This fall, 21-year-old Jurnee Farrell, a Howard University senior and a member of the universitys volleyball team donated stem cells to a complete stranger.

This fall, 21-year-old Jurnee Farrell, a Howard University senior and a member of the universitys volleyball team, was set to play in the Mid-Eastern Athletic Conference Tournament.

But instead, she was sidelined by a decision she made herself.

Two years ago, she signed up with Be the Match, a nonprofit that registers potential bone marrow and stem cell donors. When she got the call that she was a match for a 57-year-old woman with a form of leukemia, she was surprised.

At first I was like, This isnt real, Farrell recalled, but then said her decision was clear. She would follow through on the commitment she made two years ago when she signed up.

That meant undergoing a series of shots five days before the outpatient procedure, and then undergoing apheresis, a process in which the donor has blood removed through a needle in one arm, blood-forming cells are collected, and then the blood is returned through a needle in the other arm. The session can take up to eight hours.

Farrell said that the actual donation wasnt bad, but that the shots given in a series five days prior proved a little uncomfortable.

Nevertheless, she urges potential donors to sign up.

Its really not that bad, and the person whose life youre saving is probably going through so much more than you are, she said.

And while Farrell missed the tournament last month, shes back on the court already. We are going to the NCAA tournament this weekend, actually, and we play Pitt on Friday, she said. I just started practicing last week!

Farrells decision to register as a donor is one for which the staff at Be the Match is especially grateful.

Lauren Mueller, a public relations specialist with Be the Match, explained that for Caucasian patients waiting for a bone marrow or stem cell donation, the odds of finding a match are roughly 70%. For African-American patients, the odds are much lower, at 23%.

As a result, we are always looking to increase our diversity on the registry, said Mueller, who encourages people to consider registering.

It starts with a cheek swab, and Mueller said its not uncommon for years to go by before a potential donor hears that they might be a match, just as it was in Farrells case.

Your selfless action can help save a life, Mueller said.

Farrell said that shes hopeful her donation will prove successful, and that she would love to meet the recipient one day.

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Howard University athlete makes the call to donate stem cells - WTOP

I have been reading a book, The Man Who Wouldn't Die, that satirizes Silicon Valley and the venture-capital start-up culture. It's reasonably funny. I was reminded, however, that sometimes culture is crazy.

At one point a character casually describes how stem-cell therapy is obviously going to make 50-year-olds feel like 20-year-olds. Grow a new liver and you're good to go, so to speak! The author clearly meant the idea as a joke. However, that idea is out there and taken seriously. Stem cells made national headlines in the late '90s and early 2000s because of some scientific breakthroughs and their promise for future medical treatments.

Just about all of our body is made up of specialized cells. These are cells that have grown to perform some specific function. Some examples are heart muscle cells, say, or red blood cells.

Stem cells are different; they have not yet specialized. That is, they have not developed many of the specific properties that are unique to the different parts of our bodies.

Given the right environment the right chemicals, for examplethey can grow into specialized cells.

There are, of course, all sorts of important details for how stem cells grow, when they specialize and how much flexibility they have in specializing. Somehow, however, there is now an industry that has skipped all that work and is marketing stem cells as a general cure. Alzheimer's disease and joint pain are frequently mentioned. If that makes you suspicious that these clinics are targeting the elderly, you would be right.

A typical treatment involves taking stem cells from a patient's bone marrow or fat and injecting it back into a sore knee or hip or whatever needs fixing. The idea, somewhat vaguely, is that the stem cells will grow to replace whatever is worn down by age.

As is true of all good scams, this one has a good story, one that can pass as proven medicine for many patients. The treatment also benefits by treating a problem with symptoms that can come and go irregularly, so a patient can honestly claim that they recovered in, say,six months.

The body is a complicated machine. It is hard to predict what will cure or not cure any specific problem. Just because a story involving stem cells sounds reasonable is not a sufficient reason to try something.

For a typical patient, we should have good, strong, positive evidence that a treatment will help. A good story is not strong, positive evidence. In the language of science, it is a hypothesis. Potentially true.

It is worth remembering that many, many things are potentially true. We don't try them all out on ourselves when we are sick.

Many of these ideas have in fact been tested in rigorous, peer-reviewed studies. So far, the evidence for positive effects is weak.

One reason these clinics have managed to skirt the rules is that extracting material from a patient then reinjecting requires less oversight for safety. Depending on the details, this treatment is considered, for Food and Drug Administration regulations, something like plasma donation (where blood is extracted, platelets removed, then reinjected into the body).

Just because a process may be safe does not make it good medicine. Medicine is not and should not be a free market. Patients will almost never have enough knowledge to reasonably choose between treatment options.

The FDA is the federal agency in charge of ensuring companies do not offer useless or harmful procedures. Unfortunately, in 2017 the FDA decided to allow stem cell clinics a three-year grace period to describe their procedures for the FDA's evaluation. As a result of that open window, hundreds of clinics have opened nationwide, offering services for which there is no strong evidence.

In the past year the FDA has realized where the industry has gone with this treatment and begun trying to crack down. Some clinics have unsafe procedures; others are misleading patients about treatments. Many clinics are still operating, unfortunately, and it can be difficult for typical patients to recognize the difference between these treatments and proven remedies. Furthermore, these bad actors could potentially make people skeptical of stem cell-based technology entirely.

That would be a shame because there is still great promise for stem cells. That may be cold comfort for those who want a treatment immediately, but that desire is the exact motivation that has led to terrible medicine for centuries.

Christer Watson, of Fort Wayne, is a professor of physics at Manchester University. Opinions expressed are his own. He wrote this column for The Journal Gazette, where his columns normally appear the first and third Tuesday of each month.

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Stem of the problem | Columns | Journal Gazette - Fort Wayne Journal Gazette

The latest market report published by Credence Research, Inc.GlobalBlood and Bone Marrow Cancer Treatment Market Growth, Future Prospects, Competitive Analysis, 2019- 2027,the global market for the treatment of blood and bone marrow cancer was valued at US$ 38.8 billion in 2018 and is expected to reach US$ 74.9 billion by 2027, increasing to CAGR by 7.7% from 2019 to 2027.

Market Insights

Blood cancer begins in the bone marrow, an integral source of stem cells that are later differentiated between different types of blood cells in the human body. Researchers at Bristol Myers Squibb Company have indicated that around 1.85 million new cases of blood cancer will be diagnosed worldwide by 2040.

Browse the fullreport athttps://www.credenceresearch.com/report/blood-and-bone-marrow-cancer-treatment-market

Lymphoma is the largest indication in the blood and bone marrow cancer treatment market. It is prevalent across the globe in two types of Hodgkin lymphoma and non-Hodgkin lymphoma. The main variables responsible for increasing prevalence worldwide are increasing the prescription of immunosuppressive drugs for the treatment of chronic infections and genetic mutations. Leukemia occurs when the DNA of immature white blood cells is damaged by exposure to ionizing radiation, hazardous chemicals, smoking, etc. The prevalence rate of leukemia is highly variable across different ethnic groups with a male-to-female ratio of 1.4.

Chemotherapy is part of the blood and bone marrow cancer therapy market. The key factor responsible for increasing demand is the availability of its generic version at an affordable cost, which significantly reduces the health burden on sick patients. Oncologists prefer to use them in combination therapy with either radiotherapy or immunotherapy to treat patients with resistance to first-line drug therapy. Immunotherapy will be the fastest growing segment in the forecast period due to its promising drug line for the treatment of blood cancer.

North America representing a market share of 34.6% is dominating the regional segment for blood and bone marrow cancer treatment market. The chief contributing factor for its market supremacy is growing incidence of blood cancer. As per the research citings of the Leukemia and Lymphoma Society (CDC) figures after every 3 minutes 1 person in the U.S. is diagnosed with blood cancer. In 2019, approximately 176,200 people in the U.S. are diagnosed with blood cancer in the United States. Existence of key players such as F.Hoffman La-Roche Ltd., Pfizer, Inc., Novartis AG, Merck & Co., Inc., etc further strengthens the market growth. Europe holds a market share of 30.8% owing to the supportive regulatory framework provided by the European Medical Agency for the development and sale of medication for the treatment of blood cancer. Asia Pacific accounts for 18.4% market share on account of rising public health awareness related to blood cancer & its treatment and developing healthcare infrastructure.

