Scientists generate millions of nave human pluripotent stem cells, far more than have ever been produced – UB News Center

BUFFALO, N.Y. For decades, the enormous disease-curing potential of human stem cells has been thwarted by the inability to produce sufficient quantities of mature human cells in vivo in a living organism.

Now, a team led by University at Buffalo scientists has developed a method that dramatically ramps up production of mature human cells in mouse embryos. Producing human cells in vivo is critical because cells made in a petri dish often do not behave the same way that cells do in the body.

The research was published on May 13 in Science Advances.

This is fundamental research that allows us to use the mouse embryo to help us better understand human development, said Jian Feng, PhD, corresponding author and professor of physiology and biophysics in the Jacobs School of Medicine and Biomedical Sciences at UB.

Further development of our technology could enable the generation of even larger quantities of specific types of mature human cells to allow us to create more effective mouse models to study diseases that gravely affect humans, such as malaria or COVID-19, said Feng.

And because this method produces so many mature human cells, it could potentially generate materials to treat chronic diseases, such as diabetes or kidney failure, by replacing a patients damaged cells with healthy human cells or tissues.

Infectious disease applications

Feng explained that it might be possible to create a much better mouse model of the human immune system or components of the human respiratory system in order to study COVID-19, a disease that wreaks havoc in humans, but barely affects mice.

It could also be possible to use the new method to produce mice with even more mature human red blood cells. Such mice would be very effective in the study of malaria, a disease which affects only humans by destroying our red blood cells.

We have a lot of questions to answer before the technology can be useful, but this is the first time that anyone has generated so many mature human cells in a mouse embryo, said Feng.

Millions of mature human cells in 17 days

Previous efforts to produce human cells in mouse embryos have generated small amounts of immature cells that are hard to quantify. In contrast, the UB method resulted in millions of mature human cells in a mouse embryo in 17 days.

In this study, the researchers injected 10-12 nave human stem cells into a mouse blastocyst when it was 3.5 days old. The mouse embryo then generated millions of mature human cells, including red blood cells, eye cells and liver cells, as it developed.

We know that up to four percent of the total number of cells in the mouse embryo were human cells, Feng. This is a low estimate because we cannot quantify the large amount of human red blood cells generated in the mouse embryo.

He said that because these mature human red blood cells do not have a nucleus, they are not counted by the method that the scientists use to quantify the total number of cells.

The teams technique involved overcoming an important challenge: Converting human pluripotent stem cells, which can differentiate into all types of cells in the body, into a form that is compatible with the inner cell mass inside a mouse blastocyst a three-day old mouse embryo. The human stem cells are in a primed state, whereas the inner cell mass inside the mouse blastocyst is in a nave state.

When the primed human cells are put into the mouse blastocyst, they fail to develop, said Feng, noting that the mismatch between the cells different developmental stages seems to be responsible.

We wanted to see if it was possible for the human primed cells to go back to the nave state, just like the pluripotent stem cells inside a mouse blastocyst, said Feng. This is what we have done.

Our method is to transiently inhibit the mTOR kinase for three hours to shock the human primed cells to the nave state, said Feng. Blocking the mTOR kinase triggers a series of events that rewire gene expression and cellular metabolism so that the primed cells become nave.

Converting the later stage human primed stem cells back to an earlier, less developed nave state allowed the human stem cells to co-develop with the inner cell mass in a mouse blastocyst.

The injected human stem cells now develop at the much more rapid pace of the mouse embryo, supporting the generation of millions of mature human cells in 17 days, said Feng.

In addition to Feng, UB co-authors are Zhixing Hu, Hanqin Li, Houbo Jiang, Yong Ren, and Boyang Zhang of the Department of Physiology and Biophysics, and Xinyang Yu and Michael J. Buck of the Department of Biochemistry, all of the Jacobs School. Other co-authors are Jingxin Qiu and Aimee B. Stablewski of the Roswell Park Comprehensive Cancer Center.

Funding for this research was provided by NYSTEM and the Buffalo Blue Sky Initiative.

