CRISPR is a catchy acronym that originally described a naturally occurring gene editing tool, derived from a bacterial defense mechanism against viruses. Its the name on everybodys lips in the intersecting realms of science, medicine, ethics, and politics. From the moment of its discovery, CRISPR-Cas9 looked like a miraculous solution to all of the problems that gene editing efforts have experienced over decades of trial and error. This revolutionary new gene editing technique has opened the doors to both massive scientific progress and ethical controversy. Now more than ever, were seeing that CRISPR still has massive kinks to work out. Can we ever fully understand the social and scientific implications of gene editing, and should we use it in humans before we learn how to properly harness it?

What is gene editing?

The 20th century saw genetic scientists increasingly focus their pursuits on the sub-microscopic. As science delved deeper into the human body in an attempt to uncover the molecular minutiae of life, the possibility of reaching into the cell and manipulating its genetic material began to look more and more real. Even by the 1950s, evidence had been mounting for decades that deoxyribonucleic acid (DNA), an unassuming molecule residing in a central cellular compartment called the nucleus, was the physical genetic material that passed information from parent to child. Finally, in 1953, landmark work by Kings College biochemist Rosalind Franklin allowed Cambridge researchers to reveal the structure of DNA and confirm its role in heredity once and for all.

Starting from a hesitant foundation, molecular genetics exploded in both scope and popularity over subsequent decades. With the secrets of heredity increasingly out in the open, human ambition demanded that we try to bend DNA to our willand now we can. These days, targeted gene editing techniques revolve around artificially-engineered molecular tools known as nucleases, whose earliest use was in 1996not even 50 years after the discovery of DNAs structure. Engineered nucleases are often described as molecular scissors. Fundamentally, they have two main parts: one part that finds and grabs onto the target DNA within a cell, and one part that snips a piece out of that DNA.

How CRISPR works

CRISPR is similar to other directed nucleases, but its much better at its job. The CRISPR part is secondary to the systems gene editing applications; the truly important discovery, which Jennifer Doudna made in 2012, was a protein that she called CRISPR-associated protein 9, or Cas9. This protein is the nuclease tool, the pair of molecular scissors that finds, sticks to, and snips target DNAand its more accurate than anything weve ever seen before.

In bacteria, CRISPR is a section of the genome that acts as an immune memory, storing little snippets of different viruses genetic material as DNA after failed infections, like trophies. When a once-active virus attempts to invade a bacterium, the mobile helper Cas9 copies down the relevant snippet from CRISPR in the form of ribonucleic acid, or RNA. RNA is a molecule thats virtually identical to DNA, except for one extra oxygen atom. Because of this property, the RNA sequence that Cas9 holds can pair exactly, nucleotide by nucleotide, with the viral targets DNA, making it extremely efficient at finding that DNA. With a freshly transcribed RNA guide, the bacterium can deploy Cas9 to findand cut outthe corresponding section of viral genetic material, rendering the attacker harmless.

The existence of CRISPR in bacteria was old news by 2012, but Doudnas discovery of Cas9s function was revolutionary. With a little creativity and ingenuity, such a simple and accurate nuclease can be modified to be much more than just a pair of scissors. Using synthetic RNA guides and certain tweaks, Cas9 can be used to remove specific genes, cause new insertions to genomes, tag DNA sequences with fluorescent probes, and much more.

The possibilities seem endless.What if we could go into the body of a human affected by a hereditary disease and change that persons DNA to cure them? What if we could modify reproductive germ cells in human bodies (which give rise to sperm and eggs), or make targeted genetic edits in the very first cell of an embryo? Nine months of division and multiplication later, that cell would give rise to a human being whose very nature has been deliberately tweakedand their childrens nature, and their childrens. With the accuracy and accessibility of the CRISPR/Cas9 system, these ideas arent hypotheticals. In 2019, CRISPR edits in bone marrow stem cells were successfully used to cure sickle cell anemia in a Mississippi woman. Beta thalassaemia, another genetic disease of the blood, has also been treated this way. In 2018, Chinese scientist He Jiankui even claimed that he had conferred HIV immunity upon twin girls using embryonic editing.

CRISPRs complications

At first glance, CRISPR looks like a miraclebut it isnt perfect. What if some cells were affected by edits, but others werent, creating a strange genetic mosaic in a human body? What if, in trying to modify a specific gene, we accidentally hit a different section of DNA nearby? What if we got the right gene, but it also affected a different part of the body that we didnt know about?

These problems arent hypotheticals either. So-called mosaicism and off-target editing are huge concerns among CRISPR scientists. Mosaicism is of particular concern in embryonic editing. Though CRISPR injections are carried out when an embryo is single-celled, CRISPR doesnt always appear to work until after several rounds of cell divisionand it doesnt work in every cell. If not all the cells in the body are affected by gene editing that is intended to eliminate a genetic disease, the disease could remain in the body. It may be possible to combat mosaicism with faster gene editing (so that cells dont replicate before theyve had a chance to become CRISPR-modified), altering sperm and egg cells before they meet to form an embryo, and developing more precise CRISPR gene editing which is in itself a challenge, thanks to off-target editing.

In nature, a little bit of off-target editing could actually make the CRISPR-Cas9 defense system stronger with the principle of redundancy. Flexibility in the form of imprecision could allow a bacterium to neutralize viruses whose exact genetic sequences have not yet been encountered: viruses related to, but not identical to, previous attackers. In clinical and therapeutic applications, on the other hand, precision is everything. And unfortunately, as time passes, CRISPRs level of precision seems further and further off. Preprints released just this year reveal that the frequency and magnitude of CRISPRs off-target edits in human cells may be worse than we had previously known. Large proportions of cells with massive unwanted DNA deletions, losses of entire chromosomes in experimental embryos, and shuffling of genetic sequences were observed.

Of course, not only do scientists need to avoid off-target edits, but they also need to know when such undesired edits have occurred. Off-target effects can be detected by genome sequencing and computer prediction tools, but theres no perfect way to do it yetthere may still be editing misses that were, well, missing. Off-target edits themselves could be minimized by altering the RNA transcript that Cas9 carries to make it more accurate, altering Cas9 itself, or reducing the actual amount of Cas9 protein released into the cell (though this could also reduce on-target effects). Replacing Cas9 itself with other Cas variants, like smaller and more easily deliverable CasX and CasY proteins, is a promising possibility for more efficient editing, but these candidates still run into many of the same problems as Cas9. More strategies are constantly being discovered, proposed, and explored, but were still nowhere near perfect.

Perhaps most importantly, even barring any purely technical problems, is that humans remain in sheer ignorance of much of the extent and consequences of pleiotropy, a phenomenon where a genes presence or deletion has more than one effect in the human body. Even genes whose function we think we know well might have totally unexpected additional functions. On the other side of the coin, we dont have a comprehensive understanding of how many different genetic contributors there are to any given trait or disease, much less where they lie in the genome. We dont understand the way that thousands of variations across the entire genome contribute to appearance, personality, and health. Assuming that some genes are good and others are bad is morally dangerous, and scientifically reprehensible. In reality, we are not ready for genetic determinism, and may never be.

A great responsibility

Humanity has discovered a great power, but we all know what comes with great power. Questions of which edits are necessary for health (is mild Harlequin syndrome a disease or a cosmetic concern?), whether edits are ethical (should autism and homosexuality be considered curable conditions?), and the possibility of designer babies, among others, are pertinent and require thorough discussion. We also need to realize that making these types of changes isnt our decision until we can get CRISPR right, and understand the genome well enough to target particular phenotypes. Though most scientists are aware of the difficulties of CRISPR and its use is generally tightly regulated, some scientistsand laypeopleare less careful. He Jiankuis apparent miracle HIV cure led to his arrest and imprisonment for unapproved and unethical practice. Its no great surprise that his work likely fell prey to off-target effects and mosaicism; even if he got it right, his intended change could alter cognitive function, and who knows what else?

Non-scientists are getting involved too: in 2018, self-proclaimed biohacker Josiah Zayner publicly injected his own arm with what he claimed was muscle-enhancing CRISPR. Though Zayner is one of the most vocal, hes not the only one of his kind. Quieter biohackers, untrained people without a scientific background or a good understanding of how CRISPR can go wrong, are attempting to edit themselves and even their pets.

Laypeople have an unquestionable place in science: the scientific discipline needs fresh perspectives and creativity that stuffy academics cant offer. CRISPR is still in its infancy, though. Before we know much, much more about its capabilities and consequences, there can be no place for black market gene editing kits, rogue scientists altering human embryonic and germline DNA, or basement geneticists injecting Cas9 into their dogs. Who can say what effects these interventions might have, not just on edited individuals, but on the futures of entire species?

Some say that gene editing is an act of hubris, destined to backfire spectacularly and horrendously. Others believe that its our responsibility to use CRISPR to improve lives. Which of these opinions is true depends on how science walks a narrow tightrope, though Im inclined to agree with the latterand add that our responsibility is not just to master gene editing, but to make clear and public its many faults and failings. The truth, in all its complexity, needs to overcome pop sciences oversimplification and sensationalism. Promising new advances and techniques are on the horizon, but we have a long way to go. Gene editing is no joke; humanity is playing with fire. With an incredibly accurate and accessible nuclease making its way into labs and garages across the world (while its flaws continue to be uncovered year by year), it is more important than ever for the world to understand and discuss the long-reaching consequences and responsible use of gene editing technology. CRISPR is not a miracle, but gene editing may very well be the future of humanityand its on us to keep it under control.

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The Trouble With CRISPR The Strand - Strand

Bone Marrow Stem Cells Comments Off on The Trouble With CRISPR The Strand – Strand | August 10th, 2020

Brazilian researchers announced that a 36-year-old man in Brazil is HIV-free after receiving a cocktail of antiviral drugs.

If true, this unidentified case, detailed at the medical conference AIDS 2020, would be the first instance of long-term remission from HIV without a stem cell or bone marrow transplant.

But the researchers peers are sceptical, since anti-retroviral therapy, which is used to queel HIV and prevent it from developing into AIDS, has been the standard treatment for all HIV-positive people since the treatment was invented in 1995.

There will be a lot of buzz, a lot of controversy about this part everyones going to be sceptical, HIV researcher Dr. Steve Deeks told the New York Times. Am I sceptical? Of course. Am I intrigued? Absolutely.

According to the research team at the Federal University of So Paulo, the man was diagnosed with HIV in 2012 and began taking the typical antiretroviral drugs.

In 2016, he joined a clinical trial where he was given three additional drugs, including maraviroc and nicotinamide, for 11 months, in an aggressive treatment designed to flush the virus out of his body.

The man returned to the standard anti-retroviral therapy after the trial ended, and stopped taking all anti-retroviral drugs in March 2019. Every three weeks since March 2019, his blood has been tested.

The fact that he tested negative for HIV is not remarkable in itself anyone religiously taking anti-retroviral therapy for more than six months will reach an undetectable viral load.

But in this case, the researchers said they found no trace of dormant HIV-infected cells in his system.These latent cells can become active as soon as treatment stops, making people sick again.

The researchers announced that virus-detecting blood tests did not show any remaining traces of HIV in the mans blood. The man also did not show any signs of having antibodies to the virus.

Prior to this, just two people had been cured of HIV.

First was the Berlin Patient, an American man named Timothy Ray Brown, who received a bone marrow transplant in 2007 in Berlin, Germany.

Brown had leukemia, and required a bone marrow transplant to survive. His doctors sought bone marrow from someone with an HIV-resistant gene. Post-transplant, Brown suffered a series of health issues, he needed to be put in a medically induced coma, and nearly died. But not only was his cancer gone, so was his HIV. He is still alive today, with no HIV, and no need for the anti-retroviral therapy that HIV-positive people must take habitually.

