16 March 2020

Three years after receiving a stem cell transplant, a man from London is the second patient in history to be cured of HIV, doctors report.

The study, published in The Lancet HIV, reports that the so-called 'London patient' has no detectable active HIV infection in blood, semen, or tissues 30 months after stopping anti-retroviral therapy. This follow-up comes a year after the first publication in Natureannouncing he was clear of the virus (seeBioNews 990).

'We've tested a sizeable set of sites that HIV likes to hide in and they are all pretty much negative for an active virus,' the study's lead author Dr Ravindra Gupta, from the University of Cambridge, told AFP. 'We propose that these results represent the second ever case of a patient to be cured of HIV.'

Adam Castillejo who recently decided to go public with his identity in the New York Times had been diagnosed with HIV in 2003 and then additionally with advanced Hodgkin's Lymphoma in 2012. After failed rounds of chemotherapy and attempts to transplant his own stem cells, he received a donor stem cell transplant in 2016 to treat his blood cancer. Similar to the 'Berlin patient' (the first person known to be cured of HIV) in 2011, the transplant came from a donor carrying a rare genetic mutation present in less than one percent of Europeans, which prevents HIV from entering the affected cells via the CCR5 receptor.

'This is a unique position to be in, a unique and very humbling position,' Castillejo told the New York Times. 'I want to be an ambassador of hope.'

Now, 30 months after cutting off retroviral therapy, the doctors reported no active infection in Castillejo's blood, cerebrospinal fluid, semen, intestinal tissue, or lymphoid tissue. Whatever traces of virus material could be found in the system are likely so-called fossil traces, which cannot replicate and harbour no risk of reoccurrence of the infection.

Dr Gupta said that this is to be expected. 'It's quite hard to imagine that all trace of a virus that infects billions of cells was eliminated from the body,' he explained.

Scientists agree that stem cell transplants will notbe a feasible treatment for the millions of people around the world infected with the virus. With a tenpercent mortality rate,bone marrow transplants are mainly used to treat cancer patients when no other options are available, and current antiviral drugs can enable HIV patients to live long and healthy lives. However, observing these 'cured' patients could provide helpful insights for the design of future genome editing tools to treat and cure HIV.

Professor Sharon Lewin from the University of Melbourne, Australia, said the case was exciting but warned: 'We need to also place it in context - curing people of HIV via a bone marrow transplant is just not a viable option on any kind of scale. We need to constantly reiterate the importance of, prevention, early testing and treatment adherence as the pillars of the current global response to HIV/AIDS.'

More:

'London patient' declared cured of HIV after three years - BioNews

Life during a global pandemic takes on a surreal quality. The ubiquitous presence of social media and a constant fire hose of coronavirus news can make it particularly hard if you're already feeling anxious.

So, we've put together a little round-up of recent science news that we find inspiring, encouraging, and worthy of note in these trying times.

In a world first, surgeons at Oregon Health & Science Institute have used the CRISPR gene-editing technique to attempt a cure for Leber congenital amaurosis, a rare genetic condition that causes blindness in early childhood.

While we await results on how this experiment worked out, this achievement joins a list of other medical uses of the technique, including the search for a Huntington's disease cure, herpes, HIV, and immunotherapy for some types of cancer.

Living at a time when medical researchers have this powerful tool at their disposal is certainly a good news item in our books.

In February, a huge stock of 60,000 seed samples was added to the Svalbard Global Seed Vault nestled inside a mountain in Norway's Svalbard archipelago, including the first-ever heirloom seed deposit by an indigenous US tribe.

Increasing deposits to this safehouse of crops reflect growing worldwide concern about potential loss of biodiversity and food security - but these actions also demonstrate a beautiful commitment to our future generations.

The virus strains that cause influenza are shapeshifters, constantly moving beyond our ability to immunise against them - hence, we need annual flu shots to stay ahead of the disease. A 'universal' flu vaccine would give us a huge advantage in this race, and there's now a truly promising candidate on the cards.

The vaccine, called FLU-v, has successfully passed phase I and phase II clinical trials, demonstrating its safety in human subjects; it's been found to induce immune responses that last at least six months. We can't wait to see the results of the next phase of trials.

A new type of contact lens could restore the colour spectrum limitations in people whose eyes struggle to tell apart green and red hues.

This brilliant technology already exists in some cleverly designed sunglasses; soon, people might also have access to it in the highly convenient form of contacts, thanks to a team of engineers at Tel Aviv University.

'Normal' image of a tree; colour blind version; corrected version. (Sharon Karepov/Tel Aviv University)

Researchers have announced that for the second time ever, a patient carrying the HIV virus has been declared cured, with no trace of infection in his blood 30 months after he stopped traditional treatment, undergoing a specialised type of stem cell therapy.

The achievement doesn't constitute a generalised cure, because the patient also had a type of lymphoma that enabled him to receive this experimental treatment; but it demonstrates a real breakthrough in medical science, showing scientists are able to push the boundaries like never before.

The tiny South Pacific nation of Niue recently accepted a unique honour, as it became the first country to be formally accredited as an International Dark Sky Place.

This accreditation is bestowed by the International Dark-Sky Association (IDA), a conservation non-profit charged with preserving the naturally dark night-time environment, defending it from the intrusive disturbances of artificial light pollution.

There's no end of scientific research charting the negative effects of light pollution, whether on animals, plants, or human health; this honour emphasises that seeking a truly dark night sky remains as important as ever.

Hydrogen fuel is one of the more promising zero-emissions options around - if only we could produce it cheaply and without needing insane amounts of energy input.

Now, a team of researchers in Tokyo have managed to do just that, refining a method that produces hydrogen fuel using just a few basic ingredients, including light and a particular type of rust. A new study shows this method yields 25 times more hydrogen than existing methods.

Speaking of sustainability, one of the biggest challenges to widespread adoption of renewables remains the problem of large-scale storage. However, there is one excellent solution to this problem - pumped thermal electricity storage. This approach stores electricity by turning it into heat, then turning it back into electricity when needed using an engine.

Unlike pumped hydro, which requires specific geographic requirements, this type of storage can be built in many places, and it uses thermodynamic principles to store electricity in the form of heat. And the best part? It's already being tested in pilot plants.

In June 2019, an odd paper made waves after it was published in Scientific Reports. The scientific community was quick to voice their concerns over this flawed study, which claimed that the Sun's movements were the real cause of anthropogenic global warming.

Now, the editors of the well-known journal have corrected the scientific record, issuing a retraction notice that explains the errors,showing that even if something incorrect initially slips through peer review, the scientific process is still rigorous enough to fix the mistake.You can read about this fascinating case in full here.

A new report by the non-profit Project Drawdown has outlined a whopping 76 solutions the world already has at hand if we want to slow down climate change. These strategies - from shifting our means of energy production, to reducing food waste and empowering women - span across all sectors.

Furthermore, these solutions are actually cheaper than maintaining the status quo (also known as 'doing nothing'). Project Drawdown estimates that if we implemented these 76 solutions, it would result in savings of up to around US$144 trillion of avoided climate damage and pollution-related healthcare costs. Tell everyone - we can do this.

More here:

Tired of The Coronavirus? Here Are 10 Good News Stories You Need Right Now - ScienceAlert

This is a full transcript of Disabled and out of money in North Korea as first broadcast on 13 March and presented by Beth Rose

JITE- I got a few stares of course. I'm bald. I had a beard. I was in a wheelchair. I'm black. The first two that I went to said, "No, no, no, we probably can't do that." I didn't want to do something which was challenging for me only, rather than North Korea. Oh, well that's a tough place to go to.

[jingle: Ouch]

BETH-I've been so excited about bringing you this Ouch podcast. A few months ago I received an email. It said, "Hi Beth, a friend of mine, Jite Ugono has multiple sclerosis, or MS, and uses a wheelchair. He's just about to travel to North Korea. Would you like to talk to him?" "Yes," was my answer, "very much so."

I'm Beth Rose, and you're listening to the BBC Ouch podcast, and for a while Jite has been on my mind. From the day he flew to China to get his visa, to the five days he would spend in the country we know very little about. And finally, he's back. Also, just a quick note to say that this podcast was recorded long before the Corona virus outbreak.

[music]

BETH-Hello.

JITE-Hello, hi.

BETH-So how was the trip?

JITE-Everyone says surreal, but it was surreal. Being inside a communist country and being restricted. Also in a wheelchair, there are no provisions at all for wheelchair access and that kind of stuff. Most of the places I went to were only accessible by stairs, so they carried me, which was nice. And that's one of the good things about having a guide, because I had two guides and a driver.

BETH-So you said you were thinking about this trip a year ago. It's the kind of trip that most people won't even think you can do, so why did you suddenly decide to book your holiday to North Korea?

JITE-Well I've got MS so they said one of the treatments of MS could be stem cell therapy. So stem cell therapy involves chemo and the rest of it. I thought to myself why not do something as rare as stem cell therapy? It was almost like a redefinition of my identity. I didn't really want to be known solely because of MS or the treatment, because everyone's going to ask about the chemo. I wanted to do something else which was kind of equal and opposite.

BETH-It's quite rare, stem cell therapy for multiple sclerosis isn't it?

JITE-It is. I hadn't heard of it. Chemo for cancer, we know all about that, but as soon as she said chemo for this For me it was quite emotional because my mum died the year before of cancer and she went through chemo as well. It was a shock, but it was also some hope. It seems less bleak. What I have is Primary Progressive MS, a steady degradation of mobilities. And they have less treatment for that, so most other treatment comes for Secondary Remitting, when you have attacks and then you can recover.

BETH-So what does the chemo do?

JITE-Chemo reduces your immune system. So what they want to do is kind of knock out the immune system and then reintroduce the stem cells and then restart the immune system.

BETH-That sounds quite an intense treatment.

