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Mending the gap: U of T’s Molly Shoichet joins team developing new treatments for spinal cord injuries – News@UofT

By daniellenierenberg

Spinal cord injuries can be devastating and there are currentlyfew options to reverse the effects, which can include paralysis, chronic pain and loss of bladder control.

But an international team of researchers, including the University of Torontos Molly Shoichet,hopes to change that.

Over the past few years, weve made a lot of progress in tissue engineering, drug delivery and regenerative medicine, says Shoichet, a University Professor in the department of chemical engineering and applied chemistry in the Faculty of Applied Science & Engineering, the Institute of Biomedical Engineering and the Donnelly Centre for Cellular and Biomolecular Research.

With this ambitious project, we bring world leading experts together to try to do something that no one else has been able to do: promote repair and regeneration in the injured spinal cord.

Shoichet is a co-principal investigator withMend the Gap, an international collaboration of more than 30 researchers, engineers, scientists, surgeons and social scientists from Canada, the United States, Europe and Australia. The collaboration this week received $24 million from Canadas New Frontiers in Research Fund to advance their work.

The team takes its name from the fact that only a small gap, just a few centimetres long, is responsible for blocking the nerve impulses that normally flow through the spinal cord. Bridging that gap requires collaboration from some of the worlds top experts in a wide range of fields.

Shoichet is known internationally for her work on hydrogels biocompatible materials that can help facilitate tissue repair. Hydrogels can function as scaffolds, enhancing or augmenting natural processes that serve to repair damaged tissue.

Hydrogels can also serve as controlled-release mechanisms for drugs that aid healing, or to protect stem cells that are being injected into the body bykeeping them alive and healthy while they integrate into damaged tissues.

Another important line of research involves dealing with the glial scar that forms in the wake of a spinal cord injury. In the short term, this protective shield of cells and biochemicals prevents further injury in the damaged nerve, but in the long termit can serve as a barrier to nerve repair.

Shoichet and her team bring their expertise in hydrogels and local delivery strategies to deliver innovative biomolecules locally and directly to the injured spinal cord. For example, shere-engineered an enzymeto selectively degrade some of the biomolecules that make up the glial scar. This redesigned enzyme is both more stable and more active than the wild type.

By breaking through the glial scar with this new delivery strategy, the enzyme can enable other therapies from advanced drugs to stem cells to further promote tissue regeneration and repair.

The environment in the injured spinal cord is a very complicated place, says Shoichet. There are a whole range of natural processes at work some of which we want to enhance, others of which we need to find ways to circumvent. I am very excited to be part of this multidisciplinary team, which has the breadth and depth of expertise that we need to make a real difference when it comes to treating spinal cord injury.

Shoichet is the only person to be elected a fellow of all three of Canadas national academiesand is a foreign member of the U.S. National Academy of Engineering and a fellow of the Royal Society of London. She was the 2020 recipient of the Gerhard Herzberg Canada Gold Medal, Canadas highest honour for science and engineering research. She is also a member of the Order of Ontario and an Officer of the Order of Canada.

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Scots mum with MS says 50k treatment abroad is ‘last hope’ of halting disease – Daily Record

By daniellenierenberg

A mum who has lived with multiple sclerosis for over a decade says a 50,000 treatment unavailable on the NHS could be her last shot at living a life largely unhindered by the disease.

Jodie McQuillian, 32, was formally diagnosed with the chronic condition in 2015, a few years after the first signs appeared when she temporarily lost vision in her left eye.

Since then, she has undergone multiple treatments and bouts of physiotherapy in order to stave off relapses of the condition.

But the mum of one faces life in a wheelchair if she can't put a halt to the rampant flare-ups.

Multiple sclerosis, often known as MS, is a condition where the immune system mistakenly attacks nerves around the brain and spinal cord, affecting the body's ability to transmit signals properly.

Each time Jodie "relapses" - when her body launches a new attack on itself - she finds herself sapped of energy and often experiences issues with her sight and mobility.

It takes her months to recover from each flare-up, affecting the time she can spend with son Ethan, five, and her family.

And every time there's a relapse, a little bit of her doesn't come back.

Jodie, from Alloa, told the Record: "I've just had another relapse and everything I'm trying isn't really effective enough.

"Since I started my newest treatment my walking has gotten a lot worse.

"Every time you relapse you recover but it takes months and you get put on a high dose of steroids and that drains you of all your muscle.

"I'm always left a wee bit damaged from a relapse - and when I feel like I've sort of recovered they flare up again."

After experiencing a major flare-up when she gave birth to Ethan, Jodie began undergoing treatment for MS, trying every drug available on the NHS in a bid to reduce the risk of relapsing.

Despite trying Copaxone injections, Tecfidera tablets and Ocrevus infusions through a drip in her arm over the course of five years, the setbacks have continued and Jodie's outlook is bleak.

There is currently no cure for MS and while her condition is currently recurring intermittently, it is likely to become progressive later in life with little hope of recovery.

However, her last hope may lie in a new treatment known as haematopoietic stem cell transplantation, or HSCT.

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Backed by the MS Society, it is an intense chemotherapy procedure that aims to "reset" Jodie's immune system and stop it from attacking her brain and spinal cord using stem cells found in bone marrow.

The treatment was approved for use on the NHS in Scotland in 2019 but the criteria is so tight that even Jodie, with her frequent relapses, doesn't qualify for it.

Her only option has been to go private abroad - at a cost of 50,000 - and her sister Tricia has launched a GoFundMe page to crowdfund the costs of the operation.

Since publishing the page last week, over 13,000 has been donated by well-wishers, giving Jodie hope she can put a halt to her body's war against itself.

HSCT won't fix the damage done to nerves nerves in the last decade, and is not without its risks, with side-effects such as increased risk of developing cancer.

However, it should put a stop to further degeneration - and serves as Jodie's last hope to live a life relatively free of MS.

"Every relapse is like setting my body up from scratch, and it happens again and again," she added.

"If my MS becomes progressive there's not a lot of treatment available for that all.

"I know from my own research it's beneficial doing the treatment sooner rather than later - and I'm too young not to try it now.

"My next relapse will probably put me in a wheelchair. But you wouldn't look at me 90% of the time and think there's anything wrong.

"To be honest, I feel like this is the last hope. This is the most extreme treatment you can get - it's chemotherapy.

"There's not much else I can do after this but it can't wait.

"It won't be an easy fix, it's not a bounce back - but it will stop the progression, and that's the goal.

"I want to be able to live my life - that's all I ask."

Jodie's sister Tricia Moran, who spearheaded the fundraising appeal, said: "Watching Jodie go through that first episode...it was quite heartbreaking as a family to watch.

"We didn't get any answers for a long time and it really impacted on her confidence - we couldn't reassure her and felt quite helpless.

"As a family we've seen her struggle so much with her relapses and she's so aware of what she's lost.

"She can't just take Ethan to the toy shop on a whim - everything has to be planned.

"It's an understatement to say how overwhelmed we have been by the kindness of friends, family and complete strangers."

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UC Davis researchers find dual cytokine blockage as a novel treatment against graft-versus-host disease in blood stem cell transplantations – The…

By daniellenierenberg

Cytokines TNF and IL-6 can cause severely damaging inflammatory effects as a result of stem cells attacking host cells of blood cancer patients

By BRANDON NGUYEN science@theaggie.org

Researchers at the UC Davis Medical Center recently discovered a novel treatment against graft-versus-host disease (GVHD), a potentially lethal inflammatory condition that can arise following stem cell transplantation, which treats blood cancers and disorders. Allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT) to treat some blood cancers and disorders involves injecting a donors bone marrow stem cells, also known as graft, into blood cancer patients undergoing chemotherapy and radiation therapy.

Dr. William Murphy, a professor at the UC Davis School of Medicine under the Department of Dermatology and Internal Medicine and senior author of the study, further explained what GVHD is under the context of blood cancers.

If we take stem cells from another source, usually trying to match as much as we can from a related source such as a sibling, there seems to be an anti-tumor effect, Murphy said. This desired, beneficial effect from stem cell transplantation is called the graft-versus-tumor (GVT) effect. But the graft-versus-host disease means those immune cells can also attack not just the cancer, but the recipient or patient, which occurs pretty often.

The medical dilemma Murphy and his team of researchers faced involved maximizing GVT effects while minimizing GVHD during stem cell treatment to help the patient effectively fight off the tumor. Logan Vick, a graduate student under Murphys lab at the UC Davis Medical Center and a co-author of the study, talked about the major findings that help minimize GVHD in allo-HSCT patients.

