Los Angeles, United State, The report titledGlobal Hematopoietic Stem Cell Transplantation (HSCT) Marketis one of the most comprehensive and important additions to QY Researchs archive of market research studies. It offers detailed research and analysis of key aspects of the global Hematopoietic Stem Cell Transplantation (HSCT) market. The market analysts authoring this report have provided in-depth information on leading growth drivers, restraints, challenges, trends, and opportunities to offer a complete analysis of the global Hematopoietic Stem Cell Transplantation (HSCT) market. Market participants can use the analysis on market dynamics to plan effective growth strategies and prepare for future challenges beforehand. Each trend of the global Hematopoietic Stem Cell Transplantation (HSCT) market is carefully analyzed and researched about by the market analysts.

Global Hematopoietic Stem Cell Transplantation (HSCT) Market is valued at USD XX million in 2019 and is projected to reach USD XX million by the end of 2025, growing at a CAGR of XX% during the period 2019 to 2025.

Top Key Players of the Global Hematopoietic Stem Cell Transplantation (HSCT) Market : Regen Biopharma Inc, China Cord Blood Corp, CBR Systems Inc, Escape Therapeutics Inc, Cryo-Save AG, Lonza Group Ltd, Pluristem Therapeutics Inc, ViaCord Inc Hematopoietic Stem Cell Transplantation (HSCT)

>>>Download Full PDF Sample Copy of Report: (Including Full TOC, List of Tables & Figures, Chart) :

https://www.qyresearch.com/sample-form/form/1528548/global-hematopoietic-stem-cell-transplantation-hsct-market

The Essential Content Covered in the Global Hematopoietic Stem Cell Transplantation (HSCT) Market Report :

Top Key Company Profiles.Main Business and Rival InformationSWOT Analysis and PESTEL AnalysisProduction, Sales, Revenue, Price and Gross MarginMarket Size And Growth RateCompany Market Share

Global Hematopoietic Stem Cell Transplantation (HSCT) Market Segmentation By Product:, Allogeneic, Autologous Hematopoietic Stem Cell Transplantation (HSCT)

Global Hematopoietic Stem Cell Transplantation (HSCT) Market Segmentation By Application: Peripheral Blood Stem Cells Transplant (PBSCT), Bone Marrow Transplant (BMT), Cord Blood Transplant (CBT)

In terms of region, this research report covers almost all the major regions across the globe such asNorth America, Europe, South America, the Middle East, and Africa and the Asia Pacific. Europe and North America regions are anticipated to show an upward growth in the years to come. WhileHematopoietic Stem Cell Transplantation (HSCT) Market in Asia Pacific regions is likely to show remarkable growth during the forecasted period. Cutting edge technology and innovations are the most important traits of the North America region and thats the reason most of the time the US dominates the global markets.Hematopoietic Stem Cell Transplantation (HSCT) Market in South, America region is also expected to grow in near future.

Key questions answered in the report

*What will be the market size in terms of value and volume in the next five years?*Which segment is currently leading the market?*In which region will the market find its highest growth?*Which players will take the lead in the market?*What are the key drivers and restraints of the markets growth?

We provide detailed product mapping and analysis of various market scenarios. Our analysts are experts in providing in-depth analysis and breakdown of the business of key market leaders. We keep a close eye on recent developments and follow latest company news related to different players operating in the global Hematopoietic Stem Cell Transplantation (HSCT) market. This helps us to deeply analyze companies as well as the competitive landscape. Our vendor landscape analysis offers a complete study that will help you to stay on top of the competition.

Why to Buy this Report?

>>>Get Full Report in your inbox within 24 hours at USD(3900): https://www.qyresearch.com/settlement/pre/99cec1e27c598bf4f0d2c4a69d60697f,0,1,global-hematopoietic-stem-cell-transplantation-hsct-market

Table of Contents

1 Report Overview1.1 Study Scope1.2 Key Market Segments1.3 Players Covered: Ranking by Hematopoietic Stem Cell Transplantation (HSCT) Revenue1.4 Market by Type1.4.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size Growth Rate by Type: 2020 VS 20261.4.2 Allogeneic1.4.3 Autologous1.5 Market by Application1.5.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Share by Application: 2020 VS 20261.5.2 Peripheral Blood Stem Cells Transplant (PBSCT)1.5.3 Bone Marrow Transplant (BMT)1.5.4 Cord Blood Transplant (CBT)1.6 Study Objectives1.7 Years Considered 2 Global Growth Trends2.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Perspective (2015-2026)2.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Growth Trends by Regions2.2.1 Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Regions: 2015 VS 2020 VS 20262.2.2 Hematopoietic Stem Cell Transplantation (HSCT) Historic Market Share by Regions (2015-2020)2.2.3 Hematopoietic Stem Cell Transplantation (HSCT) Forecasted Market Size by Regions (2021-2026)2.3 Industry Trends and Growth Strategy2.3.1 Market Top Trends2.3.2 Market Drivers2.3.3 Market Challenges2.3.4 Porters Five Forces Analysis2.3.5 Hematopoietic Stem Cell Transplantation (HSCT) Market Growth Strategy2.3.6 Primary Interviews with Key Hematopoietic Stem Cell Transplantation (HSCT) Players (Opinion Leaders) 3 Competition Landscape by Key Players3.1 Global Top Hematopoietic Stem Cell Transplantation (HSCT) Players by Market Size3.1.1 Global Top Hematopoietic Stem Cell Transplantation (HSCT) Players by Revenue (2015-2020)3.1.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Revenue Market Share by Players (2015-2020)3.1.3 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Share by Company Type (Tier 1, Tier 2 and Tier 3)3.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Concentration Ratio3.2.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Concentration Ratio (CR5 and HHI)3.2.2 Global Top 10 and Top 5 Companies by Hematopoietic Stem Cell Transplantation (HSCT) Revenue in 20193.3 Hematopoietic Stem Cell Transplantation (HSCT) Key Players Head office and Area Served3.4 Key Players Hematopoietic Stem Cell Transplantation (HSCT) Product Solution and Service3.5 Date of Enter into Hematopoietic Stem Cell Transplantation (HSCT) Market3.6 Mergers & Acquisitions, Expansion Plans 4 Market Size by Type (2015-2026)4.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Historic Market Size by Type (2015-2020)4.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Forecasted Market Size by Type (2021-2026) 5 Market Size by Application (2015-2026)5.1 Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020)5.2 Global Hematopoietic Stem Cell Transplantation (HSCT) Forecasted Market Size by Application (2021-2026) 6 North America6.1 North America Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)6.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in North America (2019-2020)6.3 North America Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)6.4 North America Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 7 Europe7.1 Europe Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)7.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in Europe (2019-2020)7.3 Europe Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)7.4 Europe Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 8 China8.1 China Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)8.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in China (2019-2020)8.3 China Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)8.4 China Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 9 Japan9.1 Japan Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)9.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in Japan (2019-2020)9.3 Japan Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)9.4 Japan Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 10 Southeast Asia10.1 Southeast Asia Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)10.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in Southeast Asia (2019-2020)10.3 Southeast Asia Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)10.4 Southeast Asia Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 11 India11.1 India Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)11.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in India (2019-2020)11.3 India Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)11.4 India Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 12 Central & South America12.1 Central & South America Hematopoietic Stem Cell Transplantation (HSCT) Market Size (2015-2020)12.2 Hematopoietic Stem Cell Transplantation (HSCT) Key Players in Central & South America (2019-2020)12.3 Central & South America Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Type (2015-2020)12.4 Central & South America Hematopoietic Stem Cell Transplantation (HSCT) Market Size by Application (2015-2020) 13 Key Players Profiles13.1 Regen Biopharma Inc13.1.1 Regen Biopharma Inc Company Details13.1.2 Regen Biopharma Inc Business Overview13.1.3 Regen Biopharma Inc Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.1.4 Regen Biopharma Inc Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020))13.1.5 Regen Biopharma Inc Recent Development13.2 China Cord Blood Corp13.2.1 China Cord Blood Corp Company Details13.2.2 China Cord Blood Corp Business Overview13.2.3 China Cord Blood Corp Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.2.4 China Cord Blood Corp Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.2.5 China Cord Blood Corp Recent Development13.3 CBR Systems Inc13.3.1 CBR Systems Inc Company Details13.3.2 CBR Systems Inc Business Overview13.3.3 CBR Systems Inc Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.3.4 CBR Systems Inc Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.3.5 CBR Systems Inc Recent Development13.4 Escape Therapeutics Inc13.4.1 Escape Therapeutics Inc Company Details13.4.2 Escape Therapeutics Inc Business Overview13.4.3 Escape Therapeutics Inc Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.4.4 Escape Therapeutics Inc Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.4.5 Escape Therapeutics Inc Recent Development13.5 Cryo-Save AG13.5.1 Cryo-Save AG Company Details13.5.2 Cryo-Save AG Business Overview13.5.3 Cryo-Save AG Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.5.4 Cryo-Save AG Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.5.5 Cryo-Save AG Recent Development13.6 Lonza Group Ltd13.6.1 Lonza Group Ltd Company Details13.6.2 Lonza Group Ltd Business Overview13.6.3 Lonza Group Ltd Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.6.4 Lonza Group Ltd Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.6.5 Lonza Group Ltd Recent Development13.7 Pluristem Therapeutics Inc13.7.1 Pluristem Therapeutics Inc Company Details13.7.2 Pluristem Therapeutics Inc Business Overview13.7.3 Pluristem Therapeutics Inc Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.7.4 Pluristem Therapeutics Inc Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.7.5 Pluristem Therapeutics Inc Recent Development13.8 ViaCord Inc13.8.1 ViaCord Inc Company Details13.8.2 ViaCord Inc Business Overview13.8.3 ViaCord Inc Hematopoietic Stem Cell Transplantation (HSCT) Introduction13.8.4 ViaCord Inc Revenue in Hematopoietic Stem Cell Transplantation (HSCT) Business (2015-2020)13.8.5 ViaCord Inc Recent Development 14 Analysts Viewpoints/Conclusions 15 Appendix15.1 Research Methodology15.1.1 Methodology/Research Approach15.1.2 Data Source15.2 Disclaimer15.3 Author Details

About Us:

QYResearch always pursuits high product quality with the belief that quality is the soul of business. Through years of effort and supports from the huge number of customer supports, QYResearch consulting group has accumulated creative design methods on many high-quality markets investigation and research team with rich experience. Today, QYResearch has become a brand of quality assurance in the consulting industry.

More here:
Hematopoietic Stem Cell Transplantation (HSCT) Market is Thriving Worldwide with Top Companies like: China Cord Blood Corp, CBR Systems Inc, Escape...

DUARTE, Calif.--(BUSINESS WIRE)--City of Hope, a world-renowned independent research and treatment center for cancer, diabetes and other life-threatening diseases, today announced that it has licensed intellectual property relating to its pioneering chlorotoxin chimeric antigen receptor (CLTX-CAR) T cell therapy to Chimeric Therapeutics Limited, an Australian biotechnology company.

The therapy is currently being used in a phase 1 clinical trial at City of Hope to treat glioblastoma (GBM), a type of brain tumor. The first patient in the trial was recently dosed; Behnam Badie, M.D., chief of City of Hopes Division of Neurosurgery and The Heritage Provider Network Professor in Gene Therapy, is leading this innovative, first-of-its-kind trial.

Chimeric has acquired the exclusive worldwide rights to develop and commercialize certain patents relating to City of Hopes CLTX-CAR T cells, as well as to further develop the therapy for other cancers.

City of Hope is excited to enter into this agreement with Chimeric as it supports our innovative research in CAR T cell therapy and our commitment to extend these therapies to more patients, particularly those with GBM and other solid tumors that are difficult to treat, said Christine Brown, Ph.D., The Heritage Provider Network Professor in Immunotherapy and deputy director of City of Hopes T Cell Therapeutics Research Laboratory. Chimeric shares our goal of providing effective CAR T cell therapies to more patients with current unmet medical needs.

Led by Brown and Michael Barish, Ph.D., chair of City of Hopes Department of Developmental and Stem Cell Biology, and Dongrui Wang, Ph.D., a recent graduate of City of Hopes Irell & Manella Graduate School of Biological Sciences, the team developed and tested the first CAR T cell therapy using CLTX, a component of scorpion venom, to direct T cells to target brain tumor cells. The research was published this past March in Science Translational Medicine.

Chimeric is excited to join City of Hope in its quest to find more effective cancer therapies. This is an exceedingly rare opportunity to acquire a promising technology in one of the most exciting areas of immuno-oncology today, said Paul Hopper, executive chairman of Chimeric. Furthermore, the CLTX-CAR T cell therapy has completed years of preclinical research and development, and recently enrolled its first patient in a phase 1 clinical trial for brain cancer.

CARs commonly incorporate a monoclonal antibody sequence in their targeting domain, enabling CAR T cells to recognize antigens and kill tumor cells. In contrast, the CLTX-CAR uses a synthetic 36-amino acid peptide sequence first isolated from death stalker scorpion venom and now engineered to serve as the CAR recognition domain.

In this recent study, City of Hope researchers used tumor cells in resection samples from a cohort of patients with GBM to compare CLTX binding with expression of antigens currently under investigation as CAR T cell targets. They found that CLTX bound to a greater proportion of patient tumors, and cells within these tumors.

CLTX binding included the GBM stem-like cells thought to seed tumor recurrence. Consistent with these observations, CLTX-CAR T cells recognized and killed broad populations of GBM cells while ignoring nontumor cells in the brain and other organs. The study team demonstrated that CLTX-directed CAR T cells are highly effective at selectively killing human GBM cells without off-tumor targeting and toxicity in cell-based assays and in animal models.

City of Hope, a recognized leader in CAR T cell therapies for GBM and other cancers, has treated more than 500 patients since its CAR T program started in the late 1990s. The institution continues to have one of the most comprehensive CAR T cell clinical research programs in the world it currently has 30 ongoing CAR T cell clinical trials, including CAR T cell trials for HER-2 positive breast cancer that has spread to the brain, and PSCA-positive bone metastatic prostate cancer. It was the first and only cancer center to treat GBM patients with CAR T cells targeting IL13R2, and the first to administer CAR T cell therapy locally in the brain, either by direct injection at the tumor site, through intraventricular infusion into the cerebrospinal fluid, or both. In late 2019, City of Hope opened a first-in-human clinical trial for patients with recurrent GBM, combining IL13R2-CAR T cells with checkpoint inhibitors nivolumab, an anti-PD1 antibody, and ipilimumab, blocking the CTLA-4 protein.

