The medical AM segment is multifaceted, consisting of 3D printed medical devices, anatomical models, prosthetics and more. Within the industry,bioprintinghas carved out a prominent position, gaining interest across the board for its huge potentials in drug development and screening, therapeutic treatments and regenerative medicine, to name but a few. While much of the excitement surrounding bioprinting is focused on the futurewhat it could dowe want to look at what is happening now in the field that is exciting.

As part of our Medical AM Focus, we asked bioprinting leaders from across the sector what they consider to be the most exciting application for their bioprinting technologies today. In this segment, we hear from Canadian bioprinting company Aspect Biosystems.

Based in Vancouver, Aspect Biosystems is a biotech company that specializes in the microfluidic 3D bioprinting of human tissues. The company has brought to market a sophisticated bioprinting systemthe RX1 Bioprinterwhich is today used by researchers all over the globe for medical applications, including neural and cardiac research. The company also conducts its own research in-house, developing therapeutic tissue programs for orthopedic and metabolic disorders.

According to Aspect Biosystems, one of the most exciting regenerative medicine applications it is currently exploring is the bioprinting of pancreatic tissue, which could help in the treatment of diabetes patients.

The moonshot for the bioprinting industry is to create whole replacement organs, the company tells us. While this is a great goal, it is still a long way off. At Aspect, we are focused on restoration of function versus replication of entire organ structure. Many diseases and disorders are a result of missing or lost function, in which case patients dont necessarily need a whole new organthey need an implantable tissue that replaces the lost function of the damaged organ.

This is particularly true of people living with type 1 diabetes. A patient with type 1 diabetes has a pancreas that does not perform its intended functions of secreting insulin and regulating blood sugar levels. Therefore, we are not aiming to create a replacement pancreas, but rather an implantable tissue therapeutic that would restore the the intended functions of the organ.

Microfluidic-based 3D bioprinting enables the creation of well-defined microstructures, thus allowing us to print a tissue structure with specific features such as immune-protection, vascularization, or production of hormones like insulin, says Aspect Biosystems. In the case of pancreatic tissue, microfluidics gives us the ability to print a multi-layered structure with a core of insulin-producing pancreatic islet cells (stem cell-derived beta-like cells) surrounded by an immuno-protective layer.

Our goal is to use these bioprinted encapsulated pancreatic beta cells as the foundation for developing an implantable 3D tissue therapeutic. Adding an outer layer that immune cells cant penetrate, but that allows diffusion of insulin, would ensure the success of the implanted tissue therapeutic as a long-term treatment for patients with type 1 diabetes without the need for immunosuppressant therapy.

As Aspect Biosystems moves forward in its bioprinting R&D, it is supported by a recent investment of $20 million, led by VC firm Radical Ventures. This injection of capital will help the company to accelerate its bioprinting workboth for the pancreatic tissue development and beyond.

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Aspect Biosystems on the restorative capacity of bioprinting today - 3DPMN

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TheGlobalAutologous Stem Cell and Non-Stem Cell Based Therapies Marketis expected to reach USD113.04 billion by 2025, from USD 87.59 billion in 2017 growing at a CAGR of 3.7% during the forecast period of 2018 to 2025. The upcoming market report contains data for historic years 2015 & 2016, the base year of calculation is 2017 and the forecast period is 2018 to 2025.

For In depth Information Get Sample Copy of this Report @https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-autologous-stem-cell-and-non-stem-cell-based-therapies-market&raksh

Some of the major players operating in the global autologous stem cell and non-stem cell based therapies market areAntria (Cro), Bioheart, Brainstorm Cell Therapeutics, Cytori, Dendreon Corporation, Fibrocell, Genesis Biopharma, Georgia Health Sciences University, Neostem, Opexa Therapeutics, Orgenesis, Regenexx, Regeneus, Tengion, Tigenix, Virxsys and many more.

The data and information included in this Global Autologous Stem Cell And Non-Stem Cell Based Therapies business report helps businesses take sound decisions and plan about the advertising and sales promotion strategy more successfully. This Autologous Stem Cell And Non-Stem Cell Based Therapies market research report is generated by taking into account a range of objectives of market research that are vital for the clients success. This report also includes strategic profiling of key players in the market, systematic analysis of their core competencies, and draws a competitive landscape for the Healthcare industry. The Global Autologous Stem Cell And Non-Stem Cell Based Therapies business report includes market shares for global, Europe, North America, Asia Pacific and South America.

Market Definition:Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market

In autologous stem-cell transplantation persons own undifferentiated cells or stem cells are collected and transplanted back to the person after intensive therapy. These therapies are performed by means of hematopoietic stem cells, in some of the cases cardiac cells are used to fix the damages caused due to heart attacks. The autologous stem cell and non-stem cell based therapies are used in the treatment of various diseases such as neurodegenerative diseases, cardiovascular diseases, cancer and autoimmune diseases, infectious disease.

According to World Health Organization (WHO), cardiovascular disease (CVD) causes more than half of all deaths across the European Region. The disease leads to death or frequently it is caused by AIDS, tuberculosis and malaria combined in Europe. With the prevalence of cancer and diabetes in all age groups globally the need of steam cell based therapies is increasing, according to article published by the US National Library of Medicine National Institutes of Health, it was reported that around 382 million people had diabetes in 2013 and the number is growing at alarming rate which has increased the need to improve treatment and therapies regarding the diseases.

Browse Detailed TOC Herehttps://www.databridgemarketresearch.com/toc/?dbmr=global-autologous-stem-cell-and-non-stem-cell-based-therapies-market&raksh

Market Segmentation:Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market

Competitive Analysis:Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market

The global autologous stem cell and non-stem cell based therapies market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of autologous stem cell and non-stem cell based therapies market for global, Europe, North America, Asia Pacific and South America.

Major Autologous Stem Cell and Non-Stem Cell Based Therapies Market Drivers and Restraints:

Introduction of novel autologous stem cell based therapies in regenerative medicine

Reduction in transplant associated risks

Prevalence of cancer and diabetes in all age groups

High cost of autologous cellular therapies

Lack of skilled professionals

Customization of the Report:

Key benefits of buying the Autologous Stem Cell And Non-Stem Cell Based Therapies Market Report:

This Autologous Stem Cell And Non-Stem Cell Based Therapies Market report will enable both of the sides in market be an established firm or a relative new entrant. It helps the established firms to know about the moves which are being performed by their competitors and also helps the new entrants by educating them about the market situations and the industry trends. This Autologous Stem Cell And Non-Stem Cell Based Therapies Market report is quite fruitful in helping to understand the market definition and all the aspects of the market including the CAGR value and key profiles.

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Autologous Stem Cell And Non-Stem Cell Based Therapies Market 2020-2025 Booming || Leadinf Players Fibrocell, Genesis Biopharma, Georgia Health...

Cardiac Stem Cells Comments Off on Autologous Stem Cell And Non-Stem Cell Based Therapies Market 2020-2025 Booming || Leadinf Players Fibrocell, Genesis Biopharma, Georgia Health… | February 17th, 2020

Scientific discoveries and research missions beyond Earths surface are quickly moving forward. Advancements in the fields of research, space medicine, life, and physical sciences, are taking advantage of the effects of microgravity to find solutions to some big problems here on Earth. Researchers in 3D printing and bioprinting have taken advantage of space facilities that are dedicated to conducting multiple experiments in orbit, such as investigating microgravitys effects on the growth of three-dimensional, human-like tissues, creating high-quality protein crystals that will help scientists develop more effective drugs, and even growing meat with 3D printing technology.

The BioFabrication Facility (BFF) by Techshot and nScrypt (Credit: Techshot)

On November 2, 2019, a Northrop Grumman Antares rocket successfully launched a Cygnus cargo spacecraft on a mission to the International Space Station (ISS). The payload aboard the Cygnus included supplies for the 3D BioFabrication Facility (BFF), like human cells, bioinks, as well as new 3D printed ceramic fluid manifolds that replaced the previously used that were printed out of polymers. According to Lithoz the company behind the 3D printed ceramic fluid manifolds they are enabling advancements in bioprinting at the ISS.

The additive manufactured ceramics have been in service since November 2019 and Lithoz claims they have proven to provide better biocompatibility than printed polymers, resulting in larger viable structures.

Lithoz, a company specializing in the development and production of materials and AM systems for 3D printing of bone replacements and high-performance ceramics, printed the ceramic manifolds using lithography-based ceramic manufacturing (LCM) on a high-resolution CeraFab printer in collaboration with Techshot, one of the companies behind the development of the BFF. Moreover, the ceramic fluid manifolds are used inside bioreactors to provide nutrients to living materials in space by the BFF.

Testing of the ceramic 3D printed manifolds is focusing on biocompatibility, precision, durability, and overall fluid flow properties; and the latest round of microgravity bioprinting in December yielded larger biological constructs than the first BFF attempts in July.

NASA engineer Christina Koch works with the BioFabrication Facility in orbit (Credit: NASA)

Techshot and Lithoz engineers and scientists worked together to optimize the design and the manufacturing processes required to make it. Techshot Senior Scientist Carlos Chang reported that its been an absolute pleasure working with Lithoz.

While Lithoz Vice President Shawn Allan suggested that their expertise in ceramic processing really made these parts happen. The success of ceramic additive manufacturing depends on working together with design, materials, and printing. Design for ceramic additive manufacturing principles was used along with print parameter control to achieve Techshots complex fluid-handling design with the confidence needed to use the components on ISS.

Headquartered in Vienna, Austria, and founded in 2011, Lithoz offers applications and material development to its customers in cooperation with renowned research institutes all over the world, benefiting from a variety of materials ranging from alumina, zirconia, silicon nitride, silica-based for casting-core applications through medical-grade bioceramics.

This work, in particular, highlighted an ideal use case for ceramic additive manufacturing to enable the production of a special compact device that could not be produced without additive manufacturing while enabling a level of bio-compatibility and strength not achievable with printable polymers. Lithoz reported that Techshot engineers were able to interface the larger bio-structures with the Lithoz-printed ceramic manifolds and that the next steps will focus on optimized integration of these components and longer culturing of the printed biological materials. While conditioned human tissues from this mission are expected to return to Earth in early 2020 for evaluation.

Back in July 2019, Gene Boland,chief scientist atTechshot, and Ken Church, chief executive officer atnScrypt, discussed the BFF at NASAs Kennedy Space Center in Port Canaveral, Florida, how they planned to use the BFF in orbit to print cells (extracellular matrices), grow them and have them mature enough so that when they return to Earth researchers can encounter a close to full cardiac strength. Church described how a tissue of this size has never been grown here on Earth, let alone in microgravity. The 3D BFF is the first-ever 3D printer capable of manufacturing human tissue in the microgravity condition of space. Utilizing adult human cells (such as pluripotent or stem cells), the BFF can create viable tissue in space through a technology that enables it to precisely place and build ultra-fine layers of bioink layers that may be several times smaller than the width of a human hair involving the smallest print nozzles in existence.

Flight engineer Andrew Morgan works with the BioFabrication Facility (Credit: NASA)

Experts suggest that bioprinting without gravity eliminates the risk of collapse, enabling organs to grow without the need for scaffolds, offering a great alternative to some of the biggest medical challenges, like supplying bioprinted organs, providing a solution to the shortage of organs.

With NASA becoming more committed to stimulating the economy in low-Earth orbit (LEO), as well as opening up the ISS research lab to scientific investigations and experiments, we can expect to learn more about some of the most interesting discoveries that could take place 220 miles above Earth. There are already quite a few bioprinting experiments taking place on the ISS, including Allevi and Made In Spaces existing Additive Manufacturing Facility on the ISS, the ZeroG bio-extruder which allow scientists on the Allevi platform to simultaneously run experiments both on the ground and in space to observe biological differences that occur with and without gravity, and CELLINKs collaboration with Made In Space to identify 3D bioprinting development opportunities for the ISS as well as for future off-world platforms. All of these approaches are expected to have an impact on the future of medicine on Earth.

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Improvements to the BioFabrication Facility on the ISS Thanks to Lithoz - 3DPrint.com

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The following are the top rated Healthcare stocks according to Validea's Small-Cap Growth Investor model based on the published strategy of Motley Fool. This strategy looks for small cap growth stocks with solid fundamentals and strong price performance.

CORCEPT THERAPEUTICS INCORPORATED (CORT) is a small-cap growth stock in the Biotechnology & Drugs industry. The rating according to our strategy based on Motley Fool is 83% based on the firms underlying fundamentals and the stocks valuation. A score of 80% or above typically indicates that the strategy has some interest in the stock and a score above 90% typically indicates strong interest.

