Archive for the ‘Bone Marrow Stem Cells’ Category

Press Release

Gosselies, Belgium, 11February 2020, 7am CET BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the leading biotech company focused on the development of innovative cell and biological therapies to address high unmet medical needs in orthopaedics and bone diseases, announces that the Company will today present at the Annual Meeting of the Orthopaedic Research Society (ORS), in Phoenix (Arizona), USA.

The Annual ORS Meeting is the yearly summit organised by the international Orthopaedic Research Society, gathering scientists, clinicians and entrepreneurs to advance musculoskeletal research and orthopaedic care. In the oral presentation, Bone Therapeutics will highlight additional preclinical in vitro and in vivo results demonstrating the potent osteogenic properties of its allogeneic bone-forming cell therapy platform, ALLOB, to promote bone-formation and improve fracture healing in relevant models.

ALLOB is the Companys allogeneic product that consists of human bone-forming cells derived from cultured bone marrow mesenchymal stem cells of healthy adult donors, and is manufactured through a proprietary, scalable production process. ALLOB successfully completed two Phase II studies in two indications and the Company has started the CTA submission procedure with the regulatory authorities in Europe to start the PhaseIIb clinical trial in patients with difficult-to-heal tibial fractures.

Presentation Details:

Title: ALLOB, A Ready-to-use and Injectable Cryopreserved Allogenic Cell Therapy Product Derived from Bone Marrow Mesenchymal Stem Cells, Displays Potent Osteoinductive and Osteogenic Properties, Leading to Enhanced Bone Fracture HealingSpeaker: Sandra Pietri, PhD Associate Director R&D, Bone TherapeuticsSession: Podium Session 58 Bone Cell Signaling and TreatmentsDate: Tuesday, 11 February 2020Time: 8:00am 9:00am MST (4pm 5 pm CET)Location: Room West 301D, Phoenix Convention Center, Phoenix, Arizona, USA

About Bone Therapeutics

Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopedics and bone diseases. The Company has a broad, diversified portfolio of bone cell therapies and an innovative biological product in later-stage clinical development, which target markets with large unmet medical needs and limited innovation.

Bone Therapeutics is developing an off-the-shelf protein solution, JTA-004, which is entering Phase III development for the treatment of pain in knee osteoarthritis. Positive Phase IIb efficacy results in patients with knee osteoarthritis showed a statistically significant improvement in pain relief compared to a leading viscosupplement. The clinical trial application (CTA) to start the pivotal Phase III program has been submitted to the regulatory authorities in Europe and the trial is expected to start in Q1 2020.

Bone Therapeutics other core technology is based on its cutting-edge allogeneic cell therapy platform (ALLOB) which can be stored at the point of use in the hospital, and uses a unique, proprietary approach to bone regeneration, which turns undifferentiated stem cells from healthy donors into bone-forming cells. These cells can be administered via a minimally invasive procedure, avoiding the need for invasive surgery, and are produced via a proprietary, scalable cutting-edge manufacturing process. Following the promising Phase IIa efficacy and safety results for ALLOB, the Company has started the CTA submission procedure with the regulatory authorities in Europe to start the Phase IIb clinical trial with ALLOB in patients with difficult-to-heal fractures, using its optimized production process.

The ALLOB platform technology has multiple applications and will continue to be evaluated in other indications including spinal fusion, osteotomy and maxillofacial and dental applications.

Bone Therapeutics cell therapy products are manufactured to the highest GMP (Good Manufacturing Practices) standards and are protected by a broad IP (Intellectual Property) portfolio covering ten patent families as well as knowhow. The Company is based in the BioPark in Gosselies, Belgium. Further information is available at http://www.bonetherapeutics.com.

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Contacts

Bone Therapeutics SAMiguel Forte, MD, PhD, Chief Executive OfficerJean-Luc Vandebroek, Chief Financial OfficerTel: +32 (0) 71 12 10 00investorrelations@bonetherapeutics.com

International Media Enquiries:Consilium Strategic CommunicationsMarieke VermeerschTel: +44 (0) 20 3709 5701bonetherapeutics@consilium-comms.com

For French Media and Investor Enquiries:NewCap Investor Relations & Financial CommunicationsPierre Laurent, Louis-Victor Delouvrier and Arthur RouillTel: + 33 (0)1 44 71 94 94bone@newcap.eu

Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors` current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such person`s officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

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Bone Therapeutics to present preclinical data on the osteogenic properties of ALLOB in bone repair at the Annual Meeting of the Orthopaedic Research...

'This is the beginning of my new life'

I thought my cancer diagnosis was a death sentence, said Lynnette Williams-Briggs, 60, of Seaford, Delaware, who was diagnosed with advanced B-cell lymphoma in 2018.

Briggs cancer is now in complete remission thanks to successful chimeric antigen receptor CAR-T cell therapy she received in August atChristianaCaresHelen F. Graham Cancer Center & Research InstitutesBone Marrow and Stem Cell Transplant Program.

I can breathe again. This is the beginning of my new life, Williams-Briggs said following the treatment that restored her hope for a second chance at life.

She was the first patient to receive CAR-T cell therapy in Delaware. A second patient was treated in December 2019, and doctors are preparing several more patients for CAR-T cell transplants in coming weeks.

The U.S. Food and Drug Administration has approved CAR-T cell therapy to treat patients like Williams-Briggs with highly resistant, B-cell blood cancers, for whom other available options have failed.

CAR-T cell therapy is only available at select cancer centers with specialized expertise in cellular therapies that are recognized for quality by the Foundation for the Accreditation of Cellular Therapy.

The Graham Cancer Centers Bone Marrow and Stem Cell Transplant Program is the only one in Delaware that is certified to treat adult patients with advanced B-cell lymphomas and children and young adults (to age 25) with acute lymphoblastic leukemia, using an FDA-approved drug.

CAR-T cell therapy is highly personalized medicine that attempts to use the bodys natural defenses to fight against cancer. The transplant team extracts millions of T cells, from the patients bloodstream, using a specialized blood filtration process called leukapheresis. The collected T cells are flash-frozen and sent to a lab for reprogramming, and then later infused back into the patient using a process similar to a blood transfusion.

The therapy is considered a living drug with potential benefits that could last for years.

When we first met Ms. Williams-Briggs, her cancer had progressed rapidly despite a third round of chemotherapy, so we knew we had to move quickly, said Graham Cancer Center Hematologist Peter Abdelmessieh, D.O. He worked closely with the bone marrow/stem cell transplant team and Graham Cancer Center leadership over the course of just eight months to develop the CAR-T cell therapy program.

It was truly a team effort to bring CAR-T cell therapy to our community so quickly, Dr. Abdelmessieh said.

CAR-T cell therapy has been extremely effective for many patients like Williams-Briggs, whose PET scan at 90 days confirmed her remission.

The supercharged T cells Williams-Briggs received were genetically modified in the lab to sprout new surface tools that improve their ability to recognize, latch onto and destroy other cells (including cancer cells) that express a specific antigen called CD19. These reprogrammed cells continue to multiply in the body after treatment, remaining on guard to seek and destroy any new cancers that might develop.

With continued success in increasing numbers of patients, it is conceivable that in the not too distant future, CAR-T cell therapy could become the new standard of care, replacing chemotherapy and stem cell transplants for many cancers, Dr. Abdelmessieh said.

The extended recovery period for CAR-T cell therapy is generally two to three months. After the infusion, patients may spend up to three weeks in the hospital to monitor treatment response and any side effects.

During the first 30 days after leaving the hospital, patients are required to remain close to the treatment center for regular follow-up care.

The ability to offer potentially life-saving CAR-T cell therapy is one more reason our patients need not travel further than the Graham Cancer Center for state-of-the-science cancer treatment, said Nicholas J. Petrelli, M.D., Bank of America medical director of the Helen F. Graham Cancer Center & Research Institute.

The Bone Marrow and Stem Cell Transplant Program is an outstanding example of how well our clinical teams work together to drive innovation in patient care.

Although patients normally do not experience the side effects associated with chemotherapy, such as nausea, vomiting or hair loss, CAR-T cell therapy is not without risks. A common side effect, which Williams-Briggs also experienced, is cytokine release syndrome. This is an inflammatory condition that causes flu-like symptoms that may be mild or severe.

The transplant team responded quickly to manage her symptoms while she received expert care on the Bone Marrow Transplant and Oncology unit at Christiana Hospital.

From the moment I first met with my transplant team, I felt like I was part of one big loving family that extended beyond my own loved ones, Williams-Briggs said.

Dr. Abdelmessieh and my ChristianaCare family gave me hope to keep fighting when I really didnt think I would make it. I would have driven anywhere to get life-saving treatment, but I am thankful that I did not have to. I found my miracle closer to home.

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First CAR-T cell cancer therapy patient in Delaware - Dover Post

A research team featuring an expert from City University of Hong Kong (CityU) has developed a novel dual approach for the first time for concurrently rejuvenating both the cardiac muscle and vasculature of the heart by utilising two types of stem cells. The results give hope for a new treatment for repairing myocardial infarction (MI) heart.

Dr Ban Ki-won, Assistant Professor of the Department of Biomedical Sciences and his research team, including researchers from Konkuk University, The Catholic University of Korea, Pohang University of Science and Technology and T&R Biofab in South Korea, have conducted the first study of two distinct stem cell effects for cardiac repair. The two major types of stem cells employed are human bone marrow derived mesenchymal stem cells (hMSCs) and cardiomyocytes derived from human induced pluripotent stem cells (hiPSC-CMs). The research findings have been published in Nature Communications in a paper titled Dual stem cell therapy synergistically improves cardiac function and vascular regeneration following myocardial infarction.

Both cardiac muscles and vasculatures are severely damaged following MI, and so the therapeutic strategies should focus on comprehensive repair of both at the same time. But the current strategies only focus on either one, Dr Ban said.

Dr Ban said that, with limited therapeutic options for severe MI and advanced heart failure, a heart transplant was the last resort. However, such an operation is very risky, costly and subject to limited supply of suitable donors. Therefore, stem cell-based therapy has emerged as a promising therapeutic option.

In the study, the hiPSC-CMs were injected directly into the border zone of the rats heart, while the hMSCs-loaded patch was implanted on top of the infarct area, like a bandage. The results showed that this dual approach led to a significant improvement of cardiac function and an enhancement of vessel formation on a MI heart.