In 2019, the London Patient, a man named Adam Castillejo, became the second person ever to enter long-term remission from HIV. Castillejo, who was treated in London, England, had two bone marrow transplants. His recovery process was less intense, assuaging scientists concerns that Brown had only been cured because of the massive destruction to his immune system, which also rid him of HIV.

Deeks said that independent lab results will be needed to confirm these results. The Brazilian research team has offered to send the mans blood samples to other labs.

When HIV enters the body, it inserts genetic material into the DNA of its hosts immune cells. This forces the cells to make copies of the virus. Some active HIV-infected cells are created, and some latent HIV-infected cells are created. These cells are infected with HIV but are not actively producing new HIV, according to the NIH.

But there is a difference between testing negative for HIV, as some people do after taking medication that makes their HIV undetectable, and having zero traces of HIV in your RNA or DNA. In the first instance the virus is controlled within the body, but in the second instance it is entirely removed from the body.

Many researchers have announced they have cured HIV in their patients, only for the disease to return a short while later.

A baby in Mississippi stopped taking antiretroviral medication at 18 months, researchers eagerly announced that the virus was gone, and then two years later, in 2014, researchers detected HIV in the child again. In 2013, two Boston patients received bone marrow transplants, and headlines declared that they had been cured, only for the virus to resurface again.

The So Paulo Patient has gone 66 weeks without showing signs of the virus.

Read more:

Case of HIV patient in remission raises hopes for future AIDS cure

Doctors say experimental treatment may have rid man of HIV

There is no virus there that we can measure. Second HIV patient in remission becomes new hope for a cure

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Scientists say a man with HIV is the first to reach long-term remission without a bone marrow transplant, but their peers are sceptical - Business...

Bone Marrow Stem Cells Comments Off on Scientists say a man with HIV is the first to reach long-term remission without a bone marrow transplant, but their peers are sceptical – Business… | July 10th, 2020

FT819 CAR T-cell Product Candidate Derived from Clonal Master iPSC Line with Novel CD19-specific 1XX CAR Integrated into TRAC Locus

Phase 1 Clinical Study will Evaluate FT819 for Patients with Advanced B-cell Leukemias and Lymphomas

SAN DIEGO, July 09, 2020 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, announced today that the U.S. Food and Drug Administration (FDA) has cleared the Companys Investigational New Drug (IND) application for FT819, an off-the-shelf allogeneic chimeric antigen receptor (CAR) T-cell therapy targeting CD19+ malignancies. FT819 is the first-ever CAR T-cell therapy derived from a clonal master induced pluripotent stem cell (iPSC) line, and is engineered with several first-of-kind features designed to improve the safety and efficacy of CAR T-cell therapy. The Company plans to initiateclinical investigation of FT819for the treatment of patients with relapsed / refractory B-cell malignancies, including chronic lymphocytic leukemia (CLL), acute lymphoblastic leukemia (ALL), and non-Hodgkin lymphoma (NHL).

The clearance of our IND application for FT819 is a ground-breaking milestone in the field of cell-based cancer immunotherapy. Our unique ability to produce CAR T cells from a clonal master engineered iPSC line creates a pathway for more patients to gain timely access to therapies with curative potential, said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. Four years ago, we first set out under our partnership with Memorial Sloan Kettering led by Dr. Michel Sadelain to improve on the revolutionary success of patient-derived CAR T-cell therapy and bring an off-the-shelf paradigm to patients, and we are very excited to advance FT819 into clinical development.

FT819 was designed to specifically address several limitations associated with the current generation of patient- and donor-derived CAR T-cell therapies. Under a collaboration with Memorial Sloan Kettering Cancer Center (MSK) led by Michel Sadelain, M.D., Ph.D., Director, Center for Cell Engineering, and Head, Gene Expression and Gene Transfer Laboratory at MSK, the Company incorporated several first-of-kind features into FT819 including:

The multi-center Phase 1 clinical trial of FT819 is designed to determine the maximum tolerated dose of FT819 and assess its safety and clinical activity in up to 297 adult patients across three types of B-cell malignancies (CLL, ALL, and NHL). Each indication will enroll independently and evaluate three dose-escalating treatment regimens: Regimen A as a single dose of FT819; Regimen B as a single dose of FT819 with IL-2 cytokine support; and Regimen C as three fractionated doses of FT819. For each indication and regimen, dose-expansion cohorts of up to 15 patients may be enrolled to further evaluate the clinical activity of FT819.

At the American Association for Cancer Research (AACR) Virtual 2020 Meeting, the Company presented preclinical data demonstrating FT819 is comprised of CD8 T cells with uniform 1XX CAR expression and complete elimination of endogenous TCR expression. Additionally, data from functional assessments showed FT819 has antigen-specific cytolytic activity in vitro against CD19-expressing leukemia and lymphoma cell lines that is comparable to that of healthy donor-derived CAR T cells, and persists and maintains tumor clearance in the bone marrow in an in vivo disseminated xenograft model of lymphoblastic leukemia.

Fate Therapeutics has an exclusive license for all human therapeutic use to U.S. Patent No. 10,370,452 pursuant to its license agreement with MSK1, which patent covers compositions and uses of effector T cells expressing a CAR, where such T cells are derived from a pluripotent stem cell including an iPSC. In addition to the patent rights licensed from MSK, the Company owns an extensive intellectual property portfolio that broadly covers compositions and methods for the genome editing of iPSCs using CRISPR and other nucleases, including the use of CRISPR to insert a CAR in the TRAC locus for endogenous transcriptional control.

1 Fate Therapeutics haslicensedintellectual propertyfrom MSK on which Dr. Sadelain is aninventor.As a result of the licensing arrangement, MSK has financial interests related to Fate Therapeutics.

About Fate Therapeutics iPSC Product PlatformThe Companys proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with cycles of other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Companys first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Companys platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics iPSC product platform is supported by an intellectual property portfolio of over 300 issued patents and 150 pending patent applications.

About Fate Therapeutics, Inc.Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to the development of first-in-class cellular immunotherapies for cancer and immune disorders. The Company has established a leadership position in the clinical development and manufacture of universal, off-the-shelf cell products using its proprietary induced pluripotent stem cell (iPSC) product platform. The Companys immuno-oncology product candidates include natural killer (NK) cell and T-cell cancer immunotherapies, which are designed to synergize with well-established cancer therapies, including immune checkpoint inhibitors and monoclonal antibodies, and to target tumor-associated antigens with chimeric antigen receptors (CARs). The Companys immuno-regulatory product candidates include ProTmune, a pharmacologically modulated, donor cell graft that is currently being evaluated in a Phase 2 clinical trial for the prevention of graft-versus-host disease, and a myeloid-derived suppressor cell immunotherapy for promoting immune tolerance in patients with immune disorders. Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit http://www.fatetherapeutics.com.

Forward-Looking StatementsThis release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the advancement of and plans related to the Company's product candidates and clinical studies, the Companys progress, plans and timelines for the clinical investigation of its product candidates, the therapeutic potential of the Companys product candidates including FT819, and the Companys clinical development strategy for FT819. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk of difficulties or delay in the initiation of any planned clinical studies, or in the enrollment or evaluation of subjects in any ongoing or future clinical studies, the risk that the Company may cease or delay preclinical or clinical development of any of its product candidates for a variety of reasons (including requirements that may be imposed by regulatory authorities on the initiation or conduct of clinical trials or to support regulatory approval, difficulties in manufacturing or supplying the Companys product candidates for clinical testing, and any adverse events or other negative results that may be observed during preclinical or clinical development), the risk that results observed in preclinical studies of FT819 may not be replicated in ongoing or future clinical trials or studies, and the risk that FT819 may not produce therapeutic benefits or may cause other unanticipated adverse effects. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Companys actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Companys periodic filings with the Securities and Exchange Commission, including but not limited to the Companys most recently filed periodic report, and from time to time in the Companys press releases and other investor communications.Fate Therapeutics is providing the information in this release as of this date and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise.

Contact:Christina TartagliaStern Investor Relations, Inc.212.362.1200christina@sternir.com

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Fate Therapeutics Announces FDA Clearance of IND Application for First-ever iPSC-derived CAR T-Cell Therapy - GlobeNewswire

Bone Marrow Stem Cells Comments Off on Fate Therapeutics Announces FDA Clearance of IND Application for First-ever iPSC-derived CAR T-Cell Therapy – GlobeNewswire | July 10th, 2020

By David Bautz, PhD

NASDAQ:BCLI

READ THE FULL BCLI RESEARCH REPORT

Business Update

KOL Event for Alzheimers Program

On July 8, 2020, BrainStorm Cell Therapeutics, Inc. (NASDAQ:BCLI) conducted a Key Opinion Leader (KOL) webinar to discuss the companys upcoming Phase 2a clinical trial of NurOwn in patients with Alzheimers Disease (AD). The event included presentations by two of the lead investigators for the upcoming trial, Dr. Philip Scheltens, Professor of Cognitive Neurology and Director of the Alzheimer Centre at VU University Medical Center in Amsterdam, Netherlands, and Dr. Bruno Dubois, Professor of Neurology at the Neurological Institute of the Salptrire University Hospital in Paris, France. The presentation can be found here.

The companys Phase 2a trial (BCT-201-EU) is expected to enroll approximately 40 patients with prodromal to mild AD. It will be taking place at medical centers in France and the Netherlands. To be eligible for the trial, patients must have been diagnosed with prodromal to mild dementia at least six months prior to enrollment. In addition, patients must score between 20-30 on the Mini-Mental State Exam (MMSE) and have a Clinical Dementia Rating (CDR) global score of 0.5-1.0. The MMSE is a series of questions that are designed to assess a patients mental skills, with the maximum score being 30 points and a score of 20-24 suggesting mild dementia. The CDR is a scale used to characterize six domains of cognitive and functional performance with a score of 0.5 suggesting very mild dementia and a score of 1.0 suggesting mild dementia.

The primary objective of the trial is to assess the safety and tolerability of three intrathecal injections of NurOwn in AD patients. Following bone marrow aspiration during a 10-week run-in period, patients will be treated three times with NurOwn, with eight weeks between treatments. Follow-up visits will occur 12 and 26 weeks following the final injection of NurOwn for a total trial length of 52 weeks. The following figure gives an overview of the trial design.

Cerebrospinal fluid (CSF) and serum will be collected prior to treatment and again at Weeks 0, 8, and 16 to assess changes in various neurotropic, neurodegenerative, and inflammatory factors (e.g., VEGF, HGF, NfL, NfH, MCP-1, IL-6), markers associated with amyloid deposition (e.g., a40, a42), and markers of tau protein levels (e.g., p-tau, t-tau). Additional clinical outcome measures will be analyzed through administration of the following tests:

Clinical Dementia Rating ScaledSum of Boxes (CDR-SB)

Free and Cued Selective Reminding Test (FCSRT)

Neuropsychological Test Battery (NTB)

Delis-Kaplan Executive Function System (D-KEFS) subtests

Mini Mental State Examination(MMSE)

AmsterdamInstrumentalActivitiesofDailyLivingQuestionnaire-ShortVersion(A-IADL-Q-SV)

Alzheimers Disease

Alzheimers disease (AD) is the most common form of dementia in older adults. The disease is named after Dr. Alois Alzheimer, who identified the first case in a 50-year-old woman named Auguste Deter in 1902. Dr. Alzheimer followed her case until her death in 1906, at which point he first publicly reported on it (Alzheimer, 1907).

After Ms. Deters death, Dr. Alzheimer examined her brain and found many abnormal clumps (now known as amyloid plaques) and tangled bundles of fibers (now known as neurofibrillary tangles). Over the next five years, 11 similar cases were reported in the medical literature, with some of them already using the term Alzheimers disease (Berchtold et al., 1998).