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Scientists generate millions of nave human pluripotent stem cells, far more than have ever been produced - UB News Center

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

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

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

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

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

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

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

Sickle Cell Disease Data at EHA25

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

Transfusion-Dependent -Thalassemia Data at EHA25

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

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

Multiple Myeloma Data at EHA25

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Contacts

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

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

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

Regenerative Medicine Industry Outlook to 2025 Featuring Novartis, Vericel, Integra Lifesciences, Mimedx Group, Stryker, Wright Medical, Roche and…

DUBLIN, May 14, 2020 /PRNewswire/ -- The "Global Regenerative Medicine Market By Therapy (Cell-Based Immunotherapy & Cell Therapy, Gene Therapy, Others), By Application, By Material, By Cell, By Product, By Technique, By Distribution Channel, By Region, Forecast & Opportunities, 2025" report has been added to ResearchAndMarkets.com's offering.

The Global Regenerative Medicine Market is expected to register a double digit CAGR through 2025 owing to their increasing use in repair, replacement or regeneration of cells, tissues and organs. Additionally, high prevalence of chronic & genetic dieses, emergence of stem cell technology and growing aging populations are some of the key factors driving the regenerative medicine market.

Regenerative medicines deal with process of replacing, engineering or regenerating human or animal cells, tissues or organs to restore or establish normal function. They are also being used to create solutions for organs that become permanently damaged. These medicines are also used in treatment of some uncurable dieses like arthritis and diabetes.

Increasing number of cancer patients, neurodegenerative, orthopedic, and other aging-associated disorders is creating a significant demand for the regenerative medicine market globally. Various countries like United States, China and Japan are investing in stem cell research, which indicates a bright future for regenerative medicine manufacturers.

The Global Regenerative Medicine Market also faces some restraints like high treatment costs, stringent government regulations and operative inefficiency. High investment required for developing the medicine might also limit the market growth.

The market is segmented based on therapy, application, material, cell, product, technique, distribution channel and region. The application segment comprises of musculoskeletal disorders, wound care, oncology, neurology, ocular disorders, diabetes, cardiology and others. Out of them, the musculoskeletal segment is expected to dominate the market during the forecast years owing to growing use of regenerative medicines for treating musculoskeletal disorders and increasing number of orthopedic diseases.

Based on material, the regenerative medicine market is segmented into synthetic material, biologically derived material, genetically engineered material and pharmaceutical. The biologically derived material dominated the regenerative medicine market in 2019 and is expected to further hold its position in the coming years due to its unique properties. This type of material promotes cellular interactions, increases proliferation and controls the manipulation of cellular behavior.

Major players operating in the Global Regenerative Medicine Market include Novartis AG, Vericel, Integra Lifesciences, Mimedx Group, Stryker, Wright Medical, Roche, Bristol-Myers Squibb, Allergan, Corline Biomedical, Cook Biotech, Pfizer, Baxter, Boehringer Ingelheim, Caladrius Biosciences, Takara Bio, Medtronic, Osiris Therapeutics, Kite Pharma, Organogenesis and others. Due to growing demand from Asia-Pacific region, the manufacturers are focusing on countries like India and China where geriatric population is increasing rapidly.

Years considered for this report:

Objective of the Study

Key Topics Covered

1. Product Overview

2. Research Methodology

3. Impact of COVID-19 on Global Regenerative Medicine Market

4. Executive Summary

5. Voice of Customer

6. Global Regenerative Medicine Market Outlook6.1. Market Size & Forecast6.1.1. By Value & Volume6.2. Market Share & Forecast6.2.1. By Therapy (Cell-Based Immunotherapy & Cell Therapy, Gene Therapy, Tissue-Engineering, Immunomodulation therapy, Blood transfusion, Bone marrow transplantation, Plasma rich plasma therapy, Prolotherapy, Others)6.2.2. By Application (Musculoskeletal Disorders, Wound Care, Oncology, Neurology, Ocular Disorders, Diabetes, Cardiology, Others)6.2.3. By Material (Synthetic Material, Biologically Derived Material, Genetically Engineered Material, Pharmaceutical)6.2.3.1. By Synthetic Material (Biodegradable Synthetic Polymers, Scaffold, Artificial Vascular Graft Materials, Hydrogel Materials)6.2.3.2. By Biologically Derived Material (Collagen, Xenogeneic Material)6.2.3.3. By Genetically Engineered Material (Genetically Manipulated Cells, 3D Polymer Technology, Transgenic, Fibroblast, Neural Stem Cells, Gene-activated Matrices)6.2.3.4. By Pharmaceutical (Small Molecules, Biologics)6.2.4. By Cell (Autologous, Allogenic)6.2.5. By Product (Biologic, Cell -based Medical Devices, Biopharmaceutical, Biomaterial)6.2.6. By Technique (Microfracture, Mosaicplasty)6.2.7. By Distribution Channel (Hospitals, Clinics, Online, Others)6.2.8. By Region6.2.9. By Company