Pharmaceutical companies spearheading the blood and bone marrow cancer treatment market are Bristol Myers Squibb & Company, AstraZeneca, Plc., Eli Lilly & Company, Johnson and Johnson Company, F.Hoffman La-Roche Ltd., Celgene, Inc., Merck & Co., Inc., Novartis AG, Varian Medical Systems, Inc. and Pfizer, Inc.

Credence Research is providing Free Sample Get it here: https://www.credenceresearch.com/sample-request/59845

Key Market Movements:

Report Scope by Segments

By Indication Type

By Therapy Type

By Geography Segment

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Blood and Bone Marrow Cancer Treatment Market Is Expected To Reach US$ 74.9 Bn by 2027 - Credence Turbine

WASHINGTON WhenHoward University student athlete Jurnee Farrellsigned up for the Be The Match registry during a Get In The Game campus drive, she didnt know if she would ever be called upon. However, when her phone rang two years later and she discovered she was a match for a 57-year-old woman suffering from acute lymphoblastic leukemia, she did not hesitate to participate.

The football team was hosting a Be The Match booth after Coach London had donated bone marrow to his daughter, recalls Farrell. I filled out a little form and turned it in. When I got the call a few months ago, they asked if I was still interested in donating. I didnt hesitate to say yes because this is somebodys life and there was no way I could say no to that.

Farrell is a senior criminology major from Denver, Colorado, and a member of the Howard University MEAC Championship Volleyball team. Wearing jersey No. 5 as a defensive specialist, Farrell is known for her bubbly personality off the court and her intensity on the court. At senior night, the day before her donation, the entire team rallied behind her in support of her decision. Unfortunately, the timing of the donation process meant that Farrell was not able to participate in this years MEAC playoffs with her teammates, who brought home their fifth MEAC championship on Nov. 24.

Of course, we were sad to miss out on having Jurnee play in the playoffs, but this is such a worthy cause and we were happy to support her all the way, saysHead Volleyball Coach Shaun Kupferberg.What she is doing speaks directly to Howard Universitys mission of truth and service, that each student comes here not only to learn, but to make a difference in the global community. Im extremely proud of her decision.

Thanks to several medical advances, the process to donate stem cells has drastically changed over the years. In addition to bone marrow donations, doctors can also use a stem cell procedure called Peripheral Blood Stem Cell (PBSC) donation through a short 4-hour out-patient procedure where blood is circulated from one arm, into a machine and then back into the donor.

Beth Carrion, account manager for Registry Growth and Development, says individuals like Farrell help to demystify the giving process and raise awareness of the need for a more diverse donor registry. The chances of finding a match for a stem cell transplant is dependent on a persons genetic markers. Outside of a family member, finding a donor within in ones ethnicity is the next viable option. According to Be The Match, each year approximately 14,000 patients are waiting for a transplant from someone outside of their family. The current odds to match a patient with a donor in one out of 430.

Be the Match is truly thankful for our partnership with Howard University because it plays a vital role in helping the African American community have a higher rate of finding a match, says Carrion. A white person in the registry has a 78 percent chance of finding a match. For Hispanics, its 46 percent, but for African Americans, its only a 23 percent chance. We look forward to hosting more events with Howard this spring.

One week after her procedure, Farrell is back at volleyball practice with her fellow teammates, preparing for the Tournament. As she looks to finish out her senior year, she says shes also hopeful that shell get to meet the woman she helped one day.

After the donation, I can have anonymous communication, but I cannot tell my identity. After a year, they will deem the transplant successful and then we can communicate, says Farrell. I for sure want to meet her.

To join the Howard University registry, text Howard to 61474, follow the prompts and a kit will be mailed to you. You may also register online atjoin.bethematch.org/howard.

# # #

Media Contact: Alonda Thomas,Alonda.Thomas@Howard.edu

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Howard University Student Gives a Stranger the Greatest Gift for the Holidays: Life - Howard Newsroom

Hes done it - Stephen Armstrong has scooped a prestigious award for his huge fund raising efforts as his son fought a rare blood disorder.

The doting dad took home the award for Individual Fundraiser of the Year at the Anthony Nolan Supporter Awards 2019 ceremony held at the Tower of London.

The top 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.

And the award is in recognition of Stephens incredible fundraising efforts - leading a group of 19 friends and family in a series of physical challenges, all while his son Jacob was undergoing treatment.

When Jacob was diagnosed in 2017 at two-years-old, Stephen set out to find a matching stem cell donor, as well as raise awareness of the need for more people on the register.

From here Jacobs Journey was born, and through a series of challenges including the Great North Run, the Great North Bike Ride and climbing Ben Nevis, Stephen has helped raise over 20,000 for the charity.

Jacob is now four-years-old and his family have been told he does not need a transplant, but Stephen and his family want to continue raising awareness for others who arent so lucky.

When Jacob was diagnosed, we were stunned by how few people were on the stem cell donor register. I couldnt believe how a stranger in the street could potentially save our little boys life, said Stephen, 33, of Wallsend,North Tyneside.

On winning the award, Stephen said: I feel very proud- I really didnt expect it. You dont do it for recognition, but to get more people to join the register.

Stephen and mum Kirsty, 28, received the news in December 2017 that Jacob was suffering from bone marrow failure, which affects between 30 and 40 children each year.

They first became concerned about his health when they went abroad to get married and noticed he was getting bruised easily. The marks would take weeks to disappear, so when the couple returned to the UK they decided to take Jacob to the doctor for a check up.

After tests he was then diagnosed and was treated at the Great North Childrens Hospital in Newcastle, where he received two blood transfusions.

Stephen, who has raised a further 8,000 for other smaller charities, added: When we were told Jacob did not need the transplant it was the best news in the world, a total relief. He still needs check ups every three months and his consultants is keeping an eye on him. There are so few people on the stem cell donor register so I just wanted to create a ripple effect with awareness and get more people on it.

Henny Braund, Chief Executive at Anthony Nolan, said: Stephen is a hugely deserving winner of this award; his 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 Stephen and I continue to find myself inspired and humbled by the dedication and strength of supporters like him.

By raising vital funds and much needed awareness, we are curing blood cancer together. We can give families hope, and give more people a future. But without supporters like Stephen, lives cant be saved. Without him, there is no cure.

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.

To see the full shortlist, and find out more about the charity visit the website here.

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Wallsend dad who called on the public for stem cells for his son scoops top Anthony Nolan Award - Chronicle Live

2 December 2019

Stem cell therapies may become redundant in repairing cardiac function after a heart attack, suggests a new study in mice.

It showed how stem cell treatments can heal hearts by triggering an immune response which can be achieved by using a chemical instead.

'This work is paradigm-shifting because it demonstrates a mechanism to explain a perplexing phenomenon that has intrigued cardiologists as a result of decades of cardiac stem cell trials,' Dr Jonathan Epstein at the University of Pennsylvania's Perelman School of Medicine in Philadelphia told The Scientist.

Stem cell therapies to repair damaged heart tissue are currently being tested in human clinical trials. In these treatments, human stem cells are injected into the heart and this leads to an improvement in heart function. However, how this works is not fully understood.

One possibility is that the injected stem cells are incorporated into the heart tissue and repair the damage. However, the latest study, published in the journal Nature, suggests that this may not be the case. Instead, the study indicated that the repair is actually a result of triggering the innate immune response.

Researchers injected different types of stem cell or a chemical inducer (zymosan) of the innate immune response into an experimental mouse model of heart disease. They saw improvement in heart function that was similar in all cases, and showed that this repair occurs via activation of macrophage cells of the innate immune system.

'The innate immune response acutely altered cellular activity around the injured area of the heart so that it healed with a more optimised scar and improved contractile properties,' said Dr Jeffery Molkentin at the University of Cincinnati and Cincinnati Children's Hospital Medical Centre, Ohio, who led the study. 'The implications of our study are very straightforward and present important new evidence about an unsettled debate in the field of cardiovascular medicine.'

The work could open up new possibilities for optimising the treatments currently in development, as well as alternative new therapies.

'If there is a chemical off-the-shelf, it would be a much more feasible therapy [than stem cell transplants],'Dr Kory Lavine at Washington University in St Louis, Missouri, told Nature News.

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Stem cells may trigger immune repair to mend hearts - BioNews

Collagen seems to be one of the buzziest words in wellness, with just about anyones ears perked up at the mention of the wordno matter how young they are. Because when it comes to healthy skin aging, we know that prevention is everything, and delaying the process before it starts is key to maintaining a smooth, even complexion well into the autumn of life.

Enter all the collagen-promoting creams, serums, supplements, and diets the world has to offerand now, a new drug may do just the trick.