The most common early symptom of AD is a gradually worsening ability to remember new information. This is due to neurons associated with forming new memories dying off first. As neurons in other parts of the brain die, individuals experience different symptoms, which include:

Memory loss that disrupts daily life

Inability to plan or solve problems

Difficulty completing familiar tasks

Confusion with location and time

Difficulty with visual images and spatial relationships

Problems with words in speaking or writing

Withdrawal from social activities

Changes in mood, including apathy and depression

Each person progresses through AD at a different rate, and little is known about how or why there is such a marked variation, thus predicting how it will affect someone is quite difficult. One thing that is common to everyone diagnosed with AD is that his or her cognitive and functional abilities will gradually decline. As the disease progresses symptoms can include confusion, irritability, aggression, mood swings, and long-term memory loss. In the final advanced stage of the disease, people need help with the basic activities of living (e.g., bathing, dressing, eating, and using the restroom), they lose the ability to communicate, fail to recognize loved ones, and eventually become bed bound and reliant on round-the-clock care (Frstl et al., 1999). The inability to move makes them more prone to infections, including pneumonia, which are often a contributing factor to the death of those with AD.

Competing Theories for the Cause of Alzheimers

The root cause of Alzheimers is still unknown; however, it is likely to involve a number of different factors as opposed to being due to one single cause. These factors are likely a combination of genetic, environmental, and lifestyle. There are a number of hypotheses that exist to explain the cause of the disease, with the two dominant hypotheses focused on amyloid and tau.

Amyloid hypothesis: This hypothesis proposes that extracellular beta-amyloid deposits are the fundamental cause of the disease (Hardy et al., 1991). Beta-amyloid is a fragment of the larger protein amyloid precursor protein (APP), mutations of which are known to cause FAD. Several lines of evidence support the amyloid hypothesis: 1) the location of APP is on chromosome 21, while those with Down Syndrome (trisomy 21) almost all show signs of AD by 40 years of age (Lott et al., 2005); 2) APOE4 is a major genetic risk factor for AD, and while apolipoproteins enhance the breakdown of beta-amyloid, some isoforms are less capable of performing this task than others, leading to more beta-amyloid buildup on the brain (Polvikoski et al., 1995); 3) mice that harbor a mutant form of APP develop amyloid plaques and Alzheimers-like pathology (Games et al., 1995). Lastly, amyloid plaques are readily identifiable by microscopy in the brains of AD patients (Tiraboschi et al., 2004). While the brains of many older individuals develop some plaques, the brains of AD patients show severe pathological changes specifically within the temporal neocortex (Bouras et al., 1994).

Tau hypothesis: Tau is a protein located mainly within the axonal compartment of neurons and is an important element in microtubule stabilization and neurite outgrowth. In AD, a proportion of tau protein becomes abnormally phosphorylated, dissociates from axonal microtubules, and accumulates in paired helical filaments inside the neuron (Goedert et al., 1991). When this occurs, the microtubules disintegrate causing the collapse of the neurons transport system (Igbal et al., 2005). Just as with beta-amyloid plaques, tau tangles are readily observable in the brains of those affected by AD.

In addition to amyloid and tau, inflammation has been an underappreciated and often overlooked mediator in patients with AD (Akiyama et al., 2000). A multitude of inflammatory markers are found in AD patients brains and a number of studies have shown a link between chronic inflammation and an increased risk of developing AD (Walker et al., 2017; Tao et al., 2018). Thus, a treatment such as NurOwn that can decrease inflammatory mediators could prove beneficial in AD patients.

On Track to Repot Topline Data from Phase 3 ALS Trial in 4Q20

On July 2, 2020, BrainStorm announced that all doses have been administered in the pivotal Phase 3 trial ofrecen NurOwn in patients with amyotrophic lateral sclerosis (ALS) and that it remains on track to report topline data in the fourth quarter of 2020.

The ongoing randomized, double blind, placebo controlled, multi-dose Phase 3 clinical trial is testing the ability of NurOwn to alter disease progression as measured by the ALSFRS-R (NCT03280056). Cells were extracted once from each patient prior to treatment, with all administrations of NurOwn derived from the same extraction of cells due to a cryopreservation process the company developed for long-term storage of mesenchymal stem cells (MSC). Just as with the companys prior studies, there was a 3-month run-in period prior to the first treatment with two additional NurOwn treatments occurring two and four months following the first treatment. The company is focusing the trial on faster-progressing ALS patients since those patients demonstrated superior outcomes in the Phase 2 trial of NurOwn.

BrainStorm Joins Russell 2000 and Russell 3000; Granted SME Status by EMA

On June 23, 2020, BrainStorm announced that its shares would be included in the Russell 2000 Index and the Russell 3000 Index. The annual reconstitution of the Russell indexes is done to capture the 4,000 largest U.S. stocks by market capitalization.

On June 15, 2020, BrainStorm announced that the company has been granted Small and Medium-Sized Enterprise (SME) status by the European Medicines Agency (EMA). SME status allows the company to participate in a number of financial incentives including a 90-100% reduction in the EMA fee for scientific advice, clinical study protocol design, endpoints and statistical considerations, quality inspections of facilities, and fee waivers for selective EMA pre- and post-authorization regulatory filings, including Orphan Drug and PRIME designations.

Conclusion

Were excited about the potential for NurOwn in AD and we look forward to the initiation of the Phase 2a trial later in 2020. We have recently made a few changes to our model, including the inclusion of NurOwn in AD and lowering of the discount rate from 20% to 15% for all indications. We model for the company to file for approval of NurOwn in AD in 2026 and to be granted approval in 2027. We currently estimate peak sales of over $2 billion for NurOwn in AD in both the U.S. and E.U. Using a 25% probability of approval leads to an NPV of $113 million. Combined with the NPV for NurOwn in ALS ($700 million) and MS ($41 million) along with the companys current cash position and potential cash from warrants leads to a valuation for the company of a bit less than $900 million. Dividing by the companys current fully diluted share count of 35.7 million leads to a valuation of $25 per share.

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BCLI: KOL Event Gives Overview of the use of NurOwn in Alzheimer's Disease; Raising Valuation to $25/Share - Zacks Small Cap Research

Bone Marrow Stem Cells Comments Off on BCLI: KOL Event Gives Overview of the use of NurOwn in Alzheimer’s Disease; Raising Valuation to $25/Share – Zacks Small Cap Research | July 10th, 2020

BOSTON, July 09, 2020 (GLOBE NEWSWIRE) -- Ziopharm Oncology, Inc. (Ziopharm or the Company) (Nasdaq:ZIOP), today announced the initiation of a phase 1 clinical trial to evaluate CD19-specific CAR-T, using its Rapid Personalized Manufacturing (RPM) technology, as an investigational treatment for patients with relapsed CD19+ leukemias and lymphomas. The trial is now open for enrollment at The University of Texas MD Anderson Cancer Center.

In this trial, the Company utilizes its non-viral Sleeping Beauty genetic engineering technology to infuse CAR-T the day after electroporation. Ziopharms RPM CD19-specific CAR-T therapy results from the stable, non-viral insertion of DNA into the genome of resting T cells to co-express the chimeric antigen receptor (CAR), membrane-bound IL-15 (mbIL15) and a safety switch.

We are pleased to expand the scope of our clinical development with MD Anderson, as we seek to evaluate our RPM technology using CD19-specific CAR-T cells, said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of Ziopharm. RPM is a promising manufacturing solution, as T cells from the bloodstream are genetically reprogramed with DNA plasmids from the Sleeping Beauty system and then simply administered the next day.

Our CAR-T therapy can be administered at low cell doses, which may control cytokine release syndrome and is appealing for the treatment of patients including those with CD19-expressing malignancies that have relapsed after allogeneic bone marrow transplantation (BMT). There are limited effective treatment options for such patients as evidenced by the low rate of remission and poor long-term survival, Dr. Cooper added.

Up to 24 patients with advanced CD19+ leukemias and lymphomas who have relapsed after allogeneic BMT will be enrolled in this investigator-initiated trial (NCT03579888). The primary endpoint of the study is to determine the safety and maximum tolerated dose of donor-derived genetically modified CD19-specific T cells manufactured using the RPM process. An additional study is planned through Ziopharms joint venture with Eden BioCell to evaluate the RPM technology using patient-derived (autologous) CD19-specific CAR-T in Greater China.

Research reveals three-year survival for adults with CD19+ acute lymphoblastic leukemia after allogeneic BMT ranges from 30% to 65%.1 For patients with other CD19+ cancers, allogeneic BMT can provide three-year survival rates between 30% to 75%.1 Few patients experience a durable remission following allogeneic BMT, regardless of the treatment modality, with some having a median survival of only 2 to 3 months.2

About Ziopharm Oncology, Inc.Ziopharm is developing non-viral and cytokine-driven cell and gene therapies that weaponize the bodys immune system to treat the millions of people globally diagnosed with a solid tumor each year. With its multiplatform approach, Ziopharm is at the forefront of immuno-oncology with a goal to treat any type of solid tumor. Ziopharms pipeline is built for commercially scalable, cost effective T-cell receptor T-cell therapies based on its non-viral Sleeping Beauty gene transfer platform, a precisely controlled IL-12 gene therapy, and rapidly manufactured Sleeping Beauty-enabled CD19-specific CAR-T program. The Company has clinical and strategic collaborations with the National Cancer Institute, The University of Texas MD Anderson Cancer Center and Regeneron Pharmaceuticals. For more information, please visit http://www.ziopharm.com.

Forward-Looking Statements DisclaimerThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as "may," "will," "could," "expects," "plans," "anticipates," and "believes." These statements include, but are not limited to, statements regarding the progress, design and timing of the Company's research and development programs, the potential benefits of the Companys therapies, and the Companys expectations regarding the number of patients in its clinical trials. Although Ziopharms management team believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Ziopharm, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, changes in our operating plans that may impact our cash expenditures, the uncertainties inherent in research and development, future clinical data and analysis, including whether any of Ziopharms product candidates will advance further in the preclinical research or clinical trial process, including receiving clearance from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies to conduct clinical trials and whether and when, if at all, they will receive final approval from the U.S. FDA or equivalent foreign regulatory agencies and for which indication; the strength and enforceability of Ziopharms intellectual property rights; competition from other pharmaceutical and biotechnology companies as well as risk factors discussed or identified in the public filings with the Securities and Exchange Commission made by Ziopharm, including those risks and uncertainties listed in Ziopharms Quarterly Report on Form 10-Q filed by Ziopharm with the Securities and Exchange Commission. We are providing this information as of the date of this press release, and Ziopharm does not undertake any obligation to update or revise the information contained in this press release whether as a result of new information, future events or any other reason.

Investor Relations Contacts:Ziopharm Oncology:Chris TaylorVP, Investor Relations and Corporate CommunicationsT: 617.502.1881E: ctaylor@ziopharm.com

LifeSci Advisors:Mike MoyerManaging DirectorT: 617.308.4306E: mmoyer@lifesciadvisors.com

Media Relations Contact:LifeSci Communications:Patrick BurseyT: 646.876.4932E: pbursey@lifescicomms.com

1 D'Souza A, Fretham C. Current Uses and Outcomes of Hematopoietic Cell Transplantation (HCT): CIBMTR Summary Slides, 2018. Available at https://www.cibmtr.org

2 Keil F, Prinz E, Kalhs P, et al. Treatment of leukemic relapse after allogeneic stem cell transplantation with cytotoreductive chemotherapy and/or immunotherapy or second transplants. Leukemia 2001; 15:355-361.

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Ziopharm Oncology Announces Initiation of Phase 1 Trial Evaluating Rapid Personalized Manufacturing CAR-T Technology in Patients with Relapsed CD19+...