We believe this novel dual approach can potentially provide translational and clinical benefit to the field of cardiac regeneration. Based on the same principle, the protocol may also be utilised for repairing other organs including the brain, liver and pancreas in which multiple types of stem cells co-exist, Dr Ban added.

The research team is working on follow-up studies in larger animal models such as pigs. The patent application for this research result has been submitted.

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First dual stem cell therapy jointly developed by City University of Hong Kong brings new hope for cardiac repair - QS WOW News

NEW YORK, Feb. 11, 2020 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics, Inc., (BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced that the Company recently held a high level meeting with the U.S. Food and Drug Administration (FDA) to discuss potential NurOwn regulatory pathways for approval in ALS. Repeated intrathecal administration of NurOwn (autologous MSC-NTF cells) is currently being evaluated in a fully enrolled Phase 3 pivotal trial in ALS (NCT03280056).

In the planned meeting with senior Center for Biologics Evaluation and Research (CBER) leadership and several leading U.S. ALS experts, the FDA confirmed that the fully enrolled Phase 3 ALS trial is collecting relevant data critical to the assessment of NurOwn efficacy. The FDA indicated that they will look at the "totality of the evidence" in the expected Phase 3 clinical trial data. Furthermore, based on their detailed data assessment, they are committed to work collaboratively with BrainStorm to identify a regulatory pathway forward, including opportunities to expedite statistical review of data from the Phase 3 trial.

Both the FDA and BrainStorm acknowledged the urgent unmet need and the shared goal of moving much needed therapies for ALS forward as quickly as possible.

This is a key turning point in ourworktowardprovidingALSpatientswith a potential new therapy,said ChaimLebovits, President and CEO ofBrainStorm. We commend the FDA foritscommitmentto the ALS communityandtofacilitating the development, and we ultimately hope, the approvalofNurOwn.The entire BrainStorm team is grateful for the ongoing and conscientious collaboration in the quest to beat ALS.

Ralph Kern, MD, MHSc, Chief Operating Officer and Chief Medical Officer, stated, The entire team at BrainStorm has collectively worked to ensure that we conduct the finest, science-based clinical trials. We had the opportunity to communicate with Senior Leadership at the FDA and discuss how we can work together to navigate the approval process forward along a novel pathway. We appreciate their willingness and receptiveness to consider innovative approaches as we all seek to better serve the urgent unmet medical needs of the ALS community.

Brian Wallach, Co-Founder of I AM ALS stated: There is nothing more important to those living with ALS than having access to therapies that effectively combat this fatal disease. We have been working with BrainStorm for months now because we believe that NurOwn is a potentially transformative therapy in this fight. We were privileged to represent the patient voice at this meeting and are truly grateful to the company and the FDA for this critical agreement. This is a truly important moment of hope and we look forward to seeing both the Phase III data and the hopeful approval of NurOwn as soon as is possible.

About NurOwnNurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

About BrainStorm Cell Therapeutics Inc.BrainStorm Cell Therapeutics Inc.is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwnCellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement as well as through its own patents, patent applications and proprietary know-how. Autologous MSC-NTF cells have received Orphan Drug status designation from theU.S. Food and Drug Administration(U.S.FDA) and theEuropean Medicines Agency(EMA) in ALS. BrainStorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from theCalifornia Institute for Regenerative Medicine(CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S.FDAapproval of autologous MSC-NTF cells in ALS. BrainStorm received U.S.FDAclearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) inDecember 2018and has been enrolling clinical trial participants sinceMarch 2019. For more information, visit the company'swebsite.

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Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839sean.leous@icrinc.com

Or

Katie Gallagher | Account Director, PR and MarketingLaVoieHealthScience Strategic CommunicationsO: 617-374-8800 x109M: 617-792-3937kgallagher@lavoiehealthscience.com

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BrainStorm Cell Therapeutics and FDA Agree to Potential NurOwn Regulatory Pathway for Approval in ALS - Yahoo Finance

Methionine is an amino acid found in meat, eggs, and dairy. Its absorbed by T-cells that are part of our immune system. Those cells are also believed to be the immune cells that attack our myelin, creating the nerve damage that results in multiple sclerosis.

In this study, mice eating less methionine had a reduced number of a certain type of T-cell, which led to a delay in disease onset and progression. The researchers believe reducing methionine intake can actually dampen the immune cells that cause disease, leading to better outcomes.

Changing a persons diet to reduce the amount of methionine (amino acid found in food) could delay the development and progression of inflammatory and autoimmune disorders, including multiple sclerosis (MS).

That finding was described in the study Methionine Metabolism Shapes T Helper Cell Responses through Regulation of Epigenetic Reprogramming, published recently in the journal Cell Metabolism.

Click here to read the full story.

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Unlike hematopoietic stem cell transplants, in which stem cells are removed from a patients bone marrow and later infused back into the bloodstream, mesenchymal stem cell transplants (MSCT) collect those stem cells from the patients spinal column and return them there. This study concludes that MSCT is safe and that cells delivered into the spinal cord produced a significantly slower disease progression rate than did cells delivered into the bloodstream.

Transplanting patients ownmesenchymal stem cellsis a safe therapeutic approach and can delay disease progression in people with MS, a meta-analysis review shows.

The study also showed that cells transplanted to the spinal cord (intrathecal injection) were associated with significantly slower disease progression rates, compared to cells delivered into the bloodstream.

Click here to read the full story.

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Why do neurologists often use spinal taps when determining whether someone has MS? This study provides one of the reasons.

People with MS have a more diverse set of immune cells in their cerebrospinal fluid (CSF), the fluid that bathes the central nervous system, but no such diversity is seen in their blood, a study reports. Instead, MS causes changes in the activation of immune cells in the blood.

The distinct set of immune cells in MS patients CSF shows enrichment of pro-inflammatory cells that promote disease severity in MS mouse models.

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Heres encouraging news about a possible treatment that can lower the number of brain lesions in someone with MS. Keep in mind this is only a Phase 2 trial. A Phase 3 trial isnt expected until later this year. However, a news release from research sponsor Sanofisays, This molecule may be the first B-cell-targeted MS therapy that not only inhibits the peripheral immune system, but also crosses the blood-brain barrier to suppress immune cells that have migrated into the brain.

The experimental BTK inhibitor SAR442168 showed an acceptable safety profile and met its primary endpoint a significant reduction in the number of new lesions visible on a brain imaging scan in a Phase 2 trial in people with MS, study results show.

SAR442168, formerly known as PRN2246, is an oral, small molecule being co-developed by Principia Biopharmaand Sanofi Genzyme. It works by inhibiting Brutons tyrosine kinase (BTK), a protein important for the proliferation of immune cells, particularly B-cells. By blocking BTK, it is expected that SAR442168 can reduce inflammation that damages the nervous system in people with MS.

Click here to read the full story.

Did you know that some of my columns from The MS Wire are now available as audio briefings? You can listen to them here.

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Note: Multiple Sclerosis News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Multiple Sclerosis News Today or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to multiple sclerosis.

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

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MS News that Caught My Eye Last Week: Methionine, MSCT, Spinal... - Multiple Sclerosis News Today

Research into alternative stem cell sources has identified urine derived renal progenitor cells (UdRPCs) as a possible option for use in regenerative kidney therapies in the future.

Scientists have demonstrated their protocol for the reproducible isolation of kidney stem cells from human urine. These urine derived renal progenitor cells (UdRPCs) could be used to provide easier access to stem cells for regenerative kidney therapies and modelling diseases for R&D.

A shortage of donor organs and the risks and pain associated with bone marrow stem cell extractions and third trimester amniotic fluid collection have encouraged researchers to find alternative sources of stem cells. According to scientists, several laboratories have indicated urine could be an alternative source, at least for kidney stem cells, so the researchers from Heinrich Heine University-Duesseldorf (HHU) Germany,set out to complete a comprehensive molecular and cellular analysis of these cells.

UdRPCs should be considered as the choice of renal stem cells for facilitating the study of nephrogenesis, nephrotoxicity, disease modelling and drug development

Their study, published in Scientific Reports, revealed that UdRPCs isolated from ten individuals express both markers typically seen in bone marrow-derived mesenchymal stem cells (MSCs) and renal stem cells. The renal stem cell markers, according to the paper, allow UdRPCs to be differentiated into cell types present in the kidney, eg, podocytes and the proximal and distal tubules. The study also showed that these progenitor cells have similar properties to amniotic fluid-derived stem cells (AFCs).

Wasco Wruck, bioinformatician and co-author of the study, said: It is amazing that these valuable cells can be isolated from urine and comparing all the genes expressed in UdRPCs with that derived from kidney biopies we could confirm their renal and renal progenitor cell properties and origin.

According to Martina Bohndorf, a study co-author, UdRPCs can also be easily and efficiently reprogrammed into induced pluripotent stem cells using a non-viral integration-free and safe method.

Dr James Adjaye, study senior author and professor at the Institute for Stem Cell Research and Regenerative Medicine (ISRM) in the medical faculty of HHU, revealed that one of the most promising options in the near future is the use of transplantable renal stem cells (UdRPCs) for treatment of kidney diseases as a complementary option to kidney organs. He concluded that human UdRPCs should be considered as the choice of renal stem cells for facilitating the study of nephrogenesis, nephrotoxicity, disease modelling and drug development.

Read more:
Kidney stem cells isolated from urine could be regenerative therapies - Drug Target Review

Anemia also known as iron-poor blood is a condition that develops when either the blood doesn't have enough red blood cells or the concentration of hemoglobin in red blood cells is very low. Hemoglobin is the iron-containing protein in red blood cells that carries oxygen from the lungs to the rest of the body. When there are fewer red blood cells than normal or low levels of hemoglobin, the body doesn't get enough oxygen-rich blood for healthy functioning, which is what causes the symptoms of anemia.

Anemia is the most common blood disorder in the United States, affecting nearly 3 million Americans, according to the Centers for Disease Control and Prevention (CDC).