JITE-I was in hospital for a month. So I went in for chemo, I was in hospital for a week or so, first of all, came back out, did the injections, back into hospital for a month. It was tough going through, but easier when you do it in stages. You think, okay I'm going to do this chemo first, in ten days I'll do the injections. Bite size. So by the end of it it's like oh, I've done it. I think it taught me whatever I go through I have to be a bit more patient.

BETH-How long ago were you diagnosed with MS?

JITE-2009.

BETH-So you were quite young?

JITE-I'm 45 now, so yeah, the symptoms got worse maybe six or seven years ago in terms of difficulty walking. And that's the main thing. The first thing was the eyesight, so the eyes were playing up and I thought maybe I should go to the optician. It didn't really make a difference. So it got progressively worse. I did an MRI scan and then the consultant said, "Well, it could be MS." So I was kind of aware and I kind of knew that it was something quite serious. So when he came back and he said MS. You make a decision about how you're going to deal with it.

For me, it was you're not going to feel sorry for yourself because people go through worse. For me, it's only when I'm faced with stuff you realise you can do it. I didn't just want to survive. Because when you're diagnosed with stuff it's like getting through the day. Everyone says, "Oh, you're so brave. You went to work?" For me it's just one life, you can't spend it getting through the day, you want to do something else.

BETH-So was it when you were having your chemo when you were in hospital, the idea for North Korea?

JITE-It was actually the first consultation when she told me, "You're going to do stem cell therapy." They told me that I was going to be able to maybe walk with sticks and I thought, why waste it?

BETH-I feel like a lot of people would have had similar thoughts but maybe thought South of France would be quite nice?

JITE-It would have been challenging. If anyone said they were going to the South of France, oh okay. I didn't want to do something which was challenging for me only, rather than North Korea, oh well, that's a tough place to go to, regardless of whether you're in a wheelchair. It was important to me to do something which was challenging, not because of MS, not because of the wheelchair, but it was challenging.

BETH-So how do you go about booking a trip? Can you go to a travel agent?

JITE-I mean, that's what I did. So the first two that I went to said, "No, no, no. We can't do that, there's no access." And I was probably more determined. That's another lesson it taught me, it's more important for me that I wanted to do it. And no one was coming back to me to say, "Why don't you go?" So when the third person came back and said, "Actually, we could do that," the normal way of going to North Korea is through a group tour, with my condition anyway. You think about what the problems could be. Getting onto the coach. Holding people up.

So my tour was me on my own. I had two guides and a driver and that was it. They sorted out the visa to China and once you get to China you get the visa to North Korea from China.

BETH-Touching upon the issues of getting onto a bus, what is it like for you with MS? How does it manifest itself?

JITE-My balance is a problem. I can't really use my left leg at all. My eyesight's a problem. Maybe sometimes my memory and my vocabulary. They're difficulties which arose mainly because I did chemo. We know that the drugs are quite aggressive and concentrated, so they give you lots of water to dilute and because you're given that you're given drugs to help you relieve that stuff, so you're peeing like every ten minutes.

So it went down to probably once every hour and that became a problem and that affects your confidence, you're afraid to kind of go out, maybe there won't be toilets around, that's kind of what I was thinking about, going to North Korea.

BETH-Did you even know about that? Is there information about toilets or accessibility?

JITE-Not at all, not at all. It's only when I got there that I realised that the And sorry to go on about toilets, but it was important to me. [laughs] Okay, so in North Korea they had two types of toilets, they had the European toilets and then they had the Korean toilets, ground toilets, so you have to kind of balance, which I didn't even attempt. So everywhere we went to it was okay, "Is it a Korean toilet here or a European toilet?" Even the guides started to realise and started to know after a while.

BETH-I mean, that's such a gamble isn't it, not knowing the accessibility, not knowing what the toilet situation's going to be like. I'm guessing this was all in your mind?

JITE-Every problem has to have a solution. So before I went I'd got it up to you can pass an hour now, because I'd gone to the gym, I'd started doing core stuff, even in the plane, because it was ten and a half hours there. You think about the problems that you could face, it's personal of course, but also there are people around that can give you a hand.

And that was another thing, getting vaccinations was a problem, because when you do chemo and your immune system is low they don't advise that you have vaccinations. So I was intending to go to Korea in September but that was super close to my stem cell.

BETH-When you were flying, initially to China, what was going through our mind?

JITE-It was just getting through that first bit, hoping that someone's going to be there to meet me. The luggage I even took I had to make sure that I could carry. That's one of the solutions with a wheelchair, you're going to have to push the luggage as well so it can't be too big. Two pieces of hand luggage is what I took. That's what I was thinking about, I wasn't thinking about Pyongyang yet, I was thinking about how to get to China.

Beijing was packed, traffic everywhere. It was surprisingly western. The cars were German cars. In North Korea I had the guides, in China I didn't have guides, I had a person to take me from the airport to the hotel and that was it. So I didn't really have the confidence to kind of venture out. I got in a day before, so as soon as I landed in China I had to go and get the visa. As soon as you get the visa is when they give you a briefing, what you should and shouldn't do. The chap apparently had been doing it for 28 years, and no one had ever missed a briefing until me.

BETH-Ah! [laughs]

JITE-I mean, only because the person who picked me up said, "Oh, I can get the visa for you."

BETH-So they were being helpful, but actually

JITE-Yeah, so they went out and got the And I was appreciative, because getting in and out of the car was such a pain. And I am quite lazy naturally. If I can do without it then I won't do it, you know. So when they gave me an opportunity not to, oh okay. The travel agent contact in China was almost panicky on the phone, "No one's ever done this."

BETH-Wow, and I bet your heart was racing at that point.

JITE-To an extent, but I kind of knew what not to do. I mean, I'm not rude, and plus I'd seen stuff on YouTube and the guides tell you as well. So I was quite prepared. I flew into Pyongyang. The airport was a surprise. They only have a few planes that land for the day. They had one from Beijing, one from Shanghai and one from Moscow. There are soldiers everywhere, but the soldiers were, "Oh, look at this guy," I suppose maybe because I was a novelty in a sense. They'd never really seen someone in a wheelchair before. They were super helpful.

I'd met the guides at the airport as well. I got a few stares of course. I'm bald, and they have like five haircuts. I had a beard, I was in a wheelchair. I'm black. So all those things together.

BETH-So did you feel like you stuck out?

JITE-I didn't feel like I could relax, only because you feel like you're always on. I couldn't be anonymous, there's always someone watching, and that's tiring.

BETH-And did you feel like you were being watched by your guides?

JITE-Maybe the brief was to watch, but it is different when you have a relationship with people. So I didn't feel that way. I suppose they were constantly on about how great the leader is and after a while it got a bit tedious. Everyone walked around with badges. And it's difficult to tell because they spoke the language quite a bit. I don't know what they're saying.

BETH-They greeted you at the airport.

JITE-Yes.

BETH-Had they had disabled travellers before?

JITE-I don't think they had. What happens is that when you go on your own there is no camaraderie, I was mostly alone, but the advantage is you could probably get closer to people. There's good and there's bad about it.

BETH-What's it like, Pyongyang?

JITE-For me it was super quiet. I mean here we have adverts and stuff, people are selling you stuff all the time, there is different, you have pictures of the leaders surrounded by flowers and you have to respect that. If there's an image of a leader you can't really take a photo of it and you can't stand in front of it obscuring it. Or you can't crop it. Apparently they check people's phones to see what they've taken.

BETH-Did you take photos?

JITE-I took photos but they didn't check. But everywhere was empty. The place is set up for tourists but there are not many tourists. You go into a restaurant and there are people standing around. The restaurants are empty. It's bizarre.

BETH-So it's not really like a bustling city?

JITE-Not at all. Actually I went during King Il Sung who's the grandad of this present leader, it was his birthday, so there were two days of celebrations. I think there were more people on the street than normal, and then they had volunteers picking up stuff or gardening or I mean, because it's a communist environment they pay for everything but you have to work. They've got big roads, no cars.

BETH-Wow.

JITE-Yeah. The days were quite long. Maybe eight o'clock they'll come for me and then eight o'clock in the evening I'd finish. So there was always something to do and you were always with people. I think they had five channels, that was about it.

BETH-TV channels?

JITE-Five TV channels. On the channels they have the leader, Kim, pointing at stuff. He designed the theme park.

BETH-What's the tourist trail like?

JITE-There is an itinerary, so you would go to the war museum, flower exhibition. I went to their subway, it's the deepest subway in the world. So everything's the best in the world or the tallest in the world.

BETH-How did the subway compare to the tube?

JITE-It was more opulent. I only saw two of them and I think those are the two they show people, so maybe the others are less. There are chandeliers and stuff.

BETH-And the restaurants, you said you went into one, but they've got all the staff just waiting around?

JITE-Yeah, the restaurants seem to be for tourists, and because I was on my own, seven, ten people just standing around looking. I went to a casino, which was strange.

BETH-Oh, okay?

JITE-Yeah. But the casino was in the hotel. I think I was the only one in there. So when I went to North Korea I didn't take enough cash, and that was a problem obviously because no cards. So the guys were like, "You need some money? Go to the casino, you can change your money."

BETH-Oh, I thought you were going to say to like gamble and win.

JITE-At first I went to change money, but they didn't take sterling, they took US dollars and euros, but I didn't have either, so they allowed me to gamble, so I did.

BETH-Did you win? Did you get some money?

JITE-Yeah, I did. I don't want to get used to it. [laughs]

BETH-What game did you play?

JITE-Black Jack. I didn't know what was going on, but people around, they were almost cheering, and I was thinking by the time I won a hundred dollars I thought it's time to go, it's time to go. And everyone's around you willing you on and you don't want to disappoint them but you think okay, I'm going guys.

BETH-Is it expensive then, if you ran out of money and you're having to gamble to boost your-?

JITE-To boost. Okay, so I mean they have their own currency and they don't let you take the currency out.

BETH-I bet your guides quite enjoyed being in the casino.

