Adrenomyeloneuropathy is a rare genetic neuro-degenerative disease. Adrenomyeloneuropathy is the adult onset of adrenoleukodystrophy caused by the mutation in ABCD1 gene occurs usually in young boys. Adrenomyeloneuropathy disease affect the nerve cells in the spine and brain and the adrenal glands. Adrenomyeloneuropathy symptoms includes stiffness, weakness and pain in the legs. Adrenomyeloneuropathy is also known as progressive spastic paraparesis. Damage to the nerves of the legs which causes unsteadiness and fall, also the bladder, bowel and sexual organs are affected by the adrenomyeloneuropathy. Rare diseases affect vast numbers of people, with current data representing 30 million sufferers in the EU alone and 30 million affected in the US. There is no cure to Adrenomyeloneuropathy. However some treatment might stop the progression of Adrenomyeloneuropathy such as stem cell transplants. Blood testing, MRI test, vision screening and Skin biopsy and fibroblast cell culture are done for the diagnosis for the adrenomyeloneuropathy. Continued advances in the treatment of adrenomyeloneuropathy will further propel the adrenomyeloneuropathy treatment market.

Growing cases of rare disease and development of new and advanced treatment for rare disease is expected to boost the adrenomyeloneuropathy treatment market. Growing preference for healthy lifestyle and favorable government regulation spur the Adrenomyeloneuropathy treatment market in the forecast period. Development of new technology and devices for the diagnosis of genetic disorders will propel the adrenomyeloneuropathy treatment market. Rising focus on the research and development of new therapeutic and drug treatment and growing government funding for the orphan drug is expected to drive the adrenomyeloneuropathy treatment market.

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However, stringent regulations for the drug development and high cost of associated with the treatment is expected to hinder the adrenomyeloneuropathy treatment market.

The global adrenomyeloneuropathy treatment market is segmented on basis of disease type, drug type and end user and geography.

Development of novel drugs and undergoing clinical trial for the rare disease is expected to boost adrenomyeloneuropathy treatment market. More than 3,000 drugs are in active development for one of the rare disease. Progress in genomics and biomedical science for the development of rare disease drug is expected to spur the adrenomyeloneuropathy treatment market. Various pharmaceutical companies are focusing on developing drug for the low prevalence disease types and rising funding and collaboration among the key players and government is expected to spur the adrenomyeloneuropathy treatment market.

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The North America market for adrenomyeloneuropathy treatment is expected to retain its dominance, owing to increasing patient pool for rare disease, increasing government funding to accelerate the research and development for rare disease. According to Genetic and Rare Diseases Information Center, more than 25 million Americans are suffering from rare disease in United States.Europe is expected to account for the second largest share in the global adrenomyeloneuropathy treatment market owing to growing clinical trial funding programs for orphan drug development and high prevalence of adrenomyeloneuropathy and high treatment seeking rate. Asia Pacific is expected to show significant growth, owing to increasing diagnosis rate and improvement in healthcare infrastructure. China is expected to show significant growth in the adrenomyeloneuropathy treatment market, due to rising population improving R&D capability, increasing per capita heath spending. Latin America and Middle East & Africa is expected to show growth owing to lack of diagnosis and inadequate healthcare facilities and lack of skilled physicians for Adrenomyeloneuropathy Treatment market.

Examples of some of the key manufacturer present in the global adrenomyeloneuropathy treatment market are Ascend Biopharmaceuticals, Novadip Biosciences, Eureka Therapeutics, Human Longevity, Regeneus, Allogene Therapeutics, BioRestorative Therapies, Immatics Biotechnologies, NewLink Genetics, Cytori Therapeutics, Talaris Therapeutics among others.

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The Adrenomyeloneuropathy Treatment Market to grow on an emphatic note from 2019 to 2029 - PharmiWeb.com

Skin Stem Cells Comments Off on The Adrenomyeloneuropathy Treatment Market to grow on an emphatic note from 2019 to 2029 – PharmiWeb.com | December 21st, 2020

Pune, Maharashtra, India, December 18 2020 (Wiredrelease) Brandessence Market Research and Consulting Pvt ltd :Global Cell Banking Outsourcing Market is valued at USD 7122.6 Million in 2019 and expected to reach USD 18489.6 Million by 2026 with the CAGR of 14.6% over the forecast period.

Rising prevalence of cancer and infectious chronic disorders couples with growing demand for research and development in therapy viral cell banking and viral cell banking safety testing are expected to propel the growth of the Global Cell Banking Outsourcing Market.

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Scope of Global Cell Banking Outsourcing Market Report

The cell banking outsourcing is an ability that stores cells of specific genome for the purpose of future use in a product or medicinal needs to use of gene therapy, stem cell therapy, biopharmaceutical production that target on novel active sites. They are frequently containing expansive amounts of base cell material that can be utilized for various projects. The cell banking outsourcing can be used to generate detailed characterizations of cell lines and can also help mitigate cross-contamination of a cell line. Hence, the cell banking outsourcing is commonly used within fields including stem cell research and pharmaceuticals with cryopreservation being the traditional method of keeping cellular material intact. However, the cell banking is most generally used in stem cell research and therapy. The similar types of cell banking include master cell banks and working cell banks. Although, the master cell banks are expanded to form working cell banks consist of pure cells from are replicated whereas working cell banks consist of thawed cells that are replicated in cell culture.

Additionally, it is a process of replicating and storing cells for the purpose of future use. This storage of these cell samples can be utilized for research purposes and for surgical reconstruction of damaged body structures. However, the bank storage in cell banking encompasses preservation of both master and working cell banking, and their respective safety testing. The cell banking outsourcing expected to witness lucrative growth over the forecast period owing to the presence of increased research in cell line development coupled with the presence of market players providing outsourcing services for cell banking and cell line storage to different hospitals and clinical research organizations.

Global Cell Banking Outsourcing Market report is segmented on the basis of type, application, and by regional & country level. Based on type, global cell banking outsourcing market is classified as the master cell banking, viral cell banking and working cell banking. Based upon application, global cell banking outsourcing is classified into bank storage, working cell bank storage, master cell bank storage cell storage stability testing, bank preparation, bank characterization & testing and others.

The regions covered in this cell banking outsourcing market report are North America, Europe, Asia-Pacific and Rest of the World. On the basis of country level, market of cell banking outsourcing is sub divided into U.S., Mexico, Canada, U.K., France, Germany, Italy, China, Japan, India, South East Asia, GCC, Africa, etc.

Cell Banking Outsourcing Manufacturers:

Some major key players for Global Cell Banking Outsourcing Market are,

BioReliance Covance Global Stem Inc BSL Disservice Clean cells Charles River Laboratories Lonza Toxikon Corporation Cryobanks International India Wuxi Apptec Reliance Life Sciences Life Cell International Pvt. Ltd BioOutsource (Sartorious) CordLife PXTherapeutics SA SGS Life Sciences Texcell Cryo-Cell International Inc. Others

Global Cell Banking Outsourcing Market Dynamics

The rapidly increasing awareness for stem cell banking in the developing countries, and increasing governments initiatives that promote awareness for stem cell isolation and related benefits are some of the major factors driving the market growth during the forecast period. In addition, increasing application of stem cells for developing personalized medicines to minimize the spread of various chronic diseases and also the association of aging with the inability of the body to maintain tissue turnover and hemostasis has helped researchers to focus on this target population for providing relative therapies that would act effectively on the damaged cells. These factors are also supplementing the market growth. According to the World Health Organization (WHO), estimates of cancer incidence and mortality produced by the International Agency for Research on Cancer, with a focus on geographic variability across 20 globes about 18.1 million new cancer cases about 17.0 million excluding no melanoma skin cancer and 9.6 million cancer deaths in 2018. Furthermore, the master cell banks are useful for the preparation of working cell banks and thus find applicability in various research and development perspectives for stem cell therapy and gene therapy thereby resulting to section growth. The occurrence of favorable government initiatives pertaining to the R&D for development of stable cell lines, the opening of new technology for storage and description of cell lines are among the critical factors predictable to advance market growth over the forecast period.

However, the high cost associated with storing these cells in cell banks is a major challenge faced by this market which may hamper the growth of cell banking outsourcing market. In addition, the various legal challenges associate with banking a variety of cells, especially considering stem cells banking, are expected to restrain market growth. The advanced technologically cryopreservation techniques are expected to fuel the growth of this market throughout the forecast period. In spite of that, the increase in the average life expectations due to advanced medical research and improved general lifestyle of the population and straightforward regulations for the stem cell researchers are expected to create significant potential for this market in coming few years. Increasing number of adipose tissue banking can offer various opportunities of the cell banking outsourcing market.

Global Cell Banking Outsourcing Market Regional Analysis

North America is expected to dominate the global cell banking outsourcing drug market due to the highest market share owing to the increasing number biopharmaceutical companies & manufacturers and increasing awareness for the use of stem cells as therapeutic proteins and antibiotics in this region. According to the World Health Organization (WHO), the American Cancer Society epidemiologists, at least 42% of newly diagnosed cancers in the U.S. about 729,000 cases are potentially avoidable, including 19% that are caused by smoking and 18% that are caused by a combination of excess body weight, physical inactivity, excess alcohol consumption, and poor nutrition. In addition, presence of regulatory authorities that promotes continuous R&D activities is also supplementing the market growth in North America.

The Asia Pacific is expected to witness significant growth in demand over the forecast period owing to increase in number of supportive government initiatives pertaining to investments in biotechnology sector in this region. In addition, the ongoing R&D activities for cancer treatment and fertility preservation facilitate the demand for cell banking services in this region.

Key Benefits for Global Cell Banking Outsourcing Market Report

Global Cell Banking Outsourcing Market report covers in depth historical and forecast analysis.

Global Cell Banking Outsourcing Market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Market Drivers, Market Restraints, Market opportunities, Competitive Analysis, Regional and Country Level.

Global Cell Banking Outsourcing Market report helps to identify opportunities in market place.

Global Cell Banking Outsourcing Market report covers extensive analysis of emerging trends and competitive landscape.

Global Cell Banking Outsourcing Market Segmentation:

By Type:Master Cell Banking, Viral Cell Banking, Working Cell Banking

By Application:Bank Storage, Working Cell Bank Storage, Master Cell Bank Storage, Cell Storage Stability Testing, Bank Preparation, Bank Characterization & Testing

Regional & Country AnalysisNorth America, U.S., Mexico, Canada , Europe, UK, France, Germany, Italy , Asia Pacific, China, Japan, India, Southeast Asia, South America, Brazil, Argentina, Columbia, The Middle East and Africa, GCC, Africa, Rest of Middle East and Africa

Table of Content

1. Chapter Report Methodology1.1. Research Process1.2. Primary Research1.3. Secondary Research1.4. Market Size Estimates1.5. Data Triangulation1.6. Forecast Model1.7. USPs of Report1.8. Report Description

2. Chapter Global Cell Banking Outsourcing Market Overview: Qualitative Analysis2.1. Market Introduction2.2. Executive Summary2.3. Global Cell Banking Outsourcing Market Classification2.4. Market Drivers2.5. Market Restraints2.6. Market Opportunity2.7. Cell Banking Outsourcing Market: Trends2.8. Porters Five Forces Analysis2.9. Market Attractiveness Analysis

3. Chapter Global Cell Banking Outsourcing Market Overview: Quantitative Analysis

4. Chapter Global Cell Banking Outsourcing Market Analysis: Segmentation By Type

5. Chapter Global Cell Banking Outsourcing Market Analysis: Segmentation By Application

Continued.

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At 14.6% CAGR, Cell Banking Outsourcing Market 2020 Industry Analysis of Current Trends and Opportun - PharmiWeb.com

Skin Stem Cells Comments Off on At 14.6% CAGR, Cell Banking Outsourcing Market 2020 Industry Analysis of Current Trends and Opportun – PharmiWeb.com | December 21st, 2020

Launched in 2013, cruelty-free skincare brand Drunk Elephant has captured the attention of beauty experts, brands, and a strong customer following.

The company, founded by former stay-at-home mom Tiffany Masterson, is one of the fastest-growing skin care brands in the industry. According to Fashionista, this is largely thanks to its philosophy. Masterson is a believer in clean beauty, an ethos thats reflected in Drunk Elephants product line-up. Speaking to how the company came to be, Masterson said, I came up with these criteria: It has to be nontoxic or low-hazard and it has to be bioavailable, meaning able to get into your skin.

As such, all Drunk Elephant products are formulated without what Masterson deemed the Suspicious Six, ingredients that she believed her company could do without: essential oils, silicones, sodium lauryl sulfate aka SLS, drying alcohols, silicones, chemical screens, and fragrances and dyes. When it comes to ingredients, Drunk Elephant goes by the philosophy of If theres any DOUBT, its OUT.

I determined that the six ingredients I wanted to avoid, which I realized were largely there [in competitors products] for marketing, Masterson explained. Silicones make a product feel better; dyes make a product look better; essential oils and fragrance make a product smell better; SLS is a cleansing agent that penetrates your skin and irritates and strips it.

She wanted her brand to be as clean and non-irritating as possible. She even helped formulate the products herself: We went through, ingredient by ingredient, and we swapped out a couple that canceled each other out or that did the same thing, but the resulting ingredient decks were very similar to the ones I first wrote down for every product.

For Masterson, it was a perfect storm of her desire to make a clean beauty brand and evolving consumer shopping habits. Consumer mistrust in companies is on the rise, but for good reason. Last July, Johnson & Johnson was told to pay $4.7 billion to families who claimed that using its talcum powder products caused their ovarian cancer.

While natural and clean beauty are on the rise, Vox points out that the terms are not regulated by the FDA nor the Federal Trade Commission. This means that any company can use the terms for their products and define it as they wish. Clean beauty is still finding favor with consumers its a trend thats been highlighted by Whole Foods Market. People are also becoming concerned over animal ingredients in skincare and makeup, which has helped lead to the rise of vegan beauty, according to The New York Times.

But Drunk Elephants ingredients list shows that Masterson takes her all-natural ethos seriously. In addition to being clean, all products are made in the US.

The company has also resonated with customers Masterson hasnt done any traditional marketing or influencer partnerships. But, she was able to build her brand to the point where she landed in Sephora the TLC Sukari Babyfacial was actually one of the stores top-selling products of 2017.

Drunk Elephant is a cruelty-free brand, but not all products are vegan. The company uses honey in certain formulas. However, it currently offers 12 products formulated without any animal ingredients.

Drunk Elephants A-Passioni Retinol Cream combines vegan retinol with skin-nourishing ingredients like peptides, vitamin F, and plant-based ingredients like passionfruit, apricot, marula and jojoba oils, and kale. This vegan face cream was formulated to diminish the appearance of fine lines and wrinkles and sun damage while evening your skin tone and texture for a younger-looking complexion. Its free from essential oils, silicones, and fragrance.

Check it out here.