The term anemia is a broad one that represents several hundred different conditions some of them mild and treatable, others that are quite serious, said Dr. Nancy Berliner, chief of hematology at Brigham and Women's Hospital in Boston. There are three reasons that people are anemic, Berliner said: Either their body can't make enough red blood cells, something is destroying the red blood cells faster than their body can make news ones or blood loss (from menstrual periods, colon polyps or a stomach ulcer, for example) is greater than blood cell production.

There are more than 400 different types of anemia, according to the Pacific Heart, Lung & Blood Institute. Here are a few of the more common and better understood types:

Iron-deficiency anemia: The most common form of anemia is caused by low-iron levels in the body. Humans need iron to make hemoglobin, and most of that iron comes from dietary sources. Iron-deficiency anemia can result from a poor diet or from blood loss through menstruation, surgery or internal bleeding.

Pregnancy also increases the body's need for iron because more blood is needed to supply oxygen to the developing fetus, which may quickly drain the body's available iron stores, leading to a deficit. Problems absorbing iron from food because of Crohn's disease or celiac disease can also result in anemia.

Vitamin deficiency anemia: Besides iron, the body also needs two different B-vitamins folate and B12 to make enough red blood cells. Not consuming enough B12 or folate in the diet or an inability to absorb enough of these vitamins can lead to deficient red blood cell production.

Sickle cell anemia or sickle cell disease (SDC): This inherited disease causes red blood cells to become crescent-shaped rather than round. Abnormally shaped red cells can break apart easily and clog small blood vessels, resulting in a shortage of red blood cells and episodes of pain, according to the Mayo Clinic. People become chronically anemic because the sickle-shaped red cells are not pliable and can't get through blood vessels to deliver oxygen, Berliner said.

SDC occurs most often in people from parts of the world where malaria is or was common, according to the CDC; the sickle cell trait may provide protection against severe forms of malaria. In the U.S., SDC affects an estimated 100,000 Americans.

Thalassemia: Thalassemia is an inherited blood disorder that results in lower-than-normal levels of hemoglobin. This type of anemia is caused by genetic mutations in one or more of the genes that control the production of hemoglobin, according to the National Heart, Lung & Blood Institute (NHLBI).

Aplastic anemia: Aplastic anemia is a rare, life-threatening condition that develops when bone marrow stops making enough new blood cells, including red cells, white cells and platelets.

Aplastic anemia may be caused by radiation and chemotherapy treatments, which can damage stem cells in bone marrow that produce blood cells. Some medications, exposure to toxic chemicals like pesticides, viral infections and autoimmune disorders can also affect bone marrow and slow blood cell production.

Hemolytic anemias: This disorder causes red blood cells to be destroyed faster than bone marrow can replace them. Hemolytic anemias may be caused by infections, leaky heart valves, autoimmune disorders or inherited abnormalities in red blood cells, according to the American Society of Hematology.

Anemia of inflammation: Also called anemia of chronic disease, anemia of inflammation commonly occurs in people with chronic conditions that cause inflammation. This includes people with infections, rheumatoid arthritis, inflammatory bowel disease, chronic kidney disease, HIV/AIDS and certain cancers, according to the National Institute of Diabetes and Digestive and Kidney Diseases.

When a person has a disease or infection that causes inflammation, the immune system responds in a way that changes how the body works, resulting in anemia. For example, inflammation suppresses the availability of iron, so the body may not use and store the mineral normally for healthy red blood cell production, Berliner said. Inflammation may also stop the kidneys from producing a hormone that promotes red blood cell production.

The risk for anemia is higher in people with a poor diet, intestinal disorders, chronic diseases and infections. Women who are menstruating or pregnant are also prone to the disorder.

The risk of anemia increases with age, and about 10% to 12% of people over 65 are anemic, Berliner said. But the condition is not a normal part of aging, so the cause should be investigated when it's diagnosed, she said. Older adults may develop anemia from chronic diseases, such as cancer, or iron-deficiency anemia from abnormal bleeding.

According to NHLBI, the following types of people have an increased risk of developing anemia:

Mild forms of anemia may not cause any symptoms. When signs and symptoms of anemia do occur, they may include the following, according to the NHLBI:

The first test used to diagnose anemia is a complete blood count, which measures different parts and features of the blood: It shows the number and average size of red blood cells, as well as the amount of hemoglobin. A lower-than-normal red blood cell count or low levels of hemoglobin indicate anemia is present.

If more testing is needed to determine the type of anemia, a blood sample can be examined under a microscope to check for abnormalities in the size and shape of the red cells, white cells and platelets.

Related: This man's taste buds disappeared because of a blood condition

The treatment of anemia depends on the specific type of anemia, Berliner said, and anemias caused by nutritional deficiencies respond well to changes in diet. People with iron-deficiency anemia may need to take supplemental iron for several months or longer to replenish blood levels of the mineral. Some people, especially pregnant women, may find it hard to take iron because it causes side effects, such as an upset stomach or constipation, Berliner said.

For vitamin-deficiency anemias, treatment with B12 or folate from supplements (or a B12 shot) and foods, can improve levels of these nutrients in the blood, Berliner said.

Serious problems, such as aplastic anemia, which involves bone marrow failure, may be treated with medications and blood transfusions. Severe forms of thalassemia might need frequent blood transfusions.

Treatment for sickle cell anemia may include pain medications, blood transfusions or a bone marrow transplant.

Additional resources:

This article is for informational purposes only, and is not meant to offer medical advice.

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Anemia: Causes, symptoms and treatment - Livescience.com

Mesoblast Tenders Completed Biologics License Application

Mesoblast Limited (MESO) announced that it has filed a completed Biologics License Application (BLA) with the United States Food and Drug Administration for its lead allogeneic cell therapy Ryoncil. The therapy is aimed at treating children with steroid-refractory acute graft versus host disease (SR-aGVHD).

Mesoblast submitted the final module of its rolling BLA submission on January 31, 2020. This module covers various aspects related to manufacturing and quality control. The drug candidate currently has Fast Track designation assigned to it and on the basis of this tag, the company is now seeking the FDA to carry out Priority Review of its BLA.

Subject to the approval of the therapy, the company is looking to launch it in the US markets in 2020. CEO Dr. Silviu Itescu said, "This is a major corporate milestone for Mesoblast." The company is expected to use the insights gained from its Temcell product in Japan for the marketing of Ryoncil.

Acute Graft versus Host disease affects nearly 50 percent of patients given an allogeneic Bone Marrow transplant. It is estimated that nearly 30,000 patients undergo bone marrow transplants worldwide. The mortality rate for patients suffering from actual GVHD is close to 90 percent. Currently, there is no FDA approved treatment for this in the United States for children under 12.

Ryoncil has been tested on 309 children suffering from SR-aGVHD during three different studies. It was employed as salvage therapy on 241 children with SR-aGVHD (80% Grade C/D) who failed institutional standard of care. It has also been tested as first line therapy for an open label Phase 3 trial in 55 children with SR-aGVHD. RYONCIL, is an investigational therapy comprising culture-expanded mesenchymal stem cells. These stem cells are taken from the bone marrow of an unrelated donor. The drug is administered to patients as intravenous infusions.

Mesoblast specializes in developing allogeneic cellular medicines. The company uses its proprietary cell therapy technology platform for research and development purpose. It has strong drug pipeline with products such as Remestemcel-L, Revascor, MPC-06-ID and MPC-300-IV. Revascor and MPC-06-ID have completed patient enrollment for its Phase 3 trials. The former drug candidate is aimed at treating advanced chronic heart failure while the latter is targeted at treating chronic low back pain caused by degenerative disc disease. The companys Temcell and Alofisel drugs are already approved in Japan and Europe, respectively.

Mesoblast has posted strong operative results as well. The company had reported 46 percent growth in its revenue during the first quarter of 2020. Mesoblast ended the quarter with $34.5 million in cash while its pro forma cash in hand stood at $100 million. The company also reported its strategic partnership with Grunenthal, which entitles Mesoblast to receive up to $150 million in upfront and milestone payments. The collaboration will also result in commercialization milestone payments. Such milestone payments have the potential to cross $1 billion mark.

Mesoblast stock has performed strongly in the market. The stock has charted over 200 percent in the past 12 months. Currently, it is trading close to its 52-week high of $10.88 and has potential to maintain its positive trajectory as the company forges ahead with its research and development activities and marketing efforts.

Waters Corporation (WAT) reported its fourth-quarter earnings and provided guidance for 2020. The company registered $716 million in revenue for the fourth quarter, in line with the revenue of $715 million it had reported for the corresponding quarter of the previous year. Its GAAP diluted earnings per share stood at $3.12 per share, up from $2.46 on year-on-year basis.

For the full fiscal year 2019, the companys revenue stood at $2.4 billion, down 1 percent from $2.42 billion in revenue it had earned in fiscal year 2018. The EPS for the fiscal year stood at $8.69, up from $7.65 for the previous year. The non GAAP EPS also increased from $8.29 to $8.99 for fiscal year 2019.

The company reported that its sales in both the pharmaceuticals and industrial market declined by 1 percent. However, its sales into the government and academic market grew 8 percent. Chris OConnell, Chairman and Chief Executive Officer of Waters Corporation, said, We were encouraged by the increasing impact in the fourth quarter of our new products launched during 2019.

While its full-year and fourth-quarter numbers were strong, the company provided rather lackluster guidance for fiscal year 2020. Waters Corporation expects its full-year revenue to increase by 1 percent to 3 percent. Its non GAAP EPS will likely remain between $9.15 and $9.40, lower than consensus estimate of $1.75. For its first quarter, Waters Corporations non GAAP EPS for the first quarter is expected to be in the range of $1.55 and $1.65. The consensus estimate for non GAAP EPS guidance was at $1.75.

EyePoint Pharmaceuticals (EYPT) reported its new exclusive licensing deal with Equinox Science. The deal involves the development of vorolanib for treating wet age-related macular degeneration, retinal vein occlusion and diabetic retinopathy. Vorolanib is a tyrosine kinase inhibitor.

EyePoint elaborated that its drug candidate EYP 1901, which incorporates vorolanib, uses a miniaturized, sustained release and injectable intravitreal drug delivery system offering six months duration. The company has used its bioerodible Durasert technology for this purpose. EyePoint is optimistic about the combination of vorolanib with Durasert technology for delivering superior results.