JITE-The guides said, "Oh, we're not allowed in." Even when they came up to my hotel room I had to have Al Jazeera because that's the only English speaking channel, but they were almost transfixed. They were shaking their heads. Look around the world, look how happy we are type of thing. So you kind of understand why they would let Al Jazeera in, because Al Jazeera can be quite, look what's happening around the world, the protests here, the protests there.

BETH-And did you find people were willing to help you?

JITE-I think it was more because they see you as being vulnerable. "Oh, you're not comfortable, let me move your legs." So you always get somebody helping, which is not necessarily what you want all the time. Because you want to be able to be self-sufficient. Certainly in London people are a bit more patient to offer, "Okay, how can I help?" and then they stand back. In Korea it was, "Oh, we can do that for you." [laughs]

BETH-Did you see any other disabled people out and about?

JITE-No, I didn't.

BETH-No one at all?

JITE-I didn't at all. One of the guides was quite insistent on how great their society is. That's why they stay kind of thing, away from everyone else, and they obviously saw it as a good thing.

BETH-Oh, that's interesting. I was going some research, and there's a lot of reports from the UN and different charities where they say basically they send people away in an out of town community.

JITE-Yeah, they don't expect you to try. So maybe that was part of it, they were almost surprised that this person is doing something on their own.

BETH-And were they quite surprised how you just got on with everything?

JITE-Yeah, I suppose. Maybe they were. So even when I'd be going down the road people would lean over and look. They weren't rude about it. They would look, they were curious, but they weren't intrusive. And sometimes you look and they look away, except the kids, so the kids would be staring. But that's normal though, even in London you'll get kids staring. One of the guides took a video of me being lifted up the stairs, and it was quite tough to watch because you don't really see yourself as being vulnerable, except when you see it.

It's like hearing a recording of yourself and you think oh, do I sound like that? Or do I look like that? Am I really that vulnerable kind of thing? No wonder everyone helps. [laughs] It was tough to see. I didn't really see the footage until I got to the hotel and you kind of think, you know, is that how it is? They were helpful, and it sounds ungrateful almost, but it is what you think about.

It's a lack of confidence to think people only help you because you look so vulnerable. Maybe people are just nice. And that was one of the good things about going to North Korea. People say that Londoners are quite cold and I don't find that, Londoners can be helpful, and especially if you're patient enough. And MS for me does that, it allows you to be patient.

BETH-So what kinds of things is nice to have help for?

JITE-Probably getting in and out of cars. In London not so much, in London you kind of want to get strong. I know that I'm going to have to get in a car, and not everybody gives the same level of help, so you have to be self-sufficient. In North Korea there's no need. And I'm never going to be in North Korea again.

BETH-How did the access pan out? Because that was the big mystery wasn't it really? I mean, you had no idea.

JITE-It was just people lifting me. Only one place, the museum was difficult.

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Transcript: Disabled and out of money in North Korea - BBC News

In this report, the global Cancer Stem Cell Therapy market is valued at USD XX million in 2019 and is projected to reach USD XX million by the end of 2025, growing at a CAGR of XX% during the period 2019 to 2025.

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The following manufacturers are covered:AVIVA BioSciencesAdnaGenAdvanced Cell DiagnosticsSilicon Biosystems

Segment by RegionsNorth AmericaEuropeChinaJapanSoutheast AsiaIndia

Segment by TypeAutologous Stem Cell TransplantsAllogeneic Stem Cell TransplantsSyngeneic Stem Cell TransplantsOther

Segment by ApplicationHospitalClinicMedical Research InstitutionOther

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Cancer Stem Cell Therapy Market Global Size, Demand-sales, Suppliers by Key Applications 2019 Detailed Analysis and Growth Aspects on Manufacturing...

In 2020, the National Eye Institute is launching a clinical trial to test the safety of a patient-specific stem cell therapy to treat geographic atrophy, the advanced dry form of age-related macular degeneration (AMD). The protocol is the first of its kind in the United States to replace a patients eye tissue with tissue derived from induced pluripotent stem (iPS) cells engineered from a patients own blood.

If successful, this new approach to AMD treatment could prevent millions of Americans from going blind. AMD is a leading cause of vision loss in people age 65 and older. By 2050, the estimated number of people with AMD is expected to more than double from 2.07 million to 5.44 million.

The first symptoms of age-related macular degeneration are dark spots in ones central vision, which is used for daily activities such as reading, seeing faces and driving. But as the disease progresses, the spots grow larger and increase in number, which can lead to significant loss of the central vision.

There are two kinds of AMD: the neovascular, or wet, form and the geographic atrophy, or dry form. Remarkable progress has been made in the ability to prevent vision loss from the neovascular form. In particular, anti-VEGF therapy has been shown to preserve vision required for driving among about half of patients who take it for five years.

By contrast, no therapies exist for treating geographic atrophy. Should this NEI-led study, and future studies, confirm the safety and efficacy of iPS cell-derived RPE-replacement therapy, it would likely be the first therapy approved for the treatment of geographic atrophy.

To produce the therapy, we isolate cells from a patients blood and, in a lab, convert them into iPS cells. These iPS cells are theoretically capable of becoming any cell type of the body.

The iPS cells are then programmed to become retinal pigment epithelium (RPE). RPE cells are crucial for eye health because they nourish and support photoreceptors, the light-sensing cells in the retina. In geographic atrophy, RPE cells die, leading to the death of photoreceptors and blindness. The goal of the iPS cell-based therapy is to protect the health of the remaining photoreceptors by replacing dying RPE tissue with healthy iPS cell-derived RPE tissue.

We grow a single-cell layer of iPS cell-derived RPE on a biodegradable scaffold. That patch is then surgically placed next to the photoreceptors where, as we have seen in animal models, it integrates with cells of the retina and protects the photoreceptors from dying.

This years clinical trial is a phase I/IIa study, which means it will focus solely on assessing the safety and feasibility of this RPE replacement therapy. The dozen participants will have one eye treated. Importantly, everyone will already have substantial vision loss from very advanced disease, such that the therapy is not expected to be capable of significant vision restoration. Once safety is established, later study phases will involve individuals with earlier stage disease, for which we are hopeful that therapy will restore vision.

A safety concern with any stem cell-based therapy is its oncogenic potential: the ability for cells to multiply uncontrollably and form tumours. On this point, animal model studies are reassuring. When we genetically analysed the iPSC-derived RPE cells, we found no mutations linked to potential tumour growth.

Likewise, the risk of implant rejection is minimised by the fact that the therapy is derived from patient blood.

Several noteworthy innovations have occurred along the way to launching the trial. Artificial intelligence has been applied to ensure that iPS cell-derived RPE cells function similar to native RPE cells. In addition, Good Manufacturing Practices, have been developed to ensure quality control, which will be crucial for scaling up production of the therapy should it receive approval from the U.S. Food and Drug Administration. Furthermore, the iPS cell-derived RPE patch is being leveraged to develop more complex RPE/photoreceptor replacement therapies.

Potential breakthroughs in treatment cannot move forward without the support of patients willing to participate in clinical trial research. Patients who volunteer for trials such as this are the real heroes of this work because theyre doing it for altruistic reasons. The patients in this first trial are not likely to benefit, so they are doing it to help move the field forward for future patients.

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Eye health: Testing the safety of stem cell therapy for age-related macular degeneration - Open Access Government

SAN FRANCISCO, March 16, 2020 /PRNewswire/ -- The global single cell analysis marketsize is expected to reach USD 8.02 billion by 2027, registering a CAGR of 16.9% during the forecast period, according to a new report by Grand View Research, Inc. Advancements in molecular techniques which resulted in higher accuracy, ability to perform multiple omics analyses in one cell, and automation, has lowered the barriers for implementation of single-cell analysis techniques across various end-use settings. As a result, companies are investing in introducing novel solutions to accelerate the identification and quantification of genetic information in individual cells for research programs, thereby contributing to revenue growth in this market.

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This technology has addressed several research challenges with respect to biological intricacies in stem cell biology, tumor biology, immunology, and other therapeutic areas. This leads to improved therapeutic decision-making with regards to precision medicine, thereby driving the adoption of these assays in personalized therapeutic development.

The growth in research publications depicts the increasing R&D investments. Since R&D activities are considered as the foundation of innovation, investments in R&D activities signify a healthy growth prospect for the single cell analysis market. Moreover, the establishment of new single cell genomics centers in the past years is anticipated to boost the uptake of instruments and consumables for single cell analysis, thus driving the growth.

Grand View Research has segmented the global single cell analysis market on the basis of product, application, end use, and region:

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Single Cell Analysis Market Size Worth $8.02 Billion By 2027 | CAGR: 16.9%: Grand View Research, Inc. - Yahoo Finance

Immune cells by fluorescence microscopy: Blood stem cells remember a previous attack and produce more immune cells like these macrophages to fight a new infection. Credit: Sieweke lab/CIML

These findings should have a significant impact on future vaccination strategies and pave the way for new treatments of an underperforming or over-reacting immune system. The results of this research are published in Cell Stem Cell on March 12, 2020.

Stem cells in our bodies act as reservoirs of cells that divide to produce new stem cells, as well as a myriad of different types of specialized cells, required to secure tissue renewal and function. Commonly called blood stem cells, the hematopoietic stem cells (HSC) are nestled in the bone marrow, the soft tissue that is in the center of large bones such as the hips or thighs. Their role is to renew the repertoire of blood cells, including cells of the immune system which are crucial to fight infections and other diseases.

Until a decade ago, the dogma was that HSCs were unspecialized cells, blind to external signals such as infections. Only their specialized daughter cells would sense these signals and activate an immune response. But work from Prof. Michael Siewekes laboratory and others over the past years has proven this dogma wrong and shown that HSCs can actually sense external factors to specifically produce subtypes of immune cells on demand to fight an infection. Beyond their role in an emergency immune response, the question remained as to the function of HSCs in responding to repeated infectious episodes. The immune system is known to have a memory that allows it to better respond to returning infectious agents. The present study now establishes a central role for blood stem cells in this memory.