A new study published in the journal, Geroscience, found a new, unexpected formula to help with healthy aging. This specific drugcalled rapamycinhas been primarily used for patients who have undergone an organ transplant, as it helps prevent the immune system from rejecting the new organ. Rapamycin has also been used to combat a rare lung disease called lymphangioleiomyomatosis, as well as inhibiting tumor growth.

Excerpt from:
Study Finds This Medication May Reduce Wrinkles & Even Skin Tone - mindbodygreen.com

Soon youll be able to cover your imperfect flesh with more flesh. (Photo: KaoJapan)

FOX NEWS - Soon youll be able to cover your imperfect flesh with more flesh.

Japanese cosmetics company Kao Corporation has developed a custom synthetic spray-on skin to cover unwanted blemishes, moles or other marks on the natural epidermis.

READ MORE ON FOXNEWS.COM

The artificial product, called est, is composed of tiny, liquid fibers. When sprayed, the substance adheres to human skin, transforming into an extremely thin, derma-like material, the Daily Mail reports.

It has a similar elasticity to skin, and its porous, too. Water vapor and air can pass through this second skin to moisten the living dermis beneath. At its edges, est forms an even thinner bond, helping it blend in with natural flesh.

Est is set to hit the market exclusively in Japan beginning Dec 4. and will sell for roughly $532 as a diffuser and potion combination, with diffuser refills priced at $73. A lotion version will sell for $110, and everything will become available online in January, according to Japanese publication the Asahi Shimbun.

Japanese-language advertisements for the product call it Future Skin, which uses Fine Fiber Technology. Kao has plans to expand the line beginning next year and hopes to soon enter the medical market.

Until then, American consumers can check out the SkinGun by RenovaCare, which shoots a liquid mist infused with human stem cells and can help burn victims skin.

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Makeup brand offers spray-on 'skin' to cover up zits and scars - FOX 10 News Phoenix

This picture shows a spirited flying Taiwan Blue Magpie displaying a full array of flight feathers in action. Credit: Shao Huan Lang

If you took a careful look at the feathers on a chicken, youd find many different forms within the same birdeven within a single feather. The diversity of feather shapes and functions expands vastly when you consider the feathers of birds ranging from ostriches to penguins to hummingbirds. Now, researchers reporting in the journal Cell on November 27, 2019, have taken a multidisciplinary approach to understanding how all those feathers get made.

We always wonder how birds can fly and in different ways, says corresponding author Cheng-Ming Chuong of the University of Southern California, Los Angeles. Some soar like eagles, while others require rapid flapping of wings like hummingbirds. Some birds, including ostriches and penguins, dont fly at all.

This picture shows a the asymmetric vane and tapering main shaft of a single flight feather from a goshawk. Credit: Hao Howard Wu and Wen Tau Juan

Such differences in flight styles are largely due to the characteristics of their flight feathers, Chuong adds. We wanted to learn how flight feathers are made so we can understand nature better and learn principles of bioinspired architecture.

In the new study, the researchers put together a multidisciplinary team to look at feathers in many different ways, from their biophysical properties to the underlying molecular biology that allows their formation from stem cells in the skin. They examined the feathers of flightless ostriches, short-distance flying chickens, soaring ducks and eagles, and high-frequency flying sparrows. They studied the extremes by including hummingbirds and penguins. To better understand how feathers have evolved and changed over evolutionary time, the team also looked to feathers that are nearly 100 million years old, found embedded and preserved in amber in Myanmar.

Based on their findings, the researchers explain that feathers modular structure allowed birds to adapt over evolutionary time, helping them to succeed in the many different environments in which birds live today. Their structure also allows for the specialization of feathers in different parts of an individual birds body.

The flight feather is made of two highly adaptable architectural modules: the central shaft, or rachis, and the peripheral vane. The rachis is a composite beam made of a porous medulla that keeps feathers light surrounded by a rigid cortex that adds strength. Their studies show that these two components of the rachis allow for highly flexible designs that enabled to fly or otherwise get around in different ways. The researchers also revealed the underlying molecular signals, including Bmp and Ski, that guide the development of those design features.

Attached to the rachis is the feather vane. The vane is the part of the feather made up of many soft barbs that zip together. The researchers report that the vane develops using principles akin to paper cutting. As such, a single epithelial sheet produces a series of diverse, branched designs with individual barbs, each bearing many tiny hooklets that hold the vane together into a plane using a Velcro-like mechanism. Their studies show that gradients in another signaling pathway (Wnt2b) play an important role in the formation of those barbs.

To look back in time, the researchers studied recently discovered amber fossils, allowing them to explore delicate, three-dimensional feather structures. Their studies show that ancient feathers had the same basic architecture but with more primitive characteristics. For instance, adjacent barbs formed the vane with overlapping barbules, without the Velcro-like, hooklet mechanism found in living birds.

Weve learned how a simple skin can be transformed into a feather, how a prototypic feather structure can be transformed into downy, contour, or flight feathers, and how a flight feather can be modulated to adapt to different flight modes required for different living environments, Chuong says. In every corner and at different morphological scales, we were amazed at how the elegant adaption of the prototype architecture can help different birds to adapt to different new environments.

The researchers say that, in addition to helping to understand how birds have adapted over time, they hope these bioinspired architectural principles theyve uncovered can be useful in future technology design. They note that composite materials of the future could contribute toward the construction of light but robust flying drones, durable and resilient wind turbines, or better medical implants and prosthetic devices.

Team co-leader and biophysicist Wen Tau Juan of the Integrative Stem Cell Center of China Medical University Hospital, Taiwan, has already begun to explore the application of feather-inspired architectural principles in bio-material design. The team also hopes to learn even more about the molecular signals that allow the formation of such complex feather structures from epidermal stem cells that all start out the same.

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Reference: The Making of a Flight Feather: Bio-architectural Principles and Adaptation by Wei-Ling Chang, Hao Wu, Yu-Kun Chiu, Shuo Wang, Ting-Xin Jiang, Zhong-Lai Luo, Yen-Cheng Lin, Ang Li, Jui-Ting Hsu, Heng-Li Huang, How-Jen Gu, Tse-Yu Lin, Shun-Min Yang, Tsung-Tse Lee, Yung-Chi Lai, Mingxing Lei, Ming-You Shie, Cheng-Te Yao, Yi-Wen Chen, J.C. Tsai, Shyh-Jou Shieh, Yeu-Kuang Hwu, Hsu-Chen Cheng, Pin-Chi Tang, Shih-Chieh Hung, Chih-Feng Chen, Michael Habib, Randall B. Widelitz, Ping Wu, Wen-Tau Juan and Cheng-Ming Chuong, 27 November 2019, Cell.DOI: 10.1016/j.cell.2019.11.008

This work was supported by the ISCC, CMUH, Taiwan, the Drug Development Center, CMU, Higher Education Sprout Project, Ministry of Education (HESP-MOE), and grants from the National Institutes of Health, Ministry of Science and Technology, Taiwan, iEGG/Avian Genetic Resource/ABC supported by HESP-MOE, the Human Frontier Science Program, the National Natural Science Foundation of China, NSFC, Academia Sinica Research Program on Nanoscience and Nanotechnology, Top Notch Project, NCKU, and a University Advancement grant by MOE, Taiwan.

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How Flight Feathers Evolved: Study of Chickens, Ostriches, Penguins, Ducks and Eagles - SciTechDaily

Were always happy to geek out over a buzzyskin careingredient when we see it making the rounds on product labels. (See: lactic acid, rosehip oil, bakuchiol) So when we started noticing the proliferation of niacinamide, we were surprised to learn that not only has it been around for a while buttheres a decent body of research behind the multipurpose vitamin. Heres what you need to know about niacinamidesbenefits for your skin.

Niacinamide, a form of vitamin B3 also known as nicotinamide, is a water-soluble vitamin that has potent antioxidant activity and reduces inflammation, saysboard-certified dermatologistDavid Lortscher, CEO of Curology.

It would be an exaggeration to call niacinamide a cure-all, but it does have a pretty extensive range when it comes to the conditions it can treat: acne, oil regulation, fine lines and wrinkles, hyperpigmentation, enlarged pores and sun damage. Its especially good at repairing skins moisture barrier (aka its first line of defense) and protecting against environmental stressorsits even been shown to help prevent skin cancer in certain studies.