In graft-versus-host disease, something that can be picked up as a symptom is this release of cytokines, which are inflammatory proteins, Vick said. TNF and IL-6, which are two inflammatory cytokines, are often used as tools of the immune system to combat either viruses or different pathogens, but prolonged inflammation can have consequences. So by blocking these two cytokines, what we call a dual cytokine blockade, can help ameliorate GVHD.

The cytokines, TNF and IL-6, that Vick focuses on can cause a cytokine storm, which can occur during GVHD when donor immune stem cells attack the hosts healthy cells instead of the tumor and induce inflammation caused by cytokines. GVHD and the dangerous cytokine storm effect has been a problem for stem cell transplantation treatments, but Murphys team of researchers have just found a potential cure to GVHD while still maintaining the efficacy of the treatment.

Lam T. Khuat, a postdoctoral researcher at Murphys lab and the first author of the study, summarized the beneficial results from dual cytokine blockage.

Many treatments for GVHD involve suppressing the bodys immunity, which can inhibit beneficial GVT effects, Khuat said via email. For this reason, it was important to determine if blocking these cytokines impacted the GVT response. Fortunately, anti-tumor effects remained after the transplant and with the combined intervention.

Clinical methods have often employed single cytokine blockades; however, with the novel finding that dual cytokine blockades can minimize the proinflammatory responses induced by GVHD, the treatment can also be applied in other health conditions that require stem cell transplantation or reducing inflammatory side effects.

Normally, when you have an overactive immune system, whether its autoimmune disorders or GVHD or even in viral infections, the treatments sometimes blanket immunosuppression with steroids, Murphy said. Well, that works because they turn off the immune system, but in the case of cancer and viral infections, you want a working immune system. The beauty of using this double block, which can be applied in other clinical settings, is it doesnt suppress the immune system while preventing the inflammation and the damage.

Written by: Brandon Nguyen science@theaggie.org

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Bone Marrow-Derived Stem Cells (BMSCS) Market Size Is Expected To Generate Huge Revenue and Competitive Outlook Industrial IT – Industrial IT

By daniellenierenberg

Bone Marrow-Derived Stem Cells (BMSCS) Marketanalysis report gives a clear idea on various segments that are relied upon to observe the quickest business development amid the estimated forecast frame. This report indicates a professional and all-inclusive study of the market which concentrates on primary and secondary drivers, market share, competitor analysis, leading segments and geographical analysis. With the particular base year and the historic year, definite estimations and calculations are carried out in this business report. The globalBone Marrow-Derived Stem Cells (BMSCS) Marketreport displays a comprehensive study on production capacity, consumption, import, and export for all the major regions across the globe.

The report refers to standard research methodologies to offer entire and precise market analysis, statistical assessment and an upright industry projection. The Bone Marrow-Derived Stem Cells (BMSCS) market report offers a profound study derived from various analytical tools that elaborate about forthcoming opportunities to facilitate strategic and tactical business decisions to improve profitability. The report provides such enlightenment of the Bone Marrow-Derived Stem Cells (BMSCS) industry that helps to monitor the performance of the market is surrounded by the rapid evolvements and aggressive competitiveness.

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Bone marrow-derivedstem cells(BMSCS) market is expected to gain market growth in the forecast period of 2020 to 2027. Data Bridge Market Research analyses the market to growing at a CAGR of 10.4% in the above-mentioned forecast period. Increasing awareness regarding the benefits associates with the preservation of bone marrow derived stem cells will boost the growth of the market.

Later on, the report assesses gross sales (volume & value), market share, market size, market growth rate based variety of applications.The Bone Marrow-Derived Stem Cells (BMSCS) report also focuses on regional and provincial markets to analyze manufacturers, niche market segments, industry environment, raw material resources, and rivalry of the specific marketplace.

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Table of Content:

Chapter 1: Bone Marrow-Derived Stem Cells (BMSCS) Overview, Product Overview, Market Segmentation, Market Overview of Regions, Market Dynamics, Limitations, Opportunities and Industry News and Policies.

Chapter 2: PEST (Political, Economic, Social and Technological) Analysis of Bone Marrow-Derived Stem Cells (BMSCS) Market.

Chapter 3: Value Analysis, Production, Growth Rate and Price Analysis by Type of Bone Marrow-Derived Stem Cells (BMSCS).

Chapter 4: Downstream Characteristics, Consumption and Market Share by Application of Bone Marrow-Derived Stem Cells (BMSCS).

Chapter 5: Production Volume, Price, Gross Margin, and Revenue ($) of Bone Marrow-Derived Stem Cells (BMSCS) by Regions.

Chapter 6: Bone Marrow-Derived Stem Cells (BMSCS) Production, Consumption, Export, Market Trends and Competitive Landscape.

Chapter 7: Bone Marrow-Derived Stem Cells (BMSCS) Market Status and SWOT Analysis by Regions.

Chapter 8: Competitive Landscape, Product Introduction, Company Profiles, Market Distribution Status by Players of Bone Marrow-Derived Stem Cells (BMSCS).

Chapter 9: Bone Marrow-Derived Stem Cells (BMSCS) Market Analysis and Forecast by Type and Application.

Chapter 10: Market Analysis and Forecast by Regions.

Chapter 11: Industry Characteristics, Key Factors, New Entrants SWOT Analysis, Investment Feasibility Analysis.

Chapter 12: Market Conclusion.

Chapter 13: Appendix Such as Methodology and Data Resources of This Research.

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Covid-19 immunity: How antibodies, B cells, and T cells tackle omicron – Vox.com

By daniellenierenberg

Over the past two years, the United States has seen more than 63 million Covid-19 cases, with some people infected more than once. More than 240 million people in the US have received at least one dose of a Covid-19 vaccine. More than 60 million have received three.

While Covid-19 infections are never a good thing, these numbers still add up to a glimmer of good news: A large majority of Americans now have some immunity against SARS-CoV-2, the virus that causes Covid-19. Thats a big step toward defanging the disease.

When the human body is infected by the virus or encounters a fragment of the pathogen in a vaccine, our immune systems change in subtle but important ways. Across a huge swath of the population, these changes could eventually help transform Covid-19 from a world-stopping catastrophe into a mild annoyance.

Antibodies, proteins that attach to the virus, are a critical part of the immune response and are often the center of discussions about protection from Covid-19. But they rise during infection and decline naturally over time. Fortunately, antibodies are not the whole story when it comes to the immune system.

Other, longer-lasting tools against infection are hiding inside our bones. The immune system draws on stem cells living in bone marrow to produce an array of components that we dont hear as much about. They form many kinds of white blood cells that jump into action right away when they encounter a virus for the first time, and that essentially take notes to start planning for the next infection.

Its this immune system memory thats key to long-term protection against Covid-19. Whats reassuring is that as white blood cells get more practice against SARS-CoV-2, they seem to get better at containing the virus even when it evolves into new variants. That appears to be happening in the omicron wave of Covid-19.

Omicron is the most transmissible variant of the coronavirus known to date. It also appears to be better at dodging immune protection from Covid-19 vaccines. Cases have reached record levels in many parts of the United States, and hospitals are once again straining under the burden.

But the fraction of cases leading to hospitalizations and deaths appears to be far smaller compared to other variants. While there are more reports of breakthrough infections and reinfections with omicron, many previously exposed people report mild, cold-like symptoms.

One reason is that the virus itself appears to have mutated in a way that leads to fewer dangerous complications. Yet its also clear that widespread immunity is absorbing some of the worst effects of the disease, a hopeful trend that is likely to continue in 2022 and beyond.

The world is full of so many things that can make us sick viruses, bacteria, parasites, fungi, even mutated versions of our own cells. The threats are varied and unrelenting, but so too is our immune system. Its an orchestra of cells, proteins, organs, and pathways that all harmonize to keep invaders at bay. In simplified form, heres how.

When a pathogen like the coronavirus enters the body for the first time, it confronts the innate immune system, which provides generalized protection against all pathogens, but isnt always enough to prevent illness on its own. After an infection takes root, the immune system launches a more targeted response with whats known as the adaptive immune system.