Drunk Elephants C-Tango Multivitamin Eye Cream was specially formulated for the sensitive skin around your eyes. This rich, cruelty-free cream features eight peptides, five different kinds of vitamin C and cucumber extract that are said to make skin stronger. According to the company, its rich in antioxidants, ceramides and plant oils that restore and refresh appearance. It can be used in the morning or at night. This cruelty-free formula was named editors choice by Womens Health magazine.

Check it out here.

Described as a retro-style moisturizer, the Lala Retro Whipped Cream is formulated for dull, dry skin. The cream, which has a light, airy texture, is formulated with six African oils that help keep moisture locked in. It contains plantain extract to brighten and sodium hyaluronate crosspolymer that hydrates all day long. Like other Drunk Elephant products, it contains antioxidant-rich ingredients like green tea, which soothes the skin and fights signs of aging. Although its rich, the light airy texture keeps it from feeling heavy on the skin, plus it has a skin friendly pH level of 5.5.

This cream is also a three-time consecutive winner of Allures Readers Choice Awards as well as the 2017 winner of InStyle magazines Readers Choice Beauty Awards.

Check it out here.

Drunk Elephant calls the B-Hydra Intensive Hydration Gel a cool glass of water for your thirsty skin.With a light texture, this gel moisturizer was designed to brighten skintone and improve texture with provitamin B and pineapple ceramide, which helps your skin retain moisture. A blend of watermelon rind, apple, and lentil is said to provide hydration for up to 24 hours. It was designed with molecular size and pH level in mind, resulting in a bio-available moisturizer that keeps your skin looking healthy without irritating it.

Check it out here.

With a breakthrough formula, Protini Polypeptide Cream contains a proprietary lend of peptides, amino acids, and pygmy waterlily that improves skin tone, texture, and firmness. According to Drunk Elephant, the protein-rich formula reduces the appearance of fine lines, wrinkles, and sun damage they say its like adding a shot of plant-based protein to your smoothie. Its ideal for all skin types.

This cruelty-free and vegan moisturizer also won the 2018 Healthy Skin Award from Womens Health and the 2018 Skin Award from Cosmopolitan.

Check it out here.

The Shaba Complex Eye Serum is a satiny moisturizer made to tackle fine lines, wrinkles, and sun damage in the skins delicate undereye area. With ingredients like black tea ferment and copper peptides, this vegan eye serum helps smooth skin and is said to slow down the signs of aging. It also contains edelweiss stem cells and niacinamide, which work to reduce wrinkles. The antioxidant-rich ingredient Co-Q10 helps prevent premature signs of aging and fades age spots.

Check it out here.

The Beste No 9 Jelly Cleanser gets your skin clean without harsh ingredients. A blend of surfactants and emollients gets rid of makeup, excess oil, dirt, and pollution and leaves skin feeling soft when rinsed away. Free from SLS and essential oils, its ideal for all skin types, including sensitive.

This cruelty-free and vegan jelly cleanser was a winner of the 2018 Total Beauty Awards, the 2018 Cosmopolitan Skin Awards, and the 2018 Self Healthy Beauty Awards.

Check it out here.

The three-in-one Juju Bar cleanses, exfoliates, and moisturizes your skin without harsh ingredients. This mild vegan soap bar is formulated with thermal mud and bamboo powder, which help create a lather that helps get rid of dirt and oil. Its also said to minimize the appearance of your pores A superfood blend of acai and goji extracts also help fight the signs of aging while marula oil provides moisture. Its also fragrance-free and wont dry your skin out like conventional bar skin.

Check it out here.

The D-Bronzi Anti-Pollution Sunshine Serum delivers a bronzed look without the long-term consequences of sun damage. This vegan bronzer is made with a chronopeptide that is said to mimic the antioxidant effects of vitamin D, marula oil, and black currant seeds while vitamin F moisturizes and protects. Antioxidant-rich ingredients like raw cocoa powder and white tea also help protect your skin against environmental stressors like pollution while giving your skin a sun-kissed glow.

According to Drunk Elephant, a little bit goes a long way, so it should be mixed with a serum or moisturizer. Its free from fragrance, essential oil, and silicones.

Check it out here.

The T.L.C. Framboos Glycolic Night Serum is a 12 percent AHA/BHA moisturizer that helps refine and resurface skin for a more radiant complexion while reducing the appearance of fine lines, wrinkles, and discoloration. Its said to help boost the performance of other products. This cruelty-free serum is formulated with a blend of glycolic, lactic, tartaric, citric, and salicylic acids plus raspberry extract to deliver chemical exfoliation without harshness. It also contains horse chestnut and white tea to sooth the skin. While AHAs can be sensitizing, plant extracts help keep it balanced.

This vegan and cruelty-free night serum was a winner of the 2018 Shape Editor Pick, the 2018 Real Simple Road Test, and was the product winner of New Beautys Beauty Choice Awards.

Check it out here.

The T.L.C. Sukari Babyfacial is an AHA/BHA, cruelty-free and vegan facial in a bottle. Formulated with every skin type in mind, its made from a blend of glycolic, tartaric, lactic, citric, and salicylic acids that gently exfoliate. Antioxidants like matcha, apple, and milk thistle sooth while sodium hyaluronate crosspolymer and plant-based oils hydrate and nourish. It also contains chickpea flour, which brightens and balances. Niacinamide aka vitamin B3 fights the signs of aging and passion fruit seed oil combats redness. This hydrating facial is said to brighten and tone while minimizing the appearance of pores and fine lines and leaving your skin feeling baby-soft.

A customer favorite, this was a winner of the 2018 Essence Black Beauty Awards, the 2019 Teen Vogue Acne Awards, was featured in Glamour, and it was named on the Into the Gloss Top 25 list.

Check it out here.

Drunk Elephants Virgin Marula Luxury Facial Oil is a luxury moisturizer made from the antioxidants tocopherol (vitamin E), tocotrienol, phenolic compounds, and flavonoids, plus omegas 6 and 9. Described as being like rehab for your skin,this facial oil nourishes your skin to provide a youthful glow. The virgin marula oil featured is extracted from the pip of the fruit and is left pure, without fragrance or added ingredients. This quick-absorbing oil helps reverse the skins of aging, redness, and blotchiness while improving elasticity. Its also naturally anti-microbial and ideal for all skin types.

According to Drunk Elephant, its Virgin Marula Luxury Facial oil is cold-pressed using unheated water extraction, which ensures that the oil stays fresher longer. Its free from fragrance, essential oils, and silicone.

Check it out here.

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The Definitive Guide to All Things Vegan By Drunk Elephant - LIVEKINDLY

Skin Stem Cells Comments Off on The Definitive Guide to All Things Vegan By Drunk Elephant – LIVEKINDLY | December 21st, 2020

Autologous bone marrow mononuclear cell (BMMNC) transplantation has been widely studied in recent years. The fresh cell cocktail in BMMNCs, without going through the in vitro culture process, helps to establish a stable microenvironment for osteogenesis, and each cell type may play a unique role in bone regeneration. Our study compared the efficacy of concentrated fresh BMMNCs and cultured bone marrow-derived mesenchymal stem cells (BMSCs) in Beagle dogs for the first time. Fifteen-millimeter segmental bone defects were created in the animals tibia bones. In BMMNCs group, the defects were repaired with concentrated fresh BMMNCs combined with -TCP (n = 5); in cultured BMSC group, with in vitro cultured and osteo-induced BMSCs combined with -TCP (n = 5); in scaffold-only group, with a -TCP graft alone (n = 5); and in blank group, nothing was grafted (n = 3). The healing process was monitored by X-rays and single photon emission computed tomography. The animals were sacrificed 12months after surgery and their tibias were harvested and analyzed by microcomputed tomography and hard tissue histology. Moreover, the microstructure, chemical components, and microbiomechanical properties of the regenerated bone tissue were explored by multiphoton microscopy, Raman spectroscopy and nanoindentation. The results showed that BMMNCs group promoted much more bone regeneration than cultured BMSC group. The grafts in BMMNCs group were better mineralized, and they had collagen arrangement and microbiomechanical properties similar to the contralateral native tibia bone. These results indicate that concentrated fresh bone marrow mononuclear cells may be superior to in vitro expanded stem cells in segmental bone defect repair. 2020 The Authors. STEM CELLS TRANSLATIONAL MEDICINE published by Wiley Periodicals LLC on behalf of AlphaMed Press.

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Bone Marrow Stem Cells Comments Off on Comparison of concentrated fresh mononuclear cells and … | December 21st, 2020

Image: UN/Unsplash.

One of the astonishing aspects of the human response to the COVID-19 pandemic has been how quickly scientists pivoted to studying every facet of the virus in order to mitigate the loss of life and plan for a return to normalcy. At the same time, a lot of non-coronavirus research ground to a near halt.

With research labs and offices shuttered for all but essential workers, many scientists were stuck at home, their fieldwork and meetings canceled and planned experiments kicked down the road as they struggled to figure out how to keep their research programs going. Many took the opportunity to catch up on writing grants and papers; some in between caring for kids came up with strategic workarounds to keep the scientific juices flowing.

To gauge how researchers in different fields are managing,Knowable Magazine spoke with an array of scientists and technical staff among them a specialist keeping alive genetically important strains of fruit flies, the maintenance chief of an astronomical observatory working to keep telescopes safe and on standby during the lockdown, and a paediatrician struggling to manage clinical trials for a rare genetic disease. Here are a few slices of scientific life during the pandemic.

Agnieszka Czechowicz, Stanford University School of Medicine

Czechowicz is a paediatrician in Stanfords division of stem cell transplantation and regenerative medicine, where she manages a research group that develops new therapies and conducts clinical trials on rare genetic diseases.

Agnieszka Czechowiczs father suffers from severe Parkinsons disease. The coronavirus pandemic forced him to remain indoors and away from people, robbing him of the physical conditioning and social interactions he needs to cope with his disease. A recent fall left him in the hospital, bringing the additional worry that he might contract COVID-19 there and isolating him further.

For Czechowicz, his situation brought into sharp relief the challenges the coronavirus has forced upon those carrying out clinical trials, including those she is running, which involve patients traveling to hospitals around the country. Would I have him travel to any clinical site right now for a new Parkinsons treatment? she says. Absolutely not.

The pandemic forced Czechowicz to halt clinical trials she oversees for a rare genetic disease of children called Fanconi anAemia, a condition that impairs the bodys ability to repair damaged DNA and often leads to bone marrow failure and cancer. The treatment Czechowicz and colleagues are testing involves extracting blood-forming stem cells from the patients bone marrow, inserting a healthy copy of a missing or malfunctioning gene and then reinfusing those cells back into the patient.

Every aspect of what I do is massively impacted by the pandemic, Czechowicz says. One of her early-stage clinical trials involves testing the safety of the therapy. But during the initial shutdown which started in mid-March and lasted for two months her patients could not readily travel to Stanford for the necessary follow-up visits, and remote monitoring was difficult.

Theres special blood testing and bone marrow testing that we need to do. In particular, its critical to get the samples to make sure the patients, for example, arent developing leukAemia, she says. Theres no way to know that without really checking the bone marrow. She had to clear large hurdles to get her patients evaluated.

Another early-stage trial, designed to determine the effectiveness of the therapy, also had to stop enrolling new patients. Because speed is important when it comes to treating Fanconi anaemia the children are likely losing stem cells all the time any delay in treatment can be a source of great anxiety for their parents. Czechowicz had to explain to them why the trials were temporarily halted. It was really challenging to have these discussions with the families, she says.

With the easing of travel and workplace restrictions, the families began traveling to Stanford in June but with infections back on the rise, many families are becoming hesitant again, says Czechowicz. Fortunately, her trials are small, so she can guide each family through the process of safely resuming the trials and continuing with follow-up. Her own team also has to follow strict safety protocols. For example, even though her lab has 10 members, only two can be in the lab at any one time, and only one parent is allowed into the clinic with the child.

Not all clinical trials can pay such close attention to individual patients. Large trials with hundreds of patients can involve multiple sites and require much more monitoring, so resuming those remains difficult. Also, restrictions on working full bore are slowing the pipeline for new therapies. The impact of that, were not going to see for many years to come, Czechowicz says.

Abolhassan Jawahery, University of Maryland, College Park

Jawahery is a particle physicist and a member of LHCb, one of the main experiments at the Large Hadron Collider (LHC) at CERN, the particle physics laboratory near Geneva.

In December 2018, well before the coronavirus pandemic began, the LHC shut down for upgrades. Housed in a 27-kilometre-long tunnel about 100 meters underground, the LHC accelerates two beams of protons, one clockwise and one counterclockwise, and makes them collide head-on at four locations. There, four gigantic subterranean detectors ATLAS, CMS, LHCb and ALICE sift through the debris of particles created by the collisions, looking for evidence of new physics. (For example, ATLAS and CMS found the Higgs boson, the fundamental particle of the Higgs field, which gives all elementary particles their mass.)

For its next set of experiments, which aim to probe the properties of subatomic particles with greater precision, the LHC needed to increase the intensity of its proton beams. Consequently, the four detectors needed to be upgraded too, to handle the resultant higher temperatures and increased radiation at the sites of the particle collisions. The work was on track for a restart around May 2021 until the pandemic swept all the scientists careful plans away.

The LHC and its four detectors are each run by a separate collaboration. CERN, which manages the LHC, is hopeful it can restart the collider by February 2022. They think that they can get the accelerator going if there are no more major catastrophic events, says physicist Abolhassan Jawahery. But the impact on the four detectors is less clear.

For the LHCb upgrade, Jawaherys team at the University of Maryland had been working on building about 4,000 extremely sensitive electronic circuit boards. These boards have to be burned in before they can be sent to CERN. We put them in an oven, literally cooking the boards and then running extensive tests in order to get them ready so that we can put them in the accelerator and run them for 10 to 20 years, says Jawahery. And none of that could be done during the pandemic shutdown.

The team resumed its work in June, but with restrictions put in place by the state of Maryland. Jawahery runs two labs, and for months was allowed only two people at a time in one lab and three in the other, making progress extremely slow. Still, his team is fortunate that it does not depend on supplies from countries hit hard by the coronavirus. Other labs werent so lucky. Scientists in Milan, for example, built some electronics and detector components for the LHCb, and a lab at Syracuse University in New York built sensors that relied on shipments from Milan. When Milan was completely closed down at the height of the pandemic, Syracuse, too, stopped working on Milan-dependent components.

For Jawahery the lockdown had a silver lining. The LHCs most recent run had produced about 25 gigabytes of data per second but his team had found little time to analyse any of it before the pandemic. We were complaining that we were spending all our time building the new instrument and the data keeps on coming, he says. When he and his team were locked out of their labs, they turned to their data backlog. We could do actual physics, he says. We are already getting ready to publish some papers.

Gordon Gray, Princeton University

Gray is a professionalDrosophila specialist in the department of molecular biology.

Gordon Gray has been called the chef de cuisine of Princetons fly kitchen, where he has been feeding flies for 46 years. He concocts meals for millions of fruit flies, at least 150 litres each week. When the pandemic hit in March and universities around the world shut down, Princeton deemed Grays work an essential service: The Drosophilafruit flies could not be allowed to die off.