Niacinamides nourish and calm redness and inflammation,says DendyEngelman, a board-certified dermatologist in New York. She particularly likes niacinamide for dry and sensitive skin: It has similar effects to retinol by strengthening the skin barrier, but itfortifies from the get-go without sensitivity or irritation.Dr. Lortscher also has high praise: Because of its role in repairing the skin barrier, niacinamide is one of the most effective treatments for photoaging [damage caused by UV rays], according to most anti-aging research.

It starts to get technical here, but as Dr. Engelman explains it, Niacinamide helps support the cellsmetabolic system, specifically fibroblasts. We use fibroblasts tomake and repair DNA,which,in turn, activates collagen production. So by using niacinamides to boost fibroblast production, we are supporting collagen production and repairing damaged collagen.

Lots of products contain niacinamideserums, moisturizers, even cleansersand it works well in conjunction with other active ingredients, like retinol. It can be used both morning and night, though as with any goodskin careregimen, you should follow it up with a sunscreen during the day.

Niacinamide should be compatible with most otherskin careproducts and is well tolerated by all skin types,including sensitiveskin, Dr. Lortscher says. For best results, use leave-on products with niacinamide. Its safe to use around the eyes, anditmay improve the appearance of under-eye darkness and wrinkles.

Convinced yet? Check out a few of our favorite products containing the powerhouse ingredient below.

RELATED: We Ask a Derm: What Ingredients Should You Avoid If You Have Oily Skin?

Of course, the uber-popular, wallet-friendly brand is on top of it. This serum is especially helpful for congested, acne-prone skin: The niacinamides anti-inflammatory propertiescalm active breakouts, while its oil-regulating properties (and the addition of zinc, which also keeps oil in check) help keep new ones from forming.

Buy it ($6)

Nia 24 uses a patented form of niacinamide thats designed to absorb better into skin (and therefore work its magic more effectively). This rich cream strengthens the skin barrier with its namesake ingredient, plus hyaluronic acid, licorice root extract, peptides and ceramides.

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Give parched, dull skin a quick pick-me-up with a five-star-rated gel sheet mask. Reviewers rave about its glow-inducing, hydrating properties and the fact that its gentle enough for sensitive skin.

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Cursed by the ghosts of pimples past? Niacinamide, glycolic acid and NASA-developed plant stem cells (!) work together to combat hyperpigmentation and scarring.

$20 on Amazon

Derms, dry-skinned gals and makeup lovers alike know oil cleansers are a godsend forwashing off the days makeup withoutstripping any precious natural moisture. This cleanser amps up the effects with niacinamides barrier-strengthening effects, plus offers a gentle exfoliation thanks to fruit enzyme.

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SkinCeuticals serums are cult faves for a reason, and this 5 percent niacinamide serum is no exception. Its amped up with amino acids, algae extract and peptides to target the effects of environmental stress and promote collagen production.

Buy it ($112)

RELATED: The Best Face Moisturizer for Dry, Sensitive Skin, According to People Who Use Them

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Heres How Niacinamide Benefits Your Complexion (and How to Work It into Your Skin Care Routine) - Yahoo Lifestyle

Cancer | One of the most common disease in the world

In what may prove as a breakthrough in the treatment of leukaemia and other blood diseases, scientists at UCLA have discovered a protein produced by a gene known as MLLT3 and its connection to the multiplication of human blood stem cells.

The discovery which was published in a study is very much significant as cancers such as leukaemia can be effectively treated using blood stem cells, also known as Hematopoietic stem cells (HSCs), produced outside the human body and could serve as an alternative to existing treatment options such as bone marrow transplants.

Self-renewal is the process by which stem cells divide to create more cells. The study focused on a specific type of the kind: Hematopoietic stem cells (HSCs) which are present within the bone marrow where along with self-renewal, they also produce different types of blood cells such as red and white by transforming into them.

Placing HSCs in laboratory dishes after their removal from the bone marrow causes then to lose their ability to self-renew, and they either transform into other blood types or perish. It is this process that the scientists studied. Through a series of steps, the researchers studied the genes that shutdown as the cells lost their capacity to self-renew.

They discovered that the HSCs' ability to self-renew corresponded with the expression of a gene called MLLT3. They also found that MLLT3 generated a protein that instructed HSCs to retain their capacity to self-renew. As the cells divide, the protein works along with other regulatory proteins to keep vital components of the HSCs' functioning.

Employing a viral vector the researchers tried to ascertain if maintaining the MLLT3 protein levels in lab dishes would help improve the self-renewing abilities of HSCs. A viral vector is a specially designed virus that transfers genetic information to the nucleus of a cell without giving rise to a disease. Using the vector, the scientists introduced an active MLLT3 gene into HSCs. They found that there was nearly a twelvefold multiplication of working HSCs in lab dishes.

"If we think about the amount of blood stem cells needed to treat a patient, that's a significant number," said Dr. Hanna Mikkola, a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, and senior author of the study, to the UCLA Newsroom.

The UCLA scientists observed that the use of 'small molecules' organic compounds that help in the multiplication of human HSCs, improved self-renewal in general. However, the cells were unable to maintain stable MLLT3 levels and did not perform well when implanted into mice.

"Our method, which exposes blood stem cells to the small molecules and also inserts an active MLLT3 gene, created blood stem cells that integrated well into mouse bone marrow, efficiently produced all blood cell types and maintained their self-renewing ability," said Vincenzo Calvanese, a UCLA project scientist and the study's co-corresponding author, to the UCLA Newsroom.

The team noted that the self-renewal of HSCs caused by MLLT3 was at a safe rate. This means that they did not acquire potent characteristics such as mutation or excessive multiplication, or the production aberrant cells that can cause leukaemia.

Determining which proteins and constituents within the DNA of the HSCs affect the activation and deactivation of MLLT3 is the next step for the team. Also, understanding the regulation of the process using components in the lab dishes is another step. The information gathered may help find ways in which MLLT3 could be switched on and off without employing a viral vector.

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Protein that can help treatment of leukemia identified, UCLA scientists upbeat - International Business Times, Singapore Edition

MAGICAL Nature is Rebecca Bennett's first exhibition in four years following her stem cell transplant.

Running throughout December at Rydal Hall Old School Room Teashop at Ambleside, the exhibition features various media from acrylics and watercolours to photography.

Rebecca loves capturing the magic of colour and pattern within nature in her artwork. She grew up surrounded by the beautiful fells and lakes of Cumbria. Coming from an artistic family, painting and drawing from an early age was natural.

From photographs of lakes seen through the prism of a crystal ball to an enigmatic acrylic painting of a grey heron, Magical Nature includes striking images of the wildlife and countryside of Rebeccas native Lake District.

With a BA(hons) degree in Contemporary Applied Arts from Cumbria Institute of the Arts, Rebecca furthered her art practice and skills to create porcelain ceramic pieces and textile mixed media artworks.

Following university Rebecca completed a variety of successful art projects and workshops alongside her exhibitions. These included art workshops with young people at the Coniston Water Festival and Blencathra Field Studies Centre.

In 2015 Rebecca had a stem cell transplant at London's Kings College Hospital. An unrelated donor provided the stem cells to treat failing bone marrow caused by rare Gata2 deficiency and Myelodysplastic Syndrome. Painting and photography have, she says, been a great therapy throughout her transplant journey.

"When you feel up to it having something to immerse yourself in such as art and photography can be a fantastic way to help you forget about your pain and problems. For those moments, you are focused on what you are creating and can escape for a little while."

Alongside exhibiting her work for the first time since being diagnosed with her illnesses, Rebecca hopes to raise awareness of MDS and blood disorders. Donations to the charity MDS UK patient support will be made from sales of Rebeccas greeting cards and prints during the exhibition.

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Artist's first exhibition following stem cell transplant - The Westmorland Gazette

Emi receives kisses from her adoptive parents, Jason and Katie Ballard, at the NIH Clinical Center as she gets ready to receive a lifesaving hematopoietic stem cell transplant, the only way to cure her fatal immune deficiency, using cells donated by her birth mom.

Emi smiles at The Children's Inn at NIH prior to undergoing a hematopoietic stem cell transplant, the only potential cure for her rare and deadly immune deficiency.

Bethesda, Maryland, Nov. 26, 2019 (GLOBE NEWSWIRE) -- One Texas family has lots to be thankful for this Thanksgiving. Their daughter, now 13, is doing well after undergoing a bone marrow transplantthe only chance for a cure for her rare and deadly disease. But Emis story is not only a story about the triumph of medical research that is making her cure possibleits also a story about extraordinary parental love and sacrifices by her birth mom and her adoptive family that are giving this very ill girl the best chance at life. Emi's birth mom donated her stem cells to make the lifesaving transplant possible.

