The new horror flick on Netflix, Eli, released just in time for Halloween, borrows from The Exorcist and Rosemarys Baby, with touches of The Shining. And it all takes place in what looks like Downton Abbey with the cleaning staff gone.

Eli works; its scary. But the set-up using gene therapy gone awry is unfortunate, superfluous, and even offensive. (Beware, spoilers ahead)

The film opens with 11-year-old Eli dreaming about being able to go outside without his hazmat suit and breathing without his skin reddening and blistering. He awakens and hes inside, in a bubble.

David was diagnosed, four years earlier, with a rare formof severe combined immune deficiency (SCID). It slashes his ability to make the antibodies that protect against infection, unleashes inflammation that reddens his skin, while at the same time turns his immune system against his own tissues, an autoimmune response.

The parents, caring Rose and weirdo Paul, bundle Eli up to take him to a doctor whos going to cure him with a new treatment. Once at the supposedly clean Downton Abbey haunted house, Eli has a decontamination shower.

When the boy meets the doc, she explains that his immune system makes too many bad immunoglobulins, using that word instead of antibodies because it sounds more technical.Eli quickly responds, spouting out that he has mutations in the RAG1 and RAG2 genes (recombination-activating genes).

Elis body cant make the enzymes that mix and match antibody parts, and the proportions of a bunch of immune system cells and proteins go out of whack. His condition is also called Omenn syndrome.

Dr. Horn has two nurses, and all three of them wear purple uniforms.

Good news! Dr. Horn will administer viral gene therapy! I will make you better, like my other patients, she assures the boy.

Rose gingerly begins to unwrap the blue layers that encase her son, bending down and looking like Laura Dern examining dino poo in Jurassic Park or Princess Leia releasing the hologram from R2D2. Mom and boy can finally hug!!!

At night, the house creaks. Eli wanders the spooky halls, glimpsing kids in the windows, mirrors, and reflections, including ghostly girls who look like the twins at the end of the hallway in The Shining.

A redheaded girl outside, Sadie Sink, apparently escaped from playing Max on Stranger Things, seems real.

Im allergic to the world, Eli tells her. Not exactly.

The next morning, Dr. Horn blames Elis ghost sightings on a side effect from immunosuppressants. Why is she trying to suppress an immune system already so impaired?

Next Eli, who looks so much like Tom Petty that I expected him to shriek I Wont Back Down, is strapped down to a table with a contraption holding his head in place, as Dr. Frankenstein asks her nurses to take a reading.

Dr. Horn at first seems to have gotten the basic idea of gene therapy correct: introducing a working copy of the mutant gene aboard viruses into stem cells from bone marrow. And poof! Like a magic trick! itll work, she proclaims.

She proceeds to extract a hunk of pinkish gunk after drilling into a bone, as the immobilized boy twists and grimaces on the table. Satisfied, the doc plops the glob into a Petri dish.

It burns! Eli shrieks.

That means its working, replies the doc. Within seconds, the doctored viruses have apparently hit their targets.

Then Eli awakens. It all seems a dream, but its foreshadowing.

A ghost appears in a bloody nightgown.

The house breathes at night.

A scrawny, dagger-nailed hand grabs Eli and the apparition turns into his father.

When Eli writes his name on a window, the letters rearrange to spell Lie, like Redrum becoming Murder in the mirror in The Shining. Later on, with the E written like a 3, Eli scratched into various furniture surfaces in the house becomes 317317317. What can it mean?

When Eli reports these events, Dr. Horn barks, Its the medication, as if sophisticated gene therapy has suddenly become as mundane as a tab of Tylenol. Shell have to lower the dosage because the second of the three treatments is coming up.

Treatment 2 is indeed brutal. Eli is held in a contraption like the one Hannibal Lecter wears to keep him from eating people and his head bolted like hes Frankenstein.

Were confident that the gene therapy virus is correcting the mutation, Dr. Horn declares, adding that this will burn a little bit, as she presumably delivers more.

When Eli turns red and screams, she assures him that this is supposed to happen. The virus is penetrating the blood-brain barrier, as if said barrier is a superhighway requiring that the bolts hold his head still.

I was speechless.

Barrier refers to the blood vessels in the brain that are closed to large molecules, which keeps toxins out. A widely-used gene therapy vector, AAV9 (adeno-associated virus 9), has been known for a decade to naturally cross the barrier. And that doesnt require torture hardware.

Heres a photo of one of the kids I write about receiving AAV9 gene therapy for a rare neurological disease through an intravenous delivery in her hand!

The doc then attributes Elis reaction to his body initially rejecting the new cells, like any transplant. But if his gene therapy consists of viruses traipsing across the blood-brain barrier, where did cells suddenly come from? Is it the doctoring of stem cells from bone marrow that was in Elis dream, or delivering viruses into the bloodstream?

Elis nocturnal adventures continue. Hes pushed and pulled from unseen forces as the floor turns transparent, revealing scary medical people. As he keeps bellowing the doc orders Haldol and his mom pushes Valium.

The mysterious 317 opens a key pad to an inner sanctum, which looks like the set of the second Indiana Jones film. We see insects alighting, so the place was never a clean room after all.

Eli finds a notebook with case histories of the past patients and the pieces start to fit. Perry. Agnes. Lucas.

After treatment 2, the kids eyes look haunted, their complexions gray, like Eli. After treatment 3, their heads exploded.

Then the religion clues start to fall out.

Eli discovers a photo of nuns that includes his medical team. A huge iron cross sheaths a dagger. The surgical table with Eli across it resembles Christ on the cross.

One reviewer posits that the plot is about gay conversion, pointing to a scene in which Eli literally crawls out of a closet to tell his parents the truth.

The action speeds up and twists as treatment 3 looms.

Dr. Horn dons religious garb, makes the sign of the cross, flings holy water, and babbles about Jesus and the archangel. The boy, having discovered the medical records, has become a liability.

I thought I could cure Eli. The gene therapy would have worked, if he wasnt so strong. But he cant leave here! the enraged doc yells.

But when Eli is tied down and Dr. Crazy is coming at him with the dagger pulled from the cross, he suddenly summons his inner Regan MacNeil (from The Exorcist) and stops the knife in mid-air, turns it around, and forces the doctor to stab herself. She mutters may you find peace and forgiveness in the name of the Lord, channeling Father Damien KarrasThe power of Christ compels you! as he attempts to exorcise Regan.

With the plunging of the dagger, Eli, red-eyed and screaming, rips off his restraints. His parents are thrown to the floor while the nurses and the doc, somehow still living with the dagger in her chest, try to leave.

But Eli, like Anthony in the cornfield episode of the Twilight Zone, points at them and they turn in unison and then elevate, like Regan rising from her bed. The purple ones then float around the room in an eerie circle emanating an unearthly blue glow, as if theyre on one of those centripetal force amusement park rides.

A conspiracy revealed

It turns out that all are in on whats happening, even the nice-seeming mom. And Eli realizes hes never been sick.

What has she been putting inside me? What have you been putting inside me? he shrieks at his parents, conjuring images of Rosemarysdevil spawn.

At that the nurses and doc suddenly flip upside down, the horror equivalent of Regans rotating head, and slam to the floor.

What am I?

Our son.

Eli sets the nurses and doc on fire.

Are you my dad?

I prayed every day! answers dad.

Prayed to whom? the boy bellows.

The Lord didnt answer me, but your father did, Rose utters mysteriously.

And we know.

Eli never had a SCID. Its a twist on Munchausen Syndrome by Proxy, the cause of his symptoms the holy water that mom and then doc sprinkled on him.

But the ultimate cause? Dad is the devil. At that realization, Eli makes his dads head explode.

The other kids, whose bodies indeed turn up, were Elis half-siblings, even Haley. Dad the Devil got around.

Eli is fun, fast and scary. But why did the writers have to tarnish gene therapy? Why use a genetic disease at all? And especially an ultra rare one? I cant help but wonder what motivated the writers to do this.

Ive devoted the past decade to learning about families who have rare genetic diseases and have kids who have had, or wish they could have, gene therapy, writing about them, and accompanying some of them on their journeys.

In addition to my posts here and at my blog DNA Science, I wrote the only book on gene therapy, The Forever Fix, which chronicles the efforts of a few families. The first gene therapy was FDA-approved in late 2017. The technology has indeed been like the mythical phoenix bird, arising from the ashes.

For the families, I resent the use of gene therapy as a plot point.

After viewing the film the other night, I did a final Facebook check. The first thing that popped up: a photo of an exquisite child, on a page for families dealing with Sanfilippo syndrome, a devastating neurological condition.

The boy was now free of the cruel disease, free to be at peace. He was 11. Elis age.

Genetic disease, and especially attempts to treat it, shouldnt be the stuff of horror films.

Ricki Lewis is the GLPs senior contributing writer focusing on gene therapy and gene editing. She has a PhD in genetics and is a genetic counselor, science writer and author of The Forever Fix: Gene Therapy and the Boy Who Saved It, the only popular book about gene therapy. BIO. Follow her at her website or Twitter @rickilewis

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Viewpoint: Netflix's new horror movie 'Eli' is a fright. But why did they have to 'tarnish gene therapy'? - Genetic Literacy Project

Bone Marrow Stem Cells Comments Off on Viewpoint: Netflix’s new horror movie ‘Eli’ is a fright. But why did they have to ‘tarnish gene therapy’? – Genetic Literacy Project | October 30th, 2019

More than 10 million people worldwideabout 1 percent of people over age 60live with Parkinsons disease. There are treatments that can help control symptoms, but there is no cure.

The hallmark of the disease is the death of certain brain cellsneurons that produce dopamine. Most Parkinsons researchers have focused on studying these cells. But what if the disease starts elsewhere? What if it involves not only neurons but other cells that interact with neurons? In particular, what role is played by astrocytes, star-shaped cells that nurture and help form the connections, or synapses, between the neurons?

(This article by Angela Spivey, with photos by Alex Boerner, originally appeared in Duke Medical Alumni News. Read that story here.)

Thats the question a team of Duke researchers led by Cagla Eroglu, PhD, associate professor of cell biology and neurobiology, is exploring, thanks to a $1 million grant from the Chan Zuckerberg Initiative.

Sitting in her office, Eroglu picks up an orange plastic object that resembles a piece of coral, its tentacles branching this way and that. This is a model of a mouse astrocyte, she says. It can interact with 100,000 synapses at the same time. Astrocytes, she explains, infiltrate the brain, touching everything within their reach. They communicate with its synapses, regulating blood flow and metabolism.

Astrocytes from the Greek astron, meaning "star"have traditionally been thought of as support cells. But that thinking is changing. Since astrocytes are in such close contact and continuously communicating with synapses, we are beginning to appreciate that they are also fundamentally involved in regulating brain function, Eroglu says.

Collaborating with Albert La Spada, MD, PhD, Eroglu found that a certain gene known to be important in Parkinsons is more highly expressed in astrocytes than in neurons. And when the researchers mutated that gene in astrocytes, they saw some intriguing changes. This still-unpublished work laid the foundation for their proposal to the Chan Zuckerberg Initiative, which is bringing together experimental scientists from divergent fields to take a fresh look at the causes of neurodegenerative disorders.