Princetons flies include mutant and transgenic strains everything from ones that allow researchers to study the genes that influence normal development of a fly embryos organs, to those that have cancer-causing mutations. If the flies starved, researchers would need months or years to recreate these strains, says Princeton molecular biologist Elizabeth Gavis. And often, as techniques in molecular biology improve, the biologists reexamine flies they had studied earlier to get a more fine-grained understanding, making it worthwhile to preserve the strains.

Normally, if a lab had to shut down, researchers would send their flies to stock centres, such as one at Bowling Green State University in Ohio, that preserve the flies as part of their genetic library. But the stock centres couldnt handle Princetons flies, so Gray found himself on his own. Its basically catch as catch can with regards to the various labs here, just to keep things operational, he says.

For months, university pandemic restrictions have allowed only one person to be in Grays kitchen at a time. This has caused problems. Before the pandemic began, Gray, who is in his late 60s, had started training someone as a backup. But because of the one-person restriction, Gray and his trainee havent been able to work together. Gray envisions doing so soon, while wearing masks, keeping nearly 12 feet apart and communicating using hand signals.

To whip up a batch of fly food, or media, Gray uses a 50-litre steel cauldron, to which is attached a mixer that looks like an outboard motor. Gray fills the cauldron with water and adds agar, sugars, yeasts, salts and cornmeal, then brings it to a boil, all the while stirring watchfully. You dont want it to boil over, because when it does you wind up with a gigantic pancake on the floor, which you have to scoop up immediately because it gels, he says. Once the suspension cools to the right temperature, Gray adds an acid to inhibit mould, then dispenses precise amounts of the media into bottles and vials.

Even before the pandemic, Grays kitchen was isolated, to keep errant fruit flies from contaminating the pristine media. But at least he could work regular hours, because he knew the rhythms of the 10 or so fly labs he cooked for. That has changed. Labs, restricted to two occupants at a time, are now working seven days a week on rotating shifts. Gray comes in to work at all hours, because he cannot predict when each batch of fly food will run out and hell need to cook more.

He tries to work mostly at night to avoid coming into contact with others. But he still worries for his health, given his asthma and age-related risk. The relentless pandemic is taking a toll. Its exhausting, he says. It doesnt help not knowing when we will return to a sense of normalcy.

Celeste Kidd, University of California, Berkeley

Kidd is a child developmental psychologist who uses behavioural tests and computational methods to understand how children acquire knowledge.

When UC Berkeley locked down in March, Celeste Kidd found herself closeted at home, dealing simultaneously with virtual meetings and her three-year-old son. During the early days of the pandemic, Kidd kept a supply of treats handy, and when her toddler came up to her during a meeting shed sneak him some under the desk. But she hadnt accounted for how long the pandemic would last. It turns out thats not a good strategy, long term, she says. I was very literally rewarding him for bad behaviour.

Kidds son soon learned that acting up during her meetings meant more candy. I knew that would happen. I did it anyway because I didnt have the bandwidth to come up with a better solution, she says. But Kidd knew from her own research that children are also extremely flexible and can unlearn behaviours. Eventually, she had a chat with her son. First, she admitted to him that she had made a mistake by giving him candy when he disrupted her meetings, and that was bad of her. Then she brought in new rules: no candy for misbehaving and misbehaviour could even mean no treats for the rest of day. We had some meltdown moments, says Kidd. But he gets it now and he doesnt do those things.

Her son may be the only child Kidd gets to interact with during the pandemic. Thats a huge loss for her research, because the bulk of her work focuses on young children. In normal times, families would bring their children to her lab, where her research team would track their gaze as they watched videos. In one study, for example, infants about seven to nine months old would look away (demonstrating lack of interest) when the events in the video were either too complex or too simple, suggesting that infants use their cognitive resources for stimuli that have just the right amount of information.

Such work, of course, requires the presence of parent, child and researchers, all in the same room. None of that is going to happen anytime soon, she says. Those families are not going to feel comfortable coming in for a while.

Kidd is also concerned about the impact of the pandemic on younger scientists. One of her undergraduate students had spent six months working on a study aimed at exploring the complexity of kids play patterns using physical objects and their relation to working memory and other cognitive resources. The university had approved the protocol, but shelter-in-place orders went into effect the week the first child was to come for the experiment. I feel so bad for her as a young scientist, to have done all this hard work and then right when you get to the fun part, which is collecting the data and finding out if her ideas have lasting merit, she doesnt get to do that part, Kidd says.

The situation might be even worse for grad students and postdocs. All of them are experiencing a big blow to morale in general, because there is so much uncertainty about what the future holds, she says. University budget cuts mean fewer slots for graduate students and fewer jobs for postdocs. Its very hard to stay motivated and get things done when youre not sure if there will be a payoff in the future, says Kidd. Thats literally a study that we ran in the lab so were all acutely aware of it.

Maxime Boccas, ESO Paranal Observatory

Boccas is the head of maintenance, support and engineering at the European Southern Observatorys Paranal Observatory in Chile.

When the massive domes of the Very Large Telescope, a constellation of four 8-meter-class telescopes atop Mount Paranal in Chiles Atacama Desert, open to the night sky each evening and the telescopes get ready for observations, its like a dragon waking up.

When the pandemic hit in March, the dragon on Mount Paranal closed its eyes to the cosmos and slept the first shutdown in its 20-year history, which included a major earthquake in 2010 that paralyzed much of the rest of Chile. For those who had to leave Paranal, it was like being sent away from home. We spend 40% of our life here, says Maxime Boccas, who oversaw the process of ensuring an orderly shutdown of the sites scientific and technical facilities. We work and sleep here, and we stay here eight days in a row. Some of Boccass colleagues have been doing that for 20 to 25 years. Leaving Paranal was like leaving their second home. That was a weird feeling.

The skeleton staff just 20 of the normal 150 or so people remained on site kept the observatory safe, ensuring that essential systems continued working: computers that astronomers were accessing remotely, the fire detection system and the earthquake protection system essential for protecting the 8-meter-wide primary mirrors from Chiles frequent quakes. The mirrors will likely never be made again, says Boccas. All the factories that cast and polished them are dismantled. If we lost a mirror, it would take between 5 and 10 years to build up the factory again and fabricate it. So each mirror has an airbag a tube that inflates around it when the system detects tremors and other protections.

During the shutdown, astronomers kept their fingers crossed. They were anxious that no big thing, like a supernova in our galaxy, would explode, Boccas says. The heavens have been quiet, but the six-month shutdown harmed research that involves continuously monitoring the same patch of the sky for transient phenomena such as gamma ray bursts. It creates a hole in their science program, says Boccas.

The observatory began a slow return to normalcy on September 9. Boccas is overseeing the reawakening of each telescope, one at a time. The staff still less than full strength is now working in shifts that have doubled from 8 to 15 days to limit the amount of travel to and from the site. The four large telescopes are now up and running again, and Boccas hopes they will be back to working together as one by the end of January.

Boccas, his crew and a few lucky astronomers are glad to be back at Paranal. It really feels like a family and I think everyone has noticed that, he says. Even in the kitchen, they have to cook for 30 people instead of 150, so the quality of the food is different, its slightly better.

But even as people return to the observatory, Boccas worries about long-term effects of the shutdown. Given the reduced staff, he has had to cut down on the frequency of preventive maintenance tasks, such as changing belts and lubricating motors, potentially shortening the lifetime of some components. We will not know until six months, a year or three years from now, he says.

This article is part ofReset: The Science of Crisis & Recovery, an ongoing series exploring how the world is navigating the coronavirus pandemic, its consequences and the way forward. Reset is supported by a grant from the Alfred P. Sloan Foundation.

Anil Ananthaswamy is a science journalist who enjoys writing about cosmology, consciousness and climate change. Hes a 2019-20 MIT Knight Science Journalism fellow. His latest book is Through Two Doors at Once. http://www.anilananthaswamy.com.

This article originally appeared in Knowable Magazine, an independent journalistic endeavour from Annual Reviews.

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How Researchers Are Making Do in the Time of COVID-19 - The Wire Science

Bone Marrow Stem Cells Comments Off on How Researchers Are Making Do in the Time of COVID-19 – The Wire Science | December 21st, 2020

Problems soon after transplant

Many of the problems that can happen shortly after the transplant come from having the bone marrow wiped out by medicines or radiation just before the transplant. Others may be side effects of the conditioning treatments themselves.

Your transplant team can help you cope with side effects. Some can be prevented, and most can be treated to help you feel better.This is not a complete list and you should tell your doctor or transplant team about any problems you have or changes you notice. Some of these problems can be life-threatening, so its important to be able to reach your doctor or transplant team at night, on weekends, and during holidays. Ask for their after hours contact numbers to makesure you will be able to do this.

Mucositis (inflammation or sores in the mouth) is a short-term side effect that can happen with chemo and radiation. It usually gets better within a few weeks after treatment, but it can make it very painful to eat and drink.

Good nutrition is important for people with cancer. If mouth pain or sores make it hard to eat or swallow, your transplant team can help you develop a plan to manage your symptoms.

Because chemotherapy drugs can cause severe nausea and vomiting, doctors often give anti-nausea medicines at the same time as chemo to try to prevent it. As much as possible, the goal is to prevent nausea and vomiting, because its easier to prevent it than it is to stop it once it starts. Preventive treatment should start before chemo is given and should continue for as long as the chemo is likely to cause vomiting, which can be up to 7 to 10 days after the last dose.

No one drug can prevent or control chemo-related nausea and vomiting 100% of the time. In many cases, two or more medicines are used. Youll need to tell your transplant team how well the medicines are controlling your nausea and vomiting. If they arent working, they will need to be changed.

For at least the first 6 weeks after transplant, until the new stem cells start making white blood cells (engraftment), you can easily get serious infections. Bacterial infections are most common during this time, but viral infections that were controlled by your immune system can become active again. Fungal infections can also be an issue. And even infections that cause only mild symptoms in people with normal immune systems can be quite dangerous for you. This is because right after the transplant you don't have many white blood cells that are working well, and they are the primary immune cells that fight off infections.

You may be given antibiotics to try to prevent infections until your blood counts reach a certain level. For instance, pneumocystis pneumonia (often called PCP) is a common infection thats easy to catch. Even though the germ doesnt harm people with normal immune systems, for others it can cause fever, cough, and serious breathing problems. Antibiotics are often used to keep transplant patients from getting this.

Your doctor may check you before the transplant for signs of certain infections that may become active after transplant, and give you special medicines to keep those germs under control. For example, the virus called CMV (cytomegalovirus) is a common infection that many adults have or had in the past. Adults with healthy immune systems may not have any symptoms because their immune system can keep the virus under control. But, CMV can be a cause of serious pneumonia in people who have had transplants, because the transplant lowers the amount of white blood cells they have. Pneumonia from CMVmainly happens to people who were already infected with CMV, or whose donor had the virus. If neither you nor your donor had CMV, the transplant team might follow special precautions to prevent this infection while you are in the hospital.

After engraftment, the risk of infection is lower, but it still can happen. It can take 6 months to a year after transplant for the immune system to work as well as it should. It can take even longer for patients with graft-versus-host disease (GVHD, see below). It's important to talk to your cancer care team about your risk for infection during this time.

Because of the increased risk, you will be watched closely for signs of infection, such as fever, cough, shortness of breath, or diarrhea. Your doctor may check your blood often, and extra precautions will be needed to keep you from being exposed to germs. While in the hospital, everyone who enters your room must wash their hands well. They may also wear gowns, shoe coverings, gloves, and masks.

Since flowers and plants can carry bacteria and fungi, theyre not allowed in your room. For the same reason, you may be told not to eat certain fresh fruits and vegetables. All your food must be well cooked and handled very carefully by you and family members. You might need to avoid certain foods for a while.

You may also be told to avoid contact with soil, feces (stool, both human and animal), aquariums, reptiles, and exotic pets. Your team may tell you to avoid being near disturbed soil, bird droppings, or mold. You will need to wash your hands after touching pets. Your family may need to move the cats litter box away from places you eat or spend your time. Also, you should not clean pet cages or litter boxes during this time. Instead, give this task to a family member or friend.

Your transplant team will tell you and your family in detail about the precautions you need to follow. There are many viruses, bacteria, and fungi that can cause infection after your transplant. You may be at risk for some more than others.

Despite all these precautions, patients often develop fevers, one of the first signs of infection. In fact, sometimes fever is the only sign of infection, so it's very important to contact your cancer care team if you have one or if you have any other signs of infection. You'll probably be asked to take your temperature by mouth every day or twice a day for a while. And your cancer care team will let you know when you should call in your temperature to them. If you get a fever, tests will be done to look for possible causes of the infection (chest x-rays, urine tests, and blood cultures) and antibiotics will be started.

After transplant, youre at risk for bleeding because the conditioning treatment destroys your bodys ability to make platelets. Platelets are the blood cells that help blood to clot. While you wait for your transplanted stem cells to start working, your transplant team may have you follow special precautions to avoid injury and bleeding.

Platelet counts are low for at least several weeks after transplant. In the meantime, you might notice easy bruising and bleeding, such as nosebleeds and bleeding gums. If your platelet count drops below a certain level, a platelet transfusion may be needed. Youll need to follow precautions until your platelet counts stay at safe levels.

It also takes time for your bone marrow to start making red blood cells, and you might need red blood cell transfusions from time to time as you recover.

For more information on the transfusion process, see Blood Transfusion and Donation.

Pneumonitis is a type of inflammation (swelling) in lung tissue thats most common in the first 100 days after transplant. But some lung problems can happen much later even 2 or more years after transplant.

Pneumonia caused by infection happens more often, but pneumonitis may be caused by radiation, graft-versus-host disease, or chemo rather than germs. Its caused by damage to the areas between the cells of the lungs (called interstitial spaces).

Pneumonitis can be severe, especially if total body irradiation was given with chemo as part of the pre-transplant (conditioning) treatment. Chest x-rays will be taken in the hospital to watch for pneumonitis as well as pneumonia. Some doctors will do breathing tests every few months if you have graft-versus-host disease (see next section).

You should report any shortness of breath or changes in your breathing to your doctor or transplant team right away. There are many other types of lung and breathing problems that also need to be handled quickly.

Graft-versus-host disease (GVHD) can happen in allogeneic transplants when the immune cells from the donor see your body as foreign. (Remember: The recipients immune system has mostly been destroyed by conditioning treatment and cannot fight back, so the new stem cells make up most of the immune system after transplant.) The donor immune cells may attack certain organs, most often the skin, gastrointestinal (GI) tract, and liver. This can change the way the organs work and increase the chances of infection.

GVHD reactions are very common and can range from barely noticeable to life-threatening. Doctors think of GVHD as acute or chronic. Acute GVHD starts soon after transplant and lasts a short time. Chronic GVHD starts later and lasts a long time. A person could have one, both, or neither type of GVHD.