Neutralizing antibodies form the pillar of the adaptive immune system. The virus is studded with spike proteins (giving it its namesake corona, meaning crown in Latin), which attach to human cells to begin the infection process. Y-shaped antibodies can attach to the spikes on the virus and prevent it from entering cells, thereby neutralizing the pathogen. The parts of a virus that can trigger an immune response are known as antigens.

In general, neutralizing antibodies keep you from getting infected in the first place, said Lewis Lanier, chair of the microbiology and immunology department at the University of California San Francisco.

Neutralizing antibodies are picky about the parts of the virus they recognize, known as epitopes. If those attachment points on the virus change, as they do in many coronavirus variants, antibodies can become less effective. In the months following an infection or immunization, the amount of these neutralizing antibodies declines as well. Thats expected. Making antibodies takes a lot of energy, so the body makes fewer of them after an infection is gone.

That decline may sound worrisome, but the immune system has other powerful tools in its shed. To start, there are non-neutralizing antibodies. These dont directly interfere with how the virus functions, but they can help the immune system detect infected cells and mark them for destruction. This is a crucial task because viruses cant make copies of themselves on their own: They need to commandeer a host cell to reproduce. Once a virus enters a cell, its not accessible to neutralizing antibodies, but non-neutralizing antibodies that learned to recognize infected cells can still raise the alarm.

The task of eliminating infected cells falls to a group of white blood cells known as cytotoxic T cells, sometimes called killer T cells. They arise from stem cells in bone marrow and cause infected cells to self-destruct, without messing with normal cells.

T cells, they cannot prevent infection, said Lanier. The only way a T cell can recognize you have an infection is after a cell has been infected.

Helper T cells are another important white blood cell variety. They spur the production of antibodies by a different group of white blood cells called B cells. B cells form in bone marrow and then migrate to lymph nodes or the spleen.

After an infection or a vaccination, some B cells and T cells stick around, becoming memory B cells and T cells. They sit idle, sometimes for decades, waiting to see if a pathogen returns. If it does, they can quickly reactivate.

This is why we a decline in neutralizing antibody counts isnt always a disaster. Even if concentrations of neutralizing antibodies dip so low that they can no longer prevent an infection, other parts of the immune system can spool up to make sure the virus doesnt do too much damage.

There is a window of time after virus gets into the body before it really starts manifesting disease in the person, said Deborah Fuller, a professor of microbiology at the University of Washington School of Medicine. That window of time enables the immune system that has been vaccinated and has memory immune responses to recall very quickly and shut down the virus before it actually causes disease.

Some health officials now say that Covid-19 is so rampant that most people are likely to become infected at some point. Its hard to process whats actually happening right now, which is most people are going to get Covid, Janet Woodcock, acting commissioner of the Food and Drug Administration told the Senate health committee on Tuesday. What we need to do is make sure the hospitals can still function, transportation, other essential services are not disrupted while this happens.

However, waves of infection can crest just as quickly as they form. Countries like the United Kingdom and South Africa experienced awful omicron spikes but subsequently saw precipitous drops in cases thereafter. Omicron cases also appear to be leveling off in some parts of the US, a sign that a decline may be ahead.

Whether these spikes in Covid-19 cases lead to severe health outcomes hinges on the teamwork of B cells, T cells, and antibodies, and how they hold up against any new mutations in the virus. Its an area of active research for scientists.

Vaccines and prior infection may not prevent you from being infected by the next waves of variants, but it may well keep you out of the hospital, Lanier said.

For the past two years, with recurring spikes in Covid-19 cases, neutralizing antibodies have taken center stage. Were really more concerned right now in the middle of the pandemic about the durability of that antibody because what were trying to do is prevent transmission, said Fuller. But that could change.

Neutralizing antibodies remain a key benchmark for vaccines: Scientists judge the success and timing of vaccines in part by measuring the number of antibodies they provoke in our blood, and how long the antibodies stick around. When the mRNA vaccines from Moderna and Pfizer/BioNTech were in development, they demonstrated that they could elicit a high level of neutralizing antibodies. Further clinical trials showed that this translated to more than 90 percent efficacy in preventing illness.

The next test is how well antibody production ramps back up if the same virus invades again. It can take up to two weeks to generate antibodies after being exposed to a virus for the first time, but production can ramp up much faster during a second infection.

At the same time, a virus is rarely the same when it comes back. Viruses mutate frequently as they reproduce, and RNA viruses like SARS-CoV-2 are especially prone to change. Versions of the virus with distinct groupings of mutations are categorized as variants, like omicron, delta, and alpha. Our immune systems are getting stronger and faster, but changes to the virus still have the potential to throw them for a loop.

Already, some companies are developing omicron-specific vaccines, but they may not hit the market for months. The reformulated shots may be too little, too late. In the meantime, we have to rely on the immunity we already have, including boosts to our antibody counts that come from booster doses of Covid-19 vaccines.

There is still much to learn about how all the elements of the immune system work together over time to hold off Covid-19, and some of the answers will only become evident with time. And the odd behavior of omicron is forcing researchers to rethink what theyve learned.

The good news is that many aspects of our immune system also appear to handle the latest variant well. From what Ive seen, the T cell responses are still working rather well against omicron, said Brianne Barker, a vaccine researcher at Drew University. I think that weve still got a bit of time in which immune protections will remain intact.

Immunity will continue building across the population and will blunt the sharp edges of the pandemic, even as the virus changes. Covid-19 is unlikely to go away entirely. As it circulates, it will continue to mutate and may cause sporadic outbreaks. But our immune systems are making progress.

As you expose the human body, even to the same antigen over and over again, our immune system evolves as well, Fuller said. What were starting to see in people with third immunizations is an antibody [response] that is broader.

Its a good sign that improvements in our immune system are likely to outpace changes in the virus. But the pandemic has also made it clear that there is nothing about its trajectory we can take for granted. While the cells within us may shield against infection, its still a good idea to limit transmission of the virus in any other way we can. The fewer people it infects, the fewer unpleasant surprises ahead.

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My mum helped me recover from leukaemia then she was diagnosed with breast cancer. This is how – iNews

By daniellenierenberg

When Lily Whitmarsh was diagnosed with leukaemia in 2019, days after she turned 20, it came as a complete shock. My world came crashing down around me and I went into total meltdown, she says.

She had been a fit and healthy teenager, but in the run-up to her birthday began experiencing mysterious symptoms. I was constantly complaining that my legs ached and I was sometimes napping twice a day and still feeling exhausted, she recalls.

I looked extremely pale and was experiencing night sweats. I remember going out for a walk and having to stop halfway because I was so out of breath and felt dizzy.

Lily, now 21, from Gillingham in Dorset, noticed a slight pinprick rash on the bottoms of her legs and odd-looking bruises which she couldnt explain appearing randomly on her body. She went to her GP and was referred for blood tests.

Alarm bells rang when she was told her platelet count was extremely low and she was immediately sent to hospital.

After a bone marrow biopsy, Lily was diagnosed with acute lymphoblastic leukaemia. To make matters more complicated, she had a rarer subtype called Philadelphia positive, in which the leukaemia cells grow more rapidly.

She underwent chemotherapy treatment and went on to have a bone marrow transplant during the coronavirus pandemic. With no immune system, Lily knew she was extremely vulnerable and spent a lot of time shielding and avoiding people.

By the time the country went into lockdown and she left hospital after her transplant, she had already spent months in almost total isolation, only able to see close friends and family and with precautions to stay germ-free.

After having my bone marrow transplant in March 2020, my immune system was extremely weak and I had to be very careful not to pick up any bugs as my body would struggle to fight them.

When Lily went into hospital, she was only allowed visitors for about a week before the first coronavirus lockdown. Luckily, she had her mum, Lucy Shaw, by her side and says that she couldnt have coped without her support, or that of the Teenage Cancer Trust.

Every day, seven young people in the UK aged 13 to 24 hear the words you have cancer. Teenage Cancer Trust helps put them in the best possible place physically, mentally and emotionally for their cancer treatment and beyond through expert nurses and support teams.

Lily received support from the charitys youth support co-ordinator, Leonie, and says Leonie not only understood every aspect of a cancer diagnosis, but what it meant to be going through it as a young person.

Lily admits that being faced with a cancer diagnosis at such a young age, she had moments where she wondered: Why me? With so many young people being diagnosed with cancer every day now, I then had to think: why not me? I wasnt any different to anybody else before I got ill, so the denial soon wore off and I accepted my illness was a process and I just had to work through it.

Lily says that Leonie taught her to be strong and accept what was happening to her and feel more in control of her illness.

