The most common adverse reactions (20%) were fatigue (38%), musculoskeletal pain (24%), decreased appetite (22%), constipation (21%), rash (21%), and diarrhea (20%).

In KEYNOTE-045, KEYTRUDA was discontinued due to adverse reactions in 8% of 266 patients with locally advanced or metastatic urothelial carcinoma. The most common adverse reaction resulting in permanent discontinuation of KEYTRUDA was pneumonitis (1.9%). Serious adverse reactions occurred in 39% of KEYTRUDA-treated patients; those 2% were urinary tract infection, pneumonia, anemia, and pneumonitis. The most common adverse reactions (20%) in patients who received KEYTRUDA were fatigue (38%), musculoskeletal pain (32%), pruritus (23%), decreased appetite (21%), nausea (21%), and rash (20%).

Adverse reactions occurring in patients with gastric cancer were similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy.

Adverse reactions occurring in patients with esophageal cancer were similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy.

In KEYNOTE-158, KEYTRUDA was discontinued due to adverse reactions in 8% of 98 patients with recurrent or metastatic cervical cancer. Serious adverse reactions occurred in 39% of patients receiving KEYTRUDA; the most frequent included anemia (7%), fistula, hemorrhage, and infections [except urinary tract infections] (4.1% each). The most common adverse reactions (20%) were fatigue (43%), musculoskeletal pain (27%), diarrhea (23%), pain and abdominal pain (22% each), and decreased appetite (21%).

Adverse reactions occurring in patients with hepatocellular carcinoma (HCC) were generally similar to those in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy, with the exception of increased incidences of ascites (8% Grades 34) and immune-mediated hepatitis (2.9%). Laboratory abnormalities (Grades 34) that occurred at a higher incidence were elevated AST (20%), ALT (9%), and hyperbilirubinemia (10%).

Among the 50 patients with MCC enrolled in study KEYNOTE-017, adverse reactions occurring in patients with MCC were generally similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy. Laboratory abnormalities (Grades 34) that occurred at a higher incidence were elevated AST (11%) and hyperglycemia (19%).

In KEYNOTE-426, when KEYTRUDA was administered in combination with axitinib, fatal adverse reactions occurred in 3.3% of 429 patients. Serious adverse reactions occurred in 40% of patients, the most frequent (1%) were hepatotoxicity (7%), diarrhea (4.2%), acute kidney injury (2.3%), dehydration (1%), and pneumonitis (1%). Permanent discontinuation due to an adverse reaction occurred in 31% of patients; KEYTRUDA only (13%), axitinib only (13%), and the combination (8%); the most common were hepatotoxicity (13%), diarrhea/colitis (1.9%), acute kidney injury (1.6%), and cerebrovascular accident (1.2%). The most common adverse reactions (20%) were diarrhea (56%), fatigue/asthenia (52%), hypertension (48%), hepatotoxicity (39%), hypothyroidism (35%), decreased appetite (30%), palmar-plantar erythrodysesthesia (28%), nausea (28%), stomatitis/mucosal inflammation (27%), dysphonia (25%), rash (25%), cough (21%), and constipation (21%).

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FDA Oncologic Drugs Advisory Committee (ODAC) Recommends KEYTRUDA (pembrolizumab) for the Treatment of Certain Patients with High-Risk, Non-Muscle...

New technologies that could soon diagnose Alzheimers and restore speech to the paralyzed; potential new avenues in treating diabetes, multiple sclerosis and Down syndrome; and a genetic test that dramatically saved a babys life these were among the science and health topics that most engaged our readers in 2019.

Look back at these 11 stories of the past year or discover them for the first time they reflect the exciting, transformative research that takes place at UC San Francisco every day of the year.

Researchers trained a machine-learning algorithm on nearly 2,000 brain scans and then challenged it to detect early-stage Alzheimers disease in other scans. The algorithm performed with flying colors, catching the disease six years before a clinical diagnosis a lead time that may eventually help doctors treat the disease.