7. North America Regenerative Medicine Market Outlook

8. Europe Regenerative Medicine Market Outlook

9. Asia-Pacific Regenerative Medicine Market Outlook

10. Middle East & Africa Printing Ink Market Outlook

11. South America Regenerative Medicine Market Outlook

12. Market Dynamics12.1. Drivers12.2. Challenges

13. Market Trends & Developments

14. Competitive Landscape14.1. Competition Outlook14.2. Company Profiles14.2.1. Novartis AG14.2.2. Vericel14.2.3. Integra Lifesciences14.2.4. Mimedx Group14.2.5. Stryker14.2.6. Wright Medical14.2.7. Roche14.2.8. Bristol-Myers Squibb14.2.9. Allergan14.2.10. Corline Biomedical14.2.11. Cook Biotech14.2.12. Pfizer14.2.13. Baxter14.2.14. Boehringer Ingelheim14.2.15. Caladrius Biosciences14.2.16. Takara Bio14.2.17. Medtronic14.2.18. Osiris Therapeutics14.2.19. Kite Pharma14.2.20. Organogenesis

15. Strategic Recommendations

For more information about this report visit https://www.researchandmarkets.com/r/2zlobm

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

Media Contact:

Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com

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Regenerative Medicine Industry Outlook to 2025 Featuring Novartis, Vericel, Integra Lifesciences, Mimedx Group, Stryker, Wright Medical, Roche and...

Cell therapy firm boosts odds for blood cancer patients as key trial succeeds – The Times of Israel

Israels Gamida Cell, the manufacturer of a stem cell therapy that aims to increase the success of bone marrow transplants in blood cancer patients, said on Tuesday that a key late-stage clinical trial of its treatment has yielded positive results and met a major target.

Shares of the Jerusalem-based biotech firm were up 49 percent on the Nasdaq at the open of the exchange, on the news of the potentially life-saving treatment option for patients who need bone marrow transplants.

In the multinational Phase III clinical trial, conducted at more than 50 centers around the world, blood cancer patients who received bone marrow transplants were treated with the stem-cell based therapy Omidubicel, previously called NiCord.

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Those who were injected with the treatment containing expanded and enhanced stem cells had a median time of neutrophil engraftment that was significantly shorter than those that did not receive the drug: 12 days compared with 22 days for patients who received a standard umbilical cord blood transplant, the company said. Neutrophil engraftment is a measure of how quickly stem cells received in a transplant are established and begin to make healthy new cells. A rapid neutrophil engraftment has been associated with fewer infections and shorter hospitalizations.

said Julian Adams, the chief executive officer of Gamida Cell (Courtesy)

I am so thrilled and excited for Gamida and for patients, said the firms CEO Julian Adams, the chief executive officer of Gamida Cell in a phone interview. Omidubicel will transform the bone marrow transplant market we will grow the market and treat more patients and hopefully have more patients cured.

The treatment was given to cancer patients in remission who needed a transplant to stop the recurrence of the disease, he said.

Among patients who were transplanted per protocol, 96 percent of patients who received Omidubicel achieved successful neutrophil engraftment, compared to 88 percent of patients in the comparative group, the company said in a statement.

Omidubicel, which would be the first drug developed by Gamida to hit the market, is believed to increase the chances of a successful bone marrow transplantation process for patients who do not have a rapidly available, fully matched bone marrow donor.

Despite the curative potential of bone marrow transplant, it is estimated that more than 40 percent of eligible patients in the United States do not receive a transplant for various reasons, including the lack of a matched donor.

Today some high-risk blood cancers cannot be cured unless the patient undergoes a bone marrow graft. For that purpose, a perfect match needs to be found, a process that in the US takes an average of three to four months, if the patient is lucky. Sometimes, no match is found.

Umbilical cord blood collected from newborn babies contains stem cells, which can be used to treat diseases. Today cord-blood banks around the world store the cord blood. Its great advantage is that because it is so young, there is no need for a full tissue matching and a partial match is enough. Most patients generally find at least one unit of cord blood that partially matches them.

The problem is that the quantity of cells in each unit is not huge, and it is the number of stem cells in the cord blood that is critical to the success of transplantation.

Gamida overcomes this limitation by expanding the number of stem cells within one unit of umbilical cord blood and enhancing their performance.

Stem cells in bag in Gamida Cells Jerusalem lab, July 2017 (Shoshanna Solomon/TimesofIsrael)

These results have the potential to substantially move the field forward and represent an important step toward making stem cell transplantation more accessible and more successful for patients with lethal blood cancers, said Dr Mitchell Horwitz, principal investigator and professor of medicine at the Duke Cancer Institute. Shortening the time to engraftment is clinically meaningful, as it can reduce a patients time in the hospital and decrease likelihood of infection.

The trial included 125 patients aged 1265 years with acute lymphoblastic leukemia, acute myelogenous leukemia, chronic myelogenous leukemia, myelodysplastic syndrome or lymphoma. It was conducted at clinical centers in the United States, Latin America, Europe and Asia.

Omidubicel has the potential to be the first FDA-approved bone marrow transplant graft, said the CEO Adams.

There are still further steps ahead, he warned, with key secondary targets that need to be met, like evaluating if the treatment can reduce the number of infections as well as cut back on hospital days. Survival endpoints will also need to be met, he said. There is a lot of work to be done, Adams added. But the primary time-to- neutrophil-engrafment target, which the company has just met, is a milestone in a journey to bring Omidubicel to patients in the market.

The firm expects to apply for a US Food and Drug Administration license to market the product in the fourth quarter of 2020, he said. And if all goes well, then the firm will be in a position to launch Omidubicel in the US market in the second half of 2021.

Omidubicel is also being evaluated in an early clinical study in patients with severe aplastic anemia, the company said.

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Cell therapy firm boosts odds for blood cancer patients as key trial succeeds - The Times of Israel

Doctors just discovered another promising coronavirus therapy – BGR

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

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

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

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

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

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

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

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

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

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

Image Source: John Minchillo/AP/Shutterstock

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Tiziana Life Sciences Announces Data Demonstrating StemPrintER’s Superiority Compared to Oncotype DX in Providing Prognostic Information to…

This announcement contains inside information for the purposes of Article 7 of EU Regulation 596/2014.

NEW YORK and LONDON, May 14, 2020 (GLOBE NEWSWIRE) -- Tiziana Life Sciences plc (Nasdaq: TLSA) (Tiziana or the Company), a biotechnology company focused on innovative therapeutics for oncology, inflammation and infectious diseases, announces today that a new study will be presented by scientists from the European Institute of Oncology in Milan in collaboration with the Royal Marsden Hospital and Queen Mary University in London on the Companys stem cell biology-based genomic tool, StemPrintER, for the prediction of disease recurrence in breast cancer patients during a poster discussion session at the American Society of Clinical Oncology (ASCO) Virtual Conference. The study demonstrates greater refinement and superiority of StemPrintER over current market leader Oncotype DX in delivering prognostic information as part of the therapeutic decision-making process in ER+/HER2- breast cancer patients. An additional abstract from the scientists at the European Institute of Oncology will be presented in a separate poster session, which details a further refined risk model, the SPARE model, based on the combination of StemPrintER with clinical parameters for distant metastasis prediction. Both abstracts, became available May 13, 2020, while the posters and discussion session will be held during The American Society of Clinical Oncology (ASCO) Virtual Conference May 29-31, 2020.

Each of these studies independently provides significant additional information on the value of StemPrintER -- and its derivative SPARE (detailed below) -- as clinical tools to aid personalized therapeutic decision-making in women with ER+/HER2- breast cancer. Of particular note is the comparative precision of StemPrintER over Oncotype DX in predicting the potential recurrence of certain types of breast cancer following treatment. The two studies highlight the importance of the stem cell approach to develop a potentially powerful prognostic tool to predict breast cancer prognosis.

Abstract #1020, Comparison of StemPrintER, a novel biology-based genomic predictor of distant recurrence in breast cancer, with Oncotype DX in the TransATAC cohort, is an independent validation of the prognostic value of StemPrintER in a cohort of more than 800 luminal ER+/HER2- postmenopausal breast cancer patients from the international TransATAC study and a head-to-head comparison of the prognostic power of StemPrintER with Oncotype DX. Results provide independent validation of StemPrintER as a potentially powerful prognostic tool to stratify patients for the risk of early or late recurrence independently of other clinicopathological parameters. Importantly, the study also shows that StemPrintER is superior to Oncotype DX in the prediction of 10-year recurrence risk in all patients, as well as in N0 and N1-3 patients. The study further demonstrates that StemPrintER is capable of outperforming Oncotype DX in providing additional prognostic information to the standard clinicopathological parameters.

Abstract #1057, Integration of the stem cell biology-based genomic tool, StemPrintER, with clinicopathological parameters for the prediction of distant recurrence in ER+/HER2- breast cancer (BC) patients, develops a more refined risk model for distant metastasis prediction, which combines StemPrintER with tumor size (pT) and nodal status (pN). The new model is termed SPARE (StemPrintER for Personalized Adjuvant Therapy in Endocrine Receptor-Expressing Patients) and, in the analysis of a consecutive-retrospective cohort of more than 1,800 ER+/HER2- breast cancer patients with 15-year complete follow-up from the European Institute of Oncology (IEO) in Milan, revealed to be an even more powerful tool, compared to the original StemPrintER for predicting early and late distant metastasis risk independently of standard clinical parameters.

These data sets demonstrate that StemPrintER has the potential to become an essential prognostic tool that will help clinicians to tailor more or less aggressive therapy based on a more accurate risk assessment of disease recurrence compared to what we have seen to date, added Dr. Kunwar Shailubhai, CEO & CSO of Tiziana Life Sciences. This product also represents an important addition to our existing therapeutic pipeline as it opens Tiziana into the area of precision medicine, creating an entirely new business line beyond our current patented technology in offering new delivery mechanisms for monoclonal antibodies.

About StemPrintERStemPrintER is a multi-gene prognostic assay intended for the prediction of the risk of recurrence in luminal, estrogen receptor-positive HER2-negative breast cancer patients, based on the detection of 20 cancer stem cell markers. The assay has been evaluated in an initial retrospective validation study using a consecutive cohort of approximately 2,400 patients with breast cancer.

The person who arranged for the release of this information is Dr Kunwar Shailubhai, the Company's Chief Executive Officer and Chief Scientific Officer.

About Tiziana Life SciencesTiziana Life Sciences plc is adual listed (NASDAQ: TLSA & UK AIMS: TILS)biotechnology company that focuses on the discovery and development of novel molecules to treat human diseases in oncology, inflammation and infectious diseases. In addition to milciclib, the Company will be shortly initiating phase 2 studies with orally administered foralumab for Crohns Disease and nasally administered foralumab for progressive multiple sclerosis. Foralumab is the only fully human anti-CD3 monoclonal antibody (mAb) in clinical development in the world. This phase II compound has potential application in a wide range of autoimmune and inflammatory diseases, such as Crohns Disease, multiple sclerosis, type-1 diabetes (T1D), inflammatory bowel disease (IBD), psoriasis and rheumatoid arthritis, where modulation of a T-cell response is desirable. The company is accelerating development of anti-Interleukin 6 receptor (IL6R) mAb, a fully human monoclonal antibody for treatment of IL6-induced inflammation, especially for treatment of COVID-19 patients.

Receive news and updates from Tiziana Life Sciences plc by signing up to get email alerts athttps://ir.tizianalifesciences.com.

Forward-Looking StatementsCertain statements made in this announcement are forward-looking statements. These forward-looking statements are not historical facts but rather are based on the Companys current expectations, estimates, and projections about its industry; its beliefs; and assumptions. Words such as anticipates, expects, intends, plans, believes, seeks, estimates, and similar expressions are intended to identify forward-looking statements. These statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties, and other factors, some of which are beyond the Companys control, are difficult to predict, and could cause actual results to differ materially from those expressed or forecasted in the forward-looking statements. The Company cautions security holders and prospective security holders not to place undue reliance on these forward-looking statements, which reflect the view of the Company only as of the date of this announcement. The forward-looking statements made in this announcement relate only to events as of the date on which the statements are made. The Company will not undertake any obligation to release publicly any revisions or updates to these forward-looking statements to reflect events, circumstances, or unanticipated events occurring after the date of this announcement except as required by law or by any appropriate regulatory authority.

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Tiziana Life Sciences Announces Data Demonstrating StemPrintER's Superiority Compared to Oncotype DX in Providing Prognostic Information to...

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Global Animal Stem Cell Therapy Market Types, Application, and Regions, Forecast 2020- 2025. :Global Globalmarketers.biz - Cole of Duty