Company Description: Corcept Therapeutics Incorporated is a pharmaceutical company engaged in the discovery, development and commercialization of drugs for the treatment of severe metabolic, psychiatric and oncologic disorders. The Company is developing mifepristone, a compound that modulates the effects of cortisol by acting as a competitive antagonist at the glucocorticoid receptor (GR). As of December 31, 2016, it had discovered three structurally distinct series of selective cortisol modulators, all of which share mifepristone's affinity for GR but, unlike mifepristone, do not bind to the progesterone receptor. It is conducting two clinical trials of its selective cortisol modulator, CORT125134. One trial is investigating CORT125134 as a treatment for patients with Cushing syndrome. The second trial is investigating the combination of CORT125134 and nab-paclitaxel (Celgene Corporation's Abraxane) to treat patients with solid-tumor cancers.

The following table summarizes whether the stock meets each of this strategy's tests. Not all criteria in the below table receive equal weighting or are independent, but the table provides a brief overview of the strong and weak points of the security in the context of the strategy's criteria.

For a full detailed analysis using NASDAQ's Guru Analysis tool, click here

ZYNEX INC. (ZYXI) is a small-cap growth stock in the Medical Equipment & Supplies industry. The rating according to our strategy based on Motley Fool is 83% based on the firms underlying fundamentals and the stocks valuation. A score of 80% or above typically indicates that the strategy has some interest in the stock and a score above 90% typically indicates strong interest.

Company Description: Zynex, Inc. operates through the Electrotherapy and Pain Management Products segment. The Company conducts its business through its subsidiaries and the operating subsidiary is Zynex Medical, Inc. (ZMI). Its other subsidiaries include Zynex Monitoring Solutions, Inc. (ZMS) and Zynex Europe, ApS (ZEU). ZMI designs, manufactures and markets medical devices that treat chronic and acute pain, as well as activate and exercise muscles for rehabilitative purposes with electrical stimulation. ZMS is in the process of developing its blood volume monitoring product for non-invasive cardiac monitoring. ZEU intends to focus on sales and marketing its products within the international marketplace, upon receipt of necessary regulatory approvals. It markets and sells Zynex-manufactured products and distributes private labeled products. Its products include NexWave, NeuroMove, InWave, Electrodes and Batteries. ZMI devices are intended for pain management to reduce reliance on drugs and medications.

The following table summarizes whether the stock meets each of this strategy's tests. Not all criteria in the below table receive equal weighting or are independent, but the table provides a brief overview of the strong and weak points of the security in the context of the strategy's criteria.

For a full detailed analysis using NASDAQ's Guru Analysis tool, click here

INMODE LTD (INMD) is a small-cap growth stock in the Medical Equipment & Supplies industry. The rating according to our strategy based on Motley Fool is 79% based on the firms underlying fundamentals and the stocks valuation. A score of 80% or above typically indicates that the strategy has some interest in the stock and a score above 90% typically indicates strong interest.

Company Description: Inmode Ltd is an Israel-based company. It designs, develops, manufactures and commercializes energy-based, minimally-invasive surgical aesthetic and medical treatment solutions. The Company's proprietary technologies are used by physicians to remodel subdermal adipose, or fatty, tissue in a variety of procedures including fat reduction with simultaneous skin tightening, face and body contouring and ablative skin rejuvenation treatments. Its products target a wide array of procedures including simultaneous fat killing and skin tightening, permanent hair reduction, skin appearance and texture, among others. The Company's products may be used on a variety of body parts, including the face, neck, abdomen, upper arms, thighs and intimate feminine regions. It owns six product platforms: BodyTite, Optimas, Votiva, Contoura, Triton and EmbraceRF. All are market and sell traditionally to plastic and facial surgeons, aesthetic surgeons and dermatologists, among others.

The following table summarizes whether the stock meets each of this strategy's tests. Not all criteria in the below table receive equal weighting or are independent, but the table provides a brief overview of the strong and weak points of the security in the context of the strategy's criteria.

For a full detailed analysis using NASDAQ's Guru Analysis tool, click here

MEDPACE HOLDINGS INC (MEDP) is a mid-cap growth stock in the Biotechnology & Drugs industry. The rating according to our strategy based on Motley Fool is 76% based on the firms underlying fundamentals and the stocks valuation. A score of 80% or above typically indicates that the strategy has some interest in the stock and a score above 90% typically indicates strong interest.

Company Description: Medpace Holdings, Inc. is a clinical contract research organization. The Company provides clinical research-based drug and medical device development services. The Company partners with pharmaceutical, biotechnology, and medical device companies in the development and execution of clinical trials. The Company's drug development services focus on full service Phase I-IV clinical development services and include development plan design, coordinated central laboratory, project management, regulatory affairs, clinical monitoring, data management and analysis, pharmacovigilance new drug application submissions, and post-marketing clinical support. The Company also provides bio-analytical laboratory services, clinical human pharmacology, imaging services, and electrocardiography reading support for clinical trials. The Company's operations are principally based in North America, Europe, and Asia.

The following table summarizes whether the stock meets each of this strategy's tests. Not all criteria in the below table receive equal weighting or are independent, but the table provides a brief overview of the strong and weak points of the security in the context of the strategy's criteria.

For a full detailed analysis using NASDAQ's Guru Analysis tool, click here

CRISPR THERAPEUTICS AG (CRSP) is a mid-cap growth stock in the Biotechnology & Drugs industry. The rating according to our strategy based on Motley Fool is 72% based on the firms underlying fundamentals and the stocks valuation. A score of 80% or above typically indicates that the strategy has some interest in the stock and a score above 90% typically indicates strong interest.

Company Description: CRISPR Therapeutics AG is a Switzerland-based gene-editing company. It focuses on the development of transformative gene-based medicines for serious diseases. The Company develops its products using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 gene-editing platform, which allows for precise directed changes to genomic deoxyribonucleic acid (DNA). The Company has a portfolio of therapeutic programs in a range of disease areas, including hemoglobinopathies, oncology, regenerative medicine and rare diseases. Its lead product candidate is CTX001, an ex vivo CRISPR gene-edited therapy for treating patients suffering from transfusion-dependent beta thalassemia or severe sickle cell disease in which a patient's hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin in red blood cells. The Company has business operations in London and the United Kingdom, as well as research and development operations in the United States.

The following table summarizes whether the stock meets each of this strategy's tests. Not all criteria in the below table receive equal weighting or are independent, but the table provides a brief overview of the strong and weak points of the security in the context of the strategy's criteria.

For a full detailed analysis using NASDAQ's Guru Analysis tool, click here

Since its inception, Validea's strategy based on Motley Fool has returned 625.78% vs. 240.03% for the S&P 500. For more details on this strategy, click here

About Motley Fool: Brothers David and Tom Gardner often wear funny hats in public appearances, but they're hardly fools -- at least not the kind whose advice you should readily dismiss. The Gardners are the founders of the popular Motley Fool web site, which offers frank and often irreverent commentary on investing, the stock market, and personal finance. The Gardners' "Fool" really is a multi-media endeavor, offering not only its web content but also several books written by the brothers, a weekly syndicated newspaper column, and subscription newsletter services.

About Validea: Validea is an investment research service that follows the published strategies of investment legends. Validea offers both stock analysis and model portfolios based on gurus who have outperformed the market over the long-term, including Warren Buffett, Benjamin Graham, Peter Lynch and Martin Zweig. For more information about Validea, click here

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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Validea's Top Five Healthcare Stocks Based On Motley Fool - 2/16/2020 - Nasdaq

Skin Stem Cells Comments Off on Validea’s Top Five Healthcare Stocks Based On Motley Fool – 2/16/2020 – Nasdaq | February 17th, 2020

Heres the truth about serum: Its the biggest secret to having younger-looking, radiant skin.

You know all the other steps to staying healthyand showing it: wearing SPF, and applying retinol. You stay hydrated and eat healthy foods. You get lots of exercise and plenty of sleep. But, are you applying serums? They can give your skin some of the most immediate and impactful benefits.

Serum seeps into all three layers of your skin to work its magic at deeper levels than moisturizer ever could. Those moisturizing creams merely sit atop your skin. Theyre equally important, and shield skin from toxins, but they do little to reverse dark spots and smooth wrinkles. Serums, on the other hand, can correct hyperpigmentation and puffy eyes.

Serum brightens your skin complexion, and can preserve moisture levels in ways that moisturizers never could (funny, given its name). No two serums are alike in terms of ingredients and overall benefits, but almost all of them brighten your skin and smooth complexion. Here are seven of our favorite serums; apply them to clean, dried skin before moisturizers, SPFs, retinol or night creams.

If your skin suffers from redness or discoloration, then look for a corrective serum. SkinCeuticals gel serum uses thyme and cucumber extract to soothe irritation, as well as mulberry extract to even skin tone and gently hydrate the skin. If most moisturizers make your skin break out, then this is a gentle and top-notch moisturizing alternative.

Vitamin C is one of the best ingredients you can apply to skin for brighter, more even complexion. Glossiers serum is packed with it, along with magnesium, for an instant pick-me-up for tired or dull skin. Use it daily to preserve a fresh face, and to get ahead of the inevitable hangovers or early mornings.

The skin around your eyes is more sensitive than the rest of your face, and is much thinner, too hence why you quickly show signs of tiredness. (Its fluid buildup from a lack of rest, shining through your skin.) So, target the eyes with a special-blend serum that tightens and brightens at the same time. In OSEAs eye serum, hyaluronic acid preserves firmness and moisture levels, grape-seed-derived resveratrol shields this thin skin from toxins, and algae extract plumps and nourishes tired eyes (and alert eyes, if youre plenty rested already everyone benefits). It also prevents and helps reverse fine lines and other signs of aging.

Some serums are best worn during the day those that focus on hydration and pure brightening. Others, like corrective and exfoliating serums, are often best suited for bedtime so that they can sync with your bodys circadian rhythm to boost cellular turnover (and in turn guarantee that you wake up looking rested and more youthful). However, some serums cover both bases: Brickells night serum is ultra-hydrating so much so that its impact is maximized by wearing it overnight. Its loaded with plant stem cells, hyaluronic acid and vitamin C, and promises to magnify the benefits of a full nights sleep. Your friends and colleagues will notice the difference within days. (Assuming you arent pairing it with a lovely binge-drinking or chain-smoking habit.)

Theres an entire family of serums that focuses on exfoliation, by using ingredients that dissolve dead skin cells and reduce hyperpigmentation (dark spots and discoloration). Herbivores serum does so with alpha- and beta-hydroxy acids, all of which are naturally derived, despite their sciencey-sounding names. Theyre extracted from things like willow bark and sugar maple, and in turn leave skin bright, while minimizing pore size and smoothing skin texture.

Its hard to categorize Caudelies skin-brightening serum, since it uses the brands proprietary blend of ingredients (Viniferine, derived from grape vines) but they seem to deliver on their promise to brighten skin 62 times better than Vitamin C. All around the globe, this is what Caudelies spa customers pay top dollar for, and you can enjoy the same complexion-smoothing effects for less than a buck a day (assuming you just use a couple drops daily).

If your skin texture needs attention just as much as your skin tone, then look for a corrective and smoothing serum. This one from PCA Skin is pumped with peptides, and it waxes poetic on your skin to undo signs of aging (like the appearance of fine lines, wrinkles, dark spots and acne spots). It also helps skin retain moisture thanks to the inclusion of hyaluronic acid, rice bran extract, squalene, ensuring firmness and brightness.

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The Best Serums to Brighten and Refresh Dull, Tired Skin - gearpatrol.com

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Stephanie Singleton for BuzzFeed News

Do you miss being a scientist? some people ask.

Sometimes.

When people talk about science, they usually mean people in white lab coats doing things, like solving equations on the board or preparing solutions in beakers. What they mean is science as this crude mechanism of discovery by which humans refine over decades and centuries a small kernel of knowing. What they mean is grant dollars. What they mean is wild hair. What they mean is clean, aseptic, analytical. Brainy little robot people. White.

I try to be honest about my time in science about the feeling of satisfaction I had when I plotted all of my confocal data and there was a beautiful curve depicting the drop-off in signal as one moved further down the tissue of the gonad. I think about the calculations we did on scraps of paper to check the ratios of inheritance of the genes we introduced. I think of the little side room where we took our coffee and bagels. I think of the feeling of friendship and family that comes with being in a big lab, where everyone has a place, a role, an expertise, a skill. I remember the surprise I felt when people started to come to me because I knew something, because I could help. And how rare that was for me.