Lily says: The thing with cancer is, it literally doesnt care. It doesnt care about your gender, your age, your race; and in my case, it didnt care about my lifestyle either.

I just woke up one day with some dodgy cells and then, bam, youre told youve got it and it wont go away without gruelling treatment that puts your whole life on hold and makes you contemplate whether youre even going to survive.

A bone marrow donor was found for Lily using the Anthony Nolan bone marrow donor register. Three matches for her were found worldwide and all she knows about her donor is that he is a 39-year-old man from the UK.

After her transplant, the Covid-19 pandemic meant Lily had to isolate at home with her mother, sister and stepfather. She suffered severe exhaustion. I was like a newborn baby and was sleeping for 18 or 20 hours a day and my diet was bland, white food, she remembers. Having no immune system in a global pandemic isnt the ideal situation, but I felt safe knowing by not seeing people, I couldnt catch anything.

On 30 August, almost a year after her diagnosis, she celebrated her 21st birthday with an outdoor garden party and was finally able to see people, from a distance. It was a real milestone, she says.

However, in November 2020, just as Lily was making huge strides in her recovery, her mother received a diagnosis of breast cancer and had surgery that Christmas, followed by radiotherapy and chemotherapy in the new year.

My mum went through it all with me and then suddenly, our roles were reversed and I had to see her go through it all.

Between the two of us, we went through a lot that year, says Lily. But we got through it and came out the other side. My mum has finished all her treatment and is doing well.

The two women are now looking forward to a brighter 2022 and Lily says she is eternally grateful to the stranger who gave her her life back by donating his stem cells.

I count my lucky stars every day that my anonymous donor did what he did and donated his stem cells, says Lily.

Without him, I wouldnt have had another Christmas. What matters most now is spending time with my family and friends and to be able to finally start living again, not just surviving.

This random stranger did this wonderful and kind thing. He doesnt know me, but he has saved my life. Without him, I would be very poorly or not be here.

My treatment was harsh, it massively affected my life and will do for the next few years at least. But I have come so far and will forever be proud of myself for that.

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NIH researchers develop first stem cell model of albinism to study related eye conditions – National Institutes of Health

By daniellenierenberg

News Release

Tuesday, January 11, 2022

Use of patient-derived stem cells will enable high-throughput drug screening for potential therapeutics.

Researchers at the National Eye Institute (NEI) have developed the first patient-derived stem cell model for studying eye conditions related to oculocutaneous albinism (OCA). The models development is described in the January issue of the journal Stem Cell Reports. NEI is part of the National Institutes of Health.

This disease-in-a-dish system will help us understand how the absence of pigment in albinism leads to abnormal development of the retina, optic nerve fibers, and other eye structures crucial for central vision, said Aman George, Ph.D., a staff scientist in the NEI Ophthalmic Genetics and Visual Function Branch, and the lead author of the report.

OCA is a set of genetic conditions that affects pigmentation in the eye, skin, and hair due to mutation in the genes crucial to melanin pigment production. In the eye, pigment is present in the retinal pigment epithelium (RPE), and aids vision by preventing the scattering of light. The RPE is located right next to the eyes light-sensing photoreceptors and provides them nourishment and support. People with OCA lack pigmented RPE and have an underdeveloped fovea, an area within the retina that is crucial for central vision. The optic nerve carries visual signals to the brain.

People with OCA have misrouted optic nerve fibers. Scientists think that RPE plays a role in forming these structures and want to understand how lack of pigment affects their development.

Animals used to study albinism are less than ideal because they lack foveae, said Brian P. Brooks, M.D., Ph.D., NEI clinical director and chief of the Ophthalmic Genetics and Visual Function Branch. A human stem cell model that mimics the disease is an important step forward in understanding albinism and testing potential therapies to treat it.

To make the model, researchers reprogrammed skin cells from individuals without OCA and people with the two most common types of OCA (OCA1A and OCA2) into pluripotent stem cells (iPSCs). The iPSCs were then differentiated to RPE cells. The RPE cells from OCA patients were identical to RPE cells from unaffected individuals but displayed significantly reduced pigmentation.

The researchers will use the model to study how lack of pigmentation affects RPE physiology and function. In theory, if fovea development is dependent on RPE pigmentation, and pigmentation can be somehow improved, vision defects associated with abnormal fovea development could be at least partially resolved, according to Brooks.

Treating albinism at a very young age, perhaps even prenatally, when the eyes structures are forming, would have the greatest chance of rescuing vision, said Brooks. In adults, benefits might be limited to improvements in photosensitivity, for example, but children may see more dramatic effects.

The team is now exploring how to use their model for high-throughput screening of potential OCA therapies.

NEI leads the federal governments research on the visual system and eye diseases. NEI supports basic and clinical science programs to develop sight-saving treatments and address special needs of people with vision loss. For more information, visit https://www.nei.nih.gov.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

Aman George, Ruchi Sharma, Tyler Pfister, Mones Abu-Asab, Nathan Hotaling, Devika Bose, Charles DeYoung, Justin Chang, David R. Adams, Tiziana Cogliati, Kapil Bharti, Brian P. Brooks. In Vitro Disease Modeling of Oculocutaneous Albinism Type I and II Using Human Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium (2022). doi: 10.1016/j.stemcr.2021.11.01.https://www.cell.com/stem-cell-reports/fulltext/S2213-6711(21)00597-X.

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Global Stem Cell Therapy Market valued at USD 200 million is set to witness a healthy growth of 17% in the upcoming years : Medi-Tech Insights -…

By daniellenierenberg

Stem cells are the bodys raw materials. They are unspecialized cells that have ability to renew themselves through mitotic cell division and differentiate into a diverse range of specialized cell types. They are critical for the development, growth, maintenance and repair of bones, muscles, blood, brain, nerves, skin and other organs. There are several sources of stem cells:

Embryonic Stem Cells: These stem cells come from embryos that are three to five days old. These are pluripotent stem cells and can be used to regenerate or repair diseased tissues and organsAdult Stem Cells: These stem cells are found in most adult tissues (bone marrow or fat) in small numbers. As compared to embryonic stem cells, they have more limited ability to give rise to various cells of the bodyInduced Pluripotent Stem Cells: Using genetic reprogramming, adult cells are transformed by scientists into stem cells that act similar to embryonic stem cellsPerinatal Stem Cells: These stem cells are found in amniotic fluid & umbilical cord blood. They have the ability to change into specialized cells

Factors Igniting Interest in Stem Cells

To Develop Understanding of How Diseases Occur: By observing how stem cells mature into cells in nerves, bones, heart muscles and other organs and tissues, researchers and healthcare professionals may better understand how diseases and conditions developHelp in Generating Healthy Cells to Replace Diseased Cells: Stem cells possess the potential to transform into specific cells that can be used to regenerate and repair diseased or damaged tissuesTo Test Safety and Effectiveness of New Drugs: Prior to using investigational drugs on people, researchers can use stem cells to test drugs for quality & safety

Transplantation of Blood Stem Cells Most Established Stem Cell Treatment

Currently, there are only limited stem cell therapies that have been thoroughly established as safe and effective treatment. The most well-established and widely used stem cell treatment is the transplantation of blood stem cells to treat diseases and conditions of the blood and immune system, or to restore the blood system after treatments for specific cancers.

Favorable investment environment, rising clinical trials for stem cell based-therapies, increasing demand for induced pluripotent stem cells (iPSCs) as an alternative to embryonic stem cells (ESCs) and the rising demand for cell & gene therapies are some of the key factors driving the growth of the Stem Cell Therapy Market.

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Other areas/indications where stem cell therapies are being used are:

For the treatment of knee cartilage defects in patients with Osteoarthritis (OA)For the treatment of Crohns fistulaFor regeneration of subcutaneous adipose tissueFor the treatment of ALS (Amyotrophic Lateral Sclerosis)For the treatment of acute graft versus host disease (aGVHD) in children and adults, among others

Derivation of embryonic stem cells (ESCs) requires destruction of human embryos. Ethical concerns related to embryonic stem cells is one the of key factors that is likely to hamper the growth of the Stem Cell Therapy Market. Increasing number of clinics offering unproven stem cell-based treatments is another ethical issue faced in the field of stem cell-based therapies.

Stem cells have a bright future for the therapeutic world by promising stem cell therapy. We hope to see new horizon of therapeutics in the form of bone marrow transplant, skin replacement, organ development, and replacement of lost tissue such as hairs, tooth, retina and cochlear cells.