Bone Marrow Stem Cells Comments Off on Ziopharm Oncology Announces Initiation of Phase 1 Trial Evaluating Rapid Personalized Manufacturing CAR-T Technology in Patients with Relapsed CD19+… | July 10th, 2020

Due to the pandemic, we have included a special section on the Impact of COVID 19 on the Hematopoietic Stem Cells Transplantation Market which would mention How the Covid-19 is affecting the Hematopoietic Stem Cells Transplantation Industry, Market Trends and Potential Opportunities in the COVID-19 Landscape, Covid-19 Impact on Key Regions and Proposal for Hematopoietic Stem Cells Transplantation Players to Combat Covid-19 Impact.

Hematopoietic stem cell transplantation (HSCT) includes the intravenous infusion of autologous or allogeneic stem cells composed from bone marrow, or umbilical cord blood, peripheral blood to reestablish hematopoietic purpose in patients whose bone marrow or immune system is dented or flawed.

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Leading Players Hematopoietic Stem Cells Transplantation Market:

Escape Therapeutics, Lonza Group, Regen BioPharma Inc, Cesca Therapeutics Inc., Cryo-Save AG, CBR Systems, Inc., Pluristem Therapeutics Inc., China Cord Blood Corporation, and ViaCord Inc.

It contains an enormous database containing numerous market segments and sub-segments. The study also provides importance on the latest daises along with the effect of certain platforms on market growth.

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Thriving Growth of Hematopoietic Stem Cells Transplantation Market Forecast 2020 with leading players Escape Therapeutics, Lonza Group, Regen...

Bone Marrow Stem Cells Comments Off on Thriving Growth of Hematopoietic Stem Cells Transplantation Market Forecast 2020 with leading players Escape Therapeutics, Lonza Group, Regen… | July 8th, 2020

LONDON, July 6, 2020 /PRNewswire/ -- IVIRMA, a global network of fertility clinics and world-leading pioneer in fertility research, are presenting a breakthrough study at the 36th Congress of the European Society of Human Reproduction and Embryology (ESHRE) today, demonstrating the possibility of 're-awakening' the ovaries in women under 40 (38 years and below) with the lowest reproductive reserve at the ovarian level.1 The ASCOT technique (involving infusion of stem cells in the ovarian artery), which has recently been shown to be successful in low-responder patients, has now shown it can achieve pregnancy in a woman with premature ovarian failure (POF).1

The study, 'Bone marrow derived stem cells restore ovarian function and fertility in premature ovarian insufficiency women. Interim report of a randomized trial: mobilization versus ovarian injection',1 which is still ongoing, includes two study arms: one using the ASCOT technique, that is, the infusion of stem cells in the ovarian artery* and, second, a less invasive option consisting of mobilising the stem cells, and allowing them to reach the ovaries through the bloodstream directly. The preliminary results have shown that ovarian follicle development was achieved in both groups, with some patients re-starting menstruation, and a decrease in menopausal symptoms. As a result of this procedure, embryos were obtained in 2 out of the 10 participants, and even one pregnancy through the ASCOT technique was achieved.

Dr. Diaz, Medical Director, IVI London, a leading fertility specialist and co-pioneer of the world's first womb transplant, commented, "We are truly excited by these very promising results achieving ovary re-awakening and pregnancy using stem cells in a woman who previously may not have had the option to conceive using her own eggs. We continually strive to pioneer on the cutting-edge of fertility research, as we know how harrowing it can be for every person struggling to conceive. These new techniques may give us potential new options for women with premature ovarian failure, in addition to those with low ovarian reserve."

It is estimated that 1 in 100 women under 40 years of age suffer from premature ovarian failure (POF) in the UK. 2.5% of all patients with POF are adolescents.2 This premature cessation of ovarian activity is one of the most challenging scenarios in terms of reproduction and can be devastating. Now, thanks to the findings of this study, led by Dr. Sonia Herraiz, researcher at the IVI Foundation-IIS la Fe, Spain and Dr. Nuria Pellicer, gynaecologist at Hospital la Fe in Valencia, Spain, there might be hope for women suffering from this fertility issue.

Dr. Nuria Pellicer, Gynaecologist, Hospital la Fe, Valencia, Spain added, "So far, we obtained embryos in 2 of the 10 patients included and one 37-week pregnancy in the ASCOT arm, in patients with almost no chance of successful pregnancy with classic in vitro fertilisation procedures. We found that both arms promoted the development of follicles, and some patients have even recovered their menstruation, thus reducing menopausal symptoms However, these are preliminary results of an ongoing study, so we remain cautious until the study is complete. We aim to develop a technique that is as minimally invasive as possible over time and standardise it so that it can be implemented in all our clinics. We would like to make it possible to offer any woman who wishes to become a mother the possibility of doing so, even when her reproductive circumstances are unfavourable."

"This is a very encouraging line of research in which we will continue to work with a single goal: to improve assisted reproduction techniques and treatments in order to obtain the best results, however difficult the reproductive prognosis may seem," concluded Dr. Herraiz, researcher at the IVI Foundation-IIS, la Fe, Spain.

More About the Study1

In addition to this research, IVI are presenting three more studies at the ESHRE Congress:

These new techniques and other research conducted by IVI is translated and applied to the treatments available in their clinics across the world, which is in turn reflected in the achieved results. The London clinic has achieved 71.4% clinical pregnancy rates per embryos transferred in women under the age of 386 and recent data shows that with PGT-A genetic screening the evolutive pregnancy rate is 57% in women undergoing treatment at IVI London as compared to the national average of 42%.7 Furthermore, 100% of these pregnancies have been achieved through single embryo transfer, eliminating chances of multiple pregnancy and the complications that arise with it.7

More about the ASCOT technique development: 3 babies and 6 pregnancies achieved so far in low-responder patients

To date, 3 babies and 6 pregnancies have been achieved using the ASCOT technique for ovarian rejuvenation in low-responder patients with low ovarian reserve, pioneered by IVIRMA Global. The technique involves transplanting bone marrow-derived stem cells (BMDSC) into the ovarian artery, achieving a partial reversal of ageing of the ovary, the organ responsible for ovulation, and activating the dormant follicles that would otherwise remain arrested in the ovary. After its first phase in animal models to test the effectiveness of the technique with stem cells, this study went to its second phase in low-responder patients. A total of 20 patients had their stem cells mobilized, extracted from peripheral blood and implanted back into the ovary in order to reverse the ageing process and activate the dormant follicles. This technique has improved ovarian function biomarkers in 81% of low responder patients. In addition, spontaneous pregnancies occurred. In view of the success of this phase, the next stage was undertaken, which consisted of recruiting women under 38 years of age, this time with early ovarian failure (a situation with a worse reproductive prognosis that of low responders). From here the above-mentioned study arose.

IVIRMA Global and IVI London, UK

IVI was founded in 1990, as the first medical institution in Spain fully dedicated to Assisted Reproduction. Since then it has helped with the birth of more than 200,000 babies thanks to the application of the latest Assisted Reproduction technologies. In early 2017, IVI merged with RMANJ, becoming the largest Assisted Reproduction group in the world. It currently has more than 65 clinics in 9 countries and is the leading centre for Reproductive Medicine. In 2016 IVI opened its doors in London, located in the heart of the medical district.www.rmanetwork.comhttps://ivi-fertility.co.uk/

References

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Breakthrough study demonstrates the 're-awakening' of the ovaries and achieves pregnancy in woman with premature ovarian failure using stem cells -...

Bone Marrow Stem Cells Comments Off on Breakthrough study demonstrates the ‘re-awakening’ of the ovaries and achieves pregnancy in woman with premature ovarian failure using stem cells -… | July 8th, 2020

One cancer survivor looks at all the ways they might have gotten cancer, and wonders if any of those risk factors will factor into recurrence.

William Ramshaw resides in the expansive Pacific Northwest. He is a six-year survivor of pancreatic cancer and has written a memoir Gut Punched! Facing Pancreatic Cancer.

So, what would someone do to get cancer?

Use tobacco. Both my parents smoked packs a day. My brother chews. One of my best friends chain-smoked. I used to tell him, Dennis, this is going to kill you. He laughed. It did. Lost to esophageal cancer. Given those ominous Surgeon Generals Warnings, all caps and bolded, along with horrendous TV infomercials, I am mystified why people still use tobacco, but they do. Nicotine addiction is an awful thing.

Being overweight. Ok, this one is closer to home. Before getting pancreatic cancer, I had been a big guy. Not blimp-sized mind you but carrying pounds I didnt need. Post-surgery, I lost a third of my body weight, about 100 pounds. I have no butt. Yes, I can now wear those skinny jeans. Also, due to being replumbed, I cant even gain weight. Still, I wouldnt recommend my weight loss program to anyone. Its barbaric.

Getting too much sun. Growing up I plowed our orchard in my cutoffs with no shirt. By the end of summer, I was toasted brown. Looking back, I hope the layer of dirt shielded me from getting too many rays. Excess UV light is known to cause skin cancer. So far so good. I dont have any skin lesions yet.

Exposure to bad stuff. It seems everything I buy anymore comes with a warning such-and-such may cause cancer. I do pay attention but find myself unable to avoid exposure to everything that might be bad for me. At this point, I think breathing causes cancer. Should I stop breathing?

Breathing asbestos. Im a goner here too. As a former Navy-guy, I worked down in the boiler room. Hot as hell right there among gigantic steam pipes all encased in asbestos. Thankfully, so far, I have nothing other than a persistent cough and no lung cancer.

Worse draw an inside straight. (Also known as a gutshot or belly buster draw, where you have a straight but are missing the card in the middle.) While I dont play poker, getting cancer is a lot like drawing an inside straight. Our genes get messed up. Maybe we got them from a long-dead ancestor. One clicks over and we find ourselves with a losing cancer hand. Im not sure what happened in my case. My tumor was not genetically profiled. I wish it had been so I would better understand my odds at this point. Will I get another shot at a losing hand?

I know this in jest as no one tries or wants to get cancer. But its interesting to think about everything that causes cancer. Sadly, there are dozens of things. Cancer aside, I am indeed fortunate (and thankful) to be here.

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Seven 'Sure' Ways to Get Cancer - Curetoday.com

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CAR-T treatments and other immunotherapies have changed the treatment of some blood cancers, but they can target healthy cells as well as cancer cells, causing nasty side effects. Vor Biopharma is working on an engineered stem cell solution, and its raised $110 million to move its lead program into the clinic.

The Cambridge, Massachusetts-based company is developing the treatment, VOR33, for patients with acute myeloid leukemia (AML) whose disease has worsened despite undergoing chemotherapy or a stem cell transplant.

Thats the setting in which a lot of targeted agents are used. The trouble is, a great number of the targeted agents tend to failnot because they are not efficacious, but because the drug is too toxic for the patients bone marrow, Vor CEO Robert Ang told Fierce Biotech.

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The drugs home in on healthy cells as well as cancerous ones because they express the same proteins. This leads to myelosuppression, which means the bone marrow doesnt make enough white blood cells, platelets or red blood cells for the patient to survive, Ang said.

RELATED: Neon CBO Robert Ang jumps ship to take the helm at Vor Biopharma

Vors treatment is made from hematopoietic, or blood-forming, stem cells from healthy donors. The company uses gene editing to get rid of cancer drug targets in those cells that are biologically redundant, which means deleting them doesnt cause any harm. That target is CD33, in the case of VOR33.

Were trying to make the marrow treatment resistant such that the only cells that are expressing CD33 should be cancer cells, Ang said. We should be able to target them much more specifically, while minimizing the collateral damage that typically happens with these drugs.

RELATED: 5AM, JJDC get in on $42M series A round for cell therapy player Vor Biopharma

Vor believes its treatments could boost the reach of targeted therapies by improving their efficacy and increasing the amount of time patients can undergo those treatments.