For the better part of several years, I saw my labmates every day. For hours and hours. Every holiday, every break, we stayed. We worked. We supported each other. We fought. We feuded. We gossiped. We threw parties for each other. We celebrated. We said goodbye at graduations and retirements. There were people who supported me and cherished me and looked after me. People who treated me like I mattered. A lab is a family. In a way.

Science was beautiful and it was wild and it was unknowable. Science was spending days and weeks on a single experiment with no way to know if it would work and no real way to tell if it had worked. Science was like trying to find your way to a dark forest only to realize that you had always been inside of the forest and that the forest is inside of another, greater, darker forest. Science was laughing with my labmates about television the night before, about the song of the summer, about tennis, about the unruly nature of mold growing on our plates, about cheap wings at Buffalo Wild Wings. Science was being taught to think. Taught to speak. Science was a finishing school. Science was a brutal education. Science made me ruthless. Science made me understand the vast beauty of the world.

But science was also working 15 hours a day for weeks or months. Science was working weekends and holidays. Science was being called lazy for taking a break. Science was the beat of doubting silence after I answered a question put to me. Science was being told that racism was not racism. Science was being told that I was fortunate that I had running water while growing up and that I was actually privileged because there are some places that do not. Science was being told that I was mistaken for a waiter at a party because I had worn a black sweater. Science was being told that I had to work harder despite working my hardest. Science was being told that I talked too much. Science was being told that I was too loud. Science was being told that I was behind, always behind. Science was being told that I had failed but had been gifted a pass by virtue of who you are. Science was being told that I had never once been to class despite attending every session and office hour because I was mistaken for someone else.

Science was being the only black person in the program for four years. Science was saying nothing because I was tired of being corrected about the particulars of my own experience. Science was being told that I should consider moving to the other side of town where more black people live. Science was someone suggesting that I find a church in order to find community. Science was having my hair stroked and touched. Science was being told that I was articulate. Science was watching peoples eyes widen slightly in surprise when I told them what program I was in. Science was the constant humiliation of wondering if I had justified my presence or if I had made it harder for the next black person to get admitted. Science was having to worry about that in the first place.

Science was a place I ultimately left, not so much because I wanted to, but because I had to. Science is not being able to say that because I reflexively feel the rebuttal waiting on the other end of that sentence: You could have made it work if you wanted it enough. Science is not knowing whether I wanted it enough.

Does science influence your writing?

Oh, sure. I guess.

Do you write science fiction?

No, I write domestic realism.

After the above exchange, people sometimes look at me like Im joking and at any moment will drop the faade to reveal that I do in fact write and love science fiction, after all.

But no, I do not write science fiction. I think that if people knew more scientists and spent significant time in their company, they would understand that the worst possible preparation for a career as a science fiction writer is an intensive science education. My training as a scientist makes it difficult to absent myself in the way I need to in order to write good fiction. I can never turn off the part of my brain that knows about protein folding or microscopy or tissue preparation or stem cells or physics or chemistry. Writing science fiction would be an extended exercise in pedantry.

People presume that science and writing are quite different. But they are both ways of knowing. They are ways of understanding the greater mystery of the world. They are systems of knowledge and inquiry. I do not understand something until I have written it, or more accurately put, until I have written my way through it.

Science was being the only black person in the program for four years. Science was saying nothing because I was tired of being corrected about the particulars of my own experience.

I think in many ways, the best preparation for a writer is a period of prolonged and rigorous thought about a difficult and complicated question. You learn to assemble your resources. You learn to fight with yourself. You learn to quarrel on the page with your worst ideas and with the ones you hold dearest. You treat your expectations with suspicion. You demand proof. You demand evidence. You think hard about the alternate hypothesis or other explanations, and you devise strategies to root these out. You learn to live with doubt. You try to prove yourself wrong. You look for places where you have been too soft. Too vague. You eliminate language that contains falsehoods. You eliminate language that can mislead your reader. You ask questions. You pursue answers with all the energy you can muster. You try to put language to what it is you observe. You develop a stamina for iteration. You develop a thick skin. You learn to seek criticism. You treat criticism like kindness. You churn the raw material of life into something that can be understood, and when you fail, you marvel at the mystery of things.

Do you miss science?

Yes. No. Yes. No.

Sometimes, when I dont feel well, I consider the question of how to derive an expression for the degradation of a molecular species in a particular tissue under a given set of circumstances. Old calculus. I turn to YouTube lectures from MIT about thermodynamics. I think of my first winter in Madison, Wisconsin.

The first snowfall was in October. It had been a hot, rainy summer, so much so that the weather seemed to turn all at once with very little warning. I was either in the middle or at the start of my second rotation as a biochemistry graduate student, working in a biophysical chemistry lab and spending most of my day in the windowless instrument facility in the basement. My project was to deduce the effect of protein concentration on the ability of a polymer of DNA to wind itself. I spent a lot of time pipetting various liquids into each other in little cuvettes, slotting them into a machine, and then waiting for the reading. It was the kind of work to which I felt ideally suited, and I could have gone on that way forever. I had recently moved to the Midwest from Alabama to pursue a PhD, and it seemed as likely as anything else that I would go on pipetting and measuring the effect of things like DNA polymer length and protein concentration on DNA winding. It was as removed from the circumstances of my previous life as anything else, and so I didnt have a compelling reason to doubt that this would be the shape my life held.

But I remember sitting down at the desk in the lab and looking out the broad window. There was a large tree at the center of the courtyard that had recently turned yellow. Fall was there in name, but not in temperature. The labs were kept quite cold, and so I wore a sweater indoors and shucked it as soon as I got outside. But that day, I looked out of the window and saw snow drifting down. The flakes were thick and fluffy, and they seemed almost fake. It was the first time I had seen snow in years, and I was totally enamored by it. The other people in the lab were on edge because snow in October portended something dark and awful a hard winter, a long, brutal freeze. Where they saw inconvenient travel and slushy roads, I saw something beautiful if frivolous, a minor novelty. Winter came early that year, and it didnt end until the very beginning of the following summer. When I went to the lake on my birthday in early June, there was still ice in the water.

People presume that science and writing are quite different. But they are both ways of knowing.

When people ask me about my time in science, it is this day which presents itself to me in jewel-like clarity. It is the day something about my life altered irrevocably. Or perhaps it is that the snow has accumulated, the way all such moments do in life, the weight of meaning, of prophecy. Inevitability is an artifact of retrospection. It is because the snow represented a stark deviation from the previous course of events in my life, at the precise moment when my life was changing so wildly, that I remember it. It is not that the snow changed me, but it came at a point when I was starting not to resemble myself. I cannot use the snow to explain to people what my life was like in science. It has the whiff of superstition, folklore. It feels too much like a memory and not enough like an answer. I do not tell them about the snow or how it seemed a benediction at the outset of something I needed desperately to work.

It was only later that I realized this was wishful thinking, and that the snow was just snow.

Do you think youd ever go back to science?

That part of my life is over now.

Ive come to understand that what people want in such a situation is to have their own conceptions of the world confirmed. That is, they want me to say that when you leave science because you have written a novel and a book of stories and have decided to attend an MFA program in creative writing, you are doing something that is antithetical to science. People presume that it is akin to picking up and leaving your home in the middle of the night under great duress, never to return. What they want is the spectacle of the forgotten treasured item, the confirmation that something has been lost, perhaps forever.

I think if people knew what it was that I left, then theyd know better than to ask. It would be like asking someone if they were sad to have left their home with no prospect of returning. It would be like asking someone if they were sad to have left their faith behind. It would be like asking someone if they were sad to have given up some fundamental idea about who they are. It would be like asking someone if they were sad to have watched their life burn to the ground. It would be like asking someone if they were sad to have left their family and friends.

They would mind their own business if they knew.

But they do not know, and so they say things like Science, wow, thats so cool, like, do you miss it?

And I smile because that is what I have learned to do. Because explaining is too hard. Too messy. There is no clean or easy or simple way to make it known to others that I left because I had to, because it was necessary to leave that I do miss it, but I also dont because Im still that person but not that person, that every day I remind myself less of the person I was then. Its sad, like losing a memory of myself, and all those years are lost to me now, all the little tricks and habits of home dropping down and away, as I become this other person known for this other thing, and its too much in the moment to say that I miss it both more and less every day, that I become a person more capable of appreciating what is lost in the grand scheme of things but less a person who knows what it is Ive actually lost, and that there is some painful, brutal, awful misalignment in the scale of those two losses.

When people ask if I miss science, the only answer available to me is an incomplete solution to the problem: Yes. No. Sometimes. Its over now.

Brandon Taylor is the senior editor of Electric Literatures Recommended Reading and a staff writer at Literary Hub. His writing has earned him fellowships from Lambda Literary Foundation, Kimbilio Fiction, and the Tin House Summer Writer's Workshop. He holds graduate degrees from the University of Wisconsin-Madison and the University of Iowa, where he was an Iowa Arts Fellow at the Iowa Writers Workshop in fiction. Learn more about his first novel Real Life here.

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Working In Science Was A Brutal Education. Thats Why I Left. - BuzzFeed News

Skin Stem Cells Comments Off on Working In Science Was A Brutal Education. Thats Why I Left. – BuzzFeed News | February 17th, 2020

When you see Kelly Schneider and Alex Barr together, you'd think they go way back; they laugh at the same moments, seem to have inside jokes, and generally send off a vibe that they're old friends.

"Our families are both Middle Eastern, so we just have this connection. Weve just been gabbing and eating like we know each other," Schneider told Action News at her mother's house in Bloomfield Hills on Sunday.

It's where Barr and her mother came from the Boston area to meet Schneider and her family or the very first time in person, and to say thank you, since sharing something pretty personal back in August of 2018.

"I mean, she kind of is morphing into me now that she has my DNA. Thats how this works, right?" Schneider joked.

About a year earlier, in the summer of 2017 Barr, then in graduate school in the Boston area, learned she had Leukemia for the second time.

"It was just unreal. Like I couldnt even process it," Barr told Action News.

Barr didn't know it then, but Schneider had already signed up with Be The Match, and a national bone marrow registry, when she learned a close friend was diagnosed with cancer.

"We went and got tested and we donated blood. And unfortunately she did not survive. But after 4 or 5 months after she passed away, I got a call from Be The Match.

That call was on behalf of Barr, hoping Schneider might be willing to donate life-saving bone marrow.

Soon after, Schneider was getting treatment to donate stem cells from her bone marrow, to save Barr's life, who was still a stranger at the time.

All Schneider knew then was that her donation was going to help a 24-year-old from Michigan.

"How could you not? If someone needs it?" She said.

"When you hear bone marrow donation that sounds scary like theyre going to drill into your bone or something," Barr said, noting that it really wasn't as intense of a procedure as some people may think.

In this case, Schneider had to get a series of shots, the stem cells were collected, and then shipped to Boston for Barr, who is now in remission.

Its incredible. Like, I cant even describe. And I know that I would do the same," Barr said.

First, the two communicated communicated through the registry.

"We had been talking back and forth like online since September. I could tell that we would really hit it off," Barr told Action News.

Then, they decided to meet in person at Schneider's mother's house.

Not only do the two now share some of the same DNA, they keep finding other things they have in common.

Like a photo of Barr's cousin, which looks strikingly similar to Schneider.

I look like her! she said, pointing at the photo Barr brought with her.

For both Barr and Schneider, this full-circle experience is a reminder of how important the the Be The Match registry is, for the thousands of people waiting to find their life-saving donor, and just possibly, a life-long connection too.

Barr, who is now a healthy 26-year-old, is working in the health field. She works in the Hemostasis and Thrombosis Division at Beth Israel Deaconess Medical Center (BIDMC), hoping to help others who have been diagnosed with potentially terminal diseases.

Barr said her experience beating Leukemia inspired her to go into the medical field as a biologist to study diseases of the blood.

She is a currently also a volunteer with Be The Match, and conducts her own registry drives as living proof of how important the registry is and how bone marrow donations can save lives.

Click here to join the register or the learn more about the Be The Match.

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News Royal Oak native meets woman she saved with vital stem cell donation Jenn Schanz 11 - WXYZ

Bone Marrow Stem Cells Comments Off on News Royal Oak native meets woman she saved with vital stem cell donation Jenn Schanz 11 – WXYZ | February 17th, 2020

Brynn Niblock, FHSU junior in pre-med from Hoxie, swabs her cheek as part of the sign up for the Be the Match bone marrow registry Feb. 6 at Gross Memorial Coliseum.

By CRISTINA JANNEYHays Post

Usually Tiger basketball games are a time to have fun cheer the home team, eat some popcorn but students and community members at Feb. 6 game took a few minutes to stop and potentially save a life.