There are vanishingly few papers that have delved into how astrocytes are contributing to the Parkinsons disease process, says La Spada, professor of neurology and vice chair of research for the Department of Neurology. This is an area that's been under-studied, and I think that the results that we're generating are suggesting that it deserves more attention.In addition to his long experience studying neurodegenerative diseases, La Spada brings expertise in growing astrocytes from induced pluripotent stem cells (IPSCs). That process starts by growing skin cells from a skin biopsy from a Parkinsons patient. Then we use what's called a reprogramming protocol to basically revert them to stem cells that are pluripotent. Once you create the IPSCs, you could use them to make any cell you wanta muscle cell or a cardiac cell or a neuron or an astrocyte, La Spada says. The beauty of this is, it comes from the patient who has the disease of interest."

His labs expertise will only grow because of the Chan Zuckerberg Initiative, which has formed focus groups for grantees around various areas, such as stem cell modeling, CRISPR gene-editing technology, bioinformatic analysis of data sets, and more. We're meeting other researchers from around the world who are doing really unique things. It's a chance for us all to compare notes, and I think this will accelerate all of our endeavors, La Spada says.

Rounding out the team is Nicole Calakos, MD, PhD, a scientist and clinician who treats patients with movement disorders, including Parkinsons. Calakos says that when she first met Eroglu, she was intrigued by her idea that since astrocytes are involved in sculpting the language of neurons, perhaps they play a role in the events that can lead to disease.

Everybody has been fixated like a magnet on the idea that the problem is the neuron that's dying, Calakos says. Cagla said, Hey, let's think outside of the box of that dead cell. Lets consider whether astrocytes are like the soil around a plant, providing the nutrition, and allowing it to form roots, and maybe that is whats broken. Why aren't we even thinking about this critical piece of the brain?

Eroglu puts it this way: Maybe the problem is loss of connections between neurons, even before they die.

Calakos says that part of the reason she came to Duke was the close intermingling of physicians and bench scientists. Because of how the community is at Duke, Cagla and I had been exchanging ideas and collaborating over the years, she says. The Chan Zuckerberg grant is an opportunity to get together as a formal team. I think it's really forward-thinking of them to have teams of basic scientists and practicing physicians all talking to each other.

The Chan Zuckerberg Initiative was launched in December 2015 by Mark Zuckerberg, founder and CEO of Facebook, and Priscilla Chan, a pediatrician and founder and CEO of The Primary School in East Palo Alto. In addition to her clinical insight, Calakos brings expertise in electrophysiologyreal-time recording and observation of electrical signals coming from brain cells. We can listen to the language of synapses, she says. They speak in electrical currents,which we can measure. Eroglu believes that by learning all they can about how astrocytes support synaptic development and health in the normal brain, they may find ways to stop neurodegenerative diseases like Parkinsons.

We are seeing aging as a part of development, Eroglu says. If your house is built on a strong base, then it might last longer. Whereas, if you build it in another way, it may be there for a while, but gradually start to break down.

This doesn't mean that we are destined to have neurodegeneration and we can't do anything. We may be more predisposed to get the disease, but we may not get it if we have done something else in our lives that helps strengthen our brain. I strongly believe that there will be ways to stop neurodegeneration.We will find a way to strengthen the brain connections. If we can figure out the weakest link, then we could concentrate on solving that.

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The Stars in Our Brains - Duke Department of Neurology

Cardiac Stem Cells Comments Off on The Stars in Our Brains – Duke Department of Neurology | October 30th, 2019

DUBLIN--(BUSINESS WIRE)--The "Cell Based Assay & High Content Screening Markets Market Forecasts by Application, With Executive and Consultant Guides and including Customized Forecasting and Analysis 2020 to 2024" report has been added to ResearchAndMarkets.com's offering.

Cell Based Assays are a mainstay of drug development and scientific research, but growth is now accelerating as new immuno-oncology markets create unprecedented investment in the race to cure cancer. On top of this new technology is allowing Cell Based Assays to be used to measure any aspect of cell function. This market just keeps on growing with no end in sight. The workhorse of the pharmaceutical industry is becoming a central player in biotechnology.

This is a complex area but this readable report will bring the entire management team up to speed, on both the technology and the opportunity.

The technology is moving faster than the market. Genomics and Immunology are playing a role too. Find opportunities and pitfalls. Understand growth expectations and the ultimate potential market size.

Key Topics Covered

1. Introduction and Market Definition

1.1 What are Cell Based Assays?

1.2 Clinical Trial Failures

1.2.1 Immuno-oncology Plays a Leading Role in Cell Based Assays

1.3 Market Definition

1.3.1 Market Size

1.3.2 Currency

1.3.3 Years

1.4 Methodology

1.4.1 Authors

1.4.2 Sources

1.5 U.S. Medical Market and Pharmaceutical Research Spending - Perspective

1.5.1 U.S. Expenditures for Pharmaceutical Research

2. Cell Based Assays - Guide to Technology

2.1 Cell Cultures

2.1.1 Cell Lines

2.1.2 Primary Cells

2.1.3 Stem Cells

2.1.3.1 iPSC's - The Special Case

2.2 Cell Assays

2.3 Cell Viability Assays

2.3 Cell Proliferation Assays

2.4 Cytotoxicity Assays

2.5 Cell Senescence Assays

2.6 Apoptosis

2.7 Autophagy

2.8 Necrosis

2.9 Oxidative Stress

2.10 2D vs. 3D

2.11 Signalling Pathways, GPCR

2.12 Immune Regulation & Inhibition

2.13 Reporter Gene Technology

2.14 CBA Design & Development

2.15 Cell Based Assays - The Takeaway

3. Industry Overview

3.1 Players in a Dynamic Market

3.1.1 Academic Research Lab

3.1.2 Contract Research Organization

3.1.3 Genomic Instrumentation Supplier

3.1.5 Cell Line and Reagent Supplier

3.1.6 Pharmaceutical Company

3.1.7 Audit Body

3.1.8 Certification Body

4. Market Trends

4.1 Factors Driving Growth

4.1.1 Candidate Growth

4.1.2 Immuno-oncology

4.1.3 Genomic Blizzard

4.1.4 Technology Convergence

4.1.5 The Insurance Effect

4.2 Factors Limiting Growth

4.2.1 CBA Development Challenges

4.2.2 Instrument Integration

4.2.3 Protocols

4.3 Technology Development

4.3.1 3D Assays

4.3.2 Automation

4.3.3 Software

4.3.4 Primary Cells

4.3.5 Signalling and Reporter Genes

4.3.6 The Next Five Years

5. Cell Based Assays Recent Developments

5.1 Recent Developments - Importance and How to Use This Section

5.1.1 Importance of These Developments

5.1.2 How to Use This Section

6. Profiles of Key Cell Based Assay Companies

7. Global Market Size

7.1 Cell Based Assay Global Market Size by Region with Charts

7.2 Cell Based Assays Global Market Size by Type with Charts

8. Global Market by User Type

8.1 Pharmaceutical Market

8.1.1 Pharmaceutical Market by Region with Chart

8.2 Basic Research Market

8.2.1 Basic Research Market by Region with Chart

8.3 Industrial/Cosmetic Market

8.3.1 Industrial/Cosmetic Market by Region with Chart

9. Cell Based Assay by Product Class

9.1 Instrument Market

9.1.1 Instrument Market by Region with Chart

9.2 Reagent Market

9.2.1 Reagent Market by Region with Chart

9.3 Services Market

9.3.1 Services Market by Region with Chart

9.4 Software Market

9.4.1 Software Market by Region with Chart

10. Appendices

10.1 FDA Cancer Drug Approvals by Year

10.2 Clinical Trials Started 2010 to 2016

10.3 Share of Pharma R&D by Country

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/atsea9

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Global Cell Based Assay & High Content Screening Markets, 2020-2024 | Forecast by Application with Executive & Consultant Guides -...

Cardiac Stem Cells Comments Off on Global Cell Based Assay & High Content Screening Markets, 2020-2024 | Forecast by Application with Executive & Consultant Guides -… | October 30th, 2019

Non-surgical regenerative cell-based treatment uses the bodys natural healing ability to repair damaged bones, muscles, cartilage, tendons and ligaments.Knee injuries are painful and often patients are unable to walk. Our treatment protocol always uses products following FDA guidelines.Injections done with ultrasound guided needle recognition capability to ensure safety as well target the area needing treatment. Plasma; Alpha-2-Macroglobulim (A2M) is the new biologic treatment for your arthritic knee (osteoarthritis)When your hips hurt, or your knee is stiff, or your back is throbbing, that means your joint is bone on bone and there is no lubrication to ease movement.Regenerative medicine giving new hope to patients suffering from painful joint injuries such as knee, shoulder and hip with a chance to live a pain free life.Regenerative cell-based ultrasound guided injection now available to treat pain associated with joint injury. There are indications that it reduces the pain and swelling of the joints and helps lubricating and improve movements.Commonly Treated Conditions: Osteoarthritis of the Hips, Knee, and Shoulders Rotator Cuff tears of the Shoulder Meniscus, ACL and PCL tears of the kneeOur stem cell treatment using your own stem cells and with using imaging guidance ensures precise injection of stem cell, it is a highly-specialized practice.Besides treating above injuries we have advance stem cell micro-needling treatment for the following: Cell-based PRP Hair Restoration combining micro-needling with growth factors and hair follicles voluma vitamins plus BLotinyl T1, Biotin, Anti-aging and Kopexil. Non-toxin facial renewal Anti-Aging APGF Advanced Peptide Micro-needling PRP, Dual Anti-Aging Ampoules for deep hydration, more collagen to reduce wrinkles and firm skin.Dr. Ibrahim is the staff physician at Valencia Medical Center specializing in regenerative medicine, pain management, and rejuvenation. Call for a consultation at 661-222-9117.

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Hormones Control your Health, Mood and Behavior A balanced hormone means happier, healthier life and success in career and relationship. - Magazine of...

Skin Stem Cells Comments Off on Hormones Control your Health, Mood and Behavior A balanced hormone means happier, healthier life and success in career and relationship. – Magazine of… | October 29th, 2019

Michael Schumacher was said to have been admitted to a hospital in Paris the past September. Based on the news, he was transported to the medical facility by helicopter, and he was there to receive the procedure called Stem Cell Therapy.

According to The Telegraph, Schumachers latest treatment is giving his family and friends the big hope that his condition would improve. The treatment is one of the latest procedures that was developed to help people suffering from various illnesses. It uses stem cells to treat patients and help prevent disease or certain health conditions.

Did Schumi really try the Stem Cell Therapy for his condition?

Michael Schumachers health is a closely guarded matter so no knows exactly how he is doing after coming out of his coma more than five years ago. It can be recalled that he sustained a grave head injury after hitting a rock while skiing in the Alps in 2013.

Thus, it is not surprising if details of this alleged stem cell procedure are also being kept a secret by his family. They would not even confirm if he was really hospitalized for the treatment but a nurse supposedly said that Schumi is now conscious after the procedure. La Parisien via Mirror reported that an unnamed nurse claimed that the F1 legend showed signs of recovery.

"Yes he is in my service," she said. "And I can assure you that he is conscious."

Doctor may have confirmed the therapy

It was said that Dr. Philippe Menasche, a French cardiac surgeon, performed the procedure on Michael Schumacher and when the news broke out for the first time, he slammed the people who were alleging that his treatment on Schumi was only experimental.

As per Australias 7News, the doctor hit out the media for covering the treatment on Michael Schumacher and giving out false information. When the media called his advanced procedures experimental, he refuted the claims and said that he dont perform miracles.