Acute GVHD can happen 10 to 90 days after a transplant, though the average time is around 25 days.

About one-third to one-half of allogeneic transplant recipients will develop acute GVHD. Its less common in younger patients and in those with closer HLA matches between donor and the patient.

The first signs are usually a rash, burning, and redness of the skin on the palms and soles. This can spread over the entire body. Other symptoms can include:

Doctors try to prevent acute GVHD by giving drugs that suppress the immune system, such as steroids (glucocorticoids), methotrexate, cyclosporine, tacrolimus, or certain monoclonal antibodies. These drugs are given before acute GVHD starts and can help prevent serious GVHD. Still, mild GVHD will almost always happen in allogeneic transplant patients. Other drugs are being tested in different combinations for GVHD prevention.

The risk of acute GVHD can also be lowered by removing immune cells called T-cells from the donor stem cells before the transplant. But this can also increase the risk of viral infection, leukemia relapse, and graft failure (which is discussed later). Researchers are looking at new ways to remove only certain cells, called alloactivated T-cells, from donor grafts. This would reduce the severity of GVHD and still let the donor T-cells destroy any cancer cells left.

If acute GVHD does occur, it is most often mild, mainly affecting the skin. But sometimes it can be more serious, or even life-threatening.

Mild cases can often be treated with a steroid drug applied to the skin (topically) as an ointment, cream, or lotion, or with other skin treatments. More serious cases of GVHD might need to be treated with a steroid drug taken as a pill or injected into a vein. If steroids arent effective, other drugs that affect the immune system can be used.

Chronic GVHD

Chronic GVHD can start anywhere from about 90 to 600 days after the stem cell transplant. A rash on the palms of the hands or the soles of the feet is often the earliest sign. The rash can spread and is usually itchy and dry. In severe cases, the skin may blister and peel, like a bad sunburn. A fever may also develop. Other symptoms of chronic GVHD can include:

Chronic GVHD is treated with medicines that suppress the immune system, much like those used for acute GVHD. These drugs can increase your risk of infection for as long as you are treated for GVHD. Most patients with chronic GVHD can stop the immunosuppressive drugs after their symptoms improve.

Hepatic veno-occlusive disease (VOD) is a serious problem in which tiny veins and other blood vessels inside the liver become blocked. Its not common, and it only happens in people with allogeneic transplants, and mainly in those who got the drugs busulfan or melphalan as part of conditioning, or treatment that was given before the transplant.

VOD usually happens within about 3 weeks after transplant. Its more common in older people who had liver problems before the transplant, and in those with acute GVHD. It starts with yellowing skin and eyes, dark urine, tenderness below the right ribs (this is where the liver is), and quick weight gain (mostly from fluid that bloats the belly). It is life-threatening, so early diagnosis of VOD is very important. Researchers continue to find ways to try to measure a person's chances of getting VOD so that treatment can start as soon as possible.

Grafts fail when the body does not accept the new stem cells (the graft). The stem cells that were given do not go into the bone marrow and multiply like they should. Graft failure is more common when the patient and donor are not well matched and when patients get stem cells that have had the T-cells removed. It can also happen in patients who get a low number of stem cells, such as a single umbilical cord unit. Still, its not very common.

Graft failure can lead to serious bleeding and/or infection. Graft failure is suspected in patients whose counts do not start going up within 3 to 4 weeks of a bone marrow or peripheral blood transplant, or within 7 weeks of a cord blood transplant.

Although it can be very upsetting to have this happen, these people can get treated with a second dose of stem cells, if they are available. Grafts rarely fail, but if they do it can result in death.

The type of problems that can happen after a transplant depend on many factors, such as the type of transplant done, the pre-transplant chemo or radiation treatment used, the patients overall health, the patients age when the transplant was done, the length and degree of immune system suppression, and whether chronic graft-versus-host-disease (GVHD) is present and how bad it is. The problems can be caused by the conditioning treatment (the pre-transplant chemotherapy and radiation therapy), especially total body irradiation, or by other drugs used during transplant (such as the drugs that may be needed to suppress the immune system after transplant). Possible long-term risks of transplant include:

The medicines used in transplants can harm the bodys organs, such as the heart, lungs, kidneys, liver, bones/joints, and nervous system. You may need careful follow-up with close monitoring and treatment of the long-term organ problems that the transplant can cause. Some of these, like infertility, should be discussed before the transplant, so you can prepare for them.

Its important to find and quickly treat any long-term problems. Tell your doctor right away if you notice any changes or problems. Physical exams by your doctor, blood work, imaging tests, lung/breathing studies, and other tests will help look for and keep tabs on organ problems.

As transplant methods have improved, more people are living longer and doctors are learning more about the long-term results of stem cell transplant. Researchers continue to look for better ways to care for these survivors to give them the best possible quality of life.

The goal of a stem cell transplant in cancer is to prolong life and, in many cases, even cure the cancer. But in some cases, the cancer comes back (sometimes called relapse or recurrence depending on when it might occur after a transplant). Relapse or recurrence can happen a few months to a few years after transplant. It happens much more rarely 5 or more years after transplant.

If cancer comes back, treatment options are often quite limited. A lot depends on your overall health at that point, and whether the type of cancer you have responds well to drug treatment. Treatment for those who are otherwise healthy and strong may include chemotherapy or targeted therapy. Some patients who have had allogeneic transplants may be helped by getting white blood cells from the same donor (this is called donor lymphocyte infusion) to boost the graft-versus-cancer effect. Sometimes a second transplant is possible. But most of these treatments pose serious risks even to healthier patients, so those who are frail, older, or have chronic health problems are often unable to have them.

Other options may include palliative (comfort) care, or a clinical trial of an investigational treatment. Its important to know what the expected outcome of any further treatment might be, so talk with your doctor about the purpose of the treatment. Be sure you understand the benefits and risks before you decide.

Along with the possibility of the original cancer coming back (relapse) after it was treated with a stem cell transplant, there is also a chance of having a second cancer after transplant. Studies have shown that people who have had allogeneic transplants have a higher risk of second cancer than people who got a different type of stem cell transplant.

A cancer called post-transplant lymphoproliferative disease (PTLD), if it occurs, usually develops within the first year after the transplant. Other conditions and cancers that can happen are solid tumor cancers in different organs, leukemia, and myelodysplastic syndromes. These other conditions, if they occur, tend to develop a few years or longer after the transplant.

Risk factors for developing a second cancer are being studied and may include:

Successfully treating a first cancer gives a second cancer time (and the chance) to develop. No matter what type of cancer is treated, and even without the high doses used for transplant, treatments like radiation and chemo can lead to a second cancer in the future.

Post-transplant lymphoproliferative disorder (PTLD) is an out-of-control growth of lymph cells, actually a type of lymphoma, that can develop after an allogeneic stem cell transplant. Its linked to T-cells (a type of white blood cell that is part of the immune system) and the presence of Epstein-Barr virus (EBV). T-cells normally help rid the body of cells that contain viruses. When the T-cells arent working well, EBV-infected B-lymphocytes (a type of white blood cell) can grow and multiply. Most people are infected with EBV at some time during their lives, but the infection is controlled by a healthy immune system. The pre-transplant treatment given weakens the immune system, allowing the EBV infection to get out of control, which can lead to a PTLD.

Still, PTLD after allogeneic stem cell transplant is fairly rare. It most often develops within 1 to 6 months after allogeneic stem cell transplant, when the immune system is still very weak.

PTLD is life-threatening. It may show up as lymph node swelling, fever, and chills. Theres no one standard treatment, but its often treated by cutting back on immunosuppressant drugs to let the patients immune system fight back. Other treatments include white blood cell (lymphocyte) transfusions to boost the immune response, using drugs like rituximab to kill the B cells, and giving anti-viral drugs to treat the EBV.

Even though PTLD doesnt often happen after transplant, its more likely to occur with less well-matched donors and when strong suppression of the immune system is needed. Studies are being done to identify risk factors for PTLD and look for ways to prevent it in transplant patients who are at risk.

Most people who have stem cell transplants become infertile (unable to have children). This is not caused by the cells that are transplanted, but rather by the high doses of chemo and/or radiation therapy used. These treatments affect both normal and abnormal cells, and often damage reproductive organs.

If having children is important to you, or if you think it might be important in the future, talk to your doctor about ways to protect your fertility before treatment. Your doctor may be able to tell you if a particular treatment will be likely to cause infertility.

After chemo or radiation, some women may find their menstrual periods become irregular or stop completely. This doesnt always mean they cannot get pregnant, so birth control should be used before and after a transplant. The drugs used in transplants can harm a growing fetus.

The drugs used during transplant can also damage sperm, so men should use birth control to avoid starting a pregnancy during and for some time after the transplant process. Transplants may cause temporary or permanent infertility for men as well. Fertility returns in some men, but the timing is unpredictable. Men might consider storing their sperm before having a transplant.

For more information on having children after being treated for canceror sexual problems related to cancer treatment, see Fertility and Sexual Side Effects.

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Stem Cell or Bone Marrow Transplant Side Effects

Bone Marrow Stem Cells Comments Off on Stem Cell or Bone Marrow Transplant Side Effects | December 19th, 2020

18 months into the first serious clinical trials of CRISPR gene therapy for sickle cell disease and beta-thalassemiaand all patients are free from symptoms and have not needed blood transfusions.

Sickle cell disease (SCD) can cause a variety of health problems including episodes of severe pain, called vaso-occlusive crises, as well as organ damage and strokes.

Patients with transfusion-dependent thalassemia (TDT) are dependent on blood transfusions from early childhood.

The only available cure for both diseases is a bone marrow transplant from a closely related donor, an option that is not available for the vast majority of patients because of difficulty locating matched donors, the cost, and the risk of complications.

In the studies, the researchers goal is to functionally cure the blood disorders using CRISPR/Cas9 gene-editing by increasing the production of fetal hemoglobin, which produces normal, healthy red blood cells as opposed to the misshapen cells produced by faulty hemoglobin in the bodies of individuals with the disorders.

The clinical trials involve collecting stem cells from the patients. Researchers edit the stem cells using CRISPR-Cas9 and infuse the gene-modified cells into the patients. Patients remain in the hospital for approximately one month following the infusion.

Prior to receiving their modified cells, the seven patients with beta thalassemia required blood transfusions approximately every three to four weeks and the three patients with SCD suffered episodes of severe pain roughly every other month.

All the individuals with beta thalassemia have been transfusion independent since receiving the treatment, a period ranging between two and 18 months.

Similarly, none of the individuals with SCD have experienced vaso-occlusive crises since CTX001 infusion. All patients showed a substantial and sustained increase in the production of fetal hemoglobin.

15 months on, and the first patient to receive the treatment for SCD, Victoria Gray, has even been on a plane for the first time.

Before receiving CRISPR gene therapy, Gray worried that the altitude change would cause an excruciating pain attack while flying. Now she no longer worries about such things.

She told NPR of her trip to Washington, D.C: It was one of those things I was waiting to get a chance to do It was exciting. I had a window. And I got to look out the window and see the clouds and everything.

MORE: MIT Researchers Believe Theyve Developed a New Treatment for Easing the Passage of Kidney Stones

This December, theNew England Journal of Medicinepublishedthe first peer-reviewed research paperfrom the studyit focuses on Gray and the first TDT patient who was treated with an infusion of billions of edited cells into their body.

There is a great need to find new therapies for beta thalassemia and sickle cell disease, saidHaydar Frangoul, MD,Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcares TriStar Centennial Medical Center. What we have been able to do through this study is a tremendous achievement. By gene editing the patients own stem cells we may have the potential to make this therapy an option for many patients facing these blood diseases.

READ: For the First Time in the US, Surgeons Pump New Life into Dead Donor Heart for Life-Saving Transplant

Because of the precise way CRISPR-Cas9 gene editing works, Dr. Frangoul suggested the technique could potentially cure or ameliorate a variety of diseases that have genetic origins.

As GNN has reported, researchers are already using CRISPR to try and treat cancer, Parkinsons, heart disease, and HIV, as well.

Source: American Society of Hematology

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Every Patient Treated With CRISPR Gene Therapy for Blood Diseases Continues to Thrive, More Than a Year On - Good News Network

Bone Marrow Stem Cells Comments Off on Every Patient Treated With CRISPR Gene Therapy for Blood Diseases Continues to Thrive, More Than a Year On – Good News Network | December 19th, 2020

Dr. Faye Yin

Dr. Faye Yin

Melanoma is a serious and life-threatening form of cancer that begins in the skin but can spread rapidly if not treated early. We sat down with board-certified oncologist Dr. Faye Yin, an oncologist at Jersey City Medical Center, to learn more about this disease, its causes and risk factors, and why its important to protect yourself from excessive sun exposure even during the cold winter months.

What are the main risk factors for developing melanoma?

Ultraviolet, or UV, light exposure is the major risk factor. Melanoma is associated with both UVB rays, which are present in sunlight, and UVA rays, which are generated by tanning beds. Other risk factors include the presence of moles on the skin. Most are benign, but those with excessive moles should consult a dermatologist, especially if they observe any changes. Often, a mole will be removed as a precautionary measure. Age is also a risk factor; the older the person, the higher the risk. People with fair skin, freckles, and lighter hair are also more susceptible, which is why melanoma is more common in white and light-skinned people. Other risk factors include family history and the presence of a weakened immune system. Those with xeroderma pigmentosum, or XP, a rare genetic disorder, are particularly at risk because the condition affects the ability of skin cells to repair themselves after UV light exposure.

What should people do if they have any of these risk factors?

As with most risk factors impacting health, there are things you can change, and things you cannot. You cant change your skin color or family history, and you cant avoid getting older. But you can limit your exposure to UV rays. A popular catchphrase that I tell my patients, which has been promoted by the American Cancer Society, is Slip, Slop, Slap, and Wrap. Slip on a shirt, slop on some sunscreen, slap on a hat, and wrap on some sunglasses. I also recommend that people avoid using tanning beds and sun lamps. Teaching children about sun safety is especially important, because they tend to spend more time outdoors and can burn more easily. It is also important for people with risk factors to pay closer attention to their skin. Keep an eye out for abnormal moles or other skin features that appear to be unusual or changing over time, and consult a dermatologist if necessary.

Can sunlight still be dangerous during winter?

Yes whether youre skiing or just going for a walk, it is great to enjoy the sun and being outdoors in the winter, but its just as important to protect yourself from excess sun exposure in winter as it is in summer. Harmful ultraviolet rays are present year-round. They can even filter through dark cloud coverage to reach your skin, increasing your risk of melanoma. Some people may experience a bad sunburn on a winter vacation, especially if they ski in high altitudes, because UV rays are usually more intense in higher regions with a thinner atmosphere. When youre outside, any uncovered areas of your body are exposed to UV rays. So, its important to wear sunscreen even in the winter months.

Is smoking a risk factor for developing melanoma, and if so, is it mostly if youre currently smoking (for instance, what if you smoked for years and stopped?)