FHSU student health sponsored a Be the Match bone marrow registry drive.

Potential donors ages 18 to 44 answered a list of qualifying health questions on their smartphones and then swabbed their cheeks to be matched with a potential cancer sufferer in need of bone marrow transplants.

Kathy Pyke of Hays knows too the well the importance of the registry. Pyke was at Gross Memorial Coliseum the night of the drive as a volunteer handing out information to potential donors.

Her husband, Tom, was diagnosed with leukemia on March 1, 2014. Family members were tested, and they were not matches. Doctors were also unable to find a bone marrow match on the national registry. There were 6.2 million people in the registry at the time.

In lieu of a bone marrow transplant, Pyke was given donated umbilical cord blood.

Initially the treatment improved Pyke's condition. However, he ultimately died as a result of the disease on Feb. 12, 2015 at the age of 62.

Kathy said the family was rocked by Tom's illness. He was playing golf and went fishing the week before he was diagnosed with cancer.

Kathy said she wishes she could be on the registry to help another family, but her age prevents her from doing so.

"Not only for my husband," she said of the importance of the registry. "I did pray there had been a match. We stayed at the Hope Lodge that was run by the American Cancer Society in Kansas City. There were 45 apartments there and everyone there has someone who has cancer plus a caregiver in it. You just see so many lives being touched. ...

"If this is something that can help somebody, it is just an easy thing to do."

Kathy said she had a good friend who had a family member sign up for the registry, and he was able to donate to someone who had cancer in England.

Pyke said she would also like to see more hospitals participate in the cord blood bank, which is what helped her husband. At the time of Tom's illness, HaysMed was not participating in the umbilical cord blood bank.

Michelle Toogood, BSN, RN, supervisor of Hays Meds Women's andInfant Care Center/NICU, said parents wishing to participate in cord blood donation should initiate the process prior to delivery. HaysMed staff will then aid in the collection of the specimen.

"I just can't express how much people need to do this," Pyke said of signing up for the registry. "It is just so easy to swab test and they could potentially save more than one person's life. It is so easy to do and so important."

If you are identified as a match to someone suffering from cancer, you would be contacted through the registry and asked if you are willing to donate,Amanda McCord, RN at the FHSU student health center.

"Finding the perfect match is essential for people who are fighting this type of cancer," McCord said. "The closer the match the better their chances of remission and beating whatever cancer they are fighting."

There are over 70 diseases that can be treated by bone marrow transplants, according to Be the Match.

Physicians will usually look for matches among relatives first, but only 70 percent of the time are matches made from family members, McCord said.

Statistics also indicate minority patients are less likely to find matches than Caucasian patients. Be the Match is trying to boost minority participation as there are fewer minority participants in the registry at this time, McCord said.

Donating bone marrow is a little bit different for every donor, McCord said.

Most give through a Peripheral Blood Stem Cell (PBSC) donation. A machine draws blood from one arm, extracts the cells it needs, and returns the remaining blood through your other arm, according to the Be the Match website.

Others give through a marrow donation. Liquid marrow is withdrawn from the back of your pelvic bone with a needle. In this case, youll receive anesthesia and feel no pain during the procedure, the Be the Match website said.

According to Be the Match,PBSC donors may experience headaches or body aches several days before collection, but these disappear shortly after donation. Most donors feel completely recovered within a few weeks.

If you missed the Be the Match event at FHSU last week, you can contact Be the Match though its website, and the organization will send you the cheek swab kit to sign up for the registry.

The Be the Match website also has information on the donation process and a link to make monetary donations to the Be the Match program.

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FHSU partners with Be the Match for bone marrow registry event - hays Post

Bone Marrow Stem Cells Comments Off on FHSU partners with Be the Match for bone marrow registry event – hays Post | February 17th, 2020

ANN ARBOR, MI They searched the world for a match.

Bennett Sevack needed a bone marrow transplant. He was diagnosed with myelodysplastic syndrome after treatment for chronic lymphocytic leukemia. But there was no match for the Ann Arbor resident in the international registry.

So, his family started making calls.

Doctors first tried his siblings and cousins. When that didnt work, his sisters, friends and family spent six weeks calling and sharing flyers with synagogues around the world to encourage people to join the bone marrow registry, hoping someone with Sevacks Sephardic and Ashkenazi Jewish background would be willing to donate.

By fall, a partial match was found in an Italian obstetrics hospital in an umbilical cord blood sample. Soon after, another cord blood match was found in the U.S. It was enough to perform a transplant.

Sevack has gained an intense appreciation for donors.

You can save somebodys life..." he said. Its a blessing to do.

Finding an acceptable bone marrow donor is a challenge in general, but its that much harder for people of color and patients of complex ethnic backgrounds like Sevack, according to health care providers and families whove embarked on far-reaching searches for a match.

A white person has a 77% shot at finding a matching donor, according to Be The Match, a widely used donor registry. A black persons chances are 23%. For people of mixed backgrounds, the search can become more complex.

Part of the issue is the relatively narrow population of donors in the registry. Another part is the science: Certain HLA types, the protein that needs to match in bone marrow transplants, are more unique to specific backgrounds.

A closer look at plans for the new $920M University of Michigan hospital

Sevacks transplant was completed in October 2019 more than three years after his initial diagnosis, and four months after getting news that he would definitely need a transplant.

He spent eight weeks healing from his transplant and two weeks healing from a bout of graft-versus-host disease in the hematology oncology clinic on the seventh floor of Ann Arbors C.S. Mott Childrens Hospital.

On the same floor, a 9-year-old Ann Arbor-area patient undergoing chemotherapy for acute myeloid leukemia is in the same predicament that Sevack once faced. She may need a bone marrow transplant as well, but a donor match her ethnic background is Cantonese, Hispanic and Caucasian has yet to be found in the registry.

The search

Genetics play a major role in finding a good match, said Mark Vander Lugt, a pediatric bone marrow transplant specialist at C.S. Mott working with the 9-year-old. People get four HLA types from each parent, meaning donors need to match in at least six spots, though sometimes a stronger match is required.

You get combinations that are hard to find in the registry because of that, Vander Lugt said.

Not everyone needs a transplant once theyre diagnosed. Doctors often treat blood cancers and related illnesses with chemotherapy first killing off the bad cells and waiting to see if healthy cells regenerate. If they dont, a transplant replaces the bone marrow, where blood stem cells are made.

Mothers breast cancer pushes Ypsilanti woman to get preventative mastectomy

The registry is so low on certain populations that some began taking matters into their own hands. Athena Asklipiadis founded Mixed Marrow, a nonprofit that conducts outreach to encourage multi-ethnic bone marrow and blood cell donations. She said the misconceptions of donation is one of the largest challenges in getting people registered. She started by reaching out to Facebook groups and college clubs on the West Coast, where she is based. Eventually, families began reaching out to her to help in the donor search.

People use this phrase, When theres a cure for cancer or If theres a cure for cancer, Asklipiadis said. But these are actual blood diseases and cancers that are curable. Theyre curable by the selflessness of a donor and thats the beautiful thing about it.

Sevacks sister Laurel Bernstein was part of the group of relatives and supporters who called synagogues in search of donors. She estimated they called hundreds of people and added more than 1,000 to the donor registry while searching for Sevacks match.

The nine of us all felt so energized by what happened, Bernstein said. "We had not finished calling the list, even though we got the match. We went through to the end of the list to help pay it forward, to help other people, to get that much more DNA into the database.

The transplant

For Sevack, it was umbilical cord blood that saved his life. DNA from the two samples created enough HLA type matches to begin the transplant.

Vander Lugt said the preferred transplant method is still donor transplants, because cord blood units are smaller and in more limited supply than donor cells.

Misconceptions of transplants are another roadblock. A bone marrow harvest in which a donor undergoes surgery to collect marrow from the hip is widely believed to be painful. But experts describe the process as a mild discomfort with a two-to-three day recovery time, instead.

And direct harvest is no longer the only method. Many hospitals also practice apheresis, which collects the peripheral blood that can generate new bone marrow through a process that looks like dialysis. Theres no surgery involved.

Choosing between the two collection processes is based on multiple factors, including the health of the patient undergoing general anesthesia and doctor and donor preference, Vander Lugt said.

The value

Fear of the unknown is what affected Sevack the most going into treatment. He said he had an anxiety breakdown the morning of his transplant, when he realized what was about to happen.

My heart was to the moon, Sevack said. I was fully dressed and every part of me was drenched. In about eight minutes I had gone through this journey of fear because the reality of what was going to come was upon me. (My wife) calmed me down, I changed my clothes and this journey began. Theres been plenty of times when Ive been scared to death.

Finding a transplant match taught his wife Phyllis Sevack the importance of facing each day separately. Fighting cancer was about healing every day making big battles smaller.

The reality of it hits all at once, Phyllis Sevack said. "I wouldnt want anybody to be afraid to go through it because it is every day take it day by day. And as soon as Bennett got over that hes not doing everything on one day, (he got better mentally).

Now at home, Bennett Sevack may take a year to fully heal. He looks forward to small improvements in his quality of life, like an expanded diet and doing his own grocery shopping at Meijer, he said.

Even the routine blood and platelet donations he received in treatment were invaluable to him. They saved his life.

Where to donate blood in Washtenaw County during shortage

Every day, theyd give me sacks of stuff that made me sick to think about it, Sevack said. It was someones donation that was given, that was literally giving me life. People who donate would do an amazing service to someone. You may not know who that is but its somebody.

Potential donors can begin the registration process by filling out a questionnaire about their medical history at bethematch.org. A cheek swab will confirm whether you meet the medical guidelines for donation. You may remain on the registry for years before a match is found. More information is available here.

Wolverines for Life and the Michigan Medicine Bone Marrow Transplant department are hosting a registration drive from 10:30 a.m. to 5 p.m. Tuesday, Feb. 18 at the Pierpoint Commons lobby, 2101 Bonisteel Blvd., Ann Arbor.

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Mixed ethnic backgrounds make it that much harder to find a bone marrow transplant - MLive.com

Bone Marrow Stem Cells Comments Off on Mixed ethnic backgrounds make it that much harder to find a bone marrow transplant – MLive.com | February 17th, 2020

The youngest children diagnosed with cancer are just one day old. The most frequent type of cancer diagnosed is leukemia, ETV's current affairs show "Aktuaalne kaamera" reported.

"Leukemia is a disease of hematopoietic stem cells; hematopoietic stem cells are the cells which derive from all the cells that circulate in our blood - red blood cells, thrombocytes, white blood cells. Every one of them has a function and if they're not present, they're not able to fill the function," Ain Kaare, Head of the Department of Hematology and Bone Marrow Transplantation in University of Tartu said.

Kaare said that what helps children to cope with cancers is hope and optimism, and they are able to enjoy it, when they are feeling good.

These courageous children make an effort over years in order to get better. Kaare noted that there are children in the clinic who have undergone hematopoietic stem cell transplantation three times in a row, which often adults don't survive.

"In my work office, I have a picture on a wall by a boy who was five at the time, where he has written that "doctor Kaare is my friend". In the picture, there was I with a bag in my hand and in that bag there were supposed to be the hematopoietic stem cells that we transplanted to him," Kaare said.

Kaili Lellep, president of the Estonian Association of Parents of Children with Cancer (Vhihaigete laste vanemate liit) said that a child with a cancer needs a supportive parent around all the time to comfort them and explain everything if necessary, and to get their thoughts away from their illness. Children with cancer often stay in hospital for months.

On Saturday, golden ribbons and the golden light on Tallinn's TV tower and at Tallinn's Children Hospital (Tallinna lastehaigla) sent a message to hospital wards and homes alike, that seriously ill children are not alone and are being thought of, hoping that tomorrow they will be better. The golden ribbon is well-known all over the world.

"This ribbon symbolises support for children with cancer and support for the suffering of children with cancer," Lellep said.

--

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50 children diagnosed with cancer in Estonia every year - ERR News

Bone Marrow Stem Cells Comments Off on 50 children diagnosed with cancer in Estonia every year – ERR News | February 17th, 2020

Global Cord stem cell banking market is estimated to reach USD 13.8 billion by 2026 registering a healthy CAGR of 22.4%. The increasing number of parents storing their childs cord blood, acceptance of stem cell therapeutics, high applicability of stem cells are key driver to the market.