We discovered that HSCs could drive a more rapid and efficient immune response if they had previously been exposed to LPS, a bacterial molecule that mimics infection, said Dr. Sandrine Sarrazin, Inserm researcher and senior-author of the publication. Prof. Michael Sieweke, Humboldt Professor at TU Dresden, CNRS Research Director and last author of the publication, explained how they found the memory was stored within the cells: The first exposure to LPS causes marks to be deposited on the DNA of the stem cells, right around genes that are important for an immune response. Much like bookmarks, the marks on the DNA ensure that these genes are easily found, accessible and activated for a rapid response if a second infection by a similar agent was to come.

The authors further explored how the memory was inscribed on the DNA, and found C/EBP? to be the major actor, describing a new function for this factor, which is also important for emergency immune responses. Together, these findings should lead to improvements in tuning the immune system or better vaccination strategies.

The ability of the immune system to keep track of previous infections and respond more efficiently the second time they are encountered is the founding principle of vaccines. Now that we understand how blood stem cells bookmark immune response circuits, we should be able to optimize immunization strategies to broaden the protection to infectious agents. It could also more generally lead to new ways to boost the immune response when it underperforms or turn it off when it overreacts, concluded Prof. Michael Sieweke.

The research group of Prof. Michael Sieweke works at the interface of immunology and stem cell research. The scientists focus on the study of hematopoietic stem cells and macrophages, long-lived mature cells of the immune system that fulfil an important role in tissue regeneration. In 2018, Prof. Michael Sieweke received the most valuable research award in Germany: the Alexander von Humboldt Professorship, which brings top international researchers to German universities. In addition to his position as Research Director at the Centre for Immunology at the University of Marseille Luminy, he now acts as Deputy Director at the Center for Regenerative Therapies at TU Dresden (CRTD). CRTD is academic home for scientists from more than 30 nations. Their mission is to discover the principles of cell and tissue regeneration and leveraging this for recognition, treatment and reversal of diseases. The CRTD links the bench to the clinic, scientists to clinicians to pool expertise in stem cells, developmental biology, gene-editing and regeneration towards innovative therapies for neurodegenerative diseases such as Alzheimers and Parkinsons disease, hematological diseases such as leukaemia, metabolic diseases such as diabetes, retina and bone diseases.

Reference: C/EBP-Dependent Epigenetic Memory Induces Trained Immunity in Hematopoietic Stem Cells by Brengre de Laval, Julien Maurizio, Prashanth K. Kandalla, Gabriel Brisou, Louise Simonnet, Caroline Huber, Gregory Gimenez, Orit Matcovitch-Natan, Susanne Reinhardt, Eyal David, Alexander Mildner, Achim Leutz, Bertrand Nadel, Christophe Bordi, Ido Amit, Sandrine Sarrazin and Michael H.Sieweke, 12 March 2020, Cell Stem Cell.DOI: 10.1016/j.stem.2020.01.017

This study was funded by TU Dresden / CRTD through the German Excellence Initiative, the German Research Foundation as well as through an ERC Advanced Grant from the European Research Council and the Alexander von Humboldt Foundation. The study was further supported by funding from the Institut National de la Sante et de la Recherche Medicale, the Centre National de la Recherche Scientifique, Aix-Marseille University, the Agence Nationale de la Recherche, the Foundation ARC pour la Recherche sur le Cancer, an INSERM-Helmholtz cooperation programme and the Einstein Foundation.

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Newly Discovered Memory in Our Bones: Keeping a Record of Previous Infections to Boost Immunity - SciTechDaily

Global Autologous Stem Cell Based Therapies Market Report is a professional and in-depth research report on the worlds major regional market conditions of the Autologous Stem Cell Based Therapies industry, focusing on the main regions and the main countries (United States, Europe, Japan and China).

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Autologous Stem Cell Based Therapies Market 2020: Potential Growth, Challenges, Attractive Valuation | Key Players: Anterogen, Holostem Advanced...

UPDATE:

SPOKANE, Wash. -- Life-saving cells for alocal father of six are on their way to him from Poland. His family has been panicked after a travel ban was put in place by the Polish Government. They say they were told the status of the transport was stalled, and with time slipping away, they needed immediate action.

Jared Weeks was diagnosed withAcute Myeloid Leukemia back in October. His wife Janet contacted 'Help Me Hayley' on Saturday. On Sunday morning, Janet got word that the cells were on their way. She reached out to many government officials and is still trying to sort how and who helped make this happen for her husband.

"I heard that relief in (my husband's) voice and that's all I needed," she said. "I'm so thankful to everyone who shared the story, sent us prayers. I felt it. I really did. People are so overwhelmingly beautiful."

Janet says her husband will have the stem-cell transplant on Tuesday.

"I will be traveling over to Seattle on Monday evening to be there for his 're-birthday,'" she said of the procedure. "I'm so grateful."

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SPOKANE, Wash. -- A local father of six desperately needs help receiving life-saving cells provided by an overseas donor. His family says his life depends on it.

His wife Janet sent our Hayley Guenthner this 'Help Me Hayley' request:

"Dear Help Me Hayley,

My children and I are desperate to save my husband. He was diagnosed with Acute Myeloid Leukemia on 10/15/2019 (on his 42nd birthday of all days) since then he has been in the hospital. At the beginning of February we started our journey to the west side of the state to be under the care of Seattle Cancer Care Alliance and to make a long story short, we are now in the transplant stage of his disease.

My husband, Jared Weeks, went inpatient to the University of Washington Medical Center (UWMC) on behalf of the Seattle Cancer Care Alliance. He started his myeloablative chemo regimen on March 10th with the expectation of receiving an Unrelated Allogeneic Peripheral Blood Stem Cell transplant. He had the highest dose of chemotherapy to eliminate his disease and replace his immune system with a 38-year-old female peripheral blood stem cell donation from Poland. Because of the travel ban put in place by the Polish Government in response to the outbreak of the Novel COVID-19 virus, it is becoming impossible to transport these LIFE-SAVING cells that have been extracted from my husband's donor and brought back to the United States. I have left messages for Senator Cathy McMorris-Rodgers, Governor Jay Inslee, Mayor Woodward and Senator Maria Cantwell. I was able to speak personally with State Senator Shelly Short who is passing on this to some of her contacts in the cabinet. I reached out to the Polish Government agency handling the travel ban restrictions and have spoken with an Overseas Citizen Services Safety Officer out of Krakow Poland at the US Embassy-State Department. The travel ban has been put in place but I have been told that roads are still open as well as trains and planes, but as of midnight tonight (not sure if our time or their time) the borders will be closed until March 25th, and maybe extended depending on the COVID-19 outbreak. The cells have been collected from the donor and we are desperate to get them here. Please help us!! God help us.

My husband, Jared Weeks, was diagnosed with Acute Myeloid Leukemia on October 15, 2019 and is in DIRE need of these stem cells to survive.

We need some assistance from the "powers that be" to get these life-saving stem cells to my husband in Washington State ASAP.

His life depnds on it."

There have many people offering to test to see if they are a local match for Jared. Unfortunately, the family doesn't have the kind of time required to find a new donor.

"They would need to go to bethematch.org , however, it is too late in the game to be a donor for Jared but there are hundreds of others that need this life-saving donation as well," Janet said. "The HLA TYPING that is done can take weeks to complete and for Jared, we don't have that kind of time."

Janet is currently in Spokane with their children. She said she is doing everything she can to stay strong for her husband.

"(Jared) is one heck of a dad," Janet said. "He is hardworking, loves the outdoors, fishing, boating and taking his kids on adventures. He is amazing to us and is the center of gravity for our rather large family. He has been through hell and back with this cancer, and is still trusting God completely."

Seattle Cancer Cancer Care Alliance sent KHQ a statement on Jared and other cancer patients relying on life-saving bone marrow transplants during the COVID-19 outbreak.

"The COVID-19 outbreak is an evolving and fluid situation, and the global medical community is collaborating to address the needs of people who are relying on bone marrow transplants for their treatment and survival.

"Seattle Cancer Care Alliance is evaluating every patient who is currently connected with an international or USA-based donor to ensure we have an alternative solution for their treatment should the need arise.

"We are committed to continuing to coordinate with the National Marrow Donor Program and the World Marrow Donor Association, along with donor representatives in various countries, to prevent potential disruptions of critical medical transport so that every cancer patient has access to the life-saving treatment they need.

"SCCA is dedicated to providing the highest-quality cancer care, and we take that responsibility very seriously. We continue to work very closely with our alliance partners -Fred Hutch, UW Medicine and Seattle Childrens- and sharing our approach and best practices with other transplant centers around the country who may face similar unprecedented challenges."

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HELP ME HAYLEY: Spokane father of six receiving life-saving cells from Poland donor - KHQ Right Now

Londoner Jite Ugono never expected to find himself playing blackjack in a North Korean casino having run out of cash, but a few life-changing moments had led him there.

In his 30s he was diagnosed with multiple sclerosis (MS), then 10 years later in 2019 he was offered rare stem cell therapy, involving chemotherapy, on the NHS to help stall the progress of the condition.

It was hopeful, but he didnt want this complex treatment to become the main topic of conversation for friends and family so he decided to "do something equally rare, but opposite" and booked his trip to North Korea.

But would the country be ready to accept a traveller in a wheelchair and would his guides even turn up?

Presented by Beth Rose. A full transcript is available here.

Subscribe to Ouch Disability Talk podcast on BBC Sounds or say "Ask the BBC for Ouch" to your smart speaker.

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Disabled and out of money in North Korea - BBC News

By XINHUAMore by this Author

China has released the seventh version of the diagnosis and treatment guideline on the novel coronavirus disease (Covid-19).