As an oncologist, every day I tell my patients: dont smoke! Smoking is a contributing factor for many cancers, and I believe that it also affects overall skin health; I can often look at someones skin and tell whether they smoke. That having been said, we dont have evidence that smoking directly contributes to melanoma. But I always encourage patients not to smoke to stay healthy and minimize their cancer risk.

Why does having a weakened immune system count as a risk factor for melanoma?

Having a weakened immune system increases the risk of melanoma and other cancers. I have worked with many patients whose immune systems have been compromised, either by illness or in some cases due to medical treatment for other conditions. For example, immunosuppressive drugs are used after stem cell and organ transplants, to prevent the body from rejecting the transplant. Certain diseases also compromise the immune system, such as HIV. A weakened immune system increases cancer risk for two reasons. First, because the body has less ability to detect and destroy cancer cells. And secondly, because the body is more susceptible to infections that may lead to cancer.

Is gender a risk factor? If so, do we know why?

In the United States, men typically have a higher rate of melanoma than women, though this varies by age. Before age 50, the risk is higher for women, and after age 50, the risk is higher in men. We believe that this discrepancy relates to the fact that men are likely to spend more time in the sun over the course of their lifetimes. I also think that women are more likely to wear sunscreen than men, so this may play a role. In addition, men tend to have thicker skin with less fat beneath it and more collagen, and some research shows that this can make the skin more susceptible to sunlight damage. Also, some studies have shown that estrogen, which is more prevalent in women, can increase resistance to melanoma.

Are older people at higher risk for melanoma?

The risk of melanoma increases as you age. The average age for a melanoma diagnosis is age 65. But melanoma is not uncommon even among those younger than age 30. In fact, it is one of the most common cancers in young adults, especially young women. Melanoma is also more common in younger people whose families have a history of melanoma.

How does having a family history of having melanoma impact someone?

Family history is definitely a melanoma risk factor; the risk is higher among those who have one or more first-degree relatives who have had melanoma. About 10 percent of people diagnosed with melanoma have a family history. Families tend to have shared lifestyle habits, such as more frequent sun exposure, and in addition they typically have similar skin types and share certain genetic characteristics. You cant change your skin color or your genes, but you can change some factors. If you know that you are higher risk, and have a family history, pay close attention to your skin. Avoid excessive sunlight and tanning beds, and consult a dermatologist if you have concerns.

Why is UV light exposure a risk factor?

Numerous studies have shown that sun and UV light exposure is a major melanoma risk factor, especially for children and teens. Research shows that early sun exposure can damage the DNA in skin cells. Melanocytes are the cells that produce melanin, which gives skin its pigmentation, and damaging these cells can start the path to melanoma. Melanoma commonly occurs on the thighs of women, and on the trunks of men, as well as on arms and faces, which are the areas that most often receive chronic sun exposure in young people. In addition to limiting UV light exposure, people should also examine their own skin at least monthly, especially if there are high risk factors. If you see something unusual, such as a large mole or a spot youre not sure about, I will often encourage patients to take a photograph of it. You might not notice small changes over time because you get accustomed to them. But if you take a picture of a spot on your skin and compare it a month or a few months later, and you see a change, you should see a dermatologist.

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The facts about the danger of melanoma - The Hudson Reporter

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Most treatment for heart failure can only slow it down or ease your symptoms. Soon, it may be possible to fix what causes it. Doctors are testing whether stem cells can repair or replace damaged heart cells.

Stem cells can grow into many different kinds of cells. You have them in organs and tissues all over your body. They divide to replace worn-out or damaged cells, and to become new stem cells.

In the lab, scientists have turned stem cells into ones that make up blood vessel walls and linings, and into actual beating heart cells. Now theyre trying to translate that into a treatment.

Scientists have zeroed in on a few specific kinds of cells that may be helpful:

Bone marrow mononuclear cells: A mixture of cells that comes from your own bone marrow.

Cardiac-derived stem cells: Ones found in heart tissue.

Mesenchymal stromal cells: They're usually taken from bone marrow, fat, or umbilical cord blood.

Proangiogenic progenitor cells: These are in blood and bone marrow.

It's not approved to treat heart failure, yet. You can get it through a clinical trial. Thats when the research moves from the lab to the hospital to see if a treatment is safe and if it works.

If you want to try stem cell therapy, ask your doctors if there are studies that may be a good fit for you. The National Library of Medicine has a website that helps you search for all kinds of clinical trials.

Not all experimental treatments are part of a clinical trial, so make sure you understand what youre signing up for. If its a legitimate study, you shouldnt have to pay for treatment or follow-ups.

Most people testing stem cell therapy for heart failure get one treatment. Then theyre checked every few months for a year or more.

Not everyone in a trial actually gets stem cells. Researchers need to compare the results of the new treatment against what happens with a group of people who dont get it.

Doctors are testing several different methods of giving people stem cells:

Intramyocardial injection: Cells go right into the heart muscle, usually during another procedure like open-heart surgery, bypass surgery, or implanting a pacemaker.

Intracoronary infusion: A catheter puts cells into your coronary artery. It goes into a large blood vessel in your groin and threaded through your heart.

Intravenously: Cells go right into the bloodstream through a needle placed in a vein.

With any of these methods, most stem cells leave the body quickly. Researchers are looking for better ways to make them stick. One possibility is growing them into a patch that goes directly to the damaged part of the heart.

Theres no way to fix heart damage that leads to heart failure. Stem cell therapy could change that. Still, its too early to call any treatment a success. The studies done so far have been too small. They've also used very different methods.

But it does look like stem cells could help repair heart tissue. In most studies, people who got them were less likely to die or go to the hospital during the study. Their hearts worked better and their quality of life was better than for people who didnt get them.

It isnt clear how stem cells help. Doctors hope clinical trials and research will help them discover that. They're also hoping to answer many other questions, including:

If you are interested in joining a trial, talk to your doctor.

SOURCES:

International Society for Stem Cell Research: Heart Disease, Types of Stem Cells, About Clinical Trials, Stem Cell Research: What to Ask.

National Institutes of Health: Mending a Broken Heart: Stem Cells and Cardiac Repair, Can Stem Cells Repair a Damaged Heart? Stem Cell Basics, NIH Clinical Research Trials and You.

Current Cardiology Reviews: Cellular Therapy for Heart Failure.

U.S. National Library of Medicine: ClinicalTrials.gov.

U.S. Food and Drug Administration: Consumer Information on Stem Cells.

Circulation Research: Cell Therapy for Heart Failure, Safety and Efficacy of the Intravenous Infusion of Umbilical Cord Mesenchymal Stem Cells in Patients With Heart Failure: A Phase 1/2 Randomized Controlled Trial (RIMECARD Trial).

The Lancet: Ixmyelocel-T for patients with ischaemic heart failure: a prospective randomised double-blind trial.

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Cardiac Stem Cells Comments Off on Stem Cell Therapy for Heart Failure Treatment | December 17th, 2020

TAMPA, Fla. While health officials and lawmakers continue trying to steer young people away from vaping, the wide variety of enticing flavors added to these products make that a tough task. Although most of the worry over vaping comes from the risk of addiction, lung damage, and threat of switching to conventional cigarettes, a new study finds the flavoring chemicals these products use may be just as harmful as anything else. Researchers from the University of South Florida Health say vaporized flavoring molecules are toxic to the heart and damage the organs ability to beat correctly.

While other studies find that vaping is generally less harmful than smoking traditional tobacco products, the nicotine and other chemicals in e-cigarettes still damages the heart and lungs. Until now however, researchers say the impact of flavoring additives inhaled into the bloodstream remained unclear.

The flavored electronic nicotine delivery systems widely popular among teens and young adults are not harm-free, says principal investigator Dr. Sami Noujaim in a university release. Altogether, our findings in the cells and mice indicate that vaping does interfere with the normal functioning of the heart and can potentially lead to cardiac rhythm disturbances.

Dr. Noujaims study is one of the first to investigate the cardiotoxic effects of flavoring chemicals added to the e-liquids in electronic nicotine delivery systems (ENDS). ENDS include a variety of different vaping products like vape pens, mods, and pods.

Researchers define vaping as inhaling aerosols (tiny droplets) which e-cigarettes create by heating liquid nicotine and solvents like propylene glycol and vegetable glycerin. A vaping devices battery-powered heater converts this liquid into a smoke-like mix, or vapor.

The study tested how three popular e-liquid flavors fruit, cinnamon, and vanilla custard affect cardiac muscle cells (HL-1) of mice. After being exposed to e-vapor in a lab dish, the results reveal all three flavors are toxic to HL-1 cells.

The USF team also examined what happens to cardiac cells grown from human stem cells that are exposed to three types of e-vapors. The first substance containing only solvents interfered with the cells electrical activity and beating rate. The second substance, containing both nicotine and solvents, proved to be even more toxic to the heart cells.

The third substance however, containing nicotine, solvents, and vanilla custard flavoring, caused the most damage to the heart and its ability to spontaneously beat correctly. Researchers also determined that vanilla custard flavoring is the most toxic of the varieties tested.

This experiment told us that the flavoring chemicals added to vaping devices can increase harm beyond what the nicotine alone can do, Dr. Noujaim says.

The study also tested flavored vapings impact on live mice. Researchers implanted each subject with a tiny electrocardiogram device before exposing them to 60 puffs of vanilla-flavored e-vapor five days a week for 10 weeks.

Study authors looked at how this exposure impacted heart rate variability (HRV), which is the change in time intervals between successive heartbeats. The results show that HRV decreased in vaping mice compared to those only exposed to puffs of clean air.

The USF team finds vaping interferes with normal HRV by disrupting the autonomic nervous system and its control over heart rate. Mice exposed to flavored vaping are also more prone to a dangerous heart rhythm problem called ventricular tachycardia.

Researchers say they still have to confirm these results in humans. Dr. Noujaim urges policymakers to continue looking at the growing evidence that vaping is not a particularly safer alternative to smoking.

Our research matters because regulation of the vaping industry is a work in progress, Dr. Noujaim explains. The FDA needs input from the scientific community about all the possible risks of vaping in order to effectively regulate electronic nicotine delivery systems and protect the publics health. At USF Health, in particular, we will continue to examine how vaping may adversely affect cardiac health.

The study appears in the American Journal of Physiology- Heart and Circulatory Physiology.

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Flavors added to vaping devices damage the heart, vanilla custard the most toxic of all - Study Finds

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Avery Therapeutics is projected to be one of the first companies in the US to seek approval for a clinical trial using iPSC-derived technology for heart failure. The goal of this collaboration is to develop a new off-the-shelf treatment to improve the quality of life of patients suffering from heart failure, a debilitating disease that affects tens of millions of people worldwide.

The iPSCs are manufactured at I Peace's state-of-the-art GMP facility in Kyoto, Japan, under comprehensive validation programs of the facility, equipment, and processes including donor recruiting, screening, blood draw, iPSC generation, storage, and distribution. I Peace has obtained a US-based independent institutional review board (IRB) approval for its process of donor sourcing for commercial-use iPSCs. The facility is designed to be PMDA and USFDA compliant.

As Avery Therapeutics expects to expand the application of its regenerative medicine technology to various types of heart diseases and beyond, iPSCs are the key enabling technology for quality and future scalability. This agreement provides a solid foundation to improve the welfare of those suffering from diseases through advancement of tissue-engineered therapeutics.

"We are thrilled to announce this collaboration with I Peace. It is a big step forward in the development of novel cell-based therapeutics for unmet medical needs. Through this collaboration, I Peace brings deep iPSC development and manufacturing expertise to enable Avery's proprietary MyCardia cell delivery platform technology. Together we hope to positively impact millions of patients worldwide in the near future," Said Jordan Lancaster, PhD, Avery Therapeutics' CEO.

This agreement reflects an innovative collaboration involving multiple locations internationally and marks a significant milestone for both I Peace, Inc. and Avery Therapeutics to pursue one of the first US clinical trials using iPSC technology in the area of heart diseases. Koji Tanabe, PhD, founder and CEO of I Peace stated: "By combining I Peace's proprietary clinical grade iPSC technology and Avery's tissue engineering technology, we can bring the regenerative medicine dream closer to reality. We are very excited by Avery's technology and look forward to continue working together."

About I Peace, Inc

I Peace, Inc. is a global supplier of clinical and research grade iPSCs. It was founded in 2015 in Palo Alto, California, USA by Dr. Tanabe, who earned his doctorate at Kyoto University under Nobel laureate Dr. Shinya Yamanaka. I Peace's mission is to alleviate the suffering of diseased patients and help healthy people maintain a high quality of life by making cell therapy accessible to all. I Peace's state-of-the-art GMP facility and proprietary manufacturing platform enables the fully-automated mass production of discrete iPSCs from multiple donors in a single room. Increasing the available number of clinical-grade iPSC lines allows I Peace customers to take differentiation propensity into account to select the most appropriate iPSC line for their clinical research at significantly reduced cost. I Peace aims to create iPSCs for every individual that become their stem cell for life.

Founder, CEO: Koji TanabeSince: 2015Head Quarter: Palo Alto, CaliforniaJapan subsidiary: I Peace, Ltd. (Kyoto, Japan)Cell Manufacturing Facility: Kyoto, JapanWeb: https://www.ipeace.com

About Avery Therapeutics

Avery Therapeutics is a company developing advanced therapies for patients suffering from cardiovascular diseases. Avery's lead candidate is an allogeneic tissue engineered cardiac graft, MyCardia in development for treatment of chronic heart failure. Using Avery's proprietary manufacturing process MyCardia can be manufactured at scale, cryopreserved, and shipped ready to use. Avery is leveraging its proprietary tissue platform to pursue other cardiovascular indications. For more information visit: AveryThera.com. Follow Avery Therapeutics on LinkedInand Twitter.Since: 2016Headquarter: Tucson, AZWebsite: https://www.AveryThera.com

SOURCE I Peace, Inc.

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I Peace, Inc. and Avery Therapeutics announce collaboration to bring iPSC derived cell therapy for heart failure to the clinic - PRNewswire

Cardiac Stem Cells Comments Off on I Peace, Inc. and Avery Therapeutics announce collaboration to bring iPSC derived cell therapy for heart failure to the clinic – PRNewswire | December 17th, 2020

Stem cell transplants from bone marrow or blood offer lifesaving treatment. They are also taxing physically and emotionally.

Its important to know:

What are stem cells?

Blood cells are short-lived and must be replaced. Blood-forming stem cells (hematopoietic cells)divide and multiply. Some mature into one of three blood cell types:

Stem cells are found in bone marrow, in the bloodstream and in umbilical cord blood. In the bloodstream, they are called peripheral blood stem cells (PBSCs). Stem cells from any of these sources can be used in transplants.

What are stem cell transplants?

With a stem cell transplant, a doctor gives you healthy replacementcells that help you fight infection and disease. Doctors most often use stem cell transplants to treat blood disorders and blood cancers that:

A transplant is like a blood transfusion. There are three types:

How stem cell transplants work

Youundergo a process called conditioning. Chemotherapy, radiation therapy or both are used todestroycancer cells and healthy cells that could keep your body from accepting transplanted cells.

