Introduction

Sangamo Therapeutics (SGMO) is a clinical-stage biotech company focusing on the research and development of genomic medicine across 4 distinct technology platforms: gene therapy, cell therapy, in vivo genome editing, and in vivo genome regulation.

Sangamo is best-known for developing its proprietary gene-editing technology, zinc finger proteins ("ZFPs"), which is a naturally occurring class of transcription factor proteins found in humans and other species. The company has used its internal know-how and technical expertise to develop a proprietary synthetic ZFP platform with potential clinical utility in ex vivo gene-edited cell therapy, in vivo genome editing, and in vivo genome regulation.

ZFPs also can be engineered to make zinc finger nucleases ("ZFNs") which proteins that can be used to specifically modify DNA sequences by knocking in or knocking out select genes, or genome editing, and ZFP transcription-factors ("ZFP-TFs") which are proteins that can be used to selectively increase or decrease gene expression.

Sangamo is developing a series of clinical programs, which are either wholly-owned or partnered with well-established pharma and biotech companies, to focus on 3 therapeutics areas in inherited metabolic disease ("IMDs"), central nervous system ("CNS), and inflammatory and autoimmune diseases. Its full list of clinical pipeline programs is listed in Figure 1.

Figure 1 Sangamo Therapeutics' Clinical Pipeline (Source)

The company's most advanced program is an investigational gene therapy for severe hemophilia A, SB-525. SB-525 is developed under a global collaboration with Pfizer (PFE), of which the rights of SB-525 have been transferred to Pfizer to run a phase 3 trial. In December 2019, both companies announced updated initial data from the phase1/2 trial of SB-525. SB-525 was generally well-tolerated and demonstrated a sustained increase in Factor VIII activity. SB-525 has been granted RMAT, Orphan Drug, and Fast Track designation by the FDA as well as Orphan Medicinal Product Designation by the European Medicines Agency ("EMA").

Beyond SB-525, the company is also investigating 2 wholly-owned gene therapy. ST-920 is being evaluated to treat Fabry disease, a rare inherited metabolic disease, in a phase 1 study in the US and UK. SB-920 has received Orphan Drug designation by the FDA. The company also plans to advance ST-101 into clinical trials in 2021 to treat phenylketonuria ("PKU") which is a rare inherited disorder that originates from a defect in the PAH gene and results in a harmful accumulation of phenylalanine in cells throughout the body.

Sangamo is working with Sanofi (NASDAQ:SNY) to develop ex vivo gene-edited cell therapies, ST-400 and BIVV-003, for transfusion-dependent beta-thalassemia ("TDT") and sickle cell disease ("SCD") respectively. Both ST-400 and BIVV-003 are related product candidates using the same technology involving gene editing of a patient's own hematopoietic stem progenitor cells using non-viral delivery of ZFN technology.

Sangamo is the phase 1/2 study of ST-400 in 6 patients with TDT while Sanofi is recruiting the phase1/2 study evaluating BIVV-003 in patients with SCD, and Sanofi is responsible for the subsequent development, manufacturing, and commercialization of both programs.

In Dec 2009, Sangamo presented interim results for the first 3 patients ST-400. As of the data cut date, 2 more patients have been enrolled although they were not included in the interim updates. The 3 patients treated with ST-400 experienced prompt hematopoietic reconstitution, demonstrating neutrophil engraftment in 14-22 days and platelet engraftment in 22-35 days. No emerging clonal hematopoiesis had been observed as measured by on-target indel pattern monitoring in the three treated patients. The downside of the data readout is that its treatment of TDT appears to be not as efficacious as other competitors such as bluebird bio (BLUE).

Sangamo also has a global collaboration and license agreement with Kite Pharma, a wholly-owned subsidiary of Gilead Sciences (GILD), for the development of engineered cell therapies for cancer. The company is working together with Kite to design ZFNs and viral vectors to disrupt and insert select genes in T cells and natural killer cells. The first program of this agreement expected to start a clinical trial in 2020 is KITE-307, which is an allogeneic anti-CD19 CAR-T cell therapy. Given the well-documented struggles of Kite's approved autologous CAR-T, Yescarta, the success of allogeneic CAR-Ts will be very beneficial.