"My team and I are not doing an experiment, an abominable term that is not in line with a serious medical view, he told the Italian newspaper La Republica. In any case, Menasch reaction was meant to defend his stem cell therapy treatment however, he seemed to have unknowingly confirmed that he treated Michael Schumacher as well.

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Michael Schumacher: Has the racing champ recovered? Stem Cell Therapy doctor unknowingly confirmed Schumi's procedure? - EconoTimes

Cardiac Stem Cells Comments Off on Michael Schumacher: Has the racing champ recovered? Stem Cell Therapy doctor unknowingly confirmed Schumi’s procedure? – EconoTimes | October 28th, 2019

Gene therapy is a promising therapeutic procedure for genetic disorders or diseases in which defective genes are corrected, replaced, or inactivated.

In the case of adrenoleukodystrophy (ALD) a genetic disorder caused by mutations in the ABCD1 gene that damages the myelin sheath around nerve cells gene therapy may benefit patients prior to the onset, or during the early stages, of the disease by stopping the progression of demyelination. However, the therapy cannot be beneficial after the disease has worsened significantly.

Gene therapy works by introducing the correct gene sequence into cells. Since genetic material cannot enter the cell on its own, the correct gene sequence needs to be delivered using a vector. This vector can be a modified virus that has been engineered to remove its pathogenic genetic material so that it cannot cause disease, but is still able to transfer the correct gene sequence to the host cell.

The vector can be directly injected into the patients body or into host cells grown in the laboratory and then transplanted back into the patient. Upon successful viral transfer, the host cell should be able to produce the functional protein.

In ALD, the clinician first takes out the patients own stem cells (autologous) and then inserts the correctABCD1 gene sequence into these cells using a viral vector in the laboratory. The corrected stem cells that are able to produce the functional ALD protein are then implanted back into the patients body so they may develop into nerve cells in the brain. Since the patients own cells are being used, there are fewer risks than when donor stem cells are used.

Lenti-D,an investigational gene therapy developed by Bluebird Biois currently being studied in a Phase 2/3 clinical trial (NCT01896102) in the U.S., the U.K., and France. The study aims to evaluate the safety and effectiveness of Lenti-D in boys, up to 17 years old who havecerebral adrenoleukodystrophy (CALD). Based on the preliminary data from this study,the U.S. Food and Drug Administration (FDA)designated Lenti-D a breakthrough therapy for the treatment of CALD in May 2018.

A Phase 1/2 clinical trial (NCT02559830) is recruiting patients with ALD at the Shenzhen Second Peoples Hospital in Guangdong, China. The study aims to assess the safety and effectiveness of transplanting patient-derived bone marrow stem cells, which have been genetically-corrected using a lentiviral vector, for the treatment of ALD.

Another Phase 1/2 clinical trial (NCT03727555) at the Shenzhen Geno-Immune Medical Institute also in Guangdong, China is recruiting 10 patients with ALD. The study aims to evaluate the safety and effectiveness of a lentiviral vector carrying the healthy ABCD1 gene (TYF-ABCD1) injected directly into the patients brain for the treatment of ALD.

***

Adrenoleukodystrophy News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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zge has a MSc. in Molecular Genetics from the University of Leicester and a PhD in Developmental Biology from Queen Mary University of London. She worked as a Post-doctoral Research Associate at the University of Leicester for six years in the field of Behavioural Neurology before moving into science communication. She worked as the Research Communication Officer at a London based charity for almost two years.

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Gene Therapy - Adrenoleukodystrophy News

Bone Marrow Stem Cells Comments Off on Gene Therapy – Adrenoleukodystrophy News | October 28th, 2019

Still to be determined is how hospitals and other health care facilities will be reimbursed for the therapy and whether patients will have access to the therapy under health care proposals such as Medicare for All or a so-called public option.

One of the most promising cancer treatments to come along in years, CAR-T cell therapy uses the bodys own immune system to attack and kill cancer cells. The treatment involves bioengineering T cells, a white blood cell that fights foreign substances in the body, and equipping them with new Chimeric Antigen Receptors that target cancer cells.

CAR-T cell therapy has been approved by the U.S. Food and Drug Administration (FDA) for children with leukemia and adults with advanced lymphoma. The therapy is typically used alongside other, more traditional treatments such as surgery, chemotherapy, and radiation. Its use in combatting other forms of cancer is pending with the FDA, an agency known for its slow approval process.

Hefty Price Tag

There are currently two approved CAR-T treatments: Novartis Kymriah (tisagenlecleucel) and Gileads Yescarta (axicabtagene).

Like most newly introduced cutting-edge treatments, the two products come with a hefty price tag. A course of treatment of Kymriah costs $475,000 for pediatric and young adult patients with leukemia, and both are priced at $373,000 to treat lymphoma in adults, according tobiopharma.com. Under Centers for Medicare & Medicaid Services (CMS) regulations published in August, Medicare will reimburse hospitals for 65 percent of the treatments cost, or about $242,000, through Part B.

Although hospitals will likely welcome Medicares financial commitment, there still remains a sizable gap. Further complicating reimbursement is the lack of a separate Medicare billing code for CAR-T treatment, which will be handled via codes for bone marrow and stem cell transplants until a CAR-T billing code is developed, which could take up to three years.

CMS worked closely with the FDA and the National Cancer Institute in developing the new regulations, a time-consuming process rooted in the complexities of developing a reimbursement scheme and overseeing an innovative and evolving therapy.

At a July 31 Heritage Foundation panel discussion on Medicare for All, CMS Administrator Seema Verma discussed the challenges government programs face in approving innovative treatments.

Much of the problem is when Congress says you can cover durable medical treatment, supplies, and drugs, said Verma. Sounded great when they wrote that law 30 to 40 years ago but doesnt make sense in todays environment. All of these new treatments are coming out, and they dont fit nicely into the way the law has been constructed, and it creates problems for the agency.

Bonner R. Cohen,Ph.D.,(bcohen@nationalcenter.org)is a senior fellow at the National Center for Public Policy Research and a senior policy analyst with the Committee for a Constructive Tomorrow (CFACT).

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Medicare Coverage of CAR-T Cell Therapy Raises New Questions - The Heartland Institute

Bone Marrow Stem Cells Comments Off on Medicare Coverage of CAR-T Cell Therapy Raises New Questions – The Heartland Institute | October 28th, 2019

28 October 2019, 12:40

The Emmerdale actress welcomed baby Ace into the world in July of this year

Charley Webb has revealed that she's storing her baby son Ace's skin cells in an emotional Instagram post.

Read more: Strictly judge Craig Revel-Horwood blames viewers for shock Catherine exit after fierce backlash

Sharing an adorable photo of the tot, who she shares with her husband Matthew Wolfenden, she wrote: "We decided to store Aces stem cells. As parents every single one of us wants to do whats best for our children. When I was pregnant, I heard about the possibility of collecting and storing my baby's umbilical cord stem cells, which could then be used in the future should they be needed for treatment (I hope with every part of me we never need it).

"After researching, we learned that the baby's umbilical cord is a valuable source of stem cells, and these cells can be collected at birth and stored.

Read more: Coronation Streets Sally Dynevor couldn't watch Sinead's devastating death after her own cancer battle

"These could then be used as a crucial part of treating or curing an illness. Currently, there are over 80 diseases cord blood stem cells can treat. I decided to use Smart Cells to store the stem cells: the process was easy (genuinely) and they organised everything.

"Like I said, we hope we never need to use them, but it's comforting to know that we have them stored if we ever do. This is a once in a lifetime opportunity, and Im so grateful we were able to do this. Xx".

Many parents rushed to voice their approval, with one commenting: "Amazing! Such an important thing and I think every parent should consider doing this as it may save a life so respect for you. And Ace is so cute."

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Charley Webb reveals she's storing her baby son Ace's skin cells in emotional post - Heart

Skin Stem Cells Comments Off on Charley Webb reveals she’s storing her baby son Ace’s skin cells in emotional post – Heart | October 28th, 2019

NEW YORK, Oct. 22, 2019 /PRNewswire/ -- Bloomberg Philanthropies, Johns Hopkins University School of Medicine (JHUSOM), and The New York Stem Cell Foundation (NYSCF) Research Institute today announced an initiative to fundamentally advance and expand the science of precision medicine, in which diagnostic disease markers are defined with pinpoint accuracy to help researchers understand disease pathways and customize therapeutic approaches. The collaboration will combine the renowned clinical and medical expertise of Johns Hopkins with the unique stem cell technologies and research capabilities of the NYSCF Research Institute to accelerate Hopkins' pioneering Precision Medicine Initiatives.

"Johns Hopkins is working intensively to realize the great promise of precision medicine for all those in our care, locally and globally," said Johns Hopkins President Ronald J. Daniels. "This significant new collaboration with Bloomberg Philanthropies and NYSCF moves us ever closer to that aim as we join together our far-reaching research capacities to advance knowledge and deliver better health outcomes for populations and people around the world."

This collaboration will also establish an unprecedented cache of human disease models available to researchers worldwide thus promoting the real world application of precision medicine and driving a new paradigm for understanding and improving the approach to human disease.

"Bloomberg Philanthropies' mission is to ensure better, longer lives for the greatest number of people," said Michael R. Bloomberg, founder of Bloomberg LP and Bloomberg Philanthropies. "For years, Johns Hopkins University and the New York Stem Cell Foundation have shared that mission and we're honored to deepen our partnerships with them as they explore new, innovative ways to save lives through the application of precision medicine."

Diseases manifest themselves differently in different patients. To understand the basis of these differences and to tailor treatments for specific patients, researchers need more accurate biological tools. Stem cell models provide a "biological avatar" of the patient from which they were created, allowing scientists and clinicians to better understand, define, and account for differences in individual patients and groups of patients.

The new initiative will use induced pluripotent stem cells to study disease characteristics in subgroups of patients, identifying markers that lead to varying disease manifestations. For example, by examining stem cells from seemingly similar patients with different forms of multiple sclerosis, we may be able to better understand the full range of disease mechanisms and pathways.

The Johns Hopkins Precision Medicine Initiative already includes 16 Precision Medicine Centers of Excellence (PMCOE), each focusing on a specific disease, and is now working to develop 50 Precision Medicine Centers in the next five years. Johns Hopkins believes that this advancement in the study and application of precision medicine has the potential to transform the diagnosis and management of many diseases.Often, what is now categorized as a single disease is actually made up ofmultiple diseases that display similar symptoms, but require quite different therapies. Using a wide range of data sources, precision medicine seeks to better elucidate these differences, so that doctors can treat patients with precisely targeted therapies. At Johns Hopkins, dozens of researchers are bringing this idea to reality across a spectrum of debilitating and life-altering diseases.

In this collaboration, the process will begin with the full consent of patients in JHUSOM PMCOEs who wish to participate. Biological samples from the JHUSOM PMCOEs will be collected by the NYSCF Research Institute where scientists will create stem cell models of disease using the NYSCF Global Stem Cell Array, the world's first end-to-end automated system for generating human stem cells in a parallel, highly controlled process.Integrating robotics and machine learning, NYSCF's technology reprograms skin or blood cells into stem cells, differentiates them into disease-relevant cell types, and performs genome editing to unravel the genetic basis of disease.