CEO, South Korea Based Stem Cell Therapy Provider

Future Outlook of Stem Cell Therapy Market

Stem cell therapy could be the medical innovation of the century. It has emerged as a promising new approach in almost every medicine specialty. Despite an enormous amount of research being undertaken, there are still limited safe and effective treatments available to patients. This is partially because complex diseases which are currently incurable require complex treatments and a personalized approach.

However, the future growth prospects of stem cell therapy market looks promising as there are several ongoing and completed clinical trials involving stem cells which are showcasing positive outcomes.

In clinical studies and treatment attempts, stem cell therapies have been tested with the following indications:

Macular DegenerationNeurological ConditionsDiabetesGraft-versus-host disease (GvHD)Cirrhosis of the Liver, among others

Stem cell therapies are increasingly being seen as the transformative step in treating conditions with unmet needs. This, coupled with growing investment in the sector and an increasing number of stem cell donors is expected to drive the global Stem Cell Therapy market forward in the coming years.

Sources: Medi-Tech Insights Analysis, Interviews, Company Websites

For Detailed Insights on Stem Cell Therapy Market, Contact Us @ https://meditechinsights.com/contact-us/

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The role of gel wound dressings loaded with stem cells in the treatment of diabetic foot ulcers – DocWire News

By daniellenierenberg

This article was originally published here

Am J Transl Res. 2021 Dec 15;13(12):13261-13272. eCollection 2021.

ABSTRACT

Diabetic foot ulcers (DFUs) are a serious complication of diabetes and the main cause of nontraumatic lower limb amputations, resulting in a serious economic burden on society. The main causes of DFUs include peripheral neuropathy, foot deformity, chronic inflammation, and peripheral artery disease. There are many clinical approaches for the treatment of DFUs, but they are all aimed at addressing a single aetiological factor. Stem cells (SCs), which express many cytokines and a variety of nerve growth factors and modulate immunological function in the wound, may accelerate DFU healing by promoting angiogenesis, cell proliferation, and nerve growth and regulating the inflammatory response. However, the survival time of SCs without scaffold support in the wound is short. Multifunctional gel wound dressings play a critical role in skin wound healing due to their ability to maintain SC survival for a long time, provide moisture and prevent electrolyte and water loss in DFUs. Among the many methods for clinical treatment of DFUs, the most successful one is therapy with gel dressings loaded with SCs. To accelerate DFU healing, gel wound dressings loaded with SCs are needed to promote the survival and migration of SCs and increase wound contraction. This review summarizes the research advancements regarding multifunctional gel wound dressings and SCs in the treatment of DFU to demonstrate the effectiveness and safety of this combinational therapeutic strategy.

PMID:35035674 | PMC:PMC8748097

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Genome Editing Market: Rise in drug discovery and development activities to drive the market – BioSpace

By daniellenierenberg

Genome Editing Market: Snapshot

Genome editing tools have come a long way from the mid-twentieth century. In 1970s and 1980s, gene targeting was done using largely homologous combination, but was only possible in mice. Since then, the expanding science of genetic analysis and manipulation extended to all types of cells and organisms. Advent of new tools helped scientists achieve targeted DNA double-strand break (DSB) in the chromosome, and is a key pivot on which revenue generation in the genome editing market prospered. New directions for programmable genome editing emerged in the decades of the twenty-first century, expanding the arena.

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Cutting-edge platforms at various points in time continue to enrich genome editing market. Various classes of nucleases emerged, most notable of which is CRISPR-Cas. Research labs around the world have extensively used the platforms in making DSBs at any target of choice. Aside from this, agricultural sciences and medical sectors make substantial use of zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) in genome editing. Strides made in stem cell therapies, particularly in rectifying an aberrant mutation, have boosted the growth of the genome editing market. Genetic diseases such as muscular dystrophy and sickle cell disease present an incredible revenue prospect in the genome editing market. Ongoing research on novel vectors and non-vector approaches are expected to bolster the outlook of the market.

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Genomic editing refers to the strategies and techniques implemented for the modification of target genetic information of any living organism. Genome editing involves gene modification at specific areas through recombinant technology, which increases precision in insertion and decreases cell toxicity. Current advancement in genome editing is based on programmable nucleases. The genome editing market is presently witnessing significant growth due to increase in R&D expenditure, rise in government funding for genomic research, technological advancements, and growth in production of genetically modified crops. Companies have made significant investments in R&D in the past few years to develop cutting-edge technologies, such as, CRISPR and TALEN. For instance, Thermo Fisher Scientific is investing significantly in the development of its CRISPR technology for providing better efficiency and accuracy in research and also to fulfil the unmet demands in research and therapeutics. Cas9 protein and FokI protein have been combined to form a dimeric CRISPR/Cas9 RNA-guided FokI nucleases system, which is expected to have wide range of genome editing applications.

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The genome editing market is growing rapidly due to its application in a large number of areas, such as mutation, therapeutics, and agriculture biotechnology. Genome editing techniques offer large opportunities in crop improvement. However, the real potential of homologous recombination for crop improvement in targeted gene replacement therapy is yet to be realized. Homologous recombination is expected to be used as an effective methodology for crop improvement, which is not possible through transgene addition. Rise in the number of diseases and applications is likely to expand the scope of genome editing in the near future. It includes understanding the role of specific genes and processes of organ specific stem cells, such as, neural stem cells and spermatogonial stem cells. Genome editing has a significant scope to treat genetically affected cells, variety of cancers, and agents of infectious diseases such as viruses, bacteria, parasites, etc. However, genetic alteration of human germline for medicinal purpose has been debated for years. Ethical issues, comprising concern for animal welfare, can arise at all stages of generation and life span of genetically engineered animal.

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The global genome editing market can be segmented based on technology, application, end-user, and geography. In terms of technology, the genome editing market can be categorized into CRISPR, TALEN, ZFN, and other technologies. Bioinformatics has eased the process of data analysis through various technological applications. On the basis of application, the global genome editing market can be classified cell-line engineering, animal genome engineering, plant genome engineering, and others. Based on end-user, the genome editing market can be segmented into pharmaceutical and biotechnological companies and academic and clinical research organizations. In terms of region, the global genome editing market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America is projected to continue its dominance in the global genome editing market owing to high government funding for research on genetic modification in the region. Asia Pacific is a rapidly growing genome editing market due to rise in investments by key players in the region. Rise in drug discovery and development activities, coupled with increasing government initiatives toward funding small and start-up companies in the biotechnology and life sciences industry, is a major factor expected to drive the genome editing market in North America during the forecast period. Players should invest in the emerging economies and the countries of Asia-Pacific like China, South Korea, Australia, India and Singapore in which the genome editing market is expected to grow at rapid pace in future, due to growing funding in research.

Key players operating in the global genome editing market are CRISPR Therapeutics, Thermo Fisher Scientific, GenScript Corporation, Merck KgaA, Sangamo Therapeutics, Inc., Horizon Discovery Group, Integrated DNA Technologies, New England Biolabs, OriGene Technologies, Lonza Group, and Editas Medicine.

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The widespread diagnostic and serological testing is emerging as one of the key measures to mitigate the COVID-19 pandemic. The increased load on healthcare systems, social distancing, and convenience needs of individuals is anticipated to boost the growth of the North America direct-to-consumer laboratory testing market.

Topical Antibiotics Market :

Topical antibiotics have emerged as a popular drug class for the treatment and management of a range of medical conditions. Among different indications such as the skin, eye, and Bromhidrosis, the use of topical antibiotics to fight bacterial skin infection has witnessed consistent growth over the past few decades a trend that is expected to continue over the upcoming years. Research and development activities around the world are likely to fuel the growth of the global topical antibiotics market, as new topical antibiotics continue to enter the market. While the growing popularity of antiseptics could potentially hinder market growth, the growing awareness pertaining to the benefits of topical antibiotics is anticipated to boost the demand.

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Regenerative Medicine: The Promise Of Undoing The Ravages Of Time – Hackaday

By daniellenierenberg

In many ways, the human body is like any other machine in that it requires constant refueling and maintenance to keep functioning. Much of this happens without our intervention beyond us selecting what to eat that day. There are however times when due to an accident, physical illness or aging the automatic repair mechanisms of our body become overwhelmed, fail to do their task correctly, or outright fall short in repairing damage.