Under the terms of the agreement, EyePoint will take care of development and global commercialization of the treatment. However, the global commercialization will exclude China, Hong Kong, Taiwan and Macau regions. For this purpose, EyePoint will pay $1 million to Equinox Science as upfront payment. It will also pay development and regulatory milestones and post commercialization royalties.

EyePoint recently concluded a positive Type B pre investigational New Drug meeting with the FDA. The meeting clarified the pathway for a Phase 1 clinical trial. The company expects to present the data from Phase 1 trial during the second half of 2021. Nancy Lurker, President and Chief Executive Officer of EyePoint Pharmaceuticals, said, We are encouraged by the potential of vorolanib, as it demonstrated a promising Phase 1 and Phase 2 efficacy signal in prior human wAMD studies as an oral therapy and in preclinical animal studies as intravitreal EYP-1901.

EyePoint is a biopharma company specializing in developing novel ophthalmic products. The company currently has two products available in the market which are Dexycu and Yutiqu. The former is the first approved intraocular treatment for postoperative inflammation while the latter is a three-year treatment of chronic non-infectious uveitis affecting the posterior segment of the eye.

Thanks for reading. At the Total Pharma Tracker, we do more than follow biotech news. Using our IOMachine, our team of analysts work to be ahead of the curve.

That means that when the catalyst comes that will make or break a stock, weve positioned ourselves for success. And we share that positioning and all the analysis behind it with our members.

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

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Mesoblast Submits BLA, And Other News: The Good, Bad And Ugly Of Biopharma - Seeking Alpha

Stem Cell Therapy Market: Snapshot

Of late, there has been an increasing awareness regarding the therapeutic potential of stem cells for management of diseases which is boosting the growth of the stem cell therapy market. The development of advanced genome based cell analysis techniques, identification of new stem cell lines, increasing investments in research and development as well as infrastructure development for the processing and banking of stem cell are encouraging the growth of the global stem cell therapy market.

To know Untapped Opportunities in the MarketCLICK HERE NOW

One of the key factors boosting the growth of this market is the limitations of traditional organ transplantation such as the risk of infection, rejection, and immunosuppression risk. Another drawback of conventional organ transplantation is that doctors have to depend on organ donors completely. All these issues can be eliminated, by the application of stem cell therapy. Another factor which is helping the growth in this market is the growing pipeline and development of drugs for emerging applications. Increased research studies aiming to widen the scope of stem cell will also fuel the growth of the market. Scientists are constantly engaged in trying to find out novel methods for creating human stem cells in response to the growing demand for stem cell production to be used for disease management.

It is estimated that the dermatology application will contribute significantly the growth of the global stem cell therapy market. This is because stem cell therapy can help decrease the after effects of general treatments for burns such as infections, scars, and adhesion. The increasing number of patients suffering from diabetes and growing cases of trauma surgery will fuel the adoption of stem cell therapy in the dermatology segment.

Global Stem Cell Therapy Market: Overview

Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.

Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.

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Global Stem Cell Therapy Market: Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

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Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

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TMR Research is a premier provider of customized market research and consulting services to business entities keen on succeeding in todays supercharged economic climate. Armed with an experienced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

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Stem Cell Therapy Market Trends and Growth, Outlook, Research, Trends and Forecast to 2025 - Instant Tech News

Latest Research Report on Stem Cell Treatments Market size | Industry Segment by Applications (Nerve Diseases, Immunological Diseases, Musculoskeletal Disorders, Cardiovascular Diseases, Gastrointestinal Diseases and Other), by Type (Adipose Tissue-Derived Mesenchymal Stem Cells, Bone Marrow-Derived Mesenchymal Stem Cells, Cord Blood/Embryonic Stem Cells and Other Cell Sources), Regional Outlook, Market Demand, Latest Trends, Stem Cell Treatments Industry Growth, Share & Revenue by Manufacturers, Company Profiles, Forecasts 2025.Analyzes current market size and upcoming 5 years growth of this industry.

New research report to its expanding repository. The research report, titled Stem Cell Treatments Market, mainly includes a detailed segmentation of this sector, which is expected to generate massive returns by the end of the forecast period, thus showing an appreciable rate of growth over the coming years on an annual basis. The research study also looks specifically at the need for Stem Cell Treatments Market.

Our Report Offerings Include:

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Report Scope:

The study includes the profiles of key players in the Stem Cell Treatments market with a significant global and/or regional presence. The Stem Cell Treatments market competition by Top Manufacturers Covers:

By Product:

By Application:

Points Covered in The Report:

Recent Industry Trend:

The report contains the profiles of various prominent players in the Global Stem Cell Treatments Market. Different strategies implemented by these vendors have been analyzed and studied to gain a competitive edge, create unique product portfolios and increase their market share. The study also sheds light on major global industry vendors. Such essential vendors consist of both new and well-known players. Besides, the business report contains important data relating to the launch of new products on the market, specific licenses, domestic scenarios and the strategies of the organization implemented on the market.

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Stem Cell Treatments Market to Exhibit Impressive Growth of CAGR during the per - News by aeresearch

Jack Chieh and Yinnie Wong with their baby boy, born last Friday (Chinese New Year). The couple donate her baby's cord blood to the cord blood bank at B.C. Womens Hospital & Health Centre.Handout

Yinnie Wong and Jack Chiehs six-pound, 13-ounce baby boy as yet unnamed was born on an auspicious day, Jan. 24, Chinese New Year, and hes already doing good in the world.

Everyone was really happy, it is supposed to be a lucky day, said Wong.

Although the birth was a planned C-section, Wong had no control over the date hospital administrators chose for the birth. What she did have control over was the choice to donate her babys cord blood to the cord blood bank at B.C. Womens Hospital & Health Centre, which has just celebrated its fifth anniversary.

Cord blood is blood that is taken from the umbilical cord and placenta immediately after the birth of a healthy infant. Cord blood is rich in stem cells, and can be used to treat over 80 diseases, including leukemia.

According to Canadian Blood Services, ethnically diverse donors are especially needed because although Stats Canada data shows 67.7 per cent of Canadians consider their ethnic origin to be diverse, only 31 per cent of Canadians with blood in Canadas stem-cell registry are from ethnically diverse backgrounds.

Crystal Nguyen, 20, is a former B.C. Childrens Hospital patient whose life was saved by a stem-cell transplant from donated cord blood. Nguyen was first diagnosed with acute myeloid leukemia at age 12. After chemo, she went into remission for almost three years. Then the cancer returned. She was told she needed a bone-marrow transplant.

Crystal Nguyen, now 20, was first diagnosed with acute myeloid leukemia at age 12. She found a stem-cell match for a needed bone-marrow transplant through the international cord blood bank.Handout

When I relapsed I was very confused, it was kind of surreal. The main thing about being told I needed the bone-marrow stem-cell transplant was confusion, fear and anxiety.

Nguyen is of Vietnamese descent and needed a match to survive. No one in her family was a match, nor was there a stem-cell match in the Canadian cord blood bank, but a match was found thanks to the Canadian Blood Services partnerships with 47 international blood banks.

I was told it came through the international cord blood bank from somewhere very far away, said Nguyen, who has been in remission since the transplant.

When she learned the stem-cell transplant had been successful, Nguyen, who is now studying to become a pediatric oncology nurse, said it felt too good to be true.

There was a lot of happiness, joy, excitement. Donating cord blood is such a simple way to save a life.

Although cord blood can be collected and stored for a fee by private companies and reserved for the donor familys use, cord blood donated through Canadian Blood Services is available free to the public whoever needs the match.

Wong didnt hesitate when her son was born. I felt like I wanted to do it if it helps someone in the public, and if it could save lives I would have been very happy to help another child, said Wong, who is a nurse at B.C. Womens hospital.

dryan@postmedia.com

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Is there more to this story? Wed like to hear from you about this or any other stories you think we should know about. Email vantips@postmedia.com.

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Chinese New Year babys B.C. family gives gift of life in cord-blood donation - Vancouver Sun

WARSAW (AFP) Submerged in liquid nitrogen vapour at a temperature of minus 175 degrees Celsius, hundreds of thousands of stem cells from all over Europe bide their time in large steel barrels on the outskirts of Warsaw.

Present in blood drawn from the umbilical cord of a newborn baby, stem cells can help cure serious blood-related illnesses like leukaemia and lymphoma, as well as genetic conditions and immune system deficits.

Polish umbilical cord blood bank PBKM/FamiCord became the industrys leader in Europe after Swiss firm Cryo-Save went bankrupt early last year.

It is also the fifth largest in the world, according to its management, after two companies in the United States (US), a Chinese firm and one based in Singapore.

Since the first cord blood transplant was performed in France in 1988, the sector has significantly progressed, fuelling hopes.

Mum-of-two Teresa Przeborowska has firsthand experience.

At five-years-old, her son Michal was diagnosed with lymphoblastic leukaemia and needed a bone marrow transplant, the entrepreneur from northern Poland said.

The most compatible donor was his younger sister, Magdalena.

When she was born, her parents had a bag of her cord blood stored at PBKM.

More than three years later, doctors injected his sisters stem cells into Michals bloodstream. It was not quite enough for Michals needs but nicely supplemented harvested bone marrow.

As a result, Michal, who is nine, is now flourishing, both intellectually and physically, his mum told AFP.

A cord blood transplant has become an alternative to a bone marrow transplant when there is no donor available, with a lower risk of complications.

Stem cells taken from umbilical cord blood are like those taken from bone marrow, capable of producing all blood cells: red cells, platelets and immune system cells.

When used, stem cells are first concentrated, then injected into the patient. Once transfused, they produce new cells of every kind.

At the PBKM laboratory, each container holds up to 10,000 blood bags. Safe and secure, they wait to be used in the future, its Head Krzysztof Machaj, said.

The bank holds around 440,000 samples, not including those from Cryo-Save, he said.

If the need arises, the blood will be ready to use without the whole process of looking for a compatible donor and running blood tests, the biologist told AFP.

For families who have paid an initial nearly EUR600 (USD675) and then an annual EUR120 euros to have the blood taken from their newborns umbilical cords preserved for around 20 years, it is a kind of health insurance promising faster and more effective treatment if illness strikes.

But researchers also warn against unrealistic expectations.