New cells are added toyour bloodstream with an IV. The cellscollect in your bone marrow, where they produce new blood cells. Because conditioning leaves your immune system weak, you will need two to three weeks of monitoring.

Who gets a transplant?

At the OHSU Knight Cancer Institute, we consider every person with blood cancer for a stem cell transplant. It might be right for you if your cancer didnt respond to other treatment or if your cancer came back after treatment.

Our providers meet at weekly gatherings called tumor boards to develop the best treatment options for each patient.

Our team considers factors such as your:

Youll want to consider:

What disorders do transplants treat?

Bone marrow/stem cell transplants may be an option to treat:

Leukemias:

Lymphomas:

Other blood cancers and blood disorders:

Other conditions:

Types of stem cell transplants

There are two main types of transplants, each with risks and benefits. OHSU is the only place in Oregon that offers allogeneic transplants.

Autologous transplant

What is it?This type uses your own stem cells. This eliminates the risk of your body rejecting donor cells or of donor cells attacking your body. A relapse may be more likely, though, because you wont have healthy donated cells to attack any diseased cells that remain after conditioning.

How it works:Your care team collects bone marrow using a hollow needle or draws blood and uses a machine to separate out stem cells. The stem cells are frozen. After the conditioning process, the cells are transplanted using anIV drip.

Allogeneic transplant

What is it? We use cells from a donor. Sometimes your own cells are too diseased to collect and reuse. Donor cells are more aggressive in killing any diseased cells left after conditioning. The risk is that they may aggressively target your healthy cells as well, a complication called graft-versus-host disease.

How it works: After the conditioning process, we transplant healthy donor cells using anIV drip. The donor cells help your body rebuild your immune system. A donor can be a relative or someone else whose marrow matches yours.

Allogeneic transplants include:

Bone marrow donors

OHSU has participated in Be The Match: The National Marrow Donor Program since 1996. This program helps people find a lifesaving marrow or PBSC donor. Donors must meet medical guidelinesand should expect to spend 20 to 30 hours in treatment over four to six weeks.

Testing:To find the closest match, doctors will test your blood and a potential donors blood to find their human leukocyte antigen (HLA) type.

HLA markers:Everyone inherits a set of HLA markers from their parents. These markers, contained in almost all of your cells, tell your body which cells belong to you. The more matching markers you and a donor have, the better your chances of a successful transplant. OHSU has success with haploidentical transplants, however, in which as few as half the markers match.

Transplant risks

As with any procedure, transplants involve risks. Your care team will discuss these with you in detail.

Infection: Chemotherapy and radiation therapy weaken your immune system. You are at high risk of infection for up to six weeks until your new cells make healthy blood cells. Your care team will keep you in a safe environment with protection against airborne germs. You will receive safety instructions for going home.

Low platelets:Your platelets will be low for three or more weeks. We will take great care to help you avoid injury or bleeding. Some patients may need a blood transfusion to replace platelets.

Pain:Mouth or throat pain is a common side effect of chemotherapy and radiation therapy. It may cause difficulty eating or swallowing for a few weeks.

Graft failure: Transplant (graft) failure occurs when the body rejects the donor cells. This is rare with stem cell transplants but more common when HLA types are poorly matched.

Graft-versus-host disease:This happens when transplanted cells from the donor attack the recipients tissue and organs. This common complication can range from mild to life-threatening.

Organ damage:Chemotherapy and radiation can leave lasting damage. Well monitor you closely for signs of any problem.

Infertility:The chemotherapy and radiation therapy used before transplants typically result in infertility. OHSU fertility expertscan offer options to preserve your ability to have children before treatment begins.

Physical and emotional effects

Transplants are difficult. They require weeks in or near the hospital, away from work and regular activities. Our cancer social workerscan provide support to you and your family before, during and after treatment:

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Understanding Bone Marrow/Stem Cell Transplant | Knight ...

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NEW YORK, Dec. 17, 2020 /PRNewswire/ -- Physio Logic, a leading provider of integrated health services in New York City and surrounding areas, continues to demonstrate its commitment to excellence in the field of Regenerative Medicine and Stem Cell Therapy by entering into a collaboration with Regenexx, a worldwide network of specially trained physicians providing the world's most advanced, research-driven, regenerative medicine and stem cell therapy treatments. The partnership brings cutting-edge regenerative treatments to New York City residents suffering from sports injuries or degenerative diseases.

The Regenerative Medicine division of Physio Logic is led by Dr. Tanuj Palvia, MD, a specialist in regenerative medicine and interventional orthopedics focused on the treatment of musculoskeletal injuries and degenerative orthopedic conditions.

"Stem Cell Therapy is one of the most innovative treatments available today but, being so new, patients need to know they're receiving the best possible care. As a physician, I hold myself and my practice to the highest standards and, being aligned with Regenexx adds that extra assurance patients need to know they're in good hands. Whether it's a nagging sports injury or slow degeneration, you're going to get the highest quality of integrated care right here at Physio Logic," said Dr. Palvia.

Interventional Orthobiologics is a specialty that focuses on using your body's natural healing agents to treat orthopedic injuries with the goal of reducing pain and improving joint function. The variety of orthobiologics available to Regenexx physicians, such as bone marrow stem cells and platelet-rich plasma (PRP), allow them to create a treatment plan to best support your recovery. It can be used in the treatment of conditions such as arthritis and injury to ligaments, tendons, cartilage, or bone.

"Being selected to represent the Regenexx brand in New York City speaks to the quality of our facility, our providers, and the care we give our patients," said Dr. Rudy Gehrman, CEO & Founder of the Brooklyn based clinic. "Physio Logic is raising the standard of healthcare in New York and our partnership with Regenexx is an extension of the quality, integrative care we provide to every patient that walks through our door."

Regenexx physicians are required to have thousands of hours of experience performing precise, injection-based treatments using image guidance for a range of body parts and injuries. Their strict acceptance criteria means that Regenexx only chooses the most qualified physicians to join their network. Physio Logic's Interventional Pain Specialist, Dr. Tanuj Palvia, MD, is ranked among them.

To learn more about Physio Logic and Regenerative Medicine, go to https://physiologicnyc.com/regenerative-medicine/

About Physio LogicPhysio Logic brings together an expert team of open-minded medical doctors, physical therapists, chiropractors, acupuncturists, massage therapists, nutritionists, health coaches, biohackers, and Pilates instructors. Our unique collaborative approach, coupled with our ability to assess patients holistically, is used to create a custom care plan tailored to patients' needs. For more information on Physio Logic, visit https://physiologicnyc.com or call (718) 260-1000.

About RegenexxRegenexx is a nationwide network of physicians who practice Interventional Orthopedics, a new specialty that focuses on using the most advanced regenerative protocols available as an alternative to many orthopedic surgeries. Regenexx has published roughly half of the research worldwide on the use of orthobiologics for treating orthopedic injuries, and our patented treatment lab-processing and treatment protocols allow us to achieve unmatched results. Our procedures use your body's natural healing agents including blood platelets and bone marrow concentrate to repair damaged bone, muscle, cartilage, tendons and ligaments. For more information on Regenexx, visit https://regenexx.com.

Media contact:Alan Sott[emailprotected](718) 260-1000

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Physio Logic Brings Cutting-edge Regenerative Treatments for Sport Injuries and Arthritis to New York City - PRNewswire

Bone Marrow Stem Cells Comments Off on Physio Logic Brings Cutting-edge Regenerative Treatments for Sport Injuries and Arthritis to New York City – PRNewswire | December 17th, 2020

PHILADELPHIA, Dec. 17, 2020 /PRNewswire/ --Immunotherapies, such as checkpoint inhibitor drugs, have made worlds of difference for the treatment of cancer. Most clinicians and scientists understand these drugs act on what's known as the adaptive immune system, the T cells and B cells that respond to specific threats to the body.

New research from a team co-led by Penn Dental Medicine's George Hajishengallis suggests that the innate immune system, which responds more generally to bodily invaders, may be an important yet overlooked component of immunotherapy's success.

Their work, published in the journal Cell, found that "training" the innate immune system with -glucan, a compound derived from fungus, inspired the production of innate immune cells, specifically neutrophils, that were programmed to prevent or attack tumors in an animal model.

"The focus in immunotherapy is placed on adaptive immunity, like checkpoint inhibitors inhibit the interaction between cancer cells and T cells," says Hajishengallis. "The innate immune cells, or myeloid cells, have not been considered so important. Yet our work suggests the myeloid cells can play a critical role in regulating tumor behavior."

The current study builds on earlier work by Hajishengallis and a multi-institutional team of collaborators, which showed that trained immunity, elicited through exposure to the fungus-derived compound -glucan, could improve immune recovery after chemotherapy in a mouse model.

In that previous study, the researchers also showed that the "memory" of the innate immune system was held within the bone marrow, in hematopoietic stem cells that serve as precursors of myeloid cells, such as neutrophils, monocytes, and macrophages.

The team next wanted to get at the details of the mechanism by which this memory was encoded. "The fact that -glucan helps you fight tumors doesn't necessarily mean it was through trained immunity," says Hajishengallis.

To confirm that link, the researchers isolated neutrophils from mice that had received the innate immune training via exposure to -glucan and transferred them, along with cells that grow into melanoma tumors, to mice that had not received -glucan. Tumor growth was significantly dampened in animals that received cells from mice that had been trained.

-glucan is already in clinical trials for cancer immunotherapy, but the researchers say this finding suggests a novel mechanism of action with new treatment approaches.

"This is a breakthrough concept that can be therapeutically exploited for cancer immunotherapy in humans," Hajishengallis says, "specifically by transferring neutrophils from -glucan-trained donors to cancer patients who would be recipients."

Contact: Beth Adams, [emailprotected]

SOURCE Penn Dental Medicine

http://www.dental.upenn.edu

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Priming the Immune System to Fight Cancer - PRNewswire

Bone Marrow Stem Cells Comments Off on Priming the Immune System to Fight Cancer – PRNewswire | December 17th, 2020

The stock price of Creative Medical Technology Holdings Inc (OTCMKTS: CELZ) a company that engages in stem cell research and developing applications to treat male sexual dysfunction and related issues increased by 80.77% yesterday as it went from $0.0026 to $0.0047 per share. One of the biggest triggers for the stock price increase is an announcement about the company announcing the successful application of ImmCelz immunotherapy for treatment of stroke.

In an animal model of ischemia stroke, the middle cerebral artery ligation model, administration of ImmCelz resulted in 34% reduction in infarct volume, whereas control bone marrow mesenchymal stem cells reduced infarct volume by 21%. And there were improvements in functional recovery were observed using the Rotarod test.

At 28 days after induction of stroke the animals receiving ImmCelz had superior running time (92% of non-stroke controls) compared to animals that received bone marrow mesenchymal stem cells (73% of non-stroke control). And animals that received saline had a running time that was 50% of non-stroke controls.

KEY QUOTES:

The regenerative potential of immune cells that have been programmed by stem cells is a fascinating and novel area of research. Conceptual advantages of using reprogrammed T cells include higher migratory ability due to smaller size, as well as ability to replicate and potentially formregenerative memory cells.

Dr.Amit Patel, coinventor of ImmCelz

This data, which is covered by our previous filed patents, such as no. 15/987739,Generation of autologous immune modulatory cells for treatment of neurological conditions, demonstrate that immune modulation via this stem cell based method may be a novel and superior way of addressing the$30 billion dollarmarket for stroke therapeutics. The fact that this technology, which has priority back to 2017, is demonstrating such stunning results, motivates us to consider filing an Investigational New Drug Application for use in stroke.

Dr.Thomas Ichim, coinventor of the patent and Chief Scientific Officer of Creative Medical Technology

While stroke historically has been a major area of unmet medical need, the rise in stroke cases , as well as the fact that younger people are increasingly falling victim to stroke, strongly motivates us to accelerate our developmental programs and to continue to explore participation of Big Pharma in this space. We are eager to replicate the existing experiments start compiling the dossier needed to take ImmCelz into humans using the Investigational New Drug Application (IND) route through the FDA.

Timothy Warbington, President and CEO of Creative Medical Technology

Disclaimer: This content is intended for informational purposes. Before making any investment, you should do your own analysis.

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Creative Medical Technology Stock Price Increased 80.77%: Why It Happened - Pulse 2.0

Bone Marrow Stem Cells Comments Off on Creative Medical Technology Stock Price Increased 80.77%: Why It Happened – Pulse 2.0 | December 17th, 2020

Today, we take an in-depth look at an intriguing development concern with one product approved and on the market and more importantly a more lucrative candidate that appears on its way to approval in Europe. A full analysis on this Busted IPO follows in the paragraphs below.

Orchard Therapeutics (ORTX) is a London, United Kingdom-based biopharmaceutical company that IPO'd in 2018. The company is focused on developing gene therapies for rare conditions. In 2018, Orchard acquired GSK's rare disease gene therapy portfolio, which originated from a collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy. The company's unique approach involves inserting a working copy of the missing or malfunctioning gene into a patient's own blood stem cells. This approach circumvents the need for a bone marrow transplant since it leverages blood stem cells intrinsic capacity to self-renew in a patient's bone marrow and produce new blood cells of all types. The company's overarching goal is to permanently correct genetic disorders via a single treatment.

The company does have one approved product called Strimvelis, which is indicated for patients with severe combined immunodeficiency due to adenosine deaminase deficiency for whom no suitable human leukocyte antigen matched related stem cell donor is available; however, the drug has only been approved by the EMA and not the FDA. The company's pipeline is candidate rich, spanning a variety of indications that are compartmentalized into three categories: neurometabolic/neurodegenerative disorders, immunological disorders, and blood disorders. The company has a couple of late-stage candidates. Orchard Therapeutics has a market capitalization of roughly $450 million and trades for approximately $4.50 a share.

Pipeline

Source: Company Presentation

OTL-200

OTL-200 is an ex vivo autologous gene therapy in development to treat metachromatic leukodystrophy. The drug uses a modified virus to insert an operational copy of the ARSA gene into a patient's cells. OTL-200 has received rare pediatric disease designation from the FDA. MLD is a rare and deadly inherited disease. The disease is characterized by the accumulation of fats called sulfatides, which causes a breakdown in the protective fatty layer surrounding nerves in the central and peripheral nerve systems. It is estimated that 1 in 40,000 to 1 in 160,000 people have the disease worldwide. OTL-200 was developed in partnership with the San Raffaele Telethon Institute for Gene Therapy.

Source: Company Presentation

On October 16th, the company announced that it received a positive CHMP opinion for the drug, which recommended full marketing authorization for the treatment of early-onset metachromatic leukodystrophy patients in the European Union. The positive opinion will now be reviewed by the European Commission. A final decision is expected by the end of 2020. If approved, the company would be targeting a launch in the first half of 2021.