Both an academic medical center and a drug development powerhouse, City of Hope is known for creating the technology used in the development of human synthetic insulin and numerous breakthrough cancer drugs. Its unique research and development hybrid of the academic and commercial creates an infrastructure that enables City of Hope researchers to submit an average of 50 investigational new drug applications to the U.S. Food and Drug Administration each year. The institution currently holds more than 450 patent families.

"City of Hope is delighted to license this technology to Chimeric, said Sangeeta Bardhan Cook, Ph.D., City of Hope director of the Office of Technology Licensing. We are impressed with the ability of their executive team to push and bring therapies to market expeditiously. At City of Hope, our mission is to transform the future of health care. We believe Chimeric has the vision to offer innovative therapies to cancer patients.

About City of Hope

City of Hope is an independent biomedical research and treatment center for cancer, diabetes and other life-threatening diseases. Founded in 1913, City of Hope is a leader in bone marrow transplantation and immunotherapy such as CAR T cell therapy. City of Hopes translational research and personalized treatment protocols advance care throughout the world. Human synthetic insulin and numerous breakthrough cancer drugs are based on technology developed at the institution. A National Cancer Institute-designated comprehensive cancer center and a founding member of the National Comprehensive Cancer Network, City of Hope has been ranked among the nations Best Hospitals in cancer by U.S. News & World Report for 14 consecutive years. Its main campus is located near Los Angeles, with additional locations throughout Southern California. For more information about City of Hope, follow us on Facebook, Twitter, YouTube or Instagram.

View post:
City of Hope Enters Licensing Agreement With Chimeric to Develop Its Pioneering Chlorotoxin CAR T Cell Therapy - Business Wire

NEW YORK, Sept. 23, 2020 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (the "Company" or "Actinium") today announced that it has successfully completed the first dosing cohort in the Actimab-A and venetoclax combination, multi-center Phase 1 trial for patients with Relapsed or Refractory ("R/R") Acute Myeloid Leukemia (AML) age 18 and above. All patients from the first dosing cohort (0.50 uCi/kg of Actimab-A) completed treatment and cleared their initial safety evaluation, thus allowing the study to proceed to the second dose cohort of 1.0 uCi/kg Actimab-A added to venetoclax. In a poster presentation at the American Association of Cancer Research (AACR) Annual Meeting 2019, Actimab-A was shown to be synergistic with venetoclax in venetoclax resistant cell lines, by depleting MCL-1, a protein shown to mediate resistance to venetoclax. The ongoing Phase 1 study was planned to replicate this synergy in a clinical setting. Actinium plans to report study proof of concept results in 2021.

Venetoclax is a B-Cell Lymphoma 2 (BCL-2) inhibitor jointly developed and marketed by AbbVie and Genentech that is approved in combination with hypomethylating agents ("HMAs") for patients with AML. The use of venetoclax has become widespread in the treatment of fit and unfit patients with R/R AML following its inclusion in the recently expanded National Comprehensive Cancer Network ("NCCN") guidelines. Actinium's preclinical research has demonstrated that by adding Actimab-A to venetoclax, the targeted internalized radiation from Actimab-A can deliver potent AML cell killing, as well as effectively deplete MCL-1 levels. The overexpression of MCL-1, a member of the BCL-2 family which venetoclax does not inhibit, promotes resistance to venetoclax. Thus, Actimab-A reverses resistance to venetoclax and has independent anti-leukemic activity mediated by CD33 as well.

"We are pleased to confirm that the second combination trial in our CD33 program is advancing through the dose escalation study as planned. Despite approval in multiple blood cancers, including AML, most AML patients are not cured with venetoclax regimens and eventually relapse. Based on the preclinical data, synergy with venetoclax and Actimab-A should lead to higher remission rates in R/R AML," said Dr. Mark Berger, Actinium's Chief Medical Officer. "We continue to generate promising data from our broader combination program. For example, the Actimab-A combination trial with chemotherapy agent CLAG-M increased the complete response rate compared to CLAG-M alone in R/R AML patients by 60%. We expect to complete the proof of concept Actimab-A venetoclax combination trial in 2021."

This Phase 1 study is a multicenter, open label trial of Actimab-A added to venetoclax for patients with CD33 positive R/R AML. The study will continue to enroll patients that have been previously treated with venetoclax as well as venetoclax nave patients. Gary Schiller, MD, Professor, Hematology-Oncology and Director, Hematologic Malignancy/Stem Cell Transplant Program at the UCLA Medical Center is the Principal Investigator for this study. The trial is also active at the University of Louisville.

Sandesh Seth, Actinium's Chairman and Chief Executive Officer, said, "We continue to advance the CD33 program for fit and unfit R/R AML patients as there is still a significant unmet need despite multiple recently approved agents. These therapeutic agents are not curative and patients continue to experience low response rates and/or high relapse rates. Our CD33 program, which also includes the Actimab-A CLAG-M combination trial, is anchored in leveraging mechanistic synergies of Actimab-A with approved or novel therapeutic agents in order to improve patient outcomes. We look forward to multiple clinical trial updates by year-end from our three ongoing trials in R/R AML, including our Iomab-B SIERRA Phase 3 pivotal trial."

Rationale for Actimab-A Venetoclax Combination Trial

This Phase 1/2 trial is a multicenter, open label trial of Actimab-A (lintuzumab-Ac225) added to venetoclax for patients with CD33 positive relapsed/refractory (R/R) Acute Myeloid Leukemia. The Phase 1 portion of the study is designed to determine the maximum tolerated dose (MTD) of Actimab-A added to venetoclax for R/R AML. The Phase 2 portion of the trial will assess the percentage of patients with CR, CRh, or Overall Response (CR + CRh), up to six months after the start of the treatment without receiving other AML therapies. The trial will enroll R/R AML patients who have been treated with venetoclax as well as venetoclax-nave patients. At the 1.0 uCi/kg dose, Actimab-A is administered on Day 1 of each cycle for four cycles and venetoclax is taken on Days 1-21 of each cycle for up to 4 cycles. Each cycle is 28 days, with a potential to expand to 42 days to allow for full hematologic recovery. Gary Schiller, MD, Professor, Hematology-Oncology and Director, Hematologic Malignancy/Stem Cell Transplant Program at the UCLA Medical Center is the Principal Investigator for this study.

More information on the clinical trial design is available at clinicaltrials.gov (NCT03867682).

About Actinium's CD33 Program (Actimab-A)

Antibody Radiation Conjugate (ARC) Actimab-A targets the CD33 antigen that is expressed on virtually all AML cells with the antibody lintuzumab which delivers potent alpha radiation via its Actinium-225 radioisotope payload. Blood cancers like AML are highly sensitive to radiation but cannot treated with the current standard of external beam delivery because the disease is too widespread throughout the body. The combination of targeted radiation with Actimab-A potentially allows for greater cancer cell death than a standalone chemotherapy regimen such as CLAG-M or venetoclax, which are frequently used in the treatment of fit and unfit patients with relapsed or refractory AML per National Comprehensive Cancer Network (NCCN) guidelines. Prior clinical results in over 100 patients treated with Actimab-A, including a Phase 1/2 trial of 58 patients, demonstrated a safety profile with minimal non-hematologic toxicities and an unmatched ability to deliver attenuated doses of radiation internally to CD33 expressing cancer cells. In the Phase 1/2 trial, Actimab-A as a single agent produced a 69% remission rate (CR, CRi, CRp) at high doses in patients with newly diagnosed AML but Actinium elected to pursue low dose combination trials for therapeutic development based on observed myelosuppression. In the Actimab-A CLAG-M Phase 1 combination trial, the second cohort with CLAG-M plus the 0.50 uCi/kg dose showed that 86% (6/7) of patients achieved complete remission (CR/CRi) after receiving the 0.50 uCi/kg dose of Actimab-A. This is a nearly 60% increase over the remission rate reported in a trial of seventy-four patients with relapsed or refractory AML who received CLAG-M alone. The company expects trial results, including the third dose cohort, in 2020. The Actimab-A Venetoclax Phase 1 trial continues to enroll patients in a maximum tolerated dose and expects to announce proof-of-concept results in 2021.

About Actinium Pharmaceuticals, Inc. (NYSE: ATNM)

Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation. Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively deplete a patient's disease or cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, Gene Therapy or Adoptive Cell Therapy (ACT) such as CAR-T to enable engraftment of these transplanted cells with minimal toxicities. With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently. Our lead product candidate, I-131 apamistamab (Iomab-B) is being studied in the ongoing pivotal Phase 3 Study of Iomab-B in Elderly Relapsed or Refractory Acute Myeloid Leukemia (SIERRA) trial for BMT conditioning. The SIERRA trial is over fifty percent enrolled and positive single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings. I-131 apamistamab will also be studied as a targeted conditioning agent in a Phase 1/2 anti-HIV stem cell gene therapy with UC Davis and is expected to be studied with a CAR-T therapy in 2020. In addition, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Ongoing combination trials include our CD33 alpha ARC, Actimab-A, in combination with the salvage chemotherapy CLAG-M and the Bcl-2 targeted therapy venetoclax. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform. This is where our intellectual property portfolio of over 100 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes. Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc. Website: https://www.actiniumpharma.com/

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Contacts:

Investors:Clayton RobertsonActinium Pharmaceuticals, Inc.crobertson@actiniumpharma.com

Hans VitzthumLifeSci Advisors, LLCHans@LifeSciAdvisors.com(617) 535-7743

View original content to download multimedia:http://www.prnewswire.com/news-releases/actinium-pharmaceuticals-successfully-completes-first-dosing-cohort-in-the-phase-1-study-of-actimab-a-and-venetoclax-combination-therapy-in-relapsedrefractory-aml-patients-301136354.html

SOURCE Actinium Pharmaceuticals, Inc.

Company Codes: AMEX:ATNM

See the rest here:
Actinium Pharmaceuticals Successfully Completes First Dosing Cohort in the Phase 1 Study of Actimab-A and Venetoclax Combination Therapy in...

Fort Collins, Colorado Reports Globe recently added the Stem Cell Therapy Market Research Report that provides a thorough investigation of the market scenario of the market size, share, demand, growth, trends, and forecast from 2020-2027. The report covers the impact analysis of the COVID-19 pandemic. COVID-19 pandemic has affected the export-import, demands, and trends of the industry and is expected to have some economic impact on the market. The report provides a comprehensive analysis of the impact of the pandemic on the overall industry and offers insights into a post-COVID-19 market scenario.

The report primarily mentions definitions, classifications, applications, and market overview of the Stem Cell Therapy industry. It also covers product portfolios, manufacturing processes, cost analysis, structures, and gross margin of the industry. It also provides a comprehensive analysis of the key competitors and their regional spread and market size.

Global Stem Cell TherapyMarketwas valued at 117.66 million in 2019 and is projected to reach USD255.37 million by 2027, growing at a CAGR of 10.97% from 2020 to 2027.

Get a sample of the report @ https://reportsglobe.com/download-sample/?rid=33553

Competitive Analysis:

The report provides a comprehensive analysis of the companies operating in the Stem Cell Therapy market, along with their overview, business plans, strengths, and weaknesses to provide a substantial analysis of the growth through the forecast period. The evaluation provides a competitive edge and understanding of their market position and strategies undertaken by them to gain a substantial market size in the global market.

Key features of the Report:

The report covers extensive analysis of the key market players in the market, along with their business overview, expansion plans, and strategies. The key players studied in the report include:

Request a Discount on the report @ https://reportsglobe.com/ask-for-discount/?rid=33553

Additionally, the report is furnished by the advanced analytical data from SWOT analysis, Porters Five Forces Analysis, Feasibility Analysis, and Investment Return Analysis. The report also provides a detailed analysis of the mergers, consolidations, acquisitions, partnerships, and government deals. Along with this, an in-depth analysis of current and emerging trends, opportunities, threats, limitations, entry-level barriers, restraints and drivers, and estimated market growth throughout the forecast period are offered in the report.

Market Breakdown:

The market breakdown provides market segmentation data based on the availability of the data and information. The market is segmented on the basis of types and applications.

1.Stem Cell Therapy Market, By Cell Source:

Adipose Tissue-Derived Mesenchymal Stem Cells Bone Marrow-Derived Mesenchymal Stem Cells Cord Blood/Embryonic Stem Cells Other Cell Sources

2.Stem Cell Therapy Market, By Therapeutic Application:

Musculoskeletal Disorders Wounds and Injuries Cardiovascular Diseases Surgeries Gastrointestinal Diseases Other Applications

3.Stem Cell Therapy Market, By Type:

Allogeneic Stem Cell Therapy Market, By Application Musculoskeletal Disorders Wounds and Injuries Surgeries Acute Graft-Versus-Host Disease (AGVHD) Other Applications Autologous Stem Cell Therapy Market, By Application Cardiovascular Diseases Wounds and Injuries Gastrointestinal Diseases Other Applications

Request customization of the report @https://reportsglobe.com/need-customization/?rid=33553

The report provides additional analysis about the key geographical segments of the Stem Cell Therapy Market and provides analysis about their current and previous share. Current and emerging trends, challenges, opportunities, and other influencing factors are presented in the report.

Regional analysis includes an in-depth study of the key geographical regions to gain a better understanding of the market and provide an accurate analysis. The regional analysis coversNorth America, Latin America, Europe, Asia-Pacific, and the Middle East & Africa.

Objectives of the Report:

To learn more about the report, visit @ https://reportsglobe.com/product/global-stem-cell-therapy-market/

Thank you for reading our report. To learn more about report details or for customization information, please contact us. Our team will ensure that the report is customized according to your requirements.