Request for FREE sample copy or PDF Herehttps://www.databridgemarketresearch.com/request-a-sample?dbmr=global-cord-stem-cell-banking-market&raksh

Few of the major market competitors currently working in the globalcord stem cell banking marketareCBR Systems, Inc., Cordlife, Cells4Life Group LLP, Cryo-Cell International, Inc., Cryo-Save AG, Lifecell, StemCyte India Therapeutics Pvt. Ltd, Viacord, SMART CELLS PLUS., Cryoviva India, Global Cord Blood Corporation, National Cord Blood Program, Vita 34, ReeLabs Pvt. Ltd., Regrow Biosciences Pvt. Ltd. , ACROBiosystems., Americord Registry LLC., New York Blood Center, Maze Cord Blood, GoodCell., AABB, Stem Cell Cryobank, New England Cryogenic Center, Inc. among others

The data and information included in this Global Cord Stem Cell banking business report helps businesses take sound decisions and plan about the advertising and sales promotion strategy more successfully. This Cord Stem Cell banking market research report is generated by taking into account a range of objectives of market research that are vital for the clients success. This report also includes strategic profiling of key players in the market, systematic analysis of their core competencies, and draws a competitive landscape for the Healthcare industry. The Global Cord Stem Cell banking business report includes market shares for global, Europe, North America, Asia Pacific and South America.

Market Definition: Global Cord Stem Cell Banking Market

Cord stem cells banking is nothing but the storing of the cord blood cell contained in the umbilical cord and placenta of a newborn child. This cord blood contains the stem cells which can be used in future to treat disease such as leukemia, thalassemia, autoimmune diseases, and inherited metabolic disorders, and few others.

Segmentation: Global Cord Stem Cell Banking Market

Cord Stem Cell banking Market : By Storage Type

Cord Stem Cell banking Market : By Product Type

Cord Stem Cell banking Market : By Service Type

Cord Stem Cell banking Market : By Indication

Cord Stem Cell banking Market : By Source

Cord Stem Cell banking Market : By Geography

Browse Detailed TOC, Tables, Figures, Charts and Companies @https://www.databridgemarketresearch.com/toc?dbmr=global-cord-stem-cell-banking-market&raksh

Key Developments in the Cord Stem Cell banking Market:

Cord Stem Cell banking Market : Drivers

Cord Stem Cell banking Market : Restraint

Competitive Analysis: Global Cord Stem Cell Banking Market

Global cord stem cell banking market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions and others to increase their footprints in this market. The report includes market shares of cord stem cell banking market for Global, Europe, North America, Asia Pacific, South America and Middle East & Africa.

Key benefits of buying the Cord Stem Cell banking Market Report:

This Cord Stem Cell banking Market report will enable both of the sides in market be an established firm or a relative new entrant. It helps the established firms to know about the moves which are being performed by their competitors and also helps the new entrants by educating them about the market situations and the industry trends. This Cord Stem Cell banking Market report is quite fruitful in helping to understand the market definition and all the aspects of the market including the CAGR value and key profiles.

Want Full Report? Enquire Here @https://www.databridgemarketresearch.com/inquire-before-buying?dbmr=global-cord-stem-cell-banking-market&raksh

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Cord Stem Cell banking Market 2020-2026 Booming With Healthy CAGR || Key Players Cryo-Save AG, Lifecell, StemCyte India Therapeutics Pvt. Ltd,...

Bone Marrow Stem Cells Comments Off on Cord Stem Cell banking Market 2020-2026 Booming With Healthy CAGR || Key Players Cryo-Save AG, Lifecell, StemCyte India Therapeutics Pvt. Ltd,… | February 17th, 2020

Dublin, Feb. 17, 2020 (GLOBE NEWSWIRE) -- The "Stem Cell Therapy Contract Manufacturing Market, 2019-2030" report has been added to ResearchAndMarkets.com's offering.

This report features an extensive study on contract service providers engaged in the development and manufacturing of stem cell therapies. The study features in-depth analyses, highlighting the capabilities of various stem cell therapy CMOs

Advances in the fields of cell biology and regenerative medicine have led to the development of a variety of stem cell-based therapies for many cardiovascular, oncological, metabolic and musculoskeletal disorders. Driven by the revenues generated from stem cell therapies, the regenerative medicine market is anticipated to generate revenues worth USD 100 billion by 2030.

With a promising pipeline of over 200 stem cell therapy candidates, it has become essential for developers to scale up the production of such therapeutic interventions. Given that stem cell therapy manufacturing requires highly regulated, state-of-the-art technologies, it is difficult for stakeholders to establish in-house expertise for large-scale manufacturing of stem cell therapies.

As a result, stem cell therapy developers have begun outsourcing their manufacturing operations to contract manufacturing organizations (CMOs). Specifically, small and mid-sized players in this sector tend to outsource a substantial proportion of clinical and commercial-scale manufacturing processes to contract service providers. In addition, even big pharma players, with established in-house capabilities, are gradually entering into long-term business relationships with CMOs in order to optimize resource utilization and manage costs.

According to a recent Nice Insight CDMO survey, about 55% of 700 respondents claimed to have collaborated with a contract service provider for clinical and commercial-scale product development requirements. Considering the prevalent trends, we believe that the stem cell therapy manufacturing market is poised to grow at a steady pace, driven by a robust pipeline of therapy candidates and technological advances aimed at mitigating challenges posed by conventional methods of production. Amidst tough competition, the availability of cutting-edge tools and technologies has emerged as a differentiating factor and is likely to grant a competitive advantage to certain CMOs over other players in the industry.

One of the key objectives of the report was to estimate the future size of the market. Based on parameters, such as increase in number of clinical studies, target patient population, anticipated adoption of stem cell therapies and expected variation in manufacturing costs, we have provided an informed estimate of the likely evolution of the market in the mid to long term, for the period 2019-2030.

Amongst other elements, the report includes:

In order to provide a detailed future outlook, our projections have been segmented on the basis of:

Key Topics Covered

1. Preface

2. Executive Summary

3. Introduction

4. Market Overview

5. Regulatory Landscape

6. Stem Cell Therapy Contract Manufacturers in North America

7. Stem Cell Therapy Contract Manufacturers in Europe and Asia-Pacific

8. Partnerships and Collaboration

9. Contract Manufacturing Opportunity Assessment

10. Capacity Analysis

11. Demand Analysis

12. Market Forecast

13. Key Performance Indicators

14. Concluding Remark

15. Executive Insights

16. Appendix 1: Tabulated Data

17. Appendix 2: List of Companies and Organizations

For more information about this report visit https://www.researchandmarkets.com/r/rktm8d

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Stem Cell Therapy Contract Manufacturing Industry, 2019-2030 - Availability of Cutting-Edge Tools & Technologies has Emerged as a Differentiating...

IPS Cell Therapy Comments Off on Stem Cell Therapy Contract Manufacturing Industry, 2019-2030 – Availability of Cutting-Edge Tools & Technologies has Emerged as a Differentiating… | February 17th, 2020

The comprehensive report published by Persistence Market Research offers an in-depth intelligence related to the various factors that are likely to impact the demand, revenue generation, and sales of the Stem Cell-Derived Cells Market. In addition, the report singles out the different parameters that are expected to influence the overall dynamics of the Stem Cell-Derived Cells Market during the forecast period 2019 2029.

As per the findings of the presented study, the Stem Cell-Derived Cells Market is poised to surpass the value of ~US$ XX by the end of 2029 growing at a CAGR of ~XX% over the assessment period. The report includes a thorough analysis of the upstream raw materials, supply-demand ratio of the Stem Cell-Derived Cells in different regions, import-export trends and more to provide readers a fair understanding of the global market scenario.

ThisPress Release will help you to understand the Volume, growth with Impacting Trends. Click HERE To get SAMPLE PDF (Including Full TOC, Table & Figures) athttps://www.persistencemarketresearch.co/samples/28780

The report segregates the Stem Cell-Derived Cells Market into different segments to provide a detailed understanding of the various aspects of the market. The competitive analysis of the Stem Cell-Derived Cells Market includes valuable insights based on which, market players can formulate impactful growth strategies to enhance their presence in the Stem Cell-Derived Cells Market.

Key findings of the report:

The report aims to eliminate the following doubts related to the Stem Cell-Derived Cells Market:

Get Access To TOC Covering 200+ Topics athttps://www.persistencemarketresearch.co/toc/28780

key players in stem cell-derived cells market are focused on generating high-end quality cardiomyocytes as well as hepatocytes that enables end use facilities to easily obtain ready-made iPSC-derived cells. As the stem cell-derived cells market registers a robust growth due to rapid adoption in stem cellderived cells therapy products, there is a relative need for regulatory guidelines that need to be maintained to assist designing of scientifically comprehensive preclinical studies. The stem cell-derived cells obtained from human induced pluripotent stem cells (iPS) are initially dissociated into a single-cell suspension and later frozen in vials. The commercially available stem cell-derived cell kits contain a vial of stem cell-derived cells, a bottle of thawing base and culture base.

The increasing approval for new stem cell-derived cells by the FDA across the globe is projected to propel stem cell-derived cells market revenue growth over the forecast years. With low entry barriers, a rise in number of companies has been registered that specializes in offering high end quality human tissue for research purpose to obtain human induced pluripotent stem cells (iPS) derived cells. The increase in product commercialization activities for stem cell-derived cells by leading manufacturers such as Takara Bio Inc. With the increasing rise in development of stem cell based therapies, the number of stem cell-derived cells under development or due for FDA approval is anticipated to increase, thereby estimating to be the most prominent factor driving the growth of stem cell-derived cells market. However, high costs associated with the development of stem cell-derived cells using complete culture systems is restraining the revenue growth in stem cell-derived cells market.

The global Stem cell-derived cells market is segmented on basis of product type, material type, application type, end user and geographic region:

Segmentation by Product Type

Segmentation by End User

The stem cell-derived cells market is categorized based on product type and end user. Based on product type, the stem cell-derived cells are classified into two major types stem cell-derived cell kits and accessories. Among these stem cell-derived cell kits, stem cell-derived hepatocytes kits are the most preferred stem cell-derived cells product type. On the basis of product type, stem cell-derived cardiomyocytes kits segment is projected to expand its growth at a significant CAGR over the forecast years on the account of more demand from the end use segments. However, the stem cell-derived definitive endoderm cell kits segment is projected to remain the second most lucrative revenue share segment in stem cell-derived cells market. Biotechnology and pharmaceutical companies followed by research and academic institutions is expected to register substantial revenue growth rate during the forecast period.

North America and Europe cumulatively are projected to remain most lucrative regions and register significant market revenue share in global stem cell-derived cells market due to the increased patient pool in the regions with increasing adoption for stem cell based therapies. The launch of new stem cell-derived cells kits and accessories on FDA approval for the U.S. market allows North America to capture significant revenue share in stem cell-derived cells market. Asian countries due to strong funding in research and development are entirely focused on production of stem cell-derived cells thereby aiding South Asian and East Asian countries to grow at a robust CAGR over the forecast period.

Some of the major key manufacturers involved in global stem cell-derived cells market are Takara Bio Inc., Viacyte, Inc. and others.

The report covers exhaustive analysis on:

Regional analysis includes

Report Highlights:

In order to get a strategic overview of the market,Access Research Methodology Prepared By Experts athttps://www.persistencemarketresearch.co/methodology/28780

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Tags: Stem CStem Cell-Derived Cells MarketStem Cell-Derived Cells Market DynamicsStem Cell-Derived Cells Market GrowthStem Cell-Derived Cells Market KeyplayersStem Cell-Derived Cells Market Trends

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Surge in the Adoption of Stem Cell-Derived Cells to Fuel the Growth of the Stem Cell-Derived Cells Market Through the Assessment Period 2019 2029 -...

IPS Cell Therapy Comments Off on Surge in the Adoption of Stem Cell-Derived Cells to Fuel the Growth of the Stem Cell-Derived Cells Market Through the Assessment Period 2019 2029 -… | February 17th, 2020

Medical treatment involving stem cells is an ever-growing, billion-dollar industry, so chances are you have heard about it in the news. Here in the U.S. and around the world, stem cells are being used in various therapies to treat a wide variety of health problems and diseases, including dementia, autism, multiple sclerosis, cerebral palsy, osteoarthritis, cancer, heart disease, Parkinsons disease, and spinal cord injury. Treatments for such health issues may sound promising, but the risk is many of those being sold and advertised arent yet proven to be safe and effective. This is why its so important to educate yourself before jumping into any kind of stem cell treatment.

What are stem cells?

To gain a better understanding of this new age of medical research, one must first understand what stem cells are and how they work. Stem cells are special human cells that can develop into many different types of cells. They can divide and produce more of the same type of stem cells, or they can turn into different functioning cells. There are no other types of cells in the body that have this natural ability to generate new cell types.

Where do stem cells come from?