Here are some drugs and therapies that have been recommended by the guideline, and some medicines that have been found to have the potential to defeat the virus and have entered clinical trials.

Chloroquine Phosphate, a widely used anti-malaria and autoimmune disease drug, has been used for more than 70 years.

The drug has been used in treating 285 critically ill Covid-19 patients in a hospital in Wuhan, and no obvious adverse reactions have been found so far.

In the latest version of the treatment guideline, Chloroquine Phosphate is recommended for Covid-19 patients from 18 to 65.

The amount for patients over 50 kg is 500 mg per dose twice a day for seven days.

The guideline also noted that patients should take less than three antiviral drugs.

Tocilizumab, with the common brand name Actemra, is an injectable synthetic protein that blocks the effects of IL-6 in patients with rheumatoid arthritis.

IL-6 is a protein that the body produces when there is inflammation.The latest version of the guideline suggests the use of Tocilizumab in patients with an increasing level of IL-6 and with extensive lesions in both lungs or severe symptoms.

Chinese researchers have found that a cause of death for severe and critically ill patients infected with the novel coronavirus is cytokine storm, an overreaction of the immune system.

These patients are found with a higher level of IL-6 in their blood.

Last month, the increasing level of IL-6 was recommended as a warning sign that the patient's situation could possibly deteriorate.

Currently, the drug is under clinical trials in 14 hospitals in Wuhan and a total of 272 severe patients had been treated with Tocilizumab as of March 5.

Convalescent plasma, processed from the plasma collected from recovered Covid-19 patients, contains a large number of protective antibodies.

As of February 28, 245 Covid-19 patients have received the therapy and 91 cases have shown improvement in clinical indicators and symptoms.

According to health authorities, plasma therapy has proved safe and effective.

4. TRADITIONAL CHINESE MEDICINE

Traditional Chinese Medicine (TCM) has been proven effective in treating Covid-19 patients.

With TCM treatment, patients with mild symptoms have seen their fever or cough alleviated, according to medical experts.

For severely ill patients, TCM helped relieve symptoms and restore blood oxygen saturation, preventing the patients' conditions from developing into critically ill cases.

TCM decoction Qingfei Paidu Soup has been recommended to medical institutions nationwide on February 6 after data analysis on 214 cases.

As of February 29, the decoction is used in 66 designated hospitals in 10 provincial-level regions in China.

Favipiravir, an influenza drug available on overseas markets, has been put in a parallel controlled study in Shenzhen, Guangdong Province, with 80 patients enlisted.

The initial outcome of the trial shows the drug has relatively obvious efficacy and low adverse reactions.

Experts have suggested expanding the trial to further observe and study its effect.

Remdesivir, developed against Ebola infections by American pharmaceutical company Gilead Sciences, has shown fairly good antiviral activity against the novel coronavirus at the cellular level.

Cao Bin, a respiratory expert who is leading the Remdesivir programme, said on Wednesday that two trials for Remdesivir are going on smoothly and China will share the data with the international community after the programme is completed.

Clinical studies on stem cell therapy, which can inhibit the overreaction of the body's immune system, have also been carried out to treat severe patients.

As of February 21, four patients who have received the therapy have been discharged from hospital, and the trial is expected to be further expanded. Currently, three kinds of stem cells mesenchymal, lung and embryonic stem cells are used in treatments.

Researchers usually inject stem cell products into the lungs.

Meanwhile, the Chinese Academy of Sciences has developed a new stem cell drug, CAStem, which has shown promising results in animal experiments.

The research team has applied for urgent assessment by the National Medical Products Administration.

Approvals by the ethics committee, and clinical observation and evaluation, are in progress.

Several research and trials on applying stem cells to treat Covid-19 patients have been carried out in the country.

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Here's how to treat coronavirus, according to research - Daily Nation

Boston Children's Hospital researchers used an investigational gene therapy to treat heart failure in a mouse model of Barth syndrome. Barth syndrome is a rare genetic disorder in boys that results in life-threatening heart failure. It also causes weakness of the skeletal muscles and the immune system. The disease is caused by a mutation of a gene known as tafazzin or TAZ.

In 2014, William Pu and researchers at Boston Childrens Hospital collaborated with the Wyss Institute to develop a beating heart on a chip model of Barth syndrome. It used heart-muscle cells with the TAZ mutation that came from patients own skin cells. This was able to prove that TAZ was the cause of the cardiac problems. The heart muscle cells did not organize normally and the mitochondria, the cells energy engines, were disorganized, resulting in the heart muscle contracting weakly. By adding healthy TAZ genes, the cells behaved more normally.

The next step was an animal model. The results of the research were published in the journal Circulation Research.

The animal model was a hurdle in the field for a long time, Pu said. Pu is director of Basic and Translational Cardiovascular Research at Boston Childrens and a member of the Harvard Stem Cell Institute. Efforts to make a mouse model using traditional methods had been unsuccessful.

Douglas Strathdees research team at the Beatson Institute for Cancer Research in the UK recently developed animal models of Barth syndrome. Pu, research fellow Suya Wang, and colleagues characterized the knockout mice into two types. One had the TAZ gene deleted throughout the body; the other had the TAZ gene deleted just in the heart.

Most of the mice that had TAZ deleted throughout their whole bodies died before birth, likely from skeletal muscle weakness. Of those that survived, they developed progressive cardiomyopathy, where the heart muscle enlarges and is less able to pump blood. The heart also showed signs of scarring similar to humans with dilated cardiomyopathy, where the hearts left ventricle is dilated and thin-walled.

The mice that lacked TAZ only in their heart tissue that survived to birth had the same features. Electron microscopy indicated that the heart muscle cells and mitochondria were poorly organized.

Pu and Wang and their team then used gene therapy to replace TAZ in the newborn mice and in older mice, using slightly different techniques. In the newborn mice the engineered virus was injected under the skin; in the older mice it was injected intravenously. The mice who had no TAZ in their bodies and received the gene therapy survived to adulthood.

In the newborn mice receiving the gene therapy, the therapy prevented cardiac dysfunction and scarring. In the older mice receiving the therapy, it reversed the cardiac dysfunction.

The study also showed that TAZ gene therapy offered durable treatment of the cardiomyocytes and skeletal muscle cells, but only when at least 70% of the heart muscle cells had taken up the gene via the therapy. Which the researchers point out that when the therapy is developed for humans, that will be the most challenging problem. You cant just scale up the dose because of inflammatory immune responses, and multiple doses wont work either because the body develops an immune response. Maintaining the gene-corrected cell is also a problem. In the heart muscles of the treated mice, the corrected TAZ gene stayed relatively stable, but slowly dropped in skeletal muscles.

The biggest takeaway was that the gene therapy was highly effective, Pu said. We have some things to think about to maximize the percentage of muscle cell transduction, and to make sure the gene therapy is durable, particularly in skeletal muscle.

Link:

Gene Therapy Reverses Heart Failure in Animal Model of Barth Syndrome - BioSpace

PHILADELPHIA--(BUSINESS WIRE)--Century Therapeutics, developer of induced pluripotent stem cell (iPSC)-derived allogeneic cell therapies for cancer, today announced the opening of its Seattle-based Innovation Hub to develop next-generation product candidates that overcome barriers that have limited the effectiveness of cell therapies in solid tumor cancers.

The site will advance the companys novel iPSC science and allogeneic cell products by establishing expertise in data sciences and machine learning, synthetic biology, cancer biology and immuno-oncology. Centurys President of R&D, Hy Levitsky, M.D., will be based at the Seattle site, and together with Philadelphia-based Chief Scientific Officer Luis Borges, PhD., will oversee site operations and integration with the pipeline programs centered at Centurys Philadelphia headquarters.

The Innovation Hub supports not only Centurys continued pipeline growth and development, but also our expansion into Seattle, a center of excellence in cell therapies, said Lalo Flores, Chief Executive Officer of Century Therapeutics. I look forward to seeing the Century team grow, and am excited to have Luis and Hy leading the charge into this exciting new chapter.

Dr. Levitsky has extensive biotech industry experience, having previously served as Chief Scientific Officer at Juno Therapeutics in Seattle, as well as Head of Cancer Immunotherapy at Roche Pharma Research and Early Development. In addition, Dr. Levitsky earned his M.D. from Johns Hopkins University and has spent over 20 years on their faculty.

Dr. Borges has extensive cancer immunotherapy and cell therapy experience, having worked at Immunex, Amgen, Five Prime Therapeutics and Cell Medica, where as CSO he led the development of off-the-shelf CAR-cell therapies for the treatment of cancer in collaboration with the Baylor College of Medicine.

Centurys new Seattle Innovation Hub will provide the infrastructure needed to conduct in-depth analytics of product candidates in preparation for entry into the pipeline portfolio, said Dr. Levitsky.

The Hub will be key in realizing the potential of Centurys science to overcome limitations of first-generation cell therapies. Dr. Borges added, The Seattle and Philadelphia research laboratories will complement each other. With our current deep expertise in iPSC biology, immunology, cell and protein engineering, the new group in Seattle will help us transition to future generation products designed to have potent anti-tumor efficacy and robust safety windows.

About Century Therapeutics

Century Therapeutics is harnessing the power of stem cells to develop curative cell therapy products for cancer that overcome the limitations of first-generation cell therapies. Our genetically engineered, universal iPSC-derived immune effector cell products (NK, T, DC and macrophage) are designed to specifically target hematologic and solid tumor cancers. Our commitment to developing off-the-shelf cell therapies will expand patient access and provides an unparalleled opportunity to advance the course of cancer care. Century was launched in 2019 by founding investor Versant Ventures in partnership with Fujifilm and Leaps by Bayer. For more information, please visit http://www.centurytx.com.

Link:

Century Therapeutics Announces Opening of Seattle Innovation Hub - Business Wire

People with multiple sclerosis have unique concerns about the new coronavirus and the COVID-19 disease that it causes. Many of us use disease-modifying therapies (DMTs) that suppress our immune systems and give us an extra element to worry about when we plan our defense against this virus.

