FRIDAY, June 18, 2021 (HealthDay News) -- It's been more than six months since Brandy Compton last landed in a hospital emergency room.

That's an amazing medical achievement, brought about by scientific breakthroughs that have been unfortunately overshadowed by the coronavirus pandemic, experts say.

Compton, 31, was born with sickle cell disease, a genetic condition that primarily affects people of African descent.

The disease causes episodes of pain so bad that in the past, Compton had to be hospitalized frequently for full blood transfusions.

"In grade school, I was in the hospital for a week, I'd get out of the hospital for maybe a good week and a half, two weeks, and then I'd be back in the hospital for another week," recalls Compton, who lives in Hartford, Conn. "It was constant."

But last year Compton started on a once-monthly IV drug called Adakveo (crizanlizumab), one of a handful of new sickle cell drugs approved by the U.S. Food and Drug Administration just before the pandemic hit.

The drug has cut in half the amount of blood Compton requires during a transfusion, and has prevented the sort of pain crisis that would send her to an ER, she said.

As the pandemic subsides, sickle cell disease experts are now trying to spread the word about these handful of treatments that could improve and potentially extend the lives of patients.

"In the last three years or so, three new medicines got approved by the FDA with different ways of working that could actually be used together and give more preventive, disease-modifying types of approaches rather than just waiting for the bad complications to occur," said Dr. Lewis Hsu, chief medical officer of the Sickle Cell Disease Association of America.

Progress also is being made on cures that would fix the genetic error that causes sickle cell, either through a donor bone marrow transplant or gene therapy that would fix the patient's own stem cells, Hsu added.

'Jagged rocks shredding your veins'

Sickle cell disease affects the shape of a person's red blood cells, which are normally disc-shaped and flexible enough to move easily through blood vessels.

The red blood cells of a person with sickle cell are crescent-shaped, resembling a sickle. The cells are stiff and sticky, and cause pain episodes and other health problems when they clump together in different parts of the body. They also are less capable of carrying oxygen to a person's tissues, causing chronic fatigue.

"Sickle cell feels like jagged rocks shredding the inside of your veins, and your bones being crushed," Compton says.

Sickle cell disproportionately affects Black people in the United States. About 1 in 13 Black babies is born with the genetic trait for sickle cell, and about 1 in every 365 Black babies is born with sickle cell disease, according to the U.S. National Institutes of Health.

For a long time, there was no treatment at all for sickle cell, Hsu said, outside of regular blood transfusions.

"At the age of 13, I started getting blood transfusions," Compton recalls. "After that, it started getting under control. I would be able to go about a month without having to be hospitalized. That time got longer as I got older."

In 1998, the FDA approved hydroxyurea, an oral medicine that can reduce or prevent sickle cell complications in people with specific subtypes of the disease. But following that, there was a "long gap" in new treatments, Hsu said.

That ended in 2017 with the approval of L-glutamine powder, sold under the brand name Endari. Patients sprinkle a packet of this purified amino acid powder on their food or drink twice a day, Hsu said.

"Particularly, it help the red cells be healthier and have better energy stores," Hsu said.

But the two real breakthroughs occurred in November 2019, on the cusp of the pandemic, with FDA approval of two new drugs -- Adaveko and Oxbryta (voxelotor).

Adaveko essentially creates an "oil slick" in the bloodstream that keeps sickled red blood cells from clumping, explained Genice Nelson, program director of the New England Sickle Cell Institute at the University of Connecticut. She also leads Compton's care.

"It helps to improve blood flow by having the cells move along better, gliding instead of sticking to each other," Hsu said.

Showing promise at a high price

Thanks to Adaveko, Compton now only needs four units of blood every four weeks, down from seven, and does not suffer frequent pain episodes.

The other drug, Oxbryta, improves the ability of deformed red blood cells to hold onto oxygen, Nelson said.

"It inhibits the deformation of the red blood cell, so it's able to hold onto oxygen," Nelson said. "Because the red blood cell is able to hold onto oxygen, it's able to give that oxygen to the tissues within the body."

Because these drugs act in different ways, the hope is that a sickle cell patient taking two or more would receive added benefits, Hsu said.

Unfortunately, the new drugs are expensive and insurance companies have balked at paying for them, Hsu said.

For example, Adakveo costs about $10,000 a month for a patient, Nelson said. It seems like a great expense, but is likely cheaper than regular ER visits.

"If someone is in the hospital several days out of the month every month, dollar for dollar you'd rather invest it in preventing them from being in the hospital rather than trying to treat them once they're in the hospital," Nelson said.

Despite this, insurance companies have dragged their feet accepting the new drugs.

"We have great, great difficulty prescribing them and getting them authorized," Hsu said. "It's a case-by-case issue for every single prescription. It takes two months or so to get the authorizations, and then we go for refills or another prescription and they have to go through the same process again."

Experts hope that the track record of these drugs will lead insurance companies to relent.

"The data is clear there is benefit to patients being on disease-modifying therapies," said Dr. Alexis Thompson, head of hematology for the Ann & Robert Lurie Children's Hospital in Chicago. "The natural history of sickle cell disease is devastating. To not think about where the opportunities are to intervene early, to modify the natural history of the disease and really reduce suffering, is something we all need to be committed to."

Great progress also has been made in cures for sickle cell, Hsu added.

Transplants tricky, but improvements underway

For a long time, the only potential cure was a full bone marrow transplant from a genetically matched donor, usually a sibling, Hsu said. Only children could handle the stress of this cure, because their existing bone marrow had to be killed off through chemotherapy prior to the transplant.

But improved medications that inhibit immune system rejection now have made transplants also available to children who have a half-matched relative. These drugs selectively inhibit immune attack cells without harming the healthy stem cells being transplanted, Hsu said.

Over the past decade, even adult sickle cell patients have been receiving transplants, through a method that replaces most but not all of the person's bone marrow.

"This is a mixture that's enough to allow the donor to supply most of the red cells that are floating around, so they're not sickle red cells, and the tiny portion of host red cells are diluted heavily," Hsu said. "This has found to be successful and stable."

Five to seven research groups also are working on what could be the ultimate cure for sickle cell, a gene therapy that would take the person's own bone marrow and fix it to remove the genetic anomaly that causes the disease.

"You'd no longer have to find a donor for the stem cells," Hsu said. "You basically do your own donation of stem cells."

Research efforts are focused on fixing the stem cells by treating them with a genetically modified virus, or by using newly discovered methods of gene editing, Hsu said. In both cases, the person's bone marrow is removed, treated in a lab, and then put back inside them.

These efforts have met with some hurdles, with the gene therapy causing leukemia in something like 2 of every 47 cases in some instances, Hsu said.

"We do need to keep working on ways to limit the side effects or toxicity of those approaches, but one cannot argue that the early data is quite remarkable," Thompson said.

Compton, now the mother of a healthy 9-year-old boy, is hopeful that these efforts will lead to a cure, even though she doesn't expect to benefit from one at her age.

"I know about gene therapy and things like that," Compton said. "I do hope there would be a cure available."

More information

The Mayo Clinic has more about sickle cell disease.

SOURCES: Brandy Compton, Hartford, Conn.; Lewis Hsu, MD, PhD, chief medical officer, Sickle Cell Disease Association of America; Alexis Thompson, MD, MPH, head, hematology, Ann & Robert Lurie Children's Hospital, Chicago; Genice Nelson, DNP, program director, New England Sickle Cell Institute, University of Connecticut, Farmington

Read the original:
Sickle Cell Plagues Many Black Americans, But There's Hope for Better Treatments - HealthDay News

Bone Marrow Stem Cells Comments Off on Sickle Cell Plagues Many Black Americans, But There’s Hope for Better Treatments – HealthDay News | June 25th, 2021

REDWOOD CITY, Calif. and NEW YORK and BASEL, Switzerland, June 21, 2021 /PRNewswire/ --Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, andAruvant Sciences, a private company focused on developing gene therapies for rare diseases, today announced that they have entered a non-exclusive research collaboration to evaluate the use of JSP191, Jasper's anti-CD117 monoclonal antibody, as a targeted, non-toxic conditioning agent with ARU-1801, Aruvant's investigational lentiviral gene therapy for sickle cell disease (SCD). The objective of the collaboration is to evaluate the use of JSP191 as an effective and more tolerable conditioning agent that can expand the number of patients who can receive ARU-1801, a potentially curative treatment for SCD.

"This research collaboration with Aruvant is the first to use a clinical-stage antibody-based conditioning agent and a novel clinical-stage gene therapy, giving this combination a clear advantage by moving beyond the harsh conditioning agents currently used for gene therapy and establishing this next-generation potentially curative treatment as a leader in sickle cell disease," said Kevin N. Heller, M.D., executive vice president, research and development of Jasper. "Our goal is to establish JSP191 as a potential new standard of care conditioning agent, broadly in autologous gene therapy and allogeneic hematopoietic stem cell transplantation."

Gene therapies and gene editing technologies generally require that a patient's own hematopoietic stem cells first be depleted from the bone marrow to facilitate the engraftment of the new, gene-modified stem cells through a process called conditioning. Other investigational gene therapies and gene editing approaches in SCD use a high-dose chemotherapy such as busulfan for the conditioning regimen, which can place patients at prolonged risk for infection and bleeding, secondary malignancy and infertility. ARU-1801 is currently the only gene therapy that has demonstrated durable efficacy using both a lower dose of chemotherapy and a different agent than busulfan with a more limited side effect profile. The Aruvant-Jasper partnership is focused on evaluating the potential of using JSP191, a highly targeted anti-CD117 (stem cell factor receptor) monoclonal antibody agent, as the foundationof a novel conditioning regimen for use in combination with ARU-1801 to further reduce the negative side effects while maintaining efficacy.

"The unique attributes of ARU-1801 enable us to bring a potentially curative one-time therapy to individuals with sickle cell disease that can be delivered in the safest way possible," said Will Chou, M.D., Aruvant chief executive officer. "By partnering with Jasper to evaluate the use of JSP191 with ARU-1801, we are one step closer to developing a next-generation definitive therapy with an even more patient-friendly conditioning regimen. We believe that this combination may be able to further expand the number of patients who can benefit from ARU-1801 in the future, including potentially those with more moderate disease."

About JSP191 JSP191 is a humanized monoclonal antibody in clinical development as a conditioning agent that blocks stem cell factor receptor signaling leading to clearance of hematopoietic stem cells from bone marrow, creating an empty space for donor or gene-corrected transplanted stem cells to engraft. While hematopoietic cell transplantation can be curative for patients, its use is limited because standard high dose myeloablative conditioning is associated with severe toxicities and standard low dose conditioning has limited efficacy. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients. It is currently enrolling in two clinical trials for myelodysplastic syndromes (MDS)/acute myeloid leukemia (AML) and severe combined immunodeficiency (SCID) and expects to begin enrollment in four additional studies in 2021 for severe autoimmune disease, sickle cell disease, chronic granulomatous disease and Fanconi anemia patients undergoing hematopoietic cell transplantation.