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For California, a 1 percent drop in the smoking rate could mean $630 million of Medicaid savings the following year. Thats because quitting smoking can reduce many health risks relatively quickly, including heart attacks, lung disease and pregnancy complications, as well as cut long-term health risks such as cancer.

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For the first time, researchers were able to transform human stem cells into mature insulin-producing cells similar to the pancreatic beta cells destroyed by type 1 diabetes. The breakthrough came after the team applied a key tenet of biology, that form follows function, to the way they were growing the cells in the lab.

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Researchers took a new approach to studying Down syndrome focusing on the conditions effect on the protein-making machinery inside cells. In a mouse model of Down syndrome, they found that cells in the brain were tamping down on protein production, leading to cognitive deficits. They were able to activate protein production and improve memory and learning with a drug called ISRIB.

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A sophisticated brain-machine interface could one day give voice to people who have lost the ability to speak due to paralysis or other neurological damage. Researchers first mapped participants brain activity to their vocal tract movements as they made various sounds. An algorithm could then translate new brain activity into movements of the virtual vocal tract and produce realistic speech.

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Whats the best way to eat your vegetables? Should you take supplements? Low-fat, low-carb, intermittent fasting do any diets actually work? Experts weigh in on the latest science behind healthy eating and separate food fact from food fiction. Keep these evidence-based tips in mind for the holidays and in the years to come.

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Baby Quincy was deteriorating fast from an aggressive blood cancer and too sick to undergo a stem cell transplant, his only chance at a cure. Determined to leave no stone unturned, Quincys doctors ordered the UCSF500 a new comprehensive cancer gene panel test that helped to reveal an unusual genetic alteration in his cancer and identified a long-shot therapy.

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Multiple sclerosis (MS) is an autoimmune disease caused by immune cells that attack the protective coating around nerve cells. In a surprise, researchers discovered that other immune cells in the gut, known as plasma cells, can reduce the brain inflammation that results from the disease. Expanding these gut plasma cells may be a new therapeutic approach to treating MS.

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Cooking food changes not only how it tastes but how our gut microbial ecosystems respond. In mice, researchers found that cooked vegetables altered their gut microbiome and caused them to lose weight. In human participants, three days of raw or cooked diets prepared by a professional chef also changed gut microbiomes, but in different ways perhaps holding clues to how our microbes have adapted to human culinary culture.

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Alcohol-associated liver disease has become the top reason for U.S. liver transplants, making up more than one-third of liver transplants in 2016. The increase is largely due to a shift away from a common rule that required patients to abstain from alcohol and drug use for at least six months prior to transplant. A 2011 study found that transplants could be successful without this minimum sobriety period, changing the policy at many centers.

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The emotional tumult of teenage years may in part be due to transformations in the brain. Most human brain cells mature in the first years of life, but a group of neurons in the amygdala, which controls emotional responses, dont mature until adolescence and a small number remain immature throughout life. The brain may hold on to these Peter Pan neurons to keep the brains emotional responses flexible and adaptable into old age.

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In 2019, UCSF drove advancements in care delivery, scientific discovery, education, public service, and more. See the highlights

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UCSF's 11 Most Popular Health and Science Stories of 2019 - UCSF News Services

Specializing in innovative cosmetic applications for the face, eyes, and body, Dr. Zamora is a leader in minimally invasive treatments.

DENVER (PRWEB) December 18, 2019

Dr. Jack Zamora, a renowned face expert in Denver, Colorado has joined the esteemed Haute Beauty network.

The Haute Beauty Network, well known for its exclusive and luxurious lifestyle publication Haute Living is privileged to present Dr. Jack Zamora as a face expert and our newest addition to the Haute Beauty members-only network.

Haute Beauty offers a prominent collective of leading doctors. The invitation-only exclusive publication maintains elite as ever, with only two doctors in every market. This partnership allows Haute Beauty to connect its affluent readers with industry-leading aesthetic surgeons located in their area.