We are most thankful for an answer to years of prayers, Emis adoptive mom says. Emi got a new start at life, a rebirth day. Every holiday this year will be like the first. Were so grateful to the doctors, nurses and The Childrens Inn.

Emi and her family will be celebrating Thanksgiving at The Childrens Inn at NIH, a nonprofit hospitality house that provides free lodging and a wide variety of support services to families of children with rare and serious diseases whose best chance for a treatment is a clinical research study at the National Institutes of Health. Emi and her mom have spent several months at The Childrens Inn so far and bonded with other families. On Thanksgiving Day, families staying at The Childrens Inn who cannot go home for the holiday will be served a traditional Thanksgiving meal prepared by a group of dedicated volunteers.

It took two moms who love this little nugget to fight for her right to life, Emis adoptive mom says. We finally are getting to see that beautiful part of the story that we always knew was there.

Read Emis full story.

See photos of Emi and her family.

About The Childrens Inn at NIH:

The Childrens Inn at NIH provides free lodging and a wide range of supportive services to more than 1,500 children and their families every year whose best chance for a treatment is a clinical trial at the National Institutes of Health. Opened in 1990 and located across from the NIH Clinical Center, the worlds largest hospital dedicated entirely to medical research, The Childrens Inn has welcomed children from all 50 states and 94 countries. Children staying at The Childrens Inn are making important contributions to rare disease and cancer research, including the successful treatment of childhood leukemia, as well as treatments for HIV/AIDS, childhood asthma, bone and growth diseases, childhood onset schizophrenia and other mental health issues, neurofibromatosis type 1 and a wide variety of genetic and rare diseases. For more information, visit http://www.childrensinn.org. To support The Childrens Inn, make a donation at http://www.childrensinn.org/donate.

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The Best Thanksgiving - GlobeNewswire

The most raved about secret in beauty this year wasn't a magic facialist or Real Housewife-lauded injection, it was an unassuming moisturizer made by a 60-year-old German scientist who'd never worked in the industry, let alone had an Instagram following. And yet, despite having none of the traditional resources that makes a beauty brand an overnight successlike paid celebrity spokespeople, $1 million ad campaigns, or millennial pink packagingAugustinus Bader became a sleeper hit purely through word of mouth.

In the nearly two years since it launched, AB's "miracle cream" has gotten accolades from Ashley Graham, Kate Bosworth, and Victoria Beckham (who's since launched a moisturizing primer with the brand). Glamour even gave it a Beauty Award for Best Moisturizer, a highly competitive category as you can surely imagine. So before I even uncapped the weighty blue-and-copper tube of its famed The Cream, which costs a cool $265 for 50 mL, I was basically set to fall in love with itprice tag be damned.

But the only kind of hype I pay real attention to is beauty editor hype; if my product-inundated colleagues are raving about it, I figure it has to stand out from the pack. And for weeks, every single editor I knew had told me about the cream in an attitude I can only describe as reverent. I was ready to experience my own Bader-sparked miracle.

Bizarrely, I didn't immediately fall head over heels for it (don't worry, a second plot twist will follow shortly). Sure, it was a good face cream. The light texture absorbed quickly and my skin looked decent, but it didn't exactly wow me. I felt like a thin layer just wasn't doing much in the moisturizing departmentwhich, apparently, I later learned isn't even what The Cream claims to do. It's more of an overall skin rejuvenator. The brand also sells a Rich Cream for dry skin, which contains additions like avocado and argan oil to aide with extra hydration, but I began with the original thinking it'd be enough.

In order to get maximum effectiveness from the active ingredients, I did what a few other friends and makeup artists had advised and skipped all other products, except face wash. The Bader formula is based on TFC8 (Trigger Factor Complex 8), a proprietary cocktail of over 40 different ingredients, including vitamins and amino acids. It's meant to encourage regeneration and healingProfessor Bader actually discovered the formula while looking for solutions to help burn survivors heal quickerand TFC8 is supposed to activate your stem cells, which go to work to repair fine lines, dark spots, and visible pores. I had heard that the ingredients within were enough to replace all other skin care, so I devoted myself to a one-step kind of lifestyle and waited for my skin to start looking like I had just walked out of a spa. And then I waited some more...

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Augustinus Bader The Cream Review: Why It's Worth Every Cent - Glamour

Birds of a feather may flock together, but the feathers of birds differ altogether.

New research from an international team led by USC scientists set out to learn how feathers developed and helped birds spread across the world. Flight feathers, in particular, are masterpieces of propulsion and adaptation, helping penguins swim, eagles soar and hummingbirds hover.

Despite such diversity, the feather shares a common core design: a one-style-fits-all model with option trims for specialized performance. This simplicity and flexibility found in nature holds promise for engineers looking for better ways to build drones, wind turbines, medical implants and other advanced materials.

Those findings, published today in Cell, offer an in-depth look at the form and function of a feather based on a comparative analysis of their physical structure, cellular composition and evolution. The study compares feathers of 21 bird species from around the world.

Weve always wondered how birds can fly in so many different ways, and we found the difference in flight styles is largely due to the characteristics of their flight feathers, said Cheng-Ming Chuong, the studys lead author and a developmental biologist in the Department of Pathology at the Keck School of Medicine of USC. We want to learn how flight feathers are made so we can better understand nature and learn how biological architecture principles can benefit modern technology.

To gain a comprehensive understanding of the flight feather, Chuong formed a multi-disciplinary international team with Wen Tau Juan, a biophysicist at the Integrative Stem Cell Center, China Medical University in Taiwan. The work involved experts in stem cells, molecular biology, anatomy, physics, bioimaging, engineering, materials science, bioinformatics and animal science. The bird species studied include ostrich, sparrow, eagle, chickens, ducks, swallow, owl, penguin, peacock, heron and hummingbird, among others.

They compared feathers using fossils, stem cells and flight performance characteristics. They focused on the feather shaft, or rachis, that supports the feather much like a mast holds a sail, bearing the stress between wind and wing. They also focused on the vane, the lateral branches astride the shaft that give the feather its shape to flap the air. And they examined how evolution shaped the barbs, ridges and hooks that help a feather hold its form and lock with adjacent feathers like Velcro to form a wing. The goal was to understand how a simple filament appendage on dinosaurs transformed into a three-level branched structure with different functions.

We want to learn how flight feathers are made so we can better understand nature and learn how biological architecture principles can benefit modern technology.

Cheng-Ming Chuong

For birds such as ducks, eagles and sparrows that fly in different modes, the scientists noted significant differences in the feather shaft compared to ground-hugging birds. On the rigid exterior, the shaft cortex was thinner and lightweight, while the interior was filled with porous cells resembling bubble wrap, aligned into bands of various orientations and reinforced with ridges that operate like tiny lateral beams. Together, it forms a light, hollow and buoyant structure to enable flight. Cross-sections of feather shafts of different birds show highly specialized shapes and orientations of the inner core and outer cortex.

The flight feather is made of two highly adaptable architectural modules, light and strong materials that can develop into highly adaptable configurations, Chuong said.

The researchers discovered two different molecular mechanisms guiding feather growth. Cortex thickness was governed by bone morphogenetic proteins, which are molecular signals for tissue growth. The porous feather interior, or medulla, relied upon a different mechanism known as transforming growth factor-beta (TGF-b). Both components originate as stem cells in the birds skin.

By contrast, feathers in flightless birds were simpler, consisting of a dense cortex exterior that is more rigid and sturdy with fewer internal struts and cells found in flying birds. The features were especially pronounced for penguins, which use wings as paddles under the water.

As part of the study, the researchers looked at 100 million-year-old feathers, found embedded in amber in Myanmar. These fossils show early feathers lacked one key feature that modern birds have. Specifically, the researchers report that fossil feathers had barb branches and barbules, which form a feather vane by overlapping, but not hooklets. The hooklets, which act like clasps to turn fluffy feathers into a tight flat plane for high-performance flight, evolved later. The scientists also identified WNT2B, another growth factor, as the agent that controls hooklet formation. These also originated from epidermal stem cells.

Taken together, the findings show how feathered dinosaurs and early birds could form a primitive vane by overlapping barbule plates, although that wasnt aerodynamically fit to carry much load. As more complex composite features occurred in the wing, it got heavier, so feather shafts became stronger yet more lightweight, which led to stiffer feathers and sturdy wings that powered flight to carry birds around the world.