Most of us know that lizards can regrow tails, some starfish regenerate into as many new starfish as the pieces which they were chopped into, and axolotl can regenerate limbs and even parts of their brain. Yet humans too have an amazing regenerating ability, although for us it is mostly contained within the liver, which can regenerate even when three-quarters are removed.

In the field of regenerative medicine, the goal is to either induce regeneration in damaged tissues, or to replace damaged organs and tissues with externally grown ones, using the patients own genetic material. This could offer us a future in which replacement organs are always available at demand, and many types of injuries are no longer permanent, including paralysis.

Our level of understanding of human physiology and that of animals in general has massively expanded since the beginning of the 20th century when technology allowed us to examine the microscopic world in more detail than ever before. Although empirical medical science saw its beginnings as early as the Sumerian civilization of the 3rd millennium BCE, our generalized understanding of the processes and components that underlie the bodys functioning are significantly more recent.

DNA was first isolated in 1869 by Friedrich Miescher, but its structure was not described until 1953. This discovery laid the foundations for the field of molecular biology, which seeks to understand the molecular basis for biological activity. In a sense this moment can be seen as transformative as for example the transition from classical mechanics to quantum mechanics, in that it changed the focus from macroscopic observations to a more fundamental understanding of these observations.

This allowed us to massively increase our understanding of how exactly the body responds to damage, and the molecular basis for regenerative processes, as well as why humans are normally not able to regrow damaged limbs. Eventually in 1999 the term regenerative medicine was coined by William A. Haseltine, who wrote an article in 2001 on what he envisions the term to include. This would be the addressing of not only injuries and trauma from accidents and disease, but also aging-related conditions, which would address the looming demographic crisis as the average age of the worlds populations keeps increasing.

The state of the art in regenerative medicine back in 2015 was covered by Angelo S. Mao et al. (2015). This covers regenerative methods involving either externally grown tissues and organs, or the stimulating of innate regenerative capabilities. Their paper includes the biomedical discipline of tissue engineering due to the broad overlap with the field of regenerative medicine. Despite the very significant time and monetary requirement to bring a regenerative medicine product to market, Mao et al. list the FDA-approved products at that time:

While these were not miracle products by any stretch of the imagination, they do prove the effectiveness of these approaches, displaying similar or better effectiveness as existing products. While getting cells to the affected area where they can induce repair is part of the strategy, another essential part involves the extracellular matrix (ECM). These are essential structures of many tissues and organs in the body which provide not only support, but also play a role in growth and regeneration.

ECM is however non-cellular, and as such is seen as a medical device. They play a role in e.g. the healing of skin to prevent scar tissue formation, but also in the scaffolding of that other tantalizing aspect of regenerative medicine: growing entire replacement organs and body parts in- or outside of the patients body using their own cells. As an example, Mase Jr, et al. (2010) report on a 19-year old US Marine who had part of his right thigh muscle destroyed by an explosion. Four months after an ECM extracted from porcine (pig) intestinal submucossa was implanted in the area, gradual regrowth of muscle tissue was detected.

An important research area here is the development of synthetic ECM-like scaffolding, as this would make the process faster, easier and more versatile. Synthetic scaffolding makes the process of growing larger structures in vitro significantly easier as well, which is what is required to enable growing organs such as kidneys, hearts and so on. These organs would then ideally be grown from induced pluropotent stem cells (iPS), which are a patients own cells that are reverted back to an earlier state of specialization.

It should come as little surprise that as a field which brings together virtually every field that touches upon (human) biology in some fashion, regenerative medicine is not an easy one. While its one thing to study a working system, its a whole different level to get one to grow from scratch. This is why as great as it would be to have an essentially infinite supply of replacement organs by simply growing new ones from iPS cells, the complexity of a functional organ makes this currently beyond our reach.

Essentially the rule is that the less complicated the organ or tissue is, the easier it is to grow it in vitro. Ideally it would just consist out of a single type of cell, and happy develop in some growth medium without the need for an ECM. Attractive targets here are for example the cornea, where the number of people on a waiting list for a corneal transplant outnumber donor corneas significantly.

In a review by Mobaraki et al. (2019), the numerous currently approved corneal replacements as well as new methods being studied are considered. Even though artificial corneas have been in use for years, they suffer from a variety of issues, including biocompatibility issues and others that prevent long-term function. Use of donor corneas comes with shortages as the primary concern. Current regenerative research focuses on the stem cells found in the limbus zone (limbal stem cells, LSC). These seem promising for repairing ocular surface defects, which has been studied since 1977.

LSCs play a role in the regular regenerative abilities of the cornea, and provide a starting point for either growing a replacement cornea, or to repair a damaged cornea, along with the addition of an ECM as necessary. This can be done in combination with the inhibiting of the local immune response, which promotes natural wound healing. Even so, there is still a lot more research that needs to be performed before viable treatments for either repairing the cornea in situ, or growing a replacement in vitro can be approved the FDA or national equivalent.

A similar scenario can be seen with the development of artificial skin, where fortunately due to the large availability of skin on a patients body grafts (autografts) are usually possible. Even so, the application of engineered skin substitutes (ESS) would seem to be superior. This approach does not require the removal of skin (epidermis) elsewhere, and limits the amount of scar formation. It involves placing a collagen-based ECM on the wound, which is optionally seeded with keritanocytes (skin precursor cells), which accelerates wound closure.

Here the scaffolding proved to be essential in the regeneration of the skin, as reported by Tzeranis et al. (2015). This supports the evidence from other studies that show the cell adhesion to the ECM to be essential in cell regulation and development. With recent changes, it would seem that both the formation of hair follicles and nerve innervation may be solved problems.

It will likely still be a long time before we can have something like a replacement heart grown from a patients own iPS cells. Recent research has focused mostly on decellularization (leaving only the ECM) of an existing heart, and repopulating it with native cells (e.g. Glvez-Montn et al., 2012). By for example creating a synthetic scaffold and populating it with cells derived from a patients iPS cells, a viable treatment could be devised.

Possibly easier to translate into a standard treatment is the regrowth of nerves in the spinal cord after trauma, with a recent article by lvarez et al, (2021) (press release) covering recent advances in the use of artificial scaffolds that promotes nerve regeneration, reduces scarring and promotes blood vessel formation. This offers hope that one day spinal cord injures may be fully repairable.

If we were to return to the body as a machine comparison, then the human body is less of a car or piece of heavy machinery, and more of a glued-together gadget with complex circuitry and components inside. With this jump in complexity comes the need for a deeper level of understanding, and increasingly more advanced tools so that repairs can be made efficiently and with good outcomes.

Even so, regenerative medicine is already saving the lives of for example burn victims today, and improving the lives of countless others. As further advances in research continue to translate into treatments, we should see a gradual change from youll have to learn to live with that, to a more optimistic give it some time to grow back, as in the case of an injured veteran, or the victim of an accident.

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University of Alberta study shows positive signs to get patients with diabetes off injected insulin – Global News

By daniellenierenberg

Editors note: This article has been updated to remove incorrect information provided by the University of Alberta.

Researchers at the University of Alberta say they have reached a milestone in the efforts to get people with diabetes off injected insulin for good.

A recent first-in-humans clinical trial is reporting early signs that pancreatic cells grown from stem cells can be safely implanted, and in some cases, begin to produce insulin.

The trial saw 17 adults with Type 1 diabetes at six centres in Canada, the United States and Europe receive implants of pluripotent stem cell-derived pancreatic endoderm cells.

Each patient received implants of several small permeable devices filled with millions of cells each. The cells were derived from stem cells then chemically transformed into stem cells programmed to become islet cells.

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Of the 17 patients who received implants, U of A researchers said 35 per cent showed signs in their blood of insulin production after meals within six months of the implant. On top of that, 63 per cent had evidence of insulin production inside the implant devices when they were removed after a year.

This is a very positive finding, said James Shapiro, professor of surgery, medicine and surgical oncology in the University of Albertas Faculty of Medicine & Dentistry.

Its not the endgame, but its a big milestone along the road to success, demonstrating that stem cell-derived islet therapies are safe and can begin to show some signal of efficacy in patients in the clinic.

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Shapiro also led the team that developed the Edmonton Protocol in the 1990s, which developed a way to transplant donated islet cells, reducing their need for insulin. However, the U of A says patients continue to need anti-rejection drugs which can have side effects such as an increased risk of cancer and kidney damage. The number of donated islet cells is also limited.