And thats not all. In addition to protecting these transplants and the blood cells they produce from targeted drugs, Vor thinks its approach could change the way we think about bone marrow transplant.

To some degree, transplants have been relatively decentralized and less controlled A lot of hospitals develop their own unique practices as to what they think works and how to handle cells and process them, Ang said. Our product will be regulated by the FDA, so we will be able to provide controls and the proper manufacturing steps to ensure were making the best quality product for patients.

The series B will push VOR33 into clinical trials in the first half of 2021, a target the companys on track to meet despite the COVID-19 pandemic. And Vor plans to expand its portfolio beyond CD33, starting with an umbrella of targets in the myeloid space, namely in acute myeloid leukemia, myelodysplastic syndromes and related diseases.

But we are also looking beyond that to other cancers where there are similar potentially biologically redundant targets we could pursue, said Ang, who took the companys helm in August 2019.

Since then, Vor has grown from a staff of six to 50, and its about to move into new digs in west Cambridge as it moves VOR33 toward the clinic. Its got the backing of RA Capital Management, Fidelity, 5AM Ventures, Johnson & Johnson Innovation, Osage University Partners, PureTech Health, the Pagliuca Family Office and Alexandria Venture Investments to do it all.

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Vor bags $110M to move engineered cell therapy into the clinic - FierceBiotech

Bone Marrow Stem Cells Comments Off on Vor bags $110M to move engineered cell therapy into the clinic – FierceBiotech | July 7th, 2020

Hematopoietic Stem Cells Transplantation (HSCT) Marketreport provides in-depth COVID19 impact analysis ofMarket Overview, Product Scope, Market Drivers, Trends, Opportunities,Market Driving Force and Market Risks. It also profile the topmost prime manufacturers (Kite Pharma, Thermo Fisher Scientific, CellGenix Technologie Transfer, Cesca Therapeutics, R&D Systems) are analyzed emphatically by competitive landscape contrast, with respect toPrice, Sales,Capacity, Import, Export, Consumption, Gross, Gross Margin, Revenue and Market Share. Hematopoietic Stem Cells Transplantation (HSCT) industry breakdown data are shown at the regional level, to show the sales, revenue and growth by regions.Hematopoietic Stem Cells Transplantation (HSCT) Market describe Hematopoietic Stem Cells Transplantation (HSCT) Sales Channel,Distributors, Customers, Research Findings and Conclusion, Appendix and Data Source.

Key Target Audience of Hematopoietic Stem Cells Transplantation (HSCT) Market:Manufacturers of Hematopoietic Stem Cells Transplantation (HSCT), Raw material suppliers, Market research and consulting firms, Government bodies such as regulating authorities and policy makers, Organizations, forums and alliances related to Hematopoietic Stem Cells Transplantation (HSCT) market.

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In-Depth Qualitative Analyses Include Identification and Investigation Of The Following Aspects:Hematopoietic Stem Cells Transplantation (HSCT) Market Structure, Growth Drivers, Restraints and Challenges, Emerging Product Trends & Market Opportunities, Porters Fiver Forces.

Summary of Hematopoietic Stem Cells Transplantation (HSCT) Market:Hematopoietic stem cell transplants (HSCT) present to a valid treatment for several congenital and other hematopoietic system disorders, post chemotherapy, and immune sensitive diseases. HSCT is also preferred for replacement of cellular components and deficient cells. The indications for HSCT thus are wide; the most frequent indication as per reported by Worldwide Network for Blood and Marrow Transplantation Group (WNBT) (2013) is lymphoproliferative disorder (53.2% of all HSCT), 12% of whom received allogeneic and the rest received autologous transplant. Plasma cell disorders are the most frequent indication in this group. A multitude of literature published by researchers and organizations demonstrate that autologous transplant own a greater edge against allogeneic HSCT.

Over 30 years of studies in the field of blood-forming stem cells i.e. hematopoietic stem cells (HSC), researchers have developed significant understanding to use HSCs as a therapy. At present, no type of stem cell, adult, embryonic or fetal has attained such sufficient status. Hematopoietic stem cell transplantation (HSCT) is now routinely used for treating patients with malignant and non-malignant disorders of blood and the immune system. Currently, researchers have observed that through animal studies HSCs have the ability to form other cells such as blood vessels, muscles, and bone. Further application of this approach it may eventually be able to treat a wide array of conditions and replace ailing tissues. However, despite the vast experience with HSCs, researchers face major barriers in expanding their use beyond the replacement of immune and blood cells.

Hematopoietic stem cells are unable to proliferate and differentiate in-vitro. Researchers have yet to evolve an accurate method to differentiate stem cells from other cells derived from blood or bone marrow. Once such technical barriers are overcome, the avenues for realizing the full potential of HSCT. The type of transplant a person receives depends on several different factors, including the type and course of the disease, availability of suitable donors, and the patients overall health. There are three different sources of hematopoietic stem cells such as bone marrow, peripheral blood stem cells, and umbilical cord blood. The stem cell source used for a given transplant depends upon the underlying disease, the type of transplant (allogeneic or autologous), and size of the patient.

On the basis on the end users/applications,this report focuses on the status and outlook for major applications/end users, sales volume, market share and growth rate of Hematopoietic Stem Cells Transplantation (HSCT) market foreach application, including-

Leukemia Lymphoproliferative Disorders Solid Tumors Non-Malignant Disorders Others

On the basis of product,this report displays the sales volume, revenue (Million USD), product price, market share and growth rate ofeach type, primarily split into-

Autologous Transplant Allogenic Transplant

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Hematopoietic Stem Cells Transplantation (HSCT) Market: Future Innovation Ways That, Growth & Profit Analysis, Forecast By 2026 - 3rd Watch News

Bone Marrow Stem Cells Comments Off on Hematopoietic Stem Cells Transplantation (HSCT) Market: Future Innovation Ways That, Growth & Profit Analysis, Forecast By 2026 – 3rd Watch News | July 7th, 2020

Patient dosing has finished in the pivotal Phase 3 trial assessing the safety and efficacy of repeat administrations of NurOwn, acell-based therapy forpeople with amyotrophic lateral sclerosis (ALS), BrainStorm Cell Therapeutics, the therapys developer, announced.

The Phase 3 trial (NCT03280056), which enrolled about 200 participants, is underway at six U.S. sites: the University of California, Irvine;Cedars-Sinai Medical Center;California Pacific Medical Center;Massachusetts General Hospital; the University of Massachusetts Medical School; and the Mayo Clinic.

Patients were randomly assigned to either three doses of NurOwn or a placebo, both administered every other month by injections directly into the spinal canal (intrathecal) over a period of four months.

The studys main goal is to assess the safety and efficacy of NurOwn, as measured by changes in the amyotrophic lateral sclerosis functional rating scale (ALSFRS-R) score seen at 28 weeks (seven months) following the first treatment. ALSFRS-R assess such daily life abilities as swallowing, speaking, dressing and washing oneself, climbing stairs, and turning over in bed.

Secondary goals include treatment effects on the levels of several disease biomarkers, as found in samples of patients blood and cerebrospinal fluid (the liquid circulating in the brain and spinal cord).

BrainStorm expects top-line trial data to be available before the end of the year.

Completion of participant dosing in this clinical trial is an important milestone and brings us a step closer to potentially filing a biologics license application to make MSC-NTF cells [NurOwn] available to people with ALS, Chaim Lebovits, CEO of BrainStorm, said in the companyspress release.

NurOwn is a cell-based therapy that usesmesenchymal stem cells (MSCs) cells that are able to give rise to nearly all tissues found in the body, including bones, muscles, and connective tissue isolated from a patients own bone marrow.

After isolation, MSCs are grown in a lab dish and differentiated into cells that produce high levels of neurotrophic factors compounds that promote the growth and survival of nerve cells. The modified cells are then returned to patients through an injection into spinal canal.

Brainstorm is also investigating the potential of NurOwn treat other neurological disorders, including multiple sclerosis (MS), Huntingtons disease, Parkinsons disease, and autism spectrum disorder.

The open-label Phase 2 trial (NCT03799718) assessing three doses of NurOwn in people with progressive MSmay still be recruitingat two sites in the U.S. The cell-based therapy has not yet entered clinical testing for other disorders.

Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells cells that made up the lining of blood vessels found in the umbilical cord of newborns.

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Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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200 ALS Patients Finish Dosing in Phase 3 Trial of NurOwn Cell Therapy - ALS News Today

Bone Marrow Stem Cells Comments Off on 200 ALS Patients Finish Dosing in Phase 3 Trial of NurOwn Cell Therapy – ALS News Today | July 7th, 2020

ONE simple act could save this little girl's life.

Little Shahera Khan has a fault with her immune system that means her body can't produce enough white blood cells.

3

It means the toddler is susceptible to infections, meaning a seemingly innocuous bug could kill her.

Shahera's life is very different to those of other tot's her age.

She can't go to nursery, make friends or really interact with too many other kids, for fear of picking up infections.

While she loves to play with her big brother, Amaan, five, she has to take daily medication and has blood transfusions each week.

If she picks up a common cold or cough, it can leave her in hospital for days while her tiny body fights the bacteria or virus.

Now, her parents are pleading for help from strangers, to offer their little girl a chance at a normal life.

Shahera's only hope of growing up like her peers is a stem cell transplant.

Speaking to The Sun her Mum Amina said she just wants her daughter to get better and to lead a normal life.

Shahera was born a healthy child and she was okay when she was small.

She picked up bronchiolitis when she was eight months old and had to be hospitalised. We were there for a week and she also picked up an ear and nose infection.

A lot of people say she has been through the wars and she has, she hasnt had an easy childhood and theres always something new with her illness

Amina, who lives in Croydon with Shahera, her husband and Sharehas little brother said that she had Shahera at home for just a couple of days before she had to go back to hospital due to coughing fits.

She recovered at home but within two weeks the family were sent a letter that detailed she had low immunity.

Amina said that little Shahera had a rash on her face and said doctors then had to do a biopsy.

They were scratching their heads really. After a biopsy they found that she was an unusual case.

3

Her skin was pretty bad at that point and they gave her steroids and antibiotics.

At the Evelina hospital they ran further tests on Shahera, but sadly she then had to be hospitalised for bacterial sepsis.

Amina said that all of this happened during the 2018 World Cup and said she remembers it so well as temperatures were soaring and she was trying to control little Shaheras temperature.

She was so thirsty and her temperature was 38/39C.

How to join the stem cell register

Little Shahera needs a stem cell donor - here's how you can register

You can join the stem cell register online. All you need to do is fill out a registration form and you will be sent out some swabs.

You will need to take some samples, usually from the inside of your mouth and then send them back. As soon as they are received you'll be added to the register.

You'll remain on the register until you are 61-years-old.

If you are a match for someone in need charities such as Anthony Nolan will help guide you through the process.

Around 90 per cent of people are able to donate via their blood stream with 10 per cent donating from bone marrow while under general anaesthetic.

To find out more and to register you can visit the below sites:

Then the hospital found that one of her white blood cells was completely wiped out, she was very sick and it was at that point that the doctors mentioned a bone marrow transplant.

Once again Shahera was allowed to return home and she made a good recovery.

Then when she was just one her rash appeared again and more tests found that she had a rare immunodeficiency.

Shahera now has antibody immunoglobulin treatments every week and Amina said this makes a big difference to her quality of life.

3

The issue now Amina says, is that doctors need to find a way to continue to treat Shahera so she can do all the things that other little girls can.

Doctors have given the family several months to find a donor and have said the transplant could take place as early as next spring.

Finding a donor though will be a struggle for the family as just 20 per cent of patients from BAME backgrounds find the best possible donor match.

This is in comparison to 69 per cent for people with white European heritage.