To help us make wise decisions, the U.K.s MS Trust and the Italian Society of Neurology have created COVID-19 guidelines related to DMTs, and both organizations recommendations are similar. They balance concerns of abruptly ending any MS treatment with those of possibly being more susceptible to this illness because some DMTs suppress the immune system.

According to BartsMS Blog, the Italian neurologists wrote that Given the lack of knowledge or data on the COVID-19 disease course in MS patients receiving DMTs, at present there is no recommendation to stop the different DMTs and therefore expose MS patients to the risk of MS exacerbations. We, therefore, recommend continuing the current DMT specifically with [the following possible modifications]:

DMTs that can be prescribed and used as usual:

DMTs whose start or continuation might be delayed, based upon individual circumstances:

A full translation of the Italian guidance by neurology professor Gavin Giovannoni can be found here.

The MS Trust adds to its guidance Mayzent (siponimod) and Arzerra (ofatumumab), which are available by private prescription in the U.K. According to the Trust, using these DMTs could also affect your risk regarding COVID-19 and should be discussed with your neurologist or healthcare professional.

It also cautions that Gilenya (fingolimod) may increase your chances of having more severe viral and other infections, including COVID-19. However, if you are already taking fingolimod, stopping can lead to rebound MS disease activity, which in many cases would outweigh the risks of the virus. If you are considering beginning a course of fingolimod in the near future, you and your neurologist could consider an alternative DMT for the time being.

Finally, it adds that Haematopoietic stem cell transplantation (HSCT) is an intense chemotherapy treatment for MS. It aims to stop the damage MS causes by wiping out and then regrowing your immune system, using your stem cells. This treatment greatly hampers your immune system for a period of time and you and your neurologist or healthcare professional should consider delaying this treatment.

The National MS Society in the U.S. is much less specific about DMTs, saying only: People with MS should continue disease-modifying therapies (DMTs) and discuss specific risks with their MS healthcare provider prior to stopping a DMT. Neither MS Australia nor the European Multiple Sclerosis Platform offers any DMT-specific guidance.

My wife and I were planning to join our son and his family on a short cruise at the end of April. Two days ago we were still considering making this trip. Even though Im 71 and have been treated with Lemtrada, I thought that with proper precautions the risks would be minimal.

However, Dr. Anthony Fauci, the top infectious disease expert in the U.S., says that elderly people shouldnt take a cruise. Period. Dr. Fauci is a man whom I reported on for many years when I worked as a journalist I highly respect his judgment and knowledge.

Additionally, the U.S. Centers for Disease Control and Prevention has issued guidance recommending that travelers, particularly those with underlying health issues, defer all cruise ship travel worldwide. The U.S. State Department has issued the same message. Former Food and Drug Administration head Scott Gottlieb says that Everyone over 60 should become a hermit for a month.

Since then, things have become even more serious.

So, no cruise with the grandkids this year. Were postponing it until 2021. Lets all hope were out of the coronavirus woods by then. Lets hope that a vaccine for this coronavirus has been developed. Lets also hope that people who think that vaccines harm, rather than help, will see the light and get themselves and their children vaccinated for influenza, measles, and other diseases for which prevention is available. Lets do what we can to hold the line on all infectious diseases.

Youre invited to visit my personal blog at http://www.themswire.com.

***

Note: Multiple Sclerosis News Today 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. The opinions expressed in this column are not those of Multiple Sclerosis News Today or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to multiple sclerosis.

Ed Tobias is a retired broadcast journalist. Most of his 40+ year career was spent as a manager with the Associated Press in Washington, DC. Tobias was diagnosed with Multiple Sclerosis in 1980 but he continued to work, full-time, meeting interesting people and traveling to interesting places, until retiring at the end of 2012.

Link:

As COVID-19 Spreads, Here Are Disease-Modifying Therapy Guidelines - Multiple Sclerosis News Today

Heart disease is the primary cause of death worldwide, principally because the heart has minimal ability to regenerate muscle tissue. Myocardial infarction (heart attack) caused by coronary artery disease leads to heart muscle loss and replacement with scar tissue, and the heart's pumping ability is permanently reduced. Breakthroughs in stem cell biology in the 1990s and 2000s led to the hypothesis that heart muscle cells (cardiomyocytes) could be regenerated by transplanting stem cells or their derivatives. It has been 18 years since the first clinical trials of stem cell therapy for heart repair were initiated (1), mostly using adult cells. Although cell therapy is feasible and largely safe, randomized, controlled trials in patients show little consistent benefit from any of the treatments with adult-derived cells (2). In the meantime, pluripotent stem cells have produced bona fide heart muscle regeneration in animal studies and are emerging as leading candidates for human heart regeneration.

In retrospect, the lack of efficacy in these adult cell trials might have been predicted. The most common cell type delivered has been bone marrow mononuclear cells, but other transplanted cell types include bone marrow mesenchymal stromal cells and skeletal muscle myoblasts, and a few studies have used putative progenitors isolated from the adult heart itself. Although each of these adult cell types was originally postulated to differentiate directly into cardiomyocytes, none of them actually do. Indeed, with the exception of skeletal muscle myoblasts, none of these cell types survive more than a few days in the injured heart (see the figure). Unfortunately, the studies using bone marrow and adult resident cardiac progenitor cells were based on a large body of fraudulent work (3), which has led to the retraction of >30 publications. This has left clinical investigators wondering whether their trials should continue, given the lack of scientific foundation and the low but measurable risk of bleeding, stroke, and infection.

Additionally, investigators have struggled to explain the beneficial effects of adult cell therapy in preclinical animal models. Because none of these injected cell types survive and engraft in meaningful numbers or directly generate new myocardium, the mechanism has always been somewhat mysterious. Most research has focused on paracrine-mediated activation of endogenous repair mechanisms or preventing additional death of cardiomyocytes. Multiple protein factors, exosomes (small extracellular vesicles), and microRNAs have been proposed as the paracrine effectors, and an acute immunomodulatory effect has recently been suggested to underlie the benefits of adult cell therapy (4). Regardless, if cell engraftment or survival is not required, the durability of the therapy and need for actual cells versus their paracrine effectors is unclear.

Of particular importance to clinical translation is whether cell therapy is additive to optimal medical therapy. This remains unclear because almost all preclinical studies do not use standard medical treatment for myocardial infarction. Given the uncertainties about efficacy and concerns over the veracity of much of the underlying data, whether agencies should continue funding clinical trials using adult cells to treat heart disease should be assessed. Perhaps it is time for proponents of adult cardiac cell therapy to reconsider the approach.

Pluripotent stem cells (PSCs) include embryonic stem cells (ESCs) and their reprogrammed cousins, induced pluripotent stem cells (iPSCs). In contrast to adult cells, PSCs can divide indefinitely and differentiate into virtually every cell type in the human body, including cardiomyocytes. These remarkable attributes also make ESCs and iPSCs more challenging to control. Through painstaking development, cell expansion and differentiation protocols have advanced such that batches of 1 billion to 10 billion pharmaceutical-grade cardiomyocytes, at >90% purity, can be generated.

Preclinical studies indicate that PSC-cardiomyocytes can remuscularize infarcted regions of the heart (see the figure). The new myocardium persists for at least 3 months (the longest time studied), and physiological studies indicate that it beats in synchrony with host myocardium. The new myocardium results in substantial improvement in cardiac function in multiple animal models, including nonhuman primates (5). Although the mechanism of action is still under study, there is evidence that these cells directly support the heart's pumping function, in addition to providing paracrine factors. These findings are in line with the original hope for stem cell therapyto regenerate lost tissue and restore organ function. Additional effects, such as mechanically buttressing the injured heart wall, may also contribute.

Breakthroughs in cancer immunotherapy have led to the adoption of cell therapies using patient-derived (autologous) T cells that are genetically modified to express chimeric antigen receptors (CARs) that recognize cancer cell antigens. CAR T cells are the first U.S. Food and Drug Administration (FDA)approved, gene-modified cellular pharmaceutical (6). The clinical and commercial success of autologous CAR T cell transplant to treat B cell malignancies has opened doors for other complex cell therapies, including PSC derivatives. There is now a regulatory path to the clinic, private-sector funding is attracted to this field, and clinical investigators in other areas are encouraged to embrace this technology. Indeed, the first transplants of human ESC-derived cardiac progenitors, surgically delivered as a patch onto the heart's surface, have been carried out (7). In the coming years, multiple attempts to use PSC-derived cardiomyocytes to repair the human heart are likely.

What might the first human trials look like? These studies will probably employ an allogeneic (non-self), off-the-shelf, cryopreserved cell product. Although the discovery of iPSCs raised hopes for widespread use of autologous stem cell therapies, the current technology and regulatory requirements likely make this approach too costly for something as common as heart disease, although this could change as technology and regulations evolve. Given that it would take at least 6 months to generate a therapeutic dose of iPSC-derived cardiomyocytes, such cells could only be applied to patients whose infarcts are in the chronic phase where scarring (fibrosis) and ventricular remodeling are complete. Preclinical data indicate that chronic infarcts benefit less from cardiomyocyte transplantation than do those with active wound-healing processes.

Adult cells from bone marrow or the adult heart secrete beneficial paracrine factors but do not engraft in the infarcted heart. Pluripotent stem cells give rise to cardiomyocytes that engraft long term in animal models, beat in synchrony with the heart, and secrete beneficial paracrine factors. Long-term cardiomyocyte engraftment partially regenerates injured heart, which is hypothesized to bring clinical benefits.