Shapiro said the main goal of this phase of the trial was to ensure safety, but added at least one patient who had 10 devices implanted was able to significantly reduce her insulin dose, which indicates the potential effectiveness of the treatment.

Were seeing some improvement in the patients blood sugar, but these cells are being transplanted right now in only very small quantities, so were not expecting big changes in insulin requirement, Shapiro said.

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But we can see in about 65 per cent of devices that we take out from under the skin that there are human insulin-producing cells surviving, and in about a third of patients they have measurable insulin levels in the bloodstream. So its a really good first start with this treatment, Im very excited about it.

The ultimate goal of the new research is to develop an unlimited supply of islet cells that can be safely transplanted without the need for anti-rejection drugs.

Weve seen a lot of advances in the last 100 years since the Canadian discovery of insulin, Shapiro said. The race isnt over yet, but were on our last laps and I really do believe that we can cross that ribbon.

Cell-based therapies have the promise to deliver something far better than insulin therapy.

Again, were not expecting to be curing diabetes in the first wave of this, were trying to do safety testing for first patients. And we see that really is helping mankind in the future of diabetes rather than any particular one patient at this point, but it will change as we move forward.

The next step will try to determine how many stem cell-derived pancreatic cells are needed for transplant to optimize insulin production in patients with both Type 1 and Type 2 diabetes.

2022 Global News, a division of Corus Entertainment Inc.

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The first successful pig heart transplant into a human was a century in the making – Popular Science

By daniellenierenberg

Last Friday, January 7, David Bennett went into the operating room at the University of Maryland Medical Center for a surgical procedure never performed before on a human. The 57-year-old Maryland resident had been hospitalized and bedridden for months due to a life-threatening arrhythmia. His heart was failing him and he needed a new one.

Bennetts condition left him unresponsive to treatments and ineligible for the transplant list or an artificial heart pump. The physician-scientists at the Baltimore medical center, however, had anotheralbeit riskyoption: transplant a heart from a genetically-modified pig.

It was either die or do this transplant, Bennett had told surgeons at the University of Maryland Medical Center a day before the operation. I want to live. I know its a shot in the dark, but its my last choice.

On Monday, the team reported that they completed the eight-hour procedure, making Bennett the first human to successfully receive a pigs heart. Its working and it looks normal. We are thrilled, but we dont know what tomorrow will bring us. This has never been done before, Bartley Griffith, M.D., physician and director of the cardiac transplant program at the University of Maryland Medical Center who led the transplant team, told the New York Times.

While its only been five days since the operation, the surgeons say that Bennetts new pig heart was, so far, functioning as expected and his body wasnt rejecting the organ. They are still monitoring his condition closely.

I think its extremely exciting, says Robert Montgomery, M.D., transplant surgeon and director of the NYU Langone Transplant Institute, who was not involved in Bennetts operation. The results of the procedure were also personally meaningful for Montgomery, who received a heart transplant in 2018 due to a genetic disease that may also impact members of his family in the future. Its still in the early days, but still the heart seems to be functioning. And that in and of itself is an extraordinary thing.

[Related: Surgeons transplanted a pig kidney into a person, and it worked like normal]

Pig heart transplant operations are still not officially approved by the U.S. Food and Drug Administration, but the agency granted emergency authorization for the surgery on December 31. The experimental procedure comes at a time of growing need for organ transplants. More than 100,000 people in the United States are on the list to receive one, while around 17 die each day waiting, according to the latest data from the federal governments organdonor.gov. The desperate demand far exceeds the number of human organ donors.

There arent enough organs, period, Montgomery says, who was part of the team that successfully transplanted a genetically modified pig kidney in a human in 2021. Of an estimated 800,000 patients on dialysis whove developed end-stage kidney disease, only 90,000 are on the list for a human organ transplant, he points out. Pig organ transplants give another potential way to to fill that gap between the supply and the demand.

The field of research and the techniques behind animal to human organ transplants has come a long way to reach this momentfrom myth and pseudoscience to sophisticated medical application. Xenotransplantation, or grafting and transplanting of organs and tissues between two species, has a long history, says Montgomery, who has been involved in this field for more than 30 years.

It has really been considered since the dawn of transplantation, he says. People were thinking about the use of animal organs for over a hundred years.

Throughout the 19th century, chickens, rats, dogs, frogs, and other animals were used for skin grafts. Researchers continued to encounter incompatibility issues between humans and animal organs and tissues. This was because many animal species have a cell membrane sugar called galactose-1,3-galactose, commonly referred to as alpha-gal. That sugar is also on the surface of bacteria, explains Montgomery. Humans are exposed to these bacteria from birth in the GI tract, which triggers the immune system to make antibodies against alpha-gal to prevent those bacteria from entering the blood. People have a huge reserve of these antibodies just circulating in our blood all the time, Montgomery says, and those antibodies will attack animal organs because they recognize alpha-gal as a target.

Around the 1960s, surgeons began to look towards closer relatives to humans: primates. Primates are obviously much closer to humans on the evolutionary scale, and so you dont have that immediate incompatibility with alpha-gal in some of the primates, Montgomery says. A surgeon at Tulane University in New Orleans transplanted chimpanzee kidneys into patients, one of whom survived for nine months. Most famously in 1984, Baby Fae, a newborn infant with an underdeveloped heart, received a baboon heart, but her body rejected it after 20 days.

By the 1990s, the public perception towards primates as organ donors had soured. Theyre much more scarce on the planet, says Montgomery. I was at a xenotransplant meeting in the 1990s and Jane Goodall was the keynote speaker At the end of that, it was really clear to all of us that primates were not going to be the organ donors we were going to use. Concerns over zoonosis, or the transmission of disease from animal to human host, were also rising, likely because of the HIV/AIDS epidemic, Montgomery adds.

[Related: Lab-grown pig lungs are great news for the future of organ transplantation]

The scientific stage was set for swine. Pigs became prime donor candidates because of their abundance, large litters, ease of breeding, rapid growth, and generally similar organ size to humans.

Plus, most people have a much different relationship with the animal as a longtime food staple, says Montgomery, though he expects ethical concerns to continue to rise as the field progresses, such as whether or not animals should be genetically modified for transplants.

But there were two big hurdles the research field had to jump over before pigs could be a viable option: the issues with alpha-gal and the potential cross transmission of viruses, particularly the porcine endogenous retrovirus (PERV) discovered in 1997. Now, researchers have been able to genetically edit out the alpha-gal target from the pig genome. Today, people have undergone pig skin graft treatments for burns, have pig heart valves, or received pig cells, like those that help produce insulin, and have not experienced any diseases.

The genetic modification, particularly now with CRISPR, has become pretty easy, Montgomery says. Almost 200 people have received pig cells, pig stem cells, pig tissue, and skin grafts without exposure to zoonoses, he says.

The genetically modified pigs used for organ donation are bred, studied, and cared for in extremely clean facilities, and theyre surveilled for potential pathogens. Its almost like an operating room, says Montgomery. They are very humanely treated.

Up until now, most experimental transplant procedures have been done between pigs and other animals. Taking it into a living human, thats the leap, Montgomery says about the University of Maryland Medical Centers transplant. The genie is out of the bottle. Now, we really need to understand what this is going to look like in humans, and start to work on optimizing the outcomes. But time is of the essence, lets move ahead boldly.

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What the World’s First Pig to Human Heart Transplant Could Mean for the Future of Transplants – Technology Networks

By daniellenierenberg

On January 7, a 57-year-old male patient received a genetically-modified pig heart transplant at the University of Maryland Medical Center (UMMC). The surgery was a world-first and deemed the patients only chance for survival after he was declared unsuitable for a human donor transplant or an artificial heart pump. On January 10, the University of Maryland School of Medicine (UMSOM) published a news release stating that the patient was doing well, and is being carefully monitored over the next days and weeks to determine whether the transplant provides lifesaving benefits.

Dr. Bartley P. Griffith the surgeon responsible for transplanting the porcine heart into the patient and a professor in transplant surgery at UMSOM said, We are proceeding cautiously, but we are also optimistic that this first-in-the-world surgery will provide an important new option for patients in the future. Dr. Griffith leads the Cardiac Xenotransplantation Program at UMSOM alongside Dr. Muhammad M. Mohiuddin, professor of surgery at UMSOM.