"The NYSCF Research Institute has invented and scaled the most advanced methods of human cell manipulation, which is critical for studying disease at the level of the individual patient," explained NYSCF CEO Susan L. Solomon. "By combining our capabilities with Johns Hopkins' extensive clinical data and expertise, we will be able to develop effective, personalized therapies for patients suffering from diseases with a high unmet need."

The stem cells generated by NYSCF will be used to research and drive effective therapeutic and diagnostic development in a wide range of diseases that include, but are not limited to, Multiple Sclerosis, Alzheimer's, chronic renal failure, and cancers of the lung, breast, prostate, pancreas, and bladder. These stem cell lines will reside in the NYSCF Repository and serve as an extraordinary resource in perpetuity for the disease research community. This vast collection will allow scientists unprecedented insights into the biochemical and genetic mechanisms underlying different diseases and subtypes thereof, thereby illuminating avenues for effective, tailored interventions.

"Stem cell science holds enormous potential for the treatment of a wide range of diseases," said Paul B. Rothman, dean of the School of Medicine and CEO of Johns Hopkins Medicine. "By combining this approach with Johns Hopkins' groundbreaking work on precision medicine, we are creating a scientific powerhouse that will help us advance medicine and science at an even faster pace. I am excited to see the discoveries and innovations that will be produced by this collaboration."

About Bloomberg PhilanthropiesBloomberg Philanthropies invests in 510 cities and 129 countries around the world to ensure better, longer lives for the greatest number of people. The organization focuses on five key areas for creating lasting change: Arts, Education, Environment, Government Innovation, and Public Health. Bloomberg Philanthropies encompasses all of Michael R. Bloomberg's giving, including his foundation and personal philanthropy as well as Bloomberg Associates, a pro bono consultancy that works in cities around the world. In 2018, Bloomberg Philanthropies distributed $767 million. For more information, please visitbloomberg.orgor follow us on Facebook, Instagram, YouTube, and Twitter.

About The New York Stem Cell Foundation Research Institute The New York Stem Cell Foundation (NYSCF) Research Institute is an independent non-profit organization accelerating cures and better treatments for patients through stem cell research. The NYSCF global community includes over 180 researchers at leading institutions worldwide, including the NYSCF Druckenmiller Fellows, the NYSCF Robertson Investigators, the NYSCF Robertson Stem Cell Prize Recipients, and NYSCF Research Institute scientists and engineers. The NYSCF Research Institute is an acknowledged world leader in stem cell research and in developing pioneering stem cell technologies, including the NYSCF Global Stem Cell Array and in manufacturing stem cells for scientists around the globe. NYSCF focuses on translational research in an accelerator model designed to overcome barriers that slow discovery and replace silos with collaboration. For more information, visit http://www.nyscf.org or follow us on Twitter, Facebook, and Instagram.

Press Contacts:

The New York Stem Cell Foundation Research Institute David McKeon dmckeon@nyscf.org 212-365-7440

Johns Hopkins University School of Medicine Vanessa Wasta wasta@jhmi.edu

SOURCE The New York Stem Cell Foundation

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Bloomberg Philanthropies, Johns Hopkins University School of Medicine, and The New York Stem Cell Foundation Research Institute Announce an...

Skin Stem Cells Comments Off on Bloomberg Philanthropies, Johns Hopkins University School of Medicine, and The New York Stem Cell Foundation Research Institute Announce an… | October 28th, 2019

In 2018, the market size of Disposable Diabetes Devices Market is million US$ and it will reach million US$ in 2025, growing at a CAGR of from 2018; while in China, the market size is valued at xx million US$ and will increase to xx million US$ in 2025, with a CAGR of xx% during forecast period.

In this report, 2018 has been considered as the base year and 2018 to 2025 as the forecast period to estimate the market size for Disposable Diabetes Devices .

This report studies the global market size of Disposable Diabetes Devices , especially focuses on the key regions like United States, European Union, China, and other regions (Japan, Korea, India and Southeast Asia).

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This study presents the Disposable Diabetes Devices Market production, revenue, market share and growth rate for each key company, and also covers the breakdown data (production, consumption, revenue and market share) by regions, type and applications. Disposable Diabetes Devices history breakdown data from 2014 to 2018, and forecast to 2025.

For top companies in United States, European Union and China, this report investigates and analyzes the production, value, price, market share and growth rate for the top manufacturers, key data from 2014 to 2018.

In global Disposable Diabetes Devices market, the following companies are covered:

Bayer HealthcareAbbott LaboratoriesJohnson& JohnsonBecton DickinsonF.Hoffmann La-RocheNovo NordiskMedtronicSanofiARKRAYTerumo

Segment by RegionsNorth AmericaEuropeChinaJapanSoutheast AsiaIndia

Segment by TypeDiagnostics DevicesDelivery Devices

Segment by ApplicationHospitals PharmaciesRetail PharmaciesE-Commerce

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The content of the study subjects, includes a total of 15 chapters:

Chapter 1, to describe Disposable Diabetes Devices product scope, market overview, market opportunities, market driving force and market risks.

Chapter 2, to profile the top manufacturers of Disposable Diabetes Devices , with price, sales, revenue and global market share of Disposable Diabetes Devices in 2017 and 2018.

Chapter 3, the Disposable Diabetes Devices competitive situation, sales, revenue and global market share of top manufacturers are analyzed emphatically by landscape contrast.

Chapter 4, the Disposable Diabetes Devices breakdown data are shown at the regional level, to show the sales, revenue and growth by regions, from 2014 to 2018.

Chapter 5, 6, 7, 8 and 9, to break the sales data at the country level, with sales, revenue and market share for key countries in the world, from 2014 to 2018.

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Chapter 10 and 11, to segment the sales by type and application, with sales market share and growth rate by type, application, from 2014 to 2018.

Chapter 12, Disposable Diabetes Devices market forecast, by regions, type and application, with sales and revenue, from 2018 to 2024.

Chapter 13, 14 and 15, to describe Disposable Diabetes Devices sales channel, distributors, customers, research findings and conclusion, appendix and data source.

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Animal Stem Cell Therapy Market Revenue, Opportunity, Segment and Key Trends 2017 2025 - Health News Office

Skin Stem Cells Comments Off on Animal Stem Cell Therapy Market Revenue, Opportunity, Segment and Key Trends 2017 2025 – Health News Office | October 27th, 2019

Founded in 1958, the AO foundation is a medically guided, not-for-profit organisation led by an international group of surgeons specialised in the treatment of trauma and disorders of the musculoskeletal system. Today, the AO has a global network of over 200,000 health care professionals. Each year it offers over 830 educational events around the world, which are supported by nearly 9,000 faculty and are attended by over 58,000 participants. It has 20,000 surgeon members working in the fields of trauma, spine, craniomaxillofacial (CMF), veterinary, and reconstructive surgery.

The Mission of the AO foundation is promoting excellence in patient care and outcomes in trauma and musculoskeletal disorders. The focus of the AO clinical divisions, clinical unit, and Institutes, is on producing new concepts for improved fracture care, delivering evidence-based decision making, guaranteeing rigorous concept and product approval as well as timely and comprehensive dissemination of knowledge and expertise. The AO is made up from four clinical divisions (AOTrauma, AOSpine, AOCMF, AOVET), one clinical unit (AORecon), and four institutes the AO Research Institute Davos (ARI), AO Education Institute, AO Clinical Investigation & Documentation and AO Technical Commission (AOTK).

AO Research Institute Davos (ARI) is both the academic arm and the translational arm of the AO foundation. In its work to further the AO foundations mission (promoting excellence in patient care and outcomes in trauma and musculoskeletal disorders), ARIs purpose is to advance patient care through innovative orthopaedic research and development.

The goals of ARI include: Contribute high quality applied preclinical research and development (exploratory and translational) focused towards clinical applications/solutions; investigate and improve the performance of surgical procedures, devices and substances; foster a close relationship with the AO medical community, academic societies, and universities; and provide research environment / research mentorship / research support for AO clinicians.

Our Bone Regeneration focus area looks at bone healing in response to fracture involving a complex sequence of dynamic events, directed by numerous different cell types and growth factors. A critical factor for bone repair is the maintenance, or effective restoration, of an adequate blood supply, which is necessary to provide the damaged tissue with oxygen, nutrients and growth factors, as well as immune cells and mesenchymal stem cells required to repair the damage and induce new bone formation. Although bone generally has a high regenerative capacity, in some cases this inherent bone healing is compromised, which results in delaying healing or non-union of the bone fracture with increased health care costs and reduced quality of life issues for affected patients.

While a variety of risk factors have been identified that predispose a patient to an increased risk of developing delayed bone healing or non-union, it is currently not possible to identify specific at-risk patients at an early stage. Using in vitro, in vivo and microfluidic technologies, the aim of the Bone Regeneration Focus Area is to gain a greater understanding of the cellular interactions and mediators, including immunoregulation, underlying such impaired healing responses. By determining how cells such as immune cells, mesenchymal stem cells and endothelial cells normally interact during the repair process, and how this process is altered during impaired healing, we can then identify key mediators of the healing process. Our goal is to use tissue engineering and regenerative medicine approaches to promote bone healing, aimed at restoring bone integrity and its effective biomechanical properties.

In terms of this focus area, we aim at investigating the potential mechanisms leading to intervertebral disc (IVD) damage and evaluating novel biological treatment methods for IVD repair and regeneration. Acute and chronic damage to the IVD are major causes of low back pain. However, the factors that contribute to the loss of function of the IVD and the underlying pathophysiology are still poorly understood. We have established a whole IVD organ culture system with the ability to maintain entire discs with the endplates for several weeks under controlled nutrient and mechanical loading conditions.

Within this bioreactor, the beneficial or detrimental effects of nutrition, mechanical forces, and/or biochemical factors on disc cell viability and metabolic activity can be investigated. We have developed various defect and degeneration models, allowing us to design and evaluate appropriate biological treatment strategies. These include implantation of cells, delivery of anabolic, anti-catabolic or anti-inflammatory molecules, biomaterials or a combination thereof. Data from ex vivo models are also correlated to in vivo observations to identify molecular markers of IVD damage or degeneration.

To study the potential of new therapies for articular cartilage repair and regeneration, a bioreactor system applying multiaxial load to tissue-engineered constructs or osteo-chondral explants has been established. The bioreactor mimics the load and motion characteristics of an articulating joint. Chondral and osteochondral defect and disease models enable us to test tailored treatments under physiologically relevant mechanically loaded ex-vivo conditions. Cell- and material-based therapies as well as chondrogenic or anti-inflammatory factors are under investigation for cartilage repair and regeneration.

Biomaterials for skeletal repair can provide structural and mechanical features for the filling of defects, but also be a carrier for drugs, cells and biological factors. One of our goals is the development of 3D structures for bone, disc and cartilage tissue engineering, using tailored polymers and composites manufactured with additive manufacturing processes.

Our experience lies in the design of biocompatible, biodegradable polymers and their processing with controlled architecture and embedded biologics. A second field of research investigates the preparation of hyaluronan, a natural occurring biopolymer, based biomaterials which can be used to deliver drugs and cells. These injectable biodegradable materials have considerable potential in infection prophylaxis and tissues repair. We are also developing innovative technologies for the structuration and assembly of tissue-like matrices aiming to mimic for example, biological matrix mechanical and structural anisotropy. Additive manufacturing technologies will lead to the development of patient specific implants that can be tailor made to each individual case.