ABOUT DR. ZAMORADr. Jack Zamora is an oculofacial plastic surgeon, and a pioneer in plasma treatments and stem cell technology. Specializing in innovative cosmetic applications for the face, eyes, and body, Dr. Zamora is a leader in minimally invasive treatments. Graduating from Tulane University in New Orleans, he received a doctorate degree in medicine and completed his internship at Boston Medical Center (internal medicine), his residency at Boston University (ophthalmology department), and completed his fellowship at Boston University (ophthalmology and oculoplastics).

Dr. Zamora is the medical director of several locations throughout Colorado offering select surgical and non-surgical facial refinement, skin rejuvenation, and body sculpting services. Known for exceptional patient care and state-of-the-art procedures that achieve natural-looking results with as little downtime as possible, Dr. Zamora and his team work with each patient to tailor a combination of treatments for long-term results.

As the creator of J-Plazty, Dr. Zamora has received national and international attention for his revolutionary technique. J-Plazty is a minimally invasive procedure that uses Renuvion plasma energy sub-dermally to instantly tighten and rejuvenate skin anywhere on the face and body without large incisions, downtime, or the complications of traditional surgery. As an authority on skin tightening applications, Dr. Zamora has seen remarkable results with plasma and often combines it with other radiofrequency (RF) modalities for superior rejuvenation. Utilizing his plasma techniques with micro and macro-needling radiofrequency (RF), Dr. Zamora is seeing unparalleled skin shrinkage as well as tightening of extremely delicate tissue allowing for long-term improvement with less downtime

In an effort to improve the outcome of aesthetic procedures, Dr. Zamora has partnered with Vitro BioPharma to develop the worlds first ultra pure cosmetic stem cell serum, InfiniVive MD, to be used topically by plastic surgeons, cosmetic surgeons, and aestheticians throughout the United States. InfiniVive MD is the highest quality cGMP-grade cosmetic stem cell serum containing ultra pure mesenchymal stem cells and exosomes. InfiniVive MD is to be used with ablative and non- ablative lasers, plasma energy technologies, and microneedling radiofrequency. The serum provides an unprecedented improvement in fine lines and wrinkles, helps reduce the signs of aging, and helps promote accelerated healing.

Being an international trainer for J-Plazty, Apyx Medical, and Bausch Health Companies Inc., and a luminary for AMP Medical, Lutronic Medical, and Syneron ELOS, Dr. Zamora offers his expertise to physicians from around the globe. He is a regular speaker and consultant, has been featured on The Doctors TV Show, and has written on the techniques and parameters of soft tissue coagulation and subcutaneous neck skin plasma tightening. Valuing continued education, Dr. Zamora created the Jack Zamora MD Aesthetic Institute, which offers advanced aesthetic training to medical professionals and licensed aestheticians.

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Dr. Jack Zamora Partners with the Exclusive Haute Beauty Network - PR Web

ACLT (THE African Caribbean Leukaemia Trust) has been honoured by blood cancer charity Anthony Nolan, at an awards ceremony held at the Tower of London.

ACLT took home the award for the black, Asian and minority ethnic advocate of the year, in recognition of the work they have done to recruit more donors from these backgrounds to the stem cell register.

The charity is committed to providing hope to patients living with blood cancer and illnesses where a matched donor (stem cell, blood or organ) is required to save a life.

The prestigious Anthony Nolan Supporter Awards were back for their seventh year to recognise the outstanding achievements of the volunteers, fundraisers, and campaigners who help the pioneering blood cancer charity save lives.

Over 23 years, ACLT have recruited thousands of potential donors from African and Caribbean descent to the Anthony Nolan stem cell register, with many people going on to donate to patients in desperate need of a life-saving stem cell transplant.

ACLT was founded IN 1996 by Beverley De Gale OBE and Orin Lewis OBE while they were searching for a matching donor for their 6-year-old son, Daniel.

Daniel had Acute Lymphoblastic Leukaemia and needed a life-saving stem cell transplant, but at the time of his diagnosis in 1993, the number of potential donors on the register from BAME backgrounds was remarkably low. His parents were told Daniel had a 1 in 250,000 change of finding a match.