The operation at the UMMC is an example of xenotransplantation. Xenotransplantation refers to any procedure involving the transplantation, infusion or implantation of cells, tissue or organs from a nonhuman, animal source into a human.

While the surgery was the first-of-its-kind, the concept of xenotransplantation is not novel. Chris Denning, professor of stem cell biology at the University of Nottingham told the UK Science Media Centre, Only in the late 1990s did the technologies become available and have steadily been improved ever since. Various academic and industrial teams have worked in this area for over 20 years, so it is not surprising that this has now been tested.

In the 20th century, non-human primates (NHP) were explored as potentially suitable donors for xenotransplantation due to the genetic similarities between primates and humans. However, concerns such as ethical issues, transmission of infection across species and breeding difficulties halted this research. Consequently, pigs are now considered to be the most appropriate candidate species for xenotransplantation.

"Pigs are considered for several reasons, Denning said. The size and anatomy of the pig heart is roughly the same as a humans, though there are considerable differences:

He added that, despite public perception, it is also relatively easy to keep pigs in a sterile condition.

Despite these advantages, transplanting a porcine heart into a human is considerably more challenging than transplanting a human heart. There are genetic differences between pigs and humans, which can lead to immunological rejection of the organ. Pigs have a gene that produces a molecule called (1,3)galactosyl transferase, which humans do not. This triggers an immediate and aggressive immune response, called hyperacute rejection, said Denning, ultimately causing the body to reject the organ.

The xenotransplantation conducted at UMMC involved a pig that had reportedly received 10 genetic modifications in total. Its unclear at this stage exactly what genes were modified, however the news release from UMSOM states three genes responsible for rapid antibody-mediated rejection of pig organs by humans were knocked out in the pig, and six human genes responsible for immune acceptance were inserted. An additional gene was also knocked out to stop excessive growth of the heart tissue.

Knockout means that an organism has been genetically altered such that it lacks either a single base, a whole gene or several genes. Often, genetic knockouts are utilized in laboratory research to understand how certain genes function, by monitoring changes in the organism when the gene is not expressed.

The porcine heart was provided by Revivicor, a subsidiary of United Therapeutics. You might recall Revivicor as the spin-out company of PPL Therapeutics, the UK-based biotech firm behind the first cloned mammal, Dolly the sheep. In December 2021, Revivicor also supplied New York University Langone Health with a kidney from a genetically-modified pig for an investigational procedure in a deceased human donor. The donor remained on ventilator support, and was closely monitored throughout the procedure and a subsequent observation period, during which the researchers said there were no signs of rejection.

According to the UMSOM news release, it received a $15.7 million research grant to evaluate Revivicor genetically-modified pig UHearts in baboon studies. Mohiuddin and colleagues reportedly applied for permission to conduct human clinical trials of the porcine heart from the US Food and Drug Administration (FDA), but were rejected. Under normal circumstances, IMPs must be evaluated in animal studies prior to human clinical trials this is standard protocol.

However, in the instance of the 57-year-old patient, an exception was made. The FDA granted emergency authorization for the procedure under its expanded access provision. This allows for an individual to access an investigational medicinal product (IMP) outside of clinical trials when there is no alternative therapy option available.

Will it be successful? asked Denning. The fact that the human patient is alive after a few days indicates that immediate hyperacute rejection has been avoided, which is the first hurdle. Only time will tell whether there are issues with chronic rejection, caused by e.g., incompatibility of major and minor histocompatibility complexes. Continuous monitoring will be needed to monitor transmission of potential pathogens, such as porcine endogenous retroviruses or hybrid porcine/human endogenous retroviruses.

Should the patient survive and the xenotransplant prove successful, it will likely raise a lot of questions as to how regulatory bodies move forward. Individual emergency authorization procedures do not generate sufficient data for the widespread implementation of xenotransplantation clinical trials are crucial for demonstrating efficacy.

However, there are logistical hurdles associated with even trialing the procedure. Seventeen people die every day waiting for an organ transplant, according to the Health Resources & Services Administration. There is a severe shortage of organs, and a steep decline in donation has been observed during the COVID-19 global pandemic. While a proposed advantage of xenotransplants is that they could provide on-demand organs, the procedure and its unknowns make it a very high-risk surgery. How does a clinician, or regulatory body, decide that a patient has waited long enough for a human organ that they qualify for inclusion in a trial?

Furthermore, if xenotransplant clinical trials support widespread adoption of xenotransplant procedures, how do we regulate a system whereby organs are widely available? Policies on patient selection and organ allocation currently exist in healthcare systems across the world. Navigating changes to these policies will require global conversations across different regulatory bodies.

Finally, a hurdle that Denning said could be the biggest of them all is: What do the general public think? Is it acceptable to harvest organs from animals? One thing that is for sure, is the outcomes of this [patient] will be watched closely by many, Denning concluded.

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Veranome Biosystems and Cold Spring Harbor Laboratory Enter Collaboration and Licensing Agreement to Develop Advanced In-Situ Sequencing Technologies

By Dr. Matthew Watson

MOUNTAIN VIEW, Calif., Jan. 13, 2022 (GLOBE NEWSWIRE) -- Veranome Biosystems LLC announced today that it has entered a collaboration and licensing agreement with Cold Spring Harbor Laboratory (CSHL) that adds in-situ sequencing technology to Veranome’s spatial omics portfolio. Veranome now has one of the most comprehensive portfolios of assay technologies that can allow targeted mapping of cellular spatial gene expressions with multiplexed in-situ hybridization (ISH) as well as de-novo profiling of cellular transcripts using in-situ sequencing. The expanded assay portfolio coupled with Veranome’s advanced imaging capabilities will enable customers to explore a broad range of applications, including characterization of cell and gene therapies methods and CRISPR screens. Veranome will further develop these high-sensitivity assay chemistries to offer researchers the ability to profile archival FFPE tissue blocks with the same spatial resolution as fresh frozen samples when analyzed on Veranome’s Spatial Analyzer.

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REPEAT — Revelation Biosciences Inc. Announces Data Demonstrating REVTx-99 In Vitro Anti-Viral Activity Against SARS-CoV-2; Commencement of Trading…

By Dr. Matthew Watson

-A greater than 50% reduction in SARS-CoV-2 Delta viral load was observed-

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REPEAT -- Revelation Biosciences Inc. Announces Data Demonstrating REVTx-99 In Vitro Anti-Viral Activity Against SARS-CoV-2; Commencement of Trading...

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Galapagos creates new subscription right plan

By Dr. Matthew Watson

Mechelen, Belgium; 13 January 2022, 22.01 CET; regulated information – Galapagos NV (Euronext & NASDAQ: GLPG) announced today that its supervisory board created 30,000 subscription rights under a new employee subscription right plan.

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Vaccitech to Host Virtual KOL Event on VTP-300, a Potential Functional Cure for Chronic Hepatitis B (CHB) Infection, and the Broader CHB Therapeutics…

By Dr. Matthew Watson

Live webcast will be at 8:30 a.m. EST on January 25 Live webcast will be at 8:30 a.m. EST on January 25

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Corvus Pharmaceuticals Announces Partner Angel Pharmaceuticals Initiated Phase 1/1b Clinical Trial of ITK Inhibitor CPI-818 in China

By Dr. Matthew Watson

BURLINGAME, Calif., Jan. 13, 2022 (GLOBE NEWSWIRE) -- Corvus Pharmaceuticals, Inc. (Corvus or the Company) (NASDAQ: CRVS), a clinical-stage biopharmaceutical company, today announced that its partner in China, Angel Pharmaceuticals Ltd. (Angel Pharma), has treated the first patient in its Phase 1/1b clinical trial of Corvus’ small molecule ITK inhibitor CPI-818 for the treatment of relapsed/refractory T-cell lymphomas (TCL) in China.

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Corvus Pharmaceuticals Announces Partner Angel Pharmaceuticals Initiated Phase 1/1b Clinical Trial of ITK Inhibitor CPI-818 in China

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Half-Year Review of Inventiva’s Liquidity Contract with Kepler Cheuvreux

By Dr. Matthew Watson

Daix (France), Long Island City (New York), January 14, 2022 – Inventiva (Euronext Paris and Nasdaq: IVA), a clinical-stage biopharmaceutical company focused on the development of oral small molecule therapies for the treatment of NASH, mucopolysaccharidoses (MPS) and other diseases with significant unmet medical needs, today announced the half-year report of its liquidity contract with Kepler Cheuvreux.

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