The Stem Cell focus area is particularly interested in stem cell therapies for bone and cartilage that could be applied within a clinical setting. We are increasingly investigating donor variation with the aim to predictively identify the potency of cells from individual donors. In the search for biomarkers to determine patient specific healing potential, exosomes and non-coding RNA sequences such as miRNA are increasingly being used as a diagnostic and therapeutic tool. The development of a serum-based biomarker approach would dramatically improve patient specific clinical decisions.

We also aim to investigate the role of mechanical and soluble factors in the activation of mesenchymal stem cells, and the promotion of differentiation and tissue repair. We can induce chondrogenic differentiation of human MSCs purely by mechanical stimulation and this is leading to new insights into cell behavior under loading conditions. Mechanical forces can be applied by way of rehabilitation protocols and are able to modify stem cell and immune cell function. Such studies are forming the basis of the emerging field of regenerative rehabilitation. In addition to the effect of load on direct differentiation, it is known that biomechanical stimulation can modulate the cell secretome. Investigating these changes could lead to the identification of new targets that may be present during articulation. This offers new avenues for potential clinical therapies.

The Musculoskeletal Infection team focusses their research activities on Fracture-Related Infection (FRI), with goals to optimise antibiotic prophylaxis, reduce the burden of therapeutic interventions, and study the impact of co-administered medication on infection. Our studies include preclinical in vitro and in vivo studies, as well as an increasing focus on observational studies in human patients.

In collaboration with ARI colleagues in the preclinical testing facility, we now have models that can mimic an open fracture, with a chronology and fixation that more accurately reflects clinical reality. Further advancements in our animal models in the past year include the controlled delivery of antimicrobials via the use of programmable, implantable pumps to more precisely control antibiotic dosing. In addition, we have investigated in more detail the use of anti-inflammatory medication in our animal studies and found it can have a major impact on treatment outcome, and so will be a focus for future studies with clear relevance for trauma patients. The preclinical evaluation of novel anti-infective interventions under Good Laboratory Practice (GLP) conditions has also continued in the past year, with two novel antimicrobial intervention studies performed in this space in the past year.

On the in vitro side, we have begun to develop an in vitro model for Staphylococcus aureus infection that has the potential to include human immune system cell-lines. This can not only reduce future animal studies but will also allow us to test interventions in a human-specific system. The antibiotic loaded hydrogel that has been in testing in ARI for several years, has now also been tested against MRSA biofilms and continues to be superior to aqueous solutions of antibiotics. In patient samples, we have made our first preparations for a study on the impact of antibiotic therapy on the human gut and skin microbiome. This is an under explored area of immense potential for bone health and will be a multi-year investigation with expert collaborations internationally.

A Fracture-Related Infection (FRI) consensus meeting in Davos in December 2016 achieved consensus on the fundamental features of FRI, and a proposal for defining the presence of FRI was reached. The establishment of this definition offers the opportunity to standardise preclinical research, improves the reporting of clinical studies and finally of course also aids in the decision-making during daily clinical practice. In the following 18 months, the expert group shifted attention to the next phase, validating the diagnostic criteria and develop treatment principles for FRI and a consensus on diagnosis and treatment principles for FRI.

In reflecting the greater complexity of this question, and to engage with other professional organisations, the group has grown to include external partners. Joining the ARI, AOTrauma and the AOTK Anti-Infection task force (AITF), is the European Bone and Joint Infection Society (EBJIS), the Orthopaedic Trauma Association (OTA), and the Pro-Implant Foundation, as well as a broadened panel of experts with extensive clinical experience in FRI. A first meeting of the expert group took place in Zrich in February. Prior to the meeting, the group was asked to review and consider the published literature on FRI, within nine specific concepts that were then presented for discussion in dedicated sessions during the meeting. The meeting engaged 35 experts and key opinion leaders in the field of FRI. Recommendations were developed on diagnosis and treatment of FRI. These guiding principles will be made available through scientific publications and an AO Bone Infection App.

Internal fracture fixation existed but only in individual hospitals and not globally, that is where ARI and AO came in and rolled this out globally and invented many new additions to this. ARI invented compression plates, minimal invasive surgery for trauma (plates, screws, nails etc.), locking plates for fractures close to articulating joints and for osteoporotic patients.

Currently tissue engineering and regenerative medicine (TERM) is in the research stage of its life cycle and has not really translated into routine surgical practice in orthopaedics. The combination of cells and biomaterials however has great potential in repair. The main issues are again regulatory, and the best way forward would be to develop techniques that can be applied in a single surgery within the operation room. Anything beyond this window and outside the operation room will take a significant amount of time to get approval and will likely not be surgeon friendly and obviously will be very costly.

TERM has its biggest potential in orthopaedics in the areas of cartilage repair (delaying classic orthopaedics), disc regeneration (back pain being one of the largest problems globally) and in bone this could be in large bone defects, but not a major area in fracture repair, where appropriate mechanical stimulation can be used to drive the repair to optimum levels and speed (which is also in the research stage). TERM has also potential in tendon and ligament repair.

Imaging and biomarkers for diagnostics and therapy (Theranostics) will be important in early detection of diseases or complications and then to prevent further development of the disease, delaying the time until classic orthopaedics is required. This may go beyond stopping the disease and towards tissue regeneration. The earlier the detection, the more potential for TERM.

The main challenges for a researcher are in translation and the fact that large companies today exist in a more complex regulatory environment, which means they are inclined to be very risk averse. This means in practice they need to see evidence of benefits or proof-of-concept in a clinical setting. The researchers in turn need to have greater awareness of these regulatory issues relating to medical development and CE approved manufacturers, than in the past. The increasingly complex regulatory environment of course has a greater impact on small companies and spin-offs, and can be seen as having a dampening effect on innovation development. Incremental innovations or solutions to niche problems will struggle to get the funding needed to carry them through the regulatory approval process. Researchers do benefit from this too, since in an environment in which companies are inclined to be more risk averse, they place a higher premium on solutions or concepts that have been through a rigorous clinical testing process. In orthopedics, we are approaching an innovation plateau with metals, and new technologies (such as tissue engineering, which is showing good results in research at present) still need to kick in to date little has translated to the patient in this field. 3D printing may have a place in spine or craniomaxillofacial areas, but offers little benefits to trauma in the most common areas for fracture repair. Surgeons who promote patient specific implants (PSI) in joint replacement have little proof that this offers clear improvements compared to current well-tested and proven joint replacement implants. The seamless integration of digitisation and robotic help into the patient treatment work-flow is another area to grow to help the surgeons in their daily practice.

Prof R. Geoff Richards

Director

AO Research Institute Davos

geoff.richards@aofoundation.org

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https://www.aofoundation.org/Structure/research/exploratory-applied-research/research-institute/Pages/exploratory-applied-research.aspx

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Advancing patient care through innovative orthopaedics - SciTech Europa

Skin Stem Cells Comments Off on Advancing patient care through innovative orthopaedics – SciTech Europa | October 27th, 2019

23 Oct 2019

A multi-institutional group, including members of the Tau Consortium, unveiled a stem cell tool kit for scientists studying primary tauopathies. In the November 12 issue of Stem Cell Reports, researchers co-led by Celeste Karch ofWashington University, St. Louis, and Alison Goate and Sally Temple of Icahn School of Medicine in New York, describe a collection of fibroblasts, induced pluripotent stem cells, and neural precursor cells. The cells come from 140 skin samples, some given by donors with richly documented clinical histories who carry pathogenic MAPT mutations or risk variants. Others come from noncarrier family members, patients with a sporadic tauopathy, and cognitively normal controls. The set includes induced pluripotent stem cell lines from 31 donors and 21 CRISPR-engineered isogenic lines. The cells are available to other researchers for study.

These types of high-quality repositories are becoming increasingly important for the scientific community, Clive Svendsen of the Cedars-Sinai Medical Center in Los Angeles wrote to Alzforum.

This is the way the field is going, agreed Lawrence Golbe of CurePSP, New York. Golbes organization funds research into progressive nuclear palsy (PSP) and related disorders, and collaborates with the Tau Consortium on other projects. Enthusiastic about the resources potential, Golbe hopes CurePSP grantees will get an automatic pass to use the cells.

Choice Mutations. Cells in the new iPSC collection carry some of the most common MAPT mutations, covering a wide range of clinical and neuropathological phenotypes of frontotemporal lobe dementia (FTLD)-Tau. [Courtesy of Karch et al., 2019.]

Tauopathies have proven difficult to study in animal models, in part because unlike other neuropathologies, they seem to afflict only humans (Heuer et al., 2012). Moreover, while adult human brains express approximately equal amounts of the tau spliced isoforms 3R and 4R, rodents produce almost exclusively 4R (Trabzuni et al., 2012). This is problematic. For example, leading proposals to explain how tau mutations cause disease point to abnormalities in splicing and microtubule binding, which differ between isoforms. The models we had been focusing on were not capturing the complexity of MAPT in human cells, said first author Karch. As a result, human induced pluripotent stem cells (iPSCs) have been gaining popularity in the field. The NINDS Human Cell and Data Repository is helping meet the demand by offering iPSC lines derived from 10 patients harboring MAPT mutations.

However, Karch and her collaborators think the field could benefit from a larger and more diverse collection of human cells, including isogenic iPSC lines. To accomplish this, they collected skin samples from 140 people carrying MAPT pathogenic mutations or risk variants, non-mutation carriers, and patients with sporadic PSP or corticobasal syndrome (CBS), most with comprehensive clinical histories. Although a few cells came from the NINDS repository, most came from patients participating in longitudinal studies at the Memory and Aging Center at the University of California, San Francisco, and the Knight Alzheimer Disease Research Center at WashU. The clinical records of most of these patients include detailed neurological and neuropathological workups, as well as fluid biomarkers and neuroimaging data collected from MRI, A-PET, and tau-PET studies.

To capture a broad range of phenotypes associated with some of the most common MAPT mutations, the authors created 36 fibroblast lines and 29 iPSC lines from individuals carrying the P301L, S305I,IVS10+16, V337M, G389R, and R406W mutations, as well as from carriers of the A152T variant, which increases the risk for both PSP and CBS (image above). The latter could be particularly useful for dissecting the mechanisms that underlie the phenotypic differences between the two diseases. The researchers also obtained iPSC lines from two noncarrier family members, and two people who suffered from autopsy-confirmed sporadic PSP. In addition, they stored fibroblast lines from 12 patients with sporadic PSP, five with CBS, 10 with a mixed PSP/CBS presentation, and 69 cognitively normal controls.

Biopsies are available for 27 of the 31 patients whose cells were used to generate iPSCs, and autopsy data for seven, including the two cases of sporadic PSP.

Importantly, the researchers edited 21 iPSC lines using CRISPR/Cas 9. They corrected cells with these mutations: MAPT IVS10+16,P301L, S305I, R406W, and V337M. Conversely, they inserted into control iPSCs these mutations: R5H, P301L,G389R, S305I, or S305S.

The authors also created a stem cell line carrying MAPT P301S,a mutation commonly overexpressed in tauopathy mouse models but not present in the available donors, by editing the P301L line. Isogenic lines are so powerful, particularly in these diseases which are so variable in their onset and progression, even within the same family, said Karch. Gnter Hglinger and Tabea Strauss at the German Center for Neurodegenerative Disease (DZNE) in Munich agreed. Having a pool of cell lines with different disease-linked mutations and risk variants from several individuals and their isogenic control cells is an excellent resource for the research community to enlighten disease mechanisms, they wrote (full comment below).