Furthermore, the company is pursuing a regenerative medicine advanced therapy designation, and it filed an investigational new drug application or IND in the U.S., which was accepted by the FDA on November 19th. Orchard has also applied for Regenerative Medicine Advanced Therapy designation for OTL-200 to help facilitate additional dialogue with the FDA

In addition, within the neurometabolic/neurodegenerative disorders category, it was announced in September that the European Medicines Agency has granted Priority Medicines designation to OTL-203 for the treatment of mucopolysaccharidosis type I. This comes not long after 203 received Orphan Drug designation in the U.S.

Source: Company Presentation

OTL-103

OTL-103 is an ex vivo autologous gene therapy in development to treat Wiskott-Aldrich syndrome. The drug uses a modified virus to insert a working copy of the WAS gene into a patient's cells. WAS is a rare, X-linked, recessive, inherited immune disorder, which is characterized by reoccurring severe infections, autoimmunity, eczema and severe bleeding episodes. The company has received Rare Pediatric Disease designation and Regenerative Medicine Advanced Therapy designation from the FDA. OTL-103 is being developed in partnership with the San Raffaele Telethon Institute for Gene Therapy.

Looking ahead, the company is preparing to file a BLA in the U.S. and an MAA in the EU for OTL-103 in WAS in 2021.

Source: Company Presentation

As of September 30th, 2020, Orchard Therapeutics had cash and cash equivalents of $41.1 million compared to $19 million on December 31st, 2019. Research and development expenses for the third quarter were $14.6 million compared to $28.4 million in Q3 of 2019. Selling, general and administrative expenses were $12.9 million in the quarter compared to $14.2 million in the same quarter of 2019. The company did $1.9 million in product revenue for the quarter compared to $1.9 million in Q3 of 2019. Overall, the company reported a net loss of $20.2 million compared to a net loss of $36.7 million in Q3 of 2019. Management stated in the latest quarterly update that it expects its current financial position to cover its anticipated operating and capital expenditure requirements into 2022.

The company is sparsely covered in the United States despite a healthy market cap as our most names in this space domiciled overseas. The most recent recommendation comes from Oppenheimer on September 14th. The firm lowered its price target from $26 a share to $16 a share, but it maintained its overweight rating. The analyst stated that the updated price target reflects a more subdued opinion on the pace of pipeline development for MPS-1 and MPS-IIIA. It reiterated this rating on November 19th.

Both Barclays ($13 price target, down from $15 previously) and Goldman Sachs ($9 price target, down from $13) reiterated Buy ratings on ORTX albeit revising the price targets lower. Finally, on May 22nd, JPMorgan lowered its price target from $26 a share to $17 a share and maintained its overweight rating. The analyst's note did seem upbeat despite the lowered price target in that the analyst thinks that the company's pipeline possesses "broad potential".

Two things prevent Orchard from being considered for a large holding. First, the company looks like it will have to raise additional capital in the very near future. I think the company will raise capital immediately after the next positive news event. Second, overseas biotech concerns don't ever seem to get the attention from analysts or valuation from investors that companies domiciled in the States do. That said, the company has multiple shots on goal and definable potential catalysts on the near-term potential. Add in the possible wildcard of being a potential buyout target at some point, and ORTX would seem worthy of a small "watch item" stake at this time.

Bret Jensen is the Founder of and authors articles for the Biotech Forum, Busted IPO Forum, and Insiders Forum.

Author's note: I present and update my best small-cap Busted IPO stock ideas only to subscribers of my exclusive marketplace, The Busted IPO Forum. Our model portfolio has crushed the return of the Russell 2000 since its launch in the summer of 2017. To join the Busted IPO Forum community, just click on the logo below.

Disclosure: I am/we are long ORTX. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Originally posted here:
Our View On Orchard Therapeutics - Seeking Alpha

Bone Marrow Stem Cells Comments Off on Our View On Orchard Therapeutics – Seeking Alpha | December 17th, 2020

Osteonecrosis of the femoral head (ONFH) is a refractory disease characterized by compromised subchondral microcirculation, bone necrosis, and microfracture accumulation without sustained compensatory remodeling.1 Its complex etiology, variability in location (lateral, medial, or central), intra-bone edema, and inflammation add to the unpredictable prognosis. Although few patients regress spontaneously, the progressive nature and lack of curative treatment for ONFH thus far are the challenges faced in the management of ONFH.

Osteonecrosis of the femoral head typically affects relatively young, active individuals between 20 and 40 years old and follows an unrelenting course resulting in substantial loss of function.2 The Indian Society of Hip and Knee Surgeons' Registry stated that 49% of total hip arthroplasty procedures in India are due to an irreversible stage of ONFH.3 Osteonecrosis of the femoral head is idiopathic in most cases. Steroid and alcohol consumption are the second most common causes.4 The term silent hip refers to an asymptomatic hip in patients with ONFH of the contralateral hip and is at risk of developing ONFH.5

Hip and groin pain and limp when patients walk are primary indicators. Radiography, magnetic resonance imaging (MRI), and computed tomography are tools for diagnosis, prognosis, and decision-making regarding treatment of ONHF. Crescent formation, collapse and anterolateral sequestration on radiographs, and a double line presentation on T2-weighted MRI provide confirmation of ONFH diagnosis.

The Ficat and Arlet staging of ONFH from I to IV indicates the progressive involvement and progression of the femoral head toward arthritis.6 However, it does not allow prediction of the possibility of progression. Ficat and Arlet stage I with extensive involvement of the femoral head will have a high chance of further progression to collapse. Steinberg grades of ONFH allow prediction of the possibility of progression to collapse in a precollapse hip.7 The Association Research Circulation Osseous (ARCO) takes into consideration the location of the crescent, amount of cartilage depression, and the area and volume of the femoral head affected as reliable predictors of prognosis in early stage ONFH and is helpful for identifying a femoral head at risk of progression and collapse.8

The most common surgical intervention in early stage ONFH is core decompression.9 However, core decompression is notable for its lack of effectivity in preventing collapse in cases where progression is most likely to happen (ie, in cases where there is extensive involvement [more than 30%] of the anterolateral region of the femoral head and crescent sign).10 Among other surgical interventions, fibular graft (vascularized or nonvascularized) proximal femoral osteotomy has been described.11

Hernigou and Beaujean12 reported abnormalities in the mesenchymal stem cell pool, which is known for its regenerative potential, following insult to the affected hip. Gangji et al13 later reported qualitative and quantitative abnormalities of osteoblast cells within the proximal femur in ONFH patients. Thus, it is accepted that the regenerative and reparative capacity of bone in ONFH is severely compromised. However, more than two decades of experience using various orthobiologics has not been convincingly satisfying, and many groups have expressed limitations of these therapies.1416

The pathology of ONFH involves ischemic imbalance of bone remodeling due to relatively enhanced osteoclastic action and poor regenerative potential of osteogenic cells in the proximal femur. The supply of differentiated osteogenic cells (osteoblasts) over time would result in arrest of ONFH progression. Core decompression would allow revascularization, and debridement of necrotic bone decreases the time needed for creeping substitution of new bone over dead bone. With this theoretical conviction, the author planned to use and assess the efficacy of autologous bone marrowderived cultured osteoblasts following core decompression and debridement in the treatment of patients diagnosed with ARCO stages II and III ONFH.

The surgeries were conducted at various hospitals. Fifteen patients (13 male and 2 female), with a mean age of 32 years (range, 2161 years), presented with typical ONFH symptoms. Patients were diagnosed with ARCO stage II or III ONFH (9 bilateral and 6 unilateral, for a total of 24 hips) on radiograph and MRI, and were considered for a predesigned treatment protocol that involved implantation of autologous cultured osteoblasts following core decompression and debridement.

Patient consent for inclusion in the study was obtained. The types of ONFH were idiopathic (8 patients), corticosteroid-induced (6 patients), and traumatic (1 patient) (Table 1). Efficacy of the treatment was assessed based on changes on radiograph and MRI and modified Harris Hip Score (mHHS), Oxford Hip Score (OHS), and visual analog scale (VAS) score after treatment. In a few patients, computed tomography also was performed.

Table 1:

Patient Characteristics

Treatment was performed in 2 steps.

Step 1. Percutaneous bone marrow aspiration from the iliac crest was performed and collected in transport media containing anticoagulant. This was transported under temperature-monitored conditions and processed at a good manufacturing practicecertified cell processing facility to obtain a predefined osteoblast culture (4 to 5 weeks).

The ex vivo culture of osteoblasts using bone marrow from the patient involved isolation of osteoprogenitor cells, osteogenic differentiation, and then expansion. Immunophenotypic characterization was performed to ensure the cultured cells tested positive for CD44+ and CD151+ markers. Alizarin red stain test ensured the presence of calcium deposits within the osteoblasts. Alkaline phosphatase test was used as an indicator of ability to form type I collagen.

Thus, ex vivo cultured live osteoblast cells, not less than 45 million, were filled and packed in sterile vials with appropriate transport/culture medium and were made available for individualized treatment. The cell viability was ensured during transport as well as after implant until the cells were integrated.

Step 2. The surgical implantation was planned as per the availability of cultured and expanded osteoblasts (4 to 5 weeks). In the first 3 patients, the osteoblast implant was performed soon after core decompression, whereas for the remaining 12 patients, core decompression was followed by debridement with implantation.

The location of the necrotic zone and its size was approximated on MRI. The patient was placed on a fracture table, and the C-arm was positioned as for routine core decompression. The entry point of the guidewire (2.5 mm) was chosen around the vastus ridge to allow faster healing in the cancellous bone (Figure 1).

Figure 1:

Surgical process details. The arrow indicates the high entry point of the guidewire at the vastus ridge targeting the area of osteonecrosis. A, 1-cm distance from the superior cortex to prevent fracture. B, varus appearance of the proximal femur due to mild flattening of the femoral head in the anterolateral femur in early osteonecrosis of the femoral head. This is the earliest sign observed radiographically and is indicative of stage IIB.

The larger sagittal dimensions of the trochanteric area allowed for a posterior entry point to avoid a possible subtrochanteric fracture due to posterior cortical breaching during or after intervention. Special effort was made to avoid a subtrochanteric entry point. On no occasion was the posterior cortex of the femur violated. The guidewire was passed in the center of the lesion but at least 1 cm from the superior cortex. An 8-mm cannulated core drill (from the dynamic hip screw set) was used over the wire to make a tunnel until the necrotic zone. The steps of the surgical intervention are shown in Figure 2.

Figure 2:

Surgical steps. Anteroposterior C-arm image of the hip with guidewire (A). Lateral C-arm image of the hip with guidewire (B). Drilling with 8-mm dynamic hip screw core drill bit (C). Anteroposterior C-arm image during curettage (D).

The tip of the drill, when removed, showed necrotic bone (Figure 3A), which was later sent for histopathology. Bone curettes of various sizes and angles then were used to curette the sequestrum under imaging guidance. The end point of curettage was the removal of hard sclerotic bone from the femoral head. If there was a bony ridge that was difficult to curette, a reamer was used. The author attempted to leave 1 cm of subchondral bone intact to allow faster revascularization of the femoral head by removing dead sclerotic bone. Curettes were kept at least 1 cm from the joint line.

Figure 3:

Drill bit with debrided live and dead bone (A). Instruments used during surgery (B).

After curettage was complete, the tunnel was plugged using an allograft of appropriate size. All of the instruments used during surgery are shown in Figure 3B. At this point, the patient was tilted to attain a gravity-dependent position of the operative hip to avoid any backflow of the implanted cells. A spinal needle was inserted through the small hole made in the allograft plug, and the osteoblast cell gel mixture was slowly injected in the space within the femoral head. Patients were held in the same position for approximately 10 minutes to allow the cells to settle without spilling.

Postoperatively, patients were partial weight bearing for 4 weeks using a walker. They progressed to using a walking stick by week 6, and then full weight bearing was permitted by week 8. For patients treated for bilateral ONFH, use of a walker was encouraged until week 6. Physical exercises to regain muscle strength and all hip joint movements were encouraged as soon as possible.

All patients underwent regular follow-up during the rehabilitation period and thereafter at 6, 12, and 18 months, with all anteroposterior and lateral radiographs of the hip and magnetic resonance imaging completed at 18 months. Two patients were lost to follow-up thereafter, and 13 patients continued with regular follow-up visits; 3 patients had follow-up of 7 years.

At 18 months after implant, no disease progression was observed on radiographs and MRIs for all patients. Postoperative mHHS, OHS, and VAS scores improved, and all of the patients had resumed normal routine activities and daily chores. Analysis of variance for HHS, OHS, and VAS scores showed a statistically significant difference (individual as well as mean values) from baseline to 18 months after implantation (P<.5; Table 2). Three patients who underwent follow-up for 7 years after implantation were assessed via telephone for HHS and VAS scores. For 1 of these patients, HHS improved from 90 to 95, and VAS score improved from 3 to 1 at 18 months. For another patient, HHS improved from 85 to 95, and VAS score improved from 2 to 1 at 18 months. One of the patients who underwent follow-up for 7 years walked daily for 3 to 4 km.

Table 2:

Pain and Function-Related Scores

One male patient who was treated for bilateral ONFH with follow-up of 5 years showed good improvement in HHS (from 65 to 92.5) at the end of 18 months, and his VAS score improved from 9 at baseline to 3 in both hips at 18 months after treatment. At 5 years postoperatively, he reported pain only after sitting for several hours and was more comfortable using a cane when walking.

Another male patient was diagnosed with ARCO stage III of the right hip. He had extensive involvement of the central and lateral lesion (>50%) with crescent depression less than 2 mm. Although reports for direct comparison were not available at 6 years after treatment, radiographs showed no further progression, with intramedullary changes evident. The joint space was preserved, which is consistent with good clinical function (Figure 4).

Figure 4:

Patient M4. Preoperative magnetic resonance image of Association Research Circulation Osseous stage III of the right hip. Extensive involvement of the central and lateral regions (>50%), with the crescent having less than 2 mm of depression (A, B). Magnetic resonance image at 5 months after treatment (C). Preoperative anteroposterior radiograph (D). Anteroposterior radiograph at 6 years after treatment showing no further progression, with evident intramedullary changes. The joint space is preserved, which is consistent with good clinical function (E).

One female patient had a history of tuberculosis treated with anti-Koch therapyanti-tubercular therapy and corticosteroids for 9 months as the standard care. This patient presented with extensive bilateral femoral head involvement evident on radiograph and computed tomography scan. The crescent depression was 2 to 4 mm. She was diagnosed with ARCO stage II of the right hip and grade III of the left hip. Radiographs at 6 years postoperatively showed arrest of osteonecrosis progression with an otherwise high risk of collapse because the ONFH was steroid induced. Clinically, this patient was able to resume all of her routine activities, including a daily commute to work and regular yoga, floor exercises, and stationary cycling (Figure 5).