Lastly, Sangamo is also evaluating the potential of regulatory T-cells ("Tregs") genetically modified with a CAR ("CAR-Tregs") in solid organ transplantation. CAR-Treg cell therapies are being conducted in several preclinical studies in autoimmune and inflammatory diseases such as multiple sclerosis ("MS") and inflammatory bowel disease ("IBD"). The most advanced CAR-Treg cell therapy is TX200, which is an autologous treatment for the prevention of solid organ transplant rejection and the clinical trial is expected to be initiated in 2020.

Sangamo is only planning to start a new clinical trial for its in vivo genome editing programs. SB-913 is a second-generation ZFNs program that will be used to treat Mucopolysaccharidosis type II ("MPS II") and a new clinical trial is planned to start this year.

The company had previous programs from first-generation ZFNs that have been halted as they did not demonstrate enough clinical benefits. The company plans to use data from the SB-913 study to definite the next steps for its in vivo genome editing programs.

The company also has several preclinical programs evaluating their ZFP-TF technology as a novel therapeutic approach for CNS diseases. In December, Sangamo announced a collaboration with Biogen (BIIB) to develop and commercialize ST-501 for tauopathies including Alzheimer's disease, ST-502 for synucleinopathies including Parkinson's disease, a third undisclosed neuromuscular disease target, and up to 9 additional neurological disease targets. Under the terms of the agreement, Biogen will pay Sangamo $350M upfront, inclusive of a license fee and equity investment, and Sangamo is eligible to receive up to $2.37B in future milestones.

Sangamo also has a partnership with Pfizer and Takeda (NYSE:TAK) to develop and evaluate ZFP-TFs. The company is working with Pfizer to evaluate ALS and frontotemporal lobar degeneration ("FTLD") that are linked to the mutations in the C9ORF72 gene. In the partnership with Takeda, the company is evaluating a preclinical program for Huntington's disease in which ZFP-TF is designed to differentially down-regulate the mutated disease-causing huntingtin gene ("HTT gene") while preserving the expression of the normal version of the gene.

As of 31 December, 2019, cash and equivalents on hand was $385M. The amount is excluding the $350M injection from the collaboration with Biogen, and when factored in, cash on hand should comfortably be in the range of high $600-700M. This should give them a comfortable runway to fund all operations well into 2021, an important point given that the recent stock market crash which limits any secondary offering options.

Impressively, the company has managed to strike several high-profile partnerships with 5 global biotech/big pharma companies. Such partnerships not only validate Sangamo's technology and capabilities, but they also provide future avenues of funding with as much as $6.34B royalties on net product sales and potential milestone payments due to the company.

Figure 1 Sangamo Therapeutics' Partnerships (Source)

In terms of competition, the company competes with several players, particularly in the cell and gene therapy space. bluebird bio has more advanced programs in both TDT and SCD and, to date, has shown much better efficacy. There are also other companies such as CRISPR Therapeutics (CRSP) that are using an alternative gene-editing method, CRISPR/Cas9 in gene therapy. Other companies such as Editas Medicine (EDIT) and Intellia Therapeutics (NTLA) are also developing CRISPR/Cas9 for treatments in TDT and SCD, although it must be noted that these are not their lead programs.

In terms of allogeneic CAR-T, there are more established players such as Allogene Therapeutics (ALLO), Cellectis (CLLS), and Precision Biosciences (DTIL). The main difference among these companies is primarily the choice of gene-editing tools with Allogene and Cellectis using TALEN while Precision is using ARCUS. All these companies are, currently, in a similar stage of clinical development.

In addition to healthy donors derived allogeneic therapies, Fate Therapeutics (FATE) is developing allogeneic therapies from induced pluripotent stem cells ("iPSCs") as a renewable cell source. The advantage of this is that product consistency and potency will be improved, and the manufacturing process will be akin to the well-established biologics where they are produced from a single cell line. It is notable to note that Allogene is also investigating using iPSCs as a renewable cell source. Also, Atara Biotherapeutics (ATRA) is developing an Epstein-Barr Virus ("EBV")-based allogeneic T cell therapy platform. Their lead program is in Phase 3 and a BLA filing is expected by the second half of the year. That should put them in the lead position of commercializing an allogeneic T cell therapy and the company is gradually moving into allogeneic CAR-T space as well.

Sangamo is, currently, trading at a market cap of around $700M, which is almost as much as its cash position. While its cash position will eventually deplete to fund operations and clinical trials, the current valuation means that there is also no value for its technology and intellectual position. I consider it a good time to take up a small position in Sangamo, especially if investors have a time horizon of at least a year to weather the COVID-19 black swan event and wait for further clinical updates from the company.

It must be cautioned though that investing in clinical-stage biotech can be extremely risky, given the binary nature of the field. This is especially so, given the market turmoil from the COVID-19 pandemic. The pandemic has also led to several countries announcing lockdowns, which have disrupted supply chain and operations. Several clinical trials have already been delayed globally and this may impact Sangamo negatively, as their cash burn will continue even if clinical trials are delayed.

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

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Sangamo Therapeutics: Market Cap Is At A Bargain Relative To Its Cash Position - Seeking Alpha

A SIX-year-old boy has received his super cells after having a stem cell transplant to fight off cancer.

Oscar Saxelby-Lee received his transplant yesterday (March 13) from his dad, Jamie Lee, who donated his cells to his little boy.

The Hand in Hand for Oscar Facebook page announced the heart-warming news: May these super cells be the beginning of something special just like you Ozzy Bear. We hope this gift will allow you a brand-new start to good health and end the monstrous ordeals you have been faced with. You will always have us by your side, hoping, praying and fighting for the brighter future you so very much deserve.

The emotive post continued: Daddy will always be with you, both inside and out now. How amazing is that. And how utterly amazing are you. You are so so close baby, keep going. Now for the difficult part of growing them and keeping him stable, but hes got this."

Oscar experienced a few slight implications during the transplant due to a reaction, but his parents have assured he is safe and is doing well.

Oscar has been in Singapore with his parents, Olivia Saxelby and Mr Lee, receiving CAR-T therapy after the community backed their appeal to raise 500,000 to afford the life-saving treatment. This is specialised therapy, which is only available in Singapore, to combat his T-cell acute lymphoblastic leukaemia. The young Worcester boy underwent a stem cell transplant last May after a search for a donor which saw thousands across the country to sign-up to a register in a bid to help.

The Worcester News previously reported that Oscar's parents were appealing for blood donations because there's a shortage due to less people donating due to the coronavirus. However, an NHS Blood and Transplant spokesman said blood stock levels are good.

The spokesman said: Our stock levels are good and we dont believe weve seen any effect on appointments. Were asking donors to keep donating as normal and to follow the latest travel and hygiene advice. Donation help us to keep stocks good so we are well prepared for every eventuality. Were regularly reviewing the situation and working closely with the government, Public Health England and the UKs other blood donation services. We're putting in place extra measures and safety is always our number one priority.

Read the original here:

Oscar Saxelby-Lee gets 'super cells' after having stem cell transplant from dad to fight off cancer - Ledbury Reporter

A 40-year-old HIV patient has been declared cured after a promising treatment has left him with no active virus. The man, Adam Castillejo, was the subject of extensive research in early 2019 after doctors failed to find HIV in his body over an 18-month period after previously being diagnosed in 2003.

Castillejo, known by the nickname London Patient lived with the disease for many years, taking medicine to manage it since 2012. That same year he was diagnosed with Hodgkins Lymphoma, and later endured a bone marrow transplant. That operation may have ultimately cured him of HIV, and appears to have made him only the second person to ever be cured of the disease that causes AIDS.

As ScienceAlert reports, the bone marrow transplant that doctors performed on Castillejo used cells from a donor with a very special genetic quirk. The cells are thought to work against HIV in the body, but there was no guarantee that the transplant would provide any concrete benefits beyond treating the cancer.

However, it appears as though the decision to treat Castillejo with the unique stem cells worked in more ways than one, and last year doctors announced they couldnt find the virus in his body after 18 months. At the time, they were hesitant to declare the London Patient cured, but after a new round of testing returned the same results, they are more confident that the active form of the virus has indeed been defeated.

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.

While this sounds like incredible news and for Castillejo, it certainly is the treatment is not an option for everyone. With cancer limiting their options, doctors used the stem cell transplant as a last resort to keep him alive. Its a serious operation and one that was only performed because Castillejos condition was so dire.

Castillejo and the other HIV patient who had similar results, known as the Berlin Patient, may be uniquely fortunate. The doctors note that there are others who have had the same transplant performed but did not improve as rapidly as the others. There are obviously many factors at work here, and as exciting as it is to see a second person cured of this terrible disease, theres a lot more work to be done before we can say HIV has been truly beaten.

Image Source: Pius Koller/imageBROKER/REX/Shutterstock

Mike Wehner has reported on technology and video games for the past decade, covering breaking news and trends in VR, wearables, smartphones, and future tech. Most recently, Mike served as Tech Editor at The Daily Dot, and has been featured in USA Today, Time.com, and countless other web and print outlets. His love ofreporting is second only to his gaming addiction.

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HIV patient appears to be totally cured after stem cell treatment - BGR

New York, March 13, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Induced Pluripotent Stem Cells Market: Global Industry Analysis, Trends, Market Size, and Forecasts up to 2025" - https://www.reportlinker.com/p05874276/?utm_source=GNW 6% over the forecast period from 2019-2025. The study on induced pluripotent stem cells market covers the analysis of the leading geographies such as North America, Europe, Asia-Pacific, and RoW for the period of 2017 to 2025.

The report on induced pluripotent stem cells market is a comprehensive study and presentation of drivers, restraints, opportunities, demand factors, market size, forecasts, and trends in the global induced pluripotent stem cells market over the period of 2017 to 2025. Moreover, the report is a collective presentation of primary and secondary research findings.

Porters five forces model in the report provides insights into the competitive rivalry, supplier and buyer positions in the market and opportunities for the new entrants in the global induced pluripotent stem cells market over the period of 2017 to 2025. Further, IGR- Growth Matrix gave in the report brings an insight into the investment areas that existing or new market players can consider.

Report Findings1) Drivers Increased government fundings and rising industry focus on the development of novel therapies Rising interest in stem cell therapy2) Restraints High the cost associated with storage3) Opportunities Growing applications of iPS cells in several biopharmaceutical applications provides extensive potential to the key players in the market

Research Methodology

A) Primary ResearchOur primary research involves extensive interviews and analysis of the opinions provided by the primary respondents. The primary research starts with identifying and approaching the primary respondents, the primary respondents are approached include1. Key Opinion Leaders associated with Infinium Global Research2. Internal and External subject matter experts3. Professionals and participants from the industry

Our primary research respondents typically include1. Executives working with leading companies in the market under review2. Product/brand/marketing managers3. CXO level executives4. Regional/zonal/ country managers5. Vice President level executives.

B) Secondary ResearchSecondary research involves extensive exploring through the secondary sources of information available in both the public domain and paid sources. At Infinium Global Research, each research study is based on over 500 hours of secondary research accompanied by primary research. The information obtained through the secondary sources is validated through the crosscheck on various data sources.

The secondary sources of the data typically include1. Company reports and publications2. Government/institutional publications3. Trade and associations journals4. Databases such as WTO, OECD, World Bank, and among others.5. Websites and publications by research agencies

Segment CoveredThe global induced pluripotent stem cells market is segmented on the basis of derived cell type, application, and end user.

The Global Induced Pluripotent Stem Cells Market by Derived Cell Type Fibroblasts Amniotic Cells Hepatocytes Keratinocytes Others

The Global Induced Pluripotent Stem Cells Market by Application Drug Development Regenerative Medicine Toxicity Testing Academic Research

The Global Induced Pluripotent Stem Cells Market by End User Research Organizations Hospitals Biopharma Industries

Company Profiles Astellas Pharma Inc. Fate Therapeutics Inc. FUJIFILM Holdings Corporation Evotec SE Japan Tissue Engineering Co., Ltd ViaCyte, Inc. Vericel Corporation Bristol-Myers Squibb Company Aastrom Biosciences, Inc. Acelity Holdings, Inc.

What does this report deliver?1. Comprehensive analysis of the global as well as regional markets of the induced pluripotent stem cells market.2. Complete coverage of all the segments in the induced pluripotent stem cells market to analyze the trends, developments in the global market and forecast of market size up to 2025.3. Comprehensive analysis of the companies operating in the global induced pluripotent stem cells market. The company profile includes analysis of product portfolio, revenue, SWOT analysis and latest developments of the company.4. IGR- Growth Matrix presents an analysis of the product segments and geographies that market players should focus to invest, consolidate, expand and/or diversify.Read the full report: https://www.reportlinker.com/p05874276/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Global induced pluripotent stem cells market is expected to grow with a CAGR of 8.6% over the forecast period from 2019-2025 - GlobeNewswire

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Future Growth Of Animal Stem Cell Therapy Market By New Business Developments - News by aeresearch

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

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

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.

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

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.

For top companies in United States, European Union and China, this report investigates and analyzes the production, value, price, market share and growth rate for the top manufacturers, key data from 2019 to 2025.

The Cancer Stem Cell Therapy market report firstly introduced the basics: definitions, classifications, applications and market overview; product specifications; manufacturing processes; cost structures, raw materials and so on. Then it analyzed the worlds main region market conditions, including the product price, profit, capacity, production, supply, demand and market growth rate and forecast etc. In the end, the Cancer Stem Cell Therapy market report introduced new project SWOT analysis, investment feasibility analysis, and investment return analysis.

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The major players profiled in this Cancer Stem Cell Therapy market report include:

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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The study objectives of Cancer Stem Cell Therapy Market Report are:

To analyze and research the Cancer Stem Cell Therapy market status and future forecast in United States, European Union and China, involving sales, value (revenue), growth rate (CAGR), market share, historical and forecast.

To present the Cancer Stem Cell Therapy manufacturers, presenting the sales, revenue, market share, and recent development for key players.

To split the breakdown data by regions, type, companies and applications

To analyze the global and key regions Cancer Stem Cell Therapy market potential and advantage, opportunity and challenge, restraints and risks.

To identify significant trends, drivers, influence factors in global and regions

To analyze competitive developments such as expansions, agreements, new product launches, and acquisitions in the keyword market.

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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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Read 150 page research report with ToC on "Single Cell Analysis Market Size, Share & Trends Analysis Report By Product, By Application (IVF, Cancer, Immunology, Neurology, Stem Cell, Non-invasive Prenatal Diagnosis), By End Use, And Segment Forecasts, 2020 - 2027" at: https://www.grandviewresearch.com/industry-analysis/single-cell-analysis-market

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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About Grand View Research

Grand View Research, U.S.-based market research and consulting company, provides syndicated as well as customized research reports and consulting services. Registered in California and headquartered in San Francisco, the company comprises over 425 analysts and consultants, adding more than 1200 market research reports to its vast database each year. These reports offer in-depth analysis on 46 industries across 25 major countries worldwide. With the help of an interactive market intelligence platform, Grand View Research helps Fortune 500 companies and renowned academic institutes understand the global and regional business environment and gauge the opportunities that lie ahead.

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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).

Market Segmentations: Global Autologous Stem Cell Based Therapies market competition by top manufacturers, with production, price, revenue (value) and market share for each manufacturer.

Based on type, report split into Embryonic Stem Cell, Resident Cardiac Stem Cells, Umbilical Cord Blood Stem Cells.

Based on the end users/applications, this report focuses on the status and outlook for major applications/end users, consumption (sales), market share and growth rate for each application, including Neurodegenerative Disorders, Autoimmune Diseases, Cardiovascular Diseases.

The report introduces Autologous Stem Cell Based Therapies basic information including definition, classification, application, industry chain structure, industry overview, policy analysis, and news analysis. Insightful predictions for the Autologous Stem Cell Based Therapies Market for the coming few years have also been included in the report.

Autologous Stem Cell Based Therapies Market landscape and market scenario includes:

The Autologous Stem Cell Based Therapies industry development trends and marketing channels are analyzed. Finally, the feasibility of new investment projects is assessed, and overall research conclusions offered.

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

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

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."

See the article here:

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

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.

Read the original post:

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.

Read the original here:

Stem cells and the heartthe road ahead - Science Magazine