In early September 2017, my daughters came home from daycare with a stomach bug that made its way around the house. Everyone else got over it pretty quickly, but I didnt. Instead, I became the sickest Ive ever been in my life with a high fever, relentless cough, chills, and vomiting to the point where I couldnt even keep down a few sips of water.

Before this, Id been in relatively good health. At 32, Id just returned to work as an attorney in the Dallas, Texas, area after maternity leave (I'm a mom of three) and I never took sick days. But for three days, I stayed home.

All I could really do was lay in bed under the blankets, hoping whatever illness I had would pass. After several days of not being able to keep any food or water down, I finally went to urgent care. There, a doctor ran blood work, gave me Zofran (an anti-nausea med), and sent me home.

Two days later, I received a call from the urgent care office.

They wouldnt tell me anything specific about my blood work but they advised me to schedule an appointment with my primary care doctor. As I didnt have one, I scheduled an appointment with a new doctor who wouldnt be able to see me for several weeks.

As the days went on, I still couldnt keep any food down and I started researching my symptoms to try to figure out what was wrong with me. I thought maybe I had a vitamin B12 deficiency and headed into urgent care again. There, the doctor advised me to go straight to the emergency room at the hospital down the street for fluids, a blood transfusion, and an appointment with a hematologist. At the ER, I did just that and was discharged with an appointment scheduled for two days later.

The next morning, a nurse from the hematologists office called and asked if I could come in that day. She told me there was a chance that the doctor might hospitalize me, so I might want to pack a bag.

I held out hope that it wasnt a big deal, but I should have realized I was seriously ill.

I was too weak to drive, so my husband took me to my appointment. There, the hematologist told me that they needed to confirm my exact diagnosis with further testing, but based on my blood panels, I had a form of blood cancer, also known as leukemia.

Although Google had told me that this was a possibility when Id begun researching my symptoms, it had seemed so imaginable. It felt surreal. People were talking around me and about me, but I dont remember much of what they were saying. I was wheeled directly to the hospital across the street and immediately admitted. The first step was to get me into a stable condition.

Leukemia causes your body to produce an abundance of white blood cells, many of which are abnormal. And the white blood cells crowd out your red blood cells and platelets, which deprives your body of oxygen and prevents blood clotting. Because I was dehydrated and dangerously anemic, I received fluids and several units of blood. Afterwards, I felt better than I had in weeks.

The doctor ran a whole host of tests to determine which type of blood cancer I had, how widespread it was, and if I had any chromosomal mutations, as those would inform the proper treatment for me.

About a week later, I was given an official diagnosis of acute myeloid leukemia.

It's also called (AML), and it's a rapidly progressive cancer of the blood and bone marrow that affects white blood cells known as myeloid cells. Within the United States, there are over 20,000 new cases every year, but the majority of those affected are older adults. When I asked how I ended up with this condition, my hematologist explained that my case wasnt genetic but probably just random bad luck.

In most cases of AML, its not clear what exactly causes the DNA damage that in turn leads to the haywire production of abnormal white blood cells. Many people who end up with this type of blood cancer, like me, have no known risk factors (some of which include being a smoker, male, and over the age of 65). Early signs of AML, including fever, body aches, and fatigue, often seem like a case of the flu or bug.

In October 2017, a month after my stomach bug first appeared, I was readmitted to the hospital and began a round of intensive remission induction chemotherapy in order to kill the leukemia cells in my blood and bone marrow.

For my first round, I stayed in the hospital for about a month. I was not allowed to leave the clean floor, where filtered air was continuously pumped into the rooms and visitors were scanned for fever and illness before they were able to be near any patients. Staff and visitors wore face masks and plastic smocks as an additional layer of protection.

The hardest part of chemo was that my daughters were not allowed on my hospital floor.

Children under 12 weren't allowed on the "clean floor," so I wasnt able to see my daughters for almost a month. We used FaceTime a few times, which was simultaneously great and excruciating because I just wanted to reach out and snuggle them but I knew I couldnt.

Every morning, my medical team would check my blood cell counts to determine if Id need a unit of blood or platelet infusion. I couldnt be discharged from the hospital until my white blood cell count had recovered enough from the chemotherapy for it to be safe for me to leave the clean floor.

After the induction round, I was discharged. Another bone marrow biopsy showed that I was in complete remission, meaning that my blast count in my bone marrow was less than 5 percent. But I wasnt totally in the clear just yet.

For my second half of treatment, I had to undergo four more rounds of consolidation chemotherapy to destroy any remaining cancer cells in order to lower my risk of relapse. While these rounds were gentler, I still struggled. Eight weeks in, my bone marrow was so severely damaged from chemotherapy it began to fail, and I had to get a transplant.

The transplant itself only took about an hour. But after that, it was a waiting game. Every day, my blood was tested, and we waited for the new stem cells to start producing new blood cells and immune system cells. I was also monitored carefully for any signs of infection, which is a huge risk after this type of procedure. My blood cell counts steadily rose, and I was discharged from the hospital after 28 days.

By August 2018, nearly a year into my journey, I went back to work and resumed my normal life.

A few months later, in October, I participated in Light the Night, a celebratory walk and fundraiser sponsored by the Leukemia & Lymphoma Society (LLS). At the event, I heard about another LLS fundraiser, The Big Climb Dallas, where participants climb the tallest building in the city: the Bank of America Tower. Its 70 flights of stairs. and I had no idea if I could do that, but Id caught the fundraising bug. I recruited about 30 friends, family members, and coworkers to climb with me, and I made it to the top.

As the leader of one of the top fundraising teams, I was asked to join the planning committee for the Big Climb 2020, which I enthusiastically accepted. This year, I was extremely grateful to be named the Honored Hero of Big Climb Dallas 2020 and to represent survivors and supporters whose lives have been touched by blood cancer.

It was a long process to get diagnosed and treated, but two and a half years later, Im in full remission.

Funny enough, Im ultimately thankful that my daughters got sick at daycare (as strange as that might sound). If they hadnt, I may have gone several more months before getting a diagnosis and treatment. Today, Im happy to share my story in the hopes that it might help even one other person.

Read more from the original source:
My Flu Symptoms Turned Out To Be Acute Myeloid Leukemia - Women's Health

Bone Marrow Stem Cells Comments Off on My Flu Symptoms Turned Out To Be Acute Myeloid Leukemia – Women’s Health | March 12th, 2020

VANCOUVER, Washington, March 12, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn or the Company), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced today that the FDA recommended that the Company request a preliminary Breakthrough Therapy designation meeting. Meanwhile, the Company continues reporting very positive data for its mTNBC and MBC patients.

Metastatic triple-negative breast cancer (mTNBC), an aggressive histological subtype, has a poor prognosis. In addition, metastatic breast cancer (MBC) is breast cancer that has spread beyond the breast and lymph nodes to other organs in the body (typically the bones, liver, lungs, or brain). Both types of cancer pose significant challenges for patients due to their aggressiveness and limited treatment options. An integral part of CytoDyns mission and purpose is to provide effective therapeutic solutions to these patients. Clinical results from the first cancer patient in the Companys Phase 1b/2 mTNBC trial are as follows:

Patient #1: Enrolled in mTNBC Phase 1b/2 with first treatment in late September 2019. CTC (circulating tumor cells) dropped to zero after two treatments with leronlimab and carboplatin. Total CTC and EMT (Epithelial Mesenchymal Transition in Tumor Metastasis) dropped to zero after about one month of treatment with leronlimab (once-a-week 350 mg dose). Results from the patients earlier CT scan indicated a more than 25% tumor shrinkage within the first few weeks of treatment with leronlimab and carboplatin. After approximately five months of treatment with leronlimab and carboplatin, the patient not only has zero CTC and zero EMT, but also zero detectible CAML (cancer-associated microphages like cells). The patients oncologist has now ordered this patients treatment to consist only of leronlimab and has discontinued treatment with carboplatin (a chemotherapy drug). Testimony provided to the Company from the patient stated: So far my experience with leronlimab has been very positive. I didnt expect it to be so easy and tolerable with virtually ZERO side effects. The results so far have been super impressive. Im very grateful to be part of this clinical trial study and it really makes me feel hopeful that this otherwise fatal disease can be turned into a manageable disease in the near future.

Bruce Patterson, M.D., chief executive officer and founder of IncellDx, a diagnostic partner and advisor to CytoDyn, commented, The FDA recommendation for a meeting on CytoDyns BTD application is a tremendous opportunity to further discuss the mechanism of action and to summarize the promising results from patients enrolled following the submission of the application. Included in this discussion will be the recent decision by the oncologist of Patient #1 to, based on continued unremarkable changes to her condition, remove carboplatin from the patients regimen with continued therapy with leronlimab. Nader Pourhassan, Ph.D., president and chief executive officer of CytoDyn, added: Our first patient in the Phase 1b/2 trial has shown remission of the tumor and her oncologist has attributed this primarily to leronlimab and discontinued the carboplatin (a form of chemotherapy). This patients latest results of zero CTC, EMT, and CAML is unique and we now have another patient with three zeros identical to the first patient. We are very excited to continue enrolling patients and hopeful to have our first patient treated in our basket trial for 22 solid tumor cancers very soon. We are also very hopeful to have several more patients in our Phase 1b/2 mTNBC trial before our preliminary meeting with the FDA for Breakthrough Therapy designation.

About Triple-Negative Breast CancerTriple-negative breast cancer (TNBC) is a type of breast cancer characterized by the absence of the three most common types of receptors in the cancer tumor known to fuel most breast cancer growthestrogen receptors (ER), progesterone receptors (PR) and the hormone epidermal growth factor receptor 2 (HER-2) gene. TNBC cancer occurs in about 10 to 20 percent of diagnosed breast cancers and can be more aggressive and more likely to spread and recur. Since the triple-negative tumor cells lack these receptors, common treatments for breast cancer such as hormone therapy and drugs that target estrogen, progesterone, and HER-2 are ineffective.

About Leronlimab (PRO 140) The U.S. Food and Drug Administration (FDA) have granted a Fast Track designation to CytoDyn for two potential indications of leronlimab for deadly diseases. The first as a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer. Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases including NASH. Leronlimab has successfully completed nine clinical trials in over 800 people, including meeting its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab can significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

In the setting of cancer, research has shown that CCR5 plays an important role in tumor invasion and metastasis. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is therefore conducting a Phase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019. Additional research is being conducted with leronlimab in the setting of cancer and NASH with plans to conduct additional clinical studies when appropriate.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation and may be important in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to further support the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD and that blocking this receptor from recognizing certain immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted orphan drug designation to leronlimab for the prevention of GvHD.

About CytoDyn CytoDyn is a biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a key role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and in immune-mediated illnesses, such as GvHD and NASH. CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard anti-retroviral therapies in HIV-infected treatment-experienced patients. CytoDyn plans to seek FDA approval for leronlimab in combination therapy and plans to complete the filing of a Biologics License Application (BLA) in the first quarter of 2020 for that indication. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients and plans to initiate a registration-directed study of leronlimab monotherapy indication, which if successful, could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs). Moreover, results from a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients, with some patients on leronlimab monotherapy remaining virally suppressed for more than five years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is at http://www.cytodyn.com.

Forward-Looking Statements This press release contains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as believes, hopes, intends, estimates, expects, projects, plans, anticipates and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. The Companys forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i) the sufficiency of the Companys cash position, (ii) the Companys ability to raise additional capital to fund its operations, (iii) the Companys ability to meet its debt obligations, if any, (iv) the Companys ability to enter into partnership or licensing arrangements with third parties, (v) the Companys ability to identify patients to enroll in its clinical trials in a timely fashion, (vi) the Companys ability to achieve approval of a marketable product, (vii) the design, implementation and conduct of the Companys clinical trials, (viii) the results of the Companys clinical trials, including the possibility of unfavorable clinical trial results, (ix) the market for, and marketability of, any product that is approved, (x) the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companys products, (xi) regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii) general economic and business conditions, (xiii) changes in foreign, political, and social conditions, and (xiv) various other matters, many of which are beyond the Companys control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form 10-K, and any risk factors or cautionary statements included in any subsequent Form 10-Q or Form 8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CYTODYN CONTACTS

Investors: Dave Gentry, CEO RedChip Companies Office: 1.800.RED.CHIP (733.2447) Cell: 407.491.4498 dave@redchip.com

Excerpt from:
CytoDyn's First mTNBC Patient in Phase 1b/2 is in Remission and Oncologist Ordered Termination of Treatment with Carboplatin (chemotherapy drug) and...

Bone Marrow Stem Cells Comments Off on CytoDyn’s First mTNBC Patient in Phase 1b/2 is in Remission and Oncologist Ordered Termination of Treatment with Carboplatin (chemotherapy drug) and… | March 12th, 2020

Edward Chang can't read your thoughts. Whenever the neuroscientist's lab at the University of California publishes a new piece of research, there's always a familiar refrain: that he's created "mind-reading technology" or can "read your thoughts". He's not alone, it's a phrase that follows much of the research into brain-computer interfaces and speech decoding.

And no wonder, when Elon Musk's startup Neuralink claims it will eventually enable "consensual telepathy" and Facebook one of the funders of Chang's lab said it wants to let people send messages by just thinking the words, rather than tapping them out on a phone, an example of a brain-computer interface (BCI).

But Chang isn't trying to read minds; he's decoding speech in people who otherwise can't speak. "We're not really talking about reading someone's thoughts," Chang says. "Every paper or project we've done has been focusing on understanding the basic science of how the brain controls our ability to speak and understand speech. But not what we're thinking, not inner thoughts." Such research would have significant ethical implications, but it's not really possible right now anyway and may never be.

Even decoding speech isn't easy. His most recent paper, in Nature last year, aimed to translate brain signals produced by speech into words and sentences read aloud by a machine; the aim is to help people with diseases such as amyotrophic lateral sclerosis (ALS) a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. "The paper describes the ability to take brain activity in people who are speaking normally and use that to create speech synthesis it's not reading someone's thoughts," he says. "It's just reading the signals that are speaking."

The technology worked to an extent. Patients with electrodes embedded in their brains were read a question and spoke an answer. Chang's system could accurately decipher what they heard 76 per cent of the time and what they said 61 per cent of the time by looking at their motor cortex to see how the brain fired up to move their mouth and tongue. But there are caveats. The potential answers were limited to a selection, making the algorithm's job a bit easier. Plus, the patients were in hospital having brain scans for epilepsy, and could therefore speak normally; it's not clear how this translates to someone who can't speak at all.

"Our goal is to translate this technology to people who are paralysed," he says. "The big challenge is understanding somebody who's not speaking. How do you train an algorithm to do that?" It's one thing to train a model using someone you can ask to read out sentences; you scan their brain signals while they read out sentences. But how do you do that if someone can't speak?

Chang's lab is currently in the middle of a clinical trial attempting to address that "formidable challenge", but it's as yet unclear how speech signals change for those unable to speak, or if different areas of the brain need to be considered. "There are these fairly substantial issues that we have to address in terms of our scientific knowledge," he says.

Decoding such signals is challenging in part because of how little we understand about how our own brains work. And while systems can be more easily trained to move a cursor left or right, speech is complicated. "The main challenges are the huge vocabulary that characterise this task, the need of a very good signal quality achieved only by very invasive technologies and the lack of understanding on how speech is encoded in the brain," says David Valeriani of Harvard Medical School. "This latter aspect is a challenge across many BCI fields. We need to know how the brain works before being able to use it to control other technologies, such as a BCI."

And we simply don't have enough data, says Mariska van Steensel, assistant professor at UMC Utrecht. It's difficult to install brain implants, so it's not frequently done; Chang used epilepsy patients because they were already having implants to track their seizures. Sitting around waiting for a seizure to strike, a handful were willing to take part in his research out of boredom. "On these types of topics, the number of patients that are going to be implanted will stay low, because it is very difficult research and very time consuming," she says, noting that fewer than 30 people have been implanted with a BCI worldwide; her own work is based on two implants. "That is one of the reasons why progress is relatively slow," she added, suggesting a database of work could be brought together to help share information.

There's another reason this is difficult: our brains don't all respond the same. Van Steensel has two patients with implants, allowing them to make a mouse click with brain signals by thinking about moving their hands. In the first patient, with ALS, it worked perfectly. But it didn't in the second, a patient with a brain-stem stroke. "Her signals were different and less optimal for this to b e reliable," she says. "Even a single mouse click to get reliable in all situations is already difficult."

This work is different than that of startups such as NextMind and CTRL-Labs that use external, non-invasive headsets to read brain signals, but they lack the precision of an implant. "If you stay outside a concert hall, you will hear a very distorted version of what's playing inside this is one of the problems of non-invasive BCIs," says Ana Matran-Fernandez, artificial intelligence industry fellow at the University of Essex. "You will get an idea of the general tempo... of the piece that's being played, but you can't pinpoint specifically each of the instruments being played. This is the same with a BCI. At best, we will know which areas of the brain are the most active playing louder, if you will but we won't know why, and we don't necessarily know what that means for a specific person."

Still, tech industry efforts including Neuralink and Facebook aren't misplaced, says Chang, but they're addressing different problems. Those projects are looking at implant or headset technology, not the hard science that's required to make so-called mind reading possible. "I think it's important to have all of these things happening," he says. "My caveat is that's not the only part of making these things work. There's still fundamental knowledge of the brain that we need to have before any of this will work."

Until then, we won't be able to read speech, let alone inner thoughts. "Even if we were perfectly able to distinguish words someone tries to say from brain signals, this is not even close to mind reading or thought reading," van Steensel says. "We're only looking at the areas that are relevant for the motor aspects of speech production. We're not looking at thoughts I don't even think that's possible."

Edward Chang will be one of the speakers at WIRED Health in London on March 25, 2020. For more details, and to book your ticket, click here

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Excerpt from:
Why computers won't be reading your mind any time soon - Wired.co.uk

Spinal Cord Stem Cells Comments Off on Why computers won’t be reading your mind any time soon – Wired.co.uk | March 12th, 2020

First, by their nature, pluripotent stem cells can potentially be used to create any cell or tissue the body might need to counter a wide range of diseases, from diabetes to spinal cord injury, to childhood leukemia, to heart disease.

Second, pluripotent stem cells can potentially be customized to provide a perfect genetic match for any patient. This means that patients could receive transplants of tissue and cells without tissue matching and tissue rejection problems, and without the need to take powerful immune-suppressing drugs for the rest of their lives. Although this vision hasnt yet been achieved, researchers at Boston Childrens Hospital have successfully treated mouse models of human disease using this strategy and hope that the same can be done with patients.

Disease in a dish:Third, pluripotent stem cells make excellent laboratory models for studying how a disease unfolds, which helps scientists pinpoint and track the very earliest disease-causing events in cells. Immune deficiencies, Type 1 diabetes, muscular dystrophy, and myriad other disorders are rooted in fetal development. In the lab, researchers can recapture these early originsobserving where the first muscle cell comes from, or the first blood cell, and how this differs when the patient has a genetic disease. Using this information, doctors may be able to intervene and correct the genetic defect before the disease advances.

Unique applications:Each type of pluripotent stem cell has different characteristics that make it useful in different ways, and each has different lessons to teach.

Read this article:
Why are Pluripotent Stem Cells Important? Boston ...

IPS Cell Therapy Comments Off on Why are Pluripotent Stem Cells Important? Boston … | March 12th, 2020

Shinya Yamanaka's 2006 discovery of induced pluripotent stem cells (iPSCs) ignited a revolution in the field of stem cell biology (1). For the first time, nearly all human somatic tissues could be produced from iPSCs reprogrammed from blood or skin cells, in a process that took only weeks. This advance was particularly crucial for obtaining surrogate tissues from cell types that are otherwise difficult to procure and do not readily expand in vitro, such as cardiac or neural cells. Additionally, many ethical concerns are avoided, because this technology uses a patient's own genetic material to create iPSCs rather than relying on embryonic stem cells. In the aftermath of Yamanaka's discovery, entire biomedical industries have developed around the promise of using human iPSCs (hiPSCs) and their derivatives for in vitro disease modeling, drug screening, and cell therapy (2).

The hiPSC technology has had a particularly notable impact in cardiac regenerative medicine, a field where scientists and clinicians have been working to devise new methods to better understand how cardiovascular disease manifests and how to restore cardiovascular function after disease strikes (3). The heart is limited in its ability to regenerate lost cardiomyocytes (beating heart muscle cells), following an adverse event such as a heart attack (4). Cardiomyocytes derived from hiPSCs (hiPSC-CMs) may represent a potential replacement option for dead cells in such a scenario. However, certain issues remain to be addressed, such as whether hiPSC-CMs can integrate with host myocardial tissue in the long term (5).

While using hiPSC-CMs for in vivo cell therapy may become practical in the future, employing hiPSC-CMs for high-throughput drug discovery and screening is becoming a reality in the present (6). Cardiovascular diseases can be recapitulated in a dish with patient-specific hiPSC-CMs. For example, if a patient exhibits a cardiac arrhythmia caused by a genetic abnormality in a sarcomeric protein or ion channel, that same rhythm problem can be recapitulated in vitro (7). Thanks to advances in hiPSC differentiation protocols, hiPSC-CMs can now be mass-produced to study cardiovascular disease mechanisms in vitro (8).

My graduate thesis in the laboratories of Joseph Wu and Sean Wu at Stanford University focused on in vitro applications of hiPSC-CMs for cardiovascular disease modeling and for high-throughput screening of chemotherapeutic compounds to predict cardiotoxicity. I initially embarked on a project using hiPSC-CMs to model viral myocarditis, a viral infection of the heart, caused by the B3 strain of coxsackievirus (9). I began by demonstrating that hiPSC-CMs express the receptors necessary for viral internalization and subsequently found that hiPSC-CMs were highly susceptible to coxsackievirus infection, exhibiting viral cytopathic effect within hours of infection. I also identified compounds that could alleviate coxsackievirus infection on hiPSC-CMs, a translationally relevant finding, as there remains a shortage of treatments for viral myocarditis.

Using a genetically modified variant of coxsackievirus B3 expressing luciferase, I developed a screening platform for assessing the efficacy of antiviral compounds. Pretreatment with interferon-, ribavirin, or pyrrolidine dithiocarbamate markedly suppressed viral replication on hiPSC-CMs by activating intracellular antiviral response and viral protein clearance pathways. These compounds alleviated viral replication in a dose-dependent fashion at low concentrations without causing cellular toxicity.

I next sought to use hiPSC-CMs to screen anticancer chemotherapeutic compounds for their off-target cardiovascular toxicities (10). Cardiotoxicity represents a major cause of drug withdrawal from the pharmaceutical market, and several chemotherapeutic agents can cause unintended cardiovascular damage (11). Using cultured hiPSC-CMs, I evaluated 21 U.S. Food and Drug Administrationapproved tyrosine kinase inhibitors (TKIs), commonly prescribed anticancer compounds, for their cardiotoxic potential. HiPSC-CMs express the major tyrosine kinase receptor proteins such as the insulin, insulin-like growth factor (IGF), vascular endothelial growth factor (VEGF), and platelet-derived growth factor (PDGF) receptors, lending validity to this cellular model.

Initially, human induced pluripotent stem cells (hiPSCs) can be produced by reprogramming skin or blood cells by nonviral or viral reprogramming methods. Cardiac differentiation protocols allow for the creation of cardiomyocytes derived from hiPSCs (hiPSC-CMs) for downstream applications, including in vitro disease modeling, drug screening, and regenerative cell therapy.

With data from a battery of cellular apoptosis, contractility, electrophysiology, and signaling assays, I generated a cardiac safety index to help align in vitro toxicity data to clinical drug safety guidelines (12). From the safety index, I determined that a subclass of VEGF receptor 2/PDGF receptorinhibiting tyrosine kinase inhibitors, some of which exhibit toxicity clinically, also elicited cardiotoxicities in hiPSC-CMs. These manifested as substantial alterations in cellular electrophysiology, contractility, and viability when administered at clinically relevant concentrations. I also discovered that cotreatment with either IGF or insulin partially rescued TKI-induced toxicity by up-regulating antiapoptotic signaling pathways. This work could prove useful for groups aiming to develop effective screening platforms to assess new chemotherapeutic compounds for cardiotoxic side effects.

I also collaborated with the Center for the Advancement of Science in Space (CASIS) to send a sample of hiPSC-CMs to the International Space Station. As humankind ventures beyond our home planet, it is imperative that we better understand how the heart functions for long periods of time in microgravity. Analysis of these hiPSC-CMs revealed microgravity-induced alterations in metabolic gene expression and calcium handling (13).

In recent years, the stem cell field has experienced an explosion of studies using hiPSC-CMs as a model cellular system to study cardiovascular biology. As improvements in hiPSC-CM mass production continue, we will see a rise in studies using these cells for disease modeling and drug screening. Thus, although hiPSC-CM technology is in its infancy, it holds great potential to improve cardiovascular health.

PHOTO: COURTESY OF A. SHARMA

FINALIST

Arun Sharma

Arun Sharma received his undergraduate degree from Duke University and a Ph.D. from Stanford University. Having completed a postdoctoral fellowship at the Harvard Medical School, Sharma is now a senior research fellow jointly appointed at the Smidt Heart Institute and Board of Governors Regenerative Medicine Institute at the Cedars-Sinai Medical Center in Los Angeles. His research seeks to develop in vitro platforms for cardiovascular disease modeling and drug cardiotoxicity assessment. http://www.sciencemag.org/content/367/6483/1206.1

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Stem cells to help the heart - Science Magazine

Skin Stem Cells Comments Off on Stem cells to help the heart – Science Magazine | March 12th, 2020

DUBLIN, March 12, 2020 /PRNewswire/ -- The "Cell Therapy - Technologies, Markets and Companies" report from Jain PharmaBiotech has been added to ResearchAndMarkets.com's offering.

The cell-based markets was analyzed for 2018, and projected to 2028. The markets are analyzed according to therapeutic categories, technologies and geographical areas. The largest expansion will be in diseases of the central nervous system, cancer and cardiovascular disorders. Skin and soft tissue repair as well as diabetes mellitus will be other major markets.

The number of companies involved in cell therapy has increased remarkably during the past few years. More than 500 companies have been identified to be involved in cell therapy and 309 of these are profiled in part II of the report along with tabulation of 302 alliances. Of these companies, 170 are involved in stem cells.

Profiles of 72 academic institutions in the US involved in cell therapy are also included in part II along with their commercial collaborations. The text is supplemented with 67 Tables and 25 Figures. The bibliography contains 1,200 selected references, which are cited in the text.

This report contains information on the following:

The report describes and evaluates cell therapy technologies and methods, which have already started to play an important role in the practice of medicine. Hematopoietic stem cell transplantation is replacing the old fashioned bone marrow transplants. Role of cells in drug discovery is also described. Cell therapy is bound to become a part of medical practice.

Stem cells are discussed in detail in one chapter. Some light is thrown on the current controversy of embryonic sources of stem cells and comparison with adult sources. Other sources of stem cells such as the placenta, cord blood and fat removed by liposuction are also discussed. Stem cells can also be genetically modified prior to transplantation.

Cell therapy technologies overlap with those of gene therapy, cancer vaccines, drug delivery, tissue engineering and regenerative medicine. Pharmaceutical applications of stem cells including those in drug discovery are also described. Various types of cells used, methods of preparation and culture, encapsulation and genetic engineering of cells are discussed. Sources of cells, both human and animal (xenotransplantation) are discussed. Methods of delivery of cell therapy range from injections to surgical implantation using special devices.

Cell therapy has applications in a large number of disorders. The most important are diseases of the nervous system and cancer which are the topics for separate chapters. Other applications include cardiac disorders (myocardial infarction and heart failure), diabetes mellitus, diseases of bones and joints, genetic disorders, and wounds of the skin and soft tissues.

Regulatory and ethical issues involving cell therapy are important and are discussed. Current political debate on the use of stem cells from embryonic sources (hESCs) is also presented. Safety is an essential consideration of any new therapy and regulations for cell therapy are those for biological preparations.

Key Topics Covered

Part I: Technologies, Ethics & RegulationsExecutive Summary 1. Introduction to Cell Therapy2. Cell Therapy Technologies3. Stem Cells4. Clinical Applications of Cell Therapy5. Cell Therapy for Cardiovascular Disorders6. Cell Therapy for Cancer7. Cell Therapy for Neurological Disorders8. Ethical, Legal and Political Aspects of Cell therapy9. Safety and Regulatory Aspects of Cell Therapy

Part II: Markets, Companies & Academic Institutions10. Markets and Future Prospects for Cell Therapy11. Companies Involved in Cell Therapy12. Academic Institutions13. References

For more information about this report visit https://www.researchandmarkets.com/r/sy4g72

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Worldwide Cell Therapy Market Projections to 2028 - The Largest Expansion Will Be in Diseases of the Central Nervous System, Cancer and Cardiovascular...

Skin Stem Cells Comments Off on Worldwide Cell Therapy Market Projections to 2028 – The Largest Expansion Will Be in Diseases of the Central Nervous System, Cancer and Cardiovascular… | March 12th, 2020

(CNN) -

There are only two northern white rhinos left on the planet, and they're both female. Unless scientists can make a dramatic breakthrough, the entire species will die with those two individuals.

In a nondescript building just north of San Diego, California, the fight to save the northern white rhino is coming down to the wire. However, the battleground here looks less like a scene from a wildlife documentary and more akin to something out of a science fiction novel.

At the San Diego Zoo Institute for Conservation Research, an army of scientists armed with liquid nitrogen, microscopes, and ultrasound machines is working around the clock to create an unprecedented first in the conservation world: they are looking to turn frozen rhino skin cells into baby rhinos.

It's not just the science that is groundbreaking, but also the team looking to save this species. Composed mostly of women, the lab is a rarity in a field traditionally dominated by men.

Find out more about Call to Earth and the extraordinary people working for a more sustainable future

The first step in this conservation effort began more than four and a half decades ago in 1975 when scientists established the institute's "Frozen Zoo." In a small room measuring no more than 36 square meters the skin cells of more than 10,000 individuals across 1,100 species sit in giant steel tanks suspended in time, frozen in liquid nitrogen.

Among the collection are the skin samples of 12 northern white rhinos. These are vital to the group's efforts because there is such a small gene pool of living northern whites.

The population has been decimated by poachers, who target rhinos because of the belief in parts of Asia that their horns can cure various ailments. The two surviving females both live under guard at the Ol Pejeta Conservancy in Kenya. Even thoughembryos have been producedin an Italian lab using eggs extracted from the pair, any future descendants from this kind of embryo would carry the genes of those two females.

That may not be enough genetic diversity to maintain a stable population. The hope is that the skin samples of those 12 individuals at the Frozen Zoo contain enough diversity to sustain the northern white species long-term.

The arduous task for these scientists is to create a rhino population from those samples.

Marlys Houck is curator of the Frozen Zoo. She graduated high school in 1979, the same year the Frozen Zoo froze its very first northern white rhino skin cell. She later joined the institute to work on the rhino project.

"I was hired specifically to try to make the cells of the rhinos grow better because they were one of the most difficult to grow cell lines," she told CNN.

Since then, she's figured out how to successfully grow and freeze the skin cells of the northern white.

The impact of this work is not lost on her. "We're losing species so rapidly," she said. "One of the things we can do is save the living cells of these animals before it's too late."

"We're at the forefront of science today," she added. "If we do everything right ... these cells should be here 50 years from now being used for purposes that we can't even imagine today."

Marisa Korody is one of the four scientists tasked with turning these frozen cells into new life. They have to reprogram the frozen skin cells into pluripotent stem cells. In layman's terms, Korody explains that "stem cells can become any cell type in the body if they're given the right signals."

Read: Former war zones turn into wildlife 'paradise'

The aim is to ultimately turn the stem cells into sperm and eggs. The ambitious feat has only been achieved in animals by Japanese scientists. While Korody and her team have looked to that research as a road map, she admits that doing the same with rhinos is uncharted territory. "We don't really know what twists and turns we need to take in order to get from A to B," she said.

"They haven't even figured out how to do this in humans," she added. "We have as much information as we possibly can about humans. We have a fraction of that for rhinos."

Korody says being at the forefront of this kind of science has been a dream job. "This was really the first project that's trying to apply this type of science to conservation as a whole," she said.

She may spend most of her time at work looking through the lens of a microscope, but her mind is always on the final goal for the rhinos: "We want to be able to put them back into the wild one day and have them living free."

Because the remaining two female northern white rhinos can't carry a pregnancy, even if the team can create embryos, the last obstacle is finding rhinos who can carry them to term.

The woman tasked with that job is Barbara Durrant. As the director of reproductive sciences, she's spent four years studying the reproductive systems of six female southern white rhinos at the institute's sister facility, the Nikita Kahn Rhino Rescue Center.

Though the rhinos at the center are a different species, Durrant says they are the closest relative to the northern white. The aim is to eventually have them be surrogates for northern white embryos.

On any given day, Durrant can be found conducting ultrasounds to help her understand each rhino's distinct reproductive cycle. In 2019, two of the center's females gave birth to southern white babies. Both were conceived via artificial insemination, giving Durrant and the teams working on the rhino project hope for the future.

Durrant believes one reason the project works so well is because there are so many women involved. "Women are naturally collaborative with each other," she said. "Because we have so many obstacles along the way and challenges and setbacks, we support each other and we have sympathy for each other."

Read: Rare bird brought back from extinction in the wild

Houck says women tend to be naturally nurturing. "The cells are living little organisms that we're growing and tending almost every day, and I think women are drawn to taking care of something and growing it into something more."

"It's wonderful leading a team of women, and I really think they're changing the world," she added. "People are going to look back and see it was this amazing group of women who quietly, unrecognized, work at this and just get better and better."

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Meet the women racing to save the northern white rhino from extinction - KAKE

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Mar 12th 2020

BEING ABLE to see all the details of the genome at once necessarily makes medicine personal. It can also make it precise. Examining illness molecule by molecule allows pharmaceutical researchers to understand the pathways through which cells act according to the dictates of genes and environment, thus seeing deep into the mechanisms by which diseases cause harm, and finding new workings to target. The flip side of this deeper understanding is that precision brings complexity. This is seen most clearly in cancer. Once, cancers were identified by cell and tissue type. Now they are increasingly distinguished by their specific genotype that reveals which of the panoply of genes that can make a cell cancerous have gone wrong in this one. As drugs targeted against those different mutations have multiplied, so have the options for oncologists to combine them to fit their patients needs.

Cancer treatment has been the most obvious beneficiary of the genomic revolution but other diseases, including many in neurology, are set to benefit, too. Some scientists now think there are five different types of diabetes rather than two. There is an active debate about whether Parkinsons is one disease that varies a lot, or four. Understanding this molecular variation is vital when developing treatments. A drug that works well on one subtype of a disease might fail in a trial that includes patients with another subtype against which it does not work at all.

Thus how a doctor treats a disease depends increasingly on which version of the disease the patient has. The Personalised Medicine Coalition, a non-profit advocacy group, examines new drugs approved in America to see whether they require such insights in order to be used. In 2014, it found that so-called personalised medicines made up 21% of the drugs newly approved for use by Americas Food and Drug Administration (FDA). In 2018 the proportion was twice that.

Two of those cited were particularly interesting: Vitrakvi (larotrectinib), developed by Loxo Oncology, a biotech firm, and Onpattro (patisiran), developed by Alnylam Pharmaceuticals. Vitrakvi is the first to be approved from the start as tumour agnostic: it can be used against any cancer that displays the mutant protein it targets. Onpattro, which is used to treat peripheral-nerve damage, is the first of a new class of drugssmall interfering RNAs, or siRNAsto be approved. Like antisense oligonucleotides (ASOs), siRNAs are little stretches of nucleic acid that stop proteins from being made, though they use a different mechanism.

Again like ASOs, siRNAs allow you to target aspects of a disease that are beyond the reach of customary drugs. Until recently, drugs were either small molecules made with industrial chemistry or bigger ones made with biologynormally with genetically engineered cells. If they had any high level of specificity, it was against the actions of a particular protein, or class of proteins. Like other new techniques, including gene therapies and anti-sense drugs, siRNAs allow the problem to be tackled further upstream, before there is any protein to cause a problem.

Take the drugs that target the liver enzyme PCSK9. This has a role in maintaining levels of bad cholesterol in the blood; it is the protein that was discovered through studies of families in which congenitally high cholesterol levels led to lots of heart attacks. The first generation of such drugs were antibodies that stuck to the enzyme and stopped it working. However, the Medicines Company, a biotech firm recently acquired by Novartis, won approval last year for an siRNA called inclisiran that interferes with the expression of the gene PCSK9thus stopping the pesky protein from being made in the first place. Inclisiran needs to be injected only twice a year, rather than once a month, as antibodies do.

New biological insights, new ways of analysing patients and their disease and new forms of drug are thus opening up a wide range of therapeutic possibilities. Unfortunately, that does not equate to a range of new profitable opportunities.

Thanks in part to ever better diagnosis, there are now 7,000 conditions recognised as rare diseases in America, meaning that the number of potential patients is less than 200,000. More than 90% of these diseases have no approved treatment. These are the diseases that personalised, precision medicine most often goes after. Nearly 60% of the personalised medicines approved by the FDA in 2018 were for rare diseases.

Zolgensma is the most expensive drug ever brought to market.

That might be fine, were the number of diseases stable. But precision in diagnosis is increasingly turning what used to be single diseases into sets of similar-looking ones brought about by distinctly different mechanisms, and thus needing different treatment. And new diseases are still being discovered. Medical progress could, in short, produce more new diseases than new drugs, increasing unmet need.

Some of it will, eventually, be met. For one thing, there are government incentives in America and Europe for the development of drugs for rare diseases. And, especially in America, drugs for rare diseases have long been able to command premium prices. Were this not the case, Novartis would not have paid $8.7bn last year to buy AveXis, a small biotech firm, thereby acquiring Zolgensma, a gene therapy for spinal muscular atrophy (SMA). Most people with SMA lack a working copy of a gene, SMN1, which the nerve cells that control the bodys muscles need to survive. Zolgensma uses an empty virus-like particle that recognises nerve cells to deliver working copies of the gene to where it is needed. Priced at $2.1m per patient, it is the most expensive drug ever brought to market. That dubious accolade might not last long. BioMarin, another biotech firm, is considering charging as much as $3m for a forthcoming gene therapy for haemophilia.

Drug firms say such treatments are economically worthwhile over the lifetime of the patient. Four-fifths of children with the worst form of SMA die before they are four. If, as is hoped, Zolgensma is a lasting cure, then its high cost should be set against a half-century or more of life. About 200 patients had been treated in America by the end of 2019.

But if some treatments for rare diseases may turn a profit, not all will. There are some 6,000 children with SMA in America. There are fewer than ten with Jansens disease. When Dr Nizar asked companies to help develop a treatment for it, she says she was told your disease is not impactful. She wrote down the negative responses to motivate herself: Every day I need to remind myself that this is bullshit.

A world in which markets shrink, drug development gets costlier and new unmet needs are ceaselessly discovered is a long way from the utopian future envisaged by the governments and charities that paid for the sequencing of all those genomes and the establishment of the worlds biobanks. As Peter Bach, director of the Centre for Health Policy and Outcomes, an academic centre in New York, puts it with a degree of understatement: if the world needs to spend as much to develop a drug for 2,000 people as it used to spend developing one for 100,000, the population-level returns from medical research are sharply diminishing.

And it is not as if the costs of drug development have been constant. They have gone up. What Jack Scannell, a consultant and former pharmaceutical analyst at UBS, a bank, has dubbed Erooms lawEroom being Moore, backwardsshows the number of drugs developed for a given amount of R&D spending has fallen inexorably, even as the amount of biological research skyrocketed. Each generation assumes that advances in science will make drugs easier to discover; each generation duly advances science; each generation learns it was wrong.

For evidence, look at the way the arrival of genomics in the 1990s lowered productivity in drug discovery. A paper in Nature Reviews Drug Discovery by Sarah Duggers from Columbia University and colleagues argues that it brought a wealth of new leads that were difficult to prioritise. Spending rose to accommodate this boom; attrition rates for drugs in development subsequently rose because the candidates were not, in general, all that good.

Today, enthused by their big-science experience with the genome and enabled by new tools, biomedical researchers are working on exhaustive studies of all sorts of other omes, including proteomesall the proteins in a cell or body; microbiomesthe non-pathogenic bacteria living in the mouth, gut, skin and such; metabolomessnapshots of all the small molecules being built up and broken down in the body; and connectomes, which list all the links in a nervous system. The patterns they find will doubtless produce new discoveries. But they will not necessarily, in the short term, produce the sort of clear mechanistic understanding which helps create great new drugs. As Dr Scannell puts it: We have treated the diseases with good experimental models. Whats left are diseases where experiments dont replicate people. Data alone canot solve the problem.

Daphne Koller, boss of Insitro, a biotech company based in San Francisco, shares Dr Scannells scepticism about the way drug discovery has been done. A lot of candidate drugs fail, she says, because they aim for targets that are not actually relevant to the biology of the condition involved. Instead researchers make decisions based on accepted rules of thumb, gut instincts or a ridiculous mouse model that has nothing to do with what is actually going on in the relevant human diseaseeven if it makes a mouse look poorly in a similar sort of way.

But she also thinks that is changing. Among the things precision biology has improved over the past five to 10 years have been the scientists own tools. Gene-editing technologies allow genes to be changed in various ways, including letter by letter; single-cell analysis allows the results to be looked at as they unfold. These edited cells may be much more predictive of the effects of drugs than previous surrogates. Organoidsself-organised, three-dimensional tissue cultures grown from human stem cellsoffer simplified but replicable versions of the brain, pancreas, lung and other parts of the body in which to model diseases and their cures.

Insitro is editing changes into stem cellswhich can grow into any other tissueand tracking the tissues they grow into. By measuring differences in the development of very well characterised cells which differ in precisely known ways the company hopes to build more accurate models of disease in living cells. All this work is automated, and carried out on such a large scale that Dr Koller anticipates collecting many petabytes of data before using machine learning to make sense of it. She hopes to create what Dr Scannell complains biology lacks and what drug designers need: predictive models of how genetic changes drive functional changes.

There are also reasons to hope that the new upstream drugsASOs, siRNAs, perhaps even some gene therapiesmight have advantages over todays therapies when it comes to small-batch manufacture. It may also prove possible to streamline much of the testing that such drugs go through. Virus-based gene-therapy vectors and antisense drugs are basically platforms from which to deliver little bits of sequence data. Within some constraints, a platform already approved for carrying one message might be fast-tracked through various safety tests when it carries another.

One more reason for optimism is that drugs developed around a known molecule that marks out a diseasea molecular markerappear to be more successful in trials. The approval process for cancer therapies aimed at the markers of specific mutations is often much shorter now than it used to be. Tagrisso (osimertinib), an incredibly specialised drug, targets a mutation known to occur only in patients already treated for lung cancer with an older drug. Being able to specify the patients who stand to benefit with this degree of accuracy allows trials to be smaller and quicker. Tagrisso was approved less than two years and nine months after the first dose was given to a patient.

With efforts to improve the validity of models of disease and validate drug targets accurately gaining ground, Dr Scannell says he is sympathetic to the proposal that, this time, scientific innovation might improve productivity. Recent years have seen hints that Erooms law is being bent, if not yet broken.

If pharmaceutical companies do not make good on the promise of these new approaches then charities are likely to step in, as they have with various ASO treatments for inherited diseases. And they will not be shackled to business models that see the purpose of medicine as making drugs. The Gates Foundation and Americas National Institutes of Health are investing $200m towards developing treatments based on rewriting genes that could be used to tackle sickle-cell disease and HIVtreatments that have to meet the proviso of being useful in poor-country clinics. Therapies in which cells are taken out of the body, treated in some way and returned might be the basis of a new sort of business, one based around the ability to make small machines that treat individuals by the bedside rather than factories which produce drugs in bulk.

There is room in all this for individuals with vision; there is also room for luck: Dr Nizar has both. Her problem lies in PTH1R, a hormone receptor; her PTH1R gene makes a form of it which is jammed in the on position. This means her cells are constantly doing what they would normally do only if told to by the relevant hormone. A few years ago she learned that a drug which might turn the mutant receptor off (or at least down a bit) had already been characterisedbut had not seemed worth developing.

The rabbit, it is said, outruns the fox because the fox is merely running for its dinner, while the rabbit is running for its life. Dr Nizars incentives outstrip those of drug companies in a similar way. By working with the FDA, the NIH and Massachusetts General Hospital, Dr Nizar helped get a grant to make enough of the drug for toxicology studies. She will take it herself, in the first human trial, in about a years time. After that, if things go well, her childrens pain may finally be eased.

This article appeared in the Technology Quarterly section of the print edition under the headline "Kill or cure?"

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New drugs are costly and unmet need is growing - The Economist

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Astronauts are growing the beginnings of new organs on board the International Space Station.

The experiment is an attempt to grow human tissue by sending adult human stem cells into space, and allowing them to grow in space.

Eventually, it is hoped, the stem cells will develop into bone, cartilage and other organs. If that is successful, the discoveries could be used to try and grow organs for transplant, the scientists involved say.

The experiment uses weightlessness as a tool, according to Cara Thiel, one of the two researchers from the University of Zurich. The lack of gravity on board the ISS will be used to encourage the stem cells to grow into tissue in three dimensions, rather than the single-layer structures that form on Earth.

It is being conducted by the astronauts on board the ISS using a mobile mini-laboratory that was sent on a SpaceX rocket last week. The experiment will last for a month, during which scientists will watch to see how the stem cells grow.

If it is successful, they hope to switch from a small laboratory to bigger production. From there, they could use the process to generate tissue for transplants by taking cells from patients, or generating organ-like material, either ensuring that it works for a specific patients or reducing the number of animals used in experiments.

On Earth, tissue grows in monolayer cultures: generating flat, 2D tissue. But investigations both in space and Earth suggest that in microgravity, cells exhibit spatially unrestricted growth and assemble into complex 3D aggregates, said Oliver Ullrich, who is also leading the research.

Previous research has involved simulated ad real experiments, mostly using tumour cells, and placing real human stem cells into microgravity simulators. But for the next stage of the research there is no alternative to the ISS, he says, as 3D tissue formation of this kind requires several days or even weeks in microgravity.

After the month-long experiment, the scientists will get the samples back and expect to see successful formation of organoids smaller, more simple versions of organs inside the test tubes.

Scientists are still not sure why the conditions of the ISS lead to the assembly of complex 3D tissue structures. Scientists are still continuing to research how the gravitational force and the molecular machinery in the cell interact to create new and different kinds of tissue on Earth and in space.

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Made-in-space organs could soon be reality - ETHealthworld.com

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MENLO PARK, Calif. and NEW YORK, March 11, 2020 (GLOBE NEWSWIRE) -- Forty Seven Inc. (Nasdaq: FTSV) and Rocket Pharmaceuticals Inc. (Nasdaq: RCKT) announced today that they have entered into a research collaboration to pursue clinical proof-of-concept for Forty Sevens novel antibody-based conditioning regimen, FSI-174 (anti-cKIT antibody) plus magrolimab (anti-CD47 antibody), with Rockets ex vivo lentiviral vector hematopoietic stem cell (LVV HSC) gene therapy, RP-L102. The initial collaboration will evaluate this treatment regimen in Fanconi Anemia (FA), a genetic disease that affects patients capacity to produce blood cells and is associated with an increased risk of leukemia and other neoplasms. RP-L102, Rockets gene therapy approach for FA, involves treatment with patients own gene-corrected blood forming stem cells (hematopoietic stem cells, or HSCs).

Gene therapies for monogenic blood disorders have broad potential. One concern associated with these treatments is the toxicity of pre-therapy conditioning regimens that utilize cytotoxic chemotherapy and/or radiation to destroy existing HSCs and facilitate engraftment of gene-corrected HSCs. Forty Sevens all-antibody based conditioning regimen is designed to address the limitations of current pre-treatment conditioning therapies. These regimens are often associated with serious side effects, including severe infection, cognitive impairment, infertility, endocrine dysfunction, secondary malignancies and organ damage. These toxicities are especially difficult for pediatric patients and are particularly severe for patients with FA, who are more sensitive to the DNA-damaging effects of traditional conditioning agents. Preliminary data demonstrate that RP-L102 may confer efficacy without pre-treatment conditioning. The combination of RP-L102 with Forty Sevens all-antibody conditioning regimen may provide patients an alternate treatment option in situations where conditioning may be advantageous.

We are pleased to enter into this collaboration with Forty Seven, said Jonathan Schwartz, M.D., Chief Medical Officer and Senior Vice President of Rocket. RP-L102 Process B is currently being evaluated in a registrational trial without the use of conditioning. In parallel, we are assessing incorporation of a non-genotoxic conditioning regimen as a part of Rockets life-cycle management strategy. Forty Sevens novelall-antibodyconditioning regimen could also beapplied to Rockets other lentiviral programs, in which conditioning is more integral to the gene therapy approach.

We are initiating our first in human healthy volunteer study of FSI-174 in the first quarter this year, and are excited to enter into a partnership with Rocket at this time. Rocket is at the forefront of developing gene therapies for high unmet-need diseases, and this collaboration will provide an opportunity to evaluate the benefit of Forty Sevens novel conditioning regimen with Rockets RP-L102 to help FA patients, says Jens-Peter Volkmer, VP of Research at Forty Seven.

This collaboration is in line with our strategy to study our anti-cKIT and anti-CD47, all-antibody conditioning regimen in combination with several different gene therapies, and to establish clinical proof-of-concept in a broad range of transplant indications, said Mukul Agarwal, VP of Corporate Development at Forty Seven.

Maria Grazia Roncarolo, M.D., Scientific Advisor to Forty Seven, commented, The goal of my lifes work is to bring pediatric patients transformative therapies for currently incurable diseases. We believe Rocket Pharmaceuticals commitment to devastating diseases, such as FA, addresses a critical unmet need and Forty Sevens antibody conditioning creates an alternative avenue to deliver this therapy to those patients. We look forward to seeing how this collaboration may help patients in need.

Under the terms of the agreement, Rocket will provide its ex vivo LVV HSC gene therapy platform and Forty Seven will contribute its innovative antibody-based conditioning regimen for the collaboration.

About FSI-174 and MagrolimabFSI-174 is a humanized monoclonal antibody targeting cKIT, which is a receptor that is highly expressed on hematopoietic stem cells. Magrolimab is a humanized monoclonal antibody targeting CD47, which is a dont eat me signal to macrophages and is expressed on all cells. Magrolimab is currently being investigated in Phase 2 clinical trials to treat cancer and has established clinical efficacy in four indications, including myelodysplastic syndrome, acute myeloid leukemia, diffuse large B cell lymphoma and follicular lymphoma, with a favorable safety profile in over 400 patients treated, including some patients treated continuously for over two years. When combined, FSI-174 sends a positive signal to macrophages to target blood forming stem cells for removal and magrolimab disengages inhibitory signals that block phagocytosis. Combination of these antibodies has shown efficient removal of blood forming stem cells, allowing for transplantation in pre-clinical models.

About Fanconi Anemia Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have aFANC-Agene mutation, which encodes for a protein essential for DNA repair. Mutation in theFANC-Agene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Chromosome fragility induced by DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is the gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natural gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells1.

1Soulier, J.,et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 105: 1329-1336

About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. (Nasdaq: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients contending with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. Rockets pre-clinical pipeline program is for Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. For more information about Rocket, please visitwww.rocketpharma.com.

For more information, please visit http://www.rocketpharma.com or contact info@rocketpharma.com

About Forty Seven, Inc.Forty Seven, Inc.is a clinical-stage immuno-oncology company that is developing therapies targeting cancer immune evasion pathways based on technology licensed fromStanford University. Forty Sevens lead program, magrolimab, is a monoclonal antibody against the CD47 receptor, a dont eat me signal that cancer cells commandeer to avoid being ingested by macrophages. This antibody is currently being evaluated in multiple clinical studies in patients with myelodysplastic syndrome, acute myeloid leukemia, and non-Hodgkins lymphoma.

For more information, please visitwww.fortyseveninc.comor contactinfo@fortyseveninc.com.

Follow Forty Seven on social media:@FortySevenInc,LinkedIn

Rocket Cautionary Statement Regarding Forward-Looking StatementsVarious statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to successfully demonstrate the efficacy and safety of such products and pre-clinical studies and clinical trials, its gene therapy programs, the preclinical and clinical results for its product candidates, which may not support further development and marketing approval, the potential advantages of Rocket's product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's and its licensors ability to obtain, maintain and protect its and their respective intellectual property, the timing, cost or other aspects of a potential commercial launch of Rocket's product candidates, Rocket's ability to manage operating expenses, Rocket's ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended December 31, 2019, filed March 6, 2020 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

Forty Seven Cautionary Statement Regarding Forward-Looking StatementsStatements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as will, may, assess, could, believe, and similar expressions (as well as other words or expressions referencing future events, conditions, or circumstances) are intended to identify forward-looking statements. These statements include those related to the research and development plans for Rockets and Forty Sevens respective platforms and product candidates, the timing and success of Forty Sevens collaboration with Rocket, Forty Sevens plans to pursue clinical proof-of-concept for FSI-174 plus magrolimab with the LVV HSC gene therapy platform, the focus on diseases that have the potential to be corrected with the combination of RP-L102 and Forty Sevens all-antibody conditioning regimen, the tolerability and efficacy of RP-L102, FSI-174 and magrolimab, the timing and success of any future collaborations between Forty Seven and Rocket, Forty Sevens plans to continue development of FSI-174 plus magrolimab, as well as related timing for clinical trials of the same.

Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. The product candidates that Forty Seven develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all.In addition, clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release. Such product candidates may not be beneficial to patients or successfully commercialized. The failure to meet expectations with respect to any of the foregoing matters may have a negative effect on Forty Seven's stock price. Additional information concerning these and other risk factors affecting Forty Seven's business can be found in Forty Seven's periodic filings with theSecurities and Exchange Commissionatwww.sec.gov. These forward-looking statements are not guarantees of future performance and speak only as of the date hereof, and, except as required by law, Forty Seven disclaims any obligation to update these forward-looking statements to reflect future events or circumstances.

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PERTH, Australia Australian stem cell therapy company Mesoblast Ltd. plans to evaluate its allogeneic mesenchymal stem cell (MSC) candidate, remestemcel-L, in patients with acute respiratory distress syndrome (ARDS) caused by coronavirus (COVID-19) in the U.S., Australia, China and Europe.

The company is in active discussions with various governments, regulatory authorities, medical institutions and pharmaceutical companies to implement these activities.

What people are dying of is acute respiratory distress syndrome, which is the bodys immune response to the virus in the lungs, and the immune system goes haywire, and in its battle with the virus it overreacts and causes severe damage to the lungs, Mesoblast CEO Silviu Itescu told BioWorld.

Were going to be evaluating whether an injection of our cells intravenously can tone down the immune system just enough so it gets rid of the virus but doesnt destroy your lungs at the same time.

Recently published results from an investigator-initiated clinical study conducted in China reported that allogeneic MSCs cured or significantly improved functional outcomes in all seven treated patients with severe COVID-19 pneumonia.

We have now looked at our own data in lung disease in adults where half the patients had the same kind of inflammation in the lungs as you get with coronavirus, and our cells significantly reduced the inflammation and significantly improved lung function, Itescu said, noting that he is awaiting emergency use authorization to treat patients under a clinical trial protocol.

In a post-hoc analyses of a 60-patient randomized controlled study in chronic obstructive pulmonary disease (COPD), remestemcel-L infusions were well-tolerated, significantly reduced inflammatory biomarkers, and significantly improved pulmonary function in those patients with elevated inflammatory biomarkers.

Since the same inflammatory biomarkers are also elevated in COVID-19, those data suggest that remestemcel-L could be useful in the treatment of patients with ARDS due to COVID-19. The COPD study results have been submitted for presentation at an international conference, with full results to be submitted for publication shortly.

Mortality in COVID-19-infected patients with the inflammatory lung condition is reported to approach 50% and is associated with older age, co-morbidities such as diabetes, higher disease severity, and elevated markers of inflammation.

Current therapeutic interventions do not appear to be improving in-hospital survival, and remestemcel-L has potential for use in the treatment of ARDS, which is the principal cause of death in COVID-19 infection.

Itescu said he didnt know of any other stem cell companies that were doing this. He said that other companies could try the approach from a research perspective but that Mesoblast has all the patents locked down.

The companys intellectual property portfolio encompasses more than 1,000 patents or patent applications in all major markets and includes the use of MSCs obtained from any source for patients with ARDS, and for inflammatory lung disease due to coronavirus (COVID-19), influenza and other viruses.

Remestemcel-L is being studied in numerous clinical trials across several inflammatory conditions, including in elderly patients with lung disease and adults and children with steroid-refractory acute graft-vs.-host disease (aGVHD).

Mesoblasts stem cell therapy is currently being reviewed by the FDA for potential approval in the treatment of children with steroid-refractory aGVHD. The company submitted the final module of a rolling BLA in January.

Remestemcel-L is being developed for rare pediatric and adult inflammatory conditions. It is an investigational therapy comprising culture-expanded MSCs derived from the bone marrow of an unrelated donor and is administered in a series of intravenous infusions.

The stem cell therapy is believed to have immunomodulatory properties to counteract the inflammatory processes that are implicated in several diseases by down-regulating the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues, according to Mesoblast.

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The College of Engineering for the first time has five faculty members who have been awarded National Science Foundations (NSF) Faculty Early Career Development (CAREER) grants. Four of the recipients of the five-year grants, Lauren B. Andrews,Peter J. Beltramo,Jungwoo Leeand Sarah L. Perry, are assistant proferssors in chemical engineering, while Xian Duis an assistant professor in mechanical and industrial engineering.

Sanjay Raman, dean of the College of Engineering, welcomed news of the grants. These awards are a testimony to the remarkable potential of these early-career UMass engineering faculty, he says. They are also the product of strong faculty development programs at the college and university levels, and outstanding mentorship by colleagues across the college. We look forward to the impactful research and educational innovations of these rising stars in emerging areas such as therapeutics and vaccine development, tissue engineering, biomanufacturing, biosensors and flexible electronics.

Du is the principal investigator on a $571,655 grant that focuses on improvements in roll-to-roll soft lithography. He is establishing a learning-based modeling method that guides the design and control of continuous microcontact printing processes and investigates continuous pattern formation mechanisms.

Andrews, the Marvin and Eva Schlanger Faculty Fellow in chemical engineering, will do researchstudying how communities of bacteria can be engineered to have coordinated behaviors. This will have numerous applications in biomanufacturing, cell-based therapies, and medical diagnostics. Andrewss $589,060 grant will fund research into developing a new approach for effectively programming how cells in a bacterial community work together in a predictive and highly controllable way.

Beltramos $592,332 grant will support his work on understanding the interplay between lipid composition and biomolecule transport in biological membranes. This is fundamental research that could enable the development of such breakthroughs as advanced drug delivery systems, biosensors, and other biomimetic materials.

Lee says his $549,710 grant will fund research that could lead to a greater understanding through which bone remodeling and blood forming processes are functionally coupled in porus, or trabecular bone cavities, by creating tissue engineered stem cell bone marrow models.

Perrys $657,920 grant will fund a study of a groundbreaking new approach to protein stabilization based on nature-inspired strategies. Her research has the ultimate goal of boosting the accessibility of vaccines and other therapeutics, especially in developing countries, and extending the reach of temperature-stable proteins to sensing and catalysis applications.

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Five College of Engineering Faculty Win NSF CAREER Grants - UMass News and Media Relations

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Drug Target Review explores five of the latest research developments in the field of spinal cord injury (SCI) repair.

MRIs of Lumbar & Thoracic spine showing how a fracture of thoracic spine gets worse over time.

Researchers have shown that increasing energy supply to injured spinal cord neurons can promote axon regrowth and motor function restoration after a spinal cord injury (SCI).

We are the first to show that spinal cord injury results in an energy crisis that is intrinsically linked to the limited ability of damaged axons to regenerate, said Dr Zu-Hang Sheng, study co-senior author, senior principal investigator at the US National Institute of Neurological Disorders and Stroke (NINDS).

According to the team, energy levels are damaged because the mitochondria that produce adenosine triphosphate (ATP) for neurons are located in the axons. When damaged, the mitochondria are unable to produce ATP at the same level.

Nerve repair requires a significant amount of energy, said Dr Sheng. Our hypothesis is that damage to mitochondria following injury severely limits the available ATP and this energy crisis is what prevents the regrowth and repair of injured axons.

The scientists suggest that this is compounded by the anchoring of mitochondria in adult cells alongside the axons, so once damaged they are hard to replace.

Using a murine model, called a Syntaphilin knockout, where mitochondria are free to move along the axons, the researchers showed that when mitochondria are more mobile, mice have significantly more axon regrowth across the site of SCI compared to control animals. The paper also demonstrated that newly-grown axons made appropriate connections beyond the injury site, leading to functional recovery of motor tasks.

They hypothesised that increasing mitochondrial transport and thus the available energy to the injury site could enable repair of damaged nerve fibres.

When fed creatine, a compound that enhances the formation of ATP, both the control and knockout mice had increased axon regrowth following injury, compared to mice fed saline instead. More robust nerve regrowth was seen in the knockout mice that received creatine.

We were very encouraged by these results, said Dr Sheng. The regeneration that we see in our knockout mice is very significant and these findings support our hypothesis that an energy deficiency is holding back the ability of both central and peripheral nervous systems to repair after injury.

Dr Sheng highlighted that despite the promising results of the study published in Cell Metabolism, genetic manipulation was required for the best regrowth as creatine produced only modest regeneration. He concluded that further research is required to develop therapeutic compounds that are more effective in entering the nervous system and increasing energy production for the treatment of SCI.

Experiments exploring the role of immune and glial cells in wound healing and neural repair has revealed that Plexin-B2, an axon guidance protein, is essential for their organisation after SCI.

The researchers suggest their findings could aid in the development of therapies that target axon guidance pathways for treatment of SCI.

An artists impression of a macrophage.

The paper published in Nature Neuroscience reveals that Plexin-B2 on macrophages and microglia is essential for the process of corralling, where microglia and macrophages are mobilised and form a protective barrier around the site of SCI, separating healthy and necrotic tissue. In this study, researchers found that corralling begins early in the healing process and requires the ability of Plexin-B2 to steer immune cells away from colliding cells.

When they deleted Plexin-B2 from the microglia and macrophages in tissues, it led to tissue damage, inflammatory spillover and hindered axonal regeneration.

The lead investigator Dr Hongyan Jenny Zou, Professor of Neurosurgery and Neuroscience at the Icahn School of Medicine at Mount Sinai, US, said the results were quite unexpected.

She concluded that understanding the signalling pathways and interactions of glial cells with each other and the injury environment is fundamental to improving neural repair after a traumatic brain or spinal cord injury.

Another studyexploring the interactions of macrophages and microglia has revealed that in the central nervous system (CNS), microglia interfere with macrophages preventing them from moving out of damaged regions of the CNS.

We expected the macrophages would be present in the area of injury, but what surprised us was that microglia actually encapsulated those macrophages and surrounded them almost like police at a riot. It seemed like the microglia were preventing them from dispersing into areas they should not be, said Jason Plemel, a medical researcher at Canadas University of Alberta and a member of the Neuroscience and Mental Health Institute.

A microglial cell stained with Rio Hortegas silver carbonate method under the microscope.

Plemel said that more research is required to ascertain why this is happening, but they found that both the immune cells that protect the CNS, microglia and the immune cells of the peripheral immune system, macrophages, are present early after demyelination and microglia continue to accumulate at the expense of macrophages.

When we removed the microglia to understand what their role was, the macrophages entered into uninjured tissue. This suggests that when there is injury, the microglia interfere with the macrophages in our CNS and act as a barrier preventing their movement.

The scientists said that this observation was only possible because they were able to distinguish between microglia and macrophages, which has historically not been possible. Using this technique, they established than one type of microglia responded to demyelination. The results were published in Science Advances.

The indication of at least two different populations of microglia is an exciting confirmation for us, said Plemel. We are continuing to study these populations and hopefully, in time, we can learn what makes them unique in terms of function. The more we know, the closer we get to understanding what is going on (or wrong) when there is neurodegeneration or injury and being able to hypothesise treatment and prevention strategies.

Researchers suggest subpially-injecting neural precursor cells (NSCs) may reduce the risk of further injury associated with current spinal cell delivery techniques.

NSCs have the potential to differentiate into many neural cell types depending on the environment and have been the subject of investigation in both the field of SCI repair and neurodegenerative disease therapies.

subpially-injected cells are likely to accelerate and improve treatment potency in cell-replacement therapies for several spinal neurodegenerative disorders

However, the senior author of this study Dr Martin Marsala, professor in the Department of Anesthesiology at University of California (UC) San Diego School of Medicine, US, explained the current delivery techniques involve direct needle injection into the spinal parenchyma the primary cord of nerve fibres running through the vertebral column, so there is an inherent risk of (further) spinal tissue injury or intraparenchymal bleeding.

The novel technique Dr Marsala proposed in a paper published in Stem Cells Translational Medicine, is to inject these cells into the spinal subpial space an area between the pial membrane and the superficial layers of the spinal cord.

This injection technique allows the delivery of high cell numbers from a single injection, Dr Marsala explained. Cells with proliferative properties, such as glial progenitors, then migrate into the spinal parenchyma and populate over time in multiple spinal segments as well as the brain stem. Injected cells acquire the functional properties consistent with surrounding host cells.

The research collaborators suggest that subpially-injected cells are likely to accelerate and improve treatment potency in cell-replacement therapies for several spinal neurodegenerative disorders. This may include spinal traumatic injury, amyotrophic lateral sclerosis and multiple sclerosis, said study senior author Dr Joseph Ciacci, a neurosurgeon at UC San Diego Health.

The team now intend to move their experiments from rats to larger pre-clinical animal models, more anatomically similar to humans. The goal is to define the optimal cell dosing and timing of cell delivery after spinal injury, which is associated with the best treatment effect, concluded Dr Marsala.

Dr Mohamad Khazaei is the recipient of the STEM CELLS Translational Medicines (SCTM) Young Investigator Award for his work on SCI.

The award recognises advancements in the field of stem cells and regenerative medicine made by young researchers. The recipient is the principal author of an article published in SCTM that, over the course of a year, is deemed to have the most impact.

Dr Khazaeis work focuses on bringing cell-based strategies, such as NSC transplantation, into the therapeutic pipeline through generating and differentiating novel cell types using genetic and cell engineering approaches.

While we currently lack effective regenerative medicine treatment options for spinal cord injuries, Dr Khazaeis work to create a cell transplantation therapy utilising neural precursor cells is novel and provides a promising approach, said Dr Anthony Atala, Editor-in-Chief of SCTM and director of the Wake Forest Institute for Regenerative Medicine.

His winning paper details how Dr Khazaei and his team used neurons and oligodendrocytes to obtain better functional recovery after SCI.

Related topicsCell Regeneration, CNS, Disease research, Drug Delivery, Drug Discovery, Drug Targets, Neurons, Neurosciences, Regenerative Medicine, Research & Development, Therapeutics

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Exploring future spinal cord injury therapies - Drug Target Review

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Adam Castillejo revealed himself to be the London Patient. Photo: Courtesy Adam Castillejo/Facebook

A London man who still has undetectable virus 30 months after stopping antiretroviral treatment is likely the second person ever cured of HIV, according to a report presented this week at the Conference on Retroviruses and Opportunistic Infections.

Two days before the conference was set to open in Boston, organizers decided to make the meeting virtual due to concerns about the coronavirus. Researchers gave their presentations via webcasts.

At last year's CROI, Dr. Ravindra Gupta of University College London reported that the so-called London Patient, who received a bone marrow transplant using stem cells from a donor with natural resistance to HIV, had no detectable virus in his blood plasma or T cells 18 months after stopping treatment.

At this week's meeting, Gupta said that an additional year of more extensive testing had found no functional HIV in the man's blood, lymph nodes, semen, gut tissue or cerebrospinal fluid.

"After 2.5 years off antiretrovirals and lack of evidence for any active virus, this almost certainly represents cure," Gupta told the Bay Area Reporter.

A day before Gupta's presentation, the New York Times revealed that the man, Adam Castillejo, 40, had decided to go public as the London Patient. Castillejo, who grew up in Venezuela, moved to London in 2002 and was diagnosed with HIV a year later. He is now leading a healthy and active life.

"My message to everyone out there living and coping with HIV is to not give up hope," Castillejo told the B.A.R. "I do hope that me going public will give some encouragement and empower people to keep breaking the stigma associated with HIV."

Resistant T cellsLike former San Francisco resident Timothy Ray Brown, known as the Berlin Patient the only other person known to be cured of HIV Castillejo underwent a bone marrow transplant to treat advanced cancer. According to the Times story, he spoke with Brown repeatedly before deciding to reveal his identity.

In both cases, doctors searched an international registry to find donors with double copies of an uncommon genetic mutation known as CCR5-delta-32, which makes T cells resistant to most types of HIV.

Brown received two stem cell transplants to treat leukemia in 2006, first undergoing strong chemotherapy and radiation to kill off his cancerous immune cells. He stopped antiretroviral therapy at the time of his first transplant, but his viral load did not rebound as expected. Over years of testing, researchers have found no functional virus anywhere in his body. Brown has now been free of HIV for more than 13 years.

Castillejo was diagnosed with lymphoma in 2011. After five years of grueling treatment, he underwent a bone marrow transplant in May 2016. But he received less aggressive chemotherapy than Brown and was able to stay on antiretroviral therapy.

The transplant led to complete remission of his lymphoma. Post-transplant tests showed that most of his T cells now lacked the CCR5 receptors HIV uses to enter the cells. In September 2017, with no evidence of viable HIV in his blood, he stopped his antiretrovirals in a closely monitored analytic treatment interruption.

When Castillejo was last tested on March 4, his plasma viral load remained undetectable using an ultrasensitive assay. Viral load was also undetectable in his semen and cerebrospinal fluid surrounding the brain and spinal cord. Biopsies showed no evidence of functional HIV in a lymph node or in his large or small intestine. Some bits of HIV genetic material were detected in long-lived memory T cells, but Gupta said these are probably "fossils" that cannot trigger active viral replication.

If this does prove to be a second cure, experts caution that the high-risk procedure will not be an option for people with HIV who do not need the treatment for cancer. But researchers are working on ways to mimic the same effect using gene therapy to delete CCR5 receptors from T cells or stem cells that give rise to all immune cells.

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The world of Netflix's Altered Carbon is one where consciousness is no longer tethered to the physical body. It can be, and regularly is, uploaded into "cortical stacks," which are implanted at the base of the neck. In the event of death, a persons consciousness can be reloaded into a new body, known as a "sleeve." For those less fortunate, like protagonist Takeshi Kovacs, that might mean receiving a body thats not your own. In one particularly existential example from the series first episode, it might even mean a young child being uploaded into the body of an adult.

For those with means, however, the mind can be placed into a swiftly made, identical clone, allowing them to return to their lives with little interruption. We've covered what it might take to create a digital copy of a persons mind before (spoiler: it wouldnt be easy), but Altered Carbon's techno-immortality requires a second piece: the swift creation of replacement bodies.

One of the major hurdles that has kept real-world cloning from being the game changer everyone suspected it might be after the birth of Dolly, the first successfully cloned mammal, is the relatively slow development of human bodies. If you wanted to clone a 50-year-old human and get them back to the same stage of development, it would take you 50 years. That's a little too slow to make use of in the same way science fiction does.

We don't have the means to artificially age a body at a rapid pace, but what if we were able to shortcut these limitations to put it plainly, what would it take to build an adult body from scratch?

BONES

If you want to build a person from scratch, you must first make the universe. Carl Sagan said something like that, I think. Just after that, though, youll need a skeleton. Without bones, youll be left with little more than a Cronenbergian nightmare, cool in its own way, but not what were shooting for.

Today, if you have trouble with your bones, your options are limited. The first option, and in most cases the best one, is to let the bone heal itself. Your body is pretty resilient and capable of repairing most day-to-day injuries, even the ones accompanied by a sickening crack. If the injury is really bad, things get a little more medieval. Surgeons might use a series of metal plates and screws to hold your bones in place and give them time for your bodys healing processes to do their work. But those solutions only work for relatively minor injuries where the bone tissue is at least moderately intact.

When it comes to bone replacements, things are a little tougher.

Again, we can return to metal. Like the Wolverine, you might have part of your skeleton replaced or covered over with metal. This might be sufficient in specific cases, but it all feels a little crude.

Ramille Shah, Ph.D., headed a team out of Northwestern's McCormick School of Engineering to create a new material capable of instigating rapid bone regeneration. The team used 3D printers (the invention that never stops giving) and a mixture of 90 percent hydroxyapatite, a natural element of human bones, and 10 percent medical polymer to build bone constructs.

The result is a bit of artificial bone modeled in whatever shape the patient needs. It is porous, allowing for blood vessels and other tissues to easily integrate. The elastibone (perhaps the worst superhero name, trademark pending) stimulates bone regeneration and degrades over time. The intent is for the artificial structure to dissipate, leaving actual bone in its place. A technology like this would go a long way to repairing complex bone defects in all manner of patients, but is particularly promising in pediatrics, where the patients are still growing.

But, in order to truly build a bone from scratch, well need something even better. Thats where Nina Tandon and EpiBone come in.

This technology would work by taking a sample of fatty tissue, something readily available if your plan is to build a copy of an existing person, and use it to extract stem cells. Those cells would then be applied to a 3D printed scaffold of a cows bone which has been scrubbed of all its living cells. Those undifferentiated stem cells would then be placed into a bioreactor (something which sounds made up but is very real) and coaxed into growing into a fully formed bone in just a few weeks. Given enough bioreactors, and enough cows (pour one out for our fallen bovine brethren) you could feasibly grow an entire skeleton in the time it takes for you to finally fold the laundry thats been sitting in the corner of your room.

Now that youve got a skeleton, youre going to need some

ORGANS

For a long time, there weren't many ways to get a new organ if you needed one. The most commonly used method (we hope) was to get your name on a list and wait for a donor. The unfortunate reality of organ donation is that there are more people who need organs than there are organs available. Even when an organ does become available, the odds are against you that theyll match your bodys preferences, and even if you get a match, theres always the threat of rejection.

Organ transplants are a veritable miracle procedure and, while we sometimes take them for granted, they are evidence of our living in truly wizardly times in medical science. But science is never content with the status quo and humanity is forever wondering if we can further laugh in the face of nature. The preferred solution would be to develop a way to craft bespoke organs, made from the recipients' own cells.

Growing cells in a petri dish is old hat. Weve been doing that for longer than many of us have been alive. The trouble is, you can take a heart cell and induce it to multiply in a dish, but all you end up with is a dish-shaped collection of heart cells. That might be good for studying cellular biology, not so good for pumping blood through a person.

A collection of cells does not an organ make. You need something more a scaffold. Each of your organs is a complex collection of various cell types clinging to a protein structure. You can think of that structure as the framing around which the rest of a house is built. Without it, you've got little more than some insulation and drywall tossed into a haphazard stack. You need that scaffold.

There are hopes that one day well be able to build them via (drum roll please) 3D printing, but were not there yet. The level of minute detail involved is beyond our current ability. So, we have to borrow from nature.

Scientists are able to take an existing organ and strip it of its surface cells by pumping detergent through it (good for removing pesky stains and unwanted biological material). Whats left is a ghostly protein structure ready for seeding.

All that's left is to take tissue samples from the recipient and seed them onto the structure, pop it into one of those handy bioreactors, and let the cells get to work. Eventually, youll end up with an organ made of the patients own tissues. Current tests are pretty impressive, but were still a ways off from having a functioning process. The number of different tissue types involved in complex organs is a barrier and the complexity of small structures like circulatory vessels is another. Still, the technology is promising and would not only allow us to build any and all organs in record time, it would solve the organ transplant shortage and save countless lives.

So, now youve got a rigid skeleton filled with juicy oozing organs. Your neighbors are starting to wonder about the smell coming from your garage and youre grateful this abominable creature is not yet sentient because it would very likely go running for the hills. At least it would if it had

MUSCLES

Look, we all know its been a while since youve been to the gym. You bought a membership for the new year and you went a few times. You really meant well but life happened and, somehow, it all got away from you. We get it. It happens to the best of us.

While you might not have the muscle mass you wish you had, you still have quite a lot. The average persons body is comprised of somewhere between 35 and 40 percent muscle, give or take. Thats a lot. Even after all of your efforts with bioreactors, youve only managed to make 60 percent of a person. Its nothing to be scoffed at, but you arent done yet.

In order to complete next steps, youre going to need more tissue samples and a few friends from Duke University.

Using human cells that were no longer stem cells but not yet muscle cells, Nenad Bursac and Lauran Madden, an associate professor of biomedical engineering and a postdoctoral researcher, respectively, were able to successfully create functioning muscle tissues in a lab.

They grew the tissue samples and, using a 3D scaffold and a nutritive gel, ended up with working muscle fibers. These bundles of muscle fibers included receptors capable of taking in external stimuli and contracted when acted on by electricity.

For their part, the intent is not to build novel muscular structures, but to test the efficacy of drugs to treat diseases. According to Bursac, drug tests in the laboratory matched results seen in living patients. Those patients with muscular ailments could provide a tissue sample, that sample could then be grown into fiber bundles and used to test various drug treatments, ex vivo, to find a workable treatment without all the trial and error usually required.

Thanks to Bursac and the team at Duke, youve now built almost all of Takeshi Kovacs. Hes twitching and moving around on the table. He might be screaming a little, thanks to those vat-grown lungs and hes still oozing a bit. Most of all, hes embarrassed by his nakedness. A lots changed in the intervening centuries, but not the need for

SKIN

Youve got your terrible Frankensteinian gift all put together, all thats left is the wrapping. Here, too, is an area were moderately familiar with. When a patient loses skin through injury, a graft can be taken from elsewhere and used to replace the damaged tissue. It gets the job done, some skin is better than no skin of course, but theres still room for improvement.

More recently, bioengineers have had some success in growing sheets of epithelial tissue for implantation but they lacked oil and sweat glands. Again, close, but not quite. Until

A study undertaken at the RIKEN Center for Developmental Biology, led by Takashi Tsuji took cells from the gums of mice and used chemicals to revert them to a stem-cell-like state. The cells were used to grow complex skin tissues.

Once the tissues were ready, they were transplanted onto living mice and were found to develop normally. Not only did those tissues function as a protective barrier, the primary function of skin, but they also succeeded in developing hair follicles and sweat glands. Even more importantly, they successfully integrated with surrounding tissue systems like muscle groups and nerves.

There are, of course, other tissue types weve not covered, each of them important to the successful functioning of a body, but if these emerging technologies are any indication, were well on our way in those areas as well.

So, youve done it. Youve made a full-grown human from scratch in months rather than decades. All thats left is to upload a mind and youre well on your way to cyberpunk chicanery. Go forth, Kovacs, we're rooting for you. And dont mess up this body, please. It was really hard to make. Thanks.

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How to build a body from scratch, Altered Carbon-style - SYFY WIRE

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Dublin, March 10, 2020 (GLOBE NEWSWIRE) -- The "Cell Therapy - Technologies, Markets and Companies" report from Jain PharmaBiotech has been added to ResearchAndMarkets.com's offering.

The cell-based markets was analyzed for 2018, and projected to 2028. The markets are analyzed according to therapeutic categories, technologies and geographical areas. The largest expansion will be in diseases of the central nervous system, cancer and cardiovascular disorders. Skin and soft tissue repair as well as diabetes mellitus will be other major markets.

The number of companies involved in cell therapy has increased remarkably during the past few years. More than 500 companies have been identified to be involved in cell therapy and 309 of these are profiled in part II of the report along with tabulation of 302 alliances. Of these companies, 170 are involved in stem cells.

Profiles of 72 academic institutions in the US involved in cell therapy are also included in part II along with their commercial collaborations. The text is supplemented with 67 Tables and 25 Figures. The bibliography contains 1,200 selected references, which are cited in the text.

This report contains information on the following:

The report describes and evaluates cell therapy technologies and methods, which have already started to play an important role in the practice of medicine. Hematopoietic stem cell transplantation is replacing the old fashioned bone marrow transplants. Role of cells in drug discovery is also described. Cell therapy is bound to become a part of medical practice.

Stem cells are discussed in detail in one chapter. Some light is thrown on the current controversy of embryonic sources of stem cells and comparison with adult sources. Other sources of stem cells such as the placenta, cord blood and fat removed by liposuction are also discussed. Stem cells can also be genetically modified prior to transplantation.

Cell therapy technologies overlap with those of gene therapy, cancer vaccines, drug delivery, tissue engineering and regenerative medicine. Pharmaceutical applications of stem cells including those in drug discovery are also described. Various types of cells used, methods of preparation and culture, encapsulation and genetic engineering of cells are discussed. Sources of cells, both human and animal (xenotransplantation) are discussed. Methods of delivery of cell therapy range from injections to surgical implantation using special devices.

Cell therapy has applications in a large number of disorders. The most important are diseases of the nervous system and cancer which are the topics for separate chapters. Other applications include cardiac disorders (myocardial infarction and heart failure), diabetes mellitus, diseases of bones and joints, genetic disorders, and wounds of the skin and soft tissues.

Regulatory and ethical issues involving cell therapy are important and are discussed. Current political debate on the use of stem cells from embryonic sources (hESCs) is also presented. Safety is an essential consideration of any new therapy and regulations for cell therapy are those for biological preparations.

Key Topics Covered

Part I: Technologies, Ethics & RegulationsExecutive Summary 1. Introduction to Cell Therapy2. Cell Therapy Technologies3. Stem Cells4. Clinical Applications of Cell Therapy5. Cell Therapy for Cardiovascular Disorders6. Cell Therapy for Cancer7. Cell Therapy for Neurological Disorders8. Ethical, Legal and Political Aspects of Cell therapy9. Safety and Regulatory Aspects of Cell Therapy

Part II: Markets, Companies & Academic Institutions10. Markets and Future Prospects for Cell Therapy11. Companies Involved in Cell Therapy12. Academic Institutions13. References

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

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

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Cell Therapy Insights Report, 2018-2028: Markets, Technologies, Ethics, Regulations, Companies & Academic Institutions - Benzinga

Skin Stem Cells Comments Off on Cell Therapy Insights Report, 2018-2028: Markets, Technologies, Ethics, Regulations, Companies & Academic Institutions – Benzinga | March 12th, 2020

I'm just going to come out and say it: Everyone has been complimenting my skin lately. My co-workers, random peopleat the grocery store, my friends and familyeveryone. While I've never dealt with any major skin woes like acne, I still never considered myself to be someone with particularly good skin (whatever that means). My skin has always been on thedry side, and like many women of color, I've dealt with my fair share of stubborn hyperpigmentation. The sudden influx of complimentshasn't just been a nice little boost to my newly 30-year-old ego but also a testament to my current skincare routine, which I've tweaked to perfectionover the course of several months.

As a beauty editor, I have access to every product under the sun. But ever since last fall, I felt like my skin had just lost something. When I think back, it makes sense, as there was lot was happening at the time. I had moved, turned 30, gotten engaged, andmade a major professional moveall in a matter of months, and while each of these life developments was exciting and positive, I found myself overwhelmed with stress. I wasn't sleeping well, I wasn't eating properly, my skincare routine had fallen by the wayside, and all of that was showing up on my face. I was getting pimples, my skin tone was blotchy and uneven, and my skin texture was less than smooth.

But then, something started to happen around January: Every time I posted a photo of my mug on Instagram, a sea of adulation would flood into my comments. I started catching glimpses of my makeup-free face and being truly happy with what I saw staring back at me. Maybe it was the newfound self-love I'd been practicing in therapy, or maybe it was my skincare regimen, whichhadadmittedly reached an all-time level of bougie, even for me. Now, I'm at a place where I'llfreely leave the house without makeup on and am genuinely pleased with how healthy and smooth my skin looks. I'm not perfect, by any means, but Iamgenuinely happy, and I have to believe that my fresh, smooth skin has something to do with it. So here it goes: the exact skincare routine thatdelivered smooth, glowing skin in a month's time and continues to do so to this day. Try it out for yourself and let me know what you think.

Klur Gentle Matter ($22)

I thoroughly cleanse my skin at night, so I don't always use a cleanser in the morning. Most days, I find that warm water is enough. When I do feel the need to cleanse in the morning, though,this gel cleanser is the onlyone I'll reach for. It's so gentle and actually adds moisture and nutrients like green tea, dandelion, and olive oil into my skin instead ofjust pulling everything out.

SkinCeuticals C E Ferulic ($166)

Vitamin C is probably the most important component of my skincare routine right now. While there's a lot of debate around L-ascorbic acid and whether or not its potency is actually good for the skin (jury's still out on that one), I find thatmy skin responds really well to it. Vitamin E and ferulic acid round out this formula with extra skin lipid and antioxidant protection. I can alwaystell when I've gotten lazy with my vitamin C regimen because marks from old blemishes will start to deepen, and my skin will lose some of the glow and refinement that earns me an insane amount of compliments.

Bioderma Sensibio Eye Contour Gel ($20)

I'll admit that I didn't take eye cream seriously until about a year ago, and this non-intimidating tube is to thank for that change of heart. The cream inside is lightweight and easy to lightly tap into my eye area. When I'm using eye cream consistently, I notice that any fine lines in the area soften over time, giving me that smooth, even texture I'm always after.

Kiehl's Ultra Facial Cream ($32)

This moisturizer has been an on-again, off-again staple on my vanity for years now. It's unscented, lightweight, and super effective. If I'm feeling extra dry, I'll even add afew small drops of marula oil to give it an extra hit of moisture.

Victoria Beckham by Augustinus Bader Cell Rejuvenating Priming Moisturizer ($145)

This moisturizing primer is basically like a blurring filter for your skin. It has tiny sparkly particles and theproprietary TFC-8 technology found in Augustinus Bader's other famous creams (more on those later). It makes my skin look way smoother, even when I don't layer any makeup on top.

Elta MD UV Clear Broad-Spectrum SPF 46 ($28)

Say it with me: SPF, all the time, no matter what. UV protection is important for so many reasons, but for me, it's all about mitigating hyperpigmentation and making sure any scars or blemishes on my face aren't getting exposed to the sun. This sunscreen by Elta MD is a dermatologist favorite, and it's one of my favorites, too. It doesn't irritate my skin or leave an unsightly white cast.

Farmacy Green Clean Makeup Removing Cleansing Balm ($34)

I'm a makeup wearer, so my nighttime cleansing ritual has always been serious. I need every stitch ofgunk off of my face before I can relax for the evening. This cleansing balm melts even the most stubborn eye makeup with ease. I usually massage it into the rest of my face for about 30 seconds before concentrating on my eyes. After just a few seconds of gentle rubbing, any makeup is melted down to an inky oil that rinses away without leaving any residue behind.

Reflekt Daily Exfoliating Wash ($48)

This exfoliator is said to be gentle enough for daily use, and I've found that to be true for me. Although I've backed off from using it every single day, I still love how clean and soft my face feels after use. The multitasking jojoba beads are small and smooth, so they aren't at all harsh on the skin and also meltdown to impart moisture instead of stripping the skin.

IS Clinical Cleansing Complex ($44)

This slippery cleanser clings to every trace of grime to remove it while also retexturizing. When I want a flat wash at night instead of a gritty, exfoliating one, this is the cleanser for the job. I used to have a serious attitude about paying more than $20 for cleanser (come on, it's literally money down the drain), but this is the one that taught me the power of investing in a high-quality cleanser.

U Beauty Resurfacing Compound ($148)

I've been using the U Beauty Resurfacing Compound pretty consistently since it launched last winter, and I can honestly say that it's ascended to my skincare top five. It's so good. If smooth skin is your goal, you need to try this stuff. It's patent-pending siren capsules are designed to carry active ingredients wherever your skin needs them and bypass the healthy skin cells that don't. That's why you won't experience any redness, irritation, or peeling that typically arises when starting a retinoid. This has definitely been the hero product in my smooth-skin journey.

Moon Juice Beauty Shroom Plumping Jelly Serum ($58)

It was love at first pump with this magical, hyaluronic acid and mushroom-packed elixir. There aren't many products that make a big difference in your skin's texture after just one use, but this one does. Every time I use it, my skin instantly looks plumped and smoother.

IS Clinical Youth Eye Complex ($105)

This eye cream plumps and moisturizes my delicate under-eye skin before bed. It has a little retinol in it, which honestly freaked me out at first, but over time has resulted in major refinement of fine lines.

Augustinus Bader The Rich Cream ($170)

Are you sick of editors telling you how much they love this cream? Well, I'm sorry to tell you that I'm about to do it, too. When I'm running on fumes and can only manage to get my makeup off and slap one product on my face before bed, this is the indispensable one I can't ever skip. Maybe it's the stem cellstimulating TFC-8 technology, maybe it's some sort of sorcery, but all I know is my skin has legitimately changed in texture since I started using this cream. Real talk: It's worth every penny.

Dr. Dennis Gross Clinical Grade Resurfacing Liquid Peel ($95)

I love a good resurfacing peel, but I have to admit that I've calmed way down on the acids. I found my skin becoming more sensitized and reactive, and while I can't say for sure that my nightly resurfacing toners were to blame, I'm way better off since scaling back. Now, once a week, I'll do a pass of this two-step, clinical-grade lactic and glycolic acid peel, and it immediately makes my skin look smooth, bright, and alive. As with any super-potent acid compound, it's a good idea to patch test this one to make sure your skin doesn't have an adverse reaction.

Goldfaden MD Facial Detox ($65)

I love this clean detox mask because it's cooling and tingly on the skin but doesn't dry down so hard that it makes my face feel dry or depleted. Rinsing off the sulfur-infused paste feels like taking the biggest breath of fresh air.

Dr. Dennis Gross Hyaluronic Marine Hydrating Modeling Mask ($48)

If you know me at all, then you already know how obsessed I am with this modeling mask. I firmly believe that I could stay awake for three days straight, not drink any water the whole time, and still look fresh as a daisy after 20 minutes with this goop slopped on my face.

Klorane Smoothing and Relaxing Patches ($24)

Whether I'm prepping for a photo shoot, getting ready for a night out, or just looking to minimize puffy eyes after a couple of glasses of wine, these cornflower eye patches by Klorane get the job done like no other. The soothing hydrogeleye masks actually stay put so I can move around without them slipping off, which is a huge plus.

Pai Rosehip BioRegenerate Oil ($44)

Not only is this fatty acidrich rose-hip oil an ultra-luxe finishing touch to my nighttime routine, but it also helps to get rid of imperfections caused by an imbalance in my skin's pH. I know that using oil to treat breakouts sounds counterintuitive, but this oil does just as much to calm and soothe the skin as it does to moisturize it.

Osea Malibu Blemish Balm ($48)

Speaking of soothing salves, this coolingbalmfeels so good on top of congested skin. Whenever I notice a pimple or that my pores are looking rough, I'll spot-treat with this clean cream and let it penetrate into my skin tocalmany inflammation that's plaguing me. I'll work it in as the first step in my routine whenever I need it, and it really sets the tone for the entire day.

Up next, the 25 best products to keep your skin right and tight well past your 20s.

This article originally appeared on Who What Wear

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My Skin Is Kind of Perfect Right Now Thanks to This Exact 30-Day Routine - Yahoo Lifestyle

Skin Stem Cells Comments Off on My Skin Is Kind of Perfect Right Now Thanks to This Exact 30-Day Routine – Yahoo Lifestyle | March 12th, 2020

Cosmetic Skin Care Market 2018: Global Industry Insights by Global Players, Regional Segmentation, Growth, Applications, Major Drivers, Value and Foreseen till 2024

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Cosmetic Skin Care Market Research Insights 2019 Global Industry Outlook Shared in Detailed Report, Forecast to 2027 - News Times

Skin Stem Cells Comments Off on Cosmetic Skin Care Market Research Insights 2019 Global Industry Outlook Shared in Detailed Report, Forecast to 2027 – News Times | March 12th, 2020

SAN DIEGO (CNN) There are only two northern white rhinos left on the planet, and theyre both female. Unless scientists can make a dramatic breakthrough, the entire species will die with those two individuals.

In a nondescript building just north of San Diego, the fight to save the northern white rhino is coming down to the wire. However, the battleground here looks less like a scene from a wildlife documentary and more akin to something out of a science fiction novel.

At the San Diego Zoo Institute for Conservation Research, an army of scientists armed with liquid nitrogen, microscopes, and ultrasound machines is working around the clock to create an unprecedented first in the conservation world: they are looking to turn frozen rhino skin cells into baby rhinos.

Its not just the science that is groundbreaking, but also the team looking to save this species. Composed mostly of women, the lab is a rarity in a field traditionally dominated by men.

The first step in this conservation effort began more than four and a half decades ago in 1975 when scientists established the institutes Frozen Zoo. In a small room measuring no more than 36 square meters the skin cells of more than 10,000 individuals across 1,100 species sit in giant steel tanks suspended in time, frozen in liquid nitrogen.

Among the collection are the skin samples of 12 northern white rhinos. These are vital to the groups efforts because there is such a small gene pool of living northern whites.

The population has been decimated by poachers, who target rhinos because of the belief in parts of Asia that their horns can cure various ailments. The two surviving females both live under guard at the Ol Pejeta Conservancy in Kenya. Even thoughembryos have been producedin an Italian lab using eggs extracted from the pair, any future descendants from this kind of embryo would carry the genes of those two females.

That may not be enough genetic diversity to maintain a stable population. The hope is that the skin samples of those 12 individuals at the Frozen Zoo contain enough diversity to sustain the northern white species long-term.

The arduous task for these scientists is to create a rhino population from those samples.

Marlys Houck is curator of the Frozen Zoo. She graduated high school in 1979, the same year the Frozen Zoo froze its very first northern white rhino skin cell. She later joined the institute to work on the rhino project.

I was hired specifically to try to make the cells of the rhinos grow better because they were one of the most difficult to grow cell lines, she told CNN.

Since then, shes figured out how to successfully grow and freeze the skin cells of the northern white.

The impact of this work is not lost on her. Were losing species so rapidly, she said. One of the things we can do is save the living cells of these animals before its too late.

Were at the forefront of science today, she added. If we do everything right these cells should be here 50 years from now being used for purposes that we cant even imagine today.

Marisa Korody is one of the four scientists tasked with turning these frozen cells into new life. They have to reprogram the frozen skin cells into pluripotent stem cells. In laymans terms, Korody explains that stem cells can become any cell type in the body if theyre given the right signals.

Read: Former war zones turn into wildlife paradise

The aim is to ultimately turn the stem cells into sperm and eggs. The ambitious feat has only been achieved in animals by Japanese scientists. While Korody and her team have looked to that research as a road map, she admits that doing the same with rhinos is uncharted territory. We dont really know what twists and turns we need to take in order to get from A to B, she said.

They havent even figured out how to do this in humans, she added. We have as much information as we possibly can about humans. We have a fraction of that for rhinos.

Korody says being at the forefront of this kind of science has been a dream job. This was really the first project thats trying to apply this type of science to conservation as a whole, she said.

She may spend most of her time at work looking through the lens of a microscope, but her mind is always on the final goal for the rhinos: We want to be able to put them back into the wild one day and have them living free.

Because the remaining two female northern white rhinos cant carry a pregnancy, even if the team can create embryos, the last obstacle is finding rhinos who can carry them to term.

The woman tasked with that job is Barbara Durrant. As the director of reproductive sciences, shes spent four years studying the reproductive systems of six female southern white rhinos at the institutes sister facility, the Nikita Kahn Rhino Rescue Center.

Though the rhinos at the center are a different species, Durrant says they are the closest relative to the northern white. The aim is to eventually have them be surrogates for northern white embryos.

On any given day, Durrant can be found conducting ultrasounds to help her understand each rhinos distinct reproductive cycle. In 2019, two of the centers females gave birth to southern white babies. Both were conceived via artificial insemination, giving Durrant and the teams working on the rhino project hope for the future.

Durrant believes one reason the project works so well is because there are so many women involved. Women are naturally collaborative with each other, she said. Because we have so many obstacles along the way and challenges and setbacks, we support each other and we have sympathy for each other.

Read: Rare bird brought back from extinction in the wild

Houck says women tend to be naturally nurturing. The cells are living little organisms that were growing and tending almost every day, and I think women are drawn to taking care of something and growing it into something more.

Its wonderful leading a team of women, and I really think theyre changing the world, she added. People are going to look back and see it was this amazing group of women who quietly, unrecognized, work at this and just get better and better.

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Only 2 northern white rhinos left, and both are female these women are trying to save the species - KSNF/KODE - FourStatesHomepage.com

Skin Stem Cells Comments Off on Only 2 northern white rhinos left, and both are female these women are trying to save the species – KSNF/KODE – FourStatesHomepage.com | March 12th, 2020

DetailsCategory: DNA RNA and CellsPublished on Wednesday, 11 March 2020 09:50Hits: 179

-Cooperation in Product Development for Innovative Cardiac Regenerative Medicine-

March 10, 2020 I Tokyo-based Heartseed Inc. (Heartseed), a Keio University-originated biotechnology company developing induced pluripotent stem cell (iPSC)-derived cardiac regenerative medicine, and MEDIPAL HOLDINGS CORPORATION (MEDIPAL) today announced that they have entered into a capital and business alliance.

In conjunction with the alliance, MEDIPAL will acquire an equity stake in Heartseed. In addition, MEDIPAL and its wholly owned subsidiary SPLine Corporation (SPLine) will begin collaborative research with Heartseed on the logistics of Heartseeds clinical trial supplies.

Purpose of the Alliance

Heartseed is developing HS-001, allogeneic iPSC-derived cardiomyocyte spheroids for severe heart failure, which currently has no effective treatment other than heart transplantation. In preparation for the initiation of its clinical trial, Heartseed will outsource its manufacturing to Nikon CeLL innovation Co., Ltd., and are discussing transport of the cardiomyocyte spheroids with MEDIPAL.

MEDIPAL has established a distribution system in compliance with Japanese Good Distribution Practice (GDP) guidelines. MEDIPAL is a pioneer in logistics services in the growing field of regenerative medicine, and has an extensive track record to support development of regenerative medicine products and to build a logistics system for them using its ultra-low temperature transport system.

In this alliance, MEDIPAL will contribute to the improvement of patient care by promoting development of Heartseeds innovative products from the clinical trial stage with its experience and expertise in the distribution of regenerative medicine products.

Comment from Heartseed CEO Keiichi Fukuda, MD, PhD, FACC

The iPSC-derived cardiomyocyte spheroids we are developing are unique in the mechanism that cardiomyocytes are strengthened by turning them into microtissues. The spheroids will be retained and engrafted with the ventricular myocardium for a long-term and are expected to contribute sustained direct ventricular contraction (remuscularization). It is completely

different from conventional treatment methods. To deliver the treatment to patients, logistical considerations are also important, and we are pleased to partner with MEDIPAL, which has an extensive track record in distribution of cellular medicines.

Comment from MEDIPAL Representative Director, President and CEO Shuichi

Watanabe

Their investigational agent has the potential to be an innovative treatment option for patients with severe heart failure. Promoting the development and stable supply of specialty pharmaceuticals is our mission, based on MEDIPALs management philosophy of

contributing to peoples health and the advancement of society through the creation of value in distribution. In this alliance, SPLine, which performs logistical planning for specialty pharmaceuticals, will be involved from the clinical trial stage, and will also work with us in creating a distribution system to ensure safe and reliable delivery of the product to patients after its launch.

Development of HS-001

Heartseed has allogeneic iPSC-derived highly purified ventricular-specific cardiomyocyte spheroids (HS-001) as its lead pipeline candidate, and is conducting research and development for the early commercialization of cardiac regenerative medicine using iPSCs supplied by the Center for iPS Cell Research and Application (CiRA) at Kyoto University. HS-001 is the produced by differentiating into ventricular-specific cardiomyocytes from iPSCs with the most frequent human leukocyte antigen (HLA) type1 in Japanese people, and removing undifferentiated iPSCs and non-cardiomyocytes to achieve high purity. To improve the engraftment rate, these cardiomyocytes are formed into spheroids in which approximately 1,000 cardiomyocytes are aggregated.

Since 2016, Heartseed has had more than 10 meetings with the Pharmaceuticals and Medical Devices Agency (PMDA), with discussions mainly focused on details of nonclinical safety studies, manufacturing processes, and quality management that are required for initiating clinical trials. Heartseed is currently conducting the nonclinical safety studies under Good Laboratory Practice (GLP)2 standards under the agreement of the PMDA on their designs.

Prior to the company-sponsored clinical trials, investigator-initiated clinical trial plan of HS-001 at Keio University had been under review by the Keio University Certified Special Committee for Regenerative Medicine since May 2019 and was approved in February 2020. This plan will be submitted to the Health Science Council of Ministry of Health, Labor and Welfare after going through established procedures in Keio University Hospital. For 90 days from its submission to the Council, the plan will be examined for conformance with the regenerative medicine provision standards. If conformance is verified, Keio University will be notified and may then begin clinical research.

1. HLA type:White blood cell type, immune rejection is less likely when the HLA type matches.

2. GLP(Good Laboratory Practice):Standards for conducting studies to assess drug safety. These standards should be followed when conducting safety studies using animals in the preclinical stage.

Summary of HS-001

Severe heart failure, particularly heart failure with reduced ejection fraction

About Heartseed Inc.

About MEDIPAL HOLDINGS CORPORATION

As a holding company, MEDIPAL controls, administers and supports the operating activities of companies in which it holds shares in the Prescription Pharmaceutical Wholesale Business; the Cosmetics, Daily Necessities and

OTC Pharmaceutical Wholesale Business; and the Animal Health Products and

Food Processing Raw Materials Wholesale Business, and conducts business development for the MEDIPAL Group.

About SPLine Corporation

3.ALC: Area Logistics Center

4. FLC: Front Logistics Center

SOURCE: Heartseed

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Notice of Capital and Business Alliance between Heartseed and MEDIPAL HOLDINGS | DNA RNA and Cells | News Channels - PipelineReview.com

Cardiac Stem Cells Comments Off on Notice of Capital and Business Alliance between Heartseed and MEDIPAL HOLDINGS | DNA RNA and Cells | News Channels – PipelineReview.com | March 11th, 2020