The need for allogeneic cells raises the question of how to prevent immune rejection, both from innate immune responses in the acute phase of transplantation or from adaptive immune responses that develop more slowly through the detection of non-self antigens presented by major histocompatibility complexes (MHCs). A current strategy is the collection of iPSCs from patients who have homozygous MHC loci, which results in exponentially more MHC matches with the general population. However, studies in macaque monkeys suggest that MHC matching will be insufficient. In a macaque model of brain injury, immunosuppression was required to prevent rejection of MHC-matched iPSC-derived neurons (8). Similarly, MHC matching reduced the immunogenicity of iPSC-derived cardiomyocytes transplanted subcutaneously or into the hearts of rhesus macaques, but immunosuppressive drugs were still required to prevent rejection (9).

Numerous immune gene editing approaches have been proposed to circumvent rejection, including preventing MHC class I and II molecule expression, overexpressing immunomodulatory cell-surface factors, such CD47 and human leukocyte antigen E (HLA-E) and HLA-G (two human MHC molecules that promote maternal-fetal immune tolerance), or engineering cells to produce immunosuppressants such as programmed cell death ligand 1 (PDL1) and cytotoxic T lymphocyteassociated antigen 4 (CTLA4) (10). These approaches singly or in combination seem to reduce adaptive immune responses in vitro and in mouse models. Overexpressing HLA-G or CD47 also blunts the innate natural killer cellmediated response that results from deleting MHC class I genes (11). However, these manipulations are not without theoretical risks. It could be difficult to clear viral infections from an immunostealthy patch of tissue, and possible tumors resulting from engraftment of PSCs might be difficult to clear immunologically.

Ventricular arrhythmias have emerged as the major toxicity of cardiomyocyte cell therapy. Initial studies in small animals showed no arrhythmic complications (probably because their heart rates are too fast), but in large animals with human-like heart rates, arrhythmias were consistently observed (5, 12). Stereotypically, these arrhythmias arise a few days after transplantation, peak within a few weeks, and subside after 4 to 6 weeks. The arrhythmias were well tolerated in macaques (5) but were lethal in a subset of pigs (12). Electrophysiological studies indicate that these arrhythmias originate in graft regions from a source that behaves like an ectopic pacemaker. Understanding the mechanism of these arrhythmias and developing solutions are major areas of research. There is particular interest in the hypothesis that the immaturity of PSC-cardiomyocytes contributes to these arrhythmias, and that their maturation in situ caused arrhythmias to subside.

A successful therapy for heart regeneration also requires understanding the host side of the equation. PSC-derived cardiomyocytes engraft despite transplantation into injured myocardium that is ischemic with poor blood flow. Although vessels eventually grow in from the host tissue, normal perfusion is not restored. Achieving a robust arterial input will be key to restoring function, which may require cotransplanting other cell populations or tissue engineering approaches (13, 14). Most PSC-mediated cardiac cell therapy studies have been performed in the subacute window, equivalent to 2 to 4 weeks after myocardial infarction in humans. At this point, there has been insufficient time for a substantial fibrotic response. Fibrosis has multiple deleterious features, including mechanically stiffening the tissue and creating zones of electrical insulation that can cause arrhythmias. Extending this therapy to other clinical situations, such as chronic heart failure, will require additional approaches that address the preexisting fibrosis. Cell therapy may again provide an answer because CAR T cells targeted to cardiac fibroblasts reduced fibrosis (15).

Developing a human cardiomyocyte therapy for heart regeneration will push the limits of cell manufacturing. Each patient will likely require a dose of 1 billion to 10 billion cells. Given the widespread nature of ischemic heart disease, 105 to 106 patients a year are likely to need treatment, which translates to 1014 to 1016 cardiomyocytes per year. Growing cells at this scale will require introduction of next generation bioreactors, development of lower-cost media, construction of large-scale cryopreservation and banking systems, and establishment of a robust supply chain compatible with clinical-grade manufacturing practices.

Beyond PSC-cardiomyocytes, other promising approaches include reactivating cardiomyocyte division and reprogramming fibroblasts to form new cardiomyocytes. However, these approaches are at an earlier stage of development, and currently, PSC-derived cardiomyocyte therapy is the only approach that results in large and lasting new muscle grafts. The hurdles to this treatment are known, and likely addressable, thus multiple clinical trials are anticipated.

Acknowledgments: C.E.M. and W.R.M. are scientific founders of and equity holders in Sana Biotechnology. C.E.M. is an employee of Sana Biotechnology. W.R.M. is a consultant for Sana Biotechnology. C.E.M. and W.R.M. hold issued and pending patents in the field of stem cell and regenerative biology.

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Stem cells and the heartthe road ahead - Science Magazine

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

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

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

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

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

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

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

Amongst other elements, the report includes:

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

Key Topics Covered

1. Preface

2. Executive Summary

3. Introduction

4. Market Overview

5. Regulatory Landscape

6. Stem Cell Therapy Contract Manufacturers in North America

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

8. Partnerships and Collaboration

9. Contract Manufacturing Opportunity Assessment

10. Capacity Analysis

11. Demand Analysis

12. Market Forecast

13. Key Performance Indicators

14. Concluding Remark

15. Executive Insights

16. Appendix 1: Tabulated Data

17. Appendix 2: List of Companies and Organizations

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

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

CONTACT: ResearchAndMarkets.comLaura Wood, Senior Press Managerpress@researchandmarkets.comFor E.S.T Office Hours Call 1-917-300-0470For U.S./CAN Toll Free Call 1-800-526-8630For GMT Office Hours Call +353-1-416-8900

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

Gimble and his co-authors recommend the FDA re-evaluate how it categorizes tissues as structural or cellular to recognize the different safety profiles of stem cells products. They also think the FDA should work with accreditation agencies like AABB and FACT to develop meaningful accreditation standards, along with a national registry for stem cell therapies.

This measured third way seeks to carve a compromise between the FDAs regulationist faction and wild west stem cell providers -- a new ideological center that synthesizes the aspirations of two opposing parties in an effort to achieve a compromise.

It must be stated that there are serious questions as to whether the authors proposed polarity is in fact an artifice created for the specific purpose of legitimizing their third way. Upon serious inspection, the authors stated dangers of stem cell clinics may actually be a disingenuous straw man created for their own business interests.

To promote their own agenda and to gain favor with the FDA, Gimble and his co-authors seem to have thrown stem cell clinicians like Dr. Mark Berman under the proverbial bus. Berman, who is a defendant in a FDA lawsuit over his use of autologous cells, recently won a victory in federal court. The judge found that the FDA may not have regulatory authority over Bermans procedures and that a trial needs to be held to resolve the issue.

Nevertheless, the impetus behind the authors recommendations is to move forward with bringing stem cells to patients faster and in a safer manner. Regrettably, the FDA does anything but take the authors seriously. In a lengthy response to the Gimble article, Dr. Peter Marks, Director of the FDAs Center for Biologics Evaluation and Research, merely reiterates the agencys firmly-established regulationist position.

After commending the authors for their desire to accelerate the scientific investigation and development of stem cell therapies, Marks demonstrates the FDAs backward-looking posture by stating the agencys regulation of stem cells is distinct from the practice of medicine and should be left alone.

This is an existing paradigm that has been in place for decades, Marks wrote. Autologous cellular therapies do hold tremendous promise, but they will only find their way into routine clinical practice to bring benefit to all patients if they are held to the same standards to demonstrate safety and efficacy as other unproven medical products.

Marks attempts to bolster his argument by citing patient safety, the dearth of research on adipose-derived stem cells and the unethical bad actor clinics that exploit desperate patients. However, the spirit of his position reveals a resistance to any sort of change whatsoever.

Marks and the FDA are living in the past. They consider your cells to be unproven medical products. Apparently, they have yet to realize that the stem cell poles have already shifted.

A. Rahman Ford, PhD, is a lawyer and research professional. He is a graduate of Rutgers University and the Howard University School of Law, where he served as Editor-in-Chief of the Howard Law Journal. Rahman lives with chronic inflammation in his digestive tract and is unable to eat solid food. He has received stem cell treatmentin China.

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FDA Scoffs at Third Way Forward in Stem Cell Therapy - Pain News Network

The excitement about cell and gene therapies is almost tangible within the biotech and pharma industry. Over 950 companies are actively developing advanced therapies, which are expected to make exceptional improvements to peoples lives in the next decade. Although hopes are high, the industry still faces a number of challenges in cell and gene therapy manufacturing, mainly around being able to deliver these often difficult to make, complex treatments at the scale needed to meet patient demand.

The unprecedented growth of the industry, alongside the need to develop scalable manufacturing strategies, has led to a number of challenges that need to be addressed urgently. Previously, patient numbers were so small that processes were highly manual and required numerous skilled operators. However, the recent success of early gene therapy trials means upscaling now needs to be considered right from the start.

In the early days the aim was just to get to the clinic, said Lindsey Clarke, Head of Cell and Gene Therapy EMEA at Bio-Techne. Scale didnt come into it so much, but now the conversations we are having focus much more on making these complex therapies at a scale needed for a commercial medicine. There are increased efforts on finding solutions that dont just work for trials with 10 patients, but will still work at 1000 times that scale.

Life science tools and technology provider, Bio-Techne, has made it its mission to further support the cell and gene therapy industry by channeling its expertise into developing technologies that can help to scale manufacturing processes. The companys commitment is highlighted by its recent investment into a new good manufacturing practice (GMP) manufacturing facility in St Paul, Minnesota, US, that will focus on producing raw materials for use in cell and gene therapy applications.

We have realized that if all our customers are to be successful with their therapies then there will be a huge demand for raw materials, Clarke explained. So weve started building that capability, ahead of time. But its not just about supply, we are also innovating, from simple things like looking closely at the format our products come in and making them more compatible with large-scale manufacturing to whole new product ranges.

Bio-Technes investment in the new GMP manufacturing facility is a solution to meet the growing demand for raw materials needed for cell and gene therapy manufacturing. But its just one piece of a large puzzle: cell and gene therapy developers also need to consider the complex logistics required to deliver their therapies to the clinic, particularly when its an autologous therapy.