About ARU-1801 ARU-1801 is designed to address the limitations of current curative treatment options, such as low donor availability and the risk of graft-versus-host disease (GvHD) seen with allogeneic stem cell transplants. Unlike investigational gene therapies and gene editing approaches which require fully myeloablative conditioning, the unique characteristics of ARU-1801 allow it to be given with reduced intensity conditioning ("RIC"). Compared to myeloablative approaches, the lower dose chemotherapy regimen underlying RIC has the potential to reduce not only hospital length of stay, but also the risk of short- and long-term adverse events such as infection and infertility. Preliminary clinical data from the MOMENTUMstudy, an ongoing Phase 1/2 trial of ARU-1801 in patients with severe sickle cell disease, demonstrate continuing durable reductions in disease burden.

The MOMENTUM Study Aruvant is conducting the MOMENTUM study, which is evaluating ARU-1801, a one-time potentially curative investigational gene therapy for patients with SCD. This Phase 1/2 study is currently enrolling participants, and information may be found at momentumtrials.comwhich includes a patient brochure, an eligibility questionnaireand information for healthcare providers.

About Jasper Therapeutics Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The company is advancing two potentially groundbreaking programs. JSP191, a first-in-class anti-CD117 monoclonal antibody, is in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplantation. It is designed to enable safer and more effective curative allogeneic and autologous hematopoietic cell transplants and gene therapies. In parallel, Jasper Therapeutics is advancing its preclinical engineered hematopoietic stem cell (eHSC) platform, which is designed to overcome key limitations of allogeneic and autologous gene-edited stem cell grafts. Both innovative programs have the potential to transform the field and expand hematopoietic stem cell therapy cures to a greater number of patients with life-threatening cancers, genetic diseases and autoimmune diseases than is possible today. For more information, please visit us at jaspertherapeutics.com.

About Aruvant Sciences Aruvant Sciences, part of the Roivant family of companies, is a clinical-stage biopharmaceutical company focused on developing and commercializing gene therapies for the treatment of rare diseases. The company has a talented team with extensive experience in the development, manufacturing and commercialization of gene therapy products. Aruvant has an active research program with a lead product candidate, ARU-1801, in development for individuals suffering from sickle cell disease (SCD). ARU-1801, an investigational lentiviral gene therapy, is being studied in a Phase 1/2 clinical trial, the MOMENTUM study, as a one-time potentially curative treatment for SCD. Preliminary clinical data demonstrate engraftment of ARU-1801 and amelioration of SCD is possible with one dose of reduced intensity chemotherapy. The company's second product candidate, ARU-2801, is in development to cure hypophosphatasia, a devastating, ultra-orphan disorder that affects multiple organ systems and leads to high mortality when not treated. Data from pre-clinical studies with ARU-2801 shows durable improvement in disease biomarkers and increased survival. For more information on the ongoing ARU-1801 clinical study, please visit http://www.momentumtrials.comand for more on the company, please visit http://www.aruvant.com. Follow Aruvant on Facebook, Twitter @AruvantSciencesand on Instagram @Aruvant_Sciences.

About Roivant Roivant's mission is to improve the delivery of healthcare to patients by treating every inefficiency as an opportunity. Roivant develops transformative medicines faster by building technologies and developing talent in creative ways, leveraging the Roivant platform to launch Vants nimble and focused biopharmaceutical and health technology companies. For more information, please visit http://www.roivant.com.

SOURCE Aruvant Sciences andJasper Therapeutics

Home

See original here:
Jasper Therapeutics and Aruvant Announce Research Collaboration to Study JSP191, an Antibody-Based Conditioning Agent, with ARU-1801, a Novel Gene...

Bone Marrow Stem Cells Comments Off on Jasper Therapeutics and Aruvant Announce Research Collaboration to Study JSP191, an Antibody-Based Conditioning Agent, with ARU-1801, a Novel Gene… | June 25th, 2021

Actinium Pharmaceuticals Inc. (NYSE: ATNM), a clinical-stage biopharmaceutical company, is developing antibody radiation-conjugates (ARCs) to combine the targeting ability of antibodies with the cell-killing ability of radiation. The Company is a leader in the targeted radiotherapy field for cancer patients who cant tolerate chemotherapy and radiation. Actiniums lead asset, Iomab-B, is currently being studied in a pivotal Phase 3 clinical trial.

Standing out in the Field of Target Conditioning

What makes Actinium unique is in its novel approach to treatment options for cancer patients. According to the National Cancer Institute (NCI) a conditioning regimen may include chemotherapy, monoclonal antibody therapy and radiation to the entire body. It supports the patient's body to make room in the bone marrow for new blood stem cells to grow, helps prevent the body from rejecting the transplanted cells and assists with killing any cancerous cells. Actiniums targeted radiotherapies are intended to be focused missiles that hit cancer directly as opposed to a broader chemoradiation therapy that can hit many other areas that do not need to be attacked with such harsh treatments.

Among its competitors, Actinium remains the only company with a pivotal Phase 3 trial for a targeting conditioning agent and the only anti-CD45 ARC in clinical development.

Multiphase Clinical Trials and the Success of Iomab-B

In the ongoing Phase 3 SIERRA trial, Actiniums lead asset lomab-B acts as an induction and conditioning agent in patients over the age of 55 with relapsed or refractory acute myeloid leukemia (AML) prior to receiving a bone marrow transplant, also known as a hematopoietic stem cell transplant.

This multicenter trial is being conducted at over 20 leading transplant centers in the U.S., including MD Anderson, Memorial Sloan Kettering and Mayo Clinic.

Of all patients who received a therapeutic dose of Iomab-B, 100% proceeded to bone marrow transplant and engrafted, which is the first sign of success in contrast to the control arm, where only 18% of patients were able to go to transplant and engraft. Its a clear, marked difference, commented Actinium CFO Steve O'Loughlin.

Story continues

Additionally, Iomab-B was very well-tolerated. Minimal adverse effects and minimal nonrelapse transplant mortality were reported compared to the control arm, OLoughlin concluded.

In addition to Iomab-B, Actiniums drug development pipeline features Iomab-ACT, a lower dose of Iomab-B that is being studied for target conditioning in advance of CAR-T, a form of cellular therapy that weaponizes patients immune cells to attack and kill their cancer. Actinium is collaborating with Sloan Kettering to study Iomab-ACT with the institutes CD19 CAR-T therapy 19-28z in a Phase 1 trial in patients with relapsed or refractory leukemia. Actinium and Sloan Kettering have been jointly awarded grant funding from the National Institute of Health via its STTR Fast Track program.

Actiniums other clinical program, Actimab-A, which has been studied in a Phase 2 clinical trial, is now being studied in two Phase 1 combination trials: one with the salvage chemotherapy regimen CLAG-M and the other with Ventoclax, a targeted therapy jointly developed and marketed by AbbVie and Roche. Actinium is focused on continuing to expand its drug development pipeline by leveraging its Antibody Warhead Enabling (AWE) technology platform.

The AWE Technology Platform

Actinium is the leader in Ac-225-based therapies, the most powerful medical-grade radioisotope. This is a result of the Companys clinical experience, technology, intellectual property and know-how. The clinical experience encompasses over 500 patients who have been treated with Actiniums ARCs and through its clinical trials.

Actinium's AWE technology platform is used to produce ARCs, a highly potent and selective form of targeted radiotherapy. ARCs enable the precision targeting of radiation to tumors and its synergistic potential with other therapeutic modalities that cannot be matched by traditional external beam radiation, cytotoxic chemotherapy or biologic therapies.

AWE-enabled ARCs exploit the use of highly-selective targeted biological agents such as monoclonal antibodies that can seek out and bind cancer antigens found on the tumor cell surface. They deliver potent radioisotopes that are capable of producing double-strand DNA breaks for which there are currently no known resistance or repair mechanisms.

Actinium announced a collaborative research partnership with Astellas Pharma in 2018 to leverage Actiniums AWE technology platform with select Astellas targeting agents. In 2021, Astellas announced this collaboration will be focused on leveraging its select targeting agents to both image and diagnose cancers. The goal is to treat patients with Actiniums AWE technology platform using the Ac-225 radioisotope warhead.

2021 and Beyond

In 2020, Actinium became a fully-integrated, targeted radiotherapy development company by securing laboratory facilities in New York City. These new research facilities function under the guidance of Dale Ludwig, Ph.D., the Company's chief scientific and technology officer, who has over 25 years of oncology discovery research and development experience.

Currently, the SIERRA trial is being conducted at preeminent transplant centers in the U.S., and the Company has begun patient enrollment in the Phase I study of Iomab-ACT for targeted conditioning before treatment in collaboration with Memorial Sloan Kettering Cancer Center. Additionally, Actinium completed enrollment of a second dose cohort in its Actimab-A Venetoclax combination trial for patients with R/R AML, making this a very exciting year for the Company.

Actinium has an IP portfolio of over 140 patents. As of March 31, 2021, the Company had a cash balance of $72 million and as of May 18, 2021, it had a market cap of approximately $156 million. Visit https://www.actiniumpharma.com/ for current news and more information.

Actinium is a partner of Benzinga. The information in this article does not represent the investment advice of Benzinga or its writers.

See more from Benzinga

2021 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

Link:
Actinium Activates Radiation Inside the Body for Target Conditioning of Cancer Cells - Yahoo Finance

Bone Marrow Stem Cells Comments Off on Actinium Activates Radiation Inside the Body for Target Conditioning of Cancer Cells – Yahoo Finance | June 8th, 2021

Global Stem Cell Manufacturing Market: Overview

Stem cells refer to special cells created by bone marrow of an individual. The key specialty of these cells is their ability to turn into various types of blood cells. Stem cells are gaining immense impetus owing to their key role in effectual disease management and specialized research activities including genomic testing and personalized medicine. Owing to these factors, the global stem cell manufacturing market is likely to register promising growth trajectory throughout the forecast period 20202030.

Get Exclusive PDF Sample Copy Of This Report:@ https://www.tmrresearch.com/sample/sample?flag=B&rep_id=7465

The present research report performs segmentation of all the data gathered from the global stem cell manufacturing market into different sections. This segmentation is carried out based on many crucial parameters such application, product, end user, and region. Based on product, the market for stem cell manufacturing is classified into consumables, stem cell lines, and instruments.

Global Stem Cell Manufacturing Market: Growth Dynamics

Stem cells are used for various purposes such as clinical application, research applications, and cell and tissue banking applications. Thus, increased demand for the product from various end users including academic institutes, pharmaceutical and biotechnology companies, hospitals and surgical centers, cell and tissue banks, and research laboratories and contract research organizations is likely to generate lucrative avenues for vendors in the global stem cell manufacturing market in the years ahead.