Bone marrow pioneer in Poland Haematologist Wieslaw Jedrzejczak describes promoters of the treatment as sellers of hope, who make promises that are either impossible to realise in the near future or downright impossible to realise at all for biological reasons.

He compares them to makers of beauty products who swear their cream will rejuvenate the client by 20 years.

Various researches is being done on the possibility of using the stem cells to treat other diseases, notably nervous disorders. But the EuroStemCell scientist network warns that the research is not yet conclusive.

There is a list of almost 80 diseases for which stem cells could prove beneficial, US Haematologist Roger Mrowiec, who heads the clinical laboratory of the cord blood programme Vitalant in New Jersey, told AFP.

But given the present state of medicine, they are effective only for around a dozen of them, like leukaemia or cerebral palsy, he said.

Its not true, as its written sometimes, that we can already use them to fight Parkinsons disease or Alzheimers disease or diabetes.

EuroStemCell also cautions against private blood banks that advertise services to parents suggesting they should pay to freeze their childs cord blood in case its needed later in life.

Studies show it is highly unlikely that the cord blood will ever be used for their child, the network said.

It also pointed out that there could be a risk of the childs cells not being useable anyway without reintroducing the same illness.

Some countries, such as Belgium and France, are cautious and ban the storage of cord blood for private purposes. Most European Union (EU) countries however permit it while imposing strict controls.

In the early 2000s, Swiss company Cryo-Save enjoyed rapid growth.

Greeks, Hungarians, Italians, Spaniards and Swiss stored blood from their newborns with the company for 20 years on payment of UER2,500 euros upfront.

When the firm was forced to close in early 2019, clients were left wondering where their stem cells would end up.

Under a kind of back-up agreement, the samples of some 250,000 European families were transferred for storage at PBKM.

The Polish firm, founded in 2002 with PLN2million (around EUR450,000, USD525,000), has also grown quickly.

Present under the FamiCord brand in several countries, PBKM has some 35 per cent of the European market, excluding Cryo-Save assets.

Over the last 15 months, outside investors have contributed EUR63 million to the firm, PBKMs Chief Executive Jakub Baran told AFP.

But the company has not escaped controversy: the Polityka weekly recently published a critical investigative report on several private clinics that offer what was described as expensive treatment involving stem cells held by PBKM.

Read more:
Europe's guardian of stem cells and hopes, real and unrealistic - Borneo Bulletin Online

SAN ANTONIO, Feb. 3, 2020 /PRNewswire/ -- SASpine is now offering cutting edge Stem Cell Treatments to patients. For the past several years Dr. Steven Cyr, Mayo Clinic Trained Spine Surgeon, has been researching the benefits of stem cells in the treatment of multiple medical conditions including spinal disorders, specifically, conditions which involve spinal cord injury, degenerative disc disease, herniated discs, and as a supplement to enhance the success of Spinal fusions when treating instability, deformity, and fractures of the spine.

Steven J. Cyr, M.D., is a spine surgeon who has gained a reputation for surgical excellence in Texas, throughout the nation, and abroad.

Dr. Steven Cyr has been treating patients using growth factors and stem cells contained in amniotic tissue and bone marrow aspirate to provide a potential for improved success with fusion procedures, when treating herniated discs, and for arthritic or damaged joints, with remarkable success. "The goal of any medical intervention is to yield improved outcomes with the ideal result of returning a patient to normal function, when possible," states Dr Cyr. He went on to elaborate that there are times when only a structural solution can solve problems related to spinal disorders, but even in that scenario, the use of stem cells or growth factors derived from stem cell products can possibly improve the success of surgical procedures. "I have patients previously unable to jog or run return to normal function and athletic ability after injections of growth factors and stem cell products into the knee joints, hip joints, and shoulder joints," he said. "This includes high-level athletes, professional dancers, and the average weekend warrior."

There may be promise in treating patients with spinal cord injury as well. SASpine CEO, LeAnn Cyr, states, "There are reports of patients gaining significant neurological improvement after being treated with stem cells." Dr Cyr continues, "Most patients with spinal cord injuries resulting from trauma also have mechanical pressure on the nerves that result either from bone fragments or disc material compressing the spinal cord that needs to be removed along with surgical stabilization of the spinal bones. There's significant potential that stem cells bring to the equation when treating these types of patients, and I am excited about the potential that these products offer to the host of treatments to address spinal conditions and arthritic joints."

For more information about SASpine's Stem Cell Treatment Program, visit http://www.saspine.com or call (210) 487-7463 in San Antonio or (832) 919-7990 in Houston.

Related Linkswww.facebook.com/saspinewww.instagram.com/surgical.associates.in.spine

If you've been living with back pain, you're not alone. Here at SASpine, we have experienced spine specialists who are committed to improving your quality of life. (PRNewsfoto/SASpine)

View original content to download multimedia:http://www.prnewswire.com/news-releases/saspine-to-offer-stem-cell-therapy-300997355.html

SOURCE SASpine

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SASpine to offer Stem Cell Therapy - Yahoo Finance

Jack Chieh and Yinnie Wong with their baby boy, born last Friday (Chinese New Year). The couple donate her baby's cord blood to the cord blood bank at B.C. Womens Hospital & Health Centre.Handout

By Denise Ryan

Yinnie Wong and Jack Chiehs six-pound, 13-ounce baby boy as yet unnamed was born on an auspicious day, Jan. 24, Chinese New Year, and hes already doing good in the world.

Everyone was really happy, it is supposed to be a lucky day, said Wong.

Although the birth was a planned C-section, Wong had no control over the date hospital administrators chose for the birth. What she did have control over was the choice to donate her babys cord blood to thecord blood bank at B.C. Womens Hospital & Health Centre, which has just celebrated its fifth anniversary.

Cord blood is blood that is taken from the umbilical cord and placenta immediately after the birth of a healthy infant. Cord blood is rich in stem cells, and can be used to treat over 80 diseases, including leukemia.

According to Canadian Blood Services, ethnically diverse donors are especially needed because although Stats Canada data shows 67.7 per cent of Canadians consider their ethnic origin to be diverse, only 31 per cent of Canadians with blood in Canadas stem-cell registry are from ethnically diverse backgrounds.

Crystal Nguyen, 20, is a former B.C. Childrens Hospital patient whose life was saved by a stem-cell transplant from donated cord blood. Nguyen was first diagnosed with acute myeloid leukemia at age 12. After chemo, she went into remission for almost three years. Then the cancer returned. She was told she needed a bone-marrow transplant.

When I relapsed I was very confused, it was kind of surreal. The main thing about being told I needed the bone-marrow stem-cell transplant was confusion, fear and anxiety.

Nguyen is of Vietnamese descent and needed a match to survive. No one in her family was a match, nor was there a stem-cell match in the Canadian cord blood bank, but a match was found thanks to the Canadian Blood Services partnerships with 47 international blood banks.

I was told it came through the international cord blood bank from somewhere very far away, said Nguyen, who has been in remission since the transplant.

When she learned the stem-cell transplant had been successful, Nguyen, who is now studying to become a pediatric oncology nurse, said it felt too good to be true.

There was a lot of happiness, joy, excitement. Donating cord blood is such a simple way to save a life.

Although cord blood can be collected and stored for a fee by private companies and reserved for the donor familys use, cord blood donated through Canadian Blood Services is available free to the public whoever needs the match.

Wong didnt hesitate when her son was born. I felt like I wanted to do it if it helps someone in the public, and if it could save lives I would have been very happy to help another child, said Wong, who is a nurse at B.C. Womens hospital.

dryan@postmedia.com

Originally posted here:
Chinese New Year babys B.C. family gives gift of life in cord - The Province

Kenneth Pettine's stem cell product to treat OA was tested on retired Navy SEALs

FORT COLLINS, CO / ACCESSWIRE / February 3, 2020 / Kenneth Pettine is proud to announce that his revolutionary mesenchymal stem cell product to treat osteoarthritis was recently tested on 33 former Navy SEALs (one is a medal of honor recipient).

Kenneth Pettine is co-founder of Paisley Laboratories and a co-developer of a bone marrow-derived mesenchymal stem cell active growth factor and exosome product that is anticipated to revolutionize regenerative medicine.

In this study, Extracellular Vesicle Isolate Product (EVIP) was injected into 33 retired Navy SEALs to assist with knee, shoulder, elbow, ankle, and wrist osteoarthritis. At three-month follow-up, the injection appeared both safe and effective, with improvements ranging from 40% to as high as 98%. The average improvement is over 70%.

"This is extremely promising and we are motivated to continue our clinical studies to improve the quality of life for patients," says Kenneth Pettine.

Kenneth Pettine notes in his study that over 50 million Americans require daily treatment for disability and pain associated with OA. Every year, over one million total hip and knee replacements are performed in the U.S. with direct costs of over $30 billion and indirect costs of over $200 billion, with these numbers expected to double in the next three years.

In addition to this trial, Kenneth Pettine has three additional clinical studies planned to evaluate his stem cell products to treat erectile dysfunction, chronic obstructive pulmonary disease (COPD), and chronic lower back pain from painful discs.

For more information, visit https://www.kenneth-pettine.com/

About Kenneth Pettine

Dr. Kenneth Pettine is a serial entrepreneur and published clinical researcher with over 30 years of experience as an orthopedic surgeon. He holds a medical degree from the University of Colorado School of Medicine and completed his master's degree in orthopedic surgery and residency at the Mayo Clinic in Rochester, Minnesota.

In 1991, Dr. Pettine founded the Rocky Mountain Associates in Orthopedic Medicine. Kenneth Pettine is also the founder of Paisley Laboratories and the co-founder of the Society for Ambulatory Spine Surgery. In addition, he co-invented the Prestige cervical artificial disc and the Maverick Artificial Disc. Dr. Pettine is the principal investigator of 18 FDA studies involving non-fusion implants, biologics, and stem cells. He holds the only two issued U.S. patents for performing stem cell joint and spinal injections and currently has 21 additional patents pending for bone marrow derived mesenchymal stem cell applications. Kenneth Pettine is also a philanthropist and currently has a scholarship program underway to help students fund their education.