Our findings suggest the evolutionary trends of feather shaft and vane are balanced for the best flight performance of an individual bird and become part of the selective basis of speciation, the study said. The principles of functional architectures we studied here may also stimulate bio-inspired designs and fabrication of future composite materials for architectures of different scales, including wind turbines, artificial tissues, flying drones.

Chuong and Juan are co-leaders of the 31-person team, joined by co-authors Randall B. Widelitz, Shuo Wang, Michael Habib, Ting-Xin Jiang, Zhong-Lai Luo and Ping Wu of the Keck School of Medicine of USC; Wei-Ling Chang, Hao Wu, Yung-Chi Lai, Ming Xing Lei, and Shih-Chieh Hung of the China Medical University Hospital in Taiwan; Ming-You Shie, Jui-Ting Hsu, Heng-Li Huang and Yi-Wen Chen of the China Medical University, Taiwan; Chih-Feng Chen, Ping Chi Tang, Hus Chen Cheng, and Yen-Cheng Lin of the National Chung Hsing University in Taiwan; How-Jen Gu, Yu-Kun Chiu, Tse-Yu Lin, Shun-Min Yang, Tsung-Tse Lee, J.C. Tsai and Yeu-Kuang Hwu of the Institute of Physics, Academia Sinica, Taiwan; Cheng-Te Yao of the Endemic Species Research Institute, Taiwan; Shyh-Jou Shieh of the National Cheng Kung University, Taiwan; Ang Li of the University of Texas, Arlington.

Work at USC was supported by the National Institutes of Health (AR 047364, AR 060306) while team members in Taiwan were supported by grants from their own institutes and the Taiwan government.

More stories about: Biology, Research

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How birds fly: New USC study examines the evolution of flight feathers - USC News

Winter is not just for hot cocoa and cozy blankets; the season is not always kind to the skin.

When the air outside gets gold and dry, without proper care, you skin can quickly follow suit. Having dry skin is the most uncomfortable feeling in the world.

The entire winter season, dead skin cells can build up on the skin's surface, which would result in a dry and dull complexion. The cold weather can lead to chapped lips, cracked hands and dry flaky skin.

Once the temperatures drop, your skincare routine should rise to the challenge.

That's why it's important to moisturize at this time of the year. However, it's also important to keep exfoliation in your winter skincare routine, and here's why.

Exfoliation isn't something yo;ll need to put on the back burner. In fact, winter is one of the best times for exfoliations. If you aren't spending your snow days sloughing away dead skin, then you're totally missing out.

Exfoliations helps replenish the skin.

Between the dry indoor heating and the cold air outside, your skin cells need to hydrate. They easily die out faster in the colder season, so it's essential to buff away the dead cells so new cells can come in healthier. It's recommended to exfoliate at least twice a week.

Exfoliators improve the effectiveness of the moisturizers.

Because you'll be slapping extra heavy lotion for the winter, you'll want to maximize its power. Do this by exfoliating regularly. The dead cells block moisture from the layers of live skin cells that actually need it.

Exfoliating beads, salt or sugar scrubs, dry scrubbing or even common body sponges are best examples of physical exfoliants that will help tremendously.

The usual at-home scrubs are the sugar scrub and the salt scrub.

The difference between the two is that the sugar scrub is more gentle, less abrasive and tends to dissolve fast in warm water.

Whereas the salt scrub tends to be a little more aggressive because the granules are larger. With a salt scrub, you're going to want to exfoliate a little less than you would with a sugar scrub or other exfoliating beads.

Keep scrolling to check 5 of our top picks for adding some body scrub time to your tub or shower regime.

M3 Naturals Himalayan Salt Scrub

Detoxify the skin with the M3 Naturals Himalayan Salt Scrub. It is infused with collagen and stem cell that increases skin cell longevity. Combined, these deliver an anti-aging performance.

This salt scrub is made from an all-natural Himalayan pink salt with lychee fruit and almond oil that will provide moisture and cleans out impurities of the skin and will effectively remove dirt, oil and reduce the appearance of acne, scars, blackheads and cellulites.

(Photo : Amazon)

Dove Exfoliating Body Polish Body Scrub

This easy-to-find exfoliating body scub removes dull, dry skin while deeply nourishes it to restore its natural nutrients.

The product is formulated with moisturizing cream and has a whipped texture that provides a creamy coverage.

(Photo : Amazon)

Brooklyn Botany Arabica Coffee Scrub

A coffee body scrub that can be used on the face, hand and foot. It will easily remove dead skin giving you a fresher, younger and moisturized appearance.

It will also reduce the signs of aging because of the coffee's antoxidants, fighting the appearance of fine lines, sun spots and wrinkles.

(Photo : Amazon)

Majestic Pure Cosmeceuticals Sweet Orange Body Scrub

A vegan-friendly, bright and refreshing body scrub crafted with nourishing ingredeints such as sweet orange oil, dead sea salt, organic aloe vera juice and coconut oil.

Using this product can promote more supple and smooth skin, gently removing dead skin and exposing it to enriching and moisturizing minerals and nutrients.

(Photo : Amazon)

Shea Moisture Exfoliating Hand and Body Sugar Scrub

The sugar scrub is made from natural ingredeints that gently cleanses skin from impurities, pollutants and build-up. It is created with argan oil and organic raw shea butter.

It will provide your skin with intense moisture and can exfoliate your hand and body by removing dead skin cells.

(Photo : Amazon)

READ MORE: 7 Gifts that Will Not Break Your Bank Account this Christmas 2019

See Now: Famous Actors Who Turned Down Iconic Movie Roles

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5 Scrubs and Exfoliators You NEED This Winter 2019 - Enstarz

Mucopolysaccharidoses (MPS) are a group of very rare disorders, also known as orphan diseases. They belong to the group of lysosomal storage diseases which are caused by a deficiency of one of the enzymes involved in the degradation of mucopolysaccharides (the acid glycosaminoglycans or GAGs). The enzymes are coded by genes which produce deficient gene products due to gene variants in each of the two gene-alleles.

Children of two carriers as parents have a 25% risk to suffer from MPS. For many families, the birth of the first affected child is a shock and a disaster. The disease is continuously progressing, and life spans are dramatically decreased without therapy. As a result, extensive efforts are put into the cure of these fatal disorders.

Enzymes are relatively small proteins, produced in the endoplasmatic reticulum of each cell. Before reaching the locus of their function, the lysosomes, additional modifications with special sugars are performed in the Golgi apparatus (glycosylation). Via mannose-6-phosphate marker, they connect to the mannose-6-phosphat receptor on the lysosomal membrane and can reach the final locus of their function. In the lysosomes, enzymes degrade the GAG chains into the smallest molecules for recycling or excretion. Any disturbance in this process leads to the accumulation of non-degraded material, which affects many other cell functions such as homeostasis, calcium metabolism, accelerates apoptosis and induces inflammation processes.

As lysosomes are ubiquitous, any disturbance leads to storage in many different tissues and organs. MPSs are a good example for chronic progressive multi-systemic disorders. The best theoretical option for treatment of any patient is to supplement the missing enzyme which could reach any organ via blood flow and get inside the lysosomes continuing the interrupted degradation processes.

The enzymes are ubiquitous and have some tissue specific compositions. Enzymes produced in the different cells and tissues have their own characteristics and are available on site. The production of recombinant enzymes means that the artificial glycosylation is created in a uniform composition for intravenous substitution with the aim to reach the organs with the blood-flow. There is no doubt that the therapeutic efficacy is ideal for many organs, such as liver, spleen, lung, and skin. All these organs have a good blood circulation and some ability to regenerate.

However, after years of treatment with the already available enzymes, it is shown that some organs are poorly supplied with blood and renewal cycles are slow, the ability to regenerate is decreased. Organs such as bones, cartilage, muscles, cornea, heart valves, meninges or the brain do not show the hope-for effect. All MPS types with brain involvement (neuronopathic forms of MPS types I, II and VII) or predominant skeletal dysplasia (MPS types IVA and B) cannot benefit from enzyme-replacement therapy and do not show the desired improvement.

In animal studies, modifications of glycosylation can change the ability to pass into organs not yet sufficiently reached such as cartilage or bones, but tissue-specific features cannot be sufficiently considered in any artificial production of the enzymes.