Over the period of past few years, there is extensive growth in awareness pertaining to the therapeutic effectiveness of stem cells. This factor is working in favor of the expansion of the global stem cell manufacturing market. Owing to the restricted therapeutic treatment options for orphan diseases, there is considerable growth in investments toward stem cell-based technologies development by private as well as public stakeholders. This scenario is expected to help in rapid growth of the global stem cell manufacturing market in the years to come.

To get Incredible Discounts on this Report, Click Here @https://www.tmrresearch.com/sample/sample?flag=D&rep_id=7465

Global Stem Cell Manufacturing Market: Competitive Analysis

Owing to presence of many active players, the nature of stem cell manufacturing market seems to be highly fragmented. It also denotes the high level of competition in the market for stem cell manufacturing. Thus to sustain in this high competitive scenario, enterprises are executing different strategic moves including collaborations, partnerships, mergers, acquisitions, agreements, joint ventures, and new product launches. Apart from growing financial support toward research and development activities, many players working in the stem cell manufacturing market are strengthening their production capabilities. On the back of all these moves, we can conclude that the global stem cell manufacturing market will expand at moderate pace throughout the assessment period 20202030.

Global Stem Cell Manufacturing Market: Notable Development

In May 2021, University of California, San Francisco and Thermo Fisher Scientific entered into strategic alliance. The main motive of this alliance was to open cell therapy cGMP manufacturing and collaboration center.The list of important players in the global stem cell manufacturing market includes:

Merck MilliporeThermo Fisher ScientificDanaher CorporationLonza Group AGBio-Rad LaboratoriesSartorius AGStemcell TechnologiesMiltenyi BiotecFujifilm Holdings CorporationCellgenix GMBHGlobal Stem Cell Manufacturing Market: Regional Assessment

In terms of region, the global stem cell manufacturing market is spread across many regions such as Europe, North America, Latin America, the Middle East and Africa, and Asia Pacific. Of these regions, North America is one of the prominent regions in the market for stem cell manufacturing. Key factor supporting this growth include extensive research and development in the region together with increased financial support by government as well as no-government organizations for the study of stem cell applications.

To know more about the table of contents, you can click @https://www.tmrresearch.com/sample/sample?flag=T&rep_id=7465

About TMR Research:

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

Contact:TMR Research,3739 Balboa St # 1097,San Francisco, CA 94121United StatesTel: +1-415-520-1050

Browse More Related Reports :

https://telegra.ph/PEGylated-Proteins-Market-to-Partake-Significant-Development-During-2025-05-31https://www.debwan.com/blogs/89779/U-S-Clinical-Oncology-Next-Generation-Sequencing-NGS-Market-Tohttps://www.homify.co.uk/ideabooks/7964765/whole-exome-sequencing-market-to-witness-a-pronounce-growth-during-2025

The rest is here:
Stem Cell Manufacturing Market Global Industry Analysis , Scope, Opportunity and Forecast 2020 to 2030 The Courier - The Courier

Bone Marrow Stem Cells Comments Off on Stem Cell Manufacturing Market Global Industry Analysis , Scope, Opportunity and Forecast 2020 to 2030 The Courier – The Courier | June 8th, 2021

Global Hematopoietic Stem Cell Transplantation (HSCT) Market Size, Status And Forecast 2021-2028

MarketInsightsReports, a leading global market research firm, is pleased to announce its new report on Hematopoietic Stem Cell Transplantation (HSCT) market, forecast for 2021-2028, covering all aspects of the market and providing up-to-date data on current trends.

The report covers comprehensive data on emerging trends, market drivers, growth opportunities, and restraints that can change the market dynamics of the industry. It provides an in-depth analysis of the market segments which include products, applications, and competitor analysis. The report also includes a detailed study of key companies to provide insights into business strategies adopted by various players in order to sustain competition in this highly competitive environment.

(Special Offer: Available Flat 30% Discount for a limited time only):Get a Free Sample Copy of the Report:https://www.marketinsightsreports.com/reports/06022956528/2016-2028-global-hematopoietic-stem-cell-transplantation-hsct-industry-market-research-report-segment-by-player-type-application-marketing-channel-and-region/inquiry?mode=dj

With our Hematopoietic Stem Cell Transplantation (HSCT) market research reports, we offer a comprehensive overview of this sector and its dynamics. We have done extensive research on this topic and are confident that our findings will be helpful for anyone who needs some guidance or direction when making important decisions related to their companys future growth strategy.

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

This report segments the global Hematopoietic Stem Cell Transplantation (HSCT) market on the basis of Types are:

On the basis of Application, the Global Hematopoietic Stem Cell Transplantation (HSCT) market is segmented into:

For comprehensive understanding of market dynamics, the global Hematopoietic Stem Cell Transplantation (HSCT) market is analyzed across key geographies namely: United States, China, Europe, Japan, South-east Asia, India and others. Each of these regions is analyzed on basis of market findings across major countries in these regions for a macro-level understanding of the market.

Avail Discount at:

https://www.marketinsightsreports.com/reports/06022956528/2016-2028-global-hematopoietic-stem-cell-transplantation-hsct-industry-market-research-report-segment-by-player-type-application-marketing-channel-and-region/discount?mode=dj

Key Takeaways from Hematopoietic Stem Cell Transplantation (HSCT) Report

Browse the report description and TOC: https://www.marketinsightsreports.com/reports/06022956528/2016-2028-global-hematopoietic-stem-cell-transplantation-hsct-industry-market-research-report-segment-by-player-type-application-marketing-channel-and-region?mode=dj

-Key Strategic Developments: The study also includes the key strategic developments of the market, comprising R&D, new product launch, M&A, agreements, collaborations, partnerships, joint ventures, and regional growth of the leading competitors operating in the market on a global and regional scale.

-Key Market Features: The report evaluates key market features, including revenue, price, capacity, capacity utilization rate, gross, production, production rate, consumption, import/export, supply/demand, cost, market share, CAGR, and gross margin. In addition, the study offers a comprehensive study of the key market dynamics and their latest trends, along with pertinent market segments and sub-segments.

-Analytical Tools: The Global Hematopoietic Stem Cell Transplantation (HSCT) Market report includes the accurately studied and assessed data of the key industry players and their scope in the market by means of a number of analytical tools. The analytical tools such as Porters five forces analysis, SWOT analysis, feasibility study, and investment return analysis have been used to analyze the growth of the key players operating in the market.

Customization of the Report: This report can be customized as per your needs for additional data up to 3 companies or countries or 40 analyst hours.

MarketInsightsReports provides syndicated market research on industry verticals including Healthcare, Information and Communication Technology (ICT), Technology and Media, Chemicals, Materials, Energy, Heavy Industry, etc.MarketInsightsReports provides global and regional market intelligence coverage, a 360-degree market view which includes statistical forecasts, competitive landscape, detailed segmentation, key trends, and strategic recommendations.

How we have factored the effect of Covid-19 in our report:

All the reports that we list have been tracking the impact of COVID-19. Both upstream and downstream of the entire supply chain has been accounted for while doing this. Also, where possible, we will provide an additional COVID-19 update supplement/report to the report in Q3, please check for with the sales team.

IrfanTamboli (Head of Sales) Market Insights Reports

Phone: + 1704 266 3234 | +91-750-707-8687

sales@marketinsightsreports.com | irfan@marketinsightsreports.com

More:
Hematopoietic Stem Cell Transplantation (HSCT) Market Competitive Analysis with Global Trends and Demand 2021 to 2028:ViaCord Inc, Cryo-Save AG, CBR...

Bone Marrow Stem Cells Comments Off on Hematopoietic Stem Cell Transplantation (HSCT) Market Competitive Analysis with Global Trends and Demand 2021 to 2028:ViaCord Inc, Cryo-Save AG, CBR… | June 8th, 2021

Brave Nathaniel Nabena smiles from his hospital bed moments before a life-saving procedure.

The nine-year-old had a vital stem cell transplant at Great Ormond Street Hospital on Wednesday after Sunday People readers helped raised more than 215,000.

Nathaniel, battling acute myeloid leukaemia, was on a drip for 30 minutes as umbilical cord stem cells were fed into his body.

Afterwards, dad Ebi said: Nathaniel is very happy. It was amazing to finally get to this point we have all been waiting for.

The youngster was admitted a fortnight ago and had five doses of chemo over ten days to prepare him for the procedure.

How brave has Nathaniel been? Have your say in comments below

Mum Modupe, 38, was able to spend time with him before his transplant.

Consultants warn he faces weeks of sickness as his body reacts to the new cells with symptoms including vomiting and a fever.

Ebi, 45, said: His doctors hope to see improvements after five weeks. It is so hard to see him so exhausted but I dont have a choice. We are grateful to have this done. Our fingers are crossed to see what happens.

For now, Nathaniel has a compromised immune system and is susceptible to falling ill, so he will be staying on the ward.

Stars including Simon Cowell, David Walliams, Katie Price and JLS singer Aston Merrygold rallied to support him after we told of the desperate race to fund treatment.

Nathaniels left eye was removed in his home country of Nigeria a year ago, due to myeloid sarcoma cancer. He was diagnosed with AML in the UK in November after coming here to have a prosthetic eye fitted.

Nathaniel was told a stem-cell transplant was his only hope for survival but it would cost 201,000 as he is not a British citizen. Ebi and Modupe were initially told it could cost as much as 825,000 but the figure was revised after doctors waived their fees and offered to treat him in their own time.

The Mirror's newsletter brings you the latest news, exciting showbiz and TV stories, sport updates and essential political information.

The newsletter is emailed out first thing every morning, at 12noon and every evening.

Never miss a moment by signing up to our newsletter here.

The lad was admitted to GOSH on May 24 after generous Brits rushed to help the family raise cash.

Business analyst Ebi, who is staying at the hospitals family quarters, said: Ive been there the whole time. When he is not sleeping he is passing the time playing his games.

We sometimes talk about when he gets better and how exciting that will be. This is a difficult thing for him to go through, but Nathaniel is being brave, he is well in himself.

In acute myeloid leukaemia, unhealthy blood-forming stem cells grow quickly in the bone marrow.

This prevents it from making normal red blood cells, white blood cells and platelets meaning the body cannot fight infections or stop bleeding.

A stem cell transplant, also known as a bone marrow transplant, can help AML patients stimulate new bone marrow growth and restore the immune system.

Before treatment, patients need high doses of chemo and sometimes radiotherapy.

This destroys existing cancer and bone marrow cells and stops the immune system working, to cut the risk of transplant rejection.

In an allogeneic transplant, stem cells are taken from a family member, unrelated donor or umbilical cord blood. In Nathaniels case, it was from a cord.

They are then passed into the patients body through a line inserted in a large, central vein, in a process that takes up to two hours.

You can also remove stem cells from the patients body and transplant them later, after any damaged or diseased cells have been removed this is called an autologous transplant.

The survival rate after a transplant for patients with acute leukaemia in remission and using related donors is 55% to 68%, according to Medicine Net. If the donor is unrelated, it is 26% to 50%.

See original here:
Brave Nathaniel Nabena, 9, all smiles as he has life-saving procedure - thanks to you - The Mirror

Bone Marrow Stem Cells Comments Off on Brave Nathaniel Nabena, 9, all smiles as he has life-saving procedure – thanks to you – The Mirror | June 8th, 2021

Get inside Wall Street with StreetInsider Premium. Claim your 1-week free trial here.

BioRestorative Therapies invites individual and institutional investors, as well as advisors and analysts, to attend its real-time, interactive presentation at the online Emerging Growth Conference.

MELVILLE, N.Y., June 07, 2021 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (the Company or BioRestorative) (OTC: BRTX), a life sciences company focused on stem cell-based therapies, is pleased to announce that it is has been invited to present at the online Emerging Growth Conference on June 9, 2021.

The Emerging Growth Conference will be held on June 9, 2021. This live, interactive online event will give existing shareholders and the investment community the opportunity to interact with the Companys CEO, Lance Alstodt, and Vice President of Research and Development, Francisco Silva, in real time.

Mr. Alstodt will make a presentation and answer questions. Please ask your questions during the event and Mr. Alstodt will try to respond to as many as possible.

BioRestorative Therapies will be presenting at 10:45 AM Eastern time for 45 minutes.

Please register here to ensure you are able to attend the conference and receive any updates that are released:

https://goto.webcasts.com/starthere.jsp?ei=1469230&tp_key=f8b5116237&sti=brtx

If attendees are unable to join the event live on the day of the conference, an archived webcast will also be made available on EmergingGrowth.com, and the Company will also release a link to that site after the event.

About the Emerging Growth Conference

The Emerging Growth Conference is an effective way for public companies to present and communicate their new products, services and other major announcements to the investment community from the convenience of their office, in a time efficient manner.

The Conferences focus and coverage includes companies in a wide range of growth sectors, with strong management teams, innovative products and services, focused strategy, execution, and the overall potential for long term growth. Its audience includes potentially tens of thousands of individuals and institutional investors, as well as investment advisors and analysts.

All sessions will be conducted through video webcasts and will take place in the Eastern time zone.

About BioRestorative Therapies, Inc.

BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:

Disc/Spine Program (brtxDISC): Our lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders or as a complementary therapeutic to a surgical procedure. The BRTX-100 production process utilizes proprietary technology and involves collecting a patients bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patients damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. We have received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat chronic lower back pain arising from degenerative disc disease.

Metabolic Program (ThermoStem): We are developing a cell-based therapy candidate to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (BAT). BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in animals may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes.

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, those set forth in the Company's latest Form 10-K filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. Any forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.

CONTACT:Email: ir@biorestorative.com

See the article here:
BioRestorative Therapies to Present at the Emerging Growth Conference on June 9, 2021 - StreetInsider.com

Bone Marrow Stem Cells Comments Off on BioRestorative Therapies to Present at the Emerging Growth Conference on June 9, 2021 – StreetInsider.com | June 8th, 2021

The investment in bolstering defences in virtual space also remains a top priority, as the pharmaceutical industry is extremely susceptible to cyber-attacks due to the involvement of sensitive and valuable data.

Several pharmaceutical companies and research institutes including Hammersmith Medicines Research in the UK, the University of California, San Francisco (UCSF), and US-based clinical services company eResearch Technology (ERT) remained targets for cyberattacks due to their involvement in the development of COVID-19 vaccines.

GlobalData conducted to survey to assess to extent to which emerging technologies such as cybersecurity will help a company survive through the Covid-19 pandemic.

Analysis of the results found that 54% of the respondents opined that cybersecurity would play a significant role in helping companies to pull through the crisis created by the pandemic.

Cybersecuritys Role During COVID-19 Crisis

Another 33% of the surveyed companies expect cybersecurity to play a minor role during the COVID-19 crisis.

Further, 10% of the companies stated that cybersecurity will play no role during the pandemic, while 3% of the respondents were unaware of the impact of cybersecurity.

The analysis is based on responses received in GlobalData, Emerging Technologies Survey 2020 fielded between 29 May and 09 July 2020.

Customised Viral Vectors for Cell Modelling, Gene Therapy, and Vaccination Research and Development

28 Aug 2020

Pharmaceutical-Grade Water Purification Systems for the Pharmaceutical and Biopharma Markets

28 Aug 2020

Original post:
Regenerative medicine: moving next-gen treatments from lab to clinic - Pharmaceutical Technology

Bone Marrow Stem Cells Comments Off on Regenerative medicine: moving next-gen treatments from lab to clinic – Pharmaceutical Technology | June 8th, 2021

World Blood Cancer Day is marked today. The celebration of this day was instituted in 2014 because, in 1991, Methchild Ehringer could not find a match by a German non-profit organisation DKMS founded by Methchilds family. She died because she was unable to find a match. The aim was and still remains to find a potential donor for every person needy of a bone marrow/stem cell transplant.

Today, there are 10 million potential donors registered when compared to the initial 3,000. Some might remember me as the person who needs Daratumumab included in the government formulary for free medicinals. I am interested in other matters too!

World Blood Cancer Day! This day highlights a more personal issue that concerns the realisation that I do not stand alone in my blood cancer journey. An issue that cuts across the three most common blood cancers: leukaemia, lymphoma and myeloma. Of these three, the lymphomas and the myelomas are the more common. For many people, such as myself, the diagnosis of a blood cancer is a shock. Like water, blood is meant to sustain a person.

Blood cancers bring along with them a lot of uncertainty and anxiety. For some blood cancers, such as multiple myeloma, a cure does not exist. To suddenly acknowledge that what is being generated in your bone marrow and what is circulating around your body and through your own blood is threatening you and your body, is psychologically invasive in a way like no other. I felt robbed. Robbed by my own body, my own immune system and, perhaps, by my own past lifestyle choices.

Stem cell transplantation offers hope of increased longevity, when appropriate, to a good proportion of blood cancer patients. A successful stem cell transplant means time out of hospital, visiting usually only every few months for monitoring. There are two types of stem cell transplantation: autologous and allogenic.

Autologous transplantation is when a person donates to oneself. As was the case with myself and my two attempts for autologous transplantation.

For many people, such as myself, the diagnosis of a blood cancer is a shock

Allogenic requires matching a donor to a patient. This is only suitable in specific cases. The government of Malta does pay for such transplantation. Charities such as Puttinu are incredibly supportive in supporting those undergoing stem cell transplantation by providing accommodation. On World Blood Cancer Day 2021, my wish is that the Maltese public understand what I consider to be three critical issues.

First, for many, stem cell/bone marrow transplants and, increasingly, cellular innovative therapies, potentially require a donor. Second, for a portion of those requiring such intervention/therapy they cannot donate to themselves and/or find a donor from their family. Third, millions of people are required to donate their stem cells.

As of today, I am under the impression that Malta and its generally very good healthcare system does not yet, have a register for stem cell donation. I hope I am wrong. Should I be right, I urge the powers that be to strongly consider this as part of their long-term strategic vision. What I do know, however, is that Malta has the local expertise and the equipment to collect stem cell transplants.

Perhaps because it is a small-island state, Malta does not have the facility for storage. The healthcare system is probably likely, especially at this point in time, not to possess as much capacity to assure consistent and sustained storage of stem cells according to European and international gold-standard criterion. This is likely to be primarily due to space issues given that the local expertise is available and excellent.

In more recent times especially, monoclonal agents, such as Daratumumab, are increasingly offered as more frontline treatment to those with an early diagnosis of multiple myeloma, at least in other EU countries and to those able to afford payment.

Chimeric Antigen therapy (CAR-T cell), which forms part of cellular therapeutic options, is also in the pipeline.

Monoclonal therapy and CAR-T cell are consequently likely to decrease the need for stem cell transplants. This is positive but, if anything, highlights even more the need for a stem cell database, register and repository. Newer therapies generally tend to be increasingly stem cell therapy dependent in some form or other.

What I would like the reader to appreciate is that I am not a medical professional. I have been at times accused of being a dreamer but life has taught me two things.

To turn lemons into lemonade and to give without the expectation of taking or receiving.

Today, I would like to go a bit beyond my myeloma, so to speak. I want to celebrate, as a blood cancer survivor, what works for me. I urge all of you to read about blood cancers and try to empathise with all blood cancer survivors.

Above all, let us not forget their carers: spouses, children, friends, doctors, nurses and everybody else whom I have inadvertently omitted.

I, for one, would not be here, especially, without the excellent care and patience of doctors, nurses, physiotherapists, etc. alongside the emotional care, motivation and interest offered by my sons, friends, work colleagues and those generally understanding and supportive of my condition, and, yes, my Lara still needs Dara quest! Thank you.

Lara Saids dream is to set up a non-profit organisation to advocate especially for the rights of patients with myeloma, leukaemia and lymphoma for Malta and Gozo. She is here appealing to survivors, their relatives and/or carers to help her set up a patient group.

Lara Said, Multiple myeloma survivor, member, Myeloma Patients Europe

Independent journalism costs money. Support Times of Malta for the price of a coffee.

View post:
Living with blood cancer - Lara Said - Times of Malta

Bone Marrow Stem Cells Comments Off on Living with blood cancer – Lara Said – Times of Malta | May 28th, 2021

Ifeyinwa Osunkwo, MD, MPH: Pat, can you describe the steps people go through to do a bone marrow transplant to gene therapy? Set the stage to help people understand why busulfan and why were talking about mutations. Can you walk us through the whole gene therapy process?

Patrick McGann, MD, MS: With transplant or gene therapy, the term transplant could be autologous, meaning your own cell gene therapy transplant. Its not as if youre transplanting a solid organ or a kidney. Sometimes patients get confused about this. Its looks like a blood transfusion hanging when it eventually goes in. For a bone marrow transplant, the donorwhoever that iseither gets a bone marrow aspiration, where they get bone marrow cells taken from their bone marrow, or a medicine to rev up their blood cells and get blood taken just from their vein. The patient needs to get prepared because they need to get rid of all their sickle cells. They need to suppress their immune system, so they dont reject this foreign cell, which is someone elses.

We use strong chemotherapy. If you have leukemia, as a comparison, you need to kill every last leukemia cell, and you get blasted with really strong chemotherapy agents and strong immunosuppressive agents. This is usually a week or so before; the days are counted backward. The cells that go in your body are most vulnerable to infection to everything. Its a dangerous time. Thats when complications come in. If its a transplant, you get infused with that donors bone marrow cells and hope it takes. It takes some weeks time for your body to take those new cells, and youre often receiving antibiotics and getting transfused and sustaining it, because your bone marrow is still not working. Basically, your immune system is suppressed. Its a tough time.

Transplant conditioning, as this regimen is called, has gotten a little less toxicreduced conditioning is the term. But thats still serious conditioning. Even though its reduced from what it used to be, its a relative term. Gene therapy is a little different because youre giving back your own cell. The way gene therapy happens is its ex vivo, meaning they take it out of your body. There are different ways that this is being done. Many patients need to have a bone marrow aspiration or many procedures to take enough cells out of their bone marrow to take them to the lab to fix them. There have been new ways to do this with peripheral blood and a medication called plerixafor, which is much better than going to the operating room for these horrible procedures.

Those cells are then taken to the lab and edited, or whatever the mechanism of gene therapy is. You still need to ablate your bone marrow to get rid of all your sickle cells. Because if you have any or many sickle cells in there, when you give back your edited gene cells, those will just take over. You still need to suppress that bone marrow. Because its your own cells, the immune suppression isnt as much of a problem as it is with transplant. Still, its a week of pretty serious medicinebusulfan, traditionallyand youre in the hospital for less of a period of time than transplant. Its quite an ordeal. Similarly, it takes or doesnt, and you monitor over time if that gene therapy has worked and whether its sustainablecross your fingersin the long term.

Ifeyinwa Osunkwo, MD, MPH: Basically, you have 2 options. The first option, you have to kill off their own bone marrow cells using chemotherapy. Then you give them somebody elses bone marrow, like a blood transfusion. The stem cells from the other person finds its way into their bone marrow and then grows. Then you wait and see what happens. Do you fight it? Do you accept it? We know if it takes or not. For gene therapy, we take out the patient stem cells, take it to a laboratory. Its usually in New Jerseydont ask me why. They manipulate it to pick out the gene they dont want. Then they give that patient back their own modified stem cells and wait for it to grow. But you still have to wipe out that persons bone marrow, so you dont have this fight going on. Even though theyre your cells, theyre a little different with the new gene change that has been made. Its a complicated process, and its really the only way to cure your disease. Either stem cell or gene therapy. We have had some setbacks in the past and more recently, but I believe that science is going to prevail. Over time were going to get to the point where we figure out the way to do this in the safest way to make it available to the most people with sickle cell disease and other blood disorders.

Thank you so much for watching this HCPLive Peer Exchange. If you enjoyed the content, please subscribe to the e-newsletter to receive upcoming Peer Exchanges and other great content right in your in-box.

Transcript Edited for Clarity

View post:
Stem Cell vs Gene Therapy Processes in SCD - MD Magazine

Bone Marrow Stem Cells Comments Off on Stem Cell vs Gene Therapy Processes in SCD – MD Magazine | May 28th, 2021

Fighter: A year long battle but Rory is still smiling

In January 10-year-old Aurora Pile-Grays family were told they may lose their little girl as a rare and aggressive cancer took its devastating toll.

The previous November Aurora affectionately known as Rory had been declared in remission from the disease that she had been fighting since May 2020 but a cruel blow saw a severe relapse with the cancer progressing to her skull, eyes, neck, spine, liver, kidneys, lungs, abdomen and pelvis.

A discussion with the consultant over going home from hospital and preparing for end of life care took place but lion-hearted Rory was not ready to give up her fight against Burkitts Lymphoma which affects blood and bone marrow and her family were not ready to let go.

After a year-long battle with the disease, seven rounds of intensive chemotherapy, stays in Royal Marsden and Great Ormond Street hospitals and targeted therapy with trial drug Inotuzumab, Aurora was declared cancer free again at the end of April.

The Inotuzumab drug also offered the family, who live in Garlinge, a chance for Aurora to be at home despite treatment.

Mum Keisha, 28, said: We chose Inotuzumab because it meant a better quality of life for Rory, she could come home to us and her brother and sister rather than having to be in hospital.

The trial drug, and cancer all clear, opened the way for the youngster to undergo a bone marrow transplant on May 13 all the more vital as chemo had wiped out her immune system.

First there were 10 radiotherapy sessions to get through involving cranial boost, where Auroras face was bolted to the bed so she couldnt move.

The transplant which is universally referred to as a new birthday to signal a second chance at life took place at the Marsden and involved replacing old bone marrow cells that are failing to produce healthy new cells, with cells from a donor. The aim is to create a new immune system and hopefully prevent cancer returning or mutations occurring.

The stem cells Aurora now carries should begin to reproduce in her own body and allow her bone marrow to work as normal in producing healthy red cells, white cells and platelets, since her body is no longer capable of doing so after the effects of both the cancer and the treatment.

Auroras donor cells were frozen in December which meant on the day they had to be defrosted and infused within 20 minutes. Auroras donor produced nine bags of stem cells. Only four were required, leaving 5 for future use if needed.

Each bag is defrosted in a water bath around 38 degrees so that by the time its infused its not too different to body temperature. They are then put into separate syringes and pushed through a central line into the body.

The family have been told there is just a 10-20% chance that the transplant will achieve long lasting remission, but the St Crispins school youngster has so far beaten the odds and mum Keisha says her little girl is a fighter.

In her blog Growing Pains and Paper Planes, Keisha says: Shes been amazing throughout this entire journey and Im unbelievably proud to call her my daughter. Im in awe of her strength, determination and resilience and shes shown us all that sometimes the smallest hearts overcome the biggest battles.

So far Aurora has responded well to the transplant and there is evidence of engraftment where the donor cells find their way to her bone marrow and begin to make new blood cells. White cells are the first to engraft which include neutrophils, then red cells and then platelets.

Keisha said: We were worried the effects would be awful as Rory was so sensitive to the chemo but it is going really well.

Rory is up every day, listening to audio books and doing lots of colouring, cracking jokes and just being Rory.

It is such a relief. In January when we realised the cancer had spread and she could lose the use of her legs, bladder and bowel we had to talk about making her comfortable at home. But we were not ready to give up. I said it wasnt that time yet, not all the time that Rory was laughing and joking and looking forward to seeing her brother and sister. She wasnt giving up and we werent.

Now there has been this complete 360 turnaround. Everything still depends on how she takes to the transplant. On day 28 a sample will hopefully show what percentage are donor (cells) and what percentage are her own. Hopefully it will show primarily donor.

Rory is on immunosuppressants for up to a year but potentially could be able to return to school in six months time.

Keisha said: Shell go back as a Year 6 and has missed two years but has been doing schoolwork for an hour each day.

She is looking forward to it, more the social side than the work!

Rory has won the hearts of the Thanet community during her battle with cancer with many following the familys progress through Keishas blog and also donating to a fundraiser for life-saving treatment. People also signed up on the bone marrow register following Keishas highlighting of the desperate need for donors, especially people of mixed ethnicity.

Keisha said: We are unbelievably grateful and humbled by the support people have shown us over the past year. The kind words, the gestures, the gifts, the donations, the shares, the marrow registrations and the sense of community has helped us more than you will ever know.

Read more from the original source:
'Lion-hearted' ten-year-old Aurora's 'second chance' at life after cancer remission and stem cell transplant - The Isle of Thanet News

Bone Marrow Stem Cells Comments Off on ‘Lion-hearted’ ten-year-old Aurora’s ‘second chance’ at life after cancer remission and stem cell transplant – The Isle of Thanet News | May 28th, 2021

Chronic lymphocytic leukemia (CLL) is a type of blood cancer. It affects lymphocytes, a type of immature white blood cell that the body produces in the bone marrow.

People with CLL may experience discomfort, swelling, and pain in the abdomen if their spleen enlarges. In rare cases, CLL can also affect mucosal tissues, such as those lining the gastrointestinal (GI) tract.

This article discusses the link between CLL and stomach pain in more detail. It also looks at ways to prevent and treat CLL, the other possible symptoms, and the outlook for people with this condition.

People with CLL may experience various symptoms involving the abdomen or stomach, such as:

In most cases, people with CLL do not experience abdominal symptoms due to the disease until it progresses and becomes more severe.

Research suggests that CLL infiltrates and affects the GI tract in about 5.713% of cases. When CLL affects the GI tract, doctors may call it Richters syndrome.

People with CLL may experience abdominal swelling, discomfort, and tenderness as a result of their spleen becoming larger. Less commonly, they may also feel full after eating small amounts of food, as the spleen can press on the stomach, making it smaller and able to hold less.

In rare cases, CLL infiltrates the lining of the GI tract, causing inflammation and ulcers or open wounds. People may experience symptoms similar to those of inflammatory bowel disease (IBD) and malabsorption disorders. These symptoms may include diarrhea, nausea, vomiting, abdominal pain and cramping, and unintentional weight loss.

CLL is cancer that develops in lymphocytes, which are white blood cells that form in the bone marrow and help fight infection.

Lymphocytes make up most of the lymph tissues in the lymph nodes, thymus gland, adenoids, tonsils, and spleen. They are also present in the GI tract, bone marrow, and respiratory system.

CLL is a type of leukemia that develops gradually over time.

About 5075% of people with CLL do not experience noticeable symptoms. Due to this, doctors diagnose most people with CLL during routine blood work.

The symptoms of CLL often begin when the cancerous cells crowd out healthy cells in the bone marrow or migrate to other organs or tissues. When symptoms first appear, they are typically mild, but they then become increasingly severe. CLL can cause many nonspecific symptoms, so a person may feel as though they have a cold or the flu.

Possible symptoms of CLL that do not relate to the abdominal area include:

Doctors do not yet have a way to prevent leukemia. However, some types of leukemia, including CLL, may have links to toxins, such as herbicides, pesticides, radon, and tobacco exposure. People can help reduce the risk of CLL by avoiding or practicing extreme caution around these toxins.

Many people do not experience symptoms of CLL for years and do not require treatment. However, as the disease progresses, these individuals may need treatment to extend their lives.

When and how a doctor treats someones CLL depends on a few factors, including:

Chemotherapy is typically the first-line therapy for CLL.

Doctors may use chemotherapy in conjunction with other treatment options, such as monoclonal antibody therapy. This therapy binds antibodies to cancer cells and destroys them. Treatment can also include medications to treat or prevent infections or improve low blood cell levels.

For instance, some people may take a combination of the monoclonal antibody rituximab and the chemotherapy drugs fludarabine and cyclophosphamide. Alongside the oral chemotherapy medication chlorambucil, doctors use obinutuzumab or ofatumumab, which have the same drug target as rituximab.

Small molecule inhibitors, such as bendamustine hydrochloride, idelalisib, and ibrutinib, can also sometimes form part of a CLL treatment regimen.

In 2017, the Food and Drug Administration (FDA) approved the combination medication Rituxan Hyecela (rituximab and hyaluronidase human) for CLL treatment.

In recurring or aggressive cases of CLL, an individual may have a blood or bone marrow stem cell transplant. This procedure replaces diseased cells with healthy blood cells that are able to mature into bone marrow cells.

Doctors may treat CLL until the symptoms lessen and then stop treatment until the symptoms worsen again.

Many people with CLL live for many years with a high quality of life.

There is no cure for CLL, so treatment aims to extend and improve someones life by reducing their symptoms. Doctors treat many people intermittently as their symptoms reoccur.

A persons outlook depends on their age, overall health, underlying conditions, and stage of CLL. Typically, people who are over the age of 65 years or have a more advanced stage of CLL have a less positive outlook.

Genetic changes in CLL cells and increased beta-2 microglobulin protein levels in the blood can make CLL more challenging to treat, potentially affecting a persons outlook.

Doctors classify people with CLL into different risk groups depending on certain factors. Based on these risk groups, the estimated percentages of people surviving 5 years or more after their diagnosis is:

People with more advanced or severe CLL may experience abdominal swelling, discomfort, tenderness, and pain. They may also feel full after eating small amounts. More rarely, someone with CLL may develop GI tract inflammation or ulcers, which can cause symptoms such as diarrhea, nausea, vomiting, cramping, and unexplained weight loss.

Anyone who thinks that they may have CLL should speak with a doctor. People with a confirmed diagnosis who experience symptoms of more advanced or severe CLL, such as abdominal pain, should also seek medical care.

See the original post here:
Chronic lymphocytic leukemia and stomach pain: What is the link? - Medical News Today

Bone Marrow Stem Cells Comments Off on Chronic lymphocytic leukemia and stomach pain: What is the link? – Medical News Today | May 28th, 2021

Magenta Therapeutics is losing its chief medical officer and head of R&D John Davis, M.D., with his last day coming July 30.

Davis helped steer the biotechs early path as well as deals with Avrobio and base editing biotech Beam Therapeutics around its lead stem cell conditioning program, MGTA-117, and MGTA-145 as a potentially new first-line standard of care for stem cell mobilization in a broad range of diseases. His departure comes three years after he joined the company from Pfizer, where he led its early R&D.

The biotech was keen to stress in an 8-K SEC filing (but not a press release) that his departure was not related to any disagreements with the Company on any matter relating to its operations, policies, practices or any issues regarding financial disclosures, accounting or legal matters.

It will now look for a new CMO, while Davis will become an adviser to the company.

RELATED: Magenta CSO Cooke jumps to IFM Therapeutics

This isnt the first move within the R&D ranks: Magenta started 2020 by losing its chief scientific officer when Michael Cooke, Ph.D., hopped over to IFM Therapeutics. In the fall, though, it nabbed Lisa Olson, Ph.D., who previously led immunology discovery at AbbVie, as his replacement.

Conditioning is a necessary step for some gene therapies, but one that can cause side effects like nausea, hair loss and mouth sores or make patients more vulnerable to infection. Magentas platform is based on looking to improve on current methods with an antibody-drug conjugate.

MGTA0117 is made up of an anti-CD117 antibody linked to amanitin, a cell-killing toxin. It is designed to target only hematopoietic, or blood-forming, stem cells and progenitor cells.

Animal studies suggest it could clear space in bone marrow for gene-modified stem cells to take root, Magenta said. The company plans to wrap IND-enabling studies for the antibody-drug conjugate this summer.

MGTA-145, meanwhile, just finished off a midstage test earlier this month, hitting its primary endpoint in a small multiple myeloma study.

Davis' replacement will already have a lot of clinical work on their hands.

Read more:
Magenta Therapeutics' research lead Davis hits the exit for family reasons - FierceBiotech

Bone Marrow Stem Cells Comments Off on Magenta Therapeutics’ research lead Davis hits the exit for family reasons – FierceBiotech | May 28th, 2021

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Moderna, Inc., (Nasdaq: MRNA) a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, today announced new research being highlighted as part of the Companys fourth annual Science Day. Modernas Science Day is designed to provide insight into the continued diverse efforts underway at Moderna to better understand how to use mRNA as medicines and vaccines and underscores the Companys continued commitment to basic science and innovation.

Science Day gives us an opportunity to provide insights into the advancements in our platform science and our further understanding of how to use mRNA as both a vaccine and a medicine. Our investments in basic science continue to result in major steps forward in our platforms capabilities, and these have allowed us to open new therapeutic areas and new scientific directions, said Stephen Hoge, M.D., President of Moderna. Our investments in platform research along with our digital backbone and manufacturing plants have enabled us to create first-in-class mRNA medicines and vaccines. Today, we're excited to highlight our work to identify and address SARS-CoV-2 variants of concern, optimize our proprietary lipid nanoparticle (LNP) technology, and deliver mRNA to hematopoietic stem cells.

Moderna currently has 24 mRNA development programs in its portfolio with 14 having entered clinical studies. The Companys updated pipeline can be found at http://www.modernatx.com/pipeline. Moderna and collaborators have published more than 65 peer-reviewed papers.

At this years Science Day, Moderna will present new platform science and preclinical research, including:

mRNA Delivery to Hematopoietic Stem and Progenitor Cells (HSPC)

Of the nearly 30 trillion cells in the body, approximately 90% are of hematopoietic origin. Hematopoietic lineages originating in the bone marrow are intimately involved in maintaining homeostasis and human health. Nonetheless, there are hundreds of hematologic or immune-related disorders caused or exacerbated by cells of the hematopoietic lineage. In a disease setting, cells of hematopoietic origin interact with host tissues to drive chronic inflammatory and immune disorders. Because some hematopoietic stem and progenitor cells (HSPC) have self-renewal and pluripotent properties, targeting HSPC has the potential to modulate underlying chronic inflammation and immune-related disorders.

Advances in lipid nanoparticle (LNP) technology has allowed for delivery to the bone marrow following systemic LNP administration in vivo. This achievement has enabled Moderna scientists to deliver mRNA directly to bone marrow HSPC in vivo, leading to HSPC transfection and long-term modulation of all hematopoietic lineages. This represents a major milestone in impacting chronic inflammatory and immune related disease.

mRNA Engineering: Optimizing Ribosome Load

The ribosome plays a central role in orchestrating the life of mRNA inside the cell. Understanding how to modulate translation by changing mRNA sequence could enable a powerful lever to control the performance of an mRNA drug. To develop such a lever, the Moderna's scientists strove to characterize mechanistically how differences in translation drive differences in protein expression. In this research, an unexpected relationship emerged where mRNAs with low translation initiation rates conferred the highest and most durable levels of protein expression. By understanding the mechanisms that drive this behavior, the Company is taking mRNA design from a guess-and-check discipline into an engineering discipline. With tools in place, and more under development, Moderna is improving its ability to make an mRNA that generates the right amount of the right protein, for the right amount of time, in the right cell type. As Moderna builds these levers into its mRNA drugs, the Company believes it will be able to target more indications with more precision.

Addressing SARS-CoV-2 Variants of Concern

One part of the Companys strategy to remain ahead of the SARS-CoV-2 virus is to closely monitor and address emerging variants of concern and waning immunity. Moderna is using artificial intelligence (AI) and machine learning to predict escape risk. This involves developing statistical models to understand and predict escape risk, including identifying breakthrough sequences from clinical trials and real-world data, examining spike protein biophysical and pseudovirus neutralization data, studying spike mutations and lineage info, and time tracking.

Science Day Webcast Information

Moderna will host its annual Science Day for analysts and investors at 8:00 a.m. ET on Thursday, May 27. A live webcast will be available under Events and Presentations in the Investors section of the Moderna website at investors.modernatx.com. A replay of the webcast will be archived on Modernas website for one year following the presentation.

About Moderna

In 10 years since its inception, Moderna has transformed from a science research-stage company advancing programs in the field of messenger RNA (mRNA), to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across six modalities, a broad intellectual property portfolio in areas including mRNA and lipid nanoparticle formulation, and an integrated manufacturing plant that allows for both clinical and commercial production at scale and at unprecedented speed. Moderna maintains alliances with a broad range of domestic and overseas government and commercial collaborators, which has allowed for the pursuit of both groundbreaking science and rapid scaling of manufacturing. Most recently, Modernas capabilities have come together to allow the authorized use of one of the earliest and most-effective vaccines against the COVID-19 pandemic.

Modernas mRNA platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, and has allowed the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and auto-immune diseases. Today, 24 development programs are underway across these therapeutic areas, with 14 programs having entered the clinic. Moderna has been named a top biopharmaceutical employer by Science for the past six years. To learn more, visit http://www.modernatx.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding: the potential for delivery of mRNA to hematopoietic stem and progenitor cells (HSPC) in vivo; methods of detecting and interrogating HSPC in vivo; the potential for delivering LNPs to hematopoietic stem cells in vivo; the ability to optimize codons and mRNA structures to increase total protein outputs; the potential for the Company to develop processes for controlling protein expression by modifying ribosomal loads; the Companys ability to engineer LNPs capable of accessing difficult-to-transfect primary cells with efficient endosomal escape and high functional mRNA delivery; the Companys strategy for combatting COVID-19, including processes for monitoring emerging variants and waning immunity; and strategies for modeling viral escape. In some cases, forward-looking statements can be identified by terminology such as will, may, should, could, expects, intends, plans, aims, anticipates, believes, estimates, predicts, potential, continue, or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. The forward-looking statements in this press release are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Modernas control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include, among others: the fact that there has never been a commercial product utilizing mRNA technology approved for use; the fact that the rapid response technology in use by Moderna is still being developed and implemented; the safety, tolerability and efficacy profile of the Moderna COVID-19 Vaccine observed to date may change adversely in ongoing analyses of trial data or subsequent to commercialization; the Moderna COVID-19 Vaccine may prove less effective against variants of the SARS-CoV-2 virus, or the Company may be unsuccessful in developing future versions of its vaccine against these variants; despite having ongoing interactions with the FDA or other regulatory agencies, the FDA or such other regulatory agencies may not agree with the Companys regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted; Moderna may encounter delays in meeting manufacturing or supply timelines or disruptions in its distribution plans for the Moderna COVID-19 Vaccine; whether and when any biologics license applications and/or additional emergency use authorization applications may be filed in various jurisdictions and ultimately approved by regulatory authorities; potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and clinical trials, supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; and those other risks and uncertainties described under the heading Risk Factors in Modernas most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Moderna with the SEC, which are available on the SECs website at http://www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Modernas current expectations and speak only as of the date hereof.

Read more here:
Moderna Highlights Advances in Platform Science and Innovative Vaccine Research at Fourth Annual Science Day - Business Wire

Bone Marrow Stem Cells Comments Off on Moderna Highlights Advances in Platform Science and Innovative Vaccine Research at Fourth Annual Science Day – Business Wire | May 28th, 2021

REDWOOD CITY, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, today announced the initiation of a Phase 1/2 clinical trial to evaluate JSP191, the companys first-in-class anti-CD117 monoclonal antibody, as a targeted, non-toxic conditioning regimen prior to allogeneic transplant for chronic granulomatous disease (CGD). Jasper Therapeutics and the National Institute of Allergy and Infectious Diseases (NIAID) have entered into a clinical trial agreement in which NIAID will serve as the Investigational New Drug (IND) sponsor for this study.

CGD is a rare, inherited disease of the immune system that develops in infancy or early childhood and results in severe and sometimes life-threatening infections. Allogeneic hematopoietic stem cell transplant is a proven cure for CGD. However, its use is limited because current conditioning agents used to deplete stem cells in preparation for transplantation are genotoxic and associated with limited efficacy and serious adverse effects, including veno-occlusive disease, long-term infertility and secondary malignancies.

We look forward to collaborating with NIAID on this Phase 1/2 clinical trial, which should provide important information about the potential of JSP191 as a safer and more effective conditioning agent for patients with CGD undergoing hematopoietic stem cell transplant, said Kevin N. Heller, M.D., Executive Vice President, Research and Development, of Jasper Therapeutics. Through this clinical trial agreement with NIAID, as well as others with the National Institutes of Health and academic centers, we are continuing to develop JSP191 for additional pretransplant conditioning regimens beyond severe combined immunodeficiency and acute myeloid leukemia/myelodysplastic syndromes, which have demonstrated safety and efficacy in early-stage clinical trials to date.

About JSP191

JSP191 is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow, creating an empty space for donor or gene-corrected transplanted stem cells to engraft. While hematopoietic cell transplantation can be curative for patients, its use is limited because standard high dose myeloablative conditioning is associated with severe toxicities and standard low dose conditioning has limited efficacy. To date, JSP191 has been evaluated in more than 90 healthy volunteers and patients. It is currently enrolling in two clinical trials for acute myeloid leukemia (AML)/myelodysplastic syndromes (MDS) and severe combined immunodeficiency (SCID) and is scheduled to begin enrollment in three additional studies in 2021 for severe autoimmune disease, sickle cell disease and Fanconi anemia patients undergoing hematopoietic cell transplantation.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The company is advancing two potentially groundbreaking programs. JSP191, a first-in-class anti-CD117 monoclonal antibody, is in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplantation. It is designed to enable safer and more effective curative allogeneic and autologous hematopoietic cell transplants and gene therapies. In parallel, Jasper Therapeutics is advancing its preclinical engineered hematopoietic stem cell (eHSC) platform, which is designed to overcome key limitations of allogeneic and autologous gene-edited stem cell grafts. Both innovative programs have the potential to transform the field and expand hematopoietic stem cell therapy cures to a greater number of patients with life-threatening cancers, genetic diseases and autoimmune diseases than is possible today. For more information, please visit us at jaspertherapeutics.com.

Here is the original post:
Jasper Therapeutics Announces New Clinical Trial with the National Institute of Allergy and Infectious Diseases to Evaluate JSP191 in Chronic...

Bone Marrow Stem Cells Comments Off on Jasper Therapeutics Announces New Clinical Trial with the National Institute of Allergy and Infectious Diseases to Evaluate JSP191 in Chronic… | May 28th, 2021

KILLEEN, TX As blankets of snow covered the frigid ground and ice sheets laid atop of pavement, Michael or MJ Dixon was due for a sports physical at his normal pediatrician.

The doctor gave him a clearance, Chaundra Dixon, his mother explained. [The doctor] gave him a clearance and said, oh, he's healthy. He's fine.

MJ was used to getting hurt.

As a basketball, football, baseball and tennis star, its only a matter of time before one accumulates bruises and sore muscles.

However, MJ kept saying he was hurt.

His mom was convinced it was just growing pains, and so the duo worked on his stretching.

Until February 24, 2021.

She was at work on Fort Hood when MJs babysitter gave her a call.

She was just like, 'hey, I think you need to come home and get Michael, we need to get him to the hospital,' she recalled, as tears built up in her eyes thinking back to that day. He's crying non stop; he's saying he can't walk.

The panicked mother immediately picked up her son from their Killeen home and braved the icy road conditions, making it to Baylor Scott and White before EMS got to her house.

As they arrived at the hospital, they were met with COVID-19 tests. From there, physicians tested MJ for the basics, which all looked OK.

It wasnt until they ran blood work and found an abnormal amount of white blood cells in MJs body when they determined it was leukemia

About seven o'clock that night, I will never forget that, Dixon said. He was like, you're a great mom and I'm sorry, that I have to tell you, your son has leukemia, and I said leukemia? I lost it.

Living in this new reality brought on by his cancer, MJ and his family began looking for bone marrow donors.

The problem? He has less than a 25% chance of finding one due to his ethnicity.

http://www.bethematch.org

This is simply because we don't have the donor pool to pull from, Tressa Malone, a spokesperson with the Be The Match organization said. What that means is, we just need more people to join the registry. It's as simple as that.

Once you register to see if you can become a match, Malone explained that the following procedures are quite simple.

She said that if you match with a patient in need, 80% of their matches donate in a process similar to donating plasma.

You go and they take out the blood, separate the cells they need, then put it back in you.

She explained that this process is usually 3-4 hours.

The second procedure to donate is by retrieving the blood near your pelvic bone, the doctors separate the stem cells, all while youre under anesthesia.

Dr. Amy Mersiovsky, the director of nursing at Texas A&M Central Texas explained that she spent countless days caring for young kids with cancer as a pediatric nurse.

Realizing the low odds African Americans face when it comes to these donors, she explained that shes not necessarily surprised after the generations of mistrust that community typically has toward the medical field.

However, the Dixons dont go down without a fight because they know God is on their side.

Now, the family organizes bone marrow drives not only here in Central Texas, but across the county, hoping to find MJ a donor or anyone whos walking the same journey the 9-year-old boy is.

The family has set up a GoFundMe page to cover various expenses, and to see if you can be a match, you can text MJ to 61474 and a swab kit will be sent to your home.

To follow along with MJ's story, head over to his Facebook page.

Continued here:
Killeen boy with leukemia needs a bone marrow donor, but the odds of finding one are slim - KXXV News Channel 25

Bone Marrow Stem Cells Comments Off on Killeen boy with leukemia needs a bone marrow donor, but the odds of finding one are slim – KXXV News Channel 25 | May 28th, 2021

For many years now, the topic of senescent cells has been the subject of plenty of research work. Back in the 1960s the Hayflick limit was noticed in cell culture: there was an apparent limit to the number of cell divisions that could take place before the cells just sort of stalled out. For human fibroblasts, that kicks in at around fifty divisions. Over time it was worked out that a primary mechanism involved is the shortening of telomeres with each cell division, specialized nucleotide sequences out at the ends of the chromosomes, and this cellular clock phenomenon has been making its way into the public consciousness ever since.

Its strange to think, but before these experiments human cells were considered to be more or less immortal and capable of unlimited numbers of divisions. Now, there are cells like that, but that (outside of some stem cell populations and a few other special cases) is a very short working definition of cancer. Those cells do indeed seem to be able to carry on for as long as conditions permit which in the artificial world of cell culture labs, means apparently forever. Henrietta Lacks died in 1951, but HeLa cells are still with us, and can be all too vigorous when they contaminate other lines. Tumor cells can pile up mutations that will make them die off, but short of that the jams have indeed been kicked out.

Its gradually become apparent that many aging or damaged tissues have a (sometimes substantial) population of cells that have reached their limit. Theyre alive and metabolically active but not really contributing much, in a stage of permanent growth arrest. Cellular senescence is a complex phenomenon, but its importance in aging, cancer, and tissue damaged by other factors (radiation, oxygen stress, etc.) is by now undeniable. Many of these non-aging states can be traced back to early telomere damage by other mechanisms, emphasizing that as a key countdown mechanism. But its clear that senescent have a different secretory profile (cytokines, growth factors and more) from the more vigorous cells around them and a number of other protein expression differences that can be used the characterize them.

Naturally enough, thoughts have turned to targeting such cells for therapy. There are a couple of very easy-to-picture hypotheses: first, could you keep telomeres from shortening (or shortening so much) and therefore keep cells in a non-senescent state for longer, potentially delaying biological aging? And second, could you somehow target cells that have already become senescent, and would doing so improve the health of the surrounding tissue? Though pretty obvious ideas, both of these are still very much in play. For now, Im going to talk about the second one, in light of a new paper.

That ones on the kidney. Younger people can regain some kidney function after an injury, but that ability goes down with aging, as youd imagine. It also goes down in states of chronic kidney disease, or after radiation damage. This new paper shows that targeting and removing senescent cells actually starts to reverse this phenotype once youve done that, the kidney tissue after injury shows increased function, increased regenerative ability, and less development of fibrosis. This is demonstrated both in aged tissue and in younger tissue exposed to radiation damage, in human cell culture and in mouse animal models.

You may well ask: how exactly does one target senescent cells? That takes us to ABT-263 (navitoclax), shown at right. This rather hefty molecule is part of a series of AbbVie protein-protein inhibitors for the Bcl-2 (B-cell-lymphoma) family. There are several of those, and navitoclax inhibits the function of Bcl-2, Bcl-xL, and Bcl-w. All of these proteins are intimately tied up in the pathways of apoptosis, programmed cell death, which is another monstrously huge pathway all its own. But one of the questions about senescent cells is why they dont go down some apoptotic pathway and just fall on their on cellular swords, instead of hanging around forever gumming up the works.

This one, like the others in its class, was developed to cause this to happen to tumor cells as an adjunct to other types of chemotherapy, but these have also turned out to be useful against senescent cells (although not all types of them). Similar to the kidney results reported in the new paper linked above, there have been reports in lung, CNS, muscle and other tissues of broadly similar enhancements (many of these summarized in this paper). So at this point you might be wondering why we dont just go ahead and put these things into the water supply already.

Theres a problem, unfortunately. It was clear from the clinical studies of the AbbVie compounds that platelet effects were dose-limiting. Cells in that pathway are sensitive to messing with these apoptosis pathways, and while you might be able to deal with that side effect in a chemotherapy situation, it doesnt exactly make for a good-for-what-ails-you drug. Navitoclax has alsorecently been shown to have profoundly bad effects on bone density and deposition, which is the exact opposite of what youd want for an aging population.

AbbVies next generation of such compounds, though, includes venetoclax, at right, also a lunker of a molecule and now approved for several types of leukemia. It still has platelet effects, but they arent nearly as disastrous as with navitoclax, thanks to deliberately lower binding to Bcl-xL. That also makes it a bit less of a mighty sword across senescent cell types for example, it appears that you need that pathway for activity against glioblastoma cells. But it has been reported to show strong protective effects against the development of Type I diabetes through the elimination of senescent cells in the islets of Langerhans. Meanwhile, other groups are looking at turning these ligands into targeted protein degraders, which (at least in some cases) seems to decrease the platelet problems and increase senolytic activity.

And before leaving the topic, it has to be noted that there are plenty of other ways to target these cells other than the Bcl pathway (although that one seems to be one of the most developed so far). What can I say? Im 59, and I doubtless have more senescent cells than I want or need, so I (and plenty of others) are interested in the idea. The whole cellular senescence pathway presumably developed as a way to avoid slipping into a tumor phenotype the more cellular divisions, the greater the chance of something going wrong along the way. Its a tradeoff, and evolution seems more than willing to shortchange older members of the species who have generally passed on their genes to all the offspring that theyre going to. But humans have other goals. We are looking at a rather rapidly aging planet, if current demographic trends hold up, and it would be extremely desirable to have that associated with less of a disease burden. Can we split the difference?

Here is the original post:
Clearing Cellular Dead Wood | In the Pipeline - Science Magazine

Bone Marrow Stem Cells Comments Off on Clearing Cellular Dead Wood | In the Pipeline – Science Magazine | May 28th, 2021

REGULATED INFORMATION

Strong clinical progress especially in JTA-004 Phase III study thanks to high patient compliance and retention

Process development partnership and appointment of cell therapy expert Anthony Ting as CSO to further strengthen product pipeline

Gosselies, Belgium, 26May 2021, 7am CEST BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the cell therapy company addressing unmet medical needs in orthopedics and other diseases, today announces its business update for the first quarter, ended 31 March 2021.

Bone Therapeutics has continued the strong momentum into 2021, said Miguel Forte, MD, PhD, CEO of Bone Therapeutics. Bone Therapeutics mid-late stage clinical programs continue to advance largely on schedule, including the Phase IIb trial of the allogenic cell therapy platform ALLOB in difficult-to-heal tibial fractures and the Phase III trial of the enhanced viscosupplement JTA-004 in knee osteoarthritic pain. Alongside this, Bone Therapeutics has strengthened its manufacturing and R&D capabilities by signing a process development partnership with Rigenerand. It has also appointed the industry veteran Tony Ting as our new Chief Scientific Officer. Building on these achievements, Bone Therapeutics will be able to continue significant clinical and commercial advancements as we move towards the topline data of our JTA-004 Phase III study; a potential key inflection point for Bone Therapeutics.

Operational highlights

Financial highlights

Outlook for the remainder of 2021

(1) Unaudited number

About Bone Therapeutics

Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopedics and other diseases. The Company has a, diversified portfolio of cell and biologic therapies at different stages ranging from pre-clinical programs in immunomodulation to mid-to-late stage clinical development for orthopedic conditions, targeting markets with large unmet medical needs and limited innovation.

Bone Therapeutics is developing an off-the-shelf next-generation improved viscosupplement, JTA-004, which is currently in Phase III development for the treatment of pain in knee osteoarthritis. Consisting of a unique combination of plasma proteins, hyaluronic acid - a natural component of knee synovial fluid, and a fast-acting analgesic, JTA-004 intends to provide added lubrication and protection to the cartilage of the arthritic joint and to alleviate osteoarthritic pain and inflammation. Positive Phase IIb efficacy results in patients with knee osteoarthritis showed a statistically significant improvement in pain relief compared to a leading viscosupplement.

Bone Therapeutics core technology is based on its cutting-edge allogeneic cell therapy platform with differentiated bone marrow sourced Mesenchymal Stromal Cells (MSCs) which can be stored at the point of use in the hospital. Currently in pre-clinical development, BT-20, the most recent product candidate from this technology, targets inflammatory conditions, while the leading investigational medicinal product, ALLOB, represents a unique, proprietary approach to bone regeneration, which turns undifferentiated stromal cells from healthy donors into bone-forming cells. These cells are produced via the Bone Therapeutics scalable manufacturing process. Following the CTA approval by regulatory authorities in Europe, the Company has initiated patient recruitment for the Phase IIb clinical trial with ALLOB in patients with difficult tibial fractures, using its optimized production process. ALLOB continues to be evaluated for other orthopedic indications including spinal fusion, osteotomy, maxillofacial and dental.

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

For further information, please contact:

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

For Belgian Media and Investor Enquiries:BepublicCatherine HaquenneTel: +32 (0)497 75 63 56catherine@bepublic.be

International Media Enquiries:Image Box CommunicationsNeil Hunter / Michelle BoxallTel: +44 (0)20 8943 4685neil.hunter@ibcomms.agency / michelle@ibcomms.agency

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

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

Visit link:
Bone Therapeutics Provides First Quarter 2021 Business Update - GlobeNewswire

Bone Marrow Stem Cells Comments Off on Bone Therapeutics Provides First Quarter 2021 Business Update – GlobeNewswire | May 28th, 2021

A five-year-old boy has returned from New York where he is taking part in a pioneering medical trial aimed at preventing his cancer from returning.

Oliver Stephenson from West Yorkshire was given the all-clear earlier this year after battling neuroblastoma a rare cancer originating from immature nerve cells throughout 2020.

His family crowdfunded 270,000 for treatment as the schoolboy underwent chemotherapy, surgery, stem cell treatment and a bone marrow transplant.

At one point, Oliver and his dad James had to isolate together in a 15sqft hospital room for seven weeks due to the coronavirus pandemic.

Oliver has spent the past month at New York's Memorial Sloan Kettering Cancer Center, where he is on a clinical trial vaccine called Bivalent.

It is hoped the treatment will train his immune system to identify and destroy neuroblastoma cells lurking in his body to prevent the disease from returning.

The vaccine, which costs 210,000, will require Oliver to make four more trips to the US this year.

He returned from the four-week trip to New York around a week ago with his mum Laura, dad James and little brother Alfie.

Mrs Stephenson said: "Everything went great. The doctors were all amazing and Oliver has responded well so far.

"It is a clinical trial so we don't know what will happen but we are hoping that this will prevent Oliver from suffering as he has done again.

"After everything he has been through it feels great to be on a positive track."

She added the treatment was "quick and easy", explaining Oliver had three vaccines and that, other than some discomfort, he had no obvious side effects.

Due to the length of their stay, Mrs Stephenson said the family were able to make a holiday out of the trip.

She added: "Oliver and Alfie had a brilliant time in New York, it was amazing.

"Because of the pandemic everywhere was really quiet, which meant we felt very safe all the time and didn't have to queue for anything."

The pioneering treatment comes almost 18 months after Oliver was diagnosed with neuroblastoma, which affects around 100 children annually.

The coronavirus pandemic complicated the family's ordeal as Oliver and his dad had to isolate together in hospital during chemotherapy.

Mr Stephenson was at his son's bedside throughout, but the rest of his family were unable to visit due to restrictions and had to make do with video calls.

Crowdfunding pages were set up to help the family pay for treatment once options on the NHS were exhausted, as neuroblastoma has a significant chance of returning.

Mrs Stephenson added: "The incredible support and donations not just from our friends and family but around the world has been overwhelming.

"We are so grateful to everyone who has helped.

"It's been a really difficult year for us and back in 2020 Oliver's prognosis wasn't good at all.

"But he has battled through everything is doing well now, he's fit and strong and even back at school.

Despite Oliver getting the all-clear, figures show 60% of people who recovered from neuroblastoma end up relapsing.

Mrs Stephenson said: "We want to everything we can to stop that happening."

Throughout Oliver's treatment, his family has been supported by the Solving Kids Cancer charity.

Original post:
Five-year-old boy returns home after first round of cancer treatment in New York - Surrey Live

Bone Marrow Stem Cells Comments Off on Five-year-old boy returns home after first round of cancer treatment in New York – Surrey Live | May 28th, 2021

DUBLIN--(BUSINESS WIRE)--The "Global Stem Cell Therapy Market by Type (Allogeneic, Autologous), Therapeutic Application (Musculoskeletal, Wound & Injury, CVD, Autoimmune & Inflammatory), Cell Source (Adipose tissue, Bone Marrow, Placenta/Umbilical Cord) - Forecasts to 2026" report has been added to ResearchAndMarkets.com's offering.

The global stem cell therapy market is projected to reach USD 401 million by 2026 from USD 187 million in 2021, at a CAGR of 16.5% during the forecast period.

Growth in this market is majorly driven by the increasing investment in stem cell research and the rising number of GMP-certified stem cell manufacturing plants. However, factors such as ethical concerns and the high cost of stem cell research and manufacturing process likely to hinder the growth of this market.

The allogeneic stem cell therapy segment accounted for the highest growth rate in the stem cell therapy market, by type, during the forecast period

The stem cell therapy market is segmented into allogeneic and autologous stem cell therapy. Allogeneic stem therapy segment accounted for the largest share of the stem cell therapy market. The large share of this segment can be attributed to the lesser complexities involved in manufacturing allogeneic-based therapies.

This segment is also expected to grow at the highest growth rate due to the increasing number of clinical trials in manufacturing allogeneic-based products.

Bone Marrow-derived MSCs segment accounted for the highest CAGR

Based on the cell source from which stem cells are obtained, the global stem cell therapy market is segmented into four sources. These include adipose tissue-derived MSCs (mesenchymal stem cells), bone marrow-derived MSCs, placenta/umbilical cord-derived MSCs, and other cell sources (which include human corneal epithelium stem cells, peripheral arterial-derived stem cells, and induced pluripotent stem cell lines).

The bone marrow-derived MSCs segment is expected to witness the highest growth rate during the forecast period, owing to an increasing number of clinical trials focused on bone marrow-derived cell therapies and the rising demand for these cells in blood-related disorders.

Asia Pacific: The fastest-growing country in the stem cell therapy market

The stem cell therapy market is segmented into North America, Europe, Asia Pacific, RoW. The stem cell therapy market in the Asia Pacific region is expected to grow at the highest CAGR during the forecast period.

Factors such as the growing adoption of stem cell-based treatment in the region and the growing approval & commercialization of stem cell-based products for degenerative disorders drive the growth of the stem cell therapy market in the region.

Market Dynamics

Drivers

Restraints

Opportunities

Challenges

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/qiagh1

Read this article:
Stem Cell Therapy Market by Type, Therapeutic Application and Cell Source - Global Forecasts to 2026 - ResearchAndMarkets.com - Business Wire

Bone Marrow Stem Cells Comments Off on Stem Cell Therapy Market by Type, Therapeutic Application and Cell Source – Global Forecasts to 2026 – ResearchAndMarkets.com – Business Wire | May 15th, 2021