For more information, visit https://www.kenneth-pettine.com/ or https://www.kennethpettinescholarship.com/

Contact

info@kenneth-pettine.com

https://www.kenneth-pettine.com/

SOURCE: Kenneth Pettine

View source version on accesswire.com: https://www.accesswire.com/574987/Dr-Kenneth-Pettine-Announces-Verification-of-Clinical-Safety-Trial

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Dr. Kenneth Pettine Announces Verification of Clinical Safety Trial - Yahoo Finance

Jack Chieh and Yinnie Wong with their baby boy, born last Friday (Chinese New Year). The couple donate her baby's cord blood to the cord blood bank at B.C. Womens Hospital & Health Centre.Handout

Yinnie Wong and Jack Chiehs six-pound, 13-ounce baby boy as yet unnamed was born on an auspicious day, Jan. 24, Chinese New Year, and hes already doing good in the world.

Everyone was really happy, it is supposed to be a lucky day, said Wong.

Although the birth was a planned C-section, Wong had no control over the date hospital administrators chose for the birth. What she did have control over was the choice to donate her babys cord blood to the cord blood bank at B.C. Womens Hospital & Health Centre, which has just celebrated its fifth anniversary.

Cord blood is blood that is taken from the umbilical cord and placenta immediately after the birth of a healthy infant. Cord blood is rich in stem cells, and can be used to treat over 80 diseases, including leukemia.

According to Canadian Blood Services, ethnically diverse donors are especially needed because although Stats Canada data shows 67.7 per cent of Canadians consider their ethnic origin to be diverse, only 31 per cent of Canadians with blood in Canadas stem-cell registry are from ethnically diverse backgrounds.

Crystal Nguyen, 20, is a former B.C. Childrens Hospital patient whose life was saved by a stem-cell transplant from donated cord blood. Nguyen was first diagnosed with acute myeloid leukemia at age 12. After chemo, she went into remission for almost three years. Then the cancer returned. She was told she needed a bone-marrow transplant.

Crystal Nguyen, now 20, was first diagnosed with acute myeloid leukemia at age 12. She found a stem-cell match for a needed bone-marrow transplant through the international cord blood bank.Handout

When I relapsed I was very confused, it was kind of surreal. The main thing about being told I needed the bone-marrow stem-cell transplant was confusion, fear and anxiety.

Nguyen is of Vietnamese descent and needed a match to survive. No one in her family was a match, nor was there a stem-cell match in the Canadian cord blood bank, but a match was found thanks to the Canadian Blood Services partnerships with 47 international blood banks.

I was told it came through the international cord blood bank from somewhere very far away, said Nguyen, who has been in remission since the transplant.

When she learned the stem-cell transplant had been successful, Nguyen, who is now studying to become a pediatric oncology nurse, said it felt too good to be true.

There was a lot of happiness, joy, excitement. Donating cord blood is such a simple way to save a life.

Although cord blood can be collected and stored for a fee by private companies and reserved for the donor familys use, cord blood donated through Canadian Blood Services is available free to the public whoever needs the match.

Wong didnt hesitate when her son was born. I felt like I wanted to do it if it helps someone in the public, and if it could save lives I would have been very happy to help another child, said Wong, who is a nurse at B.C. Womens hospital.

dryan@postmedia.com

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Chinese New Year babys B.C. family gives gift of life in cord-blood donation - The Province

DUBLIN--(BUSINESS WIRE)--The "Stem Cell Banking - Market Analysis, Trends, and Forecasts" report has been added to ResearchAndMarkets.com's offering.

The global market for Stem Cell Banking is projected to reach US$9.9 billion by 2025, driven by their growing importance in medicine given their potential to regenerate and repair damaged tissue.

Stem cells are defined as cells with the potential to differentiate and develop into different types of cells. Different accessible sources of stem cells include embryonic stem cells, fetal stem cells, peripheral blood stem cells, umbilical cord stem cells, mesenchymal stem cells (bmMSCs) and induced pluripotent stem cells. Benefits of stem cells include ability to reverse diseases like Parkinsons by growing new, healthy and functioning brain cells; heal and regenerate tissues and muscles damaged by heart attack; address genetic defects by introducing normal cells; reduce mortality among patients awaiting donor organs for transplant by regenerating healthy cells and tissues as an alternative to donated organs. While currently valuable in bone marrow transplantation, stem cell therapy holds huge potential in treating a host of common chronic diseases such as diabetes, heart disease (myocardial infarction), Parkinsons disease, spinal cord injury, arthritis, and amyotrophic lateral sclerosis. The technology has the potential to revolutionize public health.

The growing interest in regenerative medicine which involves replacing, engineering or regenerating human cells, tissues or organs, will push up the role of stem cells. Developments in stem cells bioprocessing are important and will be key factor that will influence and help regenerative medicine research move into real-world clinical use. The impact of regenerative medicine on healthcare will be comparable to the impact of antibiotics, vaccines, and monoclonal antibodies in current clinical care. With global regenerative medicine market poised to reach over US$45 billion 2025, demand for stem cells will witness robust growth.

Another emerging application area for stem cells is in drug testing in the pharmaceutical field. New drugs in development can be safely, accurately, and effectively be tested on stem cells before commencing tests on animal and human models. Among the various types of stem cells, umbilical cord stem cells are growing in popularity as they are easy and safe to extract. After birth blood from the umbilical cord is extracted without posing risk either to the mother or the child. As compared to embryonic and fetal stem cells which are saddled with safety and ethical issues, umbilical cord is recovered postnatally and is today an inexpensive and valuable source of multipotent stem cells. Until now discarded as waste material, umbilical cord blood is today acknowledged as a valuable source of blood stem cells. The huge gap between newborns and available cord blood banks reveals huge untapped opportunity for developing and establishing a more effective banking system for making this type of stem cells viable for commercial scale production and supply. Umbilical cord and placenta contain haematopoietic blood stem cells (HSCs). These are the only cells capable of producing immune system cells (red cells, white cells and platelet).

HSCs are valuable in the treatment of blood diseases and successful bone marrow transplants. Also, unlike bone marrow stem cells, umbilical cord blood has the advantage of having 'off-the-shelf' uses as it requires no human leukocyte antigen (HLA) tissue matching. Developments in stem cell preservation will remain crucial for successful stem cell banking. Among the preservation technologies, cryopreservation remains popular. Development of additives for protecting cells from the stresses of freezing and thawing will also be important for the future of the market. The United States and Europe represent large markets worldwide with a combined share of 60.5% of the market. China ranks as the fastest growing market with a CAGR of 10.8% over the analysis period supported by the large and growing network of umbilical cord blood banks in the country. The Chinese government has, over the years, systematically nurtured the growth of umbilical cord blood (UCB) banks under the 'Developmental and Reproductive Research Initiation' program launched in 2008. Several hybrid public-private partnerships and favorable governmental licensing policies today are responsible for the current growth in this market.

Companies Mentioned

Key Topics Covered:

I. METHODOLOGY

II. EXECUTIVE SUMMARY

1. MARKET OVERVIEW

2. FOCUS ON SELECT PLAYERS

3. MARKET TRENDS & DRIVERS

4. GLOBAL MARKET PERSPECTIVE

III. MARKET ANALYSIS

GEOGRAPHIC MARKET ANALYSIS

UNITED STATES

CANADA

JAPAN

CHINA

EUROPE

FRANCE

GERMANY

ITALY

UNITED KINGDOM

REST OF EUROPE

ASIA-PACIFIC

REST OF WORLD

IV. COMPETITION

V. CURATED RESEARCH

For more information about this report visit https://www.researchandmarkets.com/r/9b2ra3

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Global Stem Cell Banking Market Analysis, Trends, and Forecasts 2019-2025 - ResearchAndMarkets.com - Business Wire

At left, image shows white blood cells (red) from one of the X-CGD clinical trial participants before gene therapy. At right, after gene therapy, white blood cells from the same patient show the presence of the chemicals (blue) needed to attack and destroy bacteria and fungus.

UCLA Broad Stem Cell Research Center/Nature Medicine

University of California - Los Angeles (UCLA) researchers are part of an international team that reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six of those patients are now in remission and have stopped other treatments. Before now, people with X-CGDwhich causes recurrent infections, prolonged hospitalizations for treatment, and a shortened lifespanhad to rely on bone marrow donations for a chance at remission.

"With this gene therapy, you can use a patient's own stem cells instead of donor cells for a transplant," said Dr. Donald Kohn, a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA and a senior author of the new paper, published Jan. 28 in the journal Nature Medicine. "This means the cells are perfectly matched to the patient and it should be a much safer transplant, without the risks of rejection."

People with chronic granulomatous disease, or CGD, have a genetic mutation in one of five genes that help white blood cells attack and destroy bacteria and fungus using a burst of chemicals. Without this defensive chemical burst, patients with the disease are much more susceptible to infections than most people. The infections can be severe to life-threatening, including infections of the skin or bone and abscesses in organs such as lungs, liver or brain. The most common form of CGD is a subtype called X-CGD, which affects only males and is caused by a mutation in a gene found on the X-chromosome.

Other than treating infections as they occur and taking rotating courses of preventive antibiotics, the only treatment option for people with CGD is to receive a bone marrow transplant from a healthy matched donor. Bone marrow contains stem cells called hematopoietic, or blood-forming, stem cells, which produce white blood cells. Bone marrow from a healthy donor can produce functioning white blood cells that effectively ward off infection. But it can be difficult to identify a healthy matched bone marrow donor and the recovery from the transplant can have complications such as graft versus host disease, and risks of infection and transplant rejection.

"Patients can certainly get better with these bone marrow transplants, but it requires finding a matched donor and even with a match, there are risks," Kohn said. Patients must take anti-rejection drugs for six to 12 months so that their bodies don't attack the foreign bone marrow.

In the new approach, Kohn teamed up with collaborators at the United Kingdom's National Health Service, France-based Genethon, the U.S. National Institute of Allergy and Infectious Diseases at the National Institutes of Health, and Boston Children's Hospital. The researchers removed hematopoietic stem cells from X-CGD patients and modified the cells in the laboratory to correct the genetic mutation. Then, the patients' own genetically modified stem cellsnow healthy and able to produce white blood cells that can make the immune-boosting burst of chemicalswere transplanted back into their own bodies. While the approach is new in X-CGD, Kohn previously pioneered a similar stem cell gene therapy to effectively cure a form of severe combined immune deficiency (also known as bubble baby disease) in more than 50 babies.