Several of the reported lines have already starred in recent studies of tauopathy mechanisms and candidate therapies (e.g., Sep 2019 conference news; Nakamura et al., 2019; Hernandez et al., 2019; Silva et al., 2019).

Karch and colleagues have partially differentiated some of the iPSCs and stored them as neural progenitor cells (NPCs), so that researchers can relatively easily thaw, expand, and differentiate them into neurons. These NPCs have proved useful for large-scale functional-genomics studies, proteomics, and genetic modifier screens (e.g., Cheng et al., 2017; Boselli et al., 2017;Tian et al., 2019).

In addition, the authors inserted a neurogenin-2 transgene into two healthy controls and two MAPT mutant stem cells, P301L and R406W. Neurogenin-2 enables low-cost, large-scale differentiation of stem cells into homogenous excitatory neurons. These transgenic cells are particularly useful for high-throughput drug screens (Wang et al., 2017; Sohn et al., 2019).

Researchers can request all the reported cells online at http://neuralsci.org/tau. They must provide a summary of experimental plans, an institutional material transfer agreement, and a nominal fee to cover maintenance and distribution costs. Karch said the process resembles that of the Coriell Institute and the NINDS repository. Our goal is to share with as few hurdles as possible, she said.

While the authors are still reprogramming fibroblasts they have already collected, they also plan to add more causative mutations, generate more isogenic lines, and obtain more cells from members of the same families to help shed light on phenotypic variability. In addition, Karch said, she hopes repository users will resubmit lines with new modifications they generate.

Jeffrey Rothstein, Johns Hopkins University, Baltimore, welcomed the new resource. I think it is great they have assembled this collection, he said. Rothstein founded and co-directs the Answer ALS research project, which has amassed 600 iPSC lines from controls and patients with amyotrophic lateral sclerosis (ALS).

Rothstein suggested the tauopathy collection may want to prioritize adding cells from donors with the most common form of disease, that is, sporadic. His group aims to generate 1,000 iPSC lines, with a large fraction representing sporadic diseasealso the most common form of ALSto identify the most prevalent disease subtypes. One strategy that has helped his group build their collection, he said, is using peripheral blood mononuclear cells instead of fibroblasts to create iPSCs. More donors are willing to donate blood than have a piece of skin punched out. In addition, iPSCs derived from blood cells are genetically more stable, he noted.

Rothstein emphasized the importance of assembling a large collection of healthy controls. Although isogenic controls are of great value, he cautioned they can be subject to artifacts. One problem is that the cell population can change due to selective pressures during CRISPR editing (Budde et al., 2017). To address this, Karch and colleagues are collecting not only modified iPSC clones, but also control clones that have gone through the editing pipeline but remain unmodified.

Stem-cell users studying tauopathies face another challenge: iPSC-derived neurons express primarily the fetal isoform of tau, 3R0N. However, citing a study that shows three-dimensional neuronal cultures switch to the adult profile relatively quickly (Miguel et al., 2019), Hglinger and Strauss wrote, [It] allows us to be optimistic that current challenges of this model system can be overcome in the future.Marina Chicurel

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Introducing: iPSC Collection from Tauopathy Patients - Alzforum

Skin Stem Cells Comments Off on Introducing: iPSC Collection from Tauopathy Patients – Alzforum | October 26th, 2019

Would you live in a city without streets? Or in a flat with no walls? Probably not and the cells in our bodies expect the same level of comfort. Today, were taking a look at the tissues that create and maintain an ideal working environment for our tissues: the extracellular matrix.

Weve had a look at the differences between animal and plant cells before (heres a refresher). One of the key differences between them is that plants reinforce their cells with thick, sturdy walls. These walls are why plant tissues such as wood can get so resilient. However, the reverse of the coin is that it also limits plant cells somewhat: a muscle made out of wood wouldnt be very effective.

Animals need cells that can perform a wide variety of activities, but these cells also need biological and mechanical support to perform their tasks. Thats where the extracellular matrix, or ECM, comes in.

The ECM is a complex mix of proteins and carbohydrates that fills the spaces between cells; it is comprised of the basement membrane and interstitial matrix. Going forward, Ill use the term ECM quite loosely to mean both the extracellular matrix and the interstitial matrix. If I dont mention the basement membrane specifically, Im probably talking about the interstitial matrix (as its the more dynamic and frankly more interesting half of the topic).

Think of the basement membrane as a sheet of plastic wrap the body stretches over every individual tissue or organ to keep everything tidy and in place. This membrane is made up of two layers of cells and its quite fibrous and hard to rip.

The interstitial matrix is, for lack of a better term, the goo that our cells live in. Most of the time, it looks and feels a bit like a clear gel. Its produced by the cells themselves, which secrete and release certain compounds around them.

The simplest definition of the extracellular matrix is that it represents the sum of non-cellular components present within all tissues and organs. As we go forward, keep in mind that the ECM isnt the same everywhere.

Although, fundamentally, the ECM is composed of water, proteins, and polysaccharides, each tissue has an ECM with a unique composition and topology that is generated during tissue development, Christian Frantz, Kathleen M. Stewart, Valerie M. Weaver, 2010.

Collagen, the most abundant protein in mammals, is the main component of the ECM. Outside the cell, collagen binds with carbohydrate molecules and assembles into long molecules called collagen fibrils. These fibrils extend through the ECM and lend flexibility and strength to the material, acting similarly to the role of rebar in reinforcing concrete (which is tough but inflexible). Collagen fibrils are flexible and tough to break, so theyre used to bind together the rest of the ECM. In humans, genetic disorders that affect collagen (such as Ehlers-Danlos syndrome) cause tissues to become fragile and tear easily.

While the ECM contains a wide range of proteins and carbohydrates, another important set of compounds alongside collagen are proteoglycans (groups of proteins tied to simple sugars). Proteoglycans come with many shapes and functions, depending on which proteins and sugars theyre made of, and perform a wide range of tasks in the ECM. They can also bind to each other, to collagen (forming cartilage), or to hyaluronic acid, making them even more versatile. As a rule of thumb, proteoglycans act as fillers and regulate the movement of molecules through the ECM among other functions.

Their overall structure looks like a tree: the sugar part of the polyglycans are twigs set on a branch (the protein), which ties to a trunk made out of polysaccharide (many-sugar) molecules. A class of proteins in the membranes of cells, called integrins, serve as connection ports between the membrane and material in the ECM (such as collagen fibers and proteoglycan-polysaccharide bundles). Beneath the membrane, integrins tie into the cells support girders (the cytoskeleton).

The type of ECM Ive described so far is your run of the mill variety that youll find in skin, around muscle fibers, in adipose tissue (fat), and so on. But each tissue has an ECM that fully supports its function blood plasma is the interstitial matrix of blood. Unlike the ECM of muscles, for example, which is meant to reduce friction and wear in the tissue, blood plasma primarily works as a medium to carry blood cells around. Blood vessels are coated with a basement membrane, and together, they form the ECM of blood. Each type of animal connective tissue has its own type of ECM, even bone.

Seeing as there are many types of ECM out there, it stands to reason that there are many functions they perform. However, by and large, there are a few functions that all ECMs fulfill.

The first and perhaps most important function is that they provide support to tissues, segregate (separate) them, and that they mediate intercellular communication. The ECM is also what regulates a cells dynamic behavior i.e. whether a cell moves around, and how. The ECM keeps cells in place so we dont simply unravel. The connections formed between the ECM and integrins on a cells membrane also function as signaling pathways.

It is also essential for the good functioning of tissues at large. The ECM creates and maintains the proper environmental conditions for cells to develop, multiply, and form functioning tissues. While the exact details are still unknown, the ECM has been found to cause tissue regrowth and healing after injury. In human fetuses, for example, the extracellular matrix works with stem cells to grow and regrow all parts of the human body. Fetuses can regrow anything that gets damaged in the womb, but since babies cant, we suspect that the matrix loses this function after full development. Researchers are looking into applying it for tissue regeneration in adults.

The ECM can also act as a storage space for various compounds. In joints, it contains more hyaluronic acid which in turn absorbs water and acts as a mechanical cushion. ECMs can also store a wide range of cellular growth factors and release them as needed. This allows our bodies to activate cell growth on a dime when needed without having to produce and ship these factors to a certain area.

It also seems to impact cell differentiation and gene expression. Cells can switch genes on or off depending on the elasticity of the ECM around them. Cells also seem to want to migrate towards stiffer areas of the ECM generally (durotaxis) from less-firm ones.

The ECM isnt very well known today, and it definitely goes unsung. But no matter how you cut it, it is a key part of biology as we know it today. Without it, both animals and plants would be formless, messy blobs quite literally. And I dont know about you but I love it when my tissues stay where theyre supposed to, the way theyre supposed to.

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The extracellular matrix, and how it keeps you in tip top shape - ZME Science

Skin Stem Cells Comments Off on The extracellular matrix, and how it keeps you in tip top shape – ZME Science | October 26th, 2019

The 4.5 trillion global food industry is currently being influenced by numerous developments. From how food is designed, grown to how its consumed, the next generation of foodtech entrepreneurs are fighting for its piece of the pie. In the meantime, funding for food tech has skyrocketed and according to a report from Dealroom.co, foodtech has created 35 unicorns globally, with a combined value of 169 billion, of which 30 billion from Europe.

Few innovations introduced by European startups are currently shaping the future of food and give us a glimpse of what the future holds for the food industry.

The Magic of Food Science

One of the most interesting developments in the food industry is the introduction of new origins of food. Have you ever imagined that food could actually be made of electricity, air and water? Well, the Finnish company Solar Foods is here to make you believe it. They have produced a nutrient-rich protein, Solein, with air, water, and electricity as its main resources, laced with bacteria. Solar Foods makes Solein by extracting CO from the air using carbon-capture technology, and then combines it with water, nutrients and vitamins, using 100 percent renewable solar energy. Science fiction? Not so much. More like science fact. The end product looks and tastes like wheat flour, with 50% protein content and 510 % fat and 2025 % carbs. Producing Solein is entirely free from agriculture it doesnt require arable land or irrigation and isnt limited by climate conditions, said Solar Foods. And the best part of it? It will never run out.

Changing the way we source ingredients brings us to the next big thing in food science meat grown in a lab. Lab-grown meat is slowly becoming an alternative food option. A few years ago, Mosa Meat got the worlds attention when it announced the first-ever lab-grown meat burger from cow cells. The spin-off company from Maastricht University introduced the cultured meat in Europe and now, one of the newest companies to enter the market is Higher Steaks. Using state-of-the-art cell culture techniques, the UK-startup develops cell-based meat that has the potential to use 99% less land, 96% less water, 45% less energy and has up to 96% less greenhouse gas emissions, all the while tasting as conventional meat. The company uses stem cells obtained via a small blood sample or a skin patch and patented protocols licensed exclusively from its American university partners, allowing them to reprogramme stem cells into tissues like muscle and fat. A single blood sample could allow indefinite production of many meat products, its website states. Around the world, the demand for clean meat is consistently growing. Optimistically, their pork sausages will reach the market by 2021.