Beverley and Orin could not sit back and watch this. They got in touch with Anthony Nolan to find out how they could help, and soon after, ACLT was born. From comedy clubs, to churches, to schools, Beverley and Orin organised recruitment events far and wide to reach out to the communities most underrepresented on the stem cell register.

In 1999, Daniel De Gale was the first black person in the UK to receive a stem cell transplant from an unrelated donor. He went on to live a full and busy life, but sadly died from multiple organ failure in 2008, due to complications with his health sustained while waiting for a transplant.

ACLT is Daniels legacy, and his parents have been committed to recruiting donors and raising awareness ever since.

In the last year alone, ACLT have run 13 recruitment events to register potential donors to the Anthony Nolan Stem Cell Register, which have seen over 800 people join. Around 75% of those donors were from BAME backgrounds, and 8 people went on to donate their stem cells to patients in need.

Beverley De Gale OBE said: ACLT have been working in partnership with Anthony Nolan since the charity was formed over 23 years ago. In this time the relationship between ACLT and Anthony Nolan has grown exponentially. Were honoured to have our work of raising awareness and registering lifesaving donors recognised with the prestigious award of BAME Advocate of the Year.

Henny Braund, Chief Executive at Anthony Nolan, said: ACLT is a hugely deserving winner of this award; their incredible support and passion for our work is a fantastic example of our charity, which is built on making lifesaving connections. It was lovely to meet some of ACLTs volunteers at the ceremony and I continue to find myself inspired and humbled by the dedication and strength of supporters like them.

By recruiting potential donors, we are curing blood cancer together. We can give families hope, and give more people a future. But without supporters like ACLT lives cant be saved. Without them, there is no cure.

The awards took place on Thursday 28 November at the Tower of London and were hosted by comedian Nish Kumar.

Anthony Nolan is the charity that finds matching stem cell donors for people with blood cancer and blood disorders and gives them a second chance at life. It also carries out ground-breaking research to save more lives and provide information and support to patients after a stem cell transplant, through its clinical nurse specialists and psychologists, who help guide patients through their recovery.

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ACLT wins National Charity Award - The Voice Online

Nonalcoholic steatohepatitis (NASH) is the most severe form of non-alcoholic fatty liver disease. It is diagnosed by conditions such as a fatty liver, liver cell damage, and inflammation. If it is not treated on time, NASH can lead to further complications such as liver fibrosis. As it is essential to cure NASH in real-time, many companies focus on the same and Promethera Biosciences based in Wallonia, Belgium is one of them.

Take a look at winners of EIT Digital Challenge 2019.

Belgian biotech startup Promethera Biosciences is one of the leading innovators in the world specialised in liver therapeutics. The company operates with the mission to bring life-saving treatments that will reduce the need for liver transplantation. It is one of the pioneers in the development of cell-based therapies that will provide both anti-fibrotic and immunomodulatory effects in the liver.

Promethera Biosciences just announced that it secured 7.5 million additional Series D funding, which adds to the recently raised 39.7 million funding. This investment was led by Sony Innovation Fund of IGV and Pegasus Tech Ventures along with MEDIPAL Holdings, the family office Six Snow, a Japanese private investor and a Belgian private investor.

With the fresh round of investment, Promethera Biosciences aims to advance its clinical programs in NASH (Non-alcoholic steatohepatitis) and ACLF (Acute-on-Chronic Liver Failure). Besides these advancements, the Belgian startup also plans to accelerate growth in the Asian markets. It is in plans to initiate clinical trials outside of Europe in NASH and ACLF in 2020.

Promethera Biosciences lead clinical program is derived from its patented allogenic live-cell platform dubbed HepaStem. The biotech startup has developed its cell therapy technologies using allogeneic stem and progenitor liver cells that are isolated, expanded, and purified from healthy human livers unsuitable for transplantation.

Besides the cell-based therapies, this startup also develops antibody technologies such as the anti TNF-R1 antibody Atrosimab in order to complement and diversify the therapeutic options.