Finding a donor would be a big relief for us, because hopefully it will maker her better

Shahera is of Bangladeshi origin and Amina said its difficult for the family to find someone from their community.

We are part of her journey and it has been difficult but we have been very patient and we are still working with consultants and professors.

At the moment she is doing as well as she can be and she has been shielding due to Covid-19.

Finding a donor would be a big relief for us, because hopefully it will maker her better.

Amina said that the family are not only campaigning to find a donor for Shahera but for others in the same situation.

What is immunoglobulin therapy?

Little Shahera has to have antibody immunoglobulin treatments to keep her well

It is a blood-based treatment that contains antibodies in order to fight against infections.

People are given this treatment when their immune systems do not produce enough antibodies.

Some patients are at a higher risk than others.

Charity PID UK states: "Clinical trials have shown that for people with immune deficiency, immunoglobulin treatments result in fewer infections, and those infections that do occur tend to be less serious.

"There is also evidence that people with immune deficiency are more likely to enjoy good health over many years if they receive immunoglobulin correctly. Finally, your wellbeing and your energy levels are likely to be better if you are on immunoglobulin.

"It may take several months before you feel these benefits.

Everything has been put on hold because of Covid but donating is such a simple process.

A lot of people have misconceptions about stem cell donation, but donors can save lives.

Due to the coronavirus lockdown little Shahera has spent lots of quality time with her family.

Amina said she is doing well and has enjoyed spending even more time with her older brother.

She likes to play cops and robbers and fireman Sam but she also enjoys doing girly things too like looking after her dolls.

Now that Shahera is a little bit older, Amina said she is more aware of her rash and will sometimes look at it and point.

She is very aware of the transfusions and understands what is happening and she is very well known in the ward.

She doesnt really cry when they prepare her for transfusions.

A lot of people say she has been through the wars and she has, she hasnt had an easy childhood and theres always something new with her illness.

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But shes a tough little girl and she is doing really well.

Sarah Rogers, register development manager at Anthony Nolan says: Little Shahera wants to go to nursery, learn and make more friends just like other toddlers olds across the UK. For this to happen she needs a stranger to donate their stem cells.

"If youre aged 16-30 you can join the register online and well send you a cheek swab in the post.

"If youre found to be a match for a patient, you could donate your stem cells and give hope to families like Shaheras. Your support could help us give a patient, their family and their friends a second chance of life."

Continued here:
One common infection could kill our baby girl but one simple act could change her life forever - The Sun

Bone Marrow Stem Cells Comments Off on One common infection could kill our baby girl but one simple act could change her life forever – The Sun | July 7th, 2020

Myelofibrosis or osteomyelofibrosis is a myeloproliferative disorder which is characterized by proliferation of abnormal clone of hematopoietic stem cells. Myelofibrosis is a rare type of chronic leukemia which affects the blood forming function of the bone marrow tissue. National Institute of Health (NIH) has listed it as a rare disease as the prevalence of myelofibrosis in UK is as low as 0.5 cases per 100,000 population. The cause of myelofibrosis is the genetic mutation in bone marrow stem cells. The disorder is found to occur mainly in the people of age 50 or more and shows no symptoms at an early stage. The common symptoms associated with myelofibrosis include weakness, fatigue, anemia, splenomegaly (spleen enlargement) and gout. However, the disease progresses very slowly and 10% of the patients eventually develop acute myeloid leukemia. Treatment options for myelofibrosis are mainly to prevent the complications associated with low blood count and splenomegaly.

To remain ahead of your competitors, request for a sample [emailprotected] https://www.persistencemarketresearch.com/samples/11341

The global market for myelofibrosis treatment is expected to grow moderately due to low incidence of a disease. However, increasing incidence of genetic disorders, lifestyle up-gradation and rise in smoking population are the factors which can boost the growth of global myelofibrosis treatment market. The high cost of therapy will the growth of global myelofibrosis treatment market.

The global market for myelofibrosis treatment is segmented on basis of treatment type, end user and geography:

To receive Methodology request here@ https://www.persistencemarketresearch.com/methodology/11341

As myelofibrosis is considered as non-curable disease treatment options mainly depend on visible symptoms of a disease. Primary stages of the myelofibrosis are treated with supportive therapies such as chemotherapy and radiation therapy. However, there are serious unmet needs in myelofibrosis treatment market due to lack of disease modifying agents. Approval of JAK1/JAK2 inhibitor Ruxolitinib in 2011 is considered as a breakthrough in myelofibrosis treatment. Stem cell transplantation for the treatment of myelofibrosis also holds tremendous potential for market growth but high cost of therapy is foreseen to limits the growth of the segment.

On the basis of treatment type, the global myelofibrosis treatment market has been segmented into blood transfusion, chemotherapy, androgen therapy and stem cell or bone marrow transplantation. Chemotherapy segment is expected to contribute major share due to easy availability of chemotherapeutic agents. Ruxolitinib is the only chemotherapeutic agent approved by the USFDA specifically for the treatment of myelofibrosis, which will drive the global myelofibrosis treatment market over the forecast period.

Geographically, global myelofibrosis treatment market is segmented into five regions viz. North America, Latin America, Europe, Asia Pacific and Middle East & Africa. Northe America is anticipated to lead the global myelofibrosis treatment market due to comparatively high prevalence of the disease in the region.

To receive extensive list of important regions, Request TOC here @ https://www.persistencemarketresearch.com/toc/11341

Some of the key market players in the global myelofibrosis treatment market are

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Growing Demand for Myelofibrosis Treatment Market to Significantly Increase Revenues Through 2022 - Jewish Life News

Bone Marrow Stem Cells Comments Off on Growing Demand for Myelofibrosis Treatment Market to Significantly Increase Revenues Through 2022 – Jewish Life News | July 6th, 2020

LONDON, July 6,2020 /PRNewswire/ --IVIRMA, a global network of fertility clinics and world-leading pioneer in fertility research, are presenting a breakthrough study at the 36th Congress of the European Society of Human Reproduction and Embryology (ESHRE) today, demonstrating the possibility of 're-awakening' the ovaries in women under 40 (38 years and below) with the lowest reproductive reserve at the ovarian level.1 The ASCOT technique (involving infusion of stem cells in the ovarian artery), which has recently been shown to be successful in low-responder patients, has now shown it can achieve pregnancy in a woman with premature ovarian failure (POF).1

The study, 'Bone marrow derived stem cells restore ovarian function and fertility in premature ovarian insufficiency women. Interim report of a randomized trial: mobilization versus ovarian injection',1 which is still ongoing, includes two study arms: one using the ASCOT technique, that is, the infusion of stem cells in the ovarian artery* and, second, a less invasive option consisting of mobilising the stem cells, and allowing them to reach the ovaries through the bloodstream directly. The preliminary results have shown that ovarian follicle development was achieved in both groups, with some patients re-starting menstruation, and a decrease in menopausal symptoms. As a result of this procedure, embryos were obtained in 2 out of the 10 participants, and even one pregnancy through the ASCOT technique was achieved.

Dr. Diaz, Medical Director, IVI London, a leading fertility specialist and co-pioneer of the world's first womb transplant, commented, "We are truly excited by these very promising results achieving ovary re-awakening and pregnancy using stem cells in a woman who previously may not have had the option to conceive using her own eggs. We continually strive to pioneer on the cutting-edge of fertility research, as we know how harrowing it can be for every person struggling to conceive. These new techniques may give us potential new options for women with premature ovarian failure, in addition to those with low ovarian reserve."

It is estimated that 1 in 100 women under 40 years of age suffer from premature ovarian failure (POF) in the UK. 2.5% of all patients with POF are adolescents.2 This premature cessation of ovarian activity is one of the most challenging scenarios in terms of reproduction and can be devastating. Now, thanks to the findings of this study, led by Dr. Sonia Herraiz, researcher at the IVI Foundation-IIS la Fe, Spain and Dr. Nuria Pellicer, gynaecologist at Hospital la Fe in Valencia, Spain, there might be hope for women suffering from this fertility issue.

Dr. Nuria Pellicer, Gynaecologist, Hospital la Fe, Valencia, Spain added, "So far, we obtained embryos in 2 of the 10 patients included and one 37-week pregnancy in the ASCOT arm, in patients with almost no chance of successful pregnancy with classic in vitro fertilisation procedures. We found that both arms promoted the development of follicles, and some patients have even recovered their menstruation, thus reducing menopausal symptoms However, these are preliminary results of an ongoing study, so we remain cautious until the study is complete. We aim to develop a technique that is as minimally invasive as possible over time and standardise it so that it can be implemented in all our clinics. We would like to make it possible to offer any woman who wishes to become a mother the possibility of doing so, even when her reproductive circumstances are unfavourable."

"This is a very encouraging line of research in which we will continue to work with a single goal: to improve assisted reproduction techniques and treatments in order to obtain the best results, however difficult the reproductive prognosis may seem,"concluded Dr. Herraiz, researcher at the IVI Foundation-IIS, la Fe, Spain.

More About the Study1

In addition to this research, IVI are presenting three more studies at the ESHRE Congress:

These new techniques and other research conducted by IVI is translated and applied to the treatments available in their clinics across the world, which is in turn reflected in the achieved results. The London clinic has achieved 71.4% clinical pregnancy rates per embryos transferred in women under the age of 386 and recent data shows that with PGT-A genetic screening the evolutive pregnancy rate is 57% in women undergoing treatment at IVI London as compared to the national average of 42%.7 Furthermore, 100% of these pregnancies have been achieved through single embryo transfer, eliminating chances of multiple pregnancy and the complications that arise with it.7

More about the ASCOT technique development: 3 babies and 6 pregnancies achieved so far in low-responder patients

To date, 3 babies and 6 pregnancies have been achieved using the ASCOT technique for ovarian rejuvenation in low-responder patients with low ovarian reserve, pioneered by IVIRMA Global. The technique involves transplanting bone marrow-derived stem cells (BMDSC) into the ovarian artery, achieving a partial reversal of ageing of the ovary, the organ responsible for ovulation, and activating the dormant follicles that would otherwise remain arrested in the ovary. After its first phase in animal models to test the effectiveness of the technique with stem cells, this study went to its second phase in low-responder patients. A total of 20 patients had their stem cells mobilized, extracted from peripheral blood and implanted back into the ovary in order to reverse the ageing process and activate the dormant follicles. This technique has improved ovarian function biomarkers in 81% of low responder patients. In addition, spontaneous pregnancies occurred. In view of the success of this phase, the next stage was undertaken, which consisted of recruiting women under 38 years of age, this time with early ovarian failure (a situation with a worse reproductive prognosis that of low responders). From here the above-mentioned study arose.

IVIRMA Global and IVI London, UK

IVI was founded in 1990, as the first medical institution in Spain fully dedicated to Assisted Reproduction. Since then it has helped with the birth of more than 200,000 babies thanks to the application of the latest Assisted Reproduction technologies. In early 2017, IVI merged with RMANJ, becoming the largest Assisted Reproduction group in the world. It currently has more than 65 clinics in 9 countries and is the leading centre for Reproductive Medicine. In 2016 IVI opened its doors in London, located in the heart of the medical district.www.rmanetwork.com https://ivi-fertility.co.uk/

References

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Breakthrough study demonstrates the 're-awakening' of the ovaries and achieves pregnancy in woman with premature ovarian failure using stem cells - PR...

Bone Marrow Stem Cells Comments Off on Breakthrough study demonstrates the ‘re-awakening’ of the ovaries and achieves pregnancy in woman with premature ovarian failure using stem cells – PR… | July 6th, 2020

Poseida Therapeutics, which is developing targeted treatments for blood-based and other cancers, set the terms for its initial public stock offering expected this week, where it is seeking to raise $150 million.