Diet for One and the Birth of Personalised Nutrition

One of the biggest advantages of nutrition in the modern age is personalisation. The basic idea is very simple: we all love food, but which food is good for us? Nutrino can give you the answer. The company unlocks the potential of nutritional data and provides its users with smart, personalised analyses of how their bodies interact with the foods they eat. By using machine learning and artificial intelligence, Nutrinos platform collects, processes, and analyses food-related data from its users, matches it with their ever-growing nutritional database and defines an individualised nutrition profile, called FoodPrint. By knowing your own FoodPrint, you will never again question what to eat. Closely related to the idea of eating the food that suits you best is the freedom to choose it. But in todays hectic world, we often forget about its importance.

Luckily, we have Gousto. Providingusers with 40 recipes on a weekly basis, this cook-at-home meal kit service delivers to your doorstep correctly portioned fresh ingredients matched to each recipe of your choice. Backed by an AI recipe recommendation tool, cooking at home has never been easier. Gousto has setanambitious target of delivering 400 million balanced and nutritious meals by 2025. As consumers growingrequest towards greater convenience in eating fresh foods and leading healthier lives increases, Gousto will reach its goal in no time.The same applies for Frichti, the most-funded food startup in France. Aiming to become the second kitchen of Parisians, Frichti offers healthy, seasonal meals at affordable prices, coupled with a fast delivery service. Now their recipe for success is expanding across Europe.

Innovations in Food Creation: 3D Food Printing

As the world goes digital, its time to digitise our kitchens as well. A Spanish startup called Natural Machines has introduced to the world a 3D food printer by the name of Foodini. Foodini uses fresh ingredients loaded into stainless steel capsules to make foods like pizza, pasta, quiche, pancakes or brownies. Not to be mistaken, a real pizza will not come of Foodini, but the dough for the pizza will be as it was prepared by an Italian grandmother. Foodini simply manages the difficult and/or time-consuming parts of handmade food preparation that often discourage people from cooking at home. The decorative potential of the device is also worth mentioning. From everyday foods to elaborate creations, each piece is visually appealing, inviting Michelin-star restaurants to boost their culinary creativity and elevate the restaurant experience.

The Rise of the Functional Beverage

One bottle. One meal. This is the new norm across Europe. Feed has made sure of that. The French startup has introduced a nutritionally complete and convenient meal packed in a bottle, containing just the right amount of protein, essential fats, carbohydrates, vitamins and minerals. All the nutrients ones body needs. Just add some water, shake it a bit and drink it. Feed is a new form of nutrition that offers you freedom. Healthy, convenient and economical, Feed will simplify your life,said Anthony Bourbon, CEO of Feed. Feed is vegan, gluten-free, lactose-free, GMO-free, nut-free and comes in the form of nutrition bars (100g), drinks (500ml), drink mixes and other products. Holding the reputation of delivering quick nutrition, it seems like meal replacements are here to stay. This just might be the end of food, as we know it.

The Future of Dining is Delivery

Welcome to Keatz, the virtual restaurant without guests. Under the slogan We cook, you enjoy. Keatz has been operating since 2016 as the latest addition to the restaurant delivery marketplace. As one of the pioneers of the Ghost restaurants concept, Keatz is up and running thanks to the ongoing popularity of food delivery platforms. Currently it operates a total of 10 virtual restaurants in Berlin, Munich, Madrid, Amsterdam and Barcelona, focusing exclusively on food made for delivery, with minimal capital expenditure and time. Their idea is rather simple. Why should you do groceries and spend time cooking if you can get a great meal delivered in 20 minutes? Living in an on-demand society, consumers are expecting to get what they need whenever they want, and wherever they want. Food is no exception to that.

Fixing Food Loss with Technology

1.3 billion tonnes of food is wasted every year, taking an enormous toll on the planet. At the same time, hunger remains one of the most urgent development challenges of our time. Luckily,consumers are becoming more environmentally conscious and plus, now they have technology to help them distribute the leftovers. This is what Karma is doing. Helping restaurants, cafes and shops to distribute their surplus food to Karma users who get to buy food at half price or more. By making a shared platform on which customers and food providers co-exist and benefit from each other, Karma has found an effective solution for tackling the issue of food waste. A win -win situation.Over 550 tonnes of food have so far been rescued and counting

Looking for startups innovating in a particular sector?If youre a corporate or investor looking for exciting startups in a specific market for a potential investment or acquisition, check out ourStartup Sourcing Service!

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The food of tomorrow the latest innovations from Europe's foodtech sector - EU-Startups

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CELLINK publishes the quarterly report for the period 1st of June 2019 31st of August 2019 and for the full year of operations 2018-2019. The report is available on the company's website: https://cellink.com/investors/ This press release presents a summary of the report.

Fourth quarter

Full year

Events during the quarter (Jun. 2019-Aug. 2019)

Events during the other financial year (Sept. 2018-May 2019)

Events after the end of the period

For further information, please contact:

Erik Gatenholm, CEO Gusten Danielsson, CFO

Phone: EU +46 73 267 00 00 Phone: +46 70 991 86 04US +1 (650) 515 5566 US +1 (857) 332 2138

Email: eg@cellink.com Email: gd@cellink.com

Important information

This information is such information as CELLINK AB is required to disclose under the EU Market Abuse Regulation. The information was submitted for publication on October 24, 2019 at. 08:30 CET.

This is a translation of the Swedish version of the press release. When in doubt, the Swedish wording prevails.

About CELLINK

CELLINK is the leading 3D bioprinter provider and the first bioink company in the world. We focus on developing and commercializing bioprinting technologies to allow researchers to print human organs and tissues for pharmaceutical and cosmetic applications. Founded in 2016 and active in more than 50 countries, CELLINK is changing the future of medicine as we know it. Visit http://www.cellink.com to learn more. CELLINK is listed on Nasdaq First North Growth Market under CLNK. Erik Penser Bank AB is the companys certified adviser, available by phone at +46 846 383 00 and by email at certifiedadviser@penser.se.

at: certifiedadviser@penser.se.

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Report on Operations 2018-2019: Strategic acquisition and strong closing of the year - BioSpace

Skin Stem Cells Comments Off on Report on Operations 2018-2019: Strategic acquisition and strong closing of the year – BioSpace | October 26th, 2019

DetailsCategory: AntibodiesPublished on Friday, 25 October 2019 10:03Hits: 345

PRINCETON, NJ, USA I October 24, 2019 I Bristol-Myers Squibb Company (NYSE: BMY) today announced that the European Commission (EC) has approved Opdivo (nivolumab) flat dosing schedule of 240 mg infused over 30 minutes every two weeks (Q2W) or 480 mg infused over 60 minutes every four weeks (Q4W) for the adjuvant treatment of adult patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.

The approval of Opdivo two and four-week flat dosing schedule in the adjuvant melanoma setting is an important milestone for patients across the European Union who now have additional treatment flexibility, said Ralu Vlad, Pharm.D, development team lead, product design and delivery, Bristol-Myers Squibb. Bristol Myers-Squibb is committed to empowering patients with cancer and their families to regain control of their lives through more flexible treatment options that fit their individual needs.

About Melanoma

Melanoma is a form of skin cancer characterized by the uncontrolled growth of pigment-producing cells (melanocytes) located in the skin. Metastatic melanoma is the deadliest form of the disease and occurs when cancer spreads beyond the surface of the skin to other organs. The incidence of melanoma has been increasing steadily for the last 30 years. In the United States, 91,270 new diagnoses of melanoma and more than 9,320 related deaths are estimated for 2018. Globally, the World Health Organization estimates that by 2035, melanoma incidence will reach 424,102, with 94,308 related deaths. Melanoma is mostly curable when treated in its very early stages; however, survival rates are roughly halved if regional lymph nodes are involved. Patients in the United States diagnosed with advanced melanoma classified as Stage IV historically have a five-year survival rate of 15% to 20% and a 10-year survival of 10% to 15%.

Adjuvant Therapy in Melanoma

Melanoma is separated into five staging categories (Stages 0- IV) based on the in-situ feature, thickness and ulceration of the tumor, whether the cancer has spread to the lymph nodes, and how far the cancer has spread beyond lymph nodes.

Stage III melanoma has generally reached the regional lymph nodes but has not yet spread to distant lymph nodes or to other parts of the body (metastasized) and requires surgical resection of the primary tumor as well as the involved lymph nodes. Some patients may also be treated with adjuvant therapy. Despite surgical intervention, most patients experience disease recurrence and progress to metastatic disease.

Bristol-Myers Squibb: Advancing Oncology Research

At Bristol-Myers Squibb, patients are at the center of everything we do. The focus of our research is to increase quality, long-term survival for patients and make cure a possibility. Through a unique multidisciplinary approach powered by translational science, we harness our deep scientific experience in oncology and Immuno-Oncology (I-O) research to identify novel treatments tailored to individual patient needs. Our researchers are developing a diverse, purposefully built pipeline designed to target different immune system pathways and address the complex and specific interactions between the tumor, its microenvironment and the immune system. We source innovation internally, and in collaboration with academia, government, advocacy groups and biotechnology companies, to help make the promise of transformational medicines, like I-O, a reality for patients.

About Opdivo

Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the bodys own immune system to help restore anti-tumor immune response. By harnessing the bodys own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.

Opdivos leading global development program is based on Bristol-Myers Squibbs scientific expertise in the field of Immuno-Oncology, and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.

In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Companys Opdivo and Yervoy combination regimen was the first Immuno-Oncology combination to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.

U.S. FDA-APPROVED INDICATIONS FOR OPDIVO

OPDIVO (nivolumab) as a single agent is indicated for the treatment of patients with unresectable or metastatic melanoma.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.

OPDIVO (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.

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

OPDIVO (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the treatment of patients with intermediate or poor risk, previously untreated advanced renal cell carcinoma (RCC).

OPDIVO (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

OPDIVO (nivolumab) is indicated for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.

OPDIVO (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

OPDIVO (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

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

OPDIVO (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.

Checkmate Trials and Patient Populations

Checkmate 037previously treated metastatic melanoma; Checkmate 066previously untreated metastatic melanoma; Checkmate 067previously untreated metastatic melanoma, as a single agent or in combination with YERVOY; Checkmate 017second-line treatment of metastatic squamous non-small cell lung cancer; Checkmate 057second-line treatment of metastatic non-squamous non-small cell lung cancer; Checkmate 032small cell lung cancer; Checkmate 025previously treated renal cell carcinoma; Checkmate 214previously untreated renal cell carcinoma, in combination with YERVOY; Checkmate 205/039classical Hodgkin lymphoma; Checkmate 141recurrent or metastatic squamous cell carcinoma of the head and neck; Checkmate 275urothelial carcinoma; Checkmate 142MSI-H or dMMR metastatic colorectal cancer, as a single agent or in combination with YERVOY; Checkmate 040hepatocellular carcinoma; Checkmate 238adjuvant treatment of melanoma.

About the Bristol-Myers Squibb and Ono Pharmaceutical Collaboration

In 2011, through a collaboration agreement with Ono Pharmaceutical Co., Bristol-Myers Squibb expanded its territorial rights to develop and commercialize Opdivo globally, except in Japan, South Korea and Taiwan, where Ono had retained all rights to the compound at the time. On July 23, 2014, Ono and Bristol-Myers Squibb further expanded the companies strategic collaboration agreement to jointly develop and commercialize multiple immunotherapies as single agents and combination regimens for patients with cancer in Japan, South Korea and Taiwan.