So where do stem cells that are used for research and medical treatments come from? The three main types of stem cells are embryonic (or pluripotent) stem cells, adult stem cells, and induced pluripotent stem cells.

Embryonic stem cells come from unused, in vitro fertilized embryos that are three to five days old. The embryos are only donated for research purposes with the informed consent of the donors. Embryonic stem cells are pluripotent, which means they can turn into any cell type in the body.

Adult stem cells are found in small numbers in developed tissues in different parts of the body, such as bone marrow, skin, and the brain. They are specific to a certain kind of tissue in the body and are limited to maintaining and repairing the tissue in which they are found. For example, liver stem cells can only make new liver tissue; they arent able to make new muscle tissue.

Induced pluripotent stem cells are another form of adult stem cells. These are stem cells that have been manipulated in a laboratory and reprogrammed to work like embryotic (or pluripotent) stem cells. While these altered adult stem cells dont appear to be clinically different from embryonic stem cells, research is still being conducted to determine if the effects they have on humans differ from actual embryonic stem cells.

Stem cells can also be found in amniotic fluid and umbilical cord blood. These stem cells have the ability to change into specialized cells like embryonic stem cells. While more research is being conducted to determine the potential of these types of stem cells, researchers already actively use these through amniocentesis procedures. In this procedure, the stem cells drawn from amniotic fluid samples of pregnant women can be screened for developmental abnormalities in a fetus.

How stem cells function

The main difference between embryonic and adult stem cells is how they function. Embryonic stem cells are more versatile. Since they can divide into more stem cells or become any type of cell in the body, they can be used to regenerate or repair a variety of diseased tissue and organs. Adult stem cells only generate the types of cells from where they are taken from in the body.

The future of stem cell research

The ability for stem cells to regenerate under the right conditions in the body or in a laboratory is why researchers and doctors have become so interested in studying them. Stem cell research is helping scientists and doctors to better understand how certain diseases occur, how to possibly generate healthy cells to replace diseased cells, and offer ways to test new drugs.

Clearly, stem cell research is showing great potential for understanding and treating a range of diseases and other health issues, but there is still a lot to learn. While there are some diseases that are showing success using stem cell treatments, many others are yet to be proven in clinical trials and should be considered highly experimental.

In our next article, various stem cell treatments, FDA regulations, and other stem cell hot topics will be explored. It will also focus on what to look for when considering stem cell therapies so people arent misled or misinformed about the benefits and risks.

For more information regarding the basics of stem cells visit these sites:

https://stemcells.nih.gov/info/basics/1.htm

https://www.mayoclinic.org/tests-procedures/bone-marrow-transplant/in-depth/stem-cells/art-20048117

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The 411 on Stem Cells: What They Are and Why It's Important to Be Educated - Legal Examiner

Spinal Cord Stem Cells Comments Off on The 411 on Stem Cells: What They Are and Why It’s Important to Be Educated – Legal Examiner | February 17th, 2020

There are two different types of stem cell transplants:

To understand these treatments, it helps to know about bone marrow and stem cells.

Bone marrow is part of our immune system which protects us from infection and disease. It is found inside our bones, mainly in the hip bone and the breast bone. The bone marrow is where stem cells are made.

Stem cells are blood cells at the earliest stage of development. All our blood cells develop from stem cells in the bone marrow. Stem cells stay inside the bone marrow and when they are fully developed they go into the bloodstream.

Blood cells do not live long. The bone marrow normally makes millions of new blood cells every day to replace blood cells as they are needed.

There are three main types of blood cells:

There are two main types of white blood cell. These are called neutrophils and lymphocytes. Neutrophils are the most common. You will hear your doctor or nurse talk about your neutrophil count during your treatment.

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What are stem cells and bone marrow? - Macmillan Cancer ...

Bone Marrow Stem Cells Comments Off on What are stem cells and bone marrow? – Macmillan Cancer … | February 16th, 2020

Early data trends from first patient dosed in the AVR-RD-04 investigational gene therapy program for cystinosis show improvements across multiple measures

Data from the Phase 1 and Phase 2 trials of AVR-RD-01 support potential long-term engraftment and durable, endogenous production of functional enzyme in patients with Fabry disease

First Phase 2 Fabry patient treated using plato gene therapy platform shows plasma enzyme activity at one month 4.0 times higher than mean activity of other Phase 2 patients treated using academic platform at same timepoint

Analyst and investor event will be webcast today, Feb. 10, 2020, at 7:00 p.m. ET, in conjunction with WORLDSymposiumTM

AVROBIO, Inc. (NASDAQ: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced new initial data from the first patient dosed in the investigational gene therapy program for cystinosis, showing improvements in early measures at three months compared to baseline. The company also unveiled new clinical data showcasing a sustained biomarker response in patients for up to 32 months after receiving the companys investigational gene therapy for Fabry disease across metrics including vector copy number (VCN), substrate levels and enzyme activity. Additionally, the company reported on the clinical debut of its platoTM gene therapy platform. These data showed improved enzyme activity, transduction efficiency and VCN in drug product manufactured using plato compared with drug product produced using the academic platform, as well as higher in vivo enzyme activity at one month in the first patient treated with plato, as compared to other patients treated using the academic platform. All these data will be presented today, during the 16th Annual WORLDSymposiumTM in Orlando, Fla.

"We have now dosed 10 patients across three trials for two lysosomal disorders and were delighted with the data were seeing. We have followed six patients in our Fabry trial for more than a year and one for nearly three years, and they are consistently producing the functional enzyme that was missing as a consequence of their genetic disease, suggesting a potentially durable effect from a single dose," said Geoff MacKay, AVROBIOs president and CEO. "Furthermore, we believe that early data from the first clinical application of plato support our decision to invest heavily from AVROBIO's earliest days in this state-of-the-art gene therapy platform. We believe these data collectively indicate that were making exciting progress toward our goal of freeing patients and families from the life-limiting symptoms and relentless progression of lysosomal disorders."

Story continues

Three-month data from first patient in investigational AVR-RD-04 trial in cystinosisAVROBIO reported initial data from the first patient dosed in the investigator-sponsored Phase 1/2 trial of the companys AVR-RD-04 investigational gene therapy for cystinosis, a progressive disease marked by the accumulation of cystine crystals in cellular organelles known as lysosomes. Patients with cystinosis accumulate the amino acid cystine, which can lead to crystal formation in the lysosomes of cells, causing debilitating symptoms including corneal damage, difficulty breathing and kidney failure, often leading to a shortened lifespan. The current standard of care for cystinosis, a burdensome treatment regimen that can amount to dozens of pills a day, may not prevent overall progression of the disease.

As of the safety data cut-off date of Jan. 27, 2020, which was approximately three months following administration of the investigational gene therapy to the first patient in the AVR-RD-04 program, there have been no reports of safety events attributed to the investigational drug product. In addition, no serious adverse events (SAEs) have been reported as of the safety data cut-off date. Adverse events did not suggest any unexpected safety signals or trends.

Three months following administration of AVR-RD-04, the first patient had a VCN of 2.0. VCN measures the average number of copies of the lentiviral-vector inserted transgene integrated into the genome of a cell and can be used to help assess the durability of a gene therapy. Initial data on another biomarker show that the patients average granulocyte cystine level -- one of the trials primary endpoints -- decreased from 7.8 nmol half cystine/mg protein two weeks after cysteamine discontinuation, to 1.5 at three months post-gene therapy.

The ongoing open-label, single-arm Phase 1/2 clinical trial evaluating the safety and efficacy of AVR-RD-04 is sponsored by AVROBIOs academic collaborators at the University of California San Diego (UCSD), led by Stephanie Cherqui, Ph.D. The trial is actively enrolling up to six participants at UCSD.

Interim data continue to support potential first line use of AVR-RD-01 in Fabry diseaseFour patients have been dosed in the Phase 2 trial (FAB-201), and five patients in the Phase 1 investigator-led trial of AVR-RD-01 in Fabry disease.

VCN data continue to be stable at 32 months following AVR-RD-01 treatment for the first patient in the Phase 1 trial, suggesting successful engraftment, which is critical to the long-term success of investigational ex vivo lentiviral gene therapies. The VCN data trend was generally consistent across the seven other Phase 1 and Phase 2 trial participants out six to 24 months.

The first three AVR-RD-01 Phase 2 patients entered the study with minimal endogenous enzyme activity. At nine, 12 and 18 months after dosing, data from these three patients indicate sustained increased leukocyte and plasma enzyme activity, suggesting that they are now producing an endogenous supply of functional alpha-galactosidase (AGA) enzyme. This enzyme is essential for breaking down globotriaosylceramide (Gb3) in cells; without it, a toxic metabolite, lyso-Gb3, may accumulate, potentially causing cardiac and kidney damage and other symptoms.

For two Phase 2 patients, data indicate that their decreased plasma lyso-Gb3 levels, a key biomarker for monitoring Fabry disease, have been sustained below their baseline at six and 18 months after dosing. The third Phase 2 patient, a cardiac variant who does not have classic Fabry disease, did not show a decrease in plasma lyso-Gb3 levels, as expected. Cardiac and kidney function measures in the Phase 2 trial remained within normal range for patients who had available 12-month data.

As previously reported, a kidney biopsy taken at 12 months post-treatment for the first patient in the Phase 2 trial showed an 87-percent reduction in Gb3 inclusions per peritubular capillary. The company believes this data point, the primary efficacy endpoint for the Phase 2 trial, supports the potential of AVR-RD-01 to reduce Gb3 levels in tissue, including in the kidney.

In the Phase 1 trial of AVR-RD-01, four of the five patients had their plasma lyso-Gb3 levels reduced between 26 and 47 percent compared to their pre-treatment baseline levels. Data from the other patient in the trial, who remains off enzyme replacement therapy (ERT), through month six showed an initial decline and at month 12 showed a 23-percent increase in lyso-Gb3 levels, as compared to pre-treatment levels. This patients lyso-Gb3 levels remain within the range for the Fabry disease patients on ERT observed in this study.

Overall, three of the five Phase 1 patients have discontinued ERT and all three remain off ERT for six, 14 and 15 months.

As of the safety data cut-off date of Nov. 26, 2019, there have been no safety events attributed to AVR-RD-01 drug product in either the Phase 1 or Phase 2 trial. Through the safety data cut-off date, four SAEs have been reported in the FAB-201 trial and two SAEs in the Phase 1 trial. The fourth Phase 2 patient, who was dosed after the safety data cut-off date, has reported an SAE, which was not attributed to AVR-RD-01 and which subsequently resolved. Across both studies, each of the SAEs has been consistent with the conditioning regimen, stem cell mobilization, underlying disease or pre-existing conditions. Pre-existing low anti-AGA antibody titers have been detected in four patients in the Phase 1 trial and a transient low titer was observed but not detectable in subsequent measures in one patient in the Phase 2 trial.

The Phase 1 trial is fully enrolled. AVROBIO continues to actively enroll the Phase 2 trial in Australia, Canada and the U.S. The FAB-201 trial is an ongoing open-label, single-arm Phase 2 clinical trial evaluating the efficacy and safety of AVR-RD-01 in eight to 12 treatment-nave patients with Fabry disease.

Successful clinical debut of platoTM gene therapy platformAVROBIO also shared preliminary results from the first two patients to receive busulfan conditioning. Conditioning is an essential step in ex vivo lentiviral gene therapy designed to clear space in the bone marrow for the cells carrying the therapeutic transgene to engraft. The conditioning regimen developed as part of AVROBIOs plato platform includes therapeutic dose monitoring to assess how rapidly the individual patient metabolizes busulfan so physicians can adjust the dose as needed, with a goal of minimizing side effects while maximizing the potential of durable engraftment.

AVROBIO is implementing its precision dosing conditioning regimen across its company-sponsored clinical trials as part of the plato platform. The fourth patient in AVROBIOs Phase 2 Fabry trial received a precision dosing conditioning regimen with busulfan as part of the plato platform, while the first patient in the investigator-led cystinosis trial received busulfan but not as part of the plato platform.

These two patients both had rapid neutrophil and platelet count recovery, with a trajectory that was similar to the patients who enrolled earlier in the Fabry trials and who received a melphalan conditioning regimen. Side effects, which included nausea, mucositis, fever, rash and hair loss, developed eight to 10 days after dosing with busulfan and then resolved quickly.

The company also reported preliminary data from the first drug product produced using the plato gene therapy platform, which was used to dose the fourth patient in the Phase 2 Fabry trial (FAB-201). Early data indicate that enzyme activity and transduction efficiency for the drug product used to dose the fourth patient were 2.2 times higher than the mean of the drug product used to dose the first three patients in FAB-201. VCN for the drug product used to dose the fourth patient was 1.8 times higher than the mean of the drug product for the first three patients dosed in FAB-201. The drug product for the first three patients in FAB-201 was manufactured using a manual process first developed by AVROBIOs academic collaborators. The automated manufacturing embedded in plato leverages optimized processes developed at AVROBIO.