How Reports Globe is different than other Market Research Providers

The inception of Reports Globe has been backed by providing clients with a holistic view of market conditions and future possibilities/opportunities to reap maximum profits out of their businesses and assist in decision making. Our team of in-house analysts and consultants works tirelessly to understand your needs and suggest the best possible solutions to fulfill your research requirements.

Our team at Reports Globe follows a rigorous process of data validation, which allows us to publish reports from publishers with minimum or no deviations. Reports Globe collects, segregates, and publishes more than 500 reports annually that cater to products and services across numerous domains.

Contact us:

Mr. Mark Willams

Account Manager

US: +1-970-672-0390

Email:[emailprotected]

Web:reportsglobe.com

Read the original here:
Stem Cell Therapy Market Size, Analytical Overview, Key Players, Growth Factors, Demand, Trends And Forecast to 2027 - The Daily Chronicle

InYourArea Community

Blood Cancer charity DKMS thanks the public in the North West for going against the grain and registering as blood stem donors.

DKMS is thanking people in the North West this Blood Cancer Awareness Month (September, 1-30 2020) for registering as blood stem cell donors during lockdown. The North West has provided the fourth highest number of registrations during lockdown. However there is still an urgent need for registrations to help meet the demand and reverse the shocking decline in new registrations in recent times and potentially help save a life.

Every 20 minutes someone in the UK is diagnosed with a blood cancer and it is the third most common cause of cancer death in the UK. A stem cell transplant can be the last hope of survival for patients and during the coronavirus outbreak, it is even more important to offer hope to people with devastating blood cancers and blood disorders, whose lives have also been harmed by the pandemic.

A blood stem cell donation from a genetically similar person can offer a second chance at life for those in need. Only 1 in 3 people with blood cancer (and in need of a transplant) will find a matching blood stem cell donor within their own family which means that 2 in 3 need to look outside of this and rely on an altruistic stranger to help give them more time with their family.

Many people are unaware of how they can donate blood stem cells. Around 90% of all donations are made through a method called peripheral blood stem cell. While just 10% of cases, donations are made through bone marrow collection, which contrary to what people think, is not extracted from the spine but from the pelvic bone.

DKMS Donor Recruitment Manager, Louise Clague, 50, from Cheshire joined the fight against blood cancer and is on a mission to register as many potential blood stem cell donors as possible after her husband, Andy Clague, died in December 2017, aged 46, following a two-year battle with non-Hodgkins lymphoma.

Louise said: Raising awareness of blood cancer and how people can become potential blood stem cell donors has partly been my way of dealing with grief and having something where you feel like youre making a difference. I have a son and a daughter who are now 14 and 17 who have lost a dad. You dont want other people to go through the same ordeal.

"We were referred to as the fit family and we have no history of the condition in the family but it happened to us. So that is part of the message, it could be you and if it happened to you and your family, would you give your blood stem cells? I think a lot of people would and I think people havent signed up yet because theyve never come across the condition."

Taking the first steps to register as a potential blood stem cell donor can be done within a few minutes from the comfort of your own home. If you are aged between 17-55 and in general good health you can sign up for a home swab kit on the DKMS website. Your swabs can then be returned with the enclosed pre-paid envelope to DKMS in order to ensure that your details are added to the UKs aligned stem cell registry.

Read the original here:
Blood Cancer charity thanks the North West for registering as donors - In Your Area

A new research report published by Fior Markets with the titleCord Blood Banking Market by Type of Bank (Public, Private), Services (Processing, Storage), Application (Cancer, Blood Disorders, Immuno-deficiency Disorders, Metabolic Disorders, Bone Marrow Failure Syndrome), End-User (Hospitals, Research Institutes, Specialty Clinics), Region, Global Industry Analysis, Market Size, Share, Growth, Trends, and Forecast 2020 to 2027.

Theglobal cord blood banking marketis expected to grow from USD 16.88 billion in 2019 to USD 25.90 billion by 2027, at a CAGR of 5.5% during the forecast period 2020-2027. The North America region dominates the market and holds the largest share in the year 2019. This growth is ascertained to the increasing investment in public banking, increasing awareness of long-term benefits, and reduction in storage costs. The Asia-Pacific region is projected to witness significant growth, owing to the increasing awareness regarding the advantages of preservation of cord blood cells in developing economies like China and India.

NOTE: Our analysts monitoring the situation across the globe explains that the market will generate remunerative prospects for producers post COVID-19 crisis. The report aims to provide an additional illustration of the latest scenario, economic slowdown, and COVID-19 impact on the overall industry.

DOWNLOAD FREE SAMPLE REPORT:https://www.fiormarkets.com/report-detail/418891/request-sample

Some of the notable players in the market are CBR Systems, Singapore Cord Blood Bank, Cryo-Save, ViaCord LLC, Global Cord Blood Corporation, Jeevan Stem Cell Foundation, LifeCell, Cordlife, Americord, and FamiCord. In April 2019, FamiCord announced a future business corporation agreement with CelluGen Biotech. This agreement will help in generating beneficial synergies with both the companies in the field of development of new stem cell-based drugs and family-based stem cell banking.

The type of bank segment is classified into public and private. The public segment holds the largest market share, owing to the various funding initiatives, and various government initiatives for encouraging expectant mothers to donate cord blood cells in public banks. The private cord blood banks are expected to witness significant growth, owing to the growing awareness among expectant parents and high demand to safeguard the health of family members. The service segment includes processing and storage. The storage segment holds the largest market share, due to the increasing number of expectant parents utilizing the preservation of newborn cord blood cells. The processing segment is expected to witness significant growth due to various technological advancements in cord blood processing methods. Based on application, the market is classified into cancer, blood disorders, immuno-deficiency disorders, metabolic disorders, and bone marrow failure syndrome. Blood disorders segment holds the largest market share. This growth is attributed to the increasing application of cord blood cells in the treatment of blood disorders and the increasing prevalence of beta-thalassemia. Immuno-deficiency disorders are expected to witness significant growth, owing to the rapid and reliable recovery of immune function, low viral transmission rate, and low risk of graft versus host diseases. The end-user segment is distributed into hospitals, research institutes, and specialty clinics. The research institute segment holds the largest market share, owing to the growth in the number of clinical trials for exploring new diseases and growing pervasiveness of genetic disorders. Hospitals segment is expected to witness significant growth, owing to the advancement in cord blood banking, increasing prevalence of genetic diseases.

The factors influencing the market growth are rising awareness about therapeutic applications of cord blood, government initiatives and funding in developed economies, increasing prevalence of life-threatening genetic diseases, and growing clinical trials in regenerative medicines. Lack of uniformity in regulatory policies, large out-of-pocket expenditure, and low acceptance rate in developing countries are the factors hampering the market growth. Increasing research concerns about cord blood application for the treatment of genetic and idiopathic disorders will provide market growth opportunities.

ACCESS FULL REPORT:https://www.fiormarkets.com/report/cord-blood-banking-market-by-type-of-bank-418891.html

About the report:The globalCord Blood Bankingmarket is analyzed on the basis of value (USD Billion), volume (K Units), export (K Units), and import (K Units). All the segments have been analyzed on global, regional and country basis. The study includes an analysis of more than 30 countries for each segment. The report offers in-depth analysis of driving factors, opportunities, restraints, and challenges for gaining the key insight of the market. The study includes porters five forces model, attractiveness analysis, raw material analysis, and competitor position grid analysis.

Customization of the Report:The report can be customized as per client requirements. For further queries, you can contact us onsales@fiormarkets.comor +1-201-465-4211. Our executives will be pleased to understand your requirements and offer you the best-suited reports.

Contact UsMark StonePhone:+1-201-465-4211Email:sales@fiormarkets.comWeb:www.fiormarkets.com

This content has been distributed via CDN Newswire press release distribution service. For press release enquires please mail us at contact@cdnnewswire.com.

Follow this link:
Global Cord Blood Banking Market is Anticipate to Reach at a CAGR of 5.5% during the forecast period - PharmiWeb.com

Transparency Market Research (TMR)has published a new report titled, Alpha mannosidosis Market Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 20192027. According to the report, the globalalpha mannosidosis marketwas valued atUS$ 7.6 Mnin2018and is projected to expand at a CAGR of9.9%from2019to2027.

Request Brochure for Report https://www.transparencymarketresearch.com/sample/sample.php?flag=B&rep_id=2501

Overview

Patent-cliff To Drive Rare Disease Market to Drive Market

Request for Analysis of COVID19 Impact on Alpha mannosidosis Market

https://www.transparencymarketresearch.com/sample/sample.php?flag=covid19&rep_id=2501

Bone Marrow Transplant Segment to Dominate Market

Hospitals End-user segment to be Highly Lucrative Segment

Buy Alpha mannosidosis Market Report https://www.transparencymarketresearch.com/checkout.php?rep_id=2501&ltype=S

Competitive Landscape

About Us

Transparency Market Research is a global market intelligence company providing global business information reports and services. Our exclusive blend of quantitative forecasting and trends analysis provides forward-looking insight for several decision makers. Our experienced team of analysts, researchers, and consultants use proprietary data sources and various tools and techniques to gather and analyze information.

Our data repository is continuously updated and revised by a team of research experts so that it always reflects latest trends and information. With a broad research and analysis capability, Transparency Market Research employs rigorous primary and secondary research techniques in developing distinctive data sets and research material for business reports.

Contact

Transparency Market Research,

90 State Street, Suite 700,

Albany, NY 12207

Tel: +1-518-618-1030

USA Canada Toll Free: 866-552-3453

Website: https://www.transparencymarketresearch.com/

View post:
Alpha mannosidosis Market projected to expand at a CAGR of 9.9% from 2019 to 2027 - The Daily Chronicle

Provided by the Pennsylvania Department of Health:

Secretary of Health Dr. Rachel Levine today reminded Pennsylvanians of the seriousness of sickle cell disease and the importance of getting tested for it. Sickle cell disease is the most common inherited blood disease.

We want people to get tested for sickle cell disease if they believe they could be a carrier of it, Levine said. We inherit traits from our parents like eye and hair color, but they also pass along internal traits like blood type and sickle cell conditions. It is important to be tested to confirm if you have sickle cell disease, so that treatment for the disease can be started right away to further protect yourself and your family.

Sickle cell disease is an inherited blood disease where an individuals red blood cells take a crescent or sickle shape. This change in shape can create blockages that prevent blood from reaching parts of the body. As a result, people with sickle cell complications can experience anemia, gallstones, stroke, chronic pain, organ damage and even premature death.

According to the Centers for Disease Control and Prevention (CDC), sickle cell disease affects approximately 100,000 Americans. This disease has a greater influence on African American and Hispanic populations but is also found among many other races and ethnicities.

Sickle cell disease is one of the 10 mandatory diseasesscreened for newborns. These screenings are conducted with the goal of eliminating or reducing death, disease and disability in newborn children. In addition, sickle cell disease can be diagnosed before birth to provide an early diagnosis and find treatment.

Treatment can help those with sickle cell disease live well and be healthy, but there is ultimately no cure for sickle cell disease. Treatment requires:

Finding good medical care and getting regular checkups;

Staying up to date on vaccinations and washing hands frequently to prevent infections;

Learning healthy habits;

Looking into clinical studies; and

Finding support and assistance.

Studies have shown that donated bone marrow or stem cell transplants have helped cure sickle cell disease in children with severe cases of the disease. This means that the healthy donated bone marrow or stem cell transplant replaces an individuals bone marrow that is not working properly. Bone marrow or stem cell transplants can be risky and for the donation to work the individual would need to be a close match like a brother or sister.

The Wolf administration has developed aprescribing guideline for the treatment of acute and chronic pain in patients with sickle cell diseaseto assist physicians treating patients with the disease. The guideline provides best practices to treat acute painful crises that occur with sickle cell disease patients as well as best practices for chronic pain care. The sickle cell disease guideline addresses the specific needs of that patient population. This can help prevent the misapplication of recommendations to populations that are outside the scope of other prescribing guidelines, including patients experiencing acute sickle cell crises. It is especially important to have resources specifically for the treatment of sickle cell disease patients as this patient population often experiences racial disparities and stigma.

Report a correction via email | Editorial standards and policies

View original post here:
PA Health Secretary: Sickle Cell Disease Treatment Hinges On Getting Testing - LevittownNow.com

DetailsCategory: DNA RNA and CellsPublished on Tuesday, 22 September 2020 10:38Hits: 148

The comprehensive cancer center has also dosed its first patient in chlorotoxin CAR T cell therapy trial

DUARTE, CA, USA I September 21, 2020 I City of Hope, a world-renowned independent research and treatment center for cancer, diabetes and other life-threatening diseases, today announced that it has licensed intellectual property relating to its pioneering chlorotoxin chimeric antigen receptor (CLTX-CAR) T cell therapy to Chimeric Therapeutics Limited, an Australian biotechnology company.

The therapy is currently being used in a phase 1 clinical trial at City of Hope to treat glioblastoma (GBM), a type of brain tumor. The first patient in the trial was recently dosed; Behnam Badie, M.D., chief of City of Hopes Division of Neurosurgery and The Heritage Provider Network Professor in Gene Therapy, is leading this innovative, first-of-its-kind trial.

Chimeric has acquired the exclusive worldwide rights to develop and commercialize certain patents relating to City of Hopes CLTX-CAR T cells, as well as to further develop the therapy for other cancers.

City of Hope is excited to enter into this agreement with Chimeric as it supports our innovative research in CAR T cell therapy and our commitment to extend these therapies to more patients, particularly those with GBM and other solid tumors that are difficult to treat, said Christine Brown, Ph.D., The Heritage Provider Network Professor in Immunotherapy and deputy director of City of Hopes T Cell Therapeutics Research Laboratory. Chimeric shares our goal of providing effective CAR T cell therapies to more patients with current unmet medical needs.

Led by Brown and Michael Barish, Ph.D., chair of City of Hopes Department of Developmental and Stem Cell Biology, and Dongrui Wang, Ph.D., a recent graduate of City of Hopes Irell & Manella Graduate School of Biological Sciences, the team developed and tested the first CAR T cell therapy using CLTX, a component of scorpion venom, to direct T cells to target brain tumor cells. The research was published this past March in Science Translational Medicine.

Chimeric is excited to join City of Hope in its quest to find more effective cancer therapies. This is an exceedingly rare opportunity to acquire a promising technology in one of the most exciting areas of immuno-oncology today, said Paul Hopper, executive chairman of Chimeric. Furthermore, the CLTX-CAR T cell therapy has completed years of preclinical research and development, and recently enrolled its first patient in a phase 1 clinical trial for brain cancer.

CARs commonly incorporate a monoclonal antibody sequence in their targeting domain, enabling CAR T cells to recognize antigens and kill tumor cells. In contrast, the CLTX-CAR uses a synthetic 36-amino acid peptide sequence first isolated from death stalker scorpion venom and now engineered to serve as the CAR recognition domain.

In this recent study, City of Hope researchers used tumor cells in resection samples from a cohort of patients with GBM to compare CLTX binding with expression of antigens currently under investigation as CAR T cell targets. They found that CLTX bound to a greater proportion of patient tumors, and cells within these tumors.

CLTX binding included the GBM stem-like cells thought to seed tumor recurrence. Consistent with these observations, CLTX-CAR T cells recognized and killed broad populations of GBM cells while ignoring nontumor cells in the brain and other organs. The study team demonstrated that CLTX-directed CAR T cells are highly effective at selectively killing human GBM cells without off-tumor targeting and toxicity in cell-based assays and in animal models.

City of Hope, a recognized leader in CAR T cell therapies for GBM and other cancers, has treated more than 500 patients since its CAR T program started in the late 1990s. The institution continues to have one of the most comprehensive CAR T cell clinical research programs in the world it currently has 30 ongoing CAR T cell clinical trials, including CAR T cell trials for HER-2 positive breast cancer that has spread to the brain, and PSCA-positive bone metastatic prostate cancer. It was the first and only cancer center to treat GBM patients with CAR T cells targeting IL13R2, and the first to administer CAR T cell therapy locally in the brain, either by direct injection at the tumor site, through intraventricular infusion into the cerebrospinal fluid, or both. In late 2019, City of Hope opened a first-in-human clinical trial for patients with recurrent GBM, combining IL13R2-CAR T cells with checkpoint inhibitors nivolumab, an anti-PD1 antibody, and ipilimumab, blocking the CTLA-4 protein.

Both an academic medical center and a drug development powerhouse, City of Hope is known for creating the technology used in the development of human synthetic insulin and numerous breakthrough cancer drugs. Its unique research and development hybrid of the academic and commercial creates an infrastructure that enables City of Hope researchers to submit an average of 50 investigational new drug applications to the U.S. Food and Drug Administration each year. The institution currently holds more than 450 patent families.

"City of Hope is delighted to license this technology to Chimeric, said Sangeeta Bardhan Cook, Ph.D., City of Hope director of the Office of Technology Licensing. We are impressed with the ability of their executive team to push and bring therapies to market expeditiously. At City of Hope, our mission is to transform the future of health care. We believe Chimeric has the vision to offer innovative therapies to cancer patients.

About City of Hope

City of Hope is an independent biomedical research and treatment center for cancer, diabetes and other life-threatening diseases. Founded in 1913, City of Hope is a leader in bone marrow transplantation and immunotherapy such as CAR T cell therapy. City of Hopes translational research and personalized treatment protocols advance care throughout the world. Human synthetic insulin and numerous breakthrough cancer drugs are based on technology developed at the institution. A National Cancer Institute-designated comprehensive cancer center and a founding member of the National Comprehensive Cancer Network, City of Hope has been ranked among the nations Best Hospitals in cancer by U.S. News & World Report for 14 consecutive years. Its main campus is located near Los Angeles, with additional locations throughout Southern California. For more information about City of Hope, follow us on Facebook, Twitter, YouTube or Instagram.

SOURCE: City of Hope

Read more:
City of Hope Enters Licensing Agreement With Chimeric to Develop Its Pioneering Chlorotoxin CAR T Cell Therapy | DNA RNA and Cells | News Channels -...

INREBIC provides new, once-daily oral option for patients affectedby rare bone marrow cancer

MONTREAL, Sept. 21, 2020 /CNW/ - Bristol Myers Squibb Canada (BMS) announced today that Health Canada has approved INREBIC (fedratinib), a new once-daily oral medication used to treat adults with an enlarged spleen and associated symptoms caused by intermediate-2 or high-risk primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis.1

INREBICis the first new treatment for patients with myelofibrosis in nearly a decade to demonstrate a clinically meaningful reduction in spleen volume for patients in the approved population affected by this serious and rare bone marrow cancer.1 A new treatment provides Canadians living with myelofibrosis, and their caregivers, with more options to find a treatment that works for them.

Myelofibrosis is a serious and rare bone marrow disorder that disrupts the body's normal production of blood cells.2 Bone marrow is gradually replaced with fibrous scar tissue, which limits the ability of the bone marrow to make blood cells.2 Currently, there are an estimated 1,400 to 2,177 Canadians who are living with myelofibrosis.3

"The approval of INREBIC represents a milestone for the way healthcare practitioners treat this rare disorder, which can have debilitating symptoms," said Dr. Vikas Gupta, Director, The Elizabeth and Tony Comper MPN Program, Princess Margaret Cancer Centre, Toronto. "Canadians living with myelofibrosis now have a new treatment option that may be better suited to their needs and has shown promise for alleviating the symptom burden associated with myelofibrosis."

INREBIC is a janus kinase (JAK) inhibitor and is the first new treatment for patients with myelofibrosis in nearly a decade.1,4 JAK proteins send signals that tell the body to make more blood cells, but myelofibrosis makes it difficult for the bone marrow to create normal blood cells, which potentially moves blood cell production to the spleen.1 However, by blocking the activity of JAK proteins, INREBIC can reduce the size of the spleen and improve symptoms.

Story continues

"As part of our commitment to Canadians living with cancer, we are excited to provide INREBIC as a new treatment option for those impacted by myelofibrosis," said Al Reba, General Manager, Bristol Myers Squibb Canada. "We hope that the option of a once-daily oral treatment will have a positive and meaningful impact on Canadians living with the disease."

Health Canada's approval of INREBIC included findings from the JAKARTA and JAKARTA2 clinical trials. The JAKARTA study, a double-blind, randomized, placebo-controlled Phase 3 study, involved patients with intermediate-2 or high-risk myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis. The JAKARTA2 study, a multicenter, open-label, single-arm Phase 2 study,involved patients previously exposed to ruxolitinib with a diagnosis of intermediate-1 with symptoms, intermediate-2 or high-risk primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis.

About MyelofibrosisMyelofibrosis is classified as a myeloproliferative neoplasm, a group of rare blood cancers that are derived from blood-forming stem cells.2Myelofibrosis can lead to anemia and thrombocytopenia, weakness, fatigue and enlargement of the spleen and liver, among other symptoms.2 In Canada, approximately 36 to 360 people will be diagnosed with myelofibrosis each year.3 Both men and women are affected, and while the disease can affect people of all ages, the median age at diagnosis is 69 years old.3

About JAKARTAJAKARTA was a double-blind, randomized, placebo-controlled Phase 3 study in patients with intermediate-2 or high-risk myelofibrosis (MF), post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis with splenomegaly and platelet count 50 x 109/L. A total of 289 patients were randomized to receive either INREBIC 500 mg (N=97), 400 mg (N=96), or placebo (N=96) once daily for at least 6 cycles. The median age was 65 years (range 27 to 86 years), 47% of patients were older than 65 years, and 59% were male. Sixty-four percent (64%) of patients had primary MF, 26% had post-polycythemia vera MF, and 10% had post-essential thrombocythemia MF. Fifty-two percent (52%) of patients had intermediate-2 risk, and 48% had high-risk disease. The median hemoglobin count at baseline was 10.2 g/dL. The median platelet count at baseline was 213.5 x 109/L; 16.3% of patients had a platelet count <100 x 109/L, and 83.7% of patients had a platelet count 100 x 109/L. Patients had a median palpable spleen length of 15 cm at baseline and a median spleen volume as measured by magnetic resonance imaging (MRI) or computed tomography (CT) of 2568 mL (range of 316 to 8244 mL) at baseline. (The median normal spleen volume is approximately 215 mL).1

The primary efficacy endpoint was the proportion of patients achieving a greater than or equal to 35% reduction from baseline in spleen volume at the End of Cycle 6 as measured by MRI or CT and confirmed 4 weeks later.1

One of the secondary endpoints was the proportion of patients with a 50% or greater reduction in Total Symptom Score (TSS) from baseline to the End of Cycle 6 as measured by the modified Myelofibrosis Symptoms Assessment Form (MFSAF) v2.0 diary.1

About JAKARTA2JAKARTA2 was a multicenter, open-label, single-arm Phase 2 study in patients previously exposed to ruxolitinib with a diagnosis of intermediate-1 with symptoms, intermediate-2 or high-risk myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis with splenomegaly and platelet count 50 x 109/L.1

A total of 97 patients were enrolled and treated with INREBIC400 mg once daily. The median age was 67 years (range 38 to 83 years) with 58% of patients older than 65 years and 55% were male. Fifty-five percent (55%) of patients had primary MF, 26% had post-polycythemia vera MF, and 19% had post-essential thrombocythemia MF. Sixteen percent (16%) of patients had intermediate-1 with symptoms, 49% had intermediate-2, and 35% had high-risk disease. The median hemoglobin count was 9.8 g/dL at baseline. The median platelet count was 147.0 x 109/L at baseline; 34.0% of patients had a platelet count <100 x 109/L, and 66.0% of patients had a platelet count 100 x 109/L. Patients had a median palpable spleen length of 18 cm at baseline and a median spleen volume as measured by magnetic resonance imaging (MRI) or computed tomography (CT) of 2893.5 mL (range of 737 to 7815 mL) at baseline.1

The median duration of prior exposure to ruxolitinib was 10.7 months (range 0.1 to 62.4 months). Seventy-one percent (71%) of patients had received doses of either 30 mg or 40 mg daily of ruxolitinib prior to study entry.1

The primary endpoint was the subject response rate, defined as the proportion of subjects who have a 35% reduction in volume of spleen size at the end of Cycle 6.1

One of the secondary endpoints was the proportion of patients with a 50% or greater reduction in Total Symptom Score (TSS) from baseline to the End of Cycle 6 as measured by the modified Myelofibrosis Symptoms Assessment Form (MFSAF) diary.1

About INREBICINREBIC (fedratinib) is indicated for the treatment of splenomegaly and/or disease related symptoms in adult patients with intermediate-2 or high-risk primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis, including patients who have been previously exposed to ruxolitinib.

About Bristol Myers Squibb Canada

Bristol Myers Squibb Canada Co. is an indirect wholly-owned subsidiary of Bristol Myers Squibb Company, a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb global operations, visitwww.bms.com. Bristol Myers Squibb Canada Co. delivers innovative medicines for serious diseases to Canadian patients in the areas of cardiovascular health, oncology, and immunoscience. Bristol Myers Squibb Canada Co. employs more than 300 people across the country. For more information, please visitwww.bmscanada.ca.

About Bristol Myers Squibb Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us atBMS.comor follow us on LinkedIn, Twitter, YouTube, Facebookand Instagram.

Celgene and Juno Therapeutics are wholly owned subsidiaries of Bristol Myers Squibb Company. In certain countries outside the U.S., due to local laws, Celgene and Juno Therapeutics are referred to as, Celgene, a Bristol Myers Squibb company and Juno Therapeutics, a Bristol Myers Squibb company.

References:

INREBICCanada Product Monograph. July 10, 2010.

Leukemia & Lymphoma Society of Canada. Myelofibrosis. Available at: https://www.llscanada.org/myeloproliferative-neoplasms/myelofibrosis. Accessed July 28, 2020.

Corinne S. Hodgson & Associates. Blood Cancer in Canada Facts & Stats 2016. Leukemia & Lymphoma Society of Canada 2016; 4-8.

Canadian MPN Group. Myelofibrosis. Available at: http://www.mpncanada.com/about-mpns/practitioner-reference/myelofibrosis/#treatment-options. Accessed July 28, 2020.

SOURCE Bristol Myers Squibb Canada Co.

View original content to download multimedia: http://www.newswire.ca/en/releases/archive/September2020/21/c8189.html

Read the original here:
Health Canada Approves INREBIC (fedratinib), First New Treatment in Nearly a Decade for Patients Living with Myelofibrosis - Yahoo Finance

Since late June, life has changed for Rylee Schroeder and her parents, Megan and Levi Schroeder.

It was then the Neosho High School senior sought help for extreme fatigue, shortness of breath, nosebleeds and more. The symptoms, combined with a previously diagnosed issue with low platelets, led Rylees pediatrician to run a series of lab tests.

The news sent the family first to Freeman Hospital West in Joplin, and then Childrens Mercy Hospital in Kansas City.

Doctors discovered Rylees low hemoglobin, platelets and white blood counts were caused by myelodysplastic syndrome. Additional testing revealed the teen has an extremely rare congenital disorder called Shwachman-Diamond syndrome and a TP53 gene mutation that led to the MDS.

Megan said her daughter is only the 36th patient known to have this combination of illnesses, which have progressed to bone marrow failure.

On Friday, Rylee received a stem cell bone marrow transplant at Childrens Mercy in Kansas City, thanks to a donation from her 12-year-old brother Colin.

Megan said her son was a 12/12 match for Rylee. Typically, doctors look for a 10/10 match, or a 5/5 match with a parent. The Be a Match Donor Registry located four 10/10 matches, two donors in the United States and two international donors.

If everything goes well with the transplant, doctors hope to see Colins stem cells begin to take hold in Rylees body between Day 14 to Day 21, post transplant.

Relying on faith

Through everything, Rylee and her family are relying on their faith, as well as support from friends at Racine Christian Church and within the community of Neosho, to get them through the tough days.

We are lucky to have a great church and thankful Sunday serves are online on Facebook, Megan said. We may not be there, but we still feel a part of it, and they are always ready to lift us up.

Many times, Ive thought Im not strong enough to do this, but Im reminded that I am. We are so tired and weary but (God) has us.

Megan said shes used to being the one to offer help through giving and volunteering. Now their family is on the other side, leaning on the support of others.

Our faith means everything to us, Megan said. Without it, we wouldnt be doing as well as we are. Its hard enough as it is. If you dont have something to believe in with your whole heart to guide you and lean on, you would just be lost. It would be overwhelming to take it on yourself.

More about Rylee

Rylee, who turned 17 shortly after her diagnosis, is active in the NHS show choir, choir, Key Club and FCA.

A member of the Racine Christian Church youth leadership team, she was also slated to go to Ireland this summer pre-pandemic with the churchs mission team.

This past Thursday, Rylee was highlighted at the NHS volleyball game for childhood cancer awareness month. On Friday, she was recognized at the home football game.

Members of her show choir helped start a Go Fund Me account earlier this summer to help the family with travel expenses to and from Kansas City. Her youth minister has since created another one to help with the coming months.

Megan said the family always helps at Solomons Dance Studio recitals because the owner, Charity, uses the lessons to teach children to love dance and to love others as well as themselves.

Rylee was able to to attend the recital to watch her sister Erin dance. On the last day of the recital, Charity asked the entire family to come forward, offering prayers over the entire situation.

Rylees small group from the church, which meets on Wednesday evenings, livechats with her, allowing the teen to continue to be part of the discussions.

Before Rylee was admitted to begin the chemo needed prior to the transplant, she asked to rededicate her life to Christ. Megan said she wanted to feel his strength renewed as she went into the fight.

Our close friends all stayed behind after church that Sunday, even though it was last minute, to be witness to her baptism, Megan said. They are some of our strongest supporters. We would be lost without them.

Megan said those friends, steeped in faith, give her strength to sit at the hospital, holding Rylees hand.

I can reach out to any of them to start praying with me to help make me feel stronger when I feel I am failing her, Megan said. I know Gods got this. He is strong all the time even when we can not be strong.

Biblical baubles

Many of Rylee Schroeders friends are wearing a Rally behind Rylee bracelet featuring Isaiah 41:10: So do not fear, for I am with you; do not be dismayed, for I am your God. I will strengthen you and help you; I will uphold you with my righteous right hand.

A family friend made them to distribute to members of the community. A limited number remain. Information about the bracelets, and Rylees journey, may be found on a Facebook page, Rally Behind Rylee.

More:
Faith for the fight: Neosho teen deals with rare combination of illnesses - Joplin Globe

INREBIC provides new, once-daily oral option for patients affected by rare bone marrow cancer

MONTREAL, Sept. 21, 2020 /CNW/ - Bristol Myers Squibb Canada (BMS) announced today that Health Canada has approved INREBIC (fedratinib), a new once-daily oral medication used to treat adults with an enlarged spleen and associated symptoms caused by intermediate-2 or high-risk primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis.1

INREBIC is the first new treatment for patients with myelofibrosis in nearly a decade to demonstrate a clinically meaningful reduction in spleen volume for patients in the approved population affected by this serious and rare bone marrow cancer.1 A new treatment provides Canadians living with myelofibrosis, and their caregivers, with more options to find a treatment that works for them.

Myelofibrosis is a serious and rare bone marrow disorder that disrupts the body's normal production of blood cells.2 Bone marrow is gradually replaced with fibrous scar tissue, which limits the ability of the bone marrow to make blood cells.2 Currently, there are an estimated 1,400 to 2,177 Canadians who are living with myelofibrosis.3

"The approval of INREBIC represents a milestone for the way healthcare practitioners treat this rare disorder, which can have debilitating symptoms," said Dr. Vikas Gupta, Director, The Elizabeth and Tony Comper MPN Program, Princess Margaret Cancer Centre, Toronto. "Canadians living with myelofibrosis now have a new treatment option that may be better suited to their needs and has shown promise for alleviating the symptom burden associated with myelofibrosis."

INREBIC is a janus kinase (JAK) inhibitor and is the first new treatment for patients with myelofibrosis in nearly a decade.1,4 JAK proteins send signals that tell the body to make more blood cells, but myelofibrosis makes it difficult for the bone marrow to create normal blood cells, which potentially moves blood cell production to the spleen.1 However, by blocking the activity of JAK proteins, INREBIC can reduce the size of the spleen and improve symptoms.

"As part of our commitment to Canadians living with cancer, we are excited to provide INREBIC as a new treatment option for those impacted by myelofibrosis," said Al Reba, General Manager, Bristol Myers Squibb Canada. "We hope that the option of a once-daily oral treatment will have a positive and meaningful impact on Canadians living with the disease."

Health Canada's approval of INREBIC included findings from the JAKARTA and JAKARTA2 clinical trials. The JAKARTA study, a double-blind, randomized, placebo-controlled Phase 3 study, involved patients with intermediate-2 or high-risk myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis. The JAKARTA2 study, a multicenter, open-label, single-arm Phase 2 study, involved patients previously exposed to ruxolitinib with a diagnosis of intermediate-1 with symptoms, intermediate-2 or high-risk primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis.

About MyelofibrosisMyelofibrosis is classified as a myeloproliferative neoplasm, a group of rare blood cancers that are derived from blood-forming stem cells.2 Myelofibrosis can lead to anemia and thrombocytopenia, weakness, fatigue and enlargement of the spleen and liver, among other symptoms.2 In Canada, approximately 36 to 360 people will be diagnosed with myelofibrosis each year.3 Both men and women are affected, and while the disease can affect people of all ages, the median age at diagnosis is 69 years old.3

About JAKARTAJAKARTA was a double-blind, randomized, placebo-controlled Phase 3 study in patients with intermediate-2 or high-risk myelofibrosis (MF), post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis with splenomegaly and platelet count 50 x 109/L. A total of 289 patients were randomized to receive either INREBIC 500 mg (N=97), 400 mg (N=96), or placebo (N=96) once daily for at least 6 cycles. The median age was 65 years (range 27 to 86 years), 47% of patients were older than 65 years, and 59% were male. Sixty-four percent (64%) of patients had primary MF, 26% had post-polycythemia vera MF, and 10% had post-essential thrombocythemia MF. Fifty-two percent (52%) of patients had intermediate-2 risk, and 48% had high-risk disease. The median hemoglobin count at baseline was 10.2 g/dL. The median platelet count at baseline was 213.5 x 109/L; 16.3% of patients had a platelet count <100 x 109/L, and 83.7% of patients had a platelet count 100 x 109/L. Patients had a median palpable spleen length of 15 cm at baseline and a median spleen volume as measured by magnetic resonance imaging (MRI) or computed tomography (CT) of 2568 mL (range of 316 to 8244 mL) at baseline. (The median normal spleen volume is approximately 215 mL).1

The primary efficacy endpoint was the proportion of patients achieving a greater than or equal to 35% reduction from baseline in spleen volume at the End of Cycle 6 as measured by MRI or CT and confirmed 4 weeks later.1

One of the secondary endpoints was the proportion of patients with a 50% or greater reduction in Total Symptom Score (TSS) from baseline to the End of Cycle 6 as measured by the modified Myelofibrosis Symptoms Assessment Form (MFSAF) v2.0 diary.1

About JAKARTA2JAKARTA2 was a multicenter, open-label, single-arm Phase 2 study in patients previously exposed to ruxolitinib with a diagnosis of intermediate-1 with symptoms, intermediate-2 or high-risk myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis with splenomegaly and platelet count 50 x 109/L.1

A total of 97 patients were enrolled and treated with INREBIC 400 mg once daily. The median age was 67 years (range 38 to 83 years) with 58% of patients older than 65 years and 55% were male. Fifty-five percent (55%) of patients had primary MF, 26% had post-polycythemia vera MF, and 19% had post-essential thrombocythemia MF. Sixteen percent (16%) of patients had intermediate-1 with symptoms, 49% had intermediate-2, and 35% had high-risk disease. The median hemoglobin count was 9.8 g/dL at baseline. The median platelet count was 147.0 x 109/L at baseline; 34.0% of patients had a platelet count <100 x 109/L, and 66.0% of patients had a platelet count 100 x 109/L. Patients had a median palpable spleen length of 18 cm at baseline and a median spleen volume as measured by magnetic resonance imaging (MRI) or computed tomography (CT) of 2893.5 mL (range of 737 to 7815 mL) at baseline.1

The median duration of prior exposure to ruxolitinib was 10.7 months (range 0.1 to 62.4 months). Seventy-one percent (71%) of patients had received doses of either 30 mg or 40 mg daily of ruxolitinib prior to study entry.1

The primary endpoint was the subject response rate, defined as the proportion of subjects who have a 35% reduction in volume of spleen size at the end of Cycle 6.1

One of the secondary endpoints was the proportion of patients with a 50% or greater reduction in Total Symptom Score (TSS) from baseline to the End of Cycle 6 as measured by the modified Myelofibrosis Symptoms Assessment Form (MFSAF) diary.1

About INREBICINREBIC (fedratinib) is indicated for the treatment of splenomegaly and/or disease related symptoms in adult patients with intermediate-2 or high-risk primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis, including patients who have been previously exposed to ruxolitinib.

About Bristol Myers Squibb Canada

Bristol Myers Squibb Canada Co. is an indirect wholly-owned subsidiary of Bristol Myers Squibb Company, a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb global operations, visit http://www.bms.com. Bristol Myers Squibb Canada Co. delivers innovative medicines for serious diseases to Canadian patients in the areas of cardiovascular health, oncology, and immunoscience. Bristol Myers Squibb Canada Co. employs more than 300 people across the country. For more information, please visit http://www.bmscanada.ca.

About Bristol Myers Squibb Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.

Celgene and Juno Therapeutics are wholly owned subsidiaries of Bristol Myers Squibb Company. In certain countries outside the U.S., due to local laws, Celgene and Juno Therapeutics are referred to as, Celgene, a Bristol Myers Squibb company and Juno Therapeutics, a Bristol Myers Squibb company.

References:

SOURCE Bristol Myers Squibb Canada Co.

More:
Health Canada Approves INREBIC (fedratinib), First New Treatment in Nearly a Decade for Patients Living with Myelofibrosis - BioSpace

Immunotherapies that are currently being studied in multiple myeloma are discussed in this section under four headings: checkpoint inhibitors, chimeric antigen receptor (CAR) T cells, bispecific antibodies, and antibody drug conjugates (fig 2).

Malignant plasma cells in most patients with multiple myeloma express the checkpoint programmed death-ligand 1, which is upregulated especially when exposed to inflammatory mediators such as interferon . Interaction of this checkpoint molecule with programmed cell death protein 1 on T cells limits their proliferation and cytotoxic activity.6869

The first study evaluating single agent nivolumab for relapsed multiple myeloma showed a response in only one of 27 patients.7071 Despite a lack of single agent activity, single arm trials combining checkpoint inhibitors with immunomodulatory imide drugs and dexamethasone because of the potential synergy72 look promising.7374

With these clinical data, three large randomized phase III trials were halted by the FDA in 2017 because of increased serious adverse events and deaths as well as decreased overall survival in the checkpoint inhibitor arm (pomalidomide and dexamethasone with and without pembrolizumab) in relapsed and refractory multiple myeloma (hazard ratio 1.61, 95% confidence interval 0.91 to 2.85),75 pomalidomide and dexamethasone with and without nivolumab in relapsed and refractory multiple myeloma (1.19, 0.64 to 2.20),76 and lenalidomide and dexamethasone with and without pembrolizumab in transplant ineligible patients with newly diagnosed multiple myeloma (2.06, 0.93 to 4.55)77).

These trials encourage caution with expedited timelines for future combination studies for drugs with limited single agent activity.7879 Future trials in multiple myeloma will need to be based on sound preclinical and clinical rationale with other partners and be conducted in heavily treated patients (with limited standard options) initially.

CAR T cells are human T cells that have been genetically modified and expanded in the laboratory before they are infused back into patients to target the tumor. The receptor on the surface of CAR T cells that targets the tumor antigens consists of several parts (fig 3): an extracellular, non-major histocompatibility complex restricted, targeting domain, usually derived from a single chain variable fragment of a monoclonal antibody; a spacer region; a transmembrane domain; an intracellular signaling domain including the CD3 activation domain; and a costimulatory domain (eg, CD28 or 4-1BB). The single chain variable fragment was originally derived from mice (hence the term chimeric), but many of the newer constructs are fully human.8081

Chimeric antigen receptor (CAR) T cell structure

CD3 positive T cells are obtained from patients (for autologous CAR T cells) or healthy donors (for allogeneic CAR T cells) via a process called leukapheresis. These T cells are expanded manifold in culture and activated using beads coated with anti-CD3 or anti-CD28 monoclonal antibodies or cell based artificial antigen presenting cells.82 The T cells are then transduced with a vector (usually either lentiviral or retroviral) that carries the gene encoding a receptor to an antigen present on the surface of tumor cells. This manufacturing process takes up to four weeks at a good manufacturing practices facility, and the CAR T cells can then be stored until needed by the patient. This delay means that the disease must not be rapidly progressing, so that the patient is able to wait until the CAR T product is ready; otherwise the patient will need bridging chemotherapy. Two to seven days before CAR T cell infusion, a patient receives lymphodepleting chemotherapy to make way for the CAR T cells that are subsequently given as an intravenous infusion. Once infused into patients, the CAR T cells encounter the antigen, proliferate, and kill the tumor cells (fig 4). These cells, therefore, combine the target specificity of a monoclonal antibody with the enhanced cytotoxicity of T cells without requiring human leucocyte antigen presentation of the target antigen.83

Chimeric antigen receptor (CAR) T cell treatment for multiple myelomasequence of events. CRS=cytokine release syndrome; ICANS=immune effector cell associated neurotoxicity syndrome

An ideal antigen is one that is widely and exclusively expressed on cancer cells but not on normal cells to enhance efficacy and reduce toxicity.8485 In multiple myeloma, most emerging immunotherapies (including CAR T cells) target B cell maturation antigen (BCMA), a type III transmembrane receptor, which is a promising target antigen.8687 BCMA is also known as tumor necrosis factor receptor superfamily member 17 or CD269. It is expressed in nearly all plasma cells (normal and malignant) although its expression is variable.88 BCMA promotes plasma cell survival and is induced during plasma cell differentiation89 by binding to ligands (a proliferation inducing ligand (APRIL) and B cell activating factor (BAFF)) that are produced by osteoclasts.90 Increased levels of soluble BCMA are associated with high tumor burden in multiple myeloma and thus worse outcomes.91

CAR T cells targeting CD19 were approved by the FDA in 2017 for refractory large B cell lymphoma9293 and acute lymphoblastic leukemia,94 and are being used in clinical practice. The first study on CAR T cell treatment directed by BCMA opened in 2014 at the US National Cancer Institute.879596 Since then, about a dozen of different early phase clinical trials have been conducted on BCMA CAR T treatment for advanced multiple myeloma.9798 A detailed review outlining the differences in the construct, manufacturing, and clinical efficacy of these different products has been published previously.8186

This review focuses on four BCMA CAR T cell products that are currently being evaluated in registration (that is, for regulatory approval) phase I/II clinical trials for patients with relapsed and refractory multiple myeloma. These products include bb2121 (now known as idecabtagene vicleucel or ide-cel), JCARH125 (now known as orvacabtagene autoleucel or orva-cel), LCAR-B38M (now known as JNJ-4528), and P-Bcma-101. FDA approvals for some of these agents are anticipated in 2020-21 for relapsed and refractory multiple myeloma (fig 5; table 2). The high overall response rates of 60-100% seen in these trials in a highly refractory population is unprecedented, although the durability of these responses is still in question.

Four major constructs of chimeric antigen receptor (CAR) T cells targeting B cell maturation antigens (BCMA), currently in multicenter clinical trials investigating multiple myeloma. This figure does not include all BCMA constructs in multiple myeloma. ScFv=single chain variable fragment; VH only=variable-heavy chain only fragments

Summary of major multicenter clinical trials investigating multiple myeloma treatments*

The most advanced CAR T cell treatment targeting a BCMA is ide-cel (bb2121), which uses a lentiviral vector for CAR insertion and includes a 4-1BB costimulatory domain as well as a murine single chain variable fragment.114 In a phase I non-randomized, open label, multicenter trial in relapsed and refractory multiple myeloma (3 prior lines of treatment) for 33 patients treated at various doses,115 researchers found an overall response rate of 85% with a median progression free survival of 11.8 months. A higher overall response rate was seen at the higher dose levels and doses of 150-450106 CAR T cells were defined as the active dose.115 This dose is being tested currently in a multicenter, single arm, open label trial to evaluate bb2121 CAR T cells further in relapsed and refractory multiple myeloma; the trial has completed enrolment of 149 patients worldwide. Preliminary results show an overall response rate of 73% (complete response rate 33%) and median progression free survival of 8.8 months in 128 patients treated at doses of 150-450106 cells (table 2).99 Fifty four patients treated at the highest dose level of 450106 cells had an overall response rate of 82% and a median progression free survival of 12.1 months.99 These results have been submitted to regulatory agencies including the FDA and European Medicines Agency for treatment for advanced multiple myeloma.

Orva-cel (JCARH125) is another second generation CAR product with a fully human B cell derived single chain variable fragment, a 4-1BB costimulatory domain, and optimized manufacturing (predefined CD4:CD8 ratio) that is derived from preclinical work at Memorial Sloan Kettering Cancer Center. The preliminary data for the multicenter phase I/II EVOLVE study were presented at the American Society of Clinical Oncology meeting in 2020. These patients had received a median of six prior treatments. They received escalating doses of 50-600106 cells. The results for 62 patients treated at the 300-600106 cells dose range showed an overall response rate of 92% (complete response rate 36%).100101 The trial is currently enrolling at the recommended phase II dose of 600106 cells (table 2).

The LCAR-B38M CAR construct was developed initially in China and is currently being pursued in the US and globally as JNJ-4528 (table 2). It consists of two llama derived variable-heavy chain only fragments that target two epitopes of BCMA designed to confer avidity. In a phase I/II study in China, researchers found deep durable responses with a median progression free survival of 19.9 months and a manageable safety profile in relapsed and refractory multiple myeloma, although the patients in this study were treated earlier in their disease course with a median of three prior lines of treatment and were therefore less heavily pre-treated.102103104105 In the US and Europe, a multicenter phase Ib/II clinical trial of this CAR construct as JNJ-4528 in relapsed and refractory multiple myeloma (3 prior lines of treatment) was conducted to confirm the findings of the LEGEND-2 study. Preliminary results of the phase Ib portion showed an overall response rate of 100% (complete response rate 86%) in patients with a median of five prior lines of treatment (table 2).106107 The phase II portion is fully enrolled, and phase II and III studies have been initiated.

P-BCMA-101 is uniquely manufactured using the non-viral piggyBac gene editing system, which is less costly, produces cells with a high percentage of favorable stem cell memory phenotype T cells, and has the ability to include a safety switch. The binding molecule for this product is not a single chain variable fragment but a small fully human fibronectin domain (Centyrin) that has higher specificity and potentially less immunogenicity. In a phase I dose escalation trial, the overall response rate was 63% with a median progression free survival of 9.5 months in 19 evaluable patients108 (table 2).

CAR T cell treatments have a unique toxicity profile where patients can develop side effects such as cytokine release syndrome and neurotoxicity that has been recently termed immune effector cell associated neurotoxicity syndrome (ICANS).116 Cytokine release syndrome has been defined as a disorder characterized by fever, tachypnea, headache, tachycardia, hypotension, rash, or hypoxia caused by the release of cytokines from cells. The American Society for Transplantation and Cellular Therapy has developed a consensus grading system for cytokine release syndrome, which depends on the severity and presence of fever, hypotension, or hypoxia (table 3).116

American Society for Transplantation and Cellular Therapy consensus grading for cytokine release syndrome (CRS)116

ICANS has been defined as a disorder involving the central nervous system following any immunotherapy that results in the activation or engagement of endogenous or infused T cells or other immune effector cells. Symptoms or signs can be progressive and could include aphasia, altered level of consciousness, impairment of cognitive skills, motor weakness, seizures, and cerebral edema.116 It includes four grades that are determined by the ICE score (immune effector cell associated encephalopathy score, which provides objectivity to grading encephalopathy), level of consciousness, seizure, motor findings, and elevated intracranial pressure or cerebral edema (table 4).116 Management of ICANS and cytokine release syndrome is based on grading and involves supportive care, steroids, and interleukin blocking agents.117118 Interleukin 6 blocking agents (tocilizumab and siltuximab) with or without steroids are the mainstay of management for cytokine release syndrome, whereas steroids are the mainstay for the management of neurotoxicity. Another potential agent for managing these symptoms includes the interleukin 1 blocking agent anakinra.119

American Society for Transplantation and Cellular Therapy consensus grading for immune effector cell associated neurotoxicity syndrome (ICANS) in adults116

All the clinical trials on BCMA CAR T cell treatments had a high incidence of cytokine release syndrome (>80%) except for P-BCMA-101, which seemed to have a substantially lower incidence (10%). Despite this, severe cytokine release syndrome (that is, grade 3) is seen in less than 10% of patients. Neurotoxicity was reported in less than 20% of patients with severe neurotoxicity (grade 3) in less than 7% of patients. Another common side effect is cytopenia, which has also been thought to be secondary to the lymphodepleting chemotherapy, ongoing CAR T cell activity, and disruption of hematopoiesis showing severe hypocellularity in the bone marrow, but most patients recover with time.120121

Early recognition of cytokine release syndrome and ICANS and prompt intervention after CAR T cell treatment is vital to prevent serious consequences, although the optimal timing for intervention and benefit of prophylactic treatment is yet unknown.122 The CAR T cell therapy associated toxicity (CARTOX) working group has developed a management approach for these syndromes, based on multidisciplinary grades.123 In cytokine release syndrome, patients with grade 1 are usually managed with supportive care, those with grade 2 are managed with the anti-interleukin 6 receptor tocilizumab with or without steroids in addition to supportive care, and those with grade 3-4 are managed in the intensive care unit with aggressive supportive care, vasopressors, oxygen, tocilizumab, and steroids. Patients with grade 1 and 2 ICANS are managed supportively but an electroencephalogram is done to rule out electrical seizures and imaging of the brain to rule out edema. Patients with grade 3 and 4 ICANS need steroids and more aggressive supportive care.120

Bispecific monoclonal antibodies direct a hosts immune system (more specifically cytotoxic T cells) against cancer cells by binding CD3 on T cells with a target protein on cancer cells (fig 6).124 A type of bispecific antibody is the bispecific T cell engager (BiTE), which differs from other bispecific antibodies by containing two different single chain variable fragments connected by a linker. BiTEs often have a short half life, requiring continuous infusion to maintain efficacy.125 The first BiTE to receive FDA approval for treatment in relapsed and refractory acute lymphoblastic leukemia is Blinatumomab, a bispecific antibody that engages T cells to CD19 positive cells.126 Because BiTEs engage and activate the patients own immune cells, they have a toxicity profile similar to CAR T cells including cytokine release syndrome and ICANS.116

Structure of a bispecific antibody. BiTEs=bispecific T cell engagers

AMG 420 (previously named BI 836909) is a novel BiTE targeting BCMA on myeloma cells and CD3 on T cells, which has induced multiple myeloma cell lysis in preclinical models.127 In the first-in-human phase I study of AMG 420 in patients with at least two lines of treatment, AMG 420 was given as a continuous infusion with a pump for four week infusions, six week cycles, and a maximum of 10 cycles. The maximum tolerated dose was 400 g/day; seven (70%) of 10 patients responded to this dose. Serious adverse events were seen in 48% of patients, which were most commonly infections; and two patients had reversible grade 3 polyneuropathies. Cytokine release syndrome developed in 38% of patients, with no toxicity in the central nervous system.109 A phase Ib trial with AMG 420 is currently ongoing and although this drug looks promising, the continuous intravenous infusions present logistical challenges for patients and healthcare systems (table 2). AMG 701 is a modified version of AMG 420 (by addition of an Fc domain) with an extended half life that is suitable for dosing once a week and is being investigated in a phase I study.128

Another BCMA bispecific antibody, CC-93269, is being studied in an ongoing phase I clinical trial. This humanized 2+1, immunoglobulin G 1 based, T cell engager binds to BCMA bivalently on myeloma cells and CD3 monovalently on T cells. The bivalent binding could lead to improved potency, tumor targeting, and retention.129 All doses (range 0.15-10 mg) were given intravenously over two hours weekly for the first three cycles, every two weeks for the next three cycles, and then monthly. The most common treatment emergent adverse events of grade 3 or higher included neutropenia, anemia, and infections. Cytokine release syndrome was seen in 77% of patients, with all events developing after the first dose and less common with subsequent doses. The incidence increased with higher doses, and only one patient had cytokine release syndrome of grade 3 or higher leading to their death. In 30 patients treated, the overall response rate was 43.3% and dose dependent. The overall response rate was 88.9% in nine patients in the highest dose cohort.110

Teclistamab (JNJ-64007957) is a humanized, immunoglobulin G-4 based, bispecific DuoBody antibody that binds to BCMA and CD3 that is being studied in a phase I clinical trial. In the dose escalation part, 78 patients received doses ranging from 0.3 g/kg to 720 g/kg. The drug is given intravenously every week, with one to three step-up doses given within one week before the full dose. The overall response rate was dose dependent with no responses at doses 0.3-19.2 g/kg, 30% at 38.4-180 g/kg, and 67% at 270 g/kg. Cytokine release syndrome was seen in 56% of patients overall and 65% patients at doses over 38.4 g/kg. The most common adverse events at grade 3 or higher that were related to treatment were cytopenias and infections (table 2).111

Antibody drug conjugates are complex molecules composed of an antibody that targets cancer cells and are linked to a biologically active cytotoxic drug (known as the payload; fig 7).125 Belantamab mafodotin (GSK2857916) is a novel humanized and afucosylated (to improve antibody dependent cell mediated cytotoxicity) antibody drug conjugate that targets BCMA. It consists of an anti-BCMA monoclonal antibody conjugated to monomethyl auristatin F, a potent microtubule inhibitor.130 This antibody drug conjugate was shown to have selective myeloma cell killing in vitro and in vivo thus setting the stage for clinical trials.130131

Structure of an antibody drug conjugate

This antibody was studied in a two part phase I study. The drug was well tolerated with no dose limiting toxicities, although corneal events (such as blurry vision, dry eyes, photophobia) were seen in about 58% of patients; these events are a known toxicity of monomethyl auristatin F.132 In the dose expansion phase, 35 patients were treated, and the overall response rate was 60% with a median progression free survival 12 months.133 In a phase II, two arm study, the antibody was used in patients with relapsed and refractory multiple myeloma who had failed at least three lines of treatment. The overall response rate was 31% at the 2.5 mg/kg dose and 34% at the 3.3 mg/kg dose, which was significantly lower than the phase I study. The corneal changes or keratopathy were seen in 70% and 75% of patients, respectively. Owing to the similar response rates with the 2.5 mg/kg and 3.3 mg/kg doses and a more favorable side effect profile with the lower dose, 2.5 mg/kg will be the dose used for future studies.112 Based on these data, belantamab is the first anti-BCMA treatment to be FDA approved for relapsed and refractory multiple myeloma patients who have received four prior treatments including an anti-CD38 monoclonal antibody, a proteasome inhibitor, and an immunomodulatory agent.

Preliminary results for another study with 18 patients treated on the belantamab, bortezomib, and dexamethasone arm was presented recently, with an overall response rate of 78%; however, all 18 patients developed grade 1-3 keratopathy.113 This visual toxicity is a unique but potentially serious side effect to this drug that needs close monitoring with an ophthalmologist. Another antibody drug conjugate, DFRF4539A, is an anti-FcRH5 (also known as FcRL5) antibody conjugated to monomethyl auristatin and has shown limited activity and high incidence of toxicity in a phase I study; therefore, it was unsuccessful for this disease (table 2).134135

Read more here:
Emerging immunotherapies in multiple myeloma - The BMJ

As induction therapies continue to improve the depth of response (DOR) in patients with multiple myeloma, it may be possible to eliminate the need forautologous stem cell transplant (ASCT) in this population, provided that deep responses with up-front therapy can be achieved, according to Jeffrey Wolf, MD.

There are a lot of reasons I would like to get rid ofautologous stem cell transplants, Wolf said. Newer drugs and newer regimens are going to allow us to get there, [provided] we [conduct] the right studies.

There has been notable growth of induction therapies within the myeloma space, particularly with regard to triplet regimens, such as lenalidomide (Revlimid), bortezomib (Velcade), and dexamethasone (RVd), as well as carfilzomib (Kyprolis), lenalidomide, and dexamethasone (KRd). Daratumumab (Darzalex) is also making great progress by quickly moving to the frontline setting for patients who are transplant ineligible, as well as for older patients who appear to tolerate the agent well, Wolf explained.

Its possible that as our induction therapies improve over the next few years, we may be able to eliminateautologous stem cell transplantation as a form ofconsolidationif we [can] get deep responses, such as minimal residual disease (MRD) [negativity] with just our initial induction therapy, said Wolf.

In an interview with OncLive, Wolf, a clinical professor within the Department of Medicine at University of California, San Francisco (UCSF) and director of the Myeloma Program at the UCSF Helen Diller Family Comprehensive Cancer Center, discusses treatment options for patients with myeloma, in addition to how MRD can be used to inform clinical decisions and improve patient outcomes.

OncLive: Could you discuss the evolution of induction therapy in multiple myeloma?

Wolf: Induction therapy for myeloma has evolved tremendously over the past few years, [especially] triplet induction [regimens, such as] RVd or KRd,based on recent studies. Daratumumab is certainly making a rapid move to be included in [frontline] therapy, especially in patients who are not eligible for transplant. It seems [that if the agent is] appropriate for them, it could be appropriate for younger patients, as well, but we dont have an FDA approval [in younger patients yet].

The goal [of treatment] is DOR, [which] is measured byMRD.

What are some of the strategies that are being used for transplant-eligible and -ineligible patients with newly diagnosed disease?

One of the main studies I presented [during my talk] was the ENDURANCE trial, which was just presented at the 2020 American Society of Clinical Onccology Virtual Meeting. The trial was what we thought of as somewhat of a flawed study that [claimed] RVd and KRd were equivalent for progression-freesurvival (PFS) [in transplant-eligible patients]. The problem is that most patients move on to transplant. What we really should have been looking at was KRd plus transplant versus RVd plus transplant. [The studys design] eliminated patients from the analysis when they underwent transplant.

In the transplant-ineligible setting, there are a lot more studies to refer to, including the studies that moved daratumumab to the frontline setting. Initially, we learned that maintenance lenalidomide was necessary even in patients who are not posttransplant. Another study showed thatdaratumumab added to bortezomib, melphalan, and prednisone(VMP) really improved PFS and overall survival (OS). Of course, [there is also the study that evaluated]daratumumab plus lenalidomide and dexamethasone (Rd) versusRd, [whereby] daratumumab adds tremendous depth and durability.

This morning I put 2 older patients on that regimen. They tolerateddaratumumab quite well and can get a DOR that is equivalent to that of a patient who undergoes transplant.

How are you navigating among the agents that are currently available in practice?

It seems complicated because there are so many options, but it tends to sort itself out by circumstance. For example, this morning I saw a transplant-ineligible patient who didnt getMedicare Part D and, therefore, I couldnt give them oral therapies such aslenalidomide. Their referring doctor gave them cyclophosphamide, bortezomib, and dexamethasone (CyBorD), which is all [intravenous]. This morning I decided to switch them tosubcutaneous daratumumab, bortezomib,dexamethasone.

Most of these regimens will give you the same responses. I often start with preexisting conditions to decide which drugs I eliminate and which drugs Im going to use instead.

What is the role of transplant in this space right now? Will transplant retain its role in future?

I started out as a transplanter 40 years ago, so its hard to say this, but Ive been thinking for the past decade that were on the verge of getting rid ofautologous stem cell transplant from myeloma. If our induction therapies are so effective, we may be able to eliminate transplant. Id like to see us conduct trials, such as the MASTER trial, led by Luciano Costa, MD, PhD, of the University of Alabamas Birmingham School of Medicine, in which patients who achieve MRD [negativity] with induction therapy do not proceed to transplant. Were going to have to do those kinds of studies to eliminate transplant. Its a fairly primitive kind of therapy in that we give high-dose therapy to wipe out as much myeloma as we can, and in doing so wipe out vulnerable bone marrow. Then, we have to [transplant] frozen stem cells from the patient. [The alkylator] leads to increased risk of secondary malignancies. Patients lose their hair, they get sick for 2 or 3 months, and theyre immunocompromised, which is not a good thing to be these days.

How might MRD be used to optimize patient outcomes?

In the world of myeloma, experts are divided over whether to use MRD to make decisions. I happen to be in the group [in favor of MRD]. Its no different than usingmonoclonal spike (m spike) orlight chain measurements to make decisions. Were already using MRD in chronic lymphocytic leukemia andacute lymphocytic leukemia to make decisions. The only restriction in myeloma is that, so far, weve only been able to [evaluate MRD with] bone marrow and not blood. If we can start measuring MRD in myeloma [through] blood, it would be as commonly used as light chain or m spike measurements to make these decisions. Itll help us immeasurably because most of the time were getting patients into complete remission (CR)which it really isnt CRand then we keep them on regimens for maintenance.

For example, when we dont know what were accomplishing, [its worth asking], Are patients getting better? Are patients staying the same? Is the MRD going up while were treating patients with drugs that clearly arent working? Should we be switching therapies? For patients who are MRD [negative] year after year, is there any reason why we have them on these therapies that are so costly and have so many adverse effects (AEs) and secondary malignancies? There are all kinds of reasons as to why we should be using MRD for measurement. I would argue that the only reason why we arent using MRD is because we have to [evaluate] it in bone marrow, which is a little more uncomfortable compared with blood, and maybe because the studies havent been completed yet. Its only a matter of 1 or 2 years before we will be using MRD to make clinical decisions. In terms of frontline therapies, using MRD might allow us to [avoid] transplant or indicate when weve given enough treatment and is time to move to maintenance. There a lot of possibilities to using MRD instead of just historically saying, this is how we do it.

Read more:
Improved Induction Therapies Could Eliminate the Need for Transplant in Myeloma - OncLive

When youre in pain, its important to find effective, long-lasting solutions that can provide short recovery periods. This is what regenerative medicine offers. Over the past decade, there has been a growing field of medicine that utilizes the bodys own healing capabilities using platelet-rich plasma and mesenchymal stem cells (MSCs). This growing field is labeled as regenerative medicine. Regenerative therapies focus on healing and help regrow damaged tissue naturally. Regenerative injection therapy is used to provide relief to musculoskeletal injuries that involve damage to ligaments, tendons, cartilage, joints, and discs.

Watch video of PRP:

PRP therapy on Better CT

PRP is safeas we are using what your body naturally produces, concentrating the desired critical components and transplanting them into the affected area for effective tissue regeneration and healing. There is no risk of rejection and very minimal overall procedural risk.

FDA regulations do not allow for the cloning of stem cells or growing them in a lab. Also, stem cells derived from fat cells are not approved by the FDA as it does not allow for manipulation. This leaves us to another rich stem cell source in our body which is bone marrow. Stem cells exist in our bodies and are rudimentary cells that can differentiate into other cells.

Think of bone marrow stem cells as the mother cell that is responsible for producing new blood cells. Bone marrow contains hundreds of growth factors and is often used for severe degenerative conditions or where PRP therapy may not be sufficient to provide the growth factors needed to provide relief.

Lastly, there are many offshoot therapies that use biologics derived from placental tissue or blood cord. These biologics are sometimes marketed as Stem cells but are not stem cells and contain zero viable cells. What they contain are growth factors that can also aid when combined with PRP or Stem Cells derived from your own body.

MSCs and PRPmay be used to target a number of conditions that could benefit from their healing and regenerative qualities. Especially when considering chronic pain, alternative solutions may be necessary if it has been difficult to find relief. Along with generalized joint pain, MSCs and PRPmay be used to target:

With so many options for joint pain out there, you may be wondering what benefits choosing stem cell therapy provides. Overall, because mesenchymal stem cell therapy utilizes biologic material harvested directly from the patients body, the general benefits include minimal risk, minimal recovery time, and minimal worry:

Avoid surgery and its many complications and risks: Stem cell therapy is a minimally invasive, non-surgical procedure.

Minimal post-procedural recovery time: One of the most time-consuming factors of any injury is not always the treatment itself, but actually the recovery time. With stem cell therapy, recovery time is minimal.

No risk of rejection: Due to using biologics extracted from the patient, there is no risk of rejection.

No communicable disease transmission: As the cells originate within your own body, there is no risk of spreading disease from or to another person.

If you are suffering from joint pain, back pain, or a debilitating condition like osteoarthritis, it is important to consider all of your available options. Our elite team of professionals can determine if you are the right candidate for MSCs. If youre interested in learning more, contact us today.

View original post here:
Regenerative Therapy by Dr. Roshni Patel on Better CT - Farmington, CT - Patch.com

Differences in biological sex can dictate lifelong disease patterns, says a new study by Michigan State University researchers that links connections between specific hormones present before and after birth with immune response and lifelong immunological disease development.

Published in the most recent edition of the Proceedings of the National Academy of Sciences, the study answers questions about why females are at increased risk for common diseases that involve or target the immune system like asthma, allergies, migraines and irritable bowel syndrome. The findings by Adam Moeser, Emily Mackey and Cynthia Jordan also open the door for new therapies and preventatives

This research shows that its our perinatal hormones, not our adult sex hormones, that have a greater influence on our risk of developing mast cell-associated disorders throughout the lifespan, says Moeser, Matilda R. Wilson Endowed Chair, professor in the Department of Large Animal Clinical Sciences and the studys principle investigator. A better understanding of how perinatal sex hormones shape lifelong mast cell activity could lead to sex-specific preventatives and therapies for mast cell-associated diseases.

Mast cells are white blood cells that play beneficial roles in the body. They orchestrate the first line of defense against infections and toxin exposure and play an important role in wound healing, according to the study, Perinatal Androgens Organize Sex Differences in Mast Cells and Attenuate Anaphylaxis Severity into Adulthood.

However, when mast cells become overreactive, they can initiate chronic inflammatory diseases and, in certain cases, death. Moesers prior research linked psychological stress to a specific mast cell receptor and overreactive immune responses.

Moeser also previously discovered sex differences in mast cells. Female mast cells store and release more inflammatory substances like proteases, histamine and serotonin, compared with males. Thus, female mast cells are more likely than male mast cells to kick-start aggressive immune responses. While this may offer females the upper hand in surviving infections, it also can put females at higher risk for inflammatory and autoimmune diseases.

IBS is an example of this, says Mackey, whose doctoral research is part of this new publication.

While approximately 25% of the U.S. population is affected by IBS, women are up to four times more likely to develop this disease than men.

Moeser, Mackey and Jordans latest research explains why these sex-biased disease patterns are observed in both adults and prepubertal children. They found that lower levels of serum histamine and less-severe anaphylactic responses occur in males because of their naturally higher levels of perinatal androgens, which are specific sex hormones present shortly before and after birth.

Mast cells are created from stem cells in our bone marrow, Moeser said. High levels of perinatal androgens program the mast cell stem cells to house and release lower levels of inflammatory substances, resulting in a significantly reduced severity of anaphylactic responses in male newborns and adults.

We then confirmed that the androgens played a role by studying males who lack functional androgen receptors, says Jordan, professor of Neuroscience and an expert in the biology of sex differences.

While high perinatal androgen levels are specific to males, the researchers found that while in utero, females exposed to male levels of perinatal androgens develop mast cells that behave more like those of males.

For these females, exposure to the perinatal androgens reduced their histamine levels and they also exhibited less-severe anaphylactic responses as adults, says Mackey, who is currently a veterinary medical student at North Carolina State University.

In addition to paving the way for improved and potentially novel therapies for sex-biased immunological and other diseases, future research based will help researchers understand how physiological and environmental factors that occur early in life can shape lifetime disease risk, particularly mast cell-mediated disease patterns.

While biological sex and adult sex hormones are known to have a major influence on immunological diseases between the sexes, were learning that the hormones that we are exposed to in utero may play a larger role in determining sex differences in mast cell-associated disease risk, both as adults and as children, Moeser said.

For more information on Moesers research, go to the Gastrointestinal Stress Biology Laboratory. Also, visit the MSU College of Veterinary Medicines website for more about its research efforts.

(Note for media: Please include the following link to the study in all online media coverage: https://www.pnas.org/content/early/2020/09/10/1915075117)

Read the original here:
New research connects the hormones we're born with to lifetime risk for immunological diseases - MSUToday

By InYourArea Community

The three generations of the Taylor family, Allan, Corey and Chris.

Three generations of a lifesaving family have defied the odds by being selected to donate their bone marrow to three complete strangers thousands of miles away.

Allan, Chris and Corey Taylor were all selected from the Welsh Bone Marrow Donor Registry as the only suitable matches in the world capable of saving patients from Africa, America and Europe respectively.

To mark World Bone Marrow Donor Day on Saturday, September 19, the Taylor family from Pontypool, Torfaen, is calling on 17-30 year olds across Wales to volunteer their lifesaving bone marrow by joining the Welsh Bone Marrow Donor Registry.

Chris Harvey, Head of the Welsh Bone Marrow Donor Registry, said:

Every day blood cancer patients around the world are desperately hoping to find a suitable bone marrow match. The requirements needed to match a patient with a bone marrow donor are very specific and this sadly means three in ten patients will never find the potentially lifesaving bone marrow donor they need.

Last year 50,000 donations were made from around 40 million volunteers signed up internationally, which shows just how rare it is to be someones match.

The Taylors really are a family of lifesavers.

Bone marrow is the soft, spongy tissue found at the centre of certain bones in your body where blood stem cells live. Blood stem cells produce all your essential blood cells, such as red blood cells to carry oxygen and white blood cells to fight infection. There are some diseases, such as some forms of leukaemia, which stop bone marrow working properly. For these patients, the best hope of recovery is to receive a bone marrow transplant.

Talking of his experience, 65 year old Allan, the eldest of the Taylor family donors said: A lot has changed since I donated back in 2005 but the constant has been the fantastic Welsh Bone Marrow Donor Registry staff who are always at hand throughout the process to look after you and to offer you reassurance and support.

Chris, 33, and the middle generation of the three donors, who currently works for Torfaen Council, recounted his experience: I was already a blood donor and decided one day to sign up to the Welsh Bone Marrow Donor Registry. I am a big Superman fan so Ive always dreamed of being a superhero. I cant fly but I was able to save a life by giving my bone marrow its as close as Ill ever get to being a hero!

My employer was absolutely fantastic and couldnt have been more supportive. The procedure, which lasted about four hours, was absolutely fine. After my donation I stayed for about 30 minutes, had a cup of tea and then I went home. I rested over the weekend and was straight back to work on the Monday.

The youngest donor in the family, 25 year old Corey, added: Theres a massive misconception and stigma out there around the bone marrow process. People seem to think they have to go through an operation type medical procedure which involves taking the bone marrow directly from your hip bone.

The reality is quite different as 85% of donations are collected through a non-surgical procedure very similar to a blood donation, it just takes a little longer and theres very little discomfort.

The PBSC process collects stem cells directly using a specialist machine. The process involves drawing blood out of one arm, extracting the stem cells, before returning the remaining blood to your other arm. Donors typically return to normal activity a day or two following the donation.

Allan Taylor summed up their achievement by saying:

I know people can talk about giving money but I think we have done something much more than that, we have potentially given three people the gift of life. You cant put a price on that! Ive done what I can. You can too.

If you are aged 17-30, do something amazing, join the Welsh Bone Marrow Donor Registry. Simply, book to give blood or call the Welsh Blood Service on 0800 252 266.

Read the original here:
Pontypool family gives three lifesaving bone marrow donations - In Your Area

Its already hard enough for blood cancer patients to find a match through the international bone marrow registry, which pairs patients with potential donors who have the right type of tissue. But if youre Black and Jewish?

For people with multiple ethnic backgrounds who need marrow or stem cell transplants, matching is even harder.

I remember the doctor saying something like if he was an Irish white boy from Ireland, he might have a better chance, Monika Clark said about her son, 24-year-old Jordan Jackson-Clark of Oakland.

Jackson-Clark, whom his mom describes as mixed ethnicity and biracial, is likely to need a bone marrow transplant after a diagnosis of leukemia two weeks ago.

It was so out of the blue, Clark said. It was so unexpected.

Jackson-Clark had experienced a few bouts of intense stomach pain over the past summer, one strong enough to send him to the ER. Clark was concerned, but she was never expecting the recent call that they got from the doctor.

Through tears, Clark described the blow of hearing the diagnosis for her son, a Berkeley High School grad who was a camp counselor at the East Bay JCC and a member of the Jewish fraternity AEPi.

Hes just a gentle, loving young man, she said.

Jackson-Clark has acute myeloid leukemia, a cancer of the blood and bone marrow. Hes in the hospital getting chemotherapy for the next few weeks. In the meantime, knowing how difficult it will be to find a match for her son, Clark is desperately trying to get the word out about the bone marrow registry.

Please step out and do something very simple to save a life, she said.

The ethnic background of a cancer patient who needs a transplant matters, because the markers used to match a donor and patient are inherited. Having the same markers as a donor makes it a lot more likely that the patients body will accept the life-saving bone marrow or stem cells.

But the makeup of the database of potential donors is mostly white. For people of color and mixed race, the percentage of matches is 23 percent, and for white Caucasians its 77 percent, Clark said.

According to the nonprofit Gift of Life, while more than 12 percent of the American population is Black, only 4 percent on the registry are, and the percentages are similarly out of proportion for other ethnic groups.

Gift of Life was founded by Jay Feinberg, who was diagnosed with leukemia more than 20 years ago and needed a bone marrow transplant from a white Ashkenazi Jew. He sought a donor match, but at that time the database was sorely lacking in diversity. Efforts since then by his organization and others have greatly increased ethnic representation in the registry, but matches for mixed-ethnicity patients remain scarce. Jackson-Clark has the best chance of being matched with another person who is Black, white and Ashkenazi, but there simply arent many in the database.

The solution is getting more potential donors into the system. Clark is asking people to get tested with a simple cheek swab through Be the Match or any other registration service not only if they think they might be a match for her son, but also for all of the other patients out there who need matches. Optimal donor ages are 18 to 44; registration is free and can be done through the mail. That puts them on the international registry of potential donors, and the more people who are on the list, the more likely it is that they could be a match for a cancer patient.

Thats why Rabbi Yigal Rosenberg of Chabad of Santa Clara held a registration drive in February and encouraged young people to get on the list. When he got a call from Gift of Life a few days later, he thought it had something to do with the event.

They said, actually, you are a match! he said.

Rosenberg had the right kind of stem cells to help a 40-year-old man based on a swab hed given 10 years previously in New Jersey. (Whether marrow or stem cells are donated depends on the patients treatment needs.)

Im like, what are the chances? Rosenberg said. Literally I just hosted an event two days ago!

He immediately said yes and began a required series of injections to boost stem-cell production checking with another rabbi to make sure it was OK to have the shots on Shabbat as well.

This is the one thing youre allowed to compromise on, in Shabbat observance, is to save a life, he said.

Then, at the beginning of September, he drove down to San Bernardino, where he was put up in a hotel. He spent one day at the donation center attached to a machine that pumped blood out, filtered out and collected the stem cells, and returned the blood to his body. Rosenberg said the experience wasnt difficult at all.

I just felt so empowered during the entire process, he said.

He even livestreamed it on Facebook as a way to encourage more registrations, and to dispel some of the fear around donation. (Whether a patient requires the donors marrow or stem cells depends on the particular treatment protocol.)

I went right back to the hotel, jumped in the Jacuzzi for a bit and took a nap, he said. The next day he was back on his way to Santa Clara to resume his duties.

Clark, a former JCC preschool teacher, said it is important for people to know that donating stem cells and even bone marrow is not as intrusive or painful as it used to be. And anyone on the registry can always decide later that theyre not ready to donate, so getting the swab does not commit them to doing so.

The greatest Rosh Hashanah gift from the Jewish and biracial communities would be to spread the word far and wide with your communities, and to please get on the donor list by sending away for a simple and free cheek swab, she said. You just might save my or someone elses childs life.

Read more:
Oakland 24-year-old seeking multiethnic bone marrow donor - The Jewish News of Northern California

"It was like a curtain dropping over your eyes when youre going into the dark," Debbie Ramirez, Odessa resident said.

ODESSA, Texas Debbie Ramirez wakes up every morning just like you and I do.

She brushes her hair, like you and I do. She puts on mascara.

And then she puts on her shoes, just like you and I do.

Except, she doesn't get ready the way you and I do. That's because Debbie Ramirez is blind.

"I can see me moving my hand right in front of me, but right here I don't see anything," Debbie Ramirez, Odessa resident said.

It was six years ago when Debbie's life changed.

"It was like a curtain dropping over your eyes when you're going into the dark," Ramirez said.

That darkness not only took her sight. Debbie lost 75% of her memory and with it most of her life.

For years she sought out help from doctors. But none had an answer.

"They all told me they didn't know why I went blind. Nobody could explain it," Ramirez said.

"I thought how am I going to live like this? I've seen my entire life, I don't know how to live blind," Ramirez said.

Then one day, a phone call changed her life once again.

"I had a very large tumor. It was the size of an orange," Ramirez said.

An Odessa doctor finally had an answer. But what would come next was worse than you could imagine.

"He rushed me to Dallas by ambulance because he said I had less than 30 days to live," Ramirez said.

Her thoughts went straight to her family.

"My children are everything," Ramirez said.

And what would happen if she didn't make it through the surgery?

But the surgery was a success. The tumor was gone.

"I was me again and I just wanted to move on with life and grasp it because I had been given a second chance," Ramirez said.

Her memory started to come back, the pain and the headaches faded away, and Debbie began to take charge of her life again.

She re-learned how to cook, clean and even how to do laundry.

And now Debbie has a chance to get her sight back through a clinical trial in Florida.

"I am just really hoping to get my sight back more than anything. It's been the hardest thing I've ever gone through," Ramirez said.

The trial would use neurons from bone marrow to generate new stem cells in her retina and optic nerves. But there's one more roadblock before Debbie can see her children and grandson.

"It's a lot of money and my insurance will not cover it because it has to do with stem cell therapy," Ramirez said.

But Debbie said she's not giving up.

Although Debbie can't see, she says one thing is clear to her.

"I feel like God has always been beside me," Ramirez said.

________________________________________________________________

If you'd like to help Debbie with her mission to get her sight back, you can reach out to her directly at 432-212-5726.

She said she will take any help she can get!

Original post:
Odessa woman hopes to gain back her sight after 6 years of being blind - NewsWest9.com

Fort Collins, Colorado The report on the Stem Cell Therapy Market provides an in-depth assessment of the Stem Cell Therapy market including technological advancements, market drivers, challenges, current and emerging trends, opportunities, threats, risks, strategic developments, product advancements, and other key features. The report covers market size estimation, share, growth rate, global position, and regional analysis of the market. The report also covers forecast estimations for investments in the Stem Cell Therapy industry from 2020 to 2027.

The report is furnished with the latest market dynamics and economic scenario in regards to the COVID-19 pandemic. The pandemic has brought about drastic changes in the economy of the world and has affected several key segments and growth opportunities. The report provides an in-depth impact analysis of the pandemic on the market to better understand the latest changes in the market and gain a futuristic outlook on a post-COVID-19 scenario.

Global Stem Cell TherapyMarketwas valued at 117.66 million in 2019 and is projected to reach USD255.37 million by 2027, growing at a CAGR of 10.97% from 2020 to 2027.

Get a sample of the report @ https://reportsglobe.com/download-sample/?rid=33553

The report provides an in-depth analysis of the key developments and innovations of the market, such as research and development advancements, product launches, mergers & acquisitions, joint ventures, partnerships, government deals, and collaborations. The report provides a comprehensive overview of the regional growth of each market player.

Additionally, the report provides details about the revenue estimation, financial standings, capacity, import/export, supply and demand ratio, production and consumption trends, CAGR, market share, market growth dynamics, and market segmentation analysis.

The report covers extensive analysis of the key market players in the market, along with their business overview, expansion plans, and strategies. The key players studied in the report include:

Furthermore, the report utilizes advanced analytical tools such as SWOT analysis and Porters Five Forces Analysis to analyze key industry players and their market scope. The report also provides feasibility analysis and investment return analysis. It also provides strategic recommendations to formulate investment strategies and provides insights for new entrants.

Request a discount on the report @ https://reportsglobe.com/ask-for-discount/?rid=33553

The report is designed with an aim to assist the reader in taking beneficial data and making fruitful decisions to accelerate their businesses. The report provides an examination of the economic scenario, along with benefits, limitations, supply, production, demands, and development rate of the market.

1.Stem Cell Therapy Market, By Cell Source:

Adipose Tissue-Derived Mesenchymal Stem Cells Bone Marrow-Derived Mesenchymal Stem Cells Cord Blood/Embryonic Stem Cells Other Cell Sources

2.Stem Cell Therapy Market, By Therapeutic Application:

Musculoskeletal Disorders Wounds and Injuries Cardiovascular Diseases Surgeries Gastrointestinal Diseases Other Applications

3.Stem Cell Therapy Market, By Type:

Allogeneic Stem Cell Therapy Market, By Application Musculoskeletal Disorders Wounds and Injuries Surgeries Acute Graft-Versus-Host Disease (AGVHD) Other Applications Autologous Stem Cell Therapy Market, By Application Cardiovascular Diseases Wounds and Injuries Gastrointestinal Diseases Other Applications

Request customization of the report @https://reportsglobe.com/need-customization/?rid=33553

Regional Analysis of the Market:

For a better understanding of the global Stem Cell Therapy market dynamics, a regional analysis of the market across key geographical areas is offered in the report. The market is spread acrossNorth America, Europe, Latin America, Asia-Pacific, and Middle East & Africa.Each region is analyzed on the basis of the market scenario in the major countries of the regions to provide a deeper understanding of the market.

Benefits of the Global Stem Cell Therapy Report:

To learn more about the report, visit @ https://reportsglobe.com/product/global-stem-cell-therapy-market/

Thank you for reading our report. To learn more about report details or for customization information, please contact us. Our team will ensure that the report is customized according to your requirements.

How Reports Globe is different than other Market Research Providers

The inception of Reports Globe has been backed by providing clients with a holistic view of market conditions and future possibilities/opportunities to reap maximum profits out of their businesses and assist in decision making. Our team of in-house analysts and consultants works tirelessly to understand your needs and suggest the best possible solutions to fulfill your research requirements.

Our team at Reports Globe follows a rigorous process of data validation, which allows us to publish reports from publishers with minimum or no deviations. Reports Globe collects, segregates, and publishes more than 500 reports annually that cater to products and services across numerous domains.

Contact us:

Mr. Mark Willams

Account Manager

US: +1-970-672-0390

Email:[emailprotected]

Web:reportsglobe.com

Read more here:
Stem Cell Therapy Market to Witness Exponential Growth by 2020-2027 | Leading Players Osiris Therapeutics, Medipost Co., Anterogen Co., Pharmicell...