About Bristol-Myers Squibb

Bristol-Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol-Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.

SOURCE: Bristol-Myers Squibb

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European Commission Approves Opdivo (nivolumab) Four-Week Dosing Schedule for the Adjuvant Treatment of Adult Patients with Melanoma with Involvement...

Skin Stem Cells Comments Off on European Commission Approves Opdivo (nivolumab) Four-Week Dosing Schedule for the Adjuvant Treatment of Adult Patients with Melanoma with Involvement… | October 26th, 2019

Patients with ultra-rare bone marrow disease are set to benefit from 1.15m grant from LifeArc and The Aplastic Anaemia Trust. The grant will support researchers from Kings College London and Kings College Hospital to test a personalised treatment for aplastic anaemia patients who have not responded to available therapies

The grant has been awarded to investigate the potential of a novel type of personalised cellular therapy to reverse the ultra-rare condition aplastic anaemia (AA). The results of this research could give new hope to people living with a severe, life-limiting form of this condition.

The grant will fund a clinical trial to investigate the safety and efficacy of using a patients own T-reg cells to restore the blood-making function of the bone marrow. This follows laboratory-based research from the team of scientists where T-reg cells from a patients own blood were collected, selected for activity and multiplied. In a test tube, these cells prevented the immune system from attacking the patients bone marrow stem cells.

AA is an ultra-rare life-threatening illness caused by the bone marrow failing to make enough of all three types of blood cells red blood cells, white blood cells and platelets. Only around 100-150 people in UK are diagnosed per year, affecting all ages but most commonly people between the ages of 10 to 20 years old and those over the age of 60 years.

People with the illness are at greater risk of infections, bleeding, and can experience extreme fatigue, which leaves them unable to carry out simple daily tasks that most people take for granted. Around one in three patients with severe AA fail to respond to existing drug treatments and the other option a bone marrow transplant is reliant on finding a suitable donor, requires life-long treatment with immunosuppression therapy and is unsuccessful in one in three people.

The trial at Kings College London and Kings College Hospital will run for three years and aims to recruit nine patients. A blood sample of the patients T-reg cells will be extracted, purified and grown in the lab before being given back to them in a higher concentration. As patients with AA are more susceptible to infection, this personalised treatment approach is more likely to avoid the risk of severe infection and inflammation.

Professor Ghulam Mufti, Department of Haematological Medicine at Kings College London and Kings College Hospital, and lead study investigator said: For patients with this ultra-rare disease, were looking for the first time at a personalised medicine approach where their own immune cells could be used to alter their disease. In AA there is a reduction in the number of T-regs and most of the ones that the AA patients do have are non-functional. Weve seen success in the laboratory by selecting and bolstering the number of functional T-reg cells. Now, with funding from LifeArc and the AAT, we can investigate the potential of this approach in treating AA patients who currently have very limited treatment options.

Dr Catriona Crombie, LifeArcs Head of Philanthropic Fund explained why the charity had approved the funding: LifeArc set up the Philanthropic fund to support translational research into rare diseases, where there is less interest from commercial organisations. Patients with AA can have limited treatment options; this opportunity with Kings College London, Kings College Hospital and the AAT has the potential to transform the lives of patients living with a severe form of the disease.

Grazina Berry, Chief Executive of the AAT said: AA can severely impact a persons quality of life. Through AATs close work with Kings College London and Kings College Hospital as a specialist centre of clinical care and research in AA, we identified the project with the most potential to directly benefit patients who are currently at a loss for solutions. We are delighted to have partnered with LifeArc and Kings College London and Kings College Hospital to progress this ground-breaking work, which could potentially enable people living with severe AA to once again lead a normal life.

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New funding to test personalised treatment for aplastic anaemia - Pharmafield

Bone Marrow Stem Cells Comments Off on New funding to test personalised treatment for aplastic anaemia – Pharmafield | October 26th, 2019

A gene therapy for a rare blood disorder has shown what the manufacturer calls the first evidence of long-term improvement associated with the disease.

New York-based Rocket Pharmaceuticals said Thursday that it had presented long-term follow-up data from the Phase I/II study of RP-L102, its gene therapy for Fanconi anemia, at the annual congress of the European Society of Cell and Gene Therapy in Barcelona, Spain. The company said it represented the first evidence of long-term improvement and stabilization in blood counts and durable mosaicism among patients who received the therapy without the use of the conditioning regimens normally used for allogeneic stem cell transplants, which the company calls Process A.

Shares of Rocket were up slightly on the Nasdaq following the news. RP-L102 is a lentiviral vector-based gene therapy. Most other gene therapies in development, and both of the currently marketed ones Spark Therapeutics Luxturna (voretigene neparvovec-rzyl) and Novartis Zolgensma (onasemnogene abeparvovec-xioi) are adeno-associated viral vector-based.

According to the data, representing four of nine patients, there were improved blood counts and long-term bone marrow mitomycin C (MMC) resistance, thereby indicating durable phenotypic correction. The data met or exceeded a 10 percent threshold that the company said the Food and Drug Administration and European Medicines Agency had agreed to for its upcoming Phase II registration study, for which it plans to start enrolling patients by the end of the year.

FA is a rare, genetic bone marrow failure disorder, half of whose patients are diagnosed before the age of 10, while about 10 percent of patients are diagnosed as adults, according to the National Organization for Rare Disorders. It is often associated with progressive deficiency of production of red and white blood cells and platelets in the bone marrow and can eventually lead to certain solid and liquid tumor cancers. It occurs in 1-in-136,000 births and is more common among Ashkenazi Jews, Spanish Roma and black South Africans.

These results indicate the feasibility of engraftment in FA patients using autologous, gene corrected [hematopoietic stem cells] in the absence of any conditioning regimen, said Dr. Juan Bueren, scientific director of the FA gene therapy program at Spains Center for Energy, Environmental and Technological Research, in a statement. This indicates the potential of this therapeutic approach as a definitive hematologic treatment, while avoiding the burdensome side effects associated with allogeneic transplant, including the risk of post-transplant mortality and a substantially higher risk of head and neck cancer.

Photo: virusowy, Getty Images

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Rocket's gene therapy shows long-term efficacy in rare blood disorder - MedCity News

Bone Marrow Stem Cells Comments Off on Rocket’s gene therapy shows long-term efficacy in rare blood disorder – MedCity News | October 26th, 2019

The worlds first cell atlas of the human developmental liver has been created, giving fresh insight into how the blood and immune systems develop in the foetus.

A high-resolution resource, it will aid our understanding of normal development and efforts to tackle diseases that can form during development, such as leukaemia and immune disorders.

The cell atlas maps how the cellular landscape within the developing liver changes between the first and second trimesters of pregnancy, including how stem cell from the liver seed other tissues, supporting the high demand for oxygen required for growth.

Researchers from the Wellcome Sanger Institute in Hinxton, the Wellcome MRC Cambridge Stem Cell Institute, University of Cambridge, Newcastle University and their collaborators created the atlas by using single cell technology to analyse 140,000 liver cells and 74,000 skin, kidney and yolk sac cells.

In adults, it is bone marrow that is primarily responsible for the creation of blood and immune cells in a process called haematopoiesis.

In early embryonic life, the yolk sac and liver play a key role in creating these cells, which then seed peripheral tissues such as skin, kidney and ultimately bone marrow.

But until now, the precise process of how blood and immune systems develop in humans has been unknown.

Isolating cells from the developing liver, the researchers were able to identify them by what genes they were expressing and discover what the cells looked like.

They tagged haematopoietic cells in sections of developmental liver using heavy metal markers in order to map them to their location.

Prof Muzlifah Haniffa, a senior author of the study from Newcastle University and senior clinical fellow at the Wellcome Sanger Institute, said: Until now research in this area has been a little bit like blindfolded people studying an elephant, with each describing just a small part of it.

This is the first time that anyone has described the whole picture, how the blood and immune systems develop in such detail. Its been an extraordinary, multidisciplinary effort that is now available as a tool for the whole scientific community.

The scientists learned that during foetal development, mother haematopoietic stem cells stay in the liver. But the liver alone cannot supply enough red blood cells, so the next generation daughter cells called progenitor cells travel to other tissues, maturing in places such as the skin. Thee, they develop into red blood cells to help meet the high demand for oxygen in the developing foetus.

Dr Elisa Laurenti, a senior author from the Wellcome MRC Cambridge Stem Cell Institute and the Department of Haematology at the University of Cambridge, said: We knew that as adults age our immune system changes. This study shows how the livers ability to make blood and immune cells changes in a very short space of time, even between seven and 17 weeks post-conception.

If we can understand what makes the stem cells in the liver so good at making red blood cells, it will have important implications for regenerative medicine.

The study, published in Nature, also involved the mapping of genes involved in immune deficiencies to reveal which cells were expressing them.

It is known that gene mutations can lead to immune disorders such as leukaemia.

A better understanding of the development of healthy liver functions could aid our understanding of how to treat such conditions.

The work is part of the ambitious effort to create the first complete Human Cell Atlas.

Dr Katrina Gold, genetics and molecular sciences portfolio manager at Wellcome, said: Our immune system is vital in helping to protect us from disease, yet we know very little about how immune cells develop and behave in the early embryo. This study is hugely important, laying a critical foundation for future research that could help improve our understanding of disorders linked to the early immune system, such as childhood leukaemias.

The Human Cell Atlas has the potential to transform our understanding of health and disease and were excited to see these first discoveries from our Wellcome-funded multidisciplinary team of scientists.

Dr Sarah Teichmann, a senior author from the Wellcome Sanger Institute, University of Cambridge and co-chair of the Human Cell Atlas organising committee, said: The first comprehensive cellular map of the developmental liver is another milestone for the Human

Cell Atlas initiative.

The data is now freely available for anyone to use and will be a great resource to better understand healthy cellular development and disease-causing genetic mutations.

Read more

Asthma treatment hope as Human Cell Atlas project creates first map of lungs

Sanger Institute scientist helps unveil blueprint for extraordinary Human Cell Atlas

AstraZeneca and Cancer Research UK launch joint Functional Genomics Centre in Cambridge

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The build-out is estimated at $9.5M.

United Therapeutics received a building permit Tuesday for a $9.5M build-out of its cell therapy facility on the second floor of Mayo Clinics Discovery and Innovation Building.

The 21,843-square-foot space will house an automated stem cell manufacturing site, which is one of the first of its kind in the country. The Whiting-Turner Contracting Co. is the project contractor.

The technology, approved by the FDA in 2018, allows the Mayo Clinic Center for Regenerative Medicine to produce cells from the bone marrow of a stem cell donor in large enough quantities to be used as treatments in clinical trials. It allows for the treatment of multiple patients at the same time.

Construction began in 2017 on the $32.4M building at 14221 Kendall Hench Drive. It held a grand opening in August.

The first floor houses three ex-vivo lung perfusion surgical suites used for lung restoration, another form of regenerative medicine. It turns donor lungs, which previously would have previously been unusable, into viable transplant organs. United Therapeutics also collaborates with Mayo Clinic on lung restoration.

The third floor houses the Life Sciences Incubator for biotech entrepreneurs, which offers coworking space, wet labs, business resources, networking and entrepreneurial training.

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United Therapeutics Receives Permit For Cell Therapy Facility Build-Out At Mayo - Pharmaceutical Online

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