Figure 5:

Patient F1. Preoperative anteroposterior radiographs (A, B). Preoperative computed tomography scans. There is extensive bilateral femoral head involvement (>30%), with 2 to 4 mm of depression of the crescent (C, D). Anteroposterior radiographs 6 months after treatment (E, F). Anteroposterior radiograph 6 years after treatment. Both femoral heads show arrest of osteonecrosis progression in a patient at high risk for collapse (G).

One male patient who was receiving long-term steroid treatment had relatively moderate improvement in HHS, from a baseline of 65 to 80 at 18 months after treatment. A female patient with bilateral ONFH had ARCO stage III in the right hip and a small, centrally located lesion (<30%) in the left hip. On radiograph and MRI, the right hip showed more than 90% involvement of the lateral, central, and medial regions but no crescent. The decision was made not to treat the left hip because it was deemed to have minimum possibility of progression. At 6 years after treatment, there was regression of necrosis. The patient has done well clinically and had a successful childbirth (Figure 6).

Figure 6:

Patient F2. Preoperative anteroposterior radiographs of the right hip showing more than 90% involvement of the medial, central, and lateral regions. There is no crescent (A, B). Preoperative magnetic resonance images (C, D). Anteroposterior radiograph at 3 months after treatment (E). Anteroposterior radiograph at 6 years after treatment (F).

Overall, the short-term and long-term results of autologous cultured osteoblast treatment along with routine procedures have been satisfactory. None of the 8 patients who underwent follow-up for 5 to 7 years showed any signs of disease progression, and none of the patients required repeat treatment or total arthroplasty.

Among invasive procedures, core decompression has been the standard of care for early stages of ONFH; however, varying degrees of improvement have been reported. Yoon et al17 and Rajagopal et al18 reported treatment of ONFH with core decompression was viable only in early stages, with the effect lasting for 2 to 3 years.

Among the biologics, platelet-rich plasma, growth factors, and bone marrow aspirate concentrate (BMAC) have been widely used along with conventional techniques such as core decompression or bone grafts.19,20 Several contributions in terms of understanding the clinical application and efficacy of biologics for the treatment of ONFH have been published during the past two decades.2123 Inherent limitations such as the absence of controlled studies, uncertainty, and heterogenicity of the composition of biologics have resulted in inconsistent results, and no treatment option has passed the regulatory approval process.24

In a recent study, Hauzeur et al25 reported obvious inefficacy of BMAC treatment in a randomized clinical trial comparing BMAC and core decompression vs core decompression alone. Their assessment criteria included clinical outcome, pain score, radiology, and the need for total hip arthroplasty.

Untreated bone marrow should be considered first-generation and processed bone marrow second-generation biological treatments for ONFH. The results using first- and second-generation biologics have been variable, and there are no long-term data and no formally approved products. Thus, curative treatment of ONFH, at least prior to the collapse stage, remains challenging.26

Kim et al27 were the first to report the clinical use of cultured osteoblasts in a single patient with bilateral ONFH (Ficat Arlet grade II); they reported a good outcome at 5 years without progression of disease. Later, Gangji et al28 compared the use of BMAC and autologous osteoblast cells in the treatment of avascular necrosis. They reported the group treated with osteoblast cells had twofold higher respondents at 36 months compared with the BMAC-treated group. These patients continued to have reduced pain until the end of the study period. Also, progression of disease from stage III to IV was more than 2 times higher in the BMAC-treated group compared with the osteoblast-treated group.28

The author proposes the evolution of biologics being used as first- and second-generation treatment, and the current modality of using autologous cultured osteoblasts as the latest and third-generation treatment. As such, the latter is the only modality that qualifies as cell-based therapy (Table 3).

Table 3:

Proposed Generations of Orthobiologics

Autologous cultured osteoblast implant is the most novel treatment modality for joint preservation. In the author's experience, 11 patients at 4 years, 6 patients at 5 years, and 3 patients at 7 years after transplant showed arrest of disease. Joint structure, biomechanics, strength, and function were regained in these patients, and they required no repeat treatment. Yet, unlike few other treatments, total arthroplasty still remains viable as a future option.

The assessment of ONFH progression on MRI after core decompression remains a sparsely studied subject. Therefore, radiographic and clinical examination during follow-up is crucial.

Autologous cultured osteoblast implantation is effective and safe for patients with ARCO stages II and III ONFH. This third-generation biologic can be considered a joint-preserving treatment in correctly chosen patients.

Patient Characteristics

Pain and Function-Related Scores

Proposed Generations of Orthobiologics

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Hip Preservation With Autologous Osteoblast Cell-Based Treatment in Osteonecrosis of the Femoral Head - Healio

Bone Marrow Stem Cells Comments Off on Hip Preservation With Autologous Osteoblast Cell-Based Treatment in Osteonecrosis of the Femoral Head – Healio | December 17th, 2020

Regeneron: Preventing Infection Among Households

Regeneron: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Assessing the Efficacy and Safety of Anti-Spike SARS-CoV-2 Monoclonal Antibodies in Preventing SARS-Cov-2 Infection in Household Contacts of Individuals Infected with SARS-CoV-2

In this multisite trial, researchers are working to determine if monoclonal antibodies made by the drug company Regeneron Pharmaceuticals can prevent COVID-19 infection among people who have been exposed by someone in their household, but have not yet developed the disease. The trial is testing the same antibody cocktail given to President Donald Trump when he was hospitalized with COVID-19, though with a different use.

In this case, the antibodies are intended to prevent people from getting sick if they have a household member with COVID, Enfield said. So far, UVA has done a good job with recruitment, which is particularly tricky in this case as you have to find people who have been exposed to COVID in their household, but who do not yet have COVID.

UVA is recruiting 40 participants for the study, each of whom will receive four injections of either the antibodies or a placebo. Participants must have been exposed to COVID-19 by someone in their household within the previous 96 hours and continue to live with that person for a month.

Its been a rapid process, and a testament to the multidisciplinary team involved, from infectious disease clinicians and researchers to cell therapy, pulmonary critical care and several other departments, Sturek said. Its been all-hands-on-deck.

As results from these and other clinical trials continue to come in, Sturek also expressed hope that we will see widespread and effective vaccine distribution sooner, rather than later.

There is a lot on the horizon, from news around vaccines to getting the first wave of vaccines to high-risk people like health care workers, he said. Every day we learn something new, and its important to stay humble, to be able to adapt and change on the fly.

Fighting this pandemic has been a huge, multidisciplinary effort, and so many people joined in to help contribute and bring new treatments to our patients, and bring new research to the field. That doesnt get done without a huge team of nurses, clinical research coordinators, pharmacists, respiratory therapists and many, many others. It is impossible to overstate the importance of all of that teamwork.

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Research That Saves Lives: Four COVID-19 Therapies Being Tested at UVA - University of Virginia

Bone Marrow Stem Cells Comments Off on Research That Saves Lives: Four COVID-19 Therapies Being Tested at UVA – University of Virginia | December 17th, 2020

Autologous hematopoietic stem cell transplant (AHSCT) induces a reduction in relapse rate and physical disability in patients with relapsing-remitting multiple sclerosis (RRMS) who respond inadequately to other treatments, a small study suggests.

The study, Selective cognitive dysfunction and physical disability improvement after autologous hematopoietic stem cell transplantation in highly active multiple sclerosis, was published in the journal Nature Scientific Reports.

AHSCT is an experimental approach to treat multiple sclerosis (MS) that is meant to rebuild a patients immune system in order to stop attacks on the brain and spinal cord.

The procedure begins with collecting a patients own (meaning autologous) healthy hematopoietic stem cells immature cells that can develop into all types of blood cells from the bone marrow. These cells are put back into the patient after a fairly non-aggressive combination of chemotherapy is given to kill the patients immune cells.

A team of researchers at the Vilnius University, in Lithuania, evaluated the effectiveness and safety of the AHSCT procedure in 24 patients (18 female, mean age 37.8 years) with highly active RRMS (mean disease duration of 8.6 years) who failed to respond to conventional therapies.

The aim of the study was to assess cognitive dysfunction and physical disability after AHSCT, to explore the potential factors influencing disability regression after the transplant, and to estimate the safety of low-dose immunosuppressive therapy in highly active relapsing MS patients.

Researchers assessed participants disability and cognition through changes in several functional measures, including the expanded disability status scale (EDSS) and the Brief International Cognitive Assessment for MS, which includes three cognitive domains measured by the symbol digit modalities test, brief visuospatial memory test revised, and California verbal learning test second edition.

Of the 24 patients, 13 (54.2%) completed a 24-month follow-up and were included in the efficacy analysis of AHSCT. From those, two (15.4%) had one relapse during the first year after AHSCT and three patients (23.1%) had one relapse during the second year after AHSCT.

The annualized relapse rate (ARR) was 2.7 one year before AHSCT and 1.9 at two years before AHSCT. After the AHSCT procedure, ARR dropped to 0.2 in the first year and to 0.3 in the second year. This represented an 89% reduction in ARR, when comparing the values at two years after AHSCT with those at two years before AHSCT.

The researchers also noted a reduction in disability progression (as measured by EDSS scores), with 84.6% of patients improving their disability score after AHSCT at month six and 76.9% at one year. Additionally, 76.9% of patients showed stable disability scores two years after the transplant.

The findings of EDSS improvement in almost 85% of the patients suggest that disability may be often at least temporarily reversible in patients with highly active [relapsing] MS if they receive suitable and well-timed treatment, the researchers wrote.

Using appropriate statistical models, researchers found that the clinical variable that explained the disability regression at months 6 and 12 after AHSCT was the disability progression over 6 months before AHSCT.

Improvements in cognition after AHSCT also were observed. Specifically, the scores of information processing speed and verbal learning, measured by the symbol digit modalities test, were significantly higher at month 12 after AHSCT (56.8) when compared to month three (48.3).

The score of brief visuospatial memory test revised that assesses visuospatial memory was slightly lower at month three (25.6) than before AHSCT (27.8), however, the difference was not significant.

The score of the California verbal learning test, which assesses verbal learning, was significantly higher at month 12 (63.6) than before AHSCT (55.2).

No new or active lesions were found on MRI after AHSCT, suggesting that all patients remained without radiological disease activity.

Furthermore, regarding safety, the incidence and severity of adverse events (side effects) after AHSCT were in the expected range and all were resolved. There were no transplant-related deaths reported.

Researchers noted several limitations to the studys findings, including the low sample size and the fact that the patientss assessment and follow-ups were provided at the same center without a comparative group.

Nonetheless, the outcomes are highly promising, as compared to conventional MS treatment, the researchers wrote. Further research is needed to replicate these findings and to assess long-term outcomes and safety of AHSCT.

Diana holds a PhD in Biomedical Sciences, with specialization in genetics, from Universidade Nova de Lisboa, Portugal. Her work has been focused on enzyme function, human genetics and drug metabolism.

Total Posts: 1,053

Patrcia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

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Stem Cell Transplant Reduces Relapses and Disability in RRMS... - Multiple Sclerosis News Today

Bone Marrow Stem Cells Comments Off on Stem Cell Transplant Reduces Relapses and Disability in RRMS… – Multiple Sclerosis News Today | December 16th, 2020

PHOENIX, Dec. 16, 2020 /PRNewswire/ --Creative Medical Technology Holdings Inc., (OTC CELZ) announced today positive preclinical data supporting the utilization of its ImmCelz cell based immunotherapy for treatment of stroke. In an animal model of ischemia stroke, the middle cerebral artery ligation model, administration of ImmCelz resulted in 34% reduction in infarct volume, whereas control bone marrow mesenchymal stem cells reduced infarct volume by 21%. Additionally, improvements in functional recovery where observed using the Rotarod test. At 28 days after induction of stroke the animals receiving ImmCelz had superior running time (92% of non-stroke controls) compared to animals which received bone marrow mesenchymal stem cells (73% of non-stroke control). Animals that received saline had a running time that was 50% of non-stroke controls.

"The regenerative potential of immune cells that have been programmed by stem cells is a fascinating and novel area of research." Said Dr. Amit Patel, coinventor of ImmCelz, and board member of the Company. "Conceptual advantages of using reprogrammed T cells include higher migratory ability due to smaller size, as well as ability to replicate and potentially form "regenerative memory cells."

"This data, which is covered by our previous filed patents, such as no. 15/987739, Generation of autologous immune modulatory cells for treatment of neurological conditions, demonstrate that immune modulation via this stem cell based method may be a novel and superior way of addressing the $30 billion dollar market for stroke therapeutics1." Said Dr. Thomas Ichim, coinventor of the patent and Chief Scientific Officer of the Company. "The fact that this technology, which has priority back to 2017, is demonstrating such stunning results, motivates us to consider filing an Investigational New Drug Application for use in stroke."

Creative Medical Technology Holdings possesses numerous issued patents in the area of cellular therapy including patent no. 10,842,815 covering use of T regulatory cells for spinal disc regeneration, patent no. 9,598,673 covering stem cell therapy for disc regeneration, patent no. 10,792,310 covering regeneration of ovaries using endothelial progenitor cells and mesenchymal stem cells, patent no. 8,372,797 covering use of stem cells for erectile dysfunction, and patent no. 7,569,385 licensed from the University of California covering a novel stem cell type.

"While stroke historically has been a major area of unmet medical need, the rise in stroke cases , as well as the fact that younger people are increasingly falling victim to stroke, strongly motivates us to accelerate our developmental programs and to continue to explore participation of Big Pharma in this space." Said Timothy Warbington, President and CEO of the Company. "We are eager to replicate the existing experiments start compiling the dossier needed to take ImmCelz into humans using the Investigational New Drug Application (IND) route through the FDA."

About Creative Medical Technology Holdings

Creative Medical Technology Holdings, Inc. is a commercial stage biotechnology company specializing in stem cell technology in the fields of urology, neurology and orthopedics and trades on the OTC under the ticker symbol CELZ. For further information about the company, please visitwww.creativemedicaltechnology.com.

Forward Looking Statements

OTC Markets has not reviewed and does not accept responsibility for the adequacy or accuracy of this release. This news release may contain forward-looking statements including but not limited to comments regarding the timing and content of upcoming clinical trials and laboratory results, marketing efforts, funding, etc. Forward-looking statements address future events and conditions and, therefore, involve inherent risks and uncertainties. Actual results may differ materially from those currently anticipated in such statements. See the periodic and other reports filed by Creative Medical Technology Holdings, Inc. with the Securities and Exchange Commission and available on the Commission's website atwww.sec.gov.

Timothy Warbington, CEO[emailprotected] CreativeMedicalHealth.com

Creativemedicaltechnology.comwww.StemSpine.comwww.Caverstem.comwww.Femcelz.com

1 Stroke Management Market Size Forecasts 2026 | Statistics Report (gminsights.com)

SOURCE Creative Medical Technology Holdings, Inc.

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Creative Medical Technology Holdings Announces Successful Application of ImmCelz Immunotherapy for Treatment of Stroke - PRNewswire

Bone Marrow Stem Cells Comments Off on Creative Medical Technology Holdings Announces Successful Application of ImmCelz Immunotherapy for Treatment of Stroke – PRNewswire | December 16th, 2020