At one month following administration of the plato-produced investigational gene therapy for the fourth patient in the Phase 2 Fabry trial, initial data show the patients plasma enzyme activity level to be 4.0 times higher than the mean activity level of the first three patients in the Phase 2 Fabry trial at the same timepoint.

The investigational drug product used to dose the first patient in the AVR-RD-04 program for cystinosis, which included a four-plasmid vector but not platos automated manufacturing process, also showed increased performance in line with the increased performance recorded for the drug product in the Fabry trial. The investigational drug product and VCN assay are different for each trial.

"We believe these data are an early, but exciting, validation of our decision to invest in technological innovation rather than build expensive bricks-and-mortar manufacturing facilities," said MacKay. "The plato platform gives us control over the production and scaling of our investigational gene therapies through an efficient, automated manufacturing system that is designed to be deployed in standard contracted sites around the world. The four-plasmid vector, conditioning regimen with precision dosing and other elements of plato are designed to optimize the safety, potency and durability of our investigational lentiviral gene therapies."

About AVROBIOs ex vivo approach to gene therapyOur investigational ex vivo gene therapies start with the patients own stem cells. In the manufacturing facility, a lentiviral vector is used to insert a therapeutic gene designed to enable the patient to produce a functional supply of the protein they lack. These cells are then infused back into the patient, where they are expected to engraft in the bone marrow and produce generations of daughter cells, each containing the therapeutic gene. This approach is designed to drive durable production of the functional protein throughout the patients body, including hard-to-reach tissues such as the brain, muscle and bone. It is a distinguishing feature of this type of gene therapy that the corrected cells are expected to cross the blood-brain barrier and thereby potentially address symptoms originating in the central nervous system.

Lentiviral vectors are differentiated from other delivery mechanisms because of their large cargo capacity and their ability to integrate the therapeutic gene directly into the patients chromosomes. This integration is designed to maintain the transgenes presence as the patients cells divide, which may improve the expected durability of the therapy and potentially enable dosing of pediatric patients, whose cells divide rapidly as they grow. Because the transgene is integrated ex vivo into patients stem cells, patients are not excluded from receiving the investigational therapy due to pre-existing antibodies to the viral vector.

Analyst and investor event and webcast informationAVROBIO will host an analyst and investor event today, Monday, Feb. 10, 2020, in conjunction with the WORLDSymposiumTM, an annual scientific meeting dedicated to lysosomal disorders, in Orlando, FL. The presentation at the event will be webcast beginning at 7:00 p.m. ET. The webcast and accompanying slides will be available under "Events and Presentations" in the Investors & Media section of the companys website at http://www.avrobio.com. An archived webcast recording of the event will be available on the website for approximately 30 days.

About AVROBIOOur mission is to free people from a lifetime of genetic disease with a single dose of gene therapy. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a program in Pompe disease. AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking StatementsThis press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as "aims," "anticipates," "believes," "could," "designed to," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, and anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled "Risk Factors" in AVROBIOs most recent Quarterly Report on Form 10-Q, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200210005767/en/

Contacts

Investor Contact: Christopher F. BrinzeyWestwicke, an ICR Company339-970-2843chris.brinzey@westwicke.com

Media Contact: Tom DonovanTen Bridge Communications857-559-3397tom@tenbridgecommunications.com

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AVROBIO Presents Positive Initial Data for its Investigational Cystinosis Program and Plato TM Platform, as well as Positive Data Out to 32 Months for...

Cardiac Stem Cells Comments Off on AVROBIO Presents Positive Initial Data for its Investigational Cystinosis Program and Plato TM Platform, as well as Positive Data Out to 32 Months for… | February 16th, 2020

Marie Antoinette, the last queen of France, is best remembered for her reckless extravagance and her tragic death. French revolutionaries executed her with a guillotine in 1793 for treason. Popular belief is that her hair turned grey the night before her death.

There are other stories and anecdotes like this which suggest that stressful experiences are correlated with the phenomenon of hair greying. Now, for the first time, Harvard University scientists have found the real mechanism behind it.

Published in the journal Nature, the study was initiated with the aim of analysing the effect of stress on various tissues. Hair and skin are the tissues that are visible from outside. So, the researchers started with effects of stress on hair colour.

Their initial hypothesis was that stress initiates an immune attack on pigment-producing cells in the hair follicles. However, when they tested it on mouse, they found those lacking immune cells (nude mouse) also exhibited hair greying. Then, their attention turned to a hormone called cortisol. It is responsible for regulating a wide range of processes through the body, including metabolism and the immune response. In response to stress, extra cortisol is released to help the body to respond appropriately. Surprisingly, when the researchers removed the adrenal gland from the miceto prevent production of cortisol-like hormone aldosteroneand triggered stress, their hair still turned grey.

Finally, the researchers tested the sympathetic nerves that branch out to each hair follicle. The sympathetic nerve system is responsible for the bodys fight-or-flight response. They found that in a stressful condition, the sympathetic nerves release a chemical called norepinephrine, which is taken up by certain stem cells in the hair follicle. Stem cells act as a reservoir for pigment-producing cells. During hair regeneration, some of these stem cells are converted into pigment-producing cells to give colour to new hair strands.

When these stem cells take norepinephrine, they are activated excessively and all of them get converted into pigment-producing cells. This would prematurely deplete the reservoir for pigment-producing cells. Once all of them are consumed, pigment regeneration would stop, resulting in permanent damage. The fight-or-flight response has been traditionally viewed as beneficial. But now it is proved that it has its own detrimental effects, too.

The study established how neurons interact at the cellular and molecular level to link stress with hair greying. The findings are expected to put light on the broader effects of stress on various body parts. The scientists will initiate new studies that seek to modify or block the damaging effects of stress.

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The ghost behind grey - THE WEEK

Skin Stem Cells Comments Off on The ghost behind grey – THE WEEK | February 16th, 2020

WHEN student Hayden Ryals saw a call for blood stem cell donors, she didn't think twice about signing up.

But she was shocked when she received a call just a year later, asking for her help to save a one-year-old girl's life.

6

Hayden, now 27, from Alabama, had the operation and instantly felt bonded to little Skye.

And two years later, the pair met for the first time, when Skye was invited to be a flower girl at Hayden's wedding.

Speaking exclusively to Fabulous Digital, Hayden tells her story...

Since I was about 16, I have always given blood. It seemed like such a small thing to do to help people. Donating only takes 10 minutes.

6

Then I went to University in Auburn, Alabama. I was walking to a lecture one day and saw a stall from Be the Match. I wandered over to see what they were about.

The lady at the stall told me they were looking for bone marrow donors. I just needed a swab in my cheek, so they would have my tissue, and then I would be put on the register. You only get called if you're a match.

I happily said yes, it only took two minutes, and didnt think much more about it.

Almost a year to the day later, in April 2016, I got a phone call. It was from Be the Match.

When I spoke to her mum Talia, it was emotional. 'Youve saved my daughters life,' she said through tears

"You have been matched with a one-year-old little girl," I was told.

At first I thought it was a mistake, surely I wouldnt be able to do something so big? But it was true, she needed me to save her life.

I had been feeling so down in life, wondering what I was supposed to be doing, I felt a surge of joy at being able to help someone.

6

I found out the little girl, Skye Savren-McCormick, wasdiagnosed withjuvenile myelomonocytic leukemia, a rare and aggressive form of blood cancer. She lived in California, miles away from me.

I immediately knew I wanted to help her.

I sent her parents a letter, through a co-ordinator, telling them I wouldnt back out.

They already felt like family to me. It was strange how connected I felt to a little girl I had never met.

6

In July 2016, we did the transfer. I went to a hospital three hours away in Birmingham Alabama and was put under general anaesthetic for an hour.

They put a hollow needle into my hip bone to extract my bone marrow. It really wasnt bad, and I didnt feel much pain afterwards.

I was told Skye responded well, but had to wait a year to contact Skye's parents.

When I eventually spoke to her mum Talia, it was emotional. "Youve saved my daughters life," she said through tears.

6

Around this time, I started dating my childhood friend Adrian Ryals, now 34. He was in the US Air Force, and was posted to Korea for a year in 2017.

Just before he left, Adrian popped the question. Delighted, we set a wedding date for June 2018.

In March 2018, I sent Skye a birthday present and asked if she would consider being my flower girl.

Becoming a blood stem cell donor

Every 20 minutes, someone is diagnosed with blood cancer in the UK.

For many, blood stem cell donation is their only chance of survival.

It's difficult to find a match, because of the millions of different stem cell combinations, so the DKMS are constantly on the look out for donors.

To become a blood stem cell donor, you just need to request a kit and do a five-minute cheek swab at home.

You can check your eligibility, and request a kit, here.

The odds are you may never be called upon.

Most blood stem cells are collected through a needle in the arm, just like giving blood.

Bone marrow collection involves stem cells being collected from the back of the hip under general anaesthetic. It takes one to two hours and most donors return to normal activities within a week. This method is only used in 10% of cases.

I knew it was a big ask, she lived a six-hour flight away, but she was so special to me.

If there was a chance she could come, I wanted her at my wedding.

Skye still lived in hospital at the time and had never flown. But in May 2018, the doctor gave her the green light to be able to come to the wedding.

6

Talia, Skye and her dad Todd flew down the Thursday before the wedding.

As we arrived at the church where we were having the rehearsal, they were already there waiting.

I was breathless and speechless to finally meet this little human, and know I was fortunate enough to have been able to help her.

I just ran up to her, got on my knees and gave her a big hug. She wasnt shy with me, as we had spoken on the phone a few times before.

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Skye didnt really understand what I had done for her, but she knew I was somebody special.

I was so honoured to have Skye be my flower girl, it made the wedding even more beautiful. I feel so lucky to have her in my life.

Earlier this week, we reported on a mother-of-the-bride who demanded her ginger bridesmaid dyed her hair for the wedding - because it "clashed" with her hair.

Read the rest here:
Bride saves babys life by donating her bone marrow then meets her for the first time as a flower girl at he - The Sun

Bone Marrow Stem Cells Comments Off on Bride saves babys life by donating her bone marrow then meets her for the first time as a flower girl at he – The Sun | February 15th, 2020

DALLAS It is more blessed to give than to receive. That's what a Bible verse says. And watching the smiles of a now healthy 8-year-old boy, that's what a recent bone marrow donor will gladly tell you too.

"I felt like the earth underneath my legs was pulled out and like I didn't know what to do," said Anitha Nagilla of Frisco of the 2017 chronic myeloid leukemia diagnosis for her then 6-year-old son Akshaj.

After chemotherapy and other treatments, he would need a bone marrow transplant to regenerate his immune system. But no one in his family, including his older sister, was a close enough match. Also, the likelihood of someone with Asian Indian heritage having a matched, available donor is only 41%.

The Nagilla family moved to north Texas from southern India 13 years ago.

WFAA

Every three minutes, someone is diagnosed with a blood cancer, like leukemia or lymphoma. For many patients, finding a bone marrow donor who is a match is their only chance for a cure. Finding a match can happen anywhere in the world at any time.

In nearby Colleyville, another immigrant, unknowingly, was about to help save the Akshaj's life.

WFAA

"Typical immigration story. Parents moved for a better life for their children and for themselves," said Dr. Prasanthi Ganesa, an oncologist who works at the Center for Cancer and Blood disorders in Fort Worth.

Dr. Ganesa moved to the U.S. as a 10-year-old child, also from southern India. She now works with adult cancer patients.

But as a medical student at Texas A&M, she and a group of friends decided to join the nationalBone Marrow Registry. The donation of blood, stem cells, and bone marrow can help people recover from a variety of cancer-related illnesses.

"At that time it was pretty simple," Dr. Ganesa said of joining the registry approximately 17 years ago. "You fill out some paperwork. It's a (saliva) swab and they have my information. So I've been on it, really not thinking about it, until I got the call. It was a surprise. It was a delightful surprise."

The surprise was that she was a match for a young boy, the same age as her own youngest child. After an outpatient procedure where marrow was extracted from a location on each hip, she went home, then back to work, and waited to hear what happened to whoever it was that received her donation.

Dr. Prasanthi Ganesa

Friday morning at Children's Medical Center in Dallas, she met him face to face.

In a meeting room all decorated for Valentine's Day, they celebrated what is also known as "National Donor Day."