The viral delivery system for the X-CGD gene therapy was developed and fine-tuned by professor Adrian Thrasher's team at Great Ormond Street Hospital, or GOSH, in London, who collaborated with Kohn. The patients ranged in age from 2 to 27 years old; four were treated at GOSH and five were treated in the US, including one patient at UCLA Health.

Two people in the new study died within three months of receiving the treatment due to severe infections that they had already been battling before gene therapy. The seven surviving patients were followed for 12 to 36 months after receiving the stem cell gene therapy. All remained free of new CGD-related infections, and six of the seven have been able to discontinue their usual preventive antibiotics.

"None of the patients had complications that you might normally see from donor cells and the results were as good as you'd get from a donor transplantor better," Kohn said.

An additional four patients have been treated since the new paper was written; all are currently free of new CGD-related infections and no complications have arisen.

Orchard Therapeutics, a biotechnology company of which Kohn is a scientific co-founder, acquired the rights to the X-CGD investigational gene therapy from Genethon. Orchard will work with regulators in the US and Europe to carry out a larger clinical trial to further study this innovative treatment. The aim is to apply for regulatory approval to make the treatment commercially available, Kohn said.

Kohn and his colleagues plan to develop similar treatments for the other forms of CGDcaused by four other genetic mutations that affect the same immune function as X-CGD.

"Beyond CGD, there are also other diseases caused by proteins missing in white blood cells that could be treated in similar ways," Kohn said.

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6 Patients with Rare Blood Disease Doing Well after Gene Therapy Clinical Trial - Lab Manager Magazine

Spinal cord injury (SCI) is a common disease with high incidence, disability rate and treatment cost. microRNA (miR)-200a is reported to inhibit Keap1 to activate Nrf2 signaling. This study aimed to explore the effects of lentivirus-mediated miR-200a gene-modified bone marrow mesenchymal stem cells (BMSCs) transplantation on the repair of SCI in a rat model. BMSCs were isolated from the bone marrow of Sprague-Dawley rats. miR-200a targeting to Keap1 was identified by luciferase-reporter gene assay. The expressions of Keap1, Nrf2, NQO-1, HO-1 and GCLC were detected by Western blotting in SCI rats. The locomotor capacity of the rats was evaluated using the Basso, Beattie and Bresnahan scale. The levels of malondialdehyde (MDA) and activities of superoxide dismutase (SOD) and catalase (CAT) were measured. miR-200a inhibited Keap-1 3 UTR activity in BMSCs. Transplantation of BMSCs with overexpression of miR-200a or si-Keap1increased locomotor function recovery of rats after SCI, while decreased MDA level, increased SOD, CAT activities and Nrf2 expression together with its downstream HO-1, NQO1, GCLC protein expressions in SCI rat. These results indicated that overexpressed miR-200a in BMSCs promoted SCI repair, which may be through regulating anti-oxidative signaling pathway. 2020 International Center for Artificial Organs and Transplantation and Wiley Periodicals, Inc.

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Upregulation of microRNA-200a in bone marrow mesenchymal stem cells enhances the repair of spinal cord injury in rats by reducing oxidative stress and...

Photo Courtesy of Michael MankowichAbove, Michael Mankowich and his wife, Kathleen, in Patagonia

NUTLEY, NJ When Nutley resident Michael Mankowichs lower back started to bother him, he figured it was a souvenir from his earlier athletic days. Mike, 58, had been a top-notch wrestler at 132 pounds at Long Islands Commack North High School. Hed been an all-American, in fact, as well as a two-time all-Ivy, three-time New York state champ and three-time EIWA tournament placer as a wrestler at Cornell University. An old wrestlers injury was all it was, he figured, a physical reminder of a quick takedown of an opponent 40 years long forgotten.

But the pain did not go away.

Mike began to see a doctor and a chiropractor, and eventually he got an MRI. The news he received at Memorial Sloan Kettering Cancer Center in February 2017 was not good. He was diagnosed with multiple myeloma, a cancer that attacks the blood plasma cells responsible for creating disease-fighting antibodies.

They figured it out quickly at Sloan, he said recently, seated with his wife, Kathleen, in their Rutgers Place home. I kept it from Kathleen.

With this news, he became withdrawn, and his wife realized something was wrong. Mike told her what he had learned, and, as so often happens when a couple puts their heads together, they found some reason for hope: multiple myeloma is a blood disease in the bone marrow and, as such, does not metastasize.

Thats where all the action takes place, in the bone marrow, Mike said. You have to keep your chin up.

For treatment, he became part of a six-month chemotherapy clinical study. Mike was glad to be in the study, because most multiple myeloma patients go on chemotherapy for three months and then undergo a stem-cell transplant. He, however, would not.

A stem-cell transplant blows out the immune system, he said.

Kathleen, an administrative coordinator at Felician University School of Nursing, said her husband, a real estate management employee, did not break stride and never missed the commute to New York City during the clinical study.

A member of Nutley High Schools Class of 1976, Kathleen got on the computer.

When your spouse is diagnosed with an incurable cancer, you do a bit of research, she said.

She discovered the Multiple Myeloma Research Foundation website and learned it was founded 30 years earlier by a woman named Kathy Giusti, who was living with the disease.

That gave me hope, Kathleen said.

She also learned about a collaboration between MMRF and CURE Media Group called Moving Mountains for Multiple Myeloma, or MM4MM.

This collaboration promotes endurance events, undertaken by multiple myeloma patients, to places like Mount Fuji, Mount Kilimanjaro and Iceland. The treks raise money for research, as well as public awareness about the disease. A patient selected to participate in one of these exotic treks had to raise funds, but the trip itself was underwritten by Celgene, a pharmaceutical company headquartered in Summit.

Mike was interested and applied in November 2018 for a spot on a team going to Patagonia. He was interviewed and accepted on condition of raising $10,000 for MMRF research. He suggested that Kathleen accompany him, and they eventually raised $30,000 through social media and by asking friends, family and neighbors.

The online MMRF page devoted to Mikes fundraising shows a photograph of him with his arms around Kathleen and their daughter, Mary, a Class of 2020 NHS student.

In a letter featured on the page, Mike informs the reader that MMRF is one of the worlds leading private funders of myeloma research, with 10 new treatments approved by the Food and Drug Administration.

In August 2019, Mike and Kathleen were flown to Oregon to meet their teammates and to get a taste of what was in store for them in Patagonia. According to the MM4MM website: Each team is carefully selected, representing a microcosm of the myeloma community patients, caregivers, health care professionals and clinical trials managers, as well as representatives from our pharma partners, from CURE Magazine and the MMRF to emphasize the collaboration necessary to drive toward cures.

The foundation sent the group to Mount Hood, Mike said. It was the first time we met. What a great group of people. There were around 15 from all over the country, and there was one other couple, but no one else from New Jersey.

Four other multiple myeloma patients were in the group, he said. he team climbed for nine hours and then headed home.

To prepare for the trip to Patagonia, a region containing part of the Andes mountain range, Mike and Kathleen began a regime of long walks. For instance, theyd walk from Nutley to South Orange and went hiking in New Yorks Harriman State Park.

The MMRF website described the journey as one of arduous adventure: This team will traverse Patagonia crossing over glaciers, through deep valleys, and ascending challenging peaks. This is a powerful and life-changing experience, as the team overcomes challenges, pushes beyond perceived limits and honors loved ones and friends living with multiple myeloma.

For the trek, the team flew to El Calafate, Argentina. As the team embarked on different climbs, documentary filmmakers accompanied them.

The hiking was physically difficult, Mike said. We hiked in rain and incredible winds. In one particular hike, as soon as you felt the winds, you hit the ground. I was surprised nobody got hurt. Some of those slopes were pretty steep. But the scenery was unworldly, and there were condors.

Both Mike and Kathleen agreed that the most memorable sight was La Condorera, which their itinerary described as a nearly vertical massif, offering a home to one of the greatest concentrations of endangered condors in the world. A massif is a group of mountains standing apart from other mountains.

It was a difficult hike, Kathleen said. Youre ready to pass out getting to the top. But its so worth it. The panorama is a view of glaciers and condors. It was spectacular.

Mike and Kathleen returned home on Nov. 16, but there were no goodbyes at the airport. The team had grown so incredibly close that everyone felt they would be seeing each other again, a feeling grounded in the knowledge that multiple myeloma can be challenged and hopefully, one day, defeated.

Our goal in all of this is that you can have multiple myeloma and still do incredible things, Kathleen said.

Its an incentive to other patients to get out there and enjoy their lives, Mike said. And find a cure for multiple myeloma. I have a little bias. I have it.

FEATURED, MOBILE

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Fighting cancer with every step to Patagonia - Essex News Daily

When David Wilsons spine snapped, leading to a cancer diagnosis, there were fears he would be left paralysed.

But the cyclist and mountaineer had his back rebuilt and went through gruelling chemotherapy and now, just a year later, he is planning a very physical challenge to raise funds for charity,

The Fort William man, who is 60, has journeyed across the globe to the far reaches of the Himalayas, Greenland, Norway and New Zealand. And now he is cycling from London to Paris.

In the summer of 2018, he was cycling the Great Divide Mountain Bike Trail a 3,083-mile off-road ride from Canada to Mexico when his back began to hurt.

He cut the trip short, putting his injury down to old age and wear and tear.

And in February last year his spine suddenly snapped due to a form of blood cancer called Multiple Myeloma.

Following his devastating diagnosis, Mr Wilson owner of the Limetree Hotel, restaurant and art gallery in Fort William was transferred to hospital in Glasgow where he underwent surgery to rebuild his spine.

He explained: I was taken from the Belford Hospital to Inverness, then transferred from Inverness to Glasgow to the surgeons there and they were able to rebuild my spine by putting in a scaffolding in my back with titanium and screws. That stage was touch and go on whether I would be paraplegic now and being an outdoors geezer I was pretty fed up about that.

Following a successful surgery, the visual artist began to slowly walk again before enduring a rigorous six month cycle of chemotherapy to attack the disease.

On September 20, he then received a stem cell transplant, giving him a new lease of life.

The father-of-two said: I had a very adventurous life.

