Spinal Cord Trauma Treatment Market: Global Industry Analysis 2012 2016 and Forecast 2017 2025is the recent report of Persistence Market Research that throws light on the overall market scenario during the period of eight years, i.e. 2017-2025. According to this report, Globalspinal cord trauma treatment marketis expected to witness significant growth during the forecast period.

This growth is expected to be primarily driven by increasing incidence of spinal cord trauma, and increasing government support to reduce the burden of spinal cord injuries. Additionally, development of nerve cells growth therapy is expected to boost the market in near future.

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The global market for spinal cord trauma treatment is is estimated to be valued atUS$ 2,276.3 Mnin terms of value by the end of 2017. The global spinal cord trauma treatment market is expected to expand at aCAGR of 3.7%over the forecast period to reach a value ofUS$ 3,036.2 Mnby 2025end.

Global Spinal Cord Trauma Treatment Market: Trends

Global Spinal Cord Trauma Treatment Market: Forecast by End User

On the basis of end user, the global spinal cord trauma treatment market is segmented into hospitals and trauma centers. Hospitals segment dominated the global spinal cord trauma treatment market in revenue terms in 2016 and is projected to continue to do so throughout the forecast period.

Hospitals and trauma centers segments are expected to approximately similar attractive index. Hospitals segment accounted for53.2%value share in 2017 and is projected to account for52.5%share by 2025 end.

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Proton Therapy Systems Market

Proton Therapy Market Segmented By Single Room, Multiple Room Set up Type with Head and Neck Cancer, Brain Cancer, Sarcoma Pediatric Cancer, Gastro-intestinal Cancer, Prostate Cancer, Lung Cancer Indication.For More Information

Trauma Fixation Devices Marketglobal trauma fixation devices market is estimated to represent more than US$ 450 Mn of the total market in 2017 and is estimated to reach little more than US$ 800 Mn by 2025 end, expanding at CAGR of 7.5% over the forecast period of 20172025.For More Information

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PMR : Spinal Cord Trauma Treatment Market Worth Will Reach US$ 3000 Mn According To Forecast By 2025 - Cole of Duty

Spinal Cord Stem Cells Comments Off on PMR : Spinal Cord Trauma Treatment Market Worth Will Reach US$ 3000 Mn According To Forecast By 2025 – Cole of Duty | May 14th, 2020

The novel coronavirus cant be killed or stopped with the current drugs that we have, the WHO said earlier this week. Dr. Anthony Fauci said separately that its virtually impossible to eradicate the virus. But there are plenty of therapies that can be used to reduce the severity of COVID-19 and shorten the recovery period.

The WHO is studying four or five of the best drugs for the new illness, but there are plenty of new lines of therapy that are discovered on a regular basis. The latest one consists of a treatment thats usually given to Duchenne muscular dystrophy patients.

Cedars-Sinai doctors have given six patients an experimental treatment consisting of cells grown from human heart tissues, according to ABC7. This therapy improved the overall condition of all patients, each of whom were critically ill before the Hail Mary treatment was administered. Four of them have come off ventilators and were discharged, while the other two are still in the hospital, but theyre alive.

Dr. Eduardo Marban and his colleagues were using the treatment for muscular dystrophy patients with heart failure before considering it for COVID-19. The novel coronavirus can do severe damage to the heart, and that may have been the reason why the doctors attempted this novel therapy.

This can only be considered anecdotal evidence at best, but the doctors are hoping that the FDA can approve a more extensive study that can evaluate the benefits of the therapy. The doctors have additional doses available in the freezer for the research.

Cells grown from human heart tissues sound a lot like stem cells, although the report doesnt refer to them as such. This wouldnt be the first time that stem cell use would prove to be helpful in COVID-19 cases. A few weeks ago, doctors from Mount Sinai reported theyve treated 12 patients using stem cells derived from bone marrow, and the therapy allowed 10 of them to come off ventilators. Those physicians also noted that further study is required.

Marban and his colleagues detailed the benefits of injections of cardiac progenitor cells (cardiosphere-derived cells or CDCs) for patients with muscular dystrophy in February 2018. Cardiosphere-derived cells are stem cells derived from cardiac tissue.

We unexpectedly found that treating the heart made the whole body better, Marban said at the time. These basic findings, which have already been translated to clinical trials, rationalize why treating the heart may also benefit skeletal muscle function in boys and young men with Duchenne.

The study showed the stem cells acted not just on the heart tissue, but also on skeletal muscle, and that the benefits persisted. We found that within a few weeks, the injected cells were undetectable, Marban said, but the benefits persisted for at least three months, which led us to discover that exosomes secreted by CDCs are responsible.

The same type of therapy was likely used to treat COVID-19 patients.

Image Source: John Minchillo/AP/Shutterstock

Chris Smith started writing about gadgets as a hobby, and before he knew it he was sharing his views on tech stuff with readers around the world. Whenever he's not writing about gadgets he miserably fails to stay away from them, although he desperately tries. But that's not necessarily a bad thing.

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Doctors just discovered another promising coronavirus therapy - BGR

Cardiac Stem Cells Comments Off on Doctors just discovered another promising coronavirus therapy – BGR | May 14th, 2020

Four of six patients in case series were weaned off respiratory support

An investigational allogeneic cell therapy using cardiosphere-derived cells (CDC) showed an acceptable safety profile with early evidence of efficacy in the treatment of very severe Covid-19 in a case series involving six patients treated at Cedars-Sinai Medical Center in Los Angeles.

All six patients treated with the intravenous allogeneic CDC formulation CAP-1002 (Capricor Therapeutics) as a compassionate therapy required respiratory support prior to treatment, with five on mechanical ventilation.

No adverse events related to the treatment were reported, and four of the six patients were successfully weaned from respiratory support and were discharged from the hospital as of late April.

The other two patients are still alive, but remain intubated, Cedars-Sinai cardiologist Raj Makkar, MD, confirmed to BreakingMED Wednesday, May 13.

While we are encouraged by these findings, it is important to point out that the only way that we can assess the efficacy of this treatment in a definitive way is with a randomized clinical trial, and that is what we intend to do, Makkar said.

He added that the clinical trial, which is in the planning stages, is likely to include Covid-19 patients who are not as critically ill as the six in the case series.

All of these patients required respiratory support and they were all on a downward trajectory when treated, he said. They were getting worse and we had nothing else to offer them.

Cardiosphere-derived cells are stromal/progenitor cells from heart tissue with a distinctive antigenic profile (CD105+, CD45-, CD90low).

In their case series, published in the journal Basic Research in Cardiology, Makkar and colleagues noted that the cells are entirely distinct from the controversial c-kit+ putative cardiac progenitors, which have been the subject of various retracted studies.

Since CDCs were first isolated in 2007, the cells have been tested in more than 200 patients in clinical trials for a variety of conditions with a good safety profile, including in young boys with Duchenne muscular dystrophy.

Makkar said the anti-inflammatory and antifibrotic properties of CDCs in animal models make them a possible target therapy for Covid-19.

The prior testing gave us reasonable confidence that this treatment was safe, he said, adding that there is also evidence of a favorable effect on the same type of proinflammatory cytokines that are up-regulated in Covid-19.

Comparisons to mesenchymal stem cells (MSCs) in pre-clinical models suggest that CDCs may also be more effective for paracrine factor secretion and myocardial remodeling.

Given the safety record of CDCs in humans, and the substantial body of evidence confirming relevant disease-modifying bioactivity, applicability to Covid-19 seemed compelling, particularly in the hyperinflammatory stage of the illness, the researchers wrote.

All six patients treated with the intravenous CDC formulation had severe, confirmed Covid-19 with respiratory failure and they were not receiving any other experimental agent, with the exception of hydroxychloroquine and tocilizumab.

Lack of clinical improvement or deterioration despite standard care was the primary reason for considering patients for treatment with CAP-1002. Exclusion criteria included known hypersensitivity to DMSO, which is a component of CAP-1002; prior stem cell therapy; pre-existing terminal illness; and need for mechanical circulatory support and dialysis.

In general, patients with multi-organ failure who were deemed to be too sick for any intervention were excluded from the study, Makkar and colleagues wrote.

All patients had acute respiratory distress syndrome (ARDS) prior to infusion, with decreased PaO2/FiO2 ratios (range 69-198; median 142), diffuse bilateral pulmonary infiltrates on chest imaging and evidence of preserved cardiac function on transthoracic echocardiography (LVEF range, 50-75%). SOFA scores ranged from 2 to 8 prior to stem cell treatment.

The six patients (age range, 19-75 years) had IV infusions of CAP-1002 containing 150 million allogeneic CDCs, and two of the six had a second dose of the treatment.

Following treatment, four patients (67%) were weaned from respiratory support and discharged from the hospital.

A contemporaneous control group of critically ill Covid-19 patients (n = 34) at our institution showed 18% overall mortality at a similar stage of hospitalization, the researchers wrote.

Ferritin was elevated in all patients at baseline (range of all patients 605.43-2991.52 ng/ml) and decreased in five of the six patients (range of all patients 252.891029.90 ng/ml).

Absolute lymphocyte counts were low in five of the six patients at baseline (range 0.260.82 103/l) but had increased in 3 of these five at last follow-up (range 0.231.02 103/l).

Administration of CAP-1002 as a compassionate therapy for patients with severe Covid-19 and significant comorbidities was safe, well tolerated without serious adverse events, and associated with clinical improvement, as evidenced by extubation (or prevention of intubation, the researchers wrote.

Stem cell therapy utilizing cardiosphere-derived cells (CDC) showed an acceptable safety profile with early evidence of efficacy in the treatment of very severe Covid-19 in an early case series involving 6 patients treated at Cedars-Sinai Medical Center, Los Angeles.

No adverse events related to the treatment were reported, and four of the six patients were successfully weaned from respiratory support and were discharged from the hospital.

Salynn Boyles, Contributing Writer, BreakingMED

Funding for this story was provided by the Smidt Family Foundation. The cell product, CAP-1002, was provided by manufacturer Capricor Therapeutics.

ResearcherEduardo Marban reported owning founders equity in Cariricor Therapeutics, and researcher Linda Marban reported being an employee and owning equity in the company.

Cat ID: 125

Topic ID: 79,125,254,930,287,728,932,570,574,730,933,125,190,926,192,927,151,928,925,934

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Cardio Stem Cell Therapy Used to Treat Critically Ill Covid-19 Patients - Physician's Weekly

Cardiac Stem Cells Comments Off on Cardio Stem Cell Therapy Used to Treat Critically Ill Covid-19 Patients – Physician’s Weekly | May 14th, 2020

Presentation of new and updated results from ongoing Phase 1/2 HGB-206 study of LentiGlobin for sickle cell disease will include additional patients treated in the study

New and updated data, including analysis of healthy red blood cell production in patients with transfusion-dependent -thalassemia following treatment with betibeglogene autotemcel (LentiGlobin for -thalassemia) to be shared

CAMBRIDGE, Mass. bluebird bio, Inc. (Nasdaq: BLUE) announced today that data from its gene therapy programs for sickle cell disease (SCD), transfusion-dependent -thalassemia (TDT) and its cell therapy program for relapsed and refractory multiple myeloma (RRMM) will be presented during the Virtual Edition of the 25th European Hematology Association (EHA25) Annual Congress.

New data from the companys Phase 1/2 HGB-206 study of LentiGlobin gene therapy for SCD will be presented, including updated data from patients in Group C.

bluebird bio will also present data from its ongoing clinical studies of betibeglogene autotemcel (formerly LentiGlobin gene therapy for -thalassemia), including the Phase 3 Northstar-2 (HGB-207) study in patients who do not have a 0/0 genotype and the Phase 3 Northstar-3 (HGB-212) study in patients who have 0/0, 0/+IVS-I-110, or +IVS-I-110/+IVS-I-110 genotypes.

Data from studies of idecabtagene vicleucel (ide-cel; bb2121), the companys anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cell therapy in development with Bristol Myers Squibb, will be presented, including an encore presentation of results from the pivotal Phase 2 KarMMa study.

Sickle Cell Disease Data at EHA25

Oral Presentation: Outcomes in patients treated with LentiGlobin for sickle cell disease (SCD) gene therapy: Updated results from the Phase 1/2 HGB-206 group C study Presenting Author: Julie Kanter, M.D., University of Alabama at Birmingham, Birmingham, Ala.

Transfusion-Dependent -Thalassemia Data at EHA25

Oral Presentation: Improvement in erythropoiesis in patients with transfusion-dependent -thalassemia following treatment with betibeglogene autotemcel (LentiGlobin for -thalassemia) in the Phase 3 HGB-207 study Presenting Author: John B. Porter, MA, M.D., FRCP, FRCPath, University College London Hospital, London, UK

Poster: Betibeglogene autotemcel (LentiGlobin) in patients with transfusion-dependent -thalassemia and 0/0, +IVS-I-110/+IVS-I-110, or 0/+IVS-I-110 genotypes: Updated results from the HGB-212 study Presenting Author: Evangelia Yannaki, M.D., George Papanicolaou Hospital, Thessaloniki, Greece

Multiple Myeloma Data at EHA25

Oral Presentation:Phase II KarMMa study: Idecabtagene vicleucel (ide-cel; bb2121), a BCMA-targeted CAR T cell therapy, in patients with relapsed and refractory multiple myeloma Presenting Author: Jesus San-Miguel, M.D., Ph.D., Clinica Universidad de Navarra, Navarra, Spain

Poster: Quality of life in patients with relapsed and refractory multiple myeloma treated with the BCMA-targeted CAR T cell therapy Idecabtagene vicleucel (ide-cel; bb2121): results from the KarMMa Trial Presenting Author: Michel Delforge, M.D., Ph.D., Leuven University College, Brussels, Belgium

Poster: Matching-adjusted indirect comparisons of efficacy outcomes for idecabtagene vicleucel from the KarMMa study vs selinexor PLUS dexamethasone (STORM part 2) and belantamab mafodotin (DREAMM-2) Presenting Author: Paula Rodriguez-Otero, M.D., Clinica Universidad de Navarra, Navarra, Spain

Poster: Baseline and postinfusion pharmcodynamic biomarkers of safety and efficacy in patients treated with idecabtagene vicleucel (ide-cel; bb2121) in the KarMMa study Presenting Author: Justine DellAringa, Bristol Myers Squibb, Seattle, Wash.

Poster: Correlation of tumor BCMA expression with response and acquired resistance to idecabtagene vicleucel in the KarMMa study in relapsed and refractory multiple myeloma Presenting Author: Nathan Martin, Bristol Myers Squibb, Seattle, Wash.

Abstracts outlining bluebird bios accepted data at the EHA25 Virtual Congress have been made available on the EHA25 conference website. On Friday, June 12 at 8:30 AM CEST, the embargo will lift for poster and oral presentations accepted for EHA25.

About betibeglogene autotemcel The European Commission granted conditional marketing authorization (CMA) for betibeglogene autotemcel, marketed as ZYNTEGLO gene therapy, for patients 12 years and older with TDT who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available. On April 28, 2020, the European Medicines Agency (EMA) renewed the CMA for ZYNTEGLO, supported by data from 32 patients treated with ZYNTEGLO including three patients with up to five years of follow-up.

TDT is a severe genetic disease caused by mutations in the -globin gene that result in reduced or significantly reduced hemoglobin (Hb). In order to survive, people with TDT maintain Hb levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

Betibeglogene autotemcel adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

Non-serious adverse events (AEs) observed during the clinical studies that were attributed to betibeglogene autotemcel were abdominal pain, thrombocytopenia, leukopenia, neutropenia, hot flush, dyspnoea, pain in extremity, and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to LentiGlobin for -thalassemia for TDT.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

The CMA for ZYNTEGLO is only valid in the 28 member states of the EU as well as Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. Food and Drug Administration granted betibeglogene autotemcel Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT. Betibeglogene autotemcel is not approved in the United States.

Betibeglogene autotemcel continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more information about the ongoing clinical studies, visit http://www.northstarclinicalstudies.com or clinicaltrials.gov and use identifier NCT02906202 for Northstar-2 (HGB-207), NCT03207009 for Northstar-3 (HGB-212).

About LentiGlobin for Sickle Cell Disease LentiGlobin for sickle cell disease is an investigational gene therapy being studied as a potential treatment for SCD. bluebird bios clinical development program for LentiGlobin for SCD includes the ongoing Phase 1/2 HGB-206 study and the ongoing Phase 3 HGB-210 study.

SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the -globin gene that leads to the production of abnormal sickle hemoglobin (HbS), causing red blood cells (RBCs) to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and painful vaso-occlusive crises (VOCs). For adults and children living with SCD, this means unpredictable episodes of excruciating pain due to vaso-occlusion as well as other acute complicationssuch as acute chest syndrome (ACS), stroke, and infections, which can contribute to early mortality in these patients.

LentiGlobin for SCD received Orphan Medicinal Product designation from the European Commission for the treatment of SCD.

The U.S. Food and Drug Administration (FDA) granted Orphan Drug status and Regenerative Medicine Advanced Therapy designation for LentiGlobin for the treatment of SCD.

LentiGlobin for SCD is investigational and has not been approved by the European Medicines Agency (EMA) or FDA.

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of betibeglogene autotemcel and LentiGlobin for SCD. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

About idecabtagene vicleucel (ide-cel; bb2121) Ide-cel is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T cell immunotherapy. The ide-cel CAR is comprised of a murine extracellular single-chain variable fragment (scFv) specific for recognizing BCMA, attached to a human CD8 hinge and transmembrane domain fused to the T cell cytoplasmic signaling domains of CD137 4-1BB and CD3- chain, in tandem. Ide-cel recognizes and binds to BCMA on the surface of multiple myeloma cells leading to CAR T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells.

In addition to the pivotal KarMMa trial evaluating ide-cel in patients with relapsed and refractory multiple myeloma, bluebird bio and Bristol Myers Squibbs broad clinical development program for ide-cel includes clinical studies (KarMMa-2, KarMMa-3, KarMMa-4) in earlier lines of treatment for patients with multiple myeloma, including newly diagnosed multiple myeloma. For more information visit clinicaltrials.gov.

Ide-cel was granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) and PRIority Medicines (PRIME) designation, as well as Accelerated Assessment status, by the European Medicines Agency for relapsed and refractory multiple myeloma.

Ide-cel is being developed as part of a Co-Development, Co-Promotion and Profit Share Agreement between Bristol Myers Squibb and bluebird bio.

Ide-cel is not approved for any indication in any geography.

About KarMMa KarMMa (NCT03361748) is a pivotal, open-label, single-arm, multicenter, multinational, Phase 2 study evaluating the efficacy and safety of ide-cel in adults with relapsed and refractory multiple myeloma in North America and Europe. The primary endpoint of the study is overall response rate as assessed by an independent review committee (IRC) according to the International Myeloma Working Group (IMWG) criteria. Complete response rate is a key secondary endpoint. Other efficacy endpoints include time to response, duration of response, progression-free survival, overall survival, minimal residual disease evaluated by Next-Generation Sequencing (NGS) assay and safety. The study enrolled 140 patients, of whom 128 received ide-cel across the target dose levels of 150-450 x 10P6P CAR+ T cells after receiving lymphodepleting chemotherapy. All enrolled patients had received at least three prior treatment regimens, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, and were refractory to their last regimen, defined as progression during or within 60 days of their last therapy.

About bluebird bio, Inc. bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, LentiGlobin, and bluebird bio are trademarks of bluebird bio, Inc.

Forward-Looking Statements This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any forward-looking statements are based on managements 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: regarding the potential for betibeglogene autotemcel to treat transfusion-dependent -thalassemia and the potential for LentiGlobin for sickle cell disease (SCD) to treat SCD; and the risk that the efficacy and safety results from our prior and ongoing clinical trials will not continue or be repeated in our ongoing or planned clinical trials. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200514005234/en/

Contacts

Media: Catherine Falcetti, 339-499-9436 cfalcetti@bluebirdbio.com Victoria von Rinteln, 617-914-8774 vvonrinteln@bluebirdbio.com

Investors: Ingrid Goldberg, 410-960-5022 Ingrid.goldberg@bluebirdbio.com Elizabeth Pingpank, 617-914-8736 epingpank@bluebirdbio.com

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bluebird bio to Present Data from Its Gene and Cell Therapy Programs During the Virtual Edition of the 25th European Hematology Association Annual...

Cardiac Stem Cells Comments Off on bluebird bio to Present Data from Its Gene and Cell Therapy Programs During the Virtual Edition of the 25th European Hematology Association Annual… | May 14th, 2020

This discovery may have clinical importance in management of heart failure.

This discovery may have clinical importance in management of heart failure.Many heart diseases are linked to oxidative stress, an overabundance of reactive oxygen species. The body reacts to reduce oxidative stress where the redox teeter-totter has gone too far up through production of endogenous antioxidants that reduce the reactive oxygen species. This balancing act is called redox homeostasis.

But what happens if the redox teeter-totter goes too far down, creating antioxidative stress, also known as reductive stress? Rajasekaran Namakkal-Soorappan, Ph.D., associate professor in the University of Alabama at Birmingham Department of Pathology, and colleagues have found that reductive stress, or RS/AS, is also pathological. This discovery, they say, may have clinical importance in management of heart failure.

They report that RS causes pathological heart enlargement and diastolic dysfunction in a mouse model. This study, published in the journal Antioxidants and Redox Signaling, was led by Namakkal-Soorappan and Pei Ping, Ph.D., David Geffen School of Medicine at the University of California-Los Angeles.

Antioxidant-based therapeutic approaches for human heart failure should consider a thorough evaluation of antioxidant levels before the treatment, they said. Our findings demonstrate that chronic RS is intolerable and adequate to induce heart failure.

The study used transgenic mice that had upregulated genes for antioxidants in the heart, which increased the amounts of antioxidant proteins and reduced glutathione, creating RS. One mouse line had low upregulation, and one had high upregulation, creating chronic low RS and chronic high RS, respectively, in the hearts of the mice.

The mice with high RS showed pathological heart changes called hypertrophic cardiomyopathy, and had an abnormally high heart ejection fraction and diastolic dysfunction at 6 months of age. Sixty percent of the high-RS mice died by 18 months of age.

The mice with low RS had normal survival rates, but they developed the heart changes at about 15 months of age, suggesting that even moderate RS can lead to irreversible damage in the heart over time.

Giving high-RS mice a chemical that blocked biosynthesis of glutathione, beginning at about 6 weeks of age, prevented RS and rescued the mice from pathological heart changes.

Gobinath Shanmugam, Ph.D., postdoctoral fellow in the UAB Department of Pathology, and Namakkal-Soorappan point out that a 2019 survey found about 77 percent of Americans are consuming dietary supplements every day, and within this group, about 58 percent are consuming antioxidants as multivitamins. Thus, a chronic consumption of antioxidant drugs by any individual without knowing their redox state might result in RS, which can induce pathology and slowly damage the heart.

In a related study, published in the journal Redox Biology, Namakkal-Soorappan looked at the impact of RS on myosatellite cells, which are also known as muscle stem cells. These cells, located near skeletal muscle fibers, are able to regenerate and differentiate into skeletal muscle after acute or chronic muscle injury. The regulation of myosatellite cells is of interest given the loss of skeletal muscle mass during aging or in chronic conditions like diabetes and AIDS.

Recently, Namakkal-Soorappan reported that tilting the redox teeter-totter to oxidative stress impaired regeneration of skeletal muscle. Now, in the Redox Biology paper, he has shown that tilting the redox to RS also causes significant inhibition of muscle satellite cell differentiation.

Rather than genetic manipulation to induce RS, as was done in the heart study, the researchers used the chemical sulforaphane or direct augmentation of intracellular glutathione to induce RS in cultured mouse myoblast cells. Both treatments inhibited myoblast differentiation. Finally, authors attempted to withdraw antioxidative stress by growing cells in medium without sulforaphane, which removes the RS and accelerates the differentiation. Namakkal-Soorappan and colleagues found that a pro-oxidative milieu, through a mild generation of reactive oxygen species, was required for myoblast differentiation.

The researchers also showed that genetic silencing of a negative regulator of the antioxidant genes also inhibited myoblast differentiation.

Co-authors with Namakkal-Soorappan and Ping, and first-author Shanmugam, in the Antioxidants and Redox Signaling study, Reductive stress causes pathological cardiac remodeling and diastolic dysfunction, are Silvio H. Litovsky and Rajesh Kumar Radhakrishnan, UAB Department of Pathology; Ding Wang, UCLA; Sellamuthu S. Gounder, Kevin Whitehead, Sarah Franklin and John R. Hoidal, University of Utah School of Medicine; Jolyn Fernandes and Dean P. Jones, Emory University, Atlanta, Georgia; Thomas W. Kensler, Fred Hutch Cancer Research Center, Seattle, Washington; Louis DellItalia, UAB Department of Medicine; Victor Darley-Usmar, UAB Department of Pathology; and E. Dale Abel, University of Iowa.

In the Redox Biology study, Reductive stress impairs myogenic differentiation, co-authors with Namakkal-Soorappan are Sandeep Balu Shelar, UAB Department of Pathology; Dean P. Jones, Emory University; and John R. Hoidal, University of Utah School of Medicine.

Support for both studies came from National Institutes of Health grants HL118067 and AG042860, American Heart Association grant BGIA 0865015F, the University of Utah, and UAB.

In the two studies, Namakkal-Soorappans name is listed as Namakkal S. Rajasekaran.

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Surplus antioxidants are pathogenic for hearts and skeletal muscle - The Mix

Cardiac Stem Cells Comments Off on Surplus antioxidants are pathogenic for hearts and skeletal muscle – The Mix | May 14th, 2020

DUBLIN--(BUSINESS WIRE)--The "Cell Therapy - Technologies, Markets and Companies" report from Jain PharmaBiotech has been added to ResearchAndMarkets.com's offering.

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

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

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

This report contains information on the following:

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

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

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

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

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

Key Topics Covered

Part I: Technologies, Ethics & Regulations

Executive Summary

1. Introduction to Cell Therapy

2. Cell Therapy Technologies

3. Stem Cells

4. Clinical Applications of Cell Therapy

5. Cell Therapy for Cardiovascular Disorders

6. Cell Therapy for Cancer

7. Cell Therapy for Neurological Disorders

8. Ethical, Legal and Political Aspects of Cell therapy

9. Safety and Regulatory Aspects of Cell Therapy

Part II: Markets, Companies & Academic Institutions

10. Markets and Future Prospects for Cell Therapy

11. Companies Involved in Cell Therapy

12. Academic Institutions

13. References

For more information about this report visit https://www.researchandmarkets.com/r/7h12ne

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The Cell Therapy Industry to 2028: Global Market & Technology Analysis, Company Profiles of 309 Players (170 Involved in Stem Cells) -...

Skin Stem Cells Comments Off on The Cell Therapy Industry to 2028: Global Market & Technology Analysis, Company Profiles of 309 Players (170 Involved in Stem Cells) -… | May 14th, 2020

Dr Shikha Sharma

Coronavirus disease (COVID-19) is an unforgettable word in 2020. World health organization has declared COVID-19 as pandemic and according to the Worldometer site, it has affected 212 countries and territories and has caused approximately 2.8 lakhs deaths so far. According to the various published scientific evidences COVID-19 is an infectious disease caused by new coronavirus that can lead to lung dysfunction. There are 7 coronaviruses that are known to cause disease in humans and among these 3 can cause the severe respiratory infection. These are severe acute respiratory syndrome coronavirus (SARS-CoV) identified in 2002 in China, Middle East respiratory syndrome coronavirus (MERS-CoV) identified in 2012 in Saudi Arabia and severe acute respiratory syndrome coronavirus2 (SARS-CoV2) commonly called COVID-19 identified in late 2019 in Wuhan, China. SARS-CoV, MERS-CoV and COVID-19 are closely related but COVID-19 spread more quickly than the other two. Over 8000 people from 29 different countries were affected with SARS-CoV epidemic during 2002-2004 while 40.78 lakhs people are affected with COVID-19 so far. In most cases, immune response (bodys defence system) triggered by the COVID-19 infection is sufficient to combat its pathogenesis leads to the recovery of patient. However, in some cases, COVID-19 infection causes highly inflammatory form of lung cells death and injury as the most dangerous phase of its pathogenesis which leads to the overproduction of inflammatory cytokines by bodys own immune cells creating cytokine storm that results in damage to the lung tissues causing pneumonia, acute respiratory distress syndrome (ARDS) and sepsis. In Pneumonia and ARDS air sac of lungs fill with fluid or pus. These complications lead to severe condition such as shortness of breath that require treatment with oxygen and ventilator. Therefore controlling inflammatory response is utmost important to prevent coronavirus lethality rate and for the longer life of a patient. Currently no specific treatment is available for COVID-19 infection but several vaccines, drugs and stem cells testing in various countries has generated hope to combat its pathogenesis. Recent breakthrough has demonstrated mesenchymal stem cells (MSCs) as cell medicine therapy to reduce COVID-19 infection.What are MSCsMSCs are multipotent adult stem cells that are capable of differentiating into various cell types such as fat cells, bone cells, liver cells, pancreatic cells, brain cells, heart cells and skin cells thus can participate in the repair and regeneration of various tissues and organs of the body. Inside the body, upon injury, MSCs migrate to the injured site and participate in the regeneration and repair of the organ either by differentiation or by paracrine secretion or both. In addition MSCs possess immunomodulatory and anti-inflammatory properties that contributes to its cell medicinal properties. MSCs can be isolated from various tissues such as bone marrow, peripheral blood, body fat, muscle, placenta, umbilical cord, umbilical cord blood, teeth and hair follicles and can be expanded ex vivo and used for transplantation for treating disease and disorders after genetic stability test.How MSCs reduce COVID-19pathogenesisAs reported by various research groups that upon intravenous injection or through mist inhalation the significant population of MSCs migrate to the lung and secrete various immunomodulatory and anti-inflammatory factors to cure lung dysfunction by normalizing immune response altered by COVID-19 and stimulate lung repair. Moreover MSCs are resistant to COVID-19 infection and can be used for autologous and allogenic transplantation.Clinical trial with MSCs for COVID-19There are several clinical trials registered with MSCs for the treatment of COVID-19 from various countries such as China, USA, UK, Germany, UAE, Jordan and Iran and some reports have been published. Approximately 100 patients have been treated with MSCs therapy from moderate to critical conditions within 10-15 days of transplantation. A first case treated with MSCs showed the recovery of 65 year old critical ill patient in Baoshan Peoples Hospital, Longling County, China. Initially the patient was treated with antiviral therapy and immunomodulator thymosin alpha1 but hasnt shown any recovery. Later after 10 days patient was diagnosed with severe pneumonia, acute respiratory distress syndrome, multiorgan injury, type2 diabetes, moderate anaemia, electrolyte disturbance, immunosuppression, acute gastrointestinal bleeding and other symptom was shifted to ICU and on ventilator. They showed that after three MSCs injections along with thymosin alpha1 lead to the recovery of patient from COVID-19 infection. FDA has approved 24 patient clinical trial in USA to test safety and efficacy of MSCs from umbilical cord to prevent COVID-19 infection. Recently, in USA three critically ill patients in ICU and on ventilator recovered from COVID-19 infection with MSCs treatment. An Israeli pharmaceutical company Pluristem therapeutics have tested MSCs therapy on 7 critically ill patient and found positive results. More recently, UAE also reported the treatment of 73 COVID-19 infected patients with stem cells. They have developed the technology to isolate the stem cells from patient blood, activate them and reintroduce them by mist inhalation. These reports are indicative that MSCs hold the potential to treat the COVID-19 infection by preventing bodys own defense system from overreacting and normalise its response to fight against COVID-19 infection. Many companies from different countries are seeking approval to begin clinical trial with stem cells against COVID-19 infection.Why are we lagging behind when we have stem cell companies/labs/facility in our country? We also produce GMP grade stem cells for transplantation. China tested the stem cell therapy on first patient when all other therapies failed and stem cells was one of option left to save the life of the patient. In India also so many deaths are happening due to COVID-19 we can also check if stem cells can reduce the mortality rate. Moreover as per some reports MSCs dont stay inside the body for more than 1-3 months and they eventually die and dont result in teratoma formation. Our government along with doctors and scientist can also formulate committee on stem cells and begin such initiative to test MSCs for the treatment of COVID-19 infection. Nevertheless, MSCs has joined the army along with the other possible interventions to prevent the COVID-19 illness.(The author is (PhD and Postdoc in Stem Cells)feedbackexcelsior@gmail.com

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Stem cells therapy A prospective treatment against coronavirus? - Daily Excelsior

Skin Stem Cells Comments Off on Stem cells therapy A prospective treatment against coronavirus? – Daily Excelsior | May 14th, 2020

Stem cells have long-held hope for many people with spinal cord injuries. Since their discovery in 1998, they have been used in thousands of studies to one day cure paralysis, but there is still no cure for those with chronic injuries. Despite this, scientists have come a long way, especially in recent years. You have likely heard about some of the research in the news, stirring more hope than ever before.

And the hope is not unfounded. The hard work and the millions of dollars going into this research is finally seeing results. Stem cells may not be the only key to finding a cure for a spinal cord injury, but they arent going anywhere and are being used in hundreds of studies around the world. Here are the ones you should know about.

The human body has millions of stem cells that can be found all over the body. Researchers at the Mayo Clinic were recently in the news for their results using fat-derived adult stem cells from the patients own body. This study recruited 10 individuals with traumatic spinal cord injuries. Each was injected with stem cells taken from the fat in their stomachs and was expanded in the lab for eight weeks. The injection was then in the lower lumbar area.

This treatment is brand new and has not been approved by the FDA, however, the study was granted special clearance. One individual in the study, a man in his fifties with an incomplete injury who had leg return and was able to walk slightly post-injury without treatment, saw a nearly 50% increase in his abilities after receiving the injection.

Researchers also made sure to wait until each person in the study had plateaued after their injuries to be sure that the results from the treatment were not results from the body still having a new injury. There is currently no further news on whether the FDA will approve this treatment for the general population.

Nearly a year ago, Japan's Health Ministry approved a trial that will involve four people with complete injuries. This stem cell trial uses induced pluripotent stem cells (IPS) taken from embryos and will be grown into two million nerve cells for each patient. These cells will then be injected into the injury site. This trial comes from Masaya Nakamura, a professor at Okano and Keio University, who saw improvements in animals after they underwent the procedure. There has that no updates on this trial since it was approved.

In Spain, a clinical trial has been underway for the last few years that uses stem cells taken from the patient's bone marrow and injected into their injury site. This research comes from Dr. Vaquero at the Puerta de Hierro University Hospital in Spain. His first trial in 2016 included people with complete injuries and his second trial in 2017 included people with incomplete injuries. Almost all patients who underwent the procedure saw some improvement, with some seeing more improvement than others. The trial is currently seeking 30 people with incomplete injuries for its next phase.

Dr. Steven Levy of MD Stem Cells is launching the SciExVR trial using a patient's stem cells from their bone marrow as well. This study is currently recruiting patients in the United States and will involve exoskeleton rehab as part of the trial. Learn more:http://mdstemcells.com/sciexvr

Dr. Wise Young, along with Rutgers University and his organization SCINetChina, has been approved for a study in the United States that will involve umbilical cord blood stem cells and oral lithium. This study will involve 27 people with complete chronic injuries levels C5-T11. You must be able to be in New Jersey for six months is chosen.

Six years ago in 2014, Dr. Raisman from Poland pioneered a study using nerve stem cells taken from the nose. These stem cells were taken from the olfactory bulb deep in the brain and were transplanted into the injury site along with nerve tissue taken from the patient ankle. This study is recruiting one person for the trial who has a perfectly severed spinal cord (by a knife or similar). The person recruited will also have to spend several years in Poland. To learn more, contacthttps://walk-again-project.org/#/en

Keep in mind that all the above stem cell trials are still trials and that they cannot promise any return of movement or sensation. It is always in your best interest to go into a trial with an open mind and to be hopeful, but be realistic at all times.

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The Latest In Stem Cell Therapy After SCI

Spinal Cord Stem Cells Comments Off on The Latest In Stem Cell Therapy After SCI | May 13th, 2020

Bioscience Americas and the Global Institute of Stem Cell Therapy and Research would like to extend a special thank you to the Christopher and Dana Reeve Foundation for their support relating to our work at the University California Irvin and the Anderson Laboratory. We have made exciting progress using stem cells to treat cervical spinal cord injuries because of their generosity.

Now, based on the results of Dr. Andersons Phase I/II clinical trial, our research partners are conducting a Phase II proof of concept trial using HuCNS-SC in cervical spinal cord injury. In this study, research participants are being treated between 10 to 23 months post-injury.

Spinal Cord Injury (SCI) is damage to the human spinal cord into three different segments of the neural tissue leading to a severe form of motor and sensory loss. The kind of damage can be differentiated as:

In most of the reported cases of SCI, damage can be due to trauma or disease. Apart from the physical damage and complete dependency on caregivers, SCI can be emotionally damaging as well. Due to dependency even on basic mobility, negative attitudes of suffering trauma forever and frequent mood swings can lead suffers to remove themselves from social participation. Thus more than 30% of the reported cases of SCI showed significant signs of depression and negative impact on the functional improvement of overall health.

How prevalent is SCI?

Since SCI is associated with the loss of mobility, paralysis, and mortality due to other opportunistic infections, it is known as one of the most critical and disastrous medical conditions. Every year around 2 million to 5 million people are reported to suffer from spinal cord injury. On an average, middle-aged and young adult males are more susceptible to SCI mainly due to avoidable causes such as road accidents, injury, falls or violence. Mortality associated with SCI has been observed to be the highest immediately after the injury than in later years. The risk of mortality doubles with the severity level and is observed to be strongly influenced by the immediate availability of the best medical care. Preventable secondary opportunistic infections are also reported to be a major cause of death in many SCI patients, especially in the lower income groups.

About 90% of patients in the age group of 20-45 have been reported to face other complications such as limited employment, decreased quality of life, and severe depression.

Factors responsible for SCI.

In general, a spinal cord injury is a result of to the severe damage to different parts of the spinal cord such as the vertebral column, ligaments or the spinal disks. This typically originates from sudden trauma to the spinal cord such as fracturing, crushing or dislocating one or more vertebrae. Additional damage has been reported due to excessive bleeding, swelling, inflammation as well as other opportunistic infections. The most common reported causes of Spinal Cord Injury are:

Symptoms Associated with SCI

In general, the severity, as well as the area of injury, are the factors to be of concern in most of the cases of Spinal Cord Injury. On the basis of severity of injury, SCI is classified as:

What goes wrong in Spinal Cord Injury?

The human spinal cord is a fragile bridge connecting the brain to the other organs of the body. The spinal cord is encased in a protective covering of spinal vertebrae of the spinal column to prevent its damage from shock or injury. Our central nervous system, i.e., brain and spinal cord, is made up of millions of cells which coordinate and communicate to pass on the information from the brain to the other organs of the body via the spinal cord. This information is passed in the form of electrical signals which are then decoded by the specific organ.

Each neuron is made up of a cellular body with a long slender projection called the nerve fiber. These fibers are attached to other fibers to form a dense network of cells. In general, neurons carrying messages down the cord from the brain to other organs of the body are known as Motor Neurons. These neurons control the muscles of some of the important internal organs of the body such as heart, stomach, intestine, etc. The neurons traveling up the cord to the brain are known as Sensory Neurons, carrying sensory information from skin, joints, and muscles to control our ability to sense, touch and regularize temperature.

These neurons are insulated from the outer side by the coating of Oligodendrocytes and myelin sheath. These cells insulate the neuron to protect them from sudden damage and shock.

If any of the above types of cells are affected due to sudden damage such as shearing, laceration, stretching or shock, then the network of cells is disturbed due to which the passage of information from the brain to the spinal cord and vice versa is halted.

How Stem Cells treatment can help.

Stem cells are the mother cells that are responsible for developing an entire human body from tiny two-celled embryos. Due to their unlimited divisions and strong power to differentiate into all the cells of different lineage, the power of stem cells has been harnessed by our technology to isolate them outside the human body, concentrate in a clean environment, and implant back.

Thus stem cells treatment involves administration of concentrated cells in the targeted area, wherein they can colonize in the damaged area, adapt the properties of resident stem cells and initiate some of the lost functions that have been compromised by the disease or injury.

Thus with our standardized, broad-based and holistic approach, it is now possible to obtain noticeable improvements in SCI cases, in the symptoms as well as their functional abilities.

Continue reading here:
Spinal Cord Injury Recovery Through Stem Cell Therapy

Spinal Cord Stem Cells Comments Off on Spinal Cord Injury Recovery Through Stem Cell Therapy | May 13th, 2020

They hold huge promise, but stem cell-based spinal cord treatments wont be clinically available in the near future

My three-year-old son was born with a very large spinal lipoma. He was considered quadriplegic. Through conventional physical and occupational therapies and surgery to remove some of the lipoma he has gained enough function to walk with a walker and use his arms. However, he is experiencing some regression as his nerves are dying.

I have saved the cord blood from his younger brother and sister. New research where mice are being paralyzed and then injected with stem cells looks very promising to us. The mice nerves that are sick or weak are being protected and strengthened. Our son needs his nerves protected from degeneration.

Conventional surgery is no longer an option because the nerve roots travel in and out of the lipoma and cannot be separated from the lipoma. Our only hope is to protect and strengthen what function he currently has.

My question is: How long before this type of stem cell therapy will be used on humans, more specifically children? And how do we get to be first in line? If it is 10 or 20 years away, there may be no way to save the function our son has worked so hard to gain. I havent read anything about risks or side effects. There have to be some, what are they? Also, are there other countries that are more aggressive in their use of stem cells on humans for treating paralysis resulting from spinal cord injury?

Barbara BourgeoisCentreville, Virginia, USA

There isnt an easy answer here, and Im not clear as to why function is being lost at this pointin particular, whether the lipoma is recurring. If this is the case, resolution of the lipoma is the main issue. In some instances, it is impossible to completely remove the tumor, severely limiting the potential benefits of secondary therapeutics (such as stem cells). However, on the topic of stem cells in particular, there are several issues to discuss.

First, there are many sources of stem cells, and this affects their potential clinical use. Cord blood-derived stem cells are probably the farthest away from potential clinical use for spinal cord injury at this point, because there has been less basic research done with them so far. Human embryonic and adult stem cell lines may be somewhat closer, but research on these in the laboratory has been somewhat mixedsome very promising results with regaining motor function, and some big potential concerns, such as causing tumor formation.

As a result, we are most likely still years away from testing these treatments in patients, even to establish safety. Some other kinds of cell treatments, such as ensheathing glial cells, are being tried in the clinic in China, Russia and Portugal based on previous laboratory research in the US. However, none of these overseas trials has been designed in accordance with US standards to rigorously test safety and efficacy, and it is very difficult to evaluate the patchy data coming out so far.

To sum up, as a researcher, I think stem cells hold a huge amount of promise, but we arent yet at a point where this work will be translated to the clinic in the immediate future.

Answered by Aileen J. Anderson ~ 1/22/2004

Posted on January 27th, 2004 in General SCI and Human Interest. Tagged: stem cells

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When Will Stem Cells Heal Spinal Cord Damage?

Spinal Cord Stem Cells Comments Off on When Will Stem Cells Heal Spinal Cord Damage? | May 13th, 2020

Akin to cystic fibrosis (CF), scientists understand that certain mutations contribute to the development of the fatal neurological disorder amyotrophic lateral sclerosis (ALS). And much like CF drugmaker Vertex, a small Cambridge, Massachusetts-based biotech is forging a path to engineering precision therapies to treat the disease that killed visionary physicist Stephen Hawking.

The company, christened QurAlis, now has $42 million in its coffers with three preclinical programs and 5 employees (including senior management) to combat an illness that has long flummoxed researchers, resulting in a couple of approved therapies over the course of decades, neither of which attacks the underlying cause of the rare progressive condition that attacks nerve cells located in the brain and spinal cord responsible for controlling voluntary muscles.

ALS garnered international attention when New York Yankees player Lou Gehrig abruptly retired from baseball in 1939, after being diagnosed with the disease. In 2014, ALS returned to the spotlight with the Ice Bucket Challenge, which involved people pouring ice-cold water over their heads, posting a video on social media, and donating funds for research on the condition.

QurAlis chief Kasper Roet, whose interest in ALS was piqued while he was working on his PhD at the Netherlands Institute for Neuroscience focusing on a treatment for spinal cord paralysis and moonlighting at the Netherlands Brain Bank as an ad-hoc autopsy team coordinator, saw an opportunity to combat ALS when Harvard scientists Kevin Eggan and Clifford Woolf pioneered some new stem cell technology.

Essentially, they found a way to take skin cells from a patient, turn them into stem cells, and turn those into the nerve cells that are degenerating. Thats the missing link, Roet said. So now we can finally use patients own cells to both do target discovery and develop potential therapeutics.

So Roet packed up his things and shifted base to Boston to learn more, with plans to head back to Europe to start a company. He never left. QurAlis was born in 2016, working out of a co-working space called LabCentral after winning a spot via an Amgen-sponsored innovation competition. The company was carved out of a collaboration with Eggans startup Q-State Biosciences, which developed laser technology to examine cell behavior examining how a neuron fires was imperative in the drug discovery process for ALS.

QurAlis, which counts Vertexs founding scientist Manuel Navia as an advisor, now has three preclinical programs. The furthest along is a therapy designed to target a specific potassium channel that is implicated in certain ALS patients the plan is to take that small molecule into the clinic next year, Roet said.

It has become really clear that if you understand why a specific tumor is developing you can develop very specific targeted therapies, he explained in an interview drawing a parallel between ALS and oncology. Thats exactly the same strategy that we are following for ALS. The genetics have shown that over 25 genes are causing the (ALS) mutations. Some of them work together, some of them are very dominant and work alone what we are doing is trying to get those specific proteins that are tied to very specific ALS populations, where we know that that specific target plays a very important and crucial role in the development of the disease.

In 2018, QurAlis scored seed funding from Amgen, Alexandria, and MP Healthcare Venture Management. The Series A injection was led by LS Polaris Innovation Fund, lead seed investor Mission BioCapital, INKEF Capital and the Dementia Discovery Fund, and co-led by Droia Ventures. Additional new investors include Mitsui Global Investment and Dolby Family Ventures, and existing investors Amgen Ventures, MP Healthcare Venture Management, and Sanford Biosciences also chipped in.

Roet is not sure how long these funds will last, particularly given the uncertainty of the coronavirus pandemic. But some of the capital will be used in hiring, given that the QurAlis team is comprised of a mere five people, including Roet.

Weve been very productive, he said. But we can definitely use some extra hands.

Read more:
Precision therapy approach secures small biotech $42M haul to combat disease that inspired the Ice Bucket Challenge - Endpoints News

Spinal Cord Stem Cells Comments Off on Precision therapy approach secures small biotech $42M haul to combat disease that inspired the Ice Bucket Challenge – Endpoints News | May 13th, 2020

DUBLIN--(BUSINESS WIRE)--The "Global Stem Cell Therapy Market By Type (Allogeneic, Autologous, Syngeneic), By Source of Stem Cells (Adipose Tissue, Bone Marrow, Neural, Embryo/Cord Blood derived, iPSCs, Others), By Application, By End Users, By Region, Forecast & Opportunities, 2025" report has been added to ResearchAndMarkets.com's offering.

The Global Stem Cell Therapy Market is expected to grow at a formidable rate of around 12% during the forecast period. The industry is segmented based on type, source of stem cells, application, end-users, company and region.

The market is driven by the growing popularity and awareness pertaining to the use of stem cells for the prevention and cure of certain life threatening diseases. Additionally, increase in number of stem cell banks and growing investments by the government and private organizations for the development of stem cell preservation infrastructure is further propelling the market across the globe.

Based on type, the market can be categorized into allogeneic, autologous and syngeneic. The allogenic type segment is expected to register the highest growth during forecast period attributable to the rising commercialization of allogeneic stem cell therapy products, wider therapeutic applications of allogeneic stem cells, easy production scale-up process, growing number of clinical trials related to allogeneic stem cell therapies, among others.

Based on end-users, the market can be bifurcated into hospitals and clinics. The hospitals segment is expected to dominate the market during the forecast years. This can be accredited to the rising preference for stem cell therapies offered by hospitals proves beneficial for the business growth. Hospitals have affiliations with research laboratories and academic institutes that carry out research activities for developing stem cell therapies. On introduction and approval of any novel stem therapy, hospitals implement it immediately.

Regionally, the stem cell therapy market has been segmented into various regions namely Asia-Pacific, North America, South America, Europe, and Middle East & Africa. Among these regions, North America is expected to dominate the overall stem cell therapy market during the next five years on account of the increasing number of clinical trials for stem cell-based products and increasing public-private funding & research grants.

Major players operating in the Global Stem Cell Therapy Market include Osiris Therapeutics, Inc., MEDIPOST Co., Ltd., Anterogen Co., Ltd., Pharmicell Co., Ltd., Holostem Terapie Avanzate S.r.l., JCR Pharmaceuticals Co., Ltd., NuVasive, Inc., RTI Surgical, Inc., AlloSource, Thermo Fisher Scientific and others. The companies are developing advanced technologies and launching new services in order to stay competitive in the market.

Years considered for this report:

Objective of the Study

Key Topics Covered

1. Product Overview

2. Research Methodology

3. Executive Summary

4. Voice of Customer

5. Global Stem Cell Therapy Market Outlook

5.1. Market Size & Forecast

5.1.1. By Value

5.2. Market Share & Forecast

5.2.1. By Type (Allogeneic, Autologous, Syngeneic)

5.2.2. By Source of Stem Cells (Adipose Tissue, Bone Marrow, Neural, Embryo/Cord Blood Derived, iPSCs, Others)

5.2.3. By Application (Musculoskeletal, Wound & Injury, Cardiovascular Disease (CVD), Surgery, Acute Graft-Versus-Host Disease, Drug Discovery & Development, Others)

5.2.4. By End Users (Hospitals v/s Clinics)

5.2.5. By Company (2019)

5.2.6. By Region

5.3. Product Market Map

6. Asia-Pacific Stem Cell Therapy Market Outlook

7. Europe Stem Cell Therapy Market Outlook

8. North America Stem Cell Therapy Market Outlook

9. South America Stem Cell Therapy Market Outlook

10. Middle East and Africa Stem Cell Therapy Market Outlook

11. Market Dynamics

11.1. Drivers

11.2. Challenges

12. Market Trends & Developments

13. Competitive Landscape

13.1. Osiris Therapeutics, Inc.

13.2. MEDIPOST Co. Ltd.

13.3. Anterogen Co. Ltd.

13.4. Pharmicell Co. Ltd.

13.5. Holostem Terapie Avanzate S.r.l.

13.6. JCR Pharmaceuticals Co. Ltd.

13.7. NuVasive, Inc.

13.8. RTI Surgical, Inc.

13.9. AlloSource

13.10. Thermo Fisher Scientific

14. Strategic Recommendations

For more information about this report visit https://www.researchandmarkets.com/r/hmawq6

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Global Stem Cell Therapy Market Forecast & Opportunities, 2025 - ResearchAndMarkets.com - Business Wire

Bone Marrow Stem Cells Comments Off on Global Stem Cell Therapy Market Forecast & Opportunities, 2025 – ResearchAndMarkets.com – Business Wire | May 13th, 2020

A Scunthorpe mum who underwent 'brutal' stem cell treatment says the hardest part was not being able to see her daughters.

Joanne While has recently passed the six month anniversary of the treatment to wipe out and then regrow her immune system.

The mum-of-three was diagnosed with Multiple Sclerosis (MS) at the age of 31, and wasn't eligible for trial treatments in the UK.

The HSCTtreatment in Mexico saw her undergo chemotherapy and then have stem cells transplanted in the hopes of stopping the damage that the MS was causing. Some MS patients have also seen their symptoms be reversed from this.

"It was a very harsh, brutal treatment. I had to be kept in a special apartment where I protected from all germs. There was a lot of sickness and just getting out of bed some days was difficult," Joanne said.

"The hardest part was being away from my family. My ex-partner was very kind in taking a months unpaid leave to come to Mexico and help me through the treatment.

"At the end of the day, all of the treatment has been worth it.

"I had a brain MRI scan two months ago which showed that the MS lesions hadnt grown since last time. My body is still recovering, so time will tell exactly how good the news is."

The HSCT (haematopoietic stem cell transplantation) treatment cost the family 45,000 including flights and visas. They launched an online fundraising page last year to help cover the costs.

With her weak immune system still being rebuilt, Joanne has been shielding since before the coronavirus outbreak began.

Her family have adopted extremely strict hygiene measures at the Scunthorpe home to keep her healthy during this critical time.

HSCT is a chemotherapy-based medical procedure that wipes out the immune system and reboots it using a patient's own stem cells, which are harvested from their blood or bone marrow,

The aim is to reset the immune system to stop it attacking the rest of the body, therefore halting the progression of the MS.

It is the only medical procedure currently available that has halted the progression of the majority of patients undertaking it.

HSCT is currently available only on a trial basis in the UK, and only for individuals who have been unsuccessful with the range ofdisease modifying therapies. Each time it fails, irreparable damage is being done and the disease continues to progress.

"Before the outbreak, it had just gotten to the point where I was able to venture out for a coffee, but of course all of that has stopped now," Joanne said.

"I had to pull my daughters out of school early to minimise the risk of them bringing the virus home. Now we regularly sanitise the house and change clothes whenever we have to enter or leave in order to keep it as clean as possible.

"My eldest daughter, who is 24, has been wonderful as my carer. She has stopped work to prevent her from catching any infections.

"Im often tired and need a three hour nap in the afternoon, which can be difficult with a five-year-old in the house. Its been a balancing act, but Im so grateful to everyone who helped me during or since the treatment.

"Shielding can be frustrating, but its all about your mindset when you look at it. Its not that I cant go outside I get to be at home in my favourite place with my daughters."

Due to her compromised immune system, Joanne has had to start her vaccinations again and has just been given those that are usually given to babies.

Tests have also shown that her white blood count has recently decreased to the point it was in Mexico, although Joanne has hopeful it will recover.

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HSCT can initially cause mobility issues and stiffness in muscles, which Joanne is having physio to manage.

She is documenting her recovery on her Facebook page 'Jo's HSCT Journey'.

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Mum's brutal stem cell treatment has 'all been worth it' as she enjoys time with family - Grimsby Live

Bone Marrow Stem Cells Comments Off on Mum’s brutal stem cell treatment has ‘all been worth it’ as she enjoys time with family – Grimsby Live | May 13th, 2020

The Covid-19 (coronavirus) pandemic is impacting society and the overall economy across the world. The impact of this pandemic is growing day by day as well as affecting the supply chain. The COVID-19 crisis is creating uncertainty in the stock market, massive slowing of supply chain, falling business confidence, and increasing panic among the customer segments. The overall effect of the pandemic is impacting the production process of several industries including Life science Industry, and many more. Trade barriers are further restraining the demand- supply outlook. As government of different regions have already announced total lockdown and temporarily shutdown of industries, the overall production process being adversely affected; thus, hinder the overall Stem Cell Therapy market globally. This report on Stem Cell Therapy market provides the analysis on impact on Covid-19 on various business segments and country markets. The report also showcase market trends and forecast to 2027, factoring the impact of Covid -19 Situation.

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Stem cell therapy is a technique which uses stem cells for the treatment of various disorders. Stem cell therapy is capable of curing broad spectrum of disorders ranging from simple to life threatening. These stem cells are obtained from different sources, such as, adipose tissue, bone marrow, embryonic stem cell and cord blood among others. Stem cell therapy is enables to treat more than 70 disorders, including degenerative as well as neuromuscular disorders. The ability of a stem cell to renew itself helps in replacing the damaged areas in the human body.

MARKET DYNAMICSIncrease in the number of stem cell banking facilities and rising awareness on the benefits of stem cell for curing various disorders are expected to drive the market during the forecast period. Rise in number of regulations to promote stem cell therapy and increase in number of funds for research in developing countries are expected to offer growth opportunities to the market during the coming years.

Key Players

The research provides answers to the following key questions:

The study conducts SWOT analysis to evaluate strengths and weaknesses of the key players in the Stem Cell Therapy market. Further, the report conducts an intricate examination of drivers and restraints operating in the market. The report also evaluates the trends observed in the parent market, along with the macro-economic indicators, prevailing factors, and market appeal according to different segments. The report also predicts the influence of different industry aspects on the Stem Cell Therapy market segments and regions.

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It is extremely vital to have an impartial understanding of market opinions for a strategy. Our insights provide a keen view on the market sentiment. We keep this reconnaissance by engaging with Key Opinion Leaders of a value chain of each industry we track.

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Our research ranks investment centers of market by considering their future demands, returns, and profit margins. Our clients can focus on most prominent investment centers by procuring our market research.

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Our research and insights help our clients identify compatible business partners.

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Stem Cell Therapy Market Segmented by Region/Country: North America, Europe, Asia Pacific, Middle East & Africa, and Central & South America

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COVID-19 Impact on STEM CELL THERAPY MARKET 2020 TO 2027-EXPANDING WORLDWIDE WITH TOP PLAYERS FUTURE BUSINESS SCOPE AND INVESTMENT ANALYSIS REPORT -...

Bone Marrow Stem Cells Comments Off on COVID-19 Impact on STEM CELL THERAPY MARKET 2020 TO 2027-EXPANDING WORLDWIDE WITH TOP PLAYERS FUTURE BUSINESS SCOPE AND INVESTMENT ANALYSIS REPORT -… | May 13th, 2020

As a skin-care brand, Augustinus Bader prides itself on releasing products not out of frivolity or trying to up its bottom line, but out of necessity. As Charles Rosier, cofounder and CEO of Augustinus Bader, puts it: "We want to only add product when we feel we are relevant," he tells Allure via phone from Paris, France, where he is currently self-isolating. "Our latest initiative was a reaction to something that we felt was needed by the consumer by everyone."

The brand's newest offering, The Hand Treatment, is meant to be just that. As it stands, given the current COVID-19 situation, we are all washing our hands (or should be) much more than we might otherwise normally do so. However, as you've also likely experienced dry skin and flaking cuticles, just as is the case with everywhere else on the body, overwashing the skin on our hands can bring on a host of issues all their own. Even if you forgo washing for an alcohol-based hand sanitizer, the effects can be just as profound if not more so.

"We are seeing more and more hand dermatitis," explains New York City-based board-certified dermatologist Dhaval Bhanusali, who is not affiliated with Augustinus Bader. "By overwashing, we strip the good oils from our skin and leave it dry and susceptible to breakdown."

Our skin's moisture barrier is responsible for keeping the good things in (like hydration and natural oils) and the bad things out (like allergens and bacteria). When the lipid barrier on the hands (or anywhere on the body) becomes too dried out, it can literally start to break down and lose functionality. Simply put, in order for our skin to function and remain healthy, its barrier must be intact. However, we're not saying that you should stop washing your hands just that it's equally important to replenish hydration after doing so.

That's where Augustinus Bader's new Hand Treatment comes into play. It's formulated with a blend of ultra-nourishing ingredients, including vitamin E, glycerin, and shea butter, as well as honey and white peony extract both of which also have natural antibacterial properties. However, as with all Bader products, the key ingredient is something called TFC8, which stands for its proprietary Trigger Factor Complex-8.

For those unfamiliar with the brand, TFC8 is not one singular ingredient but rather, a proprietary blend of "natural amino acids, high-grade vitamins, and synthesized molecules naturally found in skin," cosmetic chemist Ginger King (who is not affiliated with the brand) previously told Allure. In a nutshell, TFC8 provides an ideal environment in which our skin's stem cells can naturally regenerate it acts as a sort of guide, helping to set the skin's inherent repair system back on track.

"The TCF8 technology focuses on intrinsic repair processes based in intrinsic repair cells, which we call stem cells," Bader explains. "We wanted to develop a hand cream with our technology, which helps to protect and care for the skin."

The results of using TFC8 topically, according to Augustinus Bader himself, a German stem-cell scientist, and his many devotees (our own editor in chief among them), read like a laundry list of skin-care goals: Increased hydration, a strengthened skin barrier, faded fine lines, and dark spots. The proprietary ingredient is, of course, also formulated into the brand's other offerings: two creams for the face, one for the body, and most recently, a makeup primer collaboration with Victoria Beckham Beauty.

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Augustinus Bader Launches The Hand Treatment in Response to COVID-19 Pandemic Exclusive Details - Allure

Skin Stem Cells Comments Off on Augustinus Bader Launches The Hand Treatment in Response to COVID-19 Pandemic Exclusive Details – Allure | May 13th, 2020

CRANFORD, N.J., May 13, 2020 /PRNewswire/ -- Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a specialty pharmaceutical company focused on developing and commercializing critical care drug products, today announced that data on NoveCite MSCs will be presented this week at the American Society of Gene and Cell therapy (ASGCT) annual meeting. NC-MSCs are made by Novellus, Inc. ("Novellus"), a Cambridge-based biotechnology company, using its patented mRNA-based cell-reprogramming process. Earlier this year, Citius signed an exclusive option agreement to in-license NC-MSCs for acute respiratory distress syndrome (ARDS), including in COVID-19 patients, from Novellus.

The data to be presented show that NC-MSCs secrete higher levels of anti-inflammatory proteins compared to MSCs derived from bone marrow. From the abstract: "Comparative secretome analysis showed overexpression of multiple neuroprotective and anti-inflammatory factors, including CXCL1, VEGF-A, and CXCL5." In addition, NC-MSCs showed therapeutic benefit in an experimental autoimmune encephalomyelitis (EAE) mouse model, delaying disease progression and improving the clinical score compared to the control group, while bone marrow-derived MSCs showed no difference from the control.

"We are pleased to present these data at the annual meeting of the ASGCT," said Matt Angel, PhD, co-founder and Chief Science Officer at Novellus. "While conventional MSCs have shown promise in the treatment of inflammatory lung disease, protein secretion and manufacturability remain challenges for these approaches. The data that will be presented this week show that iPSC-derived MSCs secrete higher levels of anti-inflammatory proteins, and exhibit greater expansion potential than bone marrow-derived MSCs. We believe that these properties make iPSC-derived MSCs especially well-suited for an allogeneic cell therapy for ARDS."

"Last month Citius signed an exclusive option agreement with Novellus for worldwide development and commercial rights related to the use of these uniquely derived MSCs for the treatment of ARDS. The pre-clinical data that is being presented at the ASGCT annual meeting adds to our confidence in the higher potency of these MSCs, which we expect will result in better outcomes for patients with COVID-19 and ARDS," stated Myron Holubiak, CEO of Citius Pharmaceuticals. "We intend to study these cells in the clinic later this year to determine safety, efficacy, and the optimal dose of these cells in moderate to severe ARDS patients with COVID-19."

Citius has submitted a pre-IND meeting request and supporting briefing documents to the Center for Biologics Evaluation and Research ("CBER") of the FDA under the Coronavirus Treatment Acceleration Program (CTAP) for use of these MSCs for patients with Acute Respiratory Distress Syndrome (ARDS) due to SARS-CoV-2 disease.

Presentation Information:Title:Mesenchymal Stem Cells (MSCs) Generated Using mRNA Reprogramming Show Enhanced Growth Potential, Secretome, and Therapeutic Efficacy in a Demyelinating Disease ModelPresenter:Harris, Jasmine, Novellus, Inc.Date and Time: Wednesday, May 13 | 5:30 PM - 6:30 PM

About Acute Respiratory Distress Syndrome (ARDS)ARDS is a type of respiratory failure characterized by rapid onset of widespread inflammation in the lungs. ARDS is a rapidly progressive disease that occurs in critically ill patients most notably now in those diagnosed with COVID-19. ARDS affects approximately 200,000 patients per year in the U.S., exclusive of the current COVID-19 pandemic, and has a 30% to 50% mortality rate. ARDS is sometimes initially diagnosed as pneumonia or pulmonary edema (fluid in the lungs from heart disease). Symptoms of ARDS include shortness of breath, rapid breathing and heart rate, chest pain (particularly while inhaling), and bluish skin coloration. Among those who survive ARDS, a decreased quality of life is relatively common.

About Coronavirus Treatment Acceleration Program (CTAP)In response to the pandemic, the FDA has created an emergency program called the Coronavirus Treatment Acceleration Program (CTAP) to accelerate the development of treatments for COVID-19. By redeploying staff, the FDA is responding to COVID-19-related requests and reviewing protocols within 24 hours of receipt. The FDA said CTAP "uses every available method to move new treatments to patients as quickly as possible, while at the same time finding out whether they are helpful or harmful." In practice, that means developers of potential treatments for COVID-19 will benefit from an unusually faster track at the FDA to shorten wait times at multiple steps of the process.

About Citius Pharmaceuticals, Inc.Citius is a late-stage specialty pharmaceutical company dedicated to the development and commercialization of critical care products, with a focus on anti-infectives and cancer care. For more information, please visit http://www.citiuspharma.com.

About Novellus, Inc.Novellus is a pre-clinical stage biotechnology company developing engineered cellular medicines using its non-immunogenic mRNA, nucleic-acid delivery, gene editing, and cell reprogramming technologies. Novellus is privately held and is headquartered in Cambridge, MA. For more information, please visit http://www.novellus-inc.com.

Safe HarborThis press release may contain "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements are made based on our expectations and beliefs concerning future events impacting Citius. You can identify these statements by the fact that they use words such as "will," "anticipate," "estimate," "expect," "should," and "may" and other words and terms of similar meaning or use of future dates. Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition, and stock price. Factors that could cause actual results to differ materially from those currently anticipated are: the risk of successfully negotiating a license agreement with Novellus within the option period; our need for substantial additional funds; the ability to access the FDA's CTAP program for the MARCO trial; the estimated markets for our product candidates, including those for ARDS, and the acceptance thereof by any market; risks associated with conducting trials for our product candidates, including those expected to be required for any treatment for ARDS and our Phase III trial for Mino-Lok; risks relating to the results of research and development activities; risks associated with developing our product candidates, including any licensed from Novellus, including that preclinical results may not be predictive of clinical results and our ability to file an IND for such candidates; uncertainties relating to preclinical and clinical testing; the early stage of products under development; risks related to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; our ability to identify, acquire, close, and integrate product candidates and companies successfully and on a timely basis; our ability to attract, integrate, and retain key personnel; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions, or circumstances on which any such statement is based, except as required by law.

Contact:Andrew ScottVice President, Corporate Development(O) 908-967-6677 x105 [emailprotected]

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Citius Announces Data on NoveCite Mesenchymal Stem Cells (NC-MSCs) to be Presented at the American Society of Gene and Cell Therapy (ASGCT) Annual...

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XconomyBoston

Few drugs exist that treat amyotrophic lateral sclerosis, a progressive disease that kills the nerve cells that allow patients to initiate and control muscle movement.

QurAlis, a Cambridge, MA-based startup, has an ambitious plan to develop a number of precision therapies for the disease based on forms of the condition identified by genetic mutation or a biomarker that CEO Kasper Roet (pictured) hopes to could one day, in combination, help most ALS patients.

Now the company has raised $42 million from investors in the US, Europe, and Japanmoney that will fund a move from the LabCentral incubator in Kendall Square to its own office, more than double the companys headcount by years end, and get at least one of its programs into human testing sometime next year.

The company is leveraging stem cell research from company co-founders Kevin Eggan and Clifford Woolf, Harvard University professors whoby harvesting normal skin cells from ALS patients and turning them into cells such as the motor neurons that damages as the disease progresseshave created models with the same DNA and gene mutations as those patients in an effort to identify new therapeutics for known ALS genes.

Mutations in more than 25 human genes have been implicated in ALS, the company says, and its strategy is to systematically investigate treatments targeting specific disease-causing mechanisms in patient subgroups. Some of those genes are also believed to cause frontotemporal dementia, a common cause of dementia that QurAlis also plans to treat.

One program QurAlis is advancing is intended for patients whose neurons are damaged and killed by the overactivation of certain receptors for glutamate, a key neurotransmitter, in a process known as excitotoxicity.

The company is also working on a treatment intended to return the autophagy process, through which cells recycle unwanted or damage components, to normal functioning. To do so, QurAlis is looking to target the enzyme TBK1, which plays a key role.

Roet, in an interview, said the company views its strategy as analogous to that pursued by Bostons Vertex Pharmaceuticals (NASDAQ: VRTX), which has developed multiple drugs for forms of cystic fibrosis (CF) caused by certain mutations, and late last year received approval for a combination of those drugs for about 90 percent of all CF patients.

We have identified ALS as a disease that we think we understand now, at least for specific subgroups of patients, he said. We understand what is driving the disease and we are able to develop very specific therapies for those patients.

Eggan, Woolf, Roet, and Jonathan Fleming launched QurAlis just over two years ago with seed funding from investors including MP Healthcare Venture Management, the investment arm of Mitsubishi Tanabe Pharma; the investment arm of Amgen (NASDAQ: AMGN); and Alexandria Venture Investments. Mitsubishi Tanabe markets edaravone (Radicava), one of four FDA-approved treatments for ALS. The FDAs 2017 nod for the drug made it the only ALS therapy OKd in the past 20 years.

The Cambridge, MA-based company said the new capital, a Series A financing round, brings the total it has raised to $50.5 million. The investment was led by LS Polaris Innovation Fund, Mission BioCapital, Dutch firm Inkef Capital, and the Dementia Discovery Fund. New investors including Droia Ventures, which operates from Luxembourg and Belgium, Mitsui Global Investment, and Dolby Family Ventures also participated, as did earlier investors including Amgen, MP Healthcare, and Sanford Biosciences.

As part of the deal, LS Polariss Amy Schulman, Inkef Capitals Roel Bulthuis, Dementia Discovery Funds Jonathan Behr, and Droia Ventures Luc Dochez join Mission BioCapitals Johannes Fruehauf on the QurAlis board.

Earlier this year some of the same investors, including Amgen and Dolby Family Ventures, backed a Series A financing for EnClear Therapies, a spinout of QurAlis. That company raised $10 million to advance the development of a dialysis-like medical device designed to filter out harmful proteins in the cerebral spinal fluid of patients with neurodegenerative diseases.

Sarah de Crescenzo is an Xconomy editor based in San Diego. You can reach her at sdecrescenzo@xconomy.com.

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QurAlis Hauls In $42M to Move New ALS Therapies Into Human Testing - Xconomy

Skin Stem Cells Comments Off on QurAlis Hauls In $42M to Move New ALS Therapies Into Human Testing – Xconomy | May 13th, 2020

Novadip Biosciences Reports Positive Interim Analysis of Phase I/II Bone Non-Union Study with NVD003

Mont-Saint Guibert, Belgium, 13 May, 2020: Novadip Biosciences (Novadip or the Company), a clinical-stage biopharmaceutical company leveraging its proprietary tissue regeneration technology platform, today announces positive data from the interim analysis of its Phase I/IIa clinical trial for autologous NVD-003 in adults with non-healing fracture of the lower limb.

NVD-003 is a novel autologous cell-based osteogenic (bone healing/[bone forming]) product that has been generated from Novadips proprietary 3M tissue regeneration platform. This platform is aimed at healing damaged tissues by restoring their natural physiology and consists of a 3-dimensional, scaffold-free extracellular matrix (ECM), utilizing differentiated adipose-derived stem cells (ASCs) to restore the physiology of natural healing. NVD003 presents as a scaffold-free 3D implant to fill critical-size bone defects where healing is compromised.

This phase I/II study is investigating in five European centers the potential of NVD-003 to promote bone union in nine adults with a non-healing fracture of the lower limb. There was 100% manufacturing success for NVD-003 and grafting surgery was completed successfully in all patients without deviating from standard medical practice. To date, with a median of 12 months post-treatment, no NVD003 related safety signal has been reported. Further exploratory analysis performed on data from the first five patients to complete a six month follow up showed a strong positive trend in radiological healing with confirmed bone formation for all patients and radiologically confirmed union for three of the patients.

Prof. Gunnar Anderson (MD, PhD), Professor and Chairman Emeritus of the Department of Orthopedic Surgery at Rush University Medical Center, Chicago, Illinois and Chairman of the Scientific Advisory Board commented: The early results of this study are remarkable both clinically and for the patients and we look forward to replicating these in a larger group in the future. It is hugely encouraging that we may potentially have a future solution for these patients with unmet needs.

Dr. Denis Dufrane (MD, PhD), Chief Executive Officer, Chief Scientific Officer commented: We are encouraged by the data from this interim analysis, which demonstrates the potential of our tissue regeneration 3M3 platform to restore natural healing processes in patients with reputedly difficult to treat bone defects. We look forward to further progressing NVD-003s clinical program in bone non-union and in patients with other similar conditions with no effective treatment option and hope to provide full study results in 2025.

NVD-003 is also in clinical stage for congenital pseudarthrosis of the tibia (CPT), a rare and disabling pediatric condition with very limited treatment options and has demonstrated clinical proof-of-concept in case studies.

Novadips tissue regeneration platform drives several new classes of product candidates with an initial focus on autologous cell therapies for critical size tissue reconstruction. Allogeneic therapeutics are in development for prevalent and complex tissue defects for bone and skin tissue and exosomes/miRNA-based therapeutics are being developed for immediate (off-the-shelf) clinical use.

- End -

Notes to editors

Novadip Biosciences

Novadip Biosciences is a clinical stage biopharmaceutical company leveraging its unique 3D tissue regeneration technology platform to generate multiple product candidates to address hard and soft tissue reconstruction for patients who have limited or no treatment options. The companys proprietary 3M3 platform is a 3-dimensional, extracellular matrix that utilizes adipose-derived stem cells to deliver highly-specific growth factors and miRNAs to mimic the physiology of natural healing and creates a range of products that address specific challenges in tissue regeneration. Novadips initial focus is on critical size bone reconstruction. The company is also applying its 3M3 platform to develop truly novel off-the-shelf/allogeneic therapies to address more prevalent tissue defects and miRNA/exosome products for broader indications. For more information, visit http://www.novadip.com .

For further information, please contact:

Novadip Biosciences

Denis Dufrane

Chief Executive Officer, Chief Scientific Officer

+32 (10) 779 220

info@novadip.com

For media enquiries:

Consilium Strategic Communications

Chris Gardner, Matthew Neal, Angela Gray

+44 (0) 20 3709 5700

novadip@consilium-comms.com

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Novadip Biosciences Reports Positive Interim Analysis of Phase I/II Bone Non-Union Study with NVD003 - Yahoo Finance UK

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Carlos R. Bachier, MD

Chimeric antigen receptor (CAR) T-cell therapies quickly burst into the spotlight of hematology-oncology disease management because of their potential to illicit deep and durable responses from patients whose disease is relapsed or refractory to multiple previous lines of therapy. Relevant professional meetings and oncology publications exploded with research and news about CAR T cells, and this cellular therapy strategy is now being explored across hematologic and solid malignancies.

CAR T cells are a scientific revolution, Tania Jain, MBBS, assistant professor of oncology at Johns Hopkins University in Baltimore, Maryland, said in an interview with Targeted Therapies in Oncology (TTO). They have brought about a paradigm shift in terms of how were treating patients.

The 2 currently FDA-approved CAR T-cell therapies, axicabtagene ciloleucel (Yescarta) and tisagenlecleucel (Kymriah), are both indicated for the treatment of adult patients with relapsed or refractory large B-cell lymphoma; additionally, tisagenlecleucel is approved for patients up to 25 years with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).1-3 With a second wave of approvals likely on the horizon for therapies such as lisocabtagene maraleucel (liso-cel) and idecabtagene vicleucel (bb2121), CAR T is gaining traction and will likely play an increasingly prominent role in the future treatment paradigm in oncology.

CAR T-cell therapy administration is largely limited to the inpatient setting at both academic institutions and large accredited cancer centers, making such treatments unavailable to most patients. Other challenges with this type of therapy include its potential to cause serious toxicities resulting in organ damage and death.4

David G. Maloney, MD, PhD

Due to the promising efficacy of these agents, investigators have been working toward viable solutions to bring CAR T-cell therapies to more patients by alleviating difficulties associated with therapy delivery and patient care.

CAR T-cell therapies, both those currently approved and the many being explored in late-phase clinical trials, are produced from autologous T cells obtained from the patient receiving therapy. This personalization has led to tremendous success, yet it is a large part of why CAR T-cell therapy use remains limited to a select group of patients.

Time is an important consideration for patients who have experienced multiple relapses and may be too weakened by numerous lines of prior therapy to wait several weeks for the CAR T-cell manufacturing process. The effects of previous treatments or the disease itself can also present challenges, as manypatients are rendered lymphopenic and may be unable to produce enough T cells for harvesting. Roadblocks may remain for patients who are not limited by these factors; manufacturing success and effectiveness of the CAR T-cell product can be negatively influenced by disease-related dysfunctions of patients T cells.4

A new option, off-the-shelf CAR T-cell products, may help solve these problems. These premade products are manufactured using allogeneic donor cells (instead of autologous cells from the patient), and they present immediate advantages to clinicians, such as immediate availability, opportunity for product standardization, and decreased cost.5

The advantages [include] being able to access the cellular therapy in real time, as opposed to autologous products that havetobe manufactured,Craig S. Sauter, MD, clinical director of the Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center in New York, New York, explained in an interview withTTO. This is particularly important for patients who are not responding to therapy, which is a current requirement for treatment with CAR T cells, he added.

Findings from a phase I trial (NCT01430390) in patients with relapsed or refractory B-cell malignancies showed that patients with non-Hodgkin lymphoma (NHL) experienced durable responses with an Epstein-Barr virusspecific cytotoxic lymphocyte CAR product derived from cells harvested from third-party donors (rather than from their more precisely matched stem cell donors). All 4 patients with NHL and a single patient with chronic lymphocytic leukemia, who were treated with third-party cells, remained disease free and alive at the time of analysis, with a median follow-up of over 2 years.6

The advantages [of this type of therapy are] that it eliminates the need for apheresis [and] shipping cellular products back and forth. [Instead, clinicians] have a pharmaceutical product on the shelf for access, Sauter, who was an author on the trial, said. Another notable product being investigated in clinical trials is UCART19, an allogeneic engineered anti CD19CAR T-cell product, which is being evaluated in the phase I CALM trial in adult patients with relapsed or refractory B-cell ALL (NCT02746952) and in the phase I PALL trial of pediatric patients with relapsed or refractory CD19-positive B-cell ALL (NCT02808442). Other off-the-shelf agents are described in theTABLE.5

Issues with inpatient CAR T-cell therapy administrationinclude high demands on health care resources and strain on patients and their families. Moving treatment to the outpatient setting has the potential to reduce this strain; however,clinicians taking over care of patients receiving CAR T-cell therapy must be prepared with the proper resources to identify and manage adverse events associated with therapy.

One of the most notable risks to patients receiving CAR T-cell therapy is cytokine release syndrome (CRS), a systemic inflammatory response that is characterized by increased serum levels of inflammatory cytokines, fever, hypotension, hypoxia, and organ dysfunction.4 [CAR T] can also lead to neurological events and can cause confusion and, in some patients, seizures,Carlos R. Bachier, MD, Director of Cellular Research at Sarah Cannon Cancer Center in Nashville, Tennessee, explained in an interview with TTO.

Regardless of the infusion setting, patients require close monitoring in the hours and days following therapy administration. A review byLucrecia Yez, PhD, MS, and colleagues stated that key criteria for treating patients in the outpatient setting include an educated caregiver and necessary infrastructure allowing for outpatient visits plus adequate emergency and intensive care unit (ICU) access. Patients followed as outpatients must be given twice-daily temperature checks for a minimum of 14 days following treatment and preferably extending up to 3 to 4 weeks following infusion. Anysigns of back pain, skin rash, dizziness, chills, shortness of breath, chest pain, tachycardia, or neurological events that may indicate neurotoxicity or signs of CRS must be reported immediately so treatment can begin as quickly as possible.7

Because of the risk of CRS and neurotoxicity, both FDA-approved agents are restricted under the Risk Evaluation and Mitigation Strategy, an FDA-mandated program that builds in caution for use of agents with serious safety concerns.8,9 Therefore, 2 doses of tocilizumab (Actemra), an interleukin (IL)-6 receptor antagonistthat was approved in 2017 for management of CRS associated with CAR T-cell therapy,1,4 should be on hand for each patient before the infusion of CAR T cells. Steroids have also demonstrated efficacy against CRS, but concernssurrounding CAR T-cell suppression with these agents have established them as a second-line choice after tociluzumab.9

Immune effector cellassociated neurotoxicity syndrome (ICANS) is a group of neurologic symptoms associated with treatments such as CAR T-cell therapy. Predisposing factors include younger age, higher tumor burden, high levels of pretreatment inflammation, and history of early or high-grade CRS. Treatments for complications of ICANS vary. Some centers may prescribe prophylactic antiepileptic medications, such as levetiracetam, to prevent seizures in patients with grade 2 or higher neurologic events. AntiIL-6 therapy can be considered in patients with concurrent CRS, but corticosteroids are the preferred regimen in those with neurotoxicity alone.9

In February of this year, the investigational CAR T-cell product liso-cel was granted priority review by the FDA for the treatment of adult patients with relapsed or refractory large B-cell lymphoma who had undergone at least 2 prior therapies.10 Investigators believe that liso-cel therapy may have a place in a broad range of patients and in the outpatient setting.11

It turns out liso-cel has a low incidence of [CRS and ICANS], and they occurred relatively late compared with other products, said Bachier. Because of this low incidence, the strategy was to deliver liso-cel in an outpatient setting.

The feasibility of liso-cel administration on an outpatient basiswas evaluatedby Bachier and colleagues, and the results were presented at the 2020 Transplantation & Cellular Therapy Meetings of the American Society for Transplantation and Cellular Therapy and the Center for International Blood & Marrow Transplant Research, held February 19 to 23, 2020, in Orlando, Florida.12

The authors analyzed data from 3 clinical trials of liso-cel, with a focus on the subset of participants who were treated as outpatients. The included trials were the phase I TRANSCEND-NHL-001 (NCT02631044) and phase II OUTREACH (NCT03744676) trials in patientswhohadundergone at least 2 lines of prior treatment, as well as the PILOT study (NCT03483103), which examined liso-cel as second-line therapy in patients who were ineligible for autologous hematopoietic stem cell transplant because of age, organ function, or ECOG performance score. All 3 studies allowed outpatienttreatment, with some patients receiving their therapy in the nonuniversity setting.

This clinical trial included sites that were not a part of a university but had experience treating patients for stem cell transplant, Bachier said. Some of these sites that participated were notyour traditional university centers that had traditionally been involved in the development of these therapies.

Much caution was required in order to maximize patient safety and treatment efficacy. The approach of doing CAR T-cell therapy, in general, in the outpatient setting requires a robust clinical ability of the centers, said coauthor David G. Maloney, MD, PhD, medical director of Cellular Immunotherapy at the Immunotherapy Integrated Research Center of Fred Hutchinson Cancer Research Center in Seattle, Washington, in an interview. We were able to get people safely to the hospital, and it was rare that you would have to do escalation of care when people were admitted. Most of the time, patients could bemanagedand wereout of the ICU, withrare exceptions. But again, you still have to have the wherewithal to get patients to the ICU pot entially for aggressive care if needed.

Results of the analysis of outpatient data from the 3 trials showed that rates of toxicity and response were similar to those previously reported for the entire patient cohort (both inpatients and outpatients) of the TRANSCEND-NHL-001 trial.

Based on these results, the indication is that you can deliver [liso-cel] in the outpatient setting and the outcomes are good compared with those treated in the inpatient setting, said Bachier. Aside from that, it also showed that liso-cel could be safely administered outside of university programs and in more community-based programs, most of them being aligned [with] or part of stem cell and bone marrow transplant programs.

When planning or setting up a CAR T-cell therapy outpatient program, investigators anticipate possible barriers to successfultreatment. The greatest barrier, according to Bachier, is access to physicians and staff who are knowledgeable and trained to manage toxicities related to CART-cell therapy. These therapies still should not, in my opinion, be delivered [by clinicians in] community centers that do not have the expertise to deliver the therapies safely, he said.

Maloney added that centers should be required to have the ability to triage patients 24/7 and allow for patients to be directly admitted to the hospital if needed. In the case of the analysis of outpatient data from the 3 liso-cel trials however, he said, We found that around 30% to 40% of patients did not actually ever require hospitalization, whichis quite interesting. Most of the 60% to 70% of patients who were hospitalized were admitted for fever, he added.

In addition, sites must gain accreditation and approval, Jain pointed out.

Every center that intends to do CAR T-cell therapy is first approved by each of the companies [that manufacturethese agents], Jain said. The centers also have to be approved by FACT [Foundation for the Accreditation of Cellular Therapy], which is the same organization that approves centers for allogeneic stem cell transplant. These are some of the largest things that a center needs to go through, which takes care of things like developing standard practices and other guidelines to make sure that these [therapies] are used safely and appropriately.

As investigators and oncologists explore the feasibility of moving CAR T-cell therapy into more settings, 2 questions arise: What settings have on this therapy?

What type of training and skills do clinicians need? Like other clinicians, Sauter has concerns about new allogeneic cellular therapies,andhe hopes future research will focus on mitigating these challenges. The concern would be that these are not autologous products and there is the risk of rejection from the host immune system, he said. Strategies to circumnavigate that risk are at the forefront of investigationin off-the-shelf CAR T cells.

The research is not stopping with CAR T-cell therapy,though. Were seeing a lot of new molecules coming in that will be challenging the roles of CAR T cells, [such as] specific antibodies, which may even work in cases of CAR T-cell failure, Maloney said. We are still learning how to make those more effective and safer.

References:

1. FDA approves tisagenlecleucel for B-cell ALL and tocilizumab for cytokine releasesyndrome.FDAwebsite.PublishedAugust30,2017.AccessedApril14, 2020. bit.ly/2RC4eQ8

2. FDA approves axicabtagene ciloleucel for large B-cell lymphoma. FDA website. Published October 18, 2017. Accessed April 14, 2020. bit.ly/2yYIQOp

3. FDA approves tisagenlecleucel for adults with relapsed or refractory large B-cell lymphoma. FDA website. Published May 1, 2018. Accessed April 14, 2020. bit.ly/34zPoi8

4. Rafiq S, Hackett CS, Brentjens RJ. Engineering strategies to overcome the current roadblocks in CAR T cell therapy. Nat Rev Clin Oncol. 2020;17(3):147167. doi: 10.1038/s41571-019-0297-y

5. DepilS,DuchateauP,GruppSA,MuftiG,PoirotL.Off-the-shelfallogeneic CAR T cells: development and challenges. Nat Rev Drug Discov. 2020;19(3):185199. doi: 10.1038/s41573-019-0051-2

6. Curran KJ, Sauter CS, Kernan CS, et al. Durable remission following off-theshelf chimeric antigen receptor (CAR) T-cells in patients with relapse/refractory (R/R) B-cell malignancies. Presented at: 2020 Transplantation & Cellular Therapy Meetings; February 19-23, 2020; Orlando, FL. Abstract 120. bit.ly/2ufDYCu

7. Yez L, Snchez-Escamilla M, Perales MA. CAR T cell toxicity: current managementandfuturedirections. Hemasphere.2019;3(2):e186.doi:10.1097/ HS9.0000000000000186

8. Risk evaluation and mitigation strategies | REMS. FDA website. Updated August 8, 2019. Accessed April 14, 2020. bit.ly/2ykhLVt

9. JainT,BarM,KansagraAJ,etal.UseofchimericantigenreceptorTcell therapy in clinical practice for relapsed/refractory aggressive B cell non-Hodgkin lymphoma: an expert panel opinion from the American Society for Transplantation and Cellular Therapy. Biol Blood Marrow Transplant. 2019;25(12):2305-2321. doi: 10.1016/j.bbmt.2019.08.015

10. U.S. Food and Drug Administration (FDA) accepts for Priority Review Bristol-Myers Squibbs Biologics License Application (BLA) for lisocabtagene maraleucel (liso-cel) for adult patients with relapsed or refractory large B-cell lymphoma. News release. Bristol-Myers Squibb; February 12, 2020. Accessed April 15, 2020. bit.ly/37ruQbs

11. Helwick C. Strong activity shown for lisocabtagene maraleucel CAR T-cell therapy in aggressive large B-cell lymphoma. ASCO Post website. Published February 25, 2020. Accessed April 15, 2020. bit.ly/3eoD0pT

12. Bachier CR, Palomba ML, Abramson JA, et al. Outpatient treatment with lisocabtagene maraleucel (liso-cel) in 3 ongoing clinical studies in relapsed/refractory (R/R) large B cell non-Hodgkin lymphoma (NHL), including second-line transplant noneligible (TNE) patients: TRANSCEND NHL 001, OUTREACH, and PILOT. Presented at: 2020 Transplantation & Cellular Therapy Meetings; February 19-23, Orlando, FL. Abstract 29. bit.ly/37I7DC9

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Protocol Management, Off-the-Shelf Therapies Help Bring CAR T Into More Settings - Targeted Oncology

Skin Stem Cells Comments Off on Protocol Management, Off-the-Shelf Therapies Help Bring CAR T Into More Settings – Targeted Oncology | May 13th, 2020

Families stuck at home during lockdown are being warned about a common but dangerous garden weed which can leave children - and adults - with burns and blisters.

Giant Hogweed has been dubbed Britain's most dangerous plant because of the horrific burns it inflicts on anyone who touches it - especially children - its set to thrive in the weeks ahead thanks to recent weather conditions.

The plant grows wild as well as in gardens and is becoming common the in the UK, but when it comes into contact with skin it causes a painful blistering rash.

The recent warm weather and plenty of rain after a mild winter has created the perfect conditions for this hazardous plant to thrive.

The hogweed looks relatively attractive and is part of the carrot family, but contains toxic chemicals.

Giant hogweed, or Heracleum mantegazzianum, is a weed which has dangerous effects on human health.

Growing up to five metres tall, its sap contains toxic chemicals which react with light when in contact with human skin, causing blistering within 48 hours.

Effectively it prevents the skin from protecting itself from sunlight, which can lead to very bad sunburn and scarring.

It's actually pretty and looks a bit like cow parsley. It's got green stem spotted with dark red which varies from 38 cm in diameter. Each dark red spot on the stem surrounds a hair, and large, coarse white hairs occur at the base of the leaf stalk.

It produces white flowers clustered in an umbrella-shaped head that is up to 80 cm in diameter across its flat top.

Colette Jones, Chairwoman of Friends of Close Park where Giant Hogweed was spotted, told The Bolton News : "Children are drawn to them because they grow so tall. They break them off to use them as sticks not realising how dangerous they are."

Exposure can result in blisters, long-lasting scars, and - if it comes in contact with eyes - blindness.

The blisters will form within 48 hours - scars can last for years.

It can also cause cause long-term sunlight sensitivity in people who touch it.

Black or purplish scars may be left on your skin for years after.

Medical professionals say you should cover the affected area, and wash it with soap and water.

The blisters heal very slowly and can develop into phytophotodermatitis, a type of skin rash which flares up in sunlight.

If you feel unwell or have a severe reaction you are advised to see a doctor.

Giant hogweed was among the foreign plants introduced to Britain in the 19th century as an ornamental plant, but it's now widespread throughout the British Isles.

It's invasive, which means that it chokes off other plants and can reduce wildlife in an area.

The plant is native to the Caucasus region and Central Asia.

The Wildlife and Countryside Act 1981 made it illegal to plant or cause giant hogweed to grow in the wild.

It is found in most of the UK, along footpaths and riverbanks though it also grows in places like parks, cemeteries and wasteland.

The sap of giant hogweed has chemicals which are toxic to humans and cause photosensitivity. The sap is phototoxic and can cause phytophotodermatitis.

When they touch skin, they effectively remove any protection against the sunlight causing severe skin inflammations.

Children have been hospitalised and suffered third-degree burns to their skin before.

The severe reaction to the plant is caused by the presence of linear derivates of furanocomarin in the plant's leaves, seeds, flowers, stems and roots.

The chemicals enter the cells' nucleus forming bonds with DNA and cause cells to die.

The RHS advises caution when removing the plant - cover arms and legs, and ideally wear a face mask when working on it.

Cut plant debris, contaminated clothing and tools are potentially hazardous too.

Wash any skin that comes in contact with the plant immediately.

The Wildlife and Countryside Act 1981 made it illegal to plant or cause giant hogweed to grow in the wild. Giant hogweed clearances are carried out to remove the plant.

More here:
Warning over common garden weed that causes horrific burns - Cambridgeshire Live

Skin Stem Cells Comments Off on Warning over common garden weed that causes horrific burns – Cambridgeshire Live | May 13th, 2020