"Hi buddy, how are you?" Dr. Ganesa said as she reached out to hug 8-year-old Akshaj Nagilla. "Oh my goodness," she said as they embraced.

"Definitely is on the list of one of my happiest proudest moments in my life," Dr. Ganesa said. "And Akshaj I have you to thank for that. You know what this means right? We're gonna have to be friends forever," she said as the audience of doctors, nurses, and family and friends laughed and applauded.

Being on the marrow registry is easy. All it takes is a swab of saliva. And minority donors are needed the most. Dr. Ganesa was one of two marrow donors for Akshaj. She supplied the first donation.

Akshaj initially recovered but relapsed in the fall of 2018, when he received a second transplant from a different donor, also of Asian Indian heritage. But his family credits the first donation with starting his long road to recovery.

"She being close here in Dallas is undoubtedly remarkable, in that it happened to our family," Anitha Nagilla said.

"She saved my son's life directly. But she saved some other lives as well in the family," Nagilla said of the impact on her entire extended family. "She is a lifesaver for others as well."

Friday morning at their first meeting, the Nagilla family presented Dr. Ganesa with a gold bracelet and a card with a personal message from Akshaj inside. "I'm doing good and wish you well always", he wrote.

"How wonderful is this," Dr. Ganesa said as they embraced again.

The usually shy 8-year-old also mustered up the courage to climb up to a podium and address the entire crowd, but mostly his message was for Dr. Ganesa.

"I am very thankful because I am still alive," he said.

"I just encourage everybody of Indian origin, of southeast Asian origin, any minority group to get yourself on the registry because it could save a life," Dr. Ganesa said.

"It feels really good to be able to say I gave a part of myself and I saved this person's life. I think it's the ultimate meaning of being a human being. We are here to love all serve all. And what an opportunity that I have had. So I am so grateful that I've had that opportunity," Dr. Ganesa said.

She says she feels like she received more than she gave. As she, her own two sons and the Nagilla family gathered for a group hug, her own extended family maybe just got a lot bigger too.

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8-year-old bone marrow recipient and donor celebrate in emotional reunion - WFAA.com

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In August of 2011, Carl June and his team published a landmark paper showing their CART treatment had cleared a patient of cancer. A year-to-the-month later, Jennifer Doudna made an even bigger splash when she published the first major CRISPR paper, setting off a decade of intense research and sometimes even more intense public debate over the ethics of what the gene-editing tool could do.

Last week, June, whose CART work was eventually developed by Novartis into Kymriah, published in Sciencethe first US paper showing how the two could be brought together. It was not only one of the first time scientists have combined the groundbreaking tools, but the first peer-reviewed American paper showing how CRISPR could be used in patients.

June used CRISPR to edit the cells of three patients with advanced blood cancer, deleting the traditional T cell receptor and then erasing the PD1 gene, a move designed to unleash the immune cells. The therapy didnt cure the patients, but the cells remained in the body for a median of 9 months, a major hurdle for the therapy.

Endpoints caught up with June about the long road both he and the field took to get here, if the treatment will ever scale up, and where CRISPR and other advancements can lead it.

The interview has been condensed and edited.

Youve spoken in the past about howyou started working in this field in the mid-90s after your wife passed away from cancer. What were some of those early efforts? How did you start?

Well, I graduated from high school and had a low draft number [for the Vietnam War] and was going to go to study engineering at Stanford, but I was drafted and went into the Naval Academy in 1971, and I did that so I wouldnt have to go to the rice fields.

The war ended in 73, 74, so when I graduated in 1975, I was allowed to go to medical school, and then I had a long term commitment to the Navy because they paid for the Acadamy and Medical school. And I was interested in research and at the time, what the Navy cared about was a small scale nuclear disaster like in a submarine, and like what happened at Chernobyl and Fukushima. So they sent me to the Fred Hutchinson Cancer Center where I got trained in cancer, as a medical oncologist. I was going to open a bone marrow transplant center in Bethesda because the Navy wanted one in the event of a nuclear catastrophe.

And then in 1989, the Berlin Wall came down and there was no more Cold War. I had gone back to the Navy in 86 for the transplant center, which never happened, so then I had to work in the lab full time. But in the Navy, all the research has to be about combat and casualty. They care about HIV, so my first papers were on malaria and infectious disease. And the first CAR-T trials were on HIV in the mid-90s.

In 96, my wife got diagnosed with ovarian cancer and she was in remission for 3-4 years. I moved to the University of Pennsylvania in 1999 and started working on cancer because I wasnt allowed to do that with the Navy. My wife was obviously a lot of motivation to do that. She passed away in 2001. Then I started working with David Porter on adoptive transfer T cells.

I got my first grant to do CAR-T cells on HIV in 2004, and I learned a whole lot. I was lucky to have worked on HIV because we did the first trials using lentiviruses, which is an engineered HIV virus.

I was trained in oncology, and then because of the Navy forced to work on HIV. It was actually a blessing in disguise.

So if you hadnt been drafted, you wouldve become an engineer?

Yes. Thats what I was fully intending. My dad was a chemical engineer, my brother is an engineer. Thats what I thought I was going to do. No one in my family was ever a physician. Its one of those many quirks of fate.

Back then, we didnt have these aptitude tests. It was just haphazard. I applied to three schools Berkeley, Stanford and Caltech and I got into all three. It was just luck, fate.

And it turned out when I went to the Naval Academy, they had added a pre-med thing onto the curriculum the year before, so thats what I did when I started, I did chemistry.

I wouldve [otherwise] been in nuclear submarines. The most interesting thing in the Navy then was the nuclear sub technology.

You talked about doing the first CAR-T trials on HIV patients because thats where the funding was. Was it always in your head that this was eventually going to be something for cancer?

So I got out of the Navy in 99 and moved to Penn. I started in 98 working on treating leukemia, and then once I got to Penn, I continued working one day a week on HIV.

Its kind of a Back-to-the-Future thing because now cancer has paved out a path to show that CART cells can work and put down the manufacturing and its going to be a lot cheaper making it for HIV. I still think thats going to happen.

Jim Riley, who used to be a postdoc in my lab, has some spectacular results in monkeys with HIV models. They have a large NIH and NIAID research program.

So were going to see more and more of that. The CAR technology is going to move outside of cancer, and into autoimmune and chronic infections.

I want to jump over to cytotoxic release syndrome (CRS)because a big part of the CRISPR study was that it didnt provoke this potentially deadly adverse effect. When did you first become aware that CRS was going to be a problem?

I mean we saw it in the very first patient we treated but in all honesty, we missed it. Im an MD, but I dont see the patient and David Porter tookcare of the first three patients and our first pediatric patient,Emily Whitehead.

In our first patients, 2 out of 3, had complete remission and there were fevers and it was CRS but we thought it was just an infection, and we treated with antibiotics for 3 weeks and[eventually] it went away. And sort of miraculously he was in remission and is still in remission, 9 years later.

And then when we treated Emily. She was at a 106-degree fever over three days, and there was no infection.

Ive told this story before. My daughter has rheumatoid arthritis, and I had been president of the Clinical Immunologists Society from 2009 to 2010, and the first good drug for juvenile rheumatoid arthritisthat came out. I was invited to give the Japanese scientist Tadamitsu Kishimoto the presidential award for inventing the drug.

Then in 2012, Emily Whitehead was literally dying from CRS, she had multiple organ failures. And her labs came back and IL-6 levels were 1000x normal. It turns out the drug I was looking at for my daughter, it blocks IL-6 levels. I called the physician and I said, listen theres something actionable here, since its in your formulary to give it to her off-label.

And she gave her the appropriate dose for rheumatoid arthritis. It was miraculous. She woke up very rapidly.

Now its co-labeled. When the FDA approvedKymriah, it was co-labeled. It kind of saved the field.

How were you feeling during this time? Did you have any idea what was happening to her?

No, not until we got the cytokine levels, and then it was really clear. The cytokine levels go up and it exactly coincided. Then we retroactively checked out adults and they had adverse reactions and it easy to see. We hadnt been on the lookout because it wasnt in our mouse models.

And it appeared with those who got cured. Its one of the first on-target toxicities seen in cancer, a toxicity that happens when you get better. All the toxicities from chemotherapy are off-target: like leukopenia or hair loss.

I had a physician who had a fever of 106, I saw him on a fever when he was starting to get CRS. When the nurse came in and it said 106, they thought the thermometer must be broken. On Monday, I saw him, and said how are you feeling and he said fine. And I looked at the thermometer and histemperature was still 102.

People will willingly tolerate on-target toxicity thats very different from chemotherapy if they know it helps get them better. Thats a new principle in cancer therapy.

You had these early CART results almost at the same time that Doudna publishes the first CRISPR papers, then still in bacteria. When did you first start thinking about combining the two?

Yeah, it was published inSciencein 2012 and thats when Emily Whitehead got treated. Its an amazing thing.

Thats something so orthogonal. You think how in the heck can that ever benefit CART cells? but my lab had done the first edited cells in patients, published in 2012. And we used zinc-fingered nucleases, which were the predecessors to CRISPR. It knocked out one gene at a time, but we showed it was safe.

I was already into gene editing because it could make T cells resistant to HIV. So it was pretty obvious that there were candidates in T cells that you can knock out. And almost every lab started working on some with CRISPR, cause it was much easier.

We were the first to get full approval by the FDA, so we worked on it from 2012, had all the preclinical data by 2016, and then it takes a while to develop a lot of new assays for this as we were very cautious to optimize safety and it took longer than we wanted, but in the end, we learned a tremendous amount.

So what did we learn?

First of all our patients had advanced metastatic cancer and had had a lot of chemotherapy. The first patient had had 3 bone marrow transplants.

One thing is feasibility: could you really do all the complex engineering? So we found out we could. feasibility was passed.

Another was the fact that cas9 came out of bacteria, forms of strep and staph. Everyone has pre-existing immunity to Cas9 and we had experience from the first trial with Sangamo[with zinc-finger nucleases] where some patients had a very high fever. In that case, we had used adenoviruses, and it turned out our patients had very high levels of baseline immune response to adenoviruses, so we were worried that would happen with CRISPR, and it did not happen.

It did not have any toxicity. If it had, it would have really set the field back. If there was animmune response to cas9 and CRISPR, there couldve been a real barrier to the field.

And then, the cells survived in the patients. The furthest on, it was 9 months. The cells had a very high level of survival. In the previous trials, the cells survived less than 7 days. In our case, the half-life was 85 days. We dont know the mechanism yet.

And we found very big precision in the molecular scissors, and that was a good thing for the field. You could cut 3 different genes on 3 different chromosomes and have such high fidelity.

It [CRISPR] is living up to the hype. Its going to fix all these diseases.

Whats the potential in CAR-T, specifically?

Well theres many many genes that you can add. There are many genes that knocking outwill make the cells work better. We started with the cell receptor. There are many, I think, academics and biotechs doing this now and it should make the cells more potent and less toxic.

And more broadly, what else are you looking at for the future of CART? The week before your paper, there were the results from MD Anderson on natural killer cells.

Different cell types, natural killer cells, stem cells putting CAR molecules into stem cells, macrophages. One of my graduate students started a company to do CAR macrophages and macrophages actually eat tumor cells, as opposed to T cells that punch holes in them.

There will be different cell types and there will be many more ways to edit cells. The prime editing and base editing. All different new variations.

Youve talked about how people used to think the immuno-oncology, if it ever worked, would nevertheless be a boutique treatment. Despite all the advancements, Novartis and Gilead still have not met the sales they once hoped to grab from their CART treatments. Are you confident CART will ever be widely accessible?

Oh yeah, Novartis sales are going up. They had a hiccup launching.

Back in 96 or 97, when Genentech launched Herceptin, their commercial antibody, they couldnt meet the demand either and then they scaled up and learned how to do better cultures. So right now Novartis is using tech invented in my lab in the 1990s culture tech thats complex and requires a lot of labor, so the most expensive part is human labor. A lot can be made robotic. The scale problem will be much easier.

Thats an engineering problem that will become a thing of the past. The manufacturing problem will get a lot cheaper. Here in the US, we have a huge problem with how drugs are priced. We have a problem with pricing. Thats a political issue.

But in cell therapy, its just kind of the growth things you see in a new industry. Itll get worked out.

This article has been updated to reflect that Jim Riley conducted work on CAR in HIV.

Read more here:
Carl June on CRISPR, CART and how the Vietnam War dropped him into medicine - Endpoints News

Bone Marrow Stem Cells Comments Off on Carl June on CRISPR, CART and how the Vietnam War dropped him into medicine – Endpoints News | February 15th, 2020