Main image picture credits: Promethera Biosciences

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Promethera Biosciences, a Belgian startup that works on treating liver diseases secures funding - Silicon Canals

WeTheTrillions wants to make it easier for people - especially women - to eat healthier, with an ... [+] emphasis on fruits, vegetables, and other plant-based foods.

Its long been said that food is medicine, but the healthcare system doesnt always treat it as such. Despite the fact that we need to eat every day, multiple times a day, just to stay alive, medical schools in the U.S. provide on average only 19 hours of nutritional education throughout a doctors training.

As a result, your doctor may be able to prescribe you a medicine to treat high blood pressure, diabetes, or any number of other conditions, but they simply might not know how to advise you to try to prevent illness in the first place.

But some folks are trying to change that. WeTheTrillions is a public benefit corporation that is approaching nutritious food as not just part of a healthy lifestyle, but as one of the first to leverage technology and prescription-grade delivered meals as the frontlines of preventative healthcare to stop the epidemic of chronic conditions touching more than 60 percent of adults in the US. The company is named for the trillions of microbes in each of our gut biomes along with trillions of cells, which make up the central focus of the companys food-as-medicine because of the gut biomes role in well, virtually every part of our lives and bodies.

WeTheTrillions main product is ready-to-eat customized meal and snack options, and those who subscribe to their weekly meal plans also have access to health specialists to discuss and stay on top of their goals and concerns. They also provide software to clinicians so they can track progress and help patients reach tangible results through a customized food program. This could be for patients suffering from IBS, diabetes, anemia or any other female-related hormonal imbalances.

According to the Centers for Disease Control and Prevention, 50 percent of doctors recommendations to patients with chronic illnesses are not followed and this number goes up to more than 70 percent when it comes to lifestyle changes like dietary guidelines, says Lamiaa Bounahmidi, Founder and CEO of WeTheTrillions. Closing that gap is already making WeTheTrillions an appealing solution to hundreds of doctors across the US who see this as an immediate intervention to leverage theory and practice for preventative healthcare and help save trillions of dollars in healthcare bills - effectively staunching the leading public health crisis of the 21st century: chronic disease.

To know your eligibility for a WeTheTrillions program, you first start by filling out a 3-minute online intake quiz and then have a 20-min call with a health specialist to fully customize your plan and agree on clear target biomarkers levels to reach. The process is based on an aggregation of evidence-based randomized controlled trials to provide a fully science-driven approach beyond fad diets.

Notably, theyre also looking to set up machines in urban and rural food deserts that offer affordable options via subsidization through the Supplemental Nutrition Assistance Program and other government assistance programs. And all the food is 100% plant-based, a choice justified by medical evidence that currently points to a veggie-heavy diet for its abilities to fight heart disease, diabetes, and strokes as well as hormonal balance and gut health.

But their approach to food as preventative medicine is more specific and science-backed than simply offering healthy plant-based foods. While the meals are, theoretically, for anyone looking to eat well and improve their health, much of the emphasis is on using food to treat Polycystic ovary syndrome, menopause symptoms, fertility, and other issues pertaining to womens reproductive health. Even the general health issues WeTheTrillions seeks to address, like anemia, often disproportionately affect women, especially women of color.

Theres a reason to believe that this method of specifically targeting womens reproductive and general health could be an effective strategy in improving the overall health of society. Overwhelming evidence links maternal health to that of children, which together affect the overall health of society. A society with healthy women is more likely to have healthy children who will grow into healthy adults. WeTheTrillions emphasis on the health of female-sexed people is not exclusive, but it is strong and forward-thinking.

There are a plethora of premade or prepped meal kits on the market now, many of which are vegan, organic, and health-focused. Not all of them, however, can claim to have the medical emphasis of WeTheTrillions, with its specific issue-based meal plans and grand focus on making food accessible via work, school, physicians, and public assistance.In a way, its simplicity is inspiring: to improve the health of society at large, eat plants. The work of doctors, scientists, and public health experts boil down to some tasty vegetables on your plate customized to your unique health needs.

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WeTheTrillions Says The Future Of Public Health Is Female And Plant-Based - Forbes