The process from the patient to the clinician, to the apheresis collection, to the manufacturing site, then the complex manufacturing process and then delivery back to the patient is highly complex.

Another key challenge closely related to upscaling is the great risk of human error in manual processes. Many of the cell and gene manufacturing processes currently in place have been developed with small patient numbers in mind and involve manual steps.

Humans are an excellent source of error and risk, explained Clarke. When youre manufacturing in a GMP environment, you need highly-skilled, trained operators and there is a shortage of them out there. Automation is going to be key to address this issue. Not only does it reduce the manpower that is required, but it can also streamline the processes and make them less risky, more scalable, and reproducible as well, Clarke added.

With cell and gene therapy products, various analytical methods are used to assess critical quality attributes during development and manufacturing. These reflect the identity, potency, purity, safety, and stability of the product. However, such methods are frequently complex, non-standardized, time-consuming, and performed manually by trained operators.

Organizations such as Cell and Gene Therapy Catapult have called for the development of new analytical solutions for quality testing of advanced therapies throughout the manufacturing process. More automated analytical technologies have the potential to increase facility throughput and make quality control (QC) faster, less error-prone, more reproducible, and more GMP compliant.

Although Bio-Techne has a long-standing history of developing quality proteins, antibodies, small molecules, and immunoassays, it has expanded into automated protein analytical technologies in recent years.

For viral and non-viral vectors, Bio-Technes ProteinSimple branded platforms are rapidly being adopted by cell and gene therapy developers for assessment of vector identity, purity, and stability. Compared to traditional methods like Western blot, SDS-PAGE, and ELISA, ProteinSimples technology platform is based on capillary electrophoresis and microfluidics and provides a fully automated and accurate quantitative analysis of vectors.

We are also seeing Micro-Flow Imaging (MFI), a more common image-based analytical platform in biologics, used to characterize subvisible particles for quality control of cell and gene therapy products, explained Kamar Johnson, Commercial Development Manager in Cell and Gene Therapy at Bio-Techne. These automated platforms offer ease of use, rapid time to result, enhanced reproducibility and software that meets GMP requirements.

Collaboration lies at the heart of successful innovation. It is especially important at the interface between process development and manufacturing, said Johnson.

Not everyone is an expert in everything, we all have our particular niches of expertise, added Clarke. We believe that we need to collaborate to get the innovation that will help change the way we manufacture cell and gene therapies. Collaboration is the key to solving the challenges of the cell and gene therapy industry.

On that note, Bio-Techne recently partnered with Fresenius Kabi and Wilson Wolf to form a new joint venture that provides manufacturing technologies and processes for the development and commercialization of new cell and gene therapies.

The collaboration combines Bio-Technes expertise of proteins, reagents, media, and gene editing technologies with Fresenius Kabis Lovo cell processing system and the bioreactor expertise from Wilson Wolf with its G-Rex technology that is designed as a scalable and practical platform for personalized cell therapies.

As processes develop and technologies evolve, the cell and gene therapy space will be confronted with new challenges. At Bio-Techne, the team is keeping an eye out for interesting trends that might affect the industry.

I see the induced pluripotent stem cell (iPSC) therapy field continuing to grow with more allogeneic cell therapies being developed, says Johnson. Allogeneic manufacturing is potentially less complicated than autologous manufacturing due to the ability to provide off-the-shelf products when patients need them.

Although the challenges in cell and gene therapy manufacturing remain a problem, companies like Bio-Techne are establishing quicker, simpler, and more automated options within quality control, manufacturing, and process development.

Wherever we go, we see newer technologies supporting cell and gene therapy manufacturing, says Clarke. Within our industry, changes come so rapidly and the treatments have shown so much promise that there is a lot of focus on cell and gene therapies. This puts a lot of pressure on us as an industry to provide these treatments. I believe that collaboration is the key to tackling this problem.

To learn more about the challenges in cell and gene therapy manufacturing and how to solve them, visit Bio-Technes website or get in touch with the experts here!

Images via Shutterstock.com

Author: Larissa Warneck

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Tackling the Challenges in Cell and Gene Therapy... - Labiotech.eu

A new proof-of-concept study has found a combination of two drugs, already approved by the FDA for other uses, may boost the release of stem cells from bone marrow and accelerate the healing of broken bones. Only demonstrated in animals at this stage, the researchers suggest clinical trials could progress rapidly considering the drugs have already been demonstrated as safe in humans.

"The body repairs itself all the time, says corresponding author on the study Sara Rankin. We know that when bones break they will heal, and this requires the activation of stem cells in the bone. However, when the damage is severe, there are limits to what the body can do of its own accord.

A great deal of current research is focusing on mesenchymal stem cell (MSC) therapies. MSCs are a type of adult stem cell that can grow into a variety of different cell types including muscle, fat or bone. Many current MSC treatments in development involve extracting a small number from a patient, growing them in laboratory conditions, then injecting them back into the patient.

The new research set out to investigate whether any currently approved drugs can function to mobilize the bodys natural ability in releasing MSCs, with a view on speeding up healing of bone fractures. A study published in the journal npj Regenerative Medicine, describes the testing of two already approved drugs in a rodent spinal injury model.

The two drugs tested were an immunostimulant called Plerixafor, used to stimulate the release of stem cells from bone marrow in cancer patients, and a beta-3 adrenergic agonist developed to help bladder control.

The results suggest the duo of drugs mobilize MSCs into the bloodstream and speed up the process of bone formation and healing by enhancing the binding of calcium to the injury site. Tariq Fellous, first author on the new study, suggests the next step is to investigate whether this drug combination enhances blood MSC levels in human subjects.

We first need to see if these medications release the stem cells in healthy volunteers, before we can then test them in patients with fractures, says Fellous. We have the drugs and know they are safe to use in humans - we just need the funding for the human trials.

The researchers say prior studies have identified circulating MSCs increase in volume following injuries such as burns, bone fractures, and even heart attack. The hypothesis is that the release of MSCs is a physiological process aiding general regeneration following injury, and if circulating numbers of MSCs could be pharmacologically enhanced then a variety of types of tissue regeneration could be accelerated.

It is important to note the current study only examined increases in circulating MSCs and the rate of spine injury healing compared to no drug treatment. The current research offers no indication whether the drug duo influences nerve healing or restores movement.

So, more work is certainly necessary to understand how clinically useful these results actually are. However, as the studys co-first author Andia Redpath notes, this re-purposing of existing medicines to boost stem cell activity is an easier, cheaper, and more efficient way to enhance healing compared to other, more complex and time-consuming, stem cell treatments in development.

Rather than devising new stem cell treatments from scratch that involve lengthy and expensive trials, our approach harnesses the power of the bodys own stem cells, using existing drugs, says Redpath. We already know the treatments in our study are safe, its now just a matter of exploring further if they help our bodies heal.

The new study was published in the journal npj Regenerative Medicine.

Source: Imperial College London

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Experimental study speeds up bone healing with 2 common medications - New Atlas

Former IMPACT world champion Austin Aries was spotted backstage at Wednesdays taping of AEW Dynamite from Atlanta. Aries later told Bodyslam.net that he was representing the new stem cell therapy he works for, BioXcellerator, and did in fact meet with AEW officials, as well as Diamond Dallas Pages DDPY brand.

I work for BioX. I did have meetings about partnerships and collaborations with both DDP and DDPY Brand as well as AEW.

Aries officially announced his new position, which is as Director of the Athletic Performance Division, on Instagram.

BIG ANNOUNCEMENT!!! Many of you know that I went down to Medellin, Colombia in December for @bioxcellerator_ #stemcelltherapy, and now after a couple months, my neck and lower back feel really good. I was honestly so impressed with the entire @bioxcellerator_ operation. So much so, that upon returning I had conversations about joining the @bioxcellerator_ team in some capacity. With that, I am EXTREMELY EXCITED TO ANNOUNCE that I have officially joined BioXcellerator as Director Of Athletic Performance Division. My role will be to continue connecting high level athletes, entertainers and influencers with all the amazing benefits of @bioxcellerator_ #stemcelltherapy. As someone whose passion is helping people, I couldnt pass up the opportunity to be a part of this company that is changing lives with their cutting edge #stemcelltherapy. So, anyone whos been thinking about, or interested in finding out more about BioXcellerator stem cell therapy, Im your guy! Please DM or email me at Austin@BioXcellerator.com. Heres to all our improved health and happiness in 2020 and beyond!

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BIG ANNOUNCEMENT!!! . Many of you know that I went down to Medellin, Colombia in December for @bioxcellerator_ #stemcelltherapy, and now after a couple months, my neck and lower back feel really good. I was honestly so impressed with the entire @bioxcellerator_ operation. So much so, that upon returning I had conversations about joining the @bioxcellerator_ team in some capacity. . With that, I am EXTREMELY EXCITED TO ANNOUNCE that I have officially joined BioXcellerator as Director Of Athletic Performance Division. My role will be to continue connecting high level athletes, entertainers and influencers with all the amazing benefits of @bioxcellerator_ #stemcelltherapy. As someone whose passion is helping people, I couldnt pass up the opportunity to be a part of this company that is changing lives with their cutting edge #stemcelltherapy. . So, anyone whos been thinking about, or interested in finding out more about BioXcellerator stem cell therapy, Im your guy! Please DM or email me at Austin@BioXcellerator.com. Heres to all our improved health and happiness in 2020 and beyond! . . #bioxcellerator #bioxmen #bioxwomen #stemcell #stemcells #stemcelltherapy #medellin #colombia

A post shared by Austin Aries (@austinhealyaries) on Feb 17, 2020 at 8:49am PST

Home NEWS Austin Aries Announces Position With Stem Cell Therapy Company, Says He Met With AEW Regarding Partnership

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Austin Aries Announces Position With Stem Cell Therapy Company, Says He Met With AEW Regarding Partnership - LordsofPain.net