Avascular cartilage, heart valves and corneas cannot be reached by blood flow. Also, between blood vessels and brain tissue, several specialised cells form the blood-brain-barrier (BBB) to protect the brain from any unwanted substances in the blood. Therefore, new strategies are necessary to improve the therapeutic efficiency and to provide better outcomes for the affected patients. If patients with MPS I are diagnosed at a very young age, the best option is to treat them with haematopoietic stem cell transplantation (HSCT). Migrating stem cells can reach the brain and other organs, and then differentiate into organ-specific cells producing the missing lysosomal enzymes.

A straightforward method to overcome BBB is the direct injection of a recombinant enzyme into the cerebral fluid. This can be by lumbar puncture (intra-thecal) or intra-ventricular injections in the brain ventricles. Effects can be observed, however unfortunately not all challenges can currently be solved. The liquor flow can be reduced by thickened meninges with storage and vertebral deformities, which are typical for the disease. However, the barrier between cerebral fluid and brain tissue has still not been fully studied. The half-life of enzymes is limited, and the procedure has to be repeated regularly. The clinical trials for patients with MPS I, II, IIIA and IIIB could show some reduced or reversed progression of CNS pathology but long-term effects remain unclear.

Another possibility to overcome BBB is to fuse the enzyme proteins with macromolecules which enter the brain through receptor mediated active transport systems. This physiological transport is known for hormones, neurotransmitters and many other proteins (such as transferrin and insulin). They are transported through the BBB directly into the brain via specific receptores, so, the strategy is to fuse the natural proteins with the artificial enzymes needed in the MPS patient. It is important to note that clinical trials could potentially still show some improvement in affected MPS patients.

Another method is to conjugate the therapeutic enzymes with nano-capsules and to then ferry them across BBB via transcytosis or other transport mechanisms directly into brain cells. Pharmacological chaperones have been proven to be effective in other lysosomal storage diseases such as Gaucher or Fabry disease. Chaperones are able to stabilise three-dimensional conformation of misfolded proteins, such as enzymes. This would be the case of genetic variants causing missense mutation and exchange of only one amino acid in the protein chain. The misfolding pathology reduces stability, half-life and effect of the genetically conditioned enzyme, whereas the chaperone can reverse this disadvantage and increase the activity and efficacy of the enzyme. As a result, pharmacological chaperones are a good option for some diseases and could therefore be an option for some MPS patients in the future.

Some genetic variants cause stop-codons and the production of truncated dysfunctional peptides without any enzymatic activity and degradation within the cell. Stop-codon read through therapy aims for the genetic correction on an RNA level, resulting in the production of a sufficiently functioning gene product. It is already used for some specific mutation for patients with Duchenne muscular dystrophy, but it is too early to predict positive results for patients with MPS I.

Another possibility in the future might be the use of GAG-reducing small molecules such as Genistein, Pentosam polysulfate or Rhodamine B. They are able to influence and/or reduce the synthesis of GAGs which cannot be degraded sufficiently by the genetically changed enzymes with reduced function.

To reduce the GAGs as substrate, could be a chance to create a better relation between substrate and the impaired substrate reducing enzyme. As a result, lysosomal storage could therefore be reduced. Substrate reduction therapy is an established therapeutic concept in some of the other lysosomal storage diseases, but the usefulness in MPS disorders still needs to be proven.

The genetic corrections of DNA sequences in patient cells are no longer only future options as they have now become a reality. Gene variants causing missing or impaired functioning gene products could be replaced by correct genetic sequences and genes. This can be made as an ex vivo approach, where stem cells or fibroblast are removed from the patient and are then cultured in vitro, genetically corrected and consecutively re-injected into the patient.

The genetically corrected DNA in the re-transplanted autologous cells is able to produce correct gene products (in terms of MPS, this is the specific enzyme). The amounts of newly produced enzymes might be sufficient to positively influence the disease course of the treated patients.

An in vivo approach utilises viral vectors which invade cells, and even cell nuclei. Such viruses used are adeno-associated-viruses or lenti-viruses. Such manipulated viruses with the corrective genetic material are directly injected into the patient where they are internalised into deficient cells and are then able to produce the missing gene product. In the case of MPS, the aim is to produce enzyme proteins with sufficient concentrations and activity to prevent the storage of GAGs. Furthermore, clinical trials are underway for several MPS types and therefore, might offer a therapeutic opportunity in early life for affected patients. However, larger studies and a longer follow-up is still needed.

To conclude, MPS are rare genetic disorders and for a long time, they were linked with the myth of being untreatable diseases. Although some of the new therapeutic options are still in clinical trials and not routinely used, the present shows that many of the patients can benefit from the yet available options of HSCT and enzyme replacement therapies. These therapies have an undoubted effect for some of the MPS patients, especially if any form of therapy is started early or if the course of the disease does not affect the nervous system.

However, in the future, new therapeutic options will hopefully bring benefits to those that are not sufficiently improved; the decision of the best therapy will be made on the basis of factors such as the genetic defect, the type of MPS, and the age during treatment. This individualised and personalised therapy will improve the success of MPSs therapies.

Susanne Gerit KircherMedical University of Vienna, AustriaCenter of Pathobiochemistry and Geneticssusanne.kircher@meduniwien.ac.atwww.mps-austria.at

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Mucopolysaccharidoses: future therapies and perspectives - SciTech Europa

Although one in nine men will receive a prostate cancer diagnosis in his lifetime, cutting-edge research has allowed more men to live longer or even be cured.

One such program that sheds light on this cause is City of Hopes NoShaver November. The month-long fundraising and awareness campaign urges participants to forego shaving to start a conversation, encourage testing and raise critical funds to continue leading-edge prostate cancer research and promising new therapies conducted at City of Hope.

CURE recently spoke with Dr.Tanya Dorff, a medical oncologist at City of Hope who specializes in prostate cancer, about the campaign, her current work and where she sees the future of prostate cancer treatment shifting in the coming years.

CURE: What led you to City of Hope? What do you do there?Dorff: City of Hope was attractive to me because I am a clinical and translational researcher. The reason I came here was to work with scientists who share what we are seeing in the clinic and who incorporate the latest insights from the scientific discoveries in our laboratories into patient care. There is a real sense of mission and urgency that binds scientists to clinicians at City of Hope in a way that is unique and gratifyingly productive.First and foremost, I take care of patients who remain my central inspiration and raison detre, but I spend part of my time writing and running clinical trials that have real potential to impact how we treat patients in the future how we can do even better in the future than we do today. I lead the genitourinary cancer program, which includes fostering collaborations between the incredible physicians from urology, radiation oncology, radiology and pathology to work together both clinically and in research projects.You are Grammy-winning songwriter and vocal producer Kuk Harrells physician. Can you tell me what it was like to treat him?Kuk is such an incredible gentleman; it has been a pleasure to be part of his care team. His attitude toward treatment was one of diligence, and he has approached his illness as an opportunity for personal growth and for giving back by promoting prostate cancer awareness through his story. It has been inspiring to see him come through what was a lengthy and involved treatment with so much positive energy.How has the field of prostate cancer treatment evolved in recent years?More and more men with prostate cancer can be cured, and the men who cannot be cured with todays treatments are clearly living longer and better. This is thanks to new drug approvals in advanced, resistant prostate cancer but even more so to the application of more intensive therapy earlier in the course of the disease. This has been the biggest paradigm shift in prostate cancer over the last five years: up-front intensification in metastatic hormone sensitive prostate cancer.The next big shift in prostate cancer treatment is just now upon us molecular selection of therapies to individualize prostate cancer treatment. The most imminent example is olaparib (Lynparza), a PARP inhibitor, which worked better than standard treatment in patients with castration-resistant prostate cancer whose tumors harbor mutations in DNA repair genes. But the ingenious theranostic approach will be close behind where imaging (scans) show us whether a cancer is expressing a certain target (i.e. PSMA) and if so, a radioactive particle linked to that target is applied (i.e. Lu-177 PMSA).

What are you most hopeful for in cancer treatment in the future?I believe immunotherapy will be the way to durable remission or a cure. Here at City of Hope, we are working hard to improve the effectiveness of immunotherapy for patients with metastatic prostate cancer, studying intensive treatments such as CAR-T and bispecific T-cell engaging antibodies, among other approaches. Our scientists are looking at our patients in real time to learn why treatments work or dont work, and how to better engage the immune system. I am very hopeful that these biologic insights will eventually translate into therapeutic success such as we have seen in leukemia with CAR-T and melanoma with immune checkpoint inhibitors.What advice would you offer someone who has just received a cancer diagnosis of their own?One: Play an active role. Ask questions, and if something doesnt sound right or make sense, ask again. It is so important that patients buy into their treatment, understand and feel confident about the treatment plan. No one is perfect, not even the best doctor, and working together as a team will lead to the best success.

Two: Be a squeaky wheel. Patients who communicate symptoms in real time fare better because problems are addressed before they become more serious.

Three: Stay active. Exercise is one of the things that has been shown over and over again to help cancer survivors and cancer patients in various stages. Obviously, a conversation should occur with the treatment physicians to ensure that there are no restrictions but patients who are more active will come through treatment in better shape.

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Exploring the Future of Prostate Cancer with City of Hope - Curetoday.com

If are fasting intermittently or taking long food breaks, here's the news for you. A new study presented at the 2019 American Heart Association Scientific Sessions in Philadelphia has found the good health outcomes of intermittent fasting for cardiac catheterisation patients.The study showed that patients who practised intermittent fasting lived longer than those who didn't. In addition, they are less likely to be diagnosed with heart failure. Cardiac catheterisation is a procedure used to diagnose and treat certain cardiovascular conditions. "It is another example of how we're finding that regular fasting can lead to better health outcomes and longer lives," said the study's principal investigator Benjamin Horne, PhD, director of cardiovascular and genetic epidemiology at the Intermountain Healthcare Heart Institute.Researchers asked 2,001 intermountain patients undergoing cardiac catheterization from 2013 to 2015 a series of lifestyle questions, including whether or not they practised routine intermittent fasting. They then followed up with those patients 4.5 years later and found that routine intermittent fasters had a greater survival rate than those who did not.Because people who fast routinely also are known to engage in other healthy behaviours, the study also evaluated other parameters including demographics, socioeconomic factors, cardiac risk factors, comorbid diagnoses, medications and treatments, and other lifestyle behaviours like smoking and alcohol consumption.Correcting statistically for these factors, long-term routine fasting remained a strong predictor of better survival and lower risk of heart failure, according to researchers. While the study does not show that fasting is the causal effect for better survival, these real-world outcomes in a large population do suggest that fasting may be having an effect and urge continued study of the behaviour. "While many rapid weight loss fasting diets exist today, the different purposes of fasting in those diets and in this study should not be confused with the act of fasting," said Dr Horne. "All proposed biological mechanisms of health benefits from fasting arise from effects that occur during the fasting period or are consequences of fasting," he added.Why long-term intermittent fasting leads to better health outcomes is still largely unknown, though Dr Horne said it could be a host of factors. Fasting affects a person's levels of haemoglobin, red blood cell count, human growth hormone, and lowers sodium and bicarbonate levels, while also activating ketosis and autophagy - all factors that lead to better heart health and specifically reduce risk of heart failure and coronary heart disease. "With the lower heart failure risk that we found, which is consistent with prior mechanistic studies, this study suggests that routine fasting at a low frequency over two-thirds of the lifespan is activating the same biological mechanisms that fasting diets are proposed to rapidly activate," Dr Horne noted.Researchers speculate that fasting routinely over a period of years and even decades conditions the body to activate the beneficial mechanisms of fasting after a shorter length of time than usual.

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Can intermittent fasting help you live long? - Times of India

VANCOUVER, British Columbia, Nov. 26, 2019 (GLOBE NEWSWIRE) -- Novoheart (Novoheart or the Company) (TSXV: NVH; FWB: 3NH), a global stem cell biotechnology company, is pleased to announce a collaboration with global biopharmaceutical company AstraZeneca, in an effort to develop the worlds first human-specific in vitro, functional model of heart failure with preserved ejection fraction (HFpEF), a common condition especially among the elderly and in women, with the reported prevalence approaching 10% in women over the age of 80 years.1

Heart failure (HF) is a global pandemic with an estimated 64.3 million cases worldwide in 2017, with an increasing trend in prevalence2. The annual global economic burden of HF is estimated at over US$100 billion3. Accounting for approximately 50% of HF cases, HFpEF in particular is a major and growing public health problem worldwide, with its pathological mechanisms and diverse etiology poorly understood. Due to these complexities, models of the disease available to date, including various animal models, have limited ability to mimic the clinical presentation of HFpEF4. Therefore, drug developers lack an effective tool for preclinical testing of drug candidates for efficacy, and as a result, clinical outcomes for HFpEF have not improved over the last decades, with no effective therapies available.

In collaboration with the Cardiovascular, Renal and Metabolism therapy area of AstraZeneca, the initial phase of the project aims to establish a new in vitro model, leveraging Novohearts proprietary 3-D human ventricular cardiac organoid chamber (hvCOC) technology, that reproduces key phenotypic characteristics of HFpEF. Also known as human heart-in-a-jar, the hvCOC is the only human engineered heart tissue available on the market to date that enables clinically informative assessment of human cardiac pump performance including ejection fraction and developed pressure. Unlike animal models, engineered hvCOCs can be fabricated with specific cellular and matrix compositions, and patient-specific human induced pluripotent stem cells (iPSCs), that allow control over their physical and mechanical properties to mimic those observed in HFpEF patient hearts. Together with Novohearts proprietary hardware and software, this aims to provide a unique assay for understanding the mechanisms of HFpEF, identification of new therapeutic targets, and assessment of novel therapeutics for treating HFpEF patients. Novoheart will exclusively own the intellectual property rights to the newly developed HFpEF hvCOC model.

We are delighted to partner with AstraZeneca, an organization which has long invested in cardiovascular research and is committed to bringing new therapeutic solutions to patients with heart failure, said Novoheart CSO, Dr. Kevin Costa. We look forward to co-developing this new HFpEF hvCOC model into a powerful new tool in the worldwide battle against heart failure.

Regina Fritsche Danielson, Senior Vice President, Head of Research and Early Development, Cardiovascular, Renal and Metabolism, BioPharmaceuticals R&D, AstraZeneca, said, There are significant unmet treatment needs in patients with heart failure with preserved ejection fraction. By combining Novohearts proprietary hvCOC model with our expertise in heart failure, we aim to create the first in vitro model reproducing phenotypic characteristics of heart failure with preserved ejection fraction. This could bridge the gap between in vivo animal models and clinical trials to help accelerate the drug discovery process by providing human-specific preclinical data.

1 Heart Fail Clin. 2014; 10(3):377388.2 Lancet. 2018; 392:1789-1858.3 Int J Cardiol. 2014; 171(3):368-76.4 JACC Basic Transl Sci. 2017; 2(6):770-789.

About Novoheart:

Novoheart is a global stem cell biotechnology company pioneering an array of next-generation human heart tissue prototypes. It is the first company in the world to have engineered miniature living human heart pumps that can revolutionize drug discovery, helping to save time and money for developing new therapeutics. Also known as 'human heart-in-a-jar', Novohearts bio-artificial human heart constructs are created using state-of-the-art and proprietary stem cell and bioengineering approaches and are utilized by drug developers for accurate preclinical testing of the effectiveness and safety of new drugs, maximizing the successes in drug discovery whilst minimizing costs and harm caused to patients. With the recent acquisition of Xellera Therapeutics Limited for manufacturing Good Manufacturing Product (GMP)-grade clinical materials, Novoheart is now developing gene- and cell-based therapies as well as next-generation therapeutics for cardiac repair or regeneration.

For further information, please contact:Ronald Li, CEOinfo@novoheart.com

For media enquiries or interviews, please contact:Media Relationsmedia@novoheart.com

Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

Cautionary Note Regarding Forward-Looking Statements

Information set forth in this news release may involve forward-looking statements under applicable securities laws. Forward-looking statements are statements that relate to future, not past, events. In this context, forward-looking statements often address expected future business and financial performance, and often contain words such as "anticipate", "believe", "plan", "estimate", "expect", and "intend", statements that an action or event "may", "might", "could", "should", or "will" be taken or occur, or other similar expressions. By their nature, forward-looking statements involve known and unknown risks, uncertainties and other factors which may cause the actual results, performance or achievements, or other future events, to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Such factors include, among others, the risks identified in under the heading Risk Factors in Novohearts annual information form for the year ended June 30, 2019 or other reports and filings with the TSX Venture Exchange and applicable Canadian securities regulators. Forward-looking statements are made based on management's beliefs, estimates and opinions on the date that statements are made and the respective companies undertakes no obligation to update forward-looking statements if these beliefs, estimates and opinions or other circumstances should change, except as required by applicable securities laws. Investors are cautioned against attributing undue certainty to forward-looking statements.

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Novoheart to Co-develop First of its Kind Human Heart-in-a-Jar Model of Heart Failure with AstraZeneca - GlobeNewswire