I have been in situations where death has been very close to me, people have been killed right next to me but I have always felt there was a way in those situations that you could get out of them but with cancer like this particular cancer there is no escape. Youre in the hands of the disease and you have to take your chances when you get them.

Now in remission, Mr Wilson is aiming to defy the odds by cycling 500km from London to Paris in September, arriving in Paris exactly a year following his stem cell treatment.

He has now launched a Just Giving page in the hopes of raising 1,500 for Myeloma UK towards finding a cure for the crippling disease.

Multiple myeloma, also known simply as Myeloma, is a form of blood cancer arising from plasma cells causing problems to various areas of the body such as the spine, skill, pelvis and ribs.

It develops in abnormal plasma cells, which release a large amount of a single type of antibody known as paraprotein which has no useful function.

Myeloma affects where bone marrow is normally active in an adult, such as in the bones of the spine, skull, pelvis, the rib cage, long bones of the arms and legs and the areas around the shoulders and hips.

Each year in the UK, approximately 5,700 people are diagnosed with myeloma.

Myeloma mainly affects those over the age of 65, however it has been diagnosed in people much younger.

In the early stages of developing the disease, patients rarely experience side effects and is only diagnosed through routine blood or urine tests.

As the disease progresses, the cancer can cause a range of problems including aches and tender areas in your bones, causes bones to break and fracture easily, kidney problems and repeated infections.

Patients can also become lethargic, weak and short of breath caused as a result of anaemia.

In most diagnosed cases of myeloma it cannot be cured, however, treatment can be given to control the condition and minimise its effects for several years.

Treatment includes anti-myeloma medicines to destroy the cancer cells or control the cancer if patients suffer a relapse as well as a range of medicines to prevent or treat side effects of myeloma.

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Lochaber cyclist pledges to cycle from London to Paris in aid of charity - Press and Journal

ANN ARBORConventional thinking is that bone regeneration is left to a small number of mighty cells called skeletal stem cells, which reside within larger groups of bone marrow stromal cells.

But new findings from the University of Michigan recasts that thinking.

In a recent study, Noriaki Ono, assistant professor at the U-M School of Dentistry, and colleagues report that mature bone marrow stromal cells metamorphosed to perform in ways similar to their bone-healing stem cell cousinsbut only after an injury.

Bone fracture is an emergency for humans and all vertebrates, so the sooner cells start the business of healing damaged boneand the more cells there are to do itthe better.

Our study shows that other cells besides skeletal stem cells can do this job as well, Ono said.

In the mouse study, inert Cxcl12 cells in bone marrow responded to post-injury cellular cues by converting into regenerative cells, much like skeletal stem cells. Normally, the main job of these Cxcl12-expressing cells, widely known as CAR cells, is to secrete cytokines, which help regulate neighboring blood cells. They were recruited for healing only after an injury.

The surprise in our study is that these cells essentially did nothing in terms of making bones, when bones grow longer, Ono said. Its only when bones are injured that these cells start rushing to repair the defect.

This is important because the remarkable regenerative potential of bones is generally attributed to rare skeletal stem cells, Ono says. These new findings raise the possibility that these mighty skeletal stem cells could be generated through the transformation of the more available mature stromal cells.

These mature stromal cells are malleable and readily available throughout life, and could potentially provide an excellent cellular source for bone and tissue regeneration, Ono says.

The study appears in the journal Nature Communications.

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Originally posted here:
After a bone injury, shape-shifting cells rush to the rescue - University of Michigan News

What are progenitor cells?

Every cell in the human body, and that of other mammals, originates from stem cell precursors. Progenitor cells are descendants of stem cells that then further differentiate to create specialized cell types.There are many types of progenitor cells throughout the human body. Each progenitor cell is only capable of differentiating into cells that belong to the same tissue or organ. Some progenitor cells have one final target cell that they differentiate to, while others have the potential to terminate in more than one cell type.

Stem cells share two qualifying characteristics. Firstly, all stem cells have the potential to differentiate into multiple types of cells. Secondly, stem cells are capable of unlimited self-replication via asymmetric cell division, a process known as self-renewal.There are two broad categories of stem cells found in all mammals. The first are embryonic stem cells. These cells arise from the inner cell mass of the blastocyst in an early-stage embryo. Embryonic stem cells are the blueprint used to create every cell in the body. Because they can be used to create any type of cell, they are known as pluripotent.

The second type of stem cells found in mammals are adult stem cells (or somatic stem cells). Unlike pluripotent embryonic stem cells, adult stem cells are more limited in relation to the type of cells that they become. Unlike embryonic stem cells that could be used to create any cell, adult stem cells are limited to generating cell types within a specific lineage, such as blood cells or cells of the central nervous system. This level of differentiation potential is termed multipotent.

Stem cells create two types of progeny: more stem cells or progenitor cells. All progenitor cells are descendants of stem cells. When it comes to cell differentiation, they fall on the spectrum between stem cells and fully differentiated (mature) cells.

Whilst stem cells have indefinite replication (left) progenitor cells can at most differentiate into multiple types of specialized cell (right).

Function:

Cellular repair or maintenance

Cell Potency:

Multipotent, oligopotent, or unipotent

Self-renewal:

Limited

Origin:

Stem cells

Creates:

Further differentiated cells (either progenitor cells of mature/fully differentiated cells)

Progenitor cells are an intermediary step involved in the creation of mature cells in human tissues and organs, the blood, and the central nervous system.

The human central nervous system (CNS) contains three types of fully differentiated cells: neurons, astrocytes and oligodendrocytes. The latter two are collectively known as glial cells.Every neuron, oligodendrocyte and astrocyte in the CNS evolves from the differentiation of neural progenitor cells (NPCs). NPCs themselves are produced by multipotent neural stem cells (NSCs). Both NPCs and NSCs are termed neural precursor cells.Before the 1990s, it was believed that neurogenesis terminated early in life. More recent studies demonstrate that the brain contains stem cells that are capable of regenerating neurons and glial cells throughout the human lifecycle. These stem cells have only been found in certain brain regions, including the striatum and lateral ventricle.

Hematopoietic progenitor cells (HPCs) are an intermediate cell type in blood cell development. HPCs are immature cells that develop from hematopoietic stem cells, cells that can both self-renew and differentiate into hematopoietic progenitor cells. HPCs eventually differentiate into one of more than ten different types of mature blood cells.Hematopoietic progenitor cells are categorized based upon their cell potency, or their differentiation potential. As blood cells develop, their potency decreases.

First, hematopoietic stem cells differentiate into multipotent progenitor cells. Multipotent progenitor cells are those with the potential to differentiate into a subset of cell types. These cells then differentiate into either the common myeloid progenitor (CMP) or common lymphoid progenitor (CLP). Both CMPs and CLPs are types of oligopotent progenitor cells (progenitor cells that differentiate into only a few cell types).

CMPs and CLPs continue to differentiate along cell lines into lineage-restricted progenitor cells that become final, mature blood cells.Myeloid progenitor cells are precursors to the following types of blood cells:

Lymphoid progenitor cells (also known as lymphoblasts) are precursors to other mature blood cell types, including:

The primary role of progenitor cells is to replace dead or damaged cells. In this way, progenitor cells are necessary for repair after injury and as part of ongoing tissue maintenance. Progenitor cells also replenish blood cells and play a role in embryonic development.

Neural progenitor cells (NPCs) are being explored alongside neural stem cells for their potential to treat diseases of or injury to the central nervous system. A deeper understanding of how these cells function on a cellular and molecular basis is needed to progress from early experimental research to therapeutic use.NPCs are currently utilized in research conducted on CNS disorders, development, cell regeneration and degeneration, neuronal excitability, and therapy screening. When compared to induced pluripotent stem cells, which are cells reprogrammed into a pluripotent state, NPCs can cut down on time in some experiments.Hematopoietic progenitor cells and stem cells are being researched for their capacity to treat blood cell disorders. They are also currently used to help treat patients with a variety of malignant and non-malignant diseases via bone marrow transplants that deliver bone marrow and peripheral blood progenitor cells to patients. These procedures can assist patients in recovering from the damage caused by chemotherapy.Additionally, researchers are examining the potential of using progenitor cells to create a variety of tissues, such as blood vessels, heart valves, and electrically conductive tissue for the cardiovascular system.

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What are Progenitor Cells? Exploring Neural, Myeloid and Hematopoietic Progenitor Cells - Technology Networks

Chronic pain cost an estimated $139.3 billion in 2018. Image: iStock, Top image: Pexels

Researchers at the University of Sydney have used human stem cells to make pain-killing neurons that provide lasting relief in mice, without side effects, in a single treatment. The next step is to perform extensive safety tests in rodents and pigs, and then move to human patients suffering chronic pain within the next five years.

If the tests are successful in humans, it could be a major breakthrough in the development of new non-opioid, non-addictive pain management strategies for patients, the researchers said.

Thanks to funding from the NSW Ministry of Health, we are already moving towards testing in humans, said Professor Greg Neely, a leader in pain research at the Charles Perkins Centre and the School of Life and Environmental Sciences.

Nerve injury can lead to devastating neuropathic pain and for the majority of patients there are no effective therapies. This breakthrough means for some of these patients, we could make pain-killing transplants from their own cells, and the cells can then reverse the underlying cause of pain.

Link:
Pain treatment using human stem cells a success - News - The University of Sydney

The blood collected from the umbilical cord of the newborn is a rich source of stem cells. This blood is collected and sent to a cord blood bank, where the stem cells are separated, tested, processed, and preserved in liquid nitrogen. Technically, there is no expiry date and these stem cells can be preserved for a lifetime. Scientifically, evidence exists that they can be stored for about 20 years. The stem cells can treat around 70 blood related disorders and genetic disorders including thalassemia, sickle cell anaemia, leukaemia, and immune related disorders.

Stem cells taken from umbilical cord blood are like those taken from bone marrow, capable of producing all blood cells: red cells, platelets and immune system cells. When used, stem cells are first concentrated, then injected into the patient. Once transfused, they produce new cells of every kind.

They're capable of producing all types of blood cells: red cells, platelets and immune system cells. The stem cells can treat around 80 blood related illnesses like leukaemia, lymphomas, several genetic conditions and immune related disorders. But given the present state of medicine, they are effective only for around a dozen of them

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Watch | Stem cell banking and its benefits - The Hindu