The San Diego-based company has one drug advancing to Phase 2 clinical trials for multiple myeloma, a type of cancer that develops in certain cells in the bone marrow.

The company expects to file for investigational new drug authorization with the U.S. Food and Drug Administration for its myeloma treatment later this year.

It also is developing other cancer therapies, including treatments for solid tumors and prostate cancer.

Poseida Therapeutics looks to become the third San Diego life science company to go public in the past two months, joining Progenity and Avidity Biosciences. Its stock offering highlights that investor appetite for new biotech companies remains strong in the midst of stock market turbulence from the coronavirus pandemic.

At the end of March, the company employed 149 workers, including 86 with advanced college degrees.

Since its inception, the company has raised $334 million from life sciences institutional investors who support its drug development efforts, including $75 million from Swiss pharmaceutical giant Novartis.

Poseida set the price for its initial public stock offering at $14 to $16 per share. It plans to trade under the ticker symbol PSTX on the Nasdaq exchange. If its shares get out at the midpoint of that range, Poseida Therapeutics would achieve a market value of $890 million.

Founded in 2015, Poseida Therapeutics is making its own version of what are called CAR T cells immune cells genetically engineered to fight cancers. It is developing CAR T cell treatments made from cancer patients own cells.

The process produces stem cell memory T cells. These T cells stick around, both replenishing their population and creating more differentiated group of T cells to attack cancers. They can potentially resume activity if the cancer recurs. This persistence makes it possible to produce the activity needed to eradicate tumors, which up to this point has been beyond the reach of this type of therapy.

The company has its roots in Transposagen Biopharmaceuticals, which founder Eric Ostertag ran for 13 years. Poseida was spun out to pursue gene engineering technologies.

Other members of the companys management teams have worked in executive roles at San Diego biotech firms including Amylin Pharmaceuticals, Halozyme Therapeutics and Mirati Therapeutics.

In addition to Novartis, additional investors include Adage Capital, Aisling Capital, Boxer Capital, Fidelity, Longitude Capital, Malin Corp., Millennium Capital, Pentwater Capital, Perceptive Advisors, Schonfeld and Vivo Capital.

Bank of America Securities, Piper Sandler and William Blair are joint underwriters on the initial public stock offering.

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Poseida Therapeutics sets terms for upcoming IPO, aims to raise $150M for cancer treatments - The San Diego Union-Tribune

Bone Marrow Stem Cells Comments Off on Poseida Therapeutics sets terms for upcoming IPO, aims to raise $150M for cancer treatments – The San Diego Union-Tribune | July 6th, 2020

This Agreement will allow for immediate distribution of leronlimab to patients for the treatment of COVID-19 upon successful completion of CytoDyn’s ongoing clinical trials and FDA approval

VANCOUVER, Washington, July 06, 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 it has signed an exclusive Distribution and Supply Agreement with American Regent, Inc. (American Regent”) for the distribution of leronlimab for the treatment of COVID-19 in the United States.

Under the terms of the agreement, CytoDyn will supply leronlimab for the treatment of COVID-19 for distribution by American Regent and receive quarterly payments based on a profit-sharing arrangement.

Having this distribution agreement in place ahead of the readout from CytoDyn’s COVID-19 clinical trials further emphasizes CytoDyn’s commitment to making leronlimab immediately available to patients based on the successful completion of its ongoing clinical trials,” said Nader Pourhassan, Ph.D., President and Chief Executive Officer of CytoDyn. We are particularly happy to be partnering with a company with the proven expertise, unparalleled commercial reach and stellar reputation of American Regent.”

American Regent is looking forward to partnering with CytoDyn to provide COVID-19 patients rapid and efficient access to a potentially life-saving drug,” said Mr. Harsher Singh, American Regent’s Vice President and Chief Commercial Officer.

CytoDyn is currently enrolling a Phase 2b/3 clinical trial for 390 severe and critically ill COVID-19 patients, which is a randomized, placebo-controlled with 2:1 ratio (active drug to placebo ratio). The Company has also completed its enrollment of a Phase 2 randomized clinical trial with 75 patients in the mild-to-moderate COVID-19 population. CytoDyn has been granted more than sixty emergency Investigational New Drug (eIND) authorizations by the U.S. Food and Drug Administration (FDA) and plans to provide clinical updates for this patient population in the coming weeks.

About American Regent American Regent, Inc., a Daiichi Sankyo Group company, is a top-10 injectable manufacturer. For over 50 years, American Regent has been developing, manufacturing and supplying quality generic and branded injectables for healthcare providers. For nearly 20 years, American Regent have been a leader in IV iron therapy. American Regent is committed to U.S.-based manufacturing. In 2018, more than 99% of units supplied were manufactured in its U.S.-based facilities making it uniquely positioned to quickly mobilize and respond to shortages or changes in market needs. Speed counts. Flexibility matters. Reliability and quality are paramount. Because patients should never have to wait for the medications they need. For more information, please visit http://www.americanregent.com.

About Coronavirus Disease 2019 CytoDyn has met its 75-patient enrollment target in its Phase 2 clinical trial for COVID-19, a randomized clinical trial for mild-to-moderate COVID-19 population in the U.S. and enrollment continues in its Phase 2b/3 randomized clinical trial for severe and critically ill COVID-19 population in several hospitals throughout the country.

SARS-CoV-2 was identified as the cause of an outbreak of respiratory illness first detected in Wuhan, China. The origin of SARS-CoV-2 causing the COVID-19 disease is uncertain, and the virus is highly contagious. COVID-19 typically transmits person to person through respiratory droplets, commonly resulting from coughing, sneezing, and close personal contact. Coronaviruses are a large family of viruses, some causing illness in people and others that circulate among animals. For confirmed COVID-19 infections, symptoms have included fever, cough, and shortness of breath. The symptoms of COVID-19 may appear in as few as two days or as long as 14 days after exposure. Clinical manifestations in patients have ranged from non-existent to severe and fatal. At this time, there are minimal treatment options for COVID-19.

About Leronlimab (PRO 140) and BLA Submission for the HIV Combination Therapy The FDA has 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 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 could 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.

The Company filed its BLA for Leronlimab as a Combination Therapy for Highly Treatment Experienced HIV Patients with the FDA on April 27, 2020, and submitted additional FDA requested clinical datasets on May 11, 2020. After the FDA deems a BLA submission complete, it sets a PDUFA goal date. CytoDyn has Fast Track designation for leronlimab and a rolling review for its BLA, as previously assigned by the FDA. The Company filed a request for Priority Review designation for its BLA to shorten the FDA’s review time from 10 to 6 months, an FDA goal for BLA applications given Priority Review designation.

In the setting of cancer, research has shown that CCR5 may play a role in tumor invasion, metastases, and tumor microenvironment control. 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.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial 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 support further the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD, blocking the CCR5 receptor from recognizing specific 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 late-stage 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 critical 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 immune-mediated illnesses, such as GvHD and NASH. CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. CytoDyn filed its BLA in April 2020 to seek FDA approval for leronlimab as a combination therapy for highly treatment experienced HIV patients, and submitted additional FDA requested clinical datasets on May 11, 2020. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication. If successful, it 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. No drug-related serious site injection reactions reported in about 800 patients treated with leronlimab and no drug-related SAEs reported in patients treated with 700 mg dose of leronlimab. Moreover, a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients; some patients on leronlimab monotherapy have remained 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. Forward-looking statements specifically include statements about leronlimab, its ability to have positive health outcomes, the possible results of clinical trials, studies or other programs or ability to continue those programs, the ability to obtain regulatory approval for commercial sales, and the market for actual commercial sales. The Company’s 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 Company’s cash position, (ii) the Company’s ability to raise additional capital to fund its operations, (iii) the Company’s ability to meet its debt obligations, if any, (iv) the Company’s ability to enter into partnership or licensing arrangements with third parties, (v) the Company’s ability to identify patients to enroll in its clinical trials in a timely fashion, (vi) the Company’s ability to achieve approval of a marketable product, (vii) the design, implementation and conduct of the Company’s clinical trials, (viii) the results of the Company’s 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 Company’s 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 Company’s 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: Cristina De Leon Office: 360.980.8524, ext. 106 Mobile: 503.214.0872 cdeleon@cytodyn.com

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CytoDyn Announces Execution of Exclusive Agreement with American Regent for Distribution and Supply of Leronlimab for Treatment of COVID-19 in United...

Bone Marrow Stem Cells Comments Off on CytoDyn Announces Execution of Exclusive Agreement with American Regent for Distribution and Supply of Leronlimab for Treatment of COVID-19 in United… | July 6th, 2020

The worlds biggest listed pure-play stem cell developer has a busy slate of clinical work, notably in therapies for advanced heart failure, chronic back pain and graft-versus-host disease (GvHD).

Now these programs are approaching a thrilling denouement and, as the Demtel man enthused, theres more: Mesoblast (ASX:MSB) is also undertaking an expanded coronavirus trial after a 12-patient effort showed promising results in treating acute respiratory distress syndrome (Ards), the usual cause of death with COVID-19.

The patients received infusions of Mesoblasts allogeneic (off the shelf) mesenchymal stem cell candidate, remestemcel-L, acquired from Osiris for $106m in 2013.

Meanwhile, results from two phase III trials are expected this (September) quarter: a 566-patient effort for chronic heart failure and a 404-patient trial for chronic lower back pain caused by disc degeneration.

And in September, the US Food and Drug Administration will rule on whether or not the company can market its GvHD therapy on American shores.

Mesoblast founder and CEO Prof Silviu Itescu notes that across all its therapies the company is targeting the most severe cases where alternative therapies dont exist.

Mesoblasts proprietary process selects precursor and stem cells from the bone marrow of healthy adults, creating a master cell bank. This cell kitty is then expanded into thousands of doses for off-the-shelf use, without the need for tissue matching.

Mesoblast is targeting a common market across all its disease indications: inflammation. In the case of heart disease, tissue macrophages (cells) churn out inflammatory factors that damage heart muscle and cause fibrosis and vascular dysfunction.

The stem cells respond to severe inflammation by switching the culprit macrophages off and converting them to nice cells that actually protect the heart muscle.

This is the central mechanism in each of our disease states: heart failure, back pain, GvHD and rheumatoid arthritis, Professor Itescu says. We have the potential to make a big difference in some very big disease states where inflammation is central.

Backed by the Pratt familys listed investment vehicle Thorney Investments, Mesoblast debuted on the ASX in 2004 and reached a peak valuation of $2.5bn in 2011 before suffering a reality check.

Culprits included a phase II heart trial that failed to meet primary endpoints, a badly executed Nasdaq listing and Israel pharma house Teva Pharmaceuticals decision to walk away from a heart program partnership in 2016.

Mesoblast dual listed on the Nasdaq in November 2015, accompanied by a $US63m capital raising.

The companys Ards and GvHD programs are based on mesenchymal stem cell assets acquired from US pharma group Osiris Therapeutics in October 2013.

Mesoblasts own-developed cells are called mesenchymal precursor cells and they are being developed for rheumatoid arthritis and diabetic nephropathy, as well as the aforementioned heart failure and lower back pain programs.

Ards is bought on by an excessive immune response to the virus in the lungs. The immune cells produce inflammatory cytokines, which destroy lung tissue and can also damage the liver, kidney and heart.

Remestemcell-L has the potential to tame the cytokine storm in Ards and may offer a life-saving treatment for those unfortunate individual sufferers of COVID-19 Ards, Professor Itescu says.

Mesoblasts COVID-19 proclamations have been coming so thick and fast that its been Ard(s) just to keep up. But the core excitement cluster was around Mesoblasts April 23 disclosure of the results of the trial at New Yorks Mt Sinai Hospital, covering moderate to acute Ards cases.

Under the compassionate use protocol, the patients were treated with two infusions of remestemcel-L over the first five days.

The results? Nine of the 12 patients came off a ventilator within a median 10 days, with 83 per cent survival (the Grim Reapers spin on this is that two of them died).

In comparison, only 9 per cent of patients at one reference hospital (38 out of 445 patients) were able to come off the ventilator with standard-of-care treatment.

Another US hospital reported that only 38 patients of 320 or 12 per cent survived.

Of course, 12 people good and true are adequate numbers for a jury, but sub-optimal to comprise a statistically significant trial.

Thus, the company is enrolling 300 patients in a phase III, randomised, controlled trial of severe Ards patients at 30 sites.

The first patients were dosed in early May, with about 15 sites established as the company chases the disease from the northeast to the southern states.

Mesoblast chief medical officer Professor Fred Grossman says the company is carefully choosing hot spots such as Alabama which, as of late May had the no vacancy signs outside its intensive care wards.

The sites are recruiting quite quickly, he says. There is a tremendous interest in this study.

The trial leaders will undertake an interim analysis at 30 days, and when 30 per cent of patients have reached their primary endpoint. At that point the trial can be dumped on futility grounds, or expanded to the control group because it appears to be working.

Remestemcell-L has investigational new drug (IND) status with the US Food and Drug Administration, meaning the company swiftly can initiate trials on patients with very dismal prospects.

Long-suffering Mesoblast investors will recall that the companys shares tumbled 28 per cent in November 2018 after a 159-patient trial of Rexlemestrocel-L (Revascor) for end-stage heart failure did not meet its primary endpoint of weaning patients from left ventricle assist devices (LVADs or heart pumps).

The company claimed the endpoint was set by the independent !!! investigators and was of little real clinical interest. What really mattered was that the trial showed reduced gastrointestinal bleeding by 76 per cent and hospitalisations by 65 per cent.

Investors are now nervously awaiting the first readout of the broader 566-patient chronic heart failure trial across 59 US sites.

Mesoblast targeted patients with class three or four disease, the sickest 15 to 20 per cent of patients who have failed standard-of-care drugs.

Class three patients have a 20 per cent chance of dying within two years while with class four its a case of flip a coin that you will be around in 12 months.

At this stage, Mesoblast retains its heart treatment rights except in China, where it is partnered with Tasly Pharmaceutical.

Mesoblasts phase III back pain trial aimed to enroll 404 patients with lower back pain caused by degenerative disc disease.

The endpoint of the trial, dubbed MPC-06-ID, is an improvement in pain and function over 24 months.

As with the heart trial, results are imminent and its a toss-up as to what release will hit the ASX announcements feed first.

The company is liaising with its global back pain partner Grunenthal GmbH about the clinical protocol for a European phase III confirmatory trial.

In Japan, Mesoblast is partnered with JCR Pharmaceutical for its approved GvHD treatment called Temcell and its off and racing in that smallish but enthusiastic market.

Meanwhile, the company is angling to enter the US market for a similar GvHD treatment, branded Ryoncil.

GvHD afflicts about half of the 30,000 patients annually undergoing allogeneic bone marrow transplant, typically for blood cancers, with their bodies rejecting the alien transplant.

In March, the FDA granted priority review with a September 30 action date, but we might have a good idea of the outcome in August.

Why? Because thats when the FDAs relevant advisory committee meets to vote on the matter and the (virtual) gathering is open to the public.

A date is yet to be set. While advisory committee views are not binding on the FDA, they usually presage the final decision.

If approved, Mesoblast could be selling Ryoncil in the US by the time were carving the Christmas turkey (badly, in the case of your columnist).

Buoyed by the COVID-19 results, Mesoblast in May wasted no time tapping institutional investors for an idle $US90m ($129.6m) in a placement.

Mesoblast already had a healthy cash balance of $US60m.

The raising was struck at $3.20 a share, a modest 7 per cent discount to the prevailing price.

The funds, in the main, will be used to scale-up manufacturing of remestemcell-L and to support the phase III trial, as well as for working capital and general corporate purposes.

The company also has $US67m available through existing financing facilities and partnerships.

Mesoblast reported revenue of $US31.45m for the nine months to March 2020, up 113 per cent. The reported loss narrowed 34 per cent to $US45.3m, reflecting curtailed research and development spend by $US7.5m, or 15 per cent.

The revenue included $US5.9m of JCR royalties from Temcell sales in Japan and milestone revenue of $US25m.

The company stands to pocket up to $US150m of royalties and milestones from Grunenthal prior to any European launch of Revascor.

Successful sales could result in up to $US1bn in milestone payments.

Over the last decade, Mesoblasts ASX shares have traded as high as $9 (October 2011) and as low as $1.03 (December last year).

To the Meso-sceptics the company has promised far too much with limited commercial success, while raising $1bn since listing 16 years ago.

Dare we say that Mesoblast now looks more focused and to be getting somewhere?

When we last covered Mesoblast in March 2019, Professor Itescu said he was 95 per cent certain the company would do what no other Aussie biotech in phase III had done: win FDA drug approval.

Well, Clinuvel has stolen that Aussie first honour, but Mesoblast is well placed to get over the line with a GvHD treatment in the US, which presents a market eight times the size of Japans.

Its certainly rare for a biotech to expect results for three major trials and a key regulatory decision in the space of months.

If the heart and back pain results are definitively positive and the FDA green lights GvHD, the company hits the jackpot. If two or more of them bomb lets not go there.

Your ultra conservative columnist regards the COVID-19 stuff as the icing on the cake with an outside chance of success, especially given the hundreds of other programs in the coronavirus-busting sector.

Disclosure: Dr Boreham is not a qualified medical practitioner and does not possess a doctorate of any sort. But he hopes to become proficient in turkey carving by December 25.

This column first appeared in Biotech Daily.

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Bone marrow aspiration and trephine biopsy are usually performed on the back of the hipbone, or posterior iliac crest. An aspirate can also be obtained from the sternum (breastbone). For the sternal aspirate, the patient lies on their back, with a pillow under the shoulder to raise the chest. A trephine biopsy should never be performed on the sternum, due to the risk of injury to blood vessels, lungs or the heart.

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The need to selectively isolate and concentrate selective cells, such as mononuclear cells, allogeneic cancer cells, T cells and others, is driving the market. Over 30,000 bone marrow transplants occur every year. The explosive growth of stem cells therapies represents the largest growth opportunity for bone marrow processing systems.

Europe and North America spearheaded the market as of 2018, by contributing over 74.0% to the overall revenue. Majority of stem cell transplants are conducted in Europe, and it is one of the major factors contributing to the lucrative share in the cell harvesting system market.

In 2018, North America dominated the research landscape as more than 54.0% of stem cell clinical trials were conducted in this region. The region also accounts for the second largest number of stem cell transplantation, which is further driving the demand for harvesting in the region.

Asia Pacific is anticipated to witness lucrative growth over the forecast period, owing to rising incidence of chronic diseases and increasing demand for stem cell transplantation along with stem cell-based therapy. Japan and China are the biggest markets for harvesting systems in Asia Pacific. Emerging countries such as Mexico, South Korea, and South Africa are also expected to report lucrative growth over the forecast period. Growing investment by government bodies on stem cell-based research and increase in aging population can be attributed to the increasing demand for these therapies in these countries.

Major players operating in the global bone marrow processing systems market are ThermoGenesis (Cesca Therapeutics inc.), RegenMed Systems Inc., MK Alliance Inc., Fresenius Kabi AG, Harvest Technologies (Terumo BCT), Arthrex, Inc. and others..

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Global Bone Marrow Aspirate Concentrates Marketwas valued US$ XX Bn in 2018 and is expected to reach US$ XX Bn by 2026, at CAGR of 6.5 % during forecast period of 2019 to 2026

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Bone marrow concentrate (BMC) uses stem cells that are harvested from your own bone marrow to help the body heal itself. These cells when injected directly into an injury site, prompt a rapid and efficient restoration of the tissue, returning it to a more healthy state by stimulating the bodys natural healing response. It is non-surgical treatment for various orthopedic injuries, including mild to moderate osteoarthritis, disc degeneration and soft tissue injuries.The report study has analyzed revenue impact of COVID -19 pandemic on the sales revenue of market leaders, market followers and market disrupters in the report and same is reflected in our analysis.

Global Bone Marrow Aspirate Concentrates Market Drivers and RestrainsBone marrow-derived stem cell treatment is considered a promising and advanced therapy. It reduces the injury healing time in orthopedic diseases to five to six weeks from four to six months in case of surgery. Reduction in the healing time is a factor likely to fuel the Bone Marrow Aspirate Concentrates market during the forecast period.

Pain associated with the treatment, lack of awareness, and use of alternative treatments are major restraints to the Global Bone Marrow Aspirate Concentrates Market. Furthermore, increased investments in R&D and clinical trials attributed to slow approval processes entailing sunken costs, and marginal returns on investment for manufacturers are factors hindering Global Bone Marrow Aspirate Concentrates Market.

Global Bone Marrow Aspirate Concentrates Market key segmentationBy end-use market is divided into hospitals & clinics, pharmaceutical & biotechnology companies, Contract Research Organizations (CROs) & Contract Manufacturing Organizations (CMOs), and academic & research institutes. The hospitals & clinics segment dominated the bone marrow aspirate concentrates market in 2018 and is expected to maintain its dominance during the forecast period. The hospitals & clinics segmental growth is boosted by the biotechnology & biopharmaceutical companies in terms of revenue during the forecast period. Growth of the segment is attributed to increasing number of biotechnology companies and rising partnerships among the market players to expand globally.

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Global Bone Marrow Aspirate Concentrates Market regional analysisBy regional analysis, global bone marrow aspirate concentrates market is divided into major five geographical regions, including North America, Europe, Asia-Pacific, Latin America and Middle East and Africa. North America held largest share of the Global Bone Marrow Aspirate Concentrates market owing to technological advancements and regulatory approval for new devices, rising awareness about stem cell therapy, and number of cosmetic surgical procedures. Furthermore, Asia Pacific orthopedic market is key driver, which led to this massive and augmented growth. The orthopedic market in Asia including bone graft, spine, and bone substitute is anticipated to grow as fast as the overall orthopedic market which will further boost growth of BMAC market in the region during forecast period.

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The report also helps in understanding Global Bone Marrow Aspirate Concentrates Market dynamics, structure by analyzing the market segments, and project the Global Bone Marrow Aspirate Concentrates Market size. Clear representation of competitive analysis of key players by Bone Marrow Aspirate Concentrates Type, price, financial position, product portfolio, growth strategies, and regional presence in the Global Bone Marrow Aspirate Concentrates Market make the report investors guide.Global Bone Marrow Aspirate Concentrates Market by product type

Bone Marrow Aspirate Concentrates Systems Bone Marrow Aspirate Concentrates AccessoriesGlobal Bone Marrow Aspirate Concentrates Market Application

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Chapter One: Bone Marrow Aspirate Concentrates Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Bone Marrow Aspirate Concentrates Market Competition, by Players

Chapter Four: Global Bone Marrow Aspirate Concentrates Market Size by Regions

Chapter Five: North America Bone Marrow Aspirate Concentrates Revenue by Countries

Chapter Six: Europe Bone Marrow Aspirate Concentrates Revenue by Countries

Chapter Seven: Asia-Pacific Bone Marrow Aspirate Concentrates Revenue by Countries

Chapter Eight: South America Bone Marrow Aspirate Concentrates Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Bone Marrow Aspirate Concentrates by Countries

Chapter Ten: Global Bone Marrow Aspirate Concentrates Market Segment by Type

Chapter Eleven: Global Bone Marrow Aspirate Concentrates Market Segment by Application

Chapter Twelve: Global Bone Marrow Aspirate Concentrates Market Size